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Reuters
21-03-2025
- Business
- Reuters
Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
Summary Companies Venture capital investment in gene therapies fell to $1.4 billion in 2024 from 2021 peak of $8.2 billion Total global biopharma venture funding rose to $27 billion in 2024 from $23.2 billion in 2023 Manufacturing, commercial hurdles limit demand for some gene therapies March 21 (Reuters) - Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward sectors like obesity and cancer, as sales for some of the new treatments fall short. In the last year, some drugmakers have pulled back from the sector, including Pfizer (PFE.N), opens new tab, which recently stopped selling its gene therapy for hemophilia priced at $3.5 million per patient. Bluebird Bio, a gene therapy pioneer once worth nearly $10 billion, was sold to private equity firms for $30 million last month. In 2024, developers of gene therapies and gene-editing products raised less than $1.4 billion across 39 venture rounds, according to a data analysis from DealForma for Reuters. In 2023, they raised $3.5 billion in 60 deals, which was down 57% from the sector's peak of $8.2 billion across 122 deals in 2021. "There needs to be better ways, cheaper ways to make some of these complex products," in order for investor interest in gene therapy to return, said Subin Baral, Ernst & Young global life sciences deal leader. Companies like Novartis (NOVN.S), opens new tab say they are continuing gene therapy research. More than 95% of infants born in the U.S. with a rare neuromuscular disorder called spinal muscular atrophy are now treated with its Zolgensma gene therapy and it is being developed for older children. Vertex Pharmaceuticals (VRTX.O), opens new tab continues to see patients with sickle cell disease start the process to receive its Casgevy therapy, said Chief Operating Officer Stuart Arbuckle. He said the treatment, which had 2024 sales of just $10 million, was pursued because the company believed gene editing offered the best solution for people living with the debilitating disease. Gene therapy — the one-time process of inserting a modified gene to compensate for a faulty gene or change how a patient's cells produce proteins — is a complex procedure in which patients often have to be hospitalized, with insurance coverage not always clear. The U.S. Food and Drug Administration has supported the breakthrough treatments, which have multimillion-dollar prices. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, told Reuters he is confident "speed bumps" including scientific challenges, manufacturing difficulties and real-world application of gene therapies will be worked out. "We need to have a streamlined regulatory process... Combine that with manufacturing improvements to have a package of things to bring down cost so they are no longer cost prohibitive," Marks said earlier this month. "My hope is that we have a group that is going to be here through the next years working on this." Safety issues are also a concern even after approval. Sarepta Therapeutics (SRPT.O), opens new tab said this week a 16-year-old boy died from acute liver failure months after receiving the company's gene therapy for a rare muscular dystrophy. Weight-loss drugs, meanwhile, are forecast to see annual sales of $150 billion in coming years as new, highly-effective medicines have sparked outsized demand. Obesity therapeutics attracted $1.75 billion in venture capital last year, nearly triple 2023's total of $630 million. Overall, global biopharma venture funding rose to $27 billion in 2024 from $23.2 billion in 2023. Cancer remained the top sector at $10.3 billion, according to DealForma. ECONOMICS AND POLICY DIFFICULTIES Unlike traditional drugs that can be made in large quantities for mass distribution, gene therapies are more individualized, requiring specialized equipment and cell processing materials. "People believe in the promise of gene therapy," said Priya Chandran, biopharmaceuticals sector leader at Boston Consulting Group. "Investment is dropping because the overall economics and policy landscape has been problematic." The Alliance for Regenerative Medicine, the industry trade group for the cell and gene therapy sector, said it expects investment to be reinvigorated as new trial data emerges. "The biggest biopharma companies continue to invest as the gene therapy pipeline evolves to tackle diseases with larger patient populations," said ARM spokesman Stephen Majors. When Pfizer last month stopped selling Beqvez, its gene therapy for hemophilia B, it said demand was weak. The company in 2023 had already backed away from early-stage gene therapy research, and last year abandoned efforts on a gene treatment for muscular dystrophy after disappointing results from a late-stage trial. Bluebird sells three gene therapies in the U.S., including Lyfgenia for sickle cell disease, which competes with Vertex and CRISPR Therapeutics' ( opens new tab Casgevy. Pfizer and Bluebird did not immediately respond to requests for comment. Evidence that expensive gene therapies can provide a sustained benefit and will be accepted by patients, doctors and insurers has become more critical as major pharmaceutical companies face the looming loss of market exclusivity for top-selling medicines. Morgan Stanley estimated that around $175 billion of 2025 U.S. large-cap biopharma revenue – 35% of the total – will go off patent by the end of the decade. Companies "are trying to invest fast enough so that they can restock their pipelines and hopefully fill the gap," said EY's Baral. "These are capital allocation choices."
