Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Novo Nordisk sues Hims over unapproved Wegovy and Ozempic copies
On Monday, Novo Nordisk announced filing a lawsuit against telehealth provider Hims & Hers, accusing the firm of infringing US patents by mass marketing unapproved copies of its Wegovy and Ozempic obesity drugs. The Danish pharma company said Hims’ compounded products, including oral and injectable semaglutide, contain dangerously high levels of impurities — up to 86 percent in injections and 75 percent in pills — and pose serious health risks, including immune reactions and overdoses. The company is seeking a permanent ban on Hims’ sales of compounded versions of its patented medications and damages. Hims had offered its oral semaglutide for as little as $49 (USD) for the first month, roughly $100 cheaper than Novo’s FDA-approved Wegovy pill. Compounded drugs are custom-made, unbranded medications that contain the same active ingredient as a marketed drug, such as semaglutide, and are allowed in certain situations, such as shortages or personalized regimens, even though they are not FDA-approved. The dispute comes after semaglutide is no longer in shortage in the US, yet Novo estimates as many as 1.5 million Americans have turned to compounded alternatives. Shares of Novo rose more than 3 percent on Monday, while Hims fell more than 18 percent, reflecting investor concern over the escalating feud. – Bree Foster
Fecal microbiota capsules reduce immunotherapy toxicity and boost response in early cancer trials
Two studies published in Nature Medicine report that fecal microbiota transplantation (FMT) capsules may both reduce immunotherapy-related toxicity and improve treatment response in patients with cancer. In a Phase 1 trial of 20 patients with advanced kidney cancer, researchers at Lawson Research Institute and London Health Sciences Centre Research Institute (LHSCRI) found that adding customized FMT to immunotherapy appeared safe and was associated with fewer severe side effects such as colitis and diarrhea, which often force patients to stop treatment. Separate Phase 2 studies in 20 patients each with lung cancer and melanoma, led by investigators at Centre de recherche du Centre hospitalier de l’Université de Montréal (CRCHUM) in collaboration with Lawson and LHSCRI, showed higher response rates when FMT was combined with immunotherapy — 80 percent in lung cancer and 75 percent in melanoma — compared with roughly 39–45 percent and 50–58 percent, respectively, seen historically with immunotherapy alone. The capsules, derived from healthy donor stool and designed to restore the gut microbiome, are now being evaluated in a larger randomized Canadian trial. – Andrea Corona
Lilly expands into in vivo cell therapies with Orna acquisition
Eli Lilly is set to acquire Orna Therapeutics in a deal valued at up to $2.4 billion, giving the pharmaceutical giant access to a pioneering pipeline of in vivo CAR-T therapies for autoimmune diseases. Orna’s technology uses engineered circular RNA and lipid nanoparticles to prompt patients’ own bodies to produce therapeutic immune cells, potentially overcoming the cost and logistical challenges of traditional ex vivo CAR-T treatments. The company’s lead program, ORN-252, targets CD19 to treat B cell-driven autoimmune disorders, and early studies suggest the platform could deliver longer-lasting therapeutic effects than existing RNA or cell therapy approaches. The acquisition is part of Lilly’s broader strategy to diversify beyond its obesity franchise, building a presence in areas including immunology, oncology, ophthalmology, and gene editing. – Bree Foster
BridgeBio posts positive Phase 3 dwarfism data, intensifying competition with BioMarin and Ascendis
BridgeBio Pharma reported positive Phase 3 results for its oral FGFR3 inhibitor infigratinib in approximately 110 children with achondroplasia, showing an annualized height velocity improvement of 1.74 to 2.1cm per year over placebo after one year, with no treatment-related serious adverse events and only mild, transient cases of elevated phosphate. The company plans to meet with regulators later this year to discuss marketing applications and is also advancing the drug in hypochondroplasia. If approved, infigratinib would compete with BioMarin Pharmaceutical’s Voxzogo — the only FDA-approved therapy for achondroplasia, which generated $654 million in the first nine months of last year — and a once-weekly injectable from Ascendis Pharma currently under FDA review. Analysts described the data as potentially best-in-class and noted that comparable or superior efficacy combined with the convenience of an oral option could drive significant patient switching, helping explain a more than 15 percent premarket jump in BridgeBio’s stock and reinforcing expectations that infigratinib could materially expand the company’s valuation in a competitive but well-established market. – Andrea Corona
FDA rejects REGENXBIO gene therapy for Hunter syndrome
The FDA has rejected REGENXBIO’s gene therapy RGX-121 for Hunter syndrome, citing concerns about the trial’s patient population, reliance on natural history controls, and use of a biomarker as a surrogate endpoint. RGX-121, also known as clemidsogene lanparvovec, is a one-time therapy designed to deliver a functional copy of the IDS (iduronate-2-sulfatase) gene to the central nervous system, giving boys with Hunter syndrome a permanent source of the missing enzyme needed to break down sugars that otherwise accumulate in cells and cause heart, breathing, and neurological complications. The therapy has received Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations from the FDA, and Advanced Therapy Medicinal Product classification from the European Medicines Agency. The FDA had already delayed RGX-121’s original deadline and placed the candidate on clinical hold after a patient in a separate REGENXBIO trial developed a brain tumor, though the agency did not cite that event as a reason for rejection. REGENXBIO plans to resubmit the therapy with longer-term data, but the ruling is a major setback for families who face irreversible neurodevelopmental decline, with few treatment options available for the severe neurological manifestations of the disease. - Bree Foster
FDA declines to review Moderna’s mRNA flu shot application
The FDA has issued a refusal-to-file letter declining to review Moderna’s mRNA-based influenza vaccine, mRNA-1010, stating that the Phase 3 trial was not “adequate and well-controlled” because it used a comparator that did not reflect the “best-available standard of care” in the US, though the agency did not cite safety or efficacy concerns. Moderna publicly posted the letter, signed by top vaccine regulator Vinay Prasad, and argued the decision contradicts prior FDA guidance, noting that the agency had reviewed and not objected to the study protocol and that the company also generated separate Phase 3 data using a high-dose flu vaccine comparator. The setback adds to mounting regulatory pressure on mRNA products under current Department of Health and Human Services leadership, complicates Moderna’s plans for both its standalone flu shot and a combination COVID-flu vaccine, and raises questions about the company’s near-term financial outlook as it seeks clarity from the FDA on a path forward. – Andrea Corona
