HAE

KalVista Pharmaceuticals Reports Second Fiscal Quarter Results and Provides Operational Update

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Thursday, December 7, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, NDA, LTP, Research, Food, D, ODS, Asthma, Patient, European, New Drug Application, FDA, American College, Conference, KALV, HAE, Pharmaceutical industry, Allergy

(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 second fiscal quarter ended October 31, 2023.

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 second fiscal quarter ended October 31, 2023.
  • The increase in R&D expenses during the quarter primarily reflects the ongoing Phase 3 KONFIDENT and KONFIDENT-S trials.
  • The increase in G&A expenses was primarily due to increases in employee-related expenses, commercial strategy and supply chain expenses.
  • The decrease in the net cash and marketable securities position was due to cash consumption from operating expenses.

KalVista Pharmaceuticals Presents Real-World Data at the 2023 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology

Retrieved on: 
Tuesday, November 14, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Syringe, Real, Attack, Asthma, Lung compliance, Patient, Division, American College, Internal medicine, Hereditary angioedema, Anxiety, KALV, HAE, University, Pain, University of California, Freie Berliner Kunstausstellung, ACAAI, Rheumatology, Immunology

“We continue to learn from people living with HAE that currently available parenteral on-demand treatments are simply not meeting their needs,” said Andrew Crockett, Chief Executive Officer of KalVista.

Key Points: 
  • “We continue to learn from people living with HAE that currently available parenteral on-demand treatments are simply not meeting their needs,” said Andrew Crockett, Chief Executive Officer of KalVista.
  • “Whether this is related to always carrying their syringes or vials, challenges in preparing and administering injectable or infused treatments outside the home, or anxiety related to potential injection-site reactions and pain, patients struggle to follow treatment guidelines.
  • This can lead to non-compliance and preference for long-term prophylaxis, even in cases where on-demand treatments would otherwise suffice.
  • We also note that despite the use of long-term prophylaxis, HAE patients require HAE-related home health visits and seek ER treatment more often than would be expected.

Astria Therapeutics Reports Third Quarter Financial Results and Provides a Corporate Update

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Monday, November 13, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Webcast, Interest, Plasma kallikrein, Safety, D, Therapy, First Nations Summit, IND, Investment, PD, Experiment, Patient, Allergy, Journal, Atopic dermatitis, Hereditary angioedema, Investigational New Drug, HAE, Half-life, YTE, OX40, Pharmacodynamics, ACAAI, Pharmaceutical industry, Cryptocurrency, Asteria, Pharmacology

Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company focused on developing life-changing therapies for rare and niche allergic and immunological diseases, today reported financial results for the third quarter ended September 30, 2023 and provided a corporate update.

Key Points: 
  • Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company focused on developing life-changing therapies for rare and niche allergic and immunological diseases, today reported financial results for the third quarter ended September 30, 2023 and provided a corporate update.
  • We now expect to deliver initial proof-of-concept results in Q1 2024 from the Phase 1b/2 ALPHA-STAR trial in HAE patients.
  • STAR-0215 achieved potentially therapeutic levels in less than one day and showed an estimated half-life of up to 127 days.
  • Additional preclinical results were shared in the Journal of Pharmacology and Experimental Therapeutics that support STAR-0215’s potential as a best-in-class plasma kallikrein inhibitor.

KalVista Pharmaceuticals Announces Phase 3 KONFIDENT Trial Milestone Achieved

Retrieved on: 
Monday, November 13, 2023
Health, Other Science, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, NDA, Larynx, Regulation, Food, Clinical trial, Patient, European, New Drug Application, FDA, KALV, HAE, File, Hereditary angioedema, Pharmaceutical industry

Clinical Trial and Regulatory Updates:

Key Points: 
  • Clinical Trial and Regulatory Updates:
    KalVista has achieved the targeted number of on-treatment attacks required to complete the phase 3 KONFIDENT trial.
  • The Company also expects to file for approval in the European Union and Japan later in 2024.
  • KONFIDENT randomized a total of 136 participants from 66 sites across 20 countries, making it the largest clinical trial ever conducted in HAE based on number of subjects.
  • “We are excited to have reached the number of on-treatment attacks required for completion of KONFIDENT,” said Andrew Crockett, Chief Executive Officer of KalVista.

