Timber Pharmaceuticals Announces Publication of Sub-Analysis of Phase 2b CONTROL study in Clinical and Experimental Dermatology

Rhea-AI Summary

Timber Pharmaceuticals (TMBR) has announced positive results from a sub-analysis of its Phase 2b CONTROL study of TMB-001, a topical isotretinoin for treating congenital ichthyosis (CI).

The study found significant improvements among participants with both autosomal recessive congenital ichthyosis (ARCI) and X-linked recessive ichthyosis (XLRI) in achieving primary and secondary endpoints. In the intent-to-treat population, 33% to 100% of participants with ARCI and XLRI showed a 50% reduction in Visual Index for Ichthyosis Severity (VIIS) compared to vehicle treatments.

Results support continued development of TMB-001, which has orphan designation in Europe, as a promising alternative to systemic therapies.

Positive

• TMB-001 showed significant improvement in key endpoints versus vehicle in the CONTROL study.
• 100% of XLRI participants using TMB-001 0.05% achieved a 50% reduction in VIIS.
• TMB-001 has received orphan designation from the European Commission for ARCI and XLRI.

Negative

• None.

- Treatment with TMB-001 demonstrated greater proportions of participants achieving primary and key secondary endpoints versus vehicle regardless of the subtype of congenital ichthyosis -

BASKING RIDGE, NJ, March 20, 2023 (GLOBE NEWSWIRE) -- via NewMediaWire -- Timber Pharmaceuticals, Inc. ("Timber" or the “Company”) (NYSE American: TMBR), a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today announced the online publication of a sub-analysis of the Phase 2b CONTROL study in Clinical and Experimental Dermatology (CED), the educational journal of the British Association of Dermatologists. The CONTROL study evaluated TMB-001, a topical isotretinoin formulated using the Company’s patented IPEG™ delivery system, in subjects nine years of age and older with moderate to severe congenital ichthyosis (CI).

CI is a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin. CI subtypes display variable responses to oral retinoids. The sub-analysis was designed to assess whether the efficacy and safety of TMB-001 differed between subjects with autosomal recessive congenital ichthyosis (ARCI) and X-linked recessive ichthyosis (XLRI). Results show that treatment with TMB-001 demonstrated greater proportions of participants achieving the primary and key secondary endpoints versus vehicle regardless of the subtype of CI.

“Patients with severe CI must often combine bathing, mechanical scale removal, and topical over-the-counter emollient therapy with the possible addition of systemic retinoid therapy throughout their lifetimes to address their disease, sometimes without significant improvement,” said Christopher Bunick, M.D., Ph.D., Associate Professor of Dermatology at Yale University School of Medicine and corresponding author of the paper. “In this sub-analysis, participants in the CONTROL study with ARCI and XLRI mostly showed comparable percentage differences in responses to treatment with a novel topical isotretinoin formulation versus vehicle. That is especially encouraging because systemic retinoids do not typically demonstrate the same efficacy across subtypes of CI.”

A total of 33 participants in the CONTROL study were randomized to TMB-001 0.05%, TMB-001 0.1%, or vehicle twice daily, stratified by CI subtype, for 12 weeks. The primary endpoint was the proportion of participants with a 50% or greater reduction versus baseline in Visual Index for Ichthyosis Severity (VIIS) scaling. The key secondary endpoint was a two-grade or greater reduction in Investigator Global Assessment (IGA) scaling score versus baseline. Adverse events (AEs) were monitored. Among enrolled participants (TMB-001 0.05% [n = 11], TMB-001 0.1% [n = 10], and vehicle [n = 12]), 52% had ARCI and 48% had XLRI subtypes. The intent-to-treat (ITT) population consisted of all randomized participants who received one or more doses of the study medication. The per-protocol (PP) population included all participants who met all inclusion criteria, were treatment compliant (≥80%–120% of study drug applied), had a VIIS scaling measurement at the end of Week 12, and had no major protocol violations.

• In the ITT population, 33%/50%/17% of participants with ARCI and 100%/33%/75% of participants with XLRI who received TMB-001 0.05%/TMB-001 0.1%/vehicle, respectively, achieved VIIS-50.
• In the PP population, 100%/33%/17% of participants with ARCI and 100%/50%/75% of participants with XLRI who received TMB-001 0.05%/TMB-001 0.1%/vehicle, respectively, achieved VIIS-50.
• In the ITT population, improvement of ≥2-grade IGA score was observed in 33%/50%/0% of participants with ARCI and 83%/33%/25% of participants with XLRI who received TMB-001 0.05%/TMB-001 0.1%/vehicle, respectively.
• In the PP population, improvement of ≥2-grade IGA score was observed in 100%/67%/0% of participants with ARCI and 100%/50%/25% of participants with XLRI who received TMB-001 0.05%/TMB-001 0.1%/vehicle, respectively.
• Most AEs reported were application site reactions and were similarly distributed among both subgroups.
  

“ARCI and XLRI are severe subtypes of CI that can lead to cutaneous manifestations including large, dark scaling throughout the body and reduced quality of life,” said Alan Mendelsohn, M.D., Chief Medical Officer of Timber. “This 12-week analysis demonstrated TMB-001 reduced clinical signs and symptoms of two forms of CI and was well tolerated in participants with either ARCI or XLRI. TMB-001 has been granted orphan designation for both of these subtypes of CI by the European Commission, and the findings of this sub-analysis support our efforts to evaluate TMB-001 in a currently ongoing pivotal Phase 3 clinical trial. We look forward to advancing this program with the goal of delivering a promising alternative to systemic retinoids for patients with these two especially severe forms of CI.”

About Timber Pharmaceuticals, Inc.

Timber Pharmaceuticals, Inc. is a clinical-stage 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) and 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, 2021 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) 253-8881
adaley@berrypr.com

FAQ

What were the results of the TMB-001 CONTROL study published on March 20, 2023?

The CONTROL study showed that TMB-001 significantly improved treatment outcomes for participants with both ARCI and XLRI, exceeding the results of vehicle treatments.

What are the next steps for Timber Pharmaceuticals after the CONTROL study results?

Timber Pharmaceuticals will continue with the ongoing pivotal Phase 3 clinical trial for TMB-001, aiming to provide a viable alternative to systemic retinoids.

What is the primary endpoint of the CONTROL study for TMB-001?

The primary endpoint was achieving a 50% or greater reduction in Visual Index for Ichthyosis Severity (VIIS) scaling.

What is TMB-001 used for?

TMB-001 is a topical isotretinoin formulated to treat congenital ichthyosis, a group of rare genetic skin disorders.

Which subtypes of congenital ichthyosis were studied in the CONTROL study?

The CONTROL study evaluated participants with autosomal recessive congenital ichthyosis (ARCI) and X-linked recessive ichthyosis (XLRI).