An open-label phase III study of Egalet Corp.'s abuse-deterrent, extended-release oxycodone, Egalet-002, found the drug to be generally well tolerated by about 150 opioid-experienced patients with moderate-to-severe chronic non-cancer pain. The candidate incorporates the company's proprietary Guardian Technology, first deployed in its already-approved Arymo ER (morphine sulfate) product, to make the tablets difficult to manipulate for misuse. Company president and CEO Bob Radie said the result validates its application of the technology.

While Egalet has completed a series of studies that could be filed as a new drug application, spokeswoman Blair Clark-Schoeb told BioWorld that the company is focused on finding a partner for the program and does not intend to file an application on its own.

Shares in the Wayne, Pa.-based company (NASDAQ:EGLT) climbed nearly 29 percent in premarket trading, but fell 12.6 percent to close at $1 on Friday.

The study enrolled 281 patients at 39 clinical sites in the U.S. who had a history of moderate-to-severe chronic non-cancer pain for six months or more. It sought to administer the drug to about 150 patients for six months and at least 50 patients for one year. Its primary endpoint was the incidence of treatment-emergent adverse events during the open-label treatment period, including those leading to treatment withdrawal, and/or abnormal physical examination findings, vital signs measurements, ECGs, and clinical laboratory test results related to treatment.

Adverse event reports in the trial were generally consistent with expectations for an extended-release oxycodone formulation, the company said. No other safety and efficacy trials are required.

A separate randomized, parallel assignment, double-blind, placebo-controlled, phase III study of the drug was initiated in March 2016 to evaluate safety and efficacy in 500 patients with moderate-to-severe chronic low back pain. The company said that that trial is expected to be completed by year-end.

In February 2014, the drug received FDA fast track status for the treatment of moderate-to-severe pain. If successful, it could offer health care providers interested in different moieties – including oxycodone, morphine, hydrocodone and others – an additional option for treating chronic pain.

Egalet gained U.S. approval for Arymo ER in January 2017, but strong initial investor enthusiasm for the drug's prospects was quickly blunted by news of a label indicating that its physical and chemical properties are "expected to make abuse by injection difficult. However, abuse by the intravenous, intranasal and oral routes is still possible." (See BioWorld Today, Jan. 11, 2017.)

Top-line data released by the company in March 2016 suggest that the company or an eventual licensor may be able to secure a more favorable label. That study, run to quantify the level of effort required to manipulate Egalet-002, found that doing so required significantly more effort than manipulation of Purdue Pharma LP's Oxycontin and Mallinckrodt Inc.'s Roxicodone.

The study found Egalet-002 was rated as moderately to extremely hard to physically manipulate on a tool by tool basis and that both of the dosages tested – 10 mg and 80 mg – not only required significantly more effort to tamper as compared to both Roxicodone and Oxycontin but also damaged and/or broke more instruments used to manipulate them vs. both Roxicodone and Oxycontin. Earlier studies exploring other avenues of manipulation found Egalet-002 provided further evidence of manipulation resistance enabled by the Guardian technology.

Despite positive clinical data, conditions outside the company's control may also play a significant role in shaping the future of its products. Most visible among those forces is the recent declaration by President Donald Trump of a public health emergency driven by the nation's crisis of opioid abuse. Speaking to the issue during the company's Nov. 8 earnings call, Radie said that "exactly how that's going to unfold in terms of potential funds, support for innovation, all that I think is still being determined and ironed out at this point in time."

Beyond government, Egalet will also need to work on increasing appreciation for and understanding the value it sees in abuse-deterrent formulations, particularly as it relates to diversion, said Radie. "We've been surprised at the general lack of understanding, not just with health care professionals, but I think amongst those that make decisions within insurers and payers about just what these abuse-deterrent formulations do, the types of abuse that they deter," Radie said during the earnings call.

When it comes to diversion, he said, "they're often thinking about the covered life, but not perhaps the other covered lives associated with that same insured patient like children at home, teenagers, whom might look to get their hands on legitimately prescribed products for purposes of abuse." Expanding appreciation for the power of abuse-deterrent formulations to address that issue will take a lot of education, he said.

During its third quarter, which ended Sept. 30, Egalet reported sales of $6.7 million, up 41 percent over last year's third quarter. Following a $30 million underwritten public offering that closed in July, it had cash, cash equivalents and marketable securities totaling $102.1 million, which it said would support its operations into 2020. It narrowed its net loss during the quarter to $18.9 million vs. $26.9 million in the third quarter of 2016.