Astria Therapeutics Presents New Phase 1a Data Confirming Potential for STAR-0125 to Prevent Hereditary Angioedema Attacks with Dosing 2 or 4 Times Per Year at the 2023 American College of Allergy, Asthma, and Immunology Annual Scientific Meeting

Retrieved on: 
Friday, November 10, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Plasma kallikrein, Therapy, University, Asthma, PD, Patient, Allergy, American College, Internal medicine, Hereditary angioedema, HAE, Half-life, Pharmacodynamics, ACAAI, Pharmaceutical industry

These data confirm the potential for STAR-0215 to be dosed once every three months and every six months.

Key Points: 
  • These data confirm the potential for STAR-0215 to be dosed once every three months and every six months.
  • “These new data, including long-term follow-up from the original cohorts and initial data from new, higher dose cohorts, support our vision for STAR-0215 as a first-choice therapy for HAE.
  • These data confirm our approach to administer STAR-0215 once every three and every six months in future trials.
  • These results demonstrate early proof-of-concept in healthy subjects for STAR-0215 as a potential HAE therapy with favorable safety profile, long half-life, and durable PD.

CSL's Garadacimab, a First-in-Class Factor XIIa Inhibitor, Receives FDA and EMA Filing Acceptance

Retrieved on: 
Thursday, December 14, 2023
CSL, Safety, Senior, Food, European Medicines Agency, Regulatory affairs, BLA, Disease, MAA, Doctor, EMA, Patient, FDA, ASX, HAE, Hereditary angioedema, Biologics license application, Vaccine, Pharmaceutical industry, Lancet

KING OF PRUSSIA, Penn., Dec. 14, 2023 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced the U.S. Food and Drug Administration (FDA) has accepted the company's Biologics License Application (BLA) for garadacimab (CSL312) as a once-monthly prophylactic treatment for hereditary angioedema (HAE). The company also announced the European Medicines Agency (EMA) has accepted the submission for CSL's Marketing Authorization Application (MAA) for garadacimab. If approved, garadacimab would become the first treatment for HAE in the U.S. and EU to target activated Factor XII (FXIIa).

Key Points: 
  • The company also announced the European Medicines Agency (EMA) has accepted the submission for CSL's Marketing Authorization Application (MAA) for garadacimab.
  • If approved, garadacimab would become the first treatment for HAE in the U.S. and EU to target activated Factor XII (FXIIa).
  • Orphan-drug designation for garadacimab as a therapy for hereditary angioedema has been granted by both the FDA and the EMA.
  • The ongoing open-label extension of the VANGUARD study evaluates the long-term safety and efficacy of garadacimab (200 mg monthly) in patients with HAE.

Haemonetics to Present at 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Thursday, December 7, 2023
Event, NYSE, Conference, HAE

BOSTON, Dec. 7, 2023 /PRNewswire/ -- Haemonetics Corporation (NYSE: HAE) announced that Chris Simon, President and CEO, will present at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 9, 2024 at 9:45am Pacific time.

Key Points: 
  • BOSTON, Dec. 7, 2023 /PRNewswire/ -- Haemonetics Corporation (NYSE: HAE) announced that Chris Simon, President and CEO, will present at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 9, 2024 at 9:45am Pacific time.
  • The public may access Mr. Simon's presentation live via audio webcast at:
    The live webcast can also be accessed under the Events & Presentations section of the Company's Investor Relations website.
  • The replay of the presentation will be available for 30 days using the link provided above and on Investor Relations website shortly after the live event.

