cystic fibrosis

PTC124 proves promising in cystic fibrosis trial

John Carroll — Thu, 21 Aug 2008 10:58:58 -0400

PTC Therapeutics' PTC124 proved promising in an early stage cystic fibrosis trial in Israel. PTC124 has already spurred tremendous interest as a therapy for Duchenne muscular dystrophy. And the developer believes that the therapy could prove effective in a number of genetic diseases. Last month Genzyme paid $100 million upfront and promised to pay up to $337 million in development and sales milestones to obtain co-marketing rights to PTC124.

In the current trial, researchers tested PTC124 against a type of cystic fibrosis caused by a mutation in the salt channel gene that blocks critical protein development. The therapy stops the blocking and allows the development of the proteins. That particular type of cystic fibrosis triggers half of all cases in Israel and 10 percent of the cases in the United States. Researchers say they will now push ahead with a larger trial to test the efficacy of the drug.

"We are planning a larger long-term study that will be international, in the United States and Canada and also in Europe," said Dr. Langdon Miller, chief medical officer of PTC Therapeutics.

--read the report in the Washington Post

PTC gets $25M drug development award

Maureen Martino — Wed, 16 Jul 2008 12:22:07 -0400

PTC Therapeutics has landed a $25 million award from Cystic Fibrosis Foundation to support Phase II development of PTC124. The drug is under investigation for cystic fibrosis caused by nonsense mutations. PTC gained attention last year when it announced that PTC124 was effective in treating a particular group of patients with Duchenne muscular dystrophy triggered by nonsense mutations, which is found in about 10 to 15 percent of patients. More importantly, however, the study highlighted that PTC124 could be the key to dealing with the nonsense mutations involving more than 1,800 genetic disorders like cystic fibrosis (10 percent of cases) and Hurler's disease (70 percent of cases).

"The encouraging data from the Phase IIa studies indicate that PTC may become a very important drug for the treatment of CF in the 10 percent of patients who carry nonsense mutations," said Robert J. Beall, president and CEO of the Cystic Fibrosis Foundation. "These exciting results lead us to believe PTC124 has the potential to be one of the first oral drugs on the market to address the basic defect in cystic fibrosis." PTC was a 2007 Fierce 15 winner.

- check out the release for more

Inspire Pharma shares soar 75% on CF data

Fri, 06 Jun 2008 06:59:58 -0400

Shares of Durham, NC-based Inspire Pharmaceuticals took off this morning after researchers announced that a late-stage trial of its cystic fibrosis therapy produced promising results. Patients taking the drug experienced an improvement in lung function during the 24-week study. After hearing the news on denufosol, investors pushed up Inspire's shares by more than 75 percent.

"The data from this Phase 3 trial with denufosol are encouraging for the cystic fibrosis community because it brings us one step closer to a novel treatment that addresses the basic cystic fibrosis defect," Robert J. Beall, CEO of the Cystic Fibrosis Foundation.

- read the report in the Triangle Business Journal

Inimex raises $22M in VC

Wed, 04 Jun 2008 06:59:55 -0400

Despite a recent Ernst & Young report on the funding challenges in Canada, Inimex Pharmaceuticals managed to round up a healthy $22 million in Series B funding. The company is launching the first clinical trials of an Innate Defense Regulator (IDR) drug in patients and assessing IDR drugs in a range of disease models. IDRs are novel drugs that selectively trigger the body's innate defenses, protecting against antibiotic-resistant infections and controlling inflammation. CEO John North said IDRs peptides could treat infections related to cystic fibrosis, pneumonia, inflammation and in cancer patients undergoing chemotherapy.

Morningside Venture Investments led the round, along with new investors Roche Venture Fund, Switzerland, CA-based Astellas Venture Management, and BC-based Advantage Life Science Fund II. Additional investors also participated.

- here's Inimex's release

Inspire abandons nasal spray after drug flunks trial

Wed, 23 Apr 2008 06:59:54 -0400

Disappointed by lackluster data, Inspire Pharmaceuticals has abandoned development of its epinastine nasal spray for the treatment of seasonal allergic rhinitis. Subjects treated with the nasal spray did not achieve better outcomes than those treated with placebo. In a statement, CEO Christy Shaffer said that the company won't devote any more resources to development of the spray, and that Inspire would focus on other programs. Those include a Phase III program for dry eye, another Phase III program for cystic fibrosis, and and Phase I glaucoma study.

