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.”

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.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

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You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Yao-Chang Xu, Abbisko Therapeutics founder and CEO

Qim­ing-backed Ab­bisko makes $200M+ Hong Kong de­but, as a SPAC and Agenus spin­out al­so price on Nas­daq

Three new entities priced their public debuts late Thursday and early Friday, including a SPAC, a traditional Nasdaq IPO and a Chinese biotech joining the Hong Kong Index.

Shanghai-based Abbisko Therapeutics raised the most money of the triumvirate, garnering $226 million in its Hong Kong debut and pricing at HK$12.46, or roughly $1.60 in US dollars. The blank check company followed up with a $150 million raise, while MiNK Therapeutics priced on Nasdaq at $12 per share and a $40 million raise.

Paul Grayson, Tentarix CEO (Versant)

Phar­ma vet­er­ans re­group with $50M and a plan to dis­cov­er new mul­ti-specifics

While a horde of drugmakers develops bispecific antibodies to more directly target tumor cells — there were about 100 programs in or nearing clinical trials back in May — a new company is emerging to go one step further.

On Thursday, Tentarix Biotherapeutics unveiled a $50 million Series A round to support its next-gen multi-specifics platform. While the field has largely focused on bispecifics, which engage two targets, Tentarix believes its multifunctional programs have the potential to be even more specific, since more conditions must be met for potent activity to occur.

Tillman Gerngross, Adagio CEO

Q&A: Till­man Gern­gross ex­plains why his Covid mAb will have an edge over an al­ready crowd­ed field

If anyone knows about monoclonal antibodies, it’s serial entrepreneur, Adimab CEO, and Dartmouth professor of bioengineering Tillman Gerngross.

Even the name of Gerngross’ new antibody startup Adagio Therapeutics is meant to reflect his vision behind the development of his Covid-19 mAb: slowly, he said, explaining that “everyone else, whether it’s Regeneron, Lilly, or AstraZeneca, Vir, they all valued speed over everything.”

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