Har­vard big-shots Kevin Eggan and Clif­ford Woolf launch ALS start­up with ex­clu­sive Q-State tech

A group of well-known Har­vard pro­fes­sors has launched a new ALS spin­out to take on re­search Big Phar­ma wasn’t tack­ling fast enough.

Clif­ford Woolf

The new com­pa­ny, called QurAlis (pro­nounced “cure-al-is”), was found­ed by a cou­ple of stem cell pi­o­neers at Har­vard: Kevin Eggan (the guy who de­vel­oped the first ALS pa­tient-de­rived stem cell mod­els) and Clif­ford Woolf. The duo teamed up with two oth­er co-founders, in­clud­ing QurAlis’ CEO Kasper Roet and Jonathan Flem­ing, who heads up Eggan’s oth­er start­up Q-State Bio­sciences.

Roet tells me he worked with Eggan and Woolf in years past, but it was al­ways in the con­text of large phar­ma­ceu­ti­cal com­pa­nies.

“These phar­mas tend to re­or­ga­nize and the pro­gram we were run­ning would nev­er reach the end goal,” Roet said. “So I asked Kevin and Clif­ford if we could do some­thing our­selves.”

QurAlis, found­ed in 2016, has been qui­et­ly work­ing out of a cowork­ing space in Cam­bridge, MA, called Lab­Cen­tral af­ter win­ning free space there dur­ing an Am­gen-spon­sored in­no­va­tion com­pe­ti­tion. QurAlis is step­ping out with an undis­closed round of seed mon­ey from Am­gen, Alexan­dria, and MP Health­care Ven­ture Man­age­ment.

Kasper Roet

As the com­pa­ny’s name rather di­rect­ly sug­gests, QurAlis hopes to find cures for Amy­otroph­ic Lat­er­al Scle­ro­sis, a mo­tor neu­ron dis­ease that con­tin­ues to frus­trate re­searchers for its com­plex­i­ty. As Roet de­scribes it, ALS is rather like can­cer in that its not one dis­ease but a spec­trum of dis­or­ders with vary­ing un­der­ly­ing mech­a­nisms.

“Not all ALS pa­tients are the same, and if we want to be suc­cess­ful then we must make ther­a­pies tar­get­ed to what’s go­ing wrong in that spe­cif­ic pa­tient pop­u­la­tion,” Roet said.

QurAlis is de­vel­op­ing three drugs that tar­get sub­types of ALS, in­clud­ing a drug the com­pa­ny hopes would one-up GSK’s epilep­sy drug Trobalt (reti­ga­bine), which was pulled from the mar­ket last June due to safe­ty is­sues. QurAlis’ drug would treat over­ac­tive neu­rons and pre­vent cell death via ex­ci­to­tox­i­c­i­ty. QurAlis’ oth­er two pro­grams in­clude a drug can­di­date that would re­store a dys­func­tion­al cel­lu­lar waste clear­ance sys­tem that pro­gres­sive­ly poi­sons neu­rons, and a de­vice to re­move tox­ic pro­teins.

The com­pa­ny’s edge comes from a tech­nol­o­gy it’s li­censed from Q-State Bio­sciences. In fact, QurAlis is a bit of a spin­out from Q-State, with an ex­clu­sive li­cense to use the com­pa­ny’s plat­form tech­nol­o­gy to de­vel­op ther­a­pies for ALS. Q-State’s claim to fame is a plat­form they call Op­topatch, which us­es laser tech­nol­o­gy to ex­am­ine cell be­hav­ior. Specif­i­cal­ly, they can look at a neu­ron and ex­am­ine how it fires. And that’s use­ful for ALS re­search.

Jonathan Flem­ing

“We took stem cells from ALS pa­tients and found out they fire dif­fer­ent­ly from nor­mal healthy peo­ple,” Roet said. “Op­topatch is sen­si­tive enough to pick up these fir­ing cells, which is good for as­sess­ing if po­ten­tial ther­a­peu­tics can re­store this fir­ing and bring it back to nor­mal lev­els.”

