Tides Weekly | Global Peptide and Oligonucleotide Therapeutics News Roundup
QurAlis
Developer of New Therapies for Amyotrophic Lateral Sclerosis (ALS)
On May 31, 2024, Moderna announced that the FDA had approved the respiratory syncytial virus (RSV) vaccine mRESVIA (mRNA-1345) for marketing to prevent lower respiratory tract disease (LRTD) caused by RSV in individuals aged 60 years and older.
The approval was mainly based on the positive data from the Phase III ConquerRSV study. The results showed that the vaccine had a protective efficacy of 83.7% (95.88% CI 66.0%, 92.2%) against RSV-LRTD with two or more symptoms, and a protective efficacy of 82.4% against RSV-LRTD with three or more symptoms. During the median follow-up period of 8.6 months, the vaccine demonstrated sustained protection against RSV LRTD.
In terms of safety, no serious safety issues were reported in the Phase III study. The most common adverse reactions included injection site pain, fatigue, headache, myalgia, and arthralgia.
About Moderna
Over the more than 10 years since its establishment, Moderna has transformed from a research-stage company in the messenger RNA (mRNA) field into an enterprise with a diversified clinical portfolio of vaccines and therapeutic products across seven modalities, possessing an extensive intellectual property portfolio and integrated manufacturing facilities for rapid clinical and large-scale commercial production.
On June 3, 2024, QurAlis Corporation ("QurAlis") announced that it had reached an exclusive licensing agreement with Eli Lilly and Company. QurAlis will grant Lilly global rights to develop and commercialize QRL-204. In addition, the agreement includes a research and development (R&D) collaboration, under which both parties will utilize QurAlis' proprietary FlexASO™ splicing modulator platform to identify and develop additional UNC13A-targeted drug candidates.
QRL-204 is a potential first-in-class splice-switching antisense oligonucleotide (ASO) designed to restore function in amyotrophic lateral sclerosis.(ALS), Frontotemporal Dementia (FTD), and Other Neurodegenerative Diseases: The Function of UNC13A.
About QurAlis
QurAlis is a clinical-stage biotechnology company founded by a team of neurodegenerative biologists from Harvard Medical School and Harvard University. It is advancing antisense oligonucleotide and small molecule programs to develop breakthrough precision medicines for amyotrophic lateral sclerosis (ALS) and other genetically validated targets in neurodegenerative diseases.
On June 3, 2024, Structure Therapeutics announced positive results from the Phase IIa study of GSBR-1290, a small molecule GLP-1 receptor agonist for the treatment of obesity, as well as the PK studies for capsules and tablets.
About GSBR-1290
GSBR-1290 is an orally administered small molecule GLP-1 receptor agonist. The GLP-1 receptor is a validated drug target for the treatment of type 2 diabetes mellitus (T2DM) and obesity. Designed through the company’s structure-based drug discovery platform, GSBR-1290 acts as a biased G protein-coupled receptor (GPCR) agonist, selectively activating the G protein signaling pathway.
About Structure Therapeutics
Structure Therapeutics is a leading clinical-stage biopharmaceutical company focused on discovering and developing innovative oral treatments for chronic metabolic, cardiopulmonary diseases with significant unmet medical needs.
Recently, HDM1002 Tablets, an innovative oral small-molecule GLP-1 receptor agonist developed by Hangzhou Zhongmei Huadong Pharmaceutical Co., Ltd. ("Zhongmei Huadong"), a wholly-owned subsidiary of Huadong Medicine, completed the enrollment of all participants in its Phase II clinical trial in China for weight management in overweight or obese populations (hereinafter referred to as "weight loss indication"). The study, led by Professor Li Xiaoying from Zhongshan Hospital Affiliated with Fudan University, is being conducted across 18 centers in China and aims to evaluate the safety and efficacy of HDM1002 Tablets in adult subjects who are overweight or obese.
