IPI-940

September 25, 2017 By Mark Terry , BioSpace.com Breaking News Staff Bigger is not always better. And Pfizer ’s trying to prove it. The company launched a six-person biotech startup, SpringWorks Therapeutics , with a $103 million Series A financing. Joining Pfizer in funding the company was Bain Capital Life Sciences, Bain Capital Double Impact, OrbiMed and LifeArc , formerly known as MRC Technology . “SpringWorks Therapeutics will pursue the development of medicines across therapeutic areas, focused on diseases where there is an urgent need and the potential for the greatest impact for patients,” said Lara Sullivan , founder and president of SpringWorks Therapeutics and a former vice president at Pfizer, in a statement. “We initially have rights to four very promising experimental therapies and, over time, plan to expand our pipeline by partnering with other life science companies and academic institutions who share in our mission.” STAT notes that, “This may seem odd in that Pfizer spends literally billions of dollars a year advancing treatments of its own. But the company’s executives say they simply don’t have the resources to advance all the promising compounds that catch their eye—and they believe an independent company with the scrappy ethos of a startup will be in a better position to take on that task.” “The problem is very simple,” Sullivan told STAT . “There’s too much good science and not enough resources to advance it. If you want to see grown men cry, stop a program for budget reasons, not based on science.” Although this is a strategy that seems to be gaining steam, as evidenced by Vivek Ramaswamy ’s series of biotech startups built on drugs abandoned by big pharma, that doesn’t mean it’s necessarily easy to convince a big company to give up four of its pipeline drugs—and pay for the privilege. John Carroll, with Endpoints News , writes , “’I’ve got a couple of more gray hairs than I did a few years ago,’ Sullivan tells me, when she got started pursing this project. [ Freda ] Lewis-Hall [Pfizer’s chief medical officer] helped champion the effort, and then they built support among the company’s lawyers, accountants and scientists, who are only too aware that even in a top 10 R&D outfit like Pfizer there are far more development projects than money to fund the work.” The four compounds SpringWorks is starting with are all clinical phase, three for rare diseases, one for post-traumatic stress disorder (PTDS). They are: • PF-03084014 , or nirogacestat, a gamma-secretase inhibitor that the company will take into Phase III for desmoid tumors, a rare, slow-growing non-metastatic tumor of connective tissue. • PF-0325901 , an MEK 1/2 inhibitor for a genetic disease NF1, related to neurofibromatosis. It’s completed a Phase I/II trial and will begin a pivotal trial. • PF-054116266 , or senicapoc, for hereditary xerocytosis, which is a disease where red blood cells become dehydrated, leading to severe anemia and other potentially serious illnesses. • PF-0445784 , an FAAH inhibitor for PTSD. The company also plans to continue evaluating other drugs that may be under resourced but have high potential. Daniel Lynch will join the company as executive chairman; Sullivan is founder and president; Stephen Squinto will be acting head of Research & Development and a member of the board; Saqib Islam will be chief financial officer and chief business officer; and L. Mary Smith will be vice president, Clinical Research and Development.

October 6, 2016 By Alex Keown , BioSpace.com Breaking News Staff NEWARK, N.J. – Biotrial , the French clinical research company perhaps most infamously known for running a trial that left one patient dead and five others hospitalized earlier this year, has opened a new research facility in the United States.The new site is a 70,000 square-foot space and includes 110 hospital bed, which is larger than the company’s Rennes, France facility, which is among Europe’s largest, NPR said. It opened at the end of September. NPR reported the facility will be able to conduct up to 50 Phase I trials per year. François Peaucelle , managing director at Biotrial, told NPR the company has already completed four early stage trials at the new Newark, N.J. site and is in the planning stages to conduct 20 more in coming months. As Biotrial expands to the United States though, the company has to overcome the shadow of the fatal trials earlier this year. When Biotrial celebrated the opening of its New Jersey site, Peaucelle told NPR the Rennes tragedy was a freak accident, one he compared to a plane crash—“very rare, but it can happen.” A French health ministry report found fault with both Biotrial and the drug company, Bial-Portela . The ministry criticized what appeared to be a breakdown of responsibility between Biotrial, the contract research organization conducting the trial, and Bial, the developer of the drug. In its report, investigators from the France’s IGAS social affairs inspectorate said Biotrial “had not properly informed volunteers and had followed a flawed testing protocol.” The French ministry criticized the lab for the slow reaction time of staff. In February Touraine released initial findings of its investigation, which said the lab should have halted trials after the first patient was hospitalized. However, the ministry said the drug was administered to five more patients the next day. The French ministry investigation followed the failure of a Phase I clinical trial studying an experimental fatty acid amide hydrolase inhibitor being developed by Portugal-based Bial-Portela at a Biotrial facility. Following the ministry’s announcement, Biotrial sharply criticized that statement, saying it complied with the law and noted the drug was one developed by Bial and not them. The company also criticized the French ministry for not allowing it to provide any input into the report regarding actions it had taken in the wake of the tragedy in January. The trial that left one man dead, was an early trial of an FAAH inhibitor designed to break down endocannabinoids. The drug was administered to 108 patients, but only six were negatively affected. That trial stigma though could cause some potential customers to rethink a partnership with Biotrial. John LaMattina , the former head of research and development at Pfizer , told NPR that he would balk at working with Biotrial on a clinical trial if he were in his old position at the global drug giant. And LaMattina isn’t the only one to balk. Jean-Marc Gandon , Biotrial’s president, said after the Bial death and hospitalizations happened, the number of Phase I trials the company was conducting dropped from about 60 to six, NPR said.