An FDA advisory committee Wednesday unanimously recommendedapproval of CellPro Inc.'s Ceprate SC stem cell concentrationsystem.
Specific labeling indications will be discussed later between companyand FDA officials, but CellPro is projecting remaining issues to beresolved soon and hopes to have the product on the U.S. market bythe end of the year. The positive recommendation brings CellPronearly to the end of what's been a rough road, which included aDecember 1994 non-approvable letter from the FDA andabandonment of the product by marketing partner Corange Ltd. in1995.
Marc Ostro, a managing director at UBS Securities in New York, isamong those who believed in the technology and its utility all along.
"These guys [at CellPro] have run the gauntlet and came out smellinglike a rose," Ostro said Wednesday after the meeting of theBiological Response Modifiers Advisory Committee. "They deservean enormous amount of credit. They really put on a brilliantperformance."
Investors were rewarded for their perseverance as CellPro stockgained $4.06, or 28 percent, to close Wednesday at $18.50. It went ashigh as $20.38 in trading of 2.8 million shares.
The Ceprate system is a device that separates human stem cells(which produce blood and immune system cells) from other cells ofthe bone marrow or peripheral blood. The separated cells arereturned to a patient after chemotherapy or other marrow-destroyingprocedures to help restore bone marrow.
The science in the peripheral blood stem cell (PBSC) transplant areawas not near what it is today when Seattle-based CellPro began itsCeprate study three and a half years ago. Nobody was sure PBSCswould engraft, and they were hard to catch, said Lee Parker,CellPro's director of investor relations.
Therefore, Ostro said, the early studies may support labeling only forautologous bone marrow transplantation. "But as soon as this productis on the market it's going to be used for every application," whichbrings into play allogeneic procedures and those involving PBSCs, hesaid.
The premarket approval (PMA) application for Ceprate wassupported by May 1993 results from Phase III trials for autologousbone marrow transplants in the treatment of metastatic breast cancer.The primary endpoints were equivalent engraftment of stem cellsprocessed through the Ceprate system and the reduction ofcardiovascular side effects associated with autologous bone marrowtransplantations.
Amendments to the PMA seeking expanded labeling are expected incoming years since the device is being studied in a number of trialssponsored by the company and independent investigators. Thecompany said more than 175 institutions in the U.S. and Europe areusing the system in their studies. The most advanced company trial isa Phase III study in which about 100 of 134 patients have beenaccrued, Parker said, adding that a one-year follow-up will beneeded.
Ostro said recent approvals of Immunex Corp.'s Leukine and AmgenInc.'s Neupogen in the PBSC mobilization area should play right intothe hands of the Ceprate system. Another positive, he said, is that noother stem cell concentration system will be commercially availablefor two years.
By then, Ostro said, the market will be consolidated and people willbe committed to and comfortable with the Ceprate system.
CellPro's former Ceprate partner, Corange Ltd., of Bermuda andLondon, bought out of the relationship in May 1995, five monthsafter the FDA's non-approvable letter. Corange ended up paying $90million to CellPro over the life of the relationship and came awaywith about 2 million shares of stock.
Now CellPro has all rights to the product, which it already is sellingin Europe and Canada. In July 1995 CellPro gained CE marking forthe system, allowing it to be sold in all European EconomicCommunity member countries. Sales for the current year ending inMarch are projected at $6 million, Parker said.
Ostro pointed out sales for the quarter that ended Dec. 31, 1995,increased 60 percent over the same quarter last year and the oneending in September 1995 ($2 million vs. $1.25 million).
CellPro has a sales force of about 25 in Europe covering the 250bone marrow transplant centers on that continent. The company'scentral office is in Brussels, Belgium, and marketing subsidiaries areset up in France, Germany, Italy and Spain. Distributorships will beused to sell the product in Australia and certain countries in Asia andLatin America.
Parker said the plan is for CellPro to market directly in the U.S.,which also has about 250 transplant centers and would require a salesstaff similar to the one operating in Europe. n
-- Jim Shrine
(c) 1997 American Health Consultants. All rights reserved.