PepGen Reports Promising Splicing Correction Results in DM1 Clinical Trial
• Initial results from FREEDOM-DM1 trial demonstrate significant splicing correction of 29.1% at 10 mg/kg dose in patients with Myotonic Dystrophy Type 1, showing potential therapeutic efficacy.
• PepGen's EDO platform technology successfully delivers PGN-EDODM1 to the cellular nucleus, addressing the fundamental mechanism of DM1 disease progression.
• Company advances clinical program with ongoing 15 mg/kg cohort in FREEDOM-DM1 trial and fully enrolled 10 mg/kg cohort in CONNECT1-EDO51 study, with results expected in late 2025.
Bayer's Elinzanetant Shows Promise in Reducing Hot Flashes for Breast Cancer Patients
• Elinzanetant met primary endpoints in the Phase III OASIS 4 trial, significantly reducing the frequency of moderate to severe vasomotor symptoms (VMS) in women undergoing endocrine therapy for breast cancer.
• The study also achieved secondary endpoints, demonstrating a reduction in the severity of VMS, improvements in sleep disturbances, and enhanced menopause-related quality of life compared to placebo.
• Elinzanetant is a dual neurokinin-1 and 3 receptor antagonist, representing a potential non-hormonal treatment option for VMS in breast cancer patients and women at high risk.
• Bayer is advancing regulatory submissions for elinzanetant in the US, EU, and other global markets, with potential launch expected later this year, pending regulatory approvals.
FDA Roundup: Approvals, Breakthrough Designations, and Clinical Holds in Neurology and Rare Diseases
• The FDA granted breakthrough therapy designation to STK-001 for Dravet syndrome, highlighting its potential to improve upon current treatments by restoring NaV1.1 protein levels.
• Tolebrutinib received breakthrough therapy designation for non-relapsing secondary progressive multiple sclerosis based on phase 3 trial results showing delayed disability progression.
• The FDA placed a clinical hold on PepGen’s PGN-EDO51 phase 2 study for Duchenne muscular dystrophy, pending further clarification from the agency.
FDA Halts PepGen's DMD Trial, Sanofi's MS Drug Tolebrutinib Gains Breakthrough Status, and Sevasemten Shows Promise in Becker Muscular Dystrophy
• The FDA has placed a clinical hold on PepGen's Phase 2 CONNECT2-EDO51 trial for PGN-EDO51 in Duchenne muscular dystrophy (DMD) due to regulatory questions.
• Sanofi's tolebrutinib, a BTK inhibitor, received breakthrough therapy designation from the FDA for non-relapsing secondary progressive multiple sclerosis (nrSPMS).
• Edgewise Therapeutics' sevasemten met its primary endpoint in a Phase 2 trial for Becker muscular dystrophy, showing a significant decrease in creatine kinase.
FDA Approves Multiple Breakthrough Therapies: Crenessity for CAH, Unloxcyt for Skin Cancer
• The FDA has approved Neurocrine's Crenessity as an adjunct therapy for classic congenital adrenal hyperplasia, offering patients a way to reduce steroid treatment burden.
• Checkpoint Therapeutics received FDA approval for Unloxcyt to treat advanced cutaneous squamous cell carcinoma, targeting a $1 billion market opportunity.
• Galderma's Nemluvio gains FDA approval for moderate-to-severe atopic dermatitis in patients 12 and older, with projected peak annual sales exceeding $2 billion.
Gene Therapy Advances: From FDA Clearances to Clinical Trial Progress in Early 2025
• The FDA cleared United Therapeutics' UKidney for US trials, a gene-edited porcine kidney product intended for end-stage renal disease via xenotransplant.
• uniQure received the green light to proceed with the second dose cohort in their Phase 1/2 trial of AMT-162 for SOD1-ALS gene therapy.
• Sarepta Therapeutics' Elevidys showed sustained benefit in ambulatory patients with Duchenne muscular dystrophy in Phase 3 trial results.
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