June 2020
Volume 61, Issue 7
ARVO Annual Meeting Abstract  |   June 2020
A Phase 1b/2 Open-label Clinical Trial to Evaluate the Safety and Efficacy of AU-011 for the Treatment of Choroidal Melanoma
Author Affiliations & Notes
  • Prithvi Mruthyunjaya
    Ophthalmology, Byers Eye Institute at Stanford University, Palo Alto, California, United States
  • Amy C Schefler
    Retina Consultants of Houston, Houston, Texas, United States
  • Ivana K Kim
    Mass Eye And Ear Infirmary, Boston, Massachusetts, United States
  • Christopher Bergstrom
    Retina Consultants of Carolina, PA, Greenville, South Carolina, United States
  • Hakan Demirci
    Ophthalmology, W. K. Kellogg Eye Center, University of Michigan, Ann Arbor, Michigan, United States
  • Tony Tsai
    Retina Consultants of Sacramento, Sacramento, California, United States
  • Abdhish R Bhavsar
    Retina Center, Minneapolis, Minnesota, United States
  • Antonio Capone
    Associated Retinal Consultants, PC, Royal Oak, Michigan, United States
  • Brian Marr
    Ophthalmology, Columbia Univesrsity Medical Center, New York, New York, United States
  • Tara A McCannel
    Ophthalmology, UCLA Jules Stein Eye Institute, Los Angeles, California, United States
  • Cameron Javid
    Retina Associates, Tuscon, Arizona, United States
  • Peter G Hovland
    Colorado Retina Consultants, Denver, Colorado, United States
  • Michael I. Seider
    Ophthalmology, Permanente Medical Group, San Francisco, California, United States
  • Cadmus Rich
    Aura Biosciences, Boston, Massachusetts, United States
  • Carol L Shields
    Wills Eye Hospital, Philadelphia, Pennsylvania, United States
  • Footnotes
    Commercial Relationships   Prithvi Mruthyunjaya, Aura Biosciences (C), Castle Biosciences (C); Amy Schefler, Aura Biosciences (C); Ivana Kim, None; Christopher Bergstrom, None; Hakan Demirci, None; Tony Tsai, None; Abdhish Bhavsar, None; Antonio Capone, None; Brian Marr, Aura Biosciences (C); Tara McCannel, None; Cameron Javid, None; Peter Hovland, None; Michael Seider, None; Cadmus Rich, Aura Biosciences (E); Carol Shields, Aura Biosciences (C)
  • Footnotes
    Support  Aura Biosciences (sponsor)
Investigative Ophthalmology & Visual Science June 2020, Vol.61, 4025. doi:
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      Prithvi Mruthyunjaya, Amy C Schefler, Ivana K Kim, Christopher Bergstrom, Hakan Demirci, Tony Tsai, Abdhish R Bhavsar, Antonio Capone, Brian Marr, Tara A McCannel, Cameron Javid, Peter G Hovland, Michael I. Seider, Cadmus Rich, Carol L Shields; A Phase 1b/2 Open-label Clinical Trial to Evaluate the Safety and Efficacy of AU-011 for the Treatment of Choroidal Melanoma. Invest. Ophthalmol. Vis. Sci. 2020;61(7):4025.

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

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Purpose : Choroidal melanoma (CM) is the most common primary ocular malignancy. Most small CMs are treated with radiotherapy which is associated with several adverse effects including vision loss. The safety and efficacy of AU-011, a novel targeted investigational therapy for the treatment of CM, is beingevaluated in an open-label, multi-center, dose escalation and expansion trial.

Methods : Subjects with a clinical diagnosis of CM with tumor thickness 1.2 - 3.4 mm and largest basal diameter ≤ 16 mm received intravitreal administration of a viral-like particle bioconjugate (AU-011) at doses of 20 µg, 40 µg, or 80µg followed by light-activation with a 689 nm laser at a fluence of 50 J/cm2. Regimens consisting of 1, 2 or 3 weekly treatments with AU-011 each followed by 1 or 2 laser applications have been evaluated in 8 escalation cohorts and 1stexpansion. Enrollment is ongoing in the 2ndexpansion with subjects receiving two cycles of three weekly treatments with 80 µg /2 laser administrations separated by 12 weeks.

Results : 50 subjects have been treated in the trial. Interim results show local tumor control in 33/50 (66%) subjects (23/29 [79%] at therapeutic dose) and maintenance of visual acuity in 46/50 (92%) subjects (27/29 [93%] at therapeutic dose) with up to 24 months follow up. Linear regression was used to estimate tumor growth rates using tumor thickness measurements before and after treatment in subjects with historical documented growth (n=19). Tumor control was seen in 15/19 subjects and the post-treatment growth rate was significantly reduced compared to their historical growth rate (p=0.0056; paired t-test). There has been one treatment-related SAE of severe vision loss. Expected AEs related to treatment including intraocular inflammation and increased IOP were clinically manageable.

Conclusions : Interim results from this ongoing trial show preliminary safety and efficacy of AU-011. The therapy was well tolerated with maintenance of vision and tumor control in majority of subjects.AU-011 may offer an alternative to radiotherapy for treating small melanomas and high risk indeterminate lesions and further studies are planned to confirm the safety and efficacy.

This is a 2020 ARVO Annual Meeting abstract.


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