ATYR PHARMA, INC. (NASDAQ:LIFE) Files An 8-K Regulation FD Disclosure
Item 7.01 Regulation FD Disclosure.
On December 13, 2016, aTyr Pharma, Inc. (the Company) announced
clinical trial data in a press release, a copy of which furnished
herewith as Exhibit 99.1.
In addition, on December 13, 2016, the Company conducted a
conference call with corporate presentation materials which the
Company placed on its website.A copy of the presentation
materials is furnished herewith as Exhibit 99.2. The Company does
not undertake to update the presentation materials.
The information under this Item 7.01, including Exhibit 99.1 and
99.2, is being furnished and shall not be deemed filed for the
purposes of Section 18 of the Securities and Exchange Act of
1934, as amended, or the Exchange Act, or otherwise subject to
the liabilities of that section, nor shall they be deemed
incorporated by reference into any filing under the Securities
Act of 1933, as amended, or the Exchange Act, except as expressly
set forth by specific reference in such filing.
Item 8.01 Other Events.
In connection with the announcement of clinical trial data
described above, the Company announced clinical results from
exploratory trials assessing the safety and potential activity of
Top-line results from a completed Phase 1b/2 trial for
Interim data from an ongoing Phase 1b/2 trial with early
Interim data from an ongoing long-term safety extension
The results announced today highlight the potential of Resolaris,
an immuno-modulator of activated T cells, as a single treatment
for multiple rare myopathies with an immune component (RMIC).
Clinical Activity Assessments:
As part of clinical assessments in these studies, manual muscle
testing (MMT), a validated assessment tool that measures muscle
strength, was performed across 14 selected muscle groups. In
addition, a validated patient reported outcome measure designed
specifically for neuromuscular disease, the individualized
neuromuscular quality of life (INQoL) questionnaire, was
utilized. Note that an increase in MMT score represents an
increase in muscle strength, whereas a decrease in INQoL score
represents a decrease in disease burden. Given that the 003, 004
and 005 Trials are small and open-label in nature and that the
clinical assessments expressed in this release, MMT and INQoL,
although clinically validated, are subject to variability over
time, including intra-patient and inter-physician variability, it
is important to temper any definitive conclusions made with
respect to the clinical activity of Resolaris.
LGMD2B/FSHD (004) Trial:
This international Phase 1b/2 clinical trial at 6 clinical sites
was an open-label, intra-patient, placebo run-in, dose escalation
study designed to assess the safety, tolerability, immunogenicity
and exploratory assessments of clinical activity of intra-patient
dose escalations to twice weekly (biw) intravenous infusions of
Resolaris in LGMD2B and FSHD adult patients.Patients were
assigned to two treatment groups each with 12 weeks of treatment:
Group A: 4 patients with FSHD received infusions of
Group B: 10 patients with LGMD and 4 patients with FSHD
Manual Muscle Testing, MMT, Assessment 004 Trial:
(See Figure 1 and Figure 2)
*One patient in the
LGMD2B group (Figure 1) was wheelchair bound and did not complete
the MMT evaluation.
Individualized Quality of Life, INQoL, Assessment 004 Trial:
LGMD2B Patients: Overall INQoL score was relatively
FSHD Patients: Overall INQoL score was relatively stable
Biomarker Summary in 004 Trial:
Various exploratory biomarkers (including targeted muscle T2 and
STIR MRI and various plasma proteins) did not generally establish
sufficiently high or consistent levels or robust signals across a
sufficient number of patients to determine test article effects.
Peripheral cell based biomarkers will be assessed at a later
date. Targeted muscle T2/STIR MRI will not be prioritized as a
biomarker in the near-term.
Early Onset FSHD (003) Trial:
This ongoing international, multi-center, open-label,
intra-patient, placebo run-in, dose escalation Phase 1b/2 study
is designed to evaluate the safety, tolerability, immunogenicity
and exploratory assessments of clinical activity 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.
An interim data cut was conducted for the first four early-onset
FSHD patients that completed treatment with Resolaris (age range
of 16 to 20).
Manual Muscle Testing, MMT, Assessment 003 Trial:
(See Figure 3)
Individualized Quality of Life, INQoL, Assessment 003 Trial:
Patients INQoL scores were relatively stable. Two patients had
slight decreases in disease burden and one patient showed an
increase. The fourth patient did not have a baseline INQoL
Adult FSHD Long Term Safety Extension (005) Trial:
This ongoing international, multi-center, open-label extension
clinical trial is designed to assess the long-term safety,
effects on biomarkers and systemic exposure of Resolaris in adult
FSHD patients from the completed 002 Trial. Patients receive
weekly doses of 3.0 mg/kg on an ongoing basis.
