Biotech News

GSK studies Chinese remedies to discover new drugs

Ryan McBride — Mon, 13 Feb 2012 12:15:31 -0500

Amid GlaxoSmithKline's ($GSK) major push to expand in China, the London-based drugmaker has located a new R&D unit in that country that will investigate traditional Chinese medicines. The move is part of an effort to discover new chemical entities for treating diseases, British newspaper The Telegraph reported Sunday. Last week GSK R&D honcho Moncef Slaoui shared the plans with FierceBiotech in an interview about the drugmaker's strategy for discovering and developing new products. FierceBiotech's Slaoui interview | The Telegraph report

Chelsea shares nosedive on FDA's questions about key drug

Ryan McBride — Mon, 13 Feb 2012 12:00:22 -0500

Chelsea Therapeutics ($CHTP) CEO Simon Pedder sat on the hot seat this morning after his company's update on an ongoing FDA review of its lead drug Northera sparked concerns among some investors and analysts. Chelsea's chief spoke about the hypotension drug during the BIO CEO conference today on the heels of the worrying update.

The news sent Chelsea's shares down more than 40% at certain points this morning and its stock traded down 30.26% to $3.48 as of 11:54 a.m. EST today.

In FDA briefing documents, regulators highlighted their previously discussed concerns about the company's Northera program such as the short duration and small size of clinical trials for the drug, for which Chelsea is seeking approval to combat cases of neurogenic orthostatic hypotension in patients with Parkinson's disease and others with primary autonomic failure. U.S. regulators previously gave the company orphan status for the treatment and priority review back in November because of the lack of effective treatments for the condition, which causes dangerous drops in blood pressure when patients stand up, Reuters reported.

Among the agency's concerns about the program is the tricky business of quantifying symptomatic and clinical benefits. Because of this difficulty, and after talking to regulators in 2007, Chelsea has been measuring the effect of its drug with a patient-reported questionnaire that rates some of the symptoms of the disease such as low blood pressure, according to the company.

The FDA is expected to take action on Chelsea's application for Northera by March 28.

- see the Reuters article
- and The Wall Street Journal's report

DNA sequencing exposes new uses for Pfizer targeted cancer drugs

Ryan McBride — Mon, 13 Feb 2012 11:13:59 -0500

As pharma companies expand their use of DNA sequencing for drug R&D, researchers have uncovered some potential new targets for existing cancer drugs that are likely to spur further use of sequencing in the biopharma world. And their study has shed light on potential new uses for at least two of drug giant Pfizer's ($PFE) targeted cancer drugs.

With a cancer gene test from life sciences startup Foundation Medicine, the researchers from Dana-Farber Cancer Institute and elsewhere found that 59% of lung and colorectal cancer samples tested included genetic abnormalities that could be combated with targeted cancer drugs. A subset of lung cancer samples showed that the patients had mutations that could be targeted with Pfizer's approved kidney cancer drug Sutent, and one patient's colorectal cancer was found to have gene abnormality targeted by Pfizer's Xalkori.

The previously unknown genetic flaw in lung cancer was seen in about 2% of patient samples, but Dana-Farber lung cancer specialist Dr. Pasi Janne says that is enough to warrant further study of the Sutent in certain lung cancer patients, he told Bloomberg. Pfizer is taking an interest in the findings too, which shouldn't come as a surprise. Drugmakers and regulators have shown a willingness to move forward with drugs for small pools of patients if the drugs can deliver major benefits for those patients. With genetic tests like Foundation's catching on among drug developers, there's sure to be other new targets unearthed to propel further development of personalized cancer medicines.

"It is moving closer and closer to real personalized medicine," Janne, a co-author of the study told Bloomberg. "It is fantastic as we can tailor our therapy to the particular genetics of a patient's cancer."

- here's the release
- check out Bloomberg's article

Preclinical study triggers 'massive response' from desperate Alzheimer's patients

John Carroll — Mon, 13 Feb 2012 10:23:04 -0500

It's quite common on the R&D side of the business to see patients tracking the discovery work being done on their disease, particularly if they have an ailment that can't be effectively treated with any approved drug. Late last week, though, scientists found themselves in the spotlight after finding that an approved drug--Eisai's cancer therapy bexarotene--countered the impact of Alzheimer's in mice. And patients and their families were quick to besiege providers with requests to use the drug on an off-label basis.

