Timber Pharmaceuticals Receives European Orphan Drug Designation for TMB-001 in X-Linked Recessive Ichthyosis

Rhea-AI Summary

Timber Pharmaceuticals (NYSE American: TMBR) announced that the European Commission granted orphan drug designation for TMB-001, targeting X-linked recessive ichthyosis (XLRI). This follows a similar designation for autosomal recessive congenital ichthyosis (ARCI). TMB-001 is a topical isotretinoin formulated with the IPEG™ delivery system, currently assessed in a Phase 3 ASCEND clinical trial across multiple countries including the U.S., Canada, Italy, France, and Germany. The trial aims to enroll approximately 142 patients ages six and older. Orphan drug designation offers financial incentives and market exclusivity for therapies addressing rare diseases.

Positive

• Granted orphan drug designation for TMB-001 for XLRI, enhancing its market position.
• Ongoing Phase 3 ASCEND trial with enrollment of approximately 142 patients, indicating progress in clinical development.
• Potential to address significant unmet medical needs in rare dermatologic diseases with TMB-001.

Negative

• None.

Company Progressing With Pivotal Phase 3 ASCEND Clinical Trial in the U.S., Canada, Italy, France, and Germany

BASKING RIDGE, NJ, Feb. 24, 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 European Commission (EC) granted orphan drug designation for TMB-001 for the treatment of X-linked recessive ichthyosis (XLRI) on February 15, 2023. The EC previously granted orphan drug designation for TMB-001 for the treatment of autosomal recessive congenital ichthyosis (ARCI).

“We are pleased to receive an additional orphan drug designation in Europe for the treatment of XLRI as we continue to make steady progress with our global Phase 3 clinical trial,” said John Koconis, Chairman and Chief Executive Officer of Timber. “These orphan drug designations underscore the significant unmet needs in congenital ichthyosis (CI), which can lead to a limited range of motion, chronic itching, an inability to sweat normally, high risk of secondary infections, and impaired eyesight or hearing. We believe the targeted delivery of therapies to the epidermis and dermis may be able to minimize systemic absorption and we are committed to bringing a potential new treatment option to this rare disease community.”

TMB-001 is a topical isotretinoin, formulated using the Company’s patented IPEG™ delivery system, in development for the treatment of moderate to severe forms of CI. CI is a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin. In patients with ARCI and XLRI, cutaneous manifestations can include large, dark scaling throughout the body, which affect multiple aspects of the person's quality of life.

Timber is assessing the efficacy, pharmacokinetics and safety of TMB-001 (0.05% isotretinoin) in the ongoing pivotal Phase 3 ASCEND clinical trial at leading research centers in the U.S., Canada, Italy, France, and Germany. The study will enroll approximately 142 patients ages six years and above with moderate to severe CI.

Orphan drug designation is available for treatments for rare diseases that are life-threatening or chronically debilitating that affect fewer than five in 10,000 people across the European Union (EU). Medicines that are granted orphan drug designation by the EC qualify for financial and regulatory incentives including protocol assistance at reduced fees during product development, access to centralized marketing authorization, and 10 years of market exclusivity in the EU after product approval.

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 is TMB-001 and its significance for Timber Pharmaceuticals?

TMB-001 is a topical isotretinoin in development for treating congenital ichthyosis. Its orphan drug designation enhances its market potential.

When was the orphan drug designation for TMB-001 granted?

The European Commission granted the orphan drug designation for TMB-001 for XLRI on February 15, 2023.

What is the Phase 3 ASCEND trial for TMB-001?

The Phase 3 ASCEND trial is assessing the efficacy and safety of TMB-001 in treating moderate to severe congenital ichthyosis, enrolling around 142 patients.

What are the benefits of orphan drug designation for TMB-001?

Orphan drug designation provides financial incentives, reduced fees during development, centralized marketing authorization, and 10 years of market exclusivity post-approval.