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Parkinson’s Trial Failure Signals a Likely End for CNS Biotech Aptinyx

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Aptinyx’s approach of treating neurological disorders by targeting a key receptor in the brain has fallen short again, this time in Parkinson’s disease. With its third clinical trial failure in less than a year, the biotech said it will end the Parkinson’s program as well as a separate one in post-traumatic stress disorder.

The drug candidate for Parkinson’s, NYX-458, was in testing as a potential treatment for mild cognitive impairment or mild dementia associated with that disease or a closely related neurological disorder called dementia with Lewy bodies. The once-daily pill was being evaluated in a 99-patient, placebo-controlled study. According to preliminary results released after Monday’s market close, the Aptinyx drug did not show clinically meaningful improvement as assessed using tests to evaluate function and cognition in Parkinson’s patients.

“The results overall do not support further advancement of the development program by Aptinyx,” the company said in its announcement.

Evanston, Illinois-based Aptinyx develops small molecule drugs that target and modulate N-methyl-D-asparate (NMDA) receptors. These receptors are important to normal function of the brain and central nervous system. Aptinyx’s drugs stem from its platform technology for discovering molecules that can modulate NMDA receptors.

The Aptinyx technology has yielded drug candidates for chronic pain, Parkinson’s, and PTSD. Last April, Aptinyx reported that chronic pain drug NYX-2925 failed to distance itself from a placebo in a Phase 2 study enrolling patients with diabetic peripheral neuropathy. The company continued a mid-stage test assessing the drug in fibromyalgia. Last August, the chronic pain drug failed that study as well.

After the Parkinson’s failure, Aptinyx isn’t waiting to see the results for drug candidate NYX-783 in PTSD. The company said it will terminate that study and analyze the data to determine its next steps for that program. Aptinyx also said it will implement cost-cutting measures and explore “strategic alternatives,” which could mean a sale of the company or its assets, or perhaps a merger with a privately held biotech looking for way to enter the public markets.

Aptinyx’s drug discovery technology came from Naurex, a psychiatric drugmaker that was acquired by Allergan in 2015. That year, Aptinyx launched to develop its NMDA receptor-targeting drugs. The company went public in 2018, pricing its shares at $16 each and raising nearly $118 million. Shares of Aptinyx opened Tuesday at 20 cents apiece.

Image: Dr_Microbe, Getty Images

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