Verve Therapeutics, Inc.

Dive Brief:Intellia Therapeutics on Thursday released detailed Phase 2 results from a study of a gene-editing therapy for hereditary angioedema, drawing mixed reactions from investors and experts.The therapy, dubbed NTLA-2002, is based on the CRISPR gene editing technology that won a Nobel Prize in 2020. The treatment is designed to inactivate a gene involved in potentially life-threatening inflammatory attacks that plague patients with the disease.In the ongoing study of 27 patients, researchers found either a 75% or 77% reduction in attacks in the 16-week period after receiving therapy, depending on the dose received, Intellia said. And eight of the 11 patients in the higher dose arm were completely free of attacks after a one-time infusion. All of those patients continued to be attack-free with months of followup.Dive Insight:Shares of the company dropped 20% in early trading Thursday, likely because investors were looking for stronger data on the reduction of attacks, Stifel analyst Dae Gon Ha wrote in a note to clients. However, we think fixating on the attack rate reduction misses the bigger picture, which is that NTLA-2002 is a potentially one-time curative therapy.An editorial that accompanied the data, published in The New England Journal of Medicine, agreed. The durability of the therapeutic effect shows promise to last for the patients lifetimes, wrote Kiran Musunuru, a professor at the University of Pennsylvania and co-founder of Verve Therapeutics. We can be confident that NTLA-2002 is helping patients with hereditary angioedema.Investors had been waiting for the details on the study after Intellia announced positive top-line results in August. The latest findings build on early data that showed the therapy could reduce biological markers of the disease. Intellia said its actively screening patients for a Phase 3 trial that will use the higher dose.The Intellia treatment relies on gene editing inside the body, unlike the first-ever approved gene-editing therapy, Casgevy. For that treatment, a patients stem cells are collected, edited and then reinfused to change the patients genetic code. The hereditary angioedema therapy follows Intellias work on a medicine for transthyretin amyloidosis that supported its in vivo approach.Intellia still needs to prove its treatment can work in the Phase 3 trial. And the company will have to make the case to doctors that its worth taking a bet on a one-time, gene-editing therapy rather than the pills or injections that patients have come to depend on to control symptoms. Competitors in the field are also continuing to come up with new options on the more traditional path.Intellia says its hereditary angioedema therapy is safe for patients and offers them the chance to avoid a lifetime of preventive therapy that may not totally relieve symptoms. That possibility is a game changer, according to Paula Busse, a researcher and doctor at Mount Sinai who sees patients with hereditary angioedema and was featured on an Intellia conference call with investors and analysts. '

NEW YORK, Oct. 15, 2024 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in Verve Therapeutics, Inc. ("Verve Therapeutics" or the "Company") (NASDAQ: VERV) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of Verve Therapeutics investors who were adversely affected by alleged securities fraud between August 9, 2022 and April 1, 2024. Follow the link below to get more information and be contacted by a member of our team: VERV investors may also contact Joseph E. Levi, Esq. via email at [email protected] or by telephone at (212) 363-7500. CASE DETAILS: The filed complaint alleges that defendants made false statements and/or concealed that: (1) defendants did not fully disclose the circumstances under which the Heart-1 Phase 1b clinical trial of VERVE-101, its investigational gene editing medicine, would be halted; (2) defendants overstated the potential benefits of its proprietary lipid nanoparticle delivery system; and (3) as a result, defendants' statements about its business, operations, and prospects, were materially false and misleading and/or lacked a reasonable basis at all relevant times. WHAT'S NEXT? If you suffered a loss in Verve Therapeutics during the relevant time frame, you have until October 28, 2024 to request that the Court appoint you as lead plaintiff. Your ability to share in any recovery doesn't require that you serve as a lead plaintiff. NO COST TO YOU: If you are a class member, you may be entitled to compensation without payment of any out-of-pocket costs or fees. There is no cost or obligation to participate. WHY LEVI & KORSINSKY: Over the past 20 years, the team at Levi & Korsinsky has secured hundreds of millions of dollars for aggrieved shareholders and built a track record of winning high-stakes cases. Our firm has extensive expertise representing investors in complex securities litigation and a team of over 70 employees to serve our clients. For seven years in a row, Levi & Korsinsky has ranked in ISS Securities Class Action Services' Top 50 Report as one of the top securities litigation firms in the United States. CONTACT: Levi & Korsinsky, LLP Joseph E. Levi, Esq. Ed Korsinsky, Esq. 33 Whitehall Street, 17th Floor New York, NY 10004 [email protected] Tel: (212) 363-7500 Fax: (212) 363-7171 SOURCE Levi & Korsinsky, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

AstraZeneca is strengthening its cardiovascular disease pipeline with a $100 million upfront deal for a lipid-targeting drug from China’s CSPC Pharmaceutical Group. The UK drugmaker will get the rights to a small molecule lipoprotein(a) disruptor that’s in preclinical development. The candidate, YS2302018, can stop the formation of a lipid that plays a key role in transporting cholesterol in the bloodstream. CSPC could get up to $1.92 billion in milestones as well as tiered royalties on sales, according to an AstraZeneca press release . The company said the drug can be combined with others in its pipeline, including the small molecule PCSK9 inhibitor AZD0780. That candidate is currently in Phase 2 for dyslipidemia, which refers to a basket of diseases characterized by abnormal levels of lipids. Cardiovascular, renal and metabolic disease has been an important area of growth for AstraZeneca in recent years, with the drugmaker investing in external innovation. In November, the company put down $185 million upfront to license an oral GLP-1 agonist from China’s Eccogene. At the start of 2023, it acquired CinCor Pharma and its hypertension drug candidate, baxdrostat, for $1.8 billion . The drugmaker has also been looking more to China for assets, running counter to what some US peer companies have done. At an investor day event in May, CEO Pascal Soriot said innovation has “exploded” in the country and reiterated AstraZeneca’s commitment to tapping said innovation. His comments came in spite of heightened tensions between the US and China, which came to a head last month with the passing of the Biosecure Act. During the company’s most recent earnings call, Soriot said AstraZeneca aims to treat cardiovascular diseases “holistically,” instead of just focusing on weight loss. The company has said combination approaches will be a key part of that strategy. Other noteworthy players working on next-generation cholesterol treatments include Verve Therapeutics, which is advancing a base editing therapy for heterozygous familial hypercholesterolemia. Eli Lilly, meanwhile, has a small molecule lp(a) formation inhibitor, muvalaplin, in Phase 2 development. Novartis is also developing a Phase 3 lp(a) targeting antisense therapy that it licensed from Ionis Pharmaceuticals called pelacarsen.