Nymox Pharma's Update On Phase 3 Study Of Benign Prostatic Hyperplasia Drug

Nymox Pharmaceutical Corp., reported new positive results from a long term outcome study of its lead drug candidate to treat benign prostatic hyperplasia, NX-1207. The study, a part of the Phase 3 trial of the drug, evaluated symptomatic progress of U.S. patients involved in the Phase 1 and Phase 2 studies, initially undertaken in 2003. The inclusion criterion was all subjects enrolled in the studies had previously failed conventional drug treatments.

Benign prostatic hyperplasia or BHP is a common affliction of older men, affecting approximately half of men over age 50 and close to 90% of men by age 80, and is associated with growth in prostate size as men age. The condition causes difficulties with urination associated with aging, such as urination at night, urge to void frequently, hesitancy, weak stream, and other problems. The treatment represents a growing market with more than 100 million men worldwide estimated to suffer from BPH symptoms.

Patients treated with NX-1207 were followed-up on an unselected and as available basis and assessed for symptomatic improvement, treatment outcomes, and durability of efficacy 61/2 years after a single treatment with the drug. The data was available for 69% of the patients from the initial studies.

Overall, 55% of the men in the new outcome study reported no subsequent surgical treatment and no current drug treatment for their prostatic hyperplasia or PH and had a mean improvement of 14.3 points in AUA Symptom Score. In addition, 36% of the patients reported no other approved treatments at any time for their BPH since their original treatment with NX-1207, with a mean improvement of 14.5 points.

The sustained improvement in BPH symptom score after treatment with NX-1207 compares favorably with the 3 to 5 points reported in published studies of currently approved BPH drugs, which, unlike NX-1207 treatment, require permanent daily administration to be effective. Currently approved drugs also have undesirable side effects such as loss of libido, impotence, retrograde ejaculation, dizziness, and other problems.

Follow-up trials have also confirmed the excellent safety and side effect profile of the therapy and evidence of enduring benefit for a significant percentage of patients.

On March 30, Nymox had outlined the development program for the drug, currently in Phase 3 trials in the U.S. and had added that positive results from animal studies have shown the potential for the use of high dosages of NX-1207 for the focal treatment of clinically localized prostate cancer and of primary liver cancer.

Paul Averback MD, CEO of Nymox had said that the clinical trials of the drug in humans to date have demonstrated an excellent safety profile with no known serious drug side effects. This favorable safety data will be an advantage in the clinical trials for the treatment of prostate cancer and liver cancer. He had also added that there is a serious unmet need for a safer option for the treatment of localized prostate cancer without causing impotence or other sexual side effects and that the company is looking forward to advancing NX-1207 to treat primary liver cancer.

read more» Read more...

Qnexa weight loss drug lowers blood pressure

An experimental weight loss drug that pairs a stimulant with an epilepsy drug helped patients lose weight and keep it off for a year and also lowered blood pressure, a researcher said on Tuesday.

A consultant to Mountain View, California-based Vivus Inc said three separate studies showed patients lost more weight when they took the highest doses of Qnexa, which is up for Food and Drug Administration approval in July.

Shares in Vivus soared 13.6 percent to $11.73 a share in early afternoon trading.

Drops in blood pressure were small but clear and equated with the weight loss, said Dr. Suzanne Oparil of the University of Alabama at Birmingham.

"The weight loss is very impressive, and it is good that there is concomitant reduction in blood pressure," Oparil said in a telephone interview from a meeting of the American Society of Hypertension in New York.

Oparil, a past president of the American Heart Association, pooled and analyzed the results of three separate studies of Qnexa in nearly 8,000 overweight and obese patients.

"At week 58 there is a nice weight reduction," she said. The higher the dose, the more weight loss and the more blood pressure went down, she said.

The patients who got the highest dose along with diet and exercise advice lost about 10 percent of their body weight on average, she said.

Vivus is trying to improve on the notorious "fen-phen" combination pulled off the market in 1997 after it was found to damage the heart and cause sometimes fatal cases of pulmonary hypertension.

Improving Health

Fen-phen combined fenfluramine and phentermine. Phentermine, a stimulant now available generically, appears safe and is used at low doses in Qnexa along with the epilepsy drug topiramate, available generically and sold by Johnson & Johnson's Ortho-McNeil unit as Topamax.

Losing just 10 percent of body weight is enough to lower cholesterol and blood pressure, reduce the risk of diabetes and early death. About 68 percent of U.S. adults are overweight or obese.

In the three studies that Oparil analyzed, about 80 percent of the patients were women, most morbidly obese with weights of 220 to 260 pounds (100 to 118 kg).

Most did not have high blood pressure but among those who did and who took the highest Qnexa dose, the systolic or top number in the blood pressure reading went down an average of 9 points, compared to 4 points for the women who got placebos.

Oparil said the most common side effects were tingling and dry mouth, each seen in 19 percent of the patients who took the highest dosages.

Topiramate acts on the nervous system and tends to act as a sedative, said Oparil. This can reduce appetite. In addition, patients said the drug made carbonated drinks taste metallic and that may have helped patients avoid sugary soft drinks.

Doses of both drugs are far lower than those usually used -- when phentermine is used alone to treat obesity, for example, or when topiramate is used for epilepsy, she said.

The FDA's Endocrinologic and Metabolic Drugs Advisory Committee is tentatively scheduled to review Vivus' Qnexa on July 15, the company said.

read more» Read more...

NDA Submitted for Azilsartan Medoxomil

Takeda Pharmaceutical Company Limited (Takeda) announced today that Takeda Global Research & Development Center, Inc., U.S., submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for azilsartan medoxomil , an angiotensin II receptor blocker (ARB) under investigation for the treatment of hypertension. Discovered by Takeda, azilsartan medoxomil is designed to lower blood pressure by blocking the action of a vasopressor hormone, angiotensin II.

"The NDA submission for azilsartan medoxomil is built upon a robust data package and is a significant milestone for Takeda," said Nancy Joseph-Ridge, M.D., general manager of Takeda's Pharmaceutical Development Division. "We are proud to build upon our global expertise in the cardiovascular therapeutic area with this filing, and believe this compound, once approved, will provide an important treatment option for hypertensive patients and the health care providers who manage them."

The NDA submission was supported by seven phase 3 clinical trials involving more than 5,900 patients. The safety and efficacy of azilsartan medoxomil was studied for initial therapy as a once-daily oral monotherapy or in combination with other antihypertensive medications, including chlorthalidone and amlodipine, and was also studied in comparison to olmesartan medoxomil, valsartan and ramipril. Results from five of the pivotal phase 3 studies will be presented May 1-4, 2010, at the American Society of Hypertension Inc. (ASH), 25th Annual Scientific Meeting and Exposition in New York.

