Genelabs Tech reaches agreement with FDA on special protocol assessment for phase III trial of Prestara in lupus

Genelabs Technologies has announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for a Phase III clinical trial of Prestara (prasterone) for treatment of systemic lupus erythematosus (lupus).

The SPA documents the FDA's agreement that the design and planned analyses of the study adequately address objectives in support of a New Drug Application (NDA) submission. The FDA has further indicated to Genelabs that a positive outcome to the proposed new Phase III study in addition to evidence of efficacy from previous trials of Prestara and an overall positive risk/benefit assessment would in principle meet FDA standards for NDA approval.

"We are delighted to have reached agreement with the FDA under an SPA that this phase III clinical trial, if successful, together with the totality of data contained in Genelabs' NDA for Prestara, can meet the FDA's standards for NDA approval," stated James A. D. Smith, Genelabs President and Chief Executive Officer. "FDA has earlier designated the Prestara NDA approvable and a previously completed phase III clinical trial of Prestara in lupus has been agreed by the FDA as being positive adequate and well-controlled. Now, having reached agreement with the FDA on an SPA for a new phase III trial, we have a clear regulatory pathway for Prestara in the U.S. Genelabs is actively seeking strategic partnering for project financing, which needs to be secured in order to initiate this new phase III Prestara clinical trial. We view the SPA agreement as an important factor in moving these activities forward."

The primary endpoint for this randomized, double-blind, placebo-controlled phase III clinical trial will be time to first severe flare based on the SELENA SLEDAI composite definition of severe lupus flare. The principal secondary endpoint will be time to first treatment intervention for moderate or severe lupus flare. The study will target enrollment of approximately 500 women with active systemic lupus erythematosus who are receiving standard of care medications such as glucocorticoids, cytotoxic and immunosuppressive agents. Patients will be randomized to receive either 200 mg daily Prestara or placebo for 12-months.

"Lupus is a very severe and debilitating chronic autoimmune disease for which current treatment is inadequate. The disease progression of lupus is characterized by episodic flares usually accompanied by significant medical interventions. The impact of lupus flares themselves, as well as the drugs currently used to control them, can result in permanent tissue and organ damage. As a consequence, delaying lupus flares and interventions is a very important goal in the management of these patients," stated Kenneth E. Schwartz, M.D., Genelabs Vice President of Medical Affairs. "We are pleased that the FDA shares this view, and has been willing to commit to the clinical trial protocol in the form of an SPA agreement and has further indicated that a successful outcome to the trial could yield broad potential claims derived from both primary and secondary measures."