Oli Pharma Submits IPO Prospectus Highlighting Oligonucleotide Industry Dynamics and Pipeline Advancements in October 2024

Oct 23, 2024 10:18 CST Updated 10:18

Ribo Life Science

Small Nucleic Acid Drug Developer

Innovation in China

01 Ribo Life Science

Shenji Changhua Collaborates with Mr. Cai Lei to Establish a Joint Laboratory, Aiding in ALS Drug Development

First Clinical Trial Application for Self-Developed Small Nucleic Acid Drug to Treat IgA Nephropathy Approved by the EU

On September 14, 2024, RBD7007, an siRNA drug independently developed by Ribo Life Science targeting C5, received EU clinical trial approval and will initiate its first-in-human clinical trial in Sweden.

C5, as one of the key components of the complement system, plays a role in innate immunity, participates in immune protection of the body, and modulates adaptive immune responses. The development and progression of various immune and complement-related diseases, including IgA nephropathy and other complement immune-related conditions, have been confirmed to be associated with abnormal activation of C5. However, approved antibody drugs targeting C5 have issues with short half-lives and limited duration of effectiveness.

RBD7007 is an siRNA drug independently developed by Ribo Life Science targeting C5, based on the RIBO-GalSTARTM liver-targeting technology platform.Inhibiting C5 protein expression and blocking complement pathway activation to achieve its therapeutic effect on complement-mediated diseases.Preclinical trial data show that RBD7007 has a lasting and potent inhibitory effect on the activation of the complement pathway, while also demonstrating good safety. It is expected to bring significant therapeutic benefits to patients with IgA nephropathy and other complement-mediated diseases.

02 Bibeit

Bebetter Pharmaceuticals' First siRNA Innovative Drug BEBT-507 Clinical Trial Application Accepted by CDE

On September 13, 2024, the Investigational New Drug (IND) application for "BEBT-507 Injection," a small nucleic acid (siRNA) innovative drug independently developed by Guangzhou Bebetter Pharmaceutical Co., Ltd. for the treatment of polycythemia vera, has been accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA).

BEBT-507 Injection is the first siRNA drug developed by Bebetter Pharmaceuticals based on its self-developed small nucleic acid drug R&D platform.Aiming to treat iron metabolism disorders such as polycythemia vera by regulating iron levels in the body.Preclinical study results showed that BEBT-507 can efficiently inhibit target activity over the long term, demonstrating significant and sustained efficacy as well as good safety in animal models.

03 Emma Life Science

CNS-Targeted siRNA Therapeutics via Intravenous Administration: ER2001's First Public FIH/IIT Study Progress Draws Industry Attention

From September 12 to 14, 2024, at the joint conference of EHDN (European Huntington's Disease Network) and Enroll-HD (Huntington's Disease Global Clinical Research and Observation Platform), Professor Pei Zhong from the First Affiliated Hospital of Sun Yat-sen University was invited to present the preliminary results of the IIT study on ER2001 injection.

ER2001 Injection is the world's first investigational siRNA drug administered intravenously, achieving targeted delivery to the central nervous system by in vivo self-assembly of exosome-encapsulated siRNA crossing the blood-brain barrier.In the report, Professor Pei Zhong detailed the drug mechanism of action of ER2001 injection.Data obtained from IIT studies show that the ER2001 product has good safety and tolerability. The overall clinical scale scores of 10 treated patients improved significantly, especially in terms of total motor score, mental state, and comprehensive improvement in cognitive function. The disease progression of all patients was controlled or improved during the clinical study period.

Previously, ER2001 injection had received Orphan Drug Designation (ODD) from the FDA in 2023, and obtained IND clinical trial implied permission from both the FDA and NMPA in the first half of 2024.

Global Hotspot

01 AstraZeneca

Over $7 Billion Introduced, AstraZeneca's Antisense Therapy Eplontersen Submitted for Market Approval in China

On September 29, the CDE website showed that AstraZeneca's Eplontersen Injection had been submitted for marketing authorization in China. Eplontersen is an antisense oligonucleotide (ASO) therapy that AstraZeneca acquired from Ionis through two collaborations valued at over $7 billion each. Based on the drug’s clinical development progress in China, the indication for this submission is speculated to be: for adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM).

Eplontersen (IONIS-TTR-LRx) is a ligand-conjugated antisense oligonucleotide (LICA) drug designed to reduce the production of serum TTR for the treatment of hereditary and non-hereditary ATTR. The drug was approved for marketing in the United States in December 2023.

