Pharmaceutical companies with a set Phase III data release date very often have a run-up leading into the release date. I usually try to buy the stocks three to nine months before the catalyst date to make profit from the run-up. I screened for companies that have a Phase III data release date set for this year. I wrote the Part I of "5 Pharmaceutical Companies With Phase III Clinical Study Catalysts This Year" on August 10, Part II on August 13 and Part III on August 16. Here is a look at 5 additional companies that I found.
1. Acadia (ACAD) is a biopharmaceutical company focused on innovative treatments that address unmet medical needs in neurological and related central nervous system disorders. Acadia has a pipeline of product candidates led by pimavanserin, which is in Phase III development as a potential first-in-class treatment for Parkinson's disease psychosis. Acadia also has clinical-stage programs for chronic pain and glaucoma in collaboration with Allergan (AGN) and two preclinical programs directed at Parkinson's disease and other neurological disorders. All of Acadia's product candidates are small molecules that emanate from discoveries made using its proprietary drug discovery platform.
Upcoming Phase III catalysts
Uli Hacksell, Ph.D., Acadia's Chief Executive Officer commented on August 8:
"Our progress in the first half of 2012 established the foundation for what we believe will be an exciting and value-driving second half of the year. We look forward to reporting top-line results this fall from the ongoing pivotal -020 trial in our Phase III Parkinson's disease psychosis program with pimavanserin. Importantly, our pipeline of product candidates, led by pimavanserin, positions ACADIA with multiple product and commercial opportunities and significant growth potential."
Currently, there is no FDA-approved therapy for Parkinson's Disease Psychosis (PDP). The company believes that pimavanserin has the potential to be the first safe and effective drug that will treat PDP without compromising motor control, thereby significantly improving the quality of life for patients with Parkinson's disease. PDP is a large unmet medical need and represents what the company believes is an ideal lead indication for pimavanserin.
The company reported the second-quarter financial results on August 8 with the following highlights:
|Net loss||$5.4 million|
Acadia expects that its current cash resources and anticipated payments from its existing collaborations will be sufficient to fund its operations at least into the second quarter of 2013.
The stock has a $8.38 price target from the Point and Figure chart. I am expecting the stock to reach a $3-$5 price range after positive Phase III results this fall from the -020 trial. If we do get negative results the stock would probably drop below $1.
2. BioCryst Pharmaceuticals (BCRX) designs, optimizes and develops novel small molecule drugs that block key enzymes involved in infectious and inflammatory diseases. BioCryst currently has two late-stage development programs: peramivir, a viral neuraminidase inhibitor for the treatment of influenza, and ulodesine (BCX4208), a purine nucleoside phosphorylase (PNP) inhibitor for the treatment of gout. In addition, BioCryst is advancing two preclinical programs towards IND filings: BCX5191, a nucleoside analog inhibitor of HCV RNA polymerase (NS5B) for hepatitis C, and BCX4161, an oral inhibitor of plasma kallikrein for hereditary angioedema. Utilizing state-of-the-art structure-guided drug design and crystallography, BioCryst continues to discover innovative compounds with the goal of addressing unmet medical needs of patients and physicians.
Upcoming Phase III catalysts
BioCryst continues to enroll patients in the ongoing Phase III efficacy clinical trial of the influenza antiviral i.v. peramivir. The company plans to provide an update following the planned interim analysis, which is expected after the conclusion of the 2012 Southern Hemisphere flu season towards the end of 2012. This analysis has the purpose of re-estimating the sample size required for the primarily efficacy analysis of the study.
The company reported the second-quarter financial results on August 2 with the following highlights:
|Net loss||$12.3 million|
Based upon current trends and assumptions, as well as the company's planned operations, BioCryst expects net operating cash use to be in the range of $37 to $43 million, reflecting an increase of $5 million from previous projections, and its total operating expenses to be in the range of $57 to $69 million, unchanged from previous projections. The $5 million increase in the company's operating cash forecast is due primarily to an anticipated increase in R&D investment for the development of the hepatitis C program, as well as a lower net margin contribution related to peramivir development under the BARDA/HHS advanced development contract. BioCryst's 2012 financial results will be heavily dependent on peramivir-related operating expenses, which are largely a function of the rate of enrollment in the company's ongoing Phase III clinical trial, which in turn is dependent on the prevalence and severity of influenza in those geographies where BioCryst has clinical sites.
