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Basking Ridge, NJ – 
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:
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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.
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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%).
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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:
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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.
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For TMB-001 0.2%, ≥1- and ≥2-grade IGA reductions were observed in 80% and 60% of participants, respectively, for both treatment areas.
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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 Urban
Berry & Company Public Relations
(212) 253-8881
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