Alglucosidase Alfa

Successful first-in-human study shows YCT-529 is safe SAN DIEGO--(BUSINESS WIRE)-- 2024 BIO International Convention—YourChoice Therapeutics, Inc., pioneering hormone-free family planning products, has completed its first-in-human clinical study for YCT-529, designed to be the first hormone-free birth control pill for men. YourChoice Therapeutics CEO Akash Bakshi will present the results of the phase 1a study at the 2024 BIO International Convention on June 4 at 10:45 PDT. “We’re one step closer to delivering the world’s first male birth control pill,” said YourChoice Therapeutics chief science officer Nadja Mannowetz. “While the road to FDA approval for any new drug candidate is long, we’re very pleased with the speed, efficiency, and enthusiasm for YCT-529’s first-in-human study. The findings are powerful validation that YCT-529 is safe and well tolerated in men and lays a favorable foundation for further studies.” Innovation in birth control has been stagnant since the female birth control pill (“the Pill”) debuted 65 years ago. Since then, all new birth control options, except for the copper IUD, have relied on dosing women with hormones to prevent pregnancy. Importantly, there have been no new options for men, reinforcing the view that pregnancy prevention is “a woman’s job.” A birth control pill for men that’s hormone-free is a transformative innovation for a very deserving healthcare segment. In YCT-529’s phase 1a study, researchers studied doses up to 180 mgs, finding that all were safe and well tolerated. The next study will investigate multiple ascending doses (MAD) in a larger participant pool. YCT-529’s preclinical studies showed it was 99% effective and 100% reversible with no side effects. “The Pill turns 65 next year, marking more than a half-century of hormonal birth control with no new options for men to reduce the hormonal burden on their partners,” said Stewart McCallum, MD FACS, chief medical Officer for YourChoice Therapeutics, Inc. “This lack of modernization would be unacceptable in any area of healthcare and motivates us to advance YCT-529, which is on track to be the first hormone-free male birth control pill. The success of our phase 1a study is an important milestone on the path to delivering innovation in birth control that will benefit men and women alike.” YCT-529 is a retinoic acid receptor-alpha (RAR-alpha) inhibitor, which prevents sperm production by blocking access to vitamin A in the testes. Researchers discovered this mechanism of action in the 1930s after discovering that depriving mice, rats, and monkeys of vitamin A caused infertility. Since that time, decades of published studies have validated the pathway. “Women’s narrowing reproductive rights offer potent fuel to the fire driving male contraceptive candidates,” said YCT-529 Developer Gunda Georg, regents professor at the University of Minnesota’s College of Pharmacy and founding director of the Institutes for Therapeutics Discovery and Development. “Numerous published studies and surveys show that the world wants and needs more male contraceptive options. We also can’t deny that women are tired of bearing the burden of hormones. I’m very pleased by the speed and efficiency of YCT-529’s first-in-human study and feel confident that future studies will demonstrate similar success. Society is ready for a male contraceptive, and we applaud the organizations that recognize this and support our efforts to deliver the first hormone-free male birth control pill.” Quotient Sciences, based in the UK, conducted the phase 1a study, which began in December 2024. About YCT-529 Researchers have understood for decades that vitamin A is essential for male fertility. YCT-529 is a retinoic acid receptor-alpha (RAR-a) inhibitor that prevents production of sperm cells in the testes as well as their release by blocking RAR-alpha (one of three nuclear receptors that bind retinoic acid, a form of vitamin A). Preclinical studies have shown that YCT-529 is 99% effective at preventing mouse pregnancies and 100% reversible. YourChoice Therapeutics developed YCT-529 in collaboration with Dr. Gunda Georg, a globally recognized medicinal chemist and awardee of one of the largest NIH-funded Contraceptive Centers. The Male Contraceptive Initiative (MCI) has been committed to funding the early research and YCT-529’s phase one clinical trial in men. More information about the trial can be found at clinicaltrials.gov, NCT06094283. About YourChoice Therapeutics YourChoice Therapeutics is a clinical-stage biopharmaceutical company developing the first hormone-free birth control pill for men. Based in San Francisco, the company is advancing the only male pill in human studies. Preclinical studies showed YCT-529 performed as effectively or better than the female birth control pill. Future programs will apply the company’s patented therapeutic platform to develop hormone-free family planning products for women. Established in 2018, the company received a $15M Series A investment in 2022. For more information, visit and follow the company on LinkedIn.

Maze Therapeutics looks to have found another partner for its investigational Pompe disease drug MZE001 after a deal last year with Sanofi fell foul of antitrust regulators. In an agreement announced on Friday, Maze will receive $150 million upfront from Shionogi and is eligible for additional milestones payments and tiered sales royalties.Sanofi abandoned its plans to license the Maze molecule in December under a deal potentially worth $750 million, including $150 million upfront, after the US Federal Trade Commission (FTC) raised competition concerns. The French drugmaker already markets the Pompe disease enzyme replacement therapies Lumizyme (alglucosidase alfa), also known as Myozyme, and Nexviazyme (avalglucosidase alfa), which are both administered via biweekly intravenous infusions.Meanwhile, Shionogi is looking to strengthen its rare diseases portfolio as part of its revised medium-term business strategy. “The science behind MZE001 is differentiated and promising,” with the potential to serve as “both mono and add-on therapy to enzyme replacement therapies,” remarked Shionogi CEO Isao Teshirogi. MZE001, an oral inhibitor of GYS1, is designed to restrict glycogen accumulation in patients with Pompe disease. The drug’s Phase I data released in February last year confirmed target engagement, prompting Maze to finalise mid-stage development. As part of the deal for MZE001, Shionogi will also have global rights to related programmes and intellectual property.