AstraZeneca

BridgeBio's acoramidis, a transthyretin stabilizer, may soon compete with Pfizer's tafamidis for treating transthyretin amyloid cardiomyopathy (ATTR-CM). Acoramidis, with an FDA action date of Nov. 29, could see approval, potentially challenging Pfizer's established position in the $5.2 billion ATTR-CM market. BridgeBio's Phase III trial showed acoramidis improved survival rates and reduced hospitalizations, though it faces challenges like twice-daily dosing and lack of mortality benefit data compared to tafamidis. Emerging RNA therapies, like Alnylam's Amvuttra and Intellia's nex-z, also show promise in treating ATTR-CM.

AstraZeneca's Tagrisso (osimertinib) recommended for EU approval in treating unresectable NSCLC with EGFR mutations, based on LAURA Phase III trial results. Tagrisso extends disease progression time by over three years, addressing unmet need in unresectable EGFR-mutated lung cancer.

Eli Lilly's Muvalaplin, an oral small molecule drug, showed a reduction of lipoprotein(a)– Lp(a)– levels by up to 85% in Phase 2 trials, potentially lowering cardiovascular disease risk. Muvalaplin selectively inhibits Lp(a), a genetic risk factor for heart disease affecting 63 million Americans. The drug blocks the interaction between apolipoprotein(a) and apolipoprotein B, preventing Lp(a) formation. These results highlight a potential scientific advancement in cardiovascular disease treatment.

CHMP recommends AstraZeneca's Tagrisso for EU approval to treat locally advanced, unresectable NSCLC with EGFR exon 19 deletions or exon 21 mutations, post-platinum-based CRT. Tagrisso reduced disease progression or death risk by 84% in Phase III LAURA study.

AstraZeneca’s Tagrisso recommended for EU approval in treating EGFR-mutated, unresectable NSCLC after platinum-based CRT, based on LAURA trial results showing 84% reduction in disease progression risk. Tagrisso extends PFS to 39.1 months vs 5.6 months for placebo, with OS results ongoing.

CHMP of EMA based its positive opinion on LAURA Phase III trial results showing Tagrisso reduced disease progression or death risk by 84% compared to placebo. Median progression-free survival was 39.1 months with Tagrisso versus 5.6 months for placebo. Overall survival results are still immature. Tagrisso is approved in over 100 countries for various EGFRm NSCLC treatments.

Pharma leaders discussed clinical trial quality at SCOPE Europe, emphasizing the shift from reactive to proactive, risk-based approaches. Challenges include cultural resistance and high implementation costs, highlighting the need for senior leadership support and data-driven decision-making to ensure quality and efficiency in clinical trials.

Mitiperstat, a myeloperoxidase inhibitor, showed no significant improvement in quality of life or functional capacity in heart failure patients with mid-range to preserved ejection fraction in the ENDEAVOR trial. However, it reduced heart failure hospitalizations by 36%, suggesting further exploration in larger trials.