Jasper Therapeutics presents data from SPOTLIGHT Phase 1b/2a study
Jasper Therapeutics (JSPR) is presenting data from the 180mg cohort of the Company's SPOTLIGHT Phase 1b/2a study of subcutaneous briquilimab in adult participants with CIndU at the European Academy of Allergy and Clinical Immunology, EAACI, Annual Congress. Briquilimab administration resulted in deep disease control at 180mg, with 12 of 12 participants enrolled in the cohort achieving a clinical response within the 8-week preliminary analysis period. The efficacy observed was rapid and durable, with 8 of 12 participants achieving clinical response by week 2, and 7 of 12 participants maintaining clinical response through week 8. Briquilimab continued to be well tolerated in the study, with no serious adverse events and no grade 3 or higher adverse events reported in the 180mg cohort.
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Yahoo
5 days ago
- Yahoo
BioCryst Presents New Data on the Long-term Efficacy and Safety of ORLADEYO® (berotralstat) Across all Ages at EAACI
RESEARCH TRIANGLE PARK, N.C., June 16, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new data on the long-term efficacy and safety of ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients across all age groups. 'These data from both clinical trials and real-world settings continue to strengthen the evidence that ORLADEYO is an effective and well-tolerated long-term prophylactic treatment for HAE. Importantly, patients report not only fewer and less severe attacks, but also meaningful improvements in daily functioning and emotional well-being. This contributes to the growing body of evidence supporting the use of ORLADEYO as a long-term prophylactic option for adolescents and adults living with HAE,' said Helen Thackray, chief research and development officer of BioCryst. The following four studies were presented at the European Academy of Allergy and Clinical Immunology (EAACI) meeting in Glasgow, United Kingdom, from June 13 to 16, 2025. Berotralstat Use Reduced Number of HAE Attacks Requiring Treatment or Professional Care in Pediatric Patients: Interim Results from APeX-P The ongoing open-label APeX-P study is evaluating the pharmacokinetic, safety, and effectiveness of berotralstat in children aged 2 to 11 years with HAE. Patients were enrolled into four weight-based cohorts. Cohort 1 received a 150 mg capsule once daily; cohorts 2-4 received once-daily oral granule doses of 108 mg, 96 mg and 78 mg, respectively. The median age was 8 years (range: 3–11), with disease onset typically between ages 2–6 years. All patients received standard of care (SOC) treatment for 12 weeks prior to the study. Key results Eighty-six percent reduction in attacks requiring professional care: attacks dropped from 22 during the 12-week SOC period to three following 12 weeks of berotralstat treatment Early/rapid and sustained reduction in the rate of HAE attacks requiring on-demand treatment Mean (SEM) adjusted monthly attack rate decreased from 1.28 (0.25) during SOC to 0.38 (0.13) from day one to week four of berotralstat treatment Sustained reduction for up to 48 weeks of follow-up Berotralstat was well tolerated across all cohorts; the most common treatment-emergent adverse events were nasopharyngitis, upper respiratory tract infection, and headache. Assessment of the Effectiveness and Tolerability of Berotralstat for Long-term Prophylaxis in Hereditary Angioedema: Findings from the Berolife Study The Berolife study is an open-label observational study in France which assessed the real-world tolerability and effectiveness of oral once-daily berotralstat (150 mg) in patients with HAE aged >12 years. A total of 82 patients were enrolled, with a mean (SD) age of 40.0 (17.5) years. The mean (SD) baseline attack rate was 1.1 (1.0) attacks per month (median: 0.83), based on the six months prior to enrollment. Key results Significant reduction in monthly HAE attacks was observed at six months (in patients with follow-up data (n=37)) Median monthly attack rate decreased from 1.0 to 0.44 attacks after six months of berotralstat treatment Sustained reductions in attack frequency maintained at 12, 18, and 24 months Berotralstat was well tolerated throughout the study period Adverse events were consistent with previous clinical trial data Impact of Berotralstat on Quality of Life in Patients with Hereditary Angioedema This analysis assessed the impact of berotralstat on patient-reported quality of life (QoL) outcomes compared to placebo, using pooled data from previous Phase 3 APeX-2 and APeX-J clinical trials which showed that once-daily berotralstat 150 mg significantly reduced the frequency of HAE attacks. This QoL assessment was measured using the validated Angioedema Quality of Life Questionnaire (AE-QoL), which evaluates four key domains: functioning, fatigue/mood, fears/shame, and nutrition. Key results Significant improvements in AE-QoL total and domain scores were observed with berotralstat versus placebo at week 24 Benefits sustained through week 96, indicating lasting improvement in daily life At week 24, 60 percent of patients receiving berotralstat achieved the Minimal Clinically Important