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Santhera is passionate about developing new treatments for patients with mitochondrial diseases. Our focus is on the development of treatments for neuromuscular and neuro-ophthalmological diseases that currently lack treatment options.

Raxone in LHON: Efficacy

RHODOS Trial

All VA endpoints in the RHODOS trial indicated a beneficial effect for Raxone1

Figure based on data in Klopstock T, et al. Brain 2011; 134:2677–2686.

Visual acuity endpoints in the RHODOS trial were defined as follows:1

Visual acuity (VA) efficacy end-points between baseline and Week 24:

  1. Primary endpoint: best recovery/least worsening in VA, one value per patient.
  2. Main secondary endpoint: change in best VA, one value per patient.
  3. Pre-specified secondary endpoint: change in VA of best eye at baseline, one value per patient.
  4. Pre-specified secondary endpoint: change in VA of all eyes (both eyes of a patient considered independent), two values per patient.

Figure adapted from Klopstock T, et al. Brain 2011; 134:2677–2686.

In a predefined sub-group analysis of the RHODOS trial data, Raxone prevented progression to legal blindness (logMAR ≥ 1.0) in patients who had mild vision loss at treatment initiation, in contrast to patients who received placebo treatment1,2

Clinically relevant recovery of visual acuity (CRR of VA)

In the RHODOS trial 30% of patients had a CRR of VA from baseline when treated with Raxone compared with 10% in the placebo group after 6 months of treatment.2,*

Left hand figure based on data in the Raxone SmPC, September 2015

CRR is defined as an improvement of VA by at least 10 letters on-chart or an improvement from off-chart vision to again read five letters.3 The proportion of patients experiencing clinically relevant recovery (CRR) of vision in the RHODOS trial was assessed using responder analyses.1,4


The efficacy of Raxone has been further established in an Expanded Access Program2,3,6

Similar to the data from RHODOS, more than 30% of patients in the Expanded Access Program (EAP) experienced CRR of VA after 6 months of treatment.2 Additionally, the number of patients with CRR of VA increased further with longer treatment duration.2

Figure based on data in the Raxone SmPC, September 2015.

Long-term follow-up data for Raxone in LHON

Long-term follow-up of patients at 30 months after the end of Raxone treatment indicates that the effect of Raxone may be maintained (RHODOS-OFU).2,5

The effects of Raxone on VA observed in RHODOS persisted for a median of 30 months following treatment discontinuation.5

* Post hoc analyses (RHODOS-ITT)

References

1.

Klopstock T, et al. Brain 2011; 134:2677–2686.

2.

Raxone SmPC, September 2015. Available here. Accessed December 2015.

3.

Metz G, et al. ARVO 2014 poster (Abstract 6206).

4.

Carelli V, et al. Brain 2011; 134;e188.

5.

Klopstock T, et al. Brain 2013; 136:e230.

6.

Lyseng-Williamson, Adis Drug Evaluation, Drugs (DOI 10.1007/s40265-016-0574-3)

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information about Raxone® and other
products in Santhera’s pipeline.

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