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.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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