Tuesday, September 4, 2012

‘Orphan drug’ used to treat sleep disorders may be a potent cancer-fighting agent used for many malignancies, study finds

An inexpensive "orphan drug" used to treat sleep disorders appears to be a potent inhibitor of cancer cells, according to a new study led by scientists at Fred Hutchinson Cancer Research Center. Their novel approach, using groundbreaking technology that allows rapid analysis of the genome, has broad implications for the development of safer, more-effective cancer therapies.  

A research team led by corresponding author Carla Grandori, M.D., Ph.D., an investigator in the Hutchinson Center's Human Biology Division, used a high-speed robotic technology called high-throughput screening and a powerful genetic technique called siRNA genesilencing to uncover fatal weaknesses in cancer cells driven by an oncogene known as "Myc," which is hyperactive in many cancers, including those of the brain, breast, lung, ovary and liver.


Monday, September 3, 2012

Exelixis reports data from cabozantinib phase 3 trial on MTC

Exelixis, Inc. reported data from the phase 3 pivotal trial of cabozantinib in patients with progressive, unresectable, locally advanced or metastatic medullary thyroid cancer (MTC). The trial, known as EXAM, met its primary endpoint of improving progression-free survival (PFS), with patients in the cabozantinib (see structure) arm achieving a median PFS of 11.2 months compared with 4.0 months for patients in the placebo arm. Overall response rate (ORR), a secondary endpoint, was 28% in the cabozantinib arm and 0% in the placebo arm. Estimated PFS at one year was 47.3% with cabozantinib vs. only 7.2% with placebo. Data for overall survival (OS), another secondary endpoint, are not yet mature. Patients on the cabozantinib arm of the trial received a dose of 140 mg (free base equivalent). Adverse events were generally manageable allowing for treatment with cabozantinib for prolonged periods of time. Exelixis recently submitted a New Drug Application (NDA) for cabozantinib in MTC to the U.S. Food and Drug Administration (FDA).


Exelixis reports data from cabozantinib phase 3 trial on MTC: Exelixis, Inc.  reported data from the phase 3 pivotal trial of cabozantinib in patients with progressive, unresectable, locally advanced or metastatic medullary thyroid cancer (MTC).


Sunday, September 2, 2012

Experimental drug tested against multi-drug resistant TB

"Researchers who tested a novel type of antibiotic against multi-drug-resistant tuberculosis [MDR-TB] are reporting that nearly half of patients who got the new drug cleared the bacteria from their lung fluid in two months," according to a study published  in the New England Journal of Medicine. Japanese pharmaceutical company Otsuka developed the experimental drug, delamanid (see structure), and "also designed and financed the clinical trial, which took place in 17 medical centers across nine countries." 

 "'We've invested a lot of time and money to develop this drug, but we are not seeking robust sales growth immediately,' Masuhiro Yoshitake, Otsuka's head of tuberculosis projects, said in an interview," Bloomberg Businessweek reports. "We want to begin selling to people who know how to use the drug," he added, the news service notes. "Doctors must balance the need to fight hard-to-treat cases against prolonging the medicine's potency,"

More : http://www.nejm.org/doi/full/10.1056/NEJMoa1112433

Experimental drug tested against multi-drug resistant TB

Saturday, September 1, 2012

GSK and XenoPort receive FDA approval for Horizant® for postherpetic neuralgia

GlaxoSmithKline plc  and XenoPort, Inc. announced today that the United States (US) Food and Drug Administration (FDA) has approved Horizant® (gabapentin enacarbil see structure below) Extended-Release Tablets for the management of postherpetic neuralgia (PHN) in adults. 
 The efficacy and safety of Horizant for the management of PHN was evaluated in a single 12-week principal efficacy trial, plus two supportive studies that all met their respective primary endpoints. The three clinical studies involved 574 adult patients from the US, Canada and Germany. 