"The Alzheimer's community is very desperate for anything that shows any sign of hope or promise," Eric Hall, CEO of the Alzheimer's Foundation of America, tells The Wall Street Journal.

"To patients and families who are this motivated, the idea of an off-label pill is not a major leap," neurologist Sam Gandy told the Journal. But it's also not necessarily a good idea to start taking a cancer drug for a completely unproven use. It's not at all uncommon for a mouse study to lead investigators down a blind alley, with no utility in humans. And in this case there's no solid idea of just what the prospective dosage should be, among other issues.

One of the scientists at the center of the media storm, Gary Landreth, says he's been the subject of a "massive response." He's starting a small study involving 12 subjects in a few weeks, but that's as far as the clinical plans extend for now.

The intense response underscores just how big a market awaits any drug developer that can begin to treat Alzheimer's. Ironically, though, neuroscience has become increasingly unpopular in the biopharma world, where the science is murky and patient outcomes hard to track.

- here's the story from The Wall Street Journal

Hungry Valeant snags Eyetech in ongoing buyout spree

John Carroll — Mon, 13 Feb 2012 09:26:14 -0500

Valeant Pharmaceuticals today continued its restless buyout spree, snapping up Eyetech, a small FL-based company that sells the anti-VEGF pioneer Macugen in the U.S. And while Valeant ($VRX) didn't spell out the numbers, it carefully noted that it was forking out "significantly less than two times sales" for the company.

Macugen was the first anti-VEGF therapy approved for wet, age-related macular degeneration. It was developed by the original Eyetech, which was scooped up soon after the approval by OSI. But the treatment was quickly bested by Lucentis--an event that had been widely forecast by analysts--and a chastened OSI later spun off Eyetech in 2008 with a license to market the therapy in the U.S. Pfizer ($PFE), which acquired the ex-U.S. rights, recently made an effort to garner a fresh approval for the treatment to cover visual impairment due to diabetic macular edema. But the EMA batted down that attempt last summer.

"This acquisition of Eyetech will fit nicely with our existing ophthalmology business, which includes a preservative free Timoptic in Ocudose and Lacrisert, products obtained through our acquisition of Aton in 2010," said Valeant CEO J. Michael Pearson. "The ophthalmology market has similar characteristics to the dermatology space and is a natural extension of our development capabilities. We will continue to look for future opportunities to acquire additional products and gain important critical mass in this specialty space."

Pearson has been hungrily snapping up biotechs, acquiring Biovail in 2010 and then a string of other companies. Each new buyout has been followed by the quick elimination of any overlapping operations, as Pearson underscored a few weeks ago when he vowed to slice $200 million in costs this year.

- here's the press release

U.K. charities campaign to modify pricey regs in trials directive

John Carroll — Mon, 13 Feb 2012 08:33:39 -0500

A group of charities in the U.K. is spearheading an effort to modify the EU's Clinical Trials Directive, a set of regulations which was intended to improve the safety of drug studies and which critics maintain has driven up the cost of studies and threatened to wipe out the drug research business in Europe.

According to The Telegraph, Cancer Research UK has joined a coalition that insists the directive created a battery of onerous regulations which significantly added to the cost of research without doing anything to improve safety.

"We are driving a Rolls Royce organization down a cart track, in terms of the bureaucracy and impediments that we have to overcome along the way," Cancer Research UK chief clinician Professor Peter Johnson tells the British newspaper. "The directive has just about doubled our costs for maintaining a portfolio of trials, because the cost of manpower pretty much doubled."

As an example, Johnson cites how requirements on reporting every adverse effect in a study prevented investigators from setting out to determine whether a common nutrient--with a clear safety profile--should be added to a cancer treatment. And with the number of clinical trials in the U.K. dwindling as the drug R&D industry finds itself being slashed by Big Pharma, the critics maintain that lawmakers need to act now, before the clinical trial business in Europe finds itself outsourced entirely to other, less expensive, regions of the world.

