Sylentis – Treatment commences for first patients in Phase I/II clinical trial with SYL040012

  • SYL040012 is a new compound developed in Sylentis’s R&D programme in ophthalmological disorders such as ocular hypertension and glaucoma.
  • SYL040012 is a form of RNA interference (RNAi).
  • It reduces intraocular pressure by inhibiting the expression of ß-adrenergic receptors.

Madrid, 22 November 2010: 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 commenced a Phase I/II clinical trial with SYL040012 for treating patients with ocular hypertension, after having received authorisation in August from the Spanish Medicines and Health Products Agency.

The goal of the Phase I/II trial is to establish the tolerance and effect of SYL040012 on intraocular pressure in patients with ocular hypertension. This is the second clinical trial with SYL040012; Sylentis is the first company to perform a clinical trial in Spain based on RNAi.

The Phase I/II trial with SYL040012 will be performed at the Navarra University Clinic and the Ramón y Cajal University Hospital in Madrid on patients with intraocular pressure of 21 mm Hg or greater.

After assessing the very favourable results obtained in Phase I, which established the safety of SYL040012 in healthy volunteers, Sylentis is continuing with development of the compound.

About SYL040012

SYL040012 is a specific RNAi which targets the β-2 adrenergic receptor. It has proven effective in diminishing intraocular pressure in animal models in vivo and also in preventing glaucoma, proving to be superior to the drugs used in the control branch of the trial.

About Sylentis

Founded in 2006 as a spin-off from Grupo Zeltia subsidiary Genómica, S.A.U., Sylentis is a subsidiary of Grupo Zeltia and a pioneer in Spain in the search for new therapies based on interference RNA (RNAi). The company aims to design effective short interfering RNAs (siRNAs) using bioinformatic programmes such as SIRFINDER®, which optimises the design of siRNAs with pharmacological potential.

Sylentis initially focused on developing therapies to treat glaucoma/ocular hypertension, conditions which are addressed by the company’s most advanced compounds at present.
It also has other research lines whose compounds are in the R&D and preclinical phases.

About interference RNA (RNAi)

RNAi has arisen in recent years as a promising technology with therapeutic applications, and was recognised worldwide when its discoverers, Fire and Mello, received the Nobel Prize in 2006. Discovered in plants in the 1990s, RNAi consists of highly efficient selective and specific inhibition of gene expression. RNAi is mediated by small fragments of double-stranded RNA, consisting of 19-23 nucleotides, which promote degradation of messenger RNA (mRNA), thus inhibiting synthesis of the proteins for which they code and which are responsible for the pathology. RNAi therapy has great potential as this mechanism is used naturally by cells to regulate gene expression in a way that is both non-toxic and highly effective.

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(*) This note is also available on the Sylentis website: www.sylentis.com and on the Zeltia website:www.zeltia.com