Difference (MCID) in AE-QoL total score, compared to 52.4 percent in the placebo group Over time, the proportion of patients reaching MCID increased in the berotralstat cohort, suggesting progressive and cumulative improvements in QoL alongside reductions in HAE attack frequency Patients With HAE Report Positive Perceptions Following Berotralstat Treatment: Results from a Focus Group This qualitative study explored patient experiences with HAE, including their care journey and perceptions of treatment with berotralstat. Focus groups were conducted to gain insight into the real-world impact of berotralstat on patients' daily lives. Seven patients from France, aged 20-70 years, participated in the focus groups. All had been treated with berotralstat for at least six months, with a median age at HAE diagnosis of 28 years. Prior to starting berotralstat, the majority of participants (71 percent) had switched from previous treatments due to long-term tolerability concerns and guidance from health authorities. Key findings Participants reported notable improvements in disease control, including reduced frequency and severity of HAE attacks Minimal side effects were observed Berotralstat was described as less burdensome and easier to incorporate into daily routines compared to previous therapies Improved disease management was associated with reduced psychological distress and a greater sense of normalcy, contributing to enhanced quality of life 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 PharmaceuticalsBioCryst 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 StatementsThis press release contains forward-looking statements, including statements relating to ORLADEYO safety, 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@
Associated Press
5 days ago
- Associated Press
Pharvaris Presents Data Highlighting the Potential for Deucrictibant to Prevent and Treat Bradykinin-Mediated Angioedema Attacks at the EAACI Congress
ZUG, Switzerland, June 16, 2025 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to help address unmet needs of those living with bradykinin-mediated diseases such as hereditary angioedema (HAE) and acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH), today announced a summary of data that were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025. 'Pharvaris embraced the opportunity to engage in scientific exchange with the HAE thought leader community during EAACI as we presented data supporting the differentiated profile of deucrictibant for the prophylactic and on-demand treatment of bradykinin-mediated angioedema attacks,' said Berndt Modig, Chief Executive Officer of Pharvaris. 'Building on our R&D call from last week, we shared data demonstrating the potential for deucrictibant to address the unmet needs of people living with bradykinin-mediated angioedema beyond HAE-1/2. Deucrictibant showed sustained attack reduction and improved quality of life measures in the randomized portion of the CHAPTER-1 study, which was maintained in the open-label extension study, as well as early-onset symptom relief and complete symptom resolution in a single dose in most attacks in our ongoing RAPIDe-2 on-demand long-term extension RAPIDe-3 is the first and only phase 3 on-demand study that will explore 'end-of-progression' as a new pre-specified endpoint, which is particularly meaningful for people living with HAE. Together with the outcomes from other study endpoints, we will be able to assess the full impact of deucrictibant on an HAE attack from start to end.' Details of the presentations are outlined below: Prophylaxis Long-Term Safety and Efficacy of Oral Deucrictibant for Prophylaxis in Hereditary Angioedema: Results of the CHAPTER-1 Open-Label Extension Study, a poster presentation by Emel Aygören-Pürsün, M.D. Long-Term Prophylactic Treatment with Oral Deucrictibant Improves Disease Control and Health-Related Quality of Life in Participants with Hereditary Angioedema in the CHAPTER-1 Open-Label Extension Study, a flash talk by Markus Magerl, M.D. CHAPTER-3 Phase 3 Trial Design: Efficacy and Safety of the Oral Bradykinin B2 Receptor Antagonist Deucrictibant Extended-Release Tablet for Prophylaxis of Hereditary Angioedema Attacks, a flash talk by William Lumry, M.D. Health-Related Quality of Life and Clinical Characteristics in People Living with Hereditary Angioedema Prescribed Long Term Prophylaxis Alone and On-Demand Treatment Alone, an oral presentation by Laurence Bouillet, M.D., Ph.D. On-Demand Long-Term Safety and Efficacy of Oral Deucrictibant for Treatment of Hereditary Angioedema Attacks: Results of the RAPIDe-2 Extension Study, a thematic poster session by Henriette Farkas, M.D., Ph.D., Safety and Efficacy of Oral Deucrictibant for Treatment of Upper Airway and Laryngeal Hereditary Angioedema Attacks: Results from the RAPIDe-2 Extension Study, a flash talk by Anna Valerieva, M.D., Ph.D. Expansion Beyond HAE Clinical Validation of a Novel Kinin Biomarker Assay for Characterization of Bradykinin-Mediated Pathologies in U.S. Subjects with Hereditary Angioedema, a flash talk by Evangelia Pardali, Ph.D. Development of a Conceptual Model Supporting a Clinical Outcome Assessment Strategy