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

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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