Healthcare Industry News: Integrated BioPharma
News Release - August 5, 2010
Data from First Phase 3 Trial (EDEMA3(R)) for KALBITOR(R) (ecallantide) Published in The New England Journal of MedicineCAMBRIDGE, Mass.--(HSMN NewsFeed)--Dyax Corp. (NASDAQ: DYAX ) announced today that results from EDEMA3®, the Company’s first Phase 3 trial evaluating KALBITOR® (ecallantide) for the treatment of acute attacks of hereditary angioedema (HAE), were published in the August issue of The New England Journal of Medicine. KALBITOR, discovered and developed by Dyax, is available in the United States for the treatment of acute attacks of HAE in patients 16 years of age and older.
EDEMA3 was the first of two Phase 3 trials evaluating KALBITOR for the treatment of acute HAE attacks. Results showed a statistically significant improvement of HAE attack symptoms with KALBITOR treatment versus placebo.
“The KALBITOR clinical program, including EDEMA3 and EDEMA4®, supports the treatment benefit of KALBITOR for HAE acute attacks,” said H. Henry Li, MD, an author and Director of Immunology at the Institute for Asthma & Allergy in Wheaton, MD. “Moreover, the publication of these data will enhance understanding among the physician community about the value of KALBITOR’s unique mechanism of action, as a selective plasma kallikrein inhibitor, in resolving acute HAE symptoms.”
The overall safety profile of KALBITOR observed in EDEMA3 was similar to that of placebo. The most common adverse events occurring more often in KALBITOR-treated patients versus placebo were mild or moderate and included: headache, diarrhea, pyrexia and nasal congestion. Treatment-related adverse events and serious adverse events occurred at a similar proportion in both the KALBITOR- and placebo-treated groups.
EDEMA3 was a 72-patient, double-blind, placebo-controlled trial randomized 1:1 to receive a 30 mg subcutaneous dose of KALBITOR or placebo. Patients were observed for at least 4 hours after KALBITOR was administered. Symptoms were assessed every 15 minutes for the first two hours, every 30 minutes for the next two hours, and again at 24 hours.
EDEMA3 was the first study to assess treatment response with two HAE-specific patient-reported outcomes measures, which were developed and validated by Dyax. Prior to the EDEMA3 trial, there was no validated instrument to assess HAE symptom complexes. The Mean Symptom Complex Severity (MSCS) score provides a point-in-time measure of individual symptoms that accounts for symptom location and severity of the attack using a score from 0 to 3 (0=normal, 3=severe). The Treatment Outcome Score (TOS) provides a composite measure of treatment response on a scale of 100 to -100 (100=significant improvement, -100 significant worsening) and taking into account symptom severity at baseline.
The primary endpoint was the TOS at 4 hours after dosing with KALBITOR or placebo. The principal secondary endpoint was the change from baseline in MSCS at 4 hours. At 4 hours post treatment, statistically significant improvement of symptoms was demonstrated for KALBITOR versus placebo, as measured by both the median TOS (P=0.004) and the median change in MSCS score (P=0.01). Statistical significance in favor of KALBITOR treatment was also demonstrated at 24 hours for median TOS (P=0.007) and median change in MSCS score (P=0.04).
Hereditary angioedema (HAE) is a rare acute inflammatory condition characterized by episodes of severe, often painful swelling affecting the extremities, the gastrointestinal tract, the genitalia, and in the 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 1:10,000 to 1:50,000 individuals. Learn more at www.HAEHope.com.
About KALBITOR® (ecallantide)
KALBITOR is a plasma kallikrein inhibitor indicated for the treatment of acute attacks of hereditary angioedema (HAE) in patients 16 years of age and older. KALBITOR, which was discovered and developed by Dyax, is the first subcutaneous treatment available in the U.S. for treating acute HAE attacks.
Important KALBITOR Safety Information
Anaphylaxis has been reported after administration of KALBITOR. Because of the risk of anaphylaxis, KALBITOR should only be administered by a healthcare professional with appropriate medical support to manage anaphylaxis and hereditary angioedema. Healthcare professionals should be aware of the similarity of symptoms between hypersensitivity reactions and hereditary angioedema and patients should be monitored closely. KALBITOR should not be administered to patients with known clinical hypersensitivity to KALBITOR.
As part of product approval, Dyax has implemented a Risk Evaluation and Mitigation Strategy (REMS) program, consisting of a Medication Guide and communication plan. The goal of the REMS is to communicate the risk of anaphylaxis and the importance of distinguishing between a hypersensitivity reaction and HAE attack symptoms.
For more information about KALBITOR, including full prescribing information, visit www.KALBITOR.com.
Patients and healthcare providers can contact KALBITOR AccessSM to receive information and work with program staff to research patient insurance coverage for KALBITOR. KALBITOR Access is designed as a one-stop point of contact for information about KALBITOR. The program is staffed with dedicated insurance specialists and nurse case managers who will help coordinate patient treatment and access to KALBITOR. Patients and healthcare providers can call 1-888-4KALBITOR (1-888-452-5248) for information and to utilize these services or visit www.KALBITOR.com.
Dyax is a fully Integrated BioPharmaceutical company focused on discovering, developing and commercializing novel biotherapeutics for unmet medical needs. Dyax utilizes its proprietary drug discovery technology to identify antibody, small protein and peptide compounds for clinical development. Our lead product, DX-88, has been approved under the brand name KALBITOR® (ecallantide) in the United States for the treatment of acute attacks of hereditary angioedema in patients 16 years of age and older.
In June 2010, Dyax announced a strategic partnership with Sigma-Tau to develop and commercialize subcutaneous DX-88 for the treatment of hereditary angioedema (HAE) and other therapeutic indications throughout Europe, North Africa, Middle East and Russia. Dyax and Sigma-Tau will work together with the European Medicines Agency as they review our Marketing Authorization Application for potential approval to market DX-88 in the European Union for HAE.
DX-88 is being investigated for its therapeutic potential in other angioedema indications and, through a collaboration with Fovea Pharmaceuticals (a subsidiary of sanofi aventis), is in a Phase 1 trial for retinal vein occlusion-induced macular edema.
DX-88 and other compounds in Dyax’s pipeline were identified using its patented phage display technology, which rapidly selects compounds that bind with high affinity and specificity to therapeutic targets. Dyax leverages this technology broadly with over 70 revenue generating licenses and collaborations for therapeutic discovery, as well as in non-core areas such as affinity separations, diagnostic imaging, and research reagents. Dyax is headquartered in Cambridge, Massachusetts. For online information about Dyax Corp., please visit www.dyax.com.
This press release contains forward-looking statements, including statements regarding the prospects for therapeutic benefits and treatment advantages of KALBITOR 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 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 the prospects for therapeutic benefits and treatment advantages of KALBITOR for HAE include the risks that: others may develop technologies or products superior to KALBITOR or that are on the market before KALBITOR; KALBITOR 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 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.
Dyax, the Dyax logo, KALBITOR and HAEHope are registered trademarks and EDEMA3 and EDEMA4 are registered service marks of Dyax Corp. KALBITOR Access is a service mark of Dyax Corp.
1. Cicardi M, Levy, RJ, McNeil DL, et al. Ecallantide for the treatment of acute attacks of hereditary angioedema. NEJM. 2010. 363(6): 523-531.
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