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.”

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.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Suresh Katta, Saama CEO (via YouTube)

As AI con­tin­ues to en­tice Big Phar­ma, a Car­lyle-led drug­mak­er syn­di­cate shells out $430M for cloud com­put­ing play­er

The AI revolution permeating Big Pharma took a big financial step forward Wednesday, with VCs and major drugmakers coming together to acquire a cloud-focused company.

Led by the Carlyle Group, the investors will put up $430 million for a majority stake in Saama, a company that collects patient data to help speed along the drug development process. The investment arms of Pfizer, Merck, Amgen and McKesson all participated in the financing, in addition to other prominent life sciences VCs like Northpond.

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Raju Mohan, Ventyx Biosciences CEO

Ven­tyx sprints to Wall Street less than a year af­ter emerg­ing from stealth

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

It took seven months from exiting “quiet mode” for Ventyx Biosciences to land its very own stock ticker, raising $165 million in venture funds along the way.

Now, after pricing a massive $151.5 million IPO, the Encinitas, CA-based biotech is gunning for Phase II.

Ventyx priced close to 9.5 million shares at $16 apiece on Wednesday, the midpoint of its $15 to $17 range. CEO Raju Mohan filed the S-1 papers at the end of September, just over a week after unveiling a $114 million Series B round. He penciled in the standard figure of $100 million at first, likely knowing that in the last year, it’s been common for biotechs to raise much more than those initial estimates.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.