Latest news with #14thC1-inhibitorDeficiency&AngioedemaWorkshop
Yahoo
6 days ago
- Business
- Yahoo
BioCryst Highlights Real-world Data Showing ORLADEYO® (berotralstat) Consistently Reduces HAE Attacks Across All Ages
New data shared at the 14th C1-inhibitor Deficiency & Angioedema Workshop supports berotralstat use at all stages of life RESEARCH TRIANGLE PARK, N.C., May 30, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new data which highlights the reduction in the percentage of days with hereditary angioedema (HAE) symptoms among young children initiating berotralstat in the APeX-P trial, and the broad safety and efficacy outcomes observed across all age groups of patients taking ORLADEYO to prevent HAE attacks. 'These data underscore the potential of our oral, once-daily prophylactic treatment to deliver consistent, meaningful benefits for people living with HAE across all stages of life. From young children to adolescents and adults, ORLADEYO continues to show strong efficacy and tolerability, helping patients experience more days free from attacks. These clinical and real-world results give me confidence that we can continue to have a very positive impact on advancing care for all those living with HAE, regardless of their age,' said Helen Thackray, chief research and development officer at BioCryst. The following three studies are being presented at the 14th C1-Inhibitor Deficiency & Angioedema Workshop in Budapest, Hungary from May 29–June 1, 2025. Reduction in Percentage of Days with Angioedema Symptoms Among Young Children Initiating Berotralstat – Interim Results from ApeX-P Oral Presentation - Session IV - Saturday, May 31 - 10:30 – 12:45 (12:00-12:15) The ongoing open-label APeX-P study is evaluating berotralstat in children aged 2 to 11 years with HAE. There were four weight-based cohorts. Cohort 1 from these interim results received 150 mg capsules daily, while cohorts 2–4 received daily oral granule doses of 108, 96, and 78 mg, respectively. The median age was 8 years (range: 3–11), with disease onset typically between ages 2–6 years. Prior to enrollment, patients reported a mean (SD) of 18.0 (20.5) missed school days annually due to HAE. While on berotralstat, at least half of patients were attack-free each month. The percentage of days with angioedema symptoms dropped from a mean (SD) of 11 percent (9.4 percent) during the standard-of-care period, measured pre-study, to 4.0 percent (4.7 percent) over 12 weeks, with this improvement sustained up to 48 weeks. The most common treatment emergent adverse events (TEAEs) were nasopharyngitis, upper respiratory tract infection and headache. These interim findings suggest berotralstat may help children with HAE experience fewer symptoms and a more normal daily life. Berotralstat for the Prevention of Hereditary Angioedema Attacks: Results from the Italian Expanded Access Program Oral Presentation - Session VI- Sunday, June 1 - 08:00 – 09:30 (09:00-09:15) In the Italian expanded access program (EAP), 22 patients received berotralstat free of charge until commercial availability, with an additional 12 weeks of treatment to ensure continuity. Safety was monitored throughout, and data on breakthrough attacks, disease control, and quality of life (QoL) were collected. Among 12 patients with available data, median (mean) Angioedema Control Test (AECT) scores improved from 11.5 (12.8) at baseline to 13.0 (14.0) at month three and 16.0 (14.8) at month six, indicating better disease control. Median (mean) AE-QoL total scores decreased from 45.0 (42.3) at baseline to 40.0 (37.6) at month three and 36.2 (34.0) at month six, reflecting clinically meaningful improvements in QoL. All 15 patients still enrolled at program closure transitioned to commercially available ORLADEYO. The treatment was associated with a consistently low HAE attack rate, improved disease control and a safety profile consistent with clinical trial findings. Tolerability and Effectiveness of Berotralstat for Prophylaxis in Adolescents with Hereditary Angioedema: subgroup analysis of the Berolife Study Oral Presentation - Session VI - Sunday June 1 – 08:00-9:30 (08:30-08:45) The Berolife study, a real-world observational study in France, assessed the tolerability and effectiveness of berotralstat in patients with HAE, including a subgroup of adolescents (aged 12–17) who received 150 mg once daily. The mean (SD) baseline attack rate was 1.8 (1.1) attacks/month (median: 1.8), based on the six months prior to enrolment. After six months of treatment, the median monthly attack rate decreased to 0.55 (from 2.25 at baseline), with similar reductions sustained at 12 and 18 months. TEAEs in adolescents were consistent with those seen in the overall Berolife population and in the APeX-2 and APeX-S clinical trials. These findings support berotralstat as an effective and well-tolerated long-term prophylactic option for adolescents with HAE. 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 InformationINDICATIONORLADEYO® (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 use The 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 INFORMATION An 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; interim results of a clinical trial do not necessarily predict final results; 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@ Niamh Lyons +353 87 7745000nlyons@ in to access your portfolio
Business Wire
6 days ago
- Health
- Business Wire
