ALS
Three years after Qalsody was approved by the FDA as the first drug to target a genetic cause of ALS, some patients are reporting not only disease stabilization but also improvement of symptoms.
Biotechs that announced series B financing this year and are looking for employees as they grow include one that received over $100 million and another that has a partnership with a Big Pharma company.
Nura Bio will use the series B financing to carry two neuroprotective drug candidates through early- to mid-stage clinical studies, one targeting ALS and the other for a broader neurological profile.
After suffering the market withdrawal of its only product, Amylyx is gearing up for a pivotal Phase 3 readout in post-bariatric hypoglycemia. But the company’s driving ethos is still to treat “debilitating, devastating” neurodegenerative diseases, co-CEO Justin Klee told BioSpace.
Improved survival on display at the American Society of Clinical Oncology (ASCO) in Chicago; Pfizer’s unusual pact with China’s Innovent highlights a new type of collaboration; Eli Lilly continues its nonstop deal streak, including with Chinese biotechs; and looking ahead to this weekend’s American Diabetes Association meeting.
For Peter Pitts, a former associate commissioner at the FDA, the appointment to the board of BrainStorm Cell Therapeutics is an opportunity to fulfill a promise he made long ago to a patient with ALS.
Biogen’s Qalsody won FDA approval in 2023 to treat a rare, genetic form of amyotrophic lateral sclerosis. On Tuesday, QurAlis presented interim Phase 2 data showing the potential of a similar drug to more broadly treat the neurodegenerative disease.
Corcept Therapeutics’ amyotrophic lateral sclerosis drug was linked to an 87% reduction in the risk of death, a result the biotech hopes to replicate in an upcoming Phase 3 trial.
Trace Neuroscience, a member of BioSpace’s NextGen Class of 2026, has learned from the success of Biogen’s Qalsody and aims to bring more treatment options to the ALS community.
As Congress debates renewing the Act for ALS, it must prepare for the coming era of precision ALS medicine by prioritizing early-stage research to more quickly bring effective treatments to the market.
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