NOVARTIS

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.

Novartis v. Hetero USA Inc. (2024 WL 3757086) involves a Hatch-Waxman patent infringement action over Entresto®. The court found the ’659 patent invalid for lack of written description, denying Novartis’s injunction request pending appeal. Judge Andrews was skeptical of Novartis’s claims of irreparable harm, noting that MSN’s launch should not be blamed for other generic drug makers’ actions or Novartis’s business decisions. The court granted a temporary stay for Novartis to seek relief from the Federal Circuit.

Bruce Albala, a key figure in Alzheimer's disease drug development, passed away during his 40th wedding anniversary in Greece. Known for his work on β-secretase inhibitors, Albala led Eisai's BACE program, guiding elenbecestat through Phase 3 trials. Despite elenbecestat's limited success, Albala's pragmatic approach and advocacy for safer BACE inhibitor doses were highly regarded. He transitioned to academia in 2019, focusing on improving AD clinical trial efficiency.

FDA considers removing oral phenylephrine from OTC guidelines due to lack of efficacy, not safety concerns. Public comments invited until May 7, 2025, before final decision. Phenylephrine is in many nasal decongestants like Sudafed and Mucinex. FDA's proposal does not affect nasal spray decongestants.

Experts discuss tirzepatide's safety and benefits after a UK death linked to the drug, highlighting the need for high-quality trials and the importance of the 'yellow card scheme' for reporting side effects.