The FDA has stopped Cortexyme from studying its lead drug any further in Alzheimer's disease and other conditions, dealing a blow to the pipeline and sending shares cratering 30% Wednesday morning. The biotech will now have to focus on its second bet at treating the neurodegenerative disease.
The biotech's drug, atuzaginstat, failed a phase 2/3 clinical trial in patients with the memory-robbing disease in November 2021, but the company vowed to forge ahead anyway with a lower dose based on data that showed a reduction in Porphyromonas gingivalis infection was related to the slowing of cognitive decline in a prespecified patient population.
The 643-patient phase 2/3 finished up in October. Atuzaginstat did not meet its co-primary cognitive and functional endpoints in the 48-week treatment period. The drug was studied in the U.S., France, Spain, Poland, the U.K. and the Netherlands.
The company's shares tanked nearly 30% to $6.41 apiece as of 10:21 a.m. ET on Tuesday.
Cortexyme was also testing the lysine gingipain inhibitor in patients with Parkinson's disease and periodontal disease, in which gums and bones around the teeth are infected and inflamed. Those indications are now on hold along with the Alzheimer's tests.
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The biotech did not disclose details of the FDA's hesitance on atuzaginstat.
Cortexyme will explore other options for those two indications as well as find strategic alternatives for a different preclinical coronavirus program, the South San Francisco biotech disclosed Wednesday morning. Cortexyme had selected a 3CLpro inhibitor, COR803, to study as a treatment for COVID-19 variants and coronaviruses more broadly in July 2021.
Now that its first bet at treating Alzheimer's is sidelined, the company will focus on a second attempt through COR588, which is in a phase 1 study with results slated for next quarter. The therapy is a next generation gingipain inhibitor, according to Cortexyme.
The biotech will conserve cash to support other clinical milestones so Cortexyme can keep the lights on through 2024. More details on the biotech's pipeline and milestone timeline will be revealed later.