• Romaciclib in acute myeloid leukemia (AML):
  • In the RIVER-81 Phase II study, romaciclib combined with venetoclax (VEN) shows encouraging anti-leukemic activity, including durable responses, in patients with relapsed/refractory AML (r/r AML) after VEN-based therapy failure. These results support further clinical development at the selected dose.
  • On May 28, 2026, the Food and Drug Administration (FDA) reactivated the IND, enabling the initiation of the expansion cohort at the recommended dose of 150 mg once daily (QD).
• Romaciclib in myelofibrosis (MF):
  • In the POTAMI-61 Phase II study, romaciclib demonstrates a favorable safety profile and clinical activity in myelofibrosis (MF), both as monotherapy and in combination with ruxolitinib (RUX). The observed spleen volume reductions and hematologic tolerability support continued clinical evaluation.
  • Eight patients from POTAMI-61 will continue treatment in the ROVER-01 study, reflecting ongoing clinical benefit in a subset of patients.

KRAKOW, Poland, June 11, 2026 / Biotech Newswire / -- Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel therapies that address emerging targets in oncology, presents romaciclib (RVU120) data at the European Hematology Association Congress (EHA), June 11-14, 2026, in Stockholm, Sweden.

Hendrik Nogai, M.D., Chief Medical Officer of Ryvu Therapeutics said: We are pleased to share updated clinical data at EHA that reinforce the potential of romaciclib across hematologic malignancies. The poster presentations highlight consistent clinical activity and a manageable safety profile, both as a single agent and in rational combinations, including in difficult-to-treat patient populations. Importantly, the FDA review of the program and the recent reactivation of the RIVER-81 IND in the US as well as the continued treatment of patients from POTAMI-61 in the ROVER-01 study, are meaningful signals reflecting both regulatory progress and continued patient benefit. These milestones strengthen our confidence in romaciclib's differentiated mechanism of action and support its continued clinical advancement.

Program updates:

In the RIVER-81 trial of patients with r/r AML, Ryvu is initiating an expansion cohort of approximately 30 patients to confirm the positive efficacy demonstrated in the dose cohort of 150 mg QD romaciclib combined with 400 mg QD venetoclax. Following completion of the required regulatory steps, including an updated study protocol and submission of documentation under the existing IND, the FDA reactivated the IND for RIVER-81 study on May 28, 2026.

In the POTAMI-61 study investigating romaciclib’s efficacy in MF, eight patients will be transferred into the ROVER-01 study rollover protocol, underscoring that a subset of patients continue to derive meaningful clinical benefit from romaciclib treatment. These patients will continue to receive treatment with romaciclib as long as they benefit in ROVER-01.

EHA poster presentations:

Poster (PF529):  Updated findings from the Phase 2 RIVER-81 study of romaciclib (RVU120) combined with venetoclax in acute myeloid leukemia after first-line venetoclax and hypomethylating agent failure
Poster session date and time: Friday, June 12 (6:45 - 7:45 PM CEST)

Venetoclax (VEN) combined with hypomethylating agents is the standard first-line treatment for patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy, yet ~70% experience relapsed/refractory disease with a median survival of under three months. Romaciclib, a first-in-class CDK8/CDK19 inhibitor, has shown single-agent activity in AML and preclinical synergy with VEN through enhanced apoptotic signaling and attenuation of resistance pathways.

In the ongoing Phase 2 RIVER-81 trial, romaciclib in combination with VEN demonstrates anti-leukemic activity in patients with poor prognostic AML. While responses have been observed across dose levels, the most consistent responses were seen at romaciclib 150 mg QD + VEN 400 mg QD, including two complete remissions with (CR) and one without (CRi) full hematologic recovery, resulting in a CR/CRi rate of 43% (3/7). The mean duration of response (DoR) of the CRs was 156 days, reported with a cut-off date of 12 May 2026. Based on safety, PK, and preliminary efficacy findings, this regimen was selected as the recommended dose for expansion.

Poster (PS2008):   Romaciclib (RVU120), a selective CDK8/19 inhibitor, as monotherapy or in combination with ruxolitinib in patients with myelofibrosis: Updated results from the phase II POTAMI-61 study
Poster session date and time: Saturday, June 13 (6:45 - 7:45 PM CEST)

Myelofibrosis (MF) is driven by dysregulated JAK/STAT signaling, and while JAK inhibition with ruxolitinib (RUX) improves splenomegaly and symptoms, cytopenias and suboptimal responses remain clinical challenges. Romaciclib is a first-in-class, oral CDK8/19 inhibitor that modulates STAT-dependent transcription and shows synergy with RUX in preclinical MF models.

In the ongoing Phase II POTAMI-61 study, romaciclib administered as monotherapy or combined with RUX demonstrates a manageable safety profile in patients with MF without significant treatment-related cytopenias. Prolonged exposure, spleen volume reductions, including in patients with high-molecular-risk mutations, and favorable hematologic tolerability support continued clinical development.

The posters are available online and can be accessed via the conference website: https://ehaweb.org/ and Ryvu’s website: https://ryvu.com/

The project entitled “The conduct of a phase II, multicentre, open-label clinical trial (RIVER-81) evaluating the safety and efficacy of RVU120 in combination with venetoclax in patients with relapsed/refractory acute myeloid leukemia who have failed prior therapy with venetoclax and a hypomethylating agent.”, carried out by Ryvu Therapeutics S.A., is co-financed by the Medical Research Agency from the state budget under the “Development of targeted or personalised medicine based on medicinal products involving nucleic acids and small-molecule compounds” call (ABM/2022/6).

Total Project value: PLN 132,471,740.70
Total funding: PLN 62,268,848.90

Semantic keywords: Congresses as Topic; Clinical Trials, Phase II as Topic; Ryvu Therapeutics; romaciclib; RVU120; European Hematology Association Congress; EHA; hematologic malignancies; oncology; acute myeloid leukemia; AML; relapsed/refractory AML; r/r AML; myelofibrosis; MF; RIVER-81; POTAMI-61; ROVER-01; venetoclax; VEN; ruxolitinib; RUX; CDK8/CDK19 inhibitor; selective CDK8/CDK19 inhibitor; drug development; clinical data; program update; clinical activity; anti-leukemic activity; combination therapy; JAK inhibition; JAK/STAT signaling

About Ryvu Therapeutics
Ryvu Therapeutics is a clinical-stage drug discovery and development company focused on novel oncology therapies that address emerging targets in oncology. Internally discovered pipeline candidates at Ryvu use diverse therapeutic mechanisms driven by emerging knowledge of cancer biology, including small molecules and antibody-drug conjugates directed at kinases, synthetic lethality, and immuno-oncology targets.    
Ryvu’s most advanced program is romaciclib (RVU120, SEL120), a selective CDK8/CDK19 kinase inhibitor with the potential to treat hematological malignancies and solid tumors. Romaciclib is currently in Phase II development in combination with venetoclax for the treatment of patients with r/r AML – the RIVER-81 study. Additional studies are ongoing including medulloblastoma, the MEDWAY collaboration with the Children's Memorial Health Institute, and in myelofibrosis.
Dapolsertib (MEN1703, SEL24) is a dual PIM/FLT3 kinase inhibitor licensed to the Menarini Group that is currently being investigated in a Phase II study in diffuse large B-cell lymphoma (DLBCL) – the JASPIS-01 study. RVU305, a potentially best-in-class, brain-permeable PRMT5 inhibitor aiming to treat multiple solid tumors, has completed IND/CTA-enabling studies. Ryvu Therapeutics is also engaged in oncology collaborations with BioNTech and Exelixis. 
Ryvu was founded in 2007 and is headquartered in Kraków, Poland. It is listed on the Warsaw Stock Exchange and is a component of the sWIG80 index.

Contact

Ryvu Therapeutics
Anna Wilk
+48 532 698 425
anna.wilk@ryvu.com

Source: Biotech Newswire

• Clinical Trial approval for TCX-001 is another significant development milestone for T-CURX and marks the transition of T-CURX from a pre-clinical to a clinical-stage biotech company
• TCX-001 targeting Siglec-6 is a first-in-class autologous, non-viral investigational CAR-T therapy for r/r AML as the lead indication and for CLL. This is a testament to T-CURX strategy to develop highly differentiated CAR-T products beyond targets and indications for which CAR-T therapies are already available.
• Siglec-6 is a differentiated target present on AML-blasts and leukemic stem cells, but, unlike most other AML targets, is not expressed on healthy hematopoietic stem or progenitor cells. Siglec-6 CAR-T cell therapy has the potential to be an effective treatment for AML patients, rather than only being a “bridge to transplant”.

WÜRZBURG, Germany, June 11, 2026 / Biotech Newswire / -- Today, leading European CAR-T biotech company T-CURX announces its first clinical trial approval by Swiss agency Swissmedic for the company’s non-viral  lead CAR-T program, TCX-001, targeting Siglec-6. The  first-in-human Phase 1 study will now be initiated at University Hospital Zürich as the first clinical trial site to treat patients with relapsed/refractory (r/r) Acute Myeloid Leukemia (AML) and Chronic Lymphocytic Leukemia (CLL), including patients that are not eligible for stem cell transplantation. A complementary CTIS application will also be filed at European Medicines Agency (EMA) to extend the trial to four sites in Germany led by University Hospital Würzburg (UKW). The study will enrol up to 23 adult patients with r/r AML or CLL to determine the recommended Phase 2 dose. Primary endpoints are safety, tolerability, and dose-limiting toxicities with key secondary endpoints including overall response rate, duration of response, as well as CAR-T cell expansion and persistence.

Siglec-6 was identified as a leukemia target by T-CURX co-founder Christoph Rader through the natural anti-leukemic immune response of a CLL patient cured by allogeneic stem cell transplantation. It was characterized as a highly promising  CAR-T target in AML by Michael Hudecek, co-founder and CMO at T-CURX. In contrast to other AML targets, e.g. CD33 or CD123, Siglec-6 is not expressed on healthy hematopoeitic stem or progenitor cells. This spares the hematopoietic stem cell compartment from destruction by CAR-T cells. Based on this unique aspect, Siglec-6 CAR-T therapy could provide an effective therapy for AML as a stand alone treatment, rather than just a “bridge to transplant”.

