Santhera Presents Nonclinical Data in LHON at the Joint Congress of SOE/AAO 2011
Category: BioValleyLiestal, Switzerland - Santhera Pharmaceuticals (SIX: SANN) announced that efficacy data of Catena in a rodent animal model of Leber's Hereditary Optic Neuropathy (LHON) have been presented at the 2011 Joint Congress of the European Society of Ophthalmology (SOE) and the American Academy of Ophthalmology (AAO) in Geneva, Switzerland [1].
In line with clinical data, Catena demonstrated protection from retinal ganglion cell death and other LHON-typical retinal pathologies.
Intraocular injection of rotenone in mice results in retinal pathology comparable to the clinical presentation of LHON. Oral treatment of animals with idebenone (Catena) protected from retinal ganglion cell death and preserved retinal thickness after rotenone-injection. Furthermore, in line with the results obtained for retinal ganglion cell survival and retinal thickness, Catena suppressed elevated gliosis, a marker for cellular stress.
Rotenone is a potent inhibitor of the complex I of the mitochondrial electron transport chain. Experiments applying this rotenone-mediated retinal pathology model were conducted in collaboration with scientists at the Brain Research Institute of the Swiss Federal Institute of Technology (ETH Zurich), Switzerland.
"The animal model data presented today support the clinical efficacy data obtained from the RHODOS study and confirm our understanding of Catena®'s mode of action. The drug bypasses the deficient complex I of the mitochondrial electron transport chain, providing an alternative path for cellular energy production in retinal ganglion cells with complex I deficiency as in LHON", commented Thomas Meier, Chief Scientific Officer of Santhera. "Combined with data from the RHODOS study these data support our regulatory filing for marketing approval in Europe."
Results from the clinical study (RHODOS) were also presented at the SOE/AAO conference [2]. Data show that Catena can protect vision of patients, who are at highest risk of vision loss, and enhance the recovery of visual acuity in severely affected patients. These data are supported by secondary, clinically meaningful efficacy variables such as color contrast sensitivity, changes in retinal nerve fiber layer thickness and clinical benefit. In the RHODOS study, Catena showed consistent clinically relevant superiority over placebo.
References:
[1] Heitz F, Anklin C, Guven N: Neuroprotective activity of idebenone in a mouse model of Leber's hereditary optic neuropathy (LHON). Oral presentation given at the Joint Congress of the European Society of Ophthalmology (SOE) and the American Academy of Ophthalmology (AAO) in Geneva, June 4 to 7, 2011.
[2] Chinnery PF, Dimitriadis K, Rouleau J, Yu-Wai-Man P, Heck S., Atawan A, Chattopadhyay S, Bailie M, Schubert M, Rummey C, Metz G, Leinonen M, Griffiths PG, Andrews WT, Meier T,
Klopstock T: Efficacy and safety results of a 6-month randomized, placebo controlled study (RHODOS) of idebenone in patients with Leber' Hereditary Optic Neuropathy (LHON). Oral presentation given at the Joint Congress of the European Society of Ophthalmology (SOE) and the American Academy of Ophthalmology (AAO) in Geneva, June 4 to 7, 2011.
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 and neurodegenerative diseases, an area of high unmet medical need which includes many orphan and niche indications with no current therapy. Santhera's first product, Catena to treat Friedreich's Ataxia is marketed in Canada.
For further information, contact
Thomas Meier, Chief Scientific Officer
Phone: +41 (0)61 906 89 64
Thomas Staffelbach, Head Public & Investor Relations
Phone: +41 (0)61 906 89 47
thomas.staffelbach@santhera.com
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