Gilead Sciences Inc. today announced that the Anti-Infective Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) has recommended that aztreonam for inhalation solution be approved for the treatment of infections due to Pseudomonas aeruginosa (P. aeruginosa) in patients with cystic fibrosis (CF). The committee voted 15 to 2 that Gilead has provided sufficient evidence of the safety and efficacy of aztreonam for inhalation solution. The panel also voted 17 to 0 that aztreonam for inhalation solution 75 mg three times daily is a correct dose and regimen.
The recommendations of the Advisory Committee are not binding but will be considered by the FDA as the agency completes its review of Gilead’s application. The FDA has established a target review date, under the Prescription Drug User Fee Act (PDUFA), of February 13, 2010. In the interim, Gilead will continue to make the product available through its Expanded Access Program in the United States.
“Effectively treating infections in patients with CF is very challenging, and new treatment options are urgently needed,” said Norbert Bischofberger, PhD, Executive Vice President, Research and Development and Chief Scientific Officer, Gilead Sciences. “We will continue to work closely with the FDA as it completes its review of aztreonam for inhalation solution.”
CF is a chronic, debilitating genetic disease that affects the respiratory and digestive systems of approximately 70,000 people worldwide. Chronic pulmonary infections due to P. aeruginosa are the single greatest cause of morbidity and mortality among patients with CF.
Gilead originally submitted the NDA for the potential product in November 2007. In September 2009, the product was granted conditional marketing approval in Canada and the European Union under the trade name Cayston(R) (aztreonam lysine 75 mg powder and solvent for nebuliser solution). Applications for marketing approval of Cayston are also pending in Australia, Switzerland and Turkey.
Aztreonam for inhalation solution is an antibiotic candidate for people with cystic fibrosis who have P. aeruginosa. Aztreonam has potent in vitro activity against Gram-negative bacteria such as P. aeruginosa. Aztreonam formulated with arginine is an FDA-approved agent for intravenous administration for treating various infections. Aztreonam formulated with lysine is a proprietary formulation of aztreonam developed specifically for inhalation. It has been designated with orphan drug status in the United States and European Union.
In the United States, aztreonam for inhalation solution has not yet been determined by the FDA to be safe or efficacious in humans for its ultimate intended use.
Gilead Sciences Inc. today announced that the Anti-Infective Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) has recommended that aztreonam for inhalation solution be approved for the treatment of infections due to Pseudomonas aeruginosa (P. aeruginosa) in patients with cystic fibrosis (CF). The committee voted 15 to 2 that Gilead has provided sufficient evidence of the safety and efficacy of aztreonam for inhalation solution. The panel also voted 17 to 0 that aztreonam for inhalation solution 75 mg three times daily is a correct dose and regimen.
The recommendations of the Advisory Committee are not binding but will be considered by the FDA as the agency completes its review of Gilead’s application. The FDA has established a target review date, under the Prescription Drug User Fee Act (PDUFA), of February 13, 2010. In the interim, Gilead will continue to make the product available through its Expanded Access Program in the United States.
“Effectively treating infections in patients with CF is very challenging, and new treatment options are urgently needed,” said Norbert Bischofberger, PhD, Executive Vice President, Research and Development and Chief Scientific Officer, Gilead Sciences. “We will continue to work closely with the FDA as it completes its review of aztreonam for inhalation solution.”
CF is a chronic, debilitating genetic disease that affects the respiratory and digestive systems of approximately 70,000 people worldwide. Chronic pulmonary infections due to P. aeruginosa are the single greatest cause of morbidity and mortality among patients with CF.
Gilead originally submitted the NDA for the potential product in November 2007. In September 2009, the product was granted conditional marketing approval in Canada and the European Union under the trade name Cayston(R) (aztreonam lysine 75 mg powder and solvent for nebuliser solution). Applications for marketing approval of Cayston are also pending in Australia, Switzerland and Turkey.
Aztreonam for inhalation solution is an antibiotic candidate for people with cystic fibrosis who have P. aeruginosa. Aztreonam has potent in vitro activity against Gram-negative bacteria such as P. aeruginosa. Aztreonam formulated with arginine is an FDA-approved agent for intravenous administration for treating various infections. Aztreonam formulated with lysine is a proprietary formulation of aztreonam developed specifically for inhalation. It has been designated with orphan drug status in the United States and European Union.