- take a look at Inspire's release

Funds from non-profit groups flow to biotech R&D

Mon, 07 Apr 2008 06:59:57 -0400

Epix Pharmaceuticals said this morning that its development pact with the Cystic Fibrosis Foundation has mushroomed into a $50 million deal, adding $37.7 million to the $12 million already provided by the Foundation for new drug research. Epix will have to hit a series of goals for the money. And as a story in the Boston Globe makes clear, Boston-area biotech companies have been able to garner ever larger sums of cash to advance new therapies. Other examples include $25 million in research cash provided by the Juvenile Diabetes Research Foundation, $8.5 million from the Muscular Dystrophy Foundation and $6 million from the Multiple Myeloma Research Foundation.

"It's becoming a significant method of funding," Robert Coughlin, president of the Massachusetts Biotechnology Council, tells the Globe.

"By utilizing our proprietary in silico technology to model the CFTR ion channel, we were able to quickly discover novel CFTR modulators that are fueling our structure-based optimization approach," says Epix CEO Michael G. Kauffman.

- check out the press release
- read the article in the Boston Globe

Related Articles:
Epix shares dive after mid-stage depression failure. Report
Epix shares soar on Alzheimer's trial data. Report
Epix touts data on pulmonary hypertension drug. Report
Epix shares soar on $1.2B pact with GSK. Report
Epix anxiety drug fails in late-stage trial. Report

ALSO NOTED: Pharmaxis reports Phase II CF data; New Zealand publishes biotech industry stats; and much more...

Fri, 04 Apr 2008 07:59:50 -0400

> Pharmaxis announced today that a Phase II clinical trial in children with cystic fibrosis demonstrated excellent lung function improvement following three months treatment with Bronchitol that matched that achieved by the current marketed product, Genentech's rhDNase. Release

> New Zealand has release new statistics on the country's biotech industry. Release

> Malaysian officials say the country is on track to become a biotech hub by 2020. Report

> The FDA has allowed Eisai to resume a Phase I clinical trial of E2012, an Alzheimer's treatment. Release

> Big Pharma looks more and more like a big ostrich these days, Forbes suggests. The brouhaha over Vytorin and Zetia this week is a case in point. Report

Most popular this week: Lilly readies Phase III Alzheimer's trial. Report

And Finally... Researchers are pushing ahead with the first human trials of an ebola vaccine. Report

Developers seek alternatives to animal ingredients

Tue, 01 Apr 2008 07:59:57 -0400

The brouhaha over heparin has cast a spotlight on the use of animal products in therapies. It also underscores new development activities aimed at coming up with synthetic versions of drugs that will circumvent the need to source materials from animals. Altus Pharmaceuticals, for example, is developing an alternative to heparin with the help of financing from the Cystic Fibrosis Foundation.

When tainted heparin was first linked to the deaths of patients, investigators quickly focused on the role that an active ingredient extracted from pig intestines may have played. Most of those ingredients are now sourced in China and would be threatened by any serious viral outbreak in those animals.

- read the article in the New York Times

Related Articles:
Post-heparin, no oversight excuses. Report
Heparin recall spreads in Europe. Report
More heparin recalled. Report
Baxter: Fake heparin predated API. Report

Transave aims for proof-of-concept after $35M round

Mon, 31 Mar 2008 07:59:56 -0400

Venture capitalists Rich Kollender and Steve Kraus are joining the board at Transave after helping come up with $35 million in new venture backing to get the developer through to proof-of-concept data. Quaker BioVentures and Bessemer Venture Partners led the Series D, joined by TVM Capital, Prospect Venture Partners, Fidelity Biosciences, Forbion Capital Partners and Easton Capital.

"Proceeds from the financing will enable Transave to deliver Phase II proof of concept data for our lead clinical compound, Arikace (liposomal amikacin for inhalation). We are on track to have Arikace Phase II clinical data in cystic fibrosis patients that have Pseudomonas lung infections this year. In addition, Phase II bronchiectasis data will be available in the first half of 2009," said Tim Whitten, president and CEO of Transave.

