Item
8.01. Other Events
On
October 19, 2017, the Company announced positive topline results from its 16-week Phase 2 study of safety and efficacy of anabasum
in dermatomyositis (“DM”). The mean improvement (reduction) in the primary efficacy outcome, the Cutaneous Dermatomyositis
Disease Area and Severity Index (“CDASI”) activity score, a validated outcome measure of skin disease severity, was
9.3 points for anabasum treatment at the end of the study versus a reduction of 3.7 points for placebo treatment (p = 0.04). Anabasum
also outperformed placebo in multiple secondary efficacy outcomes studied. Anabasum was well tolerated with no severe or serious
side effects associated with the drug. No subjects dropped out.
Study
Design
The
single center, double-blind, randomized, placebo-controlled trial enrolled 22 adult subjects in a 1 to 1 ratio of anabasum to
placebo cohorts. At baseline, subjects in each cohort had a mean CDASI activity score in the severe range and skin symptoms in
the extremely severe range despite background treatment with immunosuppressive drugs in 19 of the 22 subjects. Demographic parameters,
CDASI activity scores, patient-reported outcomes, and use of immunosuppressive drugs at baseline were similar for anabasum and
placebo cohorts. Subjects received anabasum 20 mg QD through week 4, then anabasum 20 mg BID through week 12 with safety and efficacy
follow-up thereafter through week 16. All subjects remained on their background standard-of-care therapy throughout the study.
Primary
Efficacy Endpoint
Improvement
in CDASI activity scores in the anabasum cohort were ≥ 7.5 points and consistently superior to improvement in the placebo cohort
from week 6 to end of study. The mean improvement (reduction) in CDASI activity score for the anabasum cohort (20 mg QD followed
by 20 mg BID) from day 1 to end of study was 9.3 points versus 3.7 points for the placebo cohort (p = 0.04, 2-sided MMRM). The
greater degree of improvement versus placebo in CDASI activity score occurred during dosing with anabasum 20 mg BID (treatment
effect 6.3 points at end of study, p = 0.02, 2-sided MMRM). Improvement in CDASI activity score ≥ 4 points has been correlated
with improvement in patient-reported quality of life outcomes, pain and physician global assessment.
Secondary
Efficacy Endpoints and Safety Outcomes
Improvements
across multiple secondary efficacy outcomes were seen in the anabasum cohort versus the placebo cohort, including statistically
significant improvement in CDASI Damage Index (p = 0.04) and patient-reported symptoms and functioning. Anabasum was well tolerated
and demonstrated a favorable safety profile with no serious or severe side effects related to the study drug and no study discontinuations.
The
dermatomyositis trial was funded by a grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases of
the National Institutes of Health to the University of Pennsylvania Perelman School of Medicine.
Forward-
Looking Statements
This
Current Report on Form 8-K contains certain forward-looking statements within the meaning of Section 27A of the Securities Act
of 1933 and Section 21E of the Securities Exchange Act of 1934 and Private Securities Litigation Reform Act, as amended, including
those relating to the Company’s product development, clinical trials, clinical and regulatory timelines, market opportunity,
competitive position, possible or assumed future results of operations, business strategies, potential growth opportunities and
other statement that are predictive in nature. These forward-looking statements are based on current expectations, estimates,
forecasts and projections about the industry and markets in which we operate and management’s current beliefs and assumptions.
These
statements may be identified by the use of forward-looking expressions, including, but not limited to, “expect,” “anticipate,”
“intend,” “plan,” “believe,” “estimate,” “potential,” “predict,”
“project,” “should,” “would” and similar expressions and the negatives of those terms. These
statements relate to future events or our financial performance and involve known and unknown risks, uncertainties, and other
factors which may cause actual results, performance or achievements to be materially different from any future results, performance
or achievements expressed or implied by the forward-looking statements. Such factors include those set forth in the Company’s
filings with the Securities and Exchange Commission. Prospective investors are cautioned not to place undue reliance on such forward-looking
statements, which speak only as of the date of this press release. The Company undertakes no obligation to publicly update any
forward-looking statement, whether as a result of new information, future events or otherwise.