Cassava Starts Second Alzheimer's Study After PT-125 Effects Multiple Biomarkers

The company formerly known as Pain Therapeutics revealed a 100% response rate in a small Phase IIa study and hopes to confirm the novel drug's effects on all biomarkers tested in Phase IIb.

Neurons and nervous system. 3d render of nerve cells
PTI-125 may help restore normal function of neurons. • Source: Shutterstock

Cassava Sciences Inc. delivered its first set of data from a small study in Alzheimer's disease for PTI-125, its only remaining clinical-stage drug since the company changed its name from Pain Therapeutics Inc. in March. The small molecule, which has a novel mechanism of action in a disease that needs fresh treatment approaches, generated a 100% response rate in the 13-patient Phase II clinical trial and impacted all nine biomarkers assessed in the study.

Pain Therapeutics changed its name to Cassava as its focus shifted from the abuse-deterrent opioid Remoxy, which was rejected by the US Food and Drug Administration, to its early-stage Alzheimer's candidate PTI-125, which binds to misfolded versions of the protein filiman A (FLNA) to restore its normal shape and function. The National Institutes of Health have committed up to $6

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