After dropping generic injectables, company adds hemophilia-related assets of Archemix to product stable
DEERFIELD, Ill.—Baxter International Inc. announced in lateNovember that it is in the process of buying all of the hemophilia-relatedassets of Archemix, a privately held biopharma firm, and has also entered intoan exclusive license agreement for related intellectual property assets.
The lead product associated with the arrangement isARC19499, a synthetic hemophilia therapy that is injected under the skin and iscurrently 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 reducingreplacement factor therapy for patients with hemophilia A and B.
The move is a strong indication of Baxter's interest inbolstering its presence in the fight against hemophilia, a rare genetic bloodclotting disorder that primarily affects males. Current estimates are that morethan 400,000 people in the world have hemophilia. All races and economic groupsare affected equally by the disorder.
"Baxter is committed to optimizing hemophilia care andimproving the lives of people living with hemophilia around the world," saysDr. Hartmut Ehrlich, vice president of global research and development andmedical affairs for Baxter's BioScience business, in a prepared statement. "Thisanti-TFPI program is an important addition to other Baxter hemophiliadevelopment programs focusing on longer-acting rFVIII and rFIX andnon-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 isthat these molecules have the potential to be further developed forsubcutaneous administration. They are short, synthetically derivedoligonucleotides that form three-dimensional structures that bind with highspecificity and affinity to protein and non-protein targets.
Currently there isone aptamer approved by the U.S. Food and Drug Administration and available topatients today: Macugen, for the treatment of age-related macular degeneration.
The Phase I clinical trial for ARC19499 was initiated byArchemix in the United Kingdom in August 2010 and continues to enrollpatients.
People living with hemophilia do not have enough of, or aremissing, one of the blood clotting proteins naturally found in blood.
Two ofthe most common forms of hemophilia are known as type A and type B. In about 30percent of cases, there is no family history of hemophilia, and the conditionis the result of a spontaneous gene mutation.
In people with hemophilia A, clotting factor VIII is notpresent in sufficient amounts or is absent. Without enough FVIII, people withhemophilia can experience spontaneous, uncontrolled internal bleeding that ispainful, debilitating, damaging to joints and potentially fatal.
People withhemophilia B (which is sometimes also referred to as "Christmas disease") do not have sufficient amounts ofclotting factor IX.
Baxter expects to record a special pre-tax, in-processresearch and development charge of approximately $30 million in the fourthquarter of 2010 relating to an upfront payment associated with the transaction.In the future, Baxter may also make milestone-related payments to Archemix ofup to $285 million.
Subject to regulatory approvals and other conditions, thecompanies expected to complete the transaction by the end of 2010.
In late October, Hikma Pharmaceuticals, a multinationalpharmaceutical group based in London, purchased Baxter's U.S. genericinjectables business for $112 million in cash.
The deal involved productscurrently on the market and in the pipeline, as well as employees and realestate in New Jersey and Tennessee.
"Baxter's growth strategy is to focus more on differentiatedofferings derived from the company's expertise in enhanced packaging andformulation technologies, resulting in higher-margin products," explainedcompany officials at the time.
Baxter officials refused to comment for this story.
Archemixrepresentatives did not respond to requests for comment.
Baxter develops, manufactures and markets products thattreat hemophilia, immune disorders, cancer, infectious diseases, kidneydisease, trauma and other chronic and acute medical conditions.
Archemix is a privately held biopharmaceutical company basedin Cambridge, Mass. The company is leading the development of aptamers as a newclass of directed therapeutics for the prevention and treatment of chronic andacute diseases. With their unique properties, aptamers offer an alternative tobiologics and small molecules in numerous applications and hold extraordinarypotential to address unmet medical needs. The company's website states thatArchemix's business strategy is to build and advance a pipeline of proprietaryaptamer products in the core areas of cardiovascular disease, hematology andoncology.
Baxter licenses autoimmune disease therapy to Accentia
TAMPA, Fla.—Baxter also recently signed an agreement withAccentia Biopharmaceuticals Inc. to provide Accentia with the exclusive,worldwide right to purchase Baxter's cyclophosphamide, which is marketed underthe brand name Cytoxan, for the treatment of designated autoimmune diseasesincluding multiple sclerosis.
Cyclophosphamide is the active drug used in Revimmunetherapy, Accentia's proprietary system-of-care being developed for thetreatment of a broad range of autoimmune diseases.
According to the companies, further investigation ofcyclophosphamide may identify a potentially valuable treatment option forpatients with devastating autoimmune diseases, including multiple sclerosis,with high unmet medical need.
The agreement also grants Accentia with the exclusive rightfor designated indications to reference Baxter's proprietary, historical datarelated to cyclophosphamide as part of Accentia's planned clinical andregulatory development of Revimmune.
The agreement designates Baxter asAccentia's sole source of cyclophosphamide for Revimmune.
"We are pleased that, through this agreement, we will beable 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 valuabletreatment option for patients with devastating autoimmune diseases, includingmultiple sclerosis, with high unmet medical need."
"With this agreement in place, we are planning a robustclinical and regulatory development strategy to advance our mission toestablish Revimmune as a new standard-of-care treatment for patients sufferingfrom autoimmune diseases, including orphan indications with potentialaccelerated regulatory pathways, as well as major indications such as multiplesclerosis," says Accentia President Samuel S. Duffey. Financial terms of the deal were not released.