Ashvattha Therapeutics Reports Positive Phase 2 Data for Subcutaneous Migaldendranib in Retinal Diseases

Ashvattha Therapeutics has announced encouraging interim results from its phase 2 trial evaluating migaldendranib (MGB), a novel subcutaneous nanomedicine, for the treatment of retinal vascular diseases. The investigational therapy has demonstrated promising efficacy and safety in patients with wet age-related macular degeneration (AMD) and diabetic macular edema (DME) who were previously treated with anti-VEGF intravitreal (IVT) injections.

About Migaldendranib (MGB)

MGB is a VEGF receptor tyrosine kinase inhibitor delivered through Ashvattha’s hydroxyl dendrimer nanomedicine platform. Unlike current standard therapies, MGB is administered subcutaneously (subQ) and is designed to selectively target activated cells in the retina, reducing VEGF expression and fluid accumulation in both eyes simultaneously.

Study Design and Patient Population

The open-label, chronic dosing phase 2 trial enrolled 27 subjects, including:

       • 16 patients with wet AMD

       • 11 patients with DME

All participants had previously responded to anti-VEGF IVT therapy and met the study’s responder criteria. Each subject received:

       • A single anti-VEGF IVT and subQ MGB at baseline

       • Follow-up subQ MGB injections every 2 or 4 weeks for a duration of 40 weeks

The interim analysis presented data from 24 weeks of treatment in 14 patients (8 with wet AMD and 6 with DME).

Clinical Outcomes at 24 Weeks

The results, presented at the 2025 Association for Research in Vision and Ophthalmology (ARVO) annual meeting (May 4–8, Salt Lake City), demonstrated notable efficacy and safety:

Reduction in Anti-VEGF Treatment Burden

       • 79.9% reduction in IVT injections for wet AMD patients

       • 80% reduction for DME patients

       • This represents a 5.0-fold decrease in anti-VEGF treatment burden over 24 weeks

Bilateral Treatment Effect

       • 66.7% reduction in fellow eye IVT injections in wet AMD subjects

       • 85.5% reduction in fellow eye injections in DME subjects

Visual Acuity and Anatomical Improvements

       • +3 letters mean improvement in Best Corrected Visual Acuity (BCVA) for wet AMD

       • +4.5 letters improvement in BCVA for DME

       • 45.5 µm reduction in central subfield thickness (CST) for wet AMD

       • 69.1 µm reduction in CST for DME

Safety Profile

No serious or ocular adverse events related to MGB were reported during the 24-week period.

       • Injection site reactions occurred in 5 subjects (3 with wet AMD, 2 with DME)

       • Across 302 total subQ injections, only 26 injection site reactions were documented

Conclusion

These interim phase 2 results suggest that subcutaneously delivered MGB may offer a safe, effective, and less burdensome alternative to traditional IVT anti-VEGF therapies for retinal diseases such as AMD and DME. The bilateral treatment effect and meaningful reduction in injection frequency position MGB as a potential game-changer in the management of chronic retinal vascular conditions.