Sarepta Therapeutics

Axsome Therapeutics announced positive Phase 3 trial results for AXS-05, a treatment for Alzheimer's disease agitation, showing significant delay in symptom relapse and prevention of agitation. Plans to submit a New Drug Application to the FDA by 2025. AXS-05 also showed promise in narcolepsy treatment and Axsome reported record Q3 revenue.

In 2024, the FDA approved several therapies for neurological diseases, including treatments for CIDP, DMD, NMOSD, and Alzheimer's, offering new options and formulations to patients and clinicians.

Antisense oligonucleotides (ASOs) modulate gene expression via binding to mRNA, showing promise in treating genetic disorders, cancers, and inflammatory conditions. Key players include Sarepta, Ionis, and Opko Health, with limited activity in China. Startups like Remix Therapeutics lead innovation. The drug landscape focuses on oncology and CNS therapies, with major companies driving preclinical and phase II trials.

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.

UC Davis Health's documentary follows a family from Taiwan to California for their 2-year-old son's DMD gene therapy trial, testing Elevidys, a treatment replacing the faulty gene causing DMD, aiming to expand its use to younger patients.

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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Solid Biosciences Inc., focusing on Duchenne muscular dystrophy (DMD) treatments, is advancing with SGT-003, a gene therapy candidate. With a strong financial position and innovative capsid technology, it aims to disrupt the DMD treatment landscape. Despite operating at a loss, its potential for superior efficacy and safety profiles positions it as a key player in the gene therapy market.