· Comprehensive pharmacology/toxicology program comprising of multiple studies
· Phase IIa dose escalating study
· Phase IIb dose range study (Data released Feb 2010)
· PRIME ESA-sparing study demonstrated a statistically significant 35% reduction in ESAs with Triferic and by 74% in hypo-responders (20% of ESRD population which typically use 2-3 times more ESA than normal dosing) compared to placebo (Data released Feb and May 2013)
· Phase III CRUISE-1 and CRUISE-2 Efficacy clinical trials met the primary endpoint demonstrating a statistically significant mean change in hemoglobin from baseline to end-of-treatment (p value .011). Triferic also effectively delivered iron via dialysate and maintained hemoglobin without increasing iron stores (ferritin). (Data released July and September 2013)
· Received U. S. Food & Drug Administration approval on January 24, 2015 for commercial sale as an iron replacement product to maintain hemoglobin in adult patients with hemodialysis dependent chronic kidney disease.
· Received the Centers for Medicare and Medicaid Services (CMS) unique product reimbursement code Q9976 for Triferic on June 1, 2015 and launched commercially in the U.S. on September 9, 2015.
· Rockwell Medical continues to seek commercial collaborations to license and develop our unique drug therapies on a global scale. The Company 100% owns the world-wide rights to Triferic, a one-of-a-kind first to market iron drug approved by the FDA for iron replacement and maintenance of hemoglobin in hemodialysis patients. The Company believes Triferic will become the standard of care in iron maintenance therapy and address an important unmet need in the maintenance of hemoglobin in ESRD patients. As of 2014, world-wide dialysis population was 2.67 million with forecasted over 6.3% growth every year to 2020.
· Clinical developments for treatment of new iron-deficiency anemia indications have begun for: Triferic TPN Solution, Triferic PD Solution, Triferic IV Solution, and Triferic Orphan Indication
PRIME ESA sparing study has been completed and demonstrated a statistically significant 35% overall ESA reduction and by 74% in hypo-responders representing approximately 20% of ESRD population which typically use 2-3 times more ESA than normal dosing. Data released in February and May 2013.
The PRIME study data showed that regular administration of Triferic via dialysate over a 9-month period significantly reduced the amount of ESAs necessary for patients on hemodialysis compared to placebo.
The PRIME study was a multi-center, 103-patient, randomized controlled nine-month study with patients receiving either Triferic dialysate or standard (iron free) dialysate. IV iron sucrose (Venofer ®) can be dosed in either group per protocol in cases of iron deficiency. Clinical study design was aimed at demonstrating ESA sparing as well as examine maintenance of hemoglobin, iron parameters, the need for intravenous (IV) iron, and oxidative stress markers in patients receiving Triferic.
Final Phase III Long-Term Safety Study
A 718-patient Phase III crossover safety study of Triferic in dialysate was completed January 2014. This clinical program is the largest and longest cohort of any parenteral iron drug with over 100,000 separate Triferic administrations.
These safety studies were the continuation of the Cruise efficacy trials and Study SFP-6, a prospective, randomized, double-blinded, placebo-controlled, crossover, multicenter, multinational, Phase 3 study with an enrollment of 718 CKD-HD patients in the US and Canada. Both studies allowed patients to continue to receive Triferic in the open-label extensions study for up to one additional year. Consistent with expectations, a preliminary review of the safety data revealed:
Triferic reliably delivered iron in dialysate to the bone marrow.
There were no specific adverse events directly attributable to Triferic.
The adverse events during Triferic administrations were those common in CKD-HD patients.
No Difference in type, frequency, severity or resolution of adverse events between Triferic and placebo.
No anaphylaxis or hypersensitivity attributable to Triferic.
No evidence of changes in hepatic enzymes between Triferic and placebo.
No evidence of first use events or an increase in intradialytic hypotension.
Overcomes reticuloendothelial block.
Reduces need for transfusions.