Cumberland Pharmaceuticals (CPIX) Study update summary

FIGHT DMD design and results

• FIGHT DMD was a 52-week, randomized, placebo-controlled Phase 2 trial of ifetroban added to standard therapy; 41 patients aged 7+ received 300 mg/day (n=18), 100 mg/day (n=12) or placebo (n=11), with cardiac MRI LVEF as the primary endpoint.

• At 12 months, high-dose ifetroban showed a 5.4-percentage-point LVEF advantage versus placebo plus matched natural-history controls (p=0.002); the cross-study comparison was not head-to-head.

• LVEF changed +1.8 points with high dose and 0 points with low dose, versus -1.5 points with placebo, -3.6 points for placebo plus natural history, and -4.3 points for natural history.

• NT-proBNP decreased by an average 54 pg/mL with high-dose ifetroban versus control (p=0.017); cardiac troponin I also decreased, and exploratory proteomics found FGF16 +2.4-fold, TSPAN7 +2.1-fold, MYL3 -30% and MYOD1 -50%.

• The open-label extension included 21 participants through 36 months; all trial completers chose to enter the extension.

NDA plans and safety

• Plans call for a Traditional Approval NDA based on FIGHT DMD and two natural-history databases; FDA granted rolling review, allowing completed sections to be submitted before the full application.

• FDA requested additional efficacy analysis, particularly on missing data, and raised questions about substantial evidence, the LVEF endpoint, comparators and statistical analysis.

• Planned NDA materials include open-label extension cardiac MRI at months 24 and 36, preclinical data, manufacturing information and a safety database covering ~1,400 subjects across ~30 studies.

• No treatment-related serious adverse events or new safety concerns were reported through 36 months; DMD participants accumulated ~90 patient-years. Bruising, petechiae, nausea, rash and weight gain were listed as probably related adverse events.

• DMD program has Orphan Drug, Rare Pediatric Disease and Fast Track designations; FDA conditionally accepted Dyscorban as the proprietary name, and patents extend through May 2035.

Treatment context and pipeline

• Ifetroban is a once-daily oral thromboxane receptor blocker; three Phase 2 programs target Duchenne cardiomyopathy, systemic sclerosis and idiopathic pulmonary fibrosis.

• DMD cardiomyopathy has no approved therapy; current treatments slow but do not stop or reverse heart-function decline, and heart disease is a leading cause of death in DMD.

• Systemic sclerosis Phase 2a ASSIST-SSc topline results showed generally good tolerability and no treatment-related serious adverse events; the trial was not designed or powered to assess efficacy.

• Planned systemic sclerosis work includes submitting the clinical study report and completing biomarker and cardiac-imaging analyses; the Phase 2 IPF study is enrolling, and the oncology metastasis-prevention Phase 2a is complete.