TIDES Weekly Digest | Global Peptide and Oligonucleotide Therapeutics News (Sep 11–18)
Hepa Thera
Innovative Drug Developer in the Field of Hepatology
Hengrui Pharma
Innovative and High-Quality Pharmaceutical Developer
Argo
RNAi Drug Developer
Recently，Suzhou Hepa Thera Biotech Co., Ltd. (hereinafter referred to as "Hepa Thera") independently developed HT-101 injection has been included in the breakthrough therapy drugs for the treatment of chronic hepatitis B virus infection. This designation is based on the drug's significant antiviral activity and good safety profile demonstrated in early clinical trials, which is expected to provide patients with a better treatment option.
HT-101 is an innovative GalNAc-conjugated siRNA drug that achieves anti-HBV effects by specifically targeting the liver to silence HBV mRNA, blocking the synthesis of multiple viral proteins. It demonstrates significant clinical advantages. Phase 1b clinical trialResults showed that HBsAg levels continued to decline in all dose groups after two administrations, with some patients in the high-dose group achieving HBsAg clearance. Preliminary Phase 2 data further indicated that HT-101 outperformed other similar competing products both domestically and internationally in terms of onset time and S antigen clearance rate.
About Hepa Thera
Suzhou Hepa Thera Biotech Co., Ltd. was established in May 2021. It is a biotechnology company incubated by Fosun Pharma's New Drug Fund, focusing on the development of innovative drugs in the field of liver disease treatment.
2025Year September15Day，The official website of the Center for Drug Evaluation (CDE) under the National Medical Products Administration (NMPA) shows that Hengrui Pharma'sHRS-5635 InjectionProposed for inclusion in the breakthrough therapy designation for the treatment of chronic hepatitis B.
HRS-5635 is a new generation of liver-targeted siRNA drug for hepatitis B virus (HBV) independently developed by Hengrui Pharma. No similar products have been approved for marketing worldwide. Non-clinical efficacy studies show that HRS-5635 exhibits excellent antiviral activity against all HBV genotypes and can exert efficient and long-lasting antiviral effects in vivo. Non-clinical safety evaluation studies indicate that HRS-5635 has no off-target activity and is highly safe. Currently, HRS-5635 for the clinical study of chronic hepatitis B has progressed to Phase II clinical trials.
Functional cure, as the ideal treatment goal for chronic hepatitis B, aims to clear hepatitis B surface antigen (HBsAg) after a limited course of treatment, thereby significantly reducing the incidence of end-stage liver disease. However, existing treatments have limited efficacy in clearing HBsAg.The efficacy is limited, and there is an urgent need to develop more effective products or treatment regimens to further improve the functional cure rate in CHB patients.
AboutHengrui Pharma
Hengrui Pharma (Stock Code: 600276.SH, 01276.HK) was founded in 1970. It is an innovative international pharmaceutical company dedicated to the research, production, and promotion of high-quality drugs. Focusing on new drug development in areas such as oncology, metabolism and cardiovascular diseases, immunology and respiratory diseases, as well as neuroscience, it is one of the leading pharmaceutical companies with the strongest innovation capabilities in China.
2025Year September15DayArgo Biopharma Co., Ltd. (hereinafter referred to as "Argo Biopharma," "the Company") announced that its investigational small interfering RNA (siRNA) drug BW-40202 has received clinical trial approval from the National Medical Products Administration (NMPA) of China, granting permission to initiate a Phase II clinical study for paroxysmal nocturnal hemoglobinuria (PNH). The study is expected to commence in January 2026. BW-40202 is a drug targeting complement factor B (CFB) for the treatment of PNH and other complement-mediated diseases. BW-40202 is also being evaluated in clinical trials for IgA nephropathy (IgAN), with the company having already received NMPA approval in June 2025 to conduct a Phase I/IIa clinical trial for IgAN.
