Achieving Our Vision Through Life-Changing Therapeutics
Product Portfolio
Because of their unique properties and proven clinical efficacy, aptamers hold extraordinary potential in the treatment of both acute and chronic diseases.
As a component of our business strategy, we have elected to focus our proprietary product development primarily on rare hematological diseases. We aim to develop products for chronic indications as part of our partnerships & alliances with third parties.
The portfolio of programs listed below include aptamer therapeutics currently in research and development resulting from Archemix's internal efforts, its collaborations with partners, and licensees of its technology. Click on a program name below for more information about the program.
| Orphan Hematology / Cardiovascular |
ARC1779 (TTP). Our lead aptamer product candidate, ARC1779, is a PEGylated aptamer consisting of 40 nucleotides that is administered intravenously. ARC1779 is designed to inhibit the function of a protein called von Willebrand Factor, or vWF, which is, when activated, responsible for the adhesion, activation and aggregation of platelets.
In our Phase 1 clinical trial of ARC1779 in healthy volunteers, we observed that vWF activity and platelet function were maximally inhibited to the limits of assay detection in a manner that correlated to the dose and concentration of ARC1779.
We plan to evaluate ARC1779 in patients suffering from a rare blood disorder known as thrombotic thrombocytopenic purpura, or TTP. In TTP, excessive levels of activated vWF cause platelet aggregation and widespread blood clotting, which can lead to life-threatening events such as heart attack and stroke. Because ARC1779 targets activated vWF, ARC1779 can reduce or eliminate the formation of blood clots that cause the morbidity and mortality associated with TTP. There is currently no approved drug therapy for TTP.
We believe that ARC1779 for treatment of TTP meets the criteria for orphan drug designation in the United States and the European Union. Orphan drug designation is a regulatory status granted by the United States Food and Drug Administration, or FDA, and some foreign regulatory authorities, to drugs for the treatment of rare diseases or conditions. This designation typically includes a period of marketing exclusivity and other economic benefits.
We commenced a Phase 2 clinical trial of ARC1779 in March 2008 in patients suffering from TTP.
REG1 (PCI). Archemix granted a license to Regado Biosciences for the discovery and development of antidote-controlled aptamers for controlling fibrin. The REG1 anticoagulation system consists of an aptamer to Factor IX and a complementary oligonucleotide antidote.
Regado completed a Phase 1 clinical trial of the REG1 system and presented study data at the American Heart Association meeting in November 2006. In total, the trial enrolled 85 healthy volunteers. The data presented by Regado established a close correlation among aptamer dose, aptamer plasma concentration, factor IX activity and measures of anticoagulation. The antidote was also shown to reverse the pharmacologic effects of the aptamer. Regado has also completed two additional Phase 1 clinical trials involving 88 additional subjects.
Regado commenced a Phase 2 clinical trial of REG1 in the fourth quarter of 2007. The study will enroll 26 patients undergoing elective Percutaneous Coronary Intervention (PCI) to assess whether REG1 can replace standard heparin therapy during the performance of coronary balloon angioplasty dilatation and stenting on patients at low-risk for complications associated with therapy-related bleeding or heart attack.
REG1 (CABG). Archemix granted a license to Regado Biosciences for the discovery and development of antidote-controlled aptamers for controlling fibrin. The REG1 anticoagulation system consists of an aptamer to Factor IX and a complementary oligonucleotide antidote.
Regado completed a Phase 1 clinical trial of the REG1 system and presented study data at the American Heart Association meeting in November 2006. In total, the trial enrolled 85 healthy volunteers. The data presented by Regado established a close correlation among aptamer dose, aptamer plasma concentration, factor IX activity and measures of anticoagulation. The antidote was also shown to reverse the pharmacologic effects of the aptamer. Regado has also completed two additional Phase 1 clinical trials involving 88 additional subjects.
Regado commenced a Phase 2 clinical trial of REG1 in the fourth quarter of 2007 and has advised us that it expects to commence an additional Phase 2 clinical trial of REG1 in the first quarter of 2008.
NU172 (CABG/PCI). Nuvelo, Inc. is developing NU172 as an anticoagulant for use in acute cardiovascular surgeries. NU172 is being tested as a fast-acting, short half-life anti-coagulant. NU172 targets thrombin, which is a protein required for blood clotting. NU172 is designed to be administered by intravenous infusion during an acute cardiovascular surgical procedure to prevent the formation of harmful blood clots. The resulting period of anticoagulation is designed to be maintained until the infusion is stopped. With its rapid offset of action, NU172 is designed to return the body to its normal state of hemostasis shortly after the cessation of the infusion.
Nuvelo has announced that it is evaluating NU172 in IND-enabling studies and expects to initiate a Phase 1 trial in the first quarter of 2008.

Hemophilia.
