Lundbeck Starts Clinical Phase IIa With Lu AA24493 (cEPO) In Friedreich's Ataxia In A Study Also Assessing Efficacy Via Biomarkers

Main Category: Neurology / Neuroscience
Also Included In: Clinical Trials / Drug Trials;  Pharma Industry / Biotech Industry
Article Date: 07 Nov 2009 - 1:00 PST

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H. Lundbeck A/S strengthens its pipeline of pharmaceuticals in clinical development by initiating phase IIa clinical studies with the innovative project Lu AA24493 in order to evaluate safety and tolerability and to explore theoretical efficacy parameters of the drug in humans. Lundbeck expects to enrol 35-40 people suffering from Friedreich's ataxia in this study.

Lu AA24493 is a novel carbamoylated form of human erythropoietin (EPO) a modification of EPO that results in loss of haematopoietic effects but maintains the tissue protective effect. These tissue protective effects translate to very positive effects in a number of animal models for neuronal damage

The primary objective of the study is to evaluate the safety and tolerability of two weeks treatment with a fixed dose Lu AA24493 in patients with Friedreich's ataxia. However, this placebo-controlled programme may also potentially provide efficacy signals via biomarkers.

"This project in Friedreich's ataxia represents Lundbeck's focus to discover and develop innovative compounds that address unmet needs for patients. Lu AA24493 is an example of compounds with a great potential for addressing a small but very severe disease," says Executive Vice President Anders Gersel Pedersen, Head of Drug Development at Lundbeck. "Furthermore, this project represents an innovative process in obtaining proof of principle as we look for signals related to a biomarker as an early indicator of drug activity".

About Friedreich's ataxia[1]

Friedreich's ataxia is an inherited disease that causes progressive damage to the nervous system resulting in symptoms ranging from gait disturbance and speech problems to heart disease. It is named after the physician Nicholaus Friedreich, who first described the condition in the 1860s. "Ataxia," which refers to coordination problems such as clumsy or awkward movements and unsteadiness, occurs in many different diseases and conditions. The ataxia of Friedreich's ataxia results from the degeneration of nerve tissue in the spinal cord and of nerves that control muscle movement in the arms and legs. The spinal cord becomes thinner and nerve cells lose some of their myelin sheath - the insular covering on all nerve cells that helps conduct nerve impulses.

Friedreich's ataxia, although rare, is the most prevalent inherited ataxia, affecting about 1 in every 50,000 people in the Caucasian population. Males and females are affected equally.

As with many degenerative diseases of the nervous system, there is currently no cure or effective treatment for Friedreich's ataxia. However, many of the symptoms and accompanying complications can be treated to help patients maintain optimal functioning as long as possible.

About Lu AA24493

Lu AA24493 is a novel cytoprotective compound currently in development for acute ischaemic stroke. It is chemically modified EPO by carbamylation and does not bind to the erythropoietin receptor (EPOR) and is thereby without haematopoietic side-effects. Despite the lack of binding to EPOR, Lu AA24493 retains full cytoprotective properties, demonstrating that Lu AA24493 mediates its beneficial effects via a mechanism different from that of the classical EPOR.

Lundbeck was founded in 1915 by Hans Lundbeck in Copenhagen, Denmark, and employs today over 5,500 people worldwide. Lundbeck is one of the world's leading pharmaceutical companies working with CNS disorders. In 2008, the company's revenue was DKK 11.3 billion (approximately EUR 1.5 billion or USD 2.2 billion).

[1] Friedreich's Ataxia Fact Sheet; National Institute of Neurological Disorders and Stroke

Source: Lundbeck