The recommended dosage for the management of PHN in adults is 600 mg twice daily. Treatment should be initiated at a dose of 600 mg in the morning for three days followed by 600 mg twice daily (1,200 mg/day) beginning on day four. Doses must be adjusted in patients with impaired renal function. In the 12-week, controlled study in patients with PHN, somnolence and dizziness were the most frequently reported side effects. Somnolence was reported in 10% of patients treated with 1,200 mg of Horizant per day compared with 8% of patients receiving placebo. Dizziness was reported in 17% of patients receiving 1,200 mg of Horizant per day compared with 15% of patients receiving placebo.

Friday, August 31, 2012

Combination [of Vismodegib (GDC-0449) and Gemcitabine] therapy may help defeat pancreatic cancer

 In continuation of my update on (GDC-0449) Visodegib and Gemcitabine
GDC-0449 targets the Smoothened (SMO) protein in the Hedgehog signaling pathway. It was approved for use in basal cell carcinoma and is marketed as vismodegib. Kim and his colleagues felt that treating patients with pancreatic cancer first with GDC-0449 and then with the standard chemotherapeutic drug gemcitabine might disrupt the desmoplastic stroma and improve the efficacy of the chemotherapy. 

They evaluated this strategy in treatment-naive patients with advanced pancreatic cancer. Patients underwent needle biopsies of the cancer before and after taking GDC-0449 for three weeks to study the effects of GDC-0449 on the Hedgehog pathway signals, tumor stroma and pancreatic cancer stem cells. Gemcitabine was added to GDC-0449 following the second biopsy.

Thursday, August 30, 2012

Keryx Biopharmaceuticals Announces Top-Line Data from the Perifosine (KRX-0401) X-PECT Phase 3 Clinical Trial

Keryx Biopharmaceuticals, Inc. reported today that the Phase 3 "X-PECT" (Xeloda® + Perifosine Evaluation in Colorectal cancer Treatment) clinical trial evaluating perifosine (KRX-0401) + capecitabine (Xeloda) in patients with refractory advanced colorectal cancer did not meet the primary endpoint of improving overall survival versus capecitabine + placebo.

This Phase 3 trial was conducted pursuant to a Special Protocol Assessment (SPA) agreement with the FDA.  468 patients at sixty-five U.S. sites participated in this study. 

Ron Bentsur, Chief Executive Officer of Keryx, stated, "We are all extremely disappointed with the results of the study.  We thank the investigators who participated in what we believe was a well-run study, despite the outcome.  We will evaluate whether our Phase 3 study of Perifosine in relapsed/refractory multiple myeloma will continue as planned."
Mr. Bentsur commented further, "With approximately $31 million in cash as of March 31, 2012, and a well-controlled burn rate, we plan to focus our resources on the pending completion of the Zerenex (ferric citrate) long-term Phase 3 study for end stage renal disease (ESRD) patients with hyperphosphatemia, expected in the fourth quarter of 2012, and the New Drug Application (NDA) filing for Zerenex which will hopefully follow shortly thereafter."

KRX-0401 (perifosine) is in-licensed by Keryx from AeternaZentaris Inc. in the United States, Canada and Mexico.

Ref : http://investors.keryx.com/phoenix.zhtml?c=122201&p=irol-newsArticle&ID=1678920&highlight=

Wednesday, August 29, 2012

Cancer publishes Aeterna Zentaris' perifosine Phase 2 trials in RCC

RESULTS:

In the Perifosine 228 trial, 1 patient achieved a partial response (objective response rate, 4%; 95% confidence interval, 0.7%-20%), and 11 patients (46%) had stable disease as their best response. The median progression-free survival was 14.2 weeks (95% confidence interval, 7.7-21.6 weeks). In the Perifosine 231 trial, 5 patients achieved a partial response (objective response rate, 10%; 95% confidence interval, 4.5%-22.2%) and 16 patients (32%) had stable disease as their best response. The median progression-free survival was 14 weeks (95% confidence interval, 12.9, 20.7 weeks). Overall, perifosine was well tolerated, and there were very few grade 3 and 4 events. The most common toxicities included nausea, diarrhea, musculoskeletal pain, and fatigue.

