- MS is a chronic inflammatory, degenerative, demyelinating disorder of the central nervous system affecting 900,000 people in 7 major markets1. Current medications are most effective only during the inflammatory phase, and are less potent as the disease transitions to a neurodegenerative process. There are no effective disease-modifying drugs for progressive forms. No therapies appear to re-myelinate damaged neurons. (1Data per National Multiple Sclerosis Society and Global Data)
- Initiated GLP toxicology studies and manufacturing for Phase 1 human study, which is planned to start in Australia in H2 2018. Successful completion of Phase 1 is expected to support Phase 2 studies.
- SSc is a rare chronic systemic autoimmune disease, causing fibrosis of skin and internal organs. It is estimated that in the US it affects ~180,000 people2. Current therapies are not effective and have significant toxicities. There are no specific approved drugs for SSc. (2Data per Coté Orphan (now called IQVIA) prevalence research analysis)
- Successful completion of Phase 1 study (planned for Australia starting in H2 2018) is expected to support Phase 2 studies.
- Granted US FDA and EU EMA Orphan Drug Designation
- PD is a chronic, progressive neurodegenerative disorder affecting over 10 million sufferers worldwide3. This disease is characterized by damaged neurons not producing sufficient dopamine, a mechanism that is critical when transmitting impulses from the brain to the muscles. There is no current cure for PD. Initial treatments become less effective as the disease progresses. (3Parkinson’s Foundation. Statistics http://parkinson.org/Understanding-Parkinsons/Causes-and-Statistics/Statistics. Accessed May 14, 2018.)
- Preclinical proof-of-concept has been established for PD. Chemistry, formulation & manufacturing development is in progress. Clinical-enabling studies expected to start once manufacturing and formulation development completed (expected in 2019).
- HD is an inherited disease that causes progressive breakdown of nerve cells. It deteriorates patients’ physical and mental abilities to the extent that they are unable to care for themselves. It has no cure. In the U.S., approximately 30,000 people are symptomatic and more than 200,000 are at risk of inheriting the disease.4 There are no specific disease-modifying therapies for HD. (4Data per Huntington’’s Disease Society of America )
- Preclinical proof-of-concept has been established for HD. Chemistry, formulation & manufacturing development is in progress. Clinical-enabling studies expected to start once manufacturing and formulation development completed (expected in 2019).
- Granted US Orphan Drug Designation.