Med-Chemist

Vitamin D Deficiency Linked to Fatigue, Studies Show

2026-09-21T13:09:33.949+05:30

In continuation of my update on Vitamin D

Prevalence and Link to Fatigue

Approximately 40% to 75% of the teen and adult population in the United States is deficient in vitamin D, according to Raymond Francis in “The Great American Health Hoax.” ^[1] Low energy and occasional fatigue are commonly reported among individuals with low vitamin D status, according to a report on the silent epidemic of nutrient deficiencies. ^[2] The scale of the problem is vast, with estimates suggesting that nearly half of all adults may have insufficient levels, the report stated.

Vitamin D deficiency is not confined to a single demographic. Factors such as limited sun exposure, darker skin pigmentation, and indoor lifestyles contribute to widespread insufficiency, researchers said. The link between low vitamin D and fatigue has been documented in multiple studies, though many individuals remain unaware of their own deficiency status.

Mechanism: Vitamin D and Cellular Energy

Vitamin D supports mitochondrial function, which is responsible for cellular energy production, according to research cited in an article on fatigue and mitochondria. ^[3] The mitochondria act as the powerhouses of the cell, generating adenosine triphosphate (ATP) from nutrients. A deficiency may disrupt mitochondrial efficiency, contributing to feelings of lethargy, experts said. ^[4]

The role of mitochondria in energy production is central to understanding why vitamin D insufficiency can lead to persistent tiredness. As noted in the book “Dissolving Illusions,” proper mitochondrial metabolism is essential for normal cellular function, and any disruption can manifest as weakness or apathy. ^[5] Although that text focuses on vitamin C, the principle applies broadly to nutrients that support mitochondrial health, including vitamin D.

Supporting Studies

Multiple studies have reported a connection between low vitamin D levels and fatigue. A 2016 study of 200 female nurses in Iran found that low vitamin D levels accounted for 13% to 18% of reported prolonged tiredness, according to a review of nutrient deficiencies. ^[2] A 2019 study published in Nutrients similarly found that older adults with occasional fatigue had a significantly worse vitamin D status than those without fatigue, the review added. ^[2]

Other research has reinforced these findings. In a broader analysis, researchers noted that individuals frequently reporting low energy were more likely to have suboptimal vitamin D levels compared to their peers, according to an article on fatigue and mitochondrial function. ^[4] The consistency of the association across different populations suggests a robust relationship, though causation requires further investigation, scientists said.

Other Health Impacts

Beyond fatigue, vitamin D deficiency has been linked to several other health issues. Vitamin D directly modulates the body’s innate and adaptive immune responses, according to a review of the critical role of vitamin D in immune function. ^[6] Immune cells require adequate vitamin D to respond efficiently to threats, the review stated. Low levels have also been associated with increased susceptibility to infections and longer recovery times.

Vitamin D also influences bone health and cellular aging. Raymond Francis wrote that vitamin D is essential for calcium metabolism, supports bone health, helps control blood sugar, and protects against cancer. ^[1] The nutrient significantly increases the production of telomerase, an enzyme that repairs telomeres and slows aging, according to the same source. ^[1] These wide-ranging effects underscore the importance of maintaining optimal levels.

Solutions: Increasing Vitamin D Levels

Optimal vitamin D levels are considered to be above 50 nanograms per milliliter, and supplementation with 5,000 IU daily is recommended by some nutritionists, according to an interview on health testing and supplementation. ^[7] At-home blood tests can help individuals assess their current levels, the interview stated. ^[7] Testing allows for personalized dosing rather than generic recommendations.

The safety of higher vitamin D doses has been affirmed by recent reassessments. A news report noted that a “schoolboy error” in earlier guidelines led to overly cautious upper limits, and the Academy of Medicine has since acknowledged that higher intakes are not toxic for most people. ^[8] Dietary sources such as fatty fish, egg yolks, and fortified foods can also contribute, though supplementation is often the most reliable way to correct a deficiency, experts said.

Conclusion

Individuals experiencing unexplained fatigue may consider testing their vitamin D levels, clinicians advised. ^[7] Supplementation and dietary changes can help restore levels to the optimal range, though consulting a healthcare provider is recommended before starting any regimen, according to the interview. ^[7]

The evidence linking vitamin D deficiency to fatigue is supported by prevalence data, mechanistic studies, and clinical observations. Given the high rates of insufficiency in the general population, testing and appropriate supplementation offer a straightforward strategy for addressing a common but often overlooked contributor to low energy, researchers concluded.

https://en.wikipedia.org/wiki/Vitamin_D

Vitamin D Deficiency Linked to Fatigue, Studies Show

Daily orforglipron treatment reduces weight and blood sugar in seniors

2026-09-18T10:21:40.454+05:30

In continuation of my update on orforglipron

A new analysis to be presented at this year's European Congress on Obesity (ECO 2026, Istanbul, Turkey, 12-15 May) examined daily oral orforglipron treatment for the treatment of obesity, with or without diabetes, in users aged 65 years and over, with results and a safety profile similar to that seen in the ATTAIN clinical trial programme population. Lead author for this post-hoc analysis is Dr Deborah Horn, Director of the Center for Obesity Medicine and Metabolic Performance at McGovern Medical School at UTHealth Houston, Houston, Texas, USA, and colleagues.

Limited clinical data exist on the use of incretin-based obesity management medications in older adults with or without type 2 diabetes (T2D), and a lack of specific analyses related to older users could be a factor that could make providers or patients in this age group hesitant when considering therapy choices.

Orforglipron is a novel small-molecule, non-peptide, oral glucagon-like peptide-1 receptor agonist (GLP-1 RA) developed by Eli-Lilly and Company (Lilly), who sponsored these analyses. The drug was approved by the US Food and Drug Administration for chronic weight management on April 1, 2026.

Orforglipron demonstrated significant weight reduction vs. placebo in the phase 3, randomised, double-blind, multinational ATTAIN-1 and ATTAIN-2 clinical trials in participants with obesity or obesity and T2D, respectively. In this new analysis of those trials, the authors evaluated efficacy and safety of orforglipron versus placebo in a sub-group of participants aged 65 years and older from ATTAIN-1 and ATTAIN-2.

The ATTAIN-1 and ATTAIN-2 global clinical trials evaluated once-daily orforglipron 6 mg, 12 mg, or 36 mg vs. placebo as an adjunct to healthy diet and physical activity in participants from 9 and 10 countries, respectively. In this sub-group analysis, efficacy outcomes were analysed separately for each trial, and safety data were pooled. The primary endpoint was percent change in body weight from baseline to week 72.

In ATTAIN-1 and ATTAIN-2, 616 randomised participants were ≥65 years of age (n=196 and 420, respectively). Of those, 613 received treatment (orforglipron 6 mg, n=118; 12 mg, n=135; 36 mg, n=146; placebo, n=214). And 79.1% of ATTAIN-1 and 86.2% of ATTAIN-2 participants had hypertension as a comorbidity.

At Week 72, the percent change in weight from baseline in users aged 65 and over in ATTAIN-1 (participants with obesity and without T2D) was –7.9%, –11.3%, and –13.0% with orforglipron 6 mg, 12 mg, and 36 mg, respectively, vs. –1.6% with placebo, all statistically significant findings. Similar results were found for those users aged 65 years and over with T2D and obesity in ATTAIN-2: orforglipron 6 mg: –7.5%; 12 mg: –8.3%; 36 mg: –12.2%; placebo: –2.3% again with all results statistically significant.

Participants with T2D experienced reductions from baseline in glycated haemoglobin (HbA1c – a measure of blood sugar control) of –1.5%, –1.6%, and –1.7% with 6 mg, 12 mg, and 36 mg orforglipron, respectively, vs. –0.1% with placebo. BMI, waist circumference, triglycerides, non-HDL cholesterol, and health-related quality of life also improved in orforglipron vs. placebo treatment groups in participants with or without T2D.

https://en.wikipedia.org/wiki/Orforglipron

Daily orforglipron treatment reduces weight and blood sugar in seniors

Semaglutide shows sustained weight loss benefits in older adults

2026-09-17T10:33:54.771+05:30

In continuation of my update on semaglutide

A new analysis of the STEP trails carried out by semaglutide manufacturer Novo Nordisk has analysed various trials to show the safety and efficacy of the obesity drug semaglutide in older adults (over 65 years), and found similar efficacy and safety as in the general trial populations . The study is by Prof Luca Busetto from the University of Padova in Italy and colleagues including from Novo Nordisk, who sponsor this new study.

Individuals of advanced age with obesity represent a vulnerable group, often presenting with comorbidities and frailty, and being at risk of adverse events (AEs). Information on the use of glucagon-like peptide-1 (GLP-1) receptor agonists including semaglutide in this population is limited; therefore, the authors decided assessed the efficacy and safety of once-weekly subcutaneous semaglutide 2.4 mg in individuals aged 65 years and over.

The analysis pooled together data from the STEP 1, 3, 4, 5, 8 and 9 trials (only in people with obesity or overweight, not diabetes, because weight loss in obesity drug trials is always lower in people with diabetes than without – thus results cannot be compared or mixed). It included participants aged ≥65 years with body mass index of at least 30 kg/m², or at least 27 kg/m² and at least 1 obesity-related complication (without diabetes) who were randomised to receive semaglutide 2.4 mg or placebo. All participants received lifestyle intervention and, in STEP 3, intensive behavioural therapy. Endpoints were assessed from baseline to week 68 and included percentage change in body weight; proportion of participants achieving categorical body weight reductions (≥10%, ≥15%, ≥20%); change in waist circumference; proportion of participants achieving waist to height ratio (WHtR) <0.53; shift in BMI category; and changes in cardiometabolic risk factors (glucose parameters, blood pressure, serum lipids and hs-CRP). Adverse events (AEs) were also assessed.

Of the total population in the selected trials (N=4523), 358 participants (8%) were aged 65 years or older and included in the analysis (semaglutide 2.4 mg, n=248; placebo, n=110), with most (90%) being aged 65–74 years (and the others 75 years and over). Across the pooled semaglutide and placebo groups at baseline, mean age was 69 years, bodyweight was 99.0 kg, BMI was 36.6 kg/m² and waist circumference was 115 cm; 72% were female.

