Dyax Corp. and Genzyme Corporation announced final results from their EDEMA1 (Evaluating DX-88's Effects on Mitigating Angioedema) trial, the first successfully completed placebo controlled trial for the treatment of hereditary angioedema (HAE) under a US investigational new drug (IND) application.

The EDEMA1 results demonstrate that DX-88 achieved statistical significance with respect to the primary clinical endpoint of the trial; DX-88 was safe and well tolerated; and that DX-88 was effective at treating all types of HAE attacks, including potentially fatal laryngeal attacks. An oral presentation of the final results was made by Dr. Lynda Schneider, director of the Allergy Programme at Children's Hospital Boston and a lead clinical investigator for DX-88, at the Annual Meeting of the American College of Allergy, Asthma and Immunology (ACAAI).

The EDEMA1 trial was a 48-patient, phase II, double-blind placebo-controlled, dose escalating clinical trial designed to evaluate the safety and efficacy of Dyax's recombinant small protein DX-88 for the treatment of hereditary angioedema (HAE).

HAE is a rare, debilitating and life-threatening acute inflammatory condition characterized by episodes of pain and swelling. There is no approved treatment for HAE in the United States.

"The results from this study are very encouraging and demonstrate the potential of DX-88 to rapidly alleviate the symptoms of HAE attacks," Dr. Schneider commented adding, "This study represents an important advance in the understanding and management of hereditary angioedema, and I'm very excited to be working with Dyax and Genzyme on our mutual mission to bring forward a treatment for patients who are living today with the uncertainty and fear of the next attack, which could at any time turn fatal."

"We are extremely pleased with the positive EDEMA1 results, and will continue to work with the FDA on defining the shortest route to approval of DX-88 for patients suffering with HAE," stated Henry E. Blair, chairman, president and CEO of Dyax Corp. "The data presented today provide validation of the positive effect of DX-88 in HAE patients. The results represent a considerable accomplishment for Dyax and Genzyme, and are a testament to the potential benefit of targeted small proteins as novel therapeutics," he added.

DX-88 is a recombinant small protein that inhibits kallikrein in vitro with very high affinity (40 pM Ki) and unlike C1-Inh, does not inhibit any of the other serine proteases against which it was tested. Kallikrein may be the most relevant target in HAE, as it is a key enzyme in the inflammatory cascade, in which it liberates bradykinin, the intermediary of pain and swelling associated with HAE. The compound was discovered at Dyax Corp., and is being developed for the treatment of HAE in a joint venture between Dyax Corp. and Genzyme Corporation.