ROBIN: a randomised, double-masked, placebo-controlled Phase IIa study of the AOC3 inhibitor BI 1467335 in diabetic retinopathy

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2024年5月28日

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Quan Dong Nguyen,
Justis P. Ehlers,
David S. Boyer,
Xidong Jin,
Andrea Giani,
Michael S. Ehrlich,
Alexander Brucker,
Allen Hu,
Amani Fawzi,
Andrew Antoszyk,
Brian Berger,
Chirag Jhaveri,
Claire Bailey,
David Brown,
Derek Kunimoto,
Faruque Ghanchi,
Francesco Bandello,
Geeta Menon,
Harsha Sen,
James Talks,
João Figueira,
Jose Juan Escobar Barranco,
Juan Donate Lopez,
Maged Habib,
Maja Gran Erke,
Martin Weger,
Matthew Cunningham,
Monica Varano,
Nonavinakere Manjunatha,
Paul Hahn,
Pilar Calvo,
Pravin Dugel,
Raj Maturi,
Richard Rosen,
Rufino Silva,
Sergio Pagliarini,
Sobha Sivaprasad,
Sofia Androudi,
Sunil Patel

摘要

Objective

To evaluate the safety and efficacy of BI 1467335 in patients with non-proliferative diabetic retinopathy (NPDR).

Methods

ROBIN is a Phase IIa, double-masked, randomised, placebo-controlled study (NCT03238963). Patients with NPDR and without centre-involved diabetic macular oedema were included; all had a best corrected visual acuity letter score of ≥70 Early Treatment Diabetic Retinopathy Study letters in the study eye at screening. Patients received oral BI 1467335 10 mg or placebo once daily for 12 weeks. Post-treatment follow-up was 12 weeks. The primary endpoint was the proportion of patients over the 24 weeks with ocular adverse events (AEs). Secondary endpoints were the proportion of patients with ≥2-step improvement from baseline in DRSS severity level at Week 12 and the proportion of patients with non-ocular AEs at 24 weeks.

Results

Seventy-nine patients entered the study (BI 1467335, n = 40; placebo, n = 39). The proportion of patients with ocular AEs over 24 weeks was greater in the BI 1467335 versus the placebo group (35.0% vs 23.1%, respectively). Treatment-related AEs were reported for similar numbers of patients in the placebo and BI 1467335 group (7.7% vs 7.5%, respectively). At Week 12, 5.7% (n = 2) of patients in the BI 1467335 group had a 2-step improvement in DRSS severity level from baseline, compared with 0% in the placebo group.

Conclusions

BI 1467335 was well tolerated by patients with NPDR. There was a high variability in DRSS levels for individual patients over time, with no clear efficacy signal.