KalVista Pharmaceuticals Medical Congress Presentations Highlight Potential of Sebetralstat to Redefine Hereditary Angioedema Management
CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)-- KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced new sebetralstat findings related to time to end of progression of hereditary angioedema (HAE) attacks, and the effectiveness of treatment of attacks considered the most debilitating by patients, mucosal attacks, and attacks that have progressed in severity after a treatment delay. These data were presented at two congresses taking place concurrently: the 14th C1-inhibitor Deficiency & Angioedema Workshop in Budapest, Hungary, and the Eastern Allergy Conference (EAC) in Palm Beach, Florida, from May 29–June 1, 2025. Time to End of Progression of Hereditary Angioedema Attacks Treated with Sebetralstat was presented by William Lumry, M.D., Clinical Professor of Internal Medicine at the University of Texas Southwestern Medical School in Dallas and Director at Allergy and Asthma Research Associates, Dallas, Texas, United States. Time to end of attack progression (n=1591 attacks) in KONFIDENT-S: median 19.8 minutes Results from the open-label extension (KONFIDENT-S) aligned closely with the pivotal phase 3 trial, KONFIDENT, reinforcing the rapid effect of sebetralstat after absorption 'Stopping the progression of an HAE attack as early as possible is paramount to mitigating its impact on patients,' said Dr. Lumry. 'In both the KONFIDENT and KONFIDENT-S studies, sebetralstat halted attack progression in a median time of 19.8 minutes. As prior data have demonstrated near-complete plasma kallikrein inhibition 15 minutes after treatment, this analysis reveals that attacks are halted minutes after absorption. The consistency of findings across the KONFIDENT and KONFIDENT-S trials underscores the potential of sebetralstat as an effective and rapid-acting on-demand treatment for HAE attacks.' Effectiveness of sebetralstat for the on-demand treatment of mucosal hereditary angioedema attacks: interim analysis from KONFIDENT-S was presented by Henriette Farkas, M.D., PhD, DSc, Professor of Allergology and Clinical Immunology at Semmelweis University and Head of the Hungarian Angioedema Center of Reference and Excellence, Budapest, Hungary. Patients were able to self-administer sebetralstat quickly, with a median time to treatment of 20 minutes for abdominal attacks and 11.5 minutes for laryngeal attacks The median time to beginning of symptom relief was 1.3 hours for both abdominal and laryngeal attacks Of attacks reaching symptom relief within 12 hours, 96% did so before or without the need for an additional sebetralstat administration Sebetralstat was well-tolerated, even in laryngeal attacks, with no reported difficulties swallowing the oral tablet 'Mucosal HAE attacks, particularly those affecting the larynx, are a significant concern for patients and clinicians due to the risk of rapid progression and severe consequences, including the possibility of asphyxiation if left untreated,' said Dr. Farkas. 'These interim data from KONFIDENT-S demonstrate that sebetralstat provided rapid relief and resolution of both abdominal and laryngeal attacks with a favorable safety profile. Patients were able to self-administer sebetralstat very early in the course of an attack, when most attacks were still mild or moderate in severity.' Effectiveness of Sebetralstat for Severe or Very Severe Hereditary Angioedema Attacks in KONFIDENT-S was presented by H. Henry Li, M.D., Director of Immunology at the Institute for Asthma and Allergy, Wheaton, Maryland, United States. Sebetralstat was used to treat 76 attacks that had progressed to severe or very severe after a median of 2.16 hours from attack onset, demonstrating its utility in more advanced stages of HAE attacks The median time to beginning of symptom relief for these attacks was 1.36 hours, with reduction in attack severity and substantial reduction of symptom burden in a median of 1.77 hours and 9.15 hours, respectively 'When attacks are not treated early in accordance with guidelines, they can escalate and become severe,' said Dr. Li. 'We conducted this analysis of KONFIDENT-S to assess the utility of sebetralstat in more advanced stages of attacks that were associated with delayed treatment. Sebetralstat delivered symptom relief in a median time of 1.36 hours, reinforcing its potential as an on-demand treatment for challenging attack scenarios.' 'These new data underscore the potential of sebetralstat to fundamentally change HAE attack management," stated Paul Audhya, M.D., MBA, Chief Medical Officer of KalVista. "Even in real-world, high-stakes scenarios—be it mucosal attacks or severe attacks due to delayed treatment—sebetralstat consistently delivered rapid and reliable relief. The uniformity of these results, paired with an oral tablet formulation, solidifies our belief that sebetralstat can empower patients to act swiftly and recover quickly. We remain committed to bringing this innovative therapy to the HAE community as quickly as possible.' Links to all presentations can be found on the KalVista website under Publications. About Sebetralstat Sebetralstat 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 Angioedema Hereditary 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, Inc. KalVista 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 Statements This 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.
