FDA Grants Orphan Drug Status to Coya’s T-cell Therapy ALS001

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by Patricia Inacio PhD |

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Coya Therapeutics’ ALS001, a potential regulatory T-cell (Treg) therapy designed to halt amyotrophic lateral sclerosis (ALS) progression, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA).

Furthermore, a Phase 2a trial (NCT04055623) testing ALS001 versus a placebo in 12 ALS patients has been completed, and results are expected to be released by the end of the summer, the company announced.

“We look forward to showcasing our manufacturing scalability, optimizing our clinical development plans, and working with the FDA to bring ALS001 to patients as efficiently and quickly as possible,” Howard Berman, PhD, CEO of Coya Therapeutics, said in a press release.

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Orphan drug designation seeks to encourage the development of therapies for rare and serious diseases through benefits such as seven years of market exclusivity following approval and exemption from FDA application fees.

“The orphan drug designation for ALS001 underscores the medical need that exists for patients with ALS,” Berman said.

Tregs are key players in controlling the immune system, shutting down excessive inflammatory responses such as those involved in nerve cell degeneration.

Previous research led by the lab of Stanley H. Appel, MD, co-director of Houston Methodist Neurological Institute, showed a link between lower levels of Tregs and ALS progression, contributing to uncontrolled inflammation.

Building on this research, scientists at Coya developed a way to isolate Tregs from ALS patients (autologous cells) and to manipulate the cells so that they become functional and neuroprotective. The cells are then expanded in the lab into “billions” of Tregs, after which they are infused back into the patient via intravenous (into-the-vein) infusions.

These cells can also be cryopreserved without losing effectiveness, which allows a continuous supply.

The therapy was deemed safe and well-tolerated in a Phase 1 pilot trial (NCT03241784). In the three ALS patients enrolled, it showed signs of being able to slow down disease progression at both early and late stages.

Prompted by these promising results and with enough capital — $10 million series A funding and an additional $1.1 million — the Phase 2a trial was launched.

The study will compare the safety, tolerability, and activity of ALS001, given as monthly intravenous infusions for six months, versus a placebo in 12 ALS adult patients; so far, eight have been treated. A low-dose of interleukin-2, essential for the survival and suppressive activity of Tregs, will be given in parallel, three times per week.

After completing this placebo-controlled stage, patients will be invited to continue or begin monthly treatment with ALS001 infusions in the study’s six-month, open-label extension phase.

The trial’s main goal is to compare the changes in Treg function after six months in ALS001-treated vs. placebo-treated patients. Additional goals will look at changes in Treg numbers, overall disease progression, and respiratory health.

“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 Treg cell therapy — ultimately slowing and halting the progression of ALS,” Berman said.