The group may have picked a poor example to make their case, though. Even if nutrients or other additives with a clear safety profile may get a pass on some of the regulations in the directive, it's unlikely that developers would be allowed to do a study of any new drug or drug combo without being required to report on adverse events. Modifying the directive in that one respect may do little to bring down the cost of any true drug study.

- here's the story from The Telegraph

Eisai loses panel vote on new Dacogen use

John Carroll — Fri, 10 Feb 2012 11:16:36 -0500

A federal advisory panel voted down Eisai's attempt to gain an approval for Dacogen as a treatment for acute myeloid leukemia. The pharma company has been trying to gain an OK to use the drug for elderly patients for whom chemo has failed. The advisers voted 10 to 3 against, making any formal approval unlikely. Story

Mouse study triggers demands for a possible Alzheimer's treatment

John Carroll — Fri, 10 Feb 2012 10:51:39 -0500

Investigators have turned in intriguing animal data suggesting that an orphan drug used to treat lymphoma may be effective in countering Alzheimer's disease. And with the news grabbing headlines around the world, researchers say they need to race to a more conclusive understanding of how the drug works in humans as physicians field demands for the treatment.

Any animal data can only offer clues to what investigators can find in human studies. In this case, the drug--bexarotene, an Eisai drug sold as Targretin--lowered levels of beta amyloid, a long-suspected culprit behind Alzheimer's, and improved the memories of the rodents in the study.

In most cases, that might inspire years of research and clinical studies to see if the treatment works in humans. There's no real consensus on what causes the disease, which afflicts millions. And companies like J&J ($JNJ) and Eli Lilly ($LLY) are devoting hundreds of millions of dollars to major trials of late-stage products in a race to a new approval that would be worth a megablockbuster return.

In this case, though, the intense interest in finding any new therapy is complicated by the fact that bexarotene is already on the market. And the lead investigator is being queried by physicians who are being asked for a prescription now by patients willing to test it on themselves.

"We've got to work fast, and we have got to be right. We can't screw this up," Gary E. Landreth, a neuroscientist at Case Western Reserve University School of Medicine, tells The Washington Post. "It has to work in humans like it works in mice or we can pick up and go home."

- here's the story from The Washington Post
- read the Bloomberg report

Special Report: Making sense of the Alzheimer's drug pipeline

NeurogesX slammed again as FDA panel spurns pain patch

John Carroll — Fri, 10 Feb 2012 10:23:04 -0500

NeurogesX ($NGSX) finds itself on the ropes this morning, with its badly battered shares taking yet another beating after an FDA panel unanimously shot down an attempt to get an approval to use its pain patch for HIV-related neuropathy.

Earlier in the week, regulators issued an internal assessment that NeurogesX never laid out a clear set of data supporting the efficacy of the patch, and that's exactly the Achilles heel that the panel zeroed in on. The experts also decided that the risk/benefit profile was acceptable to HIV patients. Though no surprise, the panel vote triggered another selloff of shares, with the stock plunging 31% to a rock-bottom 55 cents a share.

The final decision has yet to be made, but the chances of an FDA approval at this stage are negligible. Nevertheless, the company is confident in the product.

"We will continue to work closely with the FDA to address the Advisory Committee's comments as the Agency finalizes its review of our sNDA," said NeurogesX CEO Ronald Martell. "We remain confident that Qutenza(R) has the potential to address significant, unmet medical needs and to improve the quality of life for patients with HIV-PN. We would like to thank the FDA and the Advisory Committee members for their careful and thoughtful deliberation on this matter."

- here's the press release
- get the report from Bloomberg

High price of failure drives drug development costs into the stratosphere

John Carroll — Fri, 10 Feb 2012 08:47:35 -0500

The debate over the true cost of developing new drugs has created a virtual cottage industry of analysis in recent years. Critics maintain that the pharma industry has wildly overblown costs to justify the often extraordinary price it can place on a new product. Industry officials countered by citing Tufts' figure of more than a billion dollars per new treatment, by way of illustrating that there are some very rigorous analysts out there who can vouch for the mountainous expense.