Hemophilia is a family of rare bleeding disorders that impairs the body’s ability to control the normal
blood clotting process. Persons with hemophilia have low levels, or a complete absence, of specific proteins,
called factors, in their blood that work with platelets to help the blood clot. Proper clotting of blood allows a blood vessel to heal after an injury and prevents excessive bleeding. In hemophiliacs, deficiency or absence of
normal clotting factors can cause abnormally long bleeding after an injury or surgery or spontaneous bleeding
into the joints and muscles. According to the Centers for Disease Control and Prevention, hemophilia afflicts
approximately 18,000 people in the United States. According to EvaluatePharma, drugs for the treatment of
hemophilia in major pharmaceutical markets generated approximately $4.5 billion in sales in 2006. There is
currently no cure for hemophilia. Currently approved treatments for hemophilia address the symptoms of
hemophilia rather than its cause and rely largely on drugs that replace the function of missing clotting factors.
We believe that an aptamer for treatment of hemophilia may meet the criteria for orphan drug designation in
the United States and the European Union.
We believe that there is an unmet medical need in hemophilia, which represents a significant opportunity for drug discovery. We are researching aptamers that may bind to and inhibit the function of certain proteins that we believe may play a role in hemophilia.

Sickle Cell Disease.
Sickle cell disease is a chronic and debilitating genetic blood disorder, primarily affecting individuals of
African descent, resulting in a variety of disease complications and a significantly shortened lifespan in the
majority of patients. Sickle cell disease is caused by the presence of an abnormal form of hemoglobin, the
protein inside red blood cells that is responsible for carrying oxygen, which leads to abnormally shaped red
blood cells. According to the National Heart Lung and Blood Institute, sickle cell disease affects about
70,000 people in the United States. According to the Agency for Healthcare Research and Quality’s Healthcare
Cost and Utilization Project, sickle cell disease accounted for approximately 85,000 hospitalizations in the
United States in 2005. The only approved drug for the treatment of sickle cell disease is hydroxyurea, a drug
primarily used as a cancer chemotherapeutic agent that is prescribed to treat the symptoms of only the most
severely ill patients due to its potentially serious side effects. We believe that an aptamer for treatment of
sickle cell disease may meet the criteria for orphan drug designation in the United States and the European
Union.
We believe that there is an unmet medical need in sickle cell disease, which represents a significant opportunity for drug discovery. We are researching aptamers that may bind to and inhibit the function of certain proteins that we believe may play a role in sickle cell disease.
AS1411 (AML). Antisoma plc is developing an aptamer it calls AS1411. AS1411 binds to a protein called nucleolin, which is found on the surface of cancer cells. When AS1411 binds to nucleolin, it is internalized and has been shown to kill cancer cells in a range of animal models.
Antisoma studied AS1411 in a Phase 1 clinical trial that concluded in October 2006. In total, the study enrolled 30 patients. The data presented by Antisoma at the European Society of Medical Oncology meeting in October 2006 showed that signs of anti-cancer activity were observed in patients with renal cell carcinoma. Twelve of the patients in the Phase 1 trial had this type of cancer. Of these patients, two showed a complete or partial response, both with tumor shrinkage, and seven additional patients experienced disease stabilization for two months or longer. Furthermore, no serious adverse events related to drug administration were reported at any dose level.
Antisoma has also tested AS1411 in cancerous cells extracted from patients with acute myeloid leukemia. In these studies, AS1411 killed these cancer cells, suggesting that the aptamer product candidate may have utility in treating acute myeloid leukemia. In the third quarter of 2007, Antisoma announced that it commenced a Phase 2 clinical trial of AS1411 in patients with acute myeloid leukemia.
AS1411 (Renal Cell). Antisoma plc is developing an aptamer it calls AS1411. AS1411 binds to a protein called nucleolin, which is found on the surface of cancer cells. When AS1411 binds to nucleolin, it is internalized and has been shown to kill cancer cells in a range of animal models.
Antisoma studied AS1411 in a Phase 1 clinical trial that concluded in October 2006. In total, the study enrolled 30 patients. The data presented by Antisoma at the European Society of Medical Oncology meeting in October 2006 showed that signs of anti-cancer activity were observed in patients with renal cell carcinoma. Twelve of the patients in the Phase 1 trial had this type of cancer. Of these patients, two showed a complete or partial response, both with tumor shrinkage, and seven additional patients experienced disease stabilization for two months or longer. Furthermore, no serious adverse events related to drug administration were reported at any dose level.
Antisoma has advised us that it plans to commence a separate Phase 2 clinical trial of AS1411 in patients with renal cell carcinoma in 2009.
E10030 (AMD/DR). Ophthotech is developing E-10030, an aptamer directed against platelet-derived growth factor-B (PDGF-B) for the potential treatment of AMD.
ARC1905 (AMD). Ophthotech is developing ARC1905 for use in the prevention and treatment of eye diseases. ARC1905 inhibits C5, a central component of the complement cascade, which plays multiple roles in innate immunity and inflammatory diseases. Mounting evidence suggests an important link between complement and the development of age-related macular degeneration (AMD).
|