Yahoo
22-05-2025
- Health
- Yahoo
KalVista Pharmaceuticals to Present New Sebetralstat Data With 10 Abstracts Accepted at Upcoming Medical Meetings
CAMBRIDGE, Mass. & SALISBURY, England, May 22, 2025--(BUSINESS WIRE)--KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that it will present new sebetralstat data at two upcoming congresses taking place concurrently from May 29–June 1, 2025: the 14th C1-inhibitor Deficiency & Angioedema Workshop in Budapest, Hungary, and the Eastern Allergy Conference (EAC) in Palm Beach, Florida. The presentations will include new findings on the potential of sebetralstat to rapidly relieve symptoms and halt progression of hereditary angioedema (HAE) attacks, including mucosal and severe cases, underscoring the importance of early intervention and the role of an oral on-demand treatment option. Details of the presentations are as follows: C1-inhibitor Deficiency & Angioedema Workshop The following poster presentations will take place during Poster Session I on Friday, May 30, from 3:45–5:30 pm CEST (9:45–11:30 am ET): In their own words, patient perspectives on time to feeling in control of an HAE attack (P-18): Doug Jones, Anna Valerieva, Mar Guilarte, Neil Malloy, Sally van Kooten, Markus Heckmann, Markus Magerl. Time to end of progression of hereditary angioedema attacks treated with sebetralstat (P-25): William R. Lumry, John Anderson, Jonathan A. Bernstein, Mauro Cancian, Danny M. Cohn, Henriette Farkas, Henry Li, James Hao, Michael Smith, Paolo Bajcic, Paul Audhya, Markus Magerl. Impact of injectable HAE on-demand treatments on health-related quality of life: a patient and caregiver interview study (P-29): Patrick Yong, Aleena Banerji, Paula Busse, Timothy Craig, Sorena Kiani-Alikhan, Rebekah Hall, Siu Hing Lo, Caleb Dixon, Paul Audhya, Alice Wang, Tomaz Garcez. The following oral presentation will take place during Scientific Session III on Saturday, May 31, from 2:30–2:45 pm CEST (8:30–8:45 am ET): Effectiveness of sebetralstat for the on-demand treatment of mucosal hereditary angioedema attacks: interim analysis from KONFIDENT-S (O-29): Jonathan A. Bernstein, Emel Aygören-Pürsün, Vesna Grivcheva-Panovska, Inmaculada Martinez-Saguer, Danny M. Cohn, William R. Lumry, Marc A. Riedl, Andrea Zanichelli, Laurence Bouillet, Ya-Hsiu Chuang, Michael D. Smith, Christopher M. Yea, Paul K. Audhya, Henriette Farkas. The following oral presentation will take place during Scientific Session V on Saturday, May 31, from 3:15–3:30 pm CEST (9:15–9:30 am ET): Anxiety associated with hereditary angioedema attacks: results from the phase 3 KONFIDENT trial of oral sebetralstat (O-32): Timothy J. Craig, Emel Aygören-Pürsün, Jonathan A. Bernstein, Paula J. Busse, Teresa Caballero, Danny M. Cohn, Mar Guilarte, Henriette Farkas, Douglas H. Jones, Sorena Kiani-Alikhan, Michael E. Manning, Marcus Maurer, Marc A. Riedl, Sinisa Savic, H. James Wedner, Patrick F. K. Yong, Andrea Zanichelli, Erik Hansen, James Hao, Michael D. Smith, Christopher M. Yea, Paul K. Audhya, William R. Lumry. The following poster presentation will take place during Poster Session II on Saturday, May 31, from 4:15–5:45 pm CEST (10:15–11:45 am ET): Survey results from Italy, the US, UK, and France: Anxiety in patients using injectable on-demand treatments for hereditary angioedema attacks (P-32): Mauro Cancian, Alexis Bocquet, Paula J. Busse, Timothy Craig, Tariq El-Shanawany, Tomaz Garcez, Padmalal Gurugama, Rashmi Jain, Sorena Kiani-Alikhan, Maeve O'Connor, Cristine Radojicic, Sinisa Savic, Paola Triggianese, H. James Wedner, Patrick Yong, Andrea Zanichelli, Vibha Desai, Julie Ulloa, Sherry Danese, Paul K. Audhya, Sandra Christiansen. KalVista is also sponsoring a pre-workshop symposium for healthcare professionals on Thursday, May 29, from 1:00–2:00 pm CEST (7:00–8:00 am ET) in the Magnolia room, titled "Interplay Between On-Demand Treatment Guidelines and Clinical Trials in HAE." Chaired by Dr. William Lumry, Clinical