Aastrom Biosciences, Inc. (NASDAQ:ASTM) announced that the final analysis of data from its RESTORE-CLI trial showed that the trial met its primary safety and efficacy endpoints, demonstrating a statistically significant improvement in the time to first occurrence of treatment failure at 12 months.
Shares were up 4.9% at $3.19 after hours on the news despite the fact that, like the rest of the markets, there was selling pressure during the regular session. ASTM has nearly doubled in value since we began covering it, yet we expect shares to continue rising, given the stock's multiple upcoming catalysts.
In addition, some believe the potential for Aastrom to partner ixmyelocel-T -- either in the U.S., outside the U.S., or both -- is significant. Also, Aastrom Biosciences has two clinical programs ongoing, one for vascular applications in critical limb ischemia, and another for cardiac applications in dilated cardiomyopathy, the leading cause of heart transplantation.
The RESTORE-CLI clinical trial assessed the safety and ability of ixmyelocel-T, Aastrom's expanded, bone marrow-derived cellular therapy, to restore damaged tissue in a lower extremity affected by critical limb ischemia (CLI) -- considered the end-stage form of peripheral artery disease and the result of a chronic lack of blood flow to the lower extremities resulting in a variety of debilitating conditions, including severe rest pain, non-healing wounds, and gangrene.
Said ASTM president/CEO Tim Mayleben:
We are extremely pleased to report that the RESTORE-CLI trial met its primary safety and efficacy endpoints, providing substantial confirmation that ixmyelocel-T could represent an historic advance in the treatment of CLI for hundreds of thousands of patients who currently have no good treatment options available. I believe Aastrom is well positioned to advance this promising therapy through Phase 3 clinical testing and on to regulatory review.
"Given the limited treatment options available in CLI, the results of this important Phase 2b study are encouraging for both patients and vascular physicians," said William R. Hiatt, MD, Novartis Foundation and endowed professor for cardiovascular research at the University of Colorado School of Medicine and president of CPC Clinical Research, which has been chosen to collaborate with Aastrom on the upcoming Phase 3 clinical studies for ixmyelocel-T. "I look forward to working with Aastrom on what I believe is a very well-designed Phase 3 clinical development program for ixmyelocel-T in CLI, which also represents a translational step in the development of cellular therapies to treat human disease," Hiatt added.
In the clinical trial, the assessment of TTF included four components: Major amputation of the treated leg, all-cause mortality, doubling of wound size from baseline, and de novo gangrene. All of these components together comprised the primary composite endpoint for this trial. CLI patients with tissue loss are five times more likely to experience amputation. As a result, tissue loss represents a widely accepted predictor of amputation risk in CLI patients.
The double-blind, placebo-controlled RESTORE-CLI clinical trial was the largest, fully controlled cellular therapy clinical trial ever conducted in the space; it randomized 86 men and women between the ages of 18 and 90 at 20 medical centers throughout the U.S. with a diagnosis of CLI and no previous major amputations. CLI was defined as persistent, recurring ischemic pain for at least two weeks, or ulcerations (grade 4 or 5 on the Wagner scale) or gangrene of the foot or toe. Participants in the clinical trial were randomized 2:1 to receive intramuscular injections of ixmyelocel-T or an electrolyte solution into the affected limb. Patients were followed for 12 months after treatment and otherwise received normal standard of care.
Aastrom announced plans to initiate a Phase 3 CLI clinical development program for ixmyelocel-T under special protocol assessments (SPA) and Fast Track designation by the FDA. Aastrom has been actively working with the FDA to finalize the special protocol assessment for the no option SPA and expects to conclude those discussions in the next several weeks.
Needham & Company recently issued a "Buy" rating with a $6 price target on the stock based in part on the great need for ASTM's treatment options. Jason Napodano, CFA of Zacks Equity Research feels a $4 price target is more in tune with his current valuation of the company.
CLI is the most advanced form of lower extremity peripheral arterial disease. There are about 1 million people suffering from CLI, of which 400,000 patients have no treatment option other than amputation, leading to ~100,000 major amputations per year in the U.S. CLI is associated with significant high risk of cardiovascular morbidity and mortality. No-option CLI patients are not suitable for revascularization due to non-reconstructable arterial occlusive disease, necrosis of the significant areas, a terminal illness, or very limited life expectancy. The prognosis for these patients is very poor, as ~40% of patients die within two years of the first major amputation and up to 70% of patients die within four years. There is significant unmet medical need for no-option CLI patients.
It is believed the treatment has a very good chance to reach the market by 2015 and that it will be well acceptedby patients and physicians.
Aastrom plans to present the full data from the RESTORE-CLI trial at an appropriate medical meeting later this year.