The week ended April 23 did not bring good tidings to the lone company which faced the FDA panel - Acura Pharmaceuticals Inc. (ACUR), as its share price got hammered on thumbs down votes for its investigational pain drug Acurox. But the week did cheer up a few other pharma stocks and here's a list of some of them.
Shares of Melbourne-based Prana Biotechnology Ltd. (PRAN) touched a new intraday high of $3.35 on Monday before closing the day's trading at $2.41, making it to the list of top gainers of that day.
The company is focused on developing disease modifying therapeutics for the treatment of common neurological disorders, particularly Alzheimer's, Parkinson's and Huntington's disease.
On April 19, the authoritative scientific journal, Journal of Alzheimer's Disease, published an article about the company's lead drug candidate for Alzheimer's disease, PBT2, providing new analysis that it is effective in reversing dementia symptoms.
PBT2 has completed two phase I trials and a phase IIa clinical trial in patients with mild Alzheimer's disease. The phase IIa results of PBT2 published in the The Lancet Neurology in July 2008 and July 2009, showed that patients with mild Alzheimer's Disease treated with 250mg of PBT2, experienced an overall statistically significant improvement in executive function on a Neuropsychological Test Battery, or NTB, within 12 weeks of treatment.
Improvements in executive function is strongly related to improvement in daily function and to the quality of the daily life of patients, said Jeffrey Cummings, Director, Alzheimer Disease Research Center, UCLA, and Chairman of Prana's Scientific Advisory Board.
According to Colin Masters of the Mental Health Research Institute and internationally acknowledged leader in Alzheimer's Disease research, the new results are very exciting and Prana's therapeutic strategy stands up as one of the safest and most effective means of treating the disease.
A definitive phase IIb trial of PBT2, which will involve 525 patients with mild to moderate Alzheimer's disease, is expected to be commenced before the end of this year. The company is in discussions with potential sources of finance for the upcoming trial, both with investors and pharmaceutical partners.
The company's preclinical compounds include PBT519, a brain cancer compound and several Parkinson's disease candidates, including PBT434.
Being a development-stage company, Prana has had no sales income to date, and as of December 31, 2009, had an accumulated deficit of A$75.78 million.
PRAN closed Friday's trade at $1.66, down 3.49%, on a volume of 217 thousand shares.
Cadence Pharmaceuticals Inc. (CADX) gained more than 13% on Monday to close the day's trading at $10.45, joining the list of top gainers of that day.
Despite two regulatory setbacks for its investigational pain reliever Ofirmev injection, formerly known as Acetavance, Cadence is planning to resubmit a New Drug Application for the product candidate within the next 30 days.
Cadence acquired the exclusive rights to Ofirmev injection in the United States and Canada in 2006 from Bristol-Myers Squibb Co. (BMY), which markets the product as Perfalgan in Europe and other parts of the world.
Ofirmev is an intravenous formulation of acetaminophen. Acetaminophen is the most widely used medication for the treatment of pain and fever in the United States and is available in more than 600 combination and single-ingredient prescription and over-the-counter products.
Intravenous acetaminophen is approved in approximately 80 countries, including major markets in Europe, where it is the market leader among all injectable analgesics.
Cadence submitted its New Drug Application for Ofirmev injection last May and was granted a priority review with the FDA decision date of November 13, 2009. However, the regulatory agency was unable to decide on Ofirmev within the deadline as it needed more time to review the additional pharmacology data submitted by the company, and extended the decision date to February 2010.
On February 11, 2010, the FDA issued a complete response letter for Ofirmev due to deficiencies at a facility used to manufacture the drug. The facility is operated by Cadence's third party manufacturer. However, the FDA did not cite any safety or efficacy issues, nor did it request any additional studies.
Cadence met with the FDA on April 16 to discuss the deficiencies outlined in the Complete Response Letter for Ofirmev and based on feedback received during the meeting, has planned to resubmit the NDA.
If approved, Ofirmev would become the first new intravenous analgesic approved in the United States in more than 20 years and the only approved intravenous agent for the treatment of fever in the United States.
CADX gained 1.52% on Friday to close the day's trading at $10 on a volume of 117 thousand shares.
Unilife Corp. (UNIS) rose 26% to $7.90 on Tuesday joining the list of top gainers of that day. The stock was featured in the weekly report posted on April 10 , which gives an overview of the company.
UNIS gained more than 4% on Friday to close the day's trading at $7.46 on an above-average volume of 507 thousand shares.
