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BURLINGTON, Mass.–(BUSINESS WIRE)– Dyax Corp. (NASDAQ: DYAX) today announced the online publication of scientific data for DX-2930, an investigational fully human monoclonal antibody inhibitor of plasma kallikrein (pKal), in two peer-reviewed journals. 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.
The first paper titled “A Phase 1 Study Investigating DX-2930 in Healthy Subjects” by Chyung et al. was published online in the Annals of Allergy, Asthma and Immunology. The paper describes the positive results from the first-in-human clinical study of DX-2930 which met all of its objectives of assessing safety, tolerability and pharmacokinetics of this investigational drug candidate. These data indicate that DX-2930 has a long half-life in humans and thus the potential for prophylactic inhibition of pKal activity for the therapeutic treatment of HAE. DX-2930 is now being evaluated in a Phase 1b study in patients with HAE. The complete publication can be accessed online here.
The second paper titled “Inhibition of Plasma Kallikrein by a Highly Specific, Active Site Blocking Antibody” by Kenniston et al. was published online in The Journal of Biological Chemistry. The paper describes the discovery and preclinical evaluation of DX-2930 as a long-acting inhibitor of pKal proteolytic activity. This paper is expected to appear in the September 2014 (Volume , Issue ) print issue of the journal. The complete publication can be accessed online here.
Dr. Burt Adelman, Executive Vice President of Research and Development and Chief Medical Officer, commented: “We are delighted to have two important, peer-reviewed papers published in these prestigious scientific journals. Publication of these papers confirms the significance of DX-2930 in the scientific community. These data, combined with data that will be obtained from the ongoing Phase 1b study, will inform and guide the future clinical development of DX-2930.”Read full article
RUCONEST® (C1 ESTERASE INHIBITOR [RECOMBINANT]) 50 IU/kg IS THE FIRST AND ONLY RECOMBINANT TREATMENT OPTION FOR ADULT AND ADOLESCENT PATIENTS SUFFERING FROM HEREDITARY ANGIOEDEMA.
LEIDEN, THE NETHERLANDS, RALEIGH (NC), July 17, 2014 – Pharming Group NV ( EURONEXT: PHARM) and Salix Pharmaceuticals, Ltd. (NASDAQ:SLXP), today announced that the Food and Drug Administration has approved RUCONEST® (C1 Esterase Inhibitor [Recombinant]) 50 IU/kg for the treatment of acute angioedema attacks in adult and adolescent patients with hereditary angioedema (HAE). Because of the limited number of patients with laryngeal attacks, effectiveness was not established in HAE patients with laryngeal attacks.
“We are pleased that RUCONEST® provides the HAE community with another FDA-approved option for treating painful and debilitating HAE attacks,” said Anthony Castaldo, President of the Hereditary Angioedema Association (US HAEA), a non-profit patient services and research organization with a membership of over 5,000 HAE patients in the United States.
RUCONEST® is a recombinant C1 esterase inhibitor that can be administered by the patient after receiving training by a healthcare provider. HAE attacks stem from a deficiency of the C1 inhibitor protein in the blood. HAE is a rare inherited genetic condition that is often not properly diagnosed until later in a patient’s life as the symptoms of an attack can mirror someone experiencing an allergic reaction. Severe, painful swelling can occur at any time, which means most people suffering from HAE deal with the constant fear of when their next attack might surface and how that might impair their lives and those around them.
“Results in the pivotal clinical trial demonstrate RUCONEST® is a safe and effective option for the treatment of acute hereditary angioedema attacks,” said Dr. Marc Riedl of the US HAEA Angioedema Center at the University of California – San Diego and primary investigator of the phase III study. “At the US HAEA Angioedema Center, we strive to better the lives of those suffering from hereditary angioedema and part of that is ensuring patients have access to advanced treatments that have been proven to work in clinical trials. RUCONEST is an important addition to those treatment options.”
Sijmen de Vries, CEO of Pharming, said: “The approval of RUCONEST® in the US is a very significant milestone for Pharming. For many years we have strived to make RUCONEST® – the first recombinant replacement therapy for C1Inhibitor deficiency – available to the HAE patient community in the US, because we were aware of the great value and benefit this product adds to patients’ lives. Today we are proud to have achieved this goal in the US.”
