Santhera Pharmaceuticals Holding AG (CH) - Santhera Releases Additional Clinical Data Supporting the Use of Catena in LHON

Presentations today at 2010 ANA Conference

 

Catena® shows improvement in color vision (p=0.008)Catena® consistently shows superiority over placebo in pre-specified responder analysis for visual acuity (p=0.008 to p=0.131) Changes in all four visual acuity endpoints correlate significantly with clinical benefit (ITT population p<0.001 to p=0.005)

 

Liestal, Switzerland, September 14, 2010 - Santhera Pharmaceuticals (SIX: SANN) announced today additional data and analyses of its RHODOS study in support for the use of Catena® in Leber's Hereditary Optic Neuropathy (LHON). These data show that Catena® can significantly improve color vision which is lost early in patients with LHON. The data also indicate that in pre-specified responder analyses, a higher portion of patients on Catena® had improved visual acuity compared to placebo and that changes in visual acuity outcomes significantly correlated with clinical benefit. The key findings of this first-ever placebo-controlled study in LHON will be presented today at the 2010 Meeting of the American Neurological Association (ANA) in San Francisco, California.

 

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).

 

 

Population

Analysis

Catena®

Placebo

p

Intent to Treat

Improvement in visual acuity

30 of 106 (28.3%)

10 of 58 (17.2%)

0.131

Worsening in visual acuity

18 of 106 (17.0%)

17 of 58 (29.3%)

0.075

Patients with discordant visual acuity at baseline (patients at risk of further vision loss)

Improvement in visual acuity

15 of 40 (37.5%)

1 of 20 (5.0%)

0.011

Worsening in visual acuity

8 of 40 (20.0%)

9 of 20 (45.0%)

0.067

Eyes unable to read letters on a standard chart at baseline

Could read at least 5 letters on the chart at week 24

12 of 61 (19.7%)

0 of 29 (0.0%)

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.

 

Population

Correlation

Spearman's R*

p

Intent to Treat

Best recovery in visual acuity vs CGIC

-0.32

0.005

Best visual acuity vs CGIC

-0.34

0.002

Change in visual acuity of best eye at baseline vs CGIC

-0.33

0.004

Visual acuity in all eyes vs CGIC

-0.32

<0.001

* Correlation Coefficient: negative value indicates that both visual acuity and CGIC improve.

 

References

[1] Oral presentation given at the Derek-Denny Brown New Member Symposium at the 135th Annual Meeting of the American Neurological Association, San Francisco, California, on September 14, 2010.

 

[2] Patrick Chinnery et al, Results of a 6-months randomized, placebo-controlled trial (RHODOS) with idebenone (Catena®) in Leber's Hereditary Optic Neuropathy (LHON). Poster WIP-3 presented at the 135th Annual Meeting of the American Neurological Association, San Francisco, California, on September 14, 2010.

 

About the RHODOS study

The RHODOS study (Rescue of Hereditary Optic Disease Outpatient Study) was a randomized, double blind, placebo controlled trial testing the efficacy and safety of Catena® and placebo over a six-month treatment period. The dose tested was 900 mg/day. A total of 85 patients were enrolled into the study and were randomized in a 2:1 ratio to receive either Catena® or placebo. At baseline, participants were between 14 and 64 years of age with loss of visual acuity not longer than five years prior to enrolment. The study was conducted at centers in Newcastle (UK), Munich (Germany) and Montréal (Canada).

 

About Leber's Hereditary Optic Neuropathy

Leber's Hereditary Optic Neuropathy (LHON) is an inherited atrophy of certain cells in the retina and optic nerves that leads to rapid loss of central vision and ultimately to blindness. Blurring of central vision and color desaturation usually mark the beginning of the symptomatic phase of this neuro-ophthalmological disorder. The effects of LHON are rapid and severe, typically leading to blindness within a few months of the onset. While symptoms initially develop in one eye, the second eye is usually involved within a few months. Patients are predominantly young adult males who typically have one of three different point mutations of the mitochondrial genome. These mutations lead to the reduction in cellular energy production, which in turn results in cell damage and death of certain optic nerve cells.

 

Catena® is the first drug that has ever been clinically investigated in a randomized, placebo-controlled intervention study in LHON. Given the drug's principal mode of action, Catena® may protect the retinal and optic nerve cells and thereby delay, lessen or prevent vision loss. LHON is found in all ethnic groups; an estimated 20,000 patients live in Europe and in the United States.

 

* * *

 

About Santhera

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of severe neuromuscular diseases, an area of high unmet medical need which includes many orphan indications with no current therapy. Santhera's first product, Catena® to treat Friedreich's Ataxia, is marketed in Canada. For further information, please visit www.santhera.com.

 

Catena® is a trademark of Santhera Pharmaceuticals.

 

For further information, contact

Thomas Meier, Chief Scientific Officer
Phone: +41 (0)61 906 89 64
thomas.meier@santhera.com

 

Barbara Heller, Chief Financial Officer
Phone: +41 (61) 906 89 54
barbara.heller@santhera.com

 

Thomas Staffelbach, Head Public & Investor Relations
Phone: +41 (0)61 906 89 47
thomas.staffelbach@santhera.com

 

Disclaimer/Forward-looking statements

This communication does not constitute an offer or invitation to subscribe for or purchase any secu­rities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, un­certainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

 

Posted: September 2010

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