isotretinoin topical (TMB-001) / LEO Pharma - LARVOL DELTA

The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis (clinicaltrials.gov) - P3 | N=209 | Completed | Sponsor: Timber Pharmaceuticals Inc. | Active, not recruiting ➔ Completed

Trial completion • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Efficacy, Safety and Pharmacokinetics of First 17 Subjects in Maximal Use/ Phase 3 ASCEND Trial of Topical Isotretinoin (TMB-001) for Treatment of X-linked and Autosomal Recessive Congenital Ichthyosis (EADV 2024) - No abstract available

Clinical • P3 data • PK/PD data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Preliminary Efficacy, Safety and Pharmacokinetics of First 17 Subjects in Maximal Use/ Phase 3 ASCEND Trial of Topical Isotretinoin (TMB-001) for Treatment of X-linked and Autosomal Recessive Congenital Ichthyosis (EADV-Sp 2024) - No abstract available

Clinical • P3 data • PK/PD data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis (clinicaltrials.gov) - P3 | N=209 | Active, not recruiting | Sponsor: Timber Pharmaceuticals Inc. | N=142 ➔ 209 | Recruiting ➔ Active, not recruiting

Enrollment change • Enrollment closed • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Timber Pharmaceuticals, Inc., a LEO Pharma Company, Presents Late-Breaking Preliminary Results of TMB-001 in Moderate-to-Severe Congenital Ichthyosis (CI) at the 2024 AAD Annual Meeting (Businesswire) - P3 | N=142 | ASCEND (NCT05295732) | Sponsor: Timber Pharmaceuticals Inc. | "Timber Pharmaceuticals...presents preliminary results from the ASCEND trial in a late-breaking session at the 2024 American Academy of Dermatology (AAD) Annual Meeting. The results offer a first look at the safety, efficacy and pharmacokinetic (PK) data from 17 adult and adolescent participants (aged 12-64 years).....TMB-001 results showed that, from baseline to Week 12, the mean total Investigator Global Assessment (IGA) composite scaling and fissuring score decreased from 3.2 to 1.6 (p=0.0001). In the same timeframe, 59% of patients achieved a ≥2 point decrease in IGA scores from baseline, while 82% demonstrated a clinically significant (≥1 point) improvement in IGA scores from baseline. No serious adverse events (SAEs) were reported. 65% of participants experienced at least 1 adverse event (AE), with a total of 35 events."

Late-breaking abstract • P3 data • Dermatology

The Phase 3 Vehicle-Controlled ASCEND Trial of Polyethylene Glycol Based Topical Isotretinoin (TMB-001 0.05% ointment) for Treatment of X-Linked and Autosomal Recessive Congenital Ichthyosis: Pharmacokinetic Results Compared with 80 mg Oral Isotretinoin (AAD 2024) - "Mean AUCt (ng/mL*hr) for oral ISO was 16049.9. PK results from 13 adolescent and adult subjects with RXLI or ARCI treated with TMB-001 0.05% ointment on 75-90% BSA demonstrate ~100 times lower systemic ISO concentrations, compared with plasma ISO and metabolite concentrations in HV following single dose 80 mg oral isotretinoin."

P3 data • PK/PD data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

LEO Pharma to Maintain Leadership Role at AAD in 2024 with New Late-Breaking Data Spanning Multiple Therapeutic Areas (Businesswire) - "LEO Pharma A/S...will present a breadth of new data on moderate-to-severe chronic hand eczema (CHE), congenital ichthyosis (CI) and atopic dermatitis (AD) at the upcoming 82nd American Academy of Dermatology (AAD) Annual Meeting. The event is being held from March 8th to 12th in San Diego, California....The first late-breaker presentation on March 9th will outline the key results of the DELTA-3 trial, which assessed the long-term safety and efficacy of investigational delgocitinib cream in adults with moderate-to-severe CHE....An additional late-breaker presentation on March 10th will outline the preliminary results from the maximum use arm of the ASCEND trial, which examined the efficacy, safety and pharmacokinetics of investigational TMB-001 in adult and adolescent subjects with moderate-to-severe X-linked and Autosomal Recessive CI."

Late-breaking abstract • P3 data • Atopic Dermatitis • Dermatitis • Dermatology • Immunology

The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis (clinicaltrials.gov) - P3 | N=142 | Recruiting | Sponsor: Timber Pharmaceuticals Inc. | Trial completion date: May 2024 ➔ Nov 2024 | Trial primary completion date: Mar 2024 ➔ Sep 2024

Trial completion date • Trial primary completion date • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Topical Isotretinoin (TMB-001) Treatment for 12 Weeks Did Not Result in Clinically Relevant Laboratory Abnormalities in Participants with Congenital Ichthyosis in the Phase 2b CONTROL Study. (PubMed, Dermatol Ther (Heidelb)) - P2 | "There were no clinically significant laboratory changes in participants receiving TMB-001 isotretinoin ointment through 12 weeks of treatment, which differs from reported results for systemic isotretinoin."