About Azilsartan Medoxomil

Discovered by Takeda, azilsartan medoxomil, also known as TAK-491, is an angiotensin II receptor blocker currently in development for the treatment of hypertension, or high blood pressure, either used alone or in combination with other classes of antihypertensive agents. Angiotensin II, a vasopressor, is a hormone that naturally exists within the body and plays a key role in cardiovascular function. The hormone induces contraction, or tightening, of blood vessels and thus plays an important role in mediating hypertension. The most commonly reported treatment-related adverse reactions (>/=1%) in phase 3 clinical trials were dizziness (2.1%), increased blood creatine phosphokinase (1.1%) and diarrhea (1.0%).

read more» Read more...

Transplant drug 2-year study outcomes show superior kidney function

Two-year results from phase III clinical trials show the experimental immunosuppressive drug belatacept can better preserve kidney function in kidney transplant recipients while preventing graft rejection when compared with the standard immunosuppressive drug cyclosporine.

The two-year results from the three-year BENEFIT (Belatacept Evaluation of Nephroprotection and Efficacy as First-line Immunosuppression Trial) and BENEFIT-EXT ("extended criteria") studies were presented Sunday at the American Transplant Congress in San Diego. A safety study that pooled long- term data also was presented.

In the BENEFIT trial, 666 patients were randomized to three groups and transplanted at 100 sites around the world, with 493 completing two years on treatment. In the BENEFIT-EXT study, 543 patients were randomized and transplanted, with 347 completing two years on treatment. The three treatment groups were less intensive (LI) and more intensive (MI) belatacept and a standard regimen of cyclosporine (CsA). All patients also received standard transplant regimens of the anti-T cell antibody basiliximab and drugs mycophenolate mofetil and corticosteroids.

Patient and graft survival after two years was similar among the belatacept and cyclosporine groups (94 percent MI; 95 percent LI; 91 percent CsA). The superior renal benefit of belatacept found after the first year of treatment was sustained in the second year, as measured by glomerular filtration rate. The improvement in cardiovascular/metabolic risk profile with belatacept remained in year two, with an additional beneficial effect noted in LDL cholesterol. Eight additional patients experienced an episode of acute rejection in year two (four with belatacept, four with cyclosporine), but in most cases this was successfully treated with drugs and did not lead to graft failure.

The overall incidence rate of malignancies and serious infections remained comparable across the groups. Although in the second year there remained a higher incidence of post-transplant lymphoproliferative disorder (PTLD)–five belatacept patients vs. one cyclosporine patient–the overall safety profile remained similar across the groups. No additional benefits were seen in the MI vs. the LI belatacept group.

"Our goal in transplantation is to achieve a normal life span for our patients, and to have them survive dialysis-free with a functioning transplanted organ for that life span," says Christian P. Larsen, MD, DPhil, director of the Emory Transplant Center and chair of the Department of Surgery in Emory University School of Medicine.

"Today, the median survival of a transplant remains about 8-10 years, far short of what we'd like," Larsen adds. "While the calcineurin inhibitors, cyclosporine and tacrolimus, are potent immunosuppressant drugs, they are associated with multiple toxicities that limit transplant success. We have been working for years to develop new therapies that avoid the main complications and causes of death, including cardiovascular events, infections and malignancies. Our data with belatacept indicate it can better preserve kidney function while improving the risk for these complications."

Larsen, along with fellow Emory University transplant surgeon and researcher Thomas C. Pearson, MD, DPhil, Emory professor of surgery and co-director of the kidney/pancreas transplant program at the Emory Transplant Center, made significant research contributions to the development of belatacept, in collaboration with other investigators at Emory, the Yerkes National Primate Research Center, and Bristol Myers Squibb.

A third study, which pooled safety data from phase II and phase III studies over 2.4 to 7 years, found that longer-term treatment with belatacept-based regimens was generally safe. The incidence of deaths and serious adverse events were lowest in the belatacept LI group. The overall incidence of malignancies remained low, but was slightly higher in the MI group. The incidence of herpes infections and tuberculosis (mostly in endemic areas) was low overall, but higher in the belatacept groups. Fifteen cases of PTLD occurred (13 with belatacept, 2 with cyclosporine), mainly in patients not previously exposed to Epstein-Barr virus, which many humans have as a low-level chronic infection. The researchers say PTLD might be reduced by avoiding use of belatacept in Epstein-Barr-naïve patients.

Belatacept is a "costimulation blocker" that inhibits one of two signals T cells require to trigger an immune response. It is a modified version of a fusion protein known as CTLA4-Ig, which mimics a regulatory molecule found on T cells and acts as a decoy. CTLA4-Ig (commercial name: abatecept/Orencis) is FDA approved to treat rheumatoid arthritis.

read more» Read more...

FDA aprroves a new pain drug

Shares of Pozen Inc. surged more than 21% in the after hours trading session Friday after the U.S. Food and Drug Administration or FDA approved VIMOVO delayed-release tablets for the relief of signs and symptoms of osteoarthritis, rheumatoid arthritis, and ankylosing spondylitis, and to decrease the risk of developing gastric ulcers in patients at risk of developing NSAID-associated gastric ulcers.

VIMOVO, co-developed by Pozen and UK-based drug maker AstraZeneca Plc is a fixed-dose combination of enteric-coated naproxen, a pain-relieving non-steroidal anti-inflammatory drug or NSAID, and immediate-release esomeprazole, a proton pump inhibitor or PPI.

The FDA approval was supported by data from a clinical development program, including results from the pivotal PN400-301 and PN400-302 studies, which showed patients taking VIMOVO experienced significantly fewer endoscopic gastric ulcers, compared to patients receiving enteric-coated naproxen.

Nearly 27 million US residents and 140 million people worldwide suffer from osteoarthritis, which is the most common form of arthritis. According to the company, half of osteoarthritis patients on chronic non-steroidal anti-inflammatory drugs therapy are at the risk of developing gastric ulcers associated with it.

Howard Hutchinson, M.D., Chief Medical Officer, AstraZeneca, said, “In a single pill, VIMOVO provides a proven pain reliever with a built-in PPI for arthritis patients at-risk for NSAID-associated gastric ulcers.”

In the PN400-301 and 302 studies, the primary end point was the cumulative incidence of gastric ulcers through six months. Patients received either VIMOVO or enteric-coated naproxen 500 mg, twice daily, in each of the trial, over a six-month treatment period.

Data from study PN400-301 showed a 4.1% incidence of gastric ulcers in patients taking VIMOVO, compared to 23.1% among patients taking enteric-coated naproxen. Study PN400-302 showed a 7.1% incidence of gastric ulcers among patients taking VIMOVO, compared to 24.3% with enteric-coated naproxen.

read more» Read more...

Promise Seen in Drug for Fragile X Syndrome

An experimental drug succeeded in a small clinical trial in bringing about what the researchers called substantial improvements in the behaviors associated with retardation and autism in people with fragile X syndrome, the most common inherited cause of these mental disabilities.

