Rallybio begins dosing in phase 1 study of C5 inhibitor RLYB116

Published 12/06/2025, 13:14
Rallybio begins dosing in phase 1 study of C5 inhibitor RLYB116

NEW HAVEN - Rallybio Corporation (NASDAQ:RLYB), currently trading at $0.36 and showing a market capitalization of $14.82 million, has initiated dosing in a Phase 1 confirmatory pharmacokinetic/pharmacodynamic study of RLYB116, its subcutaneously injected C5 inhibitor, according to a company press release. According to InvestingPro analysis, the company appears undervalued based on its Fair Value metrics.

The clinical-stage biotechnology company announced it will focus initially on two hematologic conditions: immune platelet transfusion refractoriness (PTR) and refractory antiphospholipid syndrome (APS), both of which currently lack approved treatments. InvestingPro data shows the company maintains a strong financial position with more cash than debt and a healthy current ratio of 14.68, providing runway for its clinical development programs.

The single-blind multiple ascending dose study will evaluate RLYB116 in two cohorts of healthy volunteers over a 4-week treatment period, with participants receiving either the drug or placebo once weekly. Cohort 1 will test a 150 mg dose while Cohort 2 will evaluate dosing up to 300 mg.

Data from the first cohort is expected in the third quarter of 2025, with second cohort results anticipated in the fourth quarter.

PTR is a condition where patients fail to achieve expected platelet count increases following multiple transfusions, increasing bleeding risks in patients with conditions like hematologic malignancies. APS is a rare autoimmune disease characterized by recurrent vascular thrombosis and pregnancy-related complications, with approximately 10% of patients continuing to experience thromboses despite anticoagulant treatment.

Stephen Uden, Chief Executive Officer of Rallybio, described the study initiation as "a significant step forward" in the company’s mission to develop therapies for these conditions, which he characterized as representing a combined market opportunity of $5 billion.

RLYB116 is designed as a once-weekly, small volume C5 inhibitor that could potentially address a range of complement-mediated diseases. While the stock has experienced a significant decline of 78% over the past year, recent momentum shows a 5% gain in the last week. Discover more insights about RLYB and 1,400+ other stocks with comprehensive Pro Research Reports, available exclusively on InvestingPro.

In other recent news, Rallybio Corporation has experienced several significant developments. The company announced the discontinuation of its RLYB212 program after Phase 2 clinical trial data failed to meet the necessary pharmacokinetic targets. This decision led to a downgrade by Citizens JMP, which adjusted its rating from "Market Outperform" to "Market Perform" due to the program’s termination. Additionally, Evercore ISI downgraded Rallybio from "Outperform" to "In Line," citing concerns with the study data related to the FNAIT program. Despite these setbacks, Rallybio is shifting its focus to RLYB116, a differentiated C5 inhibitor, with Phase 1 data expected in the latter half of 2025. Citizens JMP has maintained a "Market Perform" rating for Rallybio, expressing interest in the potential of the RLYB116 program. In corporate governance news, Rallybio held its annual shareholder meeting, where Martin W. Mackay and Paula Soteropoulos were elected as Class I directors. Shareholders also ratified Deloitte and Touche LLP as the company’s independent registered public accounting firm for the fiscal year ending December 31, 2025. These developments reflect Rallybio’s ongoing strategic adjustments and governance activities.

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