HAE

BioCryst R&D Day Highlights New Diversified Pipeline of First-in-Class/Best-in-Class Therapies with Five Programs Expected in Clinical Development in Next 24 Months

Retrieved on: 
Friday, November 3, 2023
GMG, BioCryst Pharmaceuticals, FDA, Research, Patient, Drug design, IgA nephropathy, Plasma, CAD, Lupus nephritis, KLK5, Genetic disorder, RESEARCH, C2, DME, Eculizumab, Infection, Injection, Skin, Diabetes, Autoantibody, Ryan Murphy, Edema, Netherton, R, Autoimmune hemolytic anemia, HAE, Kallikrein, Growth, Disease, Dehydration, Muscle weakness, Immune system, Hemolysis, Solubility, Netherton syndrome, Clinical trial, Paratriathlon, Eye, Ravulizumab, Cold agglutinin disease, PARK, IP, SPINK5, Fine chemical, Pharmaceutical industry, Vaccine

A live webcast of the event will be available online in the investors section of the company website at www.biocryst.com .

Key Points: 
  • A live webcast of the event will be available online in the investors section of the company website at www.biocryst.com .
  • The team is excited to share our approach, and our expanded pipeline of new programs, at our R&D Day,” said Jon Stonehouse, president and chief executive officer of BioCryst.
  • The disease is caused by the deficiency of a natural inhibitor (SPINK5) of KLK5, a serine protease responsible for regulating skin shedding.
  • Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections.

BioCryst and Clearside Biomedical Enter Partnership to Develop Avoralstat for Diabetic Macular Edema Using Clearside’s Proprietary SCS Microinjector®

Retrieved on: 
Friday, November 3, 2023
BioCryst Pharmaceuticals, SCS, Research, Research and development, Plasma, RESEARCH, DME, Diabetes, Hereditary angioedema, Edema, R, HAE, Pharming, Diabetic retinopathy, Nature and Wildlife Discovery Center, Paratriathlon, Eye, PARK, Patient, Solubility, Pharmaceutical industry, Clearside

ALPHARETTA, Ga. and RESEARCH TRIANGLE PARK, N.C., Nov. 03, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) and Clearside Biomedical, Inc. (Nasdaq: CLSD) today announced the entry into a license agreement enabling BioCryst to develop its investigational plasma kallikrein inhibitor, avoralstat, with Clearside’s SCS Microinjector® to deliver avoralstat directly to the back of the eye through the suprachoroidal space to treat patients with diabetic macular edema (DME).

Key Points: 
  • “Many DME patients are not effectively controlled with anti-VEGF therapy, so it is exciting to target plasma kallikrein by combining avoralstat with delivery into the suprachoroidal space using Clearside’s proprietary SCS Microinjector.
  • Avoralstat has high potency and low solubility, which are two characteristics important to achieving potential efficacy with reduced dosing frequency in the eye for DME patients.
  • Avoralstat was previously evaluated in an oral formulation in a Phase 3 clinical trial in patients with hereditary angioedema (HAE).
  • “Treating patients with DME by delivering avoralstat directly to the suprachoroidal space using the SCS Microinjector expands the reach of our proprietary SCS injection platform to a greater number of patients.

Pharvaris Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, November 2, 2023
FDA, Research and development, Trial of the century, IND, American College, Association of International Accountants, Food, 2023 International Conference on Climate Resilient Pakistan, ACAAI, Conference, Asthma, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Hereditary angioedema, R, Bank statement, HAE, American College of Allergy, Asthma and Immunology, ACIS, Abstract, Record, Safety, Attack, Patient, International Accounting Standards Board, Communication, Book, Administration, CIIC, Pharmaceutical industry, Cryptocurrency, Allergy

Results of nonclinical toxicology study on-track to be submitted to the FDA by the end of 2023.

Key Points: 
  • Results of nonclinical toxicology study on-track to be submitted to the FDA by the end of 2023.
  • R&D expenses were €18.5 million for the quarter ended September 30, 2023, compared to €14.1 million for the quarter ended September 30, 2022.
  • G&A expenses were €7.7 million for the quarter ended September 30, 2023, compared to €8.3 million for the quarter ended September 30, 2022.
  • Details are as follows:
    Pharvaris is a Foreign Private Issuer and prepares and reports consolidated financial statements and financial information in accordance with IFRS as issued by the International Accounting Standards Board.