The surprising results, disclosed in an interview this week by Novartis, the Swiss pharmaceutical giant that makes the drug, grew out of three decades of painstaking genetic research, leaps in the understanding of how the brain works, the advocacy of families who refused to give up, and a chance meeting between two scientists who mistakenly showed up at the same conference.

“Just three years ago, I would have said that mental retardation is a disability needing rehab, not a disorder needing medication,” said Dr. Thomas R. Insel, director of the National Institute of Mental Health, who was told of the Novartis trial results. “Any positive results from clinical trials will be amazingly hopeful.”

Dr. Mark C. Fishman, president of the Novartis Institutes for BioMedical Research, cautioned against too much optimism. The trial involved only a few dozen patients, only some of whom benefited from treatment. The drug is likely to be years away from being commercially available and could fail in further clinical trials, he said.

“We have been reluctant to make this public because we still need to do more experiments, do them correctly and in a bigger way,” Dr. Fishman said. “But our group feels pretty good about the data.”

If authenticated in further, larger trials, the results could also become a landmark in the field of autism research, since scientists speculated that the drug may help some patients with autism not caused by fragile X, perhaps becoming the first medicine to address autism’s core symptoms.

One child in five thousand is born with fragile X syndrome, with mental effects ranging from mild learning disabilities to retardation so profound that sufferers do not speak, and physical effects that include elongated faces, prominent jaws, big ears, and enlarged testes. It mostly affects boys and earned its name because, under a microscope, one arm of the X chromosome seems nearly broken, with part hanging by a thread.

The gene for fragile X was discovered in 1991. Work since then has found that fragile X patients seem to experience an overload of unchecked synaptic noise — synapses being the junctions between brain neurons. The Novartis drug and others like it are intended to lower the volume of this noise so memory formation and high-level thinking can take place, allowing children to develop normally.

The Novartis trial, which began in 2008 in Europe with data analysis completed this year, was too brief to observe effects on basic intelligence. Instead, researchers measured a range of aberrant behaviors like hyperactivity, repetitive motions, social withdrawal and inappropriate speech. They gave one set of patients the drug and another a placebo, and after a few weeks switched treatments, with both doctors and patients unaware of which pill was which.

The results of the trial were something of a jumble until Novartis scientists noticed that patients who had a particular, undisclosed biological trait improved far more than others. “The bottom line is that we showed clear improvements in behavior,” Dr. Fishman said.

Told of the results, two parents of a fragile X patient were euphoric.

“This is what we have been working for and hoping for since our son was diagnosed with fragile X 17 years ago,” said Katie Clapp, president and co-founder of the Fraxa Research Foundation, a nonprofit organization dedicated to financing fragile X research. “This may be the key to solving the mystery of autism and other developmental disorders.”

Geraldine Dawson, chief science officer at Autism Speaks, the world’s largest autism advocacy organization, said that a growing body of research suggests that the many genetic causes of autism all seem to affect synapses, suggesting that a treatment for one form of the disease might help others.

“The exciting thing about these results is that it is our hope that these same medications may have similar positive benefits for people with autism who don’t have fragile X syndrome,” Dr. Dawson said.

Between 10 percent and 15 percent of autism cases result from fragile X syndrome or some other known genetic defect. While fragile X is the most common inherited cause of mental retardation, Down syndrome — which also causes retardation — is more common but is not inherited.

The Novartis trial results were not published or peer reviewed, and for commercial reasons Dr. Fishman refused to divulge many details. Dr. Luca Santarelli, head of neuroscience at Roche, confirmed that Roche is in the midst of testing a similar medicine in fragile X patients at four sites in the United States.

“So far we like what we see,” Dr. Santarelli said in his only characterization of their study.

One reason for the euphoria surrounding the Novartis trial is that it was seen as an especially difficult test of the drug’s effects. For ethical reasons, Novartis tested the drug only in adults. But the company and outside researchers believe that such compounds may prove most effective in young children, whose brains are far more likely to respond rapidly when barriers to learning are removed.

“This is perhaps the most promising therapeutic discovery ever for a gene-based behavioral disease,” said Dr. Edward M. Scolnick, former research chief at Merck and now director of the Stanley Center for Psychiatric Research at the Broad Institute at Harvard and the Massachusetts Institute of Technology.

Dr. Scolnick has not seen the results of the Novartis trial, but was told of them and concluded that if the drugs work in fragile X, “there’s nothing to say that they won’t work in some cases of broader autism-spectrum disorders.”

An Unlikely Beginning

The roots for the Novartis results began in 1982 when Stephen T. Warren, then a graduate student in genetics at Michigan State University, was looking for a job and something to research. A friend told him about fragile X and, with the same reflection he might use to pick a novel for a long flight, he decided that he wanted to find the gene that caused it.

“I had no idea how hard this would be,” Dr. Warren said. Nine years later, Dr. Warren, then at Emory University, was part of an international team that won a fierce competition by isolating the gene. The discovery was front-page news around the world, and experts predicted that widespread fetal testing and therapies were in the offing.

The predictions were premature because, like most of genetic research, discovering how the flawed gene caused disease was far harder than anticipated and required multiple leaps in neurology and biology. And even with those, much remains mysterious.

Fragile X is caused by a genetic stutter in which a portion of the gene gets repeated like a scratched album. With each subsequent generation, the number of repeats tends to rise. So if a mother has 10 repeats, her child might have 11 or 12. For reasons that are not well understood, however, this process of repeat amplification can suddenly go haywire. So mothers who have 55 or more repeats tend to have children with hundreds.

In anyone with 200 or more repeats, the body shuts off the gene. Since genes are used to make proteins, this genetic silencing means the encoded protein is never made. The absence of this protein in cells causes the wide-ranging effects of fragile X syndrome. Those with 55 to 200 repeats are considered carriers, and recent research shows they can have severe neurological declines late in life that mimic Alzheimer’s and Parkinson’s.

Many geneticists would have moved on to other research topics after finding a disorder’s underlying gene. But Dr. Warren met affected children and their parents. Instead of family pictures, Dr. Warren’s desk displays a framed photo of a fragile X chromosome.

“I could not imagine telling someone like Katie Clapp that we were not going to pursue this research anymore,” he said.

So he kept on. Years of work by him and others found that the protein missing in those with fragile X normally seems to act as a sort of traffic cop at brain synapses, helping to stop or slow brain signaling at crucial intervals. It does this by sopping up the genetic instructions needed to produce proteins that encourage brain signaling. Regulating this flow of electronic pulses across the brain is crucial for the brain’s ability to learn and mature.

Dr. Warren was puzzling over how to recreate that synaptic traffic cop when, because of a scheduling conflict, he showed up in 2001 at the wrong scientific conference and happened to sit next to Mark F. Bear, a neuroscience professor at M.I.T. who had just given a presentation about compounds that seemed to work in synapses to speed the creation of proteins — including the one missing in fragile X patients.

