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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Yao-Chang Xu, Abbisko Therapeutics founder and CEO

Qim­ing-backed Ab­bisko makes $200M+ Hong Kong de­but, as a SPAC and Agenus spin­out al­so price on Nas­daq

Three new entities priced their public debuts late Thursday and early Friday, including a SPAC, a traditional Nasdaq IPO and a Chinese biotech joining the Hong Kong Index.

Shanghai-based Abbisko Therapeutics raised the most money of the triumvirate, garnering $226 million in its Hong Kong debut and pricing at HK$12.46, or roughly $1.60 in US dollars. The blank check company followed up with a $150 million raise, while MiNK Therapeutics priced on Nasdaq at $12 per share and a $40 million raise.

Paul Grayson, Tentarix CEO (Versant)

Phar­ma vet­er­ans re­group with $50M and a plan to dis­cov­er new mul­ti-specifics

While a horde of drugmakers develops bispecific antibodies to more directly target tumor cells — there were about 100 programs in or nearing clinical trials back in May — a new company is emerging to go one step further.

On Thursday, Tentarix Biotherapeutics unveiled a $50 million Series A round to support its next-gen multi-specifics platform. While the field has largely focused on bispecifics, which engage two targets, Tentarix believes its multifunctional programs have the potential to be even more specific, since more conditions must be met for potent activity to occur.

FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.