Good morning, everyone, and welcome to another working week. We hope the weekend respite was refreshing and invigorating, because that oh-so familiar routine of online meetings, calls, and deadlines has predictably returned. To cope, we are firing up the coffee kettle in the Pharmalot cafeteria and brewing another cup of stimulation. Our choice today is the seasonal pumpkin spice. As always, you are invited to join us. Meanwhile, here is the latest menu of tidbits to help you get started. We hope your day is enervating and challenging. And of course, do keep in touch. A little communication can go a long way. …
Confounding data from a Pfizer clinical trial have rattled the field of gene therapy for Duchenne muscular dystrophy, raising more questions about the regulatory standard used to approve a treatment from Sarepta Therapeutics, and complicating plans for other companies hoping to develop next-generation products, STAT explains. Scientifically, Pfizer’s gene therapy did what it was supposed to do. Boys who received it as part of a Phase 3 clinical trial produced significant amounts of microdystrophin, a lab-built miniature version of the gene mutated in Duchenne. In principle, that should have helped slow a disease that can rob patients of the ability to walk by age 12 and cut lifespan to under 30. Yet, the participants did not benefit at all. The outcome of multiple muscle function tests — the study’s key efficacy goals — showed no improvement compared to a placebo. The discordant trial results, presented on Oct. 12 at the World Muscle Society meeting, left many Duchenne experts perplexed.
Sanofi proceeded with a plan to sell a controlling stake in its consumer-health care unit Opella to Clayton Dubilier & Rice, entering exclusive talks for a deal that values the business at $17.39 billion, including debt, The Wall Street Journal writes. The deal would allow Sanofi to focus on its more lucrative, but riskier, innovative drugs and vaccines, and CD&R to take a 50% controlling stake in a business that is home to brands such as Allegra, Dulcolax, and IcyHot. French state investment bank Bpifrance plans to take a 2% stake in the business to address concerns raised by the French government. Sanofi plans to retain the remaining 48% stake in the business. The drugmaker laid out plans to split its consumer unit from its innovative medicines and vaccines businesses a year ago. But its disclosure earlier this month that it opened talks with CD&R sparked a political debate in France over the future of the unit, which sells over-the-counter medicines such as Doliprane, a popular painkiller. The French government obtained guarantees its demands over employment, production, and investment would be respected for Doliprane and other essential medicines to France.
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