What break? Sarep­ta vet Ed Kaye quick­ly piv­ots to the helm of an­oth­er biotech up­start with a $40M bankroll

Ed Kaye

Af­ter Ed Kaye left the helm at Sarep­ta last Au­gust, he promised his wife he’d take some time off be­fore start­ing his next job in biotech.

Then the calls start­ed com­ing in.

“The phone rang off the hook,” says Kaye, a pop­u­lar fig­ure in the bustling Cam­bridge/Boston hub who has a re­sume that cov­ers every as­pect of biotech ex­is­tence. It’s the kind of back­ground that ven­ture groups love.

“With­in weeks I had 6 of­fers,” he says with­out even a slight trace of boast­ing. One fund he talked with “said that if we had 20 peo­ple like you, we’d start 20 com­pa­nies.”

Two months in­to his “break,” Kaye stepped in­to the CEO’s job at Stoke Ther­a­peu­tics, which is com­ing out of seed mode and in­to for­mal launch phase to­day with a $40 mil­lion round com­ing from a deeply ded­i­cat­ed group of back­ers at Ap­ple Tree.

What made Stoke ir­re­sistible?

For one thing, Kaye al­ready had a close re­la­tion­ship with the key sci­en­tist in­volved, Adri­an Krain­er at Cold Spring Har­bor, that went back years.

Adri­an Krain­er

Krain­er, an RNA splic­ing ex­pert who played a key role in the dis­cov­ery of Spin­raza, had been study­ing the body’s ma­chin­ery for mak­ing pro­teins and a method us­ing an­ti­sense oligonu­cleotides to in­crease pro­tein ex­pres­sion. Adding a bioin­for­mat­ics ap­proach, the fledg­ling com­pa­ny was able to iden­ti­fy key dis­eases dri­ven by miss­ing pro­tein — and now they’re out to use the tech to ad­dress dis­eases that seem out­side the realm of such tech­nolo­gies as gene edit­ing or gene ther­a­py.

Like any good plat­form, Kaye feels that Stoke can go off in mul­ti­ple di­rec­tions. But, he’s al­so run­ning a start­up that has to iden­ti­fy the low hang­ing fruit first and then grow things up along the way. Au­to­so­mal dom­i­nant dis­eases like Dravet syn­drome of­fer a tar­get-rich en­vi­ron­ment. And Kaye likes their chances start­ing with the brain and eye, look­ing at trig­gers for blind­ness that may present an ide­al path­way to proof-of-con­cept da­ta.

As any­one who ever worked at Sarep­ta could tell you, there are a lot of el­e­ments in the biotech busi­ness be­yond your im­me­di­ate con­trol. Kaye has a new board and a backer to work with, a shot at an ex­pand­ed syn­di­cate down the road and a need to per­form. But he does have some big am­bi­tions here for his staff of 14, which will now dou­ble in size over the near term.

If all goes ac­cord­ing to plan now, Kaye ex­pects to be in the clin­ic in 2019.

“My goal is to take this all the way to com­ple­tion and build a com­pa­ny that can be sus­tain­able,” says the CEO. The next leg of that jour­ney starts now.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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David Meline, incoming Moderna CFO

Am­gen vet David Meline finds a new CFO roost at Mod­er­na, tak­ing a ride on the Covid-19 tiger as de­part­ing ex­ec cash­es out with $12M

We found out a few weeks ago that Moderna CFO Lorence Kim isn’t waiting around to see how the biotech wunderkind makes out in its frantic race to field a messenger RNA vaccine that can quell Covid-19. And now we know who’s stepping on board to take his place in the latest move in the executive suite.

David Meline, who forged his rep during a 6-year run at Amgen, slipped out the exit right after his Q2 “retirement” party in California — presumably virtual — and started the next chapter of his career at a biotech company betting big on revolutionizing the vaccine R&D space.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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