It had been a mixed week for Array Biopharma Inc. (ARRY). On Tuesday, the stock rose over 33% to close the day's trading at $4.02, making it one of the top gainers of that day after the company announced a strategic collaboration with Novartis AG (NVS) for one of its investigational cancer drugs.
The collaboration with Novartis is for worldwide development of Array's small-molecule MEK inhibitors ARRY-162, its back-up, ARRY-300, and other MEK inhibitors. Under the terms of the agreement, Array will initially receive $45 million comprising an upfront and milestone payment and is eligible to receive an additional $422 million if certain clinical, regulatory and commercial milestones are achieved.
On Thursday, Array lost 6% after UBS analyst Jeff Elliott downgraded the stock to neutral from buy noting that the company is fairly valued.
ARRY gained 2.7% on Friday to close the day's trading at $3.81 on an above-average volume of 754 thousand shares.
Shares of Neuralstem Inc. (CUR) have had a nice run this past week and touched a new intraday high of $3 on Friday before closing the day's trading at $2.81.
This bio-therapeutics company is involved in stem cell research to treat diseases of the central nervous system including ischemic spastic paraplegia, traumatic spinal cord injury, huntington's disease and amyotrophic lateral sclerosis , often referred to as lou gehrig's disease.
Last September, the FDA granted approval to NeuralStem to conduct the first human trial using neural stem cells for treatment of amyotrophic lateral sclerosis, or ALS and the first patient was treated in the trial in January of this year. The company has planned to treat a total of up to 18 patients in the first U.S. ALS clinical trial.
ALS is a progressive, fatal neurodegenerative disease that affects nerve cells in the brain, leading to the degeneration and death of the motor neurons in the spinal cord that control muscle movement. ALS affects about 30,000 people in the U.S., with about 7,000 new diagnoses per year. In pre-clinical trials, Neuralstem's cells have shown to extend the life of rats with ALS.
Shares of Akorn Inc. (AKRX) touched a new intraday high of $2.30 on Wednesday following news of FDA approval of the company's supplemental abbreviated new drug application to re-launch 3.5-g tubes of erythromycin ophthalmic ointment.
Akorn is a specialty pharmaceutical company engaged in the development, manufacture and marketing of multi-source and branded pharmaceutical products.
Erythromycin ophthalmic ointment was placed on the FDA's Drug Shortage list last September. According to the FDA site, the shortage was caused by a change in manufacturers. Erythromycin ophthalmic ointment is the only FDA-approved product for prophylaxis of ophthalmia neonatorum, a form of bacterial conjunctivitis, that affects newborn babies.
The product is also approved for the treatment of superficial ocular infections involving the conjunctiva and/or cornea caused by organisms susceptible to erythromycin. According to IMS Health, the annualized U.S. sales for Erythromycin 3.5 g based on fourth quarter 2009 data, were about $26 million.
Last week, the company won FDA approval for the generic version of opioid analgesic Dilaudid-HP 10mg/ml Injection. The company intends to begin shipping the product next month.
Akorn is optimistic about its future prospects and expects to file 20 new ANDAs with the FDA in the next twelve to eighteen months.
Excluding the impact of any 2010 product approvals, Akorn expects core business revenues in 2010 to range between $55 million and $60 million, a 25% to 35% increase over 2009.
AKRX closed Friday's trading at $2.03, down 3.79%, on an above-average volume of 692 thousand shares.
Raptor Pharmaceuticals Corp. (RPTP), which gained more than 25% to close Wednesday's trading at $1.77, was one of the top gainers of that day.
The company has three clinical development programs which it is actively developing
DR Cysteamine for the potential treatment of nephropathic cystinosis, or cystinosis, a rare genetic disorder;
DR Cysteamine for the potential treatment of non-alcoholic steatohepatitis, or NASH, a metabolic disorder of the liver and
DR Cysteamine for the potential treatment of Huntington's Disease, or HD, which has completed a phase I trial.
In November 2009, Raptor completed its phase IIb clinical trial of DR Cysteamine in cystinosis. Early this year, Raptor said that it intends to initiate its phase III clinical trial of DR Cysteamine in cystinosis at multiple sites in the US and Europe upon receiving FDA and EMEA agreements on protocol. The FDA granted orphan drug designation for DR Cysteamine for the treatment of cystinosis in 2006.
Raptor announced positive interim results from a completed phase IIa clinical trial of DR Cysteamine for non-alcoholic steatohepatitis last October. The full results have been submitted for peer review and are expected to be presented this year.
The clinical-stage product candidates for which Raptor is seeking partners are Convivia for the potential treatment of acetaldehyde toxicity resulting from ALDH2 deficiency, Tezampanel and NGX426, non-opioids for the potential treatment of migraine, acute pain, and chronic pain.