TCX-001 is a next-generation autologous CAR-T product incorporating several proprietary innovations including virus-free Sleeping  Beauty transposon gene transfer and novel CAR-linker (“matchmaker”-) technologies for high scalability and optimal potency. GMP-certified manufacturing has been established at the German Red Cross Blood donation center in Frankfurt, Germany.

Ulf Grawunder, CEO and co-founder of T-CURX said. “We are excited about the clinical trial approval for our lead CAR-T program TCX-001 leveraging highly innovative non-viral CAR-T technologies. This is an important milestone for T-CURX in our quest to develop first-in-class, transformative CAR-T therapies and to make them accessible for patients with a scalable and economical non-viral CAR-T manufacturing process.”

Michael Hudecek, CMO and co-founder of T-CURX added "TCX-001 targets Siglec-6 and is manufactured using T-CURX non-viral Sleeping Beauty platform, the same technology validated for clinical use in our prior academic clinical trials, CARAMBA-1 and LION-1, sponsored by University Hospital Würzburg. We have high hopes that TCX-001 will ultimately provide meaningful and durable benefit to r/r AML and CLL patients who have exhausted current therapies."

Upon clinical validation of ex vivo manufactured non-viral CAR-T therapy TCX-001, T-CURX will build upon this clinical expertise to also combine it with its in vivo CAR-T strategy. This development is accelerated by the  recent acquisition of Pantherna Therapeutics and integrating their proprietary LNP-based nucleic acid delivery technologies into T-CURX clinical CAR-T pipeline.

Semantic keywords: Leukemia, Lymphocytic, Chronic, B-Cell; Receptors, Chimeric Antigen; Cell- and Tissue-Based Therapy; Sialic Acid Binding Immunoglobulin-like Lectins; Leukemia, Myeloid, Acute; Clinical Trials, Phase I as Topic; T-CURX; TCX-001; non-viral CAR-T; Siglec-6; First-in-Human Phase 1 clinical trial; relapsed/refractory AML; Chronic Lymphocytic Leukemia (CLL); clinical-stage biotech company; first-in-class autologous CAR-T therapy; differentiated CAR-T products; AML-blasts; leukemic stem cells; healthy hematopoietic stem cells; CAR-T cell therapy; stem cell transplantation; Swissmedic; CAR-T cell expansion; CAR-T cell persistence; leukemia target; anti-leukemic immune response; allogeneic stem cell transplantation; next-generation autologous CAR-T product; Sleeping Beauty transposon gene transfer; CAR-linker technologies; GMP-certified manufacturing; non-viral CAR-T manufacturing process

About T-CURX
T-CURX GmbH is a private, Würzburg & Munich based, German, clinical-stage biotech company with a wholly owned subsidiary, Pantherna Therapeutics in Hennigsdorf & Berlin, Germany, specialized on development of LNP delivery technologies for nucleic acids into cells for therapeutic applications. T-CURX is financially backed by a syndicate of international VC investors and family offices from Germany and Switzerland, led by Swiss BiomedVC, with Bayernkapital (Germany), Highlight Capital (China) and i&iBio Fund (Czech Republic) as institutional co-investors. T-CURX has the vision to bring innovative CAR-T cell therapies based on cost-effective and highly scalable non-viral ex vivo and in vivo CAR-T strategies to more cancer patients in need of these effective cancer therapies. T-CURX was spun out of the laboratory of T-CURX’s co-founder Prof. Michael Hudecek at the University of Würzburg and is led by Ulf Grawunder, PhD, a seasoned serial entrepreneur as CEO, who is also one of the co-founders.

Contacts

T-CURX
Ulf Grawunder, PhD, CEO
ulf@t-curx.com

For media enquiries
+49-(0)-931-250-99-712
pr@t-curx.com

Source: Biotech Newswire

VIENNA, Austria, June 08, 2026 / Biotech Newswire / -- AOP Health has launched the phase 1 clinical trial SERHEMA-1 to evaluate the first-in-class investigative drug AOP208 in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS), cancers with a dismal prognosis. This key milestone marks the progression of the clinical development program for AOP208, which began with the first-in-human trial SERONCO-1 and builds on AOP Health’s expertise in delivering cancer stem-cell targeting agents.

AOP208 is a first-in-class (1), orally administered antagonist of serotonin receptor 1B (HTR1B). This target is implicated in tumor cell survival and treatment resistance but has not previously been exploited in cancer therapy. Preclinical data and early clinical safety data demonstrate the potential for further development of AOP208 in patients with relapsed/refractory AML and high-risk MDS, who have limited treatment options.

First clinical experience with AOP208 prompts expanded research

The initial AOP208 trial, SERONCO-1 (EU CT No: 2023-509532-25-00) in patients with solid tumors or lymphoma is being conducted in a collaboration between AOP Health and Leukos Biotech and the Vall d’Hebron Institute of Oncology, in Barcelona, with partial funding from the Spanish Ministry of Science and Innovation public-private partnership program (CPP2021-008715). The study has delivered promising early safety results supporting escalation to higher doses. (2)

Christoph Klade, Chief Scientific Officer of AOP Health explains: “After successfully advancing our phase I trial in solid tumors, we are now expanding development in blood cancers. AOP208 has a novel mechanism of action with potential for targeting leukemic stem cells and the tumor microenvironment in the bone marrow niche. This next step reflects the encouraging data generated so far and builds on our long-standing expertise in hemato-oncology.”

Phase 1 clinical trial SERHEMA-1 treats first patient

The first patient has begun treatment with AOP208 in SERHEMA-1 (EU CT No: 2025-523396-38-00), a phase I/II trial in relapsed or refractory AML and high-risk MDS. This study is taking place in specialized centers in Spain and Austria and is sponsored by AOP Health. Initiation of the SERHEMA-1 trial represents a significant step towards understanding the safety and tolerability of AOP208 and exploring its anti-tumor activity.​   

Zoltan Magyarics, Vice-President of Research & Development at AOP Health emphasized: “Advancing research in rare diseases is central to our mission. We are pleased to contribute our scientific expertise to a clinical program that seeks to expand knowledge and offer therapeutic options in an area of high unmet medical need in hemato-oncology.”

Sematic Keywords: Drug Development; Clinical Trials, Phase I as Topic; Myelodysplastic Syndromes; Leukemia, Myeloid, Acute; Receptors, Serotonin; Hematologic Neoplasms; AOP Health; phase 1 clinical trial; SERHEMA-1; acute myeloid leukemia (AML); myelodysplastic syndrome (MDS); AOP208; orally administered antagonist; serotonin receptor 1B (HTR1B); cancer therapy; clinical safety data; SERONCO-1; solid tumors; lymphoma; novel mechanism of action; tumor microenvironment; bone marrow niche; hemato-oncology; phase I/II trial; anti-tumor activity

About AOP208
AOP208 is a first-in-class, orally administered serotonin receptor 1B antagonist. AOP Health has licensed AOP208 from Leukos Biotech (a spin-off company from the Josep Carreras Leukaemia Research Institute in Barcelona) as well as the entire compound library generated to identify the drug candidate. AOP Health holds further development rights and exclusive global commercial rights including USA, Europe, Korea and Taiwan with the exclusion of mainland China. 

About AOP Health
AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company’s actions. (aop-health.com)

Contact

AOP Health
DI Isolde Fally
Isolde.Fally@aop-health.com
+43-676-500 4048
https://www.aop-health.com

References:

1. https://pubmed.ncbi.nlm.nih.gov/28193998/ and https://pubmed.ncbi.nlm.nih.gov/30224768/
2. Laimito KR, Lores GM, Nuciforo PG, Smiljkovic D, Unger M, Krejcy K, Klade C, Risueño R, Braña I. 1024eTiP LB-208/AOP208 a first-in-class HTR1B antagonist: First-in-human, dose finding phase I study for the evaluation of safety, pharmacokinetics, pharmacodynamics, and anti-tumor activity in adult patients with relapsed or refractory solid tumors or lymphoma. Annals of Oncology. 2025 Sep 1;36:S665-6.

Source: Biotech Newswire

STOCKHOLM, Sweden, June 03, 2026 / Biotech Newswire / -- Haga Bioscience (HAGA.BIO), a Swedish spatial biology startup, today announced it has closed a SEK 20.9 million (approximately USD 2.3 million) oversubscribed Seed financing round bringing total funding, including pre-seed financing, to approximately USD 3 million The company will use the capital to initiate commercialization of its spatial biology technologies designed specifically for validation and translational workflows.

The financing included participation from Almi Invest, Life Science Invest, and SU Ventures, alongside undisclosed life science industry veterans, family offices, and private investors.

Founded in 2024, Stockholm-based HAGA.BIO is focused on advancing the next phase of spatial biology by bridging the gap between spatial RNA biomarker discovery and clinical translation. While recent advances in spatial biology have enabled the discovery of large numbers of novel tissue based RNA biomarkers, validating these findings across large clinical cohorts remains a major challenge due to sample throughput and the high costs. HAGA.BIO said its technologies are specifically designed to enable cost effective scalable validation and translational workflows.

The technologies enable sensitive, specific detection of single nucleotide variants (SNVs) on RNA directly in situ, and are built on next-generation in situ hybridization methods developed by co-founders Hower Lee (CEO), Marco Grillo (CSO), and Mats Nilsson, professor at Stockholm University and scientific co-founder of the company.

Additional co-founders include Malte Kuhnemund, Daniel Gyllborg, and Xiaoyan Qian, previously at 10x Genomics. Kuhnemund and Qian co-founded Cartana in 2017, a SciLifeLab spinout whose in situ RNA sequencing technology was acquired by 10x Genomics in 2020 and integrated into the Xenium platform.

“Until now, the field has lacked robust in situ variant calling on FFPE tissue samples with high sensitivity and specificity,” said Hower Lee, co-founder and CEO of Haga Bioscience. “Addressing this challenge enables new possibilities for translational research.”

“Discovery is no longer the bottleneck,” said Mats Nilsson, professor at Stockholm University and scientific co-founder of Haga Bioscience. “The next major step for the field is translating and validating these discoveries at scale and ultimately into clinical use. That is precisely what HAGA.BIO aims to enable.”

The financing supports product development, commercialization, and collaborations with academic and industry partners to advance translational spatial biology. The company officially announced the early access program at the Cancer Research UK Cambridge Institute In Situ Hybridisation and Spatial Omics Symposium on May 20, 2026. 

Through the program, HAGA.BIO is providing early access to HAGA Pattern™, a multiplexed spatial gene expression assay, and HAGA Point™, a spatial assay for in situ SNV detection on RNA.