The two got to talking and decided to collaborate. They found that if Dr. Bear reverse-engineered his compounds, they seemed to slow brain transmissions. Instead of a traffic cop, the brain would get speed bumps. Not ideal, but perhaps adequate in lowering the synaptic noise enough to encourage learning and the moderation of the kind of synaptic traffic jams that in fragile X children can lead to seizures.

Sure enough, mice, fish and fruit flies that through genetic engineering were made to have fragile X seemed to become normal when given Dr. Bear’s compound. The Novartis compound is a member of the same drug family.

“We have been promising for a long time that unlocking the molecular basis for hereditary diseases would lead to dramatic therapeutic advances, and that promise is finally coming true,” said Dr. Francis S. Collins, director of the National Institutes of Health, in discussing the science leading up to the trial. “But it has not been easy.”

A Search for Treatment

A hundred years ago, Katie Clapp would have died giving birth to Andy, her child with fragile X.

“Andy’s head was too big to get out without a C-section, he would have killed me, and that would have taken care of the fragile X gene,” she said.

But Ms. Clapp and Andy did survive. And despite going to some of the best hospitals in the country, four years would pass before Andy’s condition was properly diagnosed.

When a doctor finally thought to do a fragile X test, Ms. Clapp and her husband, Dr. Michael Tranfaglia — both Harvard graduates with post-graduate degrees — researched the disease and came to two conclusions: fragile X was potentially treatable; and only about five researchers in the world were working toward a cure.

“And I thought, what if all five walk across the street at the same time and get hit by a Mack truck?” Ms. Clapp said. “That is not going to get us there.”

So the two started the Fraxa Research Foundation. Remarkably, their efforts seem to be paying off and may finally offer hope not only to those who with fragile X but to carriers like Andy’s sister, Laura.

“I’ve always known my kids have a chance of having it,” Laura, 18, said in a recent visit to the family’s house. “But I’m not going to have kids for at least 10 years anyway, and they’ll have a cure for by then.”

She paused, looked at her mother and said: “You’ve got 10 years.”

read more» Read more...

A new drug target in lung cancer

Lung cancer patients whose tumors over-express a cell surface molecule called CXCR4 do significantly worse than those who do not, Canadian researchers have found. Their work, reported at the 2nd European Lung Cancer Conference in Geneva, highlights the exciting possibility that the molecule could soon become a new target for personalized cancer therapy.

CXCR4 is a receptor that is found on the surface of many different cell types in the body. It plays a role in immune system signaling between cells.

In cancer, evidence that CXCR4 is involved in the growth of tumors and their spreading throughout the body (metastasis) has been growing in recent years. For example, researchers have shown in studies in mice that blocking the action of CXCR4 inhibits metastasis. But its precise role in determining outcome and metastatic tendency, especially in lung cancer, is incompletely investigated.

Dr Gwyn Bebb and colleagues from the Tom Baker Cancer Centre in Calgary, Canada set out to explore whether patients whose tumors expressed high levels of the receptor had a worse prognosis than other lung cancer patients.

They studied tumor samples from 103 patients from the Glans-Look lung cancer database who were diagnosed with stage IV non-small-cell lung cancer (cancer which had already spread to other parts of the body) between 2003 and 2006. They found that 10.7% of the tumors over-expressed CXCR4. Those over-expressers had a significantly worse clinical outcome, with a median overall survival of 2.7 months, compared to 6.1 months among low-expressers.

If confirmed in an expanded series of 170 patients from the Glans-Look database, these results will suggest that new strategies to block CXCR4 should be tested in patients whose cancers over-express the molecule, the researchers say.

"I am quite excited about the possibility of using CXCR4 as a therapeutic target, but we need to learn more about its role in each specific malignancy," Dr Bebb said.

This possibility is especially promising because CXCR4 has been well studied in the context of HIV, where it is known to be a portal for the virus's entry into immune system cells. Drugs that block CXCR4 have already been developed for HIV/AIDS patients, and Dr Bebb's group thinks these drugs could quickly and easily be tested in the cancer setting.

"This is an exciting possibility," Dr Bebb said. "It seems very likely that a better understanding of the role of CXCR4 in lung cancer will lead to new treatment strategies and might allow us to meaningfully improve treatment for some lung cancer patients in the very near future."

read more» Read more...

Santaris Plans Phase II Trial of miRNA-Targeting HCV Drug

Santaris Pharma is close to completing a second phase I study of its microRNA-targeting hepatitis C therapy SPC 3649, and expects to move the drug into phase II development later this year, a company official told RNAi News.

The official, CSO Henrik Orum, also said Santaris has dropped its near-term plan to seek a partner for the therapy, which it will instead carry on its own at least through the first phase II trial in HCV-infected patients

SPC3649 targets miR-122, the most abundantly expressed miRNA in the liver and one linked to cholesterol regulation and lipid metabolism. It is based on Santaris' locked nucleic acid technology, which comprises nucleic acid analogs in which the ribose ring is locked by a methylene bridge connecting the 2'-O atom with the 4'-O atom.

In mid-2008, Santaris moved the drug into a phase I study, making it the first to test a miRNA-targeting drug in man. According to Orum, that single ascending dose trial enrolled 64 healthy volunteers and initially examined doses of the drug up to 6.4 mg/kg.

"With the initial top dose of 6.4 [mg/kg], we didn't see anything in terms of the safety issues," he said. As such, the company amended the trial to include 9 and 12 mg/kg doses.

In addition to observing no significant adverse events associated with SPC3649, Santaris saw evidence of a pharmacological effect as measured by reductions in serum cholesterol — a biomarker for miR-122, Orum explained this week during a presentation at the IBC Life Sciences' Oligonucleotide Therapeutics Discovery conference held here.

The company also initiated a second phase I study, this time examining multiple ascending doses of the drug, which it expects to wrap up shortly, he said.

Because data from this trial have not been publicly disclosed, Orum declined to comment on them. He did say, however, that Santaris is "happy with the results" and that the company is slated to begin a phase II study in HCV-infected patients "later this year."

Both phase I studies were conducted in Denmark. He noted that the company is considering in which countries it will run the phase II trial.

Meanwhile, Santaris has made the decision to keep SPC3649 in-house after partner GlaxoSmithKline opted against licensing the drug.

In late 2007, the companies inked a deal to develop and commercialize antiviral drugs based on the LNA technology (RNAi News 12/20/2007). As part of that arrangement, GlaxoSmithKline had the option to in-license SPC3649.

But as reported by RNAi News, GlaxoSmithKline let that option expire (RNAi News 12/3/2009), leaving Santaris free to host licensing negotiations with other interested parties.

