Sarep­ta picks up a slate of pre­clin­i­cal gene ther­a­py pro­grams, seed­ing new Stride­Bio deal with $48M up­front

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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

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.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Geoff McDonough, Generation Bio president and CEO

Mod­er­na part­ners on non-vi­ral gene ther­a­py with Gen­er­a­tion Bio af­ter swing­ing gene edit­ing deals

Moderna has inked a five-year partnership with gene therapy biotech Generation Bio, it announced Thursday morning, wading deeper into the genetic medicines space as it navigates beyond its vaccine work.

Moderna will pay Generation Bio $40 million upfront and invest another $36 million into the gene therapy biotech. In exchange, Moderna can license Generation Bio’s non-viral gene therapy platforms for two immune cell programs and two liver programs, with an option for a fifth program. Moderna will fund all the research work under the partnership, and could be on the hook for milestone, fee and royalty payments totaling up to $1.8 billion, a company spokesperson tells Endpoints News.

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Steven James, Pionyr Immunotherapeutics CEO

Gilead pass­es on ful­ly ac­quir­ing Pi­o­nyr, as eyes now turn to Tizona, a fel­low sum­mer 2020 buy­out op­tion

Gilead and Pionyr Immunotherapeutics, a biotech trying to follow up on the first generation of checkpoint inhibitors, have “mutually agreed” on a rewrite to their 2020 terms, with Gilead deciding not to buy out the company.

The California biopharma waived its option to acquire the remaining 50.1% of Pionyr, which would have triggered a $315 million upfront payment and up to $1.15 billion down the road. Had Gilead waited to decide, the drugmaker would have had a potential payment to make in the near term under their agreement, a spokesperson said in an email to Endpoints News.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

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.

Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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