In the United States, aztreonam for inhalation solution has not yet been determined by the FDA to be safe or efficacious in humans for its ultimate intended use.
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12.08.2009
U.S. Food and Drug Administration staff cited concerns today with the design and analysis of clinical studies intended to support the effectiveness of one of Gilead Sciences Inc.‘s experimental cystic fibrosis drug.
Gilead, the large U.S. biotechnology company, is seeking approval for its inhaled drug aztreonam for treating respiratory symptoms and pulmonary function in some cystic fibrosis patients.
In looking at Gilead’s application ahead of an FDA advisory panel on the medicine on December 10, FDA staff reviewed two clinical studies designed to support the efficacy of Gilead’s aztreonam, a reformulated version of an intravenous antibiotic.
In one study of 211 patients, FDA staff said dropouts due to adverse events and other reasons may have led to biases favoring the Gilead drug. A company analysis of the study also may have been flawed, the staff said.
“There are concerns that assumptions used in the analysis could have been determined retrospectively,” the staff said in their 48-page briefing document.
In a second study of 164 patients, staff raised several concerns about a questionnaire used to determine how a patient’s symptoms may have been affected during the study.
The problems cited included missing data from incomplete questionnaires, and unclear and vague responses.
“There are several concerns about these questions and the responses,” the staff said.
Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system of about 70,000 people worldwide, according to the Cystic Fibrosis Foundation.
The drug won marketing approval in September in Europe and Canada under the brand name Cayston.
The FDA said in September 2008 it would not approve the drug unless the company conducted an additional trial, surprising Wall Street. Gilead’s appeal of the decision was denied, but the agency agreed to an advisory panel review of the drug, according to the company.
U.S. Food and Drug Administration staff cited concerns today with the design and analysis of clinical studies intended to support the effectiveness of one of Gilead Sciences Inc.‘s experimental cystic fibrosis drug.
Gilead, the large U.S. biotechnology company, is seeking approval for its inhaled drug aztreonam for treating respiratory symptoms and pulmonary function in some cystic fibrosis patients.
In looking at Gilead’s application ahead of an FDA advisory panel on the medicine on December 10, FDA staff reviewed two clinical studies designed to support the efficacy of Gilead’s aztreonam, a reformulated version of an intravenous antibiotic.
In one study of 211 patients, FDA staff said dropouts due to adverse events and other reasons may have led to biases favoring the Gilead drug. A company analysis of the study also may have been flawed, the staff said.
“There are concerns that assumptions used in the analysis could have been determined retrospectively,” the staff said in their 48-page briefing document.
In a second study of 164 patients, staff raised several concerns about a questionnaire used to determine how a patient’s symptoms may have been affected during the study.
The problems cited included missing data from incomplete questionnaires, and unclear and vague responses.
“There are several concerns about these questions and the responses,” the staff said.
Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system of about 70,000 people worldwide, according to the Cystic Fibrosis Foundation.
The drug won marketing approval in September in Europe and Canada under the brand name Cayston.
The FDA said in September 2008 it would not approve the drug unless the company conducted an additional trial, surprising Wall Street. Gilead’s appeal of the decision was denied, but the agency agreed to an advisory panel review of the drug, according to the company.
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09.23.2009
Gilead Sciences, Inc. announced today that the European Commission has granted conditional marketing authorization for Cayston(R) 75 mg powder and solvent for nebuliser solution (aztreonam lysine) for the suppressive therapy of chronic pulmonary infections due to Pseudomonas aeruginosa (P. aeruginosa) in patients with cystic fibrosis aged 18 years and older. Cayston will be made available in certain countries of the European Union, subject to the requirements of national authorities, beginning in early 2010.
CF is a chronic, debilitating genetic disease that affects the respiratory and digestive systems of approximately 70,000 people worldwide, including 35,000 people in the European Union. Chronic pulmonary infections due to P. aeruginosa are the single greatest cause of morbidity and mortality among patients with CF.
