Cytori Therapeutics, Inc. (NASDAQ:CYTX) today announced top-line,
preliminary data from its pivotal STAR trial of HabeoTM Cell
Therapy in patients with scleroderma. While the primary endpoint,
Cochin Hand Function Score (CHFS), did not reach statistical
significance at 24 or 48 weeks, the trial data reported clinically
meaningful improvements in the primary and secondary endpoints of
both hand function and scleroderma-associated functional
disability, for Habeo treated patients compared to placebo, in a
subgroup of patients with diffuse cutaneous scleroderma.
The U.S. multi-center STAR trial enrolled and evaluated 88
patients with scleroderma, including 51 patients within the diffuse
cutaneous subset and 37 with limited cutaneous scleroderma. In the
combined study population, the primary endpoint, specifically mean
improvement in the Cochin Hand Function Score, did not show
statistical difference between treated patients and those receiving
placebo at 24 weeks and 48 weeks as determined by both analysis of
covariance and mixed model repeated measure analysis.
The Raynaud’s Condition Score, a secondary endpoint, improved in
both the treatment and placebo group but was not statistically
different between the Habeo treated and placebo groups.
However, in the pre-specified subgroup analysis of patients with
diffuse cutaneous scleroderma, a more severe form of the disease,
improvements in the Cochin Hand Function Score and the Health
Assessment Questionnaire-Disability Index (HAQ-DI), a measure of
functional disability and an important secondary endpoint, met or
exceeded the published criteria for minimally important clinical
differences in these measures (6.5 points for Cochin1, 0.22 points
for HAQ-DI2).
Endpoint |
Timepoint |
Habeo † / ‡ |
Placebo † / ‡ |
p value † / ‡ |
All Subjects |
|
n=48 |
n=40 |
|
CHFS - mean improvement |
24 weeks |
11.5 / 11.8 |
10.2 / 9.8 |
0.442 / 0.3943 |
48 weeks |
11 / 11.3 |
8.9 / 8.51 |
0.2989 / 0.2650 |
HAQ-DI - mean improvement |
48 weeks |
0.22 / NA |
0.11 / NA |
0.105 / NA |
Diffuse Cutaneous Subgroup |
|
n=32 |
n=19 |
|
CHFS - mean improvement |
24 weeks |
12.8 / 13.3 |
8.0 / 7.2 |
0.