Mavis Ag­band­je-McKen­na Stride­Bio

Hav­ing tast­ed ear­ly suc­cess in treat­ing a num­ber of mus­cu­lar dy­s­tro­phies with gene ther­a­py, Sarep­ta is set­ting it­self up to tack­le more rare dis­eases.

The Boston-based biotech has picked Stride­Bio for a part­ner­ship ev­i­dent­ly de­signed to run years, kick­ing things off with $48 mil­lion up­front and four cen­tral ner­vous sys­tem tar­gets. The small­er part­ner is tasked with all R&D work up to the IND stage, com­ing up with the AAV-based ther­a­pies to be test­ed in Rett syn­drome (MECP2), Dravet syn­drome (SCN1A), An­gel­man syn­drome (UBE3A) and Nie­mann-Pick (NPC1).

Co-found­ed by Mavis Ag­band­je-McKen­na and Ar­avind Asokan, Stride­Bio’s pitch cen­ters around its struc­ture-dri­ven cap­sid tech­nol­o­gy drawn from non-hu­man pri­mates, which it says can bet­ter di­rect these shells to tis­sues of in­ter­est and evade neu­tral­iz­ing an­ti­bod­ies. For Sarep­ta, the lure al­so lies in the po­ten­tial to re­dose pa­tients who have re­ceived AAV-de­liv­ered gene ther­a­pies be­fore.

Ar­avind Asokan Stride­Bio

Sarep­ta’s first for­ay in­to the field was a mi­crody­s­trophin ap­proach to Duchenne mus­cu­lar dy­s­tro­phy, an ex­ten­sion of its ef­forts with its mar­ket­ed ex­on-skip­ping drug Ex­ondys-51. In a re­cent bout, it ap­peared to score high­er than Pfiz­er in a Phase Ib, buoy­ing its prospects. Pre­lim­i­nary func­tion­al da­ta on a limb gir­dle mus­cu­lar dy­s­tro­phy ac­quired from My­onexus al­so ap­peared pos­i­tive.

Its new deal al­so pro­vides an ex­clu­sive op­tion to four ad­di­tion­al ge­net­ic tar­gets for CNS or neu­ro­mus­cu­lar ail­ments, which could cost up to $42.5 mil­lion up­front. Stride­Bio may co-de­vel­op and co-com­mer­cial­ize one of the eight to­tal prod­ucts — just the right num­ber for the small biotech, which is al­so jug­gling a pre­clin­i­cal pact with Take­da fea­tur­ing three tar­gets in­clud­ing Friedre­ich’s Atax­ia.

CEO Sapan Shah talked up the re­sources and ex­per­tise they will gain for ex­pand­ing their re­search and man­u­fac­tur­ing plat­form rapid­ly.

Sapan Shah Stride­Bio

“Our part­ner­ship with Stride­Bio ex­pands our re­search port­fo­lio by up to eight new tar­gets and, through our strate­gic part­ner­ing ap­proach that has our col­lab­o­ra­tor lead all IND-en­abling re­search and de­vel­op­ment, en­sures that we gain ac­cess to new tech­nol­o­gy and tar­gets while not dis­tract­ing Sarep­ta from its near-term pri­or­i­ties,” Sarep­ta chief Doug In­gram added in a state­ment.

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.