Thursday 18 April 2013

ARMGO Pharma gets $1 million to support ARM210 preclinical work for DMD treatment

ARMGO Pharma gets $1 million to support ARM210 preclinical work for DMD treatment: ARMGO Pharma and the Muscular Dystrophy Association (MDA) today announced that $1 Million has been awarded for preclinical work in support of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for ARM210, a novel, orally available, small-molecule Rycal drug that has potential as a treatment for Duchenne muscular dystrophy (DMD). The funding award to ARMGO Pharma comes from MDA Venture Philanthropy (MVP), part of MDA's translational research program.

The project being advanced by ARMGO Pharma is focused on a novel class of small molecule drugs known as Rycals™ which target the Ryanodine Receptor (RyR), an intracellular calcium channel that becomes leaky in disease states including muscular dystrophy, contributing to loss of muscle strength and function.� Rycals have been shown in animal models of muscle disease to repair RyR-mediated calcium leak and thereby improve specific muscle force and exercise capacity.�

Glaxo Muscular Dystrophy Drug Reaches Primary Objective in Study - Bloomberg

Glaxo Muscular Dystrophy Drug Reaches Primary Objective in Study - Bloomberg: GlaxoSmithKline Plc (GSK)’s drisapersen achieved the primary objective of a mid-stage study, heating up competition with Sarepta Therapeutics Inc. (SRPT) in the race for a drug that could reverse the debilitating effects of Duchenne muscular dystrophy.
Patients taking drisapersen showed a clinically meaningful difference from those on placebo after 48 weeks of treatment, according to an abstract of the study results posted on the website of CureDuchenne, an investor in Glaxo’s partner on the drug, Dutch biotechnology company Prosensa Therapeutics BV. Glaxo will present the data at a genetics conference in Cold Spring Harbor, New York, tomorrow.

Monday 15 April 2013

Positive updates for exon skipping and other treatments - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Positive updates for exon skipping and other treatments - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: At a recent RNA and Oligonucleotide Therapeutics conference at Cold Spring Harbor, US, 10-13 April 2013, Dr John Kraus GSK Project Physician Leader presented Phase II results. The (DMD114117) study was an exploratory, unpowered, double blind, placebo-controlled clinical trial with the aim of assessing the safety and efficacy of drisapersen in 53 boys with DMD. The dosing regimens were continuous (6mg/kg/wk) and intermittent (10-week cycles of 9 doses at 6mg/kg over 6 wks, and 4 wks off drug).