Shanghai-based Yingli Pharma has secured U.S. Food and Drug Administration (FDA) approval to proceed with a global Phase III registration trial for linperlisib, a novel PI3Kδ inhibitor targeting relapsed/refractory (R/R) peripheral T-cell lymphoma (PTCL). The regulatory clearance follows a successful Type B End-of-Phase II meeting where the company discussed the drug's development program and regulatory pathway.
The pivotal study is scheduled to commence in the second quarter of 2025 and will evaluate linperlisib against physicians' choice of standard of care in patients who have received one or more prior systemic therapies. The trial will include enrollment sites across the United States and other countries.
"This is a major milestone for linperlisib," said Michael Hui, Chairman and Chief Executive Officer of Yingli Pharma. "We are very excited that linperlisib has entered the global pivotal study stage with the agreement from FDA. We will continue our mission to address patient unmet clinical needs globally and to accelerate the linperlisib clinical development program to bring more treatment options for patients with R/R PTCL."
Clinical Evidence Supporting the Phase III Trial
Linperlisib has been studied in three clinical trials specifically for R/R PTCL across China, the United States, and Europe, with more than 165 patients treated to date. These studies have demonstrated impressive clinical outcomes, including high objective response rates, high complete response rates, and promising progression-free and overall survival metrics.
Notably, the drug has shown consistent efficacy and tolerability profiles across different patient populations, establishing a strong foundation for the upcoming global registration trial. Overall, more than 6,000 patients have received linperlisib in various clinical trials and post-market settings, demonstrating a consistent and well-tolerated safety profile.
Regulatory Milestones and Market Position
Linperlisib represents a significant achievement for Yingli Pharma as the company's first drug candidate. In November 2022, the compound received approval in China for the treatment of relapsed/refractory follicular lymphoma (FL). That same year, the FDA granted Orphan Drug Designations for linperlisib in three indications: follicular lymphoma, T-cell lymphoma, and chronic lymphocytic leukemia/small lymphocytic lymphoma.
The drug has also received Breakthrough Therapy status from China's National Medical Products Administration (NMPA), making Yingli Pharma the second pharmaceutical company in China and the first based in Shanghai to receive this prestigious recognition.
Mechanism of Action and Therapeutic Potential
As a potent oral small molecule inhibitor of the delta isoform of PI3 kinase (PI3Kδ), linperlisib represents a targeted approach to treating hematologic malignancies. PI3Kδ is predominantly expressed in leukocytes and plays a crucial role in B-cell and T-cell development and function. By selectively inhibiting this enzyme, linperlisib aims to disrupt signaling pathways critical for the survival and proliferation of malignant lymphocytes.
The drug's selectivity for the delta isoform potentially contributes to its differentiated safety profile compared to earlier-generation PI3K inhibitors that target multiple isoforms.
Addressing Unmet Needs in PTCL
Peripheral T-cell lymphoma represents a heterogeneous group of aggressive non-Hodgkin lymphomas with generally poor outcomes and limited treatment options, particularly for relapsed or refractory disease. Current standard therapies often yield suboptimal response rates and durability, highlighting the significant unmet need for novel therapeutic approaches.
The advancement of linperlisib into Phase III testing marks an important step toward potentially expanding the treatment armamentarium for patients with this challenging malignancy. If successful, the global registration trial could position linperlisib as an important new option for patients who have progressed on existing therapies.
About Yingli Pharma
Founded in 2011 and headquartered in the National Biomedical Industry Base of Shanghai Zhangjiang InnoPark, Yingli Pharma is a clinical-stage biopharmaceutical company focused on developing treatments for cancer, metabolic, and immune diseases. Beyond linperlisib, the company is advancing a diverse portfolio that includes panRAS and TGFβR1 oral small molecule inhibitors for oncology, as well as URAT1 and HIPK2 inhibitors for metabolic and immune conditions.
The company's progress with linperlisib demonstrates its commitment to addressing significant unmet medical needs through innovative approaches to drug development. The upcoming Phase III trial represents a critical step in Yingli Pharma's efforts to bring this promising therapy to patients globally.
