First ALS Patient Enrolled in Phase 1 Trial of AP-101, AL-S Pharma AG Announces

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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AL-S Pharma AG, a biotech company jointly founded and financed by Neurimmune and TVM Capital Life Science, has enrolled the first patient in its Phase 1 clinical trial assessing the safety and tolerability of AP-101, an experimental therapy for amyotrophic lateral sclerosis (ALS).

AP-101 is a human monoclonal antibody that targets, and therefore seeks to reduce or eliminate, misfolded superoxide dismutase-1 (SOD1). This defective protein has been established as one of the root causes of familial ALS. AP-101 was discovered and pre-clinically validated by Neurimmune’s proprietary Reverse Translational Medicine (RTM) technology platform.

AP-101 has received orphan drug designation from the U.S. Food and Drug Administration, the European Medicines Agency (EMA) and Swissmedic for the treatment of ALS.

The trial (NCT03981536) is designed to evaluate the safety, tolerability and pharmacokinetic properties of AP-101 in patients with sporadic and familial ALS. (Pharmacokinetics refers to the study of how a drug is absorbed, distributed, metabolized, and eliminated from the body.)

The multi-center, open-label, Phase 1 study is expected to recruit 18 participants from five clinical sites in Canada.

Primary goals will include assessing the number of patients experiencing at least one treatment side effect (serious or non-serious), as well as the number of participants having abnormal vital signs, clinical lab results, physical or neurological examinations, or electrocardiograms (ECGs), from the beginning of the study up to day 84.

Secondary goals will include assessing the maximum concentration of AP-101 in the serum, as well as the time it takes for the drug to reach this concentration after being administered (again, up to day 84). Drug concentration and pharmacokinetics in the cerebrospinal fluid (CSF) also will be assessed.

“The science behind AP-101 is very intriguing and we are excited to see AP-101 advance to the clinic. Our goal is to develop the molecule into a novel therapeutic option for ALS patients,” Angela Genge, MD, said in a press release. Genge is director of the Clinical Research Unit at McGill University in Montreal, and the head of AL-S Pharma’s Clinical Advisory Board.

The biopharma company Neurimmune and the venture capital fund TVM Capital Life Science created AL-S Pharma AG in 2016 to develop AP-101. Since its foundation, ALS Pharma AG has been working with Chorus, an autonomous division of Eli Lilly, to plan the most efficient and cost-effective way to develop AP-101 as a therapy for ALS.

“Promising preclinical data with the antibody warrant clinical development of AP-101, and we hope to provide therapeutic benefit to patients suffering from ALS,” said Michael Salzmann, AL-S Pharma’s CEO and Neurimmune’s chief operating officer.

“We highly value our unique partnership with TVM Capital Life Science and Eli Lilly’s Chorus. This collaboration allows us to leverage significant clinical development know-how as well as venture capital expertise to develop our promising molecule,” Salzmann said.

“We are very thankful for the work accomplished by the AL-S Pharma executive management and the Chorus team, as well as to our partner Neurimmune and our ALS experts at the Montreal Neurological Institute led by Dr. Angela Genge and Prof. Guy Rouleau,” said Hubert Birner, managing partner at TVM Capital Life Science and Chairman of the Board of AL-S Pharma, commented.