BW-40202 is an siRNA therapy targeting CFB mRNA. It inhibits the expression of CFB mRNA through the RNA interference (RNAi) mechanism, thereby reducing serum CFB protein levels and suppressing the activity of the complement alternative pathway (CAP). Preclinical studies have shown that BW-40202 hasIt exhibits excellent pharmacological activity, significantly and persistently reducing serum CFB protein levels, effectively inhibiting CAP activity over the long term, and demonstrating favorable safety characteristics.
AboutArgo
Argo is a clinical-stage biotechnology company dedicated to developing a new generationsiRNAMedicines provide better treatment options for patients worldwide.
2025Year September15Day，Focused on developing the next generationRNAiGlobal Biopharmaceutical Company—Daru Bio (Rona Therapeutics) announced that the company's independently developedRN3161Successfully submitted to the Australian Human Research Ethics Committee (HREC) Submit a clinical trial application.RN3161Is a targetedINHBETheGalNAc-siRNA, independently developed byGAIA™The platform is designed to provide replicable, sustainable, high-quality weight loss solutions for overweight and obese populations.
RN3161Based on Dariu's self-developedGAIA™Platform development, capable of achieving precise silencingINHBEReduce fat while maintaining lean body mass, thereby distinguishing itself from existing treatment methods. Its optimizationGalNAcThe combination of delivery design and chemical modification reduces the off-target risk, and in the monkey model, effects were observed after a single low-dose administration.INHBE mRNADownregulated expression exceeding90%; and has achieved leadership in efficacy and durability. Preclinical studies show,RN3161Can achieve gene silencing effects lasting over six months, with promising potentialReducing the dosing frequency to just 1–2 times per year significantly improves patient compliance and long-term management value.AsGAIA™The Fourth Clinical Candidate Released by the PlatformRN3161 once again demonstrated Dharma's continuous advancement in differentiated RNAi therapies from research and development to clinical stages.
AboutDaru Biopharma
Rona Therapeutics, a globally leading nucleic acid innovative drug platform company, focuses on the treatment of metabolic diseases and central nervous system degenerative diseases. Since its establishment in 2021, Rona Therapeutics has been committed to developing best-in-class siRNA drugs. Currently, significant progress has been made with multiple best-in-class siRNA therapeutic drugs either in clinical trials or at the stage of initiating Investigational New Drug (IND) applications, including PCSK9 siRNA for hypercholesterolemia and APOC3 siRNA for mixed hyperlipidemia.
202On September 15, Year 5, Novo Nordisk announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had approved an update to the label of Rybelsus (oral semaglutide) to reflect the cardiovascular benefits observed in the SOUL trial. Rybelsus has become the first and only orally administered glucagon-like peptide-1 receptor agonist (GLP-1 RA) approved in the EU for type 2 diabetes that has been proven to provide cardiovascular benefits.
This approval is based onStage IIIbResults of the SOUL study. The studyAimed to evaluate the cardiovascular outcome impact of Rybelsus on adult patients with type 2 diabetes who also have atherosclerotic cardiovascular disease (ASCVD) and/or chronic kidney disease (CKD). The results showed,Oral semaglutide (Rybelsus) reduced cardiovascular death, heart attack, and stroke by 14% in adults with type 2 diabetes at high cardiovascular risk compared to placebo.
The latest results of the SOUL study will be presented at the 2025 European Association for the Study of Diabetes (EASD) Annual Meeting (September 15-19). These results include a significant reduction in hospitalization rates associated with severe adverse events for oral semaglutide compared to placebo. Additionally, further outcomes from the SOUL study will be showcased at the same conference, demonstrating that the cardiovascular benefits of oral semaglutide are unaffected by participants' Body Mass Index (BMI) or weight.
Recently, Chongqing Chenan Biotech, a subsidiary of Zhifei Biological, has developedCA111 Injection, has received the clinical trial approval notice from the National Medical Products Administration, and will conduct clinical research in adult patients who are overweight or obese, marking the project's development entering the clinical validation stage.
