Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and The Alpha-1 Project (TAP), the venture philanthropy subsidiary of the Alpha-1 Foundation, announced today that they have entered into a collaboration agreement for the continued advancement of ALN-AAT, a subcutaneously administrated RNAi therapeutic in development for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease. TAP’s mission is to work with patients, academia, pharmaceutical and biotech companies, and public health organizations in the pursuit of cures and therapies for chronic obstructive pulmonary disease (COPD) and liver disease caused by AAT deficiency. TAP is partially funding research activities for ALN-AAT. Detailed financial terms of the research agreement were not disclosed. Alnylam remains on track to file an investigational new drug (IND) application for this program in mid-2015.
“We have assembled what we believe to be the industry’s most robust pre-clinical data package supporting advancement of ALN-AAT – an ‘Enhanced Stabilization Chemistry’ or ‘ESC’-GalNAc-siRNA conjugate targeting AAT – including results in rodent models that demonstrate knockdown of the disease-causing Z-allele and significant lowering of the mutant protein (Z-AAT) burden in the liver, with associated improvements in liver function, as seen by normalization of the proliferative index, improved liver pathology, attenuated tissue fibrosis, and decreased incidence of liver tumor formation. In addition, we believe we have confirmed the activity of ALN-AAT in non-human primate models and expect our Development Candidate to achieve human target gene knockdown at doses less than 1 mg/kg with a once-monthly dose regimen or better. Since our GalNAc-siRNA platform is clinically validated based on results from other Alnylam RNAi therapeutic programs, we have a high level of confidence that ALN-AAT can achieve potent knockdown of the disease-causing Z-allele protein with subcutaneous dose administration and a favorable safety profile,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “Accordingly, we are fully committed to the continued advancement of our RNAi therapeutic to patients with AAT deficiency with associated liver disease. This new recognition from TAP, part of the leading patient advocacy group for people afflicted with AAT deficiency, brings our efforts closer to patients in need and to their caregivers. We continue to expect that we will file our IND or IND equivalent for ALN-AAT in mid-2015.”