Cubist Pharmaceuticals, Inc. (NASDAQ: CBST), a leading acute
care therapeutics company, today announced positive top-line
results from the recently completed Phase 2 studies of its CXA-201
and CB-183,315 antibiotic pipeline candidates. CXA-201 was studied
for the treatment of complicated intra-abdominal infections (cIAI)
in adults. Based on these latest top-line data, as well as data
observed in previous studies, the company plans to initiate Phase 3
studies with CXA-201 for indications in both cIAI and complicated
urinary tract infections (cUTI) by year-end 2011. CB-183,315 was
studied for the treatment of a severe and sometimes
life-threatening diarrhea caused by Clostridium difficile known as
C. difficile-associated diarrhea, or CDAD. The company remains on
track to make a formal decision of whether to initiate Phase 3
studies with CB-183,315 by year-end 2011.
“Positive results from these two Phase 2 clinical trials are
significant milestones for our pipeline, as well as our ongoing
efforts to become a global acute care leader,” said Steven Gilman,
Ph.D., Executive Vice President of Research and Development. “There
is a large and growing need for new and better antibiotics across
the globe, and we are very pleased with these positive
results.”
Positive Data for CXA-201 in cIAI
CXA-201 met its objectives of assessing safety and efficacy in
the Phase 2, multicenter, double-blind, 2:1 randomized study of 122
patients. The primary objective of the study was to determine the
clinical response of CXA-201 and metronidazole (included to cover
anaerobic bacteria) and that of meropenem in the treatment of
hospitalized patients with cIAI at the test of cure visit (7 to 14
days after the completion of study drug administration). The
secondary objectives of this study were to determine the
microbiological response of CXA-201 and metronidazole and that of
meropenem at the test-of-cure visit, to evaluate the safety profile
of CXA-201 and metronidazole, and to evaluate the pharmacokinetics
(PK) of CXA-201.
The data from this Phase 2 trial in patients with cIAI
demonstrated a clinical cure rate of 91 percent for the CXA-201
treatment arm in the clinically evaluable population, which is
consistent with the clinical cure rates that have been observed
with other drugs that have been studied in this indication. The
clinical cure rate for the study comparator, meropenem, was 94
percent in this population. Favorable clinical cure rates for the
CXA-201 treatment arm were observed across the identified baseline
pathogens, which included Escherichia coli, Klebsiella pneumonia
and Pseudomonas aeruginosa. In the microbiologically evaluable
population, the CXA-201 treatment arm demonstrated a clinical cure
rate of 89 percent and the meropenem arm demonstrated a clinical
cure rate of 96 percent. In this study, CXA-201 was safe and
well-tolerated, with the incidence and severity of adverse events
consistent with those typically seen with other cephalosporins and
in patients with this underlying medical condition.
In addition, a separate Phase 1 study was recently completed
which evaluated the lung penetration of CXA-201 using measurement
of epithelial lining fluid (ELF) concentrations. The study
demonstrated good lung penetration of CXA-101 (the cephalosporin
component of CXA-201). ELF concentrations of CXA-101 exceeded 8
µg/mL, a concentration that inhibits 99 percent of P. aeruginosa
for over 60 percent of the 8-hour dosing interval. The results
strongly support the continued development of CXA-201 for
nosocomial pneumonia. Based on this data and the overall clinical
profile of CXA-201, the company will be engaging global regulatory
authorities to discuss a CXA-201 nosocomial pneumonia Phase 3 study
design and expects to begin Phase 3 studies in this indication in
2012.
Positive data for CB-183,315 in patients with C. difficile
infections
This Phase 2 trial enrolled 209 patients and was a randomized,
double-blind, active-controlled, dose-ranging study investigating
the safety and relative efficacy of oral dose regimens of 125mg of
CB-183,315 twice a day, 250mg of CB-183,315 twice a day and 125mg
oral Vancocin® four times a day for 10 days.
