Pharma

Big Pharmas take $17M interest in “molecular Trojan horse” to cross the blood-brain barrier

The closely packed cells that form blood vessels to the brain create a physical barrier that has been one of the pharmaceutical industry’s toughest hurdles in developing drugs for neurological diseases. The blood-brain barrier blocks potentially damaging toxins from the brain, but it also keeps out small- and large-molecule drugs. An approach to getting drugs […]

The closely packed cells that form blood vessels to the brain create a physical barrier that has been one of the pharmaceutical industry’s toughest hurdles in developing drugs for neurological diseases. The blood-brain barrier blocks potentially damaging toxins from the brain, but it also keeps out small- and large-molecule drugs.

An approach to getting drugs across that barrier that was developed by prolific University of California brain researcher Dr. William Partridge has piqued the interest of Boehringer Ingelheim Venture Fund, Shire plc, Takeda Ventures Inc. and Mitsui & Co. Global Investment Inc., which have just stocked up ArmaGen Technologies with a $17 million series A.

Santa Monica, California-based ArmaGen’s solution attaches substances like monoclonal antibodies, recombinant proteins and nonviral genes to a transporter protein that is normally able to cross the barrier. That creates what the company calls a molecular Trojan horse that can safely deposit the protein to a target receptor in the brain.

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Founded eight years ago, ArmaGen has been funded thus far with $20 million in nondilutive funding. According to the company, the new money will allow for expansion of existing operations and clinical development of its drugs that target diseases of the brain and spinal cord.

Initially, the company has set its sights on the orphan diseases Mucopolysaccharidosis (MPS) types I and II. Type I, also called Hurler’s syndrome, is a progressive disorder that can cause learning disabilities, deformity of the spine, hearing loss and cardiac valvular disease in children. In type II, or Hunter’s disease, accumulation of glycosaminoglycans can cause damage in various organ systems.

These treatments have advanced to toxicology studies in primates. The Santa Monica, California company also has other preclinical candidates that address Alzheimer’s, depression, stroke and Parkinson’s disease.

But ArmaGen’s is just one way researchers have approached crossing the barrier. Another popular take is medical devices that deliver drugs intranasally like those being developed by Kurve Technology, MedInvent and Impel NeuroPharma. Other approaches include chemical modification of drugs and permeability enhancers.

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A Deep-dive Into Specialty Pharma

A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.

Rapid growth in the market for BBB technology has been forecast by several market researchers. BCC Research has noted that the carrier-mediated transport like ArmaGen’s and Angiochem’s is as an emerging technology that will grow quickly by 2015.

[Photo from ArmaGen Technologies]