Acoramidis

RLY-2608, an oral, mutant-selective PI3Kα allosteric inhibitor, addresses off-target toxicities of current modulators, currently in Ph. I for HR+/HER2- breast cancer treatment.

In 2025, genetic validation will be crucial in cardiovascular R&D, investors will focus on proven science, and patients will be more active in healthcare. Precision medicine and genetic data will drive drug development, with data becoming the gold standard for investments. Real-world data (RWD) integration will transform clinical trials, enhancing patient control over medical records.

EMA's CHMP recommended 17 drugs for market authorization in Dec 2024, including 5 orphan drugs and 6 biosimilars. Notable recommendations include Kavigale for COVID-19 prevention in immunocompromised individuals and Kostaive, a mRNA vaccine for COVID-19 prevention. CHMP also extended recommendations for several drugs and withdrew Alofisel from the market.

The CHMP recommended acoramidis, an oral TTR stabilizer, for EU marketing authorization in adult ATTR-CM patients. Acoramidis demonstrated near-complete TTR stabilization and benefits on cardiovascular endpoints in the ATTRibute-CM study. The CHMP's positive opinion offers hope for ATTR-CM patients, with a final EU decision expected soon.

The CHMP recommended acoramidis, an orally administered TTR stabilizer, for EU marketing authorization in adult patients with ATTR-CM. Acoramidis demonstrated near-complete TTR stabilization and benefits on cardiovascular endpoints in the Phase III ATTRibute-CM study. The CHMP's positive opinion offers hope for ATTR-CM patients, with the European Commission's final decision expected soon. Bayer plans to launch acoramidis in Europe in the first half of 2025.

Editas Medicine lays off 65% of staff, shelving its sickle cell disease treatment Reni-Cel; Ionis Pharmaceuticals shifts to in-house drug commercialization; GLP-1 drugs spark debate in Trump's circle; investors sue Bristol Myers Squibb over Padlock Therapeutics milestone payments.

Bayer AG gains EU regulator backing for acoramidis, an oral medicine treating transthyretin amyloidosis in cardiomyopathy patients. Acquired from BridgeBio Pharma for $310M, it aims to offset expiring patents of top-selling drugs. Approved by the FDA, Bayer plans to launch in Europe by H1 2025.

Bayer likely to launch heart drug Acoramidis in EU soon, CHMP recommends approval based on Phase III results. The drug treats ATTR-CM, showing positive impact on cardiovascular endpoints. Bayer acquired European marketing rights in March. Analysts estimate peak sales at EUR 260 million by 2030, aiding in offsetting declining sales of Xarelto.

BridgeBio Pharma announced CHMP recommended EU approval of acoramidis for ATTR-CM, following Phase 3 ATTRibute-CM study results showing a 42% reduction in mortality and cardiovascular hospitalization events, and a 50% reduction in cumulative hospitalization events at Month 30. The drug was FDA-approved as Attruby™ in 2024, the first near-complete stabilization treatment for ATTR-CM in the US. Bayer plans to launch acoramidis in Europe in H1 2025.