Audentes Therapeutics Inc(NASDAQ: BOLD) stock made an outstanding start in 2018 already generating over 30.6%

The main driver for this stock rally is the positive interim data from the first dose cohort of ASPIRO, a Phase 1 / 2 clinical trial of AT132 in patients with X-Linked Myotubular Myopathy (XLMTM).  ASPIRO is a study to evaluate the safety and preliminary efficacy of AT132 in approximately 12 XLMTM patients less than five years of age.

The results of the study exceeded the group’s expectations. Patient 1 has improved from a severely compromised baseline at the 12-week timepoint to achieve a CHOP-INTEND score and maximal inspiratory pressure that are approaching the ranges normally seen in healthy children.  The group sees this initial efficacy data as a potential for AT132 to bring a change in their patients and families living with this devastating disease. The group woud offer the next update on interim data from ASPIRO in the second quarter of 2018.

Meanwhile, Audentes Therapeutics stock delivered over 71% in the year of 2017. The group’s RECENSUS study has established one of the largest data sets of genetically-confirmed XLMTM in the world. The RECENSUS study was the first step in the clinical development program for AT132, the Audentes product candidate in development for the treatment of XLMTM.  FThe group also started INCEPTUS, a clinical assessment and Phase 1 / 2 run-in study, in July 2016 for prospectively characterize the disease course and natural history of children with XLMTM and to assess the burden of disease on XLMTM patients and caregivers.

As per the numbers, the group reported a Cash Position of over $156.0 million as of September 2017. R&D Expenses rose to $20.9 million during the third quarter of 2017 from $12.5 million in pcp on the back of their AT132 and AT307 programs. Accordingly, research and development expenses rose to $54.2 million in the nine months ended September 30, 2017, from $32.2 million for the same period in 2016. As of the third quarter of 2017, the group reported a Net loss of $25.0 million for the third quarter of 2017, from a net loss of $15.4 million for the same period in 2016.