UPDATED: Vertex sizzles with upbeat PhII data on combo therapy for cystic fibrosis

Posted under Blog, Clinical Trials, Companies, cystic fibrosis, Diagnostics, Funding, Kalydeco, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, vertex, Videos, VX-809 by rmcbride

Vertex Pharmaceuticals ($VRTX) has nailed down some promising mid-stage data for a combo treatment against the genetic lung disorder cystic fibrosis. An interim analysis of the Phase II study showed that patients on the company's drug Kalydeco and an experimental treatment had significantly improved lung function compared with those on placebo, Vertex announced.

The data indicate that Vertex might have a remedy for treating a large number of the 70,000 people worldwide with CF, and the company's share price had jumped nearly 46% to $54.69 as of 12:16 pm ET today. 

There was a 5% improvement in lung function in 46% of patients on the recently approved Kalydeco and VX-809, and 30% of patients on the combo saw at least a 10% boost in lung function, according to the company. A 4%-6% improvement in lung function in the study would be a "home run," an analyst from RBC Capital Markets wrote last month, according to Bloomberg's report on the study. Yet Forbes' pharma editor Matthew Herper cautions that the study isn't complete and the combo hasn't met everyone's expectations.

Once the company wraps the Phase II study and talks to regulators, Vertex aims to race ahead with a pivotal study of the Kalydeco-VX-809 combo for treating the root cause of the genetic disease in patients with two copies of the F508del mutation. Almost half of all patients with the disease, which causes thick and sticky mucus to build up in the lungs, have two copies of the mutation, according to the company. Kalydeco, the first drug approved to combat the underlying cause of CF, has approval for treating patients with a mutation that signifies about 4% of patients with the disease, Bloomberg reported.

"People with two copies of the F508del mutation have one of the most severe forms of cystic fibrosis. In these patients, the combination of VX-809 and Kalydeco led to significant improvements in lung function that exceeded our expectations," Dr. Chris Wright, Vertex's senior vice president, global medicines development and medical affairs, stated. "We look forward to beginning discussions with regulatory agencies later this year when we have full data from the study, with the goal of moving forward with a pivotal study as quickly as possible."

The FDA has shown that the agency will take quick action on drugs that prove effective against life-threatening diseases such as CF, which leads to premature death. As Bloomberg noted, the agency stamped an approval on Kalydeco after a three-month review.

- here's the release
- check out Bloomberg's article
- see Herper's piece
- and TheStreet's coverage

Related Articles:
Vertex nabs expedited FDA OK on breakthrough CF drug
Vertex backs up pricey new CF drug with co-pay help
One father's heartbreak inspired a $250M quest for a cure

Editor's note: Updated stock price at 12:20 pm ET on 5/7/12.

Vertex chief earned $13.4M in ’11

Posted under Biotech CEO pay, Blog, Companies, Diagnostics, Funding, Matthew Emmens, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, vertex, Videos by john

Former Vertex Pharmaceuticals ($VRTX) CEO Matthew Emmens left the chief executive position after reaping $13.4 million in total compensation last year, according to the Boston Business Journal. Despite the fact that Emmens was widely credited with ushering the biotech through a major transition--winning an approval on a game-changing new hepatitis C treatment--the earnings reflect a drop from the $14.9 million in compensation he received for 2010. Story

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

Related Articles:
Vertex outlines its survival plan for next-gen hep C combo
Vertex advancing all-oral Hep C pipeline amid big 3Q profits

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

Posted under Blog, Companies, cystic fibrosis, Diagnostics, Funding, Kalydeco, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, vertex, Videos by John Carroll 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 Related Article: UPDATED: Vertex nabs expedited FDA OK on breakthrough CF drug