France’s Transgene will hold on to control of the upcoming Phase IIb/III pivotal trial of TG4010 that is slated to get under way by the end of this year after enrolling about a thousand patients with non-small cell lung cancer. Once Novartis gets its hands on the IIb portion of the data, the pharma giant will have 90 days to decide whether it will exercise its option on the program. The data is scheduled to arrive in early 2012.

If Novartis decides to pull the trigger on the option–a deal structure that is growing increasingly common in drug development–Transgene will get a fee plus milestones along with co-promotion rights in a list of countries that includes France and China. Novartis will gain control of the program and take responsibility for further costs.

“We believe this agreement represents the best way to accelerate development and create long term value for our shareholders,” says Transgene CEO Philippe Archinard. “It is also consistent with the company’s goal of becoming a fully integrated biopharmaceutical company as under this agreement Transgene will maintain certain commercialization and manufacturing rights.”

However, not everyone was impressed with the optional, uncertain terms of the agreement, andshares in the French biotechnology company went down 12 percent. “We view today’s option announcement as slightly underwhelming, given that the ongoing burden of funding remains with Transgene for another two years,” says Nomura Code analyst Gary Waanders.

Because Novartis wants to see the outcome of a mid-stage Phase IIb clinical trial before exercising its option, Waanders said he was reviewing his “neutral” rating and fair value estimate of 21.80 euros a share.

Source: FierceBiotech

Researchers at McMaster University in Hamilton, Ontario found there were 61 percent fewer flu cases in isolated communities where children and adolescents received the seasonal influenza vaccine, compared to communities where children received an unrelated vaccine.

Targeting children with a vaccine could protect the wider population, researcher Mark Loeb and colleagues concluded in their report in the Journal of the American Medical Association.

Influenza struck 2,326 unvaccinated participants in the 46 religious Hutterite communities in western Canada that were chosen for study because they have limited outside contacts.

In communities where roughly four out of five children aged 3 to 15 were vaccinated, 3.1 percent of the people got the flu compared to a 7.6 percent infection rate in communities where no one was vaccinated against flu.

This demonstrated the widely-accepted concept of “herd immunity” — that vaccination programs can still be effective even if not everyone is vaccinated — which could have public health implications, said Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases.

“Before, the healthy adolescent kids were not among those that were highly recommended to get vaccinated,” said Fauci, whose NIAID is part of the U.S. National Institutes of Health, which helped fund the study.

“Now, this shows that even though they don’t usually get into trouble from the flu, you can get double bang for the buck by protecting not only them but the people they come in contact with.”

For instance, if there was a shortage of flu vaccine, it might be more effective to vaccinate the young, even though the flu presents less of a danger to them, because their rough-and-tumble socializing habits can easily spread viruses.

Last month, vaccine advisers to the U.S. Centers of Disease Control and Prevention recommended everyone over the age of six months should receive seasonal flu vaccines every year.

Seasonal flu is blamed for 36,000 U.S. deaths each year.

Fauci said the same universal vaccination policy might apply against the pandemic H1N1 virus known as swine flu, which the CDC estimated has killed about 12,000 Americans.

“Now that we’re talking about vaccinating everyone, this (idea of selective vaccination of children) becomes a little bit of a moot point,” Fauci added in a telephone interview.

Source: Reuters

As per the agreement, Torrent will provide AstraZeneca with a portfolio of generic medicines for which the Indian group already has licenses in a range of countries, starting initially with 18 products in nine countries. Torrent will manufacture the medicines working to AstraZeneca’s rigorous quality and process standards.

Reportedly, AstraZeneca is facing stiff competition on seven drugs by 2014, including its three biggest sellers: Nexium for ulcers, the antipsychotic Seroquel and Crestor for cholesterol. According to David Brennan, CEO of AstraZeneca, the dip in sales may result in a period of fluctuating earnings. He is of the opinion that external partnerships with generic drugmakers will help the firm in maintaining double digit growth in the emerging markets.