Yahoo
18-02-2025
- Business
- Yahoo
Vertex to Participate in Upcoming March Investor Conferences
BOSTON, February 18, 2025--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in three upcoming investor conferences. Stuart Arbuckle, Executive Vice President and Chief Operating Officer, and David Altshuler, M.D., Ph.D., Executive Vice President, Global Research, and Chief Scientific Officer will participate in a fireside chat at the TD Cowen 45th Annual Health Care Conference on Monday, March 3, 2025, at 3:10 p.m. ET. Charles Wagner, Executive Vice President and Chief Financial Officer, and Dr. Altshuler will participate in a fireside chat at the 2025 Leerink Partners Global Healthcare Conference on Monday, March 10, 2025, at 10:00 a.m. ET. Susie Lisa, Senior Vice President, Investor Relations and Miroslava Minkova, Executive Director, Investor Relations will participate in the Jefferies Biotech on the Beach Summit on Tuesday, March 11, 2025. A live webcast of management's remarks will be available through the Vertex website, in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. (VRTX-WEB) View source version on Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@ Sign in to access your portfolio
Boston Globe
30-01-2025
- Health
- Boston Globe
FDA approves Journavx, new non-opioid painkiller from Vertex: ‘An important public health milestone'
In two late-stage trials of patients who underwent tummy tuck surgery or removal of a bunion, Journavx led to a 50 percent reduction in pain, Vertex said. It acted faster than a placebo and with fewer side effects than a pill that combined acetaminophen and the opioid hydrocodone, the active ingredients in Vicodin. Advertisement Significantly, Journavx didn't provide better pain relief than the opioid combination in one of the trials and was inferior to the combination in the other trial. Nonetheless, Vertex executives say Journavx's ability to soothe pain without the risk of addiction will likely make it a blockbuster, a drug that generates sales of at least $1 billion a year. 'We think this is the sort of product that, frankly, physicians and patients have been waiting for,' said Stuart Arbuckle, Vertex's chief operating officer. The most common side effects in patients who received Journavx in the clinical trials included itching, muscle spasms, and rashes. The approval comes as the nation continues to grapple with a decades-old opioid crisis. More than 81,000 people died of opioid overdoses in the United States in 2023, according to the National Center for Health Statistics. Doctors prescribe opioids for acute pain to 40 million Americans a year, and about 85,000 of them develop opioid use disorder, according to Vertex. The company has also tested Journavx as a treatment for chronic pain, a bigger market than acute pain, but the results of clinical trials so far have been underwhelming. Advertisement Dr. Jessica McCoun, an Atlanta-based anesthesiologist and principal investigator in the trials, said she considers the new Vertex drug 'a very big tool in the toolbox' for treating pain. 'The more tools we have that we can combine together to treat a patient effectively ... That's the best we can do,' she said. A bottle of Journavx Courtesy of Vertex Among the patients who received Journavx in the clinical trials was a 50-year-old woman from the Atlanta area. She had a nose job in October 2023 and began taking the pill immediately afterward in a third Vertex trial for people who underwent a variety of surgeries. The woman, who only wanted to be identified by her first name, Samantha, because of the sensitivity of the subject, said she took Journavx for four days. She never felt any pain, she said, even though her operation was a 'pretty severe reconstruction.' 'For the level of pain I should have been in, this medicine was very, very effective,' she said. 'I think it's spectacular.' Some experts were more cautious in their assessments. Dr. Adriane Fugh-Berman, a professor of pharmacology and physiology at Georgetown University Medical Center, welcomed the FDA approval. But she contended it was premature to declare that Journavx posed no risk for addiction, saying it wasn't tested long enough in clinical trials. 'It's definitely a promising painkiller, but I haven't seen studies that lasted longer than a couple of weeks in humans,' said Fugh-Berman. Drugmakers, she went on, have made similar claims for other medicines and turned out to be wrong. She cited gabapentin, an anti-seizure medication used for nerve pain that experts say can lead to abuse and dependency. Advertisement Two market analysts had predicted the FDA would approve the drug based on the results of clinical trials, but they expressed different levels of enthusiasm. David Risinger, an analyst for the Boston investment bank Leerink Partners, called the clinical trial results 'compelling' and predicted that the medicine will generate peak annual sales of $4 billion in the 2030s. 'We think physicians and insurance companies would rather send patients home with a non-opioid pill prescription rather than an opioid pill prescription,' he said. 'It's a huge market opportunity.' Brian Skorney, an analyst with Baird, also said Journavx's approval was 'as close to a sure thing as one can get' because it reduced pain when compared to a placebo, the chief goal of the studies. But he said Vertex put its thumb on the scale for a secondary goal of the trials by comparing the drug to the equivalent of a low-dose Vicodin rather than to more potent opioids that doctors sometimes prescribe. He said it would have been more meaningful had Vertex simply compared Journavx to inexpensive over-the-counter painkillers that people have in their medicine cabinets, such as ibuprofen. 'I think people will be disappointed with, ultimately, how much it sells,' he said of Journavx. Vertex, however, seems to be confident and added 150 sales employees last year in anticipation of the FDA approval. The company has about 6,100 employees worldwide, including 4,200 in Massachusetts. Despite how common pain is, there hasn't been a significant innovation for treating it in decades. Aspirin dates to the late 19th century, while acetaminophen, ibuprofen, and naproxen sodium were invented generations ago. Advertisement Opioids date back thousands of years. But they have led to a devastating public health crisis, fueled in no small part by OxyContin, the opioid tablet made by Purdue Pharma. For years, multiple companies, including Cambridge-based Biogen, the South San Francisco biotech Genentech (which was later acquired by Roche), and the Swiss drug giant Novartis have tried unsuccessfully to develop a new class of painkillers. In 2022, Regeneron Pharmaceuticals, of Tarrytown, N.Y., shelved an experimental drug for osteoarthritis and chronic low back pain that it had long worked on after clinical trials raised safety concerns. Painkillers today fall into two major classes: anti-inflammatory analgesics, such as aspirin and ibuprofen, and opioids. Anti-inflammatories work by reducing swelling at the location of pain. Opioids change the brain's perception of pain, mimicking the natural pain-relieving chemicals known as endorphins produced in the brain. In contrast, Vertex's new drug interrupts pain signals before they reach the brain, blocking certain proteins called sodium channels that transmit pain in the peripheral nervous system. Because the non-opioid drug bypasses the central nervous system, the company says, it provides pain relief without the risk of addiction. Globe correspondent Stella Tannenbaum contributed to this report. Jonathan Saltzman can be reached at
