FierceBiotech

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

Celgene pays $15M for front-row seat on Acetylon myeloma program

John Carroll — Wed, 08 Feb 2012 17:04:57 -0500

Since its launch back in 2009, Boston-based Acetylon Pharmaceuticals has fueled its work on a next-gen HDAC inhibitor with $40 million drawn from wealthy individual backers as well as the Leukemia and Lymphoma Foundation. But with its lead program in a classic Phase Ia dose-escalation safety study, Celgene has stepped in with a $15 million strategic investment that buys it a front-row seat on new technology that could play a future role in a combo therapy utilizing Revlimid.

Celgene isn't gaining a license or option on ACY-1215, its HDAC6 enzyme inhibitor, CEO Walter Ogier emphasizes, but it's been keen to gain an observation post on the board. Acetylon, meanwhile, is adding some welcome commercial expertise to help guide its development work with the arrival of Mark Alles, Celgene's chief commercial officer, on the board.

The cash injection will help Acetylon get more aggressive with its development strategy, Ogier tells FierceBiotech. A Phase Ib study that combines its HDAC6 inhibitor with Millennium's Velcade is planned to start later in the year. A combo study with Revlimid is a distinct possibility. And Ogier says a mid-stage study could get underway in 2013--or 2014. The CEO isn't quite ready to tie himself down to a hard-and-fast schedule.

He is happy, though, to project an increase in staff from 13 to 20 as the biotech gets deeper into the clinic. And there's a chance that a partnership could be struck around the HDAC inhibitor's potential as a treatment for inflammation or neurodegeneration. There are other projects in those areas in which a "partnership would be well advised."

Acetylon has been building on the research work of a group of scientists that include Kenneth Anderson at Dana-Farber Cancer Institute and Stuart Schreiber at Harvard University. Its investors include Robert Kraft, owner of the New England Patriots, Marc A. Cohen, chairman of the board at Acetylon, along with Bruce L. Downey, former CEO of Barr Pharmaceuticals and Elena Prokupets, former CEO of Lenel Systems International.

- here's the press release