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.

Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an an­ti­body play­er fo­cused on PS­MA

Five months after Amgen CEO Bob Bradway stepped up to the M&A table and acquired Five Prime for $1.9 billion, following up with the smaller Rodeo acquisition, he’s gone back in for another biotech buyout.

This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer that has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Andrea Pfeifer, AC Immune CEO (AC Immune)

Look­ing to repli­cate Covid-19 suc­cess in neu­ro, BioN­Tech back­ers bet on AC Im­mune and its new­ly-ac­quired Parkin­son's vac­cine

The German billionaires behind BioNTech have found a new vaccine project to back.

Through their family office Athos Service, twin brothers Thomas and Andreas Strüngmann are leading a $25 million private placement into Switzerland’s AC Immune — which concurrently announced that it’s shelling out $58.7 million worth of stock to acquire Affiris’ portfolio of therapies targeting alpha-synuclein, including a vaccine candidate, for Parkinson’s disease.

Christophe Weber, Takeda CEO (Kyodo via AP Images)

Take­da flesh­es out CNS pact with pep­tide drug­mak­er, set­ting aside $3.5B in fu­ture mile­stones

One of a suite of drugmakers looking to reinvest in the neuroscience space, Takeda has been aggressive in signing on new partners to help build up its pipeline in that space. But sometimes the best partner is the one you already have.

Takeda will set aside $3.5 billion in future milestones and an undisclosed upfront payment to build out its drug discovery deal with Japanese peptide conjugate maker PeptiDream, adding neurodegeneration to the partnership’s list of CNS targets, the companies said Tuesday.

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Michael Henderson, BridgeBio CBO

Bris­tol My­ers Squibb catch­es the SHP2 wave in a new col­lab­o­ra­tion deal with Bridge­Bio

Once considered “undruggable,” the phosphatase enzyme SHP2 has seen recent interest from a suite of Big Pharmas, including AstraZeneca, Amgen, Novartis and Merck. Now Bristol Myers Squibb is getting in on the action, with a deal to pair its PD-1 superstar Opdivo with BridgeBio’s SHP2 inhibitor for difficult-to-treat cancers.

BMS and BridgeBio took the wraps off the non-exclusive, co-funded collaboration early Tuesday morning. The “catalyst,” BridgeBio CBO Michael Henderson said, was last year’s virtual JP Morgan conference, where the companies met to discuss early preclinical results they were seeing between SHP2 and immuno-oncology therapies.

Busi­ness­es and schools can man­date the use of Covid-19 vac­cines un­der EUAs, DOJ says

As public and private companies stare down the reality of the Delta variant, many are now requiring that their employees or students be vaccinated against Covid-19 prior to attending school or to returning or starting a new job. Claims that such mandates are illegal or cannot be used for vaccines under emergency use authorizations have now been dismissed.

Setting the record straight, the Department of Justice on Monday called the mandates legal in a new memo, even when used for people with vaccines that remain subject to EUAs.