Hereditary angioedema

Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to NTLA-2002 for the Treatment of Hereditary Angioedema

Retrieved on: 
Tuesday, March 21, 2023
CRISPR, Regenerative Medicine Advanced Therapy, NTLA, Health care, Hereditary angioedema, KLKB1, Intellia Therapeutics, HAE, Patient, Orphan, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, 21st Century Cures Act, Pharmaceutical industry

CAMBRIDGE, Mass., March 21, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to NTLA-2002 for the treatment of hereditary angioedema (HAE).

Key Points: 
  • CAMBRIDGE, Mass., March 21, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to NTLA-2002 for the treatment of hereditary angioedema (HAE).
  • NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to inactivate the target gene, kallikrein B1 (KLKB1), to potentially prevent life-threatening swelling attacks in people with HAE.
  • The RMAT is the third special regulatory designation received by Intellia for NTLA-2002.
  • NTLA-2002 was also granted Orphan Drug Designation by the FDA and the Innovation Passport by the U.K.

Positive Phase 2 Data from RAPIDe-1 Study of PHVS416 for the On-Demand Treatment of HAE Attacks Highlighted at the 2023 HAEi Regional Conference APAC

Retrieved on: 
Saturday, March 18, 2023
Attack, Hereditary angioedema, Safety, Bradykinin receptor B2, Softgel, II, Pain, HAE, Oral, United Kingdom, Conference, Pharmaceutical industry

The time required for preparation and administration, as well as potential occurrence of pain, discomfort, or other injection site reactions can lead to treatment delays or untreated HAE symptoms.

Key Points: 
  • The time required for preparation and administration, as well as potential occurrence of pain, discomfort, or other injection site reactions can lead to treatment delays or untreated HAE symptoms.
  • 74 participants were enrolled and 62 of them experienced 147 qualifying HAE attacks that were treated with double-blinded study drug (either placebo or PHVS416 10, 20, or 30 mg doses).
  • Participants on PHVS416 also used substantially less rescue medication compared to placebo (10 mg=18.9%, 20 mg=10.7%, 30 mg=6.5%, placebo=60.8%).
  • The presentation slides will be available on the Investors section of the Pharvaris website at: https://ir.pharvaris.com/news-events/events-presentations .

CADTH Recommends Reimbursement for BioCryst’s ORLADEYO® (berotralstat) for the Routine Prevention of Attacks in Hereditary Angioedema Patients in Canada

Retrieved on: 
Wednesday, March 8, 2023
MD, BioCryst Pharmaceuticals, PARK, Health Canada, Patient, CADTH, Disease, Hereditary angioedema, FRCPC, British Society of Cinematographers, HAE, CDEC, RESEARCH, Pharmaceutical industry, Health insurance

RESEARCH TRIANGLE PARK, N.C., March 08, 2023 (GLOBE NEWSWIRE) --  BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has issued a positive recommendation for ORLADEYO® (berotralstat) to be reimbursed for the routine prevention of attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older.

Key Points: 
  • In issuing this positive recommendation, CADTH is opening the door to many Canadian HAE patients being able to access ORLADEYO,” said Stephen Betschel, HBSc, MD, FRCPC, FAAAAI, Chair of the Canadian Hereditary Angioedema Network.
  • “BioCryst is delighted that ORLADEYO has received this positive recommendation from CADTH.
  • Health Canada authorized ORLADEYO in June 2022 for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older.
  • The full CADTH reimbursement recommendation for ORLADEYO is available on CADTH’s website .

