Pharmaceutical companies with a set Phase III data release dates usually have a runup leading into the release date. I usually try to buy the stocks 3-9 months before the catalyst date to make profit from the runup. I screened for companies that have a Phase III data release date set for this year. Here is a look at 5 companies that I found.
1. ZIOPHARM Oncology (ZIOP) is a biopharmaceutical company focused on the development and commercialization of new cancer therapies. The company's clinical programs include:
- Palifosfamide (ZIO-201), a novel DNA-targeted cancer treatment that bypasses drug resistance mediated by ALDH (aldehyde dehydrogenase), an enzyme associated with cancer stem cells, and has a favorable toxicity profile. Intravenous palifosfamide is currently being studied in a randomized, double-blinded, placebo-controlled Phase III trial (PICASSO 3) for the treatment of first-line metastatic soft tissue sarcoma and is also in a pivotal Phase 3 trial (MATISSE) for first-line metastatic small cell lung cancer. Additionally, the company is developing an oral capsule form of palifosfamide.
- IL-12 DNA, a novel DNA therapeutic that is delivered to the patient's tumor and expresses interleukin-12, a protein that controls anti-cancer immune responses. IL-12 DNA is currently in two Phase 1b studies, with plans to move into Phase II studies. ZIOPHARM's DNA therapeutics are being developed in partnership with Intrexon Corporation through a revolutionary synthetic biology platform that allows for targeted, controlled production of therapies in humans with a biologic on/off switch (the RheoSwitch Therapeutic System). Preclinical and discovery work with multiple therapeutic approaches, such as antibodies, immunotoxins and protein decoys, is expected to result in multiple clinical candidates in the next 12 to 24 months.
- Indibulin (ZIO-301) is a novel, tubulin binding agent that is expected to have several potential benefits, including oral dosing, application in multi-drug resistant tumors, no neuropathy and a tolerable toxicity profile. It is currently being studied in a Phase I/II trial in metastatic breast cancer.
- Darinaparsin (ZIO-101) is a novel mitochondrial- and hedgehog-targeted agent (organic arsenic) currently in ongoing studies with Solasia Pharma K.K.
The company announced recently that, after completing a planned, pre-specified futility analysis, the Independent Data Monitoring Committee for the PICASSO 3 trial has recommended that the trial proceed as designed and conducted. By design, there is no interim efficacy analysis. Outcome in progression-free survival, the study's primary endpoint for accelerated approval, is anticipated in the fourth quarter of 2012.
The company announced the second-quarter financial results on August 2 with the following highlights:
|Net loss||$0.3 per share|
|Shares outstanding||78.5 million|
The company expects its existing cash resources to support operations into the second half of 2013.
The stock has a $8.25 price target from the Point and Figure chart. I believe the target price is achievable after positive Phase III data in the fourth quarter.
2. Celsion (CLSN) is a leading oncology company dedicated to the development and commercialization of innovative cancer drugs including tumor-targeting treatments using focused heat energy in combination with heat-activated liposomal drug technology. Celsion has research, license, or commercialization agreements with leading institutions including the National Institutes of Health, Duke University Medical Center, University of Hong Kong, the University of Pisa, the UCLA Department of Medicine, Kyungpook National University Hospital and the Beijing Cancer Hospital.
Celsion announced on May 30 that it has reached its enrollment objective of 700 patients in the company's pivotal, Phase III HEAT Study, a multinational, randomized, double-blind, placebo-controlled clinical trial of ThermoDox in combination with radiofrequency ablation for the treatment of hepatocellular carcinoma, also known as primary liver cancer. The primary endpoint for the study is progression-free survival (PFS), and a total of 380 events of progression are required to reach the planned final analysis of the study. Celsion notes that 380 PFS events are projected to occur in late 2012. Top line results will be announced following review by the company's independent Data Monitoring Committee.
The company reported the first-quarter financial results on May 15 with the following highlights:
|Net loss||$0.19 per share|
|Shares outstanding||33.2 million|
The company continues to project that reduction in expenses will allow sufficient cash to fund operations through to the third quarter of 2013, providing Celsion with capital through key data milestones.
The stock has a $8.75 price target from the Point and Figure chart. I believe the target price is achievable after positive HEAT study results later this year or early 2013.
