this was a story back last year:
02-Nov-07 18:45 Altus Pharmaceutical unlikely to appeal FDA decision to revoke Orphan Drug designation from TheraCLEC, CFO says
Story Altus Pharmaceutical will most likely not appeal the Food and Drug Administration’s (FDA) decision to revoke its Orphan Drug designation on its TheraCLEC (ALTU 135) compound, said CFO Jonathan Lieber.
The FDA granted TheraCLEC Orphan Drug designation in 2003, however, the agency revoked this STATUS late last year because it said the number of patients exceeded the statutory limit of 200,000 patients. The FDA included HIV/AIDS patients, who may also suffer from pancreatic insufficiencies, but do not necessarily take this class of drugs. Altus can appeal the decision up to its New Drug Application (NDA) date.
Altus plans to file an NDA for TheraCLEC by the first half of 2009, Lieber said. The company started Phase III studies in 2Q this year, he said. It is holding two trials: one for efficacy and one for safety. The efficacy trial will last eight weeks and Altus plans to release top line data for the study by 2Q 2008, Lieber said. The safety trial will be complete towards the end of 2008. The company plans to enroll 150 evaluable patients for its efficacy trial and 100 for its safety trial. Patients enrolling in the safety trial can op to stay on and enroll in the efficacy trial as well.
TheraCLEC is an orally delivered enzyme replacement therapy for the treatment of malabsorption as a result of pancreatic insufficiencies, a condition that affects most patients with cystic fibrosis (CF), chronic pancreatisis and pancreatic cancer. The current standard of care uses porcine enzymes.
Lieber said the current porcine-derived drugs have been widely used for over 50 years, yet still have their limitations. The pill burden is very high, with patients having to take four or five capsules with every meal or snack. With TheraCLEC patients can take as little as one capsule with every meal or snack. Also TheraCLEC is more stable, he said.
TheraCLEC has received interest from companies who would like to partner the drug, the CFO said, and discussions have taken place. He declined to go into more detail. Altus is seeking to partner the drug in the rest of the world but prefers to keep all the North American rights, he said.
Lieber said he is not aware of another company with a compound as far along in the clinical trial process as Altus. France-based Meristem Therapeutics and Swedish-based Biovitrum have similar compounds, he said, but to his knowledge both companies have held only small Phase II trials.
The enzyme replacement market place is very well established said Matthew Casbon, Director of Marketing for competing Belgian pharmaceutical company, Solvay, whose drug CREON has a 25% market share. Generics also have a 55% market share, he added. If and when TheraCLEC is approved, Casbon said, it will have a place in the market but is unlikely to make a huge impact. Still, he added that TheraCLEC’s microbially-derived mechanism seems to be the way forward and Solvay has also considered developing a similar drug.
Altus has another compound that has also completed Phase II trials, ALTU 238, which is partnered with Genentech in North America and the latter has the option to extend this for rights for the rest of the world, he said. ALTU 238 is a crystallized formation of the Human Growth Hormone (hGH) that is designed to be administered once a week for the treatment of hGH disorders in pediatric and adult populations.
Altus Pharmaceutical has a market capitalization of USD 428m.
by Gayatri Iyer
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