CONCLUSIONS:

Although perifosine demonstrated activity in patients with advanced RCC after failure on VEGF-targeted therapy, its activity was not superior to currently available second-line agents. Nonetheless, perifosine may be worthy of further study in RCC in combination with other currently available therapies. 


Ref : http://onlinelibrary.wiley.com/doi/10.1002/cncr.27668/abstract

 Cancer publishes Aeterna Zentaris' perifosine Phase 2 trials in RCC//

Tuesday, August 28, 2012

Drug shows promise as skin cancer treatment

In continuation of my update on Vismodegib

Drug shows promise as skin cancer treatment: A new vismodegibdrug for a type of skin cancer caused by a rare genetic disease can not only substantially shrink the tumors, but can also prevent the growth of new cancers, US investigators have discovered.

Monday, August 27, 2012

Health Canada approves Sunovion’s LATUDA NDA to treat schizophrenia

In continuation of my update on Lurasidone..

Health Canada approves Sunovion’s LATUDA NDA to treat schizophrenia: Sunovion Pharmaceuticals Canada Inc. today announced that the New Drug Submission (NDS) for LATUDA (lurasidone HCl), for the treatment of adult patients with acute schizophrenia has been approved by Health Canada.

Sunday, August 26, 2012

Insomnia Drug Closer to Approval | News | Drug Discovery and Development Magazine

Merck & Co. said that its experimental insomnia drug suvorexant (see structure) helped patients fall asleep faster and stay asleep longer in two late-stage tests of the drug, seen as a potential blockbuster in a multibillion-dollar market. 

Merck said the drug worked better than a placebo at measurements including total sleep time, time to falling asleep, and continuous sleep after one month and three months of treatment. The company said patients reported better results on suvorexant compared with placebo, and their sleeping habits also were measured electronically.

Suvorexant is a new type of insomnia drug designed to help patients sleep while minimizing morning grogginess. It is one of Merck's major drug candidates. The company plans to file for U.S. marketing approval this year, and it is one of six planned product filings for Merck in 2012 and 2013.

The two trials involved more than 2,000 patients who had insomnia that was not caused by another medical problem. The most common side effects of suvorexant were tiredness and headache.
 

Saturday, August 25, 2012

Two Possible Treatments for Bipolar Disorder Found

Researchers at the University of Leeds investigating the genetic causes of bipolar disorder have identified two new drugs – one of which has already been found safe in clinical trials – that may be effective in treating the disorder.

Bipolar disorder is characterised by mood swings between mania and depression. Like autism, it is thought to be a spectrum of disorders and, although its causes are not well understood, it seems to run in families and is thought to be caused by both genetic and environmental factors.

Dr Steve Clapcote, of the Institute of Membrane and Systems Biology at the University of Leeds, who led the study, says: "We suspected from published studies of bipolar patients that levels of enzymes known as NKA or sodium pumps may be abnormal in bipolar disorder, but so far the evidence has not been convincing enough to warrant detailed clinical investigations."

The research, published today in the US journal Proceedings of the National Academy of Sciences (PNAS), used a strain of genetically modified mice that exhibit symptoms very similar to humans in the manic phase of the disorder.
The mice were bred with a particular mutation that prevents the NKA enzyme from functioning normally. When tested, the mice showed characteristics closely associated with bipolar disorder, such as increased tendency to take risks, hyperactivity, and disturbed sleep patterns. They also exhibited reduced mania when treated with anti-manic drugs.

Current drugs available to treat bipolar disorders, although usually successful, are limited to either Lithium or Valproate. They can't be matched to specific types of bipolar disorder, and can sometimes cause unpleasant side effects. There is therefore a need for treatments which can be better targeted, and which are more effective and better tolerated by patients.

