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RESEARCH TRIANGLE PARK, N.C., Jan. 26, 2015 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc., (Nasdaq:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for BCX4161, a novel, orally administered, selective inhibitor of plasma kallikrein in advanced clinical development for the treatment of hereditary angioedema.
The Fast Track designation process of the FDA is designed to facilitate the development and expedite the review and approval of drugs intended to treat serious or life threatening conditions and that address unmet medical needs. A drug that receives Fast Track designation is usually eligible for more frequent written communication and meetings with the FDA to discuss the drug’s development plan and the collection of appropriate data supporting drug approval. Priority Review and Rolling Review may be granted, if relevant criteria are met. Rolling Review allows a drug company to submit completed sections of its New Drug Application (NDA) for review by FDA on an ongoing basis, rather than wait until the entire NDA is completed and then reviewed.
“We are very pleased to have been granted orphan drug and fast track status from the FDA, as well as recently receiving a positive opinion for orphan drug designation in Europe,” said Jon P. Stonehouse, President & Chief Executive Officer of BioCryst. “BCX4161 and our second generation molecules have the potential to significantly improve HAE patient treatment and their quality of life. We look forward to reporting results from OPuS-2 and sharing updates regarding BCX7353 and our other second generation HAE assets during 2015.”Read full article
LEIDEN, THE NETHERLANDS, RALEIGH, NC, 08 January 2015 – Pharming Group NV (EURONEXT: PHARM) and Salix Pharmaceuticals, Ltd. (NASDAQ:SLXP) today announced that the first patient was treated in their Phase 2 clinical study of Ruconest®, (C1 Esterase Inhibitor [Recombinant]) 50 IU/kg, for prophylaxis in patients with hereditary angioedema (HAE).
HAE patients deficient in C1 inhibitor and with a history of at least four attacks per month are being enrolled in the randomized, double-blind study, in which 30 patients will receive Ruconest® either once or twice weekly, or placebo in each of 3 treatment periods. With the crossover design, all patients will receive each of the dosing regimens. The study will evaluate the safety and efficacy of Ruconest® when used for prophylaxis of angioedema attacks in patients with HAE.
The study will be conducted at sites in Canada, Europe, Israel, and the United States. The trial is being coordinated by principal investigators, Dr. Marco Cicardi, Professor at the University of Milan, and Dr. Marc Riedl, Associate Professor and Clinical Director of the US HAEA Angioedema Center at the University of California, San Diego.
“We are pleased to have quickly initiated the treatment phase of this important study, and look forward to its completion later in 2015,” said Bruno Giannetti, MD PhD, Chief Operating Officer of Pharming.Read full article
RESEARCH TRIANGLE PARK, N.C., Dec. 18, 2014 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc., (Nasdaq:BCRX) today announced that it has dosed the first patient in OPuS-2 (Oral ProphylaxiS-2), a blinded, randomized, placebo-controlled clinical trial of orally-administered BCX4161 in patients with hereditary angioedema (HAE).
OPuS-2 is a 12-week, three-arm, parallel cohort design trial to evaluate the efficacy and safety of two doses of BCX4161, 300 mg and 500 mg, administered three-times daily compared with placebo. This trial, to be conducted in the U.S. and selected European countries, is expected to enroll approximately 100 HAE patients. The primary efficacy endpoint for the trial will be the mean angioedema attack rate for each BCX4161 dose group compared to placebo.
“The OPuS-2 trial will provide important information on the efficacy and safety of 12 weeks of oral BCX4161 for prevention of angioedema attacks in HAE patients. OPuS-2 builds on the positive efficacy, safety, tolerability, drug exposure and kallikrein inhibition results from the OPuS-1 4-week study,” said Dr. Marc Riedl, M.D., M.S., Associate Clinical Professor at the University of California-San Diego School of Medicine, Clinical Director of the U.S. HAEA Angioedema Center and OPuS-2 Principal Investigator. “OPuS-2 has been designed as an adequate and well-controlled study and represents an important next step toward reaching our goal of improving HAE patients’ lives using oral kallikrein inhibitors,” added Dr. William P. Sheridan, Chief Medical Officer at BioCryst.
