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.

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

Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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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.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

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Bris­tol My­ers Squib­b's just-launched MS drug Zeposia makes the cut in key ul­cer­a­tive col­i­tis tri­al

In March, Zeposia became the third oral S1P modulator to secure US approval for multiple sclerosis. Now, the drug has succeeded in a key ulcerative colitis study.

The immunomodulator, akin to others in its class, controls lymphocyte trafficking by limiting the white blood cells to the lymphatic system, in the lymph nodes, and thwarting their ability to jam up lymph nodes — precluding their ability to penetrate the bloodstream and the central nervous system.

Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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Novus Ther­a­peu­tics plunges deep in­to pen­ny stock ter­ri­to­ry af­ter failed ear tri­al

After a more than 15-year run, a California-based biotech is exploring options, including a sale, after its lead experimental therapy failed an exploratory mid-stage study in patients with middle ear infections characterized by a build-up of fluid behind the eardrum.

The company, initially called Tokai Pharmaceuticals but which subsequently changed its name to Novus Therapeutics in 2017, saw its shares more than halve on Monday after the drug — OP0201— did not pass muster as an adjunct therapy to oral antibiotics in infants and children aged 6 to 24 months with acute otitis media (OM).