The stock has a $11.75 price target from the Point and Figure chart. I am expecting only a $5-$6 share price after positive interim Phase III data later this year. The stock did see some insider buying in June 2012.
3. BioDelivery Sciences International (BDSI) is a specialty pharmaceutical company that is leveraging its novel and proprietary patented drug delivery technologies to develop and commercialize, either on its own or in partnerships with third parties, new applications of proven therapeutics. BDSI is focusing on developing products to meet unmet patient needs in the areas of pain management and addiction. BDSI's pain franchise currently consists of two products utilizing the patented BEMA technology. ONSOLIS (fentanyl buccal soluble film) is approved in the U.S., Canada, and the E.U. (where it will be marketed as BREAKYL), for the management of breakthrough pain in opioid tolerant, adult patients with cancer. The commercial rights are licensed to Meda for all territories worldwide except for Taiwan (licensed to TTY Biopharm) and South Korea (licensed to Kunwha Pharmaceutical Co.). BDSI's second pain product, BEMA Buprenorphine, is being developed for the treatment of moderate to severe chronic pain and is in development in a high dose formulation with naloxone for the treatment of opioid dependence. BEMA Buprenorphine for chronic pain is licensed on a worldwide basis to Endo Pharmaceuticals.
Upcoming Phase III catalysts
BNX - Opioid Dependence. Results of the pivotal pharmacokinetic study are anticipated by the end of September.
Positive results were already obtained from a pharmacokinetic study (BNX-106) examining the effects of multiple BNX films administered concurrently. The study confirmed that the buprenorphine pharmacokinetics were nearly identical following multiple BNX films applied at one time compared to an equivalent dose administered as a single film. Additionally, there was a linear relationship in buprenorphine pharmacokinetics across the dose range of BNX administered. Regarding the naloxone component of BNX, the study demonstrated that the exposure of naloxone is similar to the reference standard, Suboxone.
Results of the aforementioned study allowed for initiation of both the pivotal pharmacokinetic study and the safety study, both of which are currently underway. The current study timeline supports the filing of an NDA for BNX in the first half of 2013.
The company reported the second-quarter financial results on August 9 with the following highlights:
|Net income||$2.5 million|
Upcoming (other) milestones
- Recruitment of two Phase III studies for BEMA Buprenorphine. BDSI and Endo expect to continue recruitment in two Phase III efficacy studies for BEMA Buprenorphine for chronic pain, one in opioid experienced and one in opioid naïve patient groups.
- Launch of BREAKYL in the E.U. BDSI expects its commercial partner, Meda, to launch BREAKYL (brand name for ONSOLIS in the E.U.) in the E.U. during the fourth quarter of 2012. This launch would result in a milestone payment to BDSI of $2.5 million.
- Exploration of Potential New Products and Technologies. In addition to supporting its marketed product and advancing its lead products in development, BDSI is also, as in the past, exploring the application of its BEMA drug delivery technology to additional pharmaceuticals. Furthermore, BDSI has been investigating potential new products or technologies to complement its existing portfolio.
The stock has a $11.88 price target from the Point and Figure chart. The stock could reach $6-$7 after positive Phase III data in September. I am not expecting the $11.88 price target to be reached before the FDA approval of BNX in late 2013 or early 2014.
