In June 2023, Lipigon Pharmaceuticals and Leaderna Therapeutics embarked on a strategic partnership to develop and commercialize Lipigon’s drug candidate Lipisense^®, aimed at treating dyslipidemias such as severe hypertriglyceridemia (SHTG). This collaboration leverages the strengths of both companies to efficiently advance the clinical progress of Lipisense^®. Recently, Leaderna received approval to start a Phase I study in China.

As part of this licensing and development agreement, Leaderna obtained the rights to develop and commercialize Lipisense^® in the Greater China region. Under the terms of the agreement, Lipigon is entitled to milestone payments and royalties up to USD 91 million. To date, Lipigon has received an up-front payment of USD 1.5 million. The first milestone is expected to be reached in 2024.

Clinical Trials and Regulatory Approvals

Leaderna is set to commence a Phase I bridging study with healthy volunteers in China, marking the initial step in the development process. The National Medical Products Administration (NMPA) recently approved the study design, and the company expects to initiate the study in the second quarter of 2024.

As of February 2024, Lipigon received approval from the Swedish Medical Authorities to proceed with its Phase II study involving patients. This first in-patient study will help advance the global development of Lipisense^®.

“The entire application process, from the submission of documents to the formal approval for clinical trial by the NMPA, was completed in less than three months. We are eager to begin the upcoming Phase I clinical trial of Lipisense^® in healthy adult subjects in China, where we will evaluate its safety, tolerability, and pharmacokinetic characteristics following a single dose administration. This randomized, double-blind, placebo-controlled study marks a significant milestone in our product development. The safety and pharmacokinetic data from this Chinese cohort will be instrumental in supporting the project’s progression into global Phase II studies,” says Dr. Jinqiao Wan, CEO of Leaderna.

Future Development Plans and Collaboration

Both parties are committed to collaborating on future development to reduce costs and minimize risks. The next development step involves a larger, multinational Phase IIa/b study on both European and Chinese subjects, timed to coincide with the completion of the ongoing Chinese Phase I and Swedish Phase II studies. The clinical teams from both companies are working closely to design a study that meets the requirements of European, American, and Asian medical authorities.

“We are very pleased to be working with Leaderna on a Phase III enabling study. They have a highly competent team, with the necessary experience in both Chinese and international drug development. Designing a study that meets the approval of global authorities is complex, but with our combined expertise, I am confident in our success,” states Dr. Stefan K. Nilsson, CEO of Lipigon.

“This next phase will serve as a critical step in our clinical development strategy as we aim to further establish the safety profile of Lipisense^® across a diverse patient population with dyslipidemias, including SHTG, and to collect essential efficacy data. These findings will be pivotal in preparing for our late-stage clinical trials,” explains Dr. Jinqiao Wan.

“The strategic partnership between Leaderna and Lipigon is engineered to leverage our complementary expertise and resources. This collaborative approach is expected to significantly expedite the global development timeline for Lipisense^®, marking a promising advancement in our mission to address dyslipidemias more effectively,” concludes Dr. Jinqiao Wan.

About Lipisense^®

Lipisense^® is an RNA therapeutics drug candidate that prevents the cells from producing the disease-promoting target protein ANGPTL4 in the liver by destroying the protein-coding RNA before the target protein has been formed. Genetic data demonstrate that ANGPTL4 is an independent risk factor for both cardiovascular disease and type 2 diabetes.

Learn more about the Lipisense^® project here.

About Leaderna

Leaderna Therapeutics Ltd. (Leaderna), a spin-off of HitGen Inc. (682222. SH), was established in July 2022 to focus on pioneering research and development of synthetic oligonucleotide therapeutics. Leveraging its advanced siRNA design, modification, evaluation, and delivery platform, Leaderna has successfully established a robust pipeline targeting metabolism, cardiovascular diseases and inflammation.