As the second quarter of 2011 gets under way, investors are taking note of the FDA’s sudden emergence of being more “new drug friendly” in the first quarter and early second quarter. The first week of April witnessed major approvals of GlaxoSmithKline’s (GSX) and XenoPort Inc.’s (XNPT) Horizant for moderate-to-severe Restless Legs Syndrome in adults as well as AstraZeneca Pharmaceuticals’ (AZN) Vendetanib for medullary thyroid cancer. Original approvals for drugs in 1Q of 2011 numbered 30 while 2010 witnessed 26 total original approvals in the same period.
The increased approvals of this friendlier FDA are driving biotech investors to find the “next big thing”. Although a run up to a PDUFA or NDA date is a preferred trading method for some, the risk of an FDA surprise approval, CRL or outright rejection is often still too much for many regardless of their long or short positions in the security to hold through the catalyst date. Rather than trusting in their views of what they know or what they think they may know about a drug’s chance with the FDA, investors may rather review preliminary or interim trial data along with analysts’ opinions to discern a trial’s success potential and analytically determine if the trials will likely meet efficacy and safety endpoints. They then take positions accordingly. The second quarter of 2011 is ripe for the picking with a host of biotech trials completing, awaiting data analysis, obtaining funding or having findings presented to the public or meeting of peers.
CorMedix Inc. (AMEX: CRMD) will be completing a 60 patient enrollment in its phase II trial of CRMD001 in 2Q 2011. The trial is evaluating the safety and efficacy of the drug to prevent contrast induced acute kidney injury in subjects undergoing x-ray evaluation for kidney disease. These patients are often given iodinated contrast dyes to enhance the x-ray images and allow for a more accurate evaluation. However, patients with renal disease often cannot tolerate these dyes, which can become toxic to them. CRMD001 is a chelating agent that binds labile iron, which appears to reduce the toxicity of the dyes. Currently, there is no FDA-approved drug for this indication so positive results here would be a key catalyst. CorMedix would have worldwide rights to market and manufacture CRMD001 if approved. Interim data of the randomized, double blind, placebo-controlled trial announced on March 31st were positive and indicated that the trial findings were statistically significant and should go to completion. With the trial completing in 2Q 2011, data evaluation will likely be complete in 2H 2011 with positive results paving the way for the already FDA-approved SPA phase III trial.
Neoprobe Corporation (AMEX: NEOP) will be filing its new drug application (NDA) for Lymphoseek possibly during the 2Q but most likely 3Q 2011. Lymphoseek will be used as a tracing agent to help surgeons identify cancerous lymph nodes in patients with breast cancer or melanoma. This third phase III trial of Lymphoseek, designated NEO3-09, completed in mid 1Q and was designed to further support the NDA and potentially expands the product’s labeling. It was a superiority trial comparing the product to currently approved dyes for the same indication. The data for NEO3-09 will be presented at the June 2011 American Society of Clinical Oncology meeting. Investors will be keying in on this data and using it to hedge their bets for NDA acceptance and product approval.
Celsion Corporation (NASDAQ: CLSN) is likely to complete its phase III trial for ThermoDox, a proprietary heat-activated liposomal encapsulation of doxorubicin, in treating hepatocellular carcinoma or primary liver cancer in 2Q 2011. ThermoDox received Fast Track Designation from the FDA and has received Orphan Drug Designation in both the U.S. and Europe. Completion of this trial, designated the “Heat Study” and its subsequent data analysis will be huge catalysts for Celsion. The trial itself is operating under the FDA’s Special Protocol Assessment, which simply means the trial's design, clinical endpoints, and statistical analyses are acceptable for FDA approval. It is also expecting to expand the ThermoDox program to include other indications (via additional trials) such as secondary liver cancers, bone cancer and recurrent chest wall breast cancer sometime in 2011. An October 10th 2010 PR noted that written guidance from the FDA stated that the Company would not be required to conduct any additional non-clinical pharmacology, safety pharmacology and general toxicology studies assuming the results of current studies are sufficient. On at least three different occasions statistical analyses of the trial data indicated that the trial should continue based on the probability of the treatment success in the indication. It should be noted that the September 2010 and February 2011 evaluations by the Data Monitoring Committee had continuation recommendations for the trial that were unanimous.
Cytosorbents (CTSO.OB) announced European Mark CE marketing approval for its cytokine filtration device, Cytosorb, on March 31st. The device was approved to remove plasma cytokines in patients with sepsis and respiratory failure. Currently, there are no approved devices in the E.U. specifically for cytokine filtration. This approval of the company’s flagship product will likely open doors for other indications in which cytokine levels are elevated such as in trauma, burn injuries, acute pancreatitis, respiratory distress syndrome, influenza and other indications. Data analysis is under way for the trial in which currently investors only know that the device was “well tolerated” and that interim data indicated a 49.1% (P = 0.01) reduction in IL-6 cytokines. Potential 2Q catalysts could be data presentation, marketing plans, partnerships and longer term plans for FDA approval. Additionally, an application by Cytosorbents to DARPA (pdf) an agency of the United States Department of Defense, could mean additionally funding for this company, which is now transitioning from development phase to marketing and manufacturing phases. The February 8th presolicitation states “DARPA is soliciting innovative research proposals to manage the life-threatening blood infection known as sepsis. The goal of the Dialysis-Like Therapeutics (DLT) program is to develop a portable device capable of controlling relevant components in the entire blood volume on clinically relevant time scales.” Compare this to CEO Dr. Philip Chan’s comments about the Mark CE marketing approval and his description of the device “To our knowledge, we are the only device approved in the E.U. specifically as a cytokine filter, opening up many opportunities to not just treat patients with sepsis, but also patients with a host of life-threatening and non-life threatening inflammatory conditions where cytokine levels are elevated.” It is certainly understandable that Cytosorbents has a better than average chance of obtaining some of this funding.
Lexicon Pharmaceuticals (NASDAQ: LXRX) plans to report Phase 2a results for LX1032 for controlling symptoms associated with carcinoid syndrome and the complementary open-label clinical trial results in 2Q 2011. It will also be initiating a phase 2b study of LX4211 for type 2 diabetes in late 2Q or early 3Q 2011. The phase 2a results for LX4211 indicated statistically significant and rapid improvements in several areas in patients with type 2 diabetes, including significant reductions of HbA1c within four weeks. A separate LX4211 tablet formulation mechanistic study also indicated increased levels of GLP-1 and PYY, important mediators of glycemic and appetite control. The potential market for LX4211 is obviously huge, and investors will be anxiously awaiting results for this study whenever it completes. A relatively under the radar company, Lexicon has drug discovery alliances with Bristol-Myers Squibb (BMY), Genentech, N.V. Organon, and Takeda Pharmaceutical Company Limited (OTCPK:TKPYY). Not only will investors be evaluating these results closely, but also some key players in larger pharmaceuticals with close ties to Lexicon.
Disclosure: I have no positions in any stocks mentioned, but may initiate a long position in CTSO.OB over the next 72 hours. I receive no compensation to write about any specific stock, sector or theme.