Ed Kaye. Stoke Therapeutics

Pre­clin­i­cal an­ti­sense biotech, led by for­mer Sarep­ta CEO Kaye, vaults on to Nas­daq with up­sized IPO

Less than a year af­ter rais­ing $90 mil­lion in its sec­ond round of fi­nanc­ing, for­mer Sarep­ta chief Ed­ward Kaye is mak­ing the leap to the Nas­daq on Wednes­day with an up­sized of­fer­ing for his pre­clin­i­cal an­ti­sense biotech, Stoke Ther­a­peu­tics.

Adri­an Krain­er Cold Spring Har­bor Labra­to­ries

The com­pa­ny in-li­censed its tech­nol­o­gy: Tar­get­ed Aug­men­ta­tion of Nu­clear Gene Out­put (TAN­GO) from Cold Spring Har­bor Lab­o­ra­to­ry’s Adri­an Krain­er, who is cred­it­ed as the in­ven­tor of Bio­gen’s $BI­IB spinal mus­cu­lar at­ro­phy drug Spin­raza. Krain­er co-found­ed Stoke and serves on its board.

As it preps an ap­pli­ca­tion to test its lead ex­per­i­men­tal ther­a­py next year, Stoke raised gross pro­ceeds of $142 mil­lion by of­fer­ing 7.9 mil­lion shares at $18, above the range of $14 to $16. It had pre­vi­ous­ly filed to of­fer 6.7 mil­lion shares, and plans to list on the Nas­daq un­der the sym­bol “$STOK.”

The holy grail of gene ther­a­pies is to de­vel­op a treat­ment that is cu­ra­tive with a sin­gle dose, but so far, re­search has shown that the lev­el of pro­tein pro­duced as a re­sult of this type of ther­a­peu­tic in­ter­ven­tion can fluc­tu­ate from pa­tient to pa­tient, its dura­bil­i­ty re­mains in ques­tion and is ex­pen­sive to man­u­fac­ture be­cause sci­en­tists must en­gi­neer virus­es to de­liv­er the gene ther­a­py to the pa­tient.

Stoke is de­vel­op­ing an­ti­sense oligonu­cleotide med­i­cines, by tak­ing aim at dis­eases ar­eas that are of­ten not amenable to tra­di­tion­al gene ther­a­py — be­cause the gene size is too big — or to gene edit­ing, as the ther­a­peu­tic needs to be titrat­able. It is bet­ting on sta­bi­liz­ing the pro­tein man­u­fac­tured by treat­ed cells with its TAN­GO tech­nol­o­gy, which is de­signed to iden­ti­fy gene seg­ments that can be tar­get­ed by drugs to di­al up the lev­el of pro­tein pro­duced. The Bed­ford, Mass­a­chu­setts-based com­pa­ny then de­vel­ops RNA drugs to har­ness these tar­gets.

Like GW Phar­ma­ceu­ti­cals’ $GW­PH keen­ly watched and 2018 ap­proved cannabis-de­rived ther­a­peu­tic Epid­i­olex, Stoke is tar­get­ing Dravet syn­drome, a rare and cat­a­stroph­ic form of in­tractable epilep­sy that be­gins in in­fan­cy.

Con­trol­ling the amount of pro­tein pro­duced fol­low­ing ther­a­py is par­tic­u­lar­ly sig­nif­i­cant in au­to­so­mal dom­i­nant dis­eases — such as ge­net­ic caus­es of epilep­sy — in which mu­ta­tions in just one copy of a gene ad­verse­ly im­pact pro­tein pro­duc­tion. An over-pro­duc­tion of pro­tein in such cas­es could be just as sin­is­ter. Stoke’s drug is de­signed to up-reg­u­late pro­duc­tion of a pro­tein miss­ing in pa­tients with Dravet syn­drome by tar­get­ing RNA splic­ing. The an­ti­sense oligonu­cleotide is sus­pend­ed in saline and de­liv­ered via spinal in­jec­tion, and will like­ly be ad­min­is­tered every four months or so.

If the FDA sanc­tions an in-hu­man study, the biotech ex­pects to ini­ti­ate a Phase I/II clin­i­cal tri­al in chil­dren and ado­les­cents in the first half of 2020 and an­tic­i­pates to have clin­i­cal da­ta, in­clud­ing pre­lim­i­nary ef­fi­ca­cy da­ta, in 2021.

As of March 31, Stoke has raised over $130 mil­lion in fund­ing, in­clud­ing in­vest­ments from Ap­ple Tree Part­ners, RTW In­vest­ments, RA Cap­i­tal Man­age­ment, Cor­morant As­set Man­age­ment, Per­cep­tive Ad­vi­sors and funds man­aged by Janus Hen­der­son In­vestors, Red­mile Group, Sphera Funds Man­age­ment and Alexan­dria Ven­ture In­vest­ments.

Illustration: Assistant Editor Kathy Wong for Endpoints News

How Pur­due's $272M ad­dic­tion pay­out fund­ed a new home for its dis­card­ed non-opi­oid re­search

Don Kyle spent more than 20 years working for Purdue Pharma, right through the US opioid epidemic that led to the company’s rise and eventual infamy. But contrary to Purdue’s focus on OxyContin, Kyle was researching non-opioid painkillers — that is, until the company shelved his research.

As the company’s legal troubles mounted, Kyle found an unlikely way to reboot the project. In 2019, he took his work to an Oklahoma State University center that’s slated to receive more than two-thirds of the state’s $272 million settlement with Purdue over claims that the drugmaker’s behavior ignited the epidemic of opioid use and abuse.