02 Biogen

Biogen and Ionis Jointly Developed ALS ASO New Drug Approved for Marketing in China via Clinical Exemption Pathway

On October 8, 2024, the latest announcement by China's National Medical Products Administration (NMPA) revealed that Biogen's tofersen injection has been approved for import to treat adult amyotrophic lateral sclerosis (ALS, also known as "Lou Gehrig's disease") caused by SOD1 gene mutations.This is the world's first SOD1-ALS etiological treatment drug, approved for marketing in China through a clinical exemption pathway.

ALS is a rare, progressive, and fatal neurodegenerative disease that leads to the loss of motor neurons in the brain and spinal cord responsible for controlling voluntary muscle movements. Patients with ALS experience muscle weakness and atrophy, causing them to lose independence and gradually the ability to move, speak, eat, and breathe. The average life expectancy for ALS patients after symptom onset is 3 to 5 years.

Tofersen is an antisense oligonucleotide (ASO) drug that reduces SOD1 protein synthesis by binding to SOD1 mRNA and causing its degradation.

03 Novartis

Novartis' siRNA Chronic Disease Drug "Inclisiran Sodium Injection" Participates in National Medical Insurance Negotiation for the First Time

The "Inclisiran Sodium Injection" (Inclisiran, brand name: Leqvio) developed by Novartis has recently submitted an application to the National Healthcare Security Administration for participation in the 2024 national healthcare security negotiation. This is also...The world's first and currently only siRNA ultra-long-acting lipid-lowering drug (for chronic diseases), participating in the national medical insurance negotiation for the first time.

[Leke Wei] was first approved for marketing by the U.S. FDA in December 2020 and received approval from the China National Medical Products Administration in August 2023.

【Leke Wei】Leqvio is priced at $6,500 per year in Europe and the United States, slightly lower than PCSK9 antibodies; the first batch of prescriptions issued in Beijing are priced at 9,988 yuan per injection, with annual costs of approximately 20,000 to 30,000 yuan.Sales were $112 million in 2022 and surged to $355 million in 2023.。

04 Wave Life of Science

Wave Life ScienceAnnouncementWVE-006 Positive Progress in Clinical Phase 1b/2a

October 16, 2024Wave Life Sciences (WVE) announced positive validation data for WVE-006 in the ongoing Phase 1b/2a RestorAATion-2 study for Alpha-1 Antitrypsin Deficiency (AATD), achieving mechanism validation with Wave’s RNA editing platform.

WVE-006 is Wave's first RNA editing project, a GalNAc-conjugated, subcutaneously administered RNA editing compound. It aims to correct a single base in the mutant SERPINA1 mRNA by converting the mutated A to I, which the cell interprets as G. This approach can reduce the aggregation of AAT protein in the liver, restore functional wild-type AAT in circulation to protect the lungs (or liver) from protease damage, and maintain the physiological regulation of AAT protein.

About Ouli Bio

Suzhou Ouli Biopharmaceutical Technology Co., Ltd. is a professional medicinal nucleic acid CRDMO.The company provides customers with "one-stop" services including nucleic acid drug discovery, laboratory R&D, process and analytical development, CMC services, API production, and drug registration. The technical team of Oulibio comes from one of the earliest teams in China that developed nucleic acid drug production processes and conducted CMC research, possessing extensive experience in project development and product registration. Currently, the company has established four industry-leading core technology platforms: solid-phase nucleic acid synthesis, chemical modification and conjugation, process development and analysis, and CMC pharmaceutical research. It has also engaged in in-depth collaborations with multiple pharmaceutical and biotechnology companies both domestically and internationally.

In 2023, the company's 3,000-square-meter GMP-compliant pilot platform officially began offering services, focusing on helping clients address critical challenges such as small nucleic acid drug process scale-up and CMC pharmaceutical research. The company will continue to provide compliant, high-quality, reliable, and efficient services to help clients enhance R&D efficiency, expedite product registration and market entry, and jointly support the entire process of small nucleic acid drugs from preclinical to commercial production.

Contact Us

Email: order@olipharma.com

Official Website: www.olipharma.com

R&D Center: No. 168, Yuanfeng Road, Yushan Town, Kunshan City, Jiangsu Province

Production Base: Building C3, Phase I Factory, No. 999 Yishanhu Road, Guoxiang, Wuzhong District, Suzhou City, Jiangsu Province, China

Common Technology Platform: Room 202, 2nd Floor, Building 2, No. 21 Baoshen South Street, Beijing Daxing District Biomedical Industry Base