Lightlake Therapeutics Inc., a small New York-based biopharma, gained some big interest following FDA approval of Narcan (naloxone hydrochloride) for emergency treatment of known or suspected opioid overdose. Lightlake developed the nasal spray, which will be marketed by partner Adapt Pharma Ltd., of Dublin.

Last year, the companies inked an exclusive global license for the product, which becomes the first naloxone-based nasal spray indicated for emergency treatment of opioid overdose. As such, the drug can be administered by nonmedical personnel, including family members and caregivers, until the patient receives professional medical care. Previously, naloxone was approved by the FDA only in injectable form to reverse the effects of opioid overdose.

Opioid overdose was a factor in nearly 24,500 deaths in the U.S. in 2013, with most of those involving the use of prescription opioids and many occurring in homes, according to the Centers for Disease Control and Prevention.

Last year, in part as a response to the storm of criticism over its approval of Zohydro ER (hydrocodone, Zogenix Inc.), the FDA approved Kaleo Inc.'s Evzio (naloxone autoinjector) to rapidly reverse an opioid overdose. (See BioWorld Today, March 14, 2014, and April 4, 2014.)

Adapt Pharma said it will make a carton containing two doses of Narcan available at an initial discounted "public interest" price of $75 to qualified first-line responders and group purchasers such as law enforcement organizations, firefighters, health departments, school districts, colleges and universities and community-based organizations.

The ready-to-use, needle-free device delivers a 4-mg dose of naloxone in a single 0.1 ml nasal spray. The product is expected to be available in retail pharmacies early next year.

"We know there's a huge unmet need," said Roger Crystal, Lightlake's CEO, noting that the company took some risk "to ensure that the development timing was as tight as possible to allow a rapid launch very shortly after approval."

The drug, approved through the 505(b)(2) pathway, received FDA fast track status earlier this year. In June, Adapt opened a rolling new drug application (NDA) submission.

As part of the deal with Adapt, which bears full responsibility for Narcan's commercialization, Lightlake stands to receive more than $55 million in undisclosed up-front and milestone payments, plus product royalties that could rise into double digits. Cash flow from its first approved product will allow Lightlake to refocus on other candidates in its pipeline while Adapt does "the heavy lifting" for Narcan, Crystal said.

'THERE ARE VERY FEW COMPELLING TREATMENTS IN THIS SPACE'

Initially based in London, Lightlake moved to the U.S. this year to be closer to its investor base and to the National Institute on Drug Abuse (NIDA), which has been a key source of grant funding and clinical trial partnerships. The company is rooted in the treatment of addictions, substance abuse and eating disorders, with a common focus on the opioid system in the brain. Its technology is based on the scientific work of its late founder, David Sinclair, in opioid antagonists.

In its early years, "we did some work in binge eating disorder and had some good data," Crystal told BioWorld Today. "But then the opportunity came along to move into stealth mode and go into the opioid overdose space. We recognized that as a tremendous opportunity for the company."

Lightlake "pivoted" into the development of Narcan, which was advanced to approval in less than three years.

"We're now going back to the roots of the company and focusing upstream in terms of addiction and other products," Crystal said.

In addition to binge eating disorder, Lightlake has earlier-stage candidates to treat bulimia nervosa and amphetamine and cocaine addiction, as well as several undisclosed candidates. The company employs the same basic science across the therapies in its pipeline but adds a novel twist for each indication.

"We're pursuing a lot of activity around the technology to establish a broader intellectual property platform, and that's something we see happening over the next 12 months," Crystal said.

Human trials in several indications also are expected to begin enrolling next year.

Other products emerging from the platform also are expected to rely on nasal delivery – Crystal called nasal formulation "key to the delivery of opioid antagonists" – but follow-on candidates likely will take the traditional NDA route since they'll mostly target chronic conditions. Nevertheless, he predicted development and potential approval will still be fairly rapid, since opioid antagonists have a well-established profile with regulators.

Lightlake expects to move the remainder of its pipeline to approval on its own, and the company is not averse to broadening its portfolio through in-licensing.

"We have no immediate need to partner," Crystal said. "Our sweet spot is to be able to get into new indications that previously weren't considered suitable for existing drugs and take them down a well-established approval pathway."

In addition to NIDA, the company has received support from nonprofit and R&D organizations, "which recognize that there are very few compelling treatments in this space," Crystal said. "We're able to leverage their resources, so we're in a very good position to carry out the key activities we need to do in the next 12 months."

A small biopharma – Lightlake has four full-time employees – that has the ability to generate product revenue also is attractive to investors, he observed. The company, which trades over-the-counter (OTCQB:LLTP), saw its shares leap 75 percent Thursday before settling back to close at $8.40 for a gain of $2.13, or 34 percent.

Long term, Crystal said, Lightlake could become a target for a biopharma with a meaningful presence in treating conditions with a psychiatric component. The company also could turn into a niche specialty pharma, targeting the small group of specialists who treat binge eating disorders and the like.

Still, "to get the full potential of any market we're looking at for the future, there will have to be some element of partnering," he said.