Celcuity secures FDA 'breakthrough therapy designation' for breast cancer drug

The U.S. Food and Drug Administration this week gave Plymouth-based Celcuity's experimental breast cancer drug a special designation that could help expedite its development.

The local biotech company announced Monday it has received Breakthrough Therapy designation from the federal agency for its gedatolisib drug for some patients with breast cancer — the second-most common cancer for women.

While this designation is not an approval, the move signals confidence in Celcuity's early data, which showed 75% of people who completed the trial had a clinical benefit.

Brian Sullivan, CEO and co-founder of Celcuity Inc., called it "a significant acknowledgement."

The agency gives breakthrough designations as a way to open lines of communication between regulators and companies working on promising — yet not entirely proven — technologies that, if successful, could help a lot of people.

"These [designations] are basically the agency's stamp to say there's an unmet need that this product could potentially resolve," said Alex Nowak, senior research analyst at Minneapolis-based Craig-Hallum Capital Group.

According to data from the Centers for Disease Control and Prevention, more than 264,000 new breast cancer cases were reported in 2019. Celcuity's drug is for women with ER+/HER- advanced metastatic breast cancer who have been through initial therapy, but whose tumors are still growing.

The company reported that the drug was generally tolerated. Less than 7% of patients experienced side effects, including hyperglycemia, diarrhea, colitis and pneumonitis.

Nowak said the breakthrough designation is a step in the right direction, but there are still many steps to go.

"It's not to say that it's a slam-dunk for approval ... [but] from an investor perspective, it is helpful," said Nowak, who rates Celcuity stock a "buy."

Celcuity is just starting its Phase 3 clinical trial for the drug. Sullivan said the trial will include about 650 patients from up to 20 countries. The company hopes to have initial results in the second half of 2024. In clinical testing, Celcuity's medication has been given with Ibrance, an FDA-approved cancer drug made by Pfizer.

The company did not start out in pharmaceuticals. It was initially working on a diagnostic testing platform for analyzing a patient's living cancer cells to find more targeted therapies that could improve outcomes for cancer patients.