Our Science
MYT-109 INCREASES MITOGENE EXPRESSION & FUNCTION
MYT-109
Our MYT-109 improves mitochondrial functions and rescues lethality in animal models of Leigh’s Syndrome. It’s a prodrug, using a proprietary technology, with improved PK compared to other fumarate based drugs. Our program has the potential for an accelerated regulatory path. Its unique mechanism of action could ameliorate ~65,000 mitochondrial patients in USA.
Defects in hundreds of Mitochondrial genes cause Orphan mitochondrial diseases
Reduced Mitochondrial Function
Mitochondrial gene defects causes reduced function resulting in diseases such as Leigh’s syndrome, LHONs & FA.
Increased Mitochondrial Function
THERAPEUTIC PIPELINE
MYT-109 TARGETS MULTIPLE MITOCHONDRIAL DISEASES
- Isolation & Synthesis
- Benefits in Animal Models
- Benefits in Patient Cells
- IND-enabling studies
- Phase 1
- Phase 2
MYT-109
MYT-109 is the first small molecule to benefit animals with different mitochondrial genetic defects, to target multiple human mitochondrial orphan indications.
INDICATIONLEIGH’S SYNDROME
Leigh’s Syndrome is a progressive neurometabolic disorder that can cause deterioration of the central nervous system, including the brain, spinal cord, and optic nerve. Leigh’s Syndrome can be caused by mutations in any of more than 75 different genes.
The prognosis for Leigh’s Syndrome is poor. Depending on the defect, individuals typically live anywhere from a few years to the mid-teens.
Leigh’s Syndrome in general is rare and is estimated to affect about 1 in 30,000 to 1 in 40,000 people at birth. Mitochondrial DNA-associated Leigh’s Syndrome, which is more rare than nuclear gene-encoded Leigh’s Syndrome, is likely to occur in about 1 in 100,000 to 1 in 140,000 births.
There is no cure for Leigh’s Syndrome. Treatments generally involve variations of vitamin and supplement therapies, often in a “cocktail” combination, and are only partially effective.
INDICATIONLHON
Leber’s Hereditary Optic Neuropathy (LHON) is an inherited mitochondrial disease, caused by a genetic mutation (variance) in the mitochondria.
The most common LHON mutation is the 11778 mutation, accounting for over 65% of all LHON cases.
About 100 people in the United States lose central vision due to LHON each year, joining the 4,000 or so Americans who are already legally blind due to LHON.
It’s estimated that about 35,000 people worldwide have LHON vision. About 25% of those affected are female; 75% male.
Thousand carry a LHON genetic mutation, and each could suddenly lose their central vision at any time as onset of vision loss can happen at any age.
In the U.S. and most other countries, there is no approved treatment for LHON.
INDICATIONFRIEDREICH’S ATAXIA
Friedreich’s Ataxia (also called FA) is a rare inherited disease caused by a defect (mutation) in a gene labeled FXN, which carries the genetic code for a protein called frataxin, which leads to progressive nervous system damage and movement problems.
It usually begins in childhood and leads to impaired muscle coordination (ataxia) that worsens over time. The damage results in awkward, unsteady movements and impaired sensory functions.
Generally, within 10 to 20 years after the appearance of the first symptoms the person is confined to a wheelchair. Friedreich’s Ataxia can shorten life expectancy, and heart disease is the most common cause of death.
About one in 50,000 people in the United States has Friedreich’s ataxia. It is estimated there are 4,000 individuals affected with FA in the U.S., and 15,000 affected individuals worldwide.
As with many degenerative diseases of the nervous system, there is currently no cure or effective treatment for Friedreich’s Ataxia.