Item 8.01 Other Events.
In connection with the announcement of clinical trial data described above, the Company announced clinical results from
its Phase 1b/2 003 trial assessing the safety and potential activity of Resolaris™ in patients with early onset facioscapulohumeral muscular dystrophy (FSHD).
The Company’s exploratory trials in rare muscular dystrophies were designed to assess:
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Potential signals of clinical activity informative for developing clinical endpoints to be assessed in later-stage, placebo-controlled efficacy trials;
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Safety and tolerability of a new biologic protein, Resolaris, in patients;
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Biomarkers in these patients; and
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Data that would support further evaluation of Resolaris in rare muscular dystrophies with an immune component.
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The 003 trial was an international, multi-center, open-label, intra-patient, placebo run-in, dose escalation Phase 1b/2 study designed to evaluate the safety, tolerability, immunogenicity and exploratory clinical assessments of Resolaris at weekly doses of 0.3, 1.0 and 3.0 mg/kg in patients with early onset FSHD for a total of 12 weeks. Eight patients, ages 16 to 20, participated in the study.
Potential Signals of Clinical Activity
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63% of patients (5 of 8) had increases from baseline in their Manual Muscle Test (MMT), a validated assessment tool that measures muscle strength, with a mean change from baseline of +3.8%.
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67% of patients measured (4 of 6) had improvement in their Individualized Neuromuscular Quality of Life (INQoL) score, a validated patient reported outcome measuring a patient’s level of disease burden. On average, patients did not have a worsening of their disease burden as measured by INQoL.
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Safety and Tolerability
Resolaris was generally well-tolerated at doses up to 3.0 mg/kg once weekly in early onset FSHD. There have been no observed signs of general immunosuppression and low-level anti-drug antibody signals did not result in clinical symptoms. Adverse events were mild or moderate in intensity. There were no clinically significant changes in other safety assessments. The Company believes the observed safety results of Resolaris to date are supportive of further advancement of Resolaris.
In addition, selected slides from the corporate presentation with respect to the clinical trial data referenced above are filed herewith as Exhibit 99.3. The Company does not undertake to update the presentation materials.
Forward-Looking Statements
This Current Report on Form 8-K contains forward-looking statements within the meaning of the Private Litigation Reform Act. Forward-looking statements are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by such safe harbor provisions for forward-looking statements and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements regarding the potential and potential therapeutic benefits of Resolaris™, the ability of the Company to successfully advance its pipeline or product candidates, undertake certain development activities (such as clinical trial enrollment and the conduct of clinical trials) and accomplish certain development goals and the timing of such activities and development goals, the timing of initiation of additional clinical trials and of reporting results from our clinical trials, the scope and strength of our intellectual property portfolio, our ability to receive regulatory approvals for, and commercialize, our product candidates reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, risks associated with the discovery, development and regulation of our Physiocrine-based product candidates, as well as those set forth in our most recent Annual Report on Form 10-K for the year ended December 31, 2016 and in our other SEC filings. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Item 9.01 Exhibits.
(d) Exhibits
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Exhibit No.
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Description
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99.1
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Press Release of aTyr Pharma, Inc. dated April 24, 2017 (furnished herewith)
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99.2
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Corporate Presentation Materials of aTyr Pharma, Inc. dated April 2017, entitled “Advancing New Therapeutic Horizons for Patients with Rare Muscular Dystrophies - Resolaris Update” (furnished herewith)
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99.3
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Selected Slides from the Corporate Presentation Materials of aTyr Pharma, Inc. dated April 2017, entitled “Advancing New Therapeutic Horizons for Patients with Rare Muscular Dystrophies - Resolaris Update” (filed herewith)
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