We are a clinical stage biopharmaceutical company focused on the discovery and development of innovative, disease-modifying therapies for neurodegenerative diseases. Neurodegenerative diseases cause a progressive loss of structure and function in the brain, leaving patients with devastating damage to their nervous system and widespread functional impairment. Although treatments may help relieve some of the physical or mental symptoms associated with neurodegenerative diseases, few of the currently available therapies slow or stop the continued loss of neurons, resulting in a critical unmet need. We are specifically focused on developing novel disease-modifying therapies to treat devastating conditions, either with large or orphan disease markets, including Parkinson’s disease, dementia with Lewy bodies, multiple system atrophy (“MSA”), amyotrophic lateral sclerosis (“ALS”, also known as Lou Gehrig’s disease), frontotemporal lobar degeneration (“FTLD”), and Alzheimer’s disease.
Our goal is to advance one new program into the clinic every year. Our lead program,
YTX-7739,
is now in Phase 1 clinical trials for the potential treatment and disease modification of Parkinson’s disease.
YTX-7739
targets an enzyme known as
stearoyl-CoA
desaturase (“SCD”). Inhibition of SCD in multiple cellular systems, including patient-derived neurons, as well as in a novel mouse model of Parkinson’s disease, has been demonstrated to overcome the toxicity of misfolded alpha-synuclein or α-synuclein, a protein strongly associated with Parkinson’s disease. In February 2021, we announced the results of a Phase 1 single ascending dose (“SAD”) study of
YTX-7739
in healthy volunteers, which evaluated a broad range of doses of
YTX-7739.
We also completed a Phase 1a multiple ascending dose (“MAD”) study in healthy volunteers, and announced results in April 2021. A Phase 1b clinical study of
YTX-7739
in patients with Parkinson’s disease has commenced as a continuation of the MAD study. The Phase 1b part of the study will assess safety, tolerability and pharmacokinetics of
YTX-7739
as well as proof of biology in patients with Parkinson’s disease by exploring biomarkers of target engagement. The study will also explore potential correlative clinical parameters such as electroencephalography and neuroimaging measurements to monitor for early effects of
YTX-7739,
noting, however, that such effects may be difficult to see given the relatively short duration of the trial (28 days) compared to the length of Parkinson’s disease progression. Early results from the Phase 1b part are anticipated in the fall of 2021. Our second program,
YTX-9184,
also inhibits SCD but is chemically distinct from
YTX-7739.
Good Laboratory Practice (“GLP”) safety pharmacology and toxicological studies for
YTX-9184
were initiated in the second quarter of 2020. We anticipate commencing the
studies of
YTX-9184
in 2021 and intend to develop
YTX-9184
for the potential treatment of dementia with Lewy bodies, a devastating neurodegenerative disease which is also characterized by the abnormal accumulation of aggregates of α-synuclein. Pending the results of the Phase 1b study in
YTX-7739
and additional preclinical data, we may choose to study
YTX-7739
for the potential treatment of dementia with Lewy bodies (or other disorders of α-synuclein) instead of
YTX-9184,
as this may provide an opportunity to see results in patients sooner, given the studies done to date of
YTX-7739.
Additionally, based on promising preclinical data in one animal model, we plan to initiate a window-of-opportunity study of an SCD inhibitor in glioblastoma multiforme patients in 2022 once further validation of the preclinical data has been achieved in a second animal model. At the center of our scientific foundation is our drug discovery engine, which is based on technology licensed from the Whitehead Institute, an affiliate of the Massachusetts Institute of Technology. This core technology, combined with investments and advancements by us, is designed to enable rapid screening to identify drugs with the potential to modify disease by overcoming toxicity in disease-causing gene networks. Toxicity in many neurodegenerative diseases results from an aberrant accumulation of misfolded proteins in the brain. We leverage our proprietary discovery engine to identify and screen novel drug targets and drug molecules for their ability to protect nerve cells from toxicity arising from misfolded proteins. To date, we have identified over 20 targets, most of which have not previously been linked to neurodegenerative diseases. We believe this discovery platform will allow us to replenish our pipeline as programs advance into clinical development.