Pre­ci­sion ther­a­py ap­proach se­cures small biotech $42M haul to com­bat dis­ease that in­spired the Ice Buck­et Chal­lenge

Akin to cys­tic fi­bro­sis (CF), sci­en­tists un­der­stand that cer­tain mu­ta­tions con­tribute to the de­vel­op­ment of the fa­tal neu­ro­log­i­cal dis­or­der amy­otroph­ic lat­er­al scle­ro­sis (ALS). And much like CF drug­mak­er Ver­tex, a small Cam­bridge, Mass­a­chu­setts-based biotech is forg­ing a path to en­gi­neer­ing pre­ci­sion ther­a­pies to treat the dis­ease that killed vi­sion­ary physi­cist Stephen Hawk­ing.

The com­pa­ny, chris­tened QurAlis, now has $42 mil­lion in its cof­fers with three pre­clin­i­cal pro­grams and 5 em­ploy­ees (in­clud­ing se­nior man­age­ment) to com­bat an ill­ness that has long flum­moxed re­searchers, re­sult­ing in a cou­ple of ap­proved ther­a­pies over the course of decades, nei­ther of which at­tacks the un­der­ly­ing cause of the rare pro­gres­sive con­di­tion that at­tacks nerve cells lo­cat­ed in the brain and spinal cord re­spon­si­ble for con­trol­ling vol­un­tary mus­cles.

ALS gar­nered in­ter­na­tion­al at­ten­tion when New York Yan­kees play­er Lou Gehrig abrupt­ly re­tired from base­ball in 1939, af­ter be­ing di­ag­nosed with the dis­ease. In 2014, ALS re­turned to the spot­light with the “Ice Buck­et Chal­lenge,” which in­volved peo­ple pour­ing ice-cold wa­ter over their heads, post­ing a video on so­cial me­dia, and do­nat­ing funds for re­search on the con­di­tion.

Kasper Roet

QurAlis chief Kasper Roet, whose in­ter­est in ALS was piqued while he was work­ing on his PhD at the Nether­lands In­sti­tute for Neu­ro­science fo­cus­ing on a treat­ment for spinal cord paral­y­sis and moon­light­ing at the Nether­lands Brain Bank as an ad-hoc au­top­sy team co­or­di­na­tor, saw an op­por­tu­ni­ty to com­bat ALS when Har­vard sci­en­tists Kevin Eggan and Clif­ford Woolf pi­o­neered some new stem cell tech­nol­o­gy.

Es­sen­tial­ly, they found a way to take skin cells from a pa­tient, turn them in­to stem cells, and turn those in­to the nerve cells that are de­gen­er­at­ing. “That’s the miss­ing link,” Roet said. “So now we can fi­nal­ly use pa­tients’ own cells to both do tar­get dis­cov­ery and de­vel­op po­ten­tial ther­a­peu­tics.”

So Roet packed up his things and shift­ed base to Boston to learn more, with plans to head back to Eu­rope to start a com­pa­ny. He nev­er left. QurAlis was born in 2016, work­ing out of a co-work­ing space called Lab­Cen­tral af­ter win­ning a spot via an Am­gen-spon­sored in­no­va­tion com­pe­ti­tion. The com­pa­ny was carved out of a col­lab­o­ra­tion with Eggan’s start­up Q-State Bio­sciences, which de­vel­oped laser tech­nol­o­gy to ex­am­ine cell be­hav­ior — ex­am­in­ing how a neu­ron fires was im­per­a­tive in the drug dis­cov­ery process for ALS.

QurAlis, which counts Ver­tex’s found­ing sci­en­tist Manuel Navia as an ad­vi­sor, now has three pre­clin­i­cal pro­grams. The fur­thest along is a ther­a­py de­signed to tar­get a spe­cif­ic potas­si­um chan­nel that is im­pli­cat­ed in cer­tain ALS pa­tients — the plan is to take that small mol­e­cule in­to the clin­ic next year, Roet said.

