Diphenylcyclopropenone Ointment for the Treatment of Common Warts

Transcription

1 Poster Presentation #4055 Diphenylcyclopropenone Ointment for the Treatment of Common Warts American Academy of Dermatology 2016 Summer Meeting Boston, MA July 29, 2016 This work was supported by Hapten Pharmaceuticals and RXi Pharmaceuticals NASDAQ: RXII

2 Diphenylcyclopropenone (DPCP) DPCP is a topical immunomodulator Topical treatment for warts, alopecia areata and cutaneous metastasis of melanoma Efficacy in the three target indications reported in peer-reviewed journals Despite extensive safe and effective use, DPCP is not an FDA approved drug Typically compounded No standardized treatment regimen or formulation 2.0% DPCP sensitization dose may lead to hyper-sensitization of patients to challenge doses DPCP is negative in Ames test 2

3 Treatment with DPCP- Examples from Literature Warts (refractory plantar & periungual) Alopecia Areata Cutaneous Metastases of Melanoma (refractory) Choi, Y et al. Ann Dermatol. 2013, 25 4: >350 patients treated for weeks Response rates of 80-85% Cotellessa, C et al. JAAD 2001;44:73-6 >200 patients treated for several months Response rates of 67-78% Damian, DL et al. J Surg Oncol 2014, 109: patients with mean of 15 months treatment 46% complete clearance and 38% partial clearance 3

4 DPCP Ointment Proprietary ointment formulation of diphenylcyclopropenone (DPCP) Sensitizing DPCP Ointment 0.4% DPCP Treatment DPCP Ointment 0.04% DPCP Provides standardized treatment regimen with low sensitization dose In-licensed from Hapten Pharmaceuticals in 2014 Small pilot Phase 2a study conducted by Hapten to evaluate clearance of warts Phase 2a study initiated in 2015 by RXi for treatment of warts Study currently enrolling Orphan-drug designation granted for malignant melanoma stage IIb to IV 4

5 Pilot Study (Hapten Pharmaceuticals) Small Phase 2a pilot study to evaluate topical DPCP ointment for the treatment of common warts Placebo controlled Enrolled subjects aged years with at least one common wart Subjects were sensitized with 0.4% DPCP ointment (or placebo) on the inner arm and on one wart Two weeks later subjects were administered once-weekly treatments of 0.04% DPCP (or placebo) on each target wart for seven weeks Endpoints Efficacy assessed by wart lesion surface area and Investigator s Global Assessment Score (IGAS) of wart clearance Safety assessed by monitoring adverse events 5

6 Pilot Study Results > 50% Clearance of Warts* Total Number of Warts DPCP Placebo P value (Fisher T-test (2-tailed)) DPCP Placebo Intent to treat 38.1% 6.7% Per protocol 47.6% 6.7% Results demonstrate a statistically significant beneficial effect as a wart treatment compared to placebo Greater than 50% clearance was observed in 47.6% of treated subjects over the seven week treatment period Drug-related adverse events were local reactions due to sensitization and challenge responses in the skin Most commonly reported were rash, pruritus, irritation, and erythema after the initial sensitization step * >50% reduction in the area of all treated warts or complete eradication of at least half of the treated warts 6

7 DPCP Ointment Clinical Trial RXI-SCP-1502 Study design based on learnings from pilot study Improved original ointment formulation for better stability and consistency Initiated Phase 2a trial in December 2015 Extended treatment period to 10 weeks Includes optional extension of treatment for subjects with >50% clearance of warts at the end of 10 weeks 7

8 Overview of Phase 2 Clinical Trial RXI-SCP-1502 Phase 2a: Subjects with up to four cutaneous warts on the hands, feet, limbs or trunk Subjects are treated with a sensitization dose (0.4% DPCP) on the inner arm and on one preselected wart lesion After a confirmed sensitization response, subjects target wart lesions will be treated with a treatment dose (0.04% DPCP), weekly for 10 weeks Primary Objectives: To evaluate the effectiveness of Sensitizing DPCP Ointment in eliciting a sensitization response in healthy subjects with common warts To evaluate the effectiveness of Treatment DPCP Ointment in the clearance of common wart lesions over a ten week treatment period with once weekly treatments Secondary Objectives: To evaluate the safety and tolerability of a treatment regimen for common warts consisting of a sensitization dose with Sensitizing DPCP Ointment and ten weekly treatments with Treatment DPCP Ointment To evaluate pharmacokinetics (PK) of DPCP after topical administration of the Sensitization DPCP Ointment in a subset of subjects Currently Enrolling: Expected to be fully enrolled by the end of

9 Authorship Karen Bulock, PhD, RXi Pharmaceuticals, Marlborough MA James Cardia, PhD, RXi Pharmaceuticals, Marlborough MA Natalie Gagne, RXi Pharmaceuticals, Marlborough MA Lyn Libertine, MD, RXi Pharmaceuticals, Marlborough MA Pamela Pavco, PhD, RXi Pharmaceuticals, Marlborough MA William Levis, MD, Hapten Pharmaceuticals LLC, New York NY Geert Cauwenbergh, Dr. Med. Sc., RXi Pharmaceuticals, Marlborough MA 9