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

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.

Novartis is calling off a UK-based trial for Leqvio in the primary prevention of cardiovascular events in patients with high cholesterol, the company confirmed on Wednesday.

The Swiss pharma giant made the decision after “careful evaluation,” a spokesperson told Endpoints News via email. The trial, dubbed ORION-17, was planned in partnership with England’s National Health Service (NHS) and was part of the company’s strategy to establish Leqvio as a standard of care in cardiovascular disease management.