Tides Weekly | Global Peptide and Oligonucleotide Therapeutics News Roundup

Recently, according to the announcement on the website of the National Medical Products Administration, two innovative hepatitis B drugs from Brii Biosciences, BRII-835 (small interfering RNA) and BRII-877 (hepatitis B-specific neutralizing antibody), are proposed to receive breakthrough therapy designation, further accelerating the pace of research towards a functional cure for hepatitis B. This follows the breakthrough therapy designation received by their BRII-179 (hepatitis B therapeutic vaccine) in November last year.

About Brii Biosciences

Brii Biosciences is a biotechnology company dedicated to developing innovative therapies for significant public health challenges that have substantial unmet patient needs, limited treatment options, and impose severe social stigma on patients. The company focuses on infectious diseases and central nervous system disorders, building a robust pipeline of innovative treatment solutions based on insights into and experiences with critical patient needs, while advancing the research of these differentiated therapies.

On May 9, 2024, according to the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration, BW-20507 Injection, a Class 1 new drug submitted by Hangzhou Argo Biopharma Co., Ltd., a subsidiary of Argo, received tacit approval from the CDE for clinical trials. The drug is intended to be developed for the treatment of chronic hepatitis B.

BW-20507 is a small interfering RNA (siRNA) drug independently developed by the company that targets the hepatitis B virus (HBV). On September 13, 2023, this therapy was reported to have initiated a Phase 1/2 clinical trial in Australia to evaluate its safety and tolerability in healthy subjects and patients with chronic hepatitis B virus infection.Sex, pharmacokinetics, and dose escalation studies.

About Argo

Argo Biopharma was founded in April 2021, focusing on the development of siRNA drugs and established by several scientists with extensive experience in siRNA drug development. The company is committed to developing a new generation of siRNA drugs to provide urgently needed and better treatment options for patients worldwide. It is reported that the Argo Biopharma team has many years of professional experience in all aspects of RNAi drug development, including nucleic acid sequence design, chemical modification, GalNAc delivery technology, extrahepatic tissue targeting delivery technology, oligonucleotide synthesis, and CMC. A complete nucleic acid drug development platform has already been established at Argo Biopharma.

On May 9, 2024, Flagship Pioneering ("Flagship") and its subsidiary Metaphore Biotechnologies ("Metaphore") jointly announced that Metaphore has reached a strategic collaboration with Novo Nordisk to co-develop up to two next-generation obesity treatments.

This collaboration falls under the broader strategic partnership between Novo Nordisk and Flagship Pioneering, which aims to jointly develop novel therapeutic solutions for cardiometabolic diseases and rare diseases. Pioneering Medicines, Flagship’s internal drug development and collaboration unit, and Novo Nordisk’s Bio Innovation Hub are co-leading this collaborative effort.Up to Two Next-Generation Weight Loss Therapies. Among them, Metaphore, Pioneering Medicines, and Novo Nordisk will jointly advance basic research and preclinical development, after which Novo Nordisk will move these projects into the clinical research stage.

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 diseases. To achieve this goal, we lead scientific breakthroughs, expand the accessibility of our medicines, and are committed to the prevention and ultimate cure of diseases. Novo Nordisk has approximately 47,000 employees across 80 countries and regions worldwide, providing products and services to more than 168 countries and regions globally.

Recently, Jiaxing Faber Newday Pharmaceutical Technology Co., Ltd. (hereinafter referred to as "Faber Newday") announced the completion of the first close of its Series A financing round, which amounted to approximately RMB 100 million. This round of financing was led by Junhao Capital, with participation from Caitong Capital and strategic investors. The proceeds will be used to advance clinical research and prepare for the commercial launch of its mature pipelines, upgrade and improve its R&D platform, and support the pre-clinical and clinical development of subsequent innovative pipeline products.

In April this year, the Phase I clinical trial of the radiopharmaceutical Technetium [99mTc]-H7ND Injection, independently developed by Farber Newsky, was officially launched.

The [99mTc]-H7ND Injection, independently developed by Faber Sky as a Class I innovative drug, is also the world's first single-photon radiopharmaceutical targeting FAP. This drug can specifically bind to FAP and highly accumulate in tumors with high FAP expression, enabling precise tumor diagnosis through single-photon emission computed tomography (SPECT/CT) imaging.

About Faber Genesis

Farber BioPharma was founded in 2016 as an innovative drug research and development company specializing in molecular imaging diagnostics and radiopharmaceutical therapy. The company owns the PharmadaX®Ⅰ High-Efficiency Target Molecule Screening and Site-Specific Radiolabeling Platform, as well as the globally leading PharmadaX®Ⅱ Platform, which combines proximal therapy with immunotherapy. Based on these platforms, Farber BioPharma has established a highly competitive and differentiated R&D pipeline, with products covering both diagnostic and therapeutic drugs.

On May 13, 2024, Avidity Biosciences registered the Phase III clinical trial of AOC 1001 on the Clinicaltrials.gov website.

TheThe Phase III clinical trial plans to enroll 150 DM1 patients, with an expected preliminary completion date of October 2026.

