24. AL-S Pharma prepping a phase 3 trial of AP-101 in ALS

Progress in developing safe and effective therapies for amyotrophic lateral sclerosis (ALS) has been painfully slow. It is caused by the death of motor neurons in the central nervous system and the spinal cord, which leads to a loss of control over voluntary muscle movement, near-total paralysis and death within two to five years of diagnosis. Most patients have what is called sporadic ALS, which has no obvious cause, but about 10% of cases arise from a genetic mutation – upwards of 40 genes have been implicated, which points to the extraordinary complexity of this condition. The heterogeneity of ALS, its biological complexity and the lack of a disease-specific biomarker have all hampered progress in tackling this disease. It still takes about a year to reach a diagnosis. Just three drugs have been approved so far. The industry pipeline is highly diverse, reflecting the diversity and complexity of the condition. The Swiss firm AL-S Pharma is one of the few firms that has reported promising clinical data. Late last year, it reported that its human antibody AP-101, which binds and clears toxic, misfolded forms of the enzyme superoxide dismutase 1 (SOD1), improved survival and delayed the need for ventilatory support in patients with familial ALS and in patients with SOD1-mutated ALS. It has now reported additional evidence linking these efficacy signals with effects on two general biomarkers of neuronal damage, serum neurofilament light chain and phosphorylated cerebrospinal neurofilament heavy chain.Angela Genge, chief medical officer at AL-S Pharma, who is also director of the ALS Center of Excellence and Patient Care at the Montreal Neurological Institute-Hospital, discussed these findings with host Cormac Sheridan and outlined the company’s plans for an upcoming phase 3 trial of AP-101. The study will employ an innovative patient stratification strategy, which could help it to identify a subset of patients who may be most likely to respond to the investigational drug.● Feedback● Subscribe (Get notified when new episodes are available. NO marketing!)● Disclaimer  ● LinkedIn