ASLAN Pharmaceuticals to Advance Novel DHODH Inhibitor Farudodstat Into Phase 2 Clinical Testing as Potential First-In-Class Treatment for Alopecia Areata

• Alopecia areata (AA) is a common autoimmune disease affecting over 700,000 patients in the US and is driven by loss of immune privilege (IP) in the hair follicles and subsequent attack by IFNγ-secreting immune cells
• Farudodstat is a potent, oral DHODH inhibitor which has been shown to inhibit IFNγ secretion, reduce immune cell proliferation, and potentially protect from IP collapse in a human AA disease model
• A Phase 2 proof-of-concept trial of farudodstat in AA expected to start enrolling patients in the US in the second quarter of 2023
• ASLAN will host a virtual R&D Day on farudodstat with Brett King MD PhD, Associate Professor of Dermatology, Yale University, to discuss the unmet medical need and current treatment landscape in AA on March 16, 2023, 10:00 – 11:30 AM ET

SAN MATEO, Calif. and SINGAPORE, Feb. 28, 2023 (GLOBE NEWSWIRE) -- ASLAN Pharmaceuticals (Nasdaq: ASLN), a clinical-stage, immunology-focused biopharmaceutical company developing innovative treatments to transform the lives of patients, today announced that it is advancing its clinical program to investigate farudodstat, an oral dihydroorotate dehydrogenase (DHODH) inhibitor, in a Phase 2 proof-of-concept trial as a potential first-in-class treatment for alopecia areata (AA).

AA is an autoimmune disease characterized by loss of immune privilege (IP) in the hair follicle, as measured by an increase in major histocompatibility complex class I (MHC I) expression, and is driven by the activation and proliferation of interferon gamma (IFNγ)-secreting immune cells which mediate the attack of hair follicles, resulting in complete or partial hair loss on the scalp and body. AA is associated with severe psychological burden and has limited treatment options, affecting over 700,000 patients in the US.

The inhibition of DHODH is an established mechanism for the treatment of several autoimmune diseases. DHODH inhibitors have been approved and widely used for Multiple Sclerosis and Rheumatoid Arthritis, however first-generation inhibitors have limited potency and safety drawbacks. Farudodstat, a highly selective DHODH inhibitor, is 30-fold more potent than approved drugs in its class and has a well-tolerated safety profile. In vitro studies have shown that farudodstat inhibits immune cell proliferation and IFNγ production. Recent translational studies in an ex vivo human AA disease model showed that farudodstat reduced MHC I protein induction, a hallmark feature of IP loss. The model also yielded other signs of efficacy suggesting that farudodstat may potentially protect against hair follicle attack by activated immune cells.

“We are excited to advance the clinical development of farudodstat, which has the potential to be a first-in-class therapy addressing the unmet needs of alopecia areata patients,” commented Dr Carl Firth, Chief Executive Officer, ASLAN Pharmaceuticals. “Despite recent advancements in treatment options, the long-term safety of therapies for AA remains a top priority for physicians. We have generated encouraging data that supports our belief that farudodstat can prevent, and potentially restore, the loss of immune privilege in hair follicles, offering a safe and effective treatment option for AA with a competitive profile.”

The Phase 2a proof-of-concept trial is a 2:1 randomized trial, with patients receiving oral doses of either farudodstat or placebo twice daily for 12 weeks, followed by a crossover treatment period. The trial will recruit about 60 patients in the US and enrollment is expected to begin in the second quarter of 2023. The interim topline readout following the first 12-week treatment period is expected in 1Q 2024 and will inform the design of the subsequent Phase 2b dose-ranging study. The primary objective of the trial is to evaluate the safety, tolerability and efficacy of farudodstat in adult subjects with severe alopecia areata.

Virtual R&D Day on farudodstat

ASLAN’s management team will host a virtual Research and Development (R&D) Day on farudodstat with Brett King MD PhD, Associate Professor of Dermatology, Yale University on Thursday, March 16, 2023, from 10:00am to 11:30am ET. Further details will follow. To register for the event, please click here. A replay of the event and presentation materials will be available on the Investor Relations section of ASLAN Pharmaceutical’s website.

About farudodstat

Farudodstat is a potent oral DHODH inhibitor that suppresses immune cell proliferation and IFNγ secretion by blocking de novo production of pyrimidines required for DNA replication. Compared to first-generation DHODH inhibitors, farudodstat has been shown to be approximately 30 times more potent in its inhibition of DHODH and limiting T cell activity and has a well-tolerated safety profile. ASLAN has generated data showing that farudodstat can protect against the loss of immune privilege in hair follicles, supporting its potential as a first-in-class, safe and effective treatment option for alopecia areata (AA). ASLAN plans to initiate a proof-of-concept study in AA in the second quarter of 2023.

About ASLAN Pharmaceuticals

ASLAN Pharmaceuticals (Nasdaq: ASLN) is a clinical-stage, immunology-focused biopharmaceutical company developing innovative treatments to transform the lives of patients. ASLAN is currently evaluating eblasakimab, a potential first-in-class antibody targeting the IL-13 receptor, in the global Phase 2b TREK-AD trial in moderate-to-severe atopic dermatitis (AD) patients and the Phase 2 TREK-DX trial in dupilumab-experienced AD patients. ASLAN is also developing farudodstat, a potent oral inhibitor of the enzyme DHODH, in alopecia areata and plans to initiate a Phase 2 proof-of-concept trial in the second quarter of 2023. ASLAN has a team in California and in Singapore. For additional information please visit www.aslanpharma.com or follow ASLAN on LinkedIn.

Forward-looking statements  

This release contains forward-looking statements. These statements are based on the current beliefs and expectations of the management of ASLAN Pharmaceuticals Limited and/or its affiliates (the "Company"). These forward-looking statements may include, but are not limited to, statements regarding the Company’s business strategy and clinical development plans; the Company’s plans to develop and commercialize eblasakimab and farudodstat; the safety and efficacy of eblasakimab and farudodstat; the Company’s plans and expected timing with respect to clinical trials, clinical trial enrollment and clinical trial results for eblasakimab and farudodstat; and the potential of eblasakimab as a first-in-class treatment for atopic dermatitis and of farudodstat as a treatment for autoimmune disease. The Company’s estimates, projections and other forward-looking statements are based on management's current assumptions and expectations of future events and trends, which affect or may affect the Company’s business, strategy, operations, or financial performance, and inherently involve significant known and unknown risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of many risks and uncertainties, which include, unexpected safety or efficacy data observed during preclinical or clinical studies; clinical site activation rates or clinical trial enrollment rates that are lower than expected; the impact of the COVID-19 pandemic or the ongoing conflict between Ukraine and Russia on the Company’s business and the global economy; general market conditions; changes in the competitive landscape; and the Company’s ability to obtain sufficient financing to fund its strategic and clinical development plans. Other factors that may cause actual results to differ from those expressed or implied in such forward-looking statements are described in the Company’s US Securities and Exchange Commission filings and reports (Commission File No. 001- 38475), including the Company’s Annual Report on Form 20-F filed with the US Securities and Exchange Commission on March 25, 2022. All statements other than statements of historical fact are forward-looking statements. The words “believe,” “may,” “might,” “could,” “will,” “aim,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “plan,” or the negative of those terms, and similar expressions that convey uncertainty of future events or outcomes are intended to identify estimates, projections, and other forward-looking statements. Estimates, projections, and other forward-looking statements speak only as of the date they were made, and, except to the extent required by law, the Company undertakes no obligation to update or review any estimate, projection, or forward-looking statement.

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