HAE

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

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

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 .

Vivasure Medical Announces FDA IDE Approval to Initiate U.S. Pivotal Study

Retrieved on: 
Monday, March 13, 2023

Vivasure Medical® Limited (“Vivasure” or the “Company”), a company pioneering novel fully absorbable technology for percutaneous vessel closure, today announced that the U.S. Food and Drug Administration (FDA) has granted an Investigational Device Exemption (IDE) to advance the company’s PATCH Clinical Study, a multi-center, single-arm, pivotal study evaluating the safety and effectiveness of the Vivasure PerQseal® Closure Device System.

Key Points: 
  • Vivasure Medical® Limited (“Vivasure” or the “Company”), a company pioneering novel fully absorbable technology for percutaneous vessel closure, today announced that the U.S. Food and Drug Administration (FDA) has granted an Investigational Device Exemption (IDE) to advance the company’s PATCH Clinical Study, a multi-center, single-arm, pivotal study evaluating the safety and effectiveness of the Vivasure PerQseal® Closure Device System.
  • The PATCH pivotal study will enroll up to 188 patients across the U.S. and Europe.
  • The company intends to use the clinical study results to support an FDA pre-market approval submission as well as multinational commercial launch of the PerQseal system for large hole vessel closure.
  • The strategic investment by Haemonetics includes an option to acquire Vivasure Medical upon completion of certain milestones.

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

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

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

(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

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.

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

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

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.

Haemonetics Introduces Next Generation "Intelligent Control" Software for Cell Saver® Elite®+ Autotransfusion System

Retrieved on: 
Thursday, March 2, 2023

This software upgrade, named Intelligent Control, offers customers key enhancements to help simplify operations, supporting enhanced efficiency and an improved user experience.

Key Points: 
  • This software upgrade, named Intelligent Control, offers customers key enhancements to help simplify operations, supporting enhanced efficiency and an improved user experience.
  • "This latest upgrade represents a significant advancement in our Cell Salvage platform and builds upon our nearly 50-year foundation as the leader in autotransfusion.
  • The Intelligent Control software enhances our SmartSuction® and Latham bowl technologies to help maximize the recovery of red blood cells," said Stewart Strong, President of Global Hospital at Haemonetics.
  • "With the new software for the Cell Saver Elite+, we are delivering customers more versatility and simplicity to best meet their autotransfusion needs."