HAGA.BIO will also participate in the 2026 European Association for Cancer Research (EACR) Congress in Budapest from June 8 to 11, 2026, where the company plans to present its latest advances in translational spatial RNA analysis. 

Semantic keywords: Investments; Capital Financing; Haga Bioscience; spatial biology; Seed financing round; commercialization; spatial RNA biomarker discovery; biomarkers; clinical translation; tissue based RNA biomarkers; scalable validation; translational workflows; single nucleotide variants (SNVs); RNA; next-generation in situ hybridization; in situ variant calling; FFPE tissue samples; translational research; product development; academic and industry partners; translational spatial biology; HAGA Pattern™; multiplexed spatial gene expression assay; HAGA Point™; spatial assay; in situ SNV detection; spatial RNA analysis; EACR Congress

Contact

HAGA.BIO
Morgane Rouault
Chief Commercial Officer 
morgane@haga.bio 
www.hagabioscience.com

Source: Biotech Newswire

• T-CURX leverages Pantherna’s deep expertise and proprietary technologies in LNP-based delivery of mRNA and DNA vectors to advance T-CURX innovative in vivo CAR-T assets into clinical trials one year earlier
• T-CURX’ proprietary and clinically validated transposon-based non-viral CAR-T technology is potentiated and de-risked by Pantherna’s proprietary PTXmRNA® and PTXΔLNP® platforms, enabling the expansion of in vivo CAR-T therapies beyond cancer indications
• T-CURX’ acquisition of Pantherna builds on a successful prior technology collaboration and preclinical validation of their combined technologies

WÜRZBURG, Germany, June 01, 2026 / Biotech Newswire / -- Today, leading CAR-T company T-CURX announces the signing of a definitive agreement for the acquisition of Pantherna Therapeutics, a German biotechnology company specializing in mRNA engineering and lipid nanoparticle (LNP) delivery technologies. Pantherna will continue to operate as Pantherna Therapeutics as a wholly owned subsidiary of T-CURX. The complementary expertises and proprietary technologies of both companies will be leveraged to accelerate clinical translation of CAR-T strategies with a clear focus on clinical validation of in vivo CAR-T therapies. T-CURX is a Würzburg-headquartered private biotech company with an additional site in Munich with strong expertise, proprietary technologies and track record for clinical translation of non-viral, transposon-based CAR-T therapies for novel and differentiated targets. T-CURX is backed by private investors and an international syndicate of blue-chip Biotech investors led by Swiss BiomedVC. T-CURX closed a USD 20.5 million Series A financing round at the end of 2025. Pantherna was supported by a group of private investors and Germany-based High-Tech Gründerfonds (HTGF), who will become new shareholders of T-CURX. Financial terms of the transaction are not disclosed.

“With this acquisition, we are bringing together two highly complementary technology platforms and teams. We are taking a decisive step forward to accelerate the clinical development of differentiated, next-generation in vivo CAR-T therapies,” said Ulf Grawunder, CEO and co-founder of T-CURX. “Our strength in clinical translation of non-viral transposon-based CAR-T therapies combined with Pantherna’s proprietary mRNA and LNP-based delivery capabilities allows us to build an efficient engine to expedite clinical development of differentiated in vivo CAR-T assets in cancer, as well as in other indications.”

“Joining T-CURX is a natural evolution of our collaboration,” said Ansgar Santel, CEO of Pantherna Therapeutics. “This integration allows us to address both stable and transient cell engineering strategies based on powerful and proven proprietary platforms. We believe this positions us uniquely to efficiently drive CAR-T innovation forward for patients in need, particularly for in vivo CAR-T strategies across a broad range of therapeutic applications.”

The combination of both companies positions T-CURX to become a leading next-generation CAR-T player, being the first European Biotech with both proprietary CAR-T and LNP-technologies under one roof, allowing integration of key capabilities across the full value chain from discovery to clinical development of CAR-T products. Post acquisition, the company will have a total headcount of 35 and will manage a collective portfolio of currently 16 patent families protecting the combined technologies and assets.

Semantic keywords: Acquisition; biotechnology; T-CURX acquires Pantherna Therapeutics;  non-viral in vivo CAR-T; LNP-based delivery; mRNA engineering; mRNA and DNA vectors; transposon-based CAR-T technology; PTXmRNA®; PTXΔLNP®; clinical translation; clinical validation; in vivo CAR-T therapies; cancer indications; proprietary technologies; lipid nanoparticle delivery technologies; differentiated in vivo CAR-T assets; next-generation CAR-T therapies; stable and transient cell engineering; therapeutic applications; clinical development

About T-CURX
T-CURX GmbH is a private, Würzburg & Munich based, German biotech company with a wholly owned subsidiary, Pantherna Therapeutics, in Hennigsdorf & Berlin, Germany, combining next-generation non-viral CAR-T and LNP-nucleic acid vector delivery technologies under one roof. T-CURX is financially backed by a syndicate of international VC investors and family offices from Germany and Switzerland, led by Swiss BiomedVC, with Bayernkapital (Germany), Highlight Capital (China) and i&iBio Fund (Czech Republic) as institutional co-investors. T-CURX has the vision to bring innovative CAR-T cell therapies based on cost-effective and highly scalable non-viral ex vivo and in vivo CAR-T strategies to more cancer patients in need of these effective cancer therapies. T-CURX was spun out of the laboratory of T-CURX’s co-founder Prof. Michael Hudecek at the University of Würzburg and is led by Ulf Grawunder, PhD, a seasoned serial entrepreneur as CEO, who is also one of the co-founders. 

About Pantherna Therapeutics
Pantherna Therapeutics GmbH, a T-CURX company,  is a biotech company from Hennigsdorf, Germany, with additional operations in Berlin, specializing in mRNA engineering and lipid nanoparticle (LNP) delivery technologies. Its proprietary PTXmRNA® and PTXΔLNP® platforms enable transient, controllable gene expression and targeted in vivo delivery of nucleic acids. Pantherna adds a crucial technology layer for potentiating T-CURX next-generation CAR-T therapies, further advancing ex vivo as well as future in vivo CAR-T strategies in oncology and also additional indications.

Contacts

T-CURX
Ulf Grawunder, PhD, CEO
ulf@t-curx.com

Pantherna Therapeutics
Ansgar Santel, PhD, CEO
a.santel@pantherna-therapeutics.com

For media enquiries
+49-(0)-931-250-99-712
pr@t-curx.com 

Source: Biotech Newswire

• The results point to new directions for the expansion of clinical applications, particularly in tumor and metabolic diseases

BUDAPEST, Hungary, May 29, 2026 / Biotech Newswire / -- HYD LLC., the organizer of the 5th International Congress on Deuterium Depletion and its satellite event, the Aqua-Synapse research program workshop, announced that researchers from 12 countries demonstrated that recognition of deuterium's regulatory role in biological systems could fundamentally rewrite current therapeutic approaches. The Aqua-Synapse research program workshop was supported by the European Commission's Research Executive Agency (REA), in Budapest.

At the scientific events, speakers from Sweden, America, England, China, Russia, Canada, and Hungary presented their latest evidence on the physiological effects of the heavy isotope of hydrogen. The results not only enriched our understanding of the regulation of biological processes but also pointed to new directions for expanding clinical applications, especially in tumor and metabolic diseases. The fourteen presentations in the conference program were given by researchers from prestigious institutions including the Karolinska Institute in Sweden, the Massachusetts Institute of Technology (MIT) in the United States, Tulane University, and the University of Lisbon.

One of the keynote speakers at the conference was Gábor I. Csonka, a professor at Tulane University, who, in studies conducted with A549 lung cancer cells, demonstrated with his co-authors that while deuterium enrichment enhances signaling pathways related to inflammation and invasion, deuterium depletion results in their systemic downregulation, directly inhibiting the function of genes responsible for cancer development. Professor Csonka identified deuterium concentration as a clear modulator of oncogene transcription. Special attention was also paid to the presentation of Roman A. Zubarev, professor of the Royal Karolinska Institute in Sweden, who refuted the traditional “passive isotope” model, according to which isotopes are passive tracers of metabolic processes. Professor Zubarev pointed out that not only hydrogen, but also heavy isotopes of oxygen, nitrogen, and carbon have an active influence on biological systems. According to his experimental evidence, the scientific community is on the verge of a dramatic paradigm shift; deuterium-modulated diets and the control of heavy isotope ratios may become key biomedical applications in the near future. Tatyana Strekalova, from the University of Lisbon and also conducting research within the Aqua-Synapse program, demonstrated the protective effects of deuterium-depleted water (DDW) in an animal model. In subjects consuming DDW, in addition to increased hedonic sensitivity (increased susceptibility to positive stimuli and pleasure), novelty discovery, and improved memory, changes in brain expression of key genes (e.g., Egr1, Per2, Homer1) that are responsible for cellular stress, neural plasticity, and regeneration were observed.

In line with international scientific findings, the chief researcher at HYD LLC., Gábor Somlyai, presented results on the decisive role of the deuterium-to-hydrogen ratio in the metabolic pathways of tumor cells and the promising application possibilities of deuterium depletion in the treatment of metabolic diseases, such as diabetes. He demonstrated that the optimal deuterium concentration for proper insulin signaling lies between 125 and 140 ppm, emphasizing that the deuterium concentration in today’s average population is significantly higher due to reduced fat and high carbohydrate intake. Dr. Somlyai also reviewed the results of prospective Phase II randomized clinical trials and retrospective clinical studies demonstrating the anticancer effects of deuterium depletion. In conclusion, he stated that integrating deuterium depletion into conventional cancer treatment protocols could reduce cancer mortality by 75–80%.

In addition to the scientific results, significant international industrial players attending the conference confirmed the global potential of the technology's practical application and the growing market significance of the procedure based on Hungarian intellectual capital.

