FDA grants clearance for phase 3 trial of Fertilo, an iPSC-based fertility treatment

Gameto's Fertilo investigational product made of ovarian support cells (OSCs) in a vial. Photo credit: Gameto

The FDA has cleared an investigational new drug (IND) application for Fertilo, according to an announcement from Gameto, a clinical-stage biotech company developing induced pluripotent stem cell (iPSC)-based therapies for women’s health. This clearance enables the launch of the first US-based phase 3 clinical trial for an iPSC-based therapy, marking a significant advancement in reproductive medicine.

Fertilo is Gameto’s ovarian support cell (OSC) technology designed to mature eggs outside the body using iPSC-derived cells. Unlike traditional methods, which require 10 to 14 days of high-dose hormonal stimulation, Fertilo uses engineered ovarian support cells to replicate the natural egg maturation process in a dish. This innovation reduces hormone injections by 80% and shortens treatment cycles to just 2-3 days, providing a less invasive and more comfortable experience for patients. The technology also lowers the risk of ovarian hyperstimulation syndrome and mitigates side effects associated with high-dose hormone treatments.

“FDA clearance to advance Fertilo into a phase 3 trial is a significant milestone for Gameto, and a landmark moment in iPSC technology,” said Dina Radenkovic, CEO and cofounder of Gameto. “We are proud to be pioneers in this field, with technology that has the potential to transform reproductive medicine. I’d like to express my gratitude to our team, whose work is focused on empowering women by offering a solution that is less invasive and improves the IVF journey. Fertilo is a solution for both men and women from all walks of life, offering millions of people new possibilities for family building at a time when declining birth rates present a growing global challenge.”

The phase 3 trial will evaluate the efficacy and safety of Fertilo in maturing healthy eggs outside the body, yielding embryos, and improving pregnancy rates at 12 weeks of pregnancy—the primary efficacy endpoint for women undergoing assisted reproductive technologies. This clearance follows the world’s first live human birth conceived using Fertilo technology, achieved at Santa Isabel Clinic in Lima, Peru, in December 2024.

Gameto plans to conduct the phase 3 clinical trial across 15 sites in the United States. The study will assess key outcomes, including embryo development, pregnancy rates, maternal health, and live birth rates as safety endpoints. Participants will undergo up to two days of hormonal injection stimulation, with up to two embryo transfers. The trial is open to couples struggling with infertility for at least one year, and participants who do not achieve pregnancy will receive a voucher for a second attempt. The phase 3 study will be a double-blind, randomized controlled trial to ensure rigorous data collection and analysis.

“Fertility clinics have been hoping for a solution that makes the journey towards parenthood shorter, safer, and more likely,” said Martin Varsavsky, cofounder and chairman of Gameto and founder of Prelude Fertility. “Fertilo’s three-day IVF and egg freezing is the answer.”

“Gameto’s technology platform opens entirely new possibilities for addressing reproductive health challenges through cell engineering-inspired innovation,” said Christian Kramme, PhD, chief scientific officer of Gameto. “With Fertilo, we’ve demonstrated the potential of iPSC-based therapies to redefine fertility care by targeting ovarian health at the cellular level. This milestone also highlights the power of iPSCs in regenerative medicine and our computational and cell engineering platform where we have broader applications including menopause and ovarian cancer.”

Gameto is pioneering the use of iPSC technology in fertility care, building on Shinya Yamanaka’s Nobel Prize-winning discovery of induced pluripotent stem cells, which revolutionized regenerative medicine. By becoming the first company to apply this technology in a phase 3 clinical trial, Gameto is setting new standards in fertility treatment and advancing the field of cell therapy.

In December 2024, Fertilo achieved a historic milestone with the world’s first live birth using its technology. By co-culturing immature eggs with Fertilo’s proprietary ovarian support cells, Gameto replicated the natural egg maturation process in a laboratory setting, offering a faster, safer, and more accessible fertility solution.

Gameto is actively working to expand the availability of Fertilo and has secured regulatory clearance in Australia, Japan, Argentina, Paraguay, Mexico, and Peru. The company also recently announced a strategic partnership with IVFAustralia, part of the Virtus Health group, to bring Fertilo to a wider patient population.

Reference:

Gameto. Gameto Announces FDA IND Clearance for Fertilo, the First iPSC-Based Therapy to Enter U.S. Phase 3 Clinical Trials. Buisnesswire. January 30, 2025. Accessed January 30, 2025. https://www.businesswire.com/news/home/20250130561032/en/Gameto-Announces-FDA-IND-Clearance-for-Fertilo-the-First-iPSC-Based-Therapy-to-Enter-U.S.-Phase-3-Clinical-Trials