* Cancer (Perifosine); and
* Renal disease (Zerenex) under Special Protocol Assessment (SPA) agreements with the FDA.
$KERX has retained all key commercial rights for its two lead compounds and has the resources to complete Phase 3 development for both of its lead compounds, which provides more leverage in discussions for potential partnerships.
1.) Perifosine (KRX-0401) (PI3K/Akt pathway inhibitor anti-cancer agent) (inhibits key signaling pathways associated w/ numerous types of cancer, including tumors that may be resistant to standard anti-cancer treatments).
* Pivotal Phase 3 (X-PECT) Clinical Trial under SPA -- In April 2010, began metastatic colorectal cancer (mCRC) pivotal trial and expects to complete study during 4Q11 w/ planned DSMB safety and futility rvw during 3Q11, North American rights to perifosine in-licensed from $AEZS, FDA Fast Track status, ClinicalTrials.gov ID NCT01097018.
* Pivotal Phase 3 Clinical Trial under SPA -- In December 2009, began relapsed / refractory multiple myeloma (MM) pivotal trial, ClinicalTrials.gov ID NCT01002248 w/ results expected during 4Q12, FDA Orphan Drug / Fast Track status, North American rights to perifosine in-licensed from AEZS.
2.) ZERENEX (ferric citrate) (an iron-based phosphate binder to treat elevated phosphorus in dialysis patients) -- Zerenex works by forming iron-phosphate complexes in the gut that are not absorbed since patients with end-stage renal disease (ESRD) are prone to electrolyte disorders such as elevated phosphorus due to the absence of normal kidney function.
Zerenex represents a niche market opportunity within a well-understood and established class of drugs (phosphate binders) that has a key differentiation of being iron-based compared to existing treatments such as PhosLo, Fosrenol, and Renagel/Renvela. In addition, Zerenex has the potential to offer an improved safety profile/patient acceptance with reduced GI/bloating side effects and approximately 50% less dosing burden (i.e. taking less pills less often) compared to existing phosphate binders.
* Pivotal Phase 3 Clinical Trials (US and EU) under SPA (and equivalent EMA designation for Europe) -- In November 2010, reported short-term efficacy study met all primary and secondary endpoints. A long-term safety study (58-week study in approx. 300 patients) was initiated in September 2010 w/ expected completion during 2Q12 to support a potential NDA filing 3Q12. Finally, Japan partner JT Torii began a Phase 3 program for that country in April 2011, which resulted in a $5 million (M) milestone payment to $KERX.
In late April, $KERX reported 1Q11 financial results which included the following highlights:
* 69.1M shares of common stock outstanding as of 5/9/11
* As of 3/31/11, $KERX reported $23.1M cash / equivalents and no debt
In addition the Phase 3 catalysts are spread out evenly with data expected in late 2011 (Perifosine colon cancer trial), mid-2012 (Zerenex long-term safety trial), and late 2012 (Perifosine multiple myeloma trial) - resulting in a steady flow of P3 clinical trial catalyst data.
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