The scientific program of the 5th ICDD conference can be viewed here: https://deuteriumdepletion.com/5th-congress-2026/scientific-program/

You can read about the Aqua-Synapse project here: https://hyd.hu/en/hyd-llc-is-participant-in-the-eu-co-financed-project-aqua-synapse/

Semantic keywords: Gábor Somlyai; 5th International Congress on Deuterium Depletion; deuterium; therapeutic approaches; Aqua-Synapse research program; regulation of biological processes; tumor diseases; metabolic diseases; signaling pathways; systemic downregulation; inflammation; genes responsible for cancer development; deuterium-modulated diets; heavy isotope ratios; deuterium-depleted water; DDW; regeneration; metabolic pathways; diabetes; Phase II randomized clinical trial; anticancer effects

Contact

HYD LLC. for Cancer Research and Drug Development
Dr. Gábor Somlyai, Managing Director and Lead Researcher
+36-1/365-1660
info@hyd.hu

Source: Biotech Newswire

Highlights:

• Neurizon announces an expansion to the size of Regimen I intended to support a larger and more informative dataset, with accelerated time to topline readout and no change in funding requirements
• Increase in sample size driven by enrolment rate exceeding original expectations and the absence of a concurrent regimen during the expected recruitment period
• Regimen I expands from 160 to 240 participants to maintain statistical power for the primary endpoint aligned with the original trial assumptions
• The increased sample size allows more robust analysis of subgroups and biomarkers and ensures a fully powered study at topline results
• Based on current enrolment momentum, Neurizon anticipates that last participant dosing will now occur in Q2 CY2027 with topline results to be released early Q3 CY2027, exceeding previous trial timeframe projection
• Expanded participant cohort cost is materially offset by a contribution of philanthropic funds from the Sean M. Healey & AMG Center for ALS at Mass General Brigham
• As a result of diligent cost management and rapid recruitment, this change is expected to have no impact on Neurizon’s costs through to topline results and only a very modest increase in the total cost of completing the full trial
• Larger, consistent dataset expected to strengthen partnering and commercial positioning for NUZ-001
• 113 participants assigned and 74 dosed as of 22 May 2026, with 64 US clinical trial sites activated and 193 participants screened under the master protocol to date
• Neurizon to host a shareholder webinar on 28 May to discuss the trial recruitment expansion in further detail

MELBOURNE, Australia, May 27, 2026 / Biotech Newswire / -- Neurizon® Therapeutics Limited (ASX: NUZ & NUZOA; OTCQB: NUZTF) ("Neurizon" or "the Company"), a clinical-stage biotech company dedicated to advancing innovative treatments for neurodegenerative diseases, is pleased to advise that Regimen I of the HEALEY ALS Platform Trial will increase its total patient cohort from 160 to 240 participants. The expansion is driven by the enrolment rate exceeding original expectations and the absence of a concurrent regimen during the expected recruitment period. Expanded design maintains statistical power for the primary endpoint aligned with the original assumptions underpinning the study and allows more robust analysis of subgroups and biomarkers.  

Importantly, the current recruitment momentum is expected to accelerate the overall trial timeline relative to previous expectations, despite the increased recruitment target. Based on current trends, the Company now anticipates last participant dosing in Q2 CY2027, with topline results expected in early Q3 CY2027.

Following an extensive review of the scientific, statistical, financial and regulatory considerations, as well as ongoing engagement with the Sean M. Healey & AMG Center, Regimen I will now comprise 180 NUZ-001 participants and 60 Regimen I placebo controls.

As of 22 May 2026, 113 participants have been assigned to Regimen I and 74 participants have been dosed. There are 64 clinical trial sites activated across the United States and 193 participants have been screened to date under the master protocol. While current recruitment momentum continues to track ahead of prior expectations, future recruitment rates may vary over time.

Based on the expanded 240-patient design, accelerated enrolment timelines and a meaningful financial contribution of philanthropic funds from the Sean M. Healey & AMG Center for ALS at Mass General Brigham, Neurizon expects the total funding requirements through to study completion to remain unchanged.

Neurizon remains focused on efficiently delivering a scientifically meaningful outcome from the HEALEY ALS Platform Trial. The expansion of the trial has no impact on Neurizon’s expected costs through to topline results, with only a modest overall increase in the total cost of completing the full trial. This increase is expected to be further offset through eligibility under the Australian Federal Government’s R&D Tax Incentive Program, together with the financial contribution of philanthropic funds from the Sean M. Healey & AMG Center for ALS at Mass General Brigham. This demonstrates Neurizon’s continued focus on cost and capital efficiency and ensures the Company remains fully funded through completion of the trial.

Neurizon believes expanding the regimen is a fiscally disciplined and strategically attractive decision to generate a larger, more statistically robust, and internally consistent ALS dataset, which eliminates reliance on placebo groups from other regimens and maintains the original statistical assumptions underpinning the study at topline results.

Interim Executive Chairman, Mr Sergio Duchini said: “The expansion of Regimen I to 240 participants reflects our considerable confidence in NUZ-001 and is underpinned by strong momentum being delivered across the HEALEY ALS Platform Trial study. Importantly, this decision maintains the statistical power for the analysis of the primary endpoint and the potential for topline results ahead of our previously stated schedule.”

“The strength of recruitment and engagement across the HEALEY ALS Platform Trial network has positioned Neurizon to proactively optimise the study design at an important point in the trial. We believe the expanded dataset will strengthen the statistical robustness, interpretability and strategic value of the program as we advance toward topline results.”

“Importantly, the larger and more internally consistent dataset is expected to enhance future regulatory, partnering and commercial opportunities for NUZ-001, while also strengthening our ability to generate valuable biomarker and translational insights relevant to the broader neurodegenerative disease landscape.”

“ALS remains an area of urgent unmet medical need, and we believe this decision further strengthens Neurizon’s positioning as we continue advancing NUZ-001 through late-stage clinical development.”

Merit Cudkowicz, MD, MSc, Director of the Healey & AMG Center and Executive Director of the Mass General Brigham Neuroscience Institute, said: "This partnership with Neurizon reflects a shared commitment to advancing meaningful science while keeping people living with ALS at the center of everything we do. We thank our patient advisory committee members and the entire ALS community for active participation in this important clinical trial.  By working collaboratively, we can accelerate progress, strengthen the research ecosystem, and ultimately deliver solutions that better serve the ALS community."

Sabrina Paganoni, MD, PhD, Co-Principal Investigator of the HEALEY ALS Platform Trial and Co-Director of the Neurological Clinical Research Institute (NCRI) said: "I want to sincerely thank the Network of Excellence for ALS (NEALS) investigators and site teams for achieving our most rapid enrollment to date — an extraordinary milestone that reflects their dedication and collaboration. I also want to recognize the coordination center at the Healey & AMG Center and key vendors for outstanding implementation and execution of the study, which made this success possible, as well as participants and their families for their dedication and support."

Webinar details:

Neurizon will host an informational webinar on Thursday, 28 May 2026 at 4:00pm AEST (2:00pm AWST) to discuss this announcement and provide additional context around the expanded enrolment in the HEALEY ALS Platform Trial. Registration details are provided below. Participants are encouraged to submit questions in advance during the registration process or via company email to enquiries@neurizon.com.

Registration Link: https://bit.ly/NUZ-001 
Date: 28 May 2026
Time: 4:00pm AEST

About the HEALEY ALS Platform Trial:

The HEALEY ALS Platform Trial (ClinicalTrials.gov identifier: NCT04297683) is a multicentre, double-blind, placebo controlled adaptive Phase 2/3 clinical trial conducted by the Sean M. Healey & AMG Center for ALS at Mass General Brigham in the United States (US), created in partnership with the Network of Excellence for ALS (NEALS). Entry into the HEALEY ALS Platform Trial is competitive, with drug candidates reviewed and selected by expert committees based on scientific merit and evidence of potential benefit in ALS. The goal of the HEALEY ALS Platform Trial is to accelerate the development of potential new ALS therapies.

Semantic keywords: Clinical Trials, Phase II as Topic; Neurodegenerative Diseases; Amyotrophic Lateral Sclerosis; Motor Neuron Disease; HEALEY ALS Platform Trial Regimen I; increase of patient cohort ; 240 participants; topline results expected in Q3 CY2027; NUZ-001; neurodegenerative diseases; enrolment rate; recruitment period; Sean M. Healey & AMG Center for ALS; Mass General Brigham; rapid recruitment; clinical trial sites; accelerated enrolment timelines; statistically robust; internally consistent ALS dataset; placebo groups; study design; interpretability; strategic value; regulatory opportunities; biomarker insights; neurodegenerative disease landscape; partnering; commercial positioning

About Neurizon Therapeutics Limited
Neurizon Therapeutics Limited (ASX: NUZ) is a clinical-stage biotechnology company dedicated to advancing treatments for neurodegenerative diseases. Neurizon is developing its lead drug candidate, NUZ-001, for the treatment of ALS, which is the most common form of motor neurone disease.  Neurizon’s strategy is to accelerate access to effective ALS treatments for patients while exploring the potential of NUZ-001 for broader neurodegenerative applications. Through international collaborations and rigorous clinical programs, Neurizon is dedicated to creating new horizons for patients and families impacted by complex neural disorders.  NUZ-001 is an investigational product and is not approved for commercial use in any jurisdiction.

Neurizon Investor Hub
We encourage you to utilise our Investor Hub for any enquiries regarding this announcement or other aspects concerning Neurizon.
This platform offers an opportunity to submit questions, share comments, and view video summaries of key announcements.
To access Neurizon Investor Hub please scan the QR code or visit https://investorhub.neurizon.com

Neurizon® is a registered trademark of Neurizon Therapeutics Limited

This announcement has been authorised for release by the Board of Neurizon Therapeutics Limited. 

Contacts

Neurizon Therapeutics
Lidija Damjanovic
Marketing & Corporate Affairs
lidija@neurizon.com 
+61 (0) 425 700 504

Australia Investor Relations
Henry Jordan
Six Degrees Investor Relations
henry.jordan@sdir.com.au 
+61 (0) 431 271 538

Source: Biotech Newswire

BERLIN, Germany, May 27, 2026 / Biotech Newswire / -- ProBioGen today announced the launch of AGE1.CR.ReX®, its next-generation avian designer cell line for stable, high-titer poxvirus production and rapid generation of recombinant modified vaccinia Ankara (MVA). Enabled for switchable gene expression and the proprietary TetherexX™ System, the new platform is designed to accelerate and simplify the development of modern poxvirus-based vaccines and oncolytic viruses while improving genetic stability and manufacturing reliability.

Built on ProBioGen’s proven GMP AGE1.CR.pIX® lineage, AGE1.CR.ReX has been engineered to enable inducible control of transgene expression during virus production. This eliminates selection pressure against difficult or unstable transgenes, a common challenge in poxvirus vector development that can otherwise result in reduced viral titers, inconsistent batch performance, and repeated plaque purification cycles.