In this trial, both doses of CB-183,315 demonstrated a high
clinical cure rate (resolution of diarrhea), comparable to
Vancocin. In the 199 patients in the modified intent-to treat
(MITT) group (patients that received study drug and had a confirmed
diagnosis of CDAD), the clinical cure rate was 92 percent and 87
percent for the CB-183,315 125mg and CB-183,315 250mg arms,
respectively, compared to 89 percent in the Vancocin arm. The
percent of patients who were clinically cured, but then had a
subsequent recurrence (the recurrence rate) was 36 percent in the
Vancocin arm and was 28 percent in the CB-183,315 125mg and 17
percent in the CB-183,315 250mg treatment arms. The reduction in
recurrence rate in the CB-183,315 250mg arm was statistically
significant compared to the Vancocin arm (p=0.035). Approximately
32 percent of patients in this trial were infected with the
hypervirulent NAP-1 strain of C. difficile. The clinical response
and recurrence rates in the subset of patients infected with the
NAP-1 strain was comparable across the CB-183,315 and Vancocin®
groups. In this trial, both doses of CB-183,315 were generally safe
and well-tolerated. Most common adverse events observed in the
CB-183,315 dose groups were headache and nausea, which occurred in
approximately 10 percent of patients.
“We are pleased with the results of the Phase 2 trials for
CXA-201 and for CB-183,315 which indicate that these
investigational agents may be promising therapies in the future for
the treatment of patients with serious bacterial infections”, said
Santosh Vetticaden, MD, PhD, Chief Medical and Development
Officer.
About Gram-negative bacteria
Gram-negative bacteria, such as P. aeruginosa, differ from
Gram-positive bacteria, such as Staphylococcus aureus, in their
cell wall structure. Gram-negative bacteria have an additional
outer wall composed of phospholipids and lipopolysaccharides. This
wall provides these bacteria an additional layer of defense for
antibiotics to traverse. The outer wall contains efflux pumps that
allow the bacteria to pump out substances toxic to the cell.
Although Gram-positive bacteria also have these pumps, the
Gram-negative strains are much more proficient, believed to be from
an adaptation for living in watery environments. Examples of
Gram-negative bacteria include Escherichia coli, P. aeruginosa,
Klebsiella pneumoniae, and Acinetobacter baumannii. The diseases
caused by Gram-negative bacteria include peritonitis, septicemia,
pneumonia, neonatal meningitis, urinary tract infections, IAI, and
burn and wound infections.
About Pseudomonas aeruginosa
Recent medical literature identifies P. aeruginosa as one of the
most prevalent Gram-negative pathogens responsible for
hospital-acquired infections, and points to its significant
virulence and steeply increasing incidences in intensive care units
(ICU). Data from the National Nosocomial Infections Surveillance of
ICUs in the United States identified P. aeruginosa as the most
frequently isolated Gram-negative strain, with an incidence almost
doubling between 1975 and 2003. For example, an increase of P.
aeruginosa from 9.6 percent to 16.3 percent was shown in nosocomial
pneumonia and from 9.3 percent to 16.3 percent in urinary tract
infections. Similar increases in P. aeruginosa-related infections
were shown by the SENTRY Antimicrobial Surveillance Program for
Europe, comparing data between 1997 and 2002. Pseudomonal
infections can involve any part of the human body, but among the
most common are urinary tract, lung, bloodstream, wound/burn, and
intra-abdominal infections. Resistance to current treatment
regimens for such infections is growing, with the increasing
appearance of P. aeruginosa strains expressing multi-drug
resistance against the commonly used first-line anti-pseudomonal
antibiotics.
About CDAD
CDAD is a disease caused by an overgrowth of, and subsequent
toxin production by, Clostridium difficile, a resident anaerobic
spore-forming Gram-positive bacterium of the lower gastrointestinal
tract. This overgrowth is caused by the use of antibiotics for the
treatment of common community and hospital acquired infections.
Although they treat the underlying infection, many antibiotics
disrupt the natural gut flora and allow Clostridium difficile to
proliferate. Clostridium difficile produces enterotoxin and
cytotoxin, which can lead to severe diarrhea, sepsis and even
death. CDAD rates and severity are increasing, due in part to the
spread of a new strain of Clostridium difficile with increased
virulence and greater resistance to fluoroquinolones. According to
an article in the October 2008 issue of the New England Journal of
Medicine, during the mid- and late-1990s, the reported incidence of
Clostridium difficile infection in acute care hospitals in the
United States remained stable at 30 to 40 cases per 100,000. In
2001, this number rose to almost 50, with subsequent increases to
the point that the number of cases of Clostridium difficile
infection that were reported in 2005 (84 per 100,000) was nearly
three times the 1996 rate (31 per 100,000).
