TIDES Weekly | Global Peptide and Oligonucleotide Therapeutics News Roundup (Aug 26–30)

RecentlyThe European Commission (EC) has approved the marketing of its respiratory syncytial virus (RSV) mRNA vaccine, mRESVIA (mRNA-1345), for the protection of adults aged 60 and above from lower respiratory tract disease caused by RSV infection (RSV-LRTD). Moderna has currently submitted applications for marketing authorization of mRNA-1345 to multiple regulatory bodies worldwide.

The approval of mRESVIA is mainly based on the positive data from the Phase 3 clinical trial ConquerRSV, which was a global study conducted across 22 countries or regions with approximately 37,000 adults aged 60 years or older enrolled.

The key trial of the vaccine reached two major efficacy endpoints. Preliminary analysis with a median follow-up of 3.7 months found that the vaccine effectiveness against RSV-LRTD defined by two or more symptoms was 83.7% (95.88% CI: 66.0-92.2), and the vaccine effectiveness against RSV-LRTD defined by three or more symptoms was 82.4% (96.36% CI: 34.8-95.3). Detailed data were published in The New England Journal of Medicine.

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 vaccine and therapeutic product portfolio encompassing seven modalities. It possesses an extensive intellectual property portfolio and integrated manufacturing facilities that enable rapid clinical and large-scale commercial production. Moderna maintains alliances with a wide range of government and commercial partners both in China and internationally, which makes it possible to pursue groundbreaking science and achieve rapid manufacturing scale-up.

August 26, 2024Rigerna announced that its self-developed Class I innovative drug "RG002C0106 Injection," a small interfering RNA (siRNA) for treating complement-related diseases, has officially received approval from the Australian Human Research Ethics Committee (HREC) on August 22 to commence Phase I clinical trial research. This Phase I clinical study aims to evaluate the safety, tolerability, and pharmacokinetic characteristics of RG002C0106 Injection in healthy volunteers.

The complement system is an important component of the human innate immune system, playing an indispensable role in defending against pathogenic microorganisms, clearing immune complexes and apoptotic substances to maintain homeostasis. Abnormalities or excessive activation of the complement system may trigger inflammatory responses, leading to self-tissue damage and subsequent related diseases, including autoimmune diseases, inflammatory diseases, kidney diseases, blood disorders, and eye diseases, all of which present significant unmet clinical needs. Complement factor C3 (Complement 3, C3) is a core factor in the complement activation pathway, and the development of inhibitors targeting complement factor C3 can effectively suppress excessive complement activation.

RG002C0106 Injection is an siRNA drug developed by Rigerna based on its proprietary core technology platforms such as RIHOST®, RICMO®, and LICOD®, utilizing its self-developed second-generation N'-acetylgalactosamine (GalNAc) conjugation technology. It targets the complement factor C3 and represents Rigerna's first pipeline to enter clinical trials. With outstanding preclinical efficacy and safety data, RG002C0106 has the potential to become the best-in-class and among the world’s first marketed siRNA drugs targeting C3.

August 26, 2024Day,The official website of the Center for Drug Evaluation (CDE) under the China National Medical Products Administration shows that Novo Nordisk's new indication application for semaglutide injection has been formally accepted by the regulatory authority. It is speculated that the indication applied for this time is related to chronic kidney disease (CKD).

Semaglutide is a long-acting GLP-1 analog that shares 94% homology with the amino acid sequence of natural GLP-1 and acts as a GLP-1 receptor agonist. Semaglutide injections in doses of 0.25mg, 0.5mg, and 1.0mg were approved in China in April 2021 for the treatment of type 2 diabetes (brand name: NovoTide, Ozempic). In June 2024, the 2.4mg semaglutide injection (brand name: NovoGain, Wegovy) was approved in China for long-term weight management.

According to the official website of the China Drug Clinical Trial and Information Disclosure Platform, Novo Nordisk has also conducted several Phase 3 clinical studies on semaglutide injection, including: an international multicenter (including China) Phase 3 clinical study (FLOW study) to evaluate the effect of semaglutide on the progression of renal damage in patients with type 2 diabetes and chronic kidney disease; an international multicenter (including China) Phase 3 clinical study to assess the impact of semaglutide on functional capacity in patients with type 2 diabetes and peripheral artery disease; an international multicenter (including China) Phase 3 clinical study to evaluate the effect of semaglutide on non-cirrhotic non-alcoholic...The impact on subjects with steatohepatitis.

About Novo Nordisk

Novo Nordisk was founded in 1923 and is a global leading biopharmaceutical company headquartered in Copenhagen, the capital of Denmark. Our goal is to drive change to defeat diabetes, obesity, rare blood diseases, endocrine disorders, and other serious chronic conditions.

On August 27, 2024, Eli Lilly announced the expansion of the supply of Zepbound, the weight-loss version of tirzepatide, to meet strong clinical demand. The new prices for the 2.5mg and 5.0mg doses are $99.75 per vial and $137.25 per vial, respectively, corresponding to monthly costs of $399 and $549, with a price reduction of more than 50%.

About Tirzepatide

Tirzepatide is a dual agonist of glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptors, capable of simultaneously activating signaling pathways mediated by GLP-1 and GIP receptors. GIP and GLP-1 are natural incretin hormones that regulate blood glucose. Tirzepatide was approved by the U.S. FDA in May 2022.(Product name: Mounjaro), used in conjunction with controlled diet and exercise to improve blood sugar control in adult patients with type 2 diabetes. In November last year, tirzepatide received another approval.FDA Approval(Product name: Zepbound), used to help obese or overweight adult patients lose weight and maintain stable weight.Ding.

