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.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.