The biotech describes Arikace as a form of the antibiotic amikacin enclosed in nanocapsules of lipid called liposomes. This "liposomal technology prolongs release of amikacin in the lung while minimizing systemic exposure."

- check out the press release

Vertex drug improves lung function in IIa CF trial

Thu, 27 Mar 2008 07:59:57 -0400

After stumbling on high investor expectations for its experimental hepatitis C therapy, telaprevir, Vertex garnered a dose of good news with the release of positive Phase IIa data on VX-770 for cystic fibrosis. Researchers reported that the small, 20-patient study revealed improved lung function in patients taking the oral drug. There was no discerned improvement in patients taking a placebo. Vertex shares jumped 10 percent on the news.

"While these are early data, it is unprecedented for an investigational oral compound for the treatment of CF to have such a marked effect on multiple measures of CF disease activity. We saw an average improvement in lung function of 10 percent in patients receiving the highest dose, compared to no observed improvement in patients who received placebo," said Frank Accurso, M.D., Director of the Cystic Fibrosis Center and Professor of Pediatrics at the University of Colorado School of Medicine in Denver.

"More than 10 years ago, the Cystic Fibrosis Foundation believed that Vertex's approach to treating CF by directly targeting CFTR function--the underlying mechanism of the disease--was an important opportunity to meaningfully change the course of cystic fibrosis," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation.

- see the release
- read the AP report on the data

Related Articles:
Red tide research points to cystic fibrosis therapy. Report
Investors disappointed in Vertex despite strong data. Report
Vertex refines terms on $300M public offering. Report

Gilead, Parion partner on pulmonary disease

Thu, 16 Aug 2007 06:59:57 -0400

Parion Sciences has signed on Gilead to partner up on pulmonary disease therapies. Gilead will pay $5 million up front and $5 million in exchange for an equity stake in Parion for worldwide commercialization rights to P-680. A successful development program would be worth up to $146 million in milestones for Parion. Just as significantly, the two companies say they will collaborate on new research into similar drugs

- see this release
- read the AP report for more info

Related Articles:
Gilead banking $1.3B for new deals. Report
Gilead notes success of trial for cystic fibrosis. Report
Gilead hesitant to tout drug for AIDS prevention. Report
Gilead shares surge on $2.5B Myogen buyout. Report

Genaera abandons two studies in pipeline switch

Thu, 05 Jul 2007 00:01:38 -0400

Genaera has changed gears on its pipeline strategy after shuttering two mid-stage studies--one for Lomucin as a therapy for cystic fibrosis and the other for squalamine to treat prostate cancer. The Lomucin study was abandoned for "reasons of futility" while squalamine was shelved due to trouble restarting the cancer trial. Genaera will now focus on trodusquemine for obesity and the anti-IL-9 asthma program licensed to MedImmune.

"We continue to be very encouraged by the progress of the MEDI-528 Phase II program in asthma," said CEO Jack Armstrong. "This is an asset that is below the radar screen of many, and we believe has greater present and future value than is currently recognized by the market. Separately, our trodusquemine Phase 1 study in obesity is progressing nicely through the initial cohorts in Study 101 to test safety and PK.

- see the release
- check out the AP report

Related Articles:
Genaera cuts jobs, focuses on two programs. Report
Genaera cans AMD therapy, slashes staff. Report
C&L: Genaera makes CEO's job permanent. Report

ALSO NOTED: Kosan forges deal with Pfizer; Genta cuts staff; and much more...

Tue, 19 Dec 2006 19:01:30 -0500

> Kosan stock surged on the news that it had inked a deal with Pfizer to develop a therapy for gastrointestinal diseases. Kosan gets $12.5 million up front and up to $72.5 million in milestones. Pfizer gains worldwide rights to KOS-2187, a compound that helps spur production of a hormone that promotes movement in the gastrointestinal tract. Release

> Fast on the heels of its announcement that the FDA did not approve its cancer therapy Genasense, Genta has laid off 34 workers, or about a third of its staff. Genta is still awaiting word on its European application for Genasense as a therapy for advanced myeloma. Report

