Item
8.01. Other Events
On
June 13, 2018 the Company announced that data from open-label extensions (“OLEs”) of its systemic sclerosis and dermatomyositis
Phase 2 studies were presented at the Annual European Congress of Rheumatology.
Systemic
Sclerosis
Study
Design
Thirty-six
subjects with diffuse cutaneous SSc received open-label dosing with lenabasum at 20 mg twice per day following 16-weeks participation
in the preceding double-blinded placebo-controlled phase of the lenabasum Phase 2 study. There was an average 20-week wash-out
from investigational product prior to the start of the OLE. Twenty-seven subjects completed 1-year follow-up in the OLE at the
time of this data-cut. Lenabasum treatment was in addition to standard-of-care treatments for SSc, including stable doses of concomitant
immunosuppressive drugs in 94% of subjects.
Efficacy
Outcomes
The
modified Rodnan Skin Score (mRSS), a physician assessment of skin involvement and the primary outcome for the upcoming Phase 3
study of lenabasum in SSc, improved by a mean of -9.4 points from baseline at the start of the Phase 2 study. The baseline mRSS
at study start was 24 points. At 1 year, 77% of subjects achieved a degree of improvement in mRSS that is considered medically
meaningful (reduction ≥ 5 points), and 50% achieved ≥ 10 points improvement in mRSS.
The
ACR Composite Response Index in diffuse cutaneous Systemic Sclerosis score (ACR CRISS) increased steadily with lenabasum treatment
and reached 92% (median), with 50% of subjects achieving a score
>
95% at 1 year. Patient-reported disability, function,
skin symptoms and global health all improved from study start and OLE start.
The
mRSS and ACR CRISS, responses exceeded those seen in the 16-week double-blind placebo-controlled phase and the 6-month time point
in the OLE.
Safety
There
were no severe or serious AEs and no clinically significant laboratory abnormalities related to the drug. Thirty-three (92%) subjects
experienced AEs, and 7 (19%) subjects experienced AEs related to lenabasum during open-label dosing. AEs that occurred in ≥
10% of subjects (n, %) were upper respiratory tract infection (8, 22%), arthralgia, skin ulcer, and urinary tract infection (5,
13.9% each), and diarrhea (4, 11.1%).
Lenabasum
has been granted Orphan Drug Designation and Fast Track status for the treatment of SSc from the FDA and Orphan Designation from
the EMA. Lenabasum is currently being evaluated in the international multicenter Phase 3 RESOLVE-1 study, a double-blind, randomized,
placebo-controlled study assessing the efficacy and safety for the treatment of diffuse cutaneous SSc.
Dermatomyositis
Study
Design
Twenty
subjects with refractory, skin-predominant DM received open-label dosing with lenabasum at 20 mg twice per day following 16-weeks
participation in the preceding double-blinded placebo-controlled part of the lenabasum Phase 2 study. There was a mean 31-week
wash-out off investigational product prior to the start of the OLE. Seventeen subjects completed 6-months (28-weeks) follow-up
in the OLE at the time of data-cut. Lenabasum treatment was in addition to standard-of-care treatments for DM, including stable
doses of concomitant immunosuppressive drugs in 91% of subjects.
Efficacy
Outcomes
At
6 months (28 weeks), the CDASI activity score improved by a mean of -15.4 points from baseline at the start of the Phase 2 double-blind,
placebo-controlled phase of the study. The baseline CDASI activity score at study start was 35 points. At 6 months, 88% of subjects
achieved reduction ≥ 5 points, which is considered medically meaningful, 82% achieved reduction ≥ 10 points, and 47% had
achieved a low CDASI activity score (≤ 14 points). Patient-reported global disease activity, global skin disease, function,
pain, and skin symptoms all improved from study start and OLE start, as did physician global disease and skin activity assessments.
Safety
There
were no severe or serious AEs and no clinically significant laboratory abnormalities related to the drug. Thirteen (65%) subjects
experienced AEs, and 5 (25%) subjects experienced AEs related to lenabasum during open-label dosing. A DM flare, which is an episode
of worsening of the disease, was the only AE that occurred in ≥ 2 subjects, occurring in 2 subjects (one of which experienced
a reduction of 14 points in CDASI activity from study start and another of which experienced an increase of 5 points from study
start).
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.