Positive Early Trial Results for Lonigutamab in Treating Thyroid Eye Disease

Acelyrin, a clinical-stage biopharmaceutical company, has reported promising results from a Phase 1/2 trial of its drug lonigutamab for the treatment of thyroid eye disease (TED). The drug is a humanized monoclonal antibody that targets the insulin-like growth factor-1 receptor (IGF-1R) and is administered subcutaneously. The trial showed significant improvements in proptosis and clinical activity score (CAS) within three weeks of the first dose.

The study involved multiple cohorts of patients with active TED. The first cohort, which was placebo-controlled, saw 50% of patients receiving lonigutamab achieve a proptosis response and 100% achieve a CAS response. The second cohort, which was open label and received a different dosing regimen, showed even higher response rates. Notably, the drug was well-tolerated with no serious adverse events reported.

The CEO of Acelyrin, Shao-Lee Lin, highlighted the significance of these findings, stating that they support the company's hypothesis that lonigutamab can offer a favorable benefit-risk profile by allowing for longer-term subcutaneous dosing. This approach aims to enhance the depth and durability of the clinical response while minimizing safety risks.

Shoaib Ugradar, a specialist in orbital and oculoplastic surgery, commented on the encouraging results, noting the potential for a clinically meaningful response as early as three weeks post-injection. He emphasized the importance of a medication with a favorable side effect profile, especially considering the long-term consequences of TED.

Following the successful proof-of-concept, Acelyrin plans to initiate a Phase 2b/3 trial in the latter half of 2024. This trial will be the first of two registrational trials for TED. The company has decided to forego a separate fiscal year 2023 earnings call, opting instead to release financial results through a press release and filing the 10-K report by April 1, 2024.

Thyroid Eye Disease is a serious autoimmune condition affecting over 100,000 people in the United States. It is characterized by inflammation and tissue expansion behind the eye, leading to symptoms such as eye bulging and double vision, with potential blindness as a severe consequence. Lonigutamab's subcutaneous administration could offer a more convenient and potentially safer treatment option for patients.

Acelyrin is a Los Angeles-based company dedicated to accelerating the development of transformative medicines. It has two programs in late-stage clinical development, including lonigutamab for TED and izokibep, a next-generation IL-17A inhibitor for various conditions.