Assistant Managing Editor

Shares of BioMS Medical Corp. fell 30.3 percent after its multiple sclerosis drug dirucotide failed to affect the relapse rate in patients with the relapsing-remitting form of the disease in a Phase II study, but the Canadian company said promising data regarding disease progression could point to dirucotide's potential success in ongoing pivotal studies in secondary progressive MS.

Results from the 15-month MINDSET-01 exploratory Phase II study, which enrolled 218 patients randomized to either drug or placebo, showed that dirucotide missed the primary endpoint, defined as annualized relapse rate, which removes the likelihood of a potential label claim for dirucotide in that setting, said Kevin Giese, president and CEO of the Edmonton, Alberta-based firm.

Though the news sent the company's shares (TSX:MS) dropping C$1.04 to close at C$2.39 (US$1.95) Friday, Giese told BioWorld Today that BioMS had "no expectation" of the drug succeeding in MS relapse. For that reason, there were no milestone payments linked to the study from dirucotide partner Eli Lilly and Co.

The two firms signed a partnership in late 2007, in which Indianapolis-based Lilly agreed to pay $87 million up front for rights to dirucotide, formerly MBP8298, with as much as $410 million in future milestones. (See BioWorld Today, Dec. 19, 2007.)

BioMS and Lilly plan to continue analyzing data from the trial, but Giese said the real value of the study was that it showed a "nice signal and met endpoints" related to disease progression, including endpoints measuring the change from baseline in the Expanded Disability Status Scale and the Multiple Sclerosis Functional Composite score, which evaluate disability and functional parameters.

BioMS has targeted disease progression as its first dirucotide program, so "we see these [MINDSET-01] data as highly supportive," Giese added, and "we think they bode well" for the company's two ongoing pivotal studies in secondary progressive MS, the first of which is expected to yield results this year.

The MAESTRO-01 study completed enrollment of 611 patients in Canada and Europe, and data are expected in the second half of this year. That study also features an open-label extension portion, and BioMS said about 95 percent of patients who have completed the treatment phase to date have elected to continue dirucotide therapy.

Separately, the 510-patient MAESTRO-03 study is ongoing in the U.S., with data expected in the second half of 2010, Giese said.

Both pivotal studies are evaluating time to progression of disease after 24 months of treatment as the primary endpoint.

In the MS space, "a great many people believe there are two components" to the disease, Giese said. One is the relapsing component, with involves inflammation, and the second is the progressive component, which involves an axonal loss mechanism.

So far, dirucotide has been shown to be "very selective and appropriate" for treating progression.

And since the recent Phase II trial showed that the drug can be safely administered to relapsing-remitting patients, that "may help to expand the potential market for dirucotide," he added. "We might be able to affect progression, at no matter what" stage of the disease.

Dirucotide is a synthetic peptide consisting of 17 amino acids with a sequence identical to that of human myelin basic protein and is designed to target patients with certain immune-response genes, specifically HLA types DR2 and DR4. BioMS has said that patients with those genes make up about 70 percent of the entire MS patient population.

Dirucotide is the company's only product.

BioMS, which reported net income of C$6.3 million (US$5.1 million), or C7 cents per share, for the third quarter of 2008, had cash and short-term investments totaling C$98.8 million as of Sept. 30.