Silviu Itescu, Mesoblast CEO

Mesoblast sends in im­proved po­ten­cy as­say, look­ing to re­sub­mit to FDA on acute graft-ver­sus-host dis­ease drug

In 2020, the FDA re­ject­ed Mesoblast’s remestem­cel-L, or Ry­on­cil, its lead can­di­date for pe­di­atric acute graft-ver­sus-host dis­ease (aGVHD) that didn’t re­spond to steroids. The FDA raised a num­ber of con­cerns, first ob­ject­ing to Mesoblast’s sin­gle arm, open-la­bel tri­al, though reg­u­la­tors strug­gled to de­scribe how a ran­dom­ized tri­al would work, since pe­di­a­tri­cians and par­ents were re­luc­tant to put chil­dren with aGVHD in a place­bo arm.

Reg­u­la­tors al­so sug­gest­ed that Mesoblast im­prove its po­ten­cy mea­sure­ments for the stem cell ther­a­py, ex­press­ing un­ease to­ward the “in­con­sis­tent batch-to-batch po­ten­cy,” ac­cord­ing to Jef­feries’ David Stan­ton at the time.

Mon­day morn­ing, Mesoblast said that it added new in­for­ma­tion on an op­ti­mized po­ten­cy as­say to the IND file on the drug, set­ting the biotech up to re­sub­mit its BLA in the near fu­ture.

In its Q2 re­port, the New York and Mel­bourne-based biotech said it was aim­ing to file its BLA by the end of the third quar­ter — though that date has just passed.

In a press state­ment, Mesoblast’s CEO Sil­viu Ites­cu said, “The im­proved process con­trols we have put in place to as­sure ro­bust and con­sis­tent com­mer­cial prod­uct, to­geth­er with a po­ten­cy as­say that pre­dicts con­sis­tent sur­vival out­comes, makes remestem­cel-L a com­pelling treat­ment for these chil­dren.”

Mesoblast al­so test­ed remestem­cel-L in Covid-19 pa­tients with ARDS, in which the lungs are in­jured and can­not pro­vide the body with enough oxy­gen. No­var­tis had ini­tial­ly part­nered with Mesoblast on that pro­gram, but ditched the biotech af­ter a Phase III tri­al was cut short by a da­ta safe­ty mon­i­tor­ing board, which said the ther­a­py was un­like­ly to reach its pri­ma­ry end­point.

But Mesoblast hasn’t giv­en up there ei­ther. Ac­cord­ing to its Q2 re­port, it’s work­ing with Van­der­bilt Uni­ver­si­ty Med­ical Cen­ter to de­sign a new tri­al for Covid pa­tients with ARDS un­der the age of 65.

And Mesoblast is try­ing again on its oth­er can­di­date, rexleme­stro­cel-L, as well. That cell ther­a­py al­so missed its pri­ma­ry end­point in a Phase III tri­al in heart fail­ure, but Mesoblast said in its Q2 re­port that it’s meet­ing with the FDA to dis­cuss a po­ten­tial way to ap­proval. In that Phase III tri­al, Mesoblast’s cell ther­a­py failed to re­duce hos­pi­tal­iza­tion rates com­pared to place­bo, but Mesoblast high­light­ed that it met a sec­ondary end­point of re­duc­ing mor­tal­i­ty rate in ear­li­er stages of heart fail­ure.

It’s al­so look­ing to start a piv­otal study for rexleme­stro­cel-L in chron­ic low­er back pain by the end of this year, ac­cord­ing to its Q2 re­port.

For­mer FDA vac­cines deputy Philip Krause joined Mesoblast’s board in March.

Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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Vac­cine doc­u­ments, young lead­ers and mar­ket tur­moil: End­points' 10 biggest sto­ries of 2022

It’s been a volatile year in the world of biopharma. Market declines reset M&A valuations, and may be beginning to tempt bigger buyers back into dealmaking. Russia’s war in Ukraine disrupted drug sales and clinical trials. A new generation of young biotech leaders emerged in the Endpoints 20(+1) Under 40. And as capital runs dry in a tough environment for raising new funds, companies big and small are taking a look at their headcounts and operations for ways to make it through lean times.

