European Commission expected to issue its decision on orphan designation in approximately 30 days
BASKING RIDGE, NJ - 
(NewMediaWire) - September 14, 2022 - 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 that the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) issued a positive opinion on the Company’s application for orphan designation for TMB-001 for the treatment of autosomal recessive congenital ichthyosis (ARCI). Based on the COMP’s opinion, the European Commission is expected to issue its decision on the orphan designation in approximately 30 days.
“We are pleased that the EMA’s COMP has issued a positive opinion for orphan designation for TMB-001, recognizing the significant unmet medical need in the ARCI community for a safe and effective treatment option,” said John Koconis, Chairman and Chief Executive Officer of Timber. “This is a first and important step towards market approval in Europe and encouraging as we work to advance TMB-001 in our ongoing pivotal Phase 3 clinical trial as rapidly as possible. I want to thank our team at Timber and all trial investigators and patients who have helped us reach this stage in development. As we wait for the European Commission to issue its decision to hopefully grant orphan designation for TMB-001 in ARCI, Timber now plans to also submit an application for EMA orphan designation for TMB-001 for the treatment of X-linked ichthyosis.”
The EMA’s orphan designation is available to companies developing 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 designation by the European Commission qualify for financial and regulatory incentives including protocol assistance at reduced fees during product development, access to centralized marketing authorization, and 10 years of marketing exclusivity in the EU after product approval.
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 congenital ichthyosis (CI) including ARCI and X-linked recessive ichthyosis (XLRI). 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 include large, dark scaling throughout the body. There are currently no FDA-approved treatments for CI. TMB-001 was previously granted breakthrough therapy designation and fast track status from the U.S. Food and Drug Administration (FDA) for the treatment of CI.
Timber is currently assessing the efficacy, pharmacokinetics and safety of TMB-001 (0.05% isotretinoin) in the ongoing pivotal Phase 3 ASCEND clinical trialat leading research centers in the U.S., Canada, Italy, France, and Germany. Timber expects to enroll approximately 142 patients with moderate to severe CI in this study.
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
View the original release on www.newmediawire.com
