Sunday November 22 2009 | Biotechnology feed | All feeds
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Sunday November 22 2009 | Biotechnology feed | All feeds
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Return to introduction on drug discovery ~ LeadDiscovery Reports Inspire’s Denufosol tetrasodium (INS37217), a selective P2Y(2) agonist showing promise for the treatment of cystic fibrosis. Denufosol tetrasodium (INS37217) is a selective P2Y(2) agonist developed by Inspire Pharmaceuticals that stimulates ciliary beat frequency as well as chloride secretion in normal and cystic fibrosis airway epithelia. These two actions combine to improve mucus hydration and clearance and consequently INS37217 is being investigated as an inhaled treatment for cystic fibrosis. Until recently lacked any novel pipeline therapies in late phase trials (see Cystic Fibrosis - Countdown to a Cure). The phase I/II study recently reported in a recent issue of Pediatr Pulmonol demonstrates the good tolerability INS37217. Since this study a phase II trial confirmed tolerability and demonstrated efficacy. Cystic Fibrosis is the most common life-shortening genetic disease and is carried as an autosomal recessive trait by about 3% of the white population. The average age of survival is continually increasing and is now above 30 resulting in adult management issues and required changes to long-term treatment strategies. Although the genetic defect underlying cystic fibrosis was identified over a decade ago cystic fibrosis has until recently lacked any novel pipeline therapies in late phase trials (see Cystic Fibrosis - Countdown to a Cure). Our alert service DailyUpdates-Internal Medicine has recently featured research involving the development of CFTR activators, reduced chloride conductance of which results from the genetic defect and causes the epithelial defects seen in cystic fibrosis. Denufosol tetrasodium (INS37217) is one of these activators and has recently entered into phase II trials. INS37217 is a selective P2Y(2) agonist developed by Inspire Pharmaceuticals that stimulates ciliary beat frequency as well as chloride secretion in normal and cystic fibrosis airway epithelia. These two actions combine to improve mucus hydration and clearance and consequently INS37217 is being investigated as an inhaled treatment for cystic fibrosis. The phase I/II study recently reported in Pediatr Pulmonol highlighted here assessed the safety and tolerability of single and repeated doses of aerosolized denufosol in subjects with cystic fibrosis. Doses up to 60mg of denufosol were well-tolerated in most subjects although some intolerability was noted among subjects with lower baseline lung function. Since the completion of this phase I/II trial, a multicenter, 28-day, phase II study has been completed. This phase II study of patients with mild cystic was a double-blind comparison of three doses of INS37217 administered three times daily for 28 days by standard jet nebulizer. The trial was designed to determine the tolerability of INS37217. The most common adverse event was cough, which occurred in 46% of subjects and was comparable across all groups, including placebo. The study was not powered to demonstrate efficacy however, at the end of four weeks of treatment, subjects receiving INS37217 had significantly better lung function compared to patients receiving placebo. A further phase II study has recently been initiated as described in a company press release featured in December by DailyUpdates. This second phase II trial is being conducted in order to gain a greater understanding of safety and tolerability of INS37217 in a broader population of CF patients, which may allow expansion of the patient population for the company’s planned phase III trial. This broader study population will include patients using antibiotics and other therapeutics currently used by CF patients and the study will also include patients with lower lung function. In addition, Inspire recently began two longer-term inhalation toxicology studies.
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Pediatr Pulmonol. 2005 Feb 9; [Epub ahead of print] LeadDiscovery and BioPortfolio aims to provide reliable, insightful analysis on the biotechnology industry. However, this information is provided "as is" and no representations or warranties either express or implied of completeness, accuracy, or of any other nature are made with respect to this information. This information is neither an offer to sell nor a solicitation to buy the securities of any company. This information contains forward-looking statements, which involve risks and uncertainties which may not be listed. The biotechnology industry is an emerging industry and the securities of the companies mentioned in this report have a very high degree of risk and volatility. For this reason, this information is supplied on the condition that the reader will make his or her own determination as to its suitability for any purpose prior to any use of this information. The employees and officers of LeadDiscovery and BioPortfolio may hold positions in some or all of the stocks discussed in this report. This abstract has been produced by LeadDiscovery Ltd. Founded by life scientists for life scientists we aim to help industry identify cutting edge drug discovery options and academic/biotech institutions maximize the potential of their research. Abstracts strictly reflect the opinion of LeadDiscovery's editorial panel. While all reasonable efforts are made to ensure the accuracy of information provided LeadDiscovery and the publisher BioPortfolio, takes no responsibility for incorrect or misleading information. LeadDiscovery is designed for educational and drug development purposes only and is not intended or designed to offer medical advice or advice of any sort, and must not be used for such purpose. The information provided through LeadDiscovery and BioPortfolio should not be used for diagnosing or treating a health problem or a disease and no reliance should be placed on any information contained in this abstract or elsewhere on LeadDiscovery's and BioPortfolio's website. It is not intended to be a substitute for professional care. If you have or suspect you may have a health problem, you should consult your physician or other health care provider. |
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