Cayston is an inhaled antibiotic that is dosed three times per day over a 28-day period. Cayston is delivered via the Altera(R) Nebuliser System, a drug-specific delivery device from the eFlow(R) Technology Platform, developed by PARI Pharma GmbH. PARI Pharma also contributed to the development of Cayston’s drug formulation for delivery with the Altera Nebuliser System.
“Cayston is an important addition to the fight against infection in people with cystic fibrosis, and its approval will bring a further treatment option to patients suffering from this debilitating disease,” said Stuart Elborn, Professor of Respiratory Medicine, Queens University of Belfast. “The cystic fibrosis community is very pleased that the EMEA has recognized the importance of bringing forward this effective new treatment.”
The approval of Cayston is based on the results of two Phase III, single-course, placebo-controlled clinical studies and the results of an open-label, multiple-course extension study of patients who participated in the other studies.
Conditional marketing authorizations are granted to products that address unmet medical needs and whose availability would result in a significant public health benefit. Consideration for full marketing authorization is contingent upon the completion of an additional ongoing Phase III study examining the efficacy and safety of Cayston compared to tobramycin nebuliser solution in CF patients with pulmonary P. aeruginosa.
Applications for marketing approval of Cayston are currently pending in Australia, Switzerland and Turkey. In September 2009, Cayston was approved in Canada. Earlier this year, the U.S. Food and Drug Administration requested that Gilead conduct an additional clinical study before resubmitting its New Drug Application. In the United States, Cayston remains an investigational compound that has not yet been approved for use.
Gilead Sciences, Inc. announced today that the European Commission has granted conditional marketing authorization for Cayston(R) 75 mg powder and solvent for nebuliser solution (aztreonam lysine) for the suppressive therapy of chronic pulmonary infections due to Pseudomonas aeruginosa (P. aeruginosa) in patients with cystic fibrosis aged 18 years and older. Cayston will be made available in certain countries of the European Union, subject to the requirements of national authorities, beginning in early 2010.
CF is a chronic, debilitating genetic disease that affects the respiratory and digestive systems of approximately 70,000 people worldwide, including 35,000 people in the European Union. Chronic pulmonary infections due to P. aeruginosa are the single greatest cause of morbidity and mortality among patients with CF.
Cayston is an inhaled antibiotic that is dosed three times per day over a 28-day period. Cayston is delivered via the Altera(R) Nebuliser System, a drug-specific delivery device from the eFlow(R) Technology Platform, developed by PARI Pharma GmbH. PARI Pharma also contributed to the development of Cayston’s drug formulation for delivery with the Altera Nebuliser System.
“Cayston is an important addition to the fight against infection in people with cystic fibrosis, and its approval will bring a further treatment option to patients suffering from this debilitating disease,” said Stuart Elborn, Professor of Respiratory Medicine, Queens University of Belfast. “The cystic fibrosis community is very pleased that the EMEA has recognized the importance of bringing forward this effective new treatment.”
The approval of Cayston is based on the results of two Phase III, single-course, placebo-controlled clinical studies and the results of an open-label, multiple-course extension study of patients who participated in the other studies.
Conditional marketing authorizations are granted to products that address unmet medical needs and whose availability would result in a significant public health benefit. Consideration for full marketing authorization is contingent upon the completion of an additional ongoing Phase III study examining the efficacy and safety of Cayston compared to tobramycin nebuliser solution in CF patients with pulmonary P. aeruginosa.
Applications for marketing approval of Cayston are currently pending in Australia, Switzerland and Turkey. In September 2009, Cayston was approved in Canada. Earlier this year, the U.S. Food and Drug Administration requested that Gilead conduct an additional clinical study before resubmitting its New Drug Application. In the United States, Cayston remains an investigational compound that has not yet been approved for use.
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10.15.2009
NanoBio Corporation today announced compelling preclinical data for NB-401, a nebulized nanoemulsion-based agent that kills highly drug-resistant strains of bacteria commonly found in cystic fibrosis patients. Currently there are limited treatment options available that effectively address these resistant bacteria. The study results are being presented today at the 2009 Annual North American Cystic Fibrosis Conference (NACFC) in Minneapolis, Minn.
