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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.Read full article
RESEARCH TRIANGLE PARK, N.C.–(BUSINESS WIRE)– BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that it has dosed the first subject in OPuS-1 (Oral ProphylaxiS-1), a Phase 2a proof of concept clinical trial of orally-administered BCX4161 in patients with hereditary angioedema (HAE).
The OPuS-1 trial will test 400 mg of BCX4161 administered three times daily for 28 days in up to 25 HAE patients who have a high frequency of attacks (≥ 1 per week), in a randomized, placebo-controlled, two-period cross-over design. The main goals for the OPuS-1 trial are to estimate BCX4161′s degree of efficacy in reducing the frequency of angioedema attacks, and to evaluate the safety and tolerability of 28 days of BCX4161 treatment.
“We look forward to obtaining the results of OPuS-1, the first trial of an oral kallikrein inhibitor in HAE patients. The safety, tolerability, drug exposure and kallikrein inhibition achieved in the Phase 1 healthy volunteer trial of BCX4161 strongly support its further evaluation in HAE patients,” said Dr. William P. Sheridan, Chief Medical Officer at BioCryst. “OPuS-1 is another step toward our goal of improving HAE patients’ lives by dramatically changing the management of this disease.”
The Phase 2a OPuS-1 trial is being conducted at up to four centers in Germany and will evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of BCX4161. Each subject will receive BCX4161 and placebo in two separate 28-day periods, with the order of therapy randomized (placebo followed by BCX4161 or BCX4161 followed by placebo). The primary efficacy endpoint for the OPuS-1 trial is the mean attack frequency in each period. Other efficacy measures include average severity of attacks, the number of attack-free days and quality of life.
As a part of BioCryst’s strategy to become a leader in the treatment of HAE, its scientists are finalizing the evaluation of multiple potent and specific second generation oral kallikrein inhibitors, with the goal of developing compounds with potential for once daily dosing. One to three candidates are expected to enter preclinical development before the end of 2013.Read full article
The treatment of hereditary angioedema (HAE) has undergone dramatic changes as newer medicines have become available in recent years. Optimal care of these patients requires a comprehensive management plan. Although several consensus papers have been published concerning the diagnosis and treatment of HAE, guidelines for a comprehensive management plan have not been developed.
To develop state-of-the-art recommendations for the treatment and management of HAE due to C1 inhibitor (C1INH) deficiency in the United States.
Members of the US Hereditary Angioedema Association Medical Advisory Board began by reviewing the literature concerning treatment of HAE. Preliminary recommendations were developed based on the literature review, discussions in a face-to-face meeting, and refinements in a series of drafts. Final recommendations reflect the unanimous consensus of the medical advisory board and the US Hereditary Angioedema Association leadership.
Recommendations are provided regarding a comprehensive care plan for HAE, including the following: development of an overall management plan, treatment of angioedema attacks, prophylactic treatment, and patient monitoring.Read full article
BURLINGTON, Mass.–(BUSINESS WIRE)– Dyax Corp. (NASDAQ: DYAX) today announced dosing of the first subject in a Phase 1 clinical study evaluating the safety and tolerability of single subcutaneous administration of DX-2930, its fully human monoclonal antibody inhibitor of plasma kallikrein. Dyax, a biopharmaceutical company focused on hereditary angioedema (HAE) and other plasma kallikrein-mediated disorders, is developing DX-2930 as a subcutaneous injection for prevention of HAE attacks. DX-2930 was discovered using Dyax’s proprietary phage display technology platform.
“There is significant need for a highly effective, safe and well tolerated prophylactic agent to treat HAE,” said Burt Adelman M.D., Executive Vice President and Chief Medical Officer at Dyax. “DX-2930 is a highly potent, long-acting, fully human monoclonal antibody that binds specifically to active plasma kallikrein. We look forward to completing this study and moving forward rapidly with this important clinical program.”
This Phase 1, single-center, randomized, double-blind, placebo-controlled study is designed to assess the safety and tolerability and to characterize the pharmacokinetics (PK) of single, subcutaneous administrations of DX-2930 in healthy subjects. Approximately 32 subjects will be enrolled into four ascending dose cohorts (n=8 per cohort) of DX-2930 or placebo. The study will be conducted at the clinical trials unit of Vince & Associates Clinical Research, a recognized “Center of Research Excellence”, located in Overland Park, Kansas.
“The successful dosing of the first subject in this Phase 1 clinical study is an important milestone for the DX-2930 development program,” said Gustav Christensen, President and CEO of Dyax. “We believe our knowledge of the plasma kallikrein pathway provides us with unique insight and a significant advantage in developing and commercializing this product candidate.”
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.Read full article
The US Hereditary Angioedema Association (HAEA) leads a nationwide advocacy organization that focuses on increasing HAE awareness and education; empowering patient access to optimal therapy; establishing a presence in the health policy legislative and regulatory environments; and fostering patient-driven clinical and translational research that includes searching for a cure. A growing organization of over
4300 members, the HAEA is governed by a proactive board of directors and works closely with a m
edical advisory board of world-class HAE physicians and researchers. Notwithstanding the dramatic
expansion in the scope of its activities over the past 13 years, the HAEA remains grounded in the fundamental goal espoused since its founding—helping patients with HAE achieve lifelong health by providing unbiased information and highly personalized patient services. The HAEA’s core activities are
centered on (1) a web site that serves as the repository for a wealth of authoritative and up-to-date information on HAE diagnosis and treatment for patients and physicians, and (2) a caring and compassionate HAEA Patient Services Team that is available around the clock to help patients with HAE-related questions, problems, or emergencies. The HAEA’s activities in 2012 and the initiati
ves it has planned for 2013 reveal that a diligent and highly motivated patient organization that partners with interested physicians and works well with industry can be a powerful force in driving
programs, activities, and research that improve patients’ quality of life.