NOVARTIS

J.P.-G. consults for Lilly, Roche, Eisai, Daiichi Sankyo, AstraZeneca, Seattle Genetics, Gilead, MSD; receives travel compensation from Roche; employed by MEDSIR. G.A. receives honoraria from MEDSIR. M.G. receives travel support and honoraria from AstraZeneca, Gilead Sciences, Roche, Pfizer. A.L.-C. receives research support, consults, speaks, and travels for Roche, Lilly, Pfizer, Novartis, Merck Sharp & Dohme, Gilead, Daiichi Sankyo; owns MEDSIR, Initia-Research. J.C. consults, receives honoraria, research funding, stock, travel expenses, and patents for Roche, Celgene, Cellestia, AstraZeneca, Seattle Genetics, Daiichi Sankyo, Erytech, Athenex, Polyphor, Lilly, Merck Sharp & Dohme, GSK, Leuko, Bioasis, Clovis Oncology, Boehringer Ingelheim, Ellipses, HiberCell, BioInvent, GEMoaB, Gilead, Menarini, Zymeworks, Reveal Genomics, Expres2ion Biotechnologies; holds patents for pharmaceutical combinations.

Autolus secures FDA approval for Aucatzyl, a CD19-targeting CAR-T therapy for relapsed or refractory B-cell precursor ALL, with no REMS requirement. Aucatzyl achieved 42% complete remission in the FELIX trial, with low rates of CRS and neurotoxicity. Competing with Novartis' Kymriah and Gilead/Kite's Tecartus, Aucatzyl aims to capture market share in the US, targeting around 3,000 patients.

Adlai Nortye (ANL) is developing Buparlisib, a PI3K inhibitor for recurrent/metastatic HNSCC post-anti-PD(L)1 treatment, with a phase III trial (BURAN) underway. Expected OS data in Q1 2025. ANL also has two other clinical drugs, AN4005 and AN0025, and a cash position of $98M as of June 2024.

The article discusses the challenges sponsors face with outsourced monitoring in clinical trials, emphasizing the need for stricter oversight and potential solutions, including reclaiming in-house monitoring to ensure patient safety and data integrity.

New Huntington's disease therapies are nearing R&D finish lines, aiming to be the first disease-modifying treatments. Despite high-profile failures, companies like Wave, Prilenia, and uniQure are progressing towards regulatory approval. The field has gained momentum after decades of trial and error, with several mid-stage clinical trials underway. The ideal therapy would selectively lower mutant huntingtin and be widely distributed in the brain, though none currently exist.

The life sciences industry impacts communities and regions, creating thousands of jobs and driving economic activity. In the U.S., it made up 1.6% of GDP in 2022, employing over a million people. Mestrinaro notes that every dollar spent generates $3 in economic activity, with each job leading to five more in the wider economy. Innovation thrives in environments that protect intellectual property and have flexible regulatory systems. The Piedmont region, home to Novartis’s Ivrea facility, exemplifies this with over 400 life sciences businesses employing around 10,000 people. Cirio emphasizes the dual benefits of life sciences in healthcare and employment, advocating for public-private collaboration to accelerate development of new treatments and technologies.

Goodwin's two-part series on Greater China's life sciences sector highlights stable M&A activity in pharmaceutical/biotech despite market fluctuations. Notable 2024 deals include Fosun Pharma's $1.71B acquisition of Shanghai Henlius Biotech and Johnson & Johnson's $850M acquisition of Proteologix, Inc. Trends indicate a shift towards CAR-T cell therapies and large molecule/biologics, with medical devices also playing a significant role. Factors driving recovery include regulatory changes, market readjustments, and the maturing of early-stage investments.

Verona Pharma, a biopharmaceutical company, focuses on respiratory disease treatments, with ensifentrine in Phase 3 trials for COPD, asthma, and cystic fibrosis. Caligan Partners LP, an activist investor, sees potential in Verona's ensifentrine, predicting significant revenue growth and possible indication expansion for non-cystic fibrosis bronchiectasis.

The global spinal muscular atrophy market is projected to grow from USD 2,013.5 million in 2024 to USD 4,823.9 million by 2031, at a CAGR of 13.3%. Key drivers include FDA approvals for drugs like Zolgensma and Evrysdi, rising awareness, and increasing gene therapies. North America is expected to dominate due to its established healthcare infrastructure.