Vertex vows to race ahead with interferon-free combo for hep C

Posted under Alios BioPharma, Blog, Companies, Diagnostics, Funding, Hepatitis C, Incivek, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, vertex, Videos by John Carroll

Determined to try and keep pace with rival next-gen hepatitis C treatments in the pipeline, Vertex ($VRTX) today unveiled a slate of somewhat positive interim midstage results for an all-oral combo regimen that includes its game-changing drug Incivek along with the experimental VX-222--its non-nucleoside polymerase inhibitor--and ribavirin.

The standard goal for new hepatitis C drugs is the sustained elimination of all signs of the virus by week 12, allowing patients to stop treatment, though developers are aiming for the fastest cure rates possible. Vertex noted that 11 of 46 treatment naïve patients with the genotype 1a and 1b virus met the criteria of having "undetectable hepatitis C virus at weeks two and eight of treatment and were therefore eligible to stop all treatment at 12 weeks. Nine of these 11 patients achieved SVR4 (undetectable hepatitis C virus four weeks after the end of all treatment)." Data showed that viral loads were below the "lower limit of quantification" for 83% of the patients at week 12.

Based on the data, Vertex says it will push ahead with a Phase IIb study of the interferon-free combo, anticipating that investigators can nail late-stage data for an NDA to the FDA as early as late 2014--keeping on an ambitious development schedule.

"Our ultimate goal is to develop well-tolerated, interferon-free treatment regimens with high viral cure rates and short treatment durations for people with hepatitis C," said Vertex CSO Peter Mueller. "We believe we're well-positioned to achieve this goal by exploring various combinations within our portfolio that includes Incivek, VX-222 and two structurally-distinct nucleotide polymerase inhibitors."

Those two nucleotide polymerase inhibitors he referred to are ALS-2200 and ALS-2158, which were licensed in from Alios and perhaps represent Vertex's best shot at gaining an edge over treatments being studied at rival companies. Vertex said today that it has begun the first 7-day viral kinetic studies of ALS-2200 and ALS-2158 in people with genotype 1 hepatitis C. Safety and viral kinetic data from these studies are expected in the second quarter of 2012, enabling the launch of midstage studies in the second half of 2012.

- here's the press release

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Merck spinout nails promising study on hep C vaccine

Posted under Blog, Companies, Diagnostics, Funding, Hepatitis C, Incivek, Medical Devices, Medical Supply, Merck, Okarios, Pharmaceuticals, Startups, Universities, vertex, Videos by John Carroll

A group of European investigators working in league with a small biotech spun out of Merck ($MRK) four years ago has claimed an early-stage success with a small human trial that has generated positive efficacy results for an experimental hepatitis C vaccine. By targeting some of the consistent internal mechanisms found behind the ever-changing façade of the hepatitis C virus, the researchers say that their new vaccine spurred an immune response similar to that seen in people with a strong natural immunity. And they believe that the results warrant further human studies to see how it might fit into future cocktail therapies for the big population of patients.

The spinout, Okairos, was set up to develop genetic vaccines. In this trial, which involved 41 subjects, scientists took cold viruses and adapted them using genetic material taken from the hepatitis C virus. The modified viruses were used to spur an immune response to hepatitis C, and the primary goal of establishing the safety of the therapeutic vaccine was accomplished.

Of course, a small Phase I study is a long, long way from pivotal data. But in a world that's looking for new cocktail approaches to hepatitis C, as well as a jab to guard against new cases, it's a promising start.

"The immune responses we've seen are exciting and we are beginning the next stage of trials," says Oxford University Professor Paul Klenerman. "While we are hopeful, it could be a long road to any vaccine that protects people against hepatitis C."

Hepatitis C has become one of the hottest fields in drug research today. The enormous success of Vertex's ($VRTX) new drug Incivek has fed hopes for future treatments that will dispense with interferon, which is linked to some common side effects which often prevent patients from taking a therapy. And that has encouraged developers who are pursuing new drugs that are likely to be paired in future combinations.

