Amylyx Pharmaceuticals

Bicara Therapeutics raised $315 million in IPO to fund a pivotal study for its head and neck cancer therapy, ficerafusp alfa, which targets EGFR and TGF-beta signaling. The drug, combined with Keytruda, showed a 54% overall response rate in advanced HNSCC patients, with 64% in HPV-negative cases. A Phase 2/3 trial is planned for 2025.

An ALS drug candidate, DNL788/SAR443820, by Sanofi and Denali Therapeutics failed a Phase 2 trial, missing the main endpoint on the ALS Functional Rating Scale-Revised. Despite strong Phase 1 results, the failure questions RIPK1 inhibition's efficacy for ALS treatment. Further insights may come from an ongoing 52-week extension study. Meanwhile, other RIPK1 inhibitors and ALS treatments continue development.

Amylyx Pharmaceuticals' shares fell after its ALS treatment failed in a phase III study. Acadia Pharmaceuticals also saw a decline due to a failed schizophrenia treatment study. Regulus Therapeutics reported positive results for a kidney disease treatment, boosting its shares. Moderna initiated a cancer therapy study, increasing its stock value. Regeneron received FDA approval to extend its cholesterol drug's label to pediatric patients.

Amylyx Pharmaceuticals' ALS drug, Relyvrio, failed in a large phase 3 trial, showing no significant benefit over placebo in improving ALS symptoms or survival. The company may withdraw it from the market, pending discussions with regulators and the ALS community. Relyvrio, approved in 2022 based on a smaller trial, has been controversial due to its high cost and uncertain efficacy.

Denali Therapeutics and Sanofi's experimental ALS drug failed in a mid-stage study to slow motor function decline, marking another setback in ALS treatment development. The drug aimed to inhibit a protein contributing to neurodegeneration. Sanofi will continue testing the drug for multiple sclerosis.

Denali Therapeutics and Sanofi's ALS drug failed to slow motor function decline in a mid-stage study, marking another setback in ALS treatment development. Despite this, Sanofi will continue testing the drug for multiple sclerosis. ALS affects thousands in the U.S., with few FDA-approved treatments available.

Aldeyra's shares fell after FDA identified issues with its NDA for reproxalap, requiring additional studies. Annovis Bio's Alzheimer’s study on buntanetap showed positive interim results. Scholar Rock plans to expand into cardiometabolic disorders with SRK-439. Evelo's psoriasis study failed, halting EDP2939 development. Amylyx faces EU setback for ALS treatment AMX0035, focusing on phase III study results.

Biogen's Qalsody, targeting SOD1-ALS, a rare genetic form of ALS, received FDA approval. It's the first treatment addressing a genetic cause of ALS, showing promise in reducing neurodegeneration markers. Administered via spinal injection, it offers hope for those affected by this progressive disease.

ALS, a fatal neurodegenerative disease, has seen limited treatment options despite its discovery over a century ago. Recent scientific advancements offer hope, with new drugs like AMX0035 showing modest benefits in slowing disease progression and extending survival. However, challenges remain in understanding ALS's biology and developing effective treatments, especially for sporadic cases without known causes. The field is witnessing increased investment and research, aiming for breakthroughs that could significantly improve patients' lives.