Processa Pharmaceuticals, Inc. announced positive top-line results on the clinical symptoms associated with gastroparesis from a 4-week Phase 2A study of PCS12852, which is being developed for the treatment of patients with gastroparesis. The trial was a placebo-controlled, randomized, dose-response study designed to evaluate the safety, efficacy, and pharmacokinetics of two dosage regimens of PCS12852 vs placebo (clinicaltrials.gov identifier NCT05270460). Processa previously announced an improvement in the primary endpoint of the study, the gastric emptying rate, in patients that received a 0.5 mg daily dose of PCS12852.

The results from this Phase 2A study also showed that clinically meaningful improvements in gastroparesis symptoms occurred in more patients (i.e., a greater percentage of patients) receiving the PCS12852 0.5 mg daily dose than the placebo daily dose. The study included 25 patients with moderate to severe gastroparesis that received a daily dose of PCS12852 (0.1 mg or 0.5 mg) or a placebo. Three patients dropped out of the study.

Two patients were in the 0.5 mg group (one because of mild-moderate AEs and one did not provide a reason) and one in the 0.1 mg group (the reason was not provided by the patient). Gastroparesis symptoms were assessed using the American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index Daily Diary (ANMS GCSI-DD), which is a validated patient-reported outcome instrument that captures the daily core symptoms of gastroparesis. Publications have stated that a reduction from the baseline of greater than 0.5 in the total ANMS GCSI-DD score is clinically significant (Revicki 2012; Parkman 2018).

The total GCSI score is the total of 5 symptom subscores which include nausea, vomiting, early satiety, postprandial fullness, and upper abdominal pain. Although the study was not powered to show a statistically significant difference from the placebo, 100% of the patients receiving the 0.5 mg daily dose of PCS12852 and no rescue medication from Day 22-28 had a clinically meaningful reduction in the total ANMS GCSI-DD score (i.e., a reduction of greater than 0.5 from baseline) while only 57% of the placebo group had a clinically meaningful reduction. In addition, the magnitude of the improvement for the total ANNMS GCSI-DD score and for the subscores was greater for the 0.5 mg PCS12852 group than the placebo group.

The 0.1 mg PCS12852 daily dose group showed little to no improvement in gastroparesis symptoms. PCS12852 was shown to be generally well-tolerated, with most AEs occurring in the 0.5 mg dose group and consisting of either a mild or moderate grade. There were no clinically significant cardiovascular, unexpected, severe, or serious adverse events reported during the study. With these positive results from the Phase 2A trial, Processa will be designing the Phase 2B trial and submitting it to their IND.

Depending on priorities, funding, and licensing/partnering opportunities a Phase 2B trial could be initiated in 2023.