Another setback for aging-focused drug development, as researchers seek to highlight positives from study data.
Longevity biotech Calico Life Sciences has reported disappointing results from its first drug trial. Developed as part of the company’s collaboration with pharma giant AbbVie, the drug, fosigotifator, failed to meet its primary and secondary endpoints in a 24-week clinical trial for the treatment of amyotrophic lateral sclerosis (ALS).
Founded in 2013 by Google, the notoriously secretive Calico was formed to understand and address the challenges of aging, and has reportedly raised more than $3.5 billion in funding to date. The company’s R&D partnership with AbbVie, reportedly worth $1 billion, focuses on aging and age-related diseases, and also has ongoing clinical trials in the oncology space.
The trial, part of the HEALEY ALS Platform Trial, involved 155 patients receiving a primary dose of fosigotifator, 79 receiving an exploratory high dose, and 126 receiving a placebo. The study aimed to evaluate the drug’s efficacy in slowing disease progression and improving respiratory function and quality of life in ALS patients.
The study results found that fosigotifator did not significantly slow disease progression compared to the placebo group, and failed to improve respiratory function or quality of life better than the placebo. Secondary endpoints, including the revised ALS functioning rating scale, showed no statistically significant improvement.
In a post on X, Insilico Medicine founder Alex Zhavoronkov said Calico’s trial failure was “very sad news,” adding that it represented “a big blow for aging research in drug discovery.” The news comes not long after BioAge Labs’ recent discontinuation of its clinical trial in obesity on safety grounds.
While the trial results were not as hoped, researchers noted some potentially promising findings from the study, particularly at the exploratory high dosage of fosigotifator, which appeared to maintain muscle strength and respiratory function for a longer duration than the placebo.
We remain deeply committed to fully understanding the effects of fosigotifator in ALS, and will further evaluate the data before determining next steps,” said Massachusetts General Hospital’s Dr Merit Cudkowicz, principal investigator and sponsor of the HEALEY ALS Platform Trial. “Every step we take brings us closer to finding effective treatments.”
“The trial has provided substantial clinical data so far around dosing and the upcoming biomarker data and subgroup analyses will contribute to future studies and help us better understand ALS,” said Northwestern University’s Dr Senda Ajroud-Driss, co-lead of the trial.
Dr Bill Cho, Calico’s head of clinical sciences, said that the study has provided valuable insights into the potential bioactivity of fosigotifator in individuals with ALS, warranting further exploration.
“We remain committed to investigating the potential of fosigotifator as a much needed treatment option for people living with ALS and for other disorders, including vanishing white matter disease and major depressive disorder which each test different scientific hypotheses,” he added.
The Calico news comes as part of a double-whammy for ALS drug development, with Denali Therapeutics also announcing that its candidate had failed as part of the same platform trial. The results highlight the ongoing challenges in developing effective treatments for neurodegenerative diseases and underscore the complexity of processes that drug developers seek to address.
Despite the recent setbacks, discovering new treatments for ALS remains a high priority for the industry, exemplified by today’s announcement of a new collaboration between Alchemab Therapeutics and Lilly to “discover, research and develop” new drugs for the devastating condition.