Professor of Internal Medicine at the University of Texas Southwestern Medical School and Medical Director of AARA Research Center, this symposium will highlight insights from Dr. Mauro Cancian (University Hospital of Padova, Italy) and Dr. Danny Cohn (University of Amsterdam, Netherlands), addressing the evolution of on-demand treatment guidelines in HAE, real-world challenges in guideline adherence, and advancements in clinical trial designs to support guideline adherence. EAC The following poster presentations will take place on Saturday, May 31, from 9:45–11:00 am ET: Effectiveness of Sebetralstat for Severe or Very Severe Hereditary Angioedema Attacks in KONFIDENT-S: Henry Li, William R. Lumry, Michael E. Manning, Marc A. Riedl, James Wedner, James Hao, Michael D. Smith, Paul K. Audhya, Jonathan A. Bernstein. On-demand Treatment of Hereditary Angioedema Attacks with Sebetralstat in Older Adults: Interim Analysis from KONFIDENT-S: Daniel F. Soteres, Alan P. Baptist, Jonathan A. Bernstein, Timothy Craig, William R. Lumry, James Hao, Michael D. Smith, Paolo Bajcic, Paul K. Audhya, Marc A. Riedl. Time to End of Progression of Hereditary Angioedema Attacks Treated with Sebetralstat: Daniel F. Soteres, William R. Lumry, John Anderson, Jonathan A. Bernstein, Mauro Cancian, Danny M. Cohn, Henriette Farkas, Henry Li, James Hao, Michael D. Smith, Paolo Bajcic, Paul K. Audhya, Markus Magerl. Long-Term Prophylaxis Compliance and Healthcare Resource Utilization in Hereditary Angioedema: A Claims Database Analysis: Raffi Tachdjian, Daniel F. Soteres, Maeve O'Connor, Chirag Maheshwari, Alice Wang, Paul K. Audhya, Timothy Craig. Links to all presentations can be found on the KalVista website under Publications. 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 Ryan BakerHead, Investor Relations(617) Molly CameronDirector, Corporate Communications(857) Error 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
Business Wire
22-05-2025
- Health
- Business Wire
KalVista Pharmaceuticals to Present New Sebetralstat Data With 10 Abstracts Accepted at Upcoming Medical Meetings
CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)-- KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that it will present new sebetralstat data at two upcoming congresses taking place concurrently from May 29–June 1, 2025: the 14th C1-inhibitor Deficiency & Angioedema Workshop in Budapest, Hungary, and the Eastern Allergy Conference (EAC) in Palm Beach, Florida. The presentations will include new findings on the potential of sebetralstat to rapidly relieve symptoms and halt progression of hereditary angioedema (HAE) attacks, including mucosal and severe cases, underscoring the importance of early intervention and the role of an oral on-demand treatment option. Details of the presentations are as follows: C1-inhibitor Deficiency & Angioedema Workshop The following poster presentations will take place during Poster Session I on Friday, May 30, from 3:45–5:30 pm CEST (9:45–11:30 am ET): In their own words, patient perspectives on time to feeling in control of an HAE attack (P-18): Doug Jones, Anna Valerieva, Mar Guilarte, Neil Malloy, Sally van Kooten, Markus Heckmann, Markus Magerl. Time to end of progression of hereditary angioedema attacks treated with sebetralstat (P-25): William R. Lumry, John Anderson, Jonathan A. Bernstein, Mauro Cancian, Danny M. Cohn, Henriette Farkas, Henry Li, James Hao, Michael Smith, Paolo Bajcic, Paul Audhya, Markus Magerl. Impact of injectable HAE on-demand treatments on health-related quality of life: a patient and caregiver interview study (P-29): Patrick Yong, Aleena Banerji, Paula Busse, Timothy Craig, Sorena Kiani-Alikhan, Rebekah Hall, Siu Hing Lo, Caleb Dixon, Paul Audhya, Alice Wang, Tomaz Garcez. The following oral presentation will take place during Scientific Session III on Saturday, May 31, from 2:30–2:45 pm CEST (8:30–8:45 am ET): Effectiveness of sebetralstat for the on-demand treatment of