Har­vard big-shots Kevin Eggan and Clif­ford Woolf launch ALS start­up with ex­clu­sive Q-State tech

A group of well-known Har­vard pro­fes­sors has launched a new ALS spin­out to take on re­search Big Phar­ma wasn’t tack­ling fast enough.

Clif­ford Woolf

The new com­pa­ny, called QurAlis (pro­nounced “cure-al-is”), was found­ed by a cou­ple of stem cell pi­o­neers at Har­vard: Kevin Eggan (the guy who de­vel­oped the first ALS pa­tient-de­rived stem cell mod­els) and Clif­ford Woolf. The duo teamed up with two oth­er co-founders, in­clud­ing QurAlis’ CEO Kasper Roet and Jonathan Flem­ing, who heads up Eggan’s oth­er start­up Q-State Bio­sciences.

Roet tells me he worked with Eggan and Woolf in years past, but it was al­ways in the con­text of large phar­ma­ceu­ti­cal com­pa­nies.

“These phar­mas tend to re­or­ga­nize and the pro­gram we were run­ning would nev­er reach the end goal,” Roet said. “So I asked Kevin and Clif­ford if we could do some­thing our­selves.”

QurAlis, found­ed in 2016, has been qui­et­ly work­ing out of a cowork­ing space in Cam­bridge, MA, called Lab­Cen­tral af­ter win­ning free space there dur­ing an Am­gen-spon­sored in­no­va­tion com­pe­ti­tion. QurAlis is step­ping out with an undis­closed round of seed mon­ey from Am­gen, Alexan­dria, and MP Health­care Ven­ture Man­age­ment.

Kasper Roet

As the com­pa­ny’s name rather di­rect­ly sug­gests, QurAlis hopes to find cures for Amy­otroph­ic Lat­er­al Scle­ro­sis, a mo­tor neu­ron dis­ease that con­tin­ues to frus­trate re­searchers for its com­plex­i­ty. As Roet de­scribes it, ALS is rather like can­cer in that its not one dis­ease but a spec­trum of dis­or­ders with vary­ing un­der­ly­ing mech­a­nisms.

“Not all ALS pa­tients are the same, and if we want to be suc­cess­ful then we must make ther­a­pies tar­get­ed to what’s go­ing wrong in that spe­cif­ic pa­tient pop­u­la­tion,” Roet said.

QurAlis is de­vel­op­ing three drugs that tar­get sub­types of ALS, in­clud­ing a drug the com­pa­ny hopes would one-up GSK’s epilep­sy drug Trobalt (reti­ga­bine), which was pulled from the mar­ket last June due to safe­ty is­sues. QurAlis’ drug would treat over­ac­tive neu­rons and pre­vent cell death via ex­ci­to­tox­i­c­i­ty. QurAlis’ oth­er two pro­grams in­clude a drug can­di­date that would re­store a dys­func­tion­al cel­lu­lar waste clear­ance sys­tem that pro­gres­sive­ly poi­sons neu­rons, and a de­vice to re­move tox­ic pro­teins.

The com­pa­ny’s edge comes from a tech­nol­o­gy it’s li­censed from Q-State Bio­sciences. In fact, QurAlis is a bit of a spin­out from Q-State, with an ex­clu­sive li­cense to use the com­pa­ny’s plat­form tech­nol­o­gy to de­vel­op ther­a­pies for ALS. Q-State’s claim to fame is a plat­form they call Op­topatch, which us­es laser tech­nol­o­gy to ex­am­ine cell be­hav­ior. Specif­i­cal­ly, they can look at a neu­ron and ex­am­ine how it fires. And that’s use­ful for ALS re­search.

Jonathan Flem­ing

“We took stem cells from ALS pa­tients and found out they fire dif­fer­ent­ly from nor­mal healthy peo­ple,” Roet said. “Op­topatch is sen­si­tive enough to pick up these fir­ing cells, which is good for as­sess­ing if po­ten­tial ther­a­peu­tics can re­store this fir­ing and bring it back to nor­mal lev­els.”

In­ter­est­ing­ly, QurAlis has at­tract­ed a num­ber of no­table ad­vi­sors, in­clud­ing the renowned struc­tur­al bi­ol­o­gist and drug de­sign ex­pert Manuel Navia, who was a found­ing sci­en­tist at Ver­tex Phar­ma­ceu­ti­cals (and who you may re­mem­ber as a key char­ac­ter from the book chron­i­cling Ver­tex’s rise: “Bil­lion Dol­lar Mol­e­cule”).

Navia is a drug hunter and the guy who solved the struc­ture of the first drug for AIDS pa­tients. Roet said he was ea­ger to con­tribute the same ef­fort for ALS pa­tients.

“Watch­ing Manuel work with our CSO Daniel El­baum is like watch­ing mag­ic,” Roet said.

The com­pa­ny, which has on­ly four full-timers on board, has enough funds in hand to car­ry the start­up through to a Se­ries A round.

Im­age: Kevin Eggan pre­sent­ing at the 2016 FU­SION sym­po­sium at Har­vard Uni­ver­si­ty.YOUTUBE

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Tony Johnson, Goldfinch Bio CEO (Goldfinch via YouTube)

Kid­ney dis­ease drug­mak­er Goldfinch Bio shuts down

Goldfinch Bio, attempting to make treatments for kidney diseases and diabetic nephropathy, is shutting down.

President and CEO Tony Johnson confirmed to Endpoints News Friday afternoon that the biotech shut down after “fundraising challenges in the current macro-environment.” Fierce Biotech first reported the news.

Johnson, who joined in 2017 after a stint as SVP of early clinical development at AstraZeneca, said in a text that the company “entered the ABC process recently,” referring to an assignment for the benefit of the creditors, which provides a different wind-down avenue than a bankruptcy.

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Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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Elon Musk (GDA via AP Images)

Neu­ralink em­ploy­ees cite lay­offs at Elon Musk’s brain-com­put­er in­ter­face start­up

At least two Neuralink employees have posted to LinkedIn in recent days saying they’ve been laid off from Elon Musk’s brain-computer interface startup, which has received backlash for animal testing.

A former staffer working on preclinical study design and an ex-lab director working on assessing the safety of Neuralink’s implanted devices (prior to human testing) announced recently they’d been laid off, specifically using that terminology. Both had worked at the startup for at least two years.

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