Cumberland's 41-Patient Ifetroban Trial In Scleroderma Backs Safety, Not Efficacy
The pilot study was not designed or powered to test whether the drug works, the company said, and most patients showed limited progression.
Cumberland Pharmaceuticals, a specialty drugmaker, said Friday that its Phase 2a trial of oral ifetroban in systemic sclerosis, a rare autoimmune disease, supports the drug's safety and tolerability but was not built to show whether it works.
Safety signal, no efficacy verdict
The 12-month ASSIST-SSc pilot randomized 31 patients to ifetroban and 10 to placebo, the company said. Of those on the drug, 22 had diffuse cutaneous disease and 9 had pulmonary arterial hypertension.
Gastrointestinal adverse events were more frequent on ifetroban than on placebo, though the company said most were mild and resolved. Bleeding-related events rose modestly, consistent with the drug's mechanism, it said.
No serious adverse event during the 12-month treatment period was assessed by the medical monitor as related to the study drug, the company said.
The trial was not designed or powered to evaluate efficacy, Cumberland said. Most patients in both groups showed limited disease progression, which reduced the ability to detect a difference between treatments.
A drugmaker with a candidate in testing
Cumberland sells branded prescription drugs in hospital acute care, gastroenterology and oncology, including Acetadote, Caldolor, Kristalose, Sancuso, Vibativ and Talicia. Ifetroban is a drug candidate it is testing in clinical studies.
The company said about 100,000 people in the U.S. have systemic sclerosis and that no approved therapy targets the disease's primary cardiac problems.
Next steps and a Monday update
Cumberland plans to submit the clinical study report to the FDA, complete biomarker studies on trial specimens and evaluate cardiac imaging from patients in the open-label extension.
Separately, the company had told investors it would update the market on ifetroban on October 26. That update concerns the drug's development for cardiomyopathy tied to Duchenne muscular dystrophy, a different program from the scleroderma study.
The next dated item is Monday, October 26, when Cumberland is due to update the market on ifetroban in Duchenne-related cardiomyopathy.
Sources
- 8-K Filing — CUMBERLAND PHARMACEUTICALS INC (CPIX) — SEC EDGAR
- Cumberland Pharmaceuticals Higher; Drug Update Slated for Oct. 26 — MarketWatch
- Cumberland Pharmaceuticals Announces ASSIST-SSc Trial Top-Line Results — prnewswire.com