“Given that cystic fibrosis patients are susceptible to respiratory failure following repeated bacterial infections of the lung, there is a tremendous need to develop new treatments that can overcome drug-resistant bacteria,” said James R. Baker, Jr., MD, NanoBio’s CEO and founder. “In laboratory studies, NB-401 is a very potent killer against these difficult strains.”
NB-401 has been shown to be highly efficacious in vitro against Pseudomonas aeruginosa, Burkholderia, Acinetobacter, Stenotrophomonas and other multidrug-resistant bacterial strains from cystic fibrosis (CF) patients, including colistin-resistant isolates of Burkholderia and Stenotrophomonas. In addition, the nanoemulsion is effective against organisms that are grown in biofilm and the sputum from CF patients. Resistance to the nanoemulsion is not anticipated based on its unique mechanism of action of interacting with the bacterial membrane and causing physical lysis of the organism.
These findings suggest that the use of NB-401 may have clinical benefit in treating CF patients. NanoBio has completed additional studies demonstrating that NB-401 can effectively be nebulized for the treatment of pulmonary infections. The company is currently conducting toxicity studies prior to advancing to clinical testing of NB-401 for the treatment of bacterial infections associated with cystic fibrosis.
NanoBio Corporation today announced compelling preclinical data for NB-401, a nebulized nanoemulsion-based agent that kills highly drug-resistant strains of bacteria commonly found in cystic fibrosis patients. Currently there are limited treatment options available that effectively address these resistant bacteria. The study results are being presented today at the 2009 Annual North American Cystic Fibrosis Conference (NACFC) in Minneapolis, Minn.
“Given that cystic fibrosis patients are susceptible to respiratory failure following repeated bacterial infections of the lung, there is a tremendous need to develop new treatments that can overcome drug-resistant bacteria,” said James R. Baker, Jr., MD, NanoBio’s CEO and founder. “In laboratory studies, NB-401 is a very potent killer against these difficult strains.”
NB-401 has been shown to be highly efficacious in vitro against Pseudomonas aeruginosa, Burkholderia, Acinetobacter, Stenotrophomonas and other multidrug-resistant bacterial strains from cystic fibrosis (CF) patients, including colistin-resistant isolates of Burkholderia and Stenotrophomonas. In addition, the nanoemulsion is effective against organisms that are grown in biofilm and the sputum from CF patients. Resistance to the nanoemulsion is not anticipated based on its unique mechanism of action of interacting with the bacterial membrane and causing physical lysis of the organism.
These findings suggest that the use of NB-401 may have clinical benefit in treating CF patients. NanoBio has completed additional studies demonstrating that NB-401 can effectively be nebulized for the treatment of pulmonary infections. The company is currently conducting toxicity studies prior to advancing to clinical testing of NB-401 for the treatment of bacterial infections associated with cystic fibrosis.
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11.03.2009
Gilead Sciences Inc. added another cystic fibrosis drug to its pipeline by licensing the preclinical epithelial sodium channel (ENaC) inhibitor P-680 from Parion Sciences Inc.
Foster City, Calif.-based Gilead will pay Durham, N.C.-based Parion up to $156 million for the drug, including a $5 million license fee, a $5 million equity purchase, research funding and potential milestone payments. Parion would also receive double-digit royalties on future sales.
In exchange, Gilead gets exclusive worldwide commercialization rights to P-680 in the treatment of pulmonary diseases including CF, chronic obstructive pulmonary disease (COPD), and non-CF bronchiectasis.Parion will complete preclinical and some Phase I development of the drug, with Gilead taking over further development. The two companies also will collaborate on a research program to identify other ENaC inhibitors.
The purpose of ENaC inhibitors, including P-680, is to block sodium channels that regulate hydration in the mucus membranes. This blockage prevents the absorption of liquid and keeps the mucosal surfaces hydrated. In the case of CF, the approach is intended to inhibit the development of thick mucus that results in infections and lung damage.
Parion’s ENaC program was spun out of the University of North Carolina at Chapel Hill by Richard Boucher, UNC chief of pulmonary medicine and founder of Inspire Pharmaceuticals Inc. Boucher teamed up with former Trimeris Inc. CEO Ross Johnson to found Parion, which was originally named CyFi Pharmaceuticals.
