Top-line data from Timber Pharmaceuticals’ Phase 2b CONTROL study show “clinically meaningful efficacy with a favorable safety profile” for investigational topical isotretinoin, says the company’s Chairman and CEO John Koconis. TMB-001 is formulated using Timber's patented IPEG delivery system, in patients with moderate to severe congenital ichthyosis (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 (X-linked recessive and lamellar ichthyosis) in patients (n=33) nine years old or older. Subjects applied TMB-001 twice daily for 12 weeks. The study was not designed or powered for statistical analysis of the endpoints and was intended to provide information for future development.

Timber reports:

• In the PP population, 100 percent (nominal p= .04) and 40 percent (nominal p= ns) of patients treated with TMB-001 0.05% and 0.1%, respectively, achieved VIIS-50 compared to 40 percent in the vehicle group.
• In the ITT population, 64 percent (nominal p= 0.17) and 40 percent (nominal p= ns) of patients treated with TMB-001 0.05% and 0.1%, respectively, achieved VIIS-50 compared to 33 percent in the vehicle group.
• In the ITT population, 55 percent (nominal p=.02) and 40 percent (nominal p=ns) of patients treated with TMB-001 0.05% and 0.1%, respectively, achieved a greater-than or equal to2 point improvement in the IGA at week 12 compared to 8 percent in the vehicle group.

"We are extremely pleased with the encouraging data generated by the Phase 2b CONTROL study of TMB-001,” Mr. Koconis says in a statement. "We look forward to additional study data and plan to present the full data set at scientific congresses or peer-reviewed journal publication in the near future."

In 2018, the 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 is planning for an end-of-Phase 2 meeting with the FDA in the beginning of 2022 and expects to begin the Phase 3 study of TMB-001 in the second quarter of 2022.