Hereditary angioedema

Orphan designation: Donidalorsen Treatment of hereditary angioedema, 19/02/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
Patient, Committee, EMA, IRIS, COMP, Hereditary angioedema, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of hereditary angioedema on 19 February 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Pharvaris to Present Deucrictibant Clinical Data at the CIIC Spring 2024 Conference

Retrieved on: 
Thursday, April 4, 2024
Safety, CIIC, Abstract, HAE, Hereditary angioedema, Conference, Bradykinin receptor B2, Pharmaceutical industry

ZUG, Switzerland, April 04, 2024 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to treat and prevent hereditary angioedema (HAE) attacks, today announced the acceptance of two abstracts for presentation at the Consortium of Independent Immunology Clinics (CIIC) Spring 2024 Conference, to be held from April 12-13, 2024, in Arlington, TX.

Key Points: 
  • ZUG, Switzerland, April 04, 2024 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to treat and prevent hereditary angioedema (HAE) attacks, today announced the acceptance of two abstracts for presentation at the Consortium of Independent Immunology Clinics (CIIC) Spring 2024 Conference, to be held from April 12-13, 2024, in Arlington, TX.
  • Title: Efficacy and Safety of Oral Deucrictibant, a Bradykinin B2 Receptor Antagonist, in Prophylaxis of Hereditary Angioedema Attacks: Results of CHAPTER-1 Phase 2 Trial
    The posters and associated presentation slides will be made available on the Investors section of the Pharvaris website at the beginning of the respective presentation sessions at: https://ir.pharvaris.com/news-events/events-presentations .

Pharvaris Announces Phase 3 Clinical Study Design for Recently Initiated RAPIDe-3 Study, and Presents Quality-of-Life Improvement and Caregiver Behavior Data at Two Recent HAE Congresses

Retrieved on: 
Monday, March 18, 2024
Angioedema, Conference, EOP, Attack, Quality of life (healthcare), Network, Safety, HAE, Hereditary angioedema, Patient, Adolescence, Heredity, Country, National Congress, Physician, Medicine, Pharmaceutical industry

“We are committed to the continued advancement of our clinical development program of deucrictibant to fulfill unmet needs of current HAE treatment,” said Berndt Modig, Chief Executive Officer of Pharvaris.

Key Points: 
  • “We are committed to the continued advancement of our clinical development program of deucrictibant to fulfill unmet needs of current HAE treatment,” said Berndt Modig, Chief Executive Officer of Pharvaris.
  • The RAPIDe-3 study is designed to assess the effectiveness of deucrictibant in addressing that unmet need.
  • RAPIDe-3 is a randomized, double-blind, placebo-controlled, crossover study, which is designed to enroll approximately 120 adolescent and adult participants globally.
  • The data illustrates that HRQoL is negatively impacted, including functional and psychological impairment, in people with HAE.

Fulcrum Therapeutics Appoints Patrick Horn M.D., Ph.D., as Chief Medical Officer

Retrieved on: 
Monday, March 18, 2024
Sickle cell disease, University, Medicine, Doctor of Philosophy, Albireo, New Drug Application, Patient, SVP, Research, Losmapimod, Senior, Homocystinuria, Facioscapulohumeral muscular dystrophy, Therapy, DRS, Hereditary angioedema, Drug development, Interim, Boston, Fulcrum, PFIC, DSM-IV codes, Pharmaceutical industry

CAMBRIDGE, Mass., March 18, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced the appointment of Patrick Horn, M.D., Ph.D., as chief medical officer, effective immediately. Dr. Horn is a seasoned executive with over 20 years of end-to-end drug development experience spanning multiple therapeutic areas, with an emphasis on rare diseases, across both large pharmaceutical and biotech companies. Interim chief medical officer, Iain Fraser, MBChB, DPhil, will continue to serve on Fulcrum’s executive leadership team as SVP of early development. Together, Drs. Horn and Fraser will be responsible for leading clinical development and overseeing regulatory strategy and execution.

Key Points: 
  • Interim chief medical officer, Iain Fraser, MBChB, DPhil, will continue to serve on Fulcrum’s executive leadership team as SVP of early development.
  • Horn and Fraser will be responsible for leading clinical development and overseeing regulatory strategy and execution.
  • His most recent role was as the Chief Medical Officer at HemoShear Therapeutics, specializing in rare metabolic diseases.
  • Prior to transitioning to industry, Dr. Horn was a practicing pediatrician at major academic institutions in Chicago.

Astria Therapeutics Announces Positive Initial Proof-of-Concept Results from the ALPHA-STAR Phase 1b/2 Trial of STAR-0215 for HAE

Retrieved on: 
Monday, March 25, 2024
Biotechnology, Infectious Diseases, Health, Pharmaceutical, Clinical Trials, Interest, Dizziness, Attack, Disease, PD, Webcast, Patient, HAE, Allergy, Quality of life, IND, Injection site reaction, Rash, Plasma kallikrein, Investment, Therapy, Hereditary angioedema, Safety, Charité, Pharmacokinetics, Pain, Multimedia, Pharmaceutical industry

Based on the positive results, Astria plans to advance STAR-0215 to Phase 3 development with trial initiation expected in Q1 2025 and top-line results expected by year-end 2026.

