Announcement • Jun 27
Cumberland Pharmaceuticals Shares Updated Results From Phase 2 FIGHT DMD Trial Evaluating Ifetroban In Duchenne Muscular Dystrophy Heart Disease Cumberland Pharmaceuticals Inc. shared updated results from its Phase 2 FIGHT DMD trial evaluating ifetroban, a novel oral therapy for Duchenne muscular dystrophy (DMD) heart disease, at the annual Parent Project Muscular Dystrophy (PPMD) conference in Orlando, Florida. The presentation highlighted key updates to the primary findings in the FIGHT DMD trial, reinforcing ifetroban's potential to address the leading cause of death in patients with DMD. The complete set of slides presented at the PPMD conference is now available. The 12-month Phase 2 FIGHT DMD trial (NCT03340675) previously demonstrated that high-dose ifetroban treatment resulted in a significant 5.4% improvement in left ventricular ejection fraction (LVEF) compared to a control group composed of placebo-treated patients combined with propensity score-matched natural history patients. The study findings also included reductions in cardiac damage markers (NT-proBNP and cardiac troponin I) in the high-dose group. All patients who completed the 12-month study opted to continue in the open-label extension. New findings from the Phase 2 FIGHT DMD trial, supported by long-term safety data from the ongoing open-label extension, further characterize the cardioprotective effect exerted by ifetroban in DMD cardiomyopathy. Through 36 months of treatment across both the Phase 2 trial and the open-label extension, ifetroban maintained a favorable safety profile in DMD patients with no treatment-related serious adverse events or new safety concerns. Evaluation of novel blood biomarkers identified a 30% reduction in MYL3 and a 50% reduction in MYOD1 (circulating markers of heart muscle and cell damage), alongside a 2.4-fold increase in FGF16 and a 2.1-fold increase in TSPAN7 (cardioprotective and tissue repair proteins) with ifetroban treatment compared with placebo. These confirmatory findings further support the potential therapeutic mechanisms for ifetroban in DMD cardiomyopathy. Ifetroban is a once-daily oral medication that works by blocking the thromboxane receptor, which plays a key role in inflammation and fibrosis. The drug has received Orphan Drug Designation, Rare Pediatric Disease Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) for the indication of cardiomyopathy associated with DMD. There is currently no approved treatment specifically targeting DMD heart disease, highlighting the critical unmet medical need in this patient population where cardiac complications are universal and represent the leading cause of death. Cumberland has secured a growing portfolio of patents protecting the product for this DMD heart disease indication. Next steps include the completion of long-term treatment analyses, and the conduct of additional supportive studies. Announcement • Jun 03
Cumberland Pharmaceuticals and Vanderbilt Health Announce Positive Results from Phase 2A Clinical Trial of Ifetroban for Metastasis Prevention in High-Risk Solid Tumors Cumberland Pharmaceuticals Inc. and Vanderbilt Health announced data from a Phase 2a clinical trial of ifetroban to prevent metastasis in high-risk solid tumors. The study's primary safety endpoint was achieved, along with favorable trends in decreased metastasis recurrence and metastasis-free survival. The randomized, double-blind, placebo-controlled Phase 2a trial evaluated the safety of ifetroban, an investigational thromboxane A2 receptor antagonist, in patients with solid tumors at high risk of early metastatic recurrence. Cancer types included breast, lung, pancreatic, soft tissue, bladder, and renal cancers. The study met its primary endpoint, demonstrating that ifetroban was safe and well-tolerated in this patient population. Rates for adverse events related to treatment were similar between placebo and ifetroban. No serious adverse events (> grade 3) in either group were identified as being related to study treatment. Treatment discontinuation rates were not statistically different between placebo and ifetroban. Although primarily a safety study and intentionally not powered for efficacy, the study compared the percentage of patients with distant metastatic recurrence 12 months after completion of therapy in both groups (10 placebo-treated and 18 ifetroban-treated participants) as a prespecified secondary endpoint. While 50% of participants experienced distant metastatic recurrence in the placebo arm, only 17% of participants experienced distant metastatic recurrence in the ifetroban arm (p=0.091). Three deaths due to distant metastatic disease occurred in the placebo arm, and none occurred in the ifetroban arm (p=0.037). The premise of this novel therapy is that antagonizing the thromboxane A2 receptor and blocking platelet activation and aggregation lessens tumor cells' ability to migrate, spread, cluster, invade distal organs, and evade immune detection. This was the first trial evaluating the effects of ifetroban in people with solid tumors with high risk for early recurrence, defined as = 50% chance of recurrence within 5 years of diagnosis. The intervention was given after all cancer-related therapies and surgical procedures had been completed; participants received the intervention for 12 months and were then followed for an additional 12 months. Among 29 participants, 10 received placebo and 19 received ifetroban. This clinical trial translated robust in silico and preclinical data to humans, confirming safety of ifetroban in patients with solid tumors and preliminarily suggesting that ifetroban may target biologic mechanisms involved in distant metastatic recurrence. A phenome-wide association study (PheWAS) was conducted by Vanderbilt