The Centers for Medicare & Medicaid Services (CMS) proposed a rule June 12 that would codify the Inflation Reduction Act of 2022’s Medicare Drug Price Negotiation Program, establish new negotiation and drug benefit policies, and modify the fixed combination drug policy – the latter of which would negatively impact biopharmas attempting to extend lifecycles of blockbuster products.
Elixiron Immunotherapeutics Inc. announced positive interim open-label phase II Alzheimer’s disease (AD) study findings of enrupatinib, an oral brain-penetrant colony-stimulating factor 1 receptor inhibitor, with plans to move the asset into a biomarker-based and placebo-controlled study.
Even though children make up a quarter of the population, healthcare technologies are not often designed with them in mind. Investment in pediatric innovation remains limited with investors often viewing returns in the space as less predictable. Nevertheless, a number of companies are looking to address this and are developing technologies for kids. Afterall, with huge investments going into longevity R&D, there is a compelling case for addressing health issues at the very early stage, delegates heard at the first annual Pediatric Innovation Summit, held as part of the HLTH Europe conference in Amsterdam on June 15.
Despite some back-and-forth with the U.S. FDA regarding inclusion in the somewhat controversial Commissioner’s National Priority Voucher (CNPV) program, Sanofi SA’s Tzield (teplizumab) gained FDA clearance, expanding use of the CD3-directed monoclonal antibody as a disease-modifying therapy for patients with recently diagnosed stage 3 type 1 diabetes (T1D).
Backers of Boston-based Elicio Therapeutics Inc. may have jumped the gun as they ran away from the firm after data were made public from the phase II Amplify-7P study testing ELI-002 7P in adjuvant KRAS-mutation-driven pancreatic ductal adenocarcinoma (PDAC) following standard locoregional therapy. Wainwright analyst Robert Burns said the results are “more nuanced than they appear” and the sell-off represents “an attractive entry point” for investors. Shares (NASDAQ:ELTX) closed June 15 at $4.08, down $10.77, or 72%, in the wake of news that the trial missed the pre-specified primary endpoint of disease-free survival (DFS) in the intent-to-treat population.
Treatment with first-generation CAR T cells regularly sent patients to the intensive care unit. Now, investigators are envisioning a future where CAR T treatment could occur on an outpatient basis. At Sunday’s late-breaking oral session of the 2026 Annual Congress of the European Hematology Association (EHA2026), Lei Fan told his audience that the first-in-human data “support further development of LB-2501 as a potential first-in-class, off-the-shelf, single infusion, no lymphodepletion, outpatient use CAR T therapy.” Fan is a professor of hematology at the First Affiliated Hospital of Nanjing Medical University.
With full phase II results in hand, MBX Biosciences Inc. backers are stacking the odds of parathyroid hormone (PTH) peptide prodrug canvuparatide (canvu) against Yorvipath (palopegteriparatide} from Ascendis Pharma A/S, a PTH analog cleared by the U.S. FDA in August 2024 for hypoparathyroidism (HP).
In a repeat move, the U.S. FDA issued yet again a complete response letter (CRL) to Camurus AB for its subcutaneous extended-release injection drug CAM-2029 (octreotide) to treat the rare chronic growth disorder acromegaly. The drug, which expects to be branded Oclaiz in the U.S. upon approval, is called Oczyesa in the EU and the U.K., where it received marketing authorization in 2025.
Ethyreal Bio Inc. has come out of stealth mode in order to disclose preclinical data for ETHY-001, its monoclonal antibody targeting thyroid stimulating hormone receptor, at the Endocrine Society’s 2026 annual meeting on June 15.
Laekna Inc. is handing ex-China rights to internally discovered PI3Kα pan-mutant selective inhibitor LAE-118 to U.S.-based Vasque Bio Inc. in a deal worth up to $527 million, marking another asset-monetization move by the Shanghai company as it looks to fund and accelerate development across its oncology and metabolic disease pipeline.