Notably, GlaxoSmithKline later said it had partnered with rival miRNA drug firm Regulus Therapeutics on a miR-122-targeting drug for indications including HCV (RNAi News 2/25/2010).

This week, however, Orum said that despite "considerable interest … [in SPC3649] we've elected to keep it as a Santaris asset until we have conducted the first trials in patients with HCV. We think there is significant value creation in that phase, and we'd obviously like to retain that within Santaris … we have patient data."

read more» Read more...

New drug for the treatment of premature ejaculation

As many as one in three men suffer from premature ejaculation (PE) at some point in their life. A new prescription drug, Priligy, may help alleviate the effects of this sexual dysfunction, at least for PE sufferers who live in the UK.

If taken 1 to 3 hours before sex, Priligy can make a man last 2 to 3 times longer during intercourse. With that said, the average time gained is only two minutes. This may not seem like much, but it makes a world of difference for men who often climax in under a minute.

The drug works by regulating serotonin levels in the brain and has been proven to increase control of one’s ejaculations for up to 24 hours. The major downfall is the price. A three pack of 30 mg tablets costs around $116 US dollars. The drug has already gone on sale in other European nations and now will be available in the UK for men 18 to 62.

Johnson & Johnson will sell the drug over the internet to patients who complete an online consultation with a doctor. This online process will make it easier for men who feel uncomfortable discussing the problem in person with their doctor to address their sexual dysfunction. It may also make it easier for women posing as their PE suffering partners to order the drug.

“Priligy has the potential to do as much for men’s sexual health as Viagra,” said Nitin Makadia, head of male sexual health at U.K.’s leading pharmacy, Lloydspharmacy. “It will give sufferers a chance to improve the quality of their relationships and their general well-being.”

Priligy has yet to be approved for sale in the United States. For now American men with PE will have to rely on the standard climax-prolonging techniques: thinking of boner-killing images (like penis guillotines) , using condoms to dull the sensation, drinking excessive amounts of alcohol, and lathering their members in topical numbing agents.

read more» Read more...

Pennsaid Launch Brings New Treatment to Knee Osteoarthritis Patients

Covidien , a leading global provider of healthcare products, has introduced Pennsaid (diclofenac sodium topical solution) 1.5% w/w to the U.S. market. पेंन्सैद is the only FDA-approved topical non-steroidal anti-inflammatory drug (NSAID) in a vehicle solution containing dimethyl sulfoxide (DMSO), a known penetrating agent. It is indicatedfor treatment of the signs and symptoms of osteoarthritis of the knee(s).

PENNSAID offers patients suffering from osteoarthritis of the knee an effective option for reducing pain and improving physical function.

"We believe Pennsaid will become an important treatment for physicians caring for patients with osteoarthritis of the knee," said Timothy R. Wright, President, Pharmaceuticals, Covidien. "The product's unique formulation with DMSO and its proven clinical effectiveness make it a convenient and innovative treatment for patients who are seeking effective pain relief and improved physical function in their daily lives."

A growing number of guidelines and review committees suggest that the use of topical NSAIDs in the treatment of knee osteoarthritis offers potential significant gastrointestinal safety benefits for certain patients. Localized treatment may provide improved safety and tolerability benefits.

In Phase III clinical trials, Pennsaid demonstrated statistically significant differences in all three primary efficacy endpoints: pain and physical function (as measured by the Western Ontario and McMaster Universities LK3.1 OA Index, or WOMAC subscale), and patient overall health assessment (POHA) / patient global assessment (PGA).

"Ten million Americans suffer from osteoarthritis of the knee, illustrating the clear need for additional treatment alternatives," said Charles Argoff, M.D., Professor of Neurology at Albany Medical College and Director of the Comprehensive Pain Program of Albany Medical Center. "Though osteoarthritis is not preventable, many people are able to manage it and maintain an active lifestyle with a well designed treatment program, including medications, exercise, and physical therapy when needed."

According to the Arthritis Foundation, moderate exercise provides multiple benefits for people with osteoarthritis -- reducing joint pain and stiffness, building strong muscle around the joints and increasing flexibility and endurance for osteoarthritic patients. Education around appropriate knee osteoarthritis treatment is especially timely, as May is National Arthritis Awareness Month.

Knee osteoarthritis is one of five leading causes of disability among adults and is a chronic condition characterized by the breakdown of the joint's cartilage. Cartilage cushions the ends of the bones and allows easy movement of joints. The breakdown of this cartilage causes the bones to rub against each other, resulting in stiffness, pain and loss of movement in the joint.

Symptoms of osteoarthritis include joint pain or stiffness following periods of either inactivity or excessive use, a grating or catching sensation during joint movement and bone spurs (small growth of new bone) in the affected joints.

Last November, Covidien with Nuvo Research announced the U.S. Food and Drug Administration (FDA) had approved the New Drug Application for Pennsaid .

read more» Read more...

Merck Buys Rights to Potential Chronic Lung Drug

Drugmaker Merck & Co. said Monday it has bought rights to help market what would be the first anti-inflammatory pill for a chronic lung condition, just a few days after European regulators recommended approval there.

The deal gives Merck rights to jointly market Daxas, made by Swiss drugmaker Nycomed, for chronic obstructive pulmonary disease in Canada and several European countries. Merck, based in Whitehouse Station, N.J., also received an exclusive distribution deal for the United Kingdom.

If approved, Daxas would be the first drug in a new class of treatments for chronic obstructive pulmonary disease, which includes bronchitis and emphysema.

However, U.S. regulators have recommended against its approval. Final decisions in the U.S. and Europe are still pending.

Daxas, a pill taken once a day, blocks an enzyme involved in the lung disorder and in related inflammatory diseases. Patients now generally take inhaled medicines including corticosteroids, which reduce inflammation, and bronchodilators, which open up airway passages in the lungs to ease breathing.

Under the deal, privately owned Nycomed will receive an undisclosed upfront fee and could get additional payments for meeting development and sales milestones.

If Daxas is approved, Merck and Nycomed will co-promote it in France, Germany, Italy, Spain, Portugal and Canada, while Merck will sell it exclusively in the United Kingdom.

Nycomed, based in Zurich, will make and distribute the product. It sells brand-name medicines for respiratory, inflammatory and gastrointestinal diseases, osteoporosis and pain, plus some over-the-counter products.

Forest Laboratories Inc. has U.S. rights to sell Daxas, for which it paid Nycomed $100 million.

Earlier this month, a panel of Food and Drug Administration lung experts voted 10-5 against approving Daxas, known chemically as roflumilast, because of concerns about potentially dangerous side effects. In a study comparing Daxas with another drug, problems including suicide, cancer and weight loss were more common among the patients receiving Daxas.

However, the panel voted 9-6 that Daxas appeared effective. An FDA decision is expected by mid-May.

Last Friday, the European Medicines Agency's Committee for Medicinal Products for Human Use recommended approval of Daxas for EU countries.

read more» Read more...

