By Brady Huggett

Altus Biologics Inc. is getting up to $25 million in funding from the Cystic Fibrosis Foundation Therapeutics Inc. to develop its product, TheraCLEC-Total, for nutrition-starved cystic fibrosis (CF) patients.

The Cystic Fibrosis Foundation Therapeutics Inc., the drug development affiliate of the Cystic Fibrosis Foundation, of Bethesda, Md., will provide access to its Therapeutics Development Network as well as the funding.

"We are bringing the drug and technology, and the Cystic Fibrosis Foundation is bringing the $25 million funding to support the program," said Peter Lanciano, president and CEO of Altus. "They will help recruit patients and bring their physicians. All CF physicians belong to their broad network."

Patients with CF have a mucous buildup in the ducts that lead from the pancreas to the digestive system, blocking the path of enzymes required for digestion. If these patients are not treated with some sort of nutritional therapy, Lanciano said, they lose weight and eventually die. That's where TheraCLEC-Total comes in.

The product is being developed as an orally delivered therapy to replace the blocked enzymes in people with CF. Altus, of Cambridge, Mass., uses protein crystallization technology to make TheraCLEC-Total resist breaking down in the digestive tract, and capable of helping those with pancreatic insufficiency.

"With our Crystalomics technology, we manage proteins in their crystalline structure, where they are extremely active in that form," Lanciano told BioWorld Today. "When a protein is in its crystalline form and can maintain it, you can protect it from degradation and destruction."

This strengthened form of the protein is then able to make its way through the digestive tract and do the job the pancreatic enzymes aren't there to do. TheraCLEC-Total is preclinical, but with the $25 million support from the Cystic Fibrosis Foundation, its trials are paid for.

"A big chunk is already guaranteed, because there are no hurdles early," Lanciano said. "We will be receiving quite a bit through the balance of the year, and then when we get the IND through, we get another portion. It is milestone driven, but forward funded. It provides funding all the way through Phase III.

"We expect to file in the second half of this year," he added. "We have met with the FDA and have been discussing the trial and our preclinical work. It's going very well so far. We expect to be in human trials by this time next year. You never know how these things are until you have gotten approvals, but the data speak pretty loudly."

There are other products to treat pancreatic insufficiency, but Lanciano said TheraCLEC-Total would have advantages over the traditional polymer-coated replacement enzymes.

Patients would have to take only three pills per day, compared to 20 to 40 with other therapies, he said, adding that the company also is developing a liquid formulation for children.

The goal is to have a TheraCLEC-Total product launch by the end of 2004, Lanciano said. And while the $25 million is specifically for TheraCLEC-Total development for CF patients, it has applications for other indications.

"The product can be for other pancreatic-insufficient people, people with AIDS, people with cancer," Lanciano said. "And we will separately be expanding the label on our own, but outside the funding. We currently have many drug development deals using our Crystalomics platform. None of them has focused on the oral route of administration; this is our first one." n