BHT-3009

• Parent company: Bayhill Therapeutics


• This is a DNA vaccine to myelin basic protein (MBP); it contains the gene for MBP and is administered by intramuscular injection. This therapy is designed to cause immune tolerance, by reprogramming the immune system to modulate the response of the antigen-specific immune cells involved with MS, and reducing the attack against the MBP in the myelin sheath.


• A Phase I/II trial of immunotherapy with BHT-3009 demonstrated that the vaccine was safe and well tolerated, and that it produced an antigen-specific immune tolerance. It also provided favorable trends on brain MRI. This study was conducted with 30 RRMS or SPMS patients, and injections were given at weeks 1, 3, 5, and 9 using three dose levels.


• A Phase II study in 267 RRMS patients compared two doses versus placebo. In the lower-dose group, a significant decrease in enhancing lesions was found as measured by MRI in patients with high levels of immune activity at time of enrollment. Injections were given at weeks 0, 2, 4, and every four weeks afterward. The higher dose was found to be ineffective.


• A Phase IIb trial for RRMS patients is planned for late 2008.