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Clinical Trial News

FDA Grants Orphan Drug Designation to COAGADEX for Rare Bleeding Disorder Associated with AL Amyloidosis

Rare Disease
  • The FDA has granted Orphan Drug Designation to Kedrion Biopharma's COAGADEX for treating acquired Factor X deficiency, a rare blood disorder affecting fewer than one in one million people globally.
  • COAGADEX, already approved in 38 countries for hereditary Factor X deficiency, will be evaluated in a new clinical trial for patients with acquired deficiency caused by AL amyloidosis.
  • The condition occurs when amyloid fibrils bind to and reduce circulating Factor X levels, significantly increasing bleeding risk in affected patients.
  • Patient recruitment is underway for the trial, which will focus on moderate and severe forms of acquired Factor X deficiency with coagulant activity levels below 50% of normal.

BioNexus Gene Lab and Fidelion Diagnostics Form Strategic Alliance to Commercialize Revolutionary Liquid Biopsy Technology in Southeast Asia

  • BioNexus Gene Lab Corp. and Fidelion Diagnostics announced a strategic cross-equity partnership to bring VitaGuard™ minimal-residual-disease platform to Southeast Asia's 680 million population.
  • The VitaGuard™ technology dramatically reduces liquid biopsy costs from over $3,000 to under $300 while achieving 0.02% variant-allele-frequency sensitivity with tumor-naïve detection capabilities.
  • The alliance targets Southeast Asia's projected 2.4 million annual cancer cases by 2030, creating a multi-billion-dollar market opportunity for affordable precision oncology.
  • BGLC will secure exclusive commercial rights across ASEAN markets and lead regulatory submissions starting in Singapore and Malaysia.

NICE Recommends Vanzacaftor-Tezacaftor-Deutivacaftor for Cystic Fibrosis Treatment in England

Drug Approval
  • NICE has approved vanzacaftor-tezacaftor-deutivacaftor (Alyftrek) for NHS funding in England to treat cystic fibrosis in patients aged 6 years and older with at least one F508del mutation.
  • Clinical trial evidence demonstrates the triple combination therapy is as effective as the current standard treatment ivacaftor-tezacaftor-elexacaftor in improving lung function, growth, weight gain, and reducing lung infections.
  • The new therapy must be funded within 30 days of final guidance publication, with cost comparisons suggesting similar expenses to existing treatments.
  • NICE determined there is sufficient evidence showing the treatment provides benefits and value for money, allowing routine use across the NHS for this patient population.

AscellaHealth and Abeona Therapeutics Launch Comprehensive Support Program for ZEVASKYN Gene Therapy

Rare DiseaseDrug Approval
  • AscellaHealth has partnered with Abeona Therapeutics to successfully commercialize ZEVASKYN, the first FDA-approved autologous cell sheet-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB).
  • The collaboration resulted in AbeonaAssist, a customized patient support program designed to address the unique clinical, operational, and reimbursement challenges of this groundbreaking cell-based gene therapy.
  • Early results demonstrate enhanced patient compliance, retention, and satisfaction rates, highlighting the effectiveness of tailored support programs for complex gene therapies.
  • ZEVASKYN incorporates the functional COL7A1 gene into patients' own skin cells to produce type VII collagen, demonstrating clinically meaningful wound healing and pain reduction with a single surgical application.

LTZ Therapeutics Partners with Eli Lilly to Advance First-in-Class Myeloid Engager Platform

Oncology
  • LTZ Therapeutics announced a strategic research collaboration with Eli Lilly to develop novel myeloid engager therapeutics against selected targets for diseases with high unmet need.
  • The agreement includes a double-digit million USD upfront payment and equity investment for LTZ, plus eligibility for preclinical, clinical, regulatory and commercial milestone payments.
  • The collaboration combines LTZ's first-in-class Myeloid Engager Platform with Lilly's expertise in research, development and commercialization to accelerate therapeutic development.
  • LTZ's platform targets myeloid biology to improve clinical outcomes in patients with cancer and autoimmune diseases through novel immunotherapy approaches.