Against this backdrop, the British-Swedish pharma major is following other big drug makers GlaxoSmithKline and Sanofi-Aventis which also have been eager to forge agreements to foray into the emerging markets to increase thier generic drug sales. AstraZeneca intends to increase generic sales in emerging markets to 25% of annual revenue over the next five years, from 13% in 2009.

Renaud Savary, head of branded generics unit at AstraZeneca, said: “Clearly, we see branded generics as a way to accelerate growth in emerging markets. The products were carefully selected in therapy areas we’re strong in. We believe we can be successful in building market share and brand equity.”

In a statement, AstraZeneca said: “Emerging markets are forecast to contribute around 70% of pharmaceutical industry growth in the next five years, and branded generics represent approximately 50% by value in these emerging markets. AstraZeneca believes it can capitalize on this opportunity and over time plans to broaden its portfolio beyond these initial 18 products.”

Tony Zook, head of AstraZeneca’s global commercial organisation, said: “In markets where consumers and physicians have a strong preference for trusted brands, we believe AstraZeneca’s long-standing reputation for quality is a sustainable competitive advantage.

“Working in partnership with Torrent will extend the range of branded medicines we can offer to patients in emerging markets, where we see continuing opportunities for our business to grow. We have chosen Torrent because of their complementary product portfolio and proven ability to manufacture to AstraZeneca’s high quality standards.”

Source: Pharmaceutical Business Review

FDA staffers recently ran a two-day workshop in Claremont, Calif., to help drug developers fill out the application to get orphan-drug status, the WSJ says in an article this morning. Another workshop is planned for the University of Minnesota in August and there’s talk about doing one in Europe.

There are roughly 350 orphan drugs currently approved, covering about 150 rare diseases. The core requirement for orphan status is that the medicine treats a disease affecting fewer than 200,000 Americans, a limited market that often makes such treatments very expensive. Last year, 250 requests for orphan designation were filed with the FDA, and 160 received it.

Of course, getting more applications doesn’t mean more drugs will make it through the FDA approval process, orphan status or not, the WSJ notes. But the FDA officials with the orphan program hope that increasing the application pool will boost the chances of getting more rare-disease treatments to market.

The first workshop drew 29 potential sponsors, three-quarters of which said they had never filed an orphan-drug application before. Not that the process is really that hard. “It’s not ‘War and Peace,’ ” an FDA official told the WSJ. “The applications are six or seven pages.”

Source: The Wall Street Journal

The DSMB concluded that the safety risk outweighs the benefits observed in these specific patient populations at this time. The DSMB review detected an infection related safety signal which included serious and opportunistic infections, some of which were fatal.

As previously announced, the FILM study in MTX-naïve RA patients was placed on clinical hold following an assessment of benefit to risk in this specific RA patient population. In addition, the BELONG study in lupus nephritis patients was previously halted due to serious and opportunistic infection signals.

“Patient safety is of the utmost importance in all of our drug development programmes. In light of the DSMB recommendations we have decided to suspend Ocrelizumab treatment in the RA clinical development programme.” said Hal Barron, MD Executive Vice President and Chief Medical Officer at Roche.

The SCRIPT trial in patients who inadequately responded to one or more TNF antagonists and the FILM trial remain blinded. A detailed analysis of all of the data will be conducted to help further inform the future of the Ocrelizumab RA clinical programme.

Ocrelizumab is also being evaluated for Relapsing Remitting Multiple Sclerosis (RRMS). The treatment in the Ocrelizumab RRMS Phase II study is on-going at this time.

Source: World Pharma News

Dr. Richard Ablin of the University of Arizona joined the ongoing debate over the blood test, saying the screening procedure is too costly and ineffective.

“I never dreamed that my discovery four decades ago would lead to such a profit-driven public health disaster,” Ablin wrote in a commentary for The New York Times.