The AGE1.CR.ReX host cell line directly addresses these issues by enabling more stable recombinant MVA generation with higher productivity and increased process robustness. As a result, developers can achieve production-ready virus candidates faster and with greater confidence. Central to this is the TetherexX System, ProBioGen’s proprietary approach for rapid generation and rescue of pure recombinant MVA. Tetherin, a cellular factor of the innate immune system, is used here as a novel selection marker to selectively eliminate parental viruses, reducing the need for operator-intensive purification steps and supporting a more reliable path toward production-ready virus candidates.

Combined with the TetherexX System, the AGE1.CR.ReX platform offers several advantages:

• Pure recombinant MVA generated in as few as three passages
• Increased reliability and success rates through robust tetherin-based selection
• High-throughput compatibility for efficient screening of vaccine candidates
• Suitable for adherent and suspension cultures in chemically defined media

The TetherexX system together with the AGE1.CR.ReX cell line build the foundation for ProBioGen’s/Minapharm’s recent initiative towards an African Bundibugyo Ebola virus (BDBV) vaccine. In addition, AGE1.CR.ReX is designed for applications where conventional systems may fail to deliver both transgene stability and high viral titers and is also well suited in the development of oncolytic viruses, personalized medicines, and for supporting pandemic preparedness programs.

"In developing AGE1.CR.ReX, we focused on solving one of the bottlenecks that may impact recombinant poxvirus generation: maintaining stable transgene expression without compromising yields," said Dr. Ingo Jordan, Vice President Vaccine Strategies at ProBioGen. "The combination of inducible transgene control and the TetherexX System enables faster development timelines and more robust industrial manufacturing workflows for vaccine and viral vector developers."

"AGE1.CR.ReX and TetherexX are essential innovations for epidemic preparedness. They open the door for MVA as one of the rapid response vaccine platforms," explained Dr. Volker Sandig, Chief Scientific Officer at ProBioGen. "Without these technologies accelerated development and reliable manufacture of an MVA-based BDBV vaccine would not be possible."

AGE1.CR.ReX is fully developed and GMP-banked at ProBioGen and is available with full technology transfer and integrated GMP manufacturing support.

For high resolution please click the image.

Semantic keywords: Cell Line; Technology; Cell Culture Techniques; Culture Media; Cell Proliferation; Cell Line Development; Cells, Cultured; Ducks; Viruses; Vaccines; Viral Vaccines; Virus Cultivation; Biological Products; Productivity; Technology; Bioreactors; Germany; ProBioGen; AGE1.CR.ReX; avian designer cell line; poxvirus production; recombinant modified vaccinia Ankara; MVA; switchable gene expression; TetherexX System; poxvirus-based vaccines; oncolytic viruses; genetic stability; manufacturing reliability; control of transgene expression; virus production; poxvirus vector development; stable recombinant MVA generation; higher productivity; process robustness; production-ready virus candidates; tetherin; tetherin-based selection; Bundibugyo Ebola virus; BDBV vaccine; personalized medicines; pandemic preparedness programs; vaccine platforms; accelerated development; reliable manufacture; GMP manufacturing support

About ProBioGen
ProBioGen is a Berlin-based expert in the development and manufacturing of biopharmaceuticals, viral vectors, and vaccines, powered by proprietary technologies that enhance product quality and features. It has developed multiple innovative vaccine platforms, including Lenti.RiGHT®, AGE1 host cell lines and the IP-protected MVA-CR19 strain, which has a unique genomic signature. Operating for over 30 years, ProBioGen runs three manufacturing lines in Berlin, where 300 employees contribute to advancing next-generation therapies and global biotech innovation.
For more information about ProBioGen, follow us on LinkedIn.

Contact

ProBioGen
Dr. Gabriele Schneider
Chief Business Officer
cdmo@probiogen.de

ProBioGen Press Contact
Sarah Wandrey
Senior Communications Manager
press@probiogen.de

Source: Biotech Newswire

Pharmaceutical service provider receives prestigious management award for the seventh year in a row

• Family-owned company once again excels in all four key business areas
• Site development and digitalization as strategic investment priorities
• Focus on new sources for skilled workers and on lifelong learning

RAVENSBURG, Germany, May 26, 2026 / Biotech Newswire / -- Vetter, a leading Contract Development and Manufacturing Organization (CDMO), has been named a Best Managed Company for the seventh year in a row. The family-owned business was the only company in this year’s competition to be newly awarded Platinum status. The prestigious prize is awarded by Deloitte Private, UBS, Frankfurter Allgemeine Zeitung and the Bundesverband der Deutschen Industrie e.V. (Federation of German Industries, BDI) to companies for outstanding achievements in leadership and management. The jury evaluates companies according to four value drivers: strategy, productivity, culture and commitment, and governance and finance. In 2026, the CDMO received the award primarily for consistent further development of its corporate strategy and its operational processes in the context of a globally challenging environment. The trophy and certificate were presented to Vetter at an awards ceremony on May 21 at the Gesellschaftshaus Palmengarten in Frankfurt, Germany.

Strategic foresight, active risk management and sustainable growth

The jury was particularly impressed by the company's consistent and ongoing strategic efforts: The successful completion of the current Vetter NExT 2029 strategic program lays a solid foundation for developing and finalizing a new, forward-looking program this year. Vetter also stood out for its clear focus on achieving ambitious climate protection targets and its ongoing development of integrated corporate reporting and risk management, which strengthens its permanent ability to act. This enables the CDMO to act with foresight and reliability, even in times of geopolitical uncertainties and their potential impact on supply chains, energy supply and other external conditions.

"Being once again recognized as a Best Managed Company underscores the strength and resilience of our strategic corporate direction," says Senator h.c. Udo J. Vetter, Chairman of the Advisory Board and member of the owner family. "Our sustainable growth is the result of long-term thinking and investments in our service portfolio, and the required infrastructure. In this way, we consistently pursue our goal of supplying patients worldwide with vital medicines – and improving their quality of life every day." Vetter Managing Director Titus Ottinger adds: "Another key area of investment for us is in the digitalization of our value-added corporate processes. The potential impact is significant. So is our core objective: to reduce the number of manual interfaces, create more seamless data flows, and increase data integrity – without compromising on compliance. In this way, we are strengthening our position as a leader in quality and technology as well as our customers’ long-term trust in us."

Environmental responsibility and continuous investment in employees

The jury also recognized Vetter’s clearly defined, ambitious climate targets, through which the company is systematically working to reduce its CO₂ emissions, among other initiatives. The CDMO successfully combines environmental responsibility with economic success. In addition, the jury highlighted the company’s comprehensive commitment to securing skilled workers and employee development. Through innovative approaches such as the Integrative Entry Program, Vetter taps into additional skilled worker potential and enables successful qualification, language training and integration. The company-wide rollout of its digital platform Vetter Learning deliberately places a clear focus on lifelong learning, knowledge transfer and the continuous professional development of its employees.

Markus Seiz, Head of the Best Managed Companies (BMC) Program in Germany at Deloitte Private, emphasizes: "We have been working with Vetter for many years now, and we are impressed by the intensity and consistency with which the management and employees engage in strategic processes. The pharmaceutical service provider is also an excellent example of how the application process itself can be used as a source of additional momentum for the company."

© Vetter Pharma International GmbH: Senator h.c. Udo J. Vetter, Chairman of the Advisory Board and member of the owner family (right) along with Vetter Managing Director Titus Ottinger (left) at the awards ceremony at the Gesellschaftshaus Palmengarten in Frankfurt, Germany.

For high resolution please click the image.

Find the Vetter press kit and more background information here.

German 

Semantic keywords: Awards and Prizes; Vetter; Contract Development and Manufacturing Organization; CDMO; global clinical manufacturing network; clinical manufacturing; Best Managed Company; Platinum status; leadership and management; Vetter NExT 2029; strategic program; climate protection targets; risk management; geopolitical uncertainties; sustainable growth; service portfolio; digitalization; data integrity; compliance; quality and technology; CO2 emissions; environmental responsibility; skilled workers; integration; continuous professional development

About Vetter
Vetter is a leading Contract Development and Manufacturing Organization (CDMO) with headquarters in Ravensburg, Germany, and production facilities in Germany, Austria, and the US. As a global player, the independent pharmaceutical service provider is also present in the Asia-Pacific markets of Japan, China, South Korea and Singapore with sales locations. Around the world, renowned pharma and biotech companies benefit from decades of experience, high quality, modern technologies, reliability, and commitment of its 7,300 employees. In close collaboration with its customers, the Vetter team helps enable the supply to patients all over the world with medicines, many of which are vital. The CDMO provides support from drug product development through clinical and commercial filling to a wide range of assembly and packaging services for vials, syringes, and cartridges. With innovative approaches, Vetter develops prefilled drug-delivery systems together with its customers to continuously improve patient safety, comfort, and compliance. Vetter takes responsibility for sustainable practices and operates as a socially and ethically responsible corporate citizen. The CDMO is a member of the UN Global Compact and Science Based Target initiative (SBTi) and received platinum status in the renowned EcoVadis ranking. Multiple awards such as the CDMO Leadership Awards, Frost & Sullivan Customer Value Leadership Award and the recognition of Best Managed Company emphasize Vetter’s commitment to sustainable business. Founded in Ravensburg in 1950, the company remains family-owned to this day. For more information, visit www.vetter-pharma.com and follow Vetter on LinkedIn.

Contact

Vetter Pharma International GmbH
Markus Kirchner
Company Spokesperson / Media Relations
Eywiesenstraße 5
88212 Ravensburg
Germany
+49 (0)751-3700-3729
PRnews@vetter-pharma.com

Source: Biotech Newswire

• Following an analysis of Company resources and positive data in r/r AML, a population of high unmet medical need, Ryvu is prioritizing rapid development of romaciclib in AML. Updated clinical data from the Phase II RIVER-81 and POTAMI-61 studies will be presented at EHA 2026 (June 11-14, Stockholm).
• The Company signed the PERO grant agreement with the National Centre for Research and Development (NCBR), securing PLN 20.0 million in non-dilutive funding for an innovative oncology target validation platform.
• Total operating revenues in Q1 2026 amounted to €5.1 million, compared to €5.0 million for the same period in 2025. EBITDA loss was €2.4 million, compared to €8.4 million in the previous year.
• As of May 15, 2026, Ryvu’s cash position was €6 million. The Company has also secured approximately €20.6 million in non-dilutive grant funding.