Clinical • Journal • P2b data • Atopic Dermatitis • Dermatology • Diabetes • Hematological Disorders • Immunology • Leukopenia • Neutropenia • Vitiligo

Characteristics and outcomes for participants with congenital ichthyosis who responded to treatment with the topical isotretinoin formulation TMB-001: Results from the Phase 2b CONTROL study. (PubMed, Clin Exp Dermatol) - "Higher DLQI and I-NRS scores at baseline were associated with participants achieving treatment success by VIIS-50 and IGA response. Lower BSA involvement was associated with VIIS-50 success."

Journal • P2b data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Phase 2B randomised CONTROL study demonstrates a novel topical isotretinoin formulation, TMB-001, is safe and effective in participants with either recessive X-linked or autosomal recessive lamellar congenital ichthyosis. (PubMed, Clin Exp Dermatol) - "Regardless of CI subtype, TMB-001 demonstrated greater proportions of participants achieving VIIS-50 and ≥2-grade IGA improvement vs vehicle."

Journal • P2b data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

"$TMBR Timber Pharmaceuticals Receives European Orphan Designation for Autosomal Recessive Congenital Ichthyosis & Positive Comments on Pediatric Investigation Plan for TMB-001 in Autosomal Recessive CI & X-Linked Rec https://t.co/Tp7ZKsmRnJ" (@stock_titan)

Clinical • Orphan drug • Atopic Dermatitis • Dermatology • Immunology • Pediatrics • Vitiligo

Congenital ichthyosis subtype analysis of primary efficacy of a novel topical isotretinoin formulation (TMB-001): Results from the Phase 2b CONTROL study in patients with recessive X-linked and autosomal recessive lamellar congenital ichthyosis (EADV 2022) - No abstract available

Clinical • P2b data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Efficacy of topical isotretinoin TMB-001 in children and adults with congenital ichthyosis: Phase 2b CONTROL study results (EADV 2022) - No abstract available

Clinical • P2b data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Efficacy and safety of topical isotretinoin (TMB-001) treatment in patients with X-linked recessive or autosomal recessive lamellar congenital ichthyosis: CONTROL study results (EADV 2022) - No abstract available

Clinical • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Effect of topical isotretinoin (TMB-001) treatment on laboratory parameters in patients with congenital ichthyosis: CONTROL study results (EADV 2022) - No abstract available

Clinical • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Effect of topical isotretinoin formulation (TMB-001) concentration on incidence of local skin reactions in patients with congenital ichthyosis: Phase 2b CONTROL study results (EADV 2022) - No abstract available

Clinical • P2b data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Change in quality-of-life measurements after treatment with topical isotretinoin formulation (TMB-001) at 12 weeks in patients with congenital ichthyosis at baseline: Phase 2b CONTROL study results (EADV 2022) - No abstract available

Clinical • HEOR • P2b data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Characteristics of patients with congenital ichthyosis achieving greater than 50% reduction in Visual Index for Ichthyosis Severity scaling score relative to baseline after topical isotretinoin formulation TMB-001: CONTROL study results (EADV 2022) - No abstract available

Clinical • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis (clinicaltrials.gov) - P3 | N=142 | Recruiting | Sponsor: Timber Pharmceuticals LLC | Not yet recruiting ➔ Recruiting

Enrollment open • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Original Isotretinoin Ointment For Congenital Ichthyosis Presentations Promise - "Amy S. Paller, MD...who modified into asked to comment on the verify, said that she modified into impressed that no significant changes from baseline laboratory scientific assessments were seen. 'If that is exclusively, then we don’t need to be monitoring these patients in the identical formula as with systemic brokers,' said Paller, who modified into fascinated about the phase 2a proof-of-thought trial of TMB-001."

Media quote

Novel isotretinoin ointment for congenital ichthyosis shows promise (MDedge Dermatology) - "Amy S. Paller, MD...who was asked to comment on the study, said that she was impressed that no significant changes from baseline laboratory clinical assessments were observed. 'If that's true, then we don't have to be monitoring these patients in the same way as with systemic agents,' said Dr. Paller, who was involved in the phase 2a proof-of-concept trial of TMB-001. 'I think that deserves more investigation. Hopefully that will be looked at in the phase 3 trial.'"

Media quote

Congenital ichthyosis subtype analysis of primary efficacy of a novel topical isotretinoin formulation (TMB-001): Results from the Phase 2b CONTROL study in patients with recessive X-linked and autoso (AAD 2022) - No abstract available

Clinical • Late-breaking abstract • P2b data • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis (clinicaltrials.gov) - P3 | N=142 | Not yet recruiting | Sponsor: Timber Pharmceuticals LLC

New P3 trial • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis (clinicaltrials.gov) - P2; N=34; Completed; Sponsor: Timber Pharmceuticals LLC; Active, not recruiting ➔ Completed

Clinical • Trial completion • Atopic Dermatitis • Dermatology • Immunology • Vitiligo