About Timber Pharmaceuticals

Timber Pharmaceuticals, Inc. (Timber) operates as a clinical-stage biopharmaceutical company. The company focuses on the development and commercialization of treatments for 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'), including X-linked recessive CI ('XLRI') and autosomal recessive CI ('ARCI') and other sclerotic skin diseases. The company's lead late-stage program is TMB-001. TMB-003 is the company's earliest stage program. Products TMB-001 TMB-001, a patented topical formulation of isotretinoin using the company's patented IPEG delivery system, completed its Phase 2b clinical trial (the 'CONTROL study') in the fourth quarter of 2021 for the treatment of moderate to severe subtypes of CI, a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin. This study demonstrated a clinically meaningful reduction in targeted and overall severity of CI along with a favorable safety profile. The efficacy, pharmacokinetics and safety of TMB-001 is being evaluated in a pivotal Phase 3 ASCEND clinical trial (the 'ASCEND study'). A prior Phase 1/2 study involving 19 patients with CI demonstrated safety and a signal of preliminary efficacy of TMB-001, as well as minimal systemic absorption. The FDA (through its Orphan Products Grant program) awarded the company a grant to support clinical trials evaluating TMB-001. On October 7, 2021, the company announced the completion of its Phase 2b trial in CI. The Phase 2b CONTROL study was a randomized, double-blind, vehicle-controlled study designed to assess the efficacy and safety of two concentrations of TMB-001 (0.05% and 0.1% isotretinoin) for the treatment of two distinct subtypes of moderate-to-severe CI XLRI and autosomal recessive congenital ichthyosis ('ARCI') in patients (n=33) nine years old or older. Subjects applied TMB-001 twice daily for 12 weeks. The primary endpoint was the reduction of targeted ichthyosis severity, determined by a 50 percent or greater reduction in the validated Visual Index for Ichthyosis Severity ('VIIS') scaling score (or VIIS-50), a clinically meaningful change. Secondary endpoints included reduction in overall ichthyosis severity, as measured by a two-point improvement using the ('IGA') scale, also considered to be a clinically relevant improvement. The study was not designed or powered for statistical analysis of the endpoints and was intended to provide information for future development. On February 3, 2022, the company announced the successful completion of an End-of-Phase 2 meeting with the FDA that resulted in a clear path to progress to a pivotal Phase 3 study for TMB-001. The clinical development program for TMB-001 includes a Phase 3 study with an efficacy arm and a maximum use pharmacokinetic arm, as well as a smaller bridging study required to bridge to the oral reference product. Based on FDA feedback at the End-of-Phase 2 meeting, the company initiated the pivotal Phase 3 ASCEND study of TMB-001 0.05% in the second quarter of 2022. The primary endpoint is the comparison of proportions of subjects with a greater than or equal to2 point change from the baseline in IGA-scaling and fissuring scores in the treatment area at 12 weeks between TMB-001 0.05% and vehicle-treated subjects. Secondary endpoints include the reduction in overall ichthyosis severity. On June 23, 2022, the company announced that the first four patients had been enrolled in the Phase 3 ASCEND study. The company expects to complete enrollment of the Phase 3 ASCEND study by September 2023 and to publish top-line data by May 2024. In March 2022, a third party presented a late-breaking presentation of a sub-analysis of the company's Phase 2b CONTROL study that evaluated TMB-001 at the American Academy of Dermatology 2022 Annual Meeting. In April 2022, the FDA granted fast track designation to TMB-001 for the treatment of XLRI and ARCI-LI. In May 2022, the FDA granted breakthrough therapy designation to TMB-001 for the treatment of CI. In June 2022, the Korean Intellectual Property Office issued a patent covering TMB-001 Korean Patent Number: 10-2406880. Additional patents are pending for TMB-001 in several other countries. On June 30, 2022, the company received a notice of allowance from the United States Patent and Trademark Office for a patent application covering TMB-001 (Application Number: 16/875,710) covering claims to the uses and methods of treating congenital ichthyosis and administering isotretinoin. In July 2022, the company filed a U.S. continuation patent application covering claims to additional subject matter for TMB-001. In October 2022, the European Commission granted orphan designation for TMB-001 for the treatment of ARCI. The European Medicines Agency (the 'EMA') Pediatric Committee also provided positive comments on the Pediatric Investigation Plan for the treatment of ARCI and XLRI. The company plans to respond to the Pediatric Committee and expect a final discussion in the second quarter of 2023, which is a prerequisite for filing a Marketing Authorization Application with the EMA. In February 2023, the European Commission granted orphan drug designation for TMB-001 for the treatment of XLRI. In March 2023, a sub-analysis of the Phase 2b CONTROL study was published online