TIDES Weekly Digest | Global Peptide and Oligonucleotide Therapeutics News (Jan 1–9)
Arnatar
Antisense Technology Drug Developer
Sirius Therapeutics
Oligonucleotide Drug Developer
Recently,Arnatar Therapeutics' self-developed new chemical entity Class I new drug, ART101 Injection, has received clinical trial implied permission from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) (Acceptance No. CXHL2401113). The drug is intended for the treatment of hypertension.
About ART101 Injection
Disclosed preclinical and clinical studies have shown that oligonucleotide drugs targeting AGT can reduce the production of hepatic AGT by inhibiting liver AGT mRNA levels, thereby decreasing systemic AGT levels and achieving the purpose of lowering blood pressure. ART101 Injection, as a siRNA drug independently developed by Arnatar, achieves a blood pressure-lowering effect by targeting and inhibiting the expression of hepatic AGT mRNA, reducing AGT protein generation.
AboutArnatarArnatar
Arnatar was founded in 2022 by a global team of scientists in the field of nucleic acid drugs. It has built a breakthrough innovative nucleic acid drug platform and continues to explore novel mechanisms of nucleic acid-based drugs, covering multiple therapeutic areas. The company is leveraging innovative antisense nucleic acid technology (Antisense Technology) and utilizing RNA interference (RNAi) and antisense oligonucleotide (ASO) platforms to modulate the expression of target genes, thereby providing opportunities for the treatment of liver, extrahepatic organ diseases, as well as common and rare diseases.
Recently, Shanghai, China, and San Diego, USA: Sirius Therapeutics (SHANGHAI) Co., Ltd. ("Sirius Therapeutics"), announced that the clinical trial application (IND) for SRSD216 has been successfully accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China. SRSD216 is a next-generation siRNA therapy for the treatment of hyperlipoproteinemia(a).
About SRSD216 Injection
SRSD216 Injection is a novel double-stranded small interfering ribonucleic acid (siRNA). It specifically modulates the LPA gene, reducing hepatic Apo(a) production and lowering circulating Lp(a) levels. Preclinical in vivo studies have shown that a single dose reduced Lp(a) levels by nearly 100%, with effects lasting over six months, and no significant safety events were observed.
About Sirius Therapeutics
Sirius Therapeutics is a clinical-stage biotechnology company dedicated to advancing human health and well-being. Focused on the global development of next-generation nucleic acid therapies for cardiometabolic diseases, the company aims to become a leader in revolutionizing the prevention and treatment of chronic conditions. Its current pipeline includes SRSD107 for thromboembolic disorders and SRSD101 for dyslipidemia, both of which have entered the clinical development stage.
December 202431Day，Eden Announces the Launch of Semaglutide Chewable Gummies, the World's First Semaglutide Chewable Gummy. Eden aims to provide the benefits of GLP-1 drugs like semaglutide in a convenient, needle-free form and promote personalized medication for patients.
Currently, multiple single-target GLP-1 and dual-target GLP-1 drugs have been launched globally, all of which, except for Rybelsus (semaglutide tablets), are injectable formulations.
About Semaglutide
Semaglutide is a GLP-1 receptor agonist that stimulates insulin production, inhibits glucagon secretion, and reduces appetite and food intake. Semaglutide was initially developed as a treatment for type 2 diabetes.ApprovedLaunched (Brand Name: Ozempic), in view of its significant effect on weight loss, the FDA in June 2021ApprovalIt is used to treat ordinary obese patients (trade name: Wegovy), which is the first new drug approved by the US FDA for controlling common obesity or overweight since 2014. The drug was subsequently approved by the EU later in the same year for treating obesity indications.
On January 2, 2025, Eli Lilly China announced the official launch of Mounjaro® (tirzepatide injection) in China, covering two indications, namely:
Adult patients with type 2 diabetes mellitus (hereinafter referred to as T2DM) whose blood glucose is still poorly controlled despite receiving metformin and/or sulfonylurea treatment on the basis of diet control and exercise.
Based on controlling diet and increasing exercise, long-term weight management is suitable for adults with an initial Body Mass Index (BMI) meeting the following criteria: ≥28kg/m² (obesity), or ≥24kg/m² (overweight) accompanied by at least one weight-related comorbidity (e.g., hypertension, dyslipidemia, hyperglycemia, obstructive sleep apnea, cardiovascular disease, etc.).