KRAKOW, Poland, May 21, 2026 / Biotech Newswire / -- Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel therapies that address emerging targets in oncology, today reported financial results for the first quarter, ended March 31, 2026.

Pawel Przewiezlikowski, co-founder and Chief Executive Officer of Ryvu Therapeutics, said: "Clinical results in RIVER-81 and POTAMI-61 Phase II studies support romaciclib’s further development both in acute myeloid leukemia and myelofibrosis. Based on resources and clinical data generated so far, we decided to prioritize the AML development path by enrolling approximately 30 additional patients in RIVER-81. Alongside romaciclib clinical development, we have demonstrated good results in our early-stage programs, including RVU305, our potentially best-in-class MTA-cooperative PRMT5 inhibitor and ONCO Prime platform. Projects developed in cooperation with our partners are progressing, with potentially important milestones ahead."

Q1 2026 SUMMARY AND RECENT CORPORATE EVENTS

Romaciclib (RVU120) clinical development plan progress

Ryvu continues to advance the clinical development of romaciclib, a first-in-class selective CDK8/CDK19 inhibitor, across hematologic malignancies. Current data support continued development of romaciclib in acute myeloid leukemia (AML) and myelofibrosis (MF). With the particularly strong data in relapsed/refractory (r/r) AML, near-term priorities will focus on development in AML, where the high unmet medical need could lead to an accelerated approval path.

• Ryvu generated further clinical insights and will present updated clinical data from the RIVER-81 and POTAMI-61 studies at the European Hematology Association (EHA) Congress 2026 (June 11-14, Stockholm, Sweden). Based on available abstracts:
  • RIVER-81: Romaciclib combined with venetoclax (VEN) shows encouraging anti-leukemic activity, including durable responses, in patients with r/r AML after VEN-based therapy failure. These results support further clinical development at the selected dose.
  • POTAMI-61: Romaciclib demonstrates a favorable safety profile and clinical activity in MF, both as monotherapy and in combination with ruxolitinib. The observed spleen volume reductions and hematologic tolerability support continued clinical evaluation.
• On May 20, following the analysis of resources and clinical data generated so far, Ryvu decided to prioritize RIVER-81 study by enrolling approximately 30 additional patients. As part of resource prioritization, the Company decided to close the POTAMI-61 study (ongoing patients will continue to receive treatment in a new roll-over study ROVER-01). Ryvu retains the option to resume a modified MF development program. This approach is intended to preserve strategic optionality for romaciclib while, in the short term, focusing resources on the clinical dataset that is expected to be most relevant for potential development partners.

New preclinical data for the ONCO Prime platform were presented at the AACR Annual Meeting 2026. Ryvu’s integrated precision oncology discovery platform leveraging patient-driven models, engineered systems, and high-throughput CRISPR/omics analytics, enables the identification of novel synthetic lethal vulnerabilities in genetically stratified colorectal cancer models, including proof-of-concept validation using a clinically approved drug. This establishes a scalable platform for drug discovery programs and strategic partnerships built on ONCO Prime’s ability to bridge functional genomics with therapeutic development.

Extension of the research collaboration with BioNTech: on March 16, 2026, Ryvu reported that it amended its research collaboration and exclusive license option agreement with BioNTech, originally disclosed in November 2022. Under the amendment, the parties agreed to extend their research collaboration by an additional period of one year until November 2028.

Grant funding for the PERO project: on March 11, 2026, the Company signed a grant agreement with the National Centre for Research and Development (in Polish: Narodowe Centrum Badań i Rozwoju; NCBR) for the PERO (“Predictive Engineering for Rational Oncology”) project. The objective of PERO is to establish an innovative technological platform for the functional validation of structural protein pockets of potential therapeutic targets in oncology. The project addresses a significant technological gap by enabling an in-depth, currently unavailable level of functional target validation based on integrated genomic, structural, and pharmacological data. The funding, PLN 20.0 million of a PLN 32.4 million project, will significantly fund the development of an innovative platform for functional target validation in oncology.

Ryvu continues to advance partnerships with Menarini Group, BioNTech, and Exelixis, under which the Company is fully reimbursed for its expenses and has the potential to receive multiple financial milestones.

KEY UPCOMING INDUSTRY AND INVESTOR EVENTS

Ryvu will host partnering and investor meetings at the following conferences:

• ASCO Annual Meeting (Chicago, IL), May 29 to June 2
• EHA Annual Congress (Stockholm, Sweden), June 12-15
• BIO International Convention (San Diego, CA), June 22-25

Q1 2026 FINANCIAL UPDATE

Cash Position – On May 15, 2026, Ryvu Therapeutics held €17.6 million in cash, cash equivalents and investment funds. In addition, the Company has secured approximately €20.6 million in non-dilutive grant funding.
Operating Revenues – In Q1 2026, Ryvu recognized total operating revenues (including grants) of €5.1 million, compared with €5.0 million in Q1 2025.
Operating costs, related primarily to research and development expenditures, excluding the valuation of NodThera shares and non-cash cost of valuation of the Incentive Program for Q1 2026, amounted to €8.0 million, compared to €11.4 million in Q1 2025.
EBITDA result – EBITDA loss in Q1 2026 was €2.4 million, compared to €8.4 million in the previous year.
Net Loss Attributable to Common Shareholders – In Q1 2026, the net loss attributable to common shareholders, excluding the non-cash cost of the Incentive Program, amounted to €2.9 million, compared to €5.8 million in the same period of the previous year.

Semantic keywords: Financial results; PERO grant; Congresses as Topic; Clinical Trials, Phase II as Topic; Ryvu Therapeutics; romaciclib; RVU120; CDK8/CDK19 inhibitor; acute myeloid leukemia; AML; relapsed/refractory acute myeloid leukemia; r/r AML; venetoclax; VEN; RIVER-81; myelofibrosis; MF; ruxolitinib; POTAMI-61; hematologic malignancies; anti-leukemic activity; favorable safety profile; EHA 2026; European Hematology Association Congress; ONCO Prime; precision oncology discovery platform; BioNTech; research collaboration; RVU305; MTA-cooperative PRMT5 inhibitor; partnerships

About Ryvu Therapeutics
Ryvu Therapeutics is a clinical-stage drug discovery and development company focused on novel oncology therapies that address emerging targets in oncology. Internally discovered pipeline candidates at Ryvu use diverse therapeutic mechanisms driven by emerging knowledge of cancer biology, including small molecules and antibody-drug conjugates directed at kinases, synthetic lethality, and immuno-oncology targets. 
Ryvu’s most advanced program is romaciclib (RVU120, SEL120), a selective CDK8/CDK19 kinase inhibitor with the potential to treat hematological malignancies and solid tumors. Romaciclib is currently in Phase II development (i) in combination with venetoclax for the treatment of patients with r/r AML – the RIVER-81 study, (ii) as a monotherapy and in combination with ruxolitinib for the treatment of patients with myelofibrosis (MF) –the POTAMI-61 study. Dapolsertib (MEN1703, SEL24) is a dual PIM/FLT3 kinase inhibitor licensed to the Menarini Group that is currently being investigated in a Phase II study in diffuse large B-cell lymphoma (DLBCL) – the JASPIS-01 study. RVU305, a potentially best-in-class, brain-permeable PRMT5 inhibitor aiming to treat multiple solid tumors, has completed IND/CTA-enabling studies. Ryvu Therapeutics is also engaged in oncology collaborations with BioNTech and Exelixis. 
Ryvu was founded in 2007 and is headquartered in Kraków, Poland. It is listed on the Warsaw Stock Exchange and is a component of the sWIG80 index.

Contact

Ryvu Therapeutics
Anna Wilk
+48 532 698 425
anna.wilk@ryvu.com 

Source: Biotech Newswire

TOKYO, Japan, May 21, 2026 / Biotech Newswire / -- United Immunity, Co., Ltd., a biotechnology company developing its proprietary Myeloid Targeting Platform™ for therapeutic in vivo engineering of disease-associated macrophages and dendritic cells, today announced the acquisition of multiple assets from Carisma Therapeutics Inc..

United Immunity plans to leverage these innovative assets in combination with its PEG-free, pullulan-coated lipid nanoparticle (P-LNP) delivery system to generate in vivo CAR-M and other genetically engineered macrophages. This integrated approach aims to advance next-generation therapies for solid tumors and fibrosis diseases.

“Both assets has novel technologies to boost the efficacy of in vivo engineered anti-tumor or anti-fibrotic macrophages,” said Masato Kishida, Chief Executive Officer of United Immunity. “Our in vivo CAR-M therapy boosted by these proprietary assets represent a promising therapeutic approach for patients with solid tumors. In addition, United Immunity’s active targeting in vivo CAR-M strategy may provide an improved safety profile by reducing the risk of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), which are frequently associated with CAR-T cell therapies.”

 

Semantic keywords: Acquisition; macrophage assets; United Immunity; Carisma Therapeutics; Fibrosis; Neoplasms; Lipid Nanoparticles; cancer; liver fibrosis; Myeloid Targeting Platform; in vivo engineering; disease-associated macrophages; dendritic cells; pullulan-coated lipid nanoparticle; P-LNP delivery system; in vivo CAR-M; genetically engineered macrophages; solid tumors; fibrosis diseases; anti-tumor macrophages; anti-fibrotic macrophages; cytokine release syndrome; CRS; immune effector cell-associated neurotoxicity syndrome; ICANS; CAR-T cell therapies

About United Immunity, Co., Ltd. 
Unite the power of immunity and nanoparticle, change the future of patients
United Immunity is a clinical stage biotechnology company developing a novel Myeloid Targeting Platform™ designed to deliver therapeutic payloads directly to disease-associated macrophages and dendritic cells. Its platform combines pullulan nanoparticles (PNPs) and PEG-free, pullulan-coated lipid nanoparticles (P-LNPs) to enable targeted delivery of a wide range of therapeutics, including small molecules, nucleic acids, peptides, and proteins. This approach aims to address major unmet needs across cancer, fibrosis, infectious, autoimmune, and inflammatory diseases.
The company’s lead program, UI-102, leverages its PNP technology to deliver a TLR7/8 small-molecule agonist to tumor-associated macrophages, with the goal of converting immunologically “cold” tumors into “hot,” immune-sensitive ones. In parallel, United Immunity is advancing its P-LNP platform to enable the in vivo generation of CAR-macrophages, opening new therapeutic possibilities for cancer, fibrosis, and autoimmune diseases.