NexMed Receives FDA Clearance for PrevOnco Phase 2 Study as First-Line Therapy for HCC

NexMed, Inc. , a specialty CRO with a pipeline of products based on the NexACT technology, today announced that the U.S. Food & Drug Administration (FDA) has cleared the Company to proceed with the proposed Phase 2 trial of PrevOnco , its proprietary cancer treatment for patients with advanced, unresectable hepatocellular carcinoma (HCC), or liver cancer.

The FDA granted PrevOnco orphan drug status in August 2008, and in March 2010, NexMed filed its Investigational New Drug application for the product candidate.

The Company also noted that in IND review communication, the FDA has given NexMed the opportunity to move PrevOnco directly into a Phase 3 trial that would support marketing approval, subject to positive study results. In order to pursue this regulatory path, NexMed would need to expand the proposed Phase 2 study design to use PrevOnco in combination with Doxorubicin as a second-line therapy for patients who have failed NEXAVAR , the currently marketed first-line anticancer treatment for patients with either HCC or advanced renal cell carcinoma (cancer of the kidney).

PrevOnco incorporates lansoprazole, which is the generic anti-ulcer compound approved under the name Prevacid(R) and marketed in the U.S. by Takeda Pharmaceuticals North America, Inc. In vitro and in vivo data generated to date has demonstrated the ability of lansoprazole to inhibit tumor cell growth and enhance survival in mouse models of cancer alone, and in combination with Doxorubicin.

Commenting on today's news, Dr. Bassam Damaj, President and Chief Executive Officer of NexMed, stated, "We are very pleased that the FDA agreed with our protocol for the HCC Phase 2 trial for PrevOnco as a first-line therapy for HCC. Additionally, we are actively assessing the suggestion made by the FDA to move directly into a Phase 3 trial, by studying PrevOnco in combination with Doxorubicin as a second-line therapy for patients who have failed NEXAVAR therapy. Following this path could be very advantageous for NexMed since advancing the drug directly into a Phase 3 study would save us at least 12-24 months in development time."

read more» Read more...

A New Drug Approved By The FDA For The Treatment Of Fibromyalgia Pain

NMS Labs launches another unique test suitable for the evaluation of toxicity vs. efficacy, the potential for physicians to establish therapeutic drug monitoring (TDM) of individual patients, and the assessment of drug diversion of a new pain management drug therapy.

NMS Labs is the only known commercial laboratory to develop and perform a clinical quantitative assay for Milnacipran (Savella®) using the highly specific and sensitive technique of liquid chromatography/tandem mass spectrometry (LC-MS/MS).

Eric Rieders, PhD, President and CEO expressed the importance of innovation at NMS Labs by saying, “We best serve the medical community by deploying our professional and technical resources to rapidly identify and develop diagnostic tests within our area of expertise that might otherwise only become available at a much later date.” Dr. Rieders added, “NMS Labs is well known for providing the rare and hard to find tests that other labs do not.”

NMS Labs has responded to the need of the pain management community by filling a void in testing options. Milnacipran is a new medication used for the treatment of pain associated with fibromyalgia. NMS Labs specializes in testing for various classes of pharmacological agents, including: analgesics; anesthetics; anticonvulsants; antidepressants; hallucinogens; muscle relaxants; NSAIDs; opioids; sedatives and hypnotics; sleep aids; and stimulants.

read more» Read more...

Tumors Deficient In A Specific Enzyme Are Sensitive To A New Drug 'Adi-Peg 20'

Polaris Group scientists and university collaborators reported at the American Association for Cancer Research (AACR), that a number of different cancers have a deficiency in a specific enzyme, argininosuccinate synthetase (ASS), which allows pegylated arginine deiminase (ADI-PEG 20) to inhibit cancer cell growth.

"Polaris has been testing this drug in melanoma patients and patients with hepatocellular carcinoma (HCC), two tumors that are ASS deficient, and we are preparing to initiate a global phase 3 clinical trial in HCC to seek regulatory approval for marketing ADI-PEG 20," said Dr. Bor-Wen Wu, CEO of Polaris. "In addition, these new results show that ADI-PEG 20 may be beneficial in a number of other tumors."

ASS is required for the production of arginine, an amino acid needed for growth and replication of cells. Normal tissue cells have normal levels of ASS and can produce sufficient arginine for their own growth and survival in the presence of ADI-PEG 20. However, growth and replication of ASS-deficient tumor cells is inhibited by ADI-PEG 20 because arginine in these cells is degraded and depleted. The studies presented at AACR have shown that various other tumors also have a deficiency in ASS. He et al, Polaris Group, reported that 30-60% of human breast, lung and gastric cancers were deficient in ASS. Treatment of these and other tumor types in cell culture, including sarcoma, leukemia and plasmacytoma, showed significant inhibition of cell growth by ADI-PEG 20. He et al also established primary renal cell carcinoma cultures from fresh human tumor biopsies which were ASS-deficient. ADI-PEG 20 inhibited growth of these cells. These findings suggest that ASS deficiency may serve as a biomarker for selecting tumors for targeted therapy with ADI-PEG 20.

Rubin et al, Cleveland Clinic, showed that 88% of 45 different human sarcomas were ASS-deficient. Treatment of two sarcomas, gastrointestinal stromal tumor (GIST) and malignant peripheral nerve sheath tumor (MPNST) with ADI-PEG 20 resulted in inhibition of proliferation.

Based on analysis by Jungbluth and collaborators, Ludwig Institute for Cancer Research New York Branch at Memorial Sloan-Kettering Cancer Center, demonstrating that ~50% of human small cell lung cancer (SCLC) tumor specimens were ASS-deficient, Kelly et al, showed that growth of ASS negative cell lines was inhibited by ADI-PEG 20 in cell culture. They further showed that ADI-PEG 20 treatment of mice bearing ASS-deficient SCLC xenograft tumors caused significant and dose dependent inhibition of tumor growth in the animals.

It has been well established that treatment of human cancer with two or more drug combinations can be much more effective than the individual drugs administered alone. Two reports provided evidence that ADI-PEG 20 plus cisplatin treatment in cell cultures of ASS-deficient malignant pleural mesothelioma (Szlosarek et al, Barts Cancer Research UK Centre) and melanoma (Feun et al, University of Miami) resulted in synergistic cytotoxic effects on the cancer cells compared to either agent alone. Additional evidence of inhibition of pancreatic xenograft tumors was presented by Bold et al, University of California at Davis. Mice bearing ASS-deficient human pancreatic tumors were treated with ADI-PEG 20 plus gemcitabine and showed that the two drugs in combination inhibited tumor growth much more effectively that either drug alone.

Based on these and other findings, ADI-PEG 20 clinical trials are planned to begin in the near future for treatment of patients with malignant mesothelioma, SCLC, sarcoma, leukemia or pancreatic cancer.

read more» Read more...

