FDA Removes Partial Clinical Hold for CTP-354, Concert Pharmaceuticals’ Novel Drug for MS, Spasticity

The U.S. Food and Drug Administration (FDA) recently notified Concert Pharmaceuticals that the company had fulfilled all necessary preclinical toxicological tests for the oral therapy CTP-354 for the treatment of multiple sclerosis, spinal cord injury, and spasticity. The authorization to administer repeated doses of the compound, in excess of 6 mg per day, signals the lifting of its partial clinical hold.

CTP-354 is a non-sedating subtype-selective GABA(A) receptor modulator that targets GABA(A) receptors, which can be found in the nervous system and can influence the transmission of determined nerve signals. Although there are drugs used to target the same receptors, such as benzodiazepines, the drug developed by Concert Pharmaceuticals is the only one to include the receptor subtype selectively for CTP-354.

Concert Pharmaceuticals announced that, with the partial removal of the FDA clinical hold, they will continue their ongoing multiple ascending dose phase 1 trial and initiate dosing the patients enrolled with 12 mg per day of CTP-354 in the third quarter of this year. CTP-354 is one of Concert’s most advanced non-partnered drugs in development, and the company’s goal is to improve the existing drugs changing its molecular structure.

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Spasticity, the main condition that the oral drug aims to treat, is a chronic condition characterized by involuntary tightness, stiffness or contraction of muscles. It occurs in patients with brain or spinal cord damage and can be a result of multiple sclerosis. There were about 12 million people suffering from spasticity in the world in 2006, according to the American Association of Neurologic Surgeons.

The markets also responded to the announcement and the company’s shares ascended after the lift. “We are encouraged by the removal of the partial clinical hold on the study and view positively CNCE’s determination to proceed onward with the ascending dose study, initiating a higher dose cohort. We look forwards to the next step in CTP-354 development with the expected Phase II study to be initiated in 2014,” analyst Joseph Pantginis commented to the Street Insider.