> Shares of Panacos were slashed this morning after it announced that an experimental HIV therapy, bevirimat, proved disappointing in a Phase IIb trial. Patient data indicated a lower than expected response rate. Release

> Gilead Sciences announced that its Phase III AIR-CF2 (CP-AI-005) study of aztreonam lysine for inhalation for the treatment of people with cystic fibrosis who have pulmonary Pseudomonas aeruginosa met its primary efficacy endpoint. Release

> Germany's MorphoSys says it has extended its development pact with Pfizer, bringing its total potential value to more than $100 million in research support and milestone payments. Report

> The FDA has approved Johnson & Johnson's Invega, a therapy for schizophrenia. Release

> Repros Therapeutics has announced that preliminary trial data for the testosterone therapy Androxal demonstrates that it is not inferior to Androgel, which is sold by Solvay. Release

> Medigene has acquired the European rights to the dermatology product Oracea for €400 million plus milestones. Report

> Labopharm says it has made a complete response to the FDA's request for more information on the once-daily painkiller tramadol. Report

> Wyeth and Progenics have begun their second Phase III trial of intravenous methylnaltrexone for temporary gastrointestinal impairment. Report

> Serono and ZymoGenetics have begun a mid-stage trial Atacicept for rheumatoid arthritis. Report

> RheoGene, a gene and cell therapy company based in Norristown, PA, and Intrexon Corporation, a biotechnology company based in Blacksburg, VA, have announced plans to merge the two firms. Release

And Finally... The Bill & Melinda Gates Foundation is giving $47 million for a coordiated assault on a group of diseases. Release | Article

ALSO NOTED: Bronchitol gains fast track; Alimera garners $15.9M in second tranche; and much more...

Sun, 26 Nov 2006 19:01:30 -0500

> The FDA has granted fast-track status to Pharmaxis' Bronchitol as a therapy for cystic fibrosis. Investors applauded the news by driving up the value of Pharmaxis' stock about 20 percent on Friday. Pharmaxis has released positive mid-stage data on Bronchitol that demonstrates an improvement in lung function among the patients taking the drug. Pharmaxis plans a complete filing for Bronchitol by 2008. Report

> Alimera Sciences has gained $15.9 million in new venture capital as it completed its second tranche of a second round of venture financing. Release

> A federal judge last week denied an attempt to gain class action status for thousands of outstanding Vioxx claims, saying the suits were far too different to be pooled together. Report

> Oscient Pharmaceuticals announced today that the FDA considers its response on Factive for community-acquired pneumonia to be complete. Report

> A French agency has granted Transgene €25 million to identify and validate new molecular biomarkers. Release

> A syndicate of investors has provided a $10 million venture debt commitment to Alba Therapeutics. Much of that money will be used to research AT-1001 for autoimmune disorders. Release

> Akorn and India's Natco Pharma have expanded a purchase and supply agreement to include a third therapy for breast cancer. Report

> Australia's Mayne Pharma has agreed to pay $8 million for the outstanding rights to Nipent, a therapy for hairy-cell leukemia. Release

> The FDA has warned AstraZeneca that its promotional material for Seroquel is misleading and can't be distributed. Report

And Finally… Researchers are studying an African spice that contains an anti-inflammatory compound that is similar to the one in Vioxx, but hopefully won't mimic damaging side effects. Article

ALSO NOTED: Pharmaxis drug gains fast track status; EC approves UCB/Schwarz acquisition; and much more...

Tue, 21 Nov 2006 19:01:30 -0500

> Belgium-based UCB has been granted permission by the European Commission to complete it's planned acquisition of Germany's Schwarz Pharma for about $5.6. Report

> Shares of Australia-based Pharmaxis surged on news that the company's cystic fibrosis drug Bronchitol has received fast track status from the FDA. Report

> A new report finds that the pharmaceutical market in India is set to grow by 10 percent by the year 2010. Release

> The National Human Genome Research Institute has granted tens of millions of dollars to three major sequencing centers. Report

> Two researchers at University College London have used printing technology to weave polymer nanofibers together in a way that may eventually allow them to develop living tissue that can be used to repair the body. Report

> Researchers at the Salk Institute for Biological Studies have published the results of a study in which they activated the Wnt signaling system in the embryo of a chicken, which allowed it to grow back a wing that had been removed. Report

And Finally… The FDA is already receiving criticism for allowing silicone implants back on the market. Report

ALSO NOTED: Nektar closes UK site; Martines resigns from NSP; and much more...