- get the BBC report
- here's the Reuters story

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Interview: Vertex CEO concerned about investors’ ‘hyper-focus’ on hep C

Posted under Blog, Companies, Diagnostics, Funding, Hepatitis C, Incivek, Jeffrey Leiden, Matt Emmens, Matthew Emmens, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, vertex, Videos by Ryan McBride

Matthew Emmens

On the heels of Vertex Pharmaceuticals' ($VRTX) big news last week about changing CEOs next year, FierceBiotech chatted with both current chief executive Matt Emmens (picture, top) and his successor Dr. Jeffrey Leiden (pictured, bottom). Both Emmens and Leiden are heavyweights in the biopharma industry, with hugely successful drug launches under their belts.

Still, Vertex needs another hit to follow the successful approval and launch of its hepatitis C drug Incivek this year, and it could have one in the experimental cystic fibrosis drug Kalydeco now under FDA review. Much of the company's stock growth of recent years has wilted over the past 6 months on fears that Incivek faces lots of competition in the years ahead from Merck's ($MRK) rival drug and interferon-free treatments against hep C in development, which analysts believe could rapidly eat into Vertex's Incivek business if approved.

Jeffrey Leiden

Emmens, a 60-year-old biopharma veteran who headed Shire ($SHPGY) prior taking over as CEO of Vertex in 2009, is handing over his chief executive duties to Dr. Leiden on Feb. 1 and will serve as executive chairman until May. Leiden, 56, has served on the board of Vertex since 2009, and he made a name for himself in past leadership roles at Abbott Labs ($ABT), where he oversaw the launch of the blockbuster rheumatoid arthritis drug Humira.

Here's an edited version of FierceBiotech's conversation with Emmens and Leiden on Tuesday:

FierceBiotech: What has been your biggest mistake as CEO of Vertex?

Emmens: You ask tough questions, you know that [laughing]. I don't see any mistakes. I don't know if I'd do anything different. People are going to second-guess a lot of things. But when you look retrospectively at things, without all the information, that's easy to do. When you're there, the decisions that you have to make at the time have quite a different face on them. I don't think I would have done anything differently knowing what I knew then. Knowing what I know now, yeah, in the drug industry I think I would have gone back and bought Genentech when it was three people.

FierceBiotech: What's your biggest concern about Vertex's business heading into 2012?

Emmens: My biggest concern is the way it's perceived by some in the investment community. I think you have a company here that has the best pipeline that I've ever seen in any company in terms of numbers of projects that are successfully proven in man and in a time frame that's not 15 years out. You see, basically, 8 projects here that could all be on the market by [2017], and you see three or four that could be on the market by [2015]. That's very nice, I think. The hyper-focus on hepatitis C has taken people's focus away from that. And I think on top of that, in hepatitis C, we've got a number of projects that could put us in an all-oral regimen in the time frame of [2014]. That's being overlooked.

FierceBiotech: Do you see yourself being a biopharma CEO again after you leave your executive role at Vertex in May?

Emmens: No. I've been working since I was 11 years old. I retired once from Shire, as you probably know. I was on the board here at Vertex and I saw a unique opportunity to use my skill set at a time that the company needed it. I would not be willing to do that for the long term again. I'll be happy to be on boards and advise and help people who are earlier in their career be successful.

FierceBiotech: According to an online poll, lots of people believe that you are the best CEO in biotech this year. Do you agree with them?

Emmens: They were obviously deluded [laughing]. I thank those who voted and it's a nice honor but I have no idea whether I agree with them or not. I did the best I could, and I try to do the best job I can everywhere I've been. And I came here to help the company along into the commercial realm and be successful, and that's happened.

[FierceBiotech's Q&As with Leiden]

FierceBiotech: What's one meaningful change that you plan to bring to Vertex as CEO?

Leiden: I've been very involved in the strategy that Matt and the team have crafted on the management side and the board side. I'm very supportive of that strategy, and I don't anticipate any major changes to it.

FierceBiotech: Did you have any doubts about whether you should take the CEO job?

