Septerna, Inc. (NASDAQ: SEPN) has recently announced the commencement of the phase 1 clinical trial for its oral small molecule, SEP-631, aimed at treating mast cell-driven diseases, including chronic spontaneous urticaria (CSU). The trial will focus on evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of SEP-631 in healthy adult volunteers.
The company's Chief Medical Officer, Jae Kim, M.D., highlighted the significance of this trial, emphasizing that mast cell-driven diseases affect millions of patients globally, often leading to inadequate symptom relief with current therapies. SEP-631, a selective oral small molecule mas-related G protein-coupled receptor x2 (MRGPRX2) negative allosteric modulator, seeks to inhibit mast cell activation by selectively blocking MRGPRX2, a critical player in mast cell activation and degranulation.
Chronic spontaneous urticaria (CSU), one of the most common mast cell-driven diseases, is characterized by the recurrent appearance of itchy, painful hives and angioedema. With no known trigger, the activation and degranulation of mast cells result in these debilitating symptoms. The phase 1 trial, which is expected to enroll up to approximately 150 healthy adult volunteers, will focus on assessing the safety and tolerability of escalating oral doses of SEP-631 in the single-ascending dose (SAD) portion and over the treatment period in the multiple-ascending dose (MAD) portion. Pharmacodynamics will be evaluated through an icatibant skin challenge.
SEP-631 has demonstrated potent and long-lasting inhibition of MRGPRX2 and blocked mediator-induced skin extravasation in preclinical studies, offering potential hope for patients suffering from CSU and other mast cell-driven diseases. Septerna, Inc. is dedicated to advancing the development of novel oral small molecule drug candidates through its proprietary Native Complex Platform™, with a focus on addressing significant unmet needs in various therapeutic areas, including endocrinology, immunology, inflammation, and metabolic diseases. As a result of these announcements, the company's shares have moved 1.46% on the market, and are now trading at a price of $11.09. Check out the company's full 8-K submission here.
