Protalix BioTherapeutics, Inc. (PLX), announced that new clinical data on taliglucerase alfa would be presented at the 8th Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2012 being held this week in San Diego, California.
As readers may know, taliglucerase alfa is the company's proprietary plant cell expressed recombinant form of human Glucocerebrosidase (GCD), which is being developed for the treatment of Gaucher disease. Protalix' novel bioreactor plant cell system is based on disposable plastic vials that provides stable, optimized conditions, with manufacturing capabilities for the entire range of proteins, including antibodies, complex enzymes and plant-derived pharmaceuticals. Its patented bioreactor system utilizes sterilized, large flexible plastic containers for culturing and harvesting cells in consecutive cycles, with central unit providing oxygen and nutrients.
The data being presented is an important signal to the market and to those who have been watching developments in regards to the marketing applications for taliglucerase alfa which have been filed in the United States, Europe, Israel, Brazil and Australia. The U.S. Food and Drug Administration Prescription Drug User Fee Act (PDUFA) has a specified target action date for taliglucerase alfa on May 1, 2012.
Last December, the firm received notification from the U.S. Food and Drug Administration (FDA) that the FDA had extended the PDUFA goal date of the New Drug Application (NDA) for taliglucerase alfa by three-months from the previous PDUFA date of February 1, 2012.
Prior to that in November 2011, the company submitted certain clinical information regarding taliglucerase alfa in response to an FDA request. This request related mainly to the presentation of select data provided in the NDA and several analysts who follow the stock feel the chances for approval are good. The data presented by the company certainly appears to bolster that sentiment.
To re-cap, long-term safety and efficacy data from the company's double-blind, follow-on extension study- statistically significant results in every case, demonstrated:
- reduction in mean spleen volume after 24 months, compared with baseline
- mean reductions in liver volume
- mean increases in hemoglobin concentration
- mean increases in platelet count
- mean reductions in chitotriosidase activity
Open-label, nine-month switchover trial, in which patients with stable disease were switched from treatment via intravenous infusions of imiglucerase (Cerezyme) to intravenous infusions of taliglucerase alfa:
- patients remained stable with regard to the efficacy endpoints -- spleen volume, liver volume, platelet count and hemoglobin concentration -- after switching to taliglucerase alfa from imiglucerase
- taliglucerase alfa was well tolerated, and no drug related serious adverse events were reported
- one patient developed neutralizing IgG antibodies that were determined to be positive in an in vitro assay, and were determined to be negative in a cell-based assay; another patient experienced a hypersensitivity reaction, which was treated in a physician's office and resolved
Long-term bone marrow responses:
- eight patients from the company's pivotal and extension trial had their fat fraction evaluated by QCSI
- at 24 months, seven of the eight patients demonstrated significant improvement in fat fraction from baseline; one patient remained unchanged
- four patients whose score for bone was determined to be "at risk" at baseline (fat fraction <0.23) were no longer classified as "at risk" according to the protocol after 24 months of treatment with taliglucerase alfa
Shares climbed over $1 since we reported in January that this data presentation catalyst was coming this week. In addition, we told readers that Canaccord Genuity life sciences analyst Ritu Baral which assigned a "BUY," $8 target to the stock noted investor concern over the recent weakness in the euro and, in a note to investors last month, highlighted biotech companies in her coverage universe with the greatest foreign currency exposure. Among them was Protalix. The Israel-based company reports in dollars. She notes an expected CHMP review date around mid-2012 for lead drug taliglucerase, a recombinant enzyme therapy for Gaucher's disease. Canaccord also notes that the drug could receive approval in Israel and Brazil, both major Gaucher's markets, shortly after U.S. approval.
We continue to see volume coming into the healthcare sector and at the top of the list for Thursday's market movers was Accuray Incorporated (ARAY) which continued to trade up $7.25+0.96 (15.26%) after announcing financial results for the second quarter of fiscal 2012 that ended December 31, 2011.
Accuray included solid revenue, positive service margins and effective management of operating expenses in their report to the market and Euan S. Thomson, Ph.D., president and chief executive officer of Accuray says his firm continues to capture competitive vault space, and as their installed base grows, so does their recurring service revenue. "We remain ahead of plan with our TomoTherapy System reliability improvements and have made significant progress on improving service gross margins," said Thomson. "Overall, we're pleased to report that we remain on track to return to profitability on a non-GAAP basis as scheduled by the end of fiscal year 2013."
Shares of Medgenics (MDGN) traded up again, this time hitting an intra-day high of $6.76. The stock is still up significantly at the end of the day despite giving some of those gains back. We told our readers about the company several days ago and the stock has seen significant volume and buying interest since. As the article pointed out, investment bankers at Nomura call this one of the top 10 biotech companies to watch over the next 5 years. We'll keep an eye on it, and remind everyone that you need to set tight stops in-case a secondary follows at any point. Technicals still look okay here, with an overall consensus of technical indicators lookin 65.71% Bullish, but I am concerned about the candle formation that we're being left with at the end of the day.
Some investors took profits off the table at Inergetics (NRTI.OB), but they may have done so too soon. There is still more upside at emerging nutritional/sports drink company and we think given the amount of interest and news flow we've seen from the company during the last few days, things are finally starting to heat up.
As you may recall, we have been following this firm and their unique technology for some time and our investment thesis has always been focused on the company's proprietary nutritional products which help athletes and others bounce back from work-outs very quickly. We believe that they are a best of breed product and that opportunities exist for them to become competitors in the nutritional supplement space. Inergetics displaced CytoSport's Muscle Milk from supplying West Point last summer. A transaction that coincides with the Food and Drug Administration issuing a warning letter to Muscle Milk maker Cytosport saying the dominant protein drink brand's labels could be misleading to consumers. Now, the firm has issued two press releases which could be setting the table for some real revenue flow and more endorsement deals.
First, they finally announced that their long awaited "Ready To Drink" product is ready and yesterday they let the market know that they will be generating some revenue through their private label channel by entering into a supply agreement with Muscle Maker Grill Restaurants. This is one of the fastest growing health food franchise operations in the United States, has 53 restaurants in six States and NRTI will power the chain's Muscle Maker Grill Ultra Pro energizer protein shakes. Sports athletes and trainers absolutely love this stuff and I'd keep my eye them as it seems like they're finally getting their head above some of the financings they did. That the stock has been climbing is very encouraging. I just thought today's news was much more significant than most realize. Note the golden cross (50 day moving average crossing over the 200 day moving average) that just formed on the chart. That is usually a very bullish sign and one we often look for when running screens.
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I have been long NRTI for over 12 months and have no plans to sell my position for some time.