NeuroImmunology

 Alkermes plc (NASDAQ: ALKS) today announced the initiation of a phase 1 clinical study of ALKS 8700, a novel monomethyl fumarate (MMF) molecule in development for the treatment of multiple sclerosis (MS). The randomized, double-blind study will evaluate the safety, tolerability and pharmacokinetics of several oral formulations of ALKS 8700 compared to both placebo and active control groups in approximately 125 healthy volunteers. ALKS 8700 is designed to rapidly and efficiently convert to MMF in the body and offer differentiated features as compared to the currently marketed dimethyl fumarate, TECFIDERA®.

This phase 1 study will investigate the pharmacokinetics and pharmacodynamics of multiple formulations and doses of ALKS 8700, and is designed to determine those suitable to progress into advanced clinical testing. The initiation of the phase 1 study follows Alkermes’ filing of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA), and the issuance of a composition of matter patent for ALKS 8700 from the United States Patent and Trademark Office (USPTO) inMarch 2014, which is expected to provide patent protection into 2033.

Abstract

Eighty percent of individuals with multiple sclerosis (MS) initially develop a clinical pattern with periodic relapses followed by remissions, called relapsing-remitting MS (RRMS). This period of fluctuating disease may last for a decade or more. Clinical relapses reflect acute inflammation in the central nervous system (CNS), composed of the brain and spinal cord. Often, different anatomic areas in the CNS are involved each time a relapse occurs, resulting in varied clinical manifestations in each instance. Relapses are nearly always followed by some degree of remission, though recovery to baseline status before the flare is often incomplete. There are nine approved drugs for treatment of RRMS. The most potent drug for inhibiting relapses, the humanized anti-α4 integrin antibody known as Natalizumab, blocks homing of mononuclear cells to the CNS. The mechanisms of action of the approved drugs for RRMS provide a strong foundation for understanding the pathobiology of the relapse. Despite substantial progress in controlling relapses with the current armamentarium of medications, there is much to learn and ever more effective and safe therapies to develop.

Advances in treating glioblastoma

Glioblastoma is the most common and most aggressive primary brain tumor in adults. Optimized standard treatment only confers a modest improvement in progression and overall survival, underscoring the pressing need for the development of novel therapies. Our understanding of glioblastoma (a molecularly heterogeneous disorder) has been accelerated in the setting of large scale genomic analyses, lending insight into potential actionable targets. Antiangiogenic therapies have been used in the treatment of glioblastoma, and our understanding of the means to optimize the role of these agents is continuing to evolve. Recently, immunotherapy has garnered increasing attention as a therapeutic approach in the treatment of gliomas. Promising novel approaches are under active development in the treatment of glioblastoma.

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