News briefing: Marius submits NDA for oral hypogonadism therapy; Celltrion's billionaire founder steps down
US regulators will soon be looking at a new testosterone replacement therapy, if Marius Pharmaceuticals has its way.
The Raleigh, North Carolina-based biotech submitted on Tuesday an NDA for Kyzatrex, an oral testosterone undecanoate soft gelatin capsule aiming to treat both primary and secondary hypogonadism in men. Should the FDA accept the application for priority review, Marius expects a PDUFA date to follow roughly six months later.
Marius estimates a patient population of about two million men in the U.S., many of whom the biotech says have given up on treatment or choose not to be treated due to a lack of safe options. But the company is hoping its Phase III safety data can sway the FDA toward approval.
In a late-stage trial, more than 96% of participants completed 90 days of treatment and achieved average testosterone levels in the normal range. Kyzatrex also demonstrated an average SBP increase of less than 2 mm Hg as measured by ABPM, the standard FDA blood-pressure assessment.
“Based on available data, Marius has generated the most compelling Phase III results in the testosterone therapy space,” Marius chairman Himanshu Shah told Endpoints News.
The most common side effect in the study was hypertension, and there were no serious adverse events related to the treatment.
Hypogonadism is the most common cause of secondary osteoporosis in men and is a common comorbidity in other diseases like Type 2 diabetes and NASH. Marius expects to file for approval in Europe in the first half of 2022. — Max Gelman
Celltrion’s billionaire founder and chairman departs for blood-testing startup
Almost two decades after starting Celltrion, co-founder Seo Jung-jin has retired from his chairman role.
Seo — the second richest man in Korea — told Korea Biomedical Review he’s taking one more stab at entrepreneurship before he retires, this time focusing on blood testing.
Having started out in contract manufacturing, Celltrion was an early mover in the biosimilars business and recently came up with an antibody for Covid-19 that was authorized for emergency use. — Amber Tong
Sanofi ponies up €7 million milestone to Innate Pharma
Innate Pharma is just about ready to cash a new check.
The French biotech announced Tuesday that Sanofi has selected a target to advance to IND-enabling studies, triggering a €7 million payment as part of its nearly half-billion dollar collaboration with the pharma giant. The target, initially dubbed IPH6101/SAR443579, is an NKp46-based NK cell engager developed with Innate’s proprietary multispecific antibody format.
As part of the collaboration, Sanofi will be responsible for all future development, manufacturing and commercialization of the program.
Innate said that the candidate has shown anti-tumor activity in pre-clinical primate models, which led to its selection. The company’s technology simultaneously targets two activating receptors, NKp46 and CD16, on NK cells, as well as a tumor antigen on cancer cells.
“We believe IPH6101/SAR443579 is the first NKp46-based NK cell engager to start development, demonstrating the next wave of scientific innovation at Innate,” Innate CSO Eric Vivier said in a statement.
Sanofi and Innate entered into their collaboration in January 2016. The pair are working on an additional ongoing research program. — Max Gelman
Tonix Pharmaceuticals joins forces with Mass General Hospital on kidney rejection
Tonix Pharmaceuticals has inked a second collaboration with Massachusetts General Hospital to work on its monoclonal antibody targeting CD40-ligand — this time focusing on organ rejection in kidney transplantation.
Anti-CD40-ligand has been shown to suppress rejection and facilitate “transplant tolerance,” according to Tatsuo Kawai, surgical director of the Living Donor Transplantation and Dialysis Access Program at MGH and professor of surgery at Harvard Medical School. Researchers in the program will look at Tonix’s TNX-1500 in kidney transplantation in “a variety of models including non-human primates,” the company said in a statement.
“There remains a significant need for new treatments to reduce the toxicity of current treatments by more selectively suppressing immune responses or inducing specific tolerance to the transplanted organ,” Kawai said.
Last year, the company teamed up with MGH to study the candidate in heart transplantation.
Despite the promise of anti-CD40-ligand therapy, first generation candidates were limited because they interacted with the FcγRII receptor, raising concerns over an increased risk of thrombosis, CEO Seth Lederman said in a statement. Second generation candidates made up for some of the problem, but ran into other issues like decreased efficacy.
“TNX-1500 is a third generation anti-CD40- ligand mAb that has been designed by protein engineering to target CD40-ligand therapeutically, while potentially decreasing FcγRII binding and the potential for thrombosis,” Lederman said. — Nicole DeFeudis