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Robert Califf, FDA commissioner (Jose Luis Magana/AP Images)

Fourth ac­cel­er­at­ed ap­proval in Duchenne? Sarep­ta gets pri­or­i­ty re­view for gene ther­a­py amid FDA scruti­ny

Sarepta is once again on the accelerated approval path for a Duchenne drug, picking up a priority review Monday morning.

The FDA granted the accelerated review to SRP-9001, Sarepta announced Monday, which would become the biotech’s fourth Duchenne drug if approved. Much like SRP-9001 will do, each of the previous three therapies went through the accelerated approval pathway. But unlike the others, SRP-9001 is a gene therapy.

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Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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In­dus­try groups, CVS pick apart FDA's pro­posed path­way for gener­ics to carve out OTC in­di­ca­tions

Pharma industry groups like the Association for Accessible Medicines (AAM) and PhRMA are raising pointed questions about an FDA plan to create a new pathway for marketing prescription drugs with an additional condition for nonprescription use (ACNU), which would require more safeguards than the current OTC pathway but essentially carve out new OTC uses for some generic drugs.

Chief among the concerns were: Insurance companies dropping coverage for the Rx version, new ACNU patents to block competition, and industry essentially governing the pathway.

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Albert Bourla, Pfizer CEO (John Thys/POOL/AFP via Getty Images)

Pfiz­er CEO un­der fire from UK watch­dog over vac­cine com­ments — re­port

Pfizer CEO Albert Bourla told the BBC last December that he had “no doubt in my mind that the benefits, completely, are in favor” of vaccinating 5- to 11-year-olds for Covid-19. Almost a year later, those comments have reportedly landed him in trouble with a UK pharma watchdog.

Children’s advocacy group UsForThem filed a complaint with the UK’s Prescription Medicines Code of Practice Authority (PMCPA) last year accusing Bourla of making “disgracefully misleading” statements during the BBC interview, including one that “Covid in schools is thriving.” At the time, UK regulators had not yet cleared the vaccine for the 5 to 11 age group, though the vaccine did have a positive opinion from the EMA’s human medicines committee.

Sanofi's new headquarters, La Maison Sanofi, in Paris (Credit: Luc Boegly)

Sanofi wel­comes 500 staffers to new Paris HQ af­ter €30M ren­o­va­tion

When Paul Hudson took the helm at Sanofi back in 2019, he promised to reinvent the pharma giant — including its Paris headquarters. This week, the company set up shop in new “state-of-the-art” digs.

La Maison Sanofi, as the new HQ is called, is officially open for business, Hudson announced on Monday. The 9,000-square-meter (just under 97,000-square-foot) space accommodates 500 employees across the company’s government and global support functions teams, including finance, HR, legal and corporate affairs — and it was built with environmental sustainability and hybrid work in mind.

Sta­da to place $50M+ in­vest­ment in a new fa­cil­i­ty in Ro­ma­nia

While Romania may conjure up images of vast mountain ranges and tales of medieval kings, one generic manufacturer has broken ground on a new facility there.

German pharma company Stada said Monday that it has placed a €50 million ($51.9 million) investment into a 100,000 square-meter (1.08 million square-foot) site in Turda, Romania, a city in the Southeast of the country. According to a Stada spokesperson in an email to Endpoints News, the company has developed only 281,500 square feet of the site so far.

Rachael Rollins (Charles Krupa/AP Images)

US seeks jail time for co-CEO of New Eng­land com­pound­ing cen­ter af­ter dead­ly 2012 fun­gal out­break

The US attorney for the district of Massachusetts late last week called on the state’s district court to sentence the former co-owner of the now-defunct New England Compounding Center to 18 months of jail time for his role in the center’s quality deviations that led to more than 100 people dead from a fungal meningitis outbreak.

Gregory Conigliaro was convicted of conspiring with more than a dozen others at NECC to deceive the FDA and misrepresent the fact that the center was only dispensing drugs pursuant to patient-specific prescriptions.