via NewMediaWire -- Timber Pharmaceuticals, Inc. ("Timber" or the
“Company”) (NYSE American: TMBR), a biopharmaceutical company
focused on the development and commercialization of treatments for
rare and orphan dermatologic diseases, today announced the
successful completion of an End-of-Phase 2 meeting with the U.S.
Food and Drug Administration (FDA) that resulted in a clear path to
progress to a pivotal Phase 3 study for its lead asset, TMB-001, a
topical isotretinoin formulated using the company’s patented IPEG™
delivery system.
Timber completed the Phase 2b CONTROL study evaluating TMB-001
in moderate to severe congenital ichthyosis (CI) in September 2021
and announced that topline data demonstrated clinically meaningful
efficacy with a favorable safety profile. Full data was
subsequently presented at the 2022 Winter Clinical Dermatology
Conference held January 14–19, 2022 in Koloa, Hawaii. Based on FDA
feedback at the End-of-Phase 2 meeting, Timber intends to initiate
a pivotal Phase 3 study of TMB-001 in the second quarter of
2022.
“We are pleased to have successfully completed our End-of-Phase
2 meeting with the FDA and are committed to delivering a potential
new option as rapidly as possible for the treatment of CI to
patients who currently have no FDA-approved treatments available,”
said John Koconis, chairman and chief executive officer of Timber.
“The full data set from our Phase 2b CONTROL study indicates that
TMB-001 may be a promising topical alternative to oral retinoids
and fully supports initiating Phase 3 investigation. We now have a
clear path forward for TMB-001 based on guidance from the FDA and,
with the completion of a recent financing, look forward to the
initiation of a robust Phase 3 trial in the coming months.”
CI is a group of rare genetic keratinization disorders that
leads to dry, thickened, and scaling skin. Patients with moderate
to severe subtypes of CI, including X-linked ichthyosis (XLRI) and
autosomal recessive congenital ichthyosis (ARCI), which includes
lamellar ichthyosis (LI), often have considerable hyperkeratosis
and skin scaling. These subtypes of CI affect about 80,000 people
in the U.S. and more than 1.5 million globally. Treatment of
moderate to severe CI represents a clear unmet need in dermatology
as there are currently no approved FDA treatments for these
conditions.
Phase 2b CONTROL study data presented at the 2022 Winter
Clinical Dermatology Conference
The randomized, parallel, double-blind, vehicle-controlled Phase
2b CONTROL study was designed to evaluate the efficacy and safety
of two concentrations of TMB-001 in patients with LI or XLRI. A
total of 33 patients were randomized (1:1:1 ratio) to receive
either TMB-001 0.05%, TMB-001 0.1%, or vehicle twice daily,
stratified by CI subtype, for 12 weeks. The intent-to-treat (ITT)
population included all patients initially enrolled in the trial,
and the per-protocol (PP) population included patients who
completed the full 12 weeks of treatment.
Based on the full data set, Timber has selected the 0.05% dose
of TMB-001 for its pivotal Phase 3 program. Importantly, the median
time to response to treatment with TMB-001 0.05% in the ITT
population was significantly shorter than vehicle (28 days for
TMB-001 0.05% versus 63.5 days for vehicle) and there were no
concerning safety signals compared with vehicle. Patients receiving
TMB-001 on average demonstrated statistically significant more
rapid relief of scaling and fissuring, with half of the patients
demonstrating relief in 28 days or less.
The primary efficacy endpoint was the proportion of patients
with Visual Index for Ichthyosis Severity (VIIS)-scaling treatment
success (VIIS-50 or a 50% reduction in the VIIS score versus
baseline). The VIIS scoring system examines four representative
areas of the body to evaluate improvement in scaling alone, which
for many patients is their primary symptom and cause of
concern.
- For the PP population, 100%, 40%, and 40% of patients receiving
TMB-001 0.05%, TMB-001 0.1%, and vehicle achieved VIIS-50,
respectively (P = 0.04 for TMB-001 0.05% vs. vehicle).
- For the ITT population, 64%, 40%, and 33% of patients receiving
TMB-001 0.05%, TMB-001 0.1%, and vehicle achieved VIIS-50,
respectively (P = 0.17 for TMB-001 0.05% vs. vehicle).
The key secondary efficacy endpoint was the proportion of
patients who achieved Investigator Global Assessment (IGA)
treatment success (≥2-grade reduction in scaling and fissuring
severity over all treated areas of the body).
- For the PP population, 100%, 60%, and 10% of patients receiving
TMB-001 0.05%, TMB-001 0.1%, and vehicle, respectively, reported a
≥2-grade IGA score improvement (P = 0.002 for TMB-001 0.05% vs
vehicle).
- For the ITT population, improvement of ≥2-grade IGA score was
observed in 55%, 40%, and 8% of patients receiving TMB-001 0.05%,
TMB-001 0.1%, and vehicle, respectively (P = 0.02 for TMB-001 0.05%
vs vehicle).
Treatment-emergent adverse events included local skin reactions
that were mild or moderate in severity, and no serious adverse
events were observed.
“These data demonstrate clinically meaningful efficacy for the
participants as well as significantly more rapid improvement in the
participants who received the TMB-001 0.05% treatment,” said Alan
Mendelsohn, M.D., Chief Medical Officer of Timber. “We thank our
investigator community and the participants in our study for
assisting us to advance this promising therapy forward.”
About Timber Pharmaceuticals, Inc.
Timber Pharmaceuticals, Inc. is a biopharmaceutical company
focused on the development and commercialization of treatments for
rare and orphan dermatologic diseases. The Company's
investigational therapies have proven mechanisms-of-action backed
by decades of clinical experience and well-established CMC
(chemistry, manufacturing, and control) and safety profiles. The
Company is initially focused on developing non-systemic treatments
for rare dermatologic diseases including congenital ichthyosis
(CI), facial angiofibromas (FAs) in tuberous sclerosis complex
(TSC), and other sclerotic skin diseases. For more information,
visit www.timberpharma.com.
Forward-Looking Statements
This press release 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 and
regulatory timelines, market opportunity, competitive position,
intellectual property rights, possible or assumed future results of
operations, business strategies, potential growth opportunities and
other statements 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 Annual Report on Form 10-K for the year
ended December 31, 2020 as well as other documents filed by the
Company from time to time thereafter 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.
For more information, contact:
Timber Pharmaceuticals, Inc. John Koconis Chairman
and Chief Executive Officer jkoconis@timberpharma.com
Investor Relations: Stephanie Prince PCG Advisory (646) 863-6341
sprince@pcgadvisory.com
Media Relations: Adam Daley Berry & Company Public Relations
(212) 253-8881adaley@berrypr.com
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