BioCryst Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, November 2, 2023
HAE, BioCryst Pharmaceuticals, RESEARCH, Growth, PARK, Patient, Pharmaceutical industry

RESEARCH TRIANGLE PARK, N.C., Nov. 02, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today reported financial results for the third quarter ended September 30, 2023, and provided a corporate update.

Key Points: 
  • - Company to host R&D Day on Friday, November 3rd to introduce additional therapies from its pipeline -
    RESEARCH TRIANGLE PARK, N.C., Nov. 02, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today reported financial results for the third quarter ended September 30, 2023, and provided a corporate update.
  • Selling, general and administrative expenses for the third quarter of 2023 increased to $50.7 million, compared to $36.9 million in the third quarter of 2022 (+37.1 percent y-o-y).
  • Interest expense was $27.3 million in the third quarter of 2023, compared to $24.8 million in the third quarter of 2022 (+10.1 percent y-o-y).
  • ET today to discuss the financial results and provide a corporate update.

Pharvaris To Present at the ACAAI 2023 Annual Scientific Meeting

Retrieved on: 
Monday, October 30, 2023
HAE, ACAAI, Abstract, EST, American College, PST, Anaheim Convention Center, Hereditary angioedema, American College of Allergy, Asthma and Immunology, Pharmaceutical industry, Mobile phone

ZUG, Switzerland, Oct. 30, 2023 (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 two abstracts for ePoster presentation at the American College of Allergy, Asthma & Immunology (ACAAI) 2023 Annual Scientific Meeting, to be held from November 9-13, 2023, at the Anaheim Convention Center in Anaheim, CA.

Key Points: 
  • ZUG, Switzerland, Oct. 30, 2023 (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 two abstracts for ePoster presentation at the American College of Allergy, Asthma & Immunology (ACAAI) 2023 Annual Scientific Meeting, to be held from November 9-13, 2023, at the Anaheim Convention Center in Anaheim, CA.
  • On November 9 at 8:30 a.m. PST (11:30 a.m. EST), the ePosters and accompanying audio voice-overs will be made available on the Investors section of the Pharvaris website at: https://ir.pharvaris.com/news-events/events-presentations .

KalVista Pharmaceuticals to Present Data at the 2023 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology

Retrieved on: 
Thursday, November 2, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Princess, Patient, Them, Q&A, American College, KALV, Monitor, ACAAI, HAE, Real, Convention, Abstract, Hereditary angioedema, 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 acceptance of multiple abstracts at the 2023 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), taking place in Anaheim, CA from November 9-13.

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 acceptance of multiple abstracts at the 2023 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), taking place in Anaheim, CA from November 9-13.
  • The presentations are:
    Understanding Why Hereditary Angioedema Patients Often Do Not Carry Their On-Demand Treatment with Them - #P078: Bob Geng, Sally van Kooten, Markus Heckmann, Sherry Danese, Ledia Goga, Cristine Radojicic.
  • Results shared as an oral poster presentation and Q&A on Friday, November 10 from 4:30 - 4:45 pm PT at Monitor 13
    Treatment of Hereditary Angioedema (HAE) with Normal C1-INH (HAE-nC1-INH) –A Real-World ACARE Survey - #P059: Marcus Maurer, Markus Magerl, Marc A. Riedl, Sherry Danese, Julie Ulloa, Vibha Desai, Paul K. Audhya.
  • Results shared as an oral poster presentation and Q&A on Sunday, November 12 from 12:20 - 12:35 pm PT at Monitor 13
    All poster presentations will take place in the ePoster Section of the Exhibit Hall.

BioCryst to Present New ORLADEYO® (berotralstat) Data at Annual Scientific Meeting of American College of Allergy, Asthma & Immunology

Retrieved on: 
Friday, October 27, 2023
American College, Anaheim Convention Center, ACAAI, RESEARCH, Hereditary angioedema, HAE, BioCryst Pharmaceuticals, Convention, Abstract, PARK, Pharmaceutical industry, Nightclub, Patient

RESEARCH TRIANGLE PARK, N.C., Oct. 27, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company will present two abstracts on oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI).

