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CorMedix Completes Phase III Enrollment for REZZAYO Antifungal Prophylaxis in Bone Marrow Transplant Patients

Orphan Drug
  • CorMedix announced completion of enrollment in the global Phase III ReSPECT trial evaluating REZZAYO (rezafungin) for preventing fungal infections in adult patients undergoing allogeneic blood and marrow transplantation.
  • The study compares once-weekly rezafungin dosing versus standard antimicrobial regimens, with the primary endpoint of fungal-free survival at day 90 and topline results expected in Q2 2026.
  • REZZAYO represents a significant market opportunity with an estimated 130,000 patients constituting the addressable market for antifungal prophylaxis in the U.S. and a total addressable market exceeding $2 billion.
  • The drug currently has orphan drug exclusivity through 2035 and patent coverage through 2038, positioning CorMedix for potential FDA approval in this high-risk patient population with critical unmet medical needs.

MediWound's NexoBrid Receives Marketing Authorization in Australia, Expanding Global Burn Treatment Access

Drug ApprovalOrphan Drug
  • Australia's Therapeutic Goods Administration has granted marketing authorization for NexoBrid, an innovative enzymatic therapy for burn treatment in both adult and pediatric patients.
  • The approval brings NexoBrid's global authorization to 45 countries worldwide, reinforcing its recognition as a new standard of care in burn management.
  • Commercial launch is expected in the fourth quarter of 2025 through exclusive partner Balance Medical, supported by MediWound's manufacturing expansion completion by year-end 2025.
  • The milestone opens opportunities for broader Asia-Pacific region expansion, where growing demand exists for advanced wound and burn treatments.

Harmony Biosciences' ZYN002 Phase 3 Trial Fails to Meet Primary Endpoint in Fragile X Syndrome

Rare DiseaseOrphan Drug
  • Harmony Biosciences announced that its Phase 3 RECONNECT study of ZYN002 in Fragile X syndrome failed to meet the primary endpoint of improvement in social avoidance due to higher than expected placebo response rates.
  • The randomized, double-blind, placebo-controlled trial enrolled 215 patients aged 3 to under 30 years with Fragile X syndrome, testing the transdermal cannabidiol gel over an 18-week treatment period.
  • Despite the setback, Harmony Biosciences remains committed to advancing its late-stage pipeline, including upcoming Phase 3 trials for pitolisant HD in narcolepsy and idiopathic hypersomnia planned for Q4 2025.
  • The failure represents a significant blow to addressing the unmet medical need in Fragile X syndrome, a rare genetic disorder affecting up to 80,000 patients in the US with no FDA-approved treatments currently available.

A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

NCT05163808Active, Not RecruitingPhase 2
Tetra Discovery Partners
Posted 3/29/2022

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

NCT05358886CompletedPhase 3
Tetra Discovery Partners
Posted 11/1/2022

Study of SPG601 in Adult Men With Fragile X Syndrome

NCT06413537CompletedPhase 2
Spinogenix
Posted 7/5/2024

Aro Biotherapeutics Completes Enrollment in Phase 1b Trial of Novel siRNA Therapy for Late-Onset Pompe Disease

Rare DiseaseOrphan DrugTargeted Therapy
  • Aro Biotherapeutics has completed enrollment in its Phase 1b clinical trial of ABX1100, a first-in-class targeted siRNA therapy for late-onset Pompe disease patients currently receiving enzyme replacement therapy.
  • ABX1100 represents a novel therapeutic approach using Centyrin-siRNA conjugate technology to inhibit glycogen synthase 1 (GYS1), targeting the root cause of glycogen accumulation in muscle tissue.
  • The trial evaluates safety, tolerability, and preliminary therapeutic activity over 20 weeks, with results expected to inform later-phase clinical studies for this orphan drug-designated treatment.
  • Current enzyme replacement therapy requires intravenous infusions lasting up to 6 hours multiple times monthly, highlighting significant unmet medical need in this rare neuromuscular disorder.

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

NCT06109948RecruitingEarly Phase 1
Aro Biotherapeutics
Posted 10/19/2023

ResVita Bio Receives FDA Clearance to Advance Novel Bacterial Therapy for Rare Skin Disease

Orphan DrugRare Disease
  • ResVita Bio completed a successful Pre-IND meeting with the FDA for RVB-003, a first-in-class therapy for Netherton Syndrome, a life-threatening genetic skin disorder with no approved treatments.
  • The company's innovative approach uses genetically engineered bacteria to continuously produce therapeutic proteins directly on the skin surface, overcoming limitations of conventional protein drugs.
  • FDA provided positive feedback on the development program, clearing the path for IND submission in the first half of 2026 and clinical efficacy readout by early 2027.

