An­oth­er myo­statin pro­gram com­busts, but Ac­celeron has plen­ty in the pipeline

Myo­statin in­hibitors — en­gi­neered to build mus­cle and im­prove mo­bil­i­ty in pa­tients — the­o­ret­i­cal­ly make sense for use in pa­tients with mus­cle-wast­ing dis­eases. But the field is lit­tered with fail­ure — and so it was hard­ly sur­pris­ing when Ac­celeron, which years ago scrapped a myo­statin pro­gram it had part­nered with Shire, dis­closed an­oth­er flopped myo­statin drug on Mon­day.

Ac­celeron’s drug, ACE-083, was be­ing test­ed in a mid-stage study in pa­tients with Char­cot-Marie-Tooth dis­ease (CMT), a group of in­her­it­ed dis­or­ders that cause nerve dam­age and are char­ac­ter­ized by small­er, weak­er mus­cles.

In the study, ACE-083 in­duced a sta­tis­ti­cal­ly sig­nif­i­cant in­crease in mean to­tal mus­cle vol­ume — meet­ing the main goal. How­ev­er, that in­crease did not trans­late to sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in any of the func­tion­al or qual­i­ty of life sec­ondary end­points, ver­sus place­bo. Ac­celeron is, there­fore, dis­con­tin­u­ing the de­vel­op­ment of the ex­per­i­men­tal com­pound, it said.

Back in Sep­tem­ber, ACE-083 failed to elic­it func­tion­al ben­e­fit in a sep­a­rate study in pa­tients with fa­cioscapu­lo­humer­al mus­cu­lar dy­s­tro­phy, a ge­net­ic mus­cle-wast­ing con­di­tion that caus­es the mus­cles of the limbs, shoul­ders, and face to weak­en.

This CMT re­sult was not sur­pris­ing, giv­en the sim­i­lar lack of func­tion­al ben­e­fit from the FSHD tri­al, SVB Leerink’s Ge­of­frey Porges wrote in a note.

The Cam­bridge, Mass­a­chu­setts-based com­pa­ny’s stock $XL­RN closed down about 6.6% at $85.29 on Mon­day and was bare­ly down 0.1% in Tues­day pre­mar­ket trad­ing.

ACE-083 is the lat­est ca­su­al­ty in a list of myo­statin ef­forts that have crashed and burned. Ac­celeron and Shire halt­ed clin­i­cal work on ACE-031 as ear­ly as 2011 and scrapped the pro­gram al­to­geth­er in 2013; No­var­tis’ bima­grum­ab failed a mid-stage study in 2016; Pfiz­er dropped the Duchenne plans for do­ma­grozum­ab fol­low­ing a dis­ap­point­ing Phase II tri­al in 2018; and last No­vem­ber, Roche aban­doned its Duchenne drug (ac­quired from Bris­tol My­ers Squibb in 2017) af­ter a fu­til­i­ty analy­sis sug­gest­ed de­vel­op­ing the com­pound fur­ther was fu­tile.

Myo­statin is a pro­tein that forms part of the trans­form­ing growth fac­tor be­ta (TGFβ) su­per­fam­i­ly — a group of pro­teins that help con­trol the growth and de­vel­op­ment of tis­sues across the body. The pro­tein nor­mal­ly re­strains mus­cle growth, en­sur­ing that mus­cles do not grow too large.

Mean­while, Ac­celeron has oth­er ar­eas of fo­cus.

The com­pa­ny in No­vem­ber se­cured the ap­proval of Cel­gene-part­nered red blood cell boost­ing drug Re­blozyl — known chem­i­cal­ly as lus­pa­ter­cept — to treat ane­mia in pa­tients with be­ta-tha­lassemia who re­quire reg­u­lar trans­fu­sions. Al­though its use in myelodys­plas­tic syn­dromes — which will like­ly de­ter­mine if the drug, pegged to even­tu­al­ly gen­er­ate glob­al peak sales of more than $3 bil­lion, is a big com­mer­cial suc­cess  — is still un­der Phase III re­view.