4. BioSante's (BPAX) corporate strategy is the development of high value medically-needed pharmaceutical products, as well as seeking to implement strategic alternatives with respect to its products and its company, including licenses, business collaborations and other business combinations or transactions with other pharmaceutical and biotechnology companies. BioSante´s products include LibiGel (transdermal testosterone gel) for the treatment of female sexual dysfunction (FSD), specifically hypoactive sexual desire disorder (HSDD), which is in Phase III clinical development. BioSante also is developing a portfolio of cancer vaccines, with 17 Phase I and Phase II clinical trials currently on-going. Four of these vaccines have been granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA). BioSante´s other products include an FDA-approved testosterone gel for male hypogonadism, which is licensed to Teva Pharmaceuticals (TEVA), and the Pill-Plus, an oral contraceptive in Phase II clinical development by Pantarhei Bioscience B.V. BioSante's first FDA-approved product, Elestrin (estradiol gel) indicated for the treatment of hot flashes associated with menopause, is marketed in the U.S. by Jazz Pharmaceuticals (JAZZ), BioSante's licensee.
Upcoming Phase III catalysts
The ninth unblinded review of safety data from the LibiGel Phase III safety study by the independent Data Monitoring Committee (DMC) is expected to occur during the third quarter 2012. BioSante remains blinded to all safety data in the Phase III safety study.
BioSante is continuing the development of two new LibiGel Phase III efficacy trials, and intends to provide additional trial design information and timing of trial initiation, as appropriate. BioSante intends to apply for an FDA Special Protocol Assessment (SPA) agreement prior to initiating the efficacy trials. It is expected that any new efficacy trials will include the same FDA-required efficacy endpoints as prior Phase III efficacy trials: an increase in the number of satisfying sexual events and sexual desire, and decreased distress associated with low desire. BioSante targets an SPA agreement by year-end.
The company reported the second-quarter financial results on August 1 with the following highlights:
|Net loss||$7.3 million|
|Shares outstanding||$20.2 million|
|Cash per share||$2.1|
The stock is currently trading below its net cash of $2.1 per share. I am not expecting the Phase III safety data due in third quarter 2012 to move the stock above $2.1.
5. Cyclacel (CYCC) is a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious diseases. Sapacitabine oral capsules is in the SEAMLESS Phase III trial being conducted under an SPA with the FDA as front-line treatment of acute myeloid leukemia (AML) in the elderly, Phase II studies for AML, myelodysplastic syndromes (MDS) and solid tumors including lung cancer and in investigator-led studies in a Phase II/III study comparing sapacitabine to low dose cytarabine as front-line treatment of elderly patients with AML or high risk MDS unfit for intensive chemotherapy and a Phase II study in chronic lymphocytic leukemia. Cyclacel's pipeline includes seliciclib oral capsules in Phase II studies for the treatment of lung cancer and nasopharyngeal cancer and in a Phase I trial in combination with sapacitabine. Cyclacel's strategy is to build a diversified biopharmaceutical business focused in hematology and oncology based on a portfolio of commercial products and a development pipeline of novel drug candidates.
Upcoming Phase III catalysts
Paul McBarron, Executive Vice President, Finance and Chief Operating Officer of Cyclacel commented on August 14:
"Cyclacel's focus remains on the successful execution of SEAMLESS, our Phase III study of sapacitabine as front-line treatment in acute myeloid leukemia (AML). With 35 clinical sites open and enrolling in the U.S., we anticipate reaching the second planned periodic safety review in late 2012 or early 2013."
The company reported the second-quarter financial results on August 14 with the following highlights:
|Net loss||$3.6 million|
The company expects that its cash resources are sufficient to meet anticipated working capital needs and fund ongoing sapacitabine clinical trials for at least the next twelve months.
Cyclacel's Goals for 2012
- Continue enrollment in the SEAMLESS pivotal Phase III study of sapacitabine in AML;
- Report updated Phase II sapacitabine data in 2nd line MDS following previous treatment with hypomethylating agents;
- Report updated Phase II sapacitabine data in AML preceded by MDS following previous treatment with hypomethylating agents for the preceding MDS;
- Report updated Phase I sapacitabine and seliciclib combination data in patients with solid tumors;
- Report updated Phase II sapacitabine data in non-small cell lung cancer (NSCLC); and
- Report survival data from the "Pick a Winner/LI-1" investigator-sponsored study in AML and other investigator-sponsored studies as they become available.
I am not expecting the SEAMLESS Phase III data review to move the stock above $1. I would only recommend a small position in the stock currently.