DEERFIELD, Ill.—Baxter International Inc. announced in late November that it is in the process of buying all of the hemophilia-related assets of Archemix, a privately held biopharma firm, and has also entered into an exclusive license agreement for related intellectual property assets.
The lead product associated with the arrangement is ARC19499, a synthetic hemophilia therapy that is injected under the skin and is currently in a Phase I clinical trial in the United Kingdom.
The therapy blocks Tissue Factor Pathway Inhibitor (TFPI) activity, thereby augmenting and improving blood clotting, potentially reducing replacement factor therapy for patients with hemophilia A and B.
The move is a strong indication of Baxter's interest in bolstering its presence in the fight against hemophilia, a rare genetic blood clotting disorder that primarily affects males. Current estimates are that more than 400,000 people in the world have hemophilia. All races and economic groups are affected equally by the disorder.
“Baxter is committed to optimizing hemophilia care and improving the lives of people living with hemophilia around the world,” says Dr. Hartmut Ehrlich, vice president of global research and development and medical affairs for Baxter’s BioScience business, in a prepared statement. “This anti-TFPI program is an important addition to other Baxter hemophilia development programs focusing on longer-acting rFVIII and rFIX and non-intravenous therapies.”
ARC19499 is part of a new therapeutic class referred to as “aptamers.” Aptamers are smaller than a protein or biologic, so the theory is that these molecules have the potential to be further developed for subcutaneous administration. They are short, synthetically derived oligonucleotides that form three-dimensional structures that bind with high specificity and affinity to protein and non-protein targets.
Currently there is one aptamer approved by the U.S. Food and Drug Administration and available to patients today: Macugen, for the treatment of age-related macular degeneration.
The Phase I clinical trial for ARC19499 was initiated by Archemix in the United Kingdom in August 2010 and continues to enroll patients.
People living with hemophilia do not have enough of, or are missing, one of the blood clotting proteins naturally found in blood.
Two of the most common forms of hemophilia are known as type A and type B. In about 30 percent of cases, there is no family history of hemophilia, and the condition is the result of a spontaneous gene mutation.
In people with hemophilia A, clotting factor VIII is not present in sufficient amounts or is absent. Without enough FVIII, people with hemophilia can experience spontaneous, uncontrolled internal bleeding that is painful, debilitating, damaging to joints and potentially fatal.
People with hemophilia B (which is sometimes also referred to as “Christmas disease”) do not have sufficient amounts of clotting factor IX.
Baxter expects to record a special pre-tax, in-process research and development charge of approximately $30 million in the fourth quarter of 2010 relating to an upfront payment associated with the transaction. In the future, Baxter may also make milestone-related payments to Archemix of up to $285 million.
Subject to regulatory approvals and other conditions, the companies expected to complete the transaction by the end of 2010.
In late October, Hikma Pharmaceuticals, a multinational pharmaceutical group based in London, purchased Baxter’s U.S. generic injectables business for $112 million in cash.
The deal involved products currently on the market and in the pipeline, as well as employees and real estate in New Jersey and Tennessee.
“Baxter’s growth strategy is to focus more on differentiated offerings derived from the company’s expertise in enhanced packaging and formulation technologies, resulting in higher-margin products,” explained company officials at the time.
Baxter officials refused to comment for this story.
Archemix representatives did not respond to requests for comment.
Baxter develops, manufactures and markets products that treat hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions.
Archemix is a privately held biopharmaceutical company based in Cambridge, Mass. The company is leading the development of aptamers as a new class of directed therapeutics for the prevention and treatment of chronic and acute diseases. With their unique properties, aptamers offer an alternative to biologics and small molecules in numerous applications and hold extraordinary potential to address unmet medical needs. The company’s website states that Archemix’s business strategy is to build and advance a pipeline of proprietary aptamer products in the core areas of cardiovascular disease, hematology and oncology.
Baxter licenses autoimmune disease therapy to Accentia
TAMPA, Fla.—Baxter also recently signed an agreement with Accentia Biopharmaceuticals Inc. to provide Accentia with the exclusive, worldwide right to purchase Baxter’s cyclophosphamide, which is marketed under the brand name Cytoxan, for the treatment of designated autoimmune diseases including multiple sclerosis.
Cyclophosphamide is the active drug used in Revimmune therapy, Accentia’s proprietary system-of-care being developed for the treatment of a broad range of autoimmune diseases.
According to the companies, further investigation of cyclophosphamide may identify a potentially valuable treatment option for patients with devastating autoimmune diseases, including multiple sclerosis, with high unmet medical need.
The agreement also grants Accentia with the exclusive right for designated indications to reference Baxter’s proprietary, historical data related to cyclophosphamide as part of Accentia’s planned clinical and regulatory development of Revimmune.
The agreement designates Baxter as Accentia’s sole source of cyclophosphamide for Revimmune.
“We are pleased that, through this agreement, we will be able to support Accentia Biopharmaceuticals in the ongoing development of Revimmune,” says Dr. Debasis Chakrabarti, Baxter’s therapeutic area leader for oncology. “Further investigation of cyclophosphamide may identify a potentially valuable treatment option for patients with devastating autoimmune diseases, including multiple sclerosis, with high unmet medical need.”
“With this agreement in place, we are planning a robust clinical and regulatory development strategy to advance our mission to establish Revimmune as a new standard-of-care treatment for patients suffering from autoimmune diseases, including orphan indications with potential accelerated regulatory pathways, as well as major indications such as multiple sclerosis,” says Accentia President Samuel S. Duffey. Financial terms of the deal were not released.