Long-Term Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: Follow-Up Data from a 3-Year Open-Label Extension Study
Background: Dermatomyositis (DM) is a rare autoimmune condition involving skin manifestations often resistant to standard treatments such as immunosuppressants and antimalarials. Biopsies show elevated inflammatory cells such as CD4+ T cells, dendritic cells, and cytokines. Lenabasum, a selective ca...
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Elsevier
2025-01-01
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| author | Caroline J. Stone Geeta Ahuja Lais Lopes Almeida Gomes Joy Poroye Daniella Forman Faden Lillian Xie Rui Feng Barbara White Victoria P. Werth |
| author_facet | Caroline J. Stone Geeta Ahuja Lais Lopes Almeida Gomes Joy Poroye Daniella Forman Faden Lillian Xie Rui Feng Barbara White Victoria P. Werth |
| author_sort | Caroline J. Stone |
| collection | DOAJ |
| description | Background: Dermatomyositis (DM) is a rare autoimmune condition involving skin manifestations often resistant to standard treatments such as immunosuppressants and antimalarials. Biopsies show elevated inflammatory cells such as CD4+ T cells, dendritic cells, and cytokines. Lenabasum, a selective cannabinoid receptor 2 agonist, has demonstrated significant benefits in treating autoimmune skin diseases. Objectives: This study utilizes data from the open-label extension (OLE) phase of the lenabasum phase 2 trial and additional post-OLE follow-up data. Key aims include evaluating the drug’s long-term effectiveness and assessing disease manifestation recurrence. Methods: The phase 2 lenabasum trial enrolled patients with treatment-resistant, skin-predominant DM. The OLE consisted of a 3-year period during which 20 patients were on the drug for the entire duration, with assessments every 8 weeks to evaluate drug safety and efficacy. Subsequently, a follow-up retrospective chart review was performed on patients who completed the OLE as well as on control subjects with DM who did not participate in the lenabasum trial. Results: By week 68, patients exhibited reductions in Cutaneous Dermatomyositis Disease Area and Severity Index activity score (−21.8), Patient Skin Activity Visual Analog Scale (−3.0), and Skindex-29 (−28.0) from OLE baseline. After OLE, 58.3% maintained stable disease, significantly higher than controls (P = .035), with 41.7% not experiencing flares compared with 91.6% of controls. In addition, 50% of patients reported sustained pruritus improvement. Conclusions: Data from OLE and subsequent follow-up periods demonstrate lenabasum’s efficacy in maintaining disease stability, reducing flares, and improving DM symptoms, suggesting that it is a promising option for patients with treatment-resistant skin-predominant DM. Trial Registration: This study was registered at clinicaltrials.gov, with NCT02466243. Study registration was first submitted on June 2, 2015. |
| format | Article |
| id | doaj-art-0cf3a28457564cb4a0e070bee14deb36 |
| institution | Kabale University |
| issn | 2667-0267 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Elsevier |
| record_format | Article |
| series | JID Innovations |
| spelling | doaj-art-0cf3a28457564cb4a0e070bee14deb362025-01-11T06:42:09ZengElsevierJID Innovations2667-02672025-01-0151100311Long-Term Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: Follow-Up Data from a 3-Year Open-Label Extension StudyCaroline J. Stone0Geeta Ahuja1Lais Lopes Almeida Gomes2Joy Poroye3Daniella Forman Faden4Lillian Xie5Rui Feng6Barbara White7Victoria P. Werth8Corporal Michael J. Crescenz Veterans Affairs Medical Center, Philadelphia, Pennsylvania, USA; Department of Dermatology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USACorporal Michael J. Crescenz Veterans Affairs Medical Center, Philadelphia, Pennsylvania, USA; Department of Dermatology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USACorporal Michael J. Crescenz Veterans Affairs Medical Center, Philadelphia, Pennsylvania, USA; Department of Dermatology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USACorporal Michael J. Crescenz Veterans Affairs Medical Center, Philadelphia, Pennsylvania, USA; Department of Dermatology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USACorporal Michael J. Crescenz Veterans Affairs Medical Center, Philadelphia, Pennsylvania, USA; Department of Dermatology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USACorporal