Ablin said that as Congress searches for ways to cut costs in the U.S. health care system, a significant savings could come from changing the way PSA is used.

“The test’s popularity has led to a hugely expensive public health disaster,” he wrote.

He said the annual bill for PSA screening is at least $3 billion, with much of it paid for by Medicare and the Veterans Administration.

“As I’ve been trying to make clear for many years now, PSA testing can’t detect prostate cancer and, more important, it can’t distinguish between the two types of prostate cancer — the one that will kill you and the one that won’t,” he wrote.

“Instead, the test simply reveals how much of the prostate antigen a man has in his blood.”

Prostate cancer is the second most common cancer in men worldwide after lung cancer, killing 254,000 men a year.

PSA is a protein made only by prostate cells, and levels can shoot up as a prostate tumor proliferates. But levels can also rise as the prostate naturally enlarges with age.

A high PSA reading is usually followed by a biopsy, which is a sample of the prostate tissue taken and examined for signs of a tumor.

SLOWLY TURNING AGAINST

Doctors have routinely recommended PSA tests to men over 50 in the belief that early diagnosis and aggressive treatment for any cancer is better than standing by and doing nothing.

But prostate cancer can often be a slow-growing tumor and men will often die of something else before the cancer becomes dangerous.

Prostate cancer treatments, including surgery or radiation, can cause incontinence and erectile dysfunction in about a third of patients. Many men also experience bowel problems.

Citing recent studies and reversals of some early screening proponents, Ablin said the medical community is slowly turning against PSA screening.

“So why is it still used? Because drug companies continue peddling the tests and advocacy groups push ‘prostate cancer awareness’ by encouraging men to get screened,” Ablin wrote.

Ablin said PSA testing does have a place, after treatment for prostate cancer and for men with a family history of prostate cancer.

“Testing should absolutely not be deployed to screen the entire population of men over the age of 50, the outcome pushed by those who stand to profit,” Ablin wrote.

He urged the medical community to “confront reality and stop the inappropriate use of PSA screening.”

Source: Reuters

The Food and Drug Administration issued its statement following the publication of case reports of atypical subtrochanteric femur fractures — or fractures in the bone just below the hip joint — in women with osteoporosis using oral bisphosphonates.

Bisphosphonates are a class of drug aimed at preventing bone fractures and offsetting bone loss associated with menopause.

They include Fosamax, Roche Holding AG’s Boniva, Novartis AG’s Reclast and Procter and Gamble Co’s Actonel.

In June 2008, the FDA requested information from all bisphosphonate drug makers related to these type of fractures. The agency said a review of the data did not show an increased risk for women using the medications.

The FDA said that, although its review of the data did not show a clear connection between bisphosphonates and atypical subtrochanteric femur fractures, the agency is working closely with outside experts to gain more insight into the issue.

Bisphosphonates, which have been on the market for roughly a decade, have raised safety concerns in the past, including heart risks.

But in 2008, the FDA said the drugs showed no overall risk of heart problems. The agency’s review followed reports in the New England Journal of Medicine of serious atrial fibrillation, a type of abnormal heartbeat.

In January, a Manhattan federal judge refused to dismiss a lawsuit alleging that Fosamax caused jaw damage to a woman during the nearly eight years she took the pill.

Merck faces a slew of lawsuits involving almost 900 cases by patients who say Fosamax caused osteonecrosis of the jaw, or death of jaw bone tissue.

“In clinical studies, Fosamax has not been associated with increased fracture risk at any skeletal site,” Merck spokesman Ron Rogers said in a statement.

“Low energy femoral shaft and subtrochanteric fractures have been reported in the medical literature as occurring in non-bisphosphonate users,” Rogers noted, adding that Merck is currently conducting studies “to further investigate the issue of low energy femoral shaft and subtrochanteric fractures.”

The FDA recommended patients keep taking their medication unless told not to by their doctor. It also recommended that healthcare professionals be aware of a “possible risk” of atypical subtrochanteric femur fractures in patients taking oral bisphosphonates.

