Coya Therapeutics receives orphan drug designation for ALS001

The FDA granted orphan drug designation to ALS001, an autologous, expanded T-cell therapy under development by Coya Therapeutics for treating ALS, according to a press release.

“The orphan drug designation for ALS001 underscores the medical need that exists for patients with ALS,” Howard Berman, PhD, CEO of Coya Therapeutics, said in the release. “Although there are currently no meaningful treatments for these patients, we are excited by our preclinical and early clinical data demonstrating ALS001’s ability to harness the neuro-protective effects of T-cell (Treg) therapy, ultimately slowing and halting the progression of ALS.”

Recent research has indicated that decreased levels of Treg may impact the drivers of ALS progression and disrupt the neurodegenerative disease process, according to the release.

In the current study of ALS001, researchers found that the drug may be effective in slowing the progression of ALS.

Coya Therapeutics has further assessed ALS001 in an ongoing randomized, placebo-controlled, phase 2a trial. Researchers are studying the biological activity, safety and tolerability of regulatory Tregs taken and expanded outside of the body and returned in 12 adults with ALS. The company expects to release the trial data later this summer, according to the press release.

Reference:

 Coya Therapeutics. Coya Therapeutics receives orphan drug designation from the FDA for ALS001, an autologous Treg cell therapy, for the treatment of ALS. Available at https://www.coyatherapeutics.com/news/10-press-release/100-oyaherapeuticseceivesrphanrugesignationfromthe20210707050501.html. Accessed July 13, 2021.