June 2021
Volume 62, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2021
Topline Results From a Multicenter, Open-label, Phase 4 Study of Repository Corticotropin Injection in Patients With Treatment-resistant Severe Non-infectious Keratitis
Author Affiliations & Notes
  • Joseph Grieco
    Mallinckrodt Pharmaceuticals, Bedminster, New Jersey, United States
  • Eugene B McLaurin
    Total Eye Care, Memphis, Tennessee, United States
  • George W Ousler
    Ora, Inc, Andover, Massachusetts, United States
  • Jingyu Liu
    Mallinckrodt Pharmaceuticals, Bedminster, New Jersey, United States
  • Oktay Kacmaz
    Mallinckrodt Pharmaceuticals, Bedminster, New Jersey, United States
  • David Wirta
    Eye Research Foundation, Newport Beach, California, United States
  • Footnotes
    Commercial Relationships   Joseph Grieco, Mallinckrodt Pharmaceuticals (E); Eugene McLaurin, Aldeyra Therapeutics (F), Allergan (F), Aurinia Pharmaceuticals (F), Hanall Biopharma (F), Mitotech (F), Ocular Therapeutix (F), ReGenTree, LLC (F), Santen Pharmaceutical Co., Ltd (F), Shire (F), Sun Pharma (F), TopiVert Pharma Limited (F); George Ousler, Mallinckrodt Pharmaceuticals (F); Jingyu Liu, Mallinckrodt Pharmaceuticals (E); Oktay Kacmaz, Mallinckrodt Pharmaceuticals (E); David Wirta, Mallinckrodt Pharmaceuticals (R)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science June 2021, Vol.62, 1275. doi:
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      Joseph Grieco, Eugene B McLaurin, George W Ousler, Jingyu Liu, Oktay Kacmaz, David Wirta; Topline Results From a Multicenter, Open-label, Phase 4 Study of Repository Corticotropin Injection in Patients With Treatment-resistant Severe Non-infectious Keratitis. Invest. Ophthalmol. Vis. Sci. 2021;62(8):1275.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Keratitis is a painful inflammation of the cornea commonly treated with immunosuppressive drugs. However, additional treatments are needed for patients in whom keratitis is not responsive to standard therapies. Repository corticotropin injection (RCI, Acthar® Gel) has anti-inflammatory and immunomodulatory effects through interaction with melanocortin receptors. The goal of this multicenter, open-label, phase 4 study was to evaluate the efficacy and safety of RCI in patients with refractory severe non-infectious keratitis (Clinicaltrials.gov, NCT04169061).

Methods : Patients ≥18 years of age with severe keratitis that is refractory to standard of care received 80 U RCI subcutaneously twice weekly for 12 weeks. Initial assessments included Impact of Dry Eye on Everyday Life (IDEEL) score, Visual Analogue Scale (VAS) for burning/stinging and eye discomfort, corneal fluorescein staining, and conjunctival lissamine green staining as measured by Ora Calibra scales. One-sample binomial tests (null hypothesis: 5%) or one-sample t tests (null hypothesis: no change) were used to perform statistical analyses, as appropriate; significance vs baseline was determined as p<0.05. Adverse events (AEs) were also evaluated.

Results : Topline results in 35 patients showed that a significant proportion (41%-65%) of patients improved ≥20% at weeks 2, 4, 6, and 12 (all measured time points) on the symptom bother score module of the IDEEL questionnaire. A clinically meaningful ≥12-point improvement on the symptom bother score module was achieved in a significant proportion (50%-56%) of patients at all measured time points. VAS score for burning/stinging significantly improved at weeks 2, 6, and 12, as did the VAS score for eye discomfort at all measured time points. Inferior, superior, and central corneal fluorescein staining detecting corneal damage significantly decreased at weeks 4, 6, and 12. Both temporal and nasal conjunctival lissamine green staining for dry eyes were significantly reduced at week 6. Most AEs were rare, with hypertension being the most common, occurring in 2 patients. No serious AEs were related to the study drug.

Conclusions : Topline results of this open-label study showed that RCI significantly reduced the signs and symptoms of refractory severe non-infectious keratitis, in alignment with patient perceptions.

This is a 2021 ARVO Annual Meeting abstract.

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