Key Points: 
  • Based on the positive results, Astria plans to advance STAR-0215 to Phase 3 development with trial initiation expected in Q1 2025 and top-line results expected by year-end 2026.
  • Initial safety and efficacy data from Q3M and Q6M dosing in the ALPHA-SOLAR trial are expected mid-2025.
  • Pending regulatory feedback, the Company expects to start a pivotal Q3M Phase 3 trial in Q1 2025, with top-line results expected by year-end 2026.
  • Interested parties may join the webcast via the Investors section of the Astria website, www.astriatx.com or with following the link https://lifescievents.com/event/astriatx/ .

KalVista Pharmaceuticals Presents Real-World Data on Burden of Treatment and HAE Attack Journey at the 2024 HAEi Regional Conference Americas

Retrieved on: 
Monday, March 18, 2024
Health, General Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Conference, Disease, Patient, LTP, Hereditary angioedema, HAE, Quality of life, Anxiety, University, KALV, Return, Treatment, Quality, Asthma, Pharmaceutical industry

KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced that it presented multiple analyses of the relationship between the time to attack treatment and the effects delays in treatment have on clinical outcomes of people living with hereditary angioedema (HAE) at the 2024 HAEi Regional Conference Americas that took place in Panama City, Panama.

Key Points: 
  • KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced that it presented multiple analyses of the relationship between the time to attack treatment and the effects delays in treatment have on clinical outcomes of people living with hereditary angioedema (HAE) at the 2024 HAEi Regional Conference Americas that took place in Panama City, Panama.
  • “These data show once again that people living with HAE still face challenges in following established guidelines for treating their attacks,” said Ben Palleiko, Chief Executive Officer of KalVista.
  • “Even short treatment delays can lead to worse clinical outcomes, and despite efficacious on-demand treatments, the fact that they need to be injected results in barriers which lead to significant underuse and resultant overuse of long-term prophylaxis.
  • (Poster Presentation)
    Anxiety Associated with On-Demand Treatment for Hereditary Angioedema (HAE) Attacks: Maeve O’Connor, Allergy, Asthma, & Immunology Relief of Charlotte, Charlotte, NC, United States (Poster Presentation)
    Both adults and adolescents with HAE reported moderate to extreme anxiety when anticipating use of parenteral on-demand treatment, irrespective of use of on-demand only or on-demand plus LTP
    Characteristics of Hereditary Angioedema Attacks Among Long-Term Prophylaxis Users: Maeve O’Connor, Allergy, Asthma, & Immunology Relief of Charlotte, Charlotte, NC, United States (Poster Presentation)
    Among HAE patients who had treated a recent attack, the location and duration of the most recent attacks were similar between long-term prophylaxis (LTP) and on-demand only users

KalVista Pharmaceuticals Awarded UK Promising Innovative Medicine (PIM) Designation for Sebetralstat

Retrieved on: 
Tuesday, March 12, 2024
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, EAMS, Patient, HAE, National Health Service, Health care, NHS, American Academy, KALV, Expanded access, Marketing, MHRA, PIM, Hereditary angioedema, Safety, AAAAI, Pharmaceutical industry

KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced the UK Medicines and Healthcare products Regulatory Agency (MHRA) has awarded the Promising Innovative Medicine (PIM) designation for sebetralstat, an investigational novel, oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE).

Key Points: 
  • KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced the UK Medicines and Healthcare products Regulatory Agency (MHRA) has awarded the Promising Innovative Medicine (PIM) designation for sebetralstat, an investigational novel, oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE).
  • The PIM is the first step in the Early Access to Medicines Scheme (EAMS) which would allow KalVista to treat patients with sebetralstat prior to receiving a Marketing Authorisation.
  • “We are proud to have sebetralstat designated as a Promising Innovative Medicine by the MHRA, which is similar to Expanded Access in the US,” said Ben Palleiko, Chief Executive Officer of KalVista.
  • KalVista recently provided phase 3 data for sebetralstat at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting, which displayed clinically and statistically significant results across all endpoints, and an excellent safety and tolerability profile.

KalVista Pharmaceuticals Reports Third Fiscal Quarter Results and Provides Operational Update

Retrieved on: 
Monday, March 11, 2024
Health, Pharmaceutical, Research, CFO, Patient, HAE, Health care, CBO, NDA, American Academy, KALV, Factor, MHRA, Clinical trial, Hereditary angioedema, Safety, Pharmacology, AAAAI, Fiscal year, Food, Pharmaceutical industry

(NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today provided an operational update and released financial results for the third fiscal quarter ended January 31, 2024.

