Multiple Class 1 New Drugs Advance in Clinical Trials Across Obesity, Oncology, and Pediatric Growth Disorders

　　【Pharmaceutical Network Product InformationOn February 2, several Class 1 new drugs developed by pharmaceutical companies in China achieved significant clinical progress, covering multiple therapeutic areas such as obesity and weight loss, relapsed or refractory hematologic malignancies, and pediatric idiopathic short stature. The advancement of these independently developed new drugs demonstrates the strong capabilities of pharmaceutical companies in China in the field of innovative drug research and development, offering new hope for patients.
 

In the field of obesity treatment, LDR2515 Injection, a Class 1 new drug developed by Leaderna Therapeutics Ltd., has been approved for clinical trials and is intended for weight management in adults who are obese or overweight.
 

Data shows that LDR2515 Injection is an siRNA drug targeting the INHBE gene. Preclinical studies indicate that LDR2515 has good safety, and subcutaneous injection can efficiently and continuously suppress INHBE expression in the liver. It is expected to achieve dosing once every six months to one year in clinical settings, offering a promising new treatment option for high-quality weight loss and metabolic health.
 

The INHBE gene is primarily expressed in the liver, and its encoded protein, Activin E, promotes lipid accumulation. Human genomic studies have shown that loss-of-function mutations in the INHBE gene can improve waist-to-hip ratio, reduce abdominal fat, enhance metabolic status, and lower the risk of cardiovascular diseases and type 2 diabetes. In obese mouse models, INHBE siRNA has been shown to induce sustained weight loss without affecting food intake. Furthermore, when used in combination with a GLP-1 receptor agonist, it enhances weight loss, mitigates muscle loss caused by the GLP-1 receptor agonist, and suppresses weight rebound after discontinuation of the GLP-1 receptor agonist.
 

In the field of hematological malignancy treatment, Jiangsu Ascentage Pharma Development Co., Ltd.'s novel drug APG-3288 tablets have been approved for clinical trials, intended for the development of treatment for relapsed/refractory hematological malignancies.
 

APG-3288 is the first novel, highly potent, and highly selective BTK degrader independently developed by Ascentage Pharma. This molecule promotes the formation of ternary complexes, subsequently leading to the proteasomal degradation of BTK. Unlike traditional BTK inhibitors, APG-3288 aims to function through degradation rather than inhibition, inducing rapid, potent, highly selective, and sustained degradation of both wild-type and various mutant forms of BTK that are resistant to existing BTK inhibitors. By blocking the BCR-BTK signaling pathway at its source, it overcomes BTK inhibitor resistance, offering a differentiated solution for BTK-targeted therapy. Preclinical study results show that, compared with other BTK degraders under development, APG-3288 demonstrates stronger BTK degradation ability, higher selectivity, and superior PK characteristics, showcasing significant potential.
 

In addition to the two aforementioned new drugs approved for clinical trials, Changchun High & New Technology Industries (Group) Co., Ltd. also announced good news. On the evening of February 2, the company issued an announcement stating that the clinical trial application for the registration of domestically produced drug GS3-007a Dry Suspension, developed by its holding subsidiary GenSci, had been accepted by the National Medical Products Administration (NMPA). GS3-007a Dry Suspension is an orally administered small-molecule growth hormone secretagogue independently developed by GenSci. It is registered as a Category 1 chemical drug and is intended for the treatment of Idiopathic Short Stature (ISS).
 

ISS is a common pediatric endocrine disorder, defined as a height below -2SD (standard deviation) of the reference values for the same age, gender, and ethnicity, excluding short stature caused by growth hormone deficiency, small-for-gestational-age, other endocrine or systemic diseases, chromosomal abnormalities, or skeletal dysplasia. The GS3-007a dry suspension, administered orally once daily, can stimulate the release of endogenous growth hormone, offering more treatment options for children with growth needs. Previously, the GS3-007a dry suspension was approved to initiate clinical trials for growth delay in children caused by endogenous growth hormone deficiency (PGHD), and the clinical trial is currently ongoing.
 

Multiple Class 1 new drugs have achieved new clinical progress, reflecting the continuous improvement of innovation and R&D capabilities of pharmaceutical companies in China, as well as the ongoing enhancement of the innovative pharmaceutical ecosystem in the country. In the future, with the smooth advancement of related clinical trials, these drugs are expected to benefit patients and help propel a leapfrog development in China’s pharmaceutical industry.
 

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