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 that it is
presenting at the European Academy of Dermatology and Venereology’s
(EADV) 30th anniversary congress being held virtually from
September 29-October 2, 2021. The topic of the Company’s
presentation is the results from its previously completed Phase 2a
study in 2018 which evaluated TMB-001 in patients with lamellar or
X-linked congenital ichthyosis (CI). TMB-001 is a topical
isotretinoin formulated using Timber’s patented IPEG™ delivery
system.
The Phase 2a study results demonstrated no concerning safety
signals and no evidence of significant systemic exposure to
isotretinoin or tretinoin after 12 weeks of treatment. Most
participants in the study experienced ≥1-grade Investigator Global
Assessment (IGA) score reduction and improvement in clinical signs
and symptoms of moderate or severe CI. Importantly, patients
experienced ≥75% improvement of one point from baseline in scaling
by IGA measurement and 100% of TMB-001 treated patients were
considered to have had their scaling clear, almost clear, or mild
by day 57.
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 expects to report topline data from the Phase 2b
CONTROL study in the fourth quarter of 2021 and is planning for an
end-of-Phase 2 meeting with the FDA by the end of the year.
“As we close in on topline data from our Phase 2b CONTROL study
evaluating our lead asset, TMB-001, we are pleased to have the
opportunity to formally present prior results from the previously
completed Phase 2a study that showed clear safety and efficacy
signals and supported continued development of our lead product
candidate,” said Alan Mendelsohn, M.D., Chief Medical Officer of
Timber. “Our IPEG delivery system is designed to maximize
isotretinoin delivery into pathologic skin layers, while minimizing
systemic absorption, and we believe these data demonstrate that
capability and look forward to confirming these results in
later-stage clinical trials.”
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, and impaired eyesight or
hearing. The management of CI is a life-long endeavor, which
remains largely symptomatic and commonly focused on reducing
scaling and/or skin lubrication with both systemic and topical
treatments.
The two-part, multicenter, double-blind Phase 2a study evaluated
the safety, tolerability, and efficacy of TMB-001 for the treatment
of CI in patients greater than 12 years of age. A total of 19
participants were randomized 1:1 to 0.1% or 0.2% concentrations of
TMB-001 and were required to have two comparable contralateral
treatment areas that covered no more than 6% of body surface and
were greater than 150cm2 with identical baseline IGA scores ≥3
(indicating moderate to severe disease severity). Seven
participants discontinued the study, including two who discontinued
due to treatment-emergent adverse events including mild application
site folliculitis and four who withdrew consent from study
participation. Other mild or moderate adverse events reported
included rash, irritation, pruritus, and pain at the application
site as well as contact dermatitis.
In part one of the study, one of the two areas was randomly
treated with either concentration of active drug, while the second
area was treated with vehicle twice daily for eight weeks.
Treatment with 0.1% concentration of TMB-001 led to a greater
proportion of participants achieving an improvement in ≥1- and
≥2-grade IGA score versus vehicle (100% vs. 66.7% and 66.7% vs.
33.3%, respectively). For 0.2% concentration of TMB-001,
participants receiving active drug achieved similar ≥1- and
≥2-grade IGA improvement versus vehicle (100% vs. 87.5% and 50% vs.
62.5%, respectively). Additional efficacy analyses from part one of
the study show the following:
- When directly comparing improvements at day 57, the proportions
of participants where IGA scores were ≥1 grade lower for 0.1% and
0.2% concentration of TMB-001 versus vehicle were 66.7% and 37.5%,
respectively.
- TMB-001 0.1% and 0.2% treatment areas showed improvements in
IGA score of ≥1 grade at day 57 versus baseline in erythema
(55.6%/62.5%), scaling (100%/75.0%), fissuring (66.7%/75.0%), and
papulation/lichenification (100%/75.0%).
- By day 57, the proportion of patients with reduction of scaling
to clear, almost clear, or mild was higher in patients treated with
TMB-001 0.1% or 0.2% when compared with vehicle (100% vs. 55.6% and
100% vs. 87.5%, respectively).
In part two of the study, both areas received the same active
treatment for four weeks. Continued active treatment areas were
assessed as clear, almost clear, or mild by IGA assessment in 85.7%
and 60% of participants for TMB-001 0.1% and 0.2% by day 84,
respectively. Additional efficacy analyses from part two of the
study show the following:
- All participants receiving TMB-001 0.1% had ≥1-grade total IGA
score reduction versus baseline in both treatment areas, and most
patients had ≥2-grade total IGA improvement versus baseline.
- For TMB-001 0.2%, ≥1- and ≥2-grade IGA reductions were observed
in 80% and 60% of participants, respectively, for both treatment
areas.
- By the end of the study, most participants in both treatment
groups had ≥1-grade IGA score improvement in all clinical signs and
symptoms, including erythema, scaling, fissuring, and
papulation/lichenification for both treatment areas.
Pharmacokinetic analyses conducted in five evaluable
participants show that plasma concentrations of isotretinoin and
tretinoin indicated systemic exposure minimally increased from
baseline four hours after initial application, and trough
concentrations measured 12 hours following application were within
range of baseline levels through day 84.
“Today the management of CI typically involves emollients to
reduce scaling and dryness and may include off-label use of topical
or oral retinoids,” said study co-author Christopher Bunick, M.D.,
Ph.D., associate professor of dermatology at Yale University School
of Medicine. “These results from a Phase 2a study demonstrate that
TMB-001 may be a promising topical alternative to oral retinoids
and support ongoing efficacy and safety investigation in these
patients.”
Results from the Phase 2a study of TMB-001, first posted on
September 2, 2021, can be found at ClinicalTrials.gov
(NCT02864082):
https://clinicaltrials.gov/ct2/show/study/NCT02864082?term=NCT02864082&draw=2&rank=1
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 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 Officer jkoconis@timberpharma.com
Investor Relations: Stephanie Prince PCG Advisory (646) 863-6341
sprince@pcgadvisory.com
Media Relations: Jenna UrbanBerry & Company Public Relations
(212) 253-8881jurban@berrypr.com
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