“RUCONEST® is a much needed treatment option for patients suffering from acute attacks of hereditary angioedema. Until now, there hasn’t been an FDA approved recombinant C1 esterase inhibitor option to treat symptoms of HAE,” said Carolyn J. Logan, President and Chief Executive Officer of Salix. “The unpredictability of HAE can make patients feel uncertain about when their next attack might strike, which is why it is important to have a medicine that can be administered by the patient that resolves an attack. Salix is proud to make RUCONEST® available.”
The FDA approval of the Biologics License Agreements (BLA) for RUCONEST® for treatment of acute angioedema attacks in patients with HAE is based on a randomized, double-blind, placebo-controlled, phase III trial (RCT) which included an open-label extension (OLE) phase and is supported by the results of two additional RCTs and two additional OLE studies. The pivotal RCT and OLE studies analyzed the results from 44 subjects who experienced 170 HAE attacks. The primary efficacy endpoint was the time to beginning of symptom relief, assessed using patient-reported responses to two questions about the change in overall severity of their HAE attack symptoms after the start of treatment. These were assessed at regular time points for each of the affected anatomical locations for up to 24 hours. To achieve the primary endpoint, a patient had to have a positive response to both questions along with persistence of improvement at the next assessment time (i.e., the same or better response).
A statistically significant difference in the time to beginning of symptom relief was observed in the intent-to-treat population (n=75) between RUCONEST and placebo (p=0.031, log-rank test); the median time to beginning of symptom relief was 90 minutes for RUCONEST patients (n=44) and 152 minutes for placebo patients (n=31).
RUCONEST® is manufactured by Pharming Group NV in the Netherlands. Salix has licensed exclusive rights from Pharming to commercialize RUCONEST® in North America and market RUCONEST® for the treatment of acute HAE attack symptoms.
Salix currently plans on making RUCONEST® accessible to patients later in 2014.Read full article
BURLINGTON, Mass.–(BUSINESS WIRE)– Dyax Corp. (NASDAQ: DYAX) today announced dosing of the first subject in its Phase 1b clinical study of DX-2930 in hereditary angioedema (HAE) patients. The study is designed to evaluate the safety, tolerability, and pharmacokinetics of multiple subcutaneous administrations of DX-2930, an investigational fully human monoclonal antibody inhibitor of plasma kallikrein. Dyax, a biopharmaceutical company focused on HAE and other plasma-kallikrein-mediated disorders, is developing DX-2930 as a subcutaneous injection for prevention of HAE attacks.
“The primary purpose of this Phase 1b study is to gain important safety and pharmacokinetic data in the target patient population,” said Burt Adelman M.D., Executive Vice President of Research and Development and Chief Medical Officer at Dyax. “The pharmacodynamic effects of DX-2930 on plasma kallikrein will also be evaluated. Data from this study will guide dosing decisions for future clinical development. We’ll also be utilizing our biomarker assay to assess the possible impact of DX-2930 on exploratory pharmacodynamic parameters such as basal plasma kallikrein activity. We anticipate reporting data from this trial early in 2015.”
This Phase 1b trial is a multi-center, randomized, double-blind, placebo-controlled, multiple ascending dose study to assess the safety, tolerability and pharmacokinetics of DX-2930 in HAE patients. Approximately 18 subjects will be enrolled into three ascending dose cohorts (30 mg, 100 mg and 300 mg) of DX-2930 or placebo. There will be 4 active drug-treated subjects and 2 placebo-treated subjects per cohort. Each subject will receive two treatments with study drug, separated by two weeks, and undergo approximately 15 weeks of follow-up after the second dose.
“Expanding our HAE product offerings is a priority for Dyax and we are executing on our clinical goals with DX-2930,” said Gustav Christensen, President and CEO of Dyax. “Data generated from the Phase 1b study will provide the basis for us to continue to advance DX-2930. We believe that, if approved, DX-2930 has the potential to be a game-changing therapy for HAE patients.”Read full article
RESEARCH TRIANGLE PARK, N.C., May 27, 2014 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc., (Nasdaq:BCRX) today announced preliminary results from its OPuS-1 (Oral ProphylaxiS-1) proof of concept Phase 2a clinical trial of orally-administered BCX4161 in patients with hereditary angioedema (HAE). The trial met the primary efficacy endpoint, several secondary endpoints and all other objectives established for the trial.
