Latest news with #RaffiTachdjian
Business Wire
22-05-2025
- Business
- Business Wire
Astria Therapeutics to Present at Upcoming Eastern Allergy Conference
BOSTON--(BUSINESS WIRE)-- Astria Therapeutics, Inc. (Nasdaq:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunologic diseases, today announced that it will present at the Eastern Allergy Conference (EAC) in Palm Beach, Florida on May 30, 2025. Raffi Tachdjian, M.D., Associate Clinical Professor of Medicine and Pediatrics in the Division of Allergy and Clinical Immunology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA), will present attack severity reduction data from the ALPHA-STAR Phase 1b/2 trial of navenibart in a presentation of poster number F40 titled, 'Treatment with Navenibart (STAR-0215) Reduces Attack Severity and Use of Rescue Medication in Patients with Hereditary Angioedema (HAE): Interim Results from the ALPHA-STAR Trial.' The presentation will take place on Friday, May 30 in a poster session beginning at 9:45am EST at The Breakers Palm Beach. About Astria Therapeutics: Astria Therapeutics is a biopharmaceutical company, and our mission is to bring life-changing therapies to patients and families affected by allergic and immunologic diseases. Our lead program, navenibart (STAR-0215), is a monoclonal antibody inhibitor of plasma kallikrein in clinical development for the treatment of hereditary angioedema. Our second program, STAR-0310, is an investigational monoclonal antibody OX40 antagonist in clinical development for the treatment of atopic dermatitis. Learn more about our company on our website, or follow us on Instagram @AstriaTx and on Facebook and LinkedIn.
Yahoo
16-05-2025
- Health
- Yahoo
BioCryst Presents New Real-world Evidence Showing Significant and Sustained Reductions in HAE Attack Rates in Adolescents and People with Severe HAE Following Initiation of ORLADEYO® (berotralstat)
RESEARCH TRIANGLE PARK, N.C., May 16, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new real-world evidence on the use of oral, once-daily ORLADEYO® (berotralstat) in adolescents and people with severe HAE showing significant and sustained reductions in HAE attack rates through 18 months of follow-up after beginning treatment with ORLADEYO in both patient populations. The real-world evidence was presented in two posters at the 2025 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR), which is being held in Montreal from May 13-16, 2025. 'The outcomes detailed in these posters show how ORLADEYO is making a difference for people living with HAE, in particular those with very severe disease and those who are adolescents. These two groups experienced far fewer attacks per month compared to baseline after starting ORLADEYO. These kinds of real-world results should give physicians as well as their HAE patients the additional confidence to improve control of their attacks,' said Dr. Raffi Tachdjian, associate clinical professor of medicine & pediatrics, division of allergy & clinical immunology, David Geffen School of Medicine, University of California Los Angeles. Significant and sustained reductions in attack rates after ORLADEYO initiation The results presented in two posters at ISPOR 2025 were from a retrospective pre-post study using outcomes collected from BioCryst's sole-source pharmacy from December 15, 2020, to January 8, 2024. The poster 'Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Patients with C1 Inhibitor Deficiency (Type I/II) and ≥8 Attacks/month' (#PCR182) detailed findings from 56 U.S. patients with HAE with C1-inhibitor deficiency (HAE-C1-INH) who received ORLADEYO. Patients experienced significantly lower HAE attack rates while on ORLADEYO in each 90-day follow-up period (1.24-1.90 attacks/month) compared to baseline (7.78-8.23 attacks/month). Patients experienced significantly fewer HAE attacks per month following ORLADEYO initiation during every 90-day follow-up period relative to baseline, including: 6.25 fewer attacks/month (95 percent confidence period (CI): [5.63, 6.87]; p<0.001) at 12 months (days 271-360) 6.43 fewer attacks/month (95 percent CI: [5.78, 7.09]; p<0.001) at 18 months (days 451-540) The poster 'Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Adolescents' (#PCR132) highlighted outcomes reported from 99 U.S. patients with HAE aged 12-17 years who received ORLADEYO. Patients had significantly lower HAE attack rates while on ORLADEYO during each 90-day follow-up period (0.36-0.76 attacks/month) compared to the mean baseline rate (2.07-2.30 attacks/month). Compared to baseline, adolescents experienced statistically significant and sustained reductions in HAE attack rates after ORLADEYO initiation during each 90-day follow-up period, including: 1.56 fewer attacks/month (95 percent CI: [0.89, 2.23]; p<0.001) at 12 months (days 271-360) 1.85 fewer attacks/month (95 percent CI: [1.12, 2.58]; p<0.001) at 18 months (days 451-540) 'We continue to generate evidence from real-world use of our oral, once-daily prophylactic therapy for HAE that supports its effectiveness in a wide range of people with HAE. Here, we show that ORLADEYO is having a positive impact on attack reduction for younger people and those with severe disease. These additional findings further underscore that ORLADEYO works well for many patients with HAE, regardless of their attack severity, age or other aspects,' said Dr. Donald S. Fong, chief medical officer of BioCryst. About ORLADEYO® (berotralstat)ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein. U.S. Indication and Important Safety Information INDICATIONORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. Limitations of useThe safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation. IMPORTANT SAFETY INFORMATIONAn increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent. The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C). Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John's wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO. ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO. The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established. There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production. To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or Please see full Prescribing Information. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance or achievements and statements relating to ORLADEYO performance and effectiveness. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions 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 could affect the forward-looking statements contained herein include: BioCryst's ability to successfully implement or maintain its commercialization plans for ORLADEYO; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; and BioCryst's ability to successfully manage its growth and compete effectively. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst's forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
10-02-2025
- Health
- Yahoo
KalVista Pharmaceuticals Presents New Sebetralstat Data at the Western Society of Allergy, Asthma & Immunology 2025 Annual Meeting
– Claims data show 40% of patients on long-term prophylaxis have gaps in prescription refills, leading to greater on-demand use and higher rate of LTP discontinuation- -Data from KONFIDENT-S show patients on LTP average 1.7 HAE attacks per month and symptom relief for treated attacks in median 1.3 hours - –Results confirm sebetralstat effective for on-demand treatment of attacks regardless of type of LTP therapy used, including an all-oral regimen– CAMBRIDGE, Mass. & SALISBURY, England, February 10, 2025--(BUSINESS WIRE)--KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced the presentation of novel data related to long-term prophylaxis and sebetralstat at the Western Society of Allergy, Asthma & Immunology (WSAAI) 2025 Annual Meeting taking place in Waimea, HI from February 9-13, 2025. Raffi Tachdjian, MD, MPH, Associate Clinical Professor of Medicine and Pediatrics in the Division of Allergy and Clinical Immunology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA), presented data on the Impact of Long-Term Prophylaxis (LTP) Adherence in Hereditary Angioedema Patients: Results of a Claims Database Analysis. According to a US commercial claims analysis, nearly 40% of HAE patients who initiated LTP had substantial refill gaps in claims over 12 months, with more than half of those discontinuing LTP. For patients with substantial refill gaps, which increase the likelihood of non-adherence, on-demand claims remained unchanged before and after one year of starting LTP. "Despite guidelines emphasizing regular assessment of patients using LTP to confirm efficacy and safety of their therapy, these data suggest many patients experience substantial lapses in refilling their LTP, which may reflect non-adherence," said Dr. Tachdjian. "We saw no decrease in on-demand claims for patients with LTP refill gaps. These findings are important as most HAE patients in the US now receive LTP, and a greater focus on monitoring appears warranted, as LTP effectiveness is not a given. These data may also help to explain why on-demand treatment volumes in HAE have remained steady despite the advent of several effective subcutaneous and oral LTP options since 2017." Marc A. Riedl, MD, Professor of Medicine and Clinical Director, US Hereditary Angioedema Association Center at the University of California, San Diego, presented data on the Safety and Effectiveness of Sebetralstat in Patients with Hereditary Angioedema Receiving Long-Term Prophylaxis: Interim Analysis from the KONFIDENT-S Open-Label Study. Participants receiving LTP treated 382 attacks with sebetralstat (mean 1.7 attacks per month), of which more than half involved the abdomen and/or larynx. Sebetralstat enabled early treatment (median 6 minutes) and early symptom relief (median 1.3 hours), and was well-tolerated, regardless of LTP mechanism of action or route of administration. "Treatment guidelines recommend that patients with HAE should consider treating all attacks regardless of LTP use and should treat attacks as early as possible," noted Dr. Riedl. "These interim results suggest that, if approved, sebetralstat could be a safe and effective oral on-demand treatment for breakthrough attacks, regardless of severity, location, or type of LTP used." Michael E. Manning, MD, allergist-immunologist at Allergy, Asthma and Immunology Associates, Ltd., Scottsdale, Arizona