Nonclinical Safety Evaluation of AMX0035 to Support Translation to Clinical Trials

Research Summary

Principal Investigators: Joshua Cohen & Justin Klee

Neurodegeneration and neurotoxic inflammation are hallmarks of ALS and together form a vicious cycle that is one of the key drivers of functional decline in patients. Our lead candidate, AMX0035, is a proprietary combination of two compounds that is designed to break this cycle, thereby halting clinical progression. Based on strong in-vitro and in-vivo results, we now seek to evaluate our promising therapeutic in patients with ALS.

Nonclinical safety studies, also called toxicology studies, are the essential FDA-required battery of tests needed to translate a therapy from the lab to the clinic. We met with the FDA in a pre-IND meeting and received consent that our safety program would be sufficient to support our first clinical trial. As of September 16, 2015, we are approximately halfway through these safety studies, and have completed:

1) The Ames Test

2) Evaluation of Maximum Tolerated Dose in Rat and Mini-pig

3) Dose Range Finding in Rat (mini-pig is currently underway)

4) Development of Bioanalytical Methods

Following completion of our nonclinical safety program in early 2016, we will file an IND with the FDA to run a first-in-human clinical trial. We are very grateful to ALS Finding a Cure for their support for these pivotal studies that will bring AMX0035 to patients in need.

http://www.fiercebiotech.com/biotech/als-upstart-amylyx-gains-3m-grant-to-start-combo-trial

http://www.prnewswire.com/news-releases/the-als-association-and-als-finding-a-cure-announce-joint-funding-support-for-amylyx-clinical-trial-of-amx0035-for-the-treatment-of-als-300300955.html

http://www.businesswire.com/news/home/20160822005017/en/Amylyx-Announces-5-Million-Series-Financing-Support