Therini Bio Initiates Phase 1b Trial of Novel Anti-Fibrin Antibody THN391 for Alzheimer's Disease

Monoclonal Antibody
  • Therini Bio has dosed the first patient in a Phase 1b trial of THN391, a first-in-class monoclonal antibody targeting fibrin-mediated neuroinflammation in Alzheimer's disease.
  • The randomized, placebo-controlled trial will evaluate safety and pharmacokinetics in 65-85 year old patients with early Alzheimer's disease and vascular risk factors.
  • THN391 demonstrated a 40-day half-life supporting monthly dosing and showed no serious adverse events in Phase 1a healthy volunteer studies.
  • Initial data from the multi-dose ascending trial is expected in mid-2026, with assessments including cognitive measures and neuroimaging biomarkers.

Formation Bio Licenses First-in-Class Anti-CD226 Monoclonal Antibody for Autoimmune Diseases

Monoclonal Antibody
  • Formation Bio has licensed worldwide rights to IMIDomics' first-in-class anti-CD226 monoclonal antibody, which has received IND clearance and will initially target ulcerative colitis.
  • The antibody targets CD226 (DNAM-1), a key immune checkpoint that regulates immune cells, offering a novel mechanistic approach for treating autoimmune conditions characterized by high treatment failure rates.
  • The licensing agreement includes upfront payments, development milestones, royalties, and equity in Riverview Bio, a new Formation Bio subsidiary created to develop this therapy.
  • The program is supported by IMIDomics' 20-year longitudinal dataset from Spain's Immune Mediated Inflammatory Diseases Consortium, providing robust mechanistic validation for clinical advancement.

Bavarian Nordic Agrees to $3 Billion Take-Private Deal with Nordic Capital and Permira

  • Vaccine maker Bavarian Nordic has agreed in principle to a roughly $3 billion take-private offer from private equity firms Nordic Capital and Permira, with completion expected in the fourth quarter.
  • The deal offers 233 Danish kroner per share, representing a 21% premium to the company's closing price before takeover talks were confirmed.
  • Bavarian Nordic has developed vaccines for mpox, smallpox, and chikungunya virus, with its Vimkunya vaccine recently launched in the U.S. and approved by the CDC.
  • The company's revenue is growing due to rising demand for traveler vaccines and continued government contract business, including recent U.S. government orders for smallpox vaccine supply.

Maryland Drug Affordability Board Flags Two Diabetes Medications as Cost Challenges

  • Maryland's Prescription Drug Affordability Board unanimously determined that Jardiance and Farxiga pose affordability challenges to the state's healthcare system.
  • The decision marks the first completion of PDAB's cost review process and opens the door for potential upper payment limits to cap state spending on these medications.
  • The board cited rising wholesale acquisition costs above inflation rates and high out-of-pocket expenses as key indicators of affordability challenges.
  • Four additional drugs including Ozempic, Dupixent, Skyrizi, and Trulicity remain under review in the board's cost analysis pipeline.

Basilea Launches Phase 3 Trial for Novel Antifungal Fosmanogepix in Invasive Mold Infections

Orphan Drug
  • Basilea Pharmaceutica has initiated FORWARD-IM, a phase 3 registrational study evaluating fosmanogepix for treating invasive mold infections in adults, with completion expected in Q1 2028.
  • Fosmanogepix is a first-in-class broad-spectrum antifungal with novel mechanism of action, showing activity against multidrug-resistant molds including Aspergillus, Fusarium, and Mucorales fungi.
  • The study will enroll approximately 220 patients across two cohorts, with the first cohort randomizing 160 patients 2:1 to fosmanogepix versus standard-of-care therapy.
  • This marks the second phase 3 study for fosmanogepix, following the FAST-IC trial initiated in September 2024 for candidemia and invasive candidiasis treatment.

A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Participants With Candidemia and/or Invasive Candidiasis.

NCT05421858RecruitingPhase 3
Basilea Pharmaceutica
Posted 12/11/2024

A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Patients With Invasive Mold Infections.

NCT06925321RecruitingPhase 3
Basilea Pharmaceutica
Posted 8/1/2025

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