HDM1002 tablets are oral small-molecule GLP-1 receptor agonists. GLP-1 receptor agonist products are relatively safe, with the benefits of weight loss, blood sugar reduction, and cardiovascular advantages, making it a relatively mature, stable, and safe target. Focusing on the GLP-1 receptor target, Huadong...The pharmaceutical industry has built a comprehensive and differentiated product pipeline that combines globally innovative drugs and biosimilars, including oral and injectable formulations, with long-acting and multi-target properties.
CloseFor HDM1002
HDM1002 tablets are an innovative small molecule drug independently developed by Zhongmei Huadong, with global intellectual property rights. It is an orally active, potent, and highly selective full agonist of the GLP-1 receptor. Preclinical studies have shown that HDM1002 can effectively activate the GLP-1 receptor, induce the production of cyclic adenosine monophosphate (cAMP), and has potent effects in improving glucose tolerance, lowering blood sugar, and reducing weight, while also demonstrating good safety.
About Huadong Medicine Co., Ltd.
Huadong Medicine Co., Ltd. (Stock Code: 000963) was established in 1993, with its headquarters in Hangzhou, Zhejiang. It was listed on the Shenzhen Stock Exchange in December 1999. After nearly 30 years of development, the company's business covers the entire pharmaceutical industry chain, with four major business segments: pharmaceuticals manufacturing, pharmaceuticals commercial, medical aesthetics, and industrial microbiology. It has grown into a large comprehensive pharmaceutical listed company integrating pharmaceutical R&D, production, and distribution.
June 5, 2024Deeply Believe Biology (Innorna), a company focused on lipid nanoparticles (LNP) Delivery Technology and InnovationRNAClinical-stage biopharmaceutical company focused on therapy development announces the self-developed world's first bivalent Respiratory Syncytial Virus (RSV）mRNAVaccineIN006Clinical Trial Application (IND) has obtained the approval from the Center for Drug Evaluation of the National Medical Products Administration of China (CDE) tacit consent.
IN006 is a bivalent RSV preventive vaccine product developed by DeepTrust Bio based on its proprietary mRNA-LNP technology platform. It can cover both RSV-A and RSV-B subgroups and may offer better broad-spectrum protection compared to monovalent vaccines. IN006 received IND approval from the U.S. Food and Drug Administration (FDA) in January 2024.Receiving CDE's IND approval marks another significant milestone for IN006. IN006 is the first China-produced RSV vaccine product to obtain clinical trial approval in China, and it is also the first non-COVID mRNA preventive vaccine product to be approved for clinical trials domestically.
About QurAlis
DeepTrust Bio was founded in 2019 and is committed to developing a world-leading LNP delivery technology platform and innovative RNA therapies to address unmet clinical needs. DeepTrust Bio has established a Diversity-Oriented LNP Library (DOLL) containing more than 5,000 ionizable lipids, which can be applied to the development of various innovative therapies, including mRNA vaccines and drugs, in vivo gene editing therapies, and cell therapies.
On June 5, 2024, Telix Pharmaceuticals, a biopharmaceutical company focused on the field of radiopharmaceuticals (RDC), disclosed the terms of its initial public offering (IPO) and plans to list on NASDAQ. Telix intends to offer 17 million shares at $11.87 per share, with an IPO amount of approximately $202 million.
Telix has two approved radiopharmaceutical diagnostic products and two additional products pending approval. In addition, the company is developing radiotherapies, including two therapies for prostate cancer, one for renal cancer, and one for glioblastoma.Therapy. Among them, 177Lu-TLX591 (a prostate cancer candidate drug) is currently undergoing Phase III trials, with interim data expected to be disclosed in the first half of 2025.
About Telix Pharmaceuticals
Telix is a biopharmaceutical company focused on the development and commercialization of diagnostic and therapeutic radiopharmaceuticals and related medical devices. Headquartered in Melbourne, Australia, Telix operates internationally in the United States, Europe (Belgium and Switzerland), and Japan. Telix is developing a portfolio of products at clinical and commercial stages, aimed at addressing significant unmet medical needs in the fields of oncology and rare diseases.