Bernard Munos at InnoThink--who we picked as one of this year's most influential players in the industry--has triggered some considerable thinking, though, about whether Tufts' numbers are far too conservative. His number: $4 billion, when you account for the extraordinarily high failure rate that has afflicted Big Pharma over the past decade. And now Forbes' Matthew Herper has gone one step further, looking back over the past 15 years and breaking out the average cost of new drug development based on inflation-adjusted budgets.

For some, like Amgen ($AMGN), the average cost was a relatively reasonable $3.7 billion. That's the kind of money that can be dwarfed by a blockbuster approval. But when you switch to a company like AstraZeneca ($AZN), which has been plagued by clinical trial failures, the rate soars to $12 billion per product. Eli Lilly ($LLY), which has an R&D budget comparable to AZ's, has a higher rate of approvals, so its average is $4.5 billion.

As Herper notes, "the main expense is failure. AstraZeneca does badly by this measure because it has had so few new drugs hit the market."

Of course, a pharma company can't easily counter consumer advocates by saying it's being forced to cover a legacy of mistakes with high prices on the rare success story. But the new analysis does help illustrate the multibillion-dollar investments being made to bring new products to the marketplace. How sustainable that may be in an era where personalized drugs replace mass-marketed blockbusters, though, will occupy the industry for some time.

- here's the story from Forbes

Special Report: The 25 most influential people in biopharma today

FDA maps out long-awaited rules for the biosimilars business

John Carroll — Fri, 10 Feb 2012 07:32:14 -0500

The FDA has finally issued its eagerly anticipated draft of rules governing the development of biosimilars, laying out a roadmap for a multibillion-dollar industry that's been rapidly taking shape under the wing of big biopharma companies and a slate of multinational players.

Analysts quickly concluded that the rules largely fit a broad framework that FDA officials have been outlining for months now. Regulators will typically require developers to provide clean animal data on toxicity and compelling PK and PD data from human studies. Additional studies may be required before a developer can win approval for an "interchangeable" therapy, which would allow payers and pharmacists to automatically switch a patient to a less expensive copy.

The rules "reads largely as we expected, although a few points read as slightly more friendly to the generics industry," ISI Group analyst Mark Schoenebaum noted, according to Reuters.

The FDA, though, was very careful to detail a high standard for biosimilars. Faced with the challenge of evaluating copies of complex biologics, which can be altered simply by changing manufacturers, regulators made it clear that these new drugs will typically require expensive late-stage human studies. The science behind the target will be understood, and the discovery and early-stage work largely unnecessary, but they left an exacting hurdle for developers to clear. And each program will have to be tailored to fit the FDA's requirements.

"Instead of starting from scratch, these companies will be starting in the middle of the process," said Rachel Sherman, FDA associate director for medical policy, according to The Wall Street Journal's article. "We're trying to send the signal that it's not one-size-fits-all. It's product-by-product."

The biosimilar industry taking shape, then, is likely to offer new products which are, at least initially, not interchangeable. Due to the heavy cost of development, these new biosimilars will be offered at a discount of around 10% to 20%, but without the drastic markdowns that obliterate a small molecule's retail value in months. And that will be welcome news inside many biopharma companies, which can now look forward to managing more limited competition after a long period of exclusivity on the market.

- here's a copy of the rules
- see the Reuters report
- read the Bloomberg story
- get the article from The Wall Street Journal

One father's heartbreak inspired a $250M quest for a cure

John Carroll — Thu, 09 Feb 2012 11:38:48 -0500

Every new drug ever approved involved years of work by dedicated teams of investigators. In the case of the new cystic fibrosis drug Kalydeco from Vertex ($VRTX), the story also includes Boston businessman Joe O'Donnell, who helped raise much of the money the Cystic Fibrosis Foundation used to back the development of the treatment.

The Boston Globe today tells the touching story of O'Donnell's passionate commitment to getting the treatment pushed to an FDA approval. The groundbreaking treatment heralds the approach of a new generation of therapies intended to help treat the full range of CF patients. And that's possible because O'Donnell devoted much of the past 30 years to raising more than $250 million for the foundation following the death of his young son Joey, a victim of CF.

