Technology
”CPT-001 has a first in class mode of action and block lipid transport in mitochondria and thereby regulate fat and glucose metabolism”

CPT-001 is in-licensed and developed by CPT.one Biotech.

The development was based on early development by Medigene, a German biotech company within diabetes, cardiological and autoimmune diseases. CPT.one Biotech production and use rights are based on a licensing agreement and patents and patent applications developed by CPT.one Biotech and focused on MS.

CPT-001 has a first in class mode of action and block lipid transport in mitochondria and thereby regulate fat and glucose metabolism. CPT-001 has been shown to be highly efficacious to prevent and treat MS in state-of-the-art animal disease models.

CPT.one Biotech has developed a unique approach relating to top international research on mitochondrial CPT1 that affect metabolism, the immune system and inflammation being key in MS. The scientific basis for the development of the drug candidate is that MS progress due to an imbalance in the metabolic, immunological, and inflammatory components.

CPT.one Biotech has a strong IP position which includes in-licensed IP from Medigene AG and pending patent applications and issued patents on the use of CPT-001 to treat MS filed by CPT.one Biotech.

CPT.one Biotech

Multiple sclerosis a dysfunction of lipid metabolism.

CPT1 is the key molecule in lipid metabolism.

CPT1 and lipid metabolism is up regulated (up to 17 times).

Several receptors use lipids for signaling.

”CPT-001 blocks lipid transport in the mitochondria and regulate fatty acid and glucose
metabolism, and the research leading to this is pioneering regarding MS treatment.”

CPT-001 was developed by German based Medigene within diabetes and heart diseases with promising phase 1 clinical trial data for safety and tolerability.

CPT.one Biotech has licensed the method for producing CPT-001 from Medigene and has further developed the candidate within MS, filed patent applications and have had several patents issued.

CPT-001 blocks lipid transport in the mitochondria and regulate fatty acid and glucose metabolism, and the research leading to this is pioneering regarding MS treatment.

CPT-001 of CPT1 reestablishes deficiencies in myelin, immunological and cognitive deficiencies. This is shown in state-of-the-art animal disease

models and transgenic models in MS. Some ethnic groups with a high occurrence of genetic mutations in CPT1 that strongly reduces CPT1 activity have a significantly lower risk of neurodegenerative diseases, including MS.

CPT-001 have neuroprotective abilities on fatty-acid metabolism and immune responses to reestablish fatty-acid metabolism and the immune response, prevention and regenerating of motoric and cognitive disease elements in MS. In summary, it is our observation in animal models that inhibition of mitochondrial fatty acid import with CPT-001 can reverse neurologic deficits and our medical hypothesis that a similar effect can be obtained in human MS patients.

Pipeline

The target is mitochondrial CPT1 and treatment reestablishes defectives in myelin sheets.

Toxicology studies have shown safety/tolerability within relevant therapeutic dosing regimes.

Ready for phase 2 clinical trials.

Research collaborations

The platform and CPT-001 is developed by Jette Goller Kloth and Aalborg University in cooperation with
Medigene AG, University of Copenhagen and University of Aarhus. Primary focus is secondary progressive multiple sclerosis (SP-MS).