Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarep­ta Ther­a­peu­tics has filed the da­ta need­ed for an FDA ac­cel­er­at­ed ap­proval, which would be the biotech’s fourth if grant­ed by the agency.

The biotech has yet to com­plete con­fir­ma­to­ry tri­als for those first three con­di­tion­al nods. The fil­ing for its fourth Duchenne mus­cu­lar dy­s­tro­phy treat­ment, dis­closed Thurs­day, is not a sur­prise. Sarep­ta said in late-Ju­ly it would do so af­ter re­leas­ing pos­i­tive re­sults for the Roche-part­nered gene ther­a­py.

Sarep­ta is ask­ing for a con­di­tion­al green­light for de­landis­tro­gene mox­epar­vovec, al­so known as SRP-9001, based on a sur­ro­gate end­point that the drug­mak­er thinks should trans­late in­to clin­i­cal ben­e­fit once borne out in a con­fir­ma­to­ry study. That tri­al, dubbed EM­BARK, is al­ready ful­ly en­rolled, the biotech said.

Af­ter get­ting three an­ti­sense oligonu­cleotide ther­a­pies across the fin­ish line, Sarep­ta is look­ing for a long-term treat­ment via gene ther­a­py, and the ap­proval re­quest comes in light of two re­cent FDA bless­ings for gene ther­a­pies out of blue­bird bio, some of the most ex­pen­sive treat­ments — ad­min­is­tered one time — in US his­to­ry.

SRP-9001 ini­tial­ly failed a por­tion of the Phase II study in ear­ly 2021, but a sec­ond part showed pa­tients had sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in mo­tor abil­i­ties and lat­er showed that mo­tor func­tion im­proved af­ter one year. — Kyle LaHu­cik

Ax­some, with first drug in the bag, re­sub­mits an­oth­er as­set for mi­graines

A lit­tle more than a month af­ter snag­ging ap­proval for its rapid-act­ing de­pres­sion drug, Ax­some Ther­a­peu­tics said it plans to re­sub­mit an­oth­er one of its as­sets fol­low­ing a “suc­cess­ful” con­fab with the FDA.

The New York City biotech in­tends to ask the FDA once again to ap­prove its acute mi­graine treat­ment, dubbed AXS-07, af­ter a Type A meet­ing with the agency in which Ax­some got clar­i­ty on the May re­jec­tion.

The is­sues cen­tered on chem­istry, man­u­fac­tur­ing and con­trols, or CMC, as Ax­some has pre­vi­ous­ly in­di­cat­ed, in­clud­ing in the week lead­ing up to the re­jec­tion.

In its new fil­ing, the biotech plans to sub­mit new CMC in­for­ma­tion, in­clud­ing new com­mer­cial scale batch­es of the drug. Ax­some doesn’t need any more clin­i­cal ef­fi­ca­cy or safe­ty da­ta, the biotech said. A six-month re­view is ex­pect­ed. — Kyle LaHu­cik

BMS work­ing with Con­cer­tAI to ad­vance an AI-pow­ered clin­i­cal tri­al plat­form

A Mass­a­chu­setts-based AI-cen­tered com­pa­ny and a large phar­ma are ad­vanc­ing a soft­ware-as-a-ser­vice dig­i­tal tri­al plat­form for on­col­o­gy pa­tients.

Con­cer­tAI’s dig­i­tal tri­al plat­form aims to sim­pli­fy as­pects of clin­i­cal tri­als such as pa­tient iden­ti­fi­ca­tion, con­sent and con­tract ne­go­ti­a­tions, among oth­er fac­tors.

The com­pa­ny an­nounced Thurs­day that Bris­tol My­ers Squibb is look­ing to put its plat­form to use. Venkat Sethu­ra­man, the se­nior vice pres­i­dent for glob­al bio­met­rics and da­ta sci­ence at Bris­tol My­ers, said in a state­ment that the com­pa­ny is plan­ning to use the plat­form to give pa­tients greater ac­cess to on­col­o­gy stud­ies.

