MADRID, March 14, 2016 /PRNewswire/ --
Sylentis, a pharmaceutical company in the PharmaMar Group
(MSE:PHM) and a pioneer in the research and development of new
drugs based on gene silencing (interference RNA, RNAi), has
presented the results of two Phase II dose-finding and efficacy
assessment clinical trials (SYL1001_II and SYL1001_III) with the
investigational medicinal product SYL1001 for treating ocular
discomfort related to dry eye syndrome.
(Logo:
http://photos.prnewswire.com/prnh/20160314/343630LOGO )
These randomised parallel group, double-masked and placebo
controlled Phase II trials took place at 8 centres in two European
countries: Spain and Estonia. A total of 127 patients with ocular
pain related to dry eye syndrome took part in the trials, which
assessed safety and efficacy of four doses of SYL1001 (0.375%,
0.75%, 1.125% and 2.25%) against placebo following 10 days of
once-per-day administration in the form of eye drops.
The results revealed that 1.125% was an optimal dose which
achieved the best primary and secondary endpoints, reducing not
only ocular pain but also conjunctival hyperaemia related to dry
eye syndrome.
The two trials also confirmed a favourable safety and tolerance
profile of SYL1001, previously observed in Phase I trial, with no
differences in the percentage of adverse events between the
assessed doses of SYL1001 and placebo group.
"These positive results support continuing clinical development
of SYL1001. Sylentis is currently designing the Phase III clinical
program which it will be validated with the relevant regulatory
authorities," said Dr Ana Isabel Jiménez, COO of Sylentis.
The results and additional analysis of these clinical trials
will be presented at the ARVO conference in May 2016.
About SYL1001
SYL1001 is a drug based on RNAi that is administered as
preservative-free eye drops; it selectively inhibits production of
the TRPV1 receptor. These receptors are ion channels that mediate
the transmission of ocular pain. SYL1001 is a small synthetic
double-stranded RNA oligonucleotide (siRNA) with a novel and highly
selective mechanism of action. Non-clinical studies conducted by
Sylentis with SYL1001 have demonstrated it has high ability to
inhibit this specific target and block the perception of ocular
pain in animals[i].
SYL1001 is a product undergoing development for the treatment or
prevention of ocular pain related to with dry eye syndrome, and has
potential to be developed for other pathologies that cause ocular
pain (corneal lesions, refractive surgery,
etc.)[ii],[iii],[iv].
About RNA interference (RNAi)
RNA interference (RNAi) is a natural cellular process that
regulates the expression of certain genes, providing a role in
innate defense and development in animal and plants. This process
is used to specifically silence genetic transcripts that encode
protein-causing diseases. The therapeutic application of targeted
siRNAs is booming given the specificity of gene silencing for a
particular protein in a given tissue and the lack of side effects.
This new approach to drug discovery is a promising technology that
is rapidly moving in the translational research
space[v].
About Sylentis
Sylentis, a company of PharmaMar (MSE:PHM), is a biotechnology
company fully owned that develops innovative therapies harnessing
the technology of post-transcriptional gene silencing or RNA
interference (RNAi). Sylentis has developed an approach to
efficiently design RNAi-based therapeutics that can be used to
silence numerous disease-causing genes. We currently have a robust
therapeutic program in ophthalmology[vi] with two
candidates under development in Phase II studies for glaucoma
(bamosiran) and ocular pain
(SYL1001)[ii],[iii],[iv].
Sylentis is also developing new products for the treatment of
several eye diseases such as ocular allergies and retina diseases.
To know more about us, please visit us at
http://www.sylentis.com.
___________________________________________
i. Martínez-Garcia C, Martínez T, Pañeda C, Gallego P, Jimenez
AI, Merayo J. Differential expression and localization of transient
receptor potential vanilloid 1 in rabbit and human eyes. Histol
Histopathol, 2013, 28(11):1507-16.
ii. Pañeda C, González V, Martínez T, Ruz V, Vargas B and
Jiménez AI. RNAi based therapies for ocular conditions. In
Proceedings of the 11th ISOPT, 2014, 25-30, Medimond, Bologna,
Italy.
iii. Martínez T, Jiménez AI, Pañeda, C. Short-interference RNAs:
becoming medicines. EXCLI Journal, 2015;14:714-46.
iv. Martínez T, González MV, Vargas B, Jiménez AI, Pañeda C.
Preclinical Development of RNAi-Inducing Oligonucleotide
Therapeutics for Eye Diseases. In RNA interference. ISBN:
978-953-51-4614-8. Ed. Intech. 2015.
v. The definition and classification of dry eye disease: report
of the Definition and Classification Subcommittee of the
International Dry Eye WorkShop (2007). Ocul Surf, 2007. 5(2): p.
75-92.
vi. Moreno-Montañés J, Sádaba B, Ruz V, Gómez-Guiu A, Zarranz J,
González MV, Pañeda C, Jimenez AI. Phase I Clinical Trial of
SYL040012, a Small Interfering RNA Targeting β-Adrenergic Receptor
2, for Lowering Intraocular Pressure. Mol Ther. 2014,
22(1):226-32.
Media Inquiries:
Paula Fdez. Alarcón - Media Relations Manager
(+34-638-79-62-15)
Investor Relations:
Telephone number: +34-914444500 or visit http://www.sylentis.com
and http://www.pharmamar.com