Bamosirán ( Syl040012 ) es uno de los 11 Farmacos Basados en la Novedosa Tecnología RNAi que Figura en un Nuevo Informe Publicado ... Según el Cual Dichos Farmacos Podrían Superar la Cantidad de Tres Billones de $$ by 2018 .

* A fecha de hoy aún no hay en el Mercado ninguna terapia basada en la RNAi .

* En este informe se centran en los 11 Farmacos con más posibilidades de salir al mercado y en el monto que podrían generar hasta 2018 .

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Wednesday April 2014, Amsterdan .

RNAi drugs market could exceed $3bn by 2018, predicts new study .

A new report forecasts the world market for RNAi therapeutics, covering both optimistic and pessimistic scenarios in those medicines’ future sales. Revenues there could exceed $3bn in 2018. That forecast and others appear in RNAi for Therapeutic Applications: Technology and Market 2014-2024, published in February 2014. That analysis predicts the future of the overall RNAi market and leading RNAi-based medicines in clinical development.

The new analysis predicts the overall market for RNAi-based drugs will expand from 2015 to 2024, with high revenues possible. Future sales performances of ribonucleic acid interference (RNAi) therapies depend on their safety and efficacy compared with the available standard of care.

Hemant Mistry, a pharmaceutical industry analyst, said: “At present, in early 2014, there exist no RNAi-based therapeutics on the market. However, there has been great interest in this technology owing to its selective and targeted mode of action. Also the high specificity and potency of RNAi allows lower doses to be administered than for other therapies used in the same indication. This is an important factor in a therapeutic setting.

“The biggest challenge drug developers face there is with drug delivery. Naked RNA-based agents are reportedly unstable in circulation and vulnerable to degradation. Also, once inside the cells, they may induce innate immune responses, which could lead to eventual cell death. Therefore specialised drug delivery systems need to be developed in tandem with the drugs, to overcome this issue.”

The new report shows optimistic and conservative revenue forecasts to 2024 at overall world, product and national level.


It estimates sales of 11 RNAi-based drugs in clinical development, including these:

■ PF-655

■ QPI-1007

■ SYL040012

■ ALN-RSV01

■ ARC-520

■ SPC3649 (Miravirsen).




That study’s research, data and analyses cover activities of Alnylam Pharmaceuticals, Arrowhead Research Corporation, RXi Pharmaceuticals, Silence Therapeutics, Tekmira Pharmaceuticals and other companies. It also contains interviews with that industry.

...

Investigational Bamosiran Significantly Reduces Intraocular Pressure in Patients With Glaucoma .

Presented at ARVO May 11th, 2014 .
Vanessa Caceres

ORLANDO, Fla -- May 11, 2014 -- A dose of a novel RNAi-based compound called bamosiran (SYL040012) significantly reduced intraocular pressure (IOP) in patients with glaucoma, researchers said here at the 2014 Annual Meeting of the Association for Vision and Research in Ophthalmology (ARVO).

Victoria Gonzalez, Sylentis, Madrid, Spain, and colleagues analysed tolerability and the IOP lowering effect of 3 different doses of bamosiran eye drops given every day over 14 days in patients with increased IOP associated with glaucoma.

For the phase 2, double-blind, multicentre study, a total of 89 patients were randomised to receive bamosiran 0.2% 80 mcg/eye/day (0.2%), bamosiran 0.75% 300 mcg/eye/day, bamosiran 2.25% 900 mcg/eye/day, or placebo.

Researchers evaluated local tolerability as assessed by conjunctival and corneal examinations daily; systemic tolerability, and effect on IOP, the latter of which was evaluated with performance of a 24-hour IOP curve prior to the first administration and then on day 14.

Bamosiran 300 mcg/eye/day led to a significant reduction in IOP at day 14 compared with placebo. “This reduction was also statistically significant when compared with the IOP curve performed during the screening period,” said Dr. Gonzalez.

Only 14.6% of patients reported an adverse event and 80% of those were mild in intensity. Headache was the most frequently reported adverse event.

“The only severe adverse event registered throughout the clinical trial was hyponatremia in 1 patient treated with bamosiran 300 mcg, but this event was not considered to be related to the investigational product,” Dr. Gonzalez reported.

The authors concluded that bamosiran was safe and well-tolerated and did not have typical beta blocker side effects such as heart rate reduction.

[Presentation title: Phase 2 of Bamosiran (SYL040012), a Novel RNAi-Based Compound for the Treatment of Increased Intraocular Pressure Associated to Glaucoma]

Bamosirán ( SYL040012 ) . Ensayo de Fase II en Pacientes con Hipertensión Ocular o Glaucoma de Ángulo Abierto .

Estudio Multicéntrico Realizado en 11 Centros :

6 España, 3 Alemania y 2 de Estonia.

Bamosirán (SYL040012) es un siRNA:

-Entra en la célula epitelio pigmentario de c. ciliar.
-Silencia el gen de la producción del humor acuoso.


*.- Patente de Sylentis (Grupo ZELTIA).



*.- Estudios previos han demostrado en humanos :

-Buena tolerancia local y sistémica.
-Efecto sobre la presión intraocular.

CONCLUSIONES :

*.- El estudio europeo fase II del Bamosiran indica que la dosis de 300 μg/día disminuye significativamente la PIO comparada:

-PIO basal.
-PIO del grupo con placebo.

*.- La tolerancia sistémica es muy buena con muy pocos efectos adversos.

...

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Bamosirán es un compuesto novedoso, resultante de la investigación de Sylentis en afecciones oftalmológicas.


*.- Está indicado para el tratamiento de la hipertensión ocular asociada a Glaucoma.


*.- El SYL040012 es una entidad química que se engloba dentro de los RNA de interferencia.

*.- Sylentis se encuentra entre las cinco primeras compañías a nivel mundial con ensayos clínicos de RNAi.