The World Health Organization selects tivanisiran as the International Nonproprietary Name for SYL1001

  • Tivanisiran is under investigation in a Phase III clinical trial, HELIX, in more than 30 European hospitals and 300 patients to determine its efficacy in dry eye syndrome


Madrid, October 19th, 2017.- Sylentis, a Pharmaceutical Company of PharmaMar Group (PHM:MSE), pioneer in the development of new compounds based on genetic silencing technology through RNA interference (RNAi), has announced that the World Health Organization (WHO) has given its approval, as disclosed in paragraph 7 of the International Nonproprietary Name in Pharmaceutical Substances (INN) selection procedure. This entails the designation of tivanisiran as the chosen INN for the compound SYL1001. The inclusion of a denomination on the Recommended Common Denominations list does not imply any endorsement at all in favor of any medical or pharmaceutical use of the drug.

Tivanisiran is an important step forward in the development of innovative compounds in the field of ophthalmology through the technology called genetic silencing, based on RNAi. Tivanisiran is administered in ophthalmic drops by the patients themselves. The Phase III trial, called HELIX, is being carried out in more than 30 hospitals in Spain, Germany, Estonia, Portugal and Italy, in 300 patients to evaluate the efficacy of this drug in the treatment of the signs and symptoms of dry eye syndrome[i]. This is an ocular pathology that, in Spain, affects more than 5 million people[ii],[iii], between 10% and 20% of the population, women in particular, and almost 100% are elderly.

The obtention of the International Nonproprietary Name for SYL1001 has been published in the World Health Organization´s last report on International Nonproprietary Names for Pharmaceutical Substances (INN). “The assignment of tivanisiran implies one step more in this molecule development and we trust in its potential as a possible therapeutic alternative to respond in a novel and efficient manner to the necessities of the patients with dry eye syndrome”, as Ana Isabel, COO and R&D Director of Sylentis, explains.

Additionally, Sylentis is also working on another compound SLY136001, in ophthalmic drops and based on the same RNAi technology, for the treatment of the degenerative retina diseases that are identified by neovascularization, such as macular degeneration, associated to age and also diabetic retinopathy.

Recently, at the Oligonucleotide Therapeutics Society 2017 Annual Meeting, the preclinical efficacy data for this product externally applied in animal models with molecular degeneration were presented. Up to now the standard treatments consist in intraocular injections, therefore this topical treatment would improve the patients´ quality of life.

Sylentis´ pipeline is directed at indications in the field of ophthalmology, with the purpose of developing innovative medication based on RNAi, that increase todays therapeutic possibilities.

For more information about the clinical trial:

For more information (only available in Spanish)
What is RNA interference?
Dry eye syndrome:

About tivanisiran (SYL1001)
Tivanisiran is a drug based on RNAi that is administered as preservative-free eye drops; it selectively inhibits production of the transient receptor potential cation channel (TRPV1). These receptors are ion channels that mediate the transmission of ocular pain. Tivanisiran 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 animals3.

Tivanisiran is a product under development for the treatment of signs and symptoms related to dry eye syndrome, and has the potential to be developed for other pathologies that cause ocular pain (corneal lesions, refractive surgery, etc.)6,[iv],[v],[vi].

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 animals 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[vii],[viii].

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 10% and 20% among people aged 50 or over, and it is more frequent in women1,2.

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 analgesics 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  and III studies for glaucoma (bamosiran)[ix] and ocular pain (SYL1001)11, respectively. 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.


[i] Benitez-Del Castillo JM, Protocol No.: SYL1001_IV. EUDRACT No: 2016-003903-79. A double-masked study of SYL1001 in patients with moderate to severe dry eye disease (DED). HELIX Study (Phase III). Version 1.1: December 14th, 2016. Sylentis SAU-Pharma Mar Group


[iii] Craig, J.P., et al., Tear Film & Ocular Surface Society. International Dry Eye WorkShop DEWS II Definition and Classification Report. Ocul Surf, 2017: p. 269-649.

[iv] Martinez-Garcia C, Martinez 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

[v] Martinez T, Gonzalez MV, Vargas B, Jimenez 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

[vi] Benitez-Del-Castillo JM, Moreno-Montañés J, Jimenez-Alfaro I, Muñoz-Negrete FJ, Turman K, Palumaa k, Sádaba B, Gonzalez MV, Ruz V, Vargas B, Pañeda C, Martinez T, Bleau AM, Jimenez AI. Safety and Efficacy Clinical Trials for SYL1001, a Novel Short Interfering for the treatment of Drye Eye Disease. Invest Ophthalmol Vis Sci. 2016 Nov 1;57(14):6447-6454

[vii] Elbashir SM1, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 2001 May 24;411(6836):494-8

[viii] Soutschek J1, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, Elbashir S, Geick A, Hadwiger P, Harborth J, John M, Kesavan V, Lavine G, Pandey RK, Racie T, Rajeev KG, Röhl J, Toudjarska I, Wang G, Wuschko S, Bumcrot D, Koteliansky V, Limmer S, Manoharan M, Vornlocher HP. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature. 2004 Nov 11;432(7014):173-8

[ix] Moreno-Montañes J, Sádaba B, Ruz V, Gomez-Guiu A, Zarranz J, Gonzalez 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

error: Content is protected !!