mucosal hereditary angioedema attacks: interim analysis from KONFIDENT-S (O-29): Jonathan A. Bernstein, Emel Aygören-Pürsün, Vesna Grivcheva-Panovska, Inmaculada Martinez-Saguer, Danny M. Cohn, William R. Lumry, Marc A. Riedl, Andrea Zanichelli, Laurence Bouillet, Ya-Hsiu Chuang, Michael D. Smith, Christopher M. Yea, Paul K. Audhya, Henriette Farkas. The following oral presentation will take place during Scientific Session V on Saturday, May 31, from 3:15–3:30 pm CEST (9:15–9:30 am ET): Anxiety associated with hereditary angioedema attacks: results from the phase 3 KONFIDENT trial of oral sebetralstat (O-32): Timothy J. Craig, Emel Aygören-Pürsün, Jonathan A. Bernstein, Paula J. Busse, Teresa Caballero, Danny M. Cohn, Mar Guilarte, Henriette Farkas, Douglas H. Jones, Sorena Kiani-Alikhan, Michael E. Manning, Marcus Maurer, Marc A. Riedl, Sinisa Savic, H. James Wedner, Patrick F. K. Yong, Andrea Zanichelli, Erik Hansen, James Hao, Michael D. Smith, Christopher M. Yea, Paul K. Audhya, William R. Lumry. The following poster presentation will take place during Poster Session II on Saturday, May 31, from 4:15–5:45 pm CEST (10:15–11:45 am ET): Survey results from Italy, the US, UK, and France: Anxiety in patients using injectable on-demand treatments for hereditary angioedema attacks (P-32): Mauro Cancian, Alexis Bocquet, Paula J. Busse, Timothy Craig, Tariq El-Shanawany, Tomaz Garcez, Padmalal Gurugama, Rashmi Jain, Sorena Kiani-Alikhan, Maeve O'Connor, Cristine Radojicic, Sinisa Savic, Paola Triggianese, H. James Wedner, Patrick Yong, Andrea Zanichelli, Vibha Desai, Julie Ulloa, Sherry Danese, Paul K. Audhya, Sandra Christiansen. KalVista is also sponsoring a pre-workshop symposium for healthcare professionals on Thursday, May 29, from 1:00–2:00 pm CEST (7:00–8:00 am ET) in the Magnolia room, titled ' Interplay Between On-Demand Treatment Guidelines and Clinical Trials in HAE.' Chaired by Dr. William Lumry, Clinical Professor of Internal Medicine at the University of Texas Southwestern Medical School and Medical Director of AARA Research Center, this symposium will highlight insights from Dr. Mauro Cancian (University Hospital of Padova, Italy) and Dr. Danny Cohn (University of Amsterdam, Netherlands), addressing the evolution of on-demand treatment guidelines in HAE, real-world challenges in guideline adherence, and advancements in clinical trial designs to support guideline adherence. EAC The following poster presentations will take place on Saturday, May 31, from 9:45–11:00 am ET: Effectiveness of Sebetralstat for Severe or Very Severe Hereditary Angioedema Attacks in KONFIDENT-S: Henry Li, William R. Lumry, Michael E. Manning, Marc A. Riedl, James Wedner, James Hao, Michael D. Smith, Paul K. Audhya, Jonathan A. Bernstein. On-demand Treatment of Hereditary Angioedema Attacks with Sebetralstat in Older Adults: Interim Analysis from KONFIDENT-S: Daniel F. Soteres, Alan P. Baptist, Jonathan A. Bernstein, Timothy Craig, William R. Lumry, James Hao, Michael D. Smith, Paolo Bajcic, Paul K. Audhya, Marc A. Riedl. Time to End of Progression of Hereditary Angioedema Attacks Treated with Sebetralstat: Daniel F. Soteres, William R. Lumry, John Anderson, Jonathan A. Bernstein, Mauro Cancian, Danny M. Cohn, Henriette Farkas, Henry Li, James Hao, Michael D. Smith, Paolo Bajcic, Paul K. Audhya, Markus Magerl. Long-Term Prophylaxis Compliance and Healthcare Resource Utilization in Hereditary Angioedema: A Claims Database Analysis: Raffi Tachdjian, Daniel F. Soteres, Maeve O'Connor, Chirag Maheshwari, Alice Wang, Paul K. Audhya, Timothy Craig. Links to all presentations can be found on the KalVista website under Publications. About Sebetralstat Sebetralstat 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 Angioedema Hereditary 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, Inc. KalVista 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 Statements This 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.
