CEO Mark McKenna (Prometheus Biosciences)

Prometheus Bio is ready for its close-up shot, fol­low­ing a $130M round to back IBD drugs now head­ed to the clin­ic

A lit­tle over a year ago, Take­da lined up a deal to col­lab­o­rate with a low-pro­file San Diego up­start called Prometheus Bio­sciences on a string of new drugs for in­flam­ma­to­ry bow­el dis­ease. The phar­ma play­er tossed an undis­closed up­front and up to $420 mil­lion in mile­stones to get the al­liance start­ed.

These days, pre­clin­i­cal deals are com­mon, mile­stones are hazy mark­ers of fi­nan­cial health and the pact didn’t get a lot of at­ten­tion. But that ob­scu­ri­ty should start to dis­si­pate this week.

To­day, Prometheus, which had been cre­at­ed by the ac­qui­si­tion of Prometheus Labs by Pre­ci­sion IBD, is show­ing off a whop­ping $130 mil­lion raise from some fa­mil­iar biotech in­vestors jump­ing in to stretch the run­way at the 4-year-old pre­clin­i­cal biotech out in­to 2023, when CEO Mark McKen­na is promis­ing to have 3-4 IBD drugs ei­ther in the clin­ic or IND ready. The first of those clin­i­cal pro­grams is slat­ed to be­gin in a mat­ter of weeks, putting McKen­na’s team on track to their first round of hu­man da­ta.

Spun out of Cedars-Sinai Med­ical Cen­ter, the com­pa­ny built a con­sid­er­able por­tion of its sci­en­tif­ic foun­da­tion around the work of Stephan Tar­gan, an IBD spe­cial­ist who found­ed the IBD Cen­ter at the med­ical cen­ter some 28 years ago.

“We lever­aged this big da­ta set built over 20 years to iden­ti­fy new tar­gets in IBD and de­vel­op com­pan­ion di­ag­nos­tics for the drugs,” says McKen­na, cit­ing 20,000 sam­ples in the da­ta bank. Us­ing bioin­for­mat­ics tech, they have been work­ing on spot­light­ing tar­get­ed ther­a­peu­tics that can do bet­ter — in a much more durable fash­ion — than the cur­rent gen­er­a­tion of IBD drugs.

Their lead pro­gram — PRA023 — is an an­ti-TL1A an­ti­body. But they’re not alone. Pfiz­er has a sim­i­lar pro­gram. But McKen­na has am­bi­tions to build a broad pipeline around IBD.

“We be­lieve we can put out a new tar­get every year,” he tells me.

You may re­call that McKen­na spent a 4-year stretch fo­cused on Bausch sub Sal­ix Phar­ma­ceu­ti­cals, where he won ku­dos for the turn­around work he ac­com­plished there. Once seen as a pos­si­ble suc­ces­sor to Bausch chief Joe Pa­pa, he now wants to take this new com­pa­ny all the way through to com­mer­cial­iza­tion. And Pa­pa has joined his board at Prometheus, watch­ing the work from a front-row seat.

It was Prometheus chair­man Tachi Ya­ma­da, a leg­end in the biotech busi­ness, who reached out to McKen­na to see if he would be in­ter­est­ed in run­ning Prometheus, af­ter watch­ing his work at Sal­ix.

Ya­ma­da, no doubt, al­so played a role in bring­ing to­geth­er a large syn­di­cate of in­vestors.

Even­tide As­set Man­age­ment and RTW In­vest­ments led the round with a slate of new in­vestors: Per­cep­tive Ad­vi­sors, Cor­morant Cap­i­tal, Cowen Health­care In­vest­ments, Point72 As­set man­age­ment and Irv­ing In­vestors. Then there are the ex­ist­ing in­vestors: As­cend Glob­al In­vest­ment Fund, Cedars-Sinai Med­ical Cen­ter and Nestlé SA. Joy Ghosh of Even­tide As­set Man­age­ment and Adam Stone of Per­cep­tive Ad­vi­sors will be join­ing the Prometheus board to watch over their in­vest­ments.

They’re back­ing a com­pa­ny that has grown to 125 staffers, in a mar­ket that might sug­gest they’re think­ing about an IPO.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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George Church (courtesy EnPlusOne BioSciences)

George Church, Wyss sci­en­tists and North­pond chal­lenge con­ven­tion­al RNA man­u­fac­tur­ing with new biotech

RNA medicine has been at the forefront for the past few years, with the first RNA silencing therapy approved in 2018, and mRNA Covid vaccines following after. But flying under the radar has been the process of actually making RNA for these treatments.

That’s what Daniel Wiegand and Jonathan Rittichier have been working on in George Church’s lab for the past six years.

Friday morning, they unveiled EnPlusOne Biosciences, a biotech built on their RNA synthesis platform. Wiegand will serve as the Watertown, MA-based biotech’s CEO, and Rittichier will be CSO. And no different from his other startups, Church will be acting as scientific advisor. Its fourth co-founder, Dan Ahlstedt, joined through a Harvard Business School program, and will be COO.

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Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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