Audentes Therapeutics Inc. (NASDAQ: BOLD) shares made a handy gain on Thursday after the company announced positive interim data for its midstage trial in patients with X-linked myotubular myopathy (XLMTM). Specifically the positive data came from the firm’s Phase 1/2 clinical trial of AT132.
XLMTM is a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age.
Ultimately these results are a positive start for the firm’s Aspiro trial. And although the data is preliminary, it does set a solid safety and efficacy profile for the trial to move forward.
There were a total of six adverse events reported in the trial, two of which were determined to be serious and both of these occurring in the same patient.
Some of the highlights thus far include physicians and caregivers having reported progressive qualitative improvements in disease severity in all treated patients. Ventilator settings (pressure, rate and volume of mechanical ventilation) have been reduced in a couple patients. All treated patients have demonstrated improvements in airway clearance control, including swallowing and coughing, which is critical to preventing aspiration.
The next update is expected in the second quarter of 2018.
Dr. Suyash Prasad, senior vice president and chief medical officer of Audentes, commented:
The early AT132 efficacy data observed in our first dose cohort of patients have exceeded our expectations. At the 12-week timepoint, Patient 1 has improved from a severely compromised baseline to achieve a CHOP-INTEND score and maximal inspiratory pressure that are approaching the ranges normally seen in healthy children. Importantly, Patient 1 has also attained several age-appropriate developmental milestones within this time period, including head-control, rolling over and sitting unassisted. While still early in the trial, we view these initial efficacy data as a promising indicator of the potential for AT132 to bring meaningful benefit to patients and families living with this devastating disease.
Shares of Audentes were up about 12% at $40.00 on Thursday, with a consensus analyst price target of $35.00 and a 52-week range of $13.90 to $41.80.