10/13/16 R&D Presentation

BETonMACE. About ⅓ enrolled. Dr. Paul Watkins, former head of the FDA liver injury department has since joined the BETonMACE DSMB. Adding Taiwan next year most of that paid for by Hepalink. 125 event futility analysis still mid-2017 estimate (Sweeney indicated H2 2017 and DM indicated H1 2017).

BETonDialysis. Launch early 2017. Phase 2b. High-risk CKD. Apabetalone. This study is a sequential cross-over trial to evaluate the safety, tolerability, and efficacy of apabetalone in CKD patients on hemodialysis. Up to 30 CKD subjects on hemodialysis, double-blinded, placebo-controlled, cross-over after 2 week washout. Proposed primary endpoint is ALP reduction. ~18 week trial.

BETonRENAL. Launch 2018. Phase 3. US Focused Trial. Apabetalone.

BETonPNH. Launch 2017. Small test population. Apabetalone. Probably Phase 2 but this wasn't indicated on the slide.

BETonFABRY. Launch 2017. Small test population. Subset of Renal Trial (not clear if Phase 2 or Phase 3). Apabetalone.

New compound(s): Milestone slide had the box checked off for "Confirm a second clinical compound IND candidate" with the added comment "Seven compounds chosen for follow-on testing." 2400 synthesized compounds in library. RVX-206, RVX-641, RVX-2101, RVX-2113 (Just happen to be 4 of DM’s favorites). Complement, Calciphylaxis, Autoimmune, FSHD/DMD, Pulmonary. No timeline given for initiating trials for these compounds. However, in bold red writing at the bottom of the slide read "Several programs are in discussions regarding licensing, partnering, etc."

Manuscripts: Four manuscripts published - Four done – Atherosclerosis, Jan2016; Metabolism, May 2016; Biochemical, June 2016; Data inBrief, July 2016. Two additional publications in process

Analysts: Addition of three new analyst in 2016 - One done (I don't think we've heard about this yet though), two in process (DM indicated they were in the audience).