Slide 6, provides a brief introduction to Chondrial. We are clinical stage biotech company with a novel [ph]
protein replacement therapy platform to address untreated serious and complex rare diseases.
Our lead candidate is
CTI-1601 for which we are also excited to announce today that we recently began dosing in a Phase 1 clinical trial in the United States for the potential treatment of patients with Freidreichs Ataxia.
CTI-1601 is the only Frataxin protein replacement therapy in clinical development today. Nine clinical studies
have shown promising results in several models of the disease including heart, brain and muscle function as well as overall survival. Ill provide more details later in my remarks.
Chondrial also has a strong board of directors and leadership team. We have a broad portfolio of I.T. with at least 12 years of market exclusivity expected
upon approval. We have also licensed and filed multiple patents around the molecule and efficacy biomarkers.
Finally, Chondrial was created, incubated
and funded by Deerfield Management who remains our primary shareholder. Id like to thank them for their continued support. Turning to slide 7, Ill provide a quick intro on our proprietary protein replacement platform.
Chondrial has demonstrated the ability to design differentiated fusion proteins utilizing various Cell Penetrating Peptides, or CPP. CPPs enable intracellular
delivery of bioactive cargoes. This technology is both highly relevant and applicable to a wide range of cargoes and can be used in a number of rare disease indications.
The non-clinical data generated by CTI-1601 has demonstrated the potential of
our platform. Before I discuss CTI-1601 in detail, I wanted to quickly provide some insights into the devastating disease that Freidreichs Ataxia or F.A. as shown on slide 8.
It is a rare disease caused by a genetic defect resulting in abnormally low levels of protoxen, a protein located in the mitochondrion. The disease affects
approximately 5,000 patients in the United States and approximately 10,000 patients in the E.U. It is a progressive, irreversible, systemic disease that affects multiple body systems particularly the brain and the heart.
The age of onset is correlated with severity and speed of progression meaning earlier onset is correlated with more drastic progression. There is often a
significant asymptomatic period and the initial symptoms which often appear between ages 10 to 15 may include unsteady posture, frequent falling and progressive difficulty in walking due to impaired ability to coordinate voluntary movements
called ataxia.
By the time symptoms occur, heart damage has already normally occurred. As the disease progresses, symptoms worsen and may include the
development of advanced lemotaxia often requiring patient confinement to a wheelchair, hypotrophic cardiomyopathy, scoliosis, fatigue, diabetes and hearing loss.
Life expectancy is around 30 to 50 years with early death usually caused by heart disease due to advanced cardiomyopathy. Unfortunately, there is no approved
therapy for F.A. and treatment is limited to symptom management. Now turning to our lead candidate. CTI-1601 targeting Freidreichs Ataxia. Slide 9 shows the structure of the fusion protein and the
process by which CTI-1601 is understood to deliver for tests into mitochondria.
Initially, a cell penetrating
peptide allows CTI-1601 to cross the cell membrane and then cross the mitochondrial membrane. There, mitochondrial processing peptidase [leads] CTI-1601 at the site
shown in the diagram. Then, the mitochondrial targeting sequence and the cell penetrating peptide leads the (inaudible) and the cell. This leads mature, active Frataxin in the cells mitochondria.