Sylentis, a PharmaMar Group company, begins a new Phase II trial with SYL1801 for patients with Age-Related Macular Degeneration

Madrid, November 23rd, 2022. – Sylentis, a PharmaMar Group Company (MSE:PHM), has announced today the recruitment of the first patient in a new Phase II dose-ranging trial with its compound SYL1801, for the treatment of patients with neovascular Age-related Macular Degeneration (AMD).

This is a randomized, double-blind Phase II trial with three parallel groups in which different dose levels will be administered to each cohort of subjects. The study has been initiated already and will enroll a total of 90 patients at different centers in several European countries.

The primary endpoint of the trial is to evaluate the effect on visual acuity of SYL1801 at all three doses, administered as a daily drop for 6 weeks in patients with neovascular AMD. In addition, secondary endpoints on disease progression and safety of SYL1801 will be evaluated.

SYL1801 represents a potential breakthrough in the development of innovative drugs through gene silencing technology based on RNA interference (RNAi): its administration in the form of eye drops blocks the synthesis of the NRARP receptor (Notch-Regulated Antichiral Repeat Domain Proteins), which is involved in choroidal neovascularization (AMD pathology) and could change the standard of care for this disease.

Currently, neovascular AMD is treated with regular eye injections of anti-VEGF agents, a very uncomfortable procedure for patients and a large investment in resources for healthcare systems[i].

The application of SYL1801 in the form of eye drops could be a new therapeutic option for the treatment of retinal diseases involving a neovascularization processes.

Age-Related Macular Degeneration (AMD)

It is estimated that the number of individuals with AMD will reach 288 million in 2040. AMD is the leading cause of irreversible visual impairment in the elderly population of developed countries, accounting for 8% of all cases of blindness worldwide. The overall prevalence of AMD among 45-85 year olds is 8.7%[ii],[iii].

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About PharmaMar

PharmaMar is a biopharmaceutical company focused on the research and development of new oncology treatments, whose mission is to improve the healthcare outcomes of patients afflicted by serious diseases with our innovative medicines. The Company is inspired by the sea, driven by science, and motivated by patients with serious diseases to improve their lives by delivering novel medicines to them. PharmaMar intends to continue to be the world leader in marine medicinal discovery, development and innovation.

PharmaMar has developed and now commercializes Yondelis® in Europe by itself, as well as Zepzelca® (lurbinectedin), in the US; and Aplidin® (plitidepsin), in Australia, with different partners. In addition, it has a pipeline of drug candidates and a robust R&D oncology program. PharmaMar has other clinical-stage programs under development for several types of solid cancers: lurbinectedin and ecubectedin. Headquartered in Madrid (Spain), PharmaMar has subsidiaries in Germany, France, Italy, Belgium, Austria, Switzerland and The United States. PharmaMar also wholly owns Sylentis, dedicated to researching therapeutic applications of gene silencing (RNAi). To learn more about PharmaMar, please visit us at

About Sylentis

Sylentis is a pharmaceutical company that develops innovative therapies based on gene silencing technologies or RNAi. This technology enables the design of molecules capable of selectively inhibiting the synthesis of disease-causing proteins. Sylentis has developed numerous therapies based on this novel technology and currently has a strong program in ophthalmology with one candidate in Phase III clinical trials: tivanisiran, for the treatment of dry eye. Sylentis is also researching and developing other new products for the treatment of various eye diseases such as ocular allergies and retinal diseases. For more information, visit

About SYL1801

SYL1801 is an RNAi-based drug administered as eye drops that selectively inhibits NRARP receptor production. NRARP, by regulating the Notch and Wnt signaling pathways in endothelial cells, plays an important role in the control of choroidal neovascularization. SYL1801 is a synthetic small double-stranded RNA (siRNA) oligonucleotide that acts through a highly selective mechanism of action. Non-clinical studies conducted by Sylentis with SYL1801 have demonstrated high capacity to inhibit this target specifically. The reduction of its expression in the retina, observed in animal models, correlates with vessel regression and decreased retinal vascular growth following topical administration.

SYL1801 is a product under development to treat age-related macular degeneration, with potential for development for other pathologies involving choroidal neovascularization.


About RNA interference (RNAi)

RNA interference is a technology that seeks to reduce abnormal protein production by silencing messenger RNA. RNAi represents a breakthrough as a new mechanism of action to address numerous pathologies. Some pathologies, such as age-related macular degeneration, are caused by an alteration of certain proteins. This technology can act by reducing or controlling in a very specific way the production of the proteins involved in each pathology. Compounds based on RNAi technology tend to have a longer effect than traditional drugs and few side effects, due to their high specificity.


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[i] Sivaprasad, S. and S. Oyetunde, Impact of injection therapy on retinal patients with diabetic macular edema or retinal vein occlusion. Clin Ophthalmol, 2016. 10: p. 939-46.

[ii] Deng, Y., et al., Therapeutic potentials of gene silencing by RNA interference: principles, challenges, and new strategies. Gene, 2014. 538(2): p. 217-27.

[iii] Jonas, J.B., C.M.G. Cheung, and S. Panda-Jonas, Updates on the Epidemiology of Age-Related Macular Degeneration. Asia Pac J Ophthalmol (Phila), 2017. 6(6): p. 493-497.

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