The European Commission Approves Label Update for TAKHZYRO® (lanadelumab), Expanding Its Use to a Broader Group of Paediatric Patients with Recurrent Attacks of Hereditary Angioedema (HAE)

Retrieved on: 
Friday, November 17, 2023
Safety, Food, European Commission, Ageing, Lanadelumab, Clinical research associate, EMA, Civil service commission, Patient, FDA, Hereditary angioedema, HAE, Angioedema, Flint Adolescent Study, Pharmaceutical industry, Takeda

ZURICH, Nov. 17, 2023 /PRNewswire/ -- Takeda (TSE:4502/NYSE:TAK) today announced the European Commission has approved TAKHZYRO® (lanadelumab) for the routine prevention of recurrent attacks of Hereditary Angioedema (HAE) in patients aged 2 years and older1, expanding its initial approved use and making it the first long-term prophylactic treatment of HAE available in  European Economic Area for patients under the age of six.2,3,4

Key Points: 
  • - TAKHZYRO® is the First Routine Prevention Treatment of HAE Approved in the EU for Patients Under the Age of Six.
  • Overall, the safety and efficacy of TAKHZYRO® in preventing Hereditary Angioedema (HAE) attacks in paediatric patients aged 2 and above was demonstrated and the benefit risk-balance was considered positive.
  • said Didier Relin, Head of International Regulatory at Takeda.
  • "With this expanded label, TAKHZYRO® offers a welcomed new preventative treatment option for the paediatric HAE patient population, and one that can be administered at home with the support of a trained caregiver."

Intellia Therapeutics Announces Third Quarter 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Thursday, November 9, 2023
Gene editing, Research, Food, Facial muscles, Intellia Therapeutics, European Medicines Agency, Hereditary angioedema, Disease, A1AT, AAV, International, Mortality, Liver, TTR, HAE, Clinical, EMA, Safety, Therapy, Patient, D, DNA, KLKB1, ATM, Regeneron Pharmaceuticals, Cardiomyopathy, Conference, CTA, Tissue, NK, Serum, AATD, NTLA, FDA, ATTR, CRISPR, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Lung, SERPINA1, Amyloidosis, Pharmaceutical industry, Medical device, Vaccine, Video game, IND

“2023 has been a year of remarkable progress in which Intellia received two IND clearances for investigational in vivo CRISPR therapies.

Key Points: 
  • “2023 has been a year of remarkable progress in which Intellia received two IND clearances for investigational in vivo CRISPR therapies.
  • With the imminent start of the NTLA-2001 MAGNITUDE Phase 3 trial, Intellia has now become a late-stage drug development company,” said Intellia President and Chief Executive Officer John Leonard, M.D.
  • In November, Intellia announced new positive interim results from the Phase 1 study of NTLA-2001.
  • Net Loss: The Company’s net loss was $122.2 million for the third quarter of 2023, compared to $113.2 million during the third quarter of 2022.

Pharvaris Presents Deucrictibant Clinical Data and Real-World HAE Treatment Satisfaction Data at ACAAI 2023 Annual Scientific Meeting

Retrieved on: 
Thursday, November 9, 2023
American College, American College of Allergy, Asthma and Immunology, EOP, Physician, ACAAI, EST, Hereditary angioedema, Poster, DSP, HAE, PST, Adelphi, Angioedema, Attack, Pharmaceutical industry

In the phase 2 RAPIDe-1 trial, PHVS416 (immediate-release deucrictibant capsules) reduced time to the onset of symptom relief and to the resolution of HAE attacks, and substantially reduced use of rescue medication.

Key Points: 
  • In the phase 2 RAPIDe-1 trial, PHVS416 (immediate-release deucrictibant capsules) reduced time to the onset of symptom relief and to the resolution of HAE attacks, and substantially reduced use of rescue medication.
  • “The real-world HAE data and post-hoc analysis of RAPIDe-1 data that will be presented at ACAAI support the compelling story for the ongoing development of PHVS416 for the on-demand treatment of HAE,” said Peng Lu, M.D., Ph.D., Chief Medical Officer of Pharvaris.
  • An oral therapy with a faster onset of action may encourage more consistent and timely treatment of HAE attacks and improved treatment satisfaction.
  • Results of the post-hoc RAPIDe-1 analysis provide additional evidence on the rapid onset of effects of PHVS416 for on-demand treatment of HAE attacks.”