Sylentis announces new preclinical and clinical data for its SYL1001 molecule during ARVO 2017

  • SYL1001 is a compound under investigation for the treatment of dry eye disease.
  • It is a product based on the novel technology RNA interference (RNAi), developed for the treatment of signs and symptoms of this illness.
  • At the Annual Congress of the Association for Research in Vision and Ophthalmology 2017, around 11,000 attendees will meet form the 7-11 of May in Baltimore, USA.

Madrid, May, 9th, 2017. In the framework of the Annual Congress of the Association for Research in Vision and Ophthalmology 2017 (ARVO), being held from the 7-11 of May in Baltimore (USA), Sylentis, a pharmaceutical company from the PharmaMar Group (MSE:PHM), presents new preclinical and clinical developments with its molecule SYL1001 for the treatment of dry eye disease. This is a product based on the novel technology RNA interference (RNAi), developed for the treatment of the signs and symptoms of this pathology.

This Congress will bring together more than 11,000 top eye and vision researchers and clinicians from around the world to explore cutting-edge basic and clinical science. “We are proud to be able to present our results to such a well prepared audience. We trust our technology, innovative in its field, and we hope that SYL1001 will soon be a real alternative for treating millions of people that suffer from dry eye disease around the world”, states Ana Isabel Jiménez, R&D Director of Sylentis.

Accordingly, Sylentis participates at this event with the presentation of data from various clinical and preclinical trials. On one hand, the stability results of SYL1001 in different commercial containers. The stability of the compound in various containers and conditions through time concludes that all the formats analyzed guarantee the integrity and specifications of the product.

On the other hand, Sylentis has presented the results of the study of this product in animal models, to evaluate its efficacy in the treatment of the symptoms and signs of dry eye disease. These patients have a deficit of mucine, a protein found in tears, a low density in the goblet cells and inflammation of the ocular surface. These studies have concluded that SYL1001, apart from reducing the feeling of ocular discomfort, it also improves the quality of the tear and reduces inflammation. In animal models, the statistical increase in the levels of mucine (MUC5A+) present on the eye´s surface has also been demonstrated, allowing for the formation of a tear film. In this same study, an increase in the density of goblet cell (those producing and secreting mucine) and also in tear secretion was observed. The inflammatory mediators of the eye surface also decreased after treatment.

Finally, the Company presents a third study that demonstrates the existing correlation between the preclinical and clinical results in the search for the optimum and most efficient dose. Both, in animal and human models, the concentration of 1.125% of SYL1001 was observed to be the most efficient, along with a very low incidence of adverse events.

Principle studies of Sylentis to be presented at ARVO 2017:

  • Stability study of SYL1001 eye drops (a siRNA compound) for DED in different containers (Posterboard Number: 476 – A0401)
    Session: 7th of May of 2017, from 13:30 to 15:15 hours. Lead author: Verónica Ruz, et al. Regulatory Affairs, Sylentis, Spain.
  • Efficacy of SYL1001 in different animal models of Dry Eye Disease (Posterboard number: 458 – A0383)
    Session: 7th of May of 2017, from 13:30 to 15:15 hours. Lead author: Ana Isabel Jiménez et al. R&D, Sylentis, Spain.
  • Clinical and preclinical study correlation for SYL1001, a new treatment for dry eye disease (Posterboard number: 2670 – A0260)
    Session: 9th of May of 2017, from 8:30 to 10:15 hours. Lead author: María Victoria González et al. Clinical Department, Sylentis, Spain.

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.

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

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.

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