BrainEver: FDA orphan drug designation for BREN-02 for the treatment of ALS MyPharma editions

BrainEver, a biotech company dedicated to the research and development of innovative therapies for the treatment of neurodegenerative diseases, today announces that the FDA (Food and Drug Administration) in the United States has granted orphan drug designation to its BREN-02 product, homeoprotein recombinant human Engrailed-1 (rhEN1), in the treatment of amyotrophic lateral sclerosis (ALS).

ALS (also known as Charcot’s disease) is a severe neurodegenerative disease that affects motor neurons, leading to progressive muscle weakness that progresses to paralysis and a life expectancy of approximately 2-4 years from onset. of illness. ALS is diagnosed in approximately 16,000 people each year in Europe and North America.

“In ALS, motor neurons degenerate and die, causing progressive muscle paralysis, including in the respiratory muscles. We have observed during preclinical studies that the lumbar intrathecal administration of rhEN1, in the early symptomatic stages, restores muscle function and prevents the death of motor neurons ”, commented Prof. Alain Prochiantz, neurobiologist, Collège de France, co-founder of BrainEver.

BREN-02 is expected to enter clinical trials in ALS patients in the second half of 2021, subject to preclinical toxicity results and regulatory review. Preclinical studies have shown that hEN1 homeoprotein is essential for the survival and maintenance of alpha motor neurons in the spinal cord that supply muscles throughout the body.

The FDA grants orphan drug status in order to encourage the development of therapies to treat, prevent or diagnose diseases or conditions that affect fewer than 200,000 people in the United States. The orphan drug designation in the United States recognizes the therapeutic potential of BREN-02 in ALS and allows BrainEver to benefit from specific measures and benefits, including a seven-year market exclusivity period if hEN1 is approved for the treatment of ALS.

Created in 2016 by Prof. Alain Prochiantz and Bernard Gilly, BrainEver is a biotechnology company specializing in the discovery and development of innovative treatments for neurodegenerative diseases. BrainEver exploits the intrinsic properties of homeoproteins, a family of transcription factors essential during embryonic and postnatal development, whose recent studies have highlighted the key role in the maintenance and survival of specific neuronal populations in adults. An important property of homeoproteins is their ability to enter cells, with privileged access to the cytoplasmic and nuclear compartments. Therefore they do not require any transport systems to reach the targeted cells or a viral expression vector. BrainEver plans to launch its first clinical study on ALS, in humans in the course of 2021. BrainEver has raised € 21 million from iBionext, bpifrance and Turenne Capital.

Source: BrainEver