In May 2014, BioCryst announced positive results from the OPuS-1 (Oral ProphylaxiS-1) proof of concept Phase 2a clinical trial of orally-administered BCX4161 in patients with HAE. The trial met the primary efficacy endpoint, several secondary endpoints and all other objectives established for the trial. 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.Read full article
RALEIGH, N.C. & LEIDEN, THE NETHERLANDS–(BUSINESS WIRE)–Salix Pharmaceuticals, Ltd. (NASDAQ:SLXP) and Pharming Group NV (EURONEXT: PHARM) today announced the launch of RUCONEST® (C1 Esterase Inhibitor [Recombinant]) 50 IU/kg in the United States for the treatment of acute angioedema attacks in adult and adolescent patients with hereditary angioedema (HAE). Effectiveness in clinical studies was not established in HAE patients with laryngeal attacks. Today’s announcement follows the July approval of the drug by the Food and Drug Administration.
“We’re excited to offer the only recombinant C1 esterase inhibitor therapy for HAE in the United States,” said Carolyn J. Logan, President and Chief Executive Officer of Salix. “RUCONEST treats the root cause of HAE attacks, which has been shown to raise C1 inhibitor levels to within the normal range. RUCONEST can be self-administered by appropriately trained patients and is effective at stopping most HAE attacks in one dose.”
RUCONEST, a recombinant C1 esterase inhibitor, can be administered by the patient after receiving training by a healthcare provider. RUCONEST is over 98 percent pure, and because it is not made from human plasma, it does not carry any known risk of passing on viruses that can be found in human blood.
HAE is a rare genetic condition that affects between 1 in 10,000 to 1 in 50,000 people. It causes episodes of swelling in various parts of the body, including the hands, feet, abdomen and face. Patients with abdominal swelling often experience severe pain, nausea and vomiting. HAE attacks stem from a deficiency of the C1 inhibitor protein in the blood. The disease is often misdiagnosed, 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.
“HAE is an especially challenging disease for patients to manage,” 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. “If left untreated, patients can experience attacks that are incredibly painful and, because of its unpredictability, HAE interferes with daily life. We’re pleased HAE patients now have another treatment option available to them.”Read full article
apid Trial Progress Enables Additional Cohorts to be Studied
BURLINGTON, Mass.–(BUSINESS WIRE)– Dyax Corp. (NASDAQ: DYAX) today announced the expansion of its ongoing Phase 1b clinical trial evaluating DX-2930 to include additional patients and dosing cohorts. Dyax is developing DX-2930, an investigational fully human monoclonal antibody inhibitor of plasma kallikrein, as a subcutaneous injection for prevention of hereditary angioedema (HAE) attacks.
The ongoing Phase 1b clinical trial is a multi-center, randomized, double-blind, placebo-controlled, multiple ascending dose study designed to assess the safety, tolerability and pharmacokinetics of DX-2930 in HAE patients. As of October 31, 2014, 21 subjects were enrolled and completed dosing in three ascending dose cohorts (30 mg, 100 mg and 300 mg) of DX-2930 or placebo. Subjects in each cohort were randomized to active drug or placebo in a 2:1 ratio. Each study subject received two doses of study drug or placebo separated by 14 days and will undergo 15 weeks of follow-up after the second dose.
In light of faster than anticipated enrollment rates, Dyax has decided to take the opportunity to further characterize DX-2930 by adding two additional cohorts. Subjects will continue to be randomized to active drug or placebo in a 2:1 ratio and the dosing regimen will remain unchanged. Active drug-treated subjects in the fourth cohort will receive 400 mg of DX-2930. The dose level of the fifth cohort will be decided based upon a review of interim data.
Addition of the new dosing cohorts is in accordance with the provisions included in the original protocol which allows enrollment into additional dose groups and/or additional patients at any dose group. Total enrollment can be increased to as many as 36 patients and data from additional patients will be used to expand the DX-2930 safety database and provide potentially informative pharmacodynamic data.
“Strong investigator support for the Phase 1b trial has allowed the study to progress rapidly and, at this time, the first three dosing cohorts are fully enrolled,” said Burt Adelman M.D., Executive Vice President of Research and Development and Chief Medical Officer at Dyax. “These additional cohorts will provide safety, pharmacokinetic and pharmacodynamic data which will further guide future clinical development of DX-2930. Importantly, we remain on track to report data from this study in early 2015, followed by a Phase 2 study which is also currently planned to begin in 2015.”
“Developing a prophylactic treatment for HAE patients is a key value driver for Dyax and we are encouraged by the rapid progress we are seeing with this trial,” said Gustav Christensen, President and Chief Executive Officer of Dyax. “We believe DX-2930’s unique product profile positions it well as a potential preventative treatment for HAE attacks.”Read full article