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AskBio Completes Enrollment in Phase 1 Gene Therapy Trial for Rare Neurological Disease MSA-P

Rare Disease
  • AskBio has completed enrollment of 11 participants in its Phase 1 REGENERATE MSA-101 trial testing AB-1005 gene therapy for multiple system atrophy-parkinsonian type (MSA-P), a rare neurodegenerative disease affecting approximately 400,000 people worldwide.
  • The randomized, double-blind, placebo-controlled trial will evaluate the safety of AB-1005, which delivers the neuroprotective factor GDNF directly to the brain's putamen region using an AAV2 viral vector.
  • This milestone follows AskBio's recent FDA Regenerative Medicine Advanced Therapy designation for AB-1005 in Parkinson's disease, based on favorable 36-month Phase 1b safety data with no product-related serious adverse events.
  • Initial results from the MSA-P trial will be available after all participants complete one year of clinical assessments, with the potential to address a significant unmet medical need as no disease-modifying treatments currently exist for MSA.

GDNF Gene Therapy for Parkinson's Disease

NCT04167540Active, Not RecruitingPhase 1
Brain Neurotherapy Bio, Inc.
Posted 4/1/2020

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)

NCT06285643RecruitingPhase 2
AskBio Inc
Posted 6/11/2024

GDNF Gene Therapy for Multiple System Atrophy

NCT04680065Active, Not RecruitingPhase 1
Brain Neurotherapy Bio, Inc.
Posted 10/3/2023

Brepocitinib Achieves First-Ever Positive Phase 3 Results in Dermatomyositis, Setting New Treatment Standard

Rare DiseaseTargeted Therapy
  • Roivant and Priovant's brepocitinib demonstrated statistically significant improvement over placebo in the Phase 3 VALOR study, achieving a mean Total Improvement Score of 46.5 versus 31.2 for placebo (p=0.0006) at 52 weeks.
  • The dual TYK2/JAK1 inhibitor showed rapid onset of action with separation from placebo as early as week 4, while enabling 62% of patients to reduce steroid use to ≤2.5 mg/day compared to 34% on placebo.
  • This represents the first positive 52-week placebo-controlled trial and first successful registrational study for a targeted therapy in dermatomyositis, addressing a critical unmet need in this debilitating autoimmune disease.
  • An NDA filing is planned for the first half of 2026, positioning brepocitinib to potentially become the first approved targeted therapy for dermatomyositis patients.

FDA Expands Vyjuvek Label for Home Use and All Ages, Boosting Krystal Biotech's Market Potential

Drug ApprovalRare Disease
  • The FDA approved an updated label for Vyjuvek allowing home administration by families and patients, expanding beyond clinic-only application for dystrophic epidermolysis bullosa treatment.
  • Vyjuvek can now be used in patients from birth, significantly broadening the addressable patient population for Krystal Biotech's gene therapy.
  • The stock surged 14% over the past week and nearly 19% in three months following the regulatory approval, with analysts viewing this as a meaningful turning point for commercial expansion.

FDA Set to Review Five Key Pediatric and Adult Therapies in Q4 2025

Drug ApprovalRare Disease
  • The FDA is scheduled to make approval decisions on five significant therapies in Q4 2025, including treatments for rare pediatric conditions and infectious diseases.
  • Navepegritide represents a potential breakthrough for achondroplasia treatment, designed as a once-weekly prodrug that provides continuous CNP exposure to inhibit the overactive FGFR3 pathway.
  • Two novel oral antibiotics, gepotidacin and zoliflodacin, are under review for gonorrhea treatment in patients 12 years and older, addressing the growing need for new therapeutic options.
  • Roflumilast cream 0.05% could expand atopic dermatitis treatment options for young children aged 2-5 years, based on phase 3 data from 652 pediatric patients.

Trial of PDE4 Inhibition With Roflumilast for the Management of Atopic Dermatitis (Integument-PED)

NCT04845620CompletedPhase 3
Arcutis Biotherapeutics, Inc.
Posted 4/7/2021

INmune Bio Achieves Commercial Manufacturing Milestone for RDEB Cell Therapy CORDStrom™

Rare Disease
  • INmune Bio successfully completed its first commercial pilot-scale manufacturing run of CORDStrom™, an umbilical cord-derived mesenchymal stromal cell therapy for treating Recessive Dystrophic Epidermolysis Bullosa (RDEB).
  • The manufacturing milestone at the Cell and Gene Therapy Catapult facility in the UK keeps the company on track for regulatory submissions, with plans to file a Marketing Authorization Application in the UK during the first half of 2026.
  • CORDStrom™ demonstrated promising results in a blinded-randomized Phase 2 Mission EB trial, showing improvements in itch, pain, wound scores, and quality of life for RDEB patients.
  • The successful manufacturing run confirms the scalability and consistency of CORDStrom™ production using a proprietary GMP-compliant process for this off-the-shelf allogeneic therapy.