"We would not be where we are, had he not been there,'' foundation CEO Robert Beall tells the Globe. "He's authentic. He's a man of his word. He's not going to ask anybody for anything that he doesn't believe in.''

It all hit home when one of the parents of a young CF victim told O'Donnell how much the new treatment had helped.

"That was the moment for me,'' O'Donnell said. "I talked to Gerry (Cheevers) and, my God, the dam broke. You get emotional. It's a long slog, and I think of Joey. It's one of the reasons he was put on this Earth.''

- here's the Globe article

Altheos grabs $12.5M more, launches glaucoma trial

Mark Hollmer — Thu, 09 Feb 2012 11:18:46 -0500

Drug developer Altheos has expanded its Series A round by an additional $12.5 million, just in time for the launch of a Phase IIa dosing trial for its glaucoma treatment.

The South San Francisco startup can now herald the fact that its "A" round raised a not-insignificant $32.5 million. Existing investors Bay City Capital, Novo A/S, Canaan Partners and others contributed the additional bucks. With all the extra cash now in hand, the company says it is well-prepared to complete a Phase II clinical program and head into Phase III for ATS907. The drug is a Rho-kinase inhibitor billed as part of a new class of glaucoma drugs.

Altheos bills the trial as a "first-in-human study" that will test ATS907 to see how safe, well-tolerated and effective it is at different doses over 28 days. About 75 patents with glaucoma or ocular hypertension will take part in the trial's first portion, according to trial details posted on Clinicaltrials.gov. Another 180 patients are envisioned for the second phase, in which they will randomly receive either ATS907 or the drug latanoprost.

In August 2010, the company secured $20 million for what turned out to be the first part of its Series A round. CEO Henry Hsu noted at the time that glaucoma patients haven't had a new class of drug approved in 15 years. ATS907 is designed to lower intraocular pressure, reducing the risk of vision loss as a result.

- here's the release
- check out details of the planned trial

Takeda opens new drug development center in China

John Carroll — Thu, 09 Feb 2012 10:41:15 -0500

Japan's Takeda is adding a new drug development center in Shanghai intended to complement the international clinical work now under way in Singapore and Cambridge, MA. Both of the existing operations, including the cancer drug developer Millennium in Cambridge, work with contract research organizations to advance new treatments through the clinic. Now the Shanghai operation will draw on the international team to study new drugs catering to the fast-growing market in China. Story

Cytomedix snaps up stem cell biotech Aldagen for a bargain price

John Carroll — Thu, 09 Feb 2012 07:54:19 -0500

Just 10 months after abandoning its second effort to go public, Aldagen is selling out to a small regenerative medicine company for just $16 million in shares and the chance to earn another chunk of stock based on the clinical success of its stem cell therapies.

The news isn't likely to spur much joy among its investors. Durham, NC-based Aldagen reportedly raised more than $60 million from venture groups, a list that includes Aurora Funds, Intersouth Partners, Harbert Venture Partners, CNF Investments and Tullis-Dickerson. They agreed to buy $5 million of shares in the acquiring company, Cytomedix, to help fund a Phase II study of their lead stroke treatment. And if the trial is successful they stand to earn the lion's share of 20 million shares of Gaithersburg, MD-based Cytomedix held out in milestones.

The buyout--which hands 17% of Cytomedix ($CMXI) to Aldagen shareholders upfront--will allow Cytomedix to expand its work in the regenerative medicine field. And the CEO--who saw Cytomedix shares jump 9% on the news this morning--had no qualms touting the bargain price on the deal.

"We view the acquisition of Aldagen as an opportunistic transaction at an attractive valuation that will allow us to build and expand our new product development efforts with Aldagen's technology, intellectual property, people and clinical expertise," says Martin P. Rosendale. "In terms of maximizing opportunity for our shareholders while managing and mitigating risk, we feel this transaction is very advantageous."

Aldagen has several development programs underway for its adult stem cell technology. Its strategy is focused on the regenerative powers of stem cells that express ALDH enzymes. Its investigators have been working on stroke, critical limb ischemia and other programs.

- here's the press release
- read the story from the News & Observer