In Remoxy, Pain Therapeutics created a safer, abuse-resistant form of twice-daily oxycodone. The sticky, high-viscosity composition of Remoxy resists injection or snorting. When frozen, crushed, or mixed in alcohol, only a small fraction of oxycodone is released compared to Oxycontin. This discourages recreational drug abuse and prevents accidental misuse by patients.
Remoxy received a Special Protocol Assessment (SPA) from the FDA in February 2006, which specifies the Phase III trial objective, design, clinical endpoints, and analyses needed to support regulatory approval. Under the terms of the SPA, just one pivotal trial is required to file a New Drug Application.
The randomized, double-blinded, placebo-controlled, multicenter trial will enroll 400 patients with moderate-to-severe osteoarthritic pain in the U.S. Patients will be randomized to either Remoxy (10 milligrams daily) or placebo for 12 weeks.
After Remoxy receives marketing approval, Pain Therapeutics has a strategic alliance with King Pharmaceuticals (www.kingpharm.com) to develop and commercialize Remoxy and other abuse-resistant opioids.
Barbier describes Pain Therapeutics’ business strategy as a "high-quality clinical development shop" that "brings in a mix of low-risk, high-reward compounds and high-risk, high-reward compounds." Remoxy, for instance, is a low-risk compound, whereas Oxytrex is a high-risk compound. "Our business strategy is to build a blended pipeline," Barbier says. "We want biotechnology and specialty pharmaceutical products under the same roof. To us, drug development is drug development. We’re indifferent whether it involves testing a reformulated version of a known compound or testing the safety and efficacy of an entirely new compound. The skills required to excel in drug development are similar in both scenarios. I think this is a unique strategy in our industry."
Pain Therapeutics scouts out programs in the pre-IND stages to in-license and develop. They maintain close ties with academic researchers who have projects in the early stages of development. "When a project is ready for prime time, we in-license it," says Barbier. This model cuts down on the high costs of early-stage research, one of the pharmaceutical industry’s greatest inefficiencies.
"Early-stage research requires a lot of smarts but also a lot of luck. Even the brightest people fail in drug discovery, which is why big pharma has not been able to corner the market for novel drugs. In contrast, drug development requires a lot of hard work, people skills, teamwork, and a laser-like focus. These are precisely our strengths," says Barbier.
For example, some aspects of the formulation used in Remoxy were in-licensed for $1 million, and Pain Therapeutics spent $12 million more to develop it. Then, King Pharmaceuticals paid $400 million plus a 20% royalty rate to develop and commercialize Remoxy.
"Our approach is highly efficient by industry standards," says Barbier, and it allowed the company to move three new compounds through Phase III trials in just six years. Pain Therapeutics expects its first product to come to market in 2008.