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President Joe Biden at the State of the Union address with Vice President Kamala Harris and House Speaker Kevin McCarthy (Patrick Semansky/AP Images)

The drug pric­ing pres­i­dent: Biden warns of ve­to for any IRA re­peal at­tempts

President Joe Biden made clear in his “finish the job” State of the Union address last night that one of those jobs to be finished is insulin prices.

Biden’s push again to tackle insulin prices, after Republicans rebuffed the idea last summer and just after Biden won Medicare drug price negotiations/caps via the Inflation Reduction Act, shows how heavily he’s leaning into this work.

Rupert Vessey, Bristol Myers Squibb head of research and early development

Up­dat­ed: R&D tur­bu­lence at Bris­tol My­ers now in­cludes the end of a $650M al­liance and the de­par­ture of a top re­search cham­pi­on

This morning biotech Dragonfly put out word that Bristol Myers Squibb has handed back all rights to its IL-12 clinical-stage drug after spending $650 million to advance it into the clinic.

The news arrives amid a turbulent R&D stage for the pharma giant, which late last week highlighted Rupert Vessey’s decision to depart this summer as head of early-stage R&D following a crucial three-year stretch after he jumped to Bristol Myers in the big Celgene buyout. During that time he struck a series of deals for Bristol Myers, and also shepherded a number of Celgene programs down the pipeline, playing a major role for a lineup of biotechs which depended on him to champion their drugs.

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Utpal Koppikar, new Verily CFO

Ex­clu­sive: Ver­i­ly wel­comes Atara Bio­ther­a­peu­tics vet­er­an as new CFO

Verily, Alphabet’s life sciences outfit, has plucked a new CFO from the ranks of Atara Biotherapeutics, the company announced on Wednesday.

Utpal Koppikar joins Verily after a nearly five-year stint as CFO and senior VP at Atara, though his résumé also boasts roles at Gilead and Amgen.

The news follows a major reshuffling at Verily, including several senior departures earlier this year and a round of layoffs.

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Singer Nick Jonas is back at work for Dexcom, this time for its new G7 glucose monitor.

Dex­com's spokescelebri­ty Nick Jonas re­turns to Su­per Bowl in new glu­cose mon­i­tor com­mer­cial

Dexcom is going back to the Super Bowl with its pop singer and patient spokesperson Nick Jonas. Jonas takes center stage as the lone figure in the 30-second commercial showcasing Dexcom’s next-generation G7 continuous glucose monitoring (CGM) device.

Jonas’ sleight-of-hand tricks populate the commercial — he pinches his empty fingers together and pops them open to reveal the small CGM — even as he ends the ad, saying, “It’s not magic. It just feels that way.” Jonas then disappears in a puff of smoke.

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Richard Francis, newly-appointed Teva CEO (Novartis via Facebook)

New Te­va CEO Richard Fran­cis repri­or­i­tizes to 'get back to growth'

Six weeks into his new role at the helm of Teva Pharmaceutical, Richard Francis said it’s time to “get back to growth,” starting with a good look at the company’s priorities.

The chief executive has kicked off a strategic review, he announced during Teva’s quarterly call, which will continue over the next several months and produce results sometime in the middle of 2023. That means some pipeline cuts may be in store, he told Endpoints News, while declining to offer much more detail.

FDA Commissioner Robert Califf on Capitol Hill, Feb. 8, 2023 (Drew Angerer/Getty Images)

FDA com­mis­sion­er floats ideas on how to bet­ter han­dle the pan­dem­ic

FDA Commissioner Rob Califf joined the heads of the CDC and NIH in the hot seat today before a key House subcommittee, explaining that there needs to be a much faster, more coordinated way to oversee vaccine safety, and that foreign biopharma inspections, halted for years due to the pandemic, are slowly ramping up again.

Califf, who stressed to the House Energy and Commerce’s Subcommittee on Health that the CDC also needs better data, made clear that the FDA’s ability to monitor the safety of vaccines “would also benefit greatly by a coordinated federal public health data reporting authority.”

Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

Drag­on­fly chief: Bris­tol My­ers shouldn’t blame IL-12’s clin­i­cal per­for­mance for de­ci­sion to scrap the deal — eco­nom­ics played a key role

Bristol Myers Squibb says the IL-12 drug they were developing out of Dragonfly Therapeutics was scrubbed from the pipeline for a simple reason: It didn’t measure up on clinical performance.

But Bill Haney, the CEO of Dragonfly, is taking issue with that.

The early-stage drug, still in Phase I development, has passed muster with Bristol Myers’ general clinical expectations, advancing successfully while still in Phase I, he says.

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Sanofi is renewing its #VaccinesForDreams campaign with more stories, such as Juan's in Argentina (Sanofi)

Sanofi re­news so­cial cam­paign to re­mind that vac­cines let peo­ple ‘Dream Big’

Sanofi is highlighting people’s dreams — both big and small — to make the point that vaccines make them possible.

The renewed “Dream Big” global social media campaign’s newest dreamer is Juan, a teacher in the Misiones rainforest in Argentina whose story is told through videos on Instagram and Sanofi’s website with the hashtag #VaccinesForDreams.

The campaign ties to Sanofi’s broader umbrella initiative “Vaccine Stories” to promote the value of vaccines and drive awareness of the need for improved vaccination coverage.

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