Conference Call with Insmed Management and
Clinical Trial Investigators Will be Held Today at 8:00 a.m.
Eastern Time
Insmed Incorporated (Nasdaq GS:INSM) today reported results from
the Company’s phase 2 clinical trial of ARIKAYCETM, or liposomal
amikacin for inhalation, for the treatment of patients with
treatment resistant nontuberculous mycobacterial (NTM) lung
infections. The randomized, double-blind, placebo-controlled phase
2 clinical trial compared ARIKAYCE (590 mg delivered once daily),
added to standard of care treatment, versus standard of care
treatment plus placebo, in 90 adult patients with treatment
resistant NTM lung disease. Eligibility for the study required
patients to have been on the American Thoracic Society/Infectious
Disease Society of America (ATS/IDSA) guideline therapy for at
least six months prior to screening and to continue to have
persistently positive mycobacterial cultures.
The primary efficacy endpoint of the study was a
semi-quantitative measurement of the change in mycobacterial
density on a seven-point scale from baseline (day one) to the end
of the randomized portion of the trial (day 84). ARIKAYCE did not
meet the pre-specified level for statistical significance although
there was a positive trend (p=0.148) in favor of ARIKAYCE. However,
ARIKAYCE did achieve statistical significance with regard to the
clinically relevant key secondary endpoint of culture conversion,
with 11 out of 44 patients treated with ARIKAYCE (added to standard
of care treatment) demonstrating negative cultures by day 84 of the
study as compared to 3 out of 45 patients treated with placebo
(added to standard of care treatment) (p=0.01).
Patients receiving ARIKAYCE experienced a greater number of
adverse events than those receiving placebo. All adverse events
experienced with ARIKAYCE were consistent with those seen in
similar patient populations receiving inhaled antibiotics. The most
common side effect was laryngeal irritation.
The Company plans to incorporate these results into discussions
with the regulatory agencies in the United States and Europe to
determine next steps for ARIKAYCE. The Company also intends to
apply for Breakthrough Therapy Designation for ARIKAYCE in the
United States based upon the culture conversion results. ARIKAYCE
has already received Orphan Drug, Qualified Infectious Disease
Product (QIDP) and Fast Track designations from the U.S. Food and
Drug Administration (FDA) for the treatment of NTM lung infections
and recently received Orphan Drug Designation from the European
Medicines Agency (EMA).
“The results relating to the key secondary endpoint of culture
conversion are encouraging, and I believe demonstrate that ARIKAYCE
has the potential to be an option for treating even the most
difficult treatment resistant patients with NTM lung infections,”
said David Griffith, M.D., Professor of Medicine, W.A and E. B.
Moncrief Distinguished Professor at The University of Texas Health
Sciences Center and a co-Principal Investigator on the study.
“We are encouraged by the achievement of culture conversion in
this trial, which we believe is the ultimate goal in the treatment
of mycobacterial infections,” said Dr. Renu Gupta, Executive Vice
President, Development and Chief Medical Officer of Insmed. “The
design of this trial was such that the patients who entered the
trial and received drug were clearly resistant to guideline
therapy, making them the most treatment-challenged NTM patients.
Therefore the hurdle for showing any improvement with a therapy is
extremely high.”
“While ARIKAYCE did not achieve statistical significance on the
primary endpoint of bacterial density reduction, we are very
pleased by the greater number of culture conversions among patients
receiving ARIKAYCE,” stated Will Lewis, President and Chief
Executive Officer of Insmed. “We now look forward to the regulatory
discussions in the US and Europe that will guide our path
forward.”
“We extend our gratitude to the investigators and their patients
who participated in this study,” concluded Mr. Lewis.
Primary Endpoint
The objective of the primary endpoint was to show a reduction in
the density of bacteria of at least one step along a seven point
scale as a means of identifying whether a trend suggestive of
ultimate culture conversion could be established. The number of
patients showing at least a one-step reduction in the treatment arm
versus those in the placebo arm was not statistically significant
(p=0.148). The design of the study’s primary endpoint was not
culture conversion because it was assumed that culture conversion
would not be achievable in 84 days treatment, particularly given
the severe, treatment-resistant patient population that was the
subject of this study.
Secondary Endpoints
ARIKAYCE achieved statistical significance with regard to the
secondary outcome of culture conversion, with 11 out of 44 patients
treated with ARIKAYCE demonstrating negative cultures by day 84 of
the study as compared to 3 out of 45 patients treated with placebo
arm (standard of care therapy only) (p=0.01). Data analyses for
additional secondary, tertiary and exploratory endpoints are
ongoing and will be discussed at a presentation at the American
Thoracic Society meeting in May.
Safety
Patients receiving ARIKAYCE experienced adverse events
consistent with those seen in similar patient populations receiving
inhaled antibiotics. Overall, mild to moderate throat irritation
was more common in the ARIKAYCE arm compared to the placebo arm.
One Suspected Unexpected Serious Adverse Reaction (SUSAR) was
observed in the ARIKAYCE arm, but there was no difference in severe
serious adverse reactions or hemoptysis between the two arms.
