Timber Pharmaceuticals Announces Publication of Results from Phase 2b CONTROL Study of FDA-Designated Breakthrough Therapy TMB-001 in the Journal of the American Academy of Dermatology

Rhea-AI Summary

Timber Pharmaceuticals has published positive results from its Phase 2b CONTROL study of TMB-001 for treating moderate to severe congenital ichthyosis (CI) in the Journal of the American Academy of Dermatology. The study showed significant efficacy with TMB-001, especially at 0.05% concentration, achieving treatment success rates of 100% in the per-protocol population. Following these promising results, Timber initiated the pivotal Phase 3 ASCEND clinical trial to further evaluate TMB-001 in this patient population. The company is optimistic about the treatment's potential to enhance the quality of life for individuals with CI.

Positive

• Results from the Phase 2b CONTROL study highlight significant efficacy for TMB-001, with 100% success in the per-protocol group at the 0.05% concentration.
• Initiation of the pivotal Phase 3 ASCEND clinical trial to gather additional data for regulatory review.
• The potential to significantly improve treatment options for approximately 80,000 people in the U.S. with CI.

Negative

• No serious adverse events were reported, but local skin reactions were observed, which could impact patient adherence.
• The ITT population demonstrated only a 64% success rate at 0.05% concentration, which may raise questions about overall efficacy.

- Previously reported study demonstrated clinically meaningful efficacy with a favorable safety profile for TMB-001 in moderate to severe congenital ichthyosis -

- Company has since initiated pivotal Phase 3 ASCEND clinical trial - 

BASKING RIDGE, NJ, Aug. 26, 2022 (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, announced that results from the previously completed Phase 2b CONTROL study that evaluated TMB-001, a topical isotretinoin formulated using the Company’s patented IPEG™ delivery system, in moderate to severe congenital ichthyosis (CI) are published in the Journal of the American Academy of Dermatology.

The paper, titled, “The CONTROL study: A randomized, double-blind vehicle-controlled Phase 2b study of novel topical isotretinoin formulation demonstrates improvement in recessive X-linked and autosomal recessive lamellar congenital ichthyosis,” highlights results that were initially announced by Timber in September 2021 demonstrating clinically meaningful efficacy with a favorable safety profile. 

“The opportunity to publish these data in a world-renowned journal reviewed by our peers in rare and orphan dermatologic diseases exemplifies the significance of our clinical development program and the potential we have to change the treatment landscape for people living with CI,” said Alan Mendelsohn, M.D., Chief Medical Officer of Timber. “Based on the success of the Phase 2b CONTROL study, we have already enrolled the first patients in the recently launched Phase 3 ASCEND clinical trial that is designed to provide the pivotal data we need to navigate the regulatory review process. We want to thank all prior and current investigators and patients who have helped us get to this point.”

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 X-linked recessive (XLRI) or autosomal recessive lamellar (ARCI-LI) subtypes of CI that affect about 80,000 people in the U.S. and lead to cutaneous manifestations that include large, dark scaling throughout the body.  

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. 

The primary efficacy endpoint was the difference in the proportion of TMB-001 and vehicle treated patients with Visual Index for Ichthyosis Severity (VIIS)-scaling treatment success (VIIS-50 or a 50% reduction in the VIIS score versus baseline). 

• 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 difference in 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) comparing TMB-001 to vehicle.

• 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.

“Hyperkeratosis and widespread scaling are characteristic findings among patients with XLRI or ARCI-LI subtypes of CI, and current management predominantly includes emollients, keratolytics, and off-label retinoids,” said lead author Joyce M.C. Teng, M.D., Ph.D., Professor and Director of Pediatric Dermatology, Stanford University. “These results support ongoing TMB-001 efficacy and safety investigation as a promising alternative to oral retinoids for patients with CI.”

Timber has since announced the initiation of the pivotal Phase 3 ASCEND clinical trial that is evaluating the efficacy, pharmacokinetics and safety of TMB-001 0.05% for the treatment of moderate to severe forms of CI in subjects 6 years of age and older. For more information about the ASCEND study, visit https://ichthyosistrial.com/. 

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 are the results of the Phase 2b CONTROL study of TMB-001?

The Phase 2b CONTROL study showed that 100% of patients using TMB-001 0.05% achieved significant treatment success in the per-protocol population.

When did Timber Pharmaceuticals initiate the Phase 3 ASCEND trial for TMB-001?

Timber Pharmaceuticals initiated the pivotal Phase 3 ASCEND clinical trial following the positive results from the Phase 2b study.

What is congenital ichthyosis and how many people does it affect in the U.S.?

Congenital ichthyosis is a rare skin disorder affecting approximately 80,000 people in the U.S. It is characterized by dry, scaling skin.

What are the next steps for Timber Pharmaceuticals after the Phase 2b CONTROL study?

Timber Pharmaceuticals will focus on gathering pivotal data from the Phase 3 ASCEND clinical trial to support regulatory approval for TMB-001.

What is the purpose of the ASCEND trial for TMB-001?

The ASCEND trial aims to evaluate the efficacy, pharmacokinetics, and safety of TMB-001 for the treatment of moderate to severe congenital ichthyosis.