Source: Reuters

What’s at stake for these companies? Amylin has the most invested. An approval could provide the drugmaker with billions in revenues for years to come. According to the Wall Street Journal, one analyst projected $2.1 billion in sales for the drug by 2015, while another industry watcher predicts $3 billion in U.S. sales. For Lilly, Byetta LAR would boost a product lineup that’s facing one of the steepest patent cliffs in the coming years. And Alkermes is set to reap 7.5 percent royalty on worldwide sales.

Xconomy’s Luke Timmerman maps out the four possible outcomes from the FDA’s decision Friday:

The FDA approves Byetta LAR with only a standard warning, though JP Morgan analyst Cory Kasimov says there’s not much chance of that happening.

Byetta LAR could be approved, but with a severe black box warning about the possibility of patients developing thyroid cancer.

Third–most likely, according to Kasimov–the FDA will delay its response and ask for additional, but minor, data on the drug. In that case, the companies could still have an approval in hand by the end of 2010.

Finally, the worst-case scenario: FDA could issue a “complete response” letter asking for major new set of data that would severely delay the drug’s approval. If that happens, look for Amylin stock to lose half its value and Alkermes to dive about 33 percent.

Needless to say, the stakes are high for all involved.

Source: FierceBiotech

Not all the panelists were convinced of the drug’s efficacy; however, most voted that the potential benefits of the drug outweighed these concerns. “I voted yes because I’ve been straight down the middle the entire time. I didn’t see substantive evidence of efficacy per the FDA regulations but there was clinical meaningful effect on the disease. You need to offer patients hope. If this offers a smidgen of hope, then it is worth approving,” one panelist said, according to TheStreet’s Adam Feuerstein. Added another, “I voted yes, opposite of my vote on the question of substantial efficacy because I don’t believe there is substantial evidence of efficacy; but if I got this disease, I’d be on the next Delta flight to Japan.”

The FDA doesn’t have to follow the panel’s recommendations, but it usually does. In a conference call, CEO Dan Welch said that if the drug is approved, it may take the company some time to ramp up production. “We chose not to make certain investments in commercial or other areas of the company until we had visibility from this meeting. So one should not expect that Esbriet would be available immediately after the approval.”

During the call, analysts also attempted to suss out how InterMune would price the biologic if approved. InterMune also manufactures Actimmune, a treatment for chronic granulomatous disease that’s been used off-label for the treatment of IPF. On-label, Actimmune runs in the range of $8,000 to $20,000 per year. When used off-label for IPF patients, the annual cost price per year shoots up to $50,000. “I don’t know what you draw from that,” noted Welch, unwilling to reveal the possible price of Esbriet. A final decision is expected May 4.

Source: FierceBiotech

The documents, made public after Bloomberg asked a judge to unseal them, show that J&J was pushing to expand use of the drug beyond its schizophrenia indication. “Schizophrenia represents only 35 percent” of antipsychotic scrips, one executive at J&J subsidiary Janssen wrote in an internal report. “Aggressive expansion of Risperdal use in other indications is therefore mandatory.”

Based on its review of the documents, Bloomberg reports that the company tried to sell Risperdal for a variety of off-label uses, including dementia, an indication for which the drug was never approved. Janssen expanded its geriatric sales force almost threefold during that time. And despite nominal prohibitions against talking about unapproved uses, at least some doctors paid to speak on Risperdal’s behalf didn’t adhere to that policy. “I always plant a shill because if I get asked a question from the audiencee, I can then speak off-label,” one such doctor told the company.

As usual in these cases of big document dumps, there’s a legitimate question of “cherry-picking” the best–or worst–examples from all those pages. A J&J spokesman tells Bloomberg that the Lousiana case that spawned these documents “should be decided on the body of evidence … not on the basis of excerpts from documents.”

Source: FiercePharma