Hereditary angioedema

BioCryst Announces Publication of Data from Open-label Extension of the APeX-2 Pivotal Trial of ORLADEYO® (berotralstat)

Retrieved on: 
Tuesday, December 19, 2023

“We are pleased to share long-term efficacy and safety data from APeX-2 as published in JACI: In Practice.

Key Points: 
  • “We are pleased to share long-term efficacy and safety data from APeX-2 as published in JACI: In Practice.
  • These data further illustrate the potential lasting outcomes that can be appreciated by patients who are treated with oral, once-daily ORLADEYO.
  • In part 3 of the study (weeks 49-96), all patients (n=81) were treated with open-label ORLADEYO at 150 mg.
  • JACI: In Practice is an official journal of the American Academy of Allergy, Asthma, and Immunology (AAAAI).

Pharvaris Presents Deucrictibant Clinical Data and Analysis of Endpoints for Trials of On-demand Treatment of HAE at the GA²LEN UCARE Conference 2023

Retrieved on: 
Friday, December 8, 2023

Prof. Markus Magerl, M.D., presented an oral session titled “ Treatment of HAE Attacks with Deucrictibant: RAPIDe-1 Phase 2 Trial Results ” on Friday, December 8, 11:18-11:26 a.m. BST (9:18-9:26 a.m. EST).

Key Points: 
  • Prof. Markus Magerl, M.D., presented an oral session titled “ Treatment of HAE Attacks with Deucrictibant: RAPIDe-1 Phase 2 Trial Results ” on Friday, December 8, 11:18-11:26 a.m. BST (9:18-9:26 a.m. EST).
  • Prof. Marcus Maurer, M.D., presented a poster titled “ Early-Onset Response to Treatment of Hereditary Angioedema Attacks with Deucrictibant ” on Friday, December 8, 7:00-8:00 p.m. BST (5:00-6:00 p.m. EST).
  • Primary and post-hoc analyses of the RAPIDe-1 study were conducted to evaluate end of progression (EoP) and symptom relief in response to treatment of HAE attacks with PHVS416.
  • The onset of symptom relief was achieved at approximately two hours and clinically meaningful improvement within two hours after administration of deucrictibant.

Pharvaris Announces Pricing of $300 Million Underwritten Offering of Ordinary Shares and Pre-funded Warrants

Retrieved on: 
Wednesday, December 6, 2023

The gross proceeds to Pharvaris from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be approximately $300 million.

Key Points: 
  • The gross proceeds to Pharvaris from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be approximately $300 million.
  • All shares and pre-funded warrants in the offering are to be sold by Pharvaris.
  • The offering is expected to close on or about December 8, 2023, subject to satisfaction of customary closing conditions.
  • The shares and pre-funded warrants are being offered by Pharvaris pursuant to an effective shelf registration statement that was previously filed with the U.S. Securities and Exchange Commission (the “SEC”).

Pharvaris Announces Positive Top-line Phase 2 Data from the CHAPTER-1 Study of Deucrictibant for the Prophylactic Treatment of HAE Attacks

Retrieved on: 
Wednesday, December 6, 2023

CHAPTER-1 ( NCT05047185 ) is a double-blind, placebo-controlled Phase 2 study evaluating the efficacy as well as the safety and tolerability of deucrictibant for long-term prophylaxis against angioedema attacks in HAE-1/2.

Key Points: 
  • CHAPTER-1 ( NCT05047185 ) is a double-blind, placebo-controlled Phase 2 study evaluating the efficacy as well as the safety and tolerability of deucrictibant for long-term prophylaxis against angioedema attacks in HAE-1/2.
  • In the study, 34 participants were enrolled globally and randomized to receive one of two doses of deucrictibant (20 mg/day or 40 mg/day) or placebo for 12 weeks of treatment.
  • Deucrictibant immediate-release capsule (PHVS416) was dosed twice-a-day as a proof-of-concept for the once-daily deucrictibant extended-release tablet (PHVS719), which is the intended formulation for the prophylactic treatment of HAE.
  • The study’s primary endpoint measured the time-normalized number of investigator-confirmed HAE attacks during the treatment period.

Pharvaris to Present at the GA²LEN UCARE Conference 2023

Retrieved on: 
Thursday, November 30, 2023

ZUG, Switzerland, Nov. 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 one abstract for oral presentation and two abstracts for poster presentation at the GA²LEN UCARE Conference 2023, to be held from December 7-9, 2023, at the Rebouças Convention Center in São Paulo, Brazil.

Key Points: 
  • ZUG, Switzerland, Nov. 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 one abstract for oral presentation and two abstracts for poster presentation at the GA²LEN UCARE Conference 2023, to be held from December 7-9, 2023, at the Rebouças Convention Center in São Paulo, Brazil.
  • Title: Treatment of HAE Attacks with Deucrictibant: RAPIDe-1 Phase2 Trial Results
    Pharvaris is a bronze level sponsor of the GA²LEN UCARE Conference 2023.
  • Following the close of the respective sessions, the presentation slides and posters will be made available on the Investors section of the Pharvaris website at: https://ir.pharvaris.com/news-events/events-presentations .

