[2022.1] PS-001 Receives US FDA IND Approval, Paving the Way for Human Clinical Trials

(January 28, 2022) Pharmasaga announced that its blockbuster novel drug for Type 2 Diabetes, PS-001, has passed review by the U.S. Food and Drug Administration (FDA). This approval allows the drug to proceed into a Phase I Single-Ascending Dose (SAD) clinical trial. The Company anticipates completing the Investigational New Drug (IND) application with the Taiwan Food and Drug Administration (TFDA) and commencing patient enrollment in the first half of this year.

Market and Unmet Medical Need

According to the 2019 report by the International Diabetes Federation (IDF), the global prevalence of diabetes has reached 9.3%, with 463 million people currently living with the disease. Type 2 Diabetes, which affects adults predominantly, accounts for approximately 90% of cases. The diabetic population is growing rapidly, having quadrupled over the past 30 years, and is projected to increase to 578 million by 2030 and 700 million by 2045. Including diabetes-related complications such as kidney failure, heart disease, and stroke, the disease causes 4.2 million deaths annually, accounting for 11.3% of total global deaths and ranking as the third leading cause of death worldwide.

Although many anti-diabetic drugs are currently available, these drugs can only control blood sugar and cannot cure diabetes, requiring patients to take medication for life. Furthermore, some patients continue to deteriorate, developing severe complications and potentially death. Due to these therapeutic limitations, Type 2 Diabetes remains an area of Unmet Medical Need. Therefore, the development of a novel drug with a new mechanism capable of curing diabetes is an urgent necessity. If PS-001 proves effective in humans, it will address this market gap, with a market potential estimated to be as high as 50 billion USD.

PS-001 Mechanism and Preclinical Efficacy

PS-001 is the culmination of over ten years of research by Academia Sinica Researcher Dr. Wen-Ching Yang and is a small-molecule drug with an entirely new mechanism for diabetes. Unlike existing drugs that only control blood sugar, PS-001 inhibits the key protein in diabetes progression, PDIA4 (Protein Disulfide Isomerase), and possesses the function of preventing pancreatic $\beta$-cell death. PS-001 has been shown in mouse models to stabilize blood glucose and glycated hemoglobin (HbA1c), reduce oxidative stress and death in $\beta$-cells, and even reverse or cure diabetes.

Clinical Outlook and Financing

Motivated by these research achievements, Pharmasaga is confident that, under the premise of PS-001's high safety profile, it will successfully complete the Phase I Single-Ascending Dose trial and commence the Multiple-Ascending Dose trial this year. Fundraising is also progressing smoothly. Looking ahead to next year, Pharmasaga is actively preparing for the Phase II clinical trial for Type 2 Diabetes patients to further validate PS-001's efficacy in humans, expand the target population, and ultimately benefit more patients.