for Acquired Angioedema due to C1 Inhibitor Deficiency, a thematic poster session by Andrea Zanichelli, M.D., Ph.D. The posters are available on the Investors section of the Pharvaris website at: About Deucrictibant Deucrictibant is a novel, potent, orally bioavailable small-molecule bradykinin B2 receptor antagonist currently in clinical development. Deucrictibant is being investigated for its potential to prevent the occurrence of bradykinin-mediated angioedema attacks and to treat the manifestations of attacks if/when they occur by inhibiting bradykinin signaling through the bradykinin B2 receptor. Pharvaris is developing two formulations of deucrictibant for oral administration: an extended-release tablet to enable sustained absorption and efficacy as prophylactic treatment, and an immediate-release capsule to enable rapid onset of activity for on-demand treatment. Deucrictibant has been granted orphan drug designation for the treatment of bradykinin-mediated angioedema by the U.S. Food and Drug Administration and orphan designation by the European Commission. About Pharvaris Pharvaris is a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to potentially address all types of bradykinin-mediated angioedema. Pharvaris intends to provide injectable-like efficacy™ and placebo-like tolerability with the convenience of oral therapies to prevent and treat bradykinin-mediated angioedema attacks. With positive data in both Phase 2 prophylaxis and on-demand studies in HAE, Pharvaris is currently evaluating the efficacy and safety of deucrictibant in a pivotal Phase 3 study for the prevention of HAE attacks (CHAPTER-3) and a pivotal Phase 3 study for the on-demand treatment of HAE attacks (RAPIDe-3). For more information, visit Forward Looking Statements This press release contains certain forward-looking statements that involve substantial risks and uncertainties. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements relating to our future plans, studies and trials, and any statements containing the words 'believe,' 'anticipate,' 'expect,' 'estimate,' 'may,' 'could,' 'should,' 'would,' 'will,' 'intend' and similar expressions. These forward-looking statements are based on management's current expectations, are neither promises nor guarantees, and involve known and unknown risks, uncertainties and other important factors that may cause Pharvaris' actual results, performance or achievements to be materially different from its expectations expressed or implied by the forward-looking statements. Such risks include but are not limited to the following: uncertainty in the outcome of our interactions with regulatory authorities, including the FDA; the expected timing, progress, or success of our clinical development programs, especially for deucrictibant immediate-release capsules and deucrictibant extended-release tablets, which are in late-stage global clinical trials; our ability to replicate the efficacy and safety demonstrated in the RAPIDe-1, RAPIDe-2, and CHAPTER-1 Phase 2 and Phase 3 studies in ongoing and future nonclinical studies and clinical trials; risks arising from epidemic diseases, which may adversely impact our business, nonclinical studies, and clinical trials; our ability to potentially use deucrictibant for alternative purposes, for example to treat C1-INH deficiency (AAE-C1INH); the outcome and timing of regulatory approvals; the value of our ordinary shares; the timing, costs and other limitations involved in obtaining regulatory approval for our product candidates, or any other product candidate that we may develop in the future; our ability to establish commercial capabilities or enter into agreements with third parties to market, sell, and distribute our product candidates; our ability to compete in the pharmaceutical industry, including with respect to existing therapies, emerging potentially competitive therapies and with competitive generic products; our ability to market, commercialize and achieve market acceptance for our product candidates; our ability to produce sufficient amounts of drug product candidates for commercialization; our ability to raise capital when needed and on acceptable terms; regulatory developments in the United States, the European Union and other jurisdictions; our ability to protect our intellectual property and know-how and operate our business without infringing the intellectual property rights or regulatory exclusivity of others; our ability to manage negative consequences from changes in applicable laws and regulations, including tax laws (including the Biosecure Act), our ability to maintain an effective system of internal control over financial reporting; changes and uncertainty in general market conditions; disruptions at the FDA and other agencies; political conditions, such as the current war between Russia and Ukraine; economic conditions, including continuing inflation concerns; and the other factors described under the headings 'Cautionary Statement Regarding Forward-Looking Statements' and 'Item 3. Key Information—D. Risk Factors' in our Annual Report on Form 20-F and other periodic filings with the U.S. Securities and Exchange Commission. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. While Pharvaris may elect to update such forward-looking statements at some point in the future, Pharvaris disclaims any obligation to do so, even if subsequent events cause its views to change. These forward-looking statements should not be relied upon as representing Pharvaris' views as of any date subsequent to the date of this press release. Contact Maggie Beller Executive Director, Head of Corporate and Investor Communications [email protected]