At week 68, there was a mean −15.4% change in body weight for semaglutide 2.4 mg vs −5.1% for placebo, and a mean −14.3 cm vs −6.0 cm change in waist circumference, respectively. Proportions of participants achieving body weight reduction thresholds in the semaglutide 2.4 mg vs placebo groups were 66.5% vs 15.5% (at least 10%), 46.8% vs 6.4% (at least 15%) and 28.6% vs 2.7% (at least 20%), respectively.

In the semaglutide 2.4 mg group, 11.3% achieved a WHtR <0.53, compared with 4.5% with placebo. A greater proportion of semaglutide-treated participants improved their BMI category from baseline to week 68 compared with placebo (see Figure full abstract). A BMI of <27 kg/m² (so called healthy weight) was achieved by 27.0% of participants in the semaglutide group vs 5.5% in the placebo group; and the proportion of elderly patients in the overweight and obesity class I, II and III categories all fell in the semaglutide group at week 68 due to the increase in participants who had reached a healthy weight.

For participants achieving both a BMI of 27 or less and a WHtR of <0.53 these values were 10.5% vs 2.7%, respectively. Greater improvements in cardiometabolic risk factors were observed with semaglutide 2.4 mg vs placebo (see Table full abstract), including blood pressure, blood fats, cholesterol and glycated haemoglobin (HbA1c – a measure of blood sugar control used in diagnosis of diabetes).

Proportions of participants experiencing AEs and serious AEs in the semaglutide 2.4 mg vs placebo groups were similar for AEs overall (89.1% vs 84.5%), but higher for semaglutide re: serious AEs - 19.0% vs 12.7%, respectively. Constipation and dizziness rates (known side effects of this class of drug) were higher with semaglutide, while fractures and hypoglycaemia were comparable to placebo, both affecting less than 1% in each group.

https://en.wikipedia.org/wiki/Semaglutide

Semaglutide shows sustained weight loss benefits in older adults

Glutamic Acid Helps Fresh-Cut Potatoes Stay Fresh by Silencing Browning Genes | Newswise

2026-09-16T14:12:53.152+05:30

In continuation of my update on Glutamic acid

The study identified a transcriptional regulatory module, StbZIP53-like2–StERF091, that suppresses the activity of key polyphenol oxidase genes linked to browning. By showing how glutamic acid activates this gene-control system, the work provides a clearer molecular basis for safer anti-browning strategies and may support the future development of quality-preserving treatments for fresh-cut potato products and other minimally processed vegetables.

Fresh-cut potatoes are increasingly popular because they fit modern food habits, but their shelf life is limited by rapid enzymatic browning after cutting. Existing methods to control browning include chemical, physical, and biological approaches, yet each has drawbacks. Some chemicals raise food-safety concerns, while physical treatments may cause off-flavors, and many biological methods are still difficult to commercialize. Earlier work had already shown that glutamic acid could reduce browning in fresh-cut potatoes, but the molecular mechanism remained unclear. That unresolved question made it necessary to investigate how glutamic acid regulates browning-related genes and upstream transcription factors in fresh-cut potato tissue.

A study (DOI:10.48130/ph-0026-0004) published in Plant Hormones on 20 March 2026 by Jingying Shi’s & Zunyang Song’s team, Shandong Agricultural University, demonstrates that glutamic acid-induced StbZIP53-like2 and StERF091 form a cooperative regulatory module that represses StPPO2 and StPPO3, thereby alleviating browning in fresh-cut potatoes.

The team first prepared fresh-cut potato shreds and compared untreated samples with samples immersed in a glutamic acid solution, then stored them at 4 °C and collected tissues across multiple time points. They measured ethylene production, performed RNA-seq and RT-qPCR analyses, and screened for transcription factors whose expression changed under glutamic acid treatment. Among 11 bZIP genes detected, StbZIP53-like2 stood out because its expression was strongly induced by glutamic acid. Subcellular localization assays showed that this protein is localized in the nucleus, consistent with its proposed role as a transcription factor. The researchers then examined whether StbZIP53-like2 directly controlled known browning-associated genes. Dual-luciferase assays showed that it repressed the promoter activity of StPPO3, while EMSA and yeast one-hybrid experiments confirmed direct binding to the C-box motif in the StPPO3 promoter. To identify interacting partners, the team screened a cDNA library and found StERF091, another glutamic acid-induced transcription factor. Yeast two-hybrid, GST pull-down, and co-immunoprecipitation assays together demonstrated that StbZIP53-like2 physically interacts with StERF091 both in vitro and in vivo. Like StbZIP53-like2, StERF091 was also localized in the nucleus. Next, the group tested the function of StERF091 in regulating browning genes. They found that StERF091 repressed the promoter activities of StPPO2 and StPPO3, but not StPPO7. EMSA and yeast one-hybrid assays further showed that StERF091 directly binds the GCC-box motifs in the StPPO2 and StPPO3 promoters. Most importantly, co-expression assays revealed that when StbZIP53-like2 and StERF091 acted together, repression of StPPO2 and StPPO3 became stronger than with either factor alone. This demonstrated that the two proteins form a functional inhibitory module that amplifies the anti-browning response triggered by glutamic acid.

Overall, the study shows that glutamic acid alleviates the browning of fresh-cut potatoes by inducing two transcriptional repressors, StbZIP53-like2 and StERF091, which cooperate to suppress StPPO2 and StPPO3. The findings move beyond a simple observation that glutamic acid works and explain why it works at the molecular level. This insight may help guide future preservation technologies aimed at extending the shelf life, visual quality, and commercial value of fresh-cut produce.

https://en.wikipedia.org/wiki/Glutamic_acid

Glutamic Acid Helps Fresh-Cut Potatoes Stay Fresh by Silencing Browning Genes

Legumes and soy foods may help reduce hypertension risk

2026-09-12T09:30:35.422+05:30

In continuation of my continuation of my update on soy ..

A higher dietary intake of soy and legumes is linked to a lower risk of high blood pressure, finds a pooled data analysis of the available evidence, published in the open access journal BMJ Nutrition Prevention & Health.

And the optimal daily amount may be around 170 g of legumes, which include peas, lentils, chickpeas and beans, and 60 to 80 g of soy foods, examples of which include tofu, soy milk, edamame, tempeh, and miso, the findings indicate.

Legumes and soy foods have been associated with an overall lower risk of cardiovascular disease, but the evidence on their potential for lowering high blood pressure is mixed and needs to be systematically quantified, explain the researchers.

To explore this further, the researchers scoured databases for relevant studies published up to June 2025, and found 10 publications that included data from 12 prospective observational studies.

Five studies were from the USA, 5 from Asia (China, Iran, South Korea and Japan), and 2 were from Europe (France and the UK). Nine studies included both men and women, 2 included only women, and 1 included only men.

The number of study participants ranged from 1152 to 88,475 and the number of cases of high blood pressure ranged from 144 to 35,375.

Pooled data analysis of the study findings showed that higher daily intake of legumes and soy foods was associated with a lower risk of developing high blood pressure.

Compared with those with a low intake of legumes, those with a high intake were 16% less likely to develop high blood pressure. Similarly, those with a high intake of soy foods were 19% less likely to develop the condition than those with a low intake.

When assessing the association between quantity and lower risk, a linear reduction (30%) emerged for legumes up to around 170 g/day, while most of the reduction in risk (28-29%) for soy foods was observed at between 60 and 80 g/day, with no further reduction in risk at higher intake.

One hundred grams of legumes/soy is equivalent to a serving size of about one cup or 5–6 tablespoons of cooked beans, peas, chickpeas, lentils, soybeans or a palm-size serving of tofu, explain the researchers.

Using World Cancer Research Fund evidence grading criteria for evaluating the likelihood of causality, the researchers consider the overall evidence to indicate a probable causal relationship between both legume and soy intake and a reduced risk of high blood pressure.

There are plausible explanations for the findings, they say. Legumes and soy are high in potassium, magnesium, and dietary fiber, all of which are known for their blood pressure lowering properties.

And recent research has suggested that the fermentation of soluble fiber from legumes and soy produces short-chain fatty acids that influence blood vessel dilation, while the isoflavone content of soy also seems to help lower blood pressure, they explain.

The researchers acknowledge various limitations to their findings, including the variability of the studies in the pooled data analysis. This included differences in legume types, levels of intake, preparation methods, dietary contexts, and the definition of high blood pressure.

"Despite these limitations, the findings of this meta-analysis have major public health implications, given the alarming global increase in hypertension prevalence," they point out.

"Current legume consumption across Europe and the UK remains below dietary recommendations, with average intakes of only 8–15 g/day, far below the recommendations of 65 to 100 g/day recommended for overall cardiovascular health," they add.

"Although further large-scale cohorts are needed for confirmation, these findings provide further evidence in support of dietary recommendations to the public to prioritise and integrate legumes and soy foods as healthy protein sources in the diet," they conclude.

"This research strengthens the evidence base for the cardioprotective benefits of plant-based diets. The authors have significantly added to the case for using legumes and soy as primary dietary strategies to mitigate the global burden of hypertension," comments Professor Sumantra Ray, chief scientist and executive director of NNEdPro Global Institute for Food, Nutrition and Health, which co-owns BMJ Nutrition Prevention & Health.

"The strengths of the study lie in its rigorous dose-response analyses, which offer practical dietary targets for use in public health guidelines and clinical practice. But we can't entirely rule out the influence of unmeasured influential factors. And the plateauing of benefits for soy at 60–80 g/day warrants further investigation, as it remains unclear if this reflects a true biological limit or is a byproduct of the smaller number of studies available for analysis."

Legumes and soy foods may help reduce hypertension risk

Nicotinamide Linked to Lower Melanoma Risk Post Skin Cancer

2026-09-11T13:41:45.459+05:30

In continuation of my update on Nicotineamide

Nicotinamide supplementation was associated with a decreased risk for melanoma among patients with a history of other skin cancers, according to a retrospective cohort study of more than 33,000 veterans.

