SANTA MONICA, Calif.—Specialty pharmaceutical company Opiant Pharmaceuticals Inc. recently announced positive data from a Phase 1 clinical study for its product candidate OPNT003, as well as its plans for a development pathway.
OPNT003 is a long-lasting, intranasal nalmefene treatment to counter opioid overdose. The product candidate is a novel formulation deliverable via non-invasive metered nasal spray rather than by injection, thanks in part to a proprietary absorption enhancer (Intravail) licensed from Aegis Therapeutics.
Nalmefene is an opioid antagonist previously given U.S. Food and Drug Administration (FDA) approval for delivery by injection for treating suspected or confirmed opioid overdose. The only medication currently FDA approved to treat opioid overdose, naloxone, has a half-life of about two hours.
The data from the Phase 1 study tied to the development of OPNT003, completed under a clinical trial agreement with the National Institute on Drug Abuse, demonstrated that the intranasal nalmefene formulation containing Intravail delivered rapid increases of the drug in plasma levels with an onset faster than that of an intramuscular injection. The compound also boasted a significantly longer half-life than that of current naloxone treatments, between 6.7 and 7.8 hours.
The company held a meeting in early February with the U.S. Food and Drug Administration (FDA) regarding its planned development program. Based on the FDA’s feedback, Opiant announced in a statement released on Feb. 8, 2018, that it intends to pursue a 505(b)(2) development path for OPNT003, and anticipates submitting a New Drug Application (NDA) for the drug and intranasal delivery device combination in 2020.
According to Opiant, the 505(b)(2) pathway allows companies to rely in part on the FDA’s findings of safety and efficacy of a previously approved product and to supplement these findings with a more limited set of their own studies to satisfy FDA requirements, as opposed to conducting the full array of preclinical and clinical studies that would typically be required.
“We are pleased with the positive outcome of this meeting and the beneficial guidance received from the FDA,” Dr. Roger Crystal, CEO of Opiant, said in a news release announcing the development plan. “We now have a well-defined development and regulatory pathway to pursue approval of OPNT003 in the U.S. for the treatment of opioid overdose.”
Aegis announced in late April that it had been granted a U.S. patent for its non-invasive metered nasal spray delivery of multiple opioid blockers such as nalmefene, naltrexone, methylnaltrexone and naloxone for rescue therapy in opioid overdose, as well as opioid pain medications such as morphine, buprenorphine, nalbuphine and others. Opiant Pharmaceuticals is Aegis’ exclusive worldwide licensee for nalmefene, naloxone and naltrexone.
Opiant owns all global development and commercial rights to OPNT003.
In a special report in the New England Journal of Medicine (Volkow and Collins, NEJM 2017), directors at the National Institutes of Health (NIH) wrote of the need for stronger and long-acting formulations of opioid antagonists to counteract the high-potency synthetic opioids that are now claiming thousands of lives each year. In 2016, synthetic opioids, including fentanyl and derivatives such as carfentanil, were responsible for more overdose deaths—more than 20,000—than either heroin or prescription opioids. Synthetic opioids are especially dangerous because their long half-life of seven to ten hours may require repeated dosing of naloxone to overdose victims to prevent relapse.
According to Opiant, a long-lasting overdose-reversal drug may reduce this burden. Furthermore, the company believes an easy-to-use nasal delivery of nalmefene with a rapid onset and long duration of action could allow non-medically trained people to administer the treatment in an emergency. If approved by the FDA, OPNT003 may be especially useful in rural areas, in communities where emergency medical responses may be hours away and in which the number of occurrences of opioid overdoses are rapidly increasing.
Opiant has submitted a grant application to the NIH to fund OPNT003’s development to an NDA-ready stage, according to Crystal, and the company is optimistic that the grant will be funded in light of the NIH’s public statements about the dire need for longer-acting overdose-reversal drugs.
“Based on its profile and the Phase 1 trial results, we believe OPNT003 has the potential to be a transformative treatment for opioid overdose, a growing U.S. health epidemic,” said Crystal.
Opiant is a specialty pharmaceutical company developing pharmacological treatments for addictions; its first product, NARCAN Nasal Spray, is approved for marketing in the United States and Canada by its partner, Adapt Pharma.