Wed, 26 Jul 2006 20:00:50 -0400

> Nektar Therapeutics has closed a site in the U.K. that employs 35 workers. Report

> Chutes & Ladders: Sergio Martines has resigned as a director, president and CEO of NSP Pharma. Release

> Inspire Pharmaceuticals has launched a Phase III clinical trial to evaluate its drug candidate, denufosol tetrasodium Inhalation Solution, in patients with mild cystic fibrosis lung disease. Release

> Pfizer has won conditional marketing approval from the EU for Sutent as a therapy against a type of advanced kidney cancer. Report

> UCB has submitted an NDA for the approval of Xyzal (levocetirizine dihydrochloride), a new prescription antihistamine for treatment of allergy symptoms. Release

> Glaxo is seeking marketing approval in the U.S. and Europe for a new medicine to treat the symptoms of seasonal allergic rhinitis and perennial allergic rhinitis. Release

And Finally… Researchers say they've found a second DNA code which may help illuminate how cells activate genes. Article

Gilead to buy Corus for $365M

Wed, 19 Jul 2006 20:01:36 -0400

Gilead Sciences has announced that it will exercise its option to buy Seattle-based Corus Pharma, a company that focuses on respiratory and infectious diseases, for $365 million. The deal is expected to close in the third quarter of this year. In April, Gilead invested $25 million into Corus Pharma and at that time retained the option to purchase the company following the release of Phase III data. Corus' lead product is Cayston, a treatment for cystic fibrosis.

- read this Seattle Post Intelligencer report for more
- for details on the original deal between Gilead and Corus, see this press release (.pdf)

Crucell halts Aerugen development

Mon, 17 Jul 2006 20:01:39 -0400

Amsterdam-based Crucell has halted development of Aerugen, an experimental vaccine for the prevention of Pseudomonas aeruginosa infection in cystic fibrosis (CF) patients. The decision is the based on the results from a Phase III trial that demonstrated the vaccine was not effective despite positive results from earlier studies. The company emphasized that this would not affect its revenue forecast for this year.

- check out the company's press release for more

ALSO NOTED: $50M IT contract at FDA; Court asked to expedite stem cell challenge; and much more...

Thu, 15 Jun 2006 20:00:50 -0400

> The FDA has issued a $50 million contract to High Performance Technologies for IT services. Report

> Lawyers for California's stem-cell research institute have asked an appeals court for an expedited review of a lower court's decision to dismiss challenges to a state bond issue to fund new research. Report

> Bears Stearns analyst John Boris says he expects the FDA to approve Endo Pharmaceuticals' Oxymorphone pain killer. Report

> Hollis-Eden Pharmaceuticals has presented data on five drug candidates for cystic fibrosis. Report

And Finally… Avian flu has claimed another victim in Indonesia, bring the country's death toll to 38 and raising concerns that the country has not effectively countered the crisis. Article

ALSO NOTED: Study finds for-profit bias; TorreyPines gets positive data in Phase I; and much more...

Tue, 16 May 2006 20:00:50 -0400

> A new study concludes that clinical trials funded by drug companies are more likely to lead to positive results than trials funded by not-for-profit groups. Report

> TorryPines says its Phase I trial for its therapeutic candidate for acute migraines has produced positive data. Report

> Cambridge, MA-based Vertex has launched a Phase I trial of VX-770 as a therapy for cystic fibrosis. Report

> Amarin has acquired global rights to a novel oral formulation of apomorphine for the treatment of "off" episodes in patients with advanced Parkinson's disease. Release

> Genentech will collaborate with Immunogen to develop a manufacturing process for Herceptin. Release

> Intercept Pharmaceuticals has garnered $41 million in equity financing. The financing was led by Milan-based Genextra. Release

> PrimeGen Biotech has created a wholly owned subsidiary--PrimeCell Therapeutics. Release

And Finally… Indonesia has reported five more deaths from bird flu. Article