The above indications were approved by the National Medical Products Administration in May and July 2024, respectively.
Mounjaro® is the world's first and currently only 1,17,18,19 approved once-weekly glucose-dependent insulinotropic polypeptide (GIP)/glucagon-like peptide-1 (GLP-1) receptor agonist for T2DM and long-term weight management. As a single-molecule peptide, Mounjaro® selectively binds to and activates GIP receptors and GLP-1 receptors, which are the natural targets of GIP and GLP-1. Mounjaro® promotes first-phase and second-phase insulin secretion in a glucose-dependent manner while reducing glucagon levels, enhancing insulin sensitivity, and delaying gastric emptying. Both GIP receptors and GLP-1 receptors are expressed in key areas of the brain that regulate appetite. Mounjaro® reduces food intake, body weight, and fat content by modulating appetite; additionally, Mounjaro® regulates lipid utilization.
About Eli Lilly and Company
Eli Lilly and Company is a globally leading pharmaceutical company engaged in the research, development, manufacturing, and marketing of medicines, committed to improving human health through innovation. Eli Lilly and Company was founded over a century ago by Colonel Eli Lilly in Indianapolis, Indiana, USA, with the founder dedicated to producing high-quality medicines to meet genuine medical needs.
January 20256Day，Arcturus Therapeutics Holdings Inc., a company focused on the development of vaccines for infectious diseases andArnatar Therapeutics, a leading messenger RNA drug company specializing in rare diseases of the liver and respiratory system, announces the launch of two Phase 2 multi-dose escalation studies for mRNA therapy programs targeting cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency.The drug administration work.
The first participants in two mRNA therapy projects have begun receiving dosing in December 2024, with interim Phase 2 data expected to be released in the first half of 2025.
About ARCT-032
ARCT-032 is an inhaled investigational mRNA therapeutic designed to express functional CFTR in the lungs of patients with cystic fibrosis (CF). ARCT-032 has received Orphan Drug Designation from the European Medicines Agency (EMA) and both Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for the treatment of cystic fibrosis. ARCT-032 leverages Arcturus' LUNAR® lipid-mediated aerosol delivery platform to deliver CFTR messenger RNA to the lungs. Lung disease is a leading cause of morbidity and mortality in patients with CF. Expressing a functional copy of CFTR mRNA in the lungs of CF patients has the potential to restore CFTR activity and mitigate downstream effects that contribute to the progression of lung disease. The ARCT-032 program is supported by preclinical data in rodents, ferrets, and non-human primates, as well as in vitro demonstration of restored CFTR expression and function in human bronchial epithelial cells.
About ARCT-810
ARCT-810 is an investigational mRNA therapeutic agent administered intravenously, designed to express a functionally normal OTC enzyme in the liver of patients with OTC deficiency. ARCT-810 has received orphan drug designation from the European Medicines Agency (EMA), including an approved Pediatric Investigation Plan (PIP), as well as orphan drug and Rare Pediatric Disease designations granted by the U.S. Food and Drug Administration (FDA) for the treatment of ornithine transcarbamylase deficiency. OTC is a key enzyme in the urea cycle that converts toxic ammonia into urea. Elevated ammonia levels can lead to metabolic crises, causing progressive and irreversible neurocognitive damage. A safe and effective mRNA therapeutic agent may restore functionally normal OTC enzyme activity in the liver, thereby eliminating the risk of future metabolic crises.
January 20257DayVIAZEN Biotechnology (Suzhou) Co., Ltd. ("VIAZEN" or "Visirna") announced that its investigational small nucleic acid Class 1 innovative drug, Plersiran Sodium Injection (VSA001), has been officially included in the priority review and approval process by the Center for Drug Evaluation of the National Medical Products Administration today. The drug is intended for the treatment of Familial Chylomicronemia Syndrome (FCS). Previously, Plersiran Sodium Injection was also included in the list of breakthrough therapy drugs by the Center for Drug Evaluation of the National Medical Products Administration.CS Indications: Currently, there are no approved drugs available on the market in China.
About Arnatar Sodium Injection
VSA001 (Pulesiran Sodium Injection) is a liver-targeted small interfering RNA (siRNA) drug that effectively and persistently silences the mRNA level of apolipoprotein C3 (APOC3) to reduce the expression of APOC3 protein. It then effectively lowers serum triglycerides (TG) and TG-rich lipoproteins (TRL) and their degradation remnants through both lipoprotein lipase (LPL)-dependent and -independent dual pathways.