The study, which won a first-prize poster award at the American Academy of Dermatology (AAD) 2026 Annual Meeting, “suggests that nicotinamide could potentially be effective for primary prevention of melanoma among patients who have had a prior nonmelanoma skin cancer,” noted Lee Wheless, MD, PhD, one of the senior authors, an assistant professor of dermatology at Vanderbilt University and a staff physician with the Tennessee Valley Healthcare System VA Medical Center, both in Nashville, Tennessee.

The effect of nicotinamide, at a dose of 500 mg twice daily, was statistically significant for invasive, but not in situ, melanoma, and it was consistent in both sun-exposed and sun-protected areas, he told Medscape Medical News.

The retrospective cohort study used data from the Veterans Affairs (VA) Corporate Data Warehouse from January 1, 2007, to December 31, 2024. It included a total of 33,581 patients with one or more prior skin cancers of any type. The cohort was predominantly White and men, with comparable mean ages (77.5 years in the exposed group vs 77.1 years in the unexposed group).

The US Department of VA offers nicotinamide, a vitamin B3 derivative, on formulary. Patients exposed to nicotinamide 500 mg twice daily were propensity score matched to unexposed patients based on “numerous skin cancer risk variables to make sure risks at specific timepoints in the patients’ lives matched up,” explained Wheless.

Baseline matching variables included age, sex, race, age at first skin cancer, number and year(s) of prior skin cancers of any type, number of dermatology visits after baseline, chronic lymphocytic leukemia, solid organ transplantation, exposure to acitretin, and field therapy.

More...

https://en.wikipedia.org/wiki/Nicotinamide

Nicotinamide Linked to Lower Melanoma Risk Post Skin Cancer

Metformin's real power may be in the gut

2026-09-09T12:02:24.854+05:30

In continuation of my uodate on metformin

For decades,physicians and scientists thought metformin, the leading type 2 diabetes medication taken by millions worldwide, mainly targets the liver to suppress glucose production. But a new Northwestern University study in mice has found this "wonder drug" instead focuses primarily on the gut, acting to prevent glucose levels rising in the blood by driving glucose utilization inside cells lining the intestine.

The body relies on glucose as a fast and versatile fuel, but too much glucose can lead to insulin resistance and ultimately damage blood vessels and organs. The study found metformin slows mitochondrial energy production in gut cells, forcing the intestine to metabolize extra sugar.

"Metformin essentially helps the intestine suck the glucose out of the bloodstream, which further highlights that the gut plays a major role in regulating blood sugar levels," said corresponding author Navdeep Chandel, professor of biochemistry and molecular genetics at Northwestern University Feinberg School of Medicine.

The study is published in Nature Metabolism.

The study builds off findings from previous work in Chandel's lab, which found metformin lowers blood sugar by blocking a specific part of the cell's energy-making machinery called mitochondrial complex I, a key enzyme in cellular respiration.

The new study furthers that work by pinpointing the specific tissue targeted by metformin. The findings suggest directing drugs or supplements to the gut could be an effective strategy for controlling blood sugar, Chandel said.

Chandel is also the David W. Cugell, MD, Professor of Medicine (Pulmonology and Critical Care), Biochemistry and Molecular Genetics and an investigator with the Chan Zuckerberg Initiative. The study's first author is Zach Sebo, a postdoctoral fellow in the Chandel lab who will soon start his own research group at the University of Kansas School of Medicine.

"Our study suggests that revisiting assumptions about metformin's mechanism may offer a more detailed understanding of how it works," Sebo said.

https://en.wikipedia.org/wiki/Metformin

Metformin's real power may be in the gut

FDA Approves Veppanu (vepdegestrant) for the Treatment of ESR1m, ER+/HER2- Advanced Breast Cancer

2026-09-08T11:08:02.199+05:30

Arvinas, Inc. (Nasdaq: ARVN), with its partner Pfizer Inc. (NYSE: PFE), announced the U.S. Food and Drug Administration (FDA) approval for Veppanu (vepdegestrant) for the treatment of adults with estrogen receptor-positive (ER+)/human epidermal growth factor receptor 2-negative (HER2-), estrogen receptor 1 (ESR1)-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy. This approval marks the first time the FDA has approved a PROteolysis TArgeting Chimera (PROTAC), a type of heterobifunctional protein degrader therapy.

Veppanu™ is the first-and-only FDA-approved PROTAC, a type of heterobifunctional protein degrader

Approval received in advance of FDA-assigned PDUFA date of June 5, 2026; Arvinas and Pfizer remain on track to announce selection of a third party

Veppanu offers a new therapeutic option in ER+/HER2-, ESR1-mutated advanced or metastatic breast cancer, where treatment resistance remains a major clinical challenge

“Today’s FDA approval is a transformative moment for Arvinas as we achieve our first approved medicine and the first-ever approved PROTAC therapy based on the technology we’ve pioneered since 2013,” said Randy Teel, Ph.D., President and Chief Executive Officer at Arvinas. “This milestone demonstrates that targeted protein degradation can translate into meaningful clinical impact. It also strengthens our confidence in the breadth and versatility of our exciting clinical pipeline across oncology, neurodegenerative, and neuromuscular diseases. We are especially encouraged by receiving FDA approval ahead of the June 5 PDUFA date and together with Pfizer, we are on track to announce selection of a third party to bring this new treatment option to patients as soon as possible.”

“For patients living with ESR1 mutant, ER+/HER2 advanced breast cancer, there have been minimal second-line treatment options once standard therapies are no longer effective,” said Erika Hamilton, M.D., Chief Development Officer, Late Phase, and Director, Breast Cancer Research, Sarah Cannon Research Institute, as well as a principal investigator of the VERITAC-2 trial. “The introduction of a new, targeted treatment is an encouraging development for this community and highlights meaningful innovation in the way this disease is treated. The approval of vepdegestrant gives clinicians another tool in the breast cancer treatment arsenal and brings renewed hope to individuals who need additional options.”

Breast cancer is the most common cancer among women worldwide, with many tumors driven by estrogen receptor signaling. While endocrine therapy remains a cornerstone of metastatic ER+/HER2- breast cancer treatment, up to 40-50% of patients treated with endocrine therapy and a CDK4/6 inhibitor have ESR1 mutations, resulting in endocrine resistance and poor prognosis. These patients often experience rapid disease progression and face limited options after first-line therapy. The FDA approval of Veppanu addresses a significant unmet need, offering a new treatment option for adults with ESR1-mutant, ER+/HER2- advanced breast cancer by targeting a key biological driver of resistance to current therapies.

“The approval of Veppanu is an important milestone for patients, their caregivers, and physicians,” said Noah Berkowitz, M.D., Ph.D., Chief Medical Officer at Arvinas. “Veppanu addresses an unmet need for patients with this aggressive form of breast cancer who have progressed on their initial therapy. Today’s approval provides a new oral treatment option that showed improved progression free survival when compared to the current standard of care, fulvestrant, which is administered via an intramuscular injection.”

Veppanu was discovered by Arvinas and jointly developed by Arvinas and Pfizer. FDA approval was granted based on data from VERITAC-2 (NCT05654623), a global, randomized, open-label, pivotal Phase 3 clinical trial evaluating vepdegestrant versus fulvestrant. In the trial, among patients with an ESR1 mutation (n=270), vepdegestrant demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS), reducing the risk of disease progression or death by 43% compared to fulvestrant. Median PFS was 5 months (95% CI: 3.7, 7.4) in the vepdegestrant arm and 2.1 months (95% CI: 1.9, 3.5) in the fulvestrant arm (hazard ratio 0.57 [95% CI: 0.42, 0.77]; p-value 0.0001). Overall survival was immature with 16% of deaths in this population at the time of the PFS analysis. The majority of adverse events (AEs) with vepdegestrant were low grade (Grade 1-2) and the most common (≥10%) adverse reactions, including laboratory abnormalities, were decreased white blood cells, increased AST, musculoskeletal pain, fatigue, decreased hemoglobin, decreased neutrophils, increased ALT, increased alkaline phosphatase, nausea, decreased blood potassium, increased bilirubin, decreased appetite, electrocardiogram QT prolonged, decreased platelets, and constipation.

Arvinas and Pfizer intend to jointly identify and select a third-party partner with the capabilities and expertise to maximize the commercial potential of Veppanu. The companies are on track to announce selection of a third party.

Arvinas was originally founded based on pioneering research at Yale University, where Professor Craig Crews, Ph.D., co-authored the first-ever paper on PROTAC protein degraders.

What is Veppanu?

Veppanu is a prescription medicine to treat people with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced breast cancer or breast cancer that has spread to other parts of the body (metastatic), and whose disease has progressed after at least one line of endocrine-based therapy.

https://en.wikipedia.org/wiki/Vepdegestrant

FDA Approves Veppanu (vepdegestrant) for the Treatment of ESR1m, ER+/HER2- Advanced Breast Cancer

Cogent Biosciences Announces Submission of New Drug Application for Bezuclastinib in Gastrointestinal Stromal Tumors (GIST)

2026-09-07T13:57:16.291+05:30

Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, announced the completion of the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib. Based on the positive results from the PEAK trial, the bezuclastinib NDA was submitted under the FDA’s Real-Time Oncology Review (RTOR) program, which is intended to enable a more streamlined review process. Bezuclastinib was also granted Breakthrough Therapy Designation as a treatment for GIST earlier this year.

Bezuclastinib NDA submitted under the FDA’s RTOR program based on positive results from Phase 3 PEAK trial; bezuclastinib previously granted Breakthrough Therapy Designation in GIST
Bezuclastinib combination demonstrated 16.5 month mPFS and 46% ORR in imatinib-resistant GIST patients, dramatically improving upon the current standard of care

“We are excited to complete our PEAK NDA submission which marks a significant step toward bringing a new therapy to patients with second-line GIST,” said Andrew Robbins, President and Chief Executive Officer. “Based on the strength of the PEAK data, we believe the bezuclastinib combination has the potential to meaningfully change the treatment landscape for these patients. We are grateful to the patients, investigators, and study teams who made this possible.”

