Weekly Rundown: CAR T cell success in solid GI tumors

Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.

CAR T cells find success in treating solid GI tumors

Chimeric antigen receptor (CAR) T cells have become life-saving therapies for many different kinds of hematological cancers, but they haven’t proved as successful in treating solid tumors — until now. In a new study published in The Lancet, researchers reported that the CAR T cell therapy, satricabtagene autoleucel (satri-cel), administered in a Phase 2 trial increased survival in people with gastric or gastro-oesophageal junction cancer (GC/GEJ) who had failed at least two prior treatments (1). The Chinese biopharmaceutical company CARsgen Therapeutics developed the therapy, which targets the protein claudin18.2, a biomarker that’s enriched on many solid cancers, including GC/GEJ (2). Of the participants treated with satri-cel, 22 percent responded to the therapy compared to just four percent who responded to the standard of care. The researchers found that both overall survival and progression-free survival were longer in the satri-cel group versus the control group. All but one of the participants treated with satri-cel experienced a high-grade adverse event on the therapy while only 63 percent of those on the standard of care did. These side effects, however, are common for CAR T cell therapies. Lisa Mielke, a cancer researcher at the Olivia Newton-John Cancer Research Institute, told Nature, “Clinicians will be used to dealing with them and managing them, but it certainly means that patients need to be monitored more closely.” – Stephanie DeMarco

A COVID-19 vaccine approval, conflicting guidance, and a resignation

Every week there is more news about the COVID-19 vaccines. Here are some of the updates from this week:

Last Saturday, the FDA approved Moderna’s next-generation COVID-19 vaccine, mNEXSPIKE® (mRNA-1283), but only for people 65 years-old and older and for those aged 12 to 64 with at least one underlying condition. The limited approval to those groups corresponds with the limitations on COVID-19 vaccines the FDA announced in a recent New England Journal of Medicine (NEJM) “Sounding Board” piece (3).
As we discussed in the Weekly Rundown two weeks ago, this new COVID-19 vaccine guidance will only allow people over 65 and those between 6 months and 64 with an underlying condition that puts them at risk of severe COVID-19 outcomes to receive updated COVID-19 vaccines. One of the underlying conditions the FDA listed was pregnancy. Then, last week, HHS Secretary Robert F. Kennedy, Jr. announced that the CDC would no longer recommend COVID-19 vaccines for children or pregnant women at all — directly contradicting the administration’s previous guidance. Both the NEJM article and this announcement were made without consulting anyone in the CDC, which has a working group dedicated to making COVID-19 vaccine recommendations. Now, the CDC website has been updated to say, “Where the parent presents with a desire for their child to be vaccinated, children 6 months and older may receive COVID-19 vaccination, informed by the clinical judgment of a healthcare provider and personal preference and circumstances.” Under this “shared decision making” guidance, insurers must still cover the cost of these vaccinations. However, according to PBS New Hour, “Experts say vaccination rates tend to be lower when health authorities use that language and doctors are less emphatic with patients about getting shots.” It does not appear that there is any similar update for pregnant people on the CDC website.
On Wednesday, Reuters reported that Lakshmi Panagiotakopoulos, a pediatric infectious disease doctor and one of the co-leaders of the CDC working group that makes COVID-19 vaccine recommendations, resigned from both the advisory group and the CDC. She wrote to the members of the working group in an email, “My career in public health and vaccinology started with a deep-seated desire to help the most vulnerable members of our population, and that is not something I am able to continue doing in this role." – Stephanie DeMarco

In a deal up to $9.5 billion, Sanofi acquires Blueprint Medicines

Sanofi announced they will acquire Blueprint Medicines for up to $9.5 billion. Blueprint’s therapies focus on mast cell disorders or solid tumors, with one rare immunology disease medication, Ayvakit, already approved by the FDA to treat three conditions: 1) adults with unresectable or metastatic gastrointestinal stromal tumors with a mutation in the platelet-derived growth factor receptor alpha gene, 2) individuals with advanced systemic mastocytosis, and 3) indolent systemic mastocytosis. Blueprint’s pipeline also includes other KIT inhibitors in early and late state development. In a statement, Sanofi Chief Executive Officer Paul Hudson said, “We are excited to welcome Blueprint’s talented people and we look forward to chasing the miracles of science together. This makes sense for science, for both companies, for healthcare professionals, and – most of all – for patients.” – Allison Whitten

Promising results from Amgen in small cell lung cancer

In a late-breaking presentation at the American Society of Clinical Oncology 2025, Amgen announced results from their Phase 2 DeLLphi-304 trial on Imdelltra, a bispecific delta-like ligand 3–directed T-cell engager immunotherapy. The data showed that for patients with small cell lung cancer who had already been treated with platinum-based chemotherapy, Imdelltra decreased the risk of death by 40 percent and also significantly increased the median overall survival by more than five months compared to standard-of-care chemotherapy. The team simultaneously published the results in NEJM (4). In a press release, Executive Vice President Jay Bradner said, "Small cell lung cancer is an extraordinarily aggressive and difficult-to-treat disease, and those living with SCLC often experience limited benefit with first line treatment … These data underscore IMDELLTRA's potential to transform patient outcomes and the small cell lung cancer treatment paradigm." – Allison Whitten

Vera Therapeutics sees positive results in rare kidney disease

On Monday, the biotechnology company Vera Therapeutics announced that their drug, atacicept, met its primary endpoint in adults with the rare kidney disease, immunoglobulin A nephropathy (IgAN). In this B cell-mediated chronic condition, the cytokines B-cell activating factor (BAFF) and A proliferation-inducing ligand (APRIL) stimulate the production of autoantibodies, which lead to the accumulation of IgA in the kidneys. This immune build up can impair the kidneys’ filtration function and eventually lead to kidney failure. In their Phase 3 study, the Vera team found that atacicept, which is a protein drug that binds to BAFF and APRIL, led to a 46 percent reduction in protein in urine from baseline and a statistically significant 42 percent reduction from placebo. In the news release, the company said that they plan to share the results with the FDA in the next few weeks. “Vera aspires to evolve the practice of kidney medicine with the hope that, one day, patients may no longer face a future of dialysis or transplantation,” said Marshall Fordyce, the Founder and Chief Executive Officer of Vera Therapeutics in the statement. – Stephanie DeMarco

Regeneron’s triple combination of drugs preserves muscle mass during weight loss from GLP-1

On Monday, Regeneron released interim results from their Phase 2 COURAGE trial aimed at treating obesity that combines semaglutide (a glucagon-like peptide-1 (GLP-1) drug) with trevogrumab (an anti-myostatin) and garetosmab (an anti-activin A antibody). The data showed that the combination led to the preservation of approximately 50 to 80 percent of lean muscle compared to semaglutide alone. However, Regeneron also reported higher percentages of treatment emergent adverse events, including severe adverse events, in the triple combination group. The rate of treatment discontinuation was 28.3 percent in patients taking the triple combination compared to 4.6 percent taking semaglutide alone. Furthermore, in the triple combination group, there were two deaths in patients with cardiovascular risk factors or underlying cardiovascular disease, though Regeneron stated that they had not identified a causal relationship to the treatment. – Allison Whitten

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