Sylentis, a PharmaMar Group Company (MSE:PHM), has announced today that it is presenting at ARVO 2023 advances on two of its ophthalmic studies based on RNA interference (RNAi) technology: Tivanisiran, for Dry Eye Disease (DED); and SYL1801, for retinal diseases such as Age-related Macular Degeneration (AMD). The Company is also displaying a poster with non-clinical data regarding an improved animal model for Choroidal Neovascularization (CNV). ARVO 2023 is being held in New Orleans, Louisiana (USA) from April 23 – 27.
Among the studies to be presented, is the abstract entitled “Long-term safety studies of tivanisiran for the treatment of Dry Eye Disease (DED)” which describes tivanisiran, a small interfering ribonucleic acid (siRNA), administered in eye drops to treat signs and symptoms of this ocular surface disease. Tivanisiran has shown local and systemic tolerability in non-clinical and clinical studies, which supports the completion of the development program.
The abstract “Stability study of a preservative-free formulation of SYL1801, a siRNA indicated for neovascular AMD, in Novelia® multidose eye dropper” presents SYL1801, a new chemical entity based on RNAi technology, developed by Sylentis to treat Choroidal Neovascularization (CNV), in patients with wet AMD. Currently, the compound is being evaluated in a Phase II clinical trial.
The abstract “Optimization and characterization of an improved laser-induced CNV animal model for the study of retinal diseases” recapitulates the CNV process, which causes retinal edema and subsequent damage to photoreceptor cells. Currently, treatments directed against vascular endothelial growth factor (anti-VEGF) are administered intravitreally to slow the progression of blindness in wet AMD. However, 30% of patients requiere continuous injections are needed to maintain visual acuity. Depending on the laser treatment, relevant biomarkers showed different behaviors over time and ocular tissues. This experimental context defines novel possible scenarios to study, not only new therapeutic agents but also alternative mechanisms of action different to traditional antibody-based angiogenic therapies (such as SYL1801 eye drops).
|Tivanisiran||Long-term safety studies of Tivanisiran for the treatment of Dry Eye Disease (DED)||Anne Marie Bleau, PhD||ABSTRACT: 3954-B0271
POSTER SESION: 418
|SYL1801||Stability study of a preservative-free formulation of SYL1801, a siRNA indicated for neovascular AMD, in Novelia®multidose eye dropper||Verónica Ruz, PhD||ABSTRACT:
POSTER SESION: 139
|SYL1801||Optimization and characterization of an improved laser-induced choroidal neovascularization animal model for the study of retinal diseases||Ana Isabel Jiménez-Antón, PhD||ABSTRACT: 2112-C0065
POSTER SESION: 252
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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 www.pharmamar.com.
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 www.sylentis.com.
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.
Tivanisiran sodium is an RNAi-based drug administered as preservative-free eye drops that selectively inhibits the production of transient receptor potential type 1 (TRPV1). These receptors are ion channels that mediate the transmission of pain and inflammation at the ocular level. Tivanisiran sodium is a synthetic small double-stranded RNA (siRNA) oligonucleotide that acts by a novel and highly selective mechanism of action. Tivanisiran is currently under Phase 3 of clinical development to treat the signs and symptoms of dry eye disease, and with the potential to be developed for other pathologies that cause eye pain (corneal injury, refractive surgery, etc.).
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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