Pivotal data from PEAK, a global, randomized Phase 3 clinical trial evaluating bezuclastinib in combination with sunitinib vs. sunitinib monotherapy in patients with GIST who have received prior treatment with imatinib, were reported in November 2025. As disclosed in the top-line results, the bezuclastinib combination demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. Additionally, the bezuclastinib combination demonstrated an unprecedented ORR in imatinib-resistant patients, with 46% of patients treated with the bezuclastinib combination achieving an objective response compared to 26% of patients treated with sunitinib. The bezuclastinib combination was generally well tolerated, and no unique risks were observed with the novel combination when compared to the known safety profile of sunitinib. Data for overall survival remains immature.

At the time of data cutoff, based on the number of ongoing patients receiving treatment on the bezuclastinib combination arm, the estimated mean duration of treatment for the bezuclastinib combination is projected to exceed 19 months.

Cogent plans to present full results from the PEAK trial at a major medical meeting during the first half of 2026. Additionally, Cogent is on track this quarter to initiate a Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib. The NDA submission with bezuclastinib in Advanced Systemic Mastocytosis (AdvSM) also remains on track for the first half of 2026.

Bezuclastinib - Expanded Access Program

Working with the FDA, Cogent has established active Expanded Access Programs (EAPs) for U.S. patients with GIST or SM who meet disease-specific criteria and could benefit from treatment with bezuclastinib or the combination of bezuclastinib and sunitinib. A growing number of sites now offer access to the bezuclastinib EAPs. For more information please visit: https://www.cogentbio.com/bezuclastinib-program-development/#our-expanded-access-policy

Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Cogent also announced today that, on March 30, 2026, the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, approved the grants of “inducement” equity awards to six new employees under the company’s 2020 Inducement Plan with a grant date of March 30, 2026. The awards were approved in accordance with Listing Rule 5635(c)(4) of the corporate governance rules of the Nasdaq Stock Market. The employees received, in the aggregate, (i) nonqualified options to purchase 21,100 shares of Cogent common stock and (ii) 15,700 restricted stock units (RSUs). Each option has a 10-year term, an exercise price equal to the closing price of Cogent’s common stock on the grant date, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of the grant date and the remainder vesting in equal monthly installments over the subsequent 36 months, provided such employee remains employed through each such vesting date. The RSUs vest annually in equal installments over 4 years from the grant date, provided such employee remains employed through each such vesting date.

https://en.wikipedia.org/wiki/Bezuclastinib

Cogent Biosciences Announces Submission of New Drug Application for Bezuclastinib in Gastrointestinal Stromal Tumors (GIST)

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

2026-09-03T10:03:42.793+05:30

Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, announced the submission to the U.S. Food and Drug Administration (FDA) of the Company's NDA for neladalkib, an investigational ALK-selective inhibitor, in TKI pre-treated advanced ALK-positive NSCLC.

New Drug Application (NDA) based on data in TKI pre-treated patients from the global ALKOVE-1 Phase 1/2 clinical trial

"The advancement of neladalkib from first clinical trial initiation to NDA submission in less than four years represents a remarkable pace in oncology drug development, underscoring the vigor and urgency our team brought to this program and our deep commitment to the ALK-positive NSCLC community," said Darlene Noci, A.L.M., Chief Development Officer at Nuvalent. "We would like to extend our sincere gratitude to the patients, families and investigators who have made this progress possible, and are committed to working closely with the FDA throughout the NDA review process toward our goal of bringing neladalkib to patients as quickly as possible."

The application is based on data in TKI pre-treated patients with advanced ALK-positive NSCLC treated with neladalkib in the global, registration-directed ALKOVE-1 Phase 1/2 clinical trial. In this population, neladalkib demonstrated encouraging overall activity, including intracranial responses, the ability to address key drivers of disease progression, and a generally well-tolerated safety profile consistent with its ALK-selective, TRK-sparing design. The company plans to share detailed results at a future medical meeting.

Neladalkib has received breakthrough therapy designation from the FDA for the treatment of patients with locally advanced or metastatic ALK-positive NSCLC who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About Neladalkib

Neladalkib is an investigational, brain-penetrant, ALK-selective inhibitor created with the aim to overcome limitations observed with currently available ALK inhibitors. Neladalkib is designed to remain active in tumors that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with single or compound treatment-emergent ALK mutations such as G1202R. In addition, neladalkib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ALK inhibitors and to drive deep, durable responses for patients across all lines of therapy. Neladalkib has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About the ALKOVE-1 Phase 1/2 Clinical Trial

The ALKOVE-1 trial (NCT05384626) is a first-in-human Phase 1/2 clinical trial for patients with advanced ALK-positive NSCLC and other solid tumors. The completed Phase 1 portion enrolled ALK-positive NSCLC patients who previously received at least one ALK TKI, or patients with other ALK-positive solid tumors who had been previously treated or for whom no satisfactory standard of care exists. The Phase 1 portion of the trial was designed to evaluate the overall safety and tolerability of neladalkib, with additional objectives including determination of the recommended Phase 2 dose (RP2D), characterization of the pharmacokinetic profile, and evaluation of preliminary anti-tumor activity. The global, single arm, open label Phase 2 portion is designed with registrational intent for TKI pre-treated patients with advanced ALK-positive NSCLC. Global enrollment in ALKOVE-1 remains ongoing for adult and adolescent patients with ALK-positive solid tumors outside of NSCLC, and adolescent patients with ALK-positive NSCLC.

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

Some Improvement Seen in Cognitive Function With Exercise, Ibuprofen in Cancer

2026-09-02T11:01:39.706+05:30

In continution of my update on Ibuprofen

For adults with cancer receiving chemotherapy, an exercise intervention and low-dose ibuprofen improved some domains of cognitive function, according to a study published online April 20 in Cancer.

Michelle C. Janelsins, Ph.D., M.P.H., from the University of Rochester in New York, and colleagues randomly assigned 86 adult participants with cancer receiving chemotherapy and reporting cognitive problems to one of four study arms for six weeks: Exercise for Cancer Patients (EXCAP)-ibuprofen, EXCAP-placebo, ibuprofen only, and placebo only. EXCAP is a home-based, low- to moderate-intensity progressive walking and resistance exercise prescription.

The researchers found that compared with the placebo group, participants in the EXCAP-placebo group had significantly better attention performance on the Trail Making Test (–21.57 seconds). Greater improvements were seen for the ibuprofen-only group versus the placebo group (difference of –11.27 seconds). Improvements on the Functional Assessment of Cancer Therapy-Cognitive Function (FACT-Cog) "comments from others" subscale were exhibited by participants in both the EXCAP-ibuprofen and EXCAP-placebo groups compared with the placebo group. Participants in the EXCAP-placebo group had a significant improvement on Rapid Visual Processing mean latency compared with the placebo group; those receiving ibuprofen had an improvement compared with those receiving placebo, which was attributed to a trend after adjustment for reading score. Compared with those not receiving ibuprofen, the ibuprofen group performed less well on the Hopkins Verbal Learning Test‐Revised delayed.

"We are encouraged by the findings of this trial that suggest possible benefits of both interventions for some cognitive domains. Clearly, we saw a more pronounced effect with exercise, which is notable considering the multiple health benefits of exercise for cancer survivors," Janelsins said in a statement.

https://en.wikipedia.org/wiki/Ibuprofen

Some Improvement Seen in Cognitive Function With Exercise, Ibuprofen in Cancer - Drugs.com MedNews

Combining Cannabinoid With Opioid Does Not Relieve Pain in Knee Osteoarthritis

2026-09-01T12:20:50.939+05:30

For patients with knee osteoarthritis, combining a cannabinoid medication with an opioid does not provide additional pain relief, according to a study published in the May issue of Anesthesiology.

Katrina R. Hamilton, Ph.D., from Johns Hopkins University School of Medicine in Baltimore, and colleagues examined the acute analgesic and drug effects of synthetic delta-9-tetrahydrocannabinol and hydromorphone, alone and in combination, in 21 individuals with knee osteoarthritis (mean age, 63.4 years). Participants received oral combinations of placebo, hydromorphone (2 mg), and dronabinol (10 mg).

The researchers found greater pressure pain threshold analgesia for hydromorphone than dronabinol and greater capsaicin and noncapsaicin sensitized mechanical temporal summation analgesia versus placebo. No significant drug-related differences were seen for clinical pain severity, thermal threshold or tolerance, temporal summation, cold pressor, conditioned pain modulation, capsaicin-induced thermal threshold, central sensitization, general pain sensitivity, or physical functioning (two-minute walking distance, Timed Up and Go, and total stair climb time). Compared with all conditions, hydromorphone impaired working memory accuracy and produced greater good effects than placebo in terms of secondary outcomes. Working memory reaction time was impaired with hydromorphone + dronabinol, which produced greater high ratings than placebo, greater drug effects than placebo + hydromorphone, and higher nausea than hydromorphone. Greater high ratings were seen for dronabinol than hydromorphone.

"Some patients believe combining cannabis with opioids can help with pain, and clinicians may recommend or prescribe it in states where cannabis is legal," Hamilton said in a statement. "Our study suggests that isn't the case and patients may experience more side effects when the drugs are combined."

https://en.wikipedia.org/wiki/Cannabinoid

https://en.wikipedia.org/wiki/Opioid

Combining Cannabinoid With Opioid Does Not Relieve Pain in Knee Osteoarthritis - Drugs.com MedNews

FDA Approves Idvynso (doravirine and islatravir) for the Treatment of HIV-1 Infection in Adults

2026-08-31T11:45:38.224+05:30

In continuation of my update on Doravirine

Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced the U.S. Food and Drug Administration (FDA) approval of Idvynso, a new, two-drug single-tablet regimen of 100 mg doravirine and 0.25 mg islatravir, for the treatment of HIV-1 infection in adults to replace the current antiretroviral regimen in those who are virologically suppressed (HIV-1 RNA less than 50 copies per mL) on a stable antiretroviral regimen with no history of virologic treatment failure and no known substitutions associated with resistance to doravirine. Idvynso is contraindicated when co-administered with drugs that are strong cytochrome P450 (CYP)3A enzyme inducers and lamivudine (3TC) or emtricitabine (FTC). Co-administration with these drugs may decrease the effectiveness of Idvynso. See additional selected safety information on the following pages. Idvynso (pronounced ihd-VIHN-soh) will be available in pharmacies after May 11.

