Ashvattha Therapeutics Announces Positive Topline 40-Week Phase 2 Results for Migaldendranib in Diabetic Macular Edema and Neovascular Age-Related Macular Degeneration

Migaldendranib (MGB) is a new class of targeted nanomedicine for the treatment of diabetic macular edema (DME) and neovascular age-related macular degeneration (nAMD) with a novel mechanism of action that normalizes VEGF expression.

End of study, 40-week results demonstrated that MGB, delivered via subcutaneous (subQ) administration, is safe and well-tolerated with no MGB treatment-related ocular or systemic serious adverse events.

This Phase 2 clinical study showed improvements in efficacy outcomes and a marked reduction in the need for supplemental intravitreal anti-VEGF injections in both study and fellow eyes of DME and nAMD participants.

MGB has the potential for safe at-home administration, exhibiting a bilateral treatment effect with a single monthly subQ administration that may be an alternative option to frequent in-office intravitreal injections.

REDWOOD CITY, Calif., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Ashvattha Therapeutics ("Ashvattha"), a clinical-stage company developing a nanomedicine therapeutic that traverses the blood-retinal barrier in areas of inflammation selectively targeting activated cells in the retina, today announced positive topline end-of-study results from its Phase 2 study of migaldendranib (MGB) for the treatment of diabetic macular edema (DME) and neovascular age-related macular degeneration (nAMD) at the 25th European Society of Retina Specialists (EURETINA) Congress.

This multicenter, chronic dosing, 40-week Phase 2 clinical trial evaluated the safety and efficacy of subcutaneous MGB, an investigational VEGF receptor tyrosine kinase inhibitor covalently linked to a hydroxyl dendrimer, designed to shut down VEGF expression in activated macrophages and microglia, and hypoxic retinal pigment epithelial cells. Previously anti-VEGF treated DME and nAMD patients were enrolled. Responders to intravitreal (IVT) anti-VEGF were identified prior to entering the 40-week MGB treatment period. Criteria for supplemental anti-VEGF IVT while receiving subQ MGB during the study were pre-defined. The participants’ worse-seeing eye was designated as the study eye.

Key findings include:

• The annualized rate of IVT injections in all study eyes (DME and nAMD) decreased from 8.4 to 1.6 per year, or a 78.6% (4.7-fold) DME (n=8) and an 83.4% (6-fold) nAMD (n=14) reduction in the need for supplemental anti-VEGF versus pre-study IVT
• A bilateral MGB treatment benefit was demonstrated with the annualized rate of IVT injections in all fellow eyes, decreasing from 8.3 to 0.9 per year, or an 89.1% (9.1-fold) reduction in the need for supplemental anti-VEGF versus pre-study IVT
• Mean change from baseline out to Week 40 was +6.1 ETDRS letters in best corrected visual acuity (BCVA) and -23.3 μm in central subfield thickness (CST) in DME study eyes
• Improvements in BCVA and CST were also observed in nAMD study eyes
• SubQ MGB is safe and well tolerated with no treatment-related systemic or ocular serious adverse events reported and no clinically significant changes from baseline in renal, hepatic, or cardiac values
  

“These Phase 2 results demonstrate the potential of MGB to address one of the biggest challenges in retinal care today – the treatment burden associated with frequent in-office intravitreal injections,” said Susan Schneider, MD, Acting Chief Medical Officer of Ashvattha Therapeutics. “With the safe systemic and ocular outcomes seen to date, MGB offers a differentiated approach that has the possibility to enable monthly at-home subcutaneous administration and could meaningfully reduce the burden of care for patients with DME and nAMD, conditions that are chronic and typically bilateral.”

Unlike current anti-VEGF therapies that require repeated intravitreal injections directly into each affected eye, subcutaneously administered MGB provides a differentiated mechanism of action by crossing the blood-retinal barrier in regions of inflammation and selectively normalizing VEGF expression in activated macrophages and microglia, and hypoxic retinal pigment epithelial cells. By reducing VEGF expression rather than blocking VEGF signaling, MGB reduces the need for anti-VEGF intravitreal injections, which must be given in the physician’s office, and should potentially improve patient compliance by a safe, at home, monthly administered product.

“Over the 40-week duration of this trial, MGB was well tolerated, and we observed improvements in both vision and retinal anatomy, along with a significant reduction in the need for supplemental intravitreal injections in patients with active nAMD and DME,” said Arshad M. Khanani, MD, MA, FASRS, Managing Partner and Director of Clinical Research at Sierra Eye Associates. “Delivering a bilateral therapeutic effect through a once-monthly subcutaneous injection represents a meaningful advancement for patients with these vision-threatening retinal diseases, with the potential to enhance real world outcomes and overall quality of life.”

About Migaldendranib
Migaldendranib (MGB) is a novel first-in-class nanomedicine in clinical development for the treatment of DME and nAMD. Administered subcutaneously, MGB has a differentiated mechanism of action that crosses the blood-retinal barrier in regions of inflammation and selectively normalizes VEGF expression in activated macrophages and microglia, and hypoxic retinal pigment epithelial cells in the retina. Phase 2 clinical trial results demonstrate that subcutaneous MGB is safe and well-tolerated across multiple doses, with the potential for convenient once-monthly at-home administration that could significantly reduce intravitreal injection burden for patients with DME and nAMD, including those with bilateral disease.

About Ashvattha Therapeutics 
Ashvattha Therapeutics is advancing a new class of clinical-stage nanomedicine therapeutics that traverse tissue barriers to selectively target and reprogram activated cells only in regions of inflammation. Our targeted nanomedicine approach seeks to redefine precision medicine, empowering a new standard of care in ophthalmology. Ashvattha Therapeutics was founded by Kannan Rangaramanujam, Sujatha Kannan, and Jeff Cleland and incubated by Natural Capital. For more information, visit: www.avttx.com

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