Clinical Research

RFS Pharma, LLC, is developing important new therapies to treat human diseases.  With expertise and initial focus on HIV and hepatitis virus infections, RFS Pharma has development programs in progress for its lead compounds. 

• Amdoxovir^® (AMDX, DAPD) - DAPD is a nucleoside analog prodrug that is deaminated by adenosine deaminase to its 2'-deoxyguanosine analog, DXG. The triphosphate form of DXG is a potent and selective inhibitor of HIV-1, including drug resistant viruses harboring clinically relevant mutations. It is also active against HIV-2 and hepatitis B virus. To date, 188 subjects have safely received DAPD in six Phase 1 and 2 studies. DAPD has an excellent toxicity profile in subjects receiving treatment for up to 96 weeks and is very effective at decreasing viral load in HIV infected individuals, including those with extensive nucleoside reverse transcriptase inhibitor (NRTI) mutations. A Phase 2 pharmacokinetic/pharmacodynamic study in HIV-infected individuals to identify a potential "nucleoside combination product" is enrolling in 4Q2006. A Phase 2b multi-international site study is planned for 1Q2007.

• APD is a nucleoside derivative being developed as potential prodrug for DXG, whose anti-HIV-1 activity was described above. APD is converted to DXG by xanthine oxidase and is undergoing late stage preclinical studies for the treatment of infections caused by HIV-1 and HBV. APD may offer increased bioavailability and aqueous solubility.

• DOT - Despite the availability of eight nucleoside analogs for the treatment of HIV infections, toxicity and virus breakthrough remain a concern. There is a need for safer, orally bioavailable once a day drugs that are thymidine kinase (TK)-dependent that are effective against thymidine analog mutations (TAMs) and multidrug resistant (MDR) HIV.  DOT was found to have unique and surprising virologic and pharmacologic properties for a thymidine analog.  DOT was markedly effective against numerous clinically relevant drug resistant mutants including HIV containing MDR and T69S insert mutations.  Its pharmacological properties are unique for a nucleoside analog. DOT has been out-licensed for further development.