BioCryst Announces Approval of ORLADEYO® (berotralstat) by the National Administration of Drugs, Foods, and Medical Devices (ANMAT) in Argentina

Retrieved on: 
Wednesday, November 29, 2023

“We continue to make strides to bring ORLADEYO to patients living with HAE in Latin America in collaboration with our partner, Pint Pharma.

Key Points: 
  • “We continue to make strides to bring ORLADEYO to patients living with HAE in Latin America in collaboration with our partner, Pint Pharma.
  • We applaud ANMAT’s decision to grant approval to our oral, once-daily prophylactic therapy for HAE, which paves the way for patients living with HAE to receive ORLADEYO to help improve management of their HAE attacks,” said Charlie Gayer, chief commercial officer of BioCryst.
  • BioCryst has an exclusive collaboration with Pint Pharma GmbH to register and promote ORLADEYO in the pan-Latin America region.
  • Under the terms of the agreement, Pint is responsible for obtaining and maintaining all marketing authorizations and for commercializing ORLADEYO in the region.

BioCryst Launches ORLADEYO® (berotralstat) in Spain

Retrieved on: 
Tuesday, November 21, 2023

RESEARCH TRIANGLE PARK, N.C., Nov. 21, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Spanish Ministry for Health (Ministerio de Sanidad) has granted marketing authorization for oral, once-daily ORLADEYO® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older.

Key Points: 
  • RESEARCH TRIANGLE PARK, N.C., Nov. 21, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Spanish Ministry for Health (Ministerio de Sanidad) has granted marketing authorization for oral, once-daily ORLADEYO® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older.
  • “We have an experienced and motivated team in Spain that is enthusiastic about the impact the availability of the first oral, once daily therapy can have for people with HAE in Spain,” said Charlie Gayer, chief commercial officer of BioCryst.
  • “Spanish patients and physicians have shared with us the need to expand treatment options and we are excited now to be in a position to support access to those who can benefit most.”

Ionis announces European licensing agreement with Otsuka for donidalorsen in hereditary angioedema

Retrieved on: 
Monday, December 18, 2023

Ionis plans to independently bring donidalorsen to U.S. patients if approved

Key Points: 
  • Ionis plans to independently bring donidalorsen to U.S. patients if approved
    CARLSBAD, Calif., Dec. 18, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has entered into a license agreement with Otsuka Pharmaceutical Co., Ltd. (Otsuka) under which Otsuka obtains exclusive rights in Europe to commercialize donidalorsen, an investigational prophylactic treatment for hereditary angioedema (HAE).
  • Ionis will maintain responsibility for the non-clinical and clinical development of donidalorsen, and Otsuka will be responsible for European regulatory filings and commercialization.
  • Ionis plans to independently launch donidalorsen in the U.S. if approved, as part of the company's strategy to deliver a steady flow of wholly owned medicines to patients.
  • The company plans to report Phase 3 results with donidalorsen for prophylactic treatment of HAE in the first half of 2024.

Pharvaris Appoints Stefan Abele, Ph.D., as Chief Technical Operations Officer

Retrieved on: 
Wednesday, November 15, 2023

ZUG, Switzerland, Nov. 15, 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 appointment of Stefan Abele, Ph.D., as Chief Technical Operations Officer.

Key Points: 
  • ZUG, Switzerland, Nov. 15, 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 appointment of Stefan Abele, Ph.D., as Chief Technical Operations Officer.
  • In this role, he will be responsible for all chemistry, manufacturing, and controls (CMC) activities, supply chain, and program management as Pharvaris progresses into late-stage clinical development.
  • Dr. Abele joins Pharvaris with more than 20 years of experience in process development, end-to-end Active Pharmaceutical Ingredients (API) supply chain, cross-functional CMC activities, GMP manufacturing, global vendor management, and people leadership.
  • “Stefan joins Pharvaris at a transformative time as we prepare to transition into a late-stage clinical company and prepare for commercialization in the coming years,” said Berndt Modig, Chief Executive Officer of Pharvaris.

Intellia Therapeutics Receives European Union Orphan Drug Designation for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema

Retrieved on: 
Tuesday, November 14, 2023

CAMBRIDGE, Mass., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative single-dose therapeutics leveraging CRISPR-based technologies, today announced that the European Commission (EC) has granted orphan drug designation to NTLA-2002 for the treatment of hereditary angioedema (HAE).

Key Points: 
  • CAMBRIDGE, Mass., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative single-dose therapeutics leveraging CRISPR-based technologies, today announced that the European Commission (EC) has granted orphan drug 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.
  • “The European Union orphan drug designation for NTLA-2002 represents another important milestone for Intellia as we continue to make rapid progress in the development of a novel, potential one-time treatment for people with hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D.
  • NTLA-2002 was also granted Orphan Drug Designation and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K.