BURLINGTON, Mass. -- (BUSINESS WIRE) -- Dyax Corp. (NASDAQ:DYAX) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its drug candidate DX-2930, its fully human monoclonal antibody inhibitor of plasma kallikrein, for use in the treatment of hereditary angioedema (HAE).
Dyax is developing DX-2930 to be a long-acting, prophylactic agent that prevents HAE attacks. Development plans include a dosage formulation that will permit infrequent self-administration by small volume, subcutaneous injection. DX-2930 is currently being studied in a placebo-controlled, dose-escalation Phase 1 trial in normal individuals. Results from this study are expected in the first quarter of 2014.
“Through our experience in the development and commercialization of KALBITOR® (ecallantide), we have gained a deep understanding of the HAE market and patient needs,” said Gustav Christensen, President and Chief Executive Officer of Dyax. “There is still a significant unmet medical need within the HAE community which we plan to address with DX-2930. Orphan drug designation is an important element of our development strategy for DX-2930 as we work to further improve the health and quality of life for individuals suffering from this painful and often debilitating condition.”
Orphan drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides FDA assistance in clinical trial design, an exemption from FDA user fees and eligibility for a seven-year period of market exclusivity in the U.S. after product approval.
Discovered using Dyax’s proprietary phage display technology platform, DX-2930 is a novel, fully human monoclonal antibody inhibitor of plasma kallikrein. Uncontrolled plasma kallikrein activity leads to excessive generation of bradykinin, a vasodilator thought to be responsible for the localized swelling, inflammation and pain characteristically associated with HAE. Preclinical studies suggest that DX-2930 will have a long half-life in humans, offering the potential for a long-acting and sustained therapeutic effect with less frequent dosing. Dyax is currently developing DX-2930 as a subcutaneous injection for the prevention of HAE attacks and the candidate is currently in a Phase 1 clinical trial.
About Hereditary Angioedema (HAE)
HAE is a rare acute inflammatory condition characterized by episodes of severe, often painful swelling affecting the extremities, gastrointestinal tract, genitalia, and larynx. HAE is caused by low or dysfunctional levels of C1 esterase inhibitor (C1-INH), a naturally occurring molecule that inhibits plasma kallikrein, a key mediator of inflammation, and other serine proteases in the blood. HAE is estimated to affect up to 1 in 50,000 individuals. Learn more at www.HAEHope.com.
Dyax is a fully integrated biopharmaceutical company focused on the discovery, development and commercialization of novel biotherapeutics for unmet medical needs. The Company's key value drivers are the KALBITOR® business, DX-2930 and Dyax’s Licensing and Funded Research Program (LFRP).
For additional information about Dyax, please visit www.dyax.com.
This press release contains forward-looking statements, including statements regarding the prospects for the Phase 1 clinical trial for DX-2930. Statements that are not historical facts are based on Dyax’s current expectations, beliefs, assumptions, estimates, forecasts and projections about the industry and markets in which Dyax and its licensees compete. The statements contained in this release are not guarantees of future performance and involve certain risks, uncertainties and assumptions, which are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed in such forward-looking statements. Important factors which may affect future performance include the risks that: DX-2930 may not show sufficient therapeutic effect or an acceptable safety profile in clinical trials or could take longer to gain regulatory approval than Dyax expects or may never gain approval; others may develop products superior to KALBITOR or DX-2930; KALBITOR and/or DX-2930 may not gain market acceptance; Dyax is dependent on the expertise, effort, priorities and contractual obligations of third parties in the manufacture, marketing, sales and distribution of KALBITOR and DX-2930; and other risk factors described or referred to Item 1A, “Risk Factors” in Dyax’s most recent Annual Report on Form 10-K and other periodic reports filed with the Securities and Exchange Commission. Dyax cautions investors not to place undue reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this release, and Dyax undertakes no obligations to update or revise these statements, except as may be required by law.
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