Med Check

Therapix Biosciences in April reported positive data from a phase 2 study assessing its cannabinoid-based drug THX-110 as a treatment for Tourette syndrome. THX-110 is a combination of the FDA-approved synthetic cannabinoid dronanbinol and the fatty acid palmitoylethanolamide.

In the open-label trial, 16 patients received a daily oral dose of THX-110 for up to 12 weeks. Most of the patients had severe, refractory Tourette syndrome and had failed to respond to such treatments as antipsychotics, behavioral therapy, and deep brain stimulation.

The patients showed an average tic reduction of 21 percent (measured by the Yale Global Tic Severity Scale Total Tic Score). Six patients experienced tic reductions of greater than 25 percent.

Twelve of the 16 patients are continuing in a 24-week extension study.

The Food and Drug Administration (FDA) in April accepted Alkermes’ New Drug Application (NDA) for ALKS 5461, a novel, once-daily medication for the adjunctive treatment of major depressive disorder (MDD) in patients with an inadequate response to standard antidepressants. ALKS 5461 is a fixed-dose combination of two opioid compounds, buprenorphine and samidorphan.

On March 30, the FDA sent a Refusal to File letter to Alkermes stating that the NDA contained insufficient evidence of overall effectiveness for the proposed indication of MDD, and that additional, well-controlled clinical trials were needed prior to the resubmission of the NDA. The FDA also requested that Alkermes conduct a bioavailability study to generate additional data that can compare ALKS 5461 and the reference listed drug, buprenorphine.

In a statement released by Alkermes, the company noted it had productive interactions with the FDA to clarify certain aspects of their NDA submission, which contained data from more than 30 clinical trials involving more than 1,500 patients with MDD. The FDA has now rescinded the refusal letter without requiring additional trials.

Indivior in late March announced that it has entered into a licensing agreement with the biotech C4X Discovery. The company has obtained exclusive rights to develop and commercialize C4X’s library of orexin-1 (OX1) receptor antagonist drugs. Preclinical studies have suggested that blocking the orexin pathway can decrease drug-seeking behavior and reduce the risk of relapse for a wide range of substances, including stimulants, opiates, and alcohol.

Under the terms of the agreement, Indivior will pay $10 million up front to C4X, with subsequent milestone payments potentially reaching $284 million if all development, regulatory, and commercial goals are achieved.

“We believe C4X’s OX1 program is one of the most promising early stage programs to potentially treat addiction and look forward to rapidly advancing lead compound C4X3256 into the clinic,” said Invidior Chief Scientific Officer Christian Heidbreder, Ph.D., in the company’s press release. “Further, we believe there is great potential for additional compounds to be discovered and developed with respect to this important mechanism that will contribute to the continued elucidation of the neurobiological underpinnings of withdrawal symptoms, drug intake, craving, relapse, and comorbid psychiatric associations.”

Pfizer reported in late March that a large, phase 4 study evaluating the effectiveness of Chantix (varenicline) for smoking cessation in adolescents failed to meet its primary endpoint.

The phase 4 study enrolled 312 nicotine-dependent adolescents aged 12 to 19. The adolescents were randomly assigned to receive either varenicline or placebo for 12 weeks. Patients in the varenicline group were further divided and assigned to take either 1 mg of varenicline twice daily (high-dose group) or 0.5 mg varenicline twice daily (low-dose group). At the study’s end, continuous four-week abstinence rates were no different between the varenicline groups and the placebo group.

The adverse event profile was similar to what has been observed in adults taking varenicline; the most common side effects were nausea, headache, vomiting, agitation, and abnormal dreams. ■