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FDA Grants Fast Track Designation to Sumitomo's Nuvisertib for Myelofibrosis Treatment

Orphan DrugRare Disease
  • The FDA has granted Fast Track Designation to nuvisertib (TP-3654), Sumitomo Pharma America's investigational PIM1 kinase inhibitor, for treating intermediate or high-risk myelofibrosis patients.
  • Updated Phase 1/2 data presented at EHA 2025 showed nuvisertib achieved 22.2% spleen volume reduction and 44.4% symptom score improvement in evaluable patients with relapsed/refractory myelofibrosis.
  • The oral selective inhibitor demonstrated significant cytokine modulation with p<0.001 correlation between anti-inflammatory effects and clinical responses, supporting its potential as monotherapy or combination treatment.
  • Myelofibrosis affects 1 in 500,000 people worldwide and represents a serious unmet medical need with limited treatment options for patients facing poor prognosis.

A Study of Oral Nuvisertib (TP-3654) in Patients With Myelofibrosis

NCT04176198RecruitingPhase 1
Sumitomo Pharma America, Inc.
Posted 12/16/2019

Mezzion Secures $20 Million to Advance Phase 3 Trial of Udenafil for Rare Congenital Heart Disease

Rare Disease
  • Mezzion Pharma secured approximately $20 million in strategic funding led by Midas-Meritz New Technology Finance Association to support the ongoing global Phase 3 FUEL-2 trial of udenafil for Fontan circulation patients.
  • The investment represents strong institutional confidence in Mezzion's late-stage pipeline, with lead investor Midas PE citing positive FUEL-1 results and expressing belief in udenafil's potential for successful Phase 3 completion and regulatory approval.
  • The financing follows growing clinical momentum, including the introduction of dedicated ICD-10 codes for Fontan-associated conditions, marking progress in addressing this long-overlooked rare congenital heart disease population.
  • Mezzion reports accelerating enrollment and growing engagement across clinical sites for the FUEL-2 trial, with plans to continue seeking additional investments to support global development efforts.

uniQure Appoints Kylie O'Keefe as Chief Customer and Strategy Officer to Lead AMT-130 Commercialization for Huntington's Disease

Rare Disease
  • uniQure has appointed Kylie O'Keefe as Chief Customer and Strategy Officer to lead the global commercialization strategy for AMT-130, the company's investigational gene therapy for Huntington's disease.
  • O'Keefe brings extensive rare disease and gene therapy experience from her role as Chief Commercial Officer at PTC Therapeutics, where she led commercial launches across more than 50 countries.
  • The appointment positions uniQure for a potential U.S. commercial launch of AMT-130 in 2026, with the therapy having potential to become the first disease-modifying treatment for Huntington's disease.
  • AMT-130 is currently in Phase I/II clinical trials and has a clear pathway toward accelerated approval in the United States.

Cellectar's Iopofosine I 131 Doubles Survival in Pediatric Brain Cancer Trial

Rare DiseaseOrphan Drug
  • Cellectar Biosciences reported that iopofosine I 131 achieved 5.4 months average progression-free survival in pediatric high-grade glioma patients, doubling the reported median of 2.25 months.
  • Patients receiving higher doses (minimum 55 mCi) showed 8.6 months overall survival, with three patients receiving additional cycles achieving 11.5 months average overall survival.
  • The radioligand therapy demonstrated a favorable safety profile with no cardiovascular, renal, or liver toxicities, and all hematologic adverse events were manageable and reversible.
  • The CLOVER-2 Phase 1 trial enrolled 14 pediatric patients with relapsed/refractory high-grade gliomas, including diffuse midline gliomas and diffuse intrinsic pontine gliomas.

Novel Targeted Radiotherapy in Pediatric Patients With Inoperable Relapsed or Refractory HGG

NCT05610891Active, Not RecruitingPhase 1
Cellectar Biosciences, Inc.
Posted 10/1/2023

Dose Escalation Study of CLR 131 in Children, Adolescents, and Young Adults With Relapsed or Refractory Malignant Tumors Including But Not Limited to Neuroblastoma, Rhabdomyosarcoma, Ewings Sarcoma, and Osteosarcoma

NCT03478462Active, Not RecruitingPhase 1
Cellectar Biosciences, Inc.
Posted 4/30/2019

Study to Compare Tivozanib in Combination With Nivolumab to Tivozanib Monotherapy in Subjects With Renal Cell Carcinoma

NCT04987203Active, Not RecruitingPhase 3
AVEO Pharmaceuticals, Inc.
Posted 9/9/2021

Cogent Biosciences Secures $400M Debt Financing to Advance Bezuclastinib Through Pivotal Trials

Precision MedicineRare Disease
  • Cogent Biosciences secured a $400 million non-dilutive debt financing facility with SLR Capital Partners, with an initial $50 million already drawn to support the company's growth strategy.
  • The company is on track to report results from three pivotal bezuclastinib trials in 2025, starting with SUMMIT results in July for NonAdvSM patients.
  • Additional funding tranches are tied to clinical milestones, with $100 million available upon successful SUMMIT and PEAK trial readouts and commercial success.
  • The financing enhances Cogent's financial flexibility as it prepares for the expected bezuclastinib launch in 2026 for genetically defined diseases.

