Sylentis Completes Phase I Trial With SYL040012 To Treat Elevated Intraocular Pressure And Glaucoma

Main Category: Eye Health / Blindness
Also Included In: Clinical Trials / Drug Trials;  Pharma Industry / Biotech Industry
Article Date: 09 Jul 2010 - 2:00 PDT

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Sylentis, a biopharmaceutical subsidiary of Grupo Zeltia (MC: ZEL) and a pioneer in the research and development of new drugs based on gene silencing (interference RNA, RNAi), has completed Phase Ia of its first clinical trial with SYL040012 in the form of ophthalmic drops to treat elevated intraocular pressure and glaucoma.

This is the first product based on RNAi technology to be developed clinically in Spain.

The trial received approval from the Spanish Medicines and Health Products Agency in June 2009, and was conducted by specialists in pharmacology and ophthalmology at Navarra University Clinic.

The trial's endpoint was to determine the tolerance and safety of SYL040012 ophthalmic drops; it was administered to 30 healthy volunteers aged 18 to 33. Patients showed excellent local and systemic tolerance to SYL040012, leading to very positive trial results. Sylentis plans to commence a Phase I/II clinical trial in patients with elevated intraocular pressure as soon as possible.

About SYL040012

In preclinical trials with SYL040012, the siRNAs administered topically to treat ocular hypertension associated with open angle glaucoma have proven effective both in vitro and in vivo. In those trials, SYL040012 effectively prevented the increase in intraocular pressure induced in the ocular hypertension model that was used. Ocular tolerance studies have shown excellent local and systemic tolerance to the administration of SYL040012 in much larger doses than required for therapeutic effectiveness.

About Sylentis

Founded in 2006 as a spin-off from Grupo Zeltia subsidiary Genomica, S.A.U., Sylentis is a subsidiary of Grupo Zeltia and a key player in the search for new therapies based on interference RNA (RNAi). Its strategy focuses on the efficient design of siRNAs using proprietary technology: SIRFINDER(R), which finds small fragments of RNAi (short interfering RNAs, siRNAs) with pharmaceutical potential by searching the appropriate sequences using bioinformatics; once the disease's target gene has been identified, SYLENTIS develops a quick and economical solution for siRNAs to silence that gene.

About interference RNA (RNAi)

Interference RNA (RNAi) has arisen in recent years as a promising technology with therapeutic applications. Discovered in plants in the 1990s, RNAi consists of highly efficient selective and specific inhibition of gene expression (Fire et al., 1998). Interference RNA is mediated by small fragments of double-stranded RNA, consisting of 19-23 nucleotides, which promote degradation of mRNA, thus inhibiting synthesis of the proteins for which they code. As this mechanism is used naturally by cells to regulate gene expression in a way that is both non-toxic and highly effective, RNAi has great therapeutic potential.

About open angle glaucoma

Primary open-angle glaucoma (POAG) is the most prevalent form of glaucoma, accounting for approximately two-thirds of all diagnosed cases of glaucoma. It is defined as a multifactorial optic neuropathy consisting of a loss of retinal ganglion cells and characteristic atrophy of the optical nerve leading to progressive, irreversible blindness. The risk factors of POAG include high intraocular pressure (IOP), a family history of the disease, and old age (Marquis and Wilson, 2005). Although the physiopathological mechanisms by which high pressure leads to neuron damage are not known, most current therapies seek to reduce IOP to a level that prevents progressive loss of vision.

Source: Zeltia

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