Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
COVID-19 vaccine cut for healthy children and pregnant women
On Tuesday, the Department of Health and Human Services (HSS) Secretary Robert F. Kennedy Jr. announced in a video on X that the CDC will no longer recommend the COVID-19 vaccine for healthy children and healthy pregnant women. The latest announcement conflicts with earlier guidance released by the FDA just last week, in which they listed pregnancy as a risk factor for severe COVID-19 outcomes. Steven Fleischman, President of the American College of Obstetricians and Gynecologists (ACOG), said in a statement, “ACOG is concerned about and extremely disappointed by the announcement that HHS will no longer recommend COVID-19 vaccination during pregnancy. As ob-gyns who treat patients every day, we have seen firsthand how dangerous COVID-19 infection can be during pregnancy and for newborns who depend on maternal antibodies from the vaccine for protection.”
The new guidance still allows children with underlying conditions to qualify for the vaccine, but it remains unclear whether healthy children who have not yet received any COVID-19 vaccines will be eligible. Speaking to The New York Times, Sean O’Leary, a pediatric vaccine expert for the American Academy of Pediatrics, said that infants from birth to age six months are at similar risk of hospitalization as adults aged 65 to 74.
The decision means that pregnant women and parents of healthy children who would like to receive a vaccine will have to pay out of pocket — which USA Today reported could cost about $200. But that’s only if they find a pharmacist still willing to vaccinate them. – Allison Whitten
HHS cancels Moderna’s $590 million contract to develop bird flu vaccines
On Wednesday evening Moderna announced that HHS terminated their contract to develop a pre-pandemic mRNA vaccine against avian influenza. In the same news release, the team at Moderna announced interim Phase 1/2 clinical trial results of their two-dose avian influenza vaccine mRNA-1018, which, when tested in 300 healthy adult participants, led to a “rapid, potent and durable immune response.” Moderna CEO Stéphane Bancel said that the company will explore alternatives to continue developing the vaccine. CNN reported that HHS Communications Director Andrew Nixon said in a statement, “The reality is that mRNA technology remains under-tested, and we are not going to spend taxpayer dollars repeating the mistakes of the last administration, which concealed legitimate safety concerns from the public.” Multiple public health experts took issue with both this characterization of mRNA vaccine technology and the decision. Epidemiologist and Director of the Pandemic Center at Brown University, Jennifer Nuzzo told NPR that this was “disappointing, but unsurprising given the politically-motivated, evidence-free rhetoric that tries to paint mRNA vaccines as being dangerous.” On X, Ashish Kumar Jha, who was the White House COVID-19 response coordinator from 2022–2023 and is the Dean of the Brown University School of Public Health said, “It was President Trump's Operation Warp Speed that gave us mRNA vaccines. These vaccines have been administered nearly 2 billion times to hundreds of millions of people around the world -- making it one of the most widely used and widely studied vaccines in human history. They are safe and work well.” – Stephanie DeMarco
FDA pauses Rocket Pharmaceuticals trial after patient dies
The FDA paused a Phase 2 trial from Rocket Pharmaceuticals after a patient passed away following an acute systemic infection related to the development of capillary leak syndrome. The trial was testing the gene therapy RP-A501 to treat the rare genetic disorder Danon disease, which is associated with heart and skeletal muscle weakness and intellectual disability. Rocket Pharmaceuticals announced in a press release that they are investigating the cause of death, and the current focus is on the addition of a novel immune suppression agent to the trial. In the statement, CEO of Rocket Pharmaceuticals Gaurav Shah said, “We are heartbroken by this loss and are fully committed to our mission to develop gene therapies that address the underlying cause of devastating diseases like Danon. We are immensely grateful for the patients and families who participate in this important research.” – Allison Whitten
Lilly’s up to $1 billion non-opioid pain drug acquisition
On Tuesday, Lilly announced that they plan to acquire SiteOne Therapeutics, a biotech company developing small molecule drugs to treat pain and other conditions characterized by neuronal hyperexcitability. SiteOne’s drugs inhibit sodium channels, and their therapy STC-004, which is ready to enter Phase 2 clinical trials, targets NaV1.8, the same sodium channel that the recently approved Vertex Pharmaceuticals’ drug, Journavx, targets. This acquisition expands the breadth of Lilly’s drug development pipeline. Mark Mintun, the Lilly Group Vice President-Neuroscience Research and Development, said in the press release about the acquisition, “Lilly is eager to continue the development of STC-004 with the outstanding SiteOne team as part of our efforts to advance novel, addiction-free pain therapies. Innovation in pain management is critical to address the unmet needs of millions of patients around the world.” – Stephanie DeMarco
Promising antidepressant effect from Gilgamesh psychedelic
Gilgamesh Pharmaceuticals announced that their Phase 2a trial of GM-2505 showed that the drug is an effective, fast-acting, and durable antidepressant for individuals with major depressive disorder. The drug works by targeting the 5-HT2A receptor to mimic the effects of psychedelics like psilocybin and LSD. In the high-dose cohort, 94 percent of patients remained in remission at day 29. In a press release, Founder and CEO of Gilgamesh Jonathan Sporn said, “These compelling results are the culmination of five years of research focused on designing and developing truly novel and optimized treatments." – Allison Whitten
Antisense oligonucleotide therapy led to clinical improvement in a rare, aggressive form of ALS
While only one to two percent of people who have amyotrophic lateral sclerosis (ALS) have the rare form of the disease called fused in sarcoma (FUS) ALS, in these patients, the disease typically manifests in adolescence or young adulthood and is caused by a mutation in the FUS gene. In an investigator-led clinical study published in The Lancet, researchers enrolled 12 people who carried the FUS variant and showed signs of motor neuron disease or electrophysiological abnormalities (1). Participants received monthly doses of an antisense oligonucleotide that targeted the pre-mRNA of the FUS gene for destruction. While most participants continued to decline, “one showed unprecedented, objective functional recovery after 10 months,” the researchers wrote, and a second participant remained asymptomatic while showing improvement in their electrical muscle response which had been abnormal prior to the therapy. In partnership with the Japanese pharma company Otsuka, Ionis Pharmaceuticals is recruiting FUS-ALS patients for a Phase 3 clinical trial to further test the efficacy of this treatment. – Stephanie DeMarco
Reference
1. Shneider, N.A. et al. Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series. Lancet 0 (2025).