📉The Swiss pharma company reported a Phase 3 miss on Sept. 4 for its antisense oligonucleotide pelacarsen in patients with cardiovascular disease. The results showed that pelacarsen reduced patients’ high levels of a molecule called lipoprotein(a), or Lp(a). But it was no better than placebo at reducing the risk of cardiovascular events. The disconnect raises questions about the link between lowering Lp(a) levels and cardiovascular outcomes. 

Less than a week later, Novartis disclosed that a separate program failed a late-stage trial in a neuromuscular disease. The antibody-oligonucleotide conjugate, called delpacibart etedesiran or del-desiran, came from Novartis’ $12 billion acquisition of Avidity Biosciences. It wasn’t statistically significantly better than placebo in patients with myotonic dystrophy type 1 (DM1). Jefferies analysts wrote that DM1 represented about a third of peak sales from the Avidity acquisition.

The study miss, plus that of pelacarsen, wiped 10% off Novartis’ Swiss-listed stock in early trading Tuesday. Novartis said that despite the back-to-back trial failures, it was still on course to meet its long-term guidance of between 5% and 6% sales growth per year through the 2020s. The failures mark the latest setbacks for Novartis, following a disclosure late last month that three patients died after taking its CAR-T candidate rapcabtagene autoleucel. Novartis paused several of its immunology and neuroscience studies as a result.

🤖The company behind Claude has made serious moves into biopharma these last few months, and it took its next step by posting a job for a corporate development lead in the industry, Andrew Dunn reports. According to the posting, Anthropic wants someone who can “source and execute acquisitions, investments, and strategic partnerships with AI-native life sciences companies” — all things that confirm its drug development ambitions.

Anthropic’s posting comes as the company has embarked on a 100-day sprint into the life sciences. As Andrew reported earlier this summer, that included a $400 million acquisition, the hiring of Nobel laureate John Jumper, and the announcement of vague plans to develop its own drug pipeline. 

But it’s still early days for Anthropic’s ambitions, emphasized by an incredibly broad range of potential interest areas. Whoever wins the new job will be responsible for setting Anthropic’s life sciences strategy, which includes areas of potential interest like drug discovery, regulatory writing, lab automation and healthcare data infrastructure. It also comes amid Anthropic’s potential IPO, which could put the company’s value as high as $2 trillion. 

🏆The science behind a new narcolepsy drug has won a Lasker Award, which is known as a precursor to a Nobel Prize. The award is shared by Masashi Yanagisawa and Emmanuel Mignot, and comes after the two researchers shared the Breakthrough Prize in 2023. Takeda’s Orzeyful, the first in a class of treatments developed based on the award-winning research, was approved for a form of narcolepsy last month.

It’s only recently that orexin agonists like Orzeyful have captured the attention of the biopharma industry. The interest is partly due to their potential use outside of narcolepsy, for conditions such as ADHD and fatigue related to diseases like multiple sclerosis and Parkinson’s. On the heels of Takeda and its newly approved drug are Alkermes and Eli Lilly, which bought Centessa Pharmaceuticals and its orexin agonist earlier this year for $6.3 billion upfront.

Mignot, who treats patients with narcolepsy, has seen dramatic benefits when providing Orzeyful to his patients. As for whether orexin agonists will treat other conditions, Mignot said it’s too early to tell. You can read more here from Lei Lei Wu.

🩸A Phase 2 study from Bristol Myers Squibb may provide some answers for how to treat patients whose cancers have progressed after taking a CAR-T therapy. This week, the company said an experimental CAR-T called arlo-cel improved response rates in patients with relapsed and refractory multiple myeloma who had received at least four previous therapies. Arlo-cel is directed toward GPRC5D, a target that is expressed in both myeloma cells and bone marrow plasma. 

Researchers believe going after GPRC5D may be particularly effective for patients who had progressed after receiving BCMA-targeting CAR-T or other BCMA-directed medicines. The FDA has approved a total of six CAR-T therapies, all of which target either CD19 or BCMA. In the study, Bristol Myers enrolled patients whose cancers had progressed after receiving a BCMA CAR-T or another BCMA-directed treatment. Read more about the study here.