Regeneron Files BLA For IL-1 Trap Rilonacept To Treat Rare Inflammatory Conditions
This article was originally published in The Pink Sheet Daily
Executive Summary
Firm plans to pursue additional indications including gout, anemia and other inflammatory diseases for rilonacept.
|
Regeneron has completed a rolling BLA submission and requested priority review for its interleukin-1 trap rilonacept for long-term treatment of cryopyrin-associated periodic syndromes, the firm announced June 8. CAPS is a spectrum of rare inherited inflammatory conditions, including familial cold autoinflammatory syndrome and Muckle-Wells Syndrome. A key feature of the conditions is that exposure to mild degrees of cold temperature can provoke an inflammatory episode that occurs within hours. CAPS is caused by a range of mutations in the gene CIAS1 that encodes the protein cryopyrin, which causes excess release of IL-1 and an inflammatory response. Rilonacept binds to IL-1, allowing the excess to be excreted by the body. "There are no medicines approved for patients suffering from CAPS," Regeneron Chief Executive Officer Leonard Schleifer said in a statement. Rilonacept has fast track status and orphan designation from FDA. Results of two sequential Phase III studies submitted in support of the BLA showed the compound produced "rapid, profound and lasting reductions of signs and symptoms" with no drug-related serious adverse events, according to an abstract presented at the American Academy of Allergy, Asthma and Immunology annual meeting Feb. 27 in San Diego. Both trials met the primary efficacy endpoint of change in disease activity scores for rash, fever/chills, joint pain, eye redness/pain and fatigue. The first randomized, double-blind, placebo-controlled trial involved 47 subjects with CAPS injected weekly for 6 weeks with either 160 mg of rilonacept or placebo. Those subjects receiving rilonacept had an 85 percent reduction in mean symptom score compared with a 13 percent reduction for those patients on placebo. In a 9-week randomized withdrawal comparison, 45 patients who were switched to placebo had a five-fold increase in mean symptom score, compared to no significant change in symptoms for those remaining on rilonacept. Results of a 24-week open-label extension of the study were expected to be submitted with the BLA, according to the company. Regeneron plans to pursue additional indications for rilonacept in gout, anemia and other inflammatory diseases, the company said at its annual shareholder meeting, also held June 8 at the company's headquarters in Tarrytown, N.Y. A 10-patient pilot study is underway in gout and the firm plans to begin a larger Phase II placebo-controlled gout study later this year. Earlier this year, Regeneron signed two nonexclusive development license deals, with Astellas and AstraZeneca, for VelocImmune , its proprietary technology for discovering fully human monoclonal antibodies (1 (Also see "Astellas Signs License For Regeneron’s VelocImmune To Bolster Antibody Platform" - Pink Sheet, 30 Mar, 2007.)). Regeneron also is co-developing with Bayer a vascular endothelial growth factor trap for eye diseases including wet age-related macular degeneration and diabetic macular edema (2 (Also see "Bayer Nails Down Deal To Share Ex-U.S. Rights For Wet AMD Treatment" - Pink Sheet, 19 Oct, 2006.)). -Pamela Taulbee ([email protected]) |