Table 2.
Summary of published pilot and Phase II studies investigating the efficacy and safety of apremilast in a variety of conditions
| Lead author | Date | Disease | Type | Treatments | Number enrolled | Outcome | Adverse effects |
|---|---|---|---|---|---|---|---|
| Papp et al50 | June 2012 | Psoriasis | Phase IIb | 10 mg, 20 mg, 30 mg BID, and placebo. | 352 | Subjects treated with apremilast at 10 mg, 20 mg, and 30 mg BID reached PASI-75 at the respective rates: 11%, 29%, and 41%. This was compared to 6% of the placebo group. | No serious AE seen. Headaches and nausea reported. |
| Papp et al45 | August 2012 | Psoriasis | Phase II | 20 mg SID, 20 mg BID, and placebo. | 259 | 24.4% of subjects treated with 20 mg BID of apremilast reached PASI-75. Both the 20 mg SID and placebo groups saw 10.3% of patients reach PASI-75. | Few more AE compared to apremilast, but most considered mild and tolerable. |
| Schett et al51 | October 2012 | Psoriatic arthritis | Phase II | 20 mg BID, 40 mg SID, and placebo. | 204 | Apremilast was more successful at achieving ACR20, 20 mg BID (43.5%), 40 mg SID (35.8%), compared to placebo (11.8%). Rerandomizing the placebo group after the 12-week period also proved that apremilast was effective. | Slightly higher incidence of AE with apremilast compared to placebo, but not significant. |
| Samrao et al59 | August 2012 | Atopic dermatitis | Pilot study | 20 mg, 30 mg BID, and no placebo or control. | 83 | Small dose-dependent effect observed with apremilast. Results were moderate, but did show statistical significance. | No serious AE seen. Headaches and nausea reported. |
| Pathan et al55 | September 2012 | AS | Phase II | 30 mg BID and placebo. | 38 | The primary endpoint was not met, but six patients treated with apremilast met ASA20, compared to three on placebo. Significant reduction in serum RANKL was observed by apremilast treatment. | Similar AE observed between apremilast and placebo. Higher rate of headaches and loose stools in the apremilast group. Two patients withdrew from the apremilast group due to severe AE. |
| Gottlieb et al52 | May 2008 | Psoriasis | Phase II | 20 mg SID and no placebo or control. | 19 | Epidermal thickness was reduced by a mean of 20.5 mm, and PASI score improved in 73.7% of patients. Apremilast reduced the number of T cells and CD11c cells. | 73.7% patients experienced at least one AE. Headaches and nausea reported. |
| Paul et al60 | August 2012 | Lichen planus | Pilot study | 20 mg BID and no placebo or control. | 10 | Three out of ten patients achieved a two-grade improvement in PGA. All patients demonstrated statistically significant clinical improvement. | No serious AE seen. Headaches and nausea reported. |
| Volf et al61 | March 2012 | Recalcitrant allergic contact or atopic dermatitis | Phase II | 20 mg BID and no placebo or control. | 10 | Two patients displayed ≥ 2 improvement of Investigator Global Assessment at week 12. Secondary endpoints of EASI-75 and EASI-50 were met by 10% and 20% of patients, respectively. | No serious AE seen. Headaches and nausea reported. |
| De Souza et al62 | October 2012 | Discoid lupus erythematosus | Phase II | 20 mg BID and no placebo or control. | 8 | Significant decrease in CLASI score after 85 days observed in patients. | No serious AE seen. Headaches and nausea reported. |
Abbreviations: BID, twice a day; PASI, Psoriasis Area and Severity Index; AE, adverse events; SID, once a day; ACR20, American College of Rheumatology; AS, ankylosing spondylitis; RANKL, receptor activator of nuclear factor kappa-B ligand; EASI, Eczema Area Severity Index; CLASI, Cutaneous Lupus Erythematosus Disease Area and Severity Index.