3 of the 9 patients enrolled from the adult FSHD (002)
Of the 4 patients who received at least 6 months of
Peripheral cell based biomarkers and other biomarkers
Safety and Tolerability Summary
As of December 1, 2016, 44 patients have received
Resolaris continues to demonstrate a favorable safety
No Serious Adverse Events (SAEs) were reported by
Adverse Events (AE) reported were in general mild or
No notable differences in AEs between adult FSHD, adult
Protocol related discontinuations
Per protocol, patients:
are not medicated before or during infusion for infusion
discontinue upon occurrence of an IRR and 4 FSHD patients
reaching Jo-1 antibody unit levels above the designated
1 LGMD patient discontinued from the 004 Trial for non-drug
All IRRs were mild to moderate and transient
Elevated Jo-1 antibody observations were without associated
After changing the infusion protocol to a 90-minute
The overall discontinuation rate in the 003, 004 and 005
In the 003, 004 and 005 Trials, low level anti-drug
After reviewing the entire clinical program for Resolaris, which
spans 44 patients in four separate interventional trials, aTyr
believes that these results are supportive of the advancement of
Resolaris as a single treatment for various rare myopathies with
an immune component. Next steps for the company include
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. In addition, aTyr
plans to meet with the FDA in 2017 to discuss a regulatory path
towards a Biologics License Application (BLA).
In 2017, aTyr looks forward to:
Emphasizing one RMIC indication in the Resolaris program
Advancing its iMod.Fc program into the clinic (including
Furthering its clinical and RD pipeline by partnering one
In addition, selected slides from 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.
This Current Report on Form 8-K contains forward-looking
statements within the meaning of the Private Securities
Litigation Reform Act of 1995 and other federal securities laws.
The use of words such as may, might, will, should, expect, plan,
anticipate, believe, estimate, project, intend, future,
potential, opportunity, or continue, and other similar
expressions are intended to identify forward-looking statements.
For example, all statements we make regarding the potential
therapeutic benefits of Physiocrines and our product candidates,
including Resolaris and iMod. Fc, the ability to successfully
advance our pipeline or product candidates, the timing within
which we expect to initiate, receive and report data from, and
complete our planned clinical trials, and our ability to receive
regulatory approvals for, and commercialize, our product
candidates, our ability to identify and discover additional
product candidates, and the ability of our intellectual property
portfolio to provide protection are forward-looking statements.
All forward-looking statements are based on estimates and
assumptions by our management that, although we believe to be
reasonable, are inherently uncertain. All forward-looking
statements are subject to risks and uncertainties that may cause
actual results to differ materially from those that we expected.
These risks, uncertainties and other factors are more fully
described in our filings with the U.S. Securities and Exchange
Commission, including our most recent Annual Report on Form 10-K,
our most recent Quarterly Report on Form 10-Q and in our
subsequent filings.The forward-looking statements in this
presentation speak only as of the date of this presentation and
neither we nor any other person assume responsibility for the
accuracy and completeness of any forward-looking statement. We
undertake no obligation to publicly update or revise any
forward-looking statement, whether as a result of new
information, future events or otherwise, except as required by
Press Release of aTyr Pharma, Inc. dated December 13,
Corporate Presentation Materials of aTyr Pharma, Inc.
Selected Slides from the Corporate Presentation Materials
About ATYR PHARMA, INC. (NASDAQ:LIFE)
aTyr Pharma, Inc. is a clinical-stage biotherapeutics company. The Company is engaged in the discovery and clinical development of medicines for patients suffering from severe, rare diseases using its Physiocrine biology, a discovered set of physiological modulators. The Company focuses on the development of Physiocrine-based therapeutics for the treatment of rare diseases, including facioscapulohumeral muscular dystrophy (FSHD) and limb-girdle muscular dystrophy (LGMD) 2B. The Company is developing Resolaris, an intravenous protein therapeutic for the treatment of rare myopathies with an immune component (RMICs). The Company is investigating Resolaris in patients with LGMD2B. The Company is conducting approximately three open label trials in patients with early onset FSHD, in adult patients with FSHD or LGMD2B and a long-term extension study in adult patients with FSHD. The Company has not generated any revenues. ATYR PHARMA, INC. (NASDAQ:LIFE) Recent Trading Information
ATYR PHARMA, INC. (NASDAQ:LIFE) closed its last trading session up +0.05 at 3.65 with 162,494 shares trading hands.