Today, Mallinckrodt, a pharmaceutical company with global headquarters in the United Kingdom, announced it will initiate a phase II proof-of-concept clinical trial to assess the efficacy of H.P. Acthar® Gel in people living with ALS. The trial is expected to begin in the first half of 2017.
The U.S. Food and Drug Administration (FDA) has granted the company’s request for a Fast Track designation for its Investigational New Drug (IND) application. The next step is to start the phase II study and they are working with the FDA closely to establish the trial design. Fast Track designation is designed to expedite the review of drugs to treat serious diseases, such as ALS with the goal to get novel drugs to people living with such diseases quicker.
H.P. Acthar® Gel is a repository corticotropin injection that is given either subcutaneously or intramuscularly. It has been approved by the FDA for the treatment of 19 indications, including the treatment of acute exacerbations of Multiple Sclerosis (MS) in adults. The drug has anti-inflammatory indications and is targeted at neuro-inflammation in ALS, which is a known disease pathway that has been shown to cause motor neuron death.
Recently, H.P. Acthar® Gel met safety and tolerability standards of four doses in an open-label pilot trial that ran for an eight-week period, with an optional 28-week, open-label extension period. This pilot trial was not designed to test for drug efficacy. The planned phase II trial will test efficacy using established measures of ALS disease progression and symptoms.
The exact trial initiation date, study sites and inclusion/exclusion criteria are yet to be released. The ALS Association remains in close contact with Mallinckrodt and will update the community with more information once it becomes available. For information about other clinical trials, please visit http://www.alsa.org/research/clinical-trials/.