SGMO up 20% this morning based on success of genomic program in HIV. SGPG has exclusive partnership to utilize SGMO's gene Rx in hemophilia, etc. Looking like a wise partnership move by Shire
stands at 10.9 according to Yahoo, down from 13..5. That is 10.9 days of average daily volume to cover. Do the shorts really want to take a chance that the BPH and cancer studies will have positive results? I doubt it. The pop with positive results will be huge. I see short covering as a positive going forward.
Although there is skepticism toward the company management, particularly its secretive approach to providing information,ClinicalTrials.gov indicates a conclusion date for both phase 3 trials as April 2014. So it is likely we will have the verdict in 2Q2014.
A Google search indicates that many urologists involved in the trials( who are not paid consultants for the company) have publicly praised the ease of use and efficacy of NX-1207. The drug only has to do significantly better then the saline placebo in the phase3 studies, which seems highly likely. The independent safety review board has already cleared the drug. Hence, I see a high likelihood of FDA approval. Urology colleagues tell me there is a strong need for a simple, office based procedure for BPH, which afflicts 40% of men over 50. The procedure will almost certainly be a money maker for urologists, and there is a huge reservoir of symptomatic men who are afraid of surgery but would be eager for a simple office procedure, so a rapid ramp-up is very likely. Add on the possibility of an effective Rx for low grade prostate cancer, and I think you have a compelling risk reward scenario.
Sentiment: Strong Buy
The phase 2 studies on nx-1207 are favorable, and the comments of several urologists are also quite favorable. The drug is being compared to a placebo, which sets a low bar. I remain optimistic that the drug will prove quite superior to the placebo injection. The safety studies have also uniformly been favorable. I am optimistic for an easy FDA approval
Iam a hematologist and attended an American society of hemaology review session provided by the Dana-Farber Cancer Institute physicians. They were quite optimistic about this unique drug. A positivee ph3 study and rapid uptake by the hematology community seemshighly likely
Sentiment: Strong Buy
I am a hematologist and attended a review course sponsored by Dana Farber Cancer institute this past Saturday. Jakafi(Incyte) has become a huge success for treating myelofibrosis; however, it exacerbates the low platelet count which frequently accompanies myelofibrosis. The studies to date suggest that Pacritinib is equally efficacious WITHOUT causing a reduction in platelets. I see great potential for pacritinib to displace Jakafi. Jakafi has propelled INCY to a market cap of 5 billion!
In multiple phase2 studies, CPX-351, an emulsified preparation of daunorubicin and cytosine arabinoside in a 5:1 fixed molar concentration, has shown superiority over conventional daunorubicin and cytosine arabinoside(3 + 7) in the Rx of adults with AML. This superiority is even more pronounced in patients with secondary AML(AML following chemotherapy for solid malignancies, AML evolving out of myelodysplastic syndrome). Hence, for its ph3 FDA study, the company wisely selected the more limited indication of secondary AML; the chances of success in secondary AML based on prior experience seems excellent. Assuming success with ph3, the company will have a long time window under its orphan drug status to get the drug approved for conventional AML. Studies are also underway in pediatric AML and adult myelodysplastic syndrome. A potential big market for a company with a tiny market cap.