Sarepta Therapeutics

2024 saw highs and lows in neuroscience drug development, including FDA approval of BMS's Cobenfy for schizophrenia and Eli Lilly's donanemab for Alzheimer's, alongside failures like Sage Therapeutics' dalzanemdor in Parkinson's, Alzheimer's, and Huntington's. Despite setbacks, progress continues in treating intractable neurological diseases.

Sarepta Therapeutics completed enrolment and dosing in the global Phase III EMERGENE trial of its gene therapy SRP-9003 for limb-girdle muscular dystrophy Type 2E/R4. The trial's primary endpoint is beta-sarcoglycan protein expression, with data expected in H1 2025. The therapy uses AAVrh74 vector to deliver a full-length beta-sarcoglycan transgene to skeletal, diaphragm, and cardiac muscle.

2024 saw over 25 pediatric FDA approvals, including givinostat for Duchenne muscular dystrophy, FluMist for influenza, clonidine hydrochloride for ADHD, dupilumab for eosinophilic esophagitis, epinephrine nasal spray for anaphylaxis, and roflumilast cream for atopic dermatitis.

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The FDA issues guidance on accelerated approvals, emphasizing sponsors must have confirmatory trials underway at approval. Labels will detail drug usefulness limitations. Accelerated approval should not be considered if adequate trials are impossible. Sponsors must conduct confirmatory trials diligently, with FDA review of protocols. FDA may withdraw approval for non-compliance, safety concerns, or misleading promotions. Examples include Pfizer's Oxbryta withdrawal and Sarepta's Elevidys market retention despite trial failure.

H.C. Wainwright raises Arrowhead Pharma's price target to $80, maintaining a Buy rating, driven by a $500M upfront and $325M equity investment from Sarepta Therapeutics. Arrowhead's pipeline includes plozasiran for familial chylomicronemia syndrome and new obesity programs, ARO-INHBE and ARO-ALK7. Despite a net loss of $599.5M in 2024, the company holds $681M in cash and investments, positioning it for future growth.

Sarepta Therapeutics, known for its focus on Duchenne muscular dystrophy, made a $500 million upfront deal with Arrowhead Pharmaceuticals, investing $325 million and up to $10 billion in milestones. The deal aims to diversify Sarepta's pipeline with RNA drugs and expand its portfolio into new disease areas. Despite Elevidys failing a confirmatory trial, Sarepta secured full FDA approval and a label expansion, generating $181 million in Q3. The deal reflects Sarepta's ability to secure regulatory success and diversify its portfolio, potentially offering new options for patients with rare diseases.

Accelerated approval, despite recent controversies, has historically provided therapeutic momentum, particularly in HIV and cancer treatments. Key issues include reliance on surrogate endpoints and confirmatory trial failures, yet the pathway has led to significant drug approvals, offering hope for diseases with high unmet needs.

Roche acquires Poseida Therapeutics for up to $1.5B, expanding into CAR-T therapies. U.S. overdose deaths decline by 14.5% to 97,000 over 12 months. Arrowhead Pharmaceuticals licenses programs to Sarepta Therapeutics for $500M upfront and $325M in equity. Recordati and Angelini Pharma consider merger to form Europe's largest generics group. FDA approves BridgeBio's heart drug for transthyretin amyloidosis, challenging Pfizer's tafamidis. Novo Holdings expected to receive EU approval for $16.5B acquisition of Catalent Pharma Solutions. Amgen's obesity drug MariTide shows 20% weight loss in Phase II. Anavex Life Sciences submits MAA to EMA for Alzheimer's treatment. Biohaven's muscle drug fails in SMA study. embecta reports strong revenues of $1.12B, discontinues insulin patch pump. Axsome Therapeutics reports positive Phase 3 results for narcolepsy drug AXS-12. Kronos Bio to lay off 83% of workforce. PTC Therapeutics sells Rare Pediatric Disease Priority Review Voucher for $150M. EMA launches medicine shortages monitoring platform. Alector lays off 41 employees after Alzheimer's drug AL002 fails. Grifols seeks to refinance €1.4B in debt. Roche's cancer drug tiragolumab fails Phase 3 trial.