Aeterna Zentaris provides therapeutics development pipeline update

Therapeutics Development Pipeline Update: Autoimmunity Modifying Biologicals: Targeted, highly specific autoimmunity modifying therapeutics for the potential treatment of neuromyelitis optica spectrum disorder and Parkinson’s disease: Recent Highlights: Demonstrated positive pre-clinical proof-of-concept in NMOSD and PD. NMOSD selected as lead development program. Entered into an R&D agreement with Massachusetts General Hospital in Boston and Dr. Michael Levy, a worldwide leading NMOSD expert. Further optimization of candidates for potential use in clinical development. Initiated ex-vivo efficacy testing to confirm the AIM Biologicals mechanism of action and efficacy in human blood samples from both NMOSD and PD patients. Next Steps and Expectations: Following the completion of the pre-clinical studies, Aeterna plans to compile a comprehensive pre-clinical data package for scientific advice meetings with regulatory authorities, expected to take place in the third quarter of 2023. Delayed Clearance Parathyroid Hormone Fusion Polypeptides: Potential treatment for chronic hypoparathyroidism. Recent Highlights: Successful verification and reproduction of previous in-vivo data from University of Sheffield, in a rat model of hypoparathyroidism, in the fourth quarter of 2022. Ongoing development of the manufacturing process for AEZS-150 with the Company’s contract development and manufacturing organization, progressing toward establishment of a master cell bank for a cell line expressing AEZS-150 and a process suitable for larger scale GMP manufacturing. Next Steps and Expectations: Continue efforts to establish master cell bank for a cell line expressing AEZS-150 to be used to conduct further pre-clinical efficacy and safety tests. Meet with regulatory authorities to determine the development path forward; Company expects meeting in mid-2023 and an abbreviated safety and toxicology program as the active principle is already approved and in use. Macimorelin Therapeutic: Ghrelin agonist in development for the treatment of ALS: Recent Highlights: Substantial progress in the development of a suitable, alternative formulation for use in ALS. Ongoing evaluation of AEZS-130 in transgenic mouse ALS models to demonstrate the therapeutic potential of macimorelin in this indication. Next Steps Proof-of-concept results from ongoing pre-clinical studies expected in the first quarter of 2023. Following potential achievement of proof-of-concept, the Company will seek to have a scientific advice meeting with regulatory authorities to discuss program development next steps. Toxicology and safety studies to support treatment over prolonged periods are ongoing and based on the substantial body of pre-clinical and clinical data already available from the successful development of macimorelin as a diagnostic in AGHD.