About Cubist
Cubist Pharmaceuticals, Inc. is a biopharmaceutical company
focused on the research, development, and commercialization of
pharmaceutical products that address unmet medical needs in the
acute care environment. In the U.S., Cubist markets CUBICIN®
(daptomycin for injection), the first antibiotic in a class of
anti-infectives called lipopeptides, and has an agreement with
Optimer Pharmaceuticals, Inc. to co-promote Optimer’s drug,
DIFICID™, in the U.S. as a treatment of Clostridium difficile
infections. The Cubist clinical product pipeline currently consists
of a Phase 2 program focused on the development of a novel
cephalosporin to address certain serious infections caused by
multi-drug resistant (MDR) Gram-negative organisms and a Phase 2
program for the treatment of CDAD (Clostridium difficile-associated
diarrhea). Cubist also is working on several pre-clinical programs
being developed to address areas of significant medical needs.
These include therapies to treat various serious bacterial
infections and agents to treat acute pain. Cubist is headquartered
in Lexington, Mass. Additional information can be found at Cubist’s
web site at www.cubist.com.
Cubist Safe Harbor Statement
This press release contains forward-looking statements regarding
the clinical development of CXA-201 and CB-183,315, including plans
to advance these candidates into Phase 3 clinical trials and plans
to advance the development of CXA-201 in the indication of
nosocomial pneumonia, and the therapeutic potential of CXA-201 and
CB-183,315. There are many factors that could cause actual results
to differ materially from those in these forward-looking
statements. These factors include the following: CXA-201 and
CB-183.315 may not show sufficient therapeutic effect or an
acceptable safety profile in Phase 3 clinical trials; CXA-201 and
CB-183.315 may not act in the way expected based on prior clinical
and pre-clinical trials; clinical trials of CXA-201 and CB-183.315
may not be successful or initiated or conducted in a timely manner
and the timing of initiation and conduct of subsequent trials is
dependent on our ability to successfully work with regulatory
authorities, including the FDA on the design of the trials, among
other things; we plan to rely, to a significant extent, on third
party clinical research organizations, or CROs, to help us conduct
clinical trials so the success and timing of the trials is
dependent our ability to work with such CROs and their performance;
the commercial market for the intended use of CXA-201 and
CB-183.315 may not be as large as Cubist anticipates; if approved,
CXA-201 and CB-183,315 will compete with products currently on the
market and may also compete with products currently in development
which may have superior efficacy and/or safety profiles as CXA-201
and CB-183,315 or have other attributes that make it difficult for
CXA-201 and CB-183,315 to succeed commercially in such markets;
technical difficulties or excessive costs relating to the
manufacture or supply of CXA-201 and CB-183.315; we plan to rely,
to a significant extent, on third party contract manufacturers and
suppliers to manufacture and supply CXA-201 and CB-183,315 on our
behalf so our ability to obtain adequate supplies of CXA-201 and
CB-183,315 is dependent on our ability to work with such third
parties and on their performance; we, and Astellas Pharma Inc.,
from which we have licensed our rights to CXA-101 and which has an
interest in the intellectual property protecting CXA-201, may not
be able to maintain and enforce such intellectual property, and we
may not be able to maintain and enforce the intellectual property
protecting CB-183.315; and we may encounter other unanticipated or
unexpected risks with respect to the development or manufacture of
CXA-201. Drug development involves a high degree of risk. Success
in pre-clinical trials or early stage clinical trials does not mean
that later stage trials will be successful. Additional factors that
could cause actual results to differ materially from those
projected or suggested in any forward-looking statements are
contained in Cubist's recent periodic filings with the Securities
and Exchange Commission, including those factors discussed under
the caption "Risk Factors" in such filings. These statements speak
only as of the date of this release, and Cubist undertakes no
obligation to update or revise these statements, except as may be
required by law.
Cubist and CUBICIN are registered trademarks of Cubist
Pharmaceuticals, Inc.
DIFICID is a trademark of Optimer Pharmaceuticals, Inc.