Yahoo
5 days ago
- Yahoo
Pharvaris Presents Data Highlighting the Potential for Deucrictibant to Prevent and Treat Bradykinin-Mediated Angioedema Attacks at the EAACI Congress
ZUG, Switzerland, June 16, 2025 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to help address unmet needs of those living with bradykinin-mediated diseases such as hereditary angioedema (HAE) and acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH), today announced a summary of data that were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025. 'Pharvaris embraced the opportunity to engage in scientific exchange with the HAE thought leader community during EAACI as we presented data supporting the differentiated profile of deucrictibant for the prophylactic and on-demand treatment of bradykinin-mediated angioedema attacks,' said Berndt Modig, Chief Executive Officer of Pharvaris. 'Building on our R&D call from last week, we shared data demonstrating the potential for deucrictibant to address the unmet needs of people living with bradykinin-mediated angioedema beyond HAE-1/2. Deucrictibant showed sustained attack reduction and improved quality of life measures in the randomized portion of the CHAPTER-1 study, which was maintained in the open-label extension study, as well as early-onset symptom relief and complete symptom resolution in a single dose in most attacks in our ongoing RAPIDe-2 on-demand long-term extension study. Finally, RAPIDe-3 is the first and only phase 3 on-demand study that will explore 'end-of-progression' as a new pre-specified endpoint, which is particularly meaningful for people living with HAE. Together with the outcomes from other study endpoints, we will be able to assess the full impact of deucrictibant on an HAE attack from start to end.' Details of the presentations are outlined below:ProphylaxisLong-Term Safety and Efficacy of Oral Deucrictibant for Prophylaxis in Hereditary Angioedema: Results of the CHAPTER-1 Open-Label Extension Study, a poster presentation by Emel Aygören-Pürsün, M.D. First-ever bradykinin B2 receptor antagonism mechanism-on-mechanism prophylactic/on-demand data supports potential for deucrictibant portfolio. The ongoing Phase 2 CHAPTER-1 open-label extension (OLE) study provides further evidence on the long-term safety and efficacy of oral deucrictibant for prevention of HAE attacks. The attack rate has remained low, irrespective of baseline attack rate, for over a year and a half in OLE participants. When evaluating mechanism-on-mechanism responses, the response to icatibant for on-demand treatment of breakthrough attacks appeared to be maintained when used for breakthrough attacks during prophylactic treatment with deucrictibant. Long-Term Prophylactic Treatment with Oral Deucrictibant Improves Disease Control and Health-Related Quality of Life in Participants with Hereditary Angioedema in the CHAPTER-1 Open-Label Extension Study, a flash talk by Markus Magerl, M.D. The impact of deucrictibant treatment on health-related quality of life (HRQoL), disease control, and treatment satisfaction during the ongoing CHAPTER-1 OLE was evaluated. All of the participants who received deucrictibant reported clinically meaningful improvements in HRQoL at the end of the randomized portion of the trial, which was maintained up to the latest timepoint assessed at the time of data cutoff (week 62) of the OLE. All of the participants in the OLE reported well controlled HAE and a high level of satisfaction with treatment. CHAPTER-3 Phase 3 Trial Design: Efficacy and Safety of the Oral Bradykinin B2 Receptor Antagonist Deucrictibant Extended-Release Tablet for Prophylaxis of Hereditary Angioedema Attacks, a flash talk by William Lumry, M.D. CHAPTER-3 is an ongoing, global, Phase 3 study designed to evaluate the efficacy and safety of once-daily, oral deucrictibant (40 mg/day) extended release (XR) tablet for prophylaxis of attacks in adolescents and adults with HAE. Results from the Phase 2 CHAPTER-1 study support the CHAPTER-3 study design. Health-Related Quality of Life and Clinical Characteristics in People Living with Hereditary Angioedema Prescribed Long Term Prophylaxis Alone and On-Demand Treatment Alone, an oral presentation by Laurence Bouillet, M.D., Ph.D. A real-world cross-sectional survey was conducted to assess the relationship between treatment and outcomes of patients with HAE type 1/2 prescribed LTP or ODT alone in a real-world setting. 