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Roche decided to close two TIGIT studies. Roche has punted a Pompe disease gene therapy from its pipeline after weighing up the competitive landscape. The Swiss drugmaker made the change in a second-quarter update that also featured news of TIGIT trial closures and a commitment not to make “huge cuts” to the organization. Spark Therapeutics began enrolling patients in a phase 1/2 trial of SPK-3006 shortly after becoming part of Roche. At one point, Spark was aiming to enroll 30 people in the study. However, the company halted enrollment in 2022 and later revealed only four patients joined the study. Roche tossed the program out of its pipeline as part of its second-quarter clear-out.  “The decision to discontinue the trial is not due to safety concerns, it was made as part of a strategic portfolio prioritization within Spark,” a spokesperson for Roche told Fierce Biotech via email. “An established standard of care exists for Pompe disease, and while there are no currently approved gene therapies for Pompe disease, investigational treatments are being evaluated by different organizations.” Astellas and AskBio, a part of Bayer, have Pompe gene therapies in clinical trials. Enzyme replacement therapies such as Sanofi’s Lumizyme and Nexviazyme are the standard of care in the rare genetic disease. SPK-3006, also known as RG6359, was the most advanced new molecular entity culled this quarter. The Swiss drugmaker also defenestrated three phase 1 programs. Anti-CD25 Treg depleter RG6292 landed on the discard pile. Roche previously stopped a monotherapy clinical trial but continued to study RG6292 in combination with its checkpoint inhibitor Tecentriq. The Swiss drugmaker also stopped studying CD19xCD28 prospect RG6333 in combination with its approved bispecific Columvi in non-Hodgkin lymphoma as well as confirmed its previously disclosed retreat from a SHP2 inhibitor. Roche used a call with the media to share an update on the fallout from the failure of its TIGIT drug candidate in a phase 2/3 non-small cell lung cancer (NSCLC) trial. Based on the setback, the company has decided to close two other studies in NSCLC, namely the phase 3 SKYSCRAPER-15 trial and the phase 2 SKYSCRAPER-05 study. “The remaining tiragolumab studies have either already completed, are already fully enrolled or are almost fully enrolled. Those studies that are obviously already quite down the path, we're going to allow those to close out,” Roche Pharmaceuticals CEO Teresa Graham said. Graham also shared an update on Roche’s work on obesity, noting the positive data the company has reported on from assets acquired from Carmot Therapeutics in recent weeks and looking forward to the start of phase 2 trials of injectable and oral candidates in 2024 and 2025, respectively.  Roche CEO Thomas Schinecker fielded a question about the cost-cutting initiatives underway at some other drugmakers. Schinecker said Roche’s head count has remained stable and, while resources could be shifted from less to more promising areas, no big changes are incoming. “There will be no announcement of any big restructurings,” Schinecker said. “We don't believe in making huge cuts in the organization because we want our team to be focused and diligent on developing medicines, and any such distraction would not be something that we would want in the organization.”

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.