OPuS-1 evaluated 400 mg of BCX4161 administered three times a day for 28 days in HAE patients with a high attack frequency (≥ 1 per week), in a randomized, placebo-controlled, two-period cross-over design. The primary goals for the trial were to estimate the degree of efficacy of BCX4161 in reducing the frequency of angioedema attacks, and to evaluate the safety and tolerability of 28 days of BCX4161 treatment.
Twenty-four patients received study drug, and all completed the study. The primary efficacy endpoint for the trial was the by-subject difference in mean angioedema attack rate on BCX4161 compared to placebo. Treatment with BCX4161 demonstrated a statistically significant mean attack rate reduction of 0.45 attacks per week versus placebo, p < 0.001. The mean attack rate per week was 0.82 on BCX4161 treatment, compared to 1.27 on placebo.
Oral administration of BCX4161 was generally safe and well tolerated, with an adverse event profile similar to that observed for placebo. There was one serious adverse event reported, an abdominal HAE attack during the placebo period. Patient dosing compliance was 98 percent.
The mean number of attack-free days during each treatment period improved from 19 for placebo to 22 for BCX4161, p=0.008. Three subjects were attack-free during the BCX4161 period, compared to none during the placebo period. Quality of life was measured by the Angioedema Quality of Life questionnaire, AeQoL, and disease activity by the Angioedema Activity Score, AAS28. For BCX4161, the mean total AeQoL score improved by 8.4 units from baseline compared to 0.5 for placebo, p=0.004, and the AAS28 was 21.4 for BCX4161 compared to 28.8 for placebo, p=0.022.
Plasma drug concentrations and the degree of plasma kallikrein inhibition achieved after oral dosing with BCX4161 in OPuS-1 HAE patients were similar to those seen in healthy subjects in the Phase 1 trial. In OPuS-1, higher drug exposure was associated with a better clinical outcome.
“OPuS-1 represents a milestone study in establishing the proof of concept that prophylaxis with an oral kallikrein inhibitor can effectively reduce attacks for patients living with HAE,” said Marcus Maurer MD, Professor of Dermatology and Allergy, Charité-Universitätsmedizin, Berlin, and the principal investigator for the study. “Existing therapies for prophylaxis of attacks in patients with HAE require frequent i.v. infusions, or the use of oral androgens that have significant long term side effects. The OPuS-1 results open up the possibility of an exciting new treatment option for this challenging disease.”
“The efficacy and safety profile of BCX4161 seen in the OPuS-1 trial strongly support its continued development,” said Dr. William P. Sheridan, Chief Medical Officer at BioCryst. “We look forward to working with clinical investigators, the HAE community and regulatory authorities in advancing BCX4161 to the next stage and starting the OPuS-2, 12-week trial later this year.”Read full article
As a Diamond Sponsor of HAE Day on May 16, 2014, CSL Behring is proud to continue its partnership with HAEi, the International Patient Organization for C1 Inhibitor Deficiencies, and patient organizations throughout the world. HAE, or, hereditary angioedema, is a rare, potentially fatal swelling disorder caused by a deficiency of C1-INH, a type of protein.
HAE Day is a global event intended to support better care and an earlier and more accurate diagnosis for HAE patients. The day focuses on engaging and educating the general public and the medical community while raising funds for further national and international HAE initiatives. HAE Day awareness events strengthen the voice of patients and unite HAE patient organizations globally.
“HAE is a serious, sometimes life-threatening medical condition that appears in all age groups in all parts of the world,” said Paul R. Perreault, Chief Executive Officer of CSL Limited, parent company of CSL Behring. “Often, people who have undiagnosed HAE struggle with symptoms for years before receiving an appropriate diagnosis and effective treatment. Others go their entire lives not realizing their condition even has a name. Building broad public awareness of HAE is an important key to solving these issues for people. Therefore, CSL Behring is proud to continue our support of HAE Day at the international level, and hope our collaborative efforts can increase awareness and effective clinical management of this debilitating disorder.”
CSL Behring HAE Day activities will take place throughout the Americas, Europe and Asia. Activities planned for this year include:
In addition, CSL Behring is a Gold Sponsor of the international HAE conference in Washington, D.C. (May 15-18). As a part of its sponsorship, CSL Behring will provide conference attendees with information regarding the company and its HAE programs.
For more information about HAE Day 2014, please visit www.haeday.org.Read full article