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A Landmark Blood Cancer Treatment Just Delivered Stunning Trial Results
For nearly 30 years, patients with essential thrombocythemia (a rare blood cancer that floods the bloodstream with excess platelets) had exactly zero new approved options. PharmaEssentia's BESREMi is aiming to end that drought, and the clinical data is staggering: 42.9% of patients hit the primary endpoint versus just 6.0% on the comparator. That's a seven-to-one advantage. Unlike older drugs that simply suppress platelet counts, BESREMi goes after the root cause by targeting the mutated stem cells driving the disease, offering the first shot at actually altering its trajectory.
Why it matters: This drug fills one of the longest treatment gaps in oncology and validates interferon-based disease modification in myeloproliferative neoplasms, a historically neglected corner of blood cancer.
Read more →Deals and M&A
Lilly Drops $2.88 Billion on Tech That Selectively Destroys Rogue Antibodies
Eli Lilly is acquiring Merida Biosciences and its platform for selectively destroying disease-causing autoantibodies while leaving healthy immune defenses intact. The $2.875 billion deal (cash, with milestones) buys one Phase 1 asset in Graves' disease plus two preclinical programs. Lilly is signaling that autoimmune disease is its next major growth pillar alongside obesity, betting that precision beats the old "carpet-bomb the immune system" playbook.
Read more →Genentech Pays Up to $2.3 Billion for an Obesity Drug That Builds Muscle
Every GLP-1 drug has the same problem: patients lose muscle along with fat. Genentech just licensed HM17321 from Hanmi Pharmaceuticals for $190 million upfront (up to $2.3 billion total) because it takes a completely different approach. The drug activates CRF2 receptors to burn fat while actively growing lean mass, a trick no semaglutide or tirzepatide can pull off. It's still Phase 1, but Genentech is assembling a full obesity roster to challenge Novo and Lilly.
Read more →Ascendis and BioMarin Settle Their Multi-Country Patent War Over YUVIWEL
After litigation spanning multiple countries and the ITC, Ascendis Pharma and BioMarin signed a binding term sheet settling their fight over achondroplasia drug YUVIWEL. Ascendis pays BioMarin 20% of U.S. net sales (18% in other markets) through May 2030 in exchange for a worldwide patent license. Both sides drop all lawsuits. BioMarin collects royalties; Ascendis gets the legal clarity to actually sell its drug.
Read more →Clinical and Regulatory
Sarepta's Gene Therapy Earns the Scariest Label in Medicine, Then Cuts 500 Jobs
Two fatal liver failures in pediatric patients just earned Sarepta's Elevidys a black box warning, the FDA's most severe safety alert. The agency also pulled the non-ambulatory indication entirely, shrinking the eligible patient pool. Sarepta is responding by cutting 36% of its workforce (roughly 500 people), shelving limb-girdle gene therapy programs, and pivoting toward siRNA-based approaches. Quarterly revenue has been in freefall since early 2025.
Read more →This Stroke Catheter Clears Brain Clots With a 77% First-Pass Rate in M1 Occlusions
Irish medtech Perfuze reported that its Millipede88 catheter achieved 88.9% reperfusion within three passes without rescue therapy in the pivotal MARRS trial, with a 77% first-pass success rate in the key M1 subgroup. When roughly 1.9 million neurons die every minute during a major stroke, getting it right on the first attempt isn't a nice-to-have; it's the difference between independence and disability. A limited U.S. launch is planned at select stroke centers.
Read more →Science and Discovery
An AI Designed 1,320 Proteins. Most of Them Actually Worked.
Anthropic's Claude autonomously designed 1,320 protein binders across 15 biological targets, and independent labs at Twist Bioscience and Adaptyv Bio confirmed 354 of them actually stuck to their intended targets. Hit rates ranged from 22.6% to 35.1%, comfortably beating the 10–15% benchmark for traditional protein design. These are still research-stage molecules (binding in a dish is a long way from a drug), but the compressed design-build-test loop hints at what production-scale AI discovery could look like.
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