Leiden: There's no question that Matt is leaving some big shoes to fill, and, frankly, so did Josh Boger. There have been two fantastic CEOs of this company. So that's always a challenge. On the other hand, I see the opportunities at Vertex looking forward are just fantastic. I've had the good fortune to look at a number of biotech and pharma CEO jobs over the last 5 or 6 years, and this was absolutely a unique opportunity because of the pipeline, the team and the proven record of execution here. The job now is to come here and execute over and over, just like they executed on [Incivek and Kalydeco].

FierceBiotech: As a physician and scientist by training, do you see yourself getting more involved in R&D than Matt has been as CEO?

Leiden: I really see my job as integrating the R&D-scientific-medical function with the commercial function. It's what I did at Abbott and I think it made a difference at Abbott. One of the problems that biopharmas have had is a disconnect between the discovery organization, which first makes the molecules, the development organization, which takes it through human trials to approval, and the commercial organization, which obviously brings [a treatment] to patients and the market. The discovery organization makes drugs that are difficult to develop. The development organization develops them in a way that makes it difficult to sell them. So the real key to the success of a modern-day biotech company is a highly integrated organization.

FierceBiotech: Does Vertex spend enough money on R&D, or too little, to keep growing?

Leiden: I've never seen an R&D organization spend as relatively little money and produce so much. The key metric at Vertex is to look at the productivity per dollars spent, and I would hold that up against any company out there--Pfizer, Merck or Genentech. We're spending an 8th to a 10th of what most of the Big Pharmas are spending and producing more.

Vertex CEO stepping down after banner year

Posted under Blog, Companies, cystic fibrosis, Diagnostics, Funding, Incivek, Matthew Emmens, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, vertex, Videos by Ryan McBride

Apparently, Matthew Emmens wanted to announce his departure from his CEO post at Vertex Pharmaceuticals ($VRTX) at what could be considered the pinnacle of his tenure at the company. Emmens, who was voted biotech's top CEO in a recent poll after a huge year at the Cambridge, MA-based drug company, plans to step down from his CEO post in February. Dr. Jeffrey Leiden, a Vertex board member and venture capitalist, has been tapped to be the company's next chief executive and will start work in that role on Feb. 1.

Emmens, who took the reins at Vertex in 2009, plans to stay on as executive chairman of the company through May 2012 and then remain on Vertex's board of directors, the company announced Thursday. A self-made success in the biopharma industry, Emmens has led Vertex through its most prosperous year of its 20-year history, with key approvals and a successful launch of the company's hepatitis C drug Incivek--a blockbuster in the making--and a potentially game-changing therapy for cystic fibrosis patients under FDA review. Yet the company faces some future challenges in the hep C market, with Pharmasset's ($VRUS) interferon-free treatment against the disease in late stage development. Pharmasset's drug could offer an effective treatment for the liver-damaging disease without the side effects of treatments on the market that rely on interferon like Incivek.

Emmens' successor, Leiden, has had front-row seats for the exciting times at Vertex as a member of the board since mid-2009. He comes to the CEO job after spending the past 5 years as a managing director of Clarus Ventures, which invests in early-stage drug developers and other life sciences companies. Leiden, 56, has a unique mix of commercial and scientific experience. Prior to becoming a VC, he was president and chief operating officer at healthcare giant Abbott Labs ($ABT), where he played a role in bringing the company's blockbuster TNF drug Humira and other treatments to market. A cardiologist and molecular biologist by training, Leiden spent previous years as a practicing physician an academic, with appointments at the University of Chicago, Harvard Medical School and Brigham and Women's Hospital.

With the commercial success of Incivek at his back, Leiden has a big opportunity to keep building Vertex. The FDA has granted priority review and an April 18 action date for the company's cystic fibrosis drug Kalydeco, a potential first approved treatment that addresses the root cause of the disease, which causes the lungs to fill with sticky mucus and other symptoms that lead to premature death. The median predicted lifespan of a CF patient is 38 years.

"I am proud of the accomplishments we have made during my almost three years at Vertex and believe the company is well-positioned to bring forward additional innovative new medicines," Emmens said in a statement this morning. "Jeff brings significant and broad leadership experience to Vertex, and I am confident that his understanding of the company, combined with his unique blend of scientific, commercial and financial expertise, will help Vertex deliver on its goals in the future."

- here's Vertex's release

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