Key Points: 
  • (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today provided an operational update and released financial results for the third fiscal quarter ended January 31, 2024.
  • Third Fiscal Quarter Financial Results:
    Revenue: No revenue was recognized for the three months ended January 31, 2024, or January 31, 2023.
  • The increase in R&D expenses during the quarter primarily reflects the ongoing Phase 3 KONFIDENT and KONFIDENT-S trials.
  • The decrease in the net cash and marketable securities position was due to cash consumption from operating expenses.

Pharvaris to Present Deucrictibant Clinical Data at Upcoming Congresses

Retrieved on: 
Wednesday, March 6, 2024
Bradykinin receptor B2, Hereditary angioedema, Real, Safety, Heredity, Treatment, Conference, HAE, Network, Abstract, National Congress, Pharmaceutical industry

ZUG, Switzerland, March 06, 2024 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a clinical-stage company developing novel, oral bradykinin B2 receptor antagonists to treat and prevent hereditary angioedema (HAE) attacks, today announced the acceptance of abstracts for presentation at two upcoming congresses: the 3rd National Congress of the Italian Network for Hereditary and Acquired Angioedema (ITACA) , to be held from March 14-16, 2024, in Milan, Italy, and the 2024 HAE International (HAEi) Regional Conference Americas , to be held from March 15-17, 2024, in Panama City, Panama.

Key Points: 
  • ZUG, Switzerland, March 06, 2024 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a clinical-stage company developing novel, oral bradykinin B2 receptor antagonists to treat and prevent hereditary angioedema (HAE) attacks, today announced the acceptance of abstracts for presentation at two upcoming congresses: the 3rd National Congress of the Italian Network for Hereditary and Acquired Angioedema (ITACA) , to be held from March 14-16, 2024, in Milan, Italy, and the 2024 HAE International (HAEi) Regional Conference Americas , to be held from March 15-17, 2024, in Panama City, Panama.
  • Details of the presentations are as follows:
    Title: Design of RAPIDe-3 Phase 3 Trial: Efficacy and Safety of the Oral Bradykinin B2 Receptor Antagonist Deucrictibant Immediate-Release Capsule in Treatment of Hereditary Angioedema Attacks
    Title: Efficacy and Safety of Bradykinin B2 Receptor Antagonism with Deucrictibant Immediate-Release Capsule for Treatment of Hereditary Angioedema Attacks: Results of RAPIDe-1 Phase 2 Trial
    Title: Efficacy and Safety of Oral Deucrictibant, a Bradykinin B2 Receptor Antagonist, in Prophylaxis of Hereditary Angioedema Attacks: Results of CHAPTER-1 Phase 2 Trial
    2024 HAEi Regional Conference Americas, Panama City, Panama, March 15-17, 2024.
  • Title: Efficacy and Safety of the Oral Bradykinin B2 Receptor Antagonist Deucrictibant Immediate-Release Capsule in Treatment of Hereditary Angioedema Attacks: RAPIDe-3 Phase 3 Trial Design
    Title: Efficacy and Safety of Bradykinin B2 Receptor Antagonism with Deucrictibant Immediate-Release Capsule for Treatment of Hereditary Angioedema Attacks: Results of RAPIDe-1 Phase 2 Trial
    Title: Efficacy and Safety of Oral Deucrictibant, a Potent Bradykinin B2 Receptor Antagonist, in Prophylaxis of Hereditary Angioedema Attacks: Results of CHAPTER-1 Phase 2 Trial
    Title: Understanding Patient Reasons not to Treat All Hereditary Angioedema (HAE) Attacks and Characteristics of Untreated HAE Attacks: Results from a Real World Survey
    The posters and presentation slides will be made available on the Investors section of the Pharvaris website at the beginning of the respective presentations at: https://ir.pharvaris.com/news-events/events-presentations .

Pharvaris to Participate in the Leerink Global Biopharma Conference 2024

Retrieved on: 
Tuesday, March 5, 2024
Fontainebleau Miami Beach, HAE, Hereditary angioedema, Conference

ZUG, Switzerland, March 05, 2024 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a clinical-stage company developing novel, oral bradykinin B2 receptor antagonists to treat and prevent hereditary angioedema (HAE) attacks, today announced that its management will participate in the Leerink Global Biopharma Conference 2024, taking place from March 11-13, 2024, at the Fontainebleau Miami Beach, Miami.

Key Points: 
  • ZUG, Switzerland, March 05, 2024 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a clinical-stage company developing novel, oral bradykinin B2 receptor antagonists to treat and prevent hereditary angioedema (HAE) attacks, today announced that its management will participate in the Leerink Global Biopharma Conference 2024, taking place from March 11-13, 2024, at the Fontainebleau Miami Beach, Miami.
  • Details are as follows:
    A live audio webcast will be available on the Investors section of the Pharvaris website at: https://ir.pharvaris.com/news-events/events-presentations .
  • The audio replay will be available on Pharvaris’ website for 30 days following the presentation.