in Clinical and Experimental Dermatology ('CED'), the educational journal of the British Association of Dermatologists. The sub-analysis was designed to assess whether the efficacy and safety of TMB-001 differed between subjects with ARCI and 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. TMB-003 The earliest stage product in the company's pipeline is TMB-003, a proprietary formulation of Sitaxsentan, a new chemical entity in the U.S., which is a selective endothelin-A receptor antagonist. It is in preclinical development as a locally applied formulation for the treatment of sclerotic skin diseases. The two disease areas under consideration include Lichen Sclerosis a rare chronic disease of vulvae and perianal areas, and Localized Scleroderma, a chromic connective tissue disease that also affects other organ systems. On January 12, 2021, the company announced that the FDA has granted orphan drug designation for TMB-003, the company's locally delivered formulation of Sitaxsentan, for the treatment of systemic sclerosis. The company is planning to pursue additional orphan drug designations in other indications in the future. BPX-01 and BPX-04 In connection with the merger with BioPharmX Corporation on May 18, 2020, the company acquired the BPX-01 and BPX-04 assets. BPX-01 is a Phase 3 ready topical minocycline for the treatment of inflammatory lesions of acne vulgaris. BPX-04 is a Phase 3 ready topical minocycline for the treatment of papulopustular rosacea. On September 15, 2020, the company had received a notice of allowance from the U.S. Patent and Trademark Office ('USPTO') for a patent application covering BPX-01 and BPX-04 (U.S. Patent Application No.: 16/514,459) and the application subsequently issued on January 5, 2021, as U.S. 10,881,672. The company is seeking to monetize these assets through a license, co-development, or sale. Material Agreements Asset Purchase Agreements with Patagonia Pharmaceuticals LLC ('Patagonia') TMB-001 On February 28, 2019, the company acquired the intellectual property rights for a topical formulation of isotretinoin for the treatment of CI and identified as TMB-001, formerly PAT-001, including the IPEG brand, from Patagonia which the company refers to as the TMB-001 Acquisition, pursuant to the Asset Acquisition Agreement. On July 20, 2022, the company entered into the Amendment to the Asset Acquisition Agreement with Patagonia, pursuant to which the company and Patagonia extended the time for the company's payment of the first milestone payment, which became payable in the third quarter of 2022 upon the commencement of patient enrollment in the company's Phase 3 ASCEND clinical trial in the second quarter of 2022. TMB-003 On June 26, 2019, the company acquired the intellectual property rights for a locally administered formulation of Sitaxsentan for the treatment of cutaneous fibrosis and/or pigmentation disorders, and identified as TMB-003, formerly PAT-S03, from Patagonia (the 'TMB-003 Acquisition'). Acquisition and Termination of License from AFT Pharmaceuticals Limited ('AFT') On July 5, 2019, the company entered into a license agreement with AFT which provided the company with an exclusive license to certain licensed patents, licensed know-how and AFT trademarks to commercialize a proprietary topical formulation of rapamycin marketed under the brand name Pascomer ('Pascomer') in the United States, Canada and Mexico; and a co-exclusive license to develop Pascomer in this territory. Concurrently, the company granted to AFT an exclusive license to commercialize Pascomer outside of its territory and co-exclusive sublicense to develop and manufacture the licensed product for commercialization outside of its territory (the 'AFT License Agreement'). On July 22, 2022, the company provided written notice to AFT of the company's decision to terminate the AFT License Agreement because there was no longer a commercially reasonable path to approval and commercialization for the Pascomer product in the United States. Additionally, following the receipt and analysis of topline data for the Phase II Clinical Trial (as defined in the AFT License Agreement) it was determined that the study failed to meet its primary efficacy endpoint. Under the AFT License Agreement, the company was required to provide 120 days' prior written notice of termination to AFT which was waived by AFT on July 25, 2022 (the 'Termination Date'). On the Termination Date, the rights and licenses to Pascomer reverted to AFT, among other things, as set forth in the AFT License Agreement. Intellectual Property and Market Protection As of December 31, 2022, the company owned or had licensed rights to at least 28 patents or patent applications, including at least 6 U.S. patent applications, and at least 22 international patent applications. The company's patent applications related to the company's product candidates in development are projected to expire between 2036 and 2043, subject to any PTE that might be available in a particular jurisdiction. Research and Development For the year ended December 31, 2022, the company's research and development expenses were $13.3 million. History Timber Pharmaceuticals, Inc. was founded in 2019 under the laws of the state of Delaware.