| | | |   | Hello, one and all. Damian here, inviting you to consider the lessons of Covid-19 trials, the future of Vertex Pharmaceuticals, and whether the pandemic will forever be a big business. | | | How to get better at doing clinical trials One of the big lessons from the pandemic is an old one: Rely on randomized controlled trials when trying to figure out which drugs work and which ones do not. Another lesson, STAT’s Matthew Herper writes, is that we need to get a whole lot better at doing those randomized controlled studies. Cue what are called platform trials.
“The only trials that have given us useful information during the pandemic have been the platform trials,” Edward Mills, the head of one of those trials, told Herper. These trials compare multiple drugs to placebo or usual care, and can be a lot cheaper than the big trials run by drug firms. They’ve also given us a lot of the most important data from the pandemic, including that steroids and monoclonal antibodies are effective against Covid, and that hydroxycholoroquine and ivermectin are not.
Read more. | Vertex’s next big idea moves forward Vertex Pharmaceuticals said yesterday that it will advance one of the most closely watched drugs in its research pipeline into a late-stage clinical trial involving patients with an inherited form of chronic kidney disease. The drug, called VX-147, has generated considerable buzz from investors because of its potential to become Vertex’s next blockbuster commercial product, following the success of its cystic fibrosis treatments.
Last December, Vertex reported preliminary but positive results for VX-147 in a small study of patients with a rare genetic kidney disease called FSGS caused by variants in the APOL1 gene. The planned Phase 3 study that will start later this month intends to confirm those early findings but also expand the potential use of VX-147 to a much larger group of patients with different types of kidney disease also caused by APOL1 gene mutations.
The planned study of approximately 400 patients will run for two years, but Vertex said regulators agreed to an interim analysis after one year that, if positive, could allow the company to seek early, or accelerated, approval. | Digital health technology delivers precision recruitment at scale in research study Evidation researchers, along with researchers from the Biomedical Advanced Research and Development Authority (BARDA) were able to 1) directly recruit subjects for a Covid-19 study via a mobile application they were already using, and 2) create an AI risk model that allows targeted recruiting of persons most likely to become infected during the study, while also recruiting a representative cohort. Read more. | Is it a good time to start a Covid company? One of the problems with developing treatments for Covid is that there are now so many that work, with many more now in the late stages of testing. But, then again, if the SARS-CoV-2 virus is going to become endemic, we may need treatments for a long time.
That explains the launch of Aerium Therapeutics by investor Omega Funds, with participation from F-Prime Capital. The exact funding amount was not disclosed, but a big hire was: CEO Rajeev Venkayya, until now the head of Takeda’s vaccine business.
The company will focus first on monoclonal antibodies for preventing Covid infection and complications in immunocompromised people, where Venkayya sees a clear regulatory path and an immediate need. But he says that his goals are much broader. “I would not have left Takeda and got on board for two monoclonals,” he told STAT. He did say, however, that he had exacted a promise that the company’s treatments, if successful, would launch at the same time in rich countries and poorer ones. “That’s what I needed to hear to make sure this is aligned with my values,” he said. | Al Sandrock got a full-time job Al Sandrock, the top scientist at Biogen who left the company last year in a dispute over its polarizing Alzheimer’s treatment, is heading to Voyager Therapeutics to be its next CEO.
As STAT’s Adam Feuerstein and Matthew Herper report, taking the job is a reset for Sandrock, who spent more than two decades at Biogen and became the face of its years-long effort to win FDA approval for Aduhelm.
He joins a company in the midst of a reset all its own. Founded in 2013, Voyager set out to develop gene therapies for neurological diseases. A series of setbacks have cut the company’s valuation by about 50% over the last three years, but Voyager’s proprietary technology has attracted partnerships from Novartis and Pfizer. Hiring Sandrock, the company said, is the next step to rebuilding Voyager’s internal pipeline.
Read more. | More reads - With new ‘brain-reading’ research, a once-tarnished scientist seeks redemption. STAT
- Trial begins for former Theranos president Ramesh ‘Sunny’ Balwani. Wall Street Journal
- Before Aduhelm, Billy Dunn was a superstar at the FDA. STAT
| Thanks for reading! Until tomorrow,  | | | |
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