About ViaGen
Viazen is a small nucleic acid drug therapy company based in China with a global outlook, aiming to build a biopharmaceutical enterprise with comprehensive capabilities in research, production, and commercialization. The company was founded in 2022 and has established a long-term strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR), an internationally leading small nucleic acid drug company.
January 20257Day，Alloy Therapeutic Announces Targeted Collaboration and Licensing Agreement with Sanofi for the Former’s Innovative and Proprietary AntiClastic Antisense Platform for Central Nervous System (CNS) Targets. Through this collaboration, Sanofi will leverage its neuroscience expertise to work with Alloy in utilizing the AntiClastic Antisense platform to deliver therapeutics to the brain, aiming to develop a new class of genetic medicines capable of crossing the blood-brain barrier.
Sanofi will pay Alloy up to $27.5 million in upfront licensing fees and near-term preclinical milestone payments. In addition, Alloy is eligible to receive over $400 million in discovery, development, and commercialization milestone payments, as well as tiered royalties on sales of any products resulting from this collaboration. This partnership highlights the shared commitment of both parties to advance innovative therapeutic drugs in the CNS field.
Sanofi will pay Alloy up to $27.5 million in upfront licensing fees and near-term preclinical milestone payments. In addition, Alloy is eligible to receive over $400 million in discovery, development, and commercialization milestone payments, as well as any products resulting from this collaboration.Layered royalties on product sales. This collaboration highlights the shared commitment of both parties to advance innovative therapeutic drugs in the CNS field.
AboutAlloy
Alloy is a biotechnology ecosystem company. Its launched AntiClastic™ ASO platform combines improvements in primary sequences with novel spatial conformations of nucleic acid drugs. The resulting AntiClastic ASO candidates exhibit enhanced potency, facilitate delivery to target RNA, minimize interactions with unintended RNA targets and inflammatory responses, thereby achieving a superior therapeutic index.
January 20258Day，VikingSirius Therapeutics, Inc. announced that it has initiated the Phase II clinical trial of VK2735, a dual agonist of glucagon-like peptide-1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors.Is an oral medication aimed at treating obesity and related metabolic diseases.
Phase 2 VENTURE Oral Dose Evaluation Trial is a 13-week randomized, double-blind, placebo-controlled multicenter trial., aimed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of VK2735 in weight reduction.
The trial will enroll approximately 280 adults classified as obese or overweight with weight-related complications. Participants will be randomly assigned to one of six dosage groups or a placebo group. The primary objective of the study is to determine the percentage change in body weight from baseline after 13 weeks of treatment, with secondary and exploratory endpoints including additional safety and efficacy measures.InIn the study, VK2735 demonstrated dose-dependent weight loss and good safety.Features.
About VK2735
VK2735 is a dual agonist targeting glucagon-like peptide-1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors, currently under development for the treatment of metabolic disorders such as obesity.
About Viking Therapeutics
Viking Therapeutics was founded in 2012, headquartered in California, USA, and went public on NASDAQ in 2015. It is a clinical-stage biopharmaceutical company focused on developing first-in-class/best-in-class therapies in the field of metabolic/endocrine diseases.
January 20259Day，Verdiva Bio, a biotechnology company developing innovative therapies for obesity and other cardiometabolic disorders, announced its launch with a $411 million oversubscribed Series A financing. Verdiva’s pipeline includes three drug candidates licensed from Arnatar, one of which is an oral GLP-1 agonist poised to enter Phase II clinical trials. This drug requires only once-weekly dosing, which Verdiva believes will differentiate it from competing products, as most similar oral drugs in development require daily administration. The other two candidates are a once-weekly oral amylin agonist and a long-acting subcutaneous amylin agonist.
About Xianweida Bio
Xianweida Bio is a clinical-stage biopharmaceutical company focused on researching and developing innovative therapies for metabolic diseases. Its R&D pipeline includes first-in-class and best-in-class drug candidates, such as the long-acting GLP-1 peptide Ecnoglutide Injection (marketing application stage), oral GLP-1 peptide Ecnoglutide (Phase I clinical trials), and the oral small-molecule GLP-1 receptor agonist XW014 (Phase I clinical trials).