Christophe Bourdon, Orphazyme

Or­p­hazyme plans to lay off most of its staff fol­low­ing a string of fail­ures for lead 'pipeline-in-a-pro­duc­t'

Ré­mi Droller

Dan­ish bio­phar­ma Or­p­hazyme has been on a roller coast­er of a ride late­ly, and the com­pa­ny plum­met­ed down a bit fur­ther on Mon­day with the an­nounce­ment that it will lay off two-thirds of its work­force.

The news comes near­ly two months af­ter ari­mo­clo­mol, the com­pa­ny’s “pipeline-in-a-prod­uct,” flopped in ALS. The drug failed to meet pri­ma­ry or sec­ondary end­points for func­tion and sur­vival. The drug was hand­ed a com­plete re­sponse let­ter from the FDA two weeks ago, and its stock plum­met­ed 56.39 % to $6.35.

Da­ta have not yet been re­leased for that tri­al.

The lay­offs are meant to sup­port “es­sen­tial ac­tiv­i­ties,” the com­pa­ny said in a press re­lease, which in­cludes pur­su­ing reg­u­la­to­ry ap­proval in Eu­rope for ari­mo­clo­mol. Ré­mi Droller, Mar­ti­jn Klei­jwegt, and An­ders Hede­gaard will all re­sign from the board of di­rec­tors as well, the com­pa­ny said.

In a state­ment, CEO Christophe Bour­don said:

As a re­sult of the re­struc­tur­ing of the com­pa­ny and our rig­or­ous cost sav­ing pro­gram, we will have to part ways with many of our most val­ued and tal­ent­ed col­leagues. I thank each of them for their strong com­mit­ment to Or­p­hazyme and ded­i­ca­tion to show­ing up for pa­tients in need. The im­me­di­ate ac­tions we are tak­ing are nec­es­sary to pro­tect and sup­port the on­go­ing ap­proval process in Eu­rope and the eval­u­a­tion of a path for­ward in the U.S.

Mar­ti­jn Klei­jwegt

In March, the com­pa­ny an­nounced that ari­mo­clo­mol failed to hit its end­points in Phase II/III tri­als for pa­tients with in­clu­sion body myosi­tis, a de­bil­i­tat­ing mus­cle-wast­ing dis­ease. Near­ly three years ago, the drug missed both pri­ma­ry end­points in its Phase III Nie­mann-Pick dis­ease type C study. There is no ap­proved med­i­cine for NPC, a rare dis­ease that is es­ti­mat­ed to im­pact 1 in 100,000 lives. Mon­day, Or­p­hazyme al­so dropped da­ta from a 36-month open-la­bel ex­ten­sion tri­al, not­ing that there was a slow of pro­gres­sion in pa­tients af­ter three years. The drug al­so demon­strat­ed a con­sis­tent safe­ty pro­file. There were 41 to­tal pa­tients that joined the tri­al, the com­pa­ny said.

An­ders Hede­gaard

The com­pa­ny al­so in­tends to ad­vance ari­mo­clo­mol in­to clin­i­cal de­vel­op­ment in Gauch­er dis­ease, the in­her­it­ed dis­or­der that caus­es sug­ar-con­tain­ing fats to ac­cu­mu­late in the lyso­somes of cells and af­fect the brain, bone mar­row, spleen and liv­er. That pro­gram is cur­rent­ly in Phase II.

The res­ig­na­tion of the board mem­bers and slash­ing of its staff are just the lat­est in a string of per­son­nel moves. In De­cem­ber 2020, CEO Kim Strat­ton re­signed abrupt­ly, and the on­ly ex­pla­na­tion of that the move came “fol­low­ing a di­a­logue ini­ti­at­ed by the board of di­rec­tors.”

Pi­o­neer­ing Click Chem­istry in Hu­mans

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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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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

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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