Orchard Therapeutics Provides Business Update and Reports 2022 Financial Results

Retrieved on: 
Monday, March 6, 2023
Civil service commission, Research, Health care, ADS, Patient, POC, American depositary receipt, BLA, HAE, Webcast, IND, Incidence, HSC, GAAP, MPS, NBS, Hereditary angioedema, Type, Medicines and Healthcare products Regulatory Agency, European Commission, Investment, FDA, Diagnosis, CGMP, Aircraft, MLD, SG, Newborn screening, Bank statement, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Therapy, Food, MHRA, Medication, EC, Orchard, Pharmaceutical industry, Cryptocurrency, Medical device, Bank, Commercial property, Atidarsagene autotemcel, G&A

ET

Key Points: 
  • ET
    BOSTON and LONDON, March 06, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced business updates along with its financial results for the quarter and year ended December 31, 2022.
  • Based on the outcome of the Type B meeting, Orchard Therapeutics subsequently requested a pre-BLA meeting which has been scheduled for the second quarter of 2023.
  • Management believes this non-GAAP information is useful for investors, taken in conjunction with Orchard’s GAAP financial statements, because it provides greater transparency regarding Orchard’s operating performance.
  • Management uses these measures, among others, to assess and analyze operational results and trends and to make financial and operational decisions.

KalVista Pharmaceuticals Reports Third Fiscal Quarter Results

Retrieved on: 
Thursday, March 9, 2023
Health, Genetics, Clinical Trials, Research, Pharmaceutical, Science, HAE, PMDA, American Academy of Allergy, Asthma, and Immunology, American Academy, Western world, Fiscal year, G&A, STP, AAAAI, Hereditary angioedema, NDA, Quality of life, Patient, Shanda, Anxiety, Annual general meeting, Public expenditure, ODT, KALV, FDA, D, Manuscript, Pharmaceutical industry, Cryptocurrency, Lancet

(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, 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 third fiscal quarter ended January 31, 2023.
  • “We have made significant advances at KalVista over this quarter, including completing a financing that funds us into 2025, well beyond our phase 3 data results, which remain on track for the second half of this year,” said Andrew Crockett, Chief Executive Officer of KalVista.
  • KalVista also received guidance from the Japanese regulatory authority (PMDA) on the clinical development pathway to a regulatory submission in that country.
  • Third Fiscal Quarter Financial Results:
    Revenue: No revenue was recognized for the three months ended January 31, 2023 or January 31, 2022.

Astria Therapeutics to Report Fourth Quarter/Full Year 2022 Financial Results and Recent Corporate Developments on Wednesday, March 22nd

Retrieved on: 
Wednesday, March 8, 2023
Biotechnology, Pharmaceutical, Health, HAE, Therapy, Software, Hereditary angioedema, Webcast, Interest, Allergy, Pharmaceutical industry

Astria Therapeutics, Inc. (NASDAQ:ATXS) a biopharmaceutical company developing STAR-0215 for the treatment of hereditary angioedema (HAE) and focused on life-changing therapies for rare and niche allergic and immunological diseases, will report fourth quarter/full year 2022 financial results before the Nasdaq Global Market open on Wednesday, March 22, 2023.

Key Points: 
  • Astria Therapeutics, Inc. (NASDAQ:ATXS) a biopharmaceutical company developing STAR-0215 for the treatment of hereditary angioedema (HAE) and focused on life-changing therapies for rare and niche allergic and immunological diseases, will report fourth quarter/full year 2022 financial results before the Nasdaq Global Market open on Wednesday, March 22, 2023.
  • Jill C. Milne, Ph.D., Chief Executive Officer, will host a conference call and webcast at 8:30am ET to provide an update on corporate developments and to discuss fourth quarter and full year 2022 financial results.
  • Interested parties may join the webcast via the Investors section of the Astria website, www.astriatx.com or with the following link: https://lifescievents.com/event/astria-therapeutics-event/ .
  • Please connect to the webcast several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be required.

Astria Therapeutics to Present at Upcoming Oppenheimer 33rd Annual Healthcare Conference

Retrieved on: 
Monday, March 6, 2023
Biotechnology, Pharmaceutical, Health, Conference, Therapy, Hereditary angioedema, Allergy, Pharmaceutical industry, Broadcasting

Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company developing STAR-0215 for hereditary angioedema and focused on life-changing therapies for rare and niche allergic and immunological diseases, today announced that Jill C. Milne, Ph.D., Chief Executive Officer, will present a corporate overview and provide information on lead program STAR-0215 at the upcoming virtual Oppenheimer 33rd Annual Healthcare Conference on Monday, March 13th at 1:20pm ET.