Del-desiran is the leading candidate therapy developed by Avidity using its Antibody Oligonucleotide Conjugates platform, aiming to address the root cause of DM1 by reducing the mRNA levels of the DMPK gene associated with the disease. It conjugates a proprietary monoclonal antibody that binds to transferrin receptor 1 (TfR1) with an siRNA targeting the DMPK gene. In preclinical studies, del-desiran successfully delivered siRNA to muscle cells, resulting in a sustained, dose-dependent reduction of DMPK mRNA levels across various muscles, including skeletal, cardiac, and smooth muscles. The U.S. FDA andThe European Medicines Agency (EMA) has granted it orphan drug designation, and the FDA has also awarded this therapy Fast Track designation.

About Avidity

Avidity's mission is to profoundly improve people's lives by providing novel RNA therapies — Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the RNA field with its proprietary AOC, which aims to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases that were previously unreachable with existing RNA therapies.

May 14, 2024Day, two studies presented at the European Congress on Obesity held in Italy showed,Novo NordiskWeight loss with Semaglutide (Wegovy) Can maintain meaningful weight loss and improve cardiovascular health for up to four years, regardless of the amount of weight lost.

The first study, published in the journal *Nature Medicine* on Monday, followed patients from Novo Nordisk's SELECT cardiovascular outcomes trial for 208 weeks and found that those treated with Wegovy were able to maintain their initial weight loss over that period.

Wegovy Patients Saw Continuous Weight Loss Over 65 Weeks and Were Able to Maintain the Weight Loss Without Rebound for Four Years. On Average, Wegovy Patients Lost 10.2% of Their Body Weight, While the Placebo Group Only Lost 1.5%. The Treatment Difference Between the Two Groups Was 8.7%, Which Was Statistically Significant.

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. To achieve this, we lead scientific breakthroughs, expand the accessibility of our medicines, and are committed to the prevention and ultimate cure of these diseases. Novo Nordisk has approximately 47,000 employees across 80 countries and regions worldwide, delivering products and services to more than 168 countries and regions globally.

On May 15, 2024, Rgenta Therapeutics announced that RAG-17, the first small nucleic acid drug independently developed by the company for central nervous system diseases, has received clinical trial approval from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration.

About RAG-17

RAG-17 is a double-stranded small interfering RNA (siRNA) targeting the SOD1 gene, which treats ALS patients caused by SOD1 mutations by reducing SOD1 protein expression. RAG-17 utilizes RA Capital's independently developed SCAD™ (Smart Chemical-Assisted Delivery) platform with independent intellectual property rights to achieve central nervous system delivery.Efficient delivery through the system. Preclinical efficacy studies have shown that treatment with RAG-17 significantly delays disease onset, extends animal survival time, and improves motor function.

About Rgenta

Ractigen Therapeutics is a platform-based new drug development company based in China and targeting the global market, committed to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is one of the few global small nucleic acid drug companies that simultaneously possess both intrahepatic and extrahepatic delivery capabilities, having developed multiple proprietary, internationally leading small nucleic acid drug delivery platform technologies, including SCAD™ and LiCO™.

On May 16, 2024, Hengrui Medicine announced that it had granted a royalty-bearing license for its proprietary GLP-1 product portfolio to Hercules CM Newco, Inc. (hereinafter referred to as "Hercules US"). Hercules US will obtain exclusive rights to develop, manufacture, and commercialize the GLP-1 product portfolio globally, excluding Greater China. As part of the consideration for the out-license transaction, Hengrui will acquire 19.9% equity in Hercules US and will receive licensing fees for the GLP-1 product portfolio from Hercules US.

In addition, according to the terms of the agreement, Hengrui will receive an upfront payment and near-term milestones totaling US$110 million, including a US$100 million upfront payment and a US$10 million near-term milestone payment upon completion of technology transfer.

Based on the clinical development progress of HRS-7535 and its first FDA approval for market launch, U.S.-based Hercules will pay Hengrui cumulative clinical development and regulatory milestone payments of up to 200 million USD.

GLP-1 Based ProductsBased on the actual annual net sales in the licensed territory, U.S.-based Hercules will pay Hengrui cumulative sales milestone payments of up to $5.725 billion.

About Hengrui Medicine

Hengrui Medicine was founded in 1970. It is an international pharmaceutical company engaged in the innovation, development, and promotion of medicines, focusing on new drug research and development in areas such as anti-tumor drugs, surgical medications, autoimmune diseases, metabolic disorders, and cardiovascular diseases.

May 16, 2024RocheAnnounces Dual GLP-1R/GIPR Agonist for the Treatment of Obesity and Type 2 DiabetesCT-388The Phase I clinical trial achieved positive results. The study found that, compared to placebo, healthy obese adult subjects who received a subcutaneous injection of CT-388 once weekly for 24 weeks experienced significant weight loss. The weight loss effect of CT-388 was clinically meaningful, with an average placebo-adjusted weight loss rate of 18.8% (p-value <0.001). At week 24, 100% of subjects treated with CT-388 lost >5% of their body weight, 85% lost >10%, 70% lost >15%, and 45% lost >20%.

About Roche

Founded in 1896 and headquartered in Basel, Switzerland, Roche has over 125 years of history. It is a global leader in biotechnology and in vitro diagnostics, as well as one of the leading companies in the industrial production of original research drugs. Roche's rapid international expansion is attributed to the entrepreneurial spirit of its founder, Fritz Hoffmann.。