Boehringer Ingelheim reveals positive results from first global study on diabetic macular ischemia

Boehringer Ingelheim has announced promising results from the HORNBILL Phase I/IIa study, which investigates BI 764524, a potential treatment for diabetic macular ischemia (DMI). DMI is a severe complication of diabetic retinopathy (DR) that can result in blindness due to inadequate blood supply to the retina. Currently, there are no approved treatments for this condition.

BI 764524 demonstrated good tolerance in patients following intravitreal injections of both single and multiple doses, achieving its primary safety goals and showing preliminary signs of efficacy. This compound employs a unique mechanism by inhibiting the Sema3A pathway to re-vascularize ischemic areas, potentially outperforming existing anti-VEGF and laser treatments.

Quan Dong Nguyen, MD, MSc, FARVO, FASRS, and Principal Investigator of the trial, highlighted the potential of early intervention to prevent vision-threatening complications like DMI in patients with DR. He noted that retinal non-perfusion, a major cause of vision loss in DR patients, had not been previously targeted for treatment until the HORNBILL study.

Ulrike Graefe-Mody, Ph.D., Head of Retinal Health at Boehringer Ingelheim, emphasized the significance of these findings. She expressed optimism that the results could pave the way for precision therapies aimed at preventing vision loss before irreversible damage occurs. Boehringer Ingelheim is preparing to launch a Phase IIb study to further investigate the safety and efficacy of BI 764524.

The HORNBILL study included two parts: a single rising dose (SRD) involving 12 patients and a multiple dose (MD) phase with 31 participants. Both phases met their primary safety endpoints regarding the number of patients experiencing dose-limiting adverse events and treatment-related adverse events. Additionally, the study met its early efficacy criteria by stabilizing the foveal avascular zone area against a sham treatment at week 16.

During the SRD phase, 12 patients received varying doses of BI 764524 via intravitreal injection. No dose-limiting events were reported, and the highest tested dose was deemed safe for use in the MD phase. In the MD phase, 31 patients were randomized to receive either BI 764524 or a sham injection. The primary endpoint in this phase was the number of drug-related adverse events. Secondary endpoints included ocular adverse events, changes in foveal avascular zone area, best-corrected visual acuity, and central retinal thickness.

Patients in the MD phase had a mean age of 59.5 years. Seven ocular adverse events were documented, with one being related to the study drug. There were no cases of intraocular inflammation or occlusive retinal vasculitis reported. The HORNBILL study successfully met its early efficacy criterion for stabilizing the foveal avascular zone area, although other secondary efficacy endpoints showed no significant changes over the short trial period.

BI 764524 is a humanized monoclonal anti-Sema3A antibody designed to re-vascularize ischemic areas and reduce retinal leakage, addressing retinal non-perfusion in various retinal diseases. It represents Boehringer Ingelheim's commitment to developing innovative treatments for retinal conditions.

Diabetic retinopathy affects one-third of people with diabetes and is the leading cause of vision loss among working-age adults. As the global incidence of diabetes rises, the prevalence of diabetic retinopathy and its complications, such as DMI, is expected to increase. DMI leads to irreversible vision loss, making the development of effective treatments crucial.

Boehringer Ingelheim continues to focus on transformative therapies in areas with high unmet medical needs. Founded in 1885, the family-owned company employs over 53,000 people and operates in more than 130 markets, with a commitment to long-term, sustainable innovation.