FDA approves new drug to treat arthritis

FDA officials have recently approved a drug known as Acterma for treating moderate to severe forms of rheumatoid arthritis in patients non-responsive to TNF inhibitors.

Rheumatoid arthritis, an autoimmune disease that causes chronic inflammation of the joints, is typically a progressive illness, leading to long-term destruction and functional disability.

There is no known cure for the condition; various drugs such as TNF inhibitors, however, are commonly prescribed for these patients aiming to reduce joint inflammation and pain.

According to the statement recently released by FDA, Actemra, given once-monthly through hour-long infusions, is the first IL-6 inhibitor approved for treating arthritis.

Also known as tocilizumab, Actemra cannot be used in combination with TNF inhibitors or other biologic treatments commonly used in arthritis. Its simultaneous use with disease-modifying drugs such as methotrexate, however, seems to be acceptable.

The main side effects of Actemra include serious infections, diverticulitis, and severe allergic reactions. Increased levels of cholesterol and blood fats are also seen in some of these patients.

Officials hope to extend the use of the drug from relatively severe conditions to earlier stages of rheumatoid arthritis (RA).

read more» Read more...

A new pill to swallow in treating depression

UnknownIt's January in Portland, dark outside more than half the time. The only glimpse of color comes from those Hawaiian Airlines billboards around town, advertising flights to tropical islands that feel financially out of reach.

An antidepressant sounds good right about now. Maybe a few bottles of antidepressants, carried close in a fanny pack and popped like mints until the weather and economy improve. There's a few problems with this pharmaceutical fantasy, though:

* Antidepressants don't work for most people who want them, according to a major new study.

* The risk of taking these pills often outweighs the benefit.

* Antidepressants are already the third-most-prescribed class of drug in the United States. This $10 billion industry doesn't need any more mildly bummed Oregon people as customers to turn a profit.

Drugs typically prescribed to treat depression work no better than placebos in most cases, according to a new analysis published last week in the Journal of the American Medical Association.

The one exception is severe depression: Most people who are depressed enough to feel suicidal or incapacitated, for example, will experience some relief from antidepressants. But the same isn't true for people with mild or moderate depression, who represent the bulk of the antidepressant market.

People who are merely singing the blues rather than drowning in them, it turns out, will find little or no more relief from their Paxil, Lexapro or Prozac than from a sugar pill.

(Why has previous research painted a rosier picture? First, drug companies have a history of downplaying negative clinical trials. Second, patients who participate in trials tend to be more depressed than the average bear, so they see more benefits from drugs. Third, just seeing a doctor can make people feel better, even when the actual medicine is ineffective.)

The new research calls the true power of happy pills into question. It certainly contradicts those drug ads about the instant, painless, magical and restorative nature of antidepressants.

It also has big implications for health care costs -- not to mention medical ethics.

Doctors wrote 164 million prescriptions for antidepressants in 2008, totaling $10 billion in U.S. sales, according to IMS Health. About 27 million Americans now take antidepressants, mostly for problems less acute than severe clinical depression.

In doing so, these patients expose themselves to side effects such as weight gain, agitation, insomnia, loss of sex drive and so on -- which in turn can lead to more health problems, doctor visits and prescriptions. Such patients might be better off exercising or spending time with friends instead.

Again, there's no question that antidepressants can be lifesavers. I've watched friends overcome crippling anxiety and even suicidal feelings while using antidepressants, usually in conjunction with therapy and exercise. When you're hurting and nothing else works, sometimes you've got to throw everything at the wall and see what sticks.

Yet this research does raise serious questions about our reliance on antidepressants and susceptibility to drug marketing. True, it's possible someone will invent a perfect pill that makes a jobless recovery in January feel like a day at the beach, but no one has yet.

Until then, the best strategy may be to bundle up, hold tight and wait for spring.

read more» Read more...

FDA OKs Genentech's new arthritis drug

Federal regulators have approved a new class of rheumatoid arthritis drug created by South San Francisco's Genentech.

The drug, called Actemra, is the first arthritis treatment to operate by blocking the activity of a protein called interleukin-6 that, when overly abundant in the body, causes inflammation in the joints associated with arthritis। The Food and Drug Administration approved the drug Friday।

"This is the ninth biologic to hit the market to treat rheumatoid arthritis, but the only one that targets IL-6," said Dr। Mark Genovese, co-chief of Stanford University's immunology and rheumatology division and the new drug's study investigator.

Genovese said the drug will provide an option for people who respond inadequately to existing therapies, such as Remicade, marketed by Johnson & Johnson, Abbott's Humira and Pfizer's Enbrel. Those treatments, which are among the top-selling drugs in the country, act on another inflammatory protein called tumor necrosis factor-alpha, or TNF.

About 30 to 50 percent of people do not respond well to TNF inhibitors, Genovese said. The reason is unknown.

Rheumatoid arthritis is an autoimmune disorder that causes pain, swelling and eventually damage to joints. About 1.3 million American adults are affected by the disease, according to the Arthritis Foundation.

Officials with the Roche Group, parent company of Genentech, estimated the cost of Actemra at $1,060 to $2,125 a month, depending on the dose. The prices of other biologics used for rheumatoid arthritis range from $194 to $2,768 per vial, the company said.

The drug will be available the week of Jan. 18, according to Roche.

read more» Read more...

Biodel Submits VIAject® for Treatment of Diabetes

Biodel, Inc. announced today that it has submitted a new drug application (NDA) to the U.S. Food and Drug Administration for clearance to market VIAject® as a treatment for diabetes.

VIAject® is Biodel’s proprietary formulation of recombinant human insulin that is designed to be absorbed into the blood faster than currently marketed rapid-acting insulin analogs. It is Biodel’s most advanced product candidate and has been tested in more than 884 patients who participated in Phase 1, 2 and 3 clinical trials of the drug in the United States, Germany and India. Biodel is seeking approval to market VIAject® in the United States as a 100 IU/cc, pH7 (neutral) injectable liquid, in 10 ml vials and 3 ml pen cartridges.

The NDA includes results from pharmacokinetic, pharmacodynamic and standardized meal studies, two pivotal 6-month Phase 3 clinical trials of VIAject® in patients with Type 1 and Type 2 diabetes, as well as interim results from the long-term, 18-month safety extension trials for patients who completed the pivotal Phase 3 clinical trials. The data from these studies consistently document the safety and efficacy of VIAject®.

Biodel’s chairman and chief executive officer, Dr. Sol Steiner, stated: “We believe our studies demonstrate that patients receiving VIAject® had faster reductions in blood glucose activity, reduced risks of hyperglycemia and hypoglycemia and less weight gain than patients who received recombinant human insulin, and that VIAject® may offer important clinical benefits to people with diabetes. I congratulate our team on submitting the NDA and reaching this important milestone in the development of our lead product candidate.”

