Fulcrum Therapeutics

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.

Pfizer's withdrawal of Oxbryta from global markets due to increased risk of deaths and complications has left the sickle cell disease community reeling. Despite recent setbacks, hope for SCD treatment lies in next-gen transplantation and gene therapy, with St. Jude developing its own gene therapy. Pfizer's inclacumab and osivelotor, Agios Pharmaceuticals' mitapivat, Novo Nordisk's etavopivat, and Fulcrum Therapeutics' pociredir are among investigational therapies in the SCD pipeline.

Pfizer's withdrawal of Oxbryta due to death risk may accelerate trials of rivals like Agios' mitapivat and Fulcrum's pociredir. Oxbryta's suspension leaves mild-moderate patients reliant on hydroxyurea. Gene therapies Lyfgenia and Casgevy target severe cases. Pfizer's stock dips amid broader challenges.

Apellis Pharmaceuticals' intravitreal pegcetacoplan for geographic atrophy (GA) treatment faces a second negative opinion from the CHMP in June 2024, despite EU retina community support and dissenting CHMP votes. Shares dropped 11.5%.

FDA approves Merck's Keytruda for first-line treatment of unresectable advanced or metastatic malignant pleural mesothelioma, based on KEYNOTE-483 study showing improved overall survival and progression-free survival.

Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo in improving upper limb function and other clinical measures in people with facioscapulohumeral muscular dystrophy (FSHD) in the REACH Phase 3 trial. The company plans to stop developing losmapimod and focus on other clinical programs.

Moderna revised its break-even forecast from 2026 to 2028, leading to significant R&D cuts and a downgrade in stock ratings by firms like Jefferies and J.P. Morgan. The company plans to focus on 10 pipeline candidates, halting development of five programs, and expects a compounded annual growth rate of over 25% from 2026 to 2028.

Fulcrum Therapeutics' stock dropped >60% after its Phase 3 REACH trial for losmapimod, a potential FSHD treatment, missed its primary endpoint, leading to the suspension of the program.

Fulcrum Therapeutics' experimental drug losmapimod failed a Phase 3 trial for muscular dystrophy FSHD, leading to suspension of its development. Despite positive signs in previous tests, losmapimod missed its main objective and secondary goals. Fulcrum's shares dropped 60%, and the company will focus on its sickle cell disease treatment, expecting Phase 1 data next year. The FDA previously suspended testing of the sickle cell therapy for six months.