Anti-amyloid antibodies: treatment for amyloidosis

Lead investigator: Scott Dessain, PhD, MD

Unmet need

Medical advances paralleling aging demographics in developed countries have created an unprecedented need for strategies to prevent and treat dementia, especially late-onset Alzheimer’s disease (AD) . The role of amyloid deposition is well established in AD etiology. The amyloid protein Aß is a therapeutic target for immunological clearance in AD.

Opportunity

LIMR scientists have generated a unique patient-derived huMab that recognizes a common structural feature of all mammalian and bacterial amyloid proteins. Recent positive reports from Eisai/Biogen on the efficacy of an Aß-targeting antibody in AD patients suggest that LIMR’s huMab may offer related therapeutic potential. Preclinical validation of the therapeutic concept to clear amyloid from brain tissue and restore its function has been published (see reference below).

Approximately 5.7 million Americans have been diagnosed with AD, and that number is expected to increase to 14 million by 2050, according to the Alzheimer’s Association. It is the sixth leading cause of death in the United States. In 2018, AD and other dementias will cost the nation about $277 billion in medical and care costs. By 2050, those costs could rise to as high as $1.1 trillion, according the Alzheimer’s Association.

The global market for AD drugs is expected to reach US $5.66 billion by 2024, up from US $3.64 billion in 2017, according to Zion Market Research.

Unique attributes

The LIMR huMab bind a universal structural fold present in nature in all amyloid proteins. This structural epitope is not readily accessed by antibodies and thus represents a rare antibody cloned from a patient. The huMab not only recognizes this universal structure but also breaks up amyloid structures.

Clinical applications

The LIMR anti-amyloid huMab offers potential applications in AD therapy.

Stage of development

The LIMR huMab has been cloned, and human hybridomas are stored. The IgG genes have been sequenced and are ready for expression in any desired expression system. Preclinical proof of concept for AD treatment or biofilm clearance has been obtained.

Intellectual property

Pending patent: U.S. provisional patent has been filed that includes the IgG sequences.

Collaboration opportunity

Develop an injectable biologic therapy for AD prevention or treatment based on initial preclinical proof of concept.

Relevant publication

Levites Y, O’Nuallain B, Puligedda RD, Ondrejcak T, Adekar SP, Chen C, Cruz PE, Rosario AM, Macy S, Mably AJ, Walsh DM, Vidal R, Solomon A, Brown D, Rowan MJ, Golde TE, Dessain SK (2015). A human monoclonal IgG that binds aß assemblies and diverse amyloids exhibits anti-amyloid activities in vitro and in vivo. J. Neurosci. 35(16):6265-76.

Contacts

Institutional contact: George C. Prendergast, PhD, LIMR President and CEO, 484.476.8475, [email protected]

L2C Partners contact: Merle Gilmore, 610.662.0940, [email protected]