William Blair slashed its price target for aTyr Pharma (NASDAQ:LIFE) to $4 from $8 but maintained its “market perform” rating after the company released data from three Phase 1/2 studies of Resolaris in rare muscle diseases. The stock closed at $2.80 on Dec. 13.
The three studies were in facioscapulohumeral muscular dystrophy (FSHD) and limb girdle muscular type 2B (LGMD 2B).
“Overall, the efficacy signals as measured with manual muscle strength testing and Individualized Neuromuscular Quality of Life Questionnaire were not particularly strong, and the fact that there was no placebo control rendered the data difficult to interpret,” writes analyst Katherine Xu.
She said aTyr plans to run an additional longer-term, placebo-controlled study in a rare muscle disease indication to definitively demonstrate efficacy and safety of Resolaris.
“While the company has identified the 3 mg/kg dose as the dose to take forward, many design elements of this future study still need to be determined, including frequency and duration of dosing, what efficacy endpoints to choose, safety parameters, sample size, and which indication to pursue,” Ms. Xu added.
