Santhera Releases Additional Clinical Data Supporting the Use of Catena in LHON

Santhera has previously reported visual acuity related endpoints of the RHODOS study. The Company has now completed pre-specified responder analyses and analysis of additional endpoints including color contrast sensitivity. The study data will be presented today by Professor Patrick Chinnery, principal investigator of RHODOS and Director of the Institute of Human Genetics at Newcastle University, United Kingdom, in an oral presentation [1] followed by a poster presentation [2] at the 2010 Meeting of the ANA.

"The data released today add to the efficacy profile of Catena in the treatment of LHON. The substantial benefit seen particularly in patients at risk of vision loss is exciting as it suggests that Catena can address this urgent unmet medical need", said Thomas Meier, Chief Scientific Officer of Santhera. "The visual acuity data together with the additional data announced today result in a consistent pattern of improvement in vision in LHON patients who received Catena in the RHODOS trial. We remain fully committed to making this important therapy available to LHON patients as soon as possible and are discussing regulatory pathways with health authorities for filings anticipated in the first half of 2011."

Significant improvement in blue-yellow and stabilization in red-green color contrast sensitivity
A hallmark of LHON is loss of color contrast sensitivity, which frequently precedes the loss of visual acuity and leads to complete color blindness. In the RHODOS study the majority of individuals for which such data were collected (39 patients) had already severely impaired color contrast sensitivity at baseline. The eyes of patients on Catena improved by a mean of 7.3% in blue-yellow color contrast between baseline and week 24, compared to those on placebo who worsened by 6.4% (p=0.008). In addition, the red-green color contrast was stabilized in patients on Catena with a trend towards improvement (p=0.239) compared to placebo.

Catena consistently superior to placebo in pre-specified responder analyses
In these pre-specified responder analyses the proportion of eyes in which the visual acuity improved or worsens by 0.2 logMAR or 2 lines (equivalent of 10 letters) or more on the standard chart is counted. A change of this magnitude is considered clinically meaningful. The study data show a consistent pattern of higher proportions of patients benefiting from Catena compared to placebo. Of particular interest are the patients with severely impaired vision, i.e. unable to read any letter on the chart at baseline. In this subgroup the vision in approximately 20% of eyes in patients receiving Catena improved to a level that they were once more able to read at least 1 full line on the chart. None of the eyes in patients in the placebo group improved to this extent (p=0.008).



Significant correlation of Clinical Global Impression of Change with primary and key secondary endpoints
In the ITT population, changes in visual acuity significantly correlated with the clinical benefit experienced by patients as determined by the Clinical Global Impression of Change (CGIC) score from baseline to week 24.



Contact
Thomas Meier Chief Scientific Officer T.: 061 906 89 64 E.: thomas.meier@santhera.com