Aeterna Zentaris Starts Preclinical Development of Macimorelin as a Potential Treatment for ALS

Aeterna Zentaris Inc. has announced the commencement of the previously announced preclinical program to qualify macimorelin for clinical development as a potential treatment option for amyotrophic lateral sclerosis (ALS; Lou Gehrig's disease). The preclinical development program is part of the recently executed Material Transfer Agreement (MTA) with The University of Queensland.

The University researchers, led by Frederik Steyn, MSc, PhD, will conduct preclinical studies in disease-related in-vitro and in-vivo models of ALS to demonstrate the therapeutic potential of macimorelin on disease progression and ALS-specific pathology. Recently, The University researchers have filed funding applications to dedicated organizations in Australia to finance parts of the abbreviated preclinical development program and to conduct a subsequent investigator-initiated clinical trial to evaluate the safety, tolerability and efficacy of macimorelin as a potential new treatment option for ALS patients.

Dr. Klaus Paulini, Chief Executive Officer of Aeterna Zentaris, commented, “The start of the preclinical phase represents a significant milestone in the development of a potential new treatment option for ALS to help patients with this terrible, incurable disease and to improve their quality of life. We look forward to supporting Dr. Steyn’s research team in addressing this significant unmet medical need.”

In the event of positive preclinical results, the Company has the exclusive right to acquire a license from The University on the therapeutic use of macimorelin for the treatment of ALS.

Aeterna Zentaris’ lead product, macimorelin, is the only United States Food and Drug Administration (“FDA”) approved oral drug indicated for the diagnosis of adult growth hormone deficiency (AGHD) and is currently marketed in the United States under the tradename Macrilen™, by Novo Nordisk. It is also licensed to Consilient Health Ltd. for the European Economic Area and United Kingdom, and Megapharm Ltd. for Israel and the Palestinian Authority. Aeterna Zentaris is currently developing macimorelin for the diagnosis of childhood-onset growth hormone deficiency (CGHD), an area of significant unmet need, in collaboration with Novo Nordisk.

Ghrelin is a natural hormone stimulating release of growth hormone. Its level is often reduced in ALS patients and stimulation of GH excretion improves survival in ALS mouse models. Ghrelin and ghrelin mimetics have already demonstrated in preclinical models stimulation of growth factors to protect motor neurons and enhance muscle building, stimulate appetite, slow metabolism to promote positive energy balance and dampen neuroinflammatory processes. Dr. Steyn’s research team at The University have confirmed these positive effects of ghrelin mimetics in several experiments. Ghrelin itself is not qualified as medicine because of rapid clearance from circulation. However, macimorelin is an orally active ghrelin agonist and could mimic the biological effect of the hormone via mechanisms similar to ghrelin.