Backed by a biotech leg­end, two young en­tre­pre­neurs tack­le one of the Holy Grails of R&D

Joshua Co­hen

It’s not of­ten that two re­cent col­lege grads can come up with enough cash and con­nec­tions to run a Phase II study of a new com­bi­na­tion drug for a tough and dead­ly dis­ease like ALS. But with some back­ing by biotech leg­end Hen­ri Ter­meer and $8 mil­lion in ven­ture cash and grant mon­ey, Justin Klee and Josh Co­hen say they’re ready to turn what start­ed out as an un­der­grad­u­ate sci­ence project at Brown in­to a clin­i­cal re­al­i­ty in a mat­ter of months at a start­up dubbed Amy­lyx.

The key com­po­nent in all this, the mon­ey to start dos­ing pa­tients, comes from a $5 mil­lion Se­ries A led by Morn­ing­side Ven­ture with con­tri­bu­tions from the ALS In­vest­ment Fund and for­mer Gen­zyme CEO Ter­meer. Com­bined with a $3 mil­lion grant from the ALS Ac­cel­er­at­ed Ther­a­peu­tics Ini­tia­tive and some dis­count pric­ing from a net­work of hos­pi­tals en­gaged in ALS work, and Klee and Co­hen say they’ve reached the thresh­old of a mid-stage tri­al that will look for both ef­fi­ca­cy and safe­ty da­ta.

Justin Klee

“It is atyp­i­cal,” Klee al­lows in our tele­phone in­ter­view this morn­ing. But the two young en­tre­pre­neurs are prepar­ing to take a shot at ALS on a bud­get that most Big Phar­mas would spend on pre­clin­i­cal work. And they say their work could have ap­pli­ca­tions for Alzheimer’s, an­oth­er dev­as­tat­ing dis­ease that has so far burned bil­lions of dol­lars in large­ly wast­ed re­search ef­forts.

ALS, or more for­mal­ly Amy­otroph­ic Lat­er­al Scle­ro­sis, has de­fied re­searchers for years now. The dis­ease first crip­ples and then typ­i­cal­ly kills its vic­tims in just a few years. Much of the work has been stymied by a fun­da­men­tal lack of un­der­stand­ing of what caus­es the dis­ease. But the two new­ly mint­ed biotech en­tre­pre­neurs say that they’re tak­ing two dif­fer­ent drugs and will look to ad­dress two dif­fer­ent man­i­fes­ta­tions of the dis­ease in an at­tempt to dis­rupt the crip­pling cas­cade of events that af­flicts pa­tients.

“We thought, why don’t we look with what we know is cor­re­lat­ing to pa­tients’ clin­i­cal de­cline,” says Klee. “In Alzheimer’s you lose neu­rons in your mem­o­ry cen­ter, the brain re­gions in­volved in mem­o­ry. The same in ALS, you lose neu­rons in the mo­tor cor­tex and spinal cord, and you lose move­ment abil­i­ty. The oth­er thing,” he adds, is that as neu­rons die, the brain im­mune sys­tem re­sponds and kills more neu­rons, caus­ing a tox­ic cy­cle that speeds the course of the dis­ease.

Amy­lyx is fo­cus­ing on the en­er­gy cri­sis in the mi­to­chon­dria and the un­fold­ed pro­tein prob­lem that caus­es a lot of the cell death, says Co­hen.

The two have honed in on a new treat­ment, AMX0035, that com­bines two drugs, sodi­um phenyl­bu­tyrate (PB) and tau­rour­sodeoxy­cholic acid (TUD­CA), aimed at tack­ling both those prob­lems at once. They have some clin­i­cal da­ta from ear­li­er stud­ies on both and enough back­ing now to test their idea that they can syn­er­gis­ti­cal­ly ad­dress ALS.

It’s a tall or­der con­cern­ing one of the Holy Grails of biotech R&D; some­thing akin to first-timer climbers tack­ling Mt. Ever­est on a shoe­string bud­get. But they say that the non­prof­it groups, es­pe­cial­ly in­volv­ing the hos­pi­tals like Mass Gen­er­al where the ex­pe­ri­enced in­ves­ti­ga­tors re­side, have made it all pos­si­ble with some key pric­ing breaks. And now, with some high pro­file con­nec­tions like Ter­meer, the vir­tu­al biotech with a staff of three full timers is start­ing out to see if they can get close to a peak that has elud­ed so many be­fore.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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Anap­tys­Bio's etokimab pro­vides more dis­ap­point­ing re­sults, rais­ing ques­tions about com­pound's fu­ture

The lead program for AnaptysBio’s in-house pipeline has hit another setback.

Etokimab, an IL-33 inhibitor, did not achieve statistically significant improvement in a Phase II trial for patients suffering from chronic rhinosinusitis with nasal polyps. Researchers measured the individuals’ bilateral nasal polyps score and sino-nasal outcome test, finding that neither improved upon a placebo after both four- and eight-week time markers, though they did demonstrate improvement over baseline levels of the examinations.

Brian Stuglik, Verastem CEO

The du­velis­ib hot pota­to is tossed to a new own­er as Ve­rastem looks to re­or­ga­nize around the pipeline

When Infinity put up duvelisib for a no-money-down instant deal, the biotech was looking for a quick exit from a clinical disaster. AbbVie had walked away from their alliance after looking at how the data stacked up in a crowded field.

And while it was approvable, it wasn’t looking pretty to anyone who thought in commercial terms.

One Big Pharma’s trash, though, was seen as a biotech treasure as a deeply troubled Verastem stepped up to grab the PI3K-delta/gamma — promising to run it across the goal lines at the FDA. And they did just that, only with little to show for it.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.