Islatravir

Doravirine

Idvynso is approved for adults with virologically suppressed HIV-1 with no history of virologic treatment failure and no known substitutions associated with resistance to doravirine
Idvynso is the first and only non-INSTI, tenofovir-free, once-daily, complete two-drug regimen to demonstrate non-inferior efficacy in a head-to-head Phase 3 trial versus three-drug regimen Biktarvyi (BIC/FTC/TAF)

“Advances in HIV treatment mean more people living with HIV are living longer — a remarkable achievement,” said Carl Baloney, Jr., president and chief executive officer of AIDS United. “People aging with HIV face additional health challenges, including managing multiple chronic conditions and medications at the same time. It is essential that management of HIV considers these factors in addition to virologic suppression when choosing an HIV treatment regimen.”

“Idvynso combines islatravir, a next-generation NRTI with multiple mechanisms of action, including translocation inhibition, with doravirine, an NNRTI with an established efficacy and safety profile. As the only two-drug, non-INSTI, tenofovir-free regimen, Idvynso expands therapeutic diversity beyond the currently available oral treatment options,” said Dr. Eliav Barr, senior vice president and chief medical officer, Merck Research Laboratories. “As the health needs of adults living with HIV change over time, Idvynso gives clinicians a new choice for HIV treatment. This approval marks an important new chapter in Merck’s long-standing commitment to research and discovery for people living with HIV.”

Idvynso is a complete regimen; co-administration with other antiretroviral medications for treatment of HIV-1 infection is not recommended. Severe skin reactions, including Stevens-Johnson syndrome/toxic epidermal necrolysis, have been reported with doravirine-containing regimens. Drug Rash with Eosinophilia and Systemic Symptoms was reported with Idvynso. Concomitant use of Idvynso and certain other drugs may result in known or potentially significant drug interactions, some of which may lead to loss of therapeutic effect of Idvynso and possible development of resistance, or possible clinically significant adverse reactions from greater exposures of a component of Idvynso. See additional selected safety information on the following pages.

“Idvynso is the first non-INSTI, tenofovir-free, two-drug regimen to demonstrate non-inferior efficacy to standard oral antiretroviral regimens, including Biktarvy. This makes Idvynso a potential alternative for people with virologically suppressed HIV who may need to switch their treatment,” said Dr. Amy Colson, director of research at Community Resource Initiative, Boston, Massachusetts.

Phase 3 studies supporting approval of Idvynso

The efficacy and safety of Idvynso is supported by Week 48 data from two randomized, active-controlled, non-inferiority trials [Trial 052 (NCT05630755) and Trial 051 (NCT05631093)] in virologically-suppressed (HIV-1 RNA less than 50 copies per mL) adults living with HIV. Participants must have been stably suppressed on their baseline regimen for at least 3 months prior to trial entry and had no history of treatment failure. Across the two trials, a total of 708 participants received once-daily Idvynso; of these, 81 (11%) participants were aged 65 years and older, including 10 (1%) aged 75 years and older.

In the double-blind Trial 052, participants were switched from Biktarvy [bictegravir/emtricitabine/tenofovir alafenamide (BIC/FTC/TAF)] to Idvynso. A total of 513 participants were randomized (2:1) and were switched to once-daily Idvynso (n=342) or remained on BIC/FTC/TAF (n=171). At baseline, participants had a mean age of 48 years (range: 19 to 77), 21% of participants were female, 61% were White, 31% were Black/African American, and 6% were Asian. A total of 23% identified as Hispanic/Latino.

In the open-label Trial 051, participants were switched from an oral ART (antiretroviral therapy) regimen to Idvynso. A total of 551 participants were randomized (2:1) and were switched to once-daily Idvynso (n=366) or remained on their baseline ART (bART) (n=185). Randomization was stratified by bART. At baseline, participants had a mean age of 50 years (range: 18 to 83), 40% of participants were female, 39% were White, 45% were Black/African American, and 5% were Asian. A total of 15% identified as Hispanic/Latino. At enrollment, 64% of the participants were receiving integrase strand transfer inhibitor (INSTI)-based regimens, 5% protease inhibitor (PI)-based regimens (including combinations with INSTI), and 30% were receiving other regimens.

https://en.wikipedia.org/wiki/Islatravir

https://en.wikipedia.org/wiki/Doravirine

FDA Approves Idvynso (doravirine and islatravir) for the Treatment of HIV-1 Infection in Adults

Injectable peptides are the new anti‑aging trend. But what evidence do we have they're safe for humans?

2026-08-29T09:27:14.802+05:30

Injectable peptides are the new anti-aging trend sweeping the beauty industry. These compounds are promoted on social media as tools for skin repair, collagen production and "cellular rejuvenation." They are widely available online from overseas sellers, despite many peptides being unregulated in Australia.

But what's in them? And are they safe?

Earlier this year, three people in the United States were fined thousands of dollars for their role in providing peptide injections, at an anti-aging festival in Las Vegas, to two women who later became critically ill. The pharmacy board was unable to determine why they got sick, and what precisely the serums contained.

Our work with colleagues at Steroid QNECT, a hotline where people can seek confidential advice about enhancement drugs, shows that people are already injecting peptides in Australia.

But regulation is not keeping up. And there are still major gaps in the evidence about whether peptides' anti-aging claims stack up, and whether they are safe for humans.

What are injectable peptides, and why are they trending?

Peptides are short chains of amino acids—the building blocks of proteins. They act as chemical messengers in the body and play a key role in many processes. These include helping repair skin and calming inflammation.

The body naturally produces peptides. Synthetic peptides are manufactured to mimic or enhance these natural functions.

Certain peptides have clear medical uses. For example, glucagon-like peptide-1 (GLP-1) drugs, such as semaglutide, are approved for diabetes and weight management—sold as Ozempic and Wegovy. This is based on strong clinical trial evidence that they are effective.

But a growing number of peptides are being marketed for cosmetic and anti-aging purposes, without approval from Australia's therapeutic goods regulator.

Peptides such as GHK-Cu, BPC-157 and TB-500 are sold online with claims they can enhance collagen production, accelerate skin repair, reduce wrinkles, and even reverse aspects of biological aging.

TB 500

BPC-157

GHK-Cu

https://en.wikipedia.org/wiki/TB-500

https://en.wikipedia.org/wiki/BPC-157

https://en.wikipedia.org/wiki/Copper_peptide_GHK-Cu

Injectable peptides are the new anti‑aging trend. But what evidence do we have they're safe for humans?

Experimental drug cuts Parkinson's-linked protein up to 60% in early trial

2026-08-27T11:34:15.839+05:30

An experimental drug designed to silence a gene strongly linked to Parkinson's disease has shown encouraging effects in a first-in-human clinical trial, according to a study published in Nature Medicine. The drug, known as BIIB094 (ION859), an antisense oligonucleotide targets LRRK2, the most common genetic contributor to Parkinson's disease; variants in LRRK2 are known to increase the risk of developing the neurodegenerative disorder, which affects nearly 10 million people worldwide

Scientists have long believed that lowering the activity of the LRRK2 protein could help slow or modify the disease, but turning that idea into a viable therapy has remained a challenge, said co-author of the study Danielle Larson, MD, '15, '18 GME, assistant professor in the Ken & Ruth Davee Department of Neurology's Division of Movement Disorders.

"This was a multicenter clinical trial looking at an antisense oligonucleotide therapy for LRRK2-specific Parkinson's disease," Larson said. "The main goal was to examine the safety of delivering this therapy to patients, with the hope that if it proved safe, future studies could evaluate whether it might slow disease progression."

The study tested whether BIIB094 could safely reduce LRRK2 levels in people with Parkinson's disease. The results suggest it can be done without serious safety concerns, Larson said.

In the randomized, placebo-controlled trial, 82 participants with Parkinson's disease were enrolled across two study segments. In the first, 40 participants received a single dose of BIIB094 or placebo. In the second, 42 participants received four doses of the drug or placebo, administered every four weeks. The therapy was delivered intrathecally: directly into the cerebrospinal fluid through a lumbar puncture.

Participants in the second section were stratified based on whether they carried a known LRRK2 genetic variant.

Across both parts of the trial, the treatment was generally well tolerated. While adverse events were common, they were mostly mild to moderate and did not limit dosing. No serious adverse events related to BIIB094 were reported, according to the study.

Beyond safety, the study provided strong evidence that BIIB094 hit its intended biological target. Analysis of cerebrospinal fluid showed that levels of LRRK2 protein fell by as much as 59% in treated participants.

These reductions were observed regardless of whether patients carried a known LRRK2 mutation, suggesting the therapy could potentially affect a broader group of Parkinson's patients, not just those with a genetic diagnosis, Larson said.

"The antisense oligonucleotide was designed specifically to reduce LRRK2 expression," Larson said. "Because overactivity of this protein kinase is thought to be part of the problem in Parkinson's disease, reducing LRRK2 levels could be protective and potentially modify the disease."

The new findings hint at a possible mechanism by which LRRK2-targeting therapies could influence the underlying disease process, rather than simply addressing symptoms, Larson said.

However, Larson cautioned that the trial was not designed to assess clinical benefits such as changes in movement, cognition, or disease progression. Larger and longer studies will be needed to determine whether reducing LRRK2 levels translates into meaningful improvements for patients.

"The next step would be a phase 2 study with a larger group of patients," Larson said. "Instead of focusing only on safety, those trials would look at efficacy, whether the therapy can slow disease progression using motor assessments and standard Parkinson's rating scales."

The findings mark an important step toward targeted treatments that address the root biology of Parkinson's disease and underscore the potential of BIIB094 and other precision-medicine approaches, Larson said.

"This is one of the first antisense oligonucleotide, or ASO, therapies in Parkinson's disease to have safety and tolerability data," Larson said. "It really paves the way for other ASO-based treatments to be developed, potentially targeting different biological pathways."

https://www.nature.com/articles/s41591-026-04262-4

E xperimental drug cuts Parkinson's-linked protein up to 60% in early trial

Immunome Announces Submission of New Drug Application to U.S. FDA for Varegacestat for the Treatment of Adults with Desmoid Tumors

2026-08-24T10:58:54.369+05:30

Immunome, Inc. (Nasdaq: IMNM), a biotechnology company committed to developing first-in-class and best-in-class targeted cancer therapies, announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for varegacestat, an investigational, oral, once-daily gamma secretase inhibitor (GSI), for the treatment of adults with desmoid tumors.

