Merck and Mayo Clinic launch AI-driven precision medicine partnership
Merck and Mayo Clinic have officially kicked off a landmark R&D agreement that pairs one of the world’s top hospital systems with a global pharma giant to rethink drug discovery. This partnership is unique because it grants Merck direct access to the Mayo Clinic Platform Orchestrate, a secure environment containing de-identified, multimodal clinical data ranging from genomic sequences to medical imaging. The goal is to move beyond simple data-crunching and toward virtual cell technologies that can predict how complex diseases like multiple sclerosis and atopic dermatitis will respond to new compounds. By leveraging advanced analytics on longitudinal patient records, the teams hope to identify therapeutic targets that have a much higher probability of success in human trials. For the practicing specialist, this signifies a future where treatment protocols are driven by computational biology rather than broad-spectrum experimentation. While the initial focus is on gastroenterology and neurology, the framework could eventually be scaled across all of internal medicine. This dry-lab revolution aims to cut years off the development cycle for biologics. However, the true test will be whether these AI-validated targets can successfully navigate the complexities of Phase 3 testing. We do not yet know how this deep-data integration will affect the pricing of the specialized therapies it produces.
Oral remibrutinib hits primary endpoint for cold and pressure-induced hives
Novartis reported today that its selective BTK inhibitor, remibrutinib, successfully met all primary endpoints in the pivotal Phase III RemIND trial for chronic inducible urticaria (CIndU). This is a big win for the estimated 29 million people globally who suffer from hives triggered by specific environmental factors like cold, friction, or exercise. The data showed that patients taking the oral pill achieved significantly higher complete response rates at 12 weeks compared to those on placebo, as confirmed by standardized provocation tests. What makes remibrutinib interesting is its ability to block the release of histamine at the source by inhibiting the Bruton’s tyrosine kinase pathway. For the practicing specialist, this could mean the first targeted oral therapy for a condition that has long been managed with subpar, high-dose antihistamines. The safety profile remained clean, with no liver toxicity signals, a common concern with older drugs in this class. Novartis has already moved to file a supplemental application with the FDA to bring this to market as soon as possible. While the drug is already approved for spontaneous hives, this new data expands its reach to the most difficult-to-treat inducible subtypes. Clinicians will need to see if the drug maintains its efficacy over years of continuous use. It remains unclear if remibrutinib will eventually replace biologics as the first-line preference for refractory cases.
FDA grants priority review for iberdomide-based multiple myeloma regimen
The FDA has officially accepted Bristol Myers Squibb’s application for iberdomide, granting it priority review status for the treatment of relapsed or refractory multiple myeloma. Iberdomide is the lead candidate for a new class of oral drugs called CELMoDs, which are essentially souped-up versions of the immunomodulatory agents clinicians have used for decades. The filing is anchored by the EXCALIBER-RRMM trial, which demonstrated that adding iberdomide to a daratumumab and dexamethasone backbone significantly improved minimal residual disease (MRD) negativity. This is a high-stakes moment for the agency, as it leans into using MRD as a surrogate endpoint to speed up approvals for blood cancers. For hematologists, this oral regimen offers a potentially more potent and manageable option for patients who have already cycled through standard therapies. The FDA has set a target action date of August 17, 2026, which could make it the first CELMoD on the market. By degrading specific transcription factors like Ikaros and Aiolos more effectively, iberdomide may overcome the resistance that often develops in late-stage disease. However, the market competition with newer CAR-T therapies and bispecific antibodies will be a factor in how it is utilized. We are still waiting for mature progression-free survival data to see if the MRD results translate into long-term remissions. The manageable safety profile seen so far suggests it could become a versatile partner in several combination strategies.
Oral infigratinib achieves growth velocity gains in Phase 3 achondroplasia study
BridgeBio Pharma has released topline Phase 3 results for infigratinib, and the numbers are impressive for children with the most common form of dwarfism. The PROPEL 3 study showed that children taking the once-daily oral pill grew an average of 2.10 cm/year faster than those on placebo, meeting the primary endpoint with high significance. Perhaps more importantly for pediatricians and families, the drug showed a significant improvement in proportionality, helping to normalize the upper-to-lower body ratio that often causes orthopedic issues. This is the first time an oral small molecule has demonstrated such high growth velocity in a randomized trial. For specialists, infigratinib offers a non-injectable alternative to current therapies, which often require daily shots that are difficult for families to maintain. The drug targets the overactive FGFR3 receptor directly, treating the condition at its molecular source rather than just managing symptoms. BridgeBio is preparing to submit its marketing applications globally in the second half of 2026. While the linear growth is the headline, the long-term impact on spinal stenosis and sleep apnea remains a point of continued study. No serious treatment-related adverse events were reported, which is a major relief for a chronic pediatric therapy. We don’t yet know if the proportionality gains will translate into better functional outcomes in adulthood.
Shingles vaccine and sildenafil identified as top Alzheimer’s repurposing leads
An international panel of 21 dementia experts just wrapped up a major review, pinpointing the shingles vaccine and sildenafil as the most promising drugs to repurpose for Alzheimer’s. The study, led by the University of Exeter, combed through 80 existing medications to find candidates that could protect the brain by targeting neuroinflammation and blood flow. The shingles vaccine emerged as the front-runner because previous observational data suggests that people who get the jab may be significantly less likely to develop dementia. Sildenafil also made the cut due to its ability to cross the blood-brain barrier and potentially reduce the buildup of toxic tau proteins. For the primary care physician or geriatrician, this highlights a potential two-for-one benefit for patients already taking these common medications. The beauty of drug repurposing is that it uses medicines with decades of established safety data, skipping years of early-stage clinical risk. The experts are now calling for large-scale randomized trials to confirm if these protective effects actually hold up in a prospective setting. While you should not be prescribing these for cognitive health just yet, the findings suggest we might have powerful dementia-fighting tools already sitting in our pharmacies. It is still unclear if newer shingles vaccines will offer the same level of neuroprotection as the older versions studied here. We also need more data on how these drugs might interact with the new amyloid-clearing monoclonals entering the market.
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