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.”

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

Endpoints Premium

Premium subscription required

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

Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.