New Drug Application (NDA) supported by positive Phase 3 RINGSIDE results, including significant improvement in progression-free survival vs. placebo (hazard ratio = 0.16, p<0.0001)
Trial also met all key secondary endpoints, with varegacestat delivering an objective response rate of 56%
Detailed Phase 3 RINGSIDE data selected for oral presentation at the 2026 ASCO Annual Meeting

“The varegacestat NDA submission marks an important milestone for Immunome. It reflects the strength of the RINGSIDE dataset and the commitment of the team advancing this program,” said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Immunome. “We believe varegacestat has the potential to provide adults with progressing desmoid tumors with a meaningful new oral treatment option, and we are grateful to the patients, families, investigators and study site teams whose participation made this submission possible.”

The NDA is supported by positive results from the global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial of varegacestat in patients with progressing desmoid tumors.

The trial met its primary endpoint of improving progression-free survival, demonstrating a statistically significant and clinically meaningful improvement vs. placebo, with an 84% reduction in the risk of disease progression or death (hazard ratio (HR) = 0.16, 95% CI: 0.071, 0.375; p<0.0001). The confirmed objective response rate (ORR) based on RECIST v1.1 was 56% with varegacestat vs. 9% with placebo (p<0.0001), as assessed by blinded independent central review.

In an exploratory analysis, varegacestat demonstrated a median best change in tumor volume of -83% vs. +11% with placebo, as assessed by blinded independent central review. In addition, the trial met all key secondary endpoints, with varegacestat achieving statistically significant improvements vs. placebo in landmark tumor volume reduction and worst pain intensity.

Varegacestat was generally well tolerated, with a manageable safety profile consistent with the GSI class. The most common adverse events for participants in the treatment arm were diarrhea (82%), fatigue (44%), rash (43%), nausea (35%) and cough (34%). Most events were grade 1 or 2.

Immunome previously announced that data from RINGSIDE has been selected for presentation in an oral abstract session at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 29-June 2, 2026 in Chicago.

About the RINGSIDE Trial

The global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial (NCT04871282) evaluated the efficacy and safety of varegacestat in patients with progressing desmoid tumors. A total of 156 patients were randomized to receive varegacestat 1.2 mg daily or placebo until disease progression or death, representing the largest randomized study in this population. The primary endpoint of the trial was progression-free survival as assessed by blinded independent central review. Statistically controlled secondary endpoints were confirmed ORR using RECIST v1.1 and change in tumor volume at week 24, both determined by blinded independent central review, as well as change in pain intensity at week 12 as determined using a patient reported outcome instrument. Additional secondary endpoints included duration of response, best reduction in tumor volume, patient-reported outcomes, and safety and tolerability. RINGSIDE includes an open-label extension phase, which is ongoing.

About Desmoid Tumors

Desmoid tumors (also known as aggressive fibromatosis or desmoid-type fibromatosis) are aggressive non-metastatic soft tissue tumors that are prone to recurrence. Approximately 1,000-1,650 people are diagnosed with desmoid tumors each year in the United States, and there are approximately 10,000-11,000 actively managed patients. Those affected face debilitating pain, deformity and, in some cases, life-threatening organ damage. The chronic pain and physical limitations associated with desmoid tumors lead to a high clinical burden and impaired quality of life. Although desmoid tumors are not considered cancerous, they often require systemic treatment to prevent permanent disability and alleviate disease burden.

About Varegacestat

Varegacestat (formerly AL102) is an investigational, oral, once-daily gamma secretase inhibitor. In December 2025, Immunome reported positive topline results for the Phase 3 RINGSIDE trial of varegacestat in adults with progressing desmoid tumors. Data from RINGSIDE have been selected for oral presentation at the 2026 ASCO Annual Meeting.

https://www.medkoo.com/products/7653

mmunome Announces Submission of New Drug Application to U.S. FDA for Varegacestat for the Treatment of Adults with Desmoid Tumors

FDA Accepts New Drug Application for Zipalertinib for the Treatment of Non-Small Cell Lung Cancer

2026-08-21T21:37:06.801+05:30

Taiho Oncology, Inc., Taiho Pharmaceutical Co., Ltd., and Cullinan Therapeutics, Inc.(Nasdaq: CGEM) announced the U.S. Food and Drug Administration (FDA) acceptance of New Drug Application (NDA) for zipalertinib for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion (ex20ins) mutations whose disease has progressed on or after platinum-based chemotherapy, with or without amivantamab. The Prescription Drug User Fee Act (PDUFA) target action date is February 27, 2027.

NDA submission based on the Phase 2b REZILIENT1 clinical trial, which demonstrated clinically meaningful and durable responses in patients with relapsed EGFR exon 20 insertion–mutated NSCLC
Prescription Drug User Fee Act (PDUFA) target action date is February 27, 2027

The NDA is supported by data from the Phase 2b part of the REZILIENT1 clinical trial of zipalertinib monotherapy in patients with NSCLC harboring EGFR ex20ins mutations who have received prior therapy. The study met its primary endpoint of objective response rate. Study results from REZILIENT1 were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and simultaneously published in the Journal of Clinical Oncology.

“Zipalertinib was discovered at Taiho Pharmaceutical Co., Ltd., and has been developed with a focus on addressing the unmet needs of patients with EGFR exon 20 insertion-mutated non-small cell lung cancer,” said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. “The FDA’s acceptance of the NDA for zipalertinib is an important milestone for this program, and we look forward to working with FDA during the review process.”

“Zipalertinib is a compound created using Taiho Pharmaceutical’s proprietary drug discovery and development technologies, Cysteinomix, with the aim of delivering a new treatment option to address high unmet medical needs,” said Takeshi Sagara, PhD, Executive Director, Board Member, Medical Affairs, Translational Development, Clinical Development, Discovery and Preclinical Research at Taiho Pharmaceutical. “The FDA’s acceptance of the NDA represents an important milestone, reflecting the scientific and clinical data accumulated to date. We will continue to work closely with Taiho Oncology, Cullinan Therapeutics and the FDA throughout the review process, with the shared goal of ultimately delivering a new treatment option to patients with non-small cell lung cancer EGFR exon 20 insertion mutations.”

“FDA acceptance of the zipalertinib NDA is an important step toward making zipalertinib available for people living with non-small cell lung cancer with EGFR exon 20 insertion mutations, who continue to face limited treatment options,” said Jeffrey Jones, MD, MBA, Chief Medical Officer, Cullinan Therapeutics. “We are deeply grateful to the patients and families who have participated in the REZILIENT program, and to the investigators, study teams, and advocates whose collaboration made achievement of this milestone possible. We believe zipalertinib has the potential to help address a significant unmet need, and we look forward to working with our partners at Taiho with the goal of bringing zipalertinib to patients waiting for new treatment options.”

Summary of Primary Study Results:

Zipalertinib demonstrated clinically meaningful efficacy in the primary efficacy population (n=176), including 51 patients who had received prior amivantamab.
The confirmed objective response rate (ORR) was 35%. Median duration of response (mDOR) was 8.8 months
In patients treated after prior platinum-based chemotherapy only (n=125), ORR was 40% with a mDOR of 8.8 months.
In exploratory subgroup analyses:
- Patients who had received prior amivantamab without other ex20ins-targeted therapy (n=30) showed a confirmed ORR of 30% and mDOR of 14.7 months.
- Patients with brain metastases (n=68) showed a confirmed ORR of 31% and a mDOR of 8.3 months.
The safety profile of zipalertinib was manageable and consistent with previously reported data.¹ The most common treatment-emergent adverse events were paronychia, rash, anemia, dermatitis acneiform, diarrhea, dry skin, nausea and stomatitis. Most treatment-emergent adverse events were grade 1 or 2 per NCI-Common Terminology Criteria for Adverse Events (CTCAE v5.0).

Zipalertinib is an oral EGFR tyrosine kinase inhibitor. Zipalertinib received Breakthrough Therapy Designation in 2021 for the treatment of patients with locally advanced or metastatic NSCLC harboring EGFR ex20ins mutations who have previously received platinum‑based systemic chemotherapy.

https://en.wikipedia.org/wiki/Zipalertinib

U.S. Food and Drug Administration Accepts New Drug Application for Zipalertinib for the Treatment of Locally Advanced or Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations

New drug combination doubles down on Alzheimer's treatments

2026-08-20T14:20:27.320+05:30

A new study has found that combining the current medications for Alzheimer's disease with small molecules derived from micronutrients found in grapes, berries, peanuts and turmeric is a safer and more effective way to treat the disease. The work is published in the journal ACS Chemical Neuroscience.

Individuals with Alzheimer's have a buildup of toxic amyloid proteins in the brain. Researchers from the School of Pharmacy at the University of Waterloo combined amyloid-destroying small molecules with anti-amyloid antibodies that are already used in Alzheimer's treatment. They found that it neutralized the clumping of proteins that accumulate in the brain, leading to better outcomes.

Alzheimer's is the major cause of dementia. Dementia affects nearly 750,000 people in Canada, with a million cases expected by 2030. Alzheimer's has no cure and current medications only relieve a patient's symptoms. Anti-amyloid antibody therapies on their own can slow the disease, but they also come with risks that can be fatal, including brain swelling and bleeding.

"We already know the small molecules resveratrol or curcumin, which are found in some common foods, block the buildup of amyloid," said Dr. Praveen Nekkar Rao, a professor in the School of Pharmacy at Waterloo. "What's new and exciting is our combination of these molecules with the anti-amyloid antibodies. This approach could allow clinicians to use lower doses of antibodies, potentially reducing the risk of serious treatment-related side effects."

Since there are few effective treatments for Alzheimer's, researchers at Waterloo studied whether using two treatments together could work better than using just one. They chose resveratrol and curcumin because they are natural compounds known to reduce amyloid buildup and inflammation.

"I was inspired by chemotherapy, which involves taking multiple medications for effective treatment," Nekkar Rao said. "Alzheimer's is a complex disease, but there are very few combination therapy approaches. Our results show that the way forward is definitely combination therapy."