Michael J. Crescenz Veterans Affairs Medical Center, Philadelphia, Pennsylvania, USA; Department of Dermatology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USADepartment of Biostatistics, Epidemiology & Informatics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USACorbus Pharmaceuticals, Norwood, Massachusetts, USACorporal Michael J. Crescenz Veterans Affairs Medical Center, Philadelphia, Pennsylvania, USA; Department of Dermatology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA; Correspondence: Victoria P. Werth, Department of Dermatology, School of Medicine, University of Pennsylvania, Perelman Center for Advanced Medicine, Suite 1-330A, 3400 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA.Background: Dermatomyositis (DM) is a rare autoimmune condition involving skin manifestations often resistant to standard treatments such as immunosuppressants and antimalarials. Biopsies show elevated inflammatory cells such as CD4+ T cells, dendritic cells, and cytokines. Lenabasum, a selective cannabinoid receptor 2 agonist, has demonstrated significant benefits in treating autoimmune skin diseases. Objectives: This study utilizes data from the open-label extension (OLE) phase of the lenabasum phase 2 trial and additional post-OLE follow-up data. Key aims include evaluating the drug’s long-term effectiveness and assessing disease manifestation recurrence. Methods: The phase 2 lenabasum trial enrolled patients with treatment-resistant, skin-predominant DM. The OLE consisted of a 3-year period during which 20 patients were on the drug for the entire duration, with assessments every 8 weeks to evaluate drug safety and efficacy. Subsequently, a follow-up retrospective chart review was performed on patients who completed the OLE as well as on control subjects with DM who did not participate in the lenabasum trial. Results: By week 68, patients exhibited reductions in Cutaneous Dermatomyositis Disease Area and Severity Index activity score (−21.8), Patient Skin Activity Visual Analog Scale (−3.0), and Skindex-29 (−28.0) from OLE baseline. After OLE, 58.3% maintained stable disease, significantly higher than controls (P = .035), with 41.7% not experiencing flares compared with 91.6% of controls. In addition, 50% of patients reported sustained pruritus improvement. Conclusions: Data from OLE and subsequent follow-up periods demonstrate lenabasum’s efficacy in maintaining disease stability, reducing flares, and improving DM symptoms, suggesting that it is a promising option for patients with treatment-resistant skin-predominant DM. Trial Registration: This study was registered at clinicaltrials.gov, with NCT02466243. Study registration was first submitted on June 2, 2015.http://www.sciencedirect.com/science/article/pii/S2667026724000584AutoimmunityClinical researchClinical trialsConnective tissue disordersDrug development |
| spellingShingle | Caroline J. Stone Geeta Ahuja Lais Lopes Almeida Gomes Joy Poroye Daniella Forman Faden Lillian Xie Rui Feng Barbara White Victoria P. Werth Long-Term Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: Follow-Up Data from a 3-Year Open-Label Extension Study JID Innovations Autoimmunity Clinical research Clinical trials Connective tissue disorders Drug development |
| title | Long-Term Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: Follow-Up Data from a 3-Year Open-Label Extension Study |
| title_full | Long-Term Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: Follow-Up Data from a 3-Year Open-Label Extension Study |
| title_fullStr | Long-Term Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: Follow-Up Data from a 3-Year Open-Label Extension Study |
| title_full_unstemmed | Long-Term Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: Follow-Up Data from a 3-Year Open-Label Extension Study |
| title_short | Long-Term Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: Follow-Up Data from a 3-Year Open-Label Extension Study |
| title_sort | long term safety and efficacy of lenabasum a cannabinoid receptor type 2 agonist in patients with dermatomyositis with refractory skin disease follow up data from a 3 year open label extension study |
| topic | Autoimmunity Clinical research Clinical trials Connective tissue disorders Drug development |
| url | http://www.sciencedirect.com/science/article/pii/S2667026724000584 |
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