L to R: Jeroen Bekaert, Ivan Dimov and Nate Fernhoff, Orca Bio (Orca)

Months af­ter achiev­ing uni­corn sta­tus, Or­ca steers in­to the fast lane with an RMAT des­ig­na­tion for cell ther­a­py can­di­date

A few months af­ter its last big catch — a $192 mil­lion Se­ries D and rare uni­corn sta­tus — Or­ca Bio has reeled in the FDA’s re­gen­er­a­tive med­i­cine ad­vanced ther­a­py (RMAT) des­ig­na­tion for its ex­per­i­men­tal cell ther­a­py to treat blood stem cell trans­plant pa­tients.

Or­ca, one of this year’s End­points 11 star­tups, was found­ed by three en­tre­pre­neurs who were in­trigued by Irv Weiss­man’s cell pu­rifi­ca­tion work while study­ing at Stan­ford. No­var­tis — then San­doz — had pur­chased Weiss­man’s spin­out SyS­temix in the ’90s, then scrapped it due to prac­ti­cal chal­lenges. But Jeroen Bekaert, Ivan Di­mov and Nate Fern­hoff saw po­ten­tial there.

Now, the biotech has RMAT and or­phan drug des­ig­na­tion to steer its cell ther­a­py can­di­date Or­ca-T through the clin­ic. RMAT des­ig­na­tion comes with the ben­e­fits of both fast track and break­through ther­a­py des­ig­na­tions. Or­ca-T en­tered a Phase I/II tri­al in pa­tients un­der­go­ing stem cell trans­plants for var­i­ous blood can­cers last No­vem­ber, ac­cord­ing to an NIH fil­ing. The biotech ex­pects to read out da­ta from that tri­al this De­cem­ber at the an­nu­al Amer­i­can So­ci­ety of Hema­tol­ogy meet­ing.

“The RMAT and ODD for Or­ca-T is up­lift­ing news for pa­tients with var­i­ous blood can­cers in­clud­ing acute myeloid leukemia, acute lym­phoid leukemia, myelodys­plas­tic syn­drome and myelofi­bro­sis,” Di­mov said in a state­ment.

While a hematopoi­et­ic stem cell trans­plant is the best op­tion for some pa­tients with blood can­cer, the pro­ce­dure can lead to dan­ger­ous com­pli­ca­tions, such as graft-ver­sus-host dis­ease, in which im­mune cells from the donor at­tack the pa­tient’s healthy cells. Or­ca-T is de­signed to re­pro­gram the dis­eased blood sys­tem, by match­ing “a spe­cif­ic com­po­si­tion of stem and im­mune cells to the im­muno­log­i­cal needs of a pa­tient.”

Or­ca’s re­cent Se­ries D brought its to­tal raise to about $300 mil­lion, and came in as the com­pa­ny’s val­u­a­tion sur­passed $1 bil­lion. Plus, the biotech touts Weiss­man, for­mer No­var­tis CEO Joe Jimenez, and Lyell head and for­mer NCI chief Rick Klaus­ner as its ad­vi­sors.

Di­mov spoke with End­points News last month about the in­spi­ra­tion be­hind the biotech’s 2016 launch:

It al­most seems un­eth­i­cal from a hu­man per­spec­tive be­cause some of the ini­tial re­sults sug­gest high­ly cu­ra­tive ef­fects and the whole thing was shut down for over a decade be­cause of some of these chal­lenges of prac­ti­cal­ly im­ple­ment­ing these things and de­ploy­ing them. So that was sort of the key dri­ver for us to get start­ed. And we al­ways knew we want­ed to ful­fill that dream all the way through and not re­al­ly stop at a cer­tain stage and leave it for some­one else.

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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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.

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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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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Bo Cumbo, new Solid Bio CEO

Sol­id Bio gets a new CEO, $75M cash and drops lead drug as big in­vestors ju­ry-rig a merg­er deal

Three months after Endpoints News broke the story that gene therapy outfit AavantiBio had gutted its CMC group in a reorganization, the biotech’s CEO has helped engineer a merger with struggling penny stock player Solid Bio. And he’s going to remain in charge of the combined operation, as Solid founder Ilan Ganot steps aside.

The merger news this morning features some high-profile investors.

Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75 million raise to add to the pool of cash Solid will have after the tie-up. That will leave Solid $215 million in cash as Bain’s Adam Koppel jumps on the board — enough to pay for ops and get through some key data milestones on their way into 2025.

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