To present data at North American Cystic Fibrosis Conference next week
CHARLESTOWN, MA, USA I October 11, 2013 I Flatley Discovery Lab, LLC today announced that it has nominated FDL169, an orally available small molecule for the treatment of Cystic Fibrosis, as a clinical candidate and initiated IND-enabling studies. Data from these studies of FDL169 will be presented next week at the 27th Annual North American Cystic Fibrosis Conference, Salt Lake City, Utah, on October 17th.
“Flatley Discovery Lab is excited to have discovered a very potent and efficacious CFTR corrector,” said Richard Fitzpatrick, Ph.D., CEO of Flatley Discovery Lab. “FDL169 has a differentiated in vivo profile from other molecules in this class of drugs which we believe will produce a superior clinical profile. We believe that a combination of FDL169 and a CFTR potentiator will have a positive impact on the lives of CF patients with the ΔF508 mutation. We anticipate completing our preclinical program by the end of 2014.”
In ex vivo human bronchial models of Cystic Fibrosis lung disease, FDL169 was shown to significantly increase chloride transport. Preliminary studies of FDL169 in rodents and dogs suggest that the molecule will have a good safety profile.
John Flatley, founder of FDL, said, “I am very excited by the progress of all our projects and we will be initiating discussions with potential partners on advancing these candidates and getting them to patients as quickly as possible.”
About Cystic Fibrosis and FDL169
Cystic Fibrosis is an autosomal recessive genetic disorder characterized by thick, sticky secretions in the lung, pancreas, liver, and skin, and chronic pulmonary infections. Cystic Fibrosis is caused by a mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, which plays a key role in maintaining ion balance across membranes. The most common mutation in patients with Cystic Fibrosis is ΔF508, which causes the protein to misfold and not conduct chloride properly. The corrector FDL169 promotes CFTR folding and processing, thereby increasing the level of functional protein on the cell surface. FDL169 restores CFTR dependent chloride transport when used alone or in combination with a drug that potentiates CFTR.
About Flatley Discovery Lab, LLC.
Flatley Discovery Lab, LLC is an independent not-for-profit biotech company whose mission is to discover and develop new therapies for Cystic Fibrosis. Flatley Discovery Lab has recently begun preclinical development of FDL169 for ΔF508 cystic fibrosis. In addition, Flatley Discovery Lab is currently developing CFTR potentiating drugs and other CFTR modulators that can be used in combination with CFTR correctors and potentiators. Flatley Discovery Lab is generously supported by the Flatley Family Foundation.
SOURCE: Flatley Discovery Lab
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To present data at North American Cystic Fibrosis Conference next week
CHARLESTOWN, MA, USA I October 11, 2013 I Flatley Discovery Lab, LLC today announced that it has nominated FDL169, an orally available small molecule for the treatment of Cystic Fibrosis, as a clinical candidate and initiated IND-enabling studies. Data from these studies of FDL169 will be presented next week at the 27th Annual North American Cystic Fibrosis Conference, Salt Lake City, Utah, on October 17th.
“Flatley Discovery Lab is excited to have discovered a very potent and efficacious CFTR corrector,” said Richard Fitzpatrick, Ph.D., CEO of Flatley Discovery Lab. “FDL169 has a differentiated in vivo profile from other molecules in this class of drugs which we believe will produce a superior clinical profile. We believe that a combination of FDL169 and a CFTR potentiator will have a positive impact on the lives of CF patients with the ΔF508 mutation. We anticipate completing our preclinical program by the end of 2014.”
In ex vivo human bronchial models of Cystic Fibrosis lung disease, FDL169 was shown to significantly increase chloride transport. Preliminary studies of FDL169 in rodents and dogs suggest that the molecule will have a good safety profile.
John Flatley, founder of FDL, said, “I am very excited by the progress of all our projects and we will be initiating discussions with potential partners on advancing these candidates and getting them to patients as quickly as possible.”
About Cystic Fibrosis and FDL169
Cystic Fibrosis is an autosomal recessive genetic disorder characterized by thick, sticky secretions in the lung, pancreas, liver, and skin, and chronic pulmonary infections. Cystic Fibrosis is caused by a mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, which plays a key role in maintaining ion balance across membranes. The most common mutation in patients with Cystic Fibrosis is ΔF508, which causes the protein to misfold and not conduct chloride properly. The corrector FDL169 promotes CFTR folding and processing, thereby increasing the level of functional protein on the cell surface. FDL169 restores CFTR dependent chloride transport when used alone or in combination with a drug that potentiates CFTR.
About Flatley Discovery Lab, LLC.
Flatley Discovery Lab, LLC is an independent not-for-profit biotech company whose mission is to discover and develop new therapies for Cystic Fibrosis. Flatley Discovery Lab has recently begun preclinical development of FDL169 for ΔF508 cystic fibrosis. In addition, Flatley Discovery Lab is currently developing CFTR potentiating drugs and other CFTR modulators that can be used in combination with CFTR correctors and potentiators. Flatley Discovery Lab is generously supported by the Flatley Family Foundation.
SOURCE: Flatley Discovery Lab
Post Views: 123
