Mallinckrodt has permanently discontinued the phase 2b PENNANT trial investigating Acthar Gel (repository corticotrophin) for the treatment of amyotrophic lateral sclerosis (ALS).
The study’s independent Data and Safety Monitoring Board (DSMB) recommended the trial be halted after observing a higher rate of pneumonia occurrence in Acthar-treated patients compared with those who received placebo. In addition, the DSMB noted “the lack of a clear efficacy signal” in those patients who completed 36 weeks of treatment. The Company agreed to permanently discontinue the study in the interest of patient safety. Patients currently receiving the investigational medicine will be tapered off prior to discontinuation and all study enrollments will be terminated immediately.
“Though the probability of success for the ALS population was acknowledged as being low, this study was initiated based on compelling analyses carried out following the completion of a small pilot study and we were hopeful it would have translated into a benefit for this group of patients in great need of effective therapies,” said Steven Romano, MD, Executive Vice President and Chief Scientific Officer at Mallinckrodt.
The PENNANT trial was evaluating the efficacy and safety of Acthar Gel in the treatment of patients aged 18 to 75 with ALS and symptom onset ≤2 years prior to the screening visit. Patients were randomized to receive either subcutaneous (SC) Acthar gel 0.2mL daily or SC placebo 0.2mL daily for 36 weeks. The primary efficacy measure was change from baseline in the ALS Functional Rating Scale-Revised, after 36 weeks.
Acthar Gel is a naturally sourced complex mixture of adrenocorticotropic hormone analogues and other pituitary peptides. It is currently approved for: the treatment of infantile spasms in patients under 2 years of age; treatment of acute exacerbations of multiple sclerosis in adults; and may be used for the following disorders and diseases: rheumatic, collagen, dermatologic, allergic states, ophthalmic, respiratory, and edematous state.
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