Yes DND, it is good news. But it was not out of the blue. The first mention of Fabry's was in Kulikowski's presentation at the October 2016 R&D day. Then, at the December 2016 Annual Meeting we first saw this slide below that foretold of a Fabry's safety/efficacy Phase 2 study. This same slide has been a part of their slide deck for most if not all of their 2017 presentations.

Current & Ongoing Studies Support Alternate Indications

Neurofibromatosis – Malignant Peripheral Nerve Sheath Tumors (MPNST): studies have examined the effect of apabetalone, in vitro and in vivo, on MPNST (potential orphan indication)

Pulmonary Arterial Hypertension: studying the effects of apabetalone on primary lung SMCs was positive, animal study of the effect of apabetalone on top of standard of care

Muscular Dystrophy/Facio Scapulo Humeral Dystrophy: We have tested apabetalone and ~20 alternate RVX compounds for target and biomarker engagement in muscle cells, we are also analyzing human muscle biopsies from patients treated with apabetalone

Calciphylaxis/Calcification: due to positive in vitro data - animal studies of calcification are ongoing (also supports CVD)

Fabrys Disease: arranging ex-vivo treatment of Fabry patient blood, to analyze the effect of apabetalone on inflammatory mediators to move into a safety/efficacy Phase 2 study

Neuroinflammation: direct effects of apabetalone demonstrate reduced inflammation and microglial activation with drug treatment and no detrimental effects on neurons – animal study is ongoing

Paroxysmal Nocturnal Hemoglobinuria (PNH): due to positive data on the effect of apabetalone on the complement cascade, plans to start a safety/efficacy trial have been initiated

Chronic Kidney Disease (CKD): proteomic analysis of data from CKD PK study is ongoing

Characteristics of BET Inhibitors: studies investigating PK/tissue distribution of apabetalone and other BET inhibitors are underway, new scientist hired to investigate distribution, formulation and route of administration of BETi for other indications and target organs