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

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Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

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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.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Daisy Robinton, Oviva Therapeutics co-founder and CEO

A new spin­out wades in­to ovar­i­an ag­ing, armed with a seed round and ex­per­i­men­tal drugs from Mass Gen­er­al

There aren’t many biotechs emphasizing women’s health, but a new spinout is trying to change that.

Oviva Therapeutics, a pipeline company of New York-based Cambrian Biopharma, emerged from stealth earlier this week to dive into the idea of extending women’s “healthspans,” or what it says is the part of a person’s life spent in generally good health, with a specific focus on ovaries. The emergence comes both with a seed financing worth $11.5 million from Cambrian, and an in-licensing agreement with Massachusetts General Hospital for a trio of patents.

Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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Paul Hudson, Sanofi CEO (via Getty)

Sanofi's $20B buy­out of Gen­zyme pays off again with Eu­ro­pean OK for first Nie­mann-Pick drug

Sanofi CEO Paul Hudson has made clear his intention to develop new rare disease drugs and broaden his company’s offerings. That effort leaped forward on Friday with the EMA’s signing off on the company’s — and the EU’s — first drug to treat the non-central nervous system manifestations of the rare and debilitating Niemann-Pick disease.

The enzyme replacement therapy, developed to replace patients’ deficient or defective enzyme, known as acid sphingomyelinase, was first developed by Genzyme, which Sanofi acquired for more than $20 billion in 2011. That acquisition has also helped Sanofi pull in sales in the field of MS.