Shares of Disc Medicine Inc. (NASDAQ:IRON) were trading at $56.94, up $9.81, or 20%, after the firm disclosed positive feedback from its end-of-phase II meeting with the FDA, supporting the regulatory path forward for bitopertin in erythropoietic protoporphyria (EPP). Regulators agreed with Watertown, Mass.-based Disc on the sufficiency of a single, randomized, double-blind, placebo-controlled trial, as well as the primary endpoint of average monthly total time in sunlight without pain during the last month after six months of treatment. EPP is a rare, debilitating, and potentially life-threatening disease caused by a mutation that affects heme biosynthesis, resulting in the accumulation of toxic, photoactive protoporphyrin IX, also known as PPIX. Bitopertin selectively inhibits glycine transporter-1.
Atossa’s breast cancer drug shows promise as preventative option
Atossa Therapeutics Inc. moved a step closer in its efforts to develop a preventative approach to breast cancer, with top-line data from a phase II study showing (Z)-endoxifen significantly reduced mammographic breast density, while demonstrating a good tolerability profile. It’s the second recent data readout for the drug, a small-molecule estrogen receptor antagonist, which hit its primary endpoint in 20 women with ER-positive/HER2-negative breast cancer, as presented at the Breast Cancer Conference in San Francisco.
BioFuture 2024: Half the population’s health needs are underserved
While the size of the market is enormous, drug development and treatments for women’s health care still lags behind what is offered for men. There has been a renaissance in the past few years, however, led by investors and companies that have wrestled with determining exactly what encompasses women’s health and how to meet its challenges. At the BioFuture conference in New York, a panel of women agreed that, like with pediatric patients in the past few years, women also should have their own precision medicines and specifically focused investment opportunities. Even what has been defined as women’s health has changed, according to Stacey Seltzer, managing partner and founder of Pontiva Healthcare Partners, as the focus has shifted from only reproductive health to other areas of health needs for women.
Australia reimburses Vazkepa a decade after US approval
For the first time, Australians have access to CSL Inc.’s Vazkepa (icosapent ethyl/Vascepa) for managing cardiovascular disease more than a decade after the drug was first approved in the U.S. As previously reported by BioWorld, Vazkepa – also marketed as Vascepa – was first approved in the U.S. in 2012 as an add-on to statins for adults with severe triglyceride levels. The EMA approved the drug in 2021. It was approved in Australia in 2022, and Amarin Corp. plc licensed the drug to CSL to market in Australia and New Zealand. Vazkepa is now listed on Australia’s Pharmaceutical Benefits Scheme to reduce the risk of cardiovascular events in adult statin-treated patients with elevated triglycerides (≥1.7 mmol/L) and established cardiovascular disease, or diabetes, and at least one other cardiovascular risk factor.
Radiopharmaceuticals, oral small-molecules for Alzheimer’s
A year out from Leqembi’s (lecanemab; Biogen Inc./Eisai Co. Ltd.) approval for Alzheimer’s disease (AD), ongoing research, coupled with artificial intelligence, is advancing both radiopharmaceuticals and small-molecule drugs for AD diagnostics and treatment, speakers at the 2024 KoNECT-MOHW-MFDS conference said. At an Alzheimer’s disease session held at the conference in Seoul, South Korea Oct. 31, speakers convened to discuss the state of the field, which was undoubtedly impacted by the entry of Leqembi as the first U.S. FDA-approved amyloid-beta targeting drug for patients with AD-related mild cognitive impairment since July 2023. Eli Lilly and Co.’s donanemab gained FDA approval as Kisunla a year later, in July 2024, becoming the second beta-amyloid antagonist for adult AD patients with early symptomatic disease.
Gene editing is Trojan horse of cancer immunotherapy
Gene editing strategies, from epigenetic engineering to cell reprogramming and genetic vaccines, are accelerating the development of new therapies that awaken the immune system to treat cancer, as presented last month in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy. Some of these advances are taking advantage of the conditions of the tumor microenvironment, where cancer cells coexist with immune cells, microorganisms and blood vessels.