SpliceBio Raises $135 Million Series B to Advance Gene Therapy for Stargardt Disease Using Novel Protein Splicing Platform

Rare Disease
  • SpliceBio secured $135 million in Series B funding co-led by EQT Life Sciences and Sanofi Ventures to advance its lead gene therapy candidate SB-007 for Stargardt disease.
  • The company's innovative Protein Splicing platform overcomes the challenge of delivering large genes by splitting them into smaller pieces that reassemble into full-length proteins.
  • SB-007 targets Stargardt disease, the most common inherited macular degeneration affecting 1 in 8,000-10,000 people with no approved treatments currently available.
  • The technology has potential to address over 1,200 known mutations causing Stargardt disease and could unlock treatments for other genetic disorders involving large genes.

SIFI Merges with Faes Farma to Expand Global Access to Akantior, First Approved Therapy for Rare Eye Infection

Orphan DrugRare DiseaseDrug Approval
  • SIFI, a leading international ophthalmic company, has agreed to merge with European pharmaceutical company Faes Farma in a transaction that positions ophthalmology as approximately 20% of the combined entity's pro-forma revenue.
  • The merger will accelerate development of Akantior (polihexanide 0.08%), the first and only approved therapy worldwide for Acanthamoeba Keratitis, particularly targeting expansion into the US market where it has orphan drug designation.
  • The combined entity will leverage commercial synergies across complementary markets in Europe and Latin America, while also advancing polihexanide development for additional orphan indications including Fungal Keratitis.
  • The transaction values SIFI at a double-digit multiple of 2024 EBITDA plus regulatory and commercial milestone-based earn-outs, with closing expected in Q3 2025 subject to regulatory approvals.

Nuevocor Receives FDA IND Clearance for Gene Therapy Targeting Rare Genetic Heart Disease

Rare Disease
  • Nuevocor announced FDA clearance of its Investigational New Drug application for NVC-001, an AAV-based gene therapy designed to treat LMNA-related dilated cardiomyopathy.
  • The therapy targets approximately 100,000 patients in the U.S. and Europe with this aggressive form of genetic heart disease that rapidly progresses to end-stage heart failure.
  • NVC-001 demonstrated significant benefits in survival and cardiac function in preclinical studies by addressing the mechanobiological root cause of disease.
  • The company plans to initiate a first-in-human Phase 1/2 clinical trial in early 2026 to evaluate safety, tolerability, and preliminary efficacy.

Gyre Therapeutics Initiates Phase 1 Trial of F230 for Pulmonary Arterial Hypertension in China

Rare Disease
  • Gyre Therapeutics has successfully dosed the first volunteer in a Phase 1 clinical trial evaluating F230, a novel endothelin A receptor antagonist, for treating pulmonary arterial hypertension in China.
  • F230 is a fully synthetic small molecule designed to selectively block the ETA receptor, targeting pulmonary vascular remodeling and pressure reduction in PAH patients.
  • This milestone marks Gyre's expansion into the PAH field, leveraging China's growing PAH market valued at $370 million in 2023 and projected to reach $480 million by 2031.
  • The Phase 1 trial will evaluate safety, tolerability, and pharmacokinetics in healthy volunteers as part of Gyre's fibrosis-first therapeutic strategy.

Vanda Pharmaceuticals Initiates First-in-Human Trial of Personalized ASO Therapy VCA-894A for Ultra-Rare CMT2S

Rare Disease
  • Vanda Pharmaceuticals has dosed the first patient in a first-in-human clinical trial evaluating VCA-894A, an antisense oligonucleotide therapy designed for a specific patient with Charcot-Marie-Tooth disease Type 2S.
  • VCA-894A targets a patient-specific IGHMBP2 gene variant and has demonstrated ability to restore gene expression levels in neuromuscular junction systems derived from the patient's own cells.
  • CMT2S is an extremely rare inherited neuromuscular disorder with an estimated prevalence of less than 1 in 1,000,000 worldwide, causing progressive muscle weakness and motor function loss.
  • This personalized medicine approach represents a potential breakthrough for addressing rare genetic disorders using nucleic acid-based platforms to restore function where crucial gene expression has been disrupted.

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