Merck snags rights to Endocyte’s ovarian cancer drug in $1B deal

Posted under Blog, Companies, companion diagnostics, Diagnostics, EC145, Endocyte, Funding, Medical Devices, Medical Supply, Merck, ovarian cancer, Partnering, personalized medicine, Pharmaceuticals, Startups, Universities, Videos by john

Merck has scooped up worldwide rights to Endocyte's ($ECYT) late-stage ovarian cancer drug in a billion-dollar deal, paying $120 million upfront and committing up to $880 million in milestones. And West Lafayette, IN-based Endocyte will also get co-promotion rights and a split of U.S. profits while earning double-digit royalties in the rest of the world.

Investors liked the sound of big round numbers, bidding up Endocyte shares by 111% once the news hit.

Merck ($MRK) says the drug--Vintafolide, or EC145--matches the profile of what it's looking for in a cancer drug, highlighting Endocyte's development of a companion diagnostic to identify patients most likely to respond to the treatment. And Endocyte says it's only months away from filing for U.S. approval. The treatment has been given orphan drug status in Europe.

Endocyte's strategy is to use an imaging agent, EC20, to identify patients whose tumors express folate receptors. Those patients who test positive are treated with EC145, which combines a chemotherapy with a conjugate that includes vitamin folate, targeting specific cancer cells. That approach has produced positive results in mid-stage studies, with folate receptor-positive patients achieving a median 5.5 month PFS rate compared to a control arm's 1.5 months.

"Vintafolide is a promising and innovative late-stage cancer drug candidate. In addition to pursuing the lead indication of platinum-resistant ovarian cancer, Merck plans to further evaluate its potential for treatment of multiple other cancer types," said Peter S. Kim, executive vice president and president at Merck Research Laboratories. "This agreement underscores our strategy of building a portfolio of oncology therapeutics that employ a companion diagnostic to facilitate selection of those patients most likely to respond to treatment."

- here's the press release

Special Report: Endocyte - Fierce's 5 Drug Delivery Companies to Watch

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Highlights from the Leerink Swann Healthcare Conference

Posted under Blog, CDER, Companies, companion diagnostics, conference, Diagnostics, DNA, Events, FDA, Funding, gene therapy, genetics, Janet Woodcock, leadership, Leerink Swann, Medical Devices, Medical Supply, Next Generation Sequencing, Pharmaceuticals, Startups, Strategy, Technology, Universities, Videos by Ben Comer

Over a hundred public companies, dozens of clinicians, and several regulators participated in investment firm Leerink Swann’s 2012 Healthcare Conference in mid-February. Press was barred from the event, but John Sullivan, Leerink’s director of research, gives PharmExec a few of the take-aways.

Everyone knows that Dr. Janet Woodcock, CDER’s director, tends to stress the importance of safety as FDA’s top mission when speaking to a public audience. During a keynote address at the Leerink Swann Healthcare Conference in February, she also spoke about the importance of transparency and communication at FDA, specifically with respect to smaller biotech companies.

John Sullivan, Leerink Swann’s director of research, was intrigued by Woodcock’s comments. With an increase in the number of smaller companies, including small biotechs, appearing before the FDA as drug sponsors, Woodcock acknowledged that a different method of interaction, with different protocols, is necessary. “When your customers are big drug companies, they’re more likely to know the language…you have career regulatory officers on board internally, and a lot more shots on goal,” says Sullivan. “You can send them away with a previously unclear piece of guidance that hasn’t been clarified, and they’ll go do more work, confident in the knowledge that they’ll be back in front of the agency with that candidate or another one. They have a certain way of interacting that’s long-term in nature.”

With smaller companies, like emerging biotechs, however, the drug candidate up for review may be a company’s only product, or one of a very few. “There’s a heightened level of urgency that I think the FDA is now recognizing,” says Sullivan. “It sounds like they are taking specific steps to make themselves more available to smaller drug sponsors, to give them more guidance.” Hopefully, this will help to minimize the risk of a company going out and conducting new trials, only to reappear two or three years later to find out that the trial wasn’t what the FDA wanted, in terms of design, says Sullivan. “I took [Woodcock’s comments] as a positive at the margin. That doesn’t mean that this class of emerging biotechs will get more approvals out of FDA, but hopefully they’ll get the right answers, perhaps a bit sooner and for a bit less money, than what they might have anticipated.”

Woodcock has also been an outspoken proponent of drug and diagnostic combinations, and in addition to panels on new prostate cancer treatments, duel eligible and healthcare exchanges, biosimilars, rheumatology and other topics, Leerink also convened a panel on clinical DNA sequencing. The panel focused on the migration of sequencing toward the clinic. The panel consensus, according to Sullivan, was that DNA sequencing would inevitably come to the clinic, but not before two things happen: “The price has to come down, and the utility of the DNA sequence information, to the clinician and his or her patient, has to go up.” Per Sullivan, more links between genetic presentation and predisposition for disease, or response to therapy, need to be made. The platforms by which cancer diagnosis and treatment are used today are “relatively low-density…they can do maybe ten genes or 50 genes,” says Sullivan. With full genome sequencing, researchers can do tens of thousands of genes at once, but “most clinicians don’t need, nor do they know what to do with that volume of information.” Panelists said that as clinicians learn more about genetic information, and the price of a full scan comes down, patients will start to be scanned more often.

