PALO ALTO, Calif.--(BUSINESS WIRE)--Bayhill Therapeutics Inc, a clinical-stage biopharmaceutical company utilizing its proprietary BHT-DNA™ platform to develop novel and targeted autoimmune and immune-mediated disease treatments, today announced the successful clearance of an IND with the US FDA for BHT-3034, a disease-modifying DNA plasmid vaccine immunotherapy for patients with the autoimmune neuromuscular junction disease myasthenia gravis. The IND was filed based on promising preclinical efficacy results showing significant disease severity attenuation and anti-acetylcholine receptor autoantibody reduction, as well as GLP toxicology studies showing no evidence of toxicity or immunogenicity. The IND also included a phase I/II dose escalation clinical study to treat up to 40 subjects with myasthenia gravis with 12 weekly doses of BHT-3034 intramuscularly.
Company founder and director Lawrence Steinman, MD, Chairman of the Interdepartmental Program in Immunology at Stanford University, stated, “We are excited by this opportunity to bring forward a novel disease modifying therapy to patients with myasthenia gravis. BHT-3034 has the potential to significantly reduce the autoimmune response in a highly specific manner and thereby demonstrate a clinically meaningful benefit.”
This represents the third compound to enter clinical testing from Bayhill Therapeutics’ proprietary BHT-DNA™ platform. The first compound, BHT-3009 for multiple sclerosis, has achieved success in a phase I/II trial where safety and antigen-specific immune tolerance was demonstrated, and in a phase II trial where a responder population of high serum anti-MBP (myelin basic protein) subjects demonstrated a > 70% reduction in brain lesions (p=0.02) and corresponding reductions in annualized clinical relapse rates compared to placebo. Over 200 patients with multiple sclerosis have been treated for periods of up to one year. The second compound, BHT-3021 for type 1 diabetes, is currently being tested in a phase I/II trial where preliminary data show excellent safety and tolerability as well as evidence of preservation of pancreatic beta cell function during dosing, as measured by serum C-peptide levels. Interim data from the study indicate that BHT-3021 may preserve beta cell function and thus attenuate disease progression, making it an exciting advance for the treatment of type 1 diabetes. Furthermore, Bayhill Therapeutics has generated a pipeline of compounds for several other autoimmune and immune-mediated diseases including Lupus and anti-Factor VIII antibody diseases.
To move its programs forward in the most efficient manner, Bayhill Therapeutics also announced the retention of P2 Partners, LLC to assist the Company in the pursuit of a strategic transaction.
About Bayhill Therapeutics
Bayhill Thereapeutics is a clinical-stage biopharmaceutical company at the forefront of developing novel and targeted treatment candidates for autoimmune diseases. Leveraging its proprietary therapeutic BHT-DNA™ platform, the Company’s product candidates have been designed to restore a patient’s immune system “tolerance” to self antigens to its normal state by selectively eliminating specific, harmful immune responses while leaving the rest of the immune system intact. Through a targeted and selective approach, Bayhill’s product candidates have the potential to deliver superior efficacy, safety and tolerability relative to current therapies. For more information please visit www.bayhilltx.com