Re: The Feb 26th DSMB PR.....re-examined.
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posted on
May 14, 2018 07:23PM
GAC wrt eGFR, the lack of progress with the CKD program IMO is disgraceful and management should be embarrassed. The positive post hoc eGFR and ALP data from prior studies in conjunction with the results from the ph1 New Zealand study has opportunity written all over it yet management has mustered up nothing except kicking the trial for patients on dialysis down the road 3 or 4 times (I’ve lost count).
While I realize there is a CKD trial embedded into BETONMACE, a ph2 renal trial was to be one of the many shorter inexpensive trials which would provide “inflection points” for the stock which DM spoke about two AGM’s ago. Below are some excerpts from the Jan 23 2017 NR regarding the ph1 NZ study. 16 months ago this certainly seemed like an outstanding avenue to broaden RVX’s opportunity base. Maybe even bring in an equity partner to explore that uncluttered space of unmet medical need in CKD. I sometimes wonder what management has been doing.... well I guess they did mange to give away the Israeli territory for free.
“....preliminary results from the New Zealand based Phase 1 trial with severe kidney (renal) impaired patients. The data showed remarkable results in reducing inflamed protein biomarkers in patients with severe kidney impairment versus healthy control patients. It is believed that this is the first time in medical history that a direct connection of this type can be made between epigenetic regulation and its potential for positive disease impact. Both the healthy group and the severely impaired kidney group received equal amounts of apabetalone.”
"It was shocking and highly encouraging to see the direct comparison of the protein data ranked by magnitude of effect in the two groups. For the first time, epigenetic and BET inhibition clinical data has been shown to differentially affect genes and proteins between advanced chronic kidney disease (CKD) patients and normal subjects," stated Donald McCaffrey, President and CEO.”
“Dr. Kamyar Kalantar-Zadeh, Chairman of the Renal Clinical Advisory Board and member of the BETonMACE Clinical Steering Committee stated, "These early results help provide a first understanding of the potential rapid effects of BET inhibition and apabetalone on key proteins that drive risk and death in Stage 4 CKD and potentially dialysis patients. Late Stage 4-5 CKD and dialysis patients represent very important groups whom currently have limited therapeutic strategies that can improve their outcomes and quality of life," Dr. Kalantar-Zadeh added.”
“About Advanced CKD & Dialysis
Advanced CKD encompasses Stages 4 & 5, and it can be alternatively defined as an estimated glomerular filtration rate (eGFR) of <30 ml/min/1.73m2. As reported in the 2016 United States Renal Data System (USRDS) Annual Report, approximately 1.4 million patients in the US have advanced CKD, 474,000 of which are on dialysis treatment. According to the USRDS, advanced CKD cost the US healthcare system approximately US$17 billion in 2014, with an average annual cost exceeding US$28,000 per patient. Additionally, dialysis treatment costs the US Medicare system approximately US$28 billion with an average annual cost exceeding US$80,000 per year.” **my add, that would be $80,000 per year per patient, once on dialysis.