“I think there was broad agreement that DNA sequencing, especially single-molecule sequencing – where you don’t have the risk of mistakes as you amplify the amount of genetic material for analysis – really holds the promise of being more accurate than other methods of interrogating DNA and RNA, so it’s attractive from the standpoint of physicians, who want to make sure they’re giving the right answer. DNA sequencing, especially single molecule sequencing, increases those odds,” says Sullivan.

On the issue of price, Illumina and Life Technologies announced in the respective launches of two platforms that can sequence an entire genome for $1,000, in one day’s time. Sullivan called Illumina, which is currently subject to a hostile takeover attempt by Roche (the bid is currently $5.7 billion), a “leader in research-based DNA sequencing today.” Ion Torrent, acquired in 2010 by Life Technologies (which was itself formed through the merger of Invitrogen Corp and Applied Biosystems, in 2008) released a mock-up photograph of a clinical DNA sequencing instrument small enough to fit in the hand of a physician. While still a ways off, the photo suggests DNA sequencing in the clinic will require a set of tools that differs from what’s currently used in the lab, by researchers, says Sullivan. “Clinicians and clinical lab staffers have very different needs than research lab PhDs,” he says. “As far as the drug industry is concerned, they’ll continue to get good ideas for new products from the genetic research studies that are going on in a million academic research labs all over the world, and they will increasingly look to pair their drugs – whether genetically derived or not – with diagnostics.”

Big trouble seen for targeted cancer drugs

Posted under Blog, Cancer Drugs, Companies, companion diagnostics, Diagnostics, DNA test, Funding, Medical Devices, Medical Supply, Novartis, personalized medicine, Pfizer, Pharmaceuticals, Roche, Startups, targeted therapies, Universities, Videos by Ryan McBride

Researchers have discovered that the genetic complexities of cancer could thwart current methods of matching a patient's tumor with targeted drugs. The study by U.K. researchers could mean that more extensive digging into the genetics of cancer will be required before dispatching targeted treatments to attack tumors, a change that could drive up the already high cost of cancer care.

With an arsenal of targeted cancer drugs at their disposal now, doctors use DNA tests, typically of a single tumor specimen from their patients, to determine which meds are the best fit to take out their tumors. Alas, U.K. researchers conducted DNA tests from multiple areas of kidney tumors and found that only one third of all the key genetic mutations were present in all tumor samples, The Wall Street Journal reported.

The study is unlikely to unhinge enthusiasm about targeted cancer meds, but the findings could prompt doctors to take extra caution before using the drugs. What's more, the results could explain how cancers eventually bounce back after gene-targeted drug treatments, Bloomberg reported. And, rather than forming to overcome the effects of targeted drugs, the mutations that drive a cancer's comeback could already be present before treatment with the meds.

The targeted cancer drug phenomenon has pushed many of the top drugmakers into action, with Pfizer ($PFE), Roche ($RHBBY), and Novartis ($NVS) among the leading developers of such meds. It's probably too early to tell how the results of the U.K. study impact those companies' fortunes, but it's worth watching how the study impacts enthusiasm in the field.

"It's a sobering finding," Andrew Futreal, a co-author of the study and former director of cancer genetics and genomics at Wellcome Trust Sanger Institute in London, said, as quoted by the WSJ. He added that, with just one biopsy, "you might miss the connection between the mutation and whatever outcome you are looking at."

- read Bloomberg's article
- check out the WSJ piece

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Roche gets key nod for targeted skin cancer drug en route to EU approval

Posted under Blog, Cancer Drugs, Companies, companion diagnostics, Diagnostics, Funding, Medical Devices, Medical Supply, Melanoma, Pharmaceuticals, Roche, Startups, Universities, Videos, Zelboraf by Ryan McBride

A standout drug for "personalized" skin cancer treatment has gained a key endorsement from European regulators. Roche ($RHHBY) said that the European Medicines Agency backed the targeted melanoma drug for approval. The Swiss drug giant hopes to secure from the European Commission in February.

The drug--developed by Daiichi Sankyo-owned biotech Plexxikon in Berkeley, CA--is intended to treat the aggressive forms of melanoma in which BRAF gene mutations are found, and Daiichi Sankyo's partner Roche is providing a companion diagnostic that screens patients for the mutation prior to treatment. The FDA approved the drug in August, after giving the market green light to Bristol-Myers Squibb's ($BMY) Yervoy for melanoma. Analysts expect sales of Zelboraf--which costs $56,000 for a six-month course of treatment--to reach nearly $1 billion in sales in 5 years, Reuters reported.

Zelboraf combats the deadliest form of skin cancer, and, unfortunately, one that has been on the rise. Globally, there were 200,000 new cases of melanoma in 2008, up from 160,000 cases in 2002, and the cancer kills about 40,000 people per year, according to figures cited by Roche. With fewer than one in four patients with the cancer expected to live beyond a year, Roche impressed clinicians last year with data that the drug, known generically as vemurafenib, reduced patients risk of dying by 63% during treatment compared to those on standard chemo treatments.

"The (Committee for Medicinal Products for Human Use) recommendation to approve Zelboraf represents an important milestone for people with metastatic melanoma who until recently had limited treatment options," Dr. Hal Barron, Roche's chief medical officer and head, global product development, said in a statement. "We are working closely with health authorities worldwide to bring Zelboraf to people with this deadly disease as soon as possible."

- here's the release
- read Reuters' coverage

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