Nuosiland Announces Positive Top-Line Results from Phase III Clinical Trial of Gene Therapy NL003 for Ulcer Indication in Critical Limb Ischemia

Recently,Beijing Northland Biotechnology Co., Ltd. (hereinafter referred to as "Northland")The first pivotal trial (hereinafter referred to as the "Ulcer Trial") for the gene therapy drug "Recombinant Human Hepatocyte Growth Factor Naked Plasmid Injection" (code name NL003) in treating severe lower limb ischemic disease has reached an important milestone. The trial has been unblinded, and the primary endpoint met expectations.

Preliminary results showed that: In terms of the complete ulcer healing rate, the primary endpoint, the NL003 group was significantly superior to the placebo control group (p<0.0001); safety was good with no significant difference between the two groups, and no serious adverse reactions related to the drug were found.

"The Ulcer Trial" is a randomized, double-blind, placebo-controlled, parallel-group study led by Peking Union Medical College Hospital in Beijing, with clinical research conducted across 24 hospitals in China. The trial enrolled its first participant in August 2019 and completed the last enrollment in June 2023, with a total of 242 participants. All follow-ups were completed by December 2023. Following the completion of data cleaning, database lock, blind review meetings, and unblinding procedures, the CRO delivered the primary results to our company on February 1, 2024. Subsequently, comprehensive analysis of the trial data will be conducted. Northland will focus resources to actively advance the New Drug Application (NDA) process. Meanwhile, another pivotal trial of NL003 for the indication of ischemic rest pain is proceeding smoothly, with results expected to be announced in the third quarter of this year.

Chairman Xu Songshan of the company stated: This drug embodies 20 years of dedication and hard work since the establishment of the company. The achievement of the expected results in the first key trial is a cause for celebration. Behind the figure "0.0001" lies the perseverance and wisdom of all researchers, and it is the result of the joint efforts of all units participating in the clinical trials. The success of the NL003 clinical trial is not only a tremendous encouragement for Northland but also boosts confidence in the research and development of innovative drugs produced in China. Thanks to all employees who have stood by us, to all partners who have contributed to the clinical trials, and to everyone in society who has supported and cared about the development of the company.

Next, Northland will continue its efforts, while preparing for the NDA submission, to accelerate the planning of industrialization and commercialization in order to bring the drug to market as soon as possible and provide clinically accessible therapeutic drugs for patients with severe lower limb ischemic diseases.

NL003 is a Class 1 gene therapy biological product independently developed by Northland, supported by the national "Major New Drug Development" project, and used for treating severe lower limb ischemic diseases.

Lower extremity ischemic disease is the most common type of peripheral arterial disease (PAD) in clinical practice. It is caused by various factors leading to stenosis or occlusion of lower extremity arteries and insufficient blood perfusion, resulting in symptoms such as intermittent claudication, pain, ulcers, or gangrene. Based on the severity of ischemia, the Rutherford classification is clinically used to categorize lower extremity ischemic diseases from mild to severe into grades 0-6, with grades 4-6 classified as critical limb ischemia (CLI).

CLI is the most severe ischemic stage of lower extremity PAD progression, with clinical symptoms mainly characterized by leg and foot rest pain, ulcers, and gangrene. As the disease progresses, infections, ulcers, and gangrene gradually worsen, and severe cases may require amputation, imposing a significant burden on patients and society. According to Frost & Sullivan data, by 2021, the number of PAD patients in China had reached 51.872 million. The risk of developing peripheral artery disease increases with age. Influenced by factors such as China's aging population, the number of people suffering from peripheral artery disease is expected to continue rising in the future. It is projected that by 2025, the number of patients will increase to 56.5626 million, among which more than 5 million will be CLI patients.