Aminex Therapeutics Secures FDA Orphan Drug Designation for AMXT 1501 + DFMO in Neuroblastoma
Aminex Therapeutics, Inc., a clinical-stage biotechnology company developing novel cancer therapies, announced that the FDA has granted Orphan Drug Designation (ODD) to AMXT 1501 in combination with difluoromethylornithine (DFMO) for the treatment of neuroblastoma. The designation highlights the therapy’s potential in addressing one of the most challenging pediatric cancers.
“Receiving Orphan Drug Designation for AMXT 1501 in combination with DFMO represents an important milestone in our mission to develop innovative therapies for children with life-threatening cancers,” said Mark Burns, PhD, Chief Scientific Officer and President of Aminex Therapeutics. “We are committed to working closely with regulators, investigators, and patient advocacy groups to accelerate clinical development for neuroblastoma and other tumor types.”
The FDA’s ODD program supports the development of treatments for rare diseases, offering incentives such as tax credits for clinical testing, user fee waivers, and seven years of market exclusivity upon approval. Neuroblastoma, which accounts for up to 15% of pediatric cancer deaths in the U.S., remains an area of urgent unmet medical need.
According to Dr. Giselle Sholler, Chief of Pediatric Hematology and Oncology at Penn State Health Children’s Hospital and Chair of the Beat Childhood Cancer Research Consortium, “We believe this combination has the potential to build upon the FDA approval of DFMO to further improve outcomes for children with neuroblastoma and other rare childhood cancers.” The Beat Childhood Cancer Research Consortium is initiating a phase I/II trial (NCT06465199) to evaluate AMXT 1501 + DFMO in pediatric patients, while Aminex also plans further studies in metastatic melanoma and breast cancer.
Eccogene Gets FDA Nod to Begin Phase IIa MOSAIC Trial of ECC4703 + ECC0509 in MASH
Eccogene, a clinical-stage biopharmaceutical company focused on oral small molecule therapies for cardiometabolic and inflammatory diseases, announced that the FDA has cleared its Investigational New Drug (IND) application to initiate MOSAIC, a phase IIa clinical trial evaluating ECC4703, ECC0509, and their combination in adults with presumed metabolic dysfunction-associated steatohepatitis (MASH).
“We are pleased to receive FDA clearance to initiate our phase IIa MOSAIC trial with ECC4703 and ECC0509 for MASH, which affects millions of patients worldwide,” said Jingye Zhou, Chief Executive Officer of Eccogene. “ECC4703, a liver-targeting THR-β full agonist, is designed to deliver improved efficacy and tolerability, while ECC0509, a novel SSAO inhibitor, addresses the inflammatory and fibrotic pathways associated with MASH.”
The MOSAIC study is a U.S.-based, multicenter, randomized, double-blind, placebo-controlled trial that will assess the efficacy and safety of ECC4703, ECC0509, and their combination over a 12-week period. The trial aims to enroll approximately 160 participants, with the primary endpoint focused on changes in liver fat content.
According to Eccogene, combining ECC4703’s metabolic benefits with ECC0509’s anti-inflammatory and anti-fibrotic properties could provide a more comprehensive approach to tackling MASH, a condition with significant unmet medical need.
Orca Bio’s Orca-T Earns FDA Priority Review for Hematologic Malignancies
Orca Bio, a late-stage biotechnology company focused on precision cell therapies, announced that the FDA has accepted its Biologics License Application (BLA) for Orca-T, its lead allogeneic T-cell immunotherapy, for the treatment of hematological malignancies, including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndromes (MDS). The FDA granted Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026.
“A stem cell transplant has been the only potentially curative option for many patients with AML, ALL, or MDS, but treatment-related toxicities often limit recovery,” said Nate Fernhoff, PhD, Co-Founder and CEO of Orca Bio. “Acceptance of the Orca-T BLA marks a pivotal moment in our ability to deliver a first-in-class therapy designed to improve survival while minimizing complications like graft-versus-host disease.”
