The company expects to report topline data for the Phase 1/2 study in March 2021.
NPC1 is a rare genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to a defect in the NPC1 protein which is responsible for cholesterol processing in the cell.
As cholesterol accumulates in cells, NPC causes symptoms in the brain, liver, spleen, lung and other organs and often leads to premature death.
There are no approved drug therapies for NPC in the United States and only one approved therapy in Europe.
The randomized, double-blind Phase 1/2 study of Trappsol Cyclo enrolled 12 patients aged 2 and above. Trappsol Cyclo was evaluated in 3 dose groups (1500, 2000, 2500 mg/kg body weight) administered IV in NPC patients aged 2-plus bi-weekly over 48 weeks.
To-date, individual and cumulative safety data show the drug to be well tolerated with a favorable safety profile for all dose groups.
Previously reported data from interim analyses show effects of IV drug administration on markers of cholesterol synthesis and metabolism, indicating clearance of cholesterol from cells.
Pharmacokinetic data show the drug in cerebrospinal fluid during and even after the end of IV infusion, at all dose levels. Lysosphingomyelin-509 in plasma demonstrates a clear downward trend over the 48-week study, with no apparent dose-relationship, further supporting the drug's ability to clear lipids from cells.
Tau, a biomarker of neurodegeneration, is reduced in cerebrospinal fluid of patients who opted for lumbar punctures at 24 and 48 weeks, suggesting a neuroprotective effect of the drug.
For the 7 patients who completed the trial as of September 2020, 6 met the first efficacy criterion of the study related to improvement in 2 domains of the 17-domain Severity Scale.
For the second efficacy outcome measure, change from baseline in global impression of disease, 5 of 7 patients improved per the Clinicians Global Impression of Improvement scale and 2 were stabilized.
Following review of the Phase 1 and Phase 1/2 data, coupled with preclinical and compassionate use data, regulatory authorities acknowledged that IV Trappsol Cyclo has the potential to treat systemic and neurologic manifestations of NPC.
Additionally, the European Medicines Paediatric Committee (EMA PDCO) noted Trappsol Cyclo may have the capacity when given intravenously to be a preventative treatment. The company has confirmation that the pivotal Phase 3 study may begin enrollment, which is expected to commence in 2Q21.
For more information about the Phase 1/2 study, visit clinicaltrials.gov and reference NCT02912793.
Cyclo Therapeutics is a clinical-stage biotechnology company whose Trappsol Cyclo, an orphan drug designated product in the United States and Europe, is the subject of three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease.
The company is planning an early phase clinical trial using Trappsol Cyclo intravenously in Alzheimer's Disease based on encouraging data from an Expanded Access program for late-onset Alzheimer's Disease (NCT03624842).
UroGen's UGN-103 IND accepted by FDA for bladder cancer treatment
MaaT Pharma reveals positive 18-month data for MaaT013 in GI-aGvHD
Innate Pharma advances Sanofi-developed NK cell engager to Phase 2 for blood cancer patients
Soligenix receives orphan drug designation from FDA for active ingredient in SuVax
Candel Therapeutics granted FDA Orphan Drug Designation for CAN-2409 in pancreatic cancer treatment
Lipogems completes patient enrolment in ARISE I US FDA IDE study
Amylyx Pharmaceuticals' AMX0035 shows promising impact on Wolfram syndrome symptoms
Roche attains CE Mark for first companion diagnostic for HER2-low metastatic breast cancer
Cadrenal Therapeutics' tecarfarin receives US FDA Orphan Drug Designation
BioCity Biopharma's BC2027 Phase one study IND application receives US FDA approval
Seres Therapeutics completes patient enrollment for SER-155 Phase 1B trial