CA111 Injection is a GIP/GLP-1 dual receptor agonist that works synergistically by activating both receptors.: GIP can stimulate insulin secretion and protect pancreatic β-cells in the presence of glucose; GLP-1 can control blood sugar, suppress appetite, and delay gastric emptying, thereby regulating blood glucose and weight.Compared with single-target similar drugs, the CA111 injection developed by ChenAn Biotech can effectively reduce the side effects of administration due to the synergistic and complementary effects of dual agonists.
As of now, only Eli Lilly's tirzepatide injection, a GIP/GLP-1 dual receptor agonist, has been approved for marketing in China, with no domestically produced products targeting the same receptors currently available.
2025Year September16Day，Lilly Announces Detailed Results from Phase 3 Clinical Trial ATTAIN-1. The study evaluated the safety and efficacy of the investigational oral GLP-1 receptor agonist orforglipron in adults who are obese or overweight with at least one weight-related comorbidity but without diabetes. At week 72, all three dose groups of orforglipron (6mg, 12mg, and 36mg) met the primary endpoint, achieving significant weight loss compared to the placebo group. Additionally, all three doses demonstrated clinically meaningful results across several key secondary endpoints compared to the placebo group, including weight reduction (≥10%, ≥15%, and ≥20%) and decreases in waist circumference.The study results were presented at the 2025 annual meeting of the European Association for the Study of Diabetes (EASD) and simultaneously published in *The NewThe New England Journal of Medicine.
AboutOrforglipron
Orforglipron is an investigational, once-daily oral small molecule (non-peptide) glucagon-like peptide-1 receptor agonist (GLP-1RA). The medication can be taken at any time of the day without dietary or hydration restrictions. It was discovered by Chugai Pharmaceutical Co., Ltd. and licensed to Eli Lilly and Company for development in 2018. Chugai Pharmaceutical and Eli Lilly jointly published the preclinical pharmacological data of this molecule.Currently, Eli Lilly is conducting a series of Phase 3 studies on orforglipron for the treatment of type 2 diabetes, as well as for weight management in overweight adults with obesity or at least one weight-related comorbidity.In addition, Eli Lilly is also studying the potential of orforglipron for treating obstructive sleep apnea and hypertension in adults with obesity.
About the ATTAIN-1 and ATTAIN Clinical Study Series
ATTAIN-1 (NCT05869903) is a 72-week randomized, double-blind, placebo-controlled Phase 3 study designed to compare the efficacy and safety of orforglipron 6mg, 12mg, and 36mg monotherapy versus placebo in overweight adults with obesity or at least one comorbidity (hypertension, dyslipidemia, obstructive sleep apnea, or cardiovascular disease) but without diabetes.
On September 16, 2025, BrightGene Bio-Medical (Suzhou) Co., Ltd. (hereinafter referred to as “the Company”) announced that its wholly-owned subsidiary, BrightGene Pharma (Suzhou) Co., Ltd. (hereinafter referred to as “BrightGene Pharma”), had recently received the “Drug Clinical Trial Approval Notice” issued by the National Medical Products Administration (hereinafter referred to as “NMPA”), approving BrightGene Pharma’s BGM0504 for clinical trials.Clinical trials were conducted in adult patients with overweight/obesity.
BGM0504 is a GLP-1 (glucagon-like peptide 1) and GIP (glucose-dependent insulinotropic polypeptide) receptor dual agonist independently developed by the company. It can activate the downstream pathways of GIP and GLP-1, producing biological effects such as blood glucose control, weight loss, and the treatment of non-alcoholic steatohepatitis (NASH), demonstrating therapeutic potential for various metabolic diseases.
BGM0504 Injection for Type 2 Diabetes and Weight Loss: Both Indications Currently in Phase III Clinical Trials in China; BGM0504 Tablets Are the Oral Formulation of BGM0504. As of the Date of This Announcement, No Similar Target Oral Formulations Have Been Approved for Marketing Worldwide.
