Successful Transplant Is ‘Exciting Step Forward’ For Curing Type 1 Diabetes

MedPage Today: Novel T1D Therapy Opens The Door To Cell Transplants Sans Immunosuppressants
For the first time, a man with type 1 diabetes (T1D) is making his own insulin after undergoing islet cell transplantation and without any use of immunosuppression, researchers reported. This was made possible with the genetic modification of allogeneic donor islet cells to avoid rejection, via CRISPR-associated protein 12b (Cas12b) editing and lentiviral transduction, according to the case report and proof-of-concept study from Per-Ola Carlsson, MD, PhD, of Uppsala University in Sweden, and colleagues. (Lou, 8/4)

More pharma and tech developments —

Stat: Stealth BioTherapeutics Contemplates Closure After 'Conflicting' FDA Signals
In the latest twist over the fate of an ultra-rare disease drug, Stealth BioTherapeutics said it received “conflicting” signals from the U.S. Food and Drug Administration about information needed to approve its therapy and, consequently, is making contingency plans to close the company. (Silverman, 8/5)

Bloomberg: Will Eli Lilly Recover From Drug, Tariff Setbacks To Hit $1 Trillion Valuation?
A year ago, Eli Lilly & Co. was poised to become the first pharmaceutical company to register a trillion-dollar market valuation. It still hasn’t cracked that ceiling. Instead, a series of weak earnings reports, a setback for its obesity drug and threats of sky-high tariffs have taken Lilly investors on a wild ride only to deposit them almost exactly where they started. Down 21% from an August record, Lilly trades at $762 a share and is worth $722 billion — not bad, but not the lofty figures investors were expecting. (Adegbesan, 8/4)

Modern Healthcare: Becton Dickinson To Invest $35M In Syringe Manufacturing
Becton Dickinson will invest more than $35 million to expand prefilled flush syringe manufacturing at its facility in Columbus, Nebraska. The company said Monday it will add BD PosiFlush prefilled flush syringe production lines and make investments in product innovation and operational efficiencies. The syringes keep catheters clear and are designed to help reduce intravenous catheter-related infections, the chance of medication errors and risk of damage to catheters. (Dubinsky, 8/4)

Stat: Mayo Clinic AI Dementia Tool, StateViewer, Draws Outside Interest
Nathan Young, a community neurologist at the Mayo Clinic, recently saw a patient whose diagnosis he couldn’t quite nail down. Parkinson’s seemed a likely possibility, but Young was concerned she might instead have a rare neurological disorder called progressive supranuclear palsy, or PSP, which can progress much more rapidly. (Palmer, 8/5)

MedPage Today: Tamiflu Findings Raise Questions About Drug Label Warning For Kids
Treatment with the antiviral oseltamivir (Tamiflu) was tied to a reduced risk of serious neuropsychiatric events in children and adolescents, an analysis of Medicaid beneficiaries in Tennessee suggested. Compared with untreated flu, the risk of serious neuropsychiatric events was lower in kids treated with oseltamivir during flu exposure periods ... and post-treatment periods ... reported James Antoon, MD, PhD, MPH, of Vanderbilt University in Nashville, Tennessee and colleagues. (George, 8/4)

MedPage Today: Nearly 17M U.S. Teens, Young Adults Eligible For GLP-1 Agonists
Though an estimated 17 million U.S. adolescents and young adults were eligible for GLP-1 receptor agonists for conditions like obesity and type 2 diabetes, many lacked insurance or a routine place for healthcare, a cross-sectional study suggested. In a sample of adolescents eligible for GLP-1 receptor agonists, 40.3% were insured by Medicaid, 40.5% were privately insured, and 7.2% were uninsured. (Henderson, 8/4)

Stat: Vertex Non-Opioid Pain Candidate Fails Phase 2 Trial 
Vertex Pharmaceuticals said Monday afternoon that its next-generation non-opioid pain reliever failed to significantly outperform placebo in a Phase 2 trial. (Mast and Wosen, 8/4)