Recently, biopharmaceutical company Fortress Biotech announced positive clinical results for CUTX-101 (copper histidinate). The data highlights the efficacy of CUTX-101 for patients with Menkes disease. As there are currently no FDA-approved therapies for Menkes disease, the potential of CUTX-101 is extremely promising. Fortress Biotech and their partner, Cyprium Therapeutics, now hope to submit a New Drug Application (NDA) by the end of 2020.
CUTX-101
To first understand the results of the trial, we need to answer one question: what is CUTX-101? The therapy is administered subcutaneously. It works to replenish copper histidinate and maintain appropriate copper levels in the body. Patients with Menkes disease often have difficulty absorbing copper. As a result, oral therapies are not immensely effective. By injecting CUTX-101, patients have easier access to copper absorption. Additionally, CUTX-101 received Fast Track and Rare Pediatric Disease designations in the US, as well as Orphan Drug designation in both Europe and North America.
In an initial Phase 1/2 clinical trial, CUTX-101 improved survival rates and neurodevelopment in patients with Menkes disease. A Phase 3 clinical trial finished enrollment.
Researchers discovered that patients who received early CUTX-101 treatment reduced the risk of death by 80% in comparison to an untreated control group. Additionally, patients in the treatment group had a median survival rate of 14.8 years, while the untreated control group’s median survival rate was 1.3 years.
Menkes Disease
Menkes disease is an inherited genetic disorder (X-linked) affecting copper metabolism. Resulting from ATP7A gene mutations, Menkes disease alters the way that the body processes copper. As a result, patients often have low levels of copper in their brain and blood. Additionally, they struggle with copper absorption. Our bodies use copper to perform certain functions. Without enough copper, and their activated enzymes, the body experiences a wealth of problems.
Approximately 1 in 35,000 male infants have Menkes disease. In extremely rare cases, it can occur in females. Menkes disease is largely fatal, with many patients dying before 3 years old. However, early treatment (within 1 month of birth) can prolong survival. Many infants with Menkes disease are born prematurely.
Symptoms include:
- Dry skin
- Brittle, sparse, or kinky hair that is gray or white
- Jaundice
- Excess bilirubin in the blood
- Failure to thrive
- Epilepsy
- Intellectual disabilities
- Blood clots
- Arterial thrombosis
- Osteoporosis
- Easy fracturing
- Emphysema
- Blindness
- Hypothermia
Learn more about Menkes disease.
