Er­ic Lan­der

Deer­field Man­age­ment and the fa­mous Broad In­sti­tute of MIT and Har­vard have come up with a nov­el plan for pur­su­ing trans­la­tion­al drug re­search work.

In the deal an­nounced this morn­ing, Deer­field will in­vest more than $50 mil­lion in­to projects at the Broad over the next five years. And the biotech in­vestor plans to fol­low up with sup­port to cre­ate new biotechs out of the re­search, with “sig­nif­i­cant ad­di­tion­al re­sources” avail­able for de­vel­op­ment work.

“Over the last decade, sci­en­tif­ic ad­vances have made it pos­si­ble to rapid­ly un­der­stand the bi­o­log­i­cal ba­sis of dis­ease at un­prece­dent­ed res­o­lu­tion,” said Er­ic Lan­der, pres­i­dent and found­ing di­rec­tor of the Broad In­sti­tute. “The chal­lenge now is to de­vel­op ways to speed the process of turn­ing these sci­en­tif­ic in­sights in­to ther­a­pies that ben­e­fit pa­tients.”

Deer­field isn’t stop­ping there. It plans to trans­fer any prof­its for its Health­care In­no­va­tions Fund over to its phil­an­thropic arm, which will be used to sup­port its non­prof­it work.

James Fly­nn

“Over the last few years, Broad In­sti­tute re­searchers have de­vel­oped a num­ber of un­prece­dent­ed bi­o­log­i­cal in­sights and ther­a­peu­tic hy­pothe­ses that are ear­ly-stage but po­ten­tial­ly trans­for­ma­tive,” said James Fly­nn, Man­ag­ing Part­ner of Deer­field. “Mov­ing these hy­pothe­ses from the lab to­ward the clin­ic re­quires sig­nif­i­cant fi­nan­cial re­sources to build on work launched by phil­an­thropy and grants, and are not yet far enough along to at­tract tra­di­tion­al ven­ture fund­ing or phar­ma­ceu­ti­cal col­lab­o­ra­tions. We are pre­pared to fund and de­vel­op these projects so Broad re­searchers can im­me­di­ate­ly pur­sue bold ideas, and have con­fi­dence that re­al break­throughs will have ad­di­tion­al sup­port to move to­ward clin­i­cal suc­cess.”

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.