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.”

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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