The BLA submission is supported by positive results from the pivotal phase III Precision-T trial (NCT04013685), a multicenter, randomized, open-label study comparing Orca-T to conventional allogeneic hematopoietic stem cell transplant (alloHSCT). The study met its primary endpoint, demonstrating a statistically significant improvement in survival free of moderate-to-severe chronic graft-versus-host disease with Orca-T.
Orca Bio emphasized that, if approved, Orca-T would be the first allogeneic T-cell immunotherapy available for AML, ALL, and MDS, offering patients a potentially transformative treatment option. “We look forward to collaborating with the FDA during the review process and advancing Orca-T to patients in need,” Fernhoff added.
Alto Neuroscience Gains FDA Fast Track for ALTO-101 in Cognitive Impairment Linked to Schizophrenia
Alto Neuroscience, Inc., a clinical-stage biopharmaceutical company developing precision medicines for neuropsychiatric disorders, announced that the FDA has granted Fast Track designation to ALTO-101 for the treatment of cognitive impairment associated with schizophrenia (CIAS). CIAS is a core feature of schizophrenia with no currently approved therapies, significantly impacting daily functioning and quality of life for millions of patients.
“Receiving Fast Track designation from the FDA underscores the critical need for new, effective treatments for patients suffering from cognitive impairment associated with schizophrenia,” said Amit Etkin, MD, PhD, Founder and CEO of Alto Neuroscience. “This designation is a significant milestone for the ALTO-101 program and validates its potential as a novel treatment approach.”
ALTO-101 is a small molecule phosphodiesterase-4 (PDE4) inhibitor that enhances cyclic adenosine monophosphate (cAMP) signaling in the brain, supporting neuronal function and synaptic plasticity. Phase I studies in healthy volunteers demonstrated pro-cognitive effects, with improvements observed in both EEG measures and cognitive performance.
Enrollment is ongoing in a phase II proof-of-concept study evaluating ALTO-101 in patients with CIAS. According to Alto, Fast Track designation could accelerate development and review, potentially enabling more frequent FDA interactions, priority review, and eligibility for accelerated approval, addressing a significant unmet need in schizophrenia care.
Taysha Gene Therapies Wins FDA Breakthrough Therapy Status for TSHA-102 in Rett Syndrome
Taysha Gene Therapies, a clinical-stage biotechnology company developing AAV-based gene therapies for severe monogenic CNS diseases, announced that the FDA has granted Breakthrough Therapy designation to TSHA-102 for the treatment of Rett syndrome. The designation follows positive clinical evidence from Part A of the REVEAL phase I/II trials, where all 12 patients demonstrated a response in gaining or regaining developmental milestones.
“Breakthrough Therapy designation highlights the FDA’s recognition of both the significant unmet medical need across Rett syndrome and the potential of TSHA-102 to redefine the treatment paradigm,” said Rumana Haque-Ahmed, Chief Regulatory Officer of Taysha. “The clinical data from Part A supports TSHA-102’s ability to improve function and enable milestone achievement across core areas of the disease, which may significantly impact patients and caregivers.”
Taysha also finalized alignment with the FDA on the REVEAL pivotal trial protocol and statistical analysis plan (SAP), resolving remaining clinical and statistical queries. Key design elements remain unchanged, including a six-month interim analysis that could expedite a Biologics License Application (BLA) submission based on the unprecedented response rates observed in Part A.
“We are pleased to have finalized FDA alignment on our pivotal trial protocol and SAP, including the six-month interim analysis that may accelerate our BLA submission,” said Sukumar Nagendran, MD, President and Head of R&D. “The regulatory progress reinforces our BLA readiness as we remain on track to initiate patient enrollment in Q4 2025 for the REVEAL pivotal trial.”
The REVEAL pivotal trial is a single-arm, open-label study evaluating a single intrathecal high-dose administration of TSHA-102 in 15 female patients aged 6–22 years. The primary endpoint assesses the percentage of patients achieving one or more defined developmental milestones across communication, fine motor, and gross motor domains, based on rigorous standardized assessments and video-evidence review.
