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Vertex Secures Italian Reimbursement for CRISPR Gene Therapy Casgevy, Expanding European Access for Blood Disorders

Rare Disease
  • Vertex Pharmaceuticals announced a reimbursement agreement with Italy's Medicines Agency (AIFA) for Casgevy, the first CRISPR/Cas9 gene-edited therapy approved for transfusion-dependent beta thalassemia and sickle cell disease in Europe.
  • Italy represents the largest TDT population in Europe with approximately 5,000 patients aged 12 and older, joining seven other countries that have secured reimbursement agreements for the transformative one-time therapy.
  • Clinical data demonstrates that over 95% of SCD patients treated with Casgevy achieve freedom from vaso-occlusive crises for at least 12 months, while nearly all TDT patients become transfusion-independent.
  • The therapy's commercial potential is significant, with analysts projecting Casgevy revenue could reach $54-81.4 million in 2025 as Vertex expands to 75 treatment centers globally.

Biotech Stocks Surge After-Hours as Investors Anticipate Key Clinical Trial Results

Rare Disease
  • MBX Biosciences shares jumped 33% after-hours ahead of Phase 2 topline results for Canvuparatide, a once-weekly therapy for chronic hypoparathyroidism, scheduled for release on Monday.
  • Structure Therapeutics gained 11.37% in extended trading as attention builds around its obesity drug aleniglipron, with Phase 2b trial results expected by end of 2025.
  • Multiple biotech companies including Cartesian Therapeutics, Rapport Therapeutics, and Rezolute saw significant after-hours gains driven by upcoming clinical milestones and positive trial developments.
  • The rally reflects strong investor appetite for clinical-stage biotechnology companies with near-term data catalysts and promising pipeline assets.

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.

Rgenta Therapeutics Receives FDA Orphan Drug Designation for RGT-61159 in Adenoid Cystic Carcinoma Treatment

OncologyRare Disease
  • The FDA has granted Orphan Drug Designation to RGT-61159, an oral small molecule inhibitor targeting the MYB oncogene through RNA splicing modulation for adenoid cystic carcinoma treatment.
  • RGT-61159 is currently being evaluated in a Phase 1a/b clinical trial for patients with advanced relapsed or refractory adenoid cystic carcinoma or colorectal cancer.
  • The designation provides significant regulatory benefits including tax credits, fee waivers, and seven years of market exclusivity if approved.
  • Adenoid cystic carcinoma affects approximately 11,000 people in the US and has limited treatment options, with MYB overexpression found in over 90% of cases.

Study of Safety and Efficacy of RGT-61159 in Adults with Relapsed/Refractory Adenoid Cystic Carcinoma (ACC) or Colorectal Carcinoma (CRC)

NCT06462183RecruitingPhase 1
Rgenta Therapeutics Inc
Posted 8/19/2024

Regeneron's Garetosmab Achieves 90% Reduction in Bone Formation in Rare Disease Trial

Monoclonal AntibodyRare DiseaseOrphan Drug
  • Regeneron's Phase 3 OPTIMA trial of garetosmab met its primary endpoint, demonstrating a 90% or greater reduction in new heterotopic ossification lesions in adults with fibrodysplasia ossificans progressiva at 56 weeks.
  • The monoclonal antibody targeting activin A achieved over 99% reduction in total volume of new bone lesions, with the Independent Data Monitoring Committee recommending placebo patients transition to active treatment.
  • The company plans to submit garetosmab for U.S. regulatory approval by the end of 2025, positioning it as a potential first-in-class therapy for this ultra-rare genetic disorder affecting fewer than 1 in 2 million individuals.

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 Multiple System Atrophy

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

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

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.

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