Timber Pharmaceuticals Reports Positive Top-line Results from Phase 2b CONTROL Study Evaluating TMB-001 in Moderate to Severe Congenital Ichthyosis
October 07 2021 - 9:11AM
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 positive top-line data from its Phase 2b
CONTROL study evaluating TMB-001, an investigational topical
isotretinoin formulated using Timber’s patented IPEG™ delivery
system, in patients with moderate to severe congenital ichthyosis
(CI).
CI is a group of rare, genetic keratinization
disorders that lead to dry, thickened, and scaling skin. People
living with CI may have limited range of motion, chronic itching,
an inability to sweat, high risk of secondary infections. There are
no approved medications for CI and the current common management of
this chronic disease is focused on reducing scaling or improving
skin lubrication with both systemic and topical
treatments.
The Phase 2b CONTROL study was a randomized,
double-blind, vehicle-controlled study designed to assess the
efficacy and safety of two concentrations of TMB-001 (0.05% and
0.1% isotretinoin) for the treatment of two distinct subtypes of
moderate-to-severe CI (X-linked recessive and lamellar ichthyosis)
in patients (n=33) nine years old or older. Subjects applied
TMB-001 twice daily for 12 weeks. The primary endpoint was the
reduction of targeted ichthyosis severity, determined by a 50
percent or greater reduction in the validated Visual Index for
Ichthyosis Severity (VIIS) scaling score (or VIIS-50), a clinically
meaningful change. Secondary endpoints included reduction in
overall ichthyosis severity, as measured by a two-point improvement
using the Investigator Global Assessment (IGA) scale, also
considered to be a clinically relevant improvement. The study was
not designed or powered for statistical analysis of the endpoints
and was intended to provide information for future
development.
Top-line results including descriptive statistics
are described below:
- In the PP population, 100 percent (nominal p= .04)
and 40 percent (nominal p= ns) of patients treated with
TMB-001 0.05% and 0.1%, respectively, achieved VIIS-50 compared to
40 percent in the vehicle group.
- In the ITT population, 64 percent (nominal p= 0.17)
and 40 percent (nominal p= ns) of patients treated with
TMB-001 0.05% and 0.1%, respectively, achieved VIIS-50 compared to
33 percent in the vehicle group.
- In the PP population, 100 percent (nominal p=.002)
and 60 percent (nominal p=ns) of patients treated with TMB-001
0.05% and 0.1%, respectively, achieved a ≥2 point improvement in
the IGA at week 12 compared to 10 percent in the vehicle
group.
- In the ITT population, 55 percent (nominal p=.02)
and 40 percent (nominal p=ns) of patients treated with TMB-001
0.05% and 0.1%, respectively, achieved a ≥2 point improvement in
the IGA at week 12 compared to 8 percent in the vehicle
group.
- TMB-001 was generally well tolerated with a similar
incidence of adverse events (AEs) across treatment groups. The most
frequent AEs were local adverse effects common for such topical
treatments. There were no treatment-related serious adverse events
(SAE).
“We are extremely pleased with the encouraging
data generated by the Phase 2b CONTROL study of TMB-001. The study
was not powered or intended to show statistical significance, but
we demonstrated clinically meaningful efficacy with a favorable
safety profile that support the continued development of our lead
product candidate,” said John Koconis, Chairman and Chief Executive
Officer of Timber. “We look forward to additional study data and
plan to present the full data set at scientific congresses or
peer-reviewed journal publication in the near future.”
“These topline results demonstrate the potential
of TMB-001 to be an important option for the treatment of CI where
no FDA-approved treatments are currently available and the standard
of care is limited to the use of emollients or keratinolytics to
reduce scaling and dryness,” said Alan Mendelsohn, M.D., Chief
Medical Officer of Timber. “We are committed to fulfilling the
unmet need of CI patients and continuing to develop TMB-001 as
quickly as possible. I would like to thank all of those who helped
us to rapidly advance the CONTROL trial during the pandemic
including the outstanding team of investigators and clinical sites
and particularly the patients who participated in the study.”
In 2018, the U.S. Food & Drug Administration
(FDA) awarded TMB-001 a $1.5 million grant to support Phase 2a and
Phase 2b clinical trials through its Orphan Products Clinical
Trials Grant program. Timber is planning for an end-of-Phase 2
meeting with the FDA in the beginning of 2022 and expects to begin
the Phase 3 study of TMB-001 in the second quarter of 2022.
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
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current beliefs and assumptions.
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"expect," "anticipate," "intend," "plan," "believe," "estimate,"
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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
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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 that may be filed by the Company from time to time 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
Officerjkoconis@timberpharma.com
Investor Relations:Stephanie PrincePCG
Advisory(646) 863-6341sprince@pcgadvisory.com
Media Relations:Jenna UrbanBerry & Company
Public Relations(212) 253-8881jurban@berrypr.com
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