Today, a brief rundown of news from Sarepta Therapeutics and Sana Biotechnology, as well as updates from Bayer, Inovio and Unnatural Products that you may have missed.
Sarepta Therapeutics has begun evaluating new safeguards meant to reduce the risk of liver damage in certain people who receive Elevidys, its Duchenne muscular dystrophy gene therapy. Last year, two teenagers with Duchenne and who could no longer walk died from acute liver failure after treatment with Elevidys, leading the FDA to limit use to younger patients. Sarepta has contended that a regimen involving the immune suppressing drug sirolimus might help prevent that outcome and, on Monday, said it's begun enrolling patients in a new arm of an ongoing study to test that theory. About 25 patients will receive the regimen, which will include 14 days of sirolimus infusions prior to Elevidys treatment and continue for about 12 weeks afterwards. Sarepta will then check for incidence of acute liver injury.
Sana Biotechnology said Friday that an experimental cell therapy it’s developing continued to show evidence of helping a Type 1 diabetes patient produce insulin 14 months after receiving treatment. Sana previously shared four-week results from the first-in-human study indicating the treatment evaded detection from the immune system and increased levels of a protein associated with insulin production. The company revealed Friday that the levels of that protein are "comparable" to what was seen after six months and surpasses totals found at months 9 and 12. Sana intends to ask U.S. regulators this year for permission to start a Phase 1 trial for the therapy, a donor-derived treatment called UP421.
Bayer said Monday that its drug Kerendia has succeeded in another Phase 3 trial, this time in people with non-diabetic chronic kidney disease. According to Bayer, a regimen involving Kerendia and standard therapies was superior to those other drugs alone on a widely used marker evaluating kidney function. Bayer didn't provide details, but said the difference was "clinically meaningful" and that the data would be submitted to health regulators. Kerendia has already succeeded in four other Phase 3 trials and is approved to treat heart failure as well as kidney disease associated with Type 2 diabetes.
Unnatural Products, a developer of “macrocyclic” peptide drugs, has raised a $45 million Series B round. The new funding announced Monday was led by The Venture Collective and involved Argenx and Merck & Co.’s Global Health Innovation Fund, among other backers. Unnatural Products is aiming its macrocycle drugs, which combine the strengths of small molecules and targeting antibodies, at historically tough-to-reach targets. It formed a potentially $1.7 billion alliance with Novartis last month.
Inovio revealed in an earnings release Friday it has "eliminated roles" not "directly" involved in advancing a therapy called INO-3107 that it's developing for recurrent respiratory papillomatosis, a rare condition causing the growth of viral warts in the larynx. Inovio is seeking an accelerated approval in the U.S., and while regulators have accepted a marketing application and set a decision deadline for Oct. 30, they've also warned that the company hasn't "provided adequate information" to justify the program's eligibility for a speedy clearance. The FDA has agreed to meet with Inovio, which, in the meantime, has trimmed spending to focus resources on INO-3107. The company had 112 full-time employees as of March 11, according to a regulatory filing.
