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Cellarity Initiates Phase 1 Trial of CLY-124, First-in-Class Oral Therapy for Sickle Cell Disease

AIRare Disease
  • Cellarity has dosed the first patient in a Phase 1 clinical trial of CLY-124, a novel oral medicine that increases fetal hemoglobin through a globin-switching mechanism to treat sickle cell disease.
  • The drug was discovered using AI-powered transcriptomic analysis and demonstrated the ability to increase fetal hemoglobin above 20% in preclinical studies without cytotoxicity.
  • CLY-124 represents a potential breakthrough for sickle cell disease treatment, offering a once-daily oral alternative to current therapies that carry dose-limiting toxicity.
  • The Phase 1 global trial will evaluate safety, tolerability, and pharmacokinetics in healthy volunteers before advancing to sickle cell disease patients.

GenSight Biologics Successfully Transfers LUMEVOQ Manufacturing to Catalent for LHON Gene Therapy

Rare Disease
  • GenSight Biologics has successfully transferred the upstream manufacturing process for LUMEVOQ, its gene therapy for Leber Hereditary Optic Neuropathy (LHON), to manufacturing partner Catalent Inc.
  • The partnership is expected to improve yield and upgrade analytical methods ahead of clinical use and regulatory submissions for the rare mitochondrial disease treatment.
  • Catalent manufactured a drug product batch released as safe for human use in November 2024, which will supply France's named patient early access program and dose-ranging study.
  • The collaboration positions GenSight for its planned global Phase III RECOVER trial beginning in 2026 and upcoming regulatory submissions to FDA and EMA.

FDA Approves Enhanced Labeling for Endari Based on Post-Marketing Pharmacokinetic Data

Drug ApprovalRare Disease
  • Emmaus Life Sciences received FDA approval for enhanced labeling of Endari (L-glutamine oral powder) based on additional post-marketing pharmacokinetic study data.
  • The updated label provides more comprehensive prescribing information, including confirmation of body weight-based dosing, no unwanted accumulation with twice-daily dosing, and flexible administration with or without food.
  • Endari remains approved to reduce acute complications of sickle cell disease in adult and pediatric patients five years and older, addressing a condition affecting approximately 100,000 people in the United States.
  • The label enhancement represents a significant improvement in clinical guidance for healthcare providers treating sickle cell disease patients with this prescription-grade L-glutamine therapy.

Phase 3 Trial Demonstrates Selumetinib Efficacy in Adults with Neurofibromatosis Type 1

Rare Disease
  • The KOMET phase 3 trial published in The Lancet shows selumetinib significantly reduces tumor size in one in five adults with neurofibromatosis type 1 plexiform neurofibromas.
  • This represents the first effective pharmacological treatment for adult NF1 patients, who previously relied solely on complex surgical interventions with limited success.
  • The randomized, double-blind study included 145 adult participants worldwide and demonstrated rapid pain reduction alongside decreased need for analgesics.
  • The innovative trial design allowed placebo participants to access selumetinib under specific conditions, enabling comprehensive efficacy assessment across different treatment timelines.

Ovid Therapeutics Sells Ganaxolone Royalty Rights to Immedica Pharma for $7 Million

Rare Disease
  • Ovid Therapeutics has entered into a definitive agreement with Immedica Pharma AB to sell its future royalty rights for ganaxolone sales outside China for $7 million in cash.
  • The transaction provides non-dilutive funding to support Ovid's ongoing operations, while Immedica strengthens its focus on ganaxolone by acquiring additional intellectual property rights.
  • Ganaxolone is an approved medicine in the EU, Great Britain, US, and China for treating epileptic seizures associated with CDKL5 deficiency disorder in patients aged 2-17 years.
  • Immedica will also assume financial responsibility for all costs related to the licensed intellectual property portfolio and plans to expand the license to include additional indications.

Former Pfizer CSO Mikael Dolsten Joins Two Biotech Boards, Advancing Gene Editing and Mast Cell Therapeutics

Gene EditingRare Disease
  • Dr. Mikael Dolsten, former Pfizer Chief Scientific Officer, has joined the boards of Arbor Biotechnologies and MC Sciences as both companies advance novel therapeutic platforms.
  • Dolsten's 16-year tenure at Pfizer included advancing over 150 drug candidates into clinical studies and leading regulatory approval of 36 medicines and vaccines.
  • Arbor Biotechnologies is progressing its lead gene editing program ABO-101 for primary hyperoxaluria type 1 into clinical trials.
  • MC Sciences is developing first-in-class mast cell-targeting therapeutics for diseases including chronic urticaria, systemic mastocytosis, and asthma.

Minovia Therapeutics Announces $180M SPAC Merger to Advance First-in-Class Mitochondrial Therapy Platform

Rare Disease
  • Minovia Therapeutics and Launch One Acquisition Corp. announced a definitive business combination agreement valued at $180 million pre-money, creating a publicly traded mitochondrial therapy company targeting the $1+ trillion longevity market.
  • The company's lead product MNV-201 has received FDA Fast Track and Rare Pediatric Disease designations for Pearson Syndrome, with clinical data from 23 patients showing significant outcomes including restored mobility and kidney function.
  • Minovia's Mitochondrial Augmentation Technology (MAT) platform addresses diseases caused by mitochondrial dysfunction, with plans to launch longevity-focused offerings through global clinic partnerships beginning in 2026.
  • The transaction is expected to close in Q4 2025, providing capital to advance clinical milestones and bring U.S.-based GMP manufacturing online by end of 2025.

Biogen Advances Salanersen to Phase 3 Studies for Spinal Muscular Atrophy After Promising Results

Rare Disease
  • Biogen's salanersen (BIIB115/ION306) demonstrated substantial slowing of neurodegeneration and clinically meaningful motor function improvements in children with spinal muscular atrophy previously treated with gene therapy.
  • The investigational antisense oligonucleotide achieved a 70% reduction in neurofilament levels at six months, with half of participants gaining new motor milestones like sitting independently or walking.
  • Based on encouraging Phase 1 data, Biogen is engaging with regulators to advance salanersen directly to registrational Phase 3 studies, potentially offering once-yearly dosing compared to current treatments.
  • The drug represents a next-generation approach to SMA treatment, leveraging the same mechanism as Spinraza but designed for greater potency and less frequent administration.

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

NCT04089566CompletedPhase 3
Biogen
Posted 3/26/2020

Galderma Launches Phase II Trials of Nemolizumab for Systemic Sclerosis and Chronic Pruritus of Unknown Origin

Monoclonal AntibodyRare Disease
  • Galderma has initiated two phase II clinical trials investigating nemolizumab, an IL-31 receptor alpha-targeting monoclonal antibody, for treating systemic sclerosis and chronic pruritus of unknown origin.
  • The systemic sclerosis trial will be a multicenter, randomized, double-blind, placebo-controlled study beginning enrollment in H2 2025 with completion anticipated in 2028.
  • The chronic pruritus of unknown origin trial will explore nemolizumab's efficacy in treating persistent itch lasting more than six weeks without identified cause, with enrollment starting in H2 2025 in the U.S.
  • Both conditions represent significant unmet medical needs, with systemic sclerosis being a potentially fatal autoimmune disease and chronic pruritus of unknown origin having no currently approved treatments.

NHS Delivers First Brain-Infused Gene Therapy for Rare AADC Deficiency, Showing Promising Results in Three-Year-Old Patient

Rare Disease
  • Three-year-old Gunreet Kaur received Upstaza (eladocagene exuparvovec), the first NHS England commissioned gene therapy infused directly into the brain, at Great Ormond Street Hospital in February 2024.
  • The treatment targets aromatic l-amino acid decarboxylase (AADC) deficiency, a rare and often fatal genetic condition that prevents dopamine production and causes severe developmental delays.
  • Following gene therapy, Gunreet demonstrated significant improvements including better head control, coordination, reduced crying, and new motor skills like rolling over.
  • The therapy uses a virus to deliver a working AADC gene directly to brain nerve cells with millimetre precision using robotic surgery assistance.

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