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.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

Bris­tol-My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.