Nav: Home

Sylentis reports positive Phase II results with SYL1001 in treating ocular pain

March 14, 2016

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.

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 the SYL1001_II (NCT01776658) and SYL1001_III (NCT02455999) trials


SYL1001_II/III are dose-finding, multi-centre randomised parallel-group double-masked placebo-controlled Phase II clinical trials to evaluate the efficacy and safety of SYL1001 in patients with ocular pain related to dry eye syndrome after ten days of treatment (one drop per day in each eye).

SYL1001_II included 61 patients at 5 centres in Spain, divided into three groups of 20 patients each, who received SYL1001 (1.125% or 2.25%) or placebo. SYL1001_III included 66 patients at 5 centres in Spain and Estonia, divided into three groups of approximately 22 patients each and treated with SYL1001 (0.375% or 0.75%) or placebo.

Primary endpoints evaluated were dry eye symptoms using OSDI (Ocular Surface Disease Index) and VAS (Visual Analogue Scale), and signs associated with this pathology (conjunctival hyperaemia and corneal fluorescence staining) after ten days of treatment.

Secondary endpoints included assessment of vital signs, blood and urine analysis, and alteration of ocular parameters (IOP, TBUT, Schirmer test, visual acuity, and assessment of the anterior segment) together with the appearance of adverse events as a measure of tolerance.

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 . 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.).

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 .

About dry eye syndrome


Dry eye syndrome is a multifactorial disease of the tear film and ocular surface that produces symptoms of ocular discomfort, eyesight disorders, and tear film instability with potential damage to the ocular surface. Dry eye syndrome is accompanied by such symptoms as ocular pain, itching, stinging, and irritation of the eye tissues. It is a characteristic disease of developed countries, associated with pollution, air conditioning, the use of contact lenses, refractive surgery and continued use of computers. Moreover, the amount and quality of tears decrease with age. Prevalence is between 5% and 30% among people aged 50 or over, and it is more frequent in women .

Dry eye can be treated with cyclosporin drops or autologous serum, but there is as yet no specific product for chronic treatment of the ocular pain related to dry eye syndrome; oral analgaesics or anaesthetics are used in general. However, the main treatment consists of artificial tears, in the form of drops, gel or creams. Preservative-free eye drops have generally been found to offer the best long-term response.
-end-
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 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.

Disclaimer


This document is a press release, not a prospectus. This document does not constitute or form part of an offering or invitation to sell or a solicitation to purchase, offer or subscribe shares of the company. Moreover, no reliance should be placed upon this document for any investment decision or contract and it does not constitute a recommendation of any type with regard to the shares of the company.

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

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.

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. Martínez T, Jiménez AI, Pañeda, C. Short-interference RNAs: becoming medicines. EXCLI Journal, 2015;14:714-46.

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. 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.

Zimmermann, T.S., et al., RNAi-mediated gene silencing in non-human primates. Nature, 2006. 441(7089):111-4. 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.

Pharmamar

Related Achieved Articles:

Findings suggest reducing target SBP to below recommended levels could significantly reduce risk
Reducing systolic blood pressure (SBP) to levels below currently recommended targets may significantly reduce the risk of cardiovascular disease (CVD) and all-cause death, according to a study published by JAMA Cardiology.
A potential cure for metastatic prostate cancer? Treatment combination shows early promise
In the past, all forms of metastatic prostate cancer have been considered incurable.
Weekly diabetes drug provides similar benefits to daily version
A weekly dipeptidyl peptidase-4 (DPP-4) inhibitor was just as effective at controlling type 2 diabetic patients' blood sugar as a daily DPP-4 inhibitor in a recent randomized clinical trial.
Manchester scientists tie the tightest knot ever achieved
Scientists at The University of Manchester have produced the most tightly knotted physical structure ever known -- a scientific achievement which has the potential to create a new generation of advanced materials.
First experimental evidence of 3-D aromaticity in stacked antiaromatic compounds
Researchers centered at Nagoya University found that antiaromatic planar norcorrole molecules can form close face-to-face interactions to give structures with increased aromaticity.
Efforts are needed to protect native species from feral cats
Feral cats are among the most damaging invasive species worldwide, particularly in Australia where they have caused the extinction of more than 20 mammal species.
Importance of universal sanitation underestimated in efforts to reduce child mortality
The value of sanitation at reducing child mortality in many low income countries has been substantially underestimated according to recent research.
Quantum computers: 10-fold boost in stability achieved
Australian engineers have created a new quantum bit which remains in a stable superposition for 10 times longer than previously achieved, dramatically expanding the time during which calculations could be performed in a future silicon quantum computer.
Simplified approach to drug development with Upsalite
For the first time, researchers have revealed the nanostructure of the mesoporous magnesium carbonate Upsalite® and pore size control was achieved without organic templates or swelling agents.
Diabetes prevention programs beneficial in improving cardio-metabolic profiles
A new study by researchers at Emory's Rollins School of Public Health and the Centers for Disease Control and Prevention (CDC) shows that lifestyle modification programs modeled on diabetes prevention programs (DPP) trials not only achieved weight reduction, but also additional metabolic benefits -specifically, reductions in blood sugar, blood pressure, and cholesterol levels.

Related Achieved Reading:

Best Science Podcasts 2019

We have hand picked the best science podcasts for 2019. Sit back and enjoy new science podcasts updated daily from your favorite science news services and scientists.
Now Playing: TED Radio Hour

Changing The World
What does it take to change the world for the better? This hour, TED speakers explore ideas on activism—what motivates it, why it matters, and how each of us can make a difference. Guests include civil rights activist Ruby Sales, labor leader and civil rights activist Dolores Huerta, author Jeremy Heimans, "craftivist" Sarah Corbett, and designer and futurist Angela Oguntala.
Now Playing: Science for the People

#521 The Curious Life of Krill
Krill may be one of the most abundant forms of life on our planet... but it turns out we don't know that much about them. For a create that underpins a massive ocean ecosystem and lives in our oceans in massive numbers, they're surprisingly difficult to study. We sit down and shine some light on these underappreciated crustaceans with Stephen Nicol, Adjunct Professor at the University of Tasmania, Scientific Advisor to the Association of Responsible Krill Harvesting Companies, and author of the book "The Curious Life of Krill: A Conservation Story from the Bottom of the World".