Core Insights - Donidalorsen is an investigational RNA-targeted medicine aimed at preventing attacks of hereditary angioedema (HAE) and is set for FDA review with a PDUFA date of August 21, 2025 [1][3] - The drug has shown significant efficacy in clinical trials, with a 96% mean reduction in HAE attack rates maintained over three years in the ongoing Phase 2 open-label extension study [4] - Ionis Pharmaceuticals is preparing for its second independent commercial launch, contingent on FDA approval, and has partnered with Otsuka for commercialization in Europe and Asia Pacific [3][4] Company Overview - Ionis Pharmaceuticals has a history of developing RNA-targeted medicines and currently has five marketed products, focusing on areas of high patient need such as neurology and cardiology [8] - The company aims to deliver innovative therapies for serious diseases, leveraging its expertise in RNA technology and disease biology [8] Industry Context - HAE is a rare genetic condition affecting over 20,000 patients in the U.S. and Europe, characterized by recurrent severe swelling [6] - Current treatments for HAE are limited, and many patients still experience breakthrough attacks, highlighting the need for effective prophylactic options like donidalorsen [3][6]

Haemonetics (HAE) is expected to deliver a year-over-year increase in earnings on higher revenues when it reports results for the quarter ended September 2024. This widely-known consensus outlook gives a good sense of the company's earnings picture, but how the actual results compare to these estimates is a powerful factor that could impact its near-term stock price.The earnings report, which is expected to be released on November 7, 2024, might help the stock move higher if these key numbers are better tha ...

Core Insights - Intellia Therapeutics, Inc. (NASDAQ: NTLA) experienced a significant stock decline of up to 20% following the release of data from its phase 1/2 study of the CRISPR/Cas9 gene editing drug NTLA-2002 [1] Company Overview - Intellia Therapeutics is focused on developing CRISPR/Cas9 gene editing therapies, with NTLA-2002 being a key product in its pipeline [1] Market Context - The article highlights the volatility in biotech stocks, particularly in response to clinical trial data releases, which can lead to substantial price fluctuations [1]

Deep attack rate reductions achieved in both dose levels tested; a single 50 mg dose resulted in a mean monthly attack rate reduction of 77% and 81% compared to placebo during weeks 1-16 and 5-16, respectivelyEight of 11 patients in the 50 mg arm were completely attack free following a one-time infusion through the latest follow-up; data support NTLA-2002’s potential to be a functional cure for hereditary angioedema (HAE)NTLA-2002 demonstrated an encouraging safety and tolerability profileData published in ...

ZUG, Switzerland, Oct. 16, 2024 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to prevent and treat hereditary angioedema (HAE) attacks, today announced the acceptance of multiple abstracts at several upcoming scientific meetings. Presentation details are as follows: American College of Allergy, Asthma, & Immunology's Annual Scientific Meeting (ACAAI), Boston, October 24-28, 2024. Seven abstracts have been accept ...

Haemonetics (HAE) has been banking on its growth drivers like the Plasma and Hospital business. New product launches also lead to market expansion. The stock carries a Zacks Rank #2 (Buy) currently. Major Factors Driving HAE Stock Haemonetics' Plasma business continues to grow, banking on an expanding end-user market for plasma-derived biopharmaceuticals. In the second quarter of 2024, U.S. collection volume growth was robust. Both North America software and Europe disposable revenues witnessed double-digit ...

NTLA-2002 Development and Clinical Trial - NTLA-2002 is a single-dose CRISPR-based gene editing therapy designed to treat hereditary angioedema (HAE) by inactivating the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein [1][4] - The HAELO Phase 3 trial is a global, randomized, double-blind, placebo-controlled study involving 60 adults with Type I or Type II HAE, with patients randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo [2] - The primary endpoint of the Phase 3 trial is the change in the number of HAE attacks from week 5 through week 28 [2] - Interim Phase 1 clinical data showed dramatic reductions in attack rates and consistent, deep, and durable reductions in kallikrein levels [3] Regulatory Designations and Potential Impact - NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. FDA, Innovation Passport by the U.K. MHRA, PRIME Designation by the European Medicines Agency, and Orphan Drug Designation by the European Commission [4] - NTLA-2002 has the potential to become the first one-time treatment for HAE, addressing the real-world needs of patients, physicians, and payors [1][4] Hereditary Angioedema (HAE) Overview - HAE is a rare, genetic disease affecting approximately one in 50,000 people, characterized by severe, recurring, and unpredictable inflammatory attacks that can be life-threatening [5] - Current treatment options for HAE include lifelong therapies requiring chronic intravenous or subcutaneous administration, often with breakthrough attacks despite treatment [5] Intellia Therapeutics Overview - Intellia Therapeutics is a leading clinical-stage gene editing company focused on CRISPR-based therapies, with in vivo programs targeting disease-causing genes directly inside the human body and ex vivo programs for cancer and autoimmune diseases [6][7] - The company is advancing its modular gene editing platform, with NTLA-2002 being its second in vivo candidate to enter late-stage clinical development [1]

BOSTON, Oct. 3, 2024 /PRNewswire/ -- Haemonetics Corporation (NYSE: HAE) announced that the Company intends to publish second quarter fiscal year 2025 financial results at 6:00 am ET on Thursday, November 7, 2024. The Company will hold a conference call with investors and analysts to discuss results and answer questions at 8:00 am ET on November 7, 2024. The call can be accessed via teleconference at: Q2 2025 Haemonetics Corporation Earnings Conference Call. Once registration is completed, participants will ...

ZUG, Switzerland, Oct. 03, 2024 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to prevent and treat hereditary angioedema (HAE) attacks, today announced the acceptance of six abstracts, two for oral presentation and four for poster presentation, at the 2024 Global Angioedema Forum – HAEi Global Leadership Workshop, taking place in Copenhagen, Denmark, from Oct. 4-5, 2024. Details are as follows: Title: Long-Term ...

Haemonetics Corporation (HAE) is well-poised to grow in the coming quarters, backed by the strong potential of the Plasma franchise. The company's Hemostasis Management franchise is evolving and helping create new opportunities for growth and diversification. Strong financial stability also buoys optimism. Meanwhile, headwinds from adverse foreign exchange effects pose concern to Haemonetics' operations. In the past year, this Zacks Rank #2 (Buy) company has lost 14.5% against the industry's 24.6% growth an ...