ABT-301 is an oral HDAC1/2/3 inhibitor shown in preclinical models to convert “cold tumors” into “hot,” enhancing immune response by increasing CD8+ T cell activity, antigen presentation, and suppressing M-MDSCs. In a prior Phase I study with 23 patients, the candidate demonstrated a clean safety profile—lacking common toxicities like neutropenia or cardiotoxicity seen with other HDAC inhibitors.
According to GlobalData, the target patient population exceeds 370,000 across key markets annually, with an estimated commercial potential of $9 billion. Anbogen plans to leverage this milestone to attract global licensing opportunities and has initiated a Series B funding round to support continued development and commercialization.
Dyne Therapeutics’ DYNE-251 Earns FDA Breakthrough Therapy Tag for Duchenne Muscular Dystrophy
Dyne Therapeutics, Inc., announced that the FDA has granted Breakthrough Therapy Designation to DYNE-251 for Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. The designation is based on promising data from the ongoing DELIVER trial, highlighting the therapy’s potential to improve functional outcomes in a population with limited options.
“This Breakthrough Therapy Designation is a testament to DYNE-251’s potential as a next-generation treatment designed to bring meaningful improvement to individuals with DMD,” said Doug Kerr, M.D., Ph.D., Chief Medical Officer at Dyne. “With sustained functional benefits seen through 18 months and near full-length dystrophin expression, we’re optimistic about its long-term impact.”
With this designation, DYNE-251 now qualifies for enhanced regulatory support, including rolling submission and potential Priority Review of its future Biologics License Application (BLA). Dyne has completed enrollment in the DELIVER trial’s Registrational Expansion Cohort (n=32), with data expected in late 2025 and a potential BLA submission in early 2026.
Notably, DYNE-251 has also received Fast Track, Orphan Drug, and Rare Pediatric Disease designations in the U.S., along with Orphan Drug status from the EMA. Dyne continues to pursue global regulatory pathways for the therapy while advancing its parallel program, DYNE-101, which also holds Breakthrough Therapy Designation for myotonic dystrophy type 1.
FDA Grants Priority Review to BMS’ BREYANZI for Relapsed/Refractory Marginal Zone Lymphoma
Bristol Myers Squibb announced that the FDA accepted the supplemental Biologics License Application (sBLA) for BREYANZI (lisocabtagene maraleucel; liso-cel) to treat adults with relapsed or refractory marginal zone lymphoma (MZL) after at least two prior lines of systemic therapy. The application has been granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) date set for December 5, 2025.
“This FDA acceptance brings us one step closer to potentially standardizing CAR T cell therapy as a treatment option for MZL,” said Rosanna Ricafort, Vice President and Senior Global Program Lead, Hematology and Cell Therapy, Bristol Myers Squibb. “Many MZL patients experience multiple relapses, and BREYANZI offers hope for lasting responses in this difficult-to-treat population.”
The submission is supported by data from the MZL cohort of the TRANSCEND FL study, a Phase II, open-label, multicenter, single-arm trial. Results, presented at the 2025 International Conference on Malignant Lymphoma (ICML), showed that BREYANZI achieved durable response rates with a consistent and manageable safety profile in patients with heavily pretreated MZL.
If approved, BREYANZI would become the first and only CAR T cell therapy indicated for MZL, addressing a critical unmet need. The FDA recently removed the Risk Evaluation and Mitigation Strategy (REMS) requirement and approved streamlined patient monitoring protocols for BREYANZI, improving accessibility while maintaining safety standards.
Clarametyx Biosciences announced that the FDA has granted both Fast Track and Qualified Infectious Disease Product (QIDP) designations for its lead candidate, CMTX-101, for the treatment of chronic bacterial pulmonary infections in people with cystic fibrosis (CF). The designations aim to expedite the development and review of therapies for serious infections and provide additional regulatory benefits, including five years of extended market exclusivity.
“The receipt of both Fast Track and QIDP designations underscores CMTX-101’s potential as an innovative therapy for chronic respiratory conditions,” said David V. Richards, CEO of Clarametyx. “With expanded support from our investors, we’re well positioned to advance both our therapeutic and vaccine pipelines, including reaching full enrollment in our lead trial by year-end.”
The ongoing phase Ib/IIa trial of CMTX-101 in CF patients is evaluating safety, pharmacokinetics, immunogenicity, and reductions in Pseudomonas aeruginosa burden. A positive interim analysis announced in June 2025 met the criteria to progress, triggering expansion of the study’s next phase and trial site network.
To support its development programs, Clarametyx also announced the closing of an upsized second tranche of Series A funding from existing investors. The funding will accelerate clinical progress of CMTX-101 and further the company’s vaccine program, which is targeting an IND submission by 2027.
Anixa Biosciences Begins FDA-Approved IND Transfer for Phase II Breast Cancer Vaccine Trial
Anixa Biosciences, in collaboration with the Cleveland Clinic, initiated the transfer of the Investigational New Drug (IND) application for its breast cancer vaccine. With the Phase I trial now completed and enrollment closed, Anixa is assuming full sponsorship to advance the program into a Phase II clinical study. The IND, originally held by Cleveland Clinic, is being transitioned to Anixa to support the next phase of clinical development.
“We are pleased with the progress and preliminary findings from our Phase I clinical trial, which show that the vaccine is well tolerated, with more than 70% of patients tested to date exhibiting protocol-defined immune responses,” said Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences. “The IND transfer represents a major step in advancing to a Phase II trial under our sponsorship.”
To support the IND transfer and regulatory planning, Anixa has engaged Advyzom, a regulatory consulting firm with expertise in FDA interactions, to serve as its U.S. regulatory agent. This strategic collaboration ensures streamlined coordination as Anixa prepares to initiate the next trial phase.
The breast cancer vaccine, developed with the Cleveland Clinic, targets α-lactalbumin—an antigen typically expressed in lactating breast tissue but also present in many breast cancers. By generating an immune response against this protein, the vaccine holds promise for both therapeutic and preventive applications in patients with α-lactalbumin–expressing tumors.