Ear­li­er this year, fo­cus al­so turned to so­tater­cept as the com­pa­ny re­port­ed pos­i­tive mid-stage da­ta in pa­tients with pul­monary ar­te­r­i­al hy­per­ten­sion (PAH). Un­like ex­ist­ing PAH ther­a­pies that are de­signed to di­late the pul­monary ar­ter­ies and re­duce blood pres­sure, so­tater­cept is en­gi­neered to re­store BM­PR-II sig­nal­ing, a key dri­ver of the dis­ease. If all goes well in piv­otal stud­ies, and the drug wins ap­proval — Porges es­ti­mates the drug could rake in glob­al sales of about $1.2 bil­lion in 2028.

“Af­ter that (FSHD) re­sult we re­moved all of the val­ue of this pro­gram from our mod­el and fore­casts, and for this rea­son, this (CMT) dis­clo­sure has no im­pact on our val­u­a­tion,” Porges said.

“It does con­firm that Ac­celeron in­vestors and man­age­ment were in­deed for­tu­nate that their so­tater­cept phase II tri­al in PAH had a pos­i­tive out­come, and the mag­ni­tude of the ben­e­fit in that tri­al, and the fu­ture de­vel­op­ment path, time­line and cost, be­come even more im­por­tant to the stock and its val­ue now.”

Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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Bob Nelsen (Lyell)

As bear mar­ket con­tin­ues to beat down biotech, ARCH clos­es a $3B ear­ly-stage fund

One of the biggest names in biotech investing has a whole lot of new money to spend.

ARCH Venture Partners closed its 12th venture fund early Wednesday morning, the firm said, bringing in almost $3 billion to invest in early-stage biotechs. The move comes about a year and a half after ARCH announced its previous fund, for almost $2 billion back in January 2021.

In a statement, ARCH managing director and co-founder Bob Nelsen appeared to brush off concerns about the broader market troubles, alluding to the downturn that’s seen several biotechs downsize and the XBI fall back to almost pre-pandemic levels.

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Hank Safferstein, Generian CEO

Astel­las sub­sidiary to part­ner with Pitts­burgh up­start in search for 'un­drug­gable' pro­teins

As Astellas continues its drive to build out its gene therapy portfolio and capabilities, a subsidiary of the Japanese pharma company has entered into a collaboration with a little-known Pittsburgh biotech.

Astellas-owned Mitobridge and Generian Pharmaceuticals announced on Wednesday that they will work together in a new deal for “undruggable” protein targets. Generian will net an undisclosed upfront payment and could get up to $180 million in milestones, should anything from its platform prove successful, as well as single-digit royalties on global net sales.

Adam Simpson, Icosavax CEO

Reel­ing from Covid flop, Icosavax says its RSV can­di­date passed ear­ly test. But in­vestors need some more con­vinc­ing

Three months separated from a disappointing readout of its Covid-19 vaccine, Icosavax is back with what it calls positive topline data for a different VLP vaccine candidate — although investors aren’t impressed.

IVX-121, a vaccine candidate for respiratory syncytial virus (RSV), appeared to generate “robust” immune responses among both young and older adults, as measured by neutralizing antibodies, and appeared generally well-tolerated, Icosavax reported.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Sanofi to cut in­sulin prices for unin­sured from $99 to $35, match­ing the in­sulin cap com­ing through Con­gress

As the House-passed bill to cap the monthly price of insulin at $35 nationwide makes its way for a Senate vote soon, Sanofi announced Wednesday morning that beginning next month it will cut the monthly price of its insulins for uninsured Americans to $35, down from $99 previously.

The announcement from Sanofi, which allows the uninsured to buy one or multiple Sanofi insulins (Lantus, Insulin Glargine U-100, Toujeo, Admelog, and Apidra) at $35 for a 30-day supply effective July 1, follows House passage (232-193) of the monthly cap in March, with just 12 Republicans voting in favor of the measure.

Scoop: Roche scraps one of two schiz­o­phre­nia PhII tri­als af­ter fail­ing the pri­ma­ry end­point

Roche has terminated one of two Phase II trials testing its drug ralmitaront in patients with schizophrenia, the Big Pharma confirmed to Endpoints News.

The study was terminated last month, according to a June 22 update to the registry on clinicaltrials.gov. Begun in September 2020, the trial was looking at ralmitaront in patients with acute schizophrenia. The trial enrolled 286 patients out of an originally planned 308.

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