AboutBori Pharma
BrightGene Bio-Medical Technology is an innovative pharmaceutical company participating in international competition, and a publicly listed company equipped with a full industrial chain product portfolio and core drug R&D technology platforms.
2025Year September17Day，Mabwell (688062.SH)Aditum Bio Fund 3, L.P. ("Aditum Bio") announced the establishment of Kalexo Bio, Inc. ("Kalexo Bio") and entered into a global exclusive licensing agreement for the dual-target siRNA innovative drug 2MW7141 in the cardiovascular field.The drug mainly targets lipid regulation in people with dyslipidemia and the prevention of high-risk cardiovascular events. Aditum Bio will provide funding to Kalexo Bio, which will collaborate with Mabwell to advance the development of 2MW7141.
According to the agreement, Mabwell Bio grants Kalexo Bio exclusive global rights for the development, manufacturing, and commercialization of 2MW7141. Mabwell Bio is entitled to receive up to 1 billion US dollars in upfront and milestone payments, as well as tiered royalties. This includes a one-time, non-refundable upfront payment and a near-term payment totaling 1,200.Million US dollars in cash. As part of the consideration, Mabwell will additionally acquire a portion of Kalexo Bio's equity under agreed conditions.
2MW7141 isMabwellA dual-target small nucleic acid drug independently developed and currently in the preclinical stage,Mainly targets lipid regulation in populations with dyslipidemia and the prevention of high-risk cardiovascular events. 2MW7141 demonstrates a clear synergistic regulatory effect, showing potent and long-lasting inhibition of the target gene in preclinical studies, with a low risk of off-target effects.
AboutMabwell
Mabwell (688062.SH) is an innovative biopharmaceutical company with a fully integrated industrial chain. Adhering to the vision of "turning innovation from a dream into reality," and fulfilling the mission of "exploring life, benefiting health," Mabwell provides patients with more effective and accessible biologic innovations through original research, addressing unmet clinical needs globally.
On September 17, 2025, Johnson & Johnson announced the latest data from the Phase III ICONIC-ADVANCE 1 and ICONIC-ADVANCE 2 studies, which evaluated the efficacy and safety of icotrokinra compared to placebo and deucravacitinib in patients with moderate to severe plaque psoriasis. These data will be presented at the 2025 European Academy of Dermatology and Venereology (EADV) Congress.
Icotrokinra is a first-in-class IL-23 receptor-targeted oral peptide designed to selectively block the IL-23 receptor, a key molecule driving inflammation in moderate-to-severe plaque psoriasis (PsO), ulcerative colitis, and other potential IL-23-mediated diseases. Icotrokinra binds to the IL-23 receptor with single-digit picomolar affinity and demonstrates potent, selective IL-23 signaling inhibition in human T cells.Activity. The drug was co-discovered by Protagonist and Johnson & Johnson and is being developed under a licensing and collaboration agreement between the two parties. From Phase II clinical trials onward, Johnson & Johnson holds the exclusive global development and commercialization rights for icotrokinra.
Johnson & Johnson has launched the Phase III ICONIC-ASCEND study, the world's first head-to-head study aimed at proving that the oral drug icotrokinra is superior to the injectable biologic ustekinumab in treating psoriasis, marking an important step forward in psoriasis research.
About Johnson & Johnson
Johnson & Johnson was founded in 1886 and is one of the most comprehensive healthcare product enterprises with a wide range of business operations globally, covering medical technology and innovative pharmaceuticals. Headquartered in New Brunswick, New Jersey, USA, Johnson & Johnson successfully completed the spin-off of its consumer health business in 2023 and announced a brand renewal. The company integrated its two major businesses—medical technology and innovative pharmaceuticals—under the Johnson & Johnson name, marking a new chapter in the century-long journey of Johnson & Johnson.