Plus: New vaccine restrictions expected as CDC committee meets |
In this week’s edition of InnovationRx, we look at possible pain treatments from cannabis, risks of new vaccine restrictions, virtual clinical trials at the Mayo Clinic, GSK’s $30 billion U.S. manufacturing commitment, and more. (Did someone forward this to you? To get it in your inbox, subscribe here.)
Despite their addictive nature, opioids continue to be a major treatment for pain due to a lack of effective alternatives. In an effort to boost new drugs, the FDA released new guidelines for non-opioid painkillers last week. But making these drugs hasn’t been easy. Vertex Pharmaceuticals received FDA approval for its non-opioid Journavx in January, then abandoned a next generation drug after a failed clinical trial earlier this summer. Acadia similarly abandoned a promising candidate after a failed trial in 2022.
One possible basis for non-opioids might be cannabis. Earlier this year, researchers at Washington University at St. Louis and Stanford published a study showing that a cannabis-derived compound successfully eased pain in mice with minimal side effects. Munich-based pharmaceutical company Vertanical is perhaps the furthest along in this quest. It is developing a cannabinoid-based extract to treat chronic pain it hopes will soon become an approved medicine, first in the European Union and eventually in the United States. The drug, currently called Ver-01, packs enough low levels of cannabinoids (including THC) to relieve pain, but not so much that patients get high. Founder Clemens Fischer, a 50-year-old medical doctor and serial pharmaceutical and supplement entrepreneur, hopes it will become the first cannabis-based painkiller prescribed by physicians and covered by insurance. Fischer founded Vertanical, with his business partner Madlena Hohlefelder, in 2017, and has invested more than $250 million of his own money in it. With a cannabis cultivation site and drug manufacturing plant in Denmark, Vertanical has successfully passed phase III clinical trials in Germany and expects a decision from German and Austrian authorities later this year. So far, the data is promising: Its drug was found to be more effective than opioid painkillers with fewer side effects, including no evidence for addiction. It hopes to launch phase III trials in the U.S. in 2026. “We believe that we will be the first non-opiate chronic pain treatment worldwide,” Fischer says. |
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 | | HHS Secretary Robert F. Kennedy, Jr. Getty Images |
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| The CDC’s immunization advisory committee—whose membership Health and Human Services Secretary Robert F. Kennedy Jr. purged in June and replaced with people more aligned with his anti-vaccine views—is slated to meet this week, starting on Thursday. On the agenda, a topic crucial to the health of the country and its pharma industry: childhood vaccinations, specifically those that protect against hepatitis B, measles and chickenpox, as well as COVID-19. Typically meetings of this committee, known by its acronym ACIP, are straightforward and off the public’s radar, but since 1964 they’ve served a vital role in setting immunization recommendations and schedules to serve public health. States generally follow this guidance in setting vaccine mandates for schools, while insurers typically follow it in determining what shots they’ll cover. Global immunization efforts saved at least 154 million lives between 1974 and 2024, more than 100 million of them children, according to a study published in The Lancet. Since his appointment, Kennedy has been pushing anti-vaccine policies at the federal level and this meeting could prove critical for U.S. immunization policy going forward. Already, he has announced plans to award a no-bid contract to Rensselaer Polytechnic Institute to ‘investigate” long-debunked links between vaccines and autism, and appointed committee members like Retsef Levi and Robert Malone, who have been outspoken opponents of COVID vaccines, which saved an estimated 14.4 million lives the first year after they were made available, and Catherine Stein, who has argued against vaccine mandates. Sources with knowledge told Forbes that ACIP may restrict its COVID-19 vaccine recommendations to people older than 75 and those younger with a narrow set of preexisting conditions. The Washington Post reported this possibility previously. “We should be very worried because ACIP isn’t ACIP anymore,” Dr. Paul Offit, director of the Vaccine Education Center at the Children’s Hospital of Philadelphia, told Forbes. “It’s essentially an arm of our Secretary of HHS who is an anti-vaccine denialist and has been for the last 20 years.” Read more here. |
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| GSK announced Tuesday that it plans to invest $30 billion in R&D and manufacturing facilities in the U.S. over the next five years. This includes $1.2 billion in advanced manufacturing and AI for next-generation biopharma factories and labs, among them a new biologics factory in Upper Merion, Pa., to produce therapies for respiratory diseases and cancer. Earlier on Tuesday, Lilly announced its plans to build a $5 billion manufacturing facility near Richmond, Va., as part of its previous commitment to spend $50 billion in U.S. capital expansion projects. Plus: Krystal Biotech’s topical gene therapy Vyjuvek for patients with dystrophic epidermolysis bullosa, known colloquially as “butterfly skin disease,” received a label expansion from the FDA that allows infants to be treated immediately on diagnosis and to do so at home without a healthcare professional. Krystal’s stock rose 8% on Monday, to $156, on the news. Forbes previously profiled Krystal cofounder Suma Krishnan. |
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| Researchers at the Mayo Clinic developed “virtual clinical trials” to predict how potential treatments for heart failure will work. These trials use computer models trained on data from nearly 60,000 heart failure patients to predict whether certain existing drugs might be good candidates for repurposing in heart disease. The approach was validated by comparing predictions to existing clinical trial data. The Mayo team published its peer-reviewed findings in npj Digital Medicine. Plus: Lila Sciences raised $235 million at a valuation of $1.2 billion to bring AI to scientific research. The firm, which was founded by Flagship Pioneering in 2023, had previously raised a $200 million seed round in March. |
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| PUBLIC HEALTH + HOSPITALS |
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| More than half of healthcare professionals plan to search for or switch to new jobs next year, according to a new Harris poll. Of the 1,504 healthcare workers surveyed, 84% said they feel “taken for granted” by their employers and just 37% said they were satisfied with their current jobs. The findings come as the industry faces both high levels of burnout and workforce shortages. Plus: Investments in disease prevention and early detection could save the U.S. healthcare system as much as $2.2 trillion by 2040, according to a new report from Deloitte. |
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