Health investigators using the BioVU biorepository, which linked a naturally occurring genetic variant in the thromboxane receptor gene (TBXA2R) to an increased risk of metastatic disease across multiple cancer types. Preclinical studies subsequently published in Molecular Cancer Therapeutics demonstrated that ifetroban reduced metastasis in several animal models without affecting tumor growth, and that the drug's effects appeared to involve strengthening of the vascular endothelial barrier and inhibiting the ability of tumor cells to migrate across blood vessel walls. The favorable safety profile of ifetroban in this patient population, combined with the efficacy signals observed in this study, supports continued investigation of ifetroban as a candidate for metastasis prevention. Results of this Phase 2a clinical trial will be used to guide the further clinical development verifying efficacy and further demonstrating safety. Ifetroban is a potent and selective thromboxane-prostanoid receptor (TPr) antagonist. It exhibits high affinity for TPr on many cell types including platelets, cardiomyocytes, vascular and airway smooth muscle, and fibroblasts, and lacks agonistic activity. Cumberland is also evaluating ifetroban in Phase 2 clinical programs for patients with Duchenne Muscular Dystrophy, Systemic Sclerosis and Idiopathic Pulmonary Fibrosis. Ifetroban has a favorable safety profile as evidenced by multiple completed clinical trials collectively enrolling over 1,400 people. Cumberland's Phase 2 clinical programs are evaluating ifetroban in patients with Duchenne Muscular Dystrophy, Systemic Sclerosis, and Idiopathic Pulmonary Fibrosis, in addition to the oncology program described in this release. Announcement • May 28
Cumberland Pharmaceuticals Launches Vibativ Injection in China in Strategic Partnership with SciClone Pharmaceuticals (Holdings) Limited Cumberland Pharmaceuticals Inc. announced the launch of its Vibativ (telavancin) injection in China, following regulatory approval and a strategic partnership with SciClone Pharmaceuticals (Holdings) Limited. The launch follows a previously announced agreement granting SciClone exclusive rights to register, promote and distribute telavancin, injection across China. The product's approval by China's National Medical Products Administration (NMPA) enables SciClone to commercialize the product in one of the world's largest and fastest-growing pharmaceutical markets. Telavancin is a patented, FDA-approved injectable anti-infective that serves as a potentially life-saving treatment for patients with serious bacterial infections, including hospital-acquired and ventilator-associated pneumonia. The therapy is designed to treat infections due to Staphylococcus aureus (S. aureus) and other Gram-positive bacteria, including Methicillin-resistant Staphylococcus aureus (MRSA) and Methicillin-sensitive Staphylococcus aureus (MSSA). Vibativ addresses a range of Gram-positive bacterial pathogens, including those that are considered difficult-to-treat and multidrug-resistant. Antimicrobial resistance continues to be a critical global health challenge, reducing the effectiveness of many existing antibiotics and increasing the need for innovative treatments. Telavancin was specifically designed to address resistant pathogens, offering potent activity against difficult-to-treat infections. The successful launch of telavancin for injection in China represents another important milestone for SciClone Pharmaceuticals in the anti-infective field. Currently, MRSA remains a serious challenge and poses a significant clinical threat, particularly in respiratory tract infections, especially those that are hospital-acquired. With its unique dual bactericidal mechanism, broad antibacterial spectrum, and once-daily dosing regimen, telavancin offers an important new anti-infective option for Chinese patients with severe infections, delivering both improved efficacy and clinical convenience. Vibativ (telavancin) injection was discovered in a research program dedicated to finding new antibiotics for serious infections due to Staphylococcus aureus (S. aureus) and other Gram-positive bacteria, including MRSA and MSSA. It is a once-daily, injectable lipoglycopeptide antibiotic with in vitro potency, bactericidal activity within six hours and penetration into target infection sites. The drug is approved in the U.S. and Saudi Arabia for the treatment of adult patients with hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP) caused by susceptible isolates of S. aureus when alternative treatments are not suitable. In addition, telavancin is approved in the U.S. and Saudi Arabia for the treatment of adult patients with complicated skin and skin structure infections (cSSSI) caused by susceptible isolates of Gram-positive bacteria, including S. aureus, both methicillin-susceptible (MSSA) and methicillin-resistant (MRSA) strains. The product labeling also describes the use of telavancin in treating patients whose pneumonia or skin infection is complicated by concurrent bacteremia. The product's proven efficacy against difficult-to-treat Gram-positive infections has been demonstrated in several large, multinational registrational studies, which involved one of the largest cohorts of patients with S. aureus infections studied to date. Importantly, these studies demonstrated significantly higher cure rates for telavancin as compared to vancomycin in HABP/VABP due to any single Gram-positive pathogen or S. aureus with vancomycin MIC =1 µg/mL. Additionally, there is extensive and well-documented evidence of the drug's in vitro potency and in vivo activity against a broad collection of Gram-positive bacterial pathogens, including those that are considered difficult-to-treat and multidrug-resistant. 