How­ev­er, both have been work­ing to­geth­er for the past two years. “As an in­dus­try leader in dis­cov­er­ing, de­vel­op­ing and de­liv­er­ing med­i­cines to help pa­tients pre­vail over se­ri­ous dis­eases, we are em­ploy­ing dig­i­tal in­no­va­tion to re­design and re­de­fine how pre- and post-ap­proval stud­ies are con­duct­ed and, to­geth­er with Con­cer­tAI, we are fun­da­men­tal­ly chang­ing the way we do clin­i­cal re­search from study de­sign, through en­roll­ment and ex­e­cu­tion,” Sethu­ra­man said in the state­ment.

Con­cer­tAI has been on a roll this year as the com­pa­ny raised a $150 mil­lion Se­ries C to not on­ly move its tech­nol­o­gy along but to rope in more users. — Tyler Patchen

Mor­phoSys touts long-term Mon­ju­vi da­ta

Mor­phoSys an­nounced Wednes­day that it has pos­i­tive mul­ti-year da­ta on its lym­phoma drug Mon­ju­vi.

In short, Mon­ju­vi (taf­a­sita­m­ab) as a monother­a­py com­bined with lenalido­mide, the gener­ic name for Bris­tol My­ers Squibb’s Revlim­id, showed “long-term ef­fi­ca­cy” in pa­tients with re­lapsed or re­frac­to­ry (R/R) dif­fuse large B-cell lym­phoma. Long-term was de­fined as at least two years, with six pa­tients treat­ed for at least five years.

At the da­ta cut­off of Feb­ru­ary 15, 2022, 27 of the 80 en­rolled pa­tients had been treat­ed for at least two years, with a me­di­an treat­ment du­ra­tion of 4.3 years. And of those 27, 23 were alive at da­ta cut­off as 13 pa­tients re­mained on treat­ment.

Back to the 27: Mor­phosys said that 23 of those 27 had a com­plete re­sponse to the treat­ment, with the oth­er four not­ing a par­tial re­sponse. How­ev­er, their state­ment did not in­di­cate if the 23 who had the com­plete re­sponse were the same as those alive at the da­ta cut­off.

But all the da­ta haven’t been re­vealed yet — ac­cord­ing to Mor­phoSys, the da­ta will be pre­sent­ed at a poster ses­sion at the on­go­ing So­ci­ety of Hema­to­log­ic On­col­o­gy con­fer­ence in Hous­ton, Texas this week­end.

“These long-term fol­low-up re­sults for L-MIND reaf­firm our be­lief that taf­a­sita­m­ab plus lenalido­mide re­mains the in-prac­tice, out­pa­tient tar­get­ed im­munother­a­py of choice for this group of pa­tients,” said out­go­ing R&D chief Malte Pe­ters via state­ment. — Paul Schloess­er

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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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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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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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.

FDA tells Catal­ent to fix is­sues at two man­u­fac­tur­ing sites on its own

The CDMO Catalent will have to fix issues at two manufacturing plants in the US and Europe that were subject to inspections by the FDA this summer, giving the company room to correct the issues without facing further regulatory action.

The FDA gave Catalent a “voluntary action indicated” response to two inspections at the contract manufacturer’s site in Bloomington, IN, and Brussels, Belgium. Fixing the issues on its own is a preferable outcome to facing an “official action indicated” response, meaning that an official warning would be sent out or a sit-down with the FDA would be required.

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Merck targets vaccine-hesitant parents in its latest 'Why Vaccines' campaign. (Image: Shutterstock)

Mer­ck­'s lat­est 'Why Vac­ci­nes' cam­paign seeks to bet­ter in­form vac­cine-hes­i­tant moms

From Hollywood couple endorsements to targeted equity efforts, Merck has been pushing the value of vaccinations, especially since the Covid-19 pandemic disruption. Now the pharma is turning to a new target — vaccine-hesitant parents, and moms in particular.

Merck’s “Why Vaccines” latest social media and digital campaign spotlights real-life new moms who have questions about vaccinating their children.

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