Antibody potential a 'SIMPLE' fact

ROTTERDAM, The Netherlands—You can never have too manyfriends, and the same seems to go for business partners, as Shire recentlyannounced yet another partnership in the area of rare genetic diseases.

Shire and biopharmaceutical company arGEN-X, which specializesin discovering and developing human monoclonal antibodies, have entered into analliance to create novel therapeutic antibody products against multipletargets. The alliance represents the first time the two companies have workedtogether.

"We are delighted to pioneer human antibodies as novel,first-in-class therapies for rare diseases with a partner of Shire's caliber.Shire's depth of experience in protein-based therapies is the perfectcomplement to our own discovery capabilities," Tim Van Hauwermeiren, CEO ofarGEN-X, said in a press release. "We are confident that our SIMPLE Antibodyplatform, which consistently delivers antibodies of therapeutic quality againstcomplex targets often intractable with other technologies, will bring significantvalue to this alliance. We believe this alliance represents an industry firstand we are looking forward to an exciting and productive collaboration withShire."

Per the terms of the agreement, arGEN-X will receive anupfront technology access fee, research funding and preclinical successpayments, and will be responsible for the discovery work, as well aspreclinical support and providing antibody development candidates for Shire.Shire will gain an option to license promising leads for additional preclinicaland clinical development as well as commercialization worldwide, and will alsopay arGEN-X fees, milestones and royalties on future product sales. Specificfinancial details were not disclosed, and no details were released as to howmany targets the alliance would focus on, or in which indications.

"As a leader in innovative therapies for rare diseases,Shire is continuing to apply new technologies to address the needs ofpatients," Philip J. Vickers, senior vice president of research and developmentat Shire Human Genetic Therapies (HGT), said in a press release. "Monoclonalantibody therapy is an underutilized approach to the treatment of rarediseases, and this novel platform has the potential to bring multiple drugcandidates into our early-stage pipeline. Partnerships such as this one witharGEN-X are an important part of our strategy to bring new therapies to thosesuffering from rare diseases worldwide."

arGEN-X's Superior Immunodiversity with Minimal Protein LeadEngineering (SIMPLE) Antibody platform is based on the active immunization ofCamelids—specifically llamas—with target antigens in order to deliver antibodyvariable regions that are essentially identical to those of human antibodies.According to arGEN-X's site, "active immunization of outbred llamas with humanproteins elicits strong, unrestricted IgG responses. The variable regions ofsuch IgGs reveals an extraordinary degree of similarity to their human antibodycounterparts." The end result is SIMPLE Antibodies that display "extremely highbinding affinities for their targets, translating into high potencies infunctional bioassays." With the platform, arGEN-X can generate full-size, humantherapeutic antibodies by combining the variable regions with human constantdomains.

"arGEN-X's SIMPLE Antibody platform is a rapid, efficientway of identifying multiple different, high-potency human antibodies, evenagainst very complex targets," says Dr. Debbie Allen, senior director ofbusiness development for arGEN-X. "Multiple discovery candidates enable you tochoose only the best possible lead for development as a therapeutic antibodyproduct. The majority of existing antibody platforms do not give this highdegree of lead choice."

Jessica Cotrone, senior director of corporate communicationsfor Shire HGT, says the company has not seen widespread use of such monoclonalantibody technology, which was "very attractive to us."

"There are many possibilities that are aligned with Shire'sfocus on the orphan disease space, meeting the needs of the specialistphysician and pursuing innovative treatment options in areas of high unmet needthat deliver value for physicians, patients and the healthcare community," saysCotrone.

Allen adds that arGEN-X expects to see the rare diseasemarket gain more attention as time goes on, "both from antibody companies andfrom other platform technology players."

"We believe this deal between arGEN-X and Shire is anindustry first, but we would not anticipate it being the last, since antibodieshave been widely accepted as a valuable class of drug across the entirebiopharmaceuticals industry," she says.