HAE

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
Hereditary angioedema, EST, Quality of life, Caregiver, Attack, Angioedema, Trial of the century, Clinical trial, University, UCSD, Safety, HAE, Pharmaceutical industry

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
Hereditary angioedema, Conference, Paulo, Abstract, HAE, Pharmaceutical industry

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
Marketing, Hereditary angioedema, Medical device, BioCryst Pharmaceuticals, Attack, National government, Pint, Patient, HAE, Pharmaceutical industry

“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
Hereditary angioedema, Physician, BioCryst Pharmaceuticals, Patient, HAE, Pharmaceutical industry

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.”

KalVista Pharmaceuticals Announces Publication of First Oral Factor XIIa Data in Frontiers in Pharmacology

Retrieved on: 
Wednesday, December 20, 2023
Health, Other Science, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Biology, KALV, KKS, System, HAE, Frontiers, 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 data from the first report of an oral, potent, and selective FXIIa inhibitor has been published in the peer-reviewed scientific journal, Frontiers in Pharmacology.

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 data from the first report of an oral, potent, and selective FXIIa inhibitor has been published in the peer-reviewed scientific journal, Frontiers in Pharmacology.
  • The article describes the pharmacology of a representative compound from KalVista’s portfolio of structurally diverse, oral Factor XII inhibitors, including:
    Potent, selective inhibition of both FXII zymogen and FXIIa, thereby suppressing both the initiation and amplification of kallikrein-kinin system (KKS) activation
    “The publication is the first report of a potent and specific FXIIa inhibitor with high oral availability in multiple species and demonstrated efficacy in HAE models in vivo,” said Andrew Crockett, Chief Executive Officer of KalVista.
  • “This once again demonstrates our scientific leadership in medicinal chemistry and KKS biology.
  • It also provides new insight on the inhibition of FXII zymogen.”
    The publication abstract can be found at the below link:

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

Retrieved on: 
Monday, December 18, 2023
RNA, Hereditary angioedema, Ionis Pharmaceuticals, Orphan, ADPKD, HAE, Patient, IONS, OLE, Pharmaceutical industry

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
Abele, GMP, Senior, D, Head, Commercial, Dishman Carbogen Amcis, Doctor of Philosophy, API, Organic, QUVIVIQ, ETH Zurich, HAE, University, Hereditary angioedema, CMC, Actelion, Active ingredient, University of Konstanz, Production, Chemistry, Idorsia

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
Intellia Therapeutics, Food, European Medicines Agency, Therapy, European Commission, PRIME, NTLA, MHRA, Disease, EMA, Civil service commission, European, FDA, Committee, HAE, Hereditary angioedema, Diagnosis, CRISPR, Health care, Orphan, Pharmaceutical industry

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.

Orchard Therapeutics Reports Third Quarter 2023 Financial Results and Highlights Recent Business Accomplishments

Retrieved on: 
Monday, November 13, 2023
Infant, Acquisition, HSC, Investment, Patient, Cell, ADS, ESGCT, U.S. FDA, FTD, Marketing, ICER, Diagnosis, R, Congress, Priority review, CVR, Food, Public, MLD, Abstract, Public health, Biomarker, MPS, Therapy, BLA, Gene, Health, HAE, Frontotemporal dementia, Hurler, Research, Disease, FDA, American depositary receipt, File, Hereditary angioedema, CTA, PDUFA, Society, Kyowa Kirin, Pharmaceutical industry, Security (finance), Fine chemical, Mobile phone, Vaccine, Dietary supplement, Cryptocurrency, Metabolism, Orchard, Atidarsagene autotemcel

Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024

Key Points: 
  • Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024
    BOSTON and LONDON, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced several business accomplishments along with its financial results for the quarter ended September 30, 2023.
  • Following the completion of the acquisition, Orchard Therapeutics will become a wholly-owned subsidiary of Kyowa Kirin.
  • The company had approximately 227.3 million ordinary shares, equivalent to 22.7 million American Depositary Shares, outstanding as of September 30, 2023.
  • As a result of the anticipated acquisition by Kyowa Kirin, Orchard Therapeutics has removed its financial guidance around Libmeldy revenue and anticipated cash runway.

BioCryst Presents New Real-world Data Showing Reduced Attack Rates in Patients with HAE with Normal C1-inhibitor Following Long-term Treatment with ORLADEYO® (berotralstat)

Retrieved on: 
Friday, November 10, 2023
Medicine, Anaheim Convention Center, SEM, Safety, BioCryst Pharmaceuticals, Attack, Month, Lanadelumab, Convention, American College, Androgen, Hereditary angioedema, HAE, Day, RESEARCH, Allergy, ACAAI, Pharmaceutical industry, Nightclub, Patient

These real-world observations suggest ORLADEYO can have a meaningful impact on the lives of people who have HAE with normal C1-INH.

Key Points: 
  • These real-world observations suggest ORLADEYO can have a meaningful impact on the lives of people who have HAE with normal C1-INH.
  • The company also announced a new post-hoc analysis from the APeX-S clinical trial that showed a sustained reduction in HAE attacks compared to patients’ self-reported baseline attack rates.
  • “The ability to compare patients’ treatment outcomes with their HAE attack rates at baseline is tremendously helpful to characterize the impact of a prophylactic therapy.
  • Patients who were treated with ORLADEYO experienced a sustained reduction in HAE attacks compared to their self-reported baseline attack rates, suggesting a reduction in disease burden and durable treatment effect.