Really nice paper in NEJM. This should be enough to convince even the most parsimonious HMO that TOBI treatment is a good idea and might even save money.
Stock hasn't been reacting to good news lately - I would assume some profit taking after its run-up in 1998. I'm holding long.
Peter
Headline: Research Published in the New England Journal of Medicine Shows
Benefits Of Chronic Aerosolized Antibiotic Therapy
======================================================================
Clinical Trial Results Demonstrate Improved Pulmonary Function, Decreased
Hospitalization Risk in Cystic Fibrosis Patients
SEATTLE, Jan. 6 /PRNewswire/ -- In two double-blind, placebo-controlled
studies published in Thursday's edition of The New England Journal of
Medicine, chronic intermittent aerosolized antibiotic therapy with TOBI(R)
(tobramycin solution for inhalation) has demonstrated significant benefits in
treating people with cystic fibrosis. TOBI was developed by PathoGenesis
Corp. (NASDAQ:PGNS) and approved for U.S. sale in December 1997.
In the largest studies of inhaled antibiotics to date, 300 milligrams of
TOBI or an inhaled placebo was administered to 520 cystic fibrosis patients
with Pseudomonas aeruginosa lung infections. TOBI or placebo was inhaled at
home twice a day in repeated cycles of 28 days on drug, 28 days off drug, for
three on-off cycles over 24 weeks. In both groups, patients were also allowed
to receive standard treatment recommended for the management of cystic
fibrosis, which may have included oral or intravenous antibiotics.
"These are the first large-scale studies in cystic fibrosis patients to
show such a sizable average improvement in lung function and reduction in
hospitalization," said lead author Bonnie Ramsey, M.D., Cystic Fibrosis Center
director at Children's Hospital and Regional Medical Center, Seattle, and
professor of pediatrics, University of Washington School of Medicine. "TOBI
has shown us that inhaled antibiotics work, and that has tremendous
implications for future treatment of cystic fibrosis."
Results at week 20 (the end of the third period of drug administration)
showed that patients treated with TOBI had an average increase in lung
function of 10 percent, versus an average 2 percent decline in the placebo
group. Pulmonary function is the best predictor of disease progression in
people with cystic fibrosis.
In addition, patients taking TOBI had decreased P. aeruginosa density in
their sputum (phlegm) and were 26 percent less likely to be hospitalized than
those in the placebo group. On average, TOBI-treated patients were
hospitalized three fewer days than patients receiving standard cystic fibrosis
therapy -- 5.1 days for TOBI patients, compared with 8.1 days for placebo
patients. TOBI patients also were 36 percent less likely to require
intravenous antipseudomonal antibiotics than patients taking placebo.
Overall adverse events were comparable but less severe in the TOBI group
than in the placebo group. Tinnitis (ringing in the ears) and voice
alteration (e.g., hoarseness) were the only adverse events reported by more
patients in the TOBI group than in the placebo group. Tinnitis was reported
by eight TOBI patients (3.1 percent) and by no placebo-treated patients. It
was transient and appeared mild or moderate in severity. Voice alteration was
reported by 33 TOBI patients (12.8 percent) and 17 placebo-treated patients
(6.5 percent). Four patients in the placebo group and none in the TOBI group
died during the studies.
"The really important statistics about TOBI are the human stories about
how the drug has made a difference in the lives of people with cystic
fibrosis," said Robert J. Beall, Ph.D., president and chief executive officer
of the Cystic Fibrosis Foundation. "Many of these patients are able to
achieve things they've never been able to achieve before."
Additional data showed greatest benefit in teenagers and females
The New England Journal of Medicine article also reported that all patient
subgroups experienced a benefit in lung function, as measured by forced
expiratory volume at one second (FEV (1)). In particular, patients who were
13 to 17 years old or who were female demonstrated a significantly larger
treatment effect than those in the other groups.
Cystic fibrosis is a life-threatening inherited disease, affecting about
70,000 children and adults worldwide. In the U.S., intermittent exacerbations
(flare-ups of pulmonary symptoms) of pseudomonal lung infections in cystic
fibrosis patients have traditionally been treated with intravenous antibiotics
in the hospital and/or at home for seven to 21 days. Despite frequent
intravenous therapy, lung function continues to decline once the lungs are
infected with P. aeruginosa, and eventually 90 percent of such patients die of
respiratory failure. The New England Journal of Medicine article concluded
that "long-term antibacterial therapy may help maintain pulmonary function."
TOBI was made possible in part by early research into the potential for
aerosolized tobramycin that was supported by a grant from the Cystic Fibrosis
Foundation (CFF) and conducted by Children's Hospital and Regional Medical
Center in Seattle. Information about the CFF is available on the Internet at
www.cff.org.
Children's Hospital and Regional Medical Center is the premier regional
pediatric care center in the Pacific Northwest, with national prominence for
its clinical expertise and affiliation with the University of Washington
School of Medicine. Children's conducts research into the development of new
treatments for childhood illnesses and diseases, serves as an academic
resource for teaching future healthcare providers, and acts as an advocate for
children's health and safety.
The developer of TOBI, Seattle-based PathoGenesis Corp. is a
pharmaceutical company that develops drugs to treat chronic infectious
diseases -- lung infections, in particular -- where there is a significant
need for improved therapy. The company markets TOBI, an inhaled antibiotic,
in the U.S. for management of cystic fibrosis patients with Pseudomonas
aeruginosa. In addition, PathoGenesis is developing other drug candidates to
treat serious chronic lung infections, including those common in cystic
fibrosis, bronchiectasis and tuberculosis patients. PathoGenesis' stock is
traded on the Nasdaq National Market System under the symbol PGNS. The
company's Web site is located at www.pathogenesis.com.
Note: This news release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995. Such
forward-looking statements are subject to known and unknown risks,
uncertainties or other factors that may cause the company's actual results to
be materially different from historical results or any results expressed or
implied by such forward-looking statements. Factors that might cause such a
difference include, but are not limited to, uncertainties related to the fact
that PathoGenesis began commercial operations only recently, its dependence on
TOBI, third party reimbursement and product pricing, government regulation,
drug development and clinical trials, competition and alternative therapies,
and other factors described in PathoGenesis' filings with the Securities and
Exchange Commission.
SOURCE PathoGenesis Corporation
-0- 01/06/99
/CONTACT: Linda Mura of Porter Novelli for PathoGenesis Corp.,
312-856-8882; or Dean Forbes of Children's Hospital, 206-368-4817; or Laurie
McHale of the Univ. of Washington, 206-543-3620; or Maryellen Thielen of
PathoGenesis Corp., 847-583-5424/
/Web site: cff.org
/Web site: pathogenesis.com |