Acadia Pharmaceuticals' Phase 3 Trial of Intranasal Carbetocin for Prader-Willi Syndrome Fails to Meet Primary Endpoint

Rare DiseaseOrphan Drug
  • Acadia Pharmaceuticals announced that its Phase 3 COMPASS PWS trial of intranasal carbetocin (ACP-101) failed to demonstrate statistically significant improvement over placebo for hyperphagia in Prader-Willi syndrome patients.
  • The 12-week randomized, placebo-controlled trial enrolled 175 children and adults aged 5-30 years with PWS, testing carbetocin 3.2 mg three times daily against placebo.
  • Despite the setback, Acadia maintains its growth outlook with two approved products projected to generate over $1 billion in net sales in 2025 and a robust pipeline including eight disclosed programs.
  • The company will discontinue further investigation of intranasal carbetocin but plans to share study data with the PWS community for future learning.

GemVax Seeks FDA Breakthrough Therapy Designation for GV1001 in Progressive Supranuclear Palsy

Orphan Drug
  • GemVax & KAEL has applied for FDA Breakthrough Therapy designation for GV1001, a potential treatment for progressive supranuclear palsy (PSP), which could significantly accelerate drug development timelines.
  • The company demonstrated GV1001's potential in a Phase 2a clinical trial, showing tolerability and a trend toward slowing disease progression with statistically significant effects compared to external controls.
  • PSP represents the most severe neurodegenerative disorder among Parkinsonian syndromes with no currently approved treatments, creating an urgent unmet medical need for patients.
  • GV1001 already holds FDA Fast Track and Orphan Drug designations, as well as EMA Orphan Drug status, positioning it for expedited regulatory pathways.

Ipsen's Bylvay Receives Japanese Approval for Rare Liver Disease PFIC, Offering First Non-Surgical Treatment Option

Drug ApprovalRare DiseaseOrphan Drug
  • Japan's Ministry of Health, Labour and Welfare has approved Bylvay (odevixibat) as the first once-daily ileal bile acid transport inhibitor for treating pruritus in progressive familial intrahepatic cholestasis patients.
  • The approval addresses a critical unmet need for approximately 100 children and infants in Japan suffering from this rare, life-threatening genetic liver disorder.
  • Clinical data from the global PEDFIC Phase III trial demonstrated that 55% of patients achieved pruritus reduction with odevixibat compared to 30% on placebo.
  • The treatment offers a non-surgical alternative for managing debilitating symptoms including severe itching, sleep disruption, and impaired cognitive development in PFIC patients.

CorestemChemon's Neuronata-R Shows Significant Benefits in ALS Slow Progressors in Phase 3 Trial

Orphan DrugPrecision Medicine
  • CorestemChemon's Phase 3 ALSummit trial of Neuronata-R demonstrated statistically significant improvements in ALS patients classified as slow progressors, with meaningful gains in functional rating scales and respiratory capacity.
  • Biomarker analysis revealed sustained reductions in neurofilament light chain and MCP-1, indicating neuroprotective effects that support a precision-medicine approach for ALS treatment.
  • The company plans to pursue FDA accelerated approval pathway discussions in Q4 2025, with a Biologics License Application submission targeted for 2026 based on the subgroup efficacy data.

Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis

NCT01363401CompletedPhase 1
Corestemchemon, Inc.
Posted 2/1/2011

Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients with ALS

NCT04745299Active, Not RecruitingPhase 3
Corestemchemon, Inc.
Posted 3/23/2021

ARTHEx Biotech Secures $87M Series B to Advance Novel RNA Therapy for Myotonic Dystrophy Type 1

Orphan DrugRare Disease
  • ARTHEx Biotech successfully closed an upsized Series B financing round totaling $87 million, led by new investor Bpifrance with participation from all existing shareholders.
  • The funding will advance ATX-01, a unique anti-miR oligonucleotide targeting microRNA23b currently in Phase I/IIa trials for Myotonic Dystrophy Type 1.
  • ATX-01 demonstrates a dual mechanism of action that increases MBNL protein production and reduces toxic DMPK mRNA, showing promising results in animal models.
  • The proceeds will support preparation for a registrational study and expand the company's pipeline of targeted RNA medicines for muscular, CNS, and cardiac conditions.

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