In­ter­est­ing­ly, QurAlis has at­tract­ed a num­ber of no­table ad­vi­sors, in­clud­ing the renowned struc­tur­al bi­ol­o­gist and drug de­sign ex­pert Manuel Navia, who was a found­ing sci­en­tist at Ver­tex Phar­ma­ceu­ti­cals (and who you may re­mem­ber as a key char­ac­ter from the book chron­i­cling Ver­tex’s rise: “Bil­lion Dol­lar Mol­e­cule”).

Navia is a drug hunter and the guy who solved the struc­ture of the first drug for AIDS pa­tients. Roet said he was ea­ger to con­tribute the same ef­fort for ALS pa­tients.

“Watch­ing Manuel work with our CSO Daniel El­baum is like watch­ing mag­ic,” Roet said.

The com­pa­ny, which has on­ly four full-timers on board, has enough funds in hand to car­ry the start­up through to a Se­ries A round.

Im­age: Kevin Eggan pre­sent­ing at the 2016 FU­SION sym­po­sium at Har­vard Uni­ver­si­ty.YOUTUBE

Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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The FDA will hus­tle up an ex­pe­dit­ed re­view for As­traZeneca’s next shot at a block­buster can­cer drug fran­chise

AstraZeneca paid a hefty price to partner with Daiichi Sankyo on their experimental antibody drug conjugate for HER2 positive breast cancer. And they’ve been rewarded with a fast ride through the FDA, with a straight shot at creating another blockbuster oncology franchise.

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Sean Parker, AP

Sean Park­er helps cre­ate a CRISPRed cell ther­a­py 2.0 play — and he’s got a high-pro­file set of lead­ers on the team

You can rack up one more high-profile debut effort in the wave of activity forming around cell therapy 2.0. It’s another appealing Bay Area group that’s attracted some of the top hands in the business to a multi-year effort to create a breakthrough. And they have $85 million in hand to make that first big step to the clinic.

Today it’s Ken Drazan and the team at South San Francisco-based ArsenalBio that are coming from behind the curtain for a public bow, backed by billionaire Sean Parker and a collection of investors that includes Beth Seidenberg’s new venture investment operation based in LA.
Drazan — a J&J Innovation vet with a long record of entrepreneurial endeavors — exited the stage in 2018 when his last mission ended as he stepped aside as president of Grail. It wasn’t long, though, before he was helping out with a business plan for ArsenalBio that revolved around the work of a large group of interconnected scientists supported by the Parker Institute for Cancer Immunology.

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CSL ac­cus­es ri­val Pharm­ing of par­tic­i­pat­ing in a scheme to rip off IP on HAE while re­cruit­ing se­nior R&D staffer

Pharming has landed in the middle of a legal donnybrook after recruiting a senior executive from a rival R&D team at CSL. The Australian pharma giant slapped Pharming with a lawsuit alleging that the Dutch biotech’s new employee, Joseph Chiao, looted a large cache of proprietary documents as he hit the exit. And they want it all back.
Federal Judge Juan Sanchez in the Eastern District Pennsylvania court issued an injunction on Tuesday prohibiting Chiao from doing any work on HAE or primary immune deficiency in his new job and demanding that he return any material from CSL that he may have in his possession. And he wants Pharming to tell its employees not to ask for any information on the forbidden topics.
For its part, Pharming fired off an indignant response this morning denying any involvement in extracting any kind of IP from CSL, adding that it’s cooperating in the internal probe that CSL has underway.

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Eli Lil­ly’s first PhI­II show­down for their $1.6B can­cer drug just flopped — what now?

When Eli Lilly plunked down $1.6 billion in cash to acquire Armo Biosciences a little more than a year ago, the stars seemed aligned in its favor. The jewel in the crown they were buying was pegilodecakin, which had cleared the proof-of-concept stage and was already in a Phase III trial for pancreatic cancer.

And that study just failed.

Lilly reported this morning that their cancer drug flopped on overall survival when added to FOLFOX (folinic acid, 5-FU, oxaliplatin), compared to FOLFOX alone among patients suffering from advanced pancreatic cancer.

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