2024-07-24
Recently, BOCG's portfolio company, Innorna, announces that China National Medical Products Administration (NMPA) has granted a Drug Clinical Trial Approval Notice for its self-developed Herpes Zoster (HZ) mRNA vaccine IN001; meanwhile, U.S. FDA orphan drug designation granted to IN022 for the Treatment of Homocystinuria after it was granted rare pediatric disease designatio.
The following news is from Innorna:
July 23, Hong Kong, Shenzhen, Nanjing - Innorna, a clinical-stage biotech company pioneering its proprietary lipid nanoparticle (LNP) technology to develop novel RNA therapeutics, today announced that China National Medical Products Administration (NMPA) has granted a Drug Clinical Trial Approval Notice for its self-developed Herpes Zoster (HZ) mRNA vaccine IN001.
IN001 is the first mRNA-based HZ vaccine approved by the NMPA for clinical trials in China. This milestone follows the Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA), making IN001 the third HZ mRNA vaccine approved for clinical trials after Moderna and Pfizer/BioNTech. The Phase I clinical study for IN001, involving healthy volunteers in New Zealand and the U.S. (NCT06375512), is currently in progress. The clinical trial in China is expected to initiate shortly, accelerating the availability of IN001 to the global population.
This approval represents a significant step towards bringing a potentially better and safer HZ vaccine to the world. Innorna remains committed to advancing innovative therapies and improving global health.
About Herpes Zoster (HZ) and IN001
Herpes Zoster (HZ) is a common infectious skin disease caused by reactivation of Varicella-Zoster Virus (VZV) after a prior primary infection manifesting as chickenpox. The clinical symptoms of HZ primarily include clustered skin lesions and neuropathic pain, which can complicate into chronic pain (i.e., postherpetic neuralgia, PHN) and other severe disorders, significantly affecting the quality of life of the patients. HZ can affect individuals of all ages, but the incidence rate significantly increases with age, especially after 50. Although antiviral therapy and symptomatic treatment can alleviate discomfort and pain symptoms for HZ patients, they cannot significantly shorten the disease course or alleviate complications. HZ vaccination is currently the most effective measure for preventing HZ and its complications. However, the safety, effectiveness, and availability of existing HZ vaccines are crucial factors limiting their application. With the global aging population, the disease burden caused by HZ is escalating. Developing a next-generation HZ vaccine with equal or superior efficacy, better safety, and simplified production processes will help improve the accessibility and coverage of the HZ vaccine, addressing this critical global public health issue.
IN001 is an innovative HZ mRNA vaccine independently developed by Innorna. Its core technology includes the mRNA sequence that expresses the VZV glycoprotein E (gE) and Innorna’s unique LNP delivery system. Preclinical studies demonstrated that IN001 can effectively induce both humoral and cellular immune responses. Compared with Shingrix®’s literature-reported data, IN001 has a better safety profile and is expected to demonstrate lower adverse reactions in clinical studies. In a head-to-head preclinical immunogenicity study, IN001 induced higher levels of cellular immune responses than Shingrix® and demonstrated excellent immunological durability. The manufacturing process of IN001 is robust, easy to scale up, and key raw materials have been standardized, resulting in a stable supply chain and significant reduction in vaccine costs, ensuring robust vaccine production for both domestic and international markets.
The development of IN001 represents a promising breakthrough in HZ vaccine research, offering greater accessibility and vaccine coverage for this disease globally. We are committed to working with regulatory agencies and partners to advance IN001 into clinical development, expedite its approval, and bring this next-generation HZ vaccine to the public as soon as possible.
HONGKONG, SHENZHEN, NANJING, CHINA, July. 19, 2024— Innorna, a clinical-stage biotech company pioneering its proprietary lipid nanoparticle (LNP) technology to develop novel RNA therapeutics, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to its investigational therapy IN022, for the treatment of classic homocystinuria (HCU). IN022 has also been granted with Rare Pediatric Disease Designation (RPDD) on July 5, 2024. Having obtained both RPDD and ODD would greatly facilitate IN022 clinical development/ approval process, therefore bring this potential therapy to HCU patients quickly.
About HCU and IN022
HCU is a rare autosomal recessive inherited sulfur amino acid metabolism disorder primarily caused by a cystathionine beta-synthase deficiency (CBS). HCU patients have a broad spectrum of age (older than three years) on presentation, with multisystemic clinical complications, particularly skeletal and connective tissue defects, osteoporosis, dislocated optic lenses, learning difficulties, and developmental delay or thromboembolism. Currently, there is no approved therapeutic drug for this disease. IN022, a mRNA therapy utilizing Innorna's proprietary mRNA-LNP technology, is designed to address the root cause of HCU resulting from a deficiency in CBS. It is expected to restore the function of the CBS enzyme, thereby normalizing homocysteine metabolism and potentially ameliorating symptoms in HCU patients.