and Past President of WSAAI, presented Sebetralstat for the Treatment of HAE Attacks in Patients Receiving Berotralstat: Interim Analysis from the KONFIDENT-S Open-Label Study. Participants receiving berotralstat treated 178 attacks with sebetralstat (mean 1.8 attacks per month). Sebetralstat enabled early treatment (median 20 minutes), early symptom relief (median 1.3 hours), and was well-tolerated with no increase in gastrointestinal side effects. "In general, patients prefer oral treatments over injectables, underscoring the strong interest in oral options for HAE," said Dr. Manning. "These results demonstrate that sebetralstat, when used as an oral on-demand treatment for attacks occurring among patients receiving berotralstat for LTP, enabled rapid treatment and symptom improvement. If approved, sebetralstat could transform the treatment landscape for physicians and patients who prefer to manage HAE without needles." "Injectable on-demand treatments have side effects and logistical obstacles that hinder adequate disease control and drive an over-reliance on LTP," said Paul Audhya, MD, MBA, Chief Medical Officer of KalVista. "LTP does not, however, always yield the anticipated reductions in attacks, possibly due to adherence issues in a chronic lifelong setting. Sebetralstat has the potential to enable early treatment of attacks, thereby halting progression at an early stage and reducing morbidity, even among patients on LTP. By overcoming the barriers imposed by current injectable on-demand therapies, sebetralstat could shift the treatment paradigm and become the foundation of HAE management." Links to all presentations can be found on the KalVista website under Publications. About the KONFIDENT Phase 3 TrialThe KONFIDENT phase 3 clinical trial was a randomized, double-blind, 3-way crossover trial evaluating the safety and efficacy of sebetralstat 300 mg and 600 mg versus placebo for the on-demand treatment of HAE in adult and pediatric patients aged 12 years and older. The trial randomized 136 HAE patients from 66 clinical sites across 20 countries, making it the largest clinical trial ever conducted in HAE. In the trial, participants treated each eligible attack with up to two doses of study drug and treated up to three attacks over the course of the study. The trial included Type I and Type II HAE patients who had at least two documented HAE attacks 90 days prior to randomization and also included patients receiving long-term prophylaxis. About the KONFIDENT-S TrialKONFIDENT-S is an open-label extension trial with numerous real-world elements evaluating the long-term safety and efficacy of sebetralstat for the on-demand treatment of HAE attacks in adults and pediatric patients aged 12 years and older with HAE Type I or Type II. KalVista is transitioning ongoing trial participants to a novel oral disintegrating tablet (ODT) formulation to support a planned 2026 sNDA filing. If approved, the ODT formulation would provide people living with HAE with an alternative, novel option for oral, on-demand treatment. About SebetralstatSebetralstat is an investigational, novel oral plasma kallikrein inhibitor for the treatment of hereditary angioedema (HAE). We have filed multiple regulatory applications seeking approval of sebetralstat as the first oral, on-demand treatment for HAE in individuals aged 12 and older, with ongoing studies exploring its use in children aged 2 to 11. If approved, sebetralstat has the potential to become the foundational therapy for HAE management worldwide. About Hereditary AngioedemaHereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. All currently approved on-demand treatment options require either intravenous or subcutaneous administration. About KalVista Pharmaceuticals, Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. Our lead investigational product is sebetralstat, a novel, oral, on-demand treatment for hereditary angioedema (HAE). Sebetralstat is under regulatory review by the U.S. FDA, with a PDUFA goal date of June 17, 2025. In addition, we have completed Marketing Authorization Applications for sebetralstat to the European Medicines Agency and multiple other global regulatory authorities. For more information about KalVista, please visit or follow us on social media at @KalVista and LinkedIn. Forward-Looking StatementsThis press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, timing or outcomes of communications with the FDA, our expectations about safety and efficacy of our product candidates and timing of clinical trials and its results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and to obtain regulatory approvals for sebetralstat and other candidates in development, the success of any efforts to commercialize sebetralstat, the ability of sebetralstat and other candidates in development to treat HAE or other diseases, and the future progress and potential success of our oral Factor XIIa program. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2024, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. View source version on Contacts Jenn SnyderVice President, Corporate Affairs(617) 448-0281jsnyder@ Ryan BakerHead, Investor Relations(617) Sign in to access your portfolio