Tezampanel has been studied in six phase II trials and has been shown to have analgesic activity. Last November, Raptor presented phase I clinical trial data on NGX426 at the 12th International Conference on the Mechanisms and Treatment of Neuropathic Pain, which suggested that NGX426 could be effective in a variety of neuropathic pain states.
Last December, the company raised $7.5 million in registered direct offering to be used for general corporate purposes, including activities related to further clinical development of DR Cysteamine and for other working capital and operational purposes.
RPTP closed Friday's trading at $1.80, up 1.12%, on an above-average volume of 158 thousand shares.
Medical technology company Delcath Systems Inc. (DCTH) was one of the top gainers on Thursday after the company reported positive results from its late-stage trial of Percutaneous Hepatic Perfusion system on patients with metastatic melanoma and inoperable liver tumors. The stock gained over 19% on Thursday to close the day's trading at $12.93.
Delcath's patented Percutaneous Hepatic Perfusion technology allows physicians to deliver significantly higher doses of anti-cancer drugs to the liver without exposing the patient's entire body to those same potent levels of drug, thereby limiting the toxic side-effects due to chemotherapy treatments.
According to the trial results, patients treated with Delcath's PHP system, survived for an average of 214 days or seven months, before the disease progression, compared to mere 70 days in the control arm, in which patients received Best Alternative Care, or BAC.
The full trial data will be presented on June 5th at the American Society of Clinical Oncology's 2010 Annual Meeting. Delcath expects to initiate its rolling NDA submission to the FDA within the next 30 days.
DCTH closed Friday's trade at $13.12, up 1.47%, on an above-average volume of 2.68 million shares.
Arca biopharma Inc. (ABIO) gained more than 27% on Friday on no news to close the day's trading at $5.28, making its entry in the list of top gainers of the day.
This biopharmaceutical company is developing genetically targeted therapies for heart failure and other cardiovascular diseases.
Last June, the FDA issued a complete response letter for Arca's investigational drug Gencaro, to treat chronic heart failure, and sought additional clinical studies. According to the company, Gencaro, a gene-specific heart failure prevention drug, targets certain genes that other heart medications do not.
After the receipt of the complete response letter, Arca submitted a study protocol for review under the FDA's Special Protocol Assessment, or SPA, process for the design of a clinical trial of Gencaro last December. Subject to outcome of the regulatory agency's review of the SPA submission, the company expects to begin the proposed trial in late 2010 or the first half of 2011. Gencaro also has a fast track designation.
Shares of medical technology company Cardium Therapeutics Inc. (CXM) rose over 17% on Friday to close the day's trading at $0.62.
Cardium has two business units, Cardium Biologics and Tissue Repair Co. Last December, the company filed a 510(k) premarket notification with the FDA seeking marketing clearance of its Excellagen product candidate based on positive data from a phase IIb clinical trial that demonstrated substantial improvements in wound healing responses in patients with non-healing diabetic foot ulcers following one or two applications of Excellagen.
Foot infections are among the most serious complications of diabetes and may even lead to amputation when infections fail to respond to therapy. Statistics reveal that about 600,000 diabetics get foot ulcers every year in the U.S. According to reports, appropriate treatment of foot ulcers may prevent up to 85% of amputations.
Apligraf from privately-held Organogenesis Inc; Dermagraft from Advanced Tissue Sciences Inc. (ATISZ.PK); Johnson & Johnson's (JNJ) Regranex Gel are some of the FDA-approved treatments for diabetic foot ulcers.
In February of this year, Cardium entered into an agreement with bioRASI, an international contract research organization, to get assistance in the evaluation of Generx clinical development opportunities within major newly-industrializing markets in Eastern Europe, Asia and Latin America, for patients with chronic coronary artery disease who are either not optimal candidates for or do not have access to costly angioplasty/stenting or cardiac bypass surgery procedures. The FDA has cleared Generx for a phase III clinical study in the U.S. for women with late stage coronary artery disease.
Being a development-stage company, Cardium has incurred losses since inception in December 2003 and as of December 31, 2009, had an accumulated deficit of about $76.2 million and total cash of $3.4 million.
Bare Facts
According to findings presented recently at the American Association for Cancer Research Annual meeting, patients who had a stomach infection of Helicobacter pylori, a common bacterium, are at risk of developing colon cancer. The American Cancer Society estimates that nearly 150,000 people in the U.S are diagnosed with colorectal cancer each year and about 50,000 die from the disease.
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