Since its founding in 2000, Parion has remained under the radar while quietly advancing its lead ENaC inhibitor, P-552, through Phase II programs in CF and xerostomia (dry mouth) associated with Sjogren’ssyndrome. Trials in more than 150 patients have shown P-552 to be well-tolerated and to cause mucus clearance in CF patients, according to Parion Director of Operations Paul Boucher.
P-552 is now slated for a Phase IIb trial in CF and additional trials in Sjogren’s syndrome, but Boucher said the timelines and detailsare still being planned.
Since ENaC is found on the mucosal surfaces of the lung, mouth, nose, eye and gastrointestinal tract, Parion’s ENaC inhibitors may havebroad utility in COPD, bronchiectasis, dry eye and many other conditions, including biodefense applications against inhaled pathogens like anthrax and plague. Boucher said the company has a “vast library” of ENaC inhibitors, some of which may be possible partnering candidates. He added that the company also has “very extensive” intellectual property in the space.
Thus far, Parion has advanced its pipeline without any venture funding, instead relying on grants from the NIH and Cystic Fibrosis Foundation Therapeutics Inc. (CFF). Earlier this week, CFF signed a $20 million venture philanthropy deal with FoldRx Pharmaceuticals Inc., bringing the foundation’s total in committed biotech funding for CF programs to about $250 million since 1998.
For Gilead, the P-680 license represents another step in the effort to increase its footprint in respiratory therapeutics. To date, most of Gilead’s success has come from antiviral products like Atripla (efavirenz 600 mg/ emtricitabine 200 mg/ tenofovir disoproxil fumarate300 mg) and Truvada (emtricitabine and tenofovir disoproxil fumarate), but Gilead spokesperson Nathan Kaiser said the company is “very committed” to building out its respiratory franchise.
As of now, that franchise consists solely of Tamiflu (oseltamivir phosphate), on which Gilead gets royalties from F. Hoffmann-La Roche Ltd.. Yet a new drug application filing is planned before the end of the year for CF drug aztreonam lysine for inhalation, which met its endpoints in two Phase III trials.
Kaiser noted that P-680 and inhaled aztreonam lysine “address different aspects of the disease.” P-680 improves mucosal clearance, while inhaled aztreonam lysine addresses Pseudomonas aeruginosa infection, a significant cause of morbidity and mortality among CF patients. The two drugs “theoretically could be used in combination,” Kaiser said.
Another target indication for Gilead, according to Kaiser, is cardiovascular disease. The company markets hypertension drug Flolan (epoprostenol sodium) and gained approval in June for pulmonary arterial hypertension drug Letairis (ambrisentan). Another hypertension drug, darusentan, is in Phase III.
Gilead acquired inhaled aztreonam lysine through its buyout of Corus Pharma Inc. and all three hypertension drugs through its buyout ofMyogen Inc. With about $2 billion in cash, equivalents and marketable securities as of the end of the second quarter, the company certainly has the money to buy more companies if it so chooses, but Kaiser was mum as to any acquisition plans.
Gilead Sciences Inc. added another cystic fibrosis drug to its pipeline by licensing the preclinical epithelial sodium channel (ENaC) inhibitor P-680 from Parion Sciences Inc.
Foster City, Calif.-based Gilead will pay Durham, N.C.-based Parion up to $156 million for the drug, including a $5 million license fee, a $5 million equity purchase, research funding and potential milestone payments. Parion would also receive double-digit royalties on future sales.
In exchange, Gilead gets exclusive worldwide commercialization rights to P-680 in the treatment of pulmonary diseases including CF, chronic obstructive pulmonary disease (COPD), and non-CF bronchiectasis.Parion will complete preclinical and some Phase I development of the drug, with Gilead taking over further development. The two companies also will collaborate on a research program to identify other ENaC inhibitors.
The purpose of ENaC inhibitors, including P-680, is to block sodium channels that regulate hydration in the mucus membranes. This blockage prevents the absorption of liquid and keeps the mucosal surfaces hydrated. In the case of CF, the approach is intended to inhibit the development of thick mucus that results in infections and lung damage.