162 physicians reported data for 601 patients from Europe and the United States, collected via the Adelphi HAE Wave II Disease Specific Programme™ (DSP). Of the 601 patients, 41% were taking LTP, and 59% were taking ODT alone. Study results showed that patients with HAE prescribed LTP in the last 12 months experienced more mild attacks than moderate or severe attacks and had significantly better health related quality of life at the time of the survey compared with those prescribed ODT alone. Analysis suggests that both LTP and ODT play important roles in HAE management and corroborates international guidelines that recommend patients with HAE on LTP must always have ODT available at all times. On-DemandLong-Term Safety and Efficacy of Oral Deucrictibant for Treatment of Hereditary Angioedema Attacks: Results of the RAPIDe-2 Extension Study, a thematic poster session by Henriette Farkas, M.D., Ph.D., Following the closure of Part A of RAPIDe-2, a Phase 2/3 study of deucrictibant for the on-demand treatment of HAE attacks, an analysis of 465 attacks from 19 participants, including 14 upper airway attacks from seven participants, was conducted. The final results from Part A of the RAPIDe-2 extension are consistent with the Phase 2 RAPIDe-1 randomized study. Deucrictibant continued to be well tolerated across all doses with no treatment-related treatment-emergent adverse events reported. The median time to onset of symptom relief was 1.1 hours, and 97.8% of attacks achieved onset of symptom relief by 12 hours. The median time to complete attack resolution was 10.6 hours, and 86.9% of attack achieved complete resolution at 24 hours. Deucrictibant data shows single-dose durability without symptom reoccurrence in most HAE attacks treated. 89.2% of the attacks that achieved symptom resolution at 24 hours were treated with a single dose of deucrictibant. Safety and Efficacy of Oral Deucrictibant for Treatment of Upper Airway and Laryngeal Hereditary Angioedema Attacks: Results from the RAPIDe-2 Extension Study, a flash talk by Anna Valerieva, M.D., Ph.D. The final data from Part A of the RAPIDe-2 study showed that safety and efficacy outcomes of treatment with deucrictibant IR were consistent for both HAE attacks affecting the upper airways, including laryngeal attacks, and HAE attacks occurring in other locations. Deucrictibant was generally well tolerated with no treatment-related treatment-emergent adverse events reported across upper airway and non-upper airway attacks. Fourteen upper airway attacks were treated by 7 participants; the median time to onset of symptom relief, as measured by Patient Global Impression of Change (PGI-C) of 'a little better', was 1.4 hours (n=14) for upper airway attacks and 1.1 hours for non-upper airway attacks (n=451). Endpoint measurements taken throughout the span of an entire attack until and including complete resolution were similar for both upper airway and non-upper airway attacks. Importantly, 92.9% of the upper airway attacks were treated with a single dose of deucrictibant. Expansion Beyond HAEClinical Validation of a Novel Kinin Biomarker Assay for Characterization of Bradykinin-Mediated Pathologies in U.S. Subjects with Hereditary Angioedema, a flash talk by Evangelia Pardali, Ph.D. Assays for an early and accurate diagnosis of bradykinin-mediated angioedema are lacking. Cold activation of plasma from people living with HAE resulted in increased levels of bradykinin compared to cold-activated plasma of healthy volunteers. The qualified kinin assay can be used to reliably characterize a bradykinin signature in people with recurrent angioedema and could become a key tool aiding identification, study, and management of bradykinin-mediated pathologies including bradykinin-mediated angioedema. As presented at the 14th C1-Inhibitor Deficiency and Angioedema Workshop, the performance of the assay does not depend on availability of 'fresh' plasma samples and the assay can also be applied in biobank samples for identification of people with bradykinin-mediated angioedema. Development of a Conceptual Model Supporting a Clinical Outcome Assessment Strategy for Acquired Angioedema due to C1 Inhibitor Deficiency, a thematic poster session by Andrea Zanichelli, M.D., Ph.D. There are currently no approved therapies for the treatment of AAE-C1INH attacks, nor patient-reported outcome measures validated in AAE-C1INH. Concept elicitation and cognitive interviews were performed to develop a conceptual model of AAE-C1INH that could reveal important disease concepts supporting a clinical outcome assessment strategy, as well as evaluating the comprehension and interpretation of PGI-C, PGI-Severity (PGI-S), patient global assessment of change (PGA-C), and PGA-Status (PGA-S), and explore perceptions of meaningful change using these measures. One hundred percent of participants considered PGI-C 'better' to be a meaningful change four hours post-treatment. Epidemiologic data and cognitive interviews further elucidate the unmet needs in bradykinin-mediated angioedema. The posters are available on the Investors section of the Pharvaris website at: About DeucrictibantDeucrictibant is a novel, potent, orally bioavailable small-molecule bradykinin B2 receptor antagonist currently in clinical development. Deucrictibant is being investigated for its potential to prevent the occurrence of bradykinin-mediated angioedema attacks and to treat the manifestations of attacks if/when they