Key Points: 
  • RESEARCH TRIANGLE PARK, N.C., Oct. 27, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company will present two abstracts on oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI).
  • The meeting will take place at the Anaheim Convention Center in Anaheim, Calif., from November 9-13, 2023.
  • Berotralstat Reduced Attack Rates in Patients with Hereditary Angioedema with Normal C1-Inhibitor: Real-World Outcomes; ePoster #P082; Friday, November 10, 5:30-5:45 p.m. PT; Monitor #13, Exhibit Hall
    Berotralstat Reduced Attack Rates Compared to Baseline in Patients with Hereditary Angioedema in APeX-S; ePoster #P064; Saturday, November 11, 12:05-12:20 p.m. PT; Monitor #12, Exhibit Hall
    In addition to displaying in the exhibit hall at the noted times, ePosters will be accessible online and on demand to registered attendees on Thursday, November 9, beginning at 8:00 a.m. PT on ACAAI’s website.

KalVista Pharmaceuticals Presents Data on HAE Attacks in Patients Receiving Long-Term Prophylaxis at the Asia Pacific Association of Allergy, Asthma and Clinical Immunology 2023 International Conference

Retrieved on: 
Tuesday, October 24, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, HAE, Conference, Asthma, LTP, Patient, KALV, Hereditary angioedema, International Conference on Population and Development, 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 presentation of a novel analysis characterizing hereditary angioedema (HAE) attacks occurring in patients receiving long-term prophylaxis (LTP) treatments at the Asia Pacific Association of Allergy, Asthma and Clinical Immunology 2023 International Conference (APAAACI) in Singapore.

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 presentation of a novel analysis characterizing hereditary angioedema (HAE) attacks occurring in patients receiving long-term prophylaxis (LTP) treatments at the Asia Pacific Association of Allergy, Asthma and Clinical Immunology 2023 International Conference (APAAACI) in Singapore.
  • A systematic literature review of interventional and observational LTP studies revealed that although LTP yields significant reductions in HAE attack frequency, many patients continue to experience attacks in all anatomic locations, including potentially life-threatening laryngeal attacks.
  • “As highlighted in HAE clinical practice guidelines, the goal of LTP is to achieve full control of disease burden.
  • “Access to safe and effective on-demand therapy is essential for all people living with HAE, including those receiving LTP.

Intellia Therapeutics Receives Priority Medicines (PRIME) Designation From the European Medicines Agency for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema

Retrieved on: 
Friday, October 13, 2023
Intellia Therapeutics, Health care, HAE, Food, NTLA, European Medicines Agency, EMA, Patient, MHRA, FDA, Therapy, CRISPR, Hereditary angioedema, PRIME, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Orphan, Pharmaceutical industry

CAMBRIDGE, Mass., Oct. 13, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to NTLA-2002 for the treatment of hereditary angioedema (HAE).

Key Points: 
  • CAMBRIDGE, Mass., Oct. 13, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to NTLA-2002 for the treatment of hereditary angioedema (HAE).
  • NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to prevent potentially life-threatening swelling attacks in people with HAE.
  • “We are very pleased the EMA has granted PRIME designation to NTLA-2002.
  • PRIME is the fourth specialty regulatory designation Intellia has received for NTLA-2002.

Pharvaris To Present at the APAAACI 2023 International Conference

Retrieved on: 
Friday, October 13, 2023
HAE, Attack, International Conference on Learning Representations, ACIS, Asthma, Hereditary angioedema, Convention center, Conference, Pharmaceutical industry, Allergy

ZUG, Switzerland, Oct. 13, 2023 (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 an abstract oral presentation at the Asia Pacific Association of Allergy, Asthma, and Clinical Immunology (APAAACI) and Allergy and Clinical Immunology Society of Singapore (ACIS) APAAACI 2023 International Conference, to be held from October 23-26, 2023, at the Suntec Singapore International Convention and Exhibition Center in Singapore.

Key Points: 
  • ZUG, Switzerland, Oct. 13, 2023 (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 an abstract oral presentation at the Asia Pacific Association of Allergy, Asthma, and Clinical Immunology (APAAACI) and Allergy and Clinical Immunology Society of Singapore (ACIS) APAAACI 2023 International Conference, to be held from October 23-26, 2023, at the Suntec Singapore International Convention and Exhibition Center in Singapore.
  • Title: Bradykinin B2 receptor antagonist deucrictibant immediate-release capsule for treatment of HAE attacks: Phase 2 results
    Following the close of the session, the slide presentation will be made available on the Investors section of the Pharvaris website at: https://ir.pharvaris.com/news-events/events-presentations .