BURLINGTON, Mass.--(BUSINESS WIRE)--Dyax Corp. (NASDAQ:DYAX) today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending DX-2930 for designation as an orphan medicinal product for the treatment of hereditary angioedema (HAE). Dyax is developing DX-2930, an investigational fully human monoclonal antibody inhibitor of plasma kallikrein (pKal), as a subcutaneous injection for prevention of HAE attacks.
“There is a significant unmet medical need for a prophylactic treatment option for HAE, a potentially life-threatening condition,” said Burt Adelman, M.D., Executive Vice President of Research and Development and Chief Medical Officer at Dyax. “Our regulatory strategy supports our global development plan for DX-2930 and our goal for providing an improved therapy for patients with HAE around the world. We look forward to initiating a Phase 3 clinical trial for DX-2930 for HAE prophylaxis by year-end 2015.”
Under the EMA guidelines, the COMP adopts an opinion on the granting of orphan drug designation, after which the opinion is submitted to the European Commission (EC) for the endorsement of the opinion. Orphan drug designation by the EC provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the EU, and where no satisfactory treatment exists, or where a treatment exists, a new treatment may provide a significant benefit to patients affected by the condition. Additionally, this designation provides up to 10 years of market exclusivity if the product candidate is approved for marketing in the European Union and the orphan designation is maintained. Orphan status also permits EMA assistance in optimizing the candidate’s clinical development through participation in clinical trial design and preparation of the product marketing application. Orphan Medicinal Product designation does not change the standards for approval.
DX-2930 has previously been granted Orphan Drug, Fast Track and Breakthrough Therapy designations from the U.S. Food and Drug Administration (FDA) for the prevention of attacks of HAE.
About DX-2930
DX-2930 is a novel, fully human monoclonal antibody inhibitor of pKal which is currently being developed as a subcutaneous injection for the prevention of HAE attacks. Uncontrolled pKal activity leads to excessive generation of bradykinin, a vasodilator thought to be responsible for the localized swelling, inflammation and pain characteristically associated with HAE.
About 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.
About Dyax
Dyax is a biopharmaceutical company focused on the development and commercialization of novel biotherapeutics for unmet medical needs. The Company is developing DX-2930, a fully human monoclonal antibody, for the prevention of HAE attacks. In March 2015, the Company reported positive safety, pharmacokinetic, biomarker and proof-of-concept efficacy results from its Phase 1b clinical trial of DX-2930 in HAE patients. Additionally, Dyax markets KALBITOR® (ecallantide) for the treatment of acute attacks of HAE in patients 12 years of age and older.
Both DX-2930 and KALBITOR were identified using Dyax's proprietary phage display technology. Dyax has broadly licensed this technology under its Licensing and Funded Research Portfolio (LFRP). The current portfolio includes one FDA approved product, Eli Lilly and Company’s CYRAMZA® (ramucirumab), for which Dyax receives royalties, and multiple product candidates in various stages of clinical development for which the Company is eligible to receive future milestones and royalties.
For additional information about Dyax, please visit www.dyax.com.
For additional information about KALBITOR, including full prescribing information, please visit www.KALBITOR.com.
Disclaimer
The press release contains forward-looking statements, including statements regarding the prospects for therapeutic benefits and treatment advantages of an investigational product, DX-2930, being developed for HAE. 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 competes. The statements contained in this press release are not guarantees of future performance and involve certain risks, uncertainties and assumptions that are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed in such forward-looking statements. There are many factors that could cause actual results to differ materially from those in these forward-looking statements. These factors include the following: the results from our Phase 1b study may not be predictive of the results or success of future clinical trials that will be required to permit application for regulatory approval of DX-2930 in the U.S. and Europe; even if DX-2930 progresses through clinical trials and gains regulatory approval, it may not gain market acceptance; others may develop technologies or products superior to DX-2930 or that reach the market before DX-2930; Dyax is dependent on the expertise, effort, priorities and contractual obligations of third parties in the manufacture, quality control, storage and clinical development of DX-2930; the costs of prosecuting, maintaining, defending and enforcing our patents and other intellectual property rights; the overall condition of the financial markets; and a variety of other risks common to our industry; changing requirements and costs associated with Dyax's planned research and development activities; competition from new and existing treatments for HAE; the uncertainty of patent and intellectual property protection; and other risk factors described or referred to in 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.
Dyax, the Dyax logo and KALBITOR are registered trademarks of Dyax Corp.
CYRAMZA is a trademark owned by or licensed to Eli Lilly and Company, its subsidiaries, or affiliates.
