September 2016
Volume 57, Issue 12
Open Access
ARVO Annual Meeting Abstract  |   September 2016
BIO201: a new drug candidate for the treatment of dry AMD
Author Affiliations & Notes
  • Valerie Fontaine
    Institut de la Vision, Paris, France
  • Elena Brazhnikova
    Institut de la Vision, Paris, France
  • Elodie Monteiro
    Institut de la Vision, Paris, France
  • Laëtitia Lesage
    Institut de la Vision, Paris, France
  • Christine Balducci
    Biophytis, Paris, France
  • Louis Guibout
    Biophytis, Paris, France
  • Laurence Feraille
    Iris-Pharma, La Gaude, France
  • Pierre-Paul Elena
    Iris-Pharma, La Gaude, France
  • Jose Sahel
    Institut de la Vision, Paris, France
  • Stanislas Veillet
    Biophytis, Paris, France
  • René Lafont
    Biophytis, Paris, France
  • Footnotes
    Commercial Relationships   Valerie Fontaine, None; Elena Brazhnikova, None; Elodie Monteiro, None; Laëtitia Lesage, None; Christine Balducci, None; Louis Guibout, None; Laurence Feraille, None; Pierre-Paul Elena, None; Jose Sahel, None; Stanislas Veillet, None; René Lafont, None
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science September 2016, Vol.57, 1119. doi:
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      Valerie Fontaine, Elena Brazhnikova, Elodie Monteiro, Laëtitia Lesage, Christine Balducci, Louis Guibout, Laurence Feraille, Pierre-Paul Elena, Jose Sahel, Stanislas Veillet, René Lafont; BIO201: a new drug candidate for the treatment of dry AMD. Invest. Ophthalmol. Vis. Sci. 2016;57(12):1119.

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

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Abstract

Purpose : Dry AMD is a major cause of visual impairment associated with aging, and no drug treatment is presently available. In collaboration with the Institut de la Vision Biophytis develops a drug candidate (BIO201) based on norbixin, a di-apo-carotenoid, as IPI. We describe here the effects of norbixin on retinal pigmented epithelium (RPE) and retina photoprotection in vitro and in vivo.

Methods : The photo-protective effect of norbixin and of other carotenoids was evaluated on primary cultures of porcine RPE cells challenged with A2E and illuminated with blue light (N=16). In vivo experiments measured the photo-protective effect of norbixin one week after blue light damage in the Abca4-/-Rdh8-/- transgenic mouse model (which accumulates rapidly A2E in RPE cells). Twenty-eight 7-week-old mice were injected intra-vitreally in one eye with 120 mM norbixin (in 0.3% DMSO) or with DMSO alone, dark-adapted during 24 hours and exposed to blue light (4000 lux) for one hour. Ten mice were used as non-illuminated controls. Full field scotopic electroretinogram was measured one week after light damage and eyes were removed for histology and photoreceptor quantification. Bioavailability was tested in C57Bl/6 mice receiving norbixin 50 mg/kg per os (in oil/DMSO 9:1) or 5 mg/kg intraperitoneally (in DMSO/tetraglycol/water 1:2:7) (N=4). Norbixin was also assayed in a standard rat blue light model of photodamage by repeated intraperitoneal injections. For statistical analyses, one-way ANOVA followed by Dunnett’s tests were performed.

Results : Norbixin showed an improved photo-protection to porcine RPE as compared with lutein or zeaxanthin. At 5 mM, norbixin protected 72.6% cells vs 36.2% and 32.3% for lutein and zeaxanthin, respectively. In vivo, norbixin significantly maintained the ERG a-wave (38% vs 15.9% for DMSO control; p<0.05) and b-wave (49% vs. 21% for DMSO control; p<0.01) after induction of light damage and protected 61% photoreceptors as compared to DMSO-injected eyes (32%; p<0.001). Norbixin had a good oral bioavailability (55%); it was recovered in the eyes, peaking at 40 ng/eye 30-60 min after oral intake and it was still detectable at 7 ng/eye after 24 hours. In the blue light rat model, norbixin was equally active as PBN, the positive control.

Conclusions : Norbixin appears promising for the development of an oral treatment of dry AMD, and the potential of BIO201 will be assessed in a forthcoming multicentric clinical trial.

This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.

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