Santhera Presents Efficacy Data on Raxone® for the Treatment of Leber’s Hereditary Optic Neuropathy

In Press Release by Cameron


March 4, 2014

Santhera Pharmaceuticals announced today that it will present data from its ongoing Expanded Access Program (EAP) with Raxone® in the treatment of Leber’s Hereditary Optic Neuropathy (LHON) at the 2014 Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS) in Rio Grande, Puerto Rico. Current data from the EAP demonstrate that 50% of patients have achieved a clinically relevant improvement in their vision and 63% of the patients were protected from further vision loss following Raxone®treatment. Raxone® was safe and well tolerated following long-term use in the EAP, which provides further evidence of safety and efficacy for Raxone® in the treatment of LHON in the routine clinical setting.

Safety and efficacy data were collected in an ongoing EAP for LHON patients with recent onset of symptoms in which they were given access to Raxone® in response to an unsolicited request from their treating physician. As of January 31, 2014, 29 physicians in Europe, the USA, Australia and New Zealand had enrolled 61 LHON patients. Participants received Raxone®typically at 900 mg/day and were assessed for safety and visual acuity (VA) during routine clinical care for up to 21 months. Currently data from 48 patients carrying the G11778A, G3460A or T14484C mutation have provided best corrected VA data from at least one on-treatment visit. Patient demographics such as distribution of mtDNA mutation carried, gender, age and extent of VA loss of enrolled patients were consistent with the known natural history of LHON. Efficacy analyses assessed (i) the proportion of patients with clinically relevant and stable improvement in VA and (ii) the proportion of patients without further deterioration in VA.

LHON patients treated with Raxone® experienced rapid, clinically relevant improvement in vision 
In the EAP, 24 of 48 patients (50%) have so far experienced a clinically relevant and stable improvement in VA defined as either (i) improvement from VA nadir of at least 10 letters (logMAR 0.2) on the ETDRS chart or (ii) improvement to reading at least 5 letters on-chart in patients with severe vision loss unable to read any letters on the ETDRS chart at nadir. In both cases, VA recovery had to be stable until the last available visit. Patients have been treated for an average of 11 months (range 3 to 21 months). Analyzed by disease-causing mtDNA mutation carried, 89% of patients with the T14484C mutation, 70% with the G3460A and 31% with the G11778A mutation have had clinically relevant recovery of vision. The percentage of Raxone® treated patients with VA recovery is markedly higher than that observed in a comparable population from a recently completed natural history case record survey of over 350 LHON cases collected from centers in Europe and the USA.

In the EAP, the average treatment effect size in patients with VA recovery was 29 letters from the VA nadir across all mtDNA mutations (49 letters for patients with the T14484C, 26 letters for patients with the G3460A and 13 letters for patients with the G11778A mtDNA mutation). The efficacy of Raxone® in promoting recovery of VA occurs rapidly, as 75% of patients have responded within the first 6 months and 83 % within 12 months of the initiation of Raxone®treatment.

Raxone® treatment protects LHON patients from further vision loss
In the EAP, there were 38 patients able to read at least 5 letters on the ETDRS chart at the time of enrolment (baseline). Of those 24 (63%) had not worsened by the clinically relevant margin (defined as 10 letters of on-chart VA or transition from on-chart VA to off-chart vision) in either eye at the last available visit. Moreover, 9 of 13 patients (69%) who were not legally blind at baseline were protected from legal blindness (defined as logMAR >=1.0) during their Raxone®treatment period.

Data will be presented at this weeks’ 40th Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS) in Puerto Rico.

“The clinical experience with Raxone® from the ongoing Expanded Access Program clearly supports previously published evidence of the safety and efficacy of Raxone® in the treatment of LHON”, commented Thomas Meier, CEO of Santhera. “With this program, we have increased greatly the long-term data available for Raxone® in this disease. These new efficacy and natural history data will be important supporting elements in the European Marketing Authorization Application which we plan to submit in the coming weeks.”