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–Based on data from ZENITH-1, the company plans to begin the phase 3 trial of oral single dose 750 mg BCX7353 for the acute treatment of hereditary angioedema attacks ( HAE) in the summer of 2019-
-The data show an excellent clinical dose response in ZENITH-1, as predicted by the pK data.
– BCX7353 was well tolerated at all doses (250 mg, 500 mg, 750 mg) in ZENITH-1
-Data presented at the annual meeting of the American Academy of Allergy, Asthma & Immunology (AAAAI) –
RESEARCH TRIANGLE PARK, NC, February 23, 2019 – BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced today that additional baseline data from the ZENITH-1 Phase 2 trial, including new data from the 250 mg and 500 mg dose cohort trials. Data from the now comprehensive trial confirm previously reported results showing that a single oral dose of 750 mg of BCX7353 was well tolerated and superior to placebo (p <0.05) compared to the majority Efficacy evaluation criteria evaluated in patients clear response to the dose between the three dose levels.
Based on the results of ZENITH-1, the company plans to meet with the US Food and Drug Administration (FDA) in the second quarter and begin a phase 3 trial with the oral dose of 750 mg BCX7353. in the summer of 2019.
"The results of ZENITH-1, with onset of action in less than an hour, a single 24-hour duration of effect and effective dose response at all dose levels are very exciting." for patients who urgently need to take an oral dose. treatment for acute attacks, "said Dr. William Sheridan, BioCryst's Chief Medical Officer.
"Based on these excellent results, we plan to quickly move the oral BCX7353 to 750 mg in a Phase 3 trial to support approval in the US and the European Union," added Sheridan.
In a press release dated September 4, 2018, the company had previously reported data on efficacy and tolerability for the 750 mg dose cohort. With the 750 mg dose, compared to placebo, symptom improvement and visual badog scale (VAS) scores were observed as early as one hour after oral administration of BCX7353, and were maintained during 24 hours. After 24 hours, the use of standard drugs was reduced by 31.6% after BCX7353 compared to placebo (p = 0.0029) and no mild symptoms or symptoms were reported in 64.1% of seizures treated with BCX7353, compared with 32.3% of treated seizures. with placebo (p = 0.0038).
In the additional data reported today at the AAAAI annual meeting, a clear dose response was observed in the range of 250 mg to 750 mg. Among doses, BCX7353 was generally safe and well tolerated, with no significant differentiation in the placebo adverse event profile.
The previous press release of September 4, 2018, containing safety and efficacy data for the 750 mg dose cohort, as well as the poster containing the additional results presented today at the AAAAI Annual Meeting, including numbers highlighting the dosage response and tolerability among the three can be found in the investor relations section of the company's website at http://ir.biocryst.com/.
ZENITH-1 test design
ZENITH-1 was a double-blind, placebo-controlled, randomized, cross-dose, and mixed-dose trial of oral BCX7353 for the acute treatment of angioedema attacks in patients with HAE. A total of 63 patients were randomized and 58 received at least one dose of drug in the blind study: 11 in the 250 mg cohort, 14 in the 500 mg cohort and 33 in the 750 mg cohort .
ZENITH-1 was designed to be compatible with modern treatment guidelines for the administration of self-administered home medications as soon as possible after the onset of symptoms. The objectives of the trial were to identify the activity against significant clinical criteria that the company could use to construct a Phase 3 registration trial, to identify the dose (s) that the company could use and to evaluate safety and tolerability.
Adults with type I or II HAE self-administered a study dose of drug blinded during three seizures; two treated with the active drug and one with a placebo, in a randomized sequence. Subjects were asked to administer the drug to the study blinded within the hour following the onset of symptoms. Subjects were free to use the short-term medications prescribed and approved, but they were asked to wait for the drug at least four hours after the study, if possible. The patient completed the trial journal in order to gather information on symptoms, EVA scores and the use of COS-based drugs before taking the drug and 1, 2, 3, 4, 8 and 24 hours after the administration of the drug to the study. In cohorts of 250 mg, 500 mg and 750 mg, respectively, 21, 25 and 64 attacks were treated with BCX7353 and 11, 11 and 31 attacks with placebo.
About the BCX7353
Discovered by BioCryst, the BCX7353 is a new, once-daily, oral selective plasma kallikrein inhibitor currently in advanced clinical development for the prevention and treatment of angioedema attacks in patients with # 39; HAE. BCX7353 was generally safe and well tolerated in the APeX-1 Phase 2 clinical trial. BioCryst is currently conducting the APeX-2 Phase 3 clinical trial and the APeX-S long-term safety clinical trial, each evaluating two oral BCX7353 dosages once a day as a preventative therapy to reduce the frequency attacks in patients with HAE. BioCryst has also completed the ZENITH-1 clinical trial. ZENITH-1 was a Phase 2 clinical validation concept trial testing oral administration of BCX7353 for the treatment of acute angioedema attacks.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals is discovering new small molecule oral drugs that treat rare diseases for which there are significant unmet medical needs and where an enzyme plays a key role in the biological pathway of the disease. BioCryst has developed several oral drug development programs for the treatment of rare diseases, including BCX7353, an oral plasma kallikrein inhibitor for the treatment of hereditary angioedema; a preclinical program with an oral ALK-2 inhibitor for the treatment of progressive bone fibrodysplasia and intravenous galidesivir, a broad-spectrum viral RNA polymerase inhibitor, as a potential treatment for Marburg virus disease and yellow fever under contracts with NIAID and HHS / BARDA. RAPIVAB® (peramivir injection), an influenza virus neuraminidase inhibitor administered intravenously for the treatment of influenza, is the first product approved by BioCryst. It has received regulatory approval in the United States, Canada, Australia, Japan, Taiwan, Korea and the European Union. Post-marketing commitments for RAPIVAB are underway. For more information, please visit the company's website at www.BioCryst.com.
Forward-looking statements
This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors that could cause BioCryst's actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views regarding future events and are based on badumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that may affect the forward-looking statements contained in this document include: current and future pre-clinical and clinical development of second-generation AHA drug candidates (including APeX-2, APeX-S, and APeX- J) may not give positive results, may be more expensive or may not go as fast as expected; that the FDA, EMA or other applicable regulatory agency may not provide regulatory approvals that may delay scheduled clinical trials or fail to obtain marketing authorization for product candidates. Please refer to the documents that BioCryst files periodically with the Securities and Exchange Commission, in particular the latest BioCryst annual report on Form 10-K, the quarterly reports on Form 10-Q and the current reports on Form 8-K, which identify all important factors. this could result in a significant difference between actual results and those contained in BioCryst's projections and forward-looking statements.
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Contact:
John Bluth
+1 919 859 7910
[email protected]
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