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, dedicated to improving human health through innovation. Founded by Colonel Eli Lilly in 1876 in Indiana, USA, the company has grown over more than a century with its founder committed to producing high-quality medicines to meet genuine healthcare needs.

Recently,BrightGene Bio-Medical Technology (688166.SH) announced the results of its independently developed GLP-1 (glucagon-like peptide 1) and GIP (glucose-dependent insulinotropic polypeptide) receptor dual agonist – BGM0504 injection in a Phase II clinical trial (CTR202232464) conducted in Chinese patients with type 2 diabetes, demonstrating excellent comprehensive metabolic benefits in terms of blood sugar reduction and weight loss.

The Phase II clinical trial of BGM0504 was a randomized, double-blind, placebo-controlled and active comparator parallel-group study with multiple ascending doses conducted in Chinese patients with type 2 diabetes. The active comparator was semaglutide injection 1.0 mg. The trial results showed that BGM0504 demonstrated superior efficacy in both glycemic control and weight loss compared to semaglutide, showcasing its exceptional therapeutic potential and market competitiveness in this head-to-head comparison. Additionally, the glycemic control efficacy of BGM0504 also surpassed the reported data for tirzepatide.According to.

BGM0504 is a GLP-1 (glucagon-like peptide 1) and GIP (glucose-dependent insulinotropic polypeptide) receptor dual agonist independently developed by BrightGene, classified as an innovative chemical drug with independent intellectual property protection that has not been marketed either within or outside China.At the current Phase II clinical trial stage, BGM0504 focuses on newly diagnosed and initially treated patients with type 2 diabetes in China, patients with poor blood glucose control despite treatment with metformin alone or metformin combined with sulfonylureas, as well as non-diabetic overweight or obese patients, offering a more targeted therapeutic strategy with significant clinical benefits.

AboutBorui Pharmaceuticals

BrightGene Bio-Medical (Suzhou) Co., Ltd. (Abbreviation: BrightGene; Stock Code: 688166) is an innovative pharmaceutical company participating in international competition. Driven by research and development, the company focuses on first-to-market generics, difficult-to-copy generics, specialty active pharmaceutical ingredients (APIs), complex formulations, and original new drugs. It continuously builds high technological barriers and gradually establishes a business system that integrates APIs with formulations, combines generics with innovative drugs, and balances the international market with the domestic market in China, aiming to meet unmet clinical needs of patients worldwide.

August 28, 2024DayNovartis announced positive results from the Phase III V-MONO study. The study aimed to evaluate the efficacy and safety of Leqvio (inclisiran) administered twice a year compared to placebo and ezetimibe in low- to moderate-risk patients with atherosclerotic cardiovascular disease (ASCVD) who had not received lipid-lowering therapy. The results showed that the study met its primary endpoint, with Leqvio reducing patients' low-density lipoprotein cholesterol (LDL-C) levels.

V-MONO is a 6-month randomized, double-blind, placebo-controlled, and active-controlled Phase III clinical study (n=350), divided into a Leqvio monotherapy group (n=174), an ezetimibe monotherapy group (n=89), and a placebo group (n=87). The primary endpoint of the study is the comparison of low-density lipoprotein cholesterol (LDL-C) levels in patients treated with Leqvio versus those in the placebo and ezetimibe groups on Day 150.DL-C) Percentage change from baseline.

About Novartis

Novartis is committed to reimagining medicine to improve people's quality of life and extend human lifespan. By leveraging our technological leadership in research and development, along with innovative accessibility initiatives, we deliver high-value medicines that alleviate the greatest disease burdens on society. In our quest for new drugs, we continuously innovate, maintaining a leading position in R&D investment within the global pharmaceutical industry. Novartis has approximately 106,000 employees from over 140 countries and regions worldwide, and nearly 800 million patients globally benefit from Novartis products.

August 29, 2024DaySuzhou Rigerna Therapeutics (hereinafter referred to as "Rigerna") has completed a new round of financing worth nearly 100 million yuan. This round of investment was led by Huage Capital, with participation from new and existing shareholders including Zhongke Chuangxing, BioCity Jinchuang, Huabo Instruments, JunTian Venture Capital, Dehou Investment, and Cao Jiaming. The proceeds from this financing will mainly be used to advance the clinical progress of Rigerna's small nucleic acid new drug pipeline, further expand the pre-clinical pipeline, and strengthen the talent team.

Rigerna has built technology platforms for chemical modification and conjugation of oligonucleotide drugs, extrahepatic chemical delivery, and hepatocyte delivery, focusing on major chronic diseases. Through independent innovation and collaboration, the company has developed multiple R&D pipelines covering metabolic cardiovascular diseases such as abnormal blood pressure, blood lipids, and blood sugar, as well as central nervous system diseases. The Lipisense project, the world’s first treatment for severe hypertriglyceridemia, has entered Phase I clinical trials; a project for long-acting hypertension treatment is about to enter clinical trials. Rigerna's successful completion of this round of financing will provide strong support for the company’s next phase of rapid development.

About Rigerna

Suzhou Rigerna Therapeutics Ltd. (Rigerna Therapeutics Ltd) is a biotechnology company co-founded by former senior executives of listed companies and overseas high-level talents introduced to China. Established in 2022, the company focuses on core technological innovations related to oligonucleotides and new drug development, assembling a high-level R&D team. Rigerna has built a small nucleic acid new drug pipeline in the fields of metabolism and cardiovascular-cerebrovascular diseases, immunity, and chronic inflammation, aiming to develop small nucleic acid drugs with potential Best-in-Class or First-in-Class qualities.