“It has be­come re­al­ly clear that if you un­der­stand why a spe­cif­ic tu­mor is de­vel­op­ing … you can de­vel­op very spe­cif­ic tar­get­ed ther­a­pies,” he ex­plained in an in­ter­view draw­ing a par­al­lel be­tween ALS and on­col­o­gy. “That’s ex­act­ly the same strat­e­gy that we are fol­low­ing for ALS. The ge­net­ics have shown that over 25 genes are caus­ing the (ALS) mu­ta­tions. Some of them work to­geth­er, some of them are very dom­i­nant and work alone — what we are do­ing is try­ing to get those spe­cif­ic pro­teins that are tied to very spe­cif­ic ALS pop­u­la­tions, where we know that that spe­cif­ic tar­get plays a very im­por­tant and cru­cial role in the de­vel­op­ment of the dis­ease.”

In 2018, QurAlis scored seed fund­ing from Am­gen, Alexan­dria, and MP Health­care Ven­ture Man­age­ment. The Se­ries A in­jec­tion was led by LS Po­laris In­no­va­tion Fund, lead seed in­vestor Mis­sion Bio­Cap­i­tal, INKEF Cap­i­tal and the De­men­tia Dis­cov­ery Fund, and co-led by Droia Ven­tures. Ad­di­tion­al new in­vestors in­clude Mit­sui Glob­al In­vest­ment and Dol­by Fam­i­ly Ven­tures, and ex­ist­ing in­vestors Am­gen Ven­tures, MP Health­care Ven­ture Man­age­ment, and San­ford Bio­sciences al­so chipped in.

Roet is not sure how long these funds will last, par­tic­u­lar­ly giv­en the un­cer­tain­ty of the coro­n­avirus pan­dem­ic. But some of the cap­i­tal will be used in hir­ing, giv­en that the QurAlis team is com­prised of a mere five peo­ple, in­clud­ing Roet.

“We’ve been very pro­duc­tive,” he said. “But we can def­i­nite­ly use some ex­tra hands.”

Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

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In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an an­ti­body play­er fo­cused on PS­MA

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This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer that has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

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According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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Andrea Pfeifer, AC Immune CEO (AC Immune)

Look­ing to repli­cate Covid-19 suc­cess in neu­ro, BioN­Tech back­ers bet on AC Im­mune and its new­ly-ac­quired Parkin­son's vac­cine

The German billionaires behind BioNTech have found a new vaccine project to back.

Through their family office Athos Service, twin brothers Thomas and Andreas Strüngmann are leading a $25 million private placement into Switzerland’s AC Immune — which concurrently announced that it’s shelling out $58.7 million worth of stock to acquire Affiris’ portfolio of therapies targeting alpha-synuclein, including a vaccine candidate, for Parkinson’s disease.

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

In a week that’s expected to see several biotechs price their IPOs, Candel Therapeutics got things kicked off Tuesday with a muted opener.

The company helmed by former GlaxoSmithKline vet Paul Peter Tak made its way to Nasdaq thanks to a $72 million raise, which was downsized by about 15% than originally anticipated, according to Renaissance Capital. Candel priced at $8 per share after initially seeking to launch in the $13 to $15 range.

Busi­ness­es and schools can man­date the use of Covid-19 vac­cines un­der EUAs, DOJ says

As public and private companies stare down the reality of the Delta variant, many are now requiring that their employees or students be vaccinated against Covid-19 prior to attending school or to returning or starting a new job. Claims that such mandates are illegal or cannot be used for vaccines under emergency use authorizations have now been dismissed.

Setting the record straight, the Department of Justice on Monday called the mandates legal in a new memo, even when used for people with vaccines that remain subject to EUAs.

Gerry Brunk (Lumira)

What will Lu­mi­ra Ven­tures do with $220M? Stay out of the com­fort zone and off the beat­en biotech path

Lumira Ventures closed its largest fund on Monday, raking in $220 million to pump into the life sciences — but instead of targeting biotech hubs like San Francisco and Boston, the company is rolling the dice on “underserved geographies” in the US and Canada.

“We find oftentimes companies located in places like Montreal, or Fort Lauderdale, FL, or Kansas City or Phoenix, AZ just have more capital efficiency and better valuations, without having to compromise anything at all in the quality of the innovation and the management talent,” co-founder and managing partner Gerry Brunk told Endpoints News.