Tides Weekly | Global Peptide and Oligonucleotide Therapeutics News Roundup
ExoRNA
miRNA Nucleic Acid Drug Developer
RecentlyExoRNA Bio (Nanjing) Co., Ltd. ("ExoRNA Bio") has received approval from the U.S. Food and Drug Administration (FDA) for the Investigational New Drug (IND) application of its nucleic acid drug "ER2001 Injection." This drug is the first candidate product developed on ExoRNA Bio's third-generation in vivo self-assembling siRNA nucleic acid delivery technology platform and is being developed to treat Huntington's disease, which currently has no cure.Previously, the drug has also received FDA Orphan Drug Designation.
About ER2001 Injection
ER2001 Injection is the first product from ExoRNA Bio's In Vivo Self-Assembled Exosome Delivery (IVSAED) technology platform. ER2001 utilizes the body’s own organs and tissues as bioreactors to "process" the compound into its active form of siRNA, which is then assembled into endogenous exosomes targeting the central nervous system that are simultaneously produced by the "bioreactor." These exosomes are secreted and "transported" to neuronal cells, where they degrade the mRNA of mutant huntingtin protein (mHTT), thereby exerting the drug’s therapeutic effect.
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, 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.
On April 16, 2024, according to the official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration, the Investigational New Drug (IND) application for the Respiratory Syncytial Virus (RSV) mRNA vaccine developed by Hefei Afana Biotechnology Co., Ltd. ("Afana Bio" for short) was accepted.
Currently, treatment options for RSV infections are limited, and there is no specific treatment method available for RSV. The early development of RSV vaccines encountered clinical failures due to safety concerns and insufficient protection. At present, there are only two RSV vaccines available on the global market: one is GSK's RSVPreF3 vaccine (Arexvy), which received FDA approval on May 3, 2023, generating $1.55 billion in sales in 2023; the other is Pfizer’s RSV vaccine (Abrysvo), approved by the FDA on May 31, 2023, with $890 million in sales for 2023. In China, no RSV vaccines have been approved for marketing yet, and both globally marketed RSV vaccines are recombinant protein subunit vaccines.Vaccine.
About Hefei Afana Biotechnology Co., Ltd.
Hefei Afana Biotechnology Co., Ltd. has an independent and well-equipped research and development center and production workshop, with a GMP production base capable of producing 100 million doses of formulations annually. Meanwhile, the company’s co-founder, Professor Wang Yucai from the University of Science and Technology of China, has been awarded funding from the National Science Fund for Distinguished Young Scholars, the High-Level Overseas Talent Fund, and the CAS Hundred Talents Program. The team has been engaged in the research of nucleic acid drug delivery systems for over 20 years, holding a leading position in both academia and the industry.
On April 17, 2024, according to the official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration, the Investigational New Drug (IND) application for the Respiratory Syncytial Virus (RSV) mRNA vaccine developed by CSPC JuStone Biopharmaceuticals Co., Ltd. ("CSPC JuStone") was accepted.
Respiratory Syncytial Virus (RSV) is a very common and contagious respiratory infection, and a leading cause of bronchiolitis and pneumonia, affecting millions worldwide each year.Affecting over 64 million people in China. According to WHO, the mortality rate for RSV infections in children under one year old is as high as 6.7%, second only to malaria.
About CSPC
Shijiazhuang Pharmaceutical Group Co., Ltd. was established in 1997. Through innovative participation worldwide, Shijiazhuang Pharmaceutical Group continuously provides better innovative outcomes for human health. It has built a diversified product system, focusing on three major sectors: finished drugs, active pharmaceutical ingredients, and functional foods. The group has also actively contributed to the prevention and treatment of the coronavirus. Its mRNA COVID-19 vaccine is the first self-developed Chinese vaccine to receive emergency use authorization in China, with two other globally novel therapeutic drugs approved for clinical trials.
April 2024On the 17th, Innorna, a clinical-stage biopharmaceutical company focusing on lipid nanoparticle (LNP) delivery technology and the development of innovative RNA therapies, announced that its investigational broad-spectrum COVID mRNA vaccine IN002.5.1 has received Phase II clinical trial approval from the U.S. Food and Drug Administration (FDA).
IN002.5.1 is a broad-spectrum protective COVID-19 mRNA vaccine developed by ExoRNA Bio based on its proprietary mRNA-LNP technology platform, utilizing ExoRNA Bio's self-developed LNP delivery system. IN002.5.1 received IND approval from the U.S. FDA in February 2023 and has completed overseas Phase I clinical trials with positive results. This Phase I clinical trial used Moderna’s mRNA COVID-19 vaccine Spikevax (mRNA-1273 and mRNA-1273.222) as a positive control to evaluate the safety, reactogenicity, and immunogenicity of this product at different dose levels (30 micrograms and 60 micrograms). Safety and immunogenicity results at 28 days post-vaccination showed that this product demonstrated overall good safety and tolerability. The incidence of local/systemic solicited adverse events and the levels of induced humoral and cellular immune responses were comparable to those of Moderna’s mRNA COVID-19 vaccine Spikevax, with some clinical indicators showing better results. No grade three or higher adverse events occurred during the study.Adverse reactions and serious adverse events.
About ExoRNA Bio
Strand Therapeutics, established in 2019, is committed to developing a world-leading LNP delivery technology platform and innovative RNA therapies to address unmet clinical needs. Strand Therapeutics has built a Diversity-Oriented Lipid Library (DOLL) containing more than 5,000 ionizable lipids, which can be applied to the development of various innovative therapies, including mRNA vaccines and drugs, in vivo gene-editing therapies, and in vivo cell therapies. Based on its proprietary mRNA and LNP technology platforms, Strand Therapeutics has developed multiple internal R&D pipelines in the fields of infectious disease vaccines, rare diseases, and cancer immunotherapy. At the same time, it has established collaborations with several biopharmaceutical companies both within and outside China to explore the broader therapeutic potential of this technology.
2On April 17, 2024, Eli Lilly announced positive results from the Phase III SURMOUNT-OSA clinical trial of its GLP-1R/GIPR dual agonist Tirzepatide for the treatment of moderate to severe obstructive sleep apnea (OSA) with obesity. Compared with placebo, Tirzepatide injection (10mg or 15mg) significantly reduced the Apnea-Hypopnea Index (AHI) in subjects, achieving the primary endpoint. AHI records the number of times per hour of sleep that a subject's breathing is restricted or airflow is completely blocked, and is used to assess the severity of OSA and the effectiveness of treatment outcomes.
Tirzepatide is currently the only GLP-1R/GIPR class drug approved for chronic weight management. It is commercialized under the brand name Zepbound in the United States and under the brand name Mounjaro in some global markets outside the U.S. The successful Phase III trial for the OSA indication means that Tirzepatide will open upAnother market beyond weight loss.
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 medical needs.
April 18, 2024Lilly and Regeneron have registered a Phase II clinical trial on Clinicaltrials.gov for the combination therapy of tirzepatide and leptin receptor (LEPR) antibody Mibavademab in treating obesity.
This Phase II clinical trial plans to enroll 360 obese patients, with tirzepatide monotherapy as the control.
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 more than a century ago by Colonel Eli Lilly in Indianapolis, Indiana, in 1876, the company's founder committed to producing high-quality medicines to meet genuine medical needs.
