Tides Weekly Recap: Global Peptide and Oligonucleotide Therapeutics News (June 3–12, 2024)
ExoRNA
miRNA Nucleic Acid Drug Developer
On June 3, 2024, the Center for Drug Evaluation of China's National Medical Products Administration approved ExoRNA Bio (Nanjing) Co., Ltd.'s ER2001 Injection for clinical trials. ER2001 Injection is classified as a Category 1 therapeutic biological product and is the first pipeline product developed based on ExoRNA Bio’s proprietary IVSA™ technology platform, intended for the treatment of Huntington's disease. Administered intravenously, ER2001 Injection undergoes in vivo self-assembly after processing in the liver, enabling targeted siRNA delivery to the central nervous system and thereby achieving therapeutic effects.
About Huntington's Disease
Huntington's disease is a rare and fatal neurodegenerative disorder caused by the abnormal expansion of CAG triplet codon repeats in the first exon of the huntingtin (HTT) gene located on the short arm of chromosome 4. The Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition) indicate that the prevalence of Huntington's disease worldwide is approximately 2.7/100,000.
About ExoRNA Bio
ExoRNA Bio focuses on the globally leading third-generation in vivo self-assembled exosome delivery RNA nucleic acid drug technology, which boasts unique advantages such as multi-organ targeting, the ability to cross the blood-brain barrier, high safety, and low cost, providing a one-stop solution to the current delivery challenges of RNA drugs.
June 7, 2024Boehringer Ingelheim (BI) announced breakthrough results from a subgroup analysis of the Phase II clinical trial of glucagon/glucagon-like peptide-1 (GLP-1) receptor dual agonist survodutide (BI 456906) for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). After 48 weeks of treatment, up to 64.5% of patients with stage 2 or 3 fibrosis (F2-F3) (moderate to advanced scarring) achieved fibrosis improvement without MASH worsening, compared to 25.9% in the placebo group.
This Phase II, double-blind, placebo-controlled study evaluated the safety of survodutide at three doses (2.4mg, 4.8mg, 6.0mg) for the treatment of MASH.Tolerability. The primary endpoint of the study is the proportion of participants who achieve histological improvement in MASH without worsening of fibrosis after 48 weeks of treatment.
About Survodutide
Survodutide is a GCGR/GLP-1R dual agonist administered once weekly via subcutaneous injection. It is jointly developed by Boehringer Ingelheim and Zealand Pharma and is part of Boehringer Ingelheim's research and development portfolio in the field of metabolic, cardiovascular, and renal diseases.
About Boehringer Ingelheim
Boehringer Ingelheim is committed to researching breakthrough therapies aimed at transforming lives and safeguarding the health of generations. As a global leading biopharmaceutical company driven by research and development, the company demonstrates its value through innovation in areas with highly unmet medical needs.
On June 8, 2024, Eli Lilly and Company announcedTirzepatideDetailed results of the Phase 2 clinical trial, SYNERGY-NASH, for the treatment of patients with Metabolic Dysfunction-Associated Steatohepatitis (MASH).Efficacy evaluation showed that the trial met its primary endpoint, with patients receiving 5 mg, 10 mg, and 15 mg over 52 weeks of treatment.TirzepatideAmong the treated patients, 51.8%, 62.8%, and 73.3% achieved MASH resolution without worsening of liver fibrosis, compared to 13.2% in the placebo group. The trial data were initially presented at this year's European Association for the Study of the Liver (EASL) annual meeting.And simultaneously published in the New England Journal of Medicine (NEJM).
About Tirzepatide
Tirzepatide is a once-weekly injectable GIPR and GLP-1R agonist. GLP-1 and GIP are two natural incretins, and studies have shown that GIP can reduce food intake and increase energy expenditure, thereby reducing body weight. When GIP is used in combination with a GLP-1 receptor agonist, it may have an impact on patients' blood glucose levels and body weight.Greater Impact.
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, in 1876, with the founder dedicated to producing high-quality medicines to meet real healthcare needs.
On June 12, 2024, Avidity Biosciences announced preliminary positive data from the Phase 1/2 clinical trial FORTITUDE for its investigational antibody-oligonucleotide conjugate (AOC) therapy delpacibart braxlosiran (del-brax, AOC 1020). The data showed that patients with facioscapulohumeral muscular dystrophy (FSHD) who received the therapy had a consistent downregulation of over 50% in genes regulated by DUX4 and demonstrated a trend toward improved muscle function. Based on these positive results, Avidity plans to accelerate the initiation of the registration cohort in the FORTITUDE study.
Del-brax is the first investigational AOC therapy aimed at treating the root cause of FSHD, consisting of a proprietary transferrin receptor 1 (TfR1)-targeting monoclonal antibody conjugated with an siRNA targeting DUX4 mRNA. FSHD is a rare genetic disorder characterized by lifelong, progressive loss of muscle function, significant pain, fatigue, and progressive disability. Currently, there are no approved therapies for FSHD.
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.
June 12, 2024Livzon PharmaceuticalUnder its subsidiary Xinbei River PharmaceuticalSemaglutideThe marketing application for the injection has been accepted by the Center for Drug Evaluation of the National Medical Products Administration.
Semaglutide Injection is a biosimilar independently developed by the company. The indication for this registration application is: for adult patients with type 2 diabetes whose blood glucose is inadequately controlled on metformin and/or sulfonylurea therapy in addition to diet and exercise, as well as for reducing cardiovascular risk in patients with type 2 diabetes.The risk of major adverse cardiovascular events (cardiovascular death, non-fatal myocardial infarction, or non-fatal stroke) in human patients.
AboutLivzon Pharmaceutical Group Inc.
Livzon Pharmaceutical Group Inc. was founded in January 1985. It is a comprehensive pharmaceutical group company integrating the research and development, production, and sales of pharmaceutical products. The company regards R&D innovation as the cornerstone of sustainable development, continuously focusing on new molecules and cutting-edge technologies in the global new drug R&D field. Based on clinical value and differentiated forward-looking strategies, it has laid out innovative drugs and high-barrier complex formulations, focusing on fields such as the digestive tract, assisted reproduction, mental health, and tumor immunology. This has resulted in a complete product cluster and a differentiated product pipeline covering the entire R&D cycle.