With a potential U.S. market for Chronic Kidney Disease (CKD) and End Stage Renal Disease (ESRD) patients of almost $1bb, based on 5mm CKD patients above age 65, of whom ~50% are in stage 3-5, there is ample opportunity for a few under-appreciated stories focusing on this market to see real traction as we head into 2H/13. I've highlighted a few of the more notable players in the space, and some ones that may be under the radar, including Keryx (KERX), AMAG Pharma (AMAG), La Jolla Pharma (OTC:LJPC), Dynavax (DVAX), and Opko (OPK).
Keryx: Keryx will be filing its U.S. New Drug Application (NDA) in 2H/13 for Zerenex, following Phase III data coming later this year from the Japanese trial for CKD and dialysis patients, data from Dr. Lewis at the Word Nephrology Conference in June, and at the ASN conference in November (5-10 in Atlanta). I expect the European filing (MAA) to be filed shortly after this NDA submission. Zerenex should thus launch in mid-2014. KERX had initiated study for CKD in early '13, with enrollment expected to take 6 months and then dosing for 12 months, enrolling 150 stage 3 to 5 patients for a duration of 3 months, with primary endpoint as change in territin, and serum phosphorus. My view is that KERX may potentially require partnering up to expand beyond the ESRD setting into the Stage 3-5 CKD setting, as it is a much larger venture, but the data will drive that decision and value inflection likely in 2H/13.
AMAG Pharmaceuticals: AMAG's lead product, Feraheme, is a marketed Intravenous therapy, indicated for CKD and in development for iron-deficiency anemia, a very common nutritional defect, for which it is on track for its 4Q/13 label expansion. This expansion would mark a significant advance relative to competitors, and position the company well to compete against iron dextran. The only other major player in the area, Luitpold, has a July '13 Prescription Drug User Fee Act date after receiving its 3rd complete response letter from the FDA.
La Jolla: LJPC is developing LJPC-101, an immunosuppressant and a subcutaneous formulation of its lead GCS-100 product (an inhibitor designed to sequester & eliminate circulating levels of galectin-3), for renal transplantation and end-stage renal disease. Over-expression of galectin 3 has been implicated in chronic organ failure and cancer, thus modulation of the activity of G-3 is an attractive target for therapy in these diseases. LJPC has indicated that it has initiated the Phase II portion of the clinical trial for '101 in 2Q/13, after completing the Phase I portion on May 6th. As per Clinicaltrials.gov, the study is still enrolling across several states.
Other Players To Watch:
Dynavax Technologies: Dynavax, with a new CEO [previously President of Pharma Europe at GlaxoSmithKline (GSK)], should be progressing with their Heplisav vaccine, which is indicated for Hepatitis B, but has a supplemental application in CKD patients, and will be submitted to the FDA following their June '13 initial meeting. Previously, the FDA had issued its complete response letter in which it asked for more safety data in healthy adults, and some issues with their manufacturing process. If DVAX can gain clearance from the FDA for a narrow filing for CKD that could be completed right away (which management indicated on its March call) and potentially, no new data would even need to be submitted.
Opko: Opko will be reporting its Phase III data for CTAP101, indicated for secondary hyperparathyroidism in patients with stage 3 or 4 CKD and vitamin D insufficiency, in early 2014. While it's a bit early for this company, it's on the radar to watch setting up into 2H/13 as the data leaks start to play out, perhaps as early as ASN in November.
Summary: The CKD opportunity in the U.S. is significant and underappreciated, and there are a few notable ways to play these stock catalysts in 2H/13. Meaningful opportunities include KERX's Zerenex data at ASN, AMAG's Feraheme label expansion in 4Q/13, La Jolla's '101 phase II data read-out in 4Q, DVAX's potential FDA clearance for Heplisav, and Opko's ph III data for CTAP 101.