3. Vanda Pharmaceuticals (VNDA) is a biopharmaceutical company focused on the development and commercialization of products for the treatment of central nervous system disorders.
The tasimelteon Non-24 program continues to advance towards the goal of a projected mid-2013 NDA filing with the FDA. Vanda is in continuing discussions with the FDA to confirm the path and requirements for this regulatory submission. The SET Phase III efficacy study is fully enrolled and Vanda expects to report top-line results by the end of 2012. Vanda expects to report top-line results from the RESET Phase III efficacy study in the first quarter of 2013.
The company reported the second-quarter financial results on August 2 with the following highlights:
|Net loss||$0.28 per share|
|Shares outstanding||28.2 million|
|Cash per share||$5.13|
The stock is currently trading below the $5.13 net cash per share level. The company expects to report top-line results from the SET study by the end of 2012.
4. Amicus Therapeutics (FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare diseases. The company is developing orally-administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of human genetic diseases. Amicus' late-stage programs for lysosomal storage disorders include migalastat HCl monotherapy in Phase III for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase II for Fabry disease; and AT2220 co-administered with ERT in Phase II for Pompe disease.
Migalastat HCl monotherapy is in Phase III development for Fabry disease in patients with genetic mutations that are amenable to chaperone monotherapy. Amicus and GlaxoSmithKline (GSK) are currently conducting Phase III global registration studies (Study 011 and Study 012) of migalastat HCl monotherapy.
Study 011 is a randomized, placebo-controlled study with a six-month, double-blind primary treatment period and a six-month, open-label follow-up period. The primary endpoint is interstitial capillary globotriaosylceramide (GL-3) as measured in kidney biopsy. The six-month primary treatment period was completed in a total of 63 patients during the second quarter 2012. These patients received kidney biopsies at baseline and month six. All 63 of these patients are continuing in the six-month follow-up period, and all of these patients are expected to have 12-month kidney biopsies by year-end 2012.
Amicus and GSK have recently engaged in encouraging interactions with the FDA regarding the planned NDA for migalastat HCl. The agency indicated it would consider safety and efficacy data from both the six- and 12-month kidney biopsies to support conditional approval under subpart H. In order to preserve the integrity and availability of clinical data for the open-label follow-up period, Amicus and GSK have jointly determined that the unblinding and analysis of the data from the primary six-month treatment arm will not occur prior to the fourth quarter 2012. Both companies remain blinded to the results at this time.
Study 012 is a randomized, open-label, Phase III study targeting approximately 50 total patients (30 to switch to migalastat HCl and 20 to remain on ERT). Final enrollment continues to be expected by year-end 2012.
The company reported the second-quarter financial results on August 7 with the following highlights:
|Net loss||$0.2 per share|
Amicus expects to end 2012 with at least $90 million in cash, cash equivalents and marketable securities which is expected to fund its current operating plan beyond 2013.
The stock has a $15.25 price target from the Point and Figure chart. I believe the target price is achievable after the FDA approval of Migalastat HCl monotherapy.
5. Keryx Biopharmaceuticals (KERX) is focused on the acquisition, development and commercialization of medically important pharmaceutical products for the treatment of renal disease. Keryx is developing Zerenex (ferric citrate), an oral, ferric iron-based compound that has the capacity to bind to phosphate and form non-absorbable complexes. The Phase III clinical program of Zerenex for the treatment of hyperphosphatemia (elevated phosphate levels) in patients with end-stage renal disease is being conducted pursuant to a Special Protocol Assessment agreement with the FDA.
Ron Bentsur, the company's Chief Executive Officer, said:
"We are well-positioned to complete the Phase III long-term study for Zerenex in the fourth quarter of 2012 and we expect to have New Drug Application filings across all three key phosphate binder territories, the U.S., EU and Japan, in the first quarter of 2013. Importantly, we are operating with a well-controlled burn rate, and we believe that our current cash will be sufficient to take us through these key milestones."
The company reported the second-quarter financial results on August 8 with the following highlights:
|Net loss||$0.02 per share|
|Shares outstanding||71.5 million|
I believe the stock could trade at a $4-$5 level after positive results from the company's Phase III study of Zerenex later this year or early 2013.