aTyr Pharma Inc. (NASDAQ: LIFE) saw its shares drop early on Tuesday after the company announced clinical results from its exploratory trials assessing the safety and potential activity of Resolaris. Ultimately, the announced results highlight the potential of Resolaris as an immuno-modulator of activated T cells and a single treatment for multiple rare myopathies with an immune component (RMIC). Despite this good news, the results may be somewhat shaky.
These results can be summed up by saying that they were supportive of the hypothesis that administering a naturally occurring muscle homeostasis protein with immuno-modulator activities, which is normally secreted by skeletal muscle cells, has the potential to help patients across many rare myopathies with divergent genetic etiologies of disease.
However, it must be noted that the manual muscle testing score and the individualized neuromuscular quality of life questionnaire, which were used as assessments in this trial, are subject to variability over time. These are clinically validated methods, but they are variable over time, whether intra-patient and inter-physician.
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The next steps for the company are completing the evaluation of the 003, 004 and 005 biomarker data, particularly peripheral cell-based data using one or more mechanistic assays currently under development at aTyr for agonists of the Resokine pathway and T cell activity. Future trials will be designed using one or more of these mechanistic assays, as well as the option to assess local immune components in skeletal muscle directly with biopsies.
Additionally, aTyr plans to meet with the FDA in 2017 to discuss a regulatory path towards a Biologics License Application.
John Mendlein, Ph.D., CEO of aTyr Pharma, commented:
Congratulations to our team, collaborators and patients that helped us accomplish the fundamental objectives for these clinical trials: (1) to demonstrate the safety and tolerability of our product candidate, Resolaris, across different RMICs and (2) to explore different readouts of potential clinical activity and product candidate activity in different RMICs. We are very pleased to see RMIC patients with two entirely different genetic etiologies showing improvement in muscle strength in 3 months as measured by manual muscle testing. Taken together, our clinical data supports the exciting potential of Resolaris as a single treatment for multiple rare myopathies with an immune component and we believe our clinical results will help inform and direct the future clinical development of Resolaris in RMICs. Finally, we wish to personally thank all the FSHD and LGMD patients and health care professionals who have participated in our trials.
Shares of aTyr were trading down 15% at $3.10 on Tuesday, with a consensus analyst price target of $6.67 and a 52-week trading range of $2.48 to $10.93.
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