Genentech Inc. announced late Wednesday that it hascompleted Phase III trials of Pulmozyme, its recombinanthuman DNase, for treating cystic fibrosis (CF).
The results of the 968-patient study demonstrated thatPulmozyme improves lung function and reduces the rate ofrespiratory infection in CF patients, with no major adverse sideeffects.
Although the South San Francisco, Calif., company (NYSE:GNE)has yet to file a product license application (PLA) onPulmozyme with the FDA -- which it is "proceeding quickly" toaccomplish by March 1993 -- analysts predict a quick time-to-approval and market penetration for the product's firstindication, to treat cystic fibrosis.
Jim McCamant, editor of the Medical Technology Stock Letter inBerkeley, Calif., said he likes the market for Pulmozyme for CFbecause "they know exactly who the potential patients are.
"The market is focused, it's easy to sell into, and there will behigh market penetration," he said, "like the markets for humangrowth hormone and erythropoietin."
Genentech has already penetrated Pulmozyme's potentialmarket. The company has worked closely with the CysticFibrosis Foundation and the medical community during thesetrials, and will provide pre-approved Pulmozyme to "the mostappropriate patients" under an expanded-access program.
Although he is awaiting further examination of clinical trialdata, Cowen & Co. analyst David Stone told BioWorld he "wouldexpect very rapid market penetration (for Pulmozyme) and fullpenetration two years after product launch, at which time itmay account for as much as half of Genentech's total productsales."
Genentech said it will present a thorough data analysis in lateJanuary at the Intermountain Thoracic Society Meeting.
Analyst Denise Gilbert of Smith Barney agreed that Pulmozymewill account for a big chunk of Genentech's future sales --about 30 percent to 35 percent by 1995. And Merrill Lynchanalyst Stuart Weisbrod predicted that Pulmozyme, along withSynergen's Inc.'s Antril, will be one of biotech's two majorproducts in 1993. Although it's risky to place a dollar value onsales of a product that is barely out of Phase III trials, analystsplace the market at somewhere between $100 million and$300 million on the low end, with a high end of as much as $1billion.
Worldwide, about 50,000 to 60,000 people suffer from cysticfibrosis, the most common fatal genetic disease amongCaucasians. So a product such as DNase, which acts byliquefying the lung secretions that build up and impairbreathing in CF patients, would offer the first symptomatictreatment for the disease.
But so might SLPI (secretory leukocyte proteinase inhibitor),which Synergen (NASDAQ:SYGN) has in "advanced clinicals" forCF. And "some people think the two would be complementarytreatments at different stages in the disease," Stone said.
Beyond those treatments lie protein replacement and genetherapy, both of which are now in early stages of development.In fact, three gene therapy trial protocols for cystic fibrosis arecoming before the NIH Recombinant DNA Advisory Committeetoday and Friday.
Eventually, products such as Pulmozyme may be used withprotein replacement or gene therapy, or the newer approaches"may obviate the need for symptomatic treatment for cysticfibrosis," Stone told BioWorld.
By that time, however, "Genentech will have developed otherindications (such as for chronic bronchitis and other chroniclung diseases)," Stone said.
Genentech stock was up 13 cents a share on Wednesday to $38.
-- Jennifer Van Brunt Senior Editor
(c) 1997 American Health Consultants. All rights reserved.