The Leeds researchers found that the mice showed decreased activity of the NKA enzyme, as well as increased activity of a protein called ERK. Drugs known to have an effect on these two elements were administered to the mice, including Rostafuroxin and SL327 (see structure right), and both reduced their mania-like behaviour.

"Rostafuroxin (see structure left) has been found to be safe in clinical trials for treating high blood pressure," explained Dr Clapcote. "No one has previously looked at this drug's effects on the brain, but our mouse studies show there's a possibility that it might also be suitable for people with mania. Similarly, SL327, which is known to inhibit ERK activity, was also found to reduce manic behaviour in the mice."


"We think there is enough evidence now to start screening people with bipolar disorder to look for genetic mutations in the same NKA enzyme as that affected in our mice," says Dr Clapcote. "This will help us identify whether there is a group of bipolar patients that may be responsive to the novel treatments we have tested in the mice."....

Friday, August 24, 2012

Reformulated Copaxone Meets Goals........

Teva Pharmaceutical Industries Ltd. said that a new version of its multiple sclerosis drug Copaxone met its goals in a late-stage clinical trial.....

We know that, COPAXONE is the brand name for glatiramer acetate (formerly known as copolymer-1). Glatiramer acetate, the active ingredient of COPAXONE (glatiramer acetate) , consists of the acetate salts of synthetic polypeptides, containing four naturally occurring amino acids: L-glutamic acid, L-alanine, L-tyrosine, and L-lysine with an average molar fraction of 0.141, 0.427, 0.095, and 0.338, respectively. The average molecular weight of glatiramer acetate is 5,000 – 9,000 daltons. Glatiramer acetate is identified by specific antibodies.

Chemically, glatiramer acetate is designated L-glutamic acid polymer with L-alanine, L-lysine and L-tyrosine, acetate (salt). Its structural formula is:

(Glu, Ala, Lys, Tyr)x•xCH3COOH
(C5H9NO4•C3H7NO2•C6H14N2O2•C9H11NO3)x•xC2H4O2
CAS - 147245-92-9

COPAXONE (glatiramer acetate) is a clear, colorless to slightly yellow, sterile, nonpyrogenic solution for subcutaneous injection. Each 1 mL of solution contains 20 mg of glatiramer acetate and 40 mg of mannitol. The pH range of the solution is approximately 5.5 to 7.0. The  biological activity of COPAXONE (glatiramer acetate) is determined by its ability to block the induction of experimental autoimmune encephalomyelitis (EAE) in mice.


Thursday, August 23, 2012

New action for ancient heart drug


Since the 13th century, the herb and poisonous plant Foxglove has been used to cleanse wounds and its dried leaves were carefully brewed by Native Americans to treat leg swelling caused by heart problems. Digoxin (see structure), the active ingredient in digitalis, or the poisonous plant Foxglove, can enhance the body's own protective mechanism against high blood pressure and heart failure.
 
High blood pressure can be prevented by reducing salt intake, being active and keeping a healthy weight, but about 1 in 3 Americans has high blood pressure, also called hypertension, which can damage the body in many ways.  Most current treatments prevent excess hormone and stress signals that can lead to high blood pressure and heart failure.

But recent studies have found that the body has the ability to keep excess stimulation in check through production of a family of inhibitors called RGS proteins. Researchers looked for ways to "re-purpose" old drugs to tap into this protective mechanism which is lost among some individuals with high blood pressure and heart failure. 

Ref; http://www.uofmhealth.org/news/digoxin-0613


Tuesday, August 21, 2012

Promising preliminary data for axitinib in metastatic kidney cancer

In continuation of my update on Axitinib

Promising preliminary data for axitinib in metastatic kidney cancer: Preliminary study data show that axitinib may be an effective first-line treatment for metastatic renal cell carcinoma, particularly in patients with high therapeutic drug exposure and a rise in blood pressure during the first 2 weeks of treatment, researchers report.

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