Sirnaomics Receives Safe to Proceed Letter for U.S. FDA Investigational New Drug Application for Systemic RNAi Therapeutic STP707 in Primary Sclerosing Cholangitis (PSC)

Author: Sirnaomics Ltd. / 2023-07-24 00:04 / Source: Sirnaomics Ltd.

Sirnaomics Receives Safe to Proceed Letter for U.S. FDA Investigational New Drug Application for Systemic RNAi Therapeutic STP707 in Primary Sclerosing Cholangitis (PSC)

HONG KONG,Feb. 16,2022 -- Sirnaomics Ltd. (the "Company" or "Sirnaomics",stock code: 2257.HK),a leading biopharmaceutical company in discovery and development of RNAi therapeutics,announced that they have received the "safe to proceed" letter from the U.S. Food and Drug Administration (FDA) for the Company's investigational new drug (IND) application for STP707,its lead siRNA (small interfering RNA) drug candidate. The Company anticipates initiating in the second quarter of 2022 a Phase I study to evaluate the safety,tolerability,and pharmacokinetics of STP707,administered intravenously in healthy volunteers.

STP707 takes advantage of a dual-targeted RNAi inhibitory property against TGF-β1 and COX-2 gene expressions in cells,with a proprietary intravenous administration formulation of PNP (HKP+H),targeting various types of liver cells including liver sinusoidal endothelial cells and Kupffer cells. These two types of cells are heavily involved in liver fibrosis pathology especially when both cells overexpress TGF-β1 and COX-2. A series of preclinical animal efficacy studies with intravenously delivered TGF-β1 and COX-2 siRNAs have shown target gene knockdown in liver with potential antifibrosis activity. Further preclinical pharm/tox studies with rodent and non-human primate models have provided a favorable safety profile of STP707 for advancing to a clinical test.

"With the FDA's safe to proceed letter of our IND for STP707,Sirnaomics will further study this promising treatment in patients with PSC,a rare disease with limited medical options," said Patrick Lu,Ph.D.,founder,Chairman of the Board,Executive Director,President and CEO of Sirnaomics. "It's a notable milestone as we continue to develop siRNA therapeutics for indications with significant unmet medical needs."

"Patients with PSC experience progressive fibrosis of the liver,and there are currently no approved therapies that effectively alter the natural history of the disease," explained Michael Molyneaux M.D.,Executive Director and Chief Medical Officer at Sirnaomics. "Unfortunately,this leads to the need for liver transplant in most patients. These patients could benefit significantly from non-surgical options for this rare disease. Our data suggest that STP707 drives robust response in preclinical models,and we expect that the upcoming Phase I clinical trial will allow us to gain further insights into the potential dosing and safety of this therapeutic candidate for the treatment of PSC."

For more information about Sirnaomics' clinical trials please visit the Company's website at www.sirnaomics.com.

About Sirnaomics

Sirnaomics is an RNA therapeutics biopharmaceutical company with product candidates in preclinical and clinical stages that focuses on the discovery and development of innovative drugs for indications with medical needs and large market opportunities. Sirnaomics is the first clinical-stage RNA therapeutics company to have a strong presence in both China and the United States,and also the first company to achieve positive Phase IIa clinical outcomes in oncology for an RNAi therapeutics for its core product,STP705. Learn more at www.sirnaomics.com.

CONTACT:

Michael Molyneaux,MD,MBA

Executive Director and Chief Medical Officer,Sirnaomics Ltd.

Email: MichaelMolyneaux@sirnaomics.com

Investor Relations:

Nigel Yip

Chief Financial Officer,China,Sirnaomics Ltd.

Email: NigelYip@sirnaomics.com

US Media Contact:

Mark Corbae

Tel: +1 203 682 8288

Email: Mark.Corbae@westwicke.com