About VIAject®

VIAject® is Biodel’s proprietary formulation of injectable human insulin for use at mealtime and in insulin pumps. It has been shown to be absorbed into the blood faster than currently marketed rapid-acting insulin analogs. VIAject®’s formulation allows insulin to disassociate, or separate, from the six molecule (hexameric) form to the single molecule (monomeric) form and inhibit re-association to the hexameric form, promoting more rapid delivery of insulin into the blood to produce its desired biological effects.

About the NDA

Since VIAject® is a modified form of recombinant human insulin, Biodel is submitting its new drug application for VIAject® under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act which governs the review of an NDA for a modified form of a previously approved product. This section of the act allows a pharmaceutical company to submit an NDA based in whole or in part on published literature or on the FDA’s finding of safety and efficacy of one or more previously approved drugs and may result in approval of a drug based on fewer clinical or nonclinical studies than would be required under a full NDA. Although no insulin product has been approved under this section of the act, Biodel believes its NDA for VIAject® qualifies for review under section 505(b)(2).

About Diabetes

Diabetes is a disease characterized by abnormally high levels of blood glucose and inadequate levels of insulin. Glucose is a simple sugar used by all the cells of the body to produce energy and support life. Humans need a minimum level of glucose in their blood at all times to stay alive. Insulin is a peptide hormone naturally secreted by the pancreas to regulate the body’s management of glucose. When a healthy individual begins a meal, the pancreas releases a natural spike of insulin called the first-phase insulin release, which is critical to the body’s overall control of glucose. All patients with Type 1 diabetes must treat themselves with mealtime insulin injections to compensate for the lack of natural pancreatic first-phase insulin release. As the disease progresses, patients with Type 2 diabetes also require mealtime insulin. However, none of the currently marketed mealtime insulin products adequately mimics the first-phase insulin release. As a result, patients using insulin typically have inadequate levels of insulin in their systems at the start of a meal and too much insulin in their systems between meals. This, in turn, results in the lack of adequate glucose control associated with diabetes. The long-term adverse effects of elevated glucose levels include blindness, loss of kidney function, nerve damage and loss of sensation and poor circulation in the periphery, which in some severe cases, may lead to amputations.

About Biodel Inc.

Biodel is a specialty biopharmaceutical company focused on the development and commercialization of innovative treatments for endocrine disorders such as diabetes. Biodel's product candidates are developed using technology which reformulates existing FDA-approved peptide drugs. For further information regarding Biodel, please visit the company's website at www.biodel.com.

read more» Read more...

Submission of a New Drug Application for Pancreatic Enzyme Replacement Therapy

A New Drug Application for the pancreatic enzyme replacement therapy agent for pancreatic exocrine insufficiency, SA-001 (API name: Pancrelipase) that Solvay Seiyaku K. K. co-developed with Eisai Co., Ltd. was submitted by Solvay Seiyaku on December 22, 2009 in Japan.

The agent is a high performance, acid resistant enteric-coated pancreatic enzyme obtained by formulating highly purified pancrelipase with a proprietary technology. It is marketed as Creon(R)/Kreon(R) in 75 countries outside Japan including the United States, the United Kingdom and Germany. Creon/Kreon is the global market leader in pancreatic enzyme replacement therapy for pancreatic exocrine insufficiency due to cystic fibrosis, chronic pancreatitis, and pancreatectomy and other conditions. After obtaining its manufacturing and marketing authorization for the agent, distribution in Japan will be conducted by Eisai. Solvay Seiyaku retains co-promotion rights with Eisai.

The agent significantly improved the difference of fat absorption ratio calculated from fat intake and discharged fat in stool before and after administration in its double blind placebo controlled clinical study in patients with pancreatic exocrine insufficiency due to chronic pancreatitis or pancreatectomy conducted in Japan. Furthermore, the agent showed significant improvements of the nutrition evaluation scale in its long term study. Also, the agent showed important improvements of fat absorption ratio and nutrition evaluation scale in a clinical study in patients with pancreatic exocrine insufficiency caused by cystic fibrosis.

Solvay Seiyaku is a Japanese entity of Solvay Pharmaceuticals, which is the pharmaceutical sector of the chemical and pharmaceutical global company, Solvay Group. It is a development-driven pharmaceutical company that seeks to fulfill carefully selected, unmet needs in the therapeutic areas of neuroscience, cardio-metabolic, and gastroenterology.

With the proton pump inhibitor Pariet(R) and gastritis/gastric-ulcer treatment Selbex(R) amongst its main products in Japan, Eisai considers gastroenterology as an area of therapeutic focus. It aims to enhance this area even further through the addition of SA-001 to its product portfolio.

Solvay Seiyaku and Eisai work in tandem to bring SA-001 as a new treatment option to contribute to the treatment of patients with pancreatic exocrine insufficiency and to improving their quality of life.

read more» Read more...

FDA Approves Azaya Therapeutics Investigational New Drug Application

Azaya Therapeutics, Inc. announced today that the Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for its lead product "ATI-1123," a novel and improved formulation of Taxotere® (docetaxel), a leading chemotherapy drug with worldwide annual sales of over $2.8 billion.

Azaya’s Phase I clinical study will now open for enrollment at two premier cancer research centers in Texas: South Texas Accelerated Research Therapeutics at the START Center for Cancer Care in San Antonio and The Mary Crowley Cancer Research Center in Dallas. Patients with solid tumors that have not responded to treatment with other anti-cancer agents will be enrolled in the study. The study is designed as an open-label, dose-escalation study that will determine the safety, tolerability and pharmacokinetics of the drug. The study also provides for the collection of efficacy data.

"The FDA’s acceptance of our IND package is an important milestone in the commercialization of this important and innovative new cancer treatment," said Michael T. Dwyer, president and CEO of Azaya. "Developing a cancer treatment such as ours takes an extraordinary amount of time and effort, and I am pleased to initiate our Phase I trial for patient enrollment," he added.

Azaya utilizes a proprietary manufacturing process to produce liposome-encapsulated chemotherapeutics that eliminates the use of toxic carriers and incorporates a naturally occurring protein to stabilize the liposomal encapsulated drug. Preclinical studies have demonstrated improvement in the toxicology profile and an enhanced efficacy of ATI-1123 compared to Taxotere® in a number of tumor models.

Azaya Therapeutics is a San Antonio-based emerging pharmaceutical company whose innovative technology platform makes safer and more effective cancer treatments. Its unique and proprietary Protein Stabilized Nanoparticle (PSN™) platform addresses the significant problems associated with delivery of water insoluble drugs. Azaya has leveraged this platform to develop differentiated drug products for the global oncology market. The company’s management is highly experienced in biotechnology and oncology and has a track record of obtaining FDA drug approvals and successfully building companies.

read more» Read more...