Key Points: 
  • Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company developing STAR-0215 for hereditary angioedema and focused on life-changing therapies for rare and niche allergic and immunological diseases, today announced that Jill C. Milne, Ph.D., Chief Executive Officer, will present a corporate overview and provide information on lead program STAR-0215 at the upcoming virtual Oppenheimer 33rd Annual Healthcare Conference on Monday, March 13th at 1:20pm ET.
  • A webcast of the presentation can be accessed at the following link: https://wsw.com/webcast/oppenheimer27/atxs/2819488 .
  • An archived replay of the presentation will be available in the investors section of www.astriatx.com for 30 days following the event.

Astria Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Thursday, March 2, 2023
Biotechnology, Health, Science, Pharmaceutical, Research, Employment, Therapy, Hereditary angioedema, Allergy, Pharmaceutical industry, Health insurance

Astria Therapeutics, Inc. (Nasdaq:ATXS), a biopharmaceutical company developing STAR-0215 for the treatment of hereditary angioedema and focused on life-changing therapies for rare and niche allergic and immunological diseases, granted stock options to purchase 10,000 shares of Astria’s common stock on March 1, 2023 under Astria’s 2022 Inducement Stock Incentive Plan.

Key Points: 
  • Astria Therapeutics, Inc. (Nasdaq:ATXS), a biopharmaceutical company developing STAR-0215 for the treatment of hereditary angioedema and focused on life-changing therapies for rare and niche allergic and immunological diseases, granted stock options to purchase 10,000 shares of Astria’s common stock on March 1, 2023 under Astria’s 2022 Inducement Stock Incentive Plan.
  • The 2022 Inducement Stock Incentive Plan is used exclusively for the grant of equity awards to individuals who were not previously an employee of Astria.
  • The options were granted as an inducement material to an employee or employees entering into employment with Astria in accordance with Nasdaq Listing Rule 5635(c)(4).
  • The options are subject to the terms and conditions of the 2022 Inducement Stock Incentive Plan and the terms and conditions of an award agreement covering the grant.

Intellia Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for NTLA-2002, an In Vivo CRISPR-Based Investigational Therapy for the Treatment of Hereditary Angioedema (HAE)

Retrieved on: 
Thursday, March 2, 2023
Genome, HAE, Intellia Therapeutics, NTLA, FDA, KLKB1, Patient, Therapy, Investigational New Drug, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Hereditary angioedema, CRISPR, Food, IND, Pharmaceutical industry

NTLA-2002 is an in vivo genome editing candidate designed to inactivate the target gene, kallikrein B1 (KLKB1), to permanently reduce plasma kallikrein protein activity and thus prevent HAE attacks after a single-dose treatment.

Key Points: 
  • NTLA-2002 is an in vivo genome editing candidate designed to inactivate the target gene, kallikrein B1 (KLKB1), to permanently reduce plasma kallikrein protein activity and thus prevent HAE attacks after a single-dose treatment.
  • “The NTLA-2002 IND clearance marks an important milestone for Intellia as we continue our track record of execution as the leader in the genome editing field.
  • We are thrilled to advance the development of NTLA-2002 in the U.S. and are working to rapidly enroll patients in the Phase 2 portion of the study.
  • We look forward to presenting additional data from the first-in-human, Phase 1 portion of the study later this year.”

Pharvaris to Present in Upcoming March Investor Conferences

Retrieved on: 
Thursday, March 2, 2023
HAE, Conference, Hereditary angioedema, Pharmaceutical industry, Online shopping

ZUG, Switzerland, March 02, 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 that management will participate in two upcoming investor conferences in March:

Key Points: 
  • ZUG, Switzerland, March 02, 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 that management will participate in two upcoming investor conferences in March:
    A live audio webcast of the Oppenheimer 33rd Annual Healthcare Conference presentation will be available on the Investors section of the Pharvaris website at: https://ir.pharvaris.com/news-events/events-presentations.
  • A replay will be available on Pharvaris’ website for 30 days following the presentation.