Inspire Pharmaceuticals Inc. said following a productive meeting with the FDA, it has decided to compile existing data from three trials of its dry-eye product, INS365 Ophthalmic, and submit it for regulatory approval.
Inspire's stock (NASDAQ:ISPH) shot up $2.02 Wednesday, or 53.6 percent, to close at $5.79.
INS365 is an eye drop administered four times a day in patients who suffer from chronic dry eye, a condition involving abnormalities and deficiencies in the tear film due to a variety of causes, Durham, N.C.-based Inspire said. There are no pharmacologically active treatments for the condition, which is commonly found in post-menopausal women.
The company will officially submit its application following a "pre-NDA" (new drug application) meeting expected to take place over the next several months, Mary Bennett, Inspire's vice president of operations and communications, told BioWorld Today. Under the best of circumstances, Inspire hopes for marketing clearance next year, followed by a 2004 launch.
News of the pending NDA, announced early Wednesday, relates to a healthy set of data submitted to the agency by Inspire.
"We submitted a comprehensive package of all the existing clinical data and were invited to a face-to-face in Rockville [FDA headquarters] to discuss the package that included 1,200 dry-eye patients who had participated in Phase II and Phase III studies," Christy Shaffer, CEO of Inspire, said in a conference call. "We believe the package we submitted was very strong for a variety of reasons. We have consistently shown statistical significance in corneal staining, our 2 percent solution was consistently the most effective concentration and the removal of patients from treatment for a week resulted in a worsening of their staining scores in several studies."
Inspire's NDA will consist of data from a Phase II study, referred to as 103, and two Phase III trials, referred to as 104 and 105. An ongoing Phase III, called 108, will be designated a Phase IIIb supportive study.
Study 103 was a 158-patient trial that demonstrated statistical significance in corneal staining. While study 104, the first of the pivotal trials, did not meet its primary endpoint, it did meet statistical significance for corneal staining by the secondary statistical analysis at six weeks, Bennett said, adding that when the company did the combination analysis of 104 and 105, it "also saw a statistically significant improvement in corneal staining."
Study 104 caused Inspire some trouble in January when the company released the preliminary analysis indicating that the study would not reach its primary objective. Inspire's stock tumbled 73 percent, or $11.36, on the news to close at $4.16. But six months later, the stock shot up 102.7 percent when Shaffer said data from study 105 indicated that INS365 Ophthalmic 2 percent demonstrated a highly statistically significant improvement (p<0.001) over placebo for the primary objective endpoint of corneal staining. (See BioWorld Today, Jan. 17, 2002, and June 19, 2002.)
While dry-eye sufferers have few choices in remedies today, by the time INS365 enters the market, there may be some friendly competition.
Indeed, Inspire's partner, Allergan Inc., of Irvine, Calif., has its own product in development, called Restasis. Allergan is Inspire's partner on INS365 in the U.S. and the remainder of the world outside Asia, where Santen Pharmaceutical Co. Ltd. has the rights. Inspire officials said Restasis and INS365 are on the same timeline. (See BioWorld Today, June 28, 2001.)
David Steinberg, managing director of Deutsche Bank Securities in San Francisco, told BioWorld Today that under the contract with Allergan, Inspire will receive royalties on Restasis sales.
Both Steinberg and Greg Mossinghoff, president of Inspire, said Allergan is estimating that peak sales of Restasis could reach the $300 million to $500 million range.
Steinberg estimates INS365's worldwide peak will hit $400 million.
Regarding competition between the two products, Steinberg said Restasis may be positioned toward the more severe patients, and INS365 might better serve mild to moderate patients. "But it's really too early to make a definitive assessment about that," he said.
Mossinghoff would not discuss specific milestones paid by Allergan, however, he did say the initial deal was valued at $39 million.
"We haven't gotten a whole lot so far," he said. "We have not released milestones, but what we have said is that we do get milestones for key regulatory hurdles and in this case, NDA filings and approval both trigger milestones."