For more information, please visit www.unitedimmunity.co.jp/eng/
www.linkedin.com/company/unitedimmunitycoltd

Contact

United Immunity
Masato Kishida, CEO
info@unitedimmunity.co.jp

Source: Biotech Newswire

Ceremonial groundbreaking kicks off the first construction phase

• Pharmaceutical service provider advances its expansion with a 50,000 sqm facility
• Strategic investments in the EU and US strengthen Vetter’s global footprint 
• Groundbreaking ceremony featuring state and regional representatives, and project partners

SAARLOUIS and RAVENSBURG, Germany, May 13, 2026 / Biotech Newswire / -- Vetter, a leading Contract Development and Manufacturing Organization (CDMO), is celebrating the start of construction on its new site in Saarlouis with a traditional groundbreaking ceremony today. During the first construction phase, the company will build a state-of-the-art facility for the commercial production of injectable drugs on the 95-acre industrial site. In total, Vetter will invest almost half a billion euros in the site by the planned commissioning in 2031, underscoring its role as an industry leader for quality and technology. Once operational, the facility will create 400 to 500 new jobs, a figure that could rise to 1,500 by the final stage of the first construction phase. Ultimately, up to 2,000 jobs could be created.

In the first expansion phase, Vetter will build a 50,000 sqm manufacturing site, along with production-related facilities including laboratories and warehouses. Initially, three state-of-the-art cleanrooms are planned for the aseptic production of innovative and complex drug products: two for filling pre-sterilized syringes and one for filling vials.

Executives and officials at the groundbreaking ceremony

Senator h.c. Udo J. Vetter, Chairman of the Advisory Board and member of the owner family: "Today's groundbreaking ceremony marks a decisive step for the future of our family business. With our new location in Saarlouis, we are setting the course for further sustainable growth. We are creating the conditions to further expand our capacities in the field of complex drug product manufacturing. This underlines our continuous approach to offer our international customers the resources, quality and reliability they need and are accustomed to from us."

Anke Rehlinger, Minister President of Saarland: “Today is a good day for Saarland. With Vetter, a strong and innovative company, new opportunities are opening up for the region and its people. Here in Saarlouis, it is clear, that structural change succeeds when we actively shape it.”

Jürgen Barke, Minister of Economic Affairs, Innovation, Digitalization and Energy of Saarland: “An advanced technology investment of this kind positions us as one of the most sought-after locations for the pharmaceutical industry and demonstrates how transformation succeeds: with new, sustainable jobs and a strong, future-oriented industry. This will set off ripple effects unlike anything we have experienced before.”

Global investments in the future

For over 75 years, Vetter has stood for quality, innovation and responsibility, helping patients worldwide to achieve a better quality of life. In order to meet the growing demand and increasing market requirements, the CDMO is sustainably investing in its pharmaceutical production sites in Germany, Austria and the US. With its new location in Saarlouis, Vetter is significantly expanding its capacities once again.

© Vetter Pharma International GmbH: Representatives of the pharmaceutical service provider Vetter, the economic development agency gwSaar, and political representatives from the city, district, and state at the symbolic groundbreaking ceremony for the new production site in Saarlouis (from left to right): Nicholas H. Vetter, Thomas Schuck, Henryk Badack, Jürgen Barke, Gunther Strothe, Anke Rehlinger, Senator h.c. Udo J. Vetter, Marc Speicher, Patrik Lauer, Wolfgang Kerkhoff.

For high resolution please click the image.

Please find the Vetter press kit and more background information here. 

Semantic keywords: Manufacturing and Industrial Facilities; Vetter; global clinical manufacturing network; clinical manufacturing; Vetter; Contract Development and Manufacturing Organization; CDMO; production site; Saarlouis; commercial production; injectable drugs; manufacturing site; global footprint; cleanrooms; aseptic production; complex drug products; pre-sterilized syringes; drug product manufacturing; capacity expansion; sustainable growth

About Vetter
Vetter is a leading Contract Development and Manufacturing Organization (CDMO) with headquarters in Ravensburg, Germany, and production facilities in Germany, Austria, and the US. As a global player, the independent pharmaceutical service provider is also present in the Asia-Pacific markets of Japan, China, South Korea and Singapore with sales locations. Around the world renowned pharma and biotech companies benefit from decades of experience, high quality, modern technologies, reliability, and commitment of its 7,300 employees. In close collaboration with its customers, the Vetter team helps enable the supply to patients all over the world with medicines, many of which are vital. The CDMO provides support from drug product development through clinical and commercial filling to a wide range of assembly and packaging services for vials, syringes, and cartridges. With innovative approaches, Vetter develops prefilled drug-delivery systems together with its customers to continuously improve patient safety, comfort, and compliance. Vetter takes responsibility for sustainable practices and operates as a socially and ethically responsible corporate citizen. The CDMO is a member of the UN Global Compact and Science Based Target initiative (SBTi) and received platinum status in the renowned EcoVadis ranking. Multiple awards such as the CDMO Leadership Awards, Frost & Sullivan Customer Value Leadership Award and the recognition of Best Managed Company emphasize Vetter’s commitment to sustainable business. Founded in Ravensburg in 1950, the company remains family-owned to this day. For more information, visit www.vetter-pharma.com and follow Vetter on LinkedIn.

Contact

Vetter Pharma International GmbH
Markus Kirchner
Company Spokesperson / Media Relations
Eywiesenstraße 5
88212 Ravensburg
Germany
+49 (0)751-3700-3729
PRnews@vetter-pharma.com

Source: Biotech Newswire

Highlights:

• Bellberry Human Research Ethics Committee (HREC) approves Neurizon’s Phase 1 formulation study supporting development of the NUZ-001 oral liquid formulation and continued advancement of NUZ-001 for amyotrophic lateral sclerosis (ALS)
• Oral liquid formulation developed to improve treatment accessibility, administration flexibility and continuity of treatment as ALS progresses and swallowing difficulties increase
• Study designed to generate additional pharmacokinetic, safety and tolerability data to support clinical, regulatory and commercial development activities for NUZ-001
• Study forms part of Neurizon’s broader NUZ-001 development program, alongside ongoing participation in the Phase 2/3 HEALEY ALS Platform Trial in the United States

MELBOURNE, Australia, May 13, 2026 / Biotech Newswire / -- Neurizon® Therapeutics Limited (ASX: NUZ & NUZOA; OTCQB: NUZTF) (“Neurizon” or “the Company”), is pleased to announce that the Bellberry Human Research Ethics Committee (HREC) has approved the Company’s Phase 1 formulation study supporting development of the NUZ-001 oral liquid formulation, representing a further milestone in the development of NUZ-001 for ALS.

Neurizon previously announced development of an oral liquid formulation of NUZ-001 as part of its broader development strategy for ALS (refer to announcement 26 June 2025). ALS is associated with progressive impairment in speech and swallowing function (dysphagia), increasing the importance of flexible treatment administration options as the disease progresses.

The oral liquid formulation is intended to improve treatment accessibility, administration flexibility and continuity of treatment for people living with ALS, particularly as swallowing difficulties progress, supporting longer-term treatment administration across different stages of the disease.

The study will enrol 32 healthy volunteers in Australia in a randomised, four-arm Phase 1 clinical trial evaluating NUZ-001 oral liquid and tablet formulations under fed and fasted conditions. The study is designed to generate pharmacokinetic, safety and tolerability data, alongside exploratory biomarker and palatability data, to support formulation development and the broader clinical and regulatory development program for NUZ-001.

Interim Executive Chairman, Sergio Duchini commented: “Receipt of HREC approval marks another important milestone in the advancement of the NUZ-001 development program and reflects Neurizon’s commitment to developing patient-centred therapeutic solutions in ALS.”

“The oral liquid formulation was developed in direct response to the practical challenges faced by people living with ALS, particularly as swallowing difficulties become more prominent during disease progression. In addition to supporting flexibility and continuity of treatment, the formulation is intended to improve the overall practicality of administration for patients, caregivers and clinical teams.”

“The advancement of the oral liquid formulation reflects Neurizon’s broader strategy to support long-term treatment accessibility and expand the potential utility of NUZ-001 across different stages of disease progression.”

Study initiation is targeted for Q3 CY2026, subject to completion of remaining operational and site initiation activities, with study completion anticipated in Q4 CY2026.

This announcement has been authorized for release by the Board of Neurizon Therapeutics Limited.

Semantic keywords: Neurodegenerative Diseases; Amyotrophic Lateral Sclerosis; Motor Neuron Disease; Deglutition Disorders; Speech; Neurizon Therapeutics; NUZ-001; Phase 1 formulation study; oral liquid formulation; Bellberry Human Research Ethics Committee; HREC;  ALS; treatment accessibility; administration flexibility; swallowing difficulties; dysphagia; clinical development; regulatory development; commercial development; HEALEY ALS Platform Trial; Phase 2/3; formulation development; patient-centred therapeutic solutions

About Neurizon Therapeutics Limited
Neurizon Therapeutics Limited (ASX: NUZ) is a clinical-stage biotechnology company dedicated to advancing treatments for neurodegenerative diseases. Neurizon is developing its lead drug candidate, NUZ-001, for the treatment of ALS, which is the most common form of motor neurone disease.  Neurizon’s strategy is to accelerate access to effective ALS treatments for patients while exploring the potential of NUZ-001 for broader neurodegenerative applications. Through international collaborations and rigorous clinical programs, Neurizon is dedicated to creating new horizons for patients and families impacted by complex neural disorders.  NUZ-001 is an investigational product and is not approved for commercial use in any jurisdiction.

Neurizon Investor Hub
We encourage you to utilise our Investor Hub for any enquiries regarding this announcement or other aspects concerning Neurizon.
This platform offers an opportunity to submit questions, share comments, and view video summaries of key announcements.
To access Neurizon Investor Hub please scan the QR code or visit https://investorhub.neurizon.com

Neurizon® is a registered trademark of Neurizon Therapeutics Limited

This announcement has been authorized for release by the Board of Neurizon Therapeutics Limited.