Parion’s ENaC program was spun out of the University of North Carolina at Chapel Hill by Richard Boucher, UNC chief of pulmonary medicine and founder of Inspire Pharmaceuticals Inc. Boucher teamed up with former Trimeris Inc. CEO Ross Johnson to found Parion, which was originally named CyFi Pharmaceuticals.
Since its founding in 2000, Parion has remained under the radar while quietly advancing its lead ENaC inhibitor, P-552, through Phase II programs in CF and xerostomia (dry mouth) associated with Sjogren’ssyndrome. Trials in more than 150 patients have shown P-552 to be well-tolerated and to cause mucus clearance in CF patients, according to Parion Director of Operations Paul Boucher.
P-552 is now slated for a Phase IIb trial in CF and additional trials in Sjogren’s syndrome, but Boucher said the timelines and detailsare still being planned.
Since ENaC is found on the mucosal surfaces of the lung, mouth, nose, eye and gastrointestinal tract, Parion’s ENaC inhibitors may havebroad utility in COPD, bronchiectasis, dry eye and many other conditions, including biodefense applications against inhaled pathogens like anthrax and plague. Boucher said the company has a “vast library” of ENaC inhibitors, some of which may be possible partnering candidates. He added that the company also has “very extensive” intellectual property in the space.
Thus far, Parion has advanced its pipeline without any venture funding, instead relying on grants from the NIH and Cystic Fibrosis Foundation Therapeutics Inc. (CFF). Earlier this week, CFF signed a $20 million venture philanthropy deal with FoldRx Pharmaceuticals Inc., bringing the foundation’s total in committed biotech funding for CF programs to about $250 million since 1998.
For Gilead, the P-680 license represents another step in the effort to increase its footprint in respiratory therapeutics. To date, most of Gilead’s success has come from antiviral products like Atripla (efavirenz 600 mg/ emtricitabine 200 mg/ tenofovir disoproxil fumarate300 mg) and Truvada (emtricitabine and tenofovir disoproxil fumarate), but Gilead spokesperson Nathan Kaiser said the company is “very committed” to building out its respiratory franchise.
As of now, that franchise consists solely of Tamiflu (oseltamivir phosphate), on which Gilead gets royalties from F. Hoffmann-La Roche Ltd.. Yet a new drug application filing is planned before the end of the year for CF drug aztreonam lysine for inhalation, which met its endpoints in two Phase III trials.
Kaiser noted that P-680 and inhaled aztreonam lysine “address different aspects of the disease.” P-680 improves mucosal clearance, while inhaled aztreonam lysine addresses Pseudomonas aeruginosa infection, a significant cause of morbidity and mortality among CF patients. The two drugs “theoretically could be used in combination,” Kaiser said.
Another target indication for Gilead, according to Kaiser, is cardiovascular disease. The company markets hypertension drug Flolan (epoprostenol sodium) and gained approval in June for pulmonary arterial hypertension drug Letairis (ambrisentan). Another hypertension drug, darusentan, is in Phase III.
Gilead acquired inhaled aztreonam lysine through its buyout of Corus Pharma Inc. and all three hypertension drugs through its buyout ofMyogen Inc. With about $2 billion in cash, equivalents and marketable securities as of the end of the second quarter, the company certainly has the money to buy more companies if it so chooses, but Kaiser was mum as to any acquisition plans.
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12.02.2009
Genta Incorporated announced it will supply Ganite® (gallium nitrate injection) for a new clinical trial that will be initiated in patients with cystic fibrosis (CF) who may develop serious infections.
Infection is the most common cause of death in CF patients. Frequently, these infections are due to bacteria known as Pseudomonas aeruginosa, and patients are commonly treated with years of antibiotic therapy to control such infections. However, prolonged antibiotic use has greatly increased resistance due to genetic mutations. In addition, Pseudomonas has evolved protective mechanisms known as “biofilms” that encase colonies of bacteria and prevent antibiotics from reaching infection sites. Bacteria in biofilms are far more resistant to being killed. Recent information has suggested that gallium may be used as an anti-bacterial agent against Pseudomonas, in part due to its ability to disrupt biofilms.