occur by inhibiting bradykinin signaling through the bradykinin B2 receptor. Pharvaris is developing two formulations of deucrictibant for oral administration: an extended-release tablet to enable sustained absorption and efficacy as prophylactic treatment, and an immediate-release capsule to enable rapid onset of activity for on-demand treatment. Deucrictibant has been granted orphan drug designation for the treatment of bradykinin-mediated angioedema by the U.S. Food and Drug Administration and orphan designation by the European Commission. About PharvarisPharvaris is a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to potentially address all types of bradykinin-mediated angioedema. Pharvaris intends to provide injectable-like efficacy™ and placebo-like tolerability with the convenience of oral therapies to prevent and treat bradykinin-mediated angioedema attacks. With positive data in both Phase 2 prophylaxis and on-demand studies in HAE, Pharvaris is currently evaluating the efficacy and safety of deucrictibant in a pivotal Phase 3 study for the prevention of HAE attacks (CHAPTER-3) and a pivotal Phase 3 study for the on-demand treatment of HAE attacks (RAPIDe-3). For more information, visit Forward Looking StatementsThis press release contains certain forward-looking statements that involve substantial risks and uncertainties. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements relating to our future plans, studies and trials, and any statements containing the words 'believe,' 'anticipate,' 'expect,' 'estimate,' 'may,' 'could,' 'should,' 'would,' 'will,' 'intend' and similar expressions. These forward-looking statements are based on management's current expectations, are neither promises nor guarantees, and involve known and unknown risks, uncertainties and other important factors that may cause Pharvaris' actual results, performance or achievements to be materially different from its expectations expressed or implied by the forward-looking statements. Such risks include but are not limited to the following: uncertainty in the outcome of our interactions with regulatory authorities, including the FDA; the expected timing, progress, or success of our clinical development programs, especially for deucrictibant immediate-release capsules and deucrictibant extended-release tablets, which are in late-stage global clinical trials; our ability to replicate the efficacy and safety demonstrated in the RAPIDe-1, RAPIDe-2, and CHAPTER-1 Phase 2 and Phase 3 studies in ongoing and future nonclinical studies and clinical trials; risks arising from epidemic diseases, which may adversely impact our business, nonclinical studies, and clinical trials; our ability to potentially use deucrictibant for alternative purposes, for example to treat C1-INH deficiency (AAE-C1INH); the outcome and timing of regulatory approvals; the value of our ordinary shares; the timing, costs and other limitations involved in obtaining regulatory approval for our product candidates, or any other product candidate that we may develop in the future; our ability to establish commercial capabilities or enter into agreements with third parties to market, sell, and distribute our product candidates; our ability to compete in the pharmaceutical industry, including with respect to existing therapies, emerging potentially competitive therapies and with competitive generic products; our ability to market, commercialize and achieve market acceptance for our product candidates; our ability to produce sufficient amounts of drug product candidates for commercialization; our ability to raise capital when needed and on acceptable terms; regulatory developments in the United States, the European Union and other jurisdictions; our ability to protect our intellectual property and know-how and operate our business without infringing the intellectual property rights or regulatory exclusivity of others; our ability to manage negative consequences from changes in applicable laws and regulations, including tax laws (including the Biosecure Act), our ability to maintain an effective system of internal control over financial reporting; changes and uncertainty in general market conditions; disruptions at the FDA and other agencies; political conditions, such as the current war between Russia and Ukraine; economic conditions, including continuing inflation concerns; and the other factors described under the headings 'Cautionary Statement Regarding Forward-Looking Statements' and 'Item 3. Key Information—D. Risk Factors' in our Annual Report on Form 20-F and other periodic filings with the U.S. Securities and Exchange Commission. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. While Pharvaris may elect to update such forward-looking statements at some point in the future, Pharvaris disclaims any obligation to do so, even if subsequent events cause its views to change. These forward-looking statements should not be relied upon as representing Pharvaris' views as of any date subsequent to the date of this press release. CONTACT: Contact Maggie Beller Executive Director, Head of Corporate and Investor Communications 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