Instances of hearing loss or tinnitus, a side effect more commonly
associated with intravenous dosing of amikacin, were evenly
balanced between the ARIKAYCE and placebo arms. The study
population was chronically ill with a mean age of 61.
Clinical Trial Design
Mycobacterial density is a measurement currently used in
clinical practice to assess the progress or decline of those
patients with recalcitrant NTM. Following the randomized portion of
the study, all eligible patients had the option to receive ARIKAYCE
once daily for an additional 84 days in an open-label design.
Patients in the trial were stratified for either Mycobacterium
avium complex (MAC) infections or Mycobacterium abscessus
infections. These pathogens collectively account for approximately
85% of all patients with NTM lung disease in the U.S.
Stratification was also performed based on patients with cystic
fibrosis versus those who do not have cystic fibrosis.
Conference Call and Webcast
Insmed management will host an investment community conference
call today at 8:00 a.m. Eastern time, featuring several clinical
trial investigators who are experts in NTM. Shareholders and other
interested parties may participate in the call by dialing
888-803-5993 (domestic) or 706-634-5454 (international) and
referencing conference ID number 19390649. The call will be webcast
live and archived at http://investor.insmed.com/events.cfm.
A replay of the conference call will be accessible two hours
after its completion through April 1, 2014 by dialing 855-859-2056
(domestic) or 404-537-3406 (international) and referencing
conference ID number 19390649. The call will also be archived for
90 days on the Company’s website at www.insmed.com.
About Nontuberculous Mycobacteria (NTM)
Nontuberculous mycobacteria (NTM) are organisms found in the
soil and water that can cause serious lung disease in susceptible
individuals, for which there are currently limited effective
treatments and no approved therapies. The prevalence of NTM disease
is reported to be increasing, and according to reports from the
American Thoracic Society is believed to be greater than that of
tuberculosis in the U.S. According to the National Center for
Biotechnology Information, epidemiological studies show that
presence of NTM infection is increasing in developing countries,
perhaps because of the implementation of tap water. Women with
characteristic phenotype are believed to be at higher risk of
acquiring NTM infection along with patients with defects on cystic
fibrosis transmembrane conductance regulators.
NTM lung disease is often a chronic condition that can lead to
progressive inflammation and lung damage, and is characterized by
bronchiectasis and cavitary disease. NTM infections often require
lengthy hospital stays for medical management. Treatment usually
involves multi-drug regimens that can be poorly tolerated and have
limited effectiveness, especially in patients with severe disease
or in those who have failed prior treatment attempts. According to
a company-sponsored patient chart study conducted by Clarity Pharma
Research, approximately 50,000 patients suffering from NTM lung
disease visited physician offices in the U.S. during 2011.
About ARIKAYCE®
ARIKAYCE is a form of the antibiotic amikacin, which is enclosed
in nanocapsules of lipid called liposomes. This advanced pulmonary
liposome technology prolongs the release of amikacin in the lungs
while minimizing systemic exposure. The treatment uses
biocompatible lipids endogenous to the lung that are formulated
into small (0.3 micron), charge-neutral liposomes. ARIKAYCE is
administered once-daily using an optimized, investigational eFlow®
Nebulizer System manufactured by PARI Pharma GmbH, a novel, highly
efficient and portable aerosol delivery system.
This is the first controlled clinical trial of an antibiotic in
patients suffering from NTM lung infections. There are no drugs
approved by the FDA for the treatment of this chronic, debilitating
disease.
About eFlow® Technology and PARI Pharma
ARIKAYCE is delivered by an investigational eFlow® Nebulizer
System developed by PARI Pharma and optimized specifically for
ARIKAYCE. The optimized device uses eFlow Technology to enable
highly efficient aerosolization of medication including liposomal
formulations via a vibrating, perforated membrane that includes
thousands of laser-drilled holes. Compared with other nebulization
technologies, eFlow Technology produces aerosols with a very high
density of active drug, a precisely defined droplet size and a high
proportion of respirable droplets delivered in the shortest
possible period of time. eFlow Technology is not an ultrasonic
nebulizer technology and is not a general purpose electronic
aerosol generator nebulizer technology. Combined with its quiet
mode of operation, small size, light weight and battery use, eFlow
Technology reduces the burden of taking daily, inhaled
treatments.
About Insmed
Insmed Incorporated is a biopharmaceutical company dedicated to
improving the lives of patients battling serious lung diseases.
Insmed is focused on the development and commercialization of
ARIKAYCE™, or liposomal amikacin for inhalation, for at least two
identified orphan patient populations: patients with nontuberculous
mycobacteria (NTM) lung infections and cystic fibrosis (CF)
patients with Pseudomonas aeruginosa lung infections. For more
information, please visit http://www.insmed.com.
Forward-looking Statements
This release contains forward-looking statements. Words, and
variations of words, such as “intend,” “expect,” “will,”
“anticipate,” “believe,” “continue,” “propose” and similar
expressions are intended to identify forward-looking statements.