The researchers emphasize that the study does not suggest that people should start consuming resveratrol or curcumin to prevent or treat dementia. You would have to consume an unsafe amount in order to reach the brain. The next phase of the research will focus on designing next-generation drugs that can reach the brain more effectively, interact favorably with amyloids and pair seamlessly with antibody treatments.

https://pubs.acs.org/doi/10.1021/acschemneuro.5c00760

New drug combination doubles down on Alzheimer's treatments

U.S. FDA Grants Full Approval of Kite’s Tecartus for Adult Patients with Relapsed or Refractory Mantle Cell Lymphoma

2026-08-18T09:29:43.234+05:30

The U.S. Food and Drug Administration (FDA) has granted traditional (full) approval to Kite’s CAR T-cell therapy, Tecartus (brexucabtagene autoleucel), for adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL). The full approval now includes efficacy, safety and pharmacokinetic data from Cohort 3 of the ZUMA-2 study in patients who are R/R after one or more lines of therapy and who are Bruton tyrosine kinase inhibitor (BTKi)-naïve.

Today’s action converts the R/R MCL indication to full approval based on the totality of evidence from ZUMA-2, including confirmatory data from Cohort 3, which demonstrated high response rates, durable efficacy outcomes and a manageable safety profile consistent with prior experience. This milestone fulfills Kite’s post-marketing requirement for verification and description of clinical benefit in a confirmatory trial under the FDA’s Accelerated Approval pathway for Tecartus in R/R MCL.

“The full approval of brexu-cel in relapsed or refractory mantle cell lymphoma, along with the inclusion of Cohort 3 data in the label, reinforces our confidence in the overall profile of brexu-cel,” said Michael Wang, M.D., ZUMA-2 Lead Investigator and Professor, Department of Lymphoma and Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center. “The Cohort 3 results showed high response rates, including deep remissions, in patients who were Bruton tyrosine kinase inhibitor-naïve, with a manageable safety profile consistent with prior experience. These data provide important information to help guide treatment decisions in the relapsed or refractory setting for appropriate patients.”

MCL is a rare form of non-Hodgkin lymphoma (NHL) that arises from cells originating in the “mantle zone” of the lymph node and predominantly affects men over the age of 60. Approximately 33,000 people worldwide are diagnosed with MCL each year. MCL is highly aggressive following relapse, with many patients’ disease progressing following therapy.

“The full approval of Tecartus for adults with relapsed or refractory mantle cell lymphoma reflects meaningful progress,” said Gallia Levy, MD, PhD, Senior Vice President and Global Head of Development, Kite. “For those whose cancer has returned, the data support Tecartus as a second-line treatment option with the potential to deliver long-term remission.”

Data Supporting Full Approval of Tecartus in R/R MCL

ZUMA-2 is a single-arm, open-label, multicenter study evaluating Tecartus in adult patients with R/R MCL. Cohorts 1 and 2 evaluated Tecartus in patients who had previously received up to five lines of therapy including anthracycline- or bendamustine-containing chemotherapy, an anti-CD20 antibody and a BTKi. Cohort 3 evaluated Tecartus in patients who had received up to five prior lines of therapy and were BTKi-naïve. A total of 82 patients were treated in Cohorts 1 and 2, and 86 patients were treated in Cohort 3. The primary endpoint across the study was objective response rate (ORR) per the Lugano Classification (2014), as assessed by an Independent Radiologic Review Committee.

U.S. FDA Grants Full Approval of Kite’s Tecartus for Adult Patients with Relapsed or Refractory Mantle Cell Lymphoma

FDA Approves Revtorpyk (gedatolisib) for the Treatment of HR+/HER2-, PIK3CA Wild-Type Locally Advanced or Metastatic Breast Cancer

2026-08-17T11:14:40.586+05:30

Celcuity Inc. (Nasdaq: CELC), a biotechnology company focused on developing and commercializing targeted therapies for multiple solid tumor indications, announced the U.S. Food and Drug Administration (“FDA”) approval of Revtorpyk (gedatolisib) for the treatment of patients with hormone receptor positive (“HR+”), human epidermal growth factor receptor 2 negative (“HER2-”), locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after treatment with at least one line of endocrine therapy in the metastatic setting. Revtorpyk is the only inhibitor of class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2 to receive FDA approval.

Revtorpyk is the first and only FDA-approved therapy that inhibits all class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2
In the Phase 3 VIKTORIA-1 trial, Revtorpyk combined with palbociclib and fulvestrant and Revtorpyk combined with fulvestrant reduced the risk of disease progression or death by 76% and 67%, respectively, compared to fulvestrant among patients with PIK3CA wild-type advanced or metastatic breast cancer

“The PI3K/AKT/mTOR, or PAM, pathway is one of the most important targets in cancer, but comprehensively inhibiting it has stymied researchers and drug developers for nearly two decades,” said Brian Sullivan, CEO and co-founder of Celcuity. “Revtorpyk addressed this 20-year challenge by becoming the first pan-PI3K, mTORC1/2 inhibitor approved by the FDA. We are thankful for the opportunity to make this important new therapy available to patients with HR+/HER2- locally advanced or metastatic breast cancer.”

HR+/HER2- breast cancer is the most common subtype of breast cancer, accounting for approximately 70% of all breast cancers.1 Among this breast cancer subtype, approximately 60% have PIK3CA wild-type disease.2

“For patients with HR+/HER2- locally advanced or metastatic breast cancer, there is an urgent need for new treatment options that can meaningfully increase the likelihood of survival without disease progression or death,” said Sara Hurvitz, MD, Senior Vice President, Clinical Research Division, Fred Hutchinson Cancer Center, Smith Family Endowed Chair in Women’s Health and Professor and Head, Division of Hematology and Oncology, University of Washington, School of Medicine and co-principal investigator for the VIKTORIA-1 trial. “With the approval of Revtorpyk, oncologists now have an effective new treatment option for these patients.”

The approval of Revtorpyk is based on positive clinical results from the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 trial, an open-label, global, randomized clinical trial evaluating the efficacy and safety of Revtorpyk plus fulvestrant, with or without palbociclib, for the treatment of patients with locally advanced or metastatic HR+/HER2- breast cancer following progression on or after CDK4/6 therapy and an aromatase inhibitor. In the VIKTORIA-1 trial, median progression free survival (“PFS”) with the Revtorpyk triplet (Revtorpyk plus palbociclib and fulvestrant) was 9.3 months versus 2.0 months with fulvestrant, an incremental improvement of 7.3 months (HR=0.24; 95% CI: 0.17-0.35; p<0.0001). The objective response rate (“ORR”) of the Revtorpyk triplet was 32% compared to 1% with fulvestrant and the median duration of response (“DOR”) was 17.5 months. For the Revtorpyk doublet (Revtorpyk plus fulvestrant), the median PFS was 7.4 months versus 2.0 months with fulvestrant, an incremental improvement of 5.4 months (HR=0.33; 95% CI: 0.24-0.48; p<0.0001). The ORR of the Revtorpyk doublet was 28% and the median DOR was 12.0 months. The median DOR was not determinable for fulvestrant because there was only one objective response.

“Today’s approval of Revtorpyk addresses a significant unmet need for the thousands of patients affected each year by HR+/HER2-, PIK3CA wild-type locally advanced or metastatic breast cancer whose disease has progressed after endocrine therapy,” said Igor Gorbatchevsky, MD, Chief Medical Officer of Celcuity. “We are deeply grateful to the patients and their caregivers, investigators and clinical study teams, and Celcuity team members who made this advancement possible.”

Celcuity anticipates commercial launch in late Q3 2026. Based on the company’s commitment to ensuring broad, affordable, and unrestricted patient access to Revtorpyk, we have designed a comprehensive patient support program. To make Revtorpyk available to patients prior to commercial launch, those who are eligible will be able to enroll in Celcuity’s expanded access program.

Celcuity plans to submit in Q3 2026 a supplemental New Drug Application (“sNDA”) to the FDA for Revtorpyk for the treatment of HR+/HER2-, PIK3CA mutated, locally advanced or metastatic breast cancer, following at least one line of endocrine therapy based on results from the mutant cohort of the Phase 3 VIKTORIA-1 trial. Results for this study were recently presented in a late-breaking abstract oral session at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. Following the sNDA submission, Celcuity intends to submit VIKTORIA-1 data for marketing authorization of gedatolisib to other regulatory authorities around the world.

Revtorpyk is also being studied in the ongoing Phase 3 VIKTORIA-2 clinical trial incorporating two independent studies evaluating two separate patient cohorts with HR+/HER2- locally advanced or metastatic breast cancer who are treatment-naive in the advanced setting.

About Revtorpyk (gedatolisib)

Revtorpyk is a kinase inhibitor of class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2, resulting in downstream inhibition of multiple effectors, including AKT.3.4.5

Revtorpyk is in development for the first-line treatment of HR+/HER2- locally advanced or metastatic breast cancer and for the second-line treatment of metastatic castration resistant prostate cancer.

Indication Statement

Revtorpyk (gedatolisib) is a kinase inhibitor indicated in combination with fulvestrant, with or without palbociclib, for the treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after treatment with at least one line of endocrine therapy in the metastatic setting.

https://en.wikipedia.org/wiki/Gedatolisib

FDA Approves Revtorpyk (gedatolisib) for the Treatment of HR+/HER2-, PIK3CA Wild-Type Locally Advanced or Metastatic Breast Cancer

FDA Accepts New Drug Application for Genentech’s Giredestrant in ESR1-Mutated, ER-Positive Advanced Breast Cancer

2026-08-14T12:32:18.985+05:30

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced the U.S. Food and Drug Administration (FDA) acceptance of the company’s New Drug Application for giredestrant, an investigational oral therapy, in combination with everolimus for the treatment of adult patients with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated locally advanced or metastatic breast cancer following recurrence or progression on a prior endocrine-based regimen. The FDA is expected to make a decision on the approval by December 18, 2026. Giredestrant plus everolimus could be the first and only oral selective estrogen receptor degrader (SERD) combination approved in the post-cyclin-dependent kinase (CDK)4/6 inhibitor setting.

