Neu­ro up­start launch­es out of RA Cap­i­tal’s low-pro­file in­cu­ba­tor with Big Phar­ma vets, clin­i­cal drugs and $80M

As a pain re­searcher of 20-plus years, Va­lerie Moris­set doesn’t get ex­cit­ed about new drug ideas eas­i­ly.

So when An­drew Levin from RA Cap­i­tal called to talk about a pro­drug of the en­do­cannabi­noid palmi­toylethanolamide, she was skep­ti­cal. Moris­set had just swapped out a ca­reer in biotech — most re­cent­ly head­ing up bi­ol­o­gy and trans­la­tion­al med­i­cine for Con­ver­gence Phar­ma­ceu­ti­cals through its ac­qui­si­tion by Bio­gen — for a VC perch at Bridge Val­ley Ven­tures , and to­geth­er with Si­mon Tate, the man­ag­ing di­rec­tor of In­ter­me­di­ate Cap­i­tal Group, they dived in for three months of due dili­gence.

Va­lerie Moriset

“At first, it is true, our in­tent was to ba­si­cal­ly kill it and move along to the next op­por­tu­ni­ty,” she told End­points News.

Rather, she would be­come the pres­i­dent and CSO of Eliem, the start­up tasked with steer­ing this — ETX-810 — and three oth­er neu­ro drugs to the fin­ish line with $80 mil­lion in fresh fund­ing.

Bob Azel­by, the for­mer chief com­mer­cial of­fi­cer at Juno and and re­cent chief of Alder who helped ne­go­ti­ate a $1.95 bil­lion buy­out by Lund­beck, is on board as CEO, lead­ing an of­fice in Seat­tle while Moris­set man­ages an R&D team in Cam­bridge, UK. A clin­i­cal de­vel­op­ment crew is al­so based in the US, where all tri­als are cur­rent­ly planned.

En­do­cannabi­noids — en­doge­nous neu­ro­trans­mit­ters that bind to cannabi­noid re­cep­tors — had en­thused Big Phar­ma for some years, send­ing them off on a search for CB2 ag­o­nists, pe­riph­er­al CB1 or oth­ers, but the ef­forts have large­ly turned up emp­ty, if not out­right dan­ger­ous. Oth­ers have tried go­ing af­ter a mas­ter mod­u­la­tor in­stead.

PEA, as Moris­set al­ready knew, is part of this group. What she was sur­prised to find, though, was the “ab­solute wealth” of clin­i­cal lit­er­a­ture on us­ing the nat­u­ral­ly oc­cur­ring mol­e­cule in a num­ber of chron­ic pain con­di­tions. Af­ter ask­ing a pro­fes­sor at the Uni­ver­si­ty of Rome to re­an­a­lyze da­ta from one of the biggest stud­ies avail­able and con­firm a dose-re­sponse re­la­tion­ship be­tween the ex­po­sure of PEA and the re­duc­tion of pain, they were con­vinced.

An­drew Levin

Tak­ing a pro­drug ap­proach, she not­ed, con­fers a three-fold high­er ex­po­sure, a longer half life, and bet­ter drug prop­er­ties over­all. They will first test it in two Phase IIa tri­als for lum­bosacral radic­u­lar pain (chron­ic sci­at­i­ca) and di­a­bet­ic pe­riph­er­al neu­ro­path­ic pain.

But Eliem wants to be more than a pain com­pa­ny; the fo­cus they’re giv­ing them­selves is neu­ronal ex­citabil­i­ty dis­or­ders, which could be any­thing from de­pres­sion to epilep­sy.

The com­mon theme of these dis­eases is that they arise from an im­bal­ance be­tween ex­ci­ta­tion and in­hi­bi­tion, not­ed Moris­set, an elec­tro­phys­i­ol­o­gist by back­ground.

“There is a lot of lit­er­a­ture de­scrib­ing pain, epilep­sy as hy­per­ex­citabil­i­ty dis­or­ders where ba­si­cal­ly neu­rons are fir­ing too fast,” she added.

ETX-155, a next-gen­er­a­tion GABAA pos­i­tive al­losteric mod­u­la­tor al­so from RA Cap­i­tal, is now in Phase I tri­als with plans to go in­to mid-stage stud­ies for ma­jor de­pres­sive dis­or­der, hor­mone-re­lat­ed mood dis­or­ders and fo­cal on­set seizures. Then there’s a pre­clin­i­cal Kv7.⅔ chan­nel open­er dis­cov­ered in house and an­oth­er ear­ly-stage re­search pro­gram.

Alder alum Erin Lavelle is tak­ing up the COO post, while phar­ma vets Amy Chap­pell and Jo Palmer-Phillips are fill­ing the chief med­ical of­fi­cer and chief de­vel­op­ment of­fi­cer po­si­tions, re­spec­tive­ly. Levin, who is cred­it­ed as co-founder along­side Moris­set, is chair­ing the board of di­rec­tors, which al­so in­volves Tate of ICG and Liam Rat­cliffe from Ac­cess Biotech­nol­o­gy, the two in­vestors who joined RA Cap­i­tal for the Se­ries A.

Bob Azel­by

“When you have a re­al­ly ex­pe­ri­enced team with clin­i­cal­ly val­i­dat­ed mech­a­nism of ac­tion, it’s a lot about ex­e­cu­tion,” Azel­by said, “mak­ing sure that you’re get­ting the right tri­al set up and ex­e­cute the right tri­al.”

It’s not just about the tech­ni­cal skills, ei­ther. As Eliem grows from the cur­rent head­count of 18 to 25 or 30 by the end of the year, Azel­by said a key at­tribute he will be look­ing for in re­cruit­ment is re­silience.

“You got­ta get peo­ple that are com­ing here and un­der­stand that, you know what? The boat may rock a lit­tle bit right be­cause bi­ol­o­gy,” he said. “And you want those folks that get ex­cit­ed when an is­sue aris­es so they can dig in and solve it.”

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.

From left: James Brown, Michael Chambers, John Ballantyne

Alde­vron founders back a biotech start­up that's look­ing to end the moral de­bate over cell lines once and for all

For millions of Catholics around the world, the development of new vaccines to combat Covid-19 has sparked a moral dilemma. All the approved vaccines in use relied — in some fashion — on cell lines that were derived from aborted fetal tissue.

While church leaders accepted the vaccines and recommended their use to end the pandemic, a number also highlighted their preference for the mRNA vaccines from Pfizer/BioNTech and Moderna over the J&J and AstraZeneca shots, which they noted were more heavily dependent on cell lines that they found morally objectionable.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”