The initial clinical trial using a gallium compound for this purpose will involve patients with cystic fibrosis who will be treated at the University of Washington in Seattle, WA and the University of Iowa, Iowa City, IA. If initial results are promising, additional trials will be conducted with additional medical centers and a larger number of patients. Genta has provided cross-reference to its Investigational New Drug (IND) exemption for Ganite®, and the Company will supply the drug at no cost to patients in the trial. The study is also supported by grants awarded by the Orphan Products Division of the Food and Drug Administration (FDA) and by the Cystic Fibrosis Foundation.
“Work from our center has established that gallium can be used as a ‘Trojan horse’ that interferes with how iron is used by Pseudomonas”, said Dr. Christopher H. Goss, Associate Professor of Medicine at the University of Washington, who is the Principal Investigator of the trial. “Our preliminary data suggest that gallium exploits potentially vulnerable mechanisms in Pseudomonas by disrupting biofilms and killing antibiotic-resistant strains of bacteria. This study is the first formal pharmacokinetic and safety evaluation of gallium in patients with cystic fibrosis.”
“This trial represents the rapid clinical translation of enormously promising observations”, said Dr. Raymond P. Warrell, Jr., Genta’s Chief Executive Officer. “Initial results from our first CF patient showed that systemic treatment achieved target gallium levels in sputum, and that these levels could be sustained for a prolonged duration. Pseudomonas infections in CF are exceptionally difficult to eradicate. If initial results of the IV drug in this trial are promising, future patients might also benefit from extended therapy that could be afforded by one of our oral gallium compounds.”
Further background on this work from the Universities of Washington and Iowa can be accessed here: http://www.jci.org/articles/view/30783/pdf.
Genta Incorporated announced it will supply Ganite® (gallium nitrate injection) for a new clinical trial that will be initiated in patients with cystic fibrosis (CF) who may develop serious infections.
Infection is the most common cause of death in CF patients. Frequently, these infections are due to bacteria known as Pseudomonas aeruginosa, and patients are commonly treated with years of antibiotic therapy to control such infections. However, prolonged antibiotic use has greatly increased resistance due to genetic mutations. In addition, Pseudomonas has evolved protective mechanisms known as “biofilms” that encase colonies of bacteria and prevent antibiotics from reaching infection sites. Bacteria in biofilms are far more resistant to being killed. Recent information has suggested that gallium may be used as an anti-bacterial agent against Pseudomonas, in part due to its ability to disrupt biofilms.
The initial clinical trial using a gallium compound for this purpose will involve patients with cystic fibrosis who will be treated at the University of Washington in Seattle, WA and the University of Iowa, Iowa City, IA. If initial results are promising, additional trials will be conducted with additional medical centers and a larger number of patients. Genta has provided cross-reference to its Investigational New Drug (IND) exemption for Ganite®, and the Company will supply the drug at no cost to patients in the trial. The study is also supported by grants awarded by the Orphan Products Division of the Food and Drug Administration (FDA) and by the Cystic Fibrosis Foundation.
“Work from our center has established that gallium can be used as a ‘Trojan horse’ that interferes with how iron is used by Pseudomonas”, said Dr. Christopher H. Goss, Associate Professor of Medicine at the University of Washington, who is the Principal Investigator of the trial. “Our preliminary data suggest that gallium exploits potentially vulnerable mechanisms in Pseudomonas by disrupting biofilms and killing antibiotic-resistant strains of bacteria. This study is the first formal pharmacokinetic and safety evaluation of gallium in patients with cystic fibrosis.”
“This trial represents the rapid clinical translation of enormously promising observations”, said Dr. Raymond P. Warrell, Jr., Genta’s Chief Executive Officer. “Initial results from our first CF patient showed that systemic treatment achieved target gallium levels in sputum, and that these levels could be sustained for a prolonged duration. Pseudomonas infections in CF are exceptionally difficult to eradicate. If initial results of the IV drug in this trial are promising, future patients might also benefit from extended therapy that could be afforded by one of our oral gallium compounds.”
Further background on this work from the Universities of Washington and Iowa can be accessed here: http://www.jci.org/articles/view/30783/pdf.
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