Filing acceptance based on Phase III data showing giredestrant plus everolimus reduced the risk of disease progression or death by 44% and 62% in ITT and ESR1-mutated populations, respectively, versus standard-of-care endocrine therapy plus everolimus

Strength of evERA data demonstrate potential for giredestrant combination to help address resistance to standard-of-care therapies, and could be the first and only oral SERD combination approved in the post-CDK4/6 inhibitor setting

“The clinically meaningful benefit seen with giredestrant could enable an important new treatment option to help delay disease progression or death in people with advanced, ER-positive breast cancer,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “This acceptance marks a first step towards establishing the giredestrant combination as a new standard of care in this population.”

The filing acceptance is based on the Phase III evERA Breast Cancer study results, which showed that giredestrant plus everolimus reduced the risk of disease progression or death by 44% and 62% in the intention-to-treat (ITT) and ESR1-mutated populations, respectively, compared with standard-of-care endocrine therapy plus everolimus. In the ESR1-mutated population, the median progression-free survival (PFS) was 9.99 months compared with 5.45 months in the giredestrant and comparator arm, respectively (stratified hazard ratio [HR]=0.38, 95% CI: 0.27-0.54, p-value=<0.0001). In the ITT population, the median PFS was 8.77 months compared with 5.49 months in the giredestrant and comparator arms, respectively (HR=0.56, 95% CI: 0.44-0.71, p-value=<0.0001).

Overall survival (OS) data were immature at the time of analysis, but a clear positive trend has been observed in the ITT (HR=0.69, 95% CI: 0.47-1.00, p-value=0.0473) and ESR1-mutated populations (HR=0.62, 95% CI: 0.38-1.02, p-value=0.0566). Follow-up for OS will continue to the next analysis. Adverse events for the giredestrant combination were manageable and consistent with the known safety profiles of the individual medicines. No unexpected safety findings were observed, including no photopsia.

Data from evERA are being used to support filing submissions to other global health authorities.

ER-positive breast cancer accounts for approximately 70% of breast cancer cases. Resistance to endocrine therapies, particularly in the post-CDK inhibitor setting, increases the risk of disease progression and is associated with poor outcomes. Oral combination therapies, such as giredestrant plus everolimus, could address this by targeting two different signaling pathways while helping to minimize the impact of treatment on people’s lives without the need for injections.

evERA was the first positive Phase III readout for giredestrant, followed by lidERA Breast Cancer in the early-stage setting. The scientific rationale for lidERA was supported by prior results in the neoadjuvant setting, including the coopERA trial showing that giredestrant was superior to an aromatase inhibitor in reducing malignant cell division (Ki67 levels). This growing body of evidence underscores the potential of giredestrant to become a new standard-of-care endocrine therapy across ER-positive early-stage and advanced breast cancer. In the coming weeks, Genentech will submit the giredestrant Phase III lidERA data in early-stage breast cancer to the FDA. The persevERA readout in first-line ER-positive breast cancer is expected in the first half of this year, which will provide further evidence for giredestrant in the ER-positive breast cancer treatment paradigm.

Our extensive giredestrant clinical development program spans multiple treatment settings and lines of therapy, reflecting our commitment to deliver innovative medicines to as many people with ER-positive breast cancer as possible.

https://en.wikipedia.org/wiki/Giredestrant

FDA Accepts New Drug Application for Genentech’s Giredestrant in ESR1-Mutated, ER-Positive Advanced Breast Cancer

Xspray Pharma Re-Submits its FDA Application for Dasynoc

2026-08-13T11:13:22.980+05:30

Xspray Pharma AB (publ) has re-submitted its application for market approval for Dasynoc® to the U.S. Food and Drug Administration (FDA). Dasynoc® is the company's lead product candidate – an amorphous dasatinib for the treatment of leukemia. The resubmission includes the additional information requested by the FDA. A new PDUFA-date is expected to be set for August 2026 at the latest.

Xspray Pharma has submitted its response to the CRL (Complete Response Letter) received from the FDA in October 2025. The CRL related to the company’s NDA application (New Drug Application) for market approval in the United States for Dasynoc, an amorphous dasatinib for the treatment of CML (chronic myeloid leukemia) and ALL (acute lymphoblastic leukemia).

The current resubmission includes additional data specifically designed to address FDA’s concerns regarding risk for medication error. A qualitative human factors study has been completed to address this, confirming high prescriber comprehension using the new tablet strengths. The requested documentation confirming the effectiveness of previously implemented manufacturing measures was also submitted.

The GMP-observations in the company’s third-party manufacturing facility are being handled by the facility itself, and corrective measures are being implemented. Xspray welcomes the recent acquisition of the facility by Benta Group, a well-established company with core operations in pharmaceutical contract manufacturing and a strong reputation for quality, regulatory compliance and operational standards.

“Although the progress and approval process of the facility is not under our direct control, current information that is available to us reassures us that the issues in the facility will be resolved. We maintain our plan to launch two products, Dasynoc and XS003 nilotinib during the second half of 2026,” comments Per Andersson, CEO of Xspray Pharma.

The Dasynoc application will now undergo FDA review. Once the FDA formally confirms acceptance of the application, a new PDUFA date will be announced, expected no later than August 2026.

https://en.wikipedia.org/wiki/Dasatinib

Xspray Pharma Re-Submits its FDA Application for Dasynoc

Telix Resubmits NDA to U.S. FDA for TLX101-Px (Pixclara) Brain Cancer Imaging Candidate

2026-08-10T13:53:14.432+05:30

Telix Pharmaceuticals Limited (ASX: TLX, NASDAQ: TLX, “Telix”) announced the resubmission of a New Drug Application (NDA) to the United States (U.S.) Food and Drug Administration (FDA) for TLX101-Px, (Pixclara®1, Floretyrosine F 18 or 18F-FET), an investigational PET2 imaging agent for the characterization of recurrent or progressive glioma (brain cancer) from treatment related changes in both adult and pediatric patients.

Telix has resubmitted the NDA with the additional data requested by the FDA. The Company believes, based on the Type A meeting and ongoing consultation with the FDA, that the additional data and statistical analysis, along with the primary data set provided in the original submission, appropriately addresses the Complete Response Letter3.

Given the potential to address significant unmet medical need, TLX101-Px has been granted Orphan Drug4 and Fast Track5 designations by the FDA. PET imaging with 18F-FET is already included in international clinical practice guidelines for the imaging of gliomas6, however there is currently no FDA-approved targeted amino acid PET agent for adult and pediatric brain cancer imaging commercially available in the U.S.

Dr. David N. Cade, Telix Group Chief Medical Officer, said, “We appreciate the FDA’s recognition of the critical unmet need to improve the diagnosis and management of glioma, particularly in the post-treatment setting. Our resubmission is supported by an extensive and compelling data set – particularly so for an orphan indication. We are grateful to our global clinical collaborators, who share our commitment to ensuring patients in the U.S. can benefit from this important patient management tool.”

Maggie Haynes, Executive Director, Head for the Cure Foundation, added: “Our community is encouraged by the FDA’s ongoing engagement and guidance to the sponsor and support for the Expanded Access Program for TLX101-Px. We are hopeful of an expedited review, so this important and proven imaging option can become available to those who urgently need it.”

https://en.wikipedia.org/wiki/Fluoroethyl-L-tyrosine_(18F)

T elix Resubmits NDA to U.S. FDA for TLX101-Px (Pixclara) Brain Cancer Imaging Candidate

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

2026-08-05T10:17:10.970+05:30

Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced the submission to the U.S. Food and Drug Administration (FDA) of the Company's NDA for neladalkib, an investigational ALK-selective inhibitor, in TKI pre-treated advanced ALK-positive NSCLC.

New Drug Application (NDA) based on data in TKI pre-treated patients from the global ALKOVE-1 Phase 1/2 clinical trial

About Neladalkib

https://go.drugbank.com/drugs/DB16688

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

Aldeyra Therapeutics Receives Complete Response Letter from the U.S. Food and Drug Administration for the Reproxalap New Drug Application for the Treatment of Signs and Symptoms of Dry Eye Disease

2026-08-03T12:02:31.740+05:30

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to discovering and developing innovative therapies designed to treat immune-mediated diseases, announced receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) of reproxalap, an investigational drug candidate, for the treatment of dry eye disease. The CRL stated that there is “a lack of substantial evidence consisting of adequate and well-controlled investigations … that the drug product will have the effect it purports or is represented to have under the conditions of use prescribed, recommended, or suggested in its proposed labeling” and that “the application has failed to demonstrate efficacy in adequate and well controlled studies in the treatment of signs and symptoms of dry eye disease.” The letter also stated that the “inconsistency of study results raises serious concerns about the reliability and meaningfulness of the positive findings” and that the “totality of evidence from the completed clinical trials does not support the effectiveness of the product.” Consistent with prior NDA reviews of reproxalap, no safety or manufacturing concerns were identified.

During the NDA review, label drafts were provided by the FDA in December 2025 and again in March 2026. Aldeyra does not believe that label negotiations were completed.

The FDA recommended that the reasons for failure in certain trials be explored, and that populations or certain conditions in which reproxalap may be effective be identified. The FDA did not recommend conducting additional trials or request submission of additional confirmatory evidence. As such, Aldeyra does not currently expect to pursue additional clinical trials, and intends to expeditiously request a Type A meeting to understand the actions needed for NDA approval. Per Prescription Drug User Fee Act (PDUFA) goals, the target Type A meeting date is within 30 days of receipt of the meeting request.

“To the thousands of American and Canadian patients who participated in our clinical trials and to the tens of millions of patients with dry eye disease worldwide, I want to assure you that we will work with urgency to support the FDA in enabling market access to what is, to our knowledge, the only drug with clinical activity within minutes of administration in patients with dry eye disease, a condition that is today treated with medications that require weeks or months of treatment to achieve even modest improvement,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra.

https://go.drugbank.com/drugs/DB16688

Aldeyra Therapeutics Receives Complete Response Letter from the U.S. Food and Drug Administration for the Reproxalap New Drug Application for the Treatment of Signs and Symptoms of Dry Eye Disease