Insmed stock soars after FDA ends hold on key lung drug

Posted under Arikace, Blog, Companies, cystic fibrosis, Diagnostics, FDA, Funding, Insmed, Medical Devices, Medical Supply, Pharmaceuticals, Pipeline, Regulatory, Startups, Universities, Videos by rmcbride

Drug developer Insmed has rallied on news that U.S. regulators have lifted the clinical hold on its experimental lung-disease treatment Arikace. The company's stock price skyrocketed nearly 63% to $4.40 per share as of 12:07 p.m. ET after the announcement. 

This ends the clinical hold the FDA had put on the inhaled antibiotic for treating cystic fibrosis patients with Pseudomonas lung infections, the Monmouth Junction, NJ-based developer reported. Early this year, regulators withdrew a hold on the developmental treatment in patients with non-tuberculous mycobacteria, Reuters reported.

Lifting the holds is a big deal for Insmed. The company has scrapped programs in cancer and other diseases, deciding to gamble most of its resources on Arikace, an inhaled liposomal formulation of amikacin. With the clinical holds removed, the company met eye-to-eye with regulators on a revised CF trial population involving certain adults with chronic Pseudomonas infections, and the company is in talks to hammer out other details of a Phase III study.

Cystic fibrosis, which affects about 30,000 people in the U.S., is a chronic genetic disease that clogs the lungs with sticky mucus and often leads to frequent respiratory infections.

"We are pleased that FDA has lifted the clinical hold on the Arikace studies in both CF and, as previously disclosed, in patients with [non-tuberculosis mycobacteria] lung disease," Timothy Whitten, Insmed's president and CEO, stated. "We believe that Arikace has the potential to be an important treatment option for" those patients.

- here's the company's release
- check out Reuters' article

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

U.K. green lights study of pioneering gene therapy against cystic fibrosis

Posted under Blog, Clinical Trials, Companies, cystic fibrosis, Diagnostics, drug delivery systems, Funding, gene therapy, genetic mutation, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

Researchers in the U.K. plan to launch a mid-stage clinical trial this month for an inhaled gene therapy to combat cystic fibrosis (CF). The U.K.'s National Institute for Health Research and Medical Research Council have ponied up £3.1 million ($4.9 million) in funding to get the trial off the ground.

U.K. Cystic Fibrosis Gene Therapy Consortium, a nonprofit research group, will coordinate the 130-patient trial, which the consortium called the largest of its kind and will include patients aged 12 and up. Patients in the double-blind, placebo-controlled study will get a dose of the gene therapy or a placebo once per month over the course of a year, with results expected in spring 2014, according to the group's release.

Cystic fibrosis is a genetic disease that causes a buildup of thick, sticky mucus in the lungs and leads to respiratory infections and other debilitating symptoms. In the U.K. the only available treatments address only the symptoms of the chronic condition, and CF patients have an average life expectancy of 41 years, the PharmaTimes reports. The consortium has been working for more than a decade on their gene therapy, which is inhaled into the lungs to deliver molecules of DNA into cells to replace the defective gene that causes CF.

The Medical Research Council has also come up with £1.2 million ($1.9 million) to fund research of a modified virus as a vehicle to provide improved delivery of the gene therapy.

CF patients are forced to take a battery of treatments to control the symptoms of their disease, yet in recent years, companies such as Vertex Pharmaceuticals ($VRTX) have advanced treatments that address the root genetic cause of the disease rather than just the symptoms. The FDA in January stamped an approval on Vertex's Kalydeco, a targeted drug for about 4% of CF patients in the U.S. who have the G551D mutation. And the drug developer is now working on other targeted approaches to address the root cause of the disease.

- get the consortium's release
- read the PharmaTimes article

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UPDATED: Vertex nabs expedited FDA OK on breakthrough CF drug
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Langer upstart rakes in fresh venture cash for mucus-targeting drugs

Posted under Blog, Companies, cystic fibrosis, Diagnostics, Funding, Kala Pharmaceuticals, Medical Devices, Medical Supply, ocular diseases, Pharmaceuticals, Robert Langer, Startups, Universities, Videos by John Carroll

An upstart biotech company with intriguing new formulation technologies and a roster of influential backers has rounded up $6.2 million in additional seed capital. Kala Pharmaceuticals put out word about the new venture funds this morning, noting that Lux Capital, Polaris Venture Partners, Third Rock Ventures and Lighthouse Capital Partners all chipped in some of the cash. 

There are now a dozen full timers at the Waltham, MA-based biotech, says business development chief Kevin Pojasek. Most of the staffers are Ph.D.-level scientists working with the mucus-penetrating particle technology that Justin Hanes--a Johns Hopkins investigator and nanomedicine expert--initially crafted by studying the particular viruses which were able to get through the mucus barrier. Using the technology they can target a drug specifically to mucus tissue and then calibrate how long it remains active there. The technology has a wide range of possible applications, but its potential is particularly strong in cystic fibrosis, ocular disease and ailments associated with the gastrointestinal tract and female reproductive system.

Kala can now work on reformulating existing therapies, partner with biopharma on their new chemical entities and also revive failed development programs with the new approach, says Pojasek. And they're already in talks aimed at establishing partnerships with key players.

The scientific team behind Kala is a close-knit group. Hanes is a former student of the ubiquitous Robert Langer at MIT, who stepped in as a co-founder of the biotech and helped open doors with Polaris, says Pojasek. Dr. Colin Gardner, a Kala co-founder and the former CSO at TransForm Pharmaceuticals/Johnson & Johnson, did his postdoc with Langer.  

Kala has garnered a bit more than $11 million in total seed money, says Pojasek, and also announced today that it has won two grants from separate divisions of the National Institutes of Health that will advance its cystic fibrosis and ocular disease programs. Kala now has the cash needed to get through the first quarter of 2013, buying time to start working on collaborations and putting together an A round.

- here's the press release