Investors are cautioned that such statements in this release,
including statements relating to the status, results and timing of
clinical trials and clinical data, the anticipated benefits of
Insmed’s products, the anticipated timing of regulatory
submissions, and the ability to obtain required regulatory
approvals, the ability to obtain a Breakthrough Therapy Designation
for ARIKAYCE in the U.S., bring products to market and successfully
commercialize products constitute forward-looking statements that
involve risks and uncertainties that could cause actual results to
differ materially from those in the forward-looking statements.
Such risks and uncertainties include, without limitation, failure
or delay of European, Canadian, U.S. Food and Drug Administration
and other regulatory reviews and approvals, competitive
developments affecting the Company’s product candidates, delays in
product development or clinical trials or other studies, patent
disputes and other intellectual property developments relating to
the Company’s product candidates, unexpected regulatory actions,
delays or requests, the failure of clinical trials or other studies
or results of clinical trials or other studies that do not meet
expectations, the fact that subsequent analyses of clinical trial
or study data may lead to different (including less favorable)
interpretations of trial or study results or may identify important
implications of a trial or study that are not reflected in
Company’s prior disclosures, and the fact that trial or study
results or subsequent analyses may be subject to differing
interpretations by regulatory agencies, the inability to
successfully develop the Company’s product candidates or receive
necessary regulatory approvals, inability to make product
candidates commercially successful, changes in anticipated
expenses, changes in the Company’s financing requirements or
ability raise additional capital, and other risks and challenges
detailed in the Company’s filings with the U.S. Securities and
Exchange Commission, including its Annual Report on Form 10-K
for the year ended December 31, 2013. Investors are cautioned
not to place undue reliance on any forward-looking statements that
speak only as of the date of this news release. The Company
undertakes no obligation to update these forward-looking statements
to reflect events or circumstances or changes in its
expectations.
Select ARIKAYCE™ Clinical Data
TARGET NTM (TR02-112 STUDY): Change from
Baseline on the Full Semi Quantitative Scale for Mycobacterial
Culture, mITT Population
Day 84
ARIKAYCE590 mg QD
(N=44)
Placebo(N=45)
Overall(N=89)
-6 1 (2.3) 0 1 (1.1) -5 0 0
0 -4 1 (2.3) 0 1 (1.1) -3 3
(6.8) 1 (2.2) 4 (4.5) -2 4 (9.1) 6
(13.3) 10 (11.2) -1 9 (20.5) 6 (13.3)
15 (16.9) 0 20 (45.5) 20 (44.4) 40 (44.9) +1
3 (6.8) 7 (15.6) 10 (11.2) +2 0
3 (6.7) 3 (3.4) +3 1 (2.3) 0 1 (1.1) +4
1 (2.3) 2 (4.4) 3 (3.4) +5 0 0
0 +6 0 0 0 +7 (Death) 1* (2.3)
0 1 (1.1) * Death determined to be not related to
study drug; data handling rules require any death to be scored as a
+7
TARGET NTM (TR02-112 STUDY): Summary of
semi quantitative data for mITT population
ARIKAYCE
Placebo # of Patients
CumulativeSum ofChanges
onthe Scale
# of Patients
CumulativeSum ofChanges
onthe Scale
Patients with decrease in bacterialdensity
on the scale
18 -36 13 -21
Patients with no change on the scale 20 0
20 0
Patients with increase in bacterialdensity
on the scale
5 +10 12 +21
Patient death 1* +7 0
0
* Death determined to be not related to
study drug, data handling rules require any death to be scored as a
+7
ARIKAYCE: TARGET NTM (TR02-112 STUDY):
Overview of Adverse Events
ARIKAYCE590 mg
QD(N=44)
Placebo(N=45)
Overall(N=89) Number (%) of subjects
with treatment-emergent adverse events 41 (93.2)
40 (88.9) 81 (91.0)
Number of treatment-emergent
adverse events 240 140 380
Number (%) of subjects with treatment-emergent adverse events by
maximum severity Grade 1: Mild 12 (27.3) 25
(55.6) 37 (41.6) Grade 2: Moderate 24 (54.5)
10 (22.2) 34 (38.2) Grade 3: Severe 4 (9.1) 5
(11.1) 9 (10.1) Grade 4: Life Threatening or Disabling
0 0 0 Grade 5: Death 1* (2.3) 0
1 (1.1)
Number (%) of subjects with treatment-emergent adverse events by
seriousness Serious 8 (18.2) 4 (8.9) 12
(13.5) Not Serious 33 (75.0) 36 (80.0) 69
(77.5) Number of treatment-emergent serious adverse events
12 5 17
Number (%) of subjects with treatment-emergent adverse
events by strongest relationship to study drug [1] Related
32 (72.7) 17 (37.8) 49 (55.1) Not Related
9 (20.5) 23 (51.1) 32 (36.0) Number of
subjects who discontinued 9 0 9 * Death
determined to be not related to study drug
Photos/Multimedia Gallery Available:
http://www.businesswire.com/multimedia/home/20140326005630/en/
For Insmed IncorporatedLHAAnne Marie
Fields, 212-838-3777Senior Vice Presidentafields@lhai.comorBruce
Voss, 310-691-7100Managing Directorbvoss@lhai.com
Insmed (NASDAQ:INSM)
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