Contacts

Neurizon Therapeutics
Lidija Damjanovic
Marketing & Corporate Affairs
lidija@neurizon.com 
+61 (0) 425 700 504

Australia Investor Relations
Henry Jordan
Six Degrees Investor Relations
henry.jordan@sdir.com.au 
+61 (0) 431 271 538 

Source: Biotech Newswire

• Romaciclib combined with venetoclax (VEN) shows encouraging anti-leukemic activity, including durable responses, in patients with relapsed/refractory acute myeloid leukemia after VEN-based therapy failure (RIVER-81). These results support further clinical development at the selected dose.
• Romaciclib demonstrates a favorable safety profile and clinical activity in myelofibrosis, both as monotherapy and in combination with ruxolitinib (POTAMI-61). The observed spleen volume reductions and hematologic tolerability support continued clinical evaluation.

KRAKOW, Poland, May 12, 2026 / Biotech Newswire / -- Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel therapies that address emerging targets in oncology, today announced it will present romaciclib (RVU120) data at the European Hematology Association Congress (EHA), June 11-14, 2026, in Stockholm, Sweden.

Hendrik Nogai, M.D., Chief Medical Officer of Ryvu Therapeutics said: "We are happy to share new data that confirm the strength and potential of romaciclib across hematologic malignancies. The results to be presented at EHA highlight consistent clinical activity and a manageable safety profile, both as a single agent and in rationale combinations, including in difficult-to-treat patient populations. These findings reinforce our confidence in romaciclib’s differentiated mechanism of action and support its continued clinical advancement."

 Details on the abstract presentations are as follows:

Title:  Updated findings from the phase 2 RIVER-81 study of romaciclib (RVU120) combined with venetoclax in acute myeloid leukemia after first-line venetoclax and hypomethylating agent failure
Session date and time: Friday, June 12 (6:45 - 7:45 PM CEST)

Venetoclax (VEN) combined with hypomethylating agents is the standard first-line treatment for patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy, yet ~70% experience relapsed/refractory disease with a median survival of under 3 months. Romaciclib, a first-in-class CDK8/CDK19 inhibitor, has shown single-agent activity in AML and preclinical synergy with VEN through enhanced apoptotic signaling and attenuation of resistance pathways.

In the ongoing Phase 2 RIVER-81 trial, romaciclib in combination with VEN demonstrates anti-leukemic activity in patients with poor prognostic AML, with the most consistent responses at romaciclib 150 mg QD + VEN 400 mg QD, including durable CR/CRi, reported with a cut-off date of 09 February 2026. Based on safety, PK, and preliminary efficacy findings, this regimen was selected as the recommended dose for expansion. Further evaluation is planned to better define durability of response and clinical benefit.

Title:  Romaciclib (RVU120), a selective CDK8/19 inhibitor, as monotherapy or in combination with ruxolitinib in patients with myelofibrosis: Updated results from the phase II POTAMI-61 study
Session date and time:  Saturday, June 13 (6:45 - 7:45 PM CEST)

Myelofibrosis (MF) is driven by dysregulated JAK/STAT signaling, and while JAK inhibition with ruxolitinib (RUX) improves splenomegaly and symptoms, cytopenias and suboptimal responses remain clinical challenges. Romaciclib (RVU120) is a first-in-class, oral CDK8/19 inhibitor that modulates STAT-dependent transcription and showed synergy with RUX in preclinical MF models.

In the ongoing Phase II POTAMI-61 study, romaciclib administered as monotherapy or combined with RUX demonstrates a manageable safety profile in patients with MF without significant treatment-related cytopenias. Prolonged exposure, spleen volume reductions, including in patients with high-molecular-risk mutations, and favorable hematologic tolerability support continued clinical development and further evaluation in expansion cohorts.

The abstracts are now available online and can be obtained from the conference site:  https://ehaweb.org/

The project entitled “The conduct of a phase II, multicentre, open-label clinical trial (RIVER-81) evaluating the safety and efficacy of RVU120 in combination with venetoclax in patients with relapsed/refractory acute myeloid leukemia who have failed prior therapy with venetoclax and a hypomethylating agent.”, carried out by Ryvu Therapeutics S.A., is co-financed by the Medical Research Agency from the state budget under the “Development of targeted or personalised medicine based on medicinal products involving nucleic acids and small-molecule compounds” call (ABM/2022/6).

Total Project value: PLN 132,471,740.70
Total funding: PLN 62,268,848.90

Semantic keywords: Congresses as Topic; Clinical Trials, Phase II as Topic; Clinical Trials, Phase I as Topic; venetoclax; ruxolitinib; Primary Myelofibrosis; Hematologic Neoplasms; Leukemia, Myeloid, Acute; Lymphoma; Poland; Ryvu Therapeutics; romaciclib; RVU120; European Hematology Association Congress; EHA; clinical data; hematologic malignancies; relapsed/refractory acute myeloid leukemia; acute myeloid leukemia; AML; venetoclax; VEN; VEN-based therapy; RIVER-81; anti-leukemic activity; myelofibrosis; ruxolitinib; RUX; POTAMI-61; CDK8/CDK19 inhibitor

About Ryvu Therapeutics
Ryvu Therapeutics is a clinical-stage drug discovery and development company focused on novel oncology therapies that address emerging targets in oncology. Internally discovered pipeline candidates at Ryvu use diverse therapeutic mechanisms driven by emerging knowledge of cancer biology, including small molecules and antibody-drug conjugates directed at kinases, synthetic lethality, and immuno-oncology targets. 
Ryvu’s most advanced program is romaciclib (RVU120, SEL120), a selective CDK8/CDK19 kinase inhibitor with the potential to treat hematological malignancies and solid tumors. Romaciclib is currently in Phase II development (i) in combination with venetoclax for the treatment of patients with r/r AML – the RIVER-81 study, (ii) as a monotherapy and in combination with ruxolitinib for the treatment of patients with myelofibrosis (MF) – the POTAMI-61 study, (iii) as a monotherapy for the treatment of patients with lower-risk myelodysplastic syndromes (LR-MDS) – the REMARK study. Dapolsertib (MEN1703, SEL24) is a dual PIM/FLT3 kinase inhibitor licensed to the Menarini Group that is currently being investigated in a Phase II study in diffuse large B-cell lymphoma (DLBCL) – the JASPIS-01 study. RVU305, a potentially best-in-class, brain-permeable, MTA-cooperative PRMT5 inhibitor aiming to treat multiple solid tumors, is currently in IND/CTA-enabling studies. Ryvu Therapeutics is also engaged in oncology collaborations with BioNTech and Exelixis.
Ryvu was founded in 2007 and is headquartered in Kraków, Poland. It is listed on the Warsaw Stock Exchange and is a component of the sWIG80 index.

Contact

Ryvu Therapeutics
Anna Wilk
+48 532 698 425
anna.wilk@ryvu.com

Source: Biotech Newswire

VIENNA, Austria and BUDAPEST, Hungary, May 12, 2026 / Biotech Newswire / -- AOP Health, a global pharmaceutical enterprise group focused on integrated therapies for rare diseases and critical care, today announced a strategic partnership with VRG Therapeutics, an AI-driven developer for miniproteins based in Budapest. The collaboration aims to advance a novel Kv1.3 potassium channel inhibitor, a key regulator of immune cell activity, for use in inflammation and immunology (I&I) indications.

The collaboration centers on a next-generation miniprotein candidate discovered using VRG Therapeutics’ AI-MPRO platform, an AI-based system to design and test new miniproteins.  This platform combines artificial intelligence-driven protein design with experimental validation to generate highly selective, target-specific miniproteins.
The partners will focus on generating additional preclinical proof-of-concept data in selected indications to support progression towards first-in-human studies.

Targeting Kv1.3 with a Novel Therapeutic Approach
Kv1.3 is a clinically validated target that plays a critical role in the regulation of pathogenic immune cells in chronic inflammatory and autoimmune disorders. Selective inhibition of Kv1.3 holds significant promise in modulating immune responses that drive disease while reducing side effects. The partners leverage the exceptional selectivity and precision of the miniprotein modality to overcome the limitations of previous advancements in the Kv1.3 field.

Combining Platform Innovation with Clinical Development Expertise
The partnership brings together VRG Therapeutics’ AI-driven miniprotein discovery capabilities with AOP Health’s 30 years of experience in developing therapies for indications in areas of high unmet medical need.
For AOP Health, the agreement marks an expansion of its external R&D activities into a complex and scientifically active area of immunology. For VRG Therapeutics, the collaboration represents an important validation of its AI-MPRO platform and supports the advancement of its technology toward clinical application.  

“VRG’s AI-MPRO platform has demonstrated the ability to generate highly selective candidates against complex biological targets,” said Martin Steinhart, CEO of AOP Health Group. “We see strong potential in targeting Kv1.3 for autoimmune and inflammatory diseases and look forward to advancing this program toward clinical evaluation.”

“With its established development capabilities and strong track record in addressing unmet medical needs, AOP Health is an ideal partner for this program.” commented Zalán Péterfi, Chairman of the Board and Managing Director at VRG Therapeutics. “Advancing together the Kv1.3 program, we intend to expand our partnership to fully exploit the platform potential.”

Sematic Keywords: International Cooperation; Rare Diseases; Autoimmune Diseases; Potassium Channels; Immunity; Inflammation; Artificial Intelligence; AOP Health; VRG Therapeutics; co-development partnership; immunology program; AI-driven ; developer of miniproteins; Kv1.3 ; potassium channel inhibitor; immune cell activity; inflammatory diseases; inflammation and immunology; I&I indications; AI-MPRO platform; artificial intelligence-driven protein design; target-specific miniproteins; preclinical proof-of-concept; pathogenic immune cells; chronic inflammatory disorders; autoimmune disorders; selective inhibition; immune responses

About AOP Health
AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company’s actions. (aop-health.com)

About VRG
VRG Therapeutics is an innovative biotechnology company headquartered in Budapest, focused on the discovery and development of next-generation miniprotein therapeutics targeting challenging biological mechanisms. The company leverages its proprietary AI-MPRO platform, which integrates computational protein design, high-throughput screening, and machine learning–driven analytics to generate highly selective and potent drug candidates. With a strong scientific foundation and an experienced team, VRG Therapeutics is advancing a growing pipeline of therapeutic programs with the potential to deliver differentiated best-in-class and first-in-class treatments.

Contact

AOP Health
Mag. Nina Roth
Nina.Roth@aop-health.com
+43-676-3131509
https://www.aop-health.com

Source: Biotech Newswire