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