CAMBRIDGE, Mass.-- Catabasis Pharmaceuticals, Inc. (CATB), a clinical-stage biopharmaceutical company (“Catabasis”), and Sarepta Therapeutics, Inc. (SRPT), a commercial-stage developer of innovative RNA-targeted therapeutics (“Sarepta”), today announced a joint research collaboration to explore a combination drug treatment approach for Duchenne muscular dystrophy (DMD). The two companies will contribute their respective expertise to study an exon skipping treatment developed by Sarepta, together with an oral NF-kB inhibition treatment developed by Catabasis in a mouse model of DMD.

CAMBRIDGE, Mass., Sept. 28, 2016  -- Aegerion Pharmaceuticals, Inc. (AEGR), a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases, today announced that Japan’s Ministry of Health, Labor & Welfare (MHLW) has approved JUXTAPID for patients with homozygous familial hypercholesterolemia (HoFH).

 

HoFH is a serious, rare genetic disease that impairs the function of the receptor responsible for removing LDL-C (“bad” cholesterol) from the body. A loss of LDL receptor function results in extreme evaluation of blood cholesterol levels. HoFH patients often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.

NEW YORK, Sept. 28, 2016 -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer, today announced that it has signed a material transfer agreement with Biogen (Cambridge, MA) under which Biogen will test Anavex’s lead drug candidate, ANAVEX 2-73 in an oligodendrocyte precursor cell (OPC) differentiation assay. A satisfactory result from the OPC assay study may lead to an in vivo remyelination study using a chemical demyelination model.

 

“Battling demyelinating diseases such as multiple sclerosis requires an understanding of the processes that cause remyelination to fail.  Remyelination of demyelinated axons is typically a function of oligodendrocyte precursor cells. These studies will examine the therapeutic role ANAVEX 2-73 may play in permitting remyelination in the brain,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.

CAMBRIDGE, Mass., Sept. 26, 2016  -- Infinity Pharmaceuticals, Inc.(INFI) today announced initial clinical and new preclinical data for IPI-549, an orally administered immuno-oncology development candidate that selectively inhibits PI3K-gamma. Preliminary Phase 1 results from nine patients with advanced solid tumors show that the safety, pharmacokinetics and pharmacodynamics of IPI-549 monotherapy treatment appear favorable. Additionally, new preclinical data demonstrate that IPI-549 can reverse tumor resistance to checkpoint inhibitors, providing additional rationale for the planned evaluation of IPI-549 in combination with a checkpoint inhibitor in the ongoing Phase 1 study. These data are being presented in a poster session at the Second CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference: Translating Science into Survival taking place in New York City.