Argo Biopharma Advances siRNA Therapy BW-40202 to Phase II Trial for Paroxysmal Nocturnal Hemoglobinuria

Rare Disease
  • Argo Biopharma received NMPA approval for a Phase II clinical trial of BW-40202, an siRNA therapy targeting complement factor B for treating paroxysmal nocturnal hemoglobinuria.
  • The investigational therapy utilizes RNA interference to suppress complement factor B expression in the liver, demonstrating significant and durable suppression of serum CFB protein in preclinical studies.
  • BW-40202 addresses a critical unmet need in PNH treatment, where thrombosis remains the most life-threatening complication responsible for 40-67% of PNH-related deaths.
  • The Phase II study is expected to commence in January 2026, while the drug is also being evaluated in a Phase I study for IgA Nephropathy.

Ocugen Partners with Kwangdong for OCU400 Gene Therapy in Korea with $7.5M Upfront Deal

Rare Disease
  • Ocugen has executed a licensing agreement with Kwangdong Pharmaceutical for exclusive Korean rights to OCU400, a novel modifier gene therapy for retinitis pigmentosa.
  • The deal includes upfront fees and development milestones totaling up to $7.5 million, with potential sales milestones of $180 million or more over the first 10 years of commercialization.
  • OCU400 is currently in Phase 3 clinical development with a target U.S. BLA filing in 2026, representing a potential one-time therapy for life to treat retinitis pigmentosa.
  • The Korean market represents an estimated 7,000 individuals with retinitis pigmentosa, approximately 7% of the U.S. market size.

SOM Biotech Secures European Orphan Drug Designation for Huntington's Disease Therapy SOM3355

Rare Disease
  • SOM Biotech received positive opinion from the European Medicines Agency's Committee for Orphan Medical Products for SOM3355, a novel therapy for Huntington's disease that already holds orphan drug designation in the US.
  • SOM3355 features a unique multimodal mechanism combining mild beta-blocking activity with VMAT1 and VMAT2 inhibition, showing encouraging results in proof-of-concept and phase 2b studies.
  • The therapy aims to address the complex symptomatology of Huntington's disease with a streamlined approach, potentially reducing polypharmacy and improving patient adherence compared to current symptom-specific treatments.
  • The company plans to advance SOM3355 through phase 3 clinical studies globally as part of its final development stages.

Monopar's ALXN1840 Shows Sustained Neurological Benefits in Long-Term Wilson Disease Study

Rare Disease
  • Monopar Therapeutics presented new long-term data on ALXN1840 (tiomolybdate choline) at the 150th American Neurological Association Annual Meeting, demonstrating sustained neurological improvement in Wilson disease patients over 6 years.
  • The pooled analysis from three clinical trials (n=255) showed statistically significant neurologic improvement on the Unified Wilson Disease Rating Scale, with patients crossing over from standard of care showing additional neurological benefits.
  • ALXN1840 demonstrated a favorable safety profile with less than 1% of patients experiencing drug-related neurological serious adverse events across more than 645 patient-years of treatment.
  • The findings complement recent hepatic efficacy data presented at EASL, underscoring ALXN1840's potential for treating both neurological and hepatic manifestations of Wilson disease.

Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

NCT04573309CompletedPhase 2
Alexion Pharmaceuticals, Inc.
Posted 9/7/2020

Real-World LOTUS Study Shows Sustained Efficacy of DAYBUE in Rett Syndrome Patients Over 12 Months

Real World EvidenceRare Disease
  • Acadia Pharmaceuticals published interim results from the LOTUS study showing 71-90% of Rett syndrome patients experienced behavioral improvements with DAYBUE treatment over 12 months.
  • The real-world observational study of 227 patients aged 1-60 years demonstrated improvements in nonverbal communication, alertness, and social interaction consistent with previous clinical trials.
  • Gastrointestinal side effects remained manageable with diarrhea incidence varying from 23-50% in early weeks to 26-38% in later months, with most cases contained in diapers.
  • Quality of life scores improved with a median change of 4.6 points on the QI-Disability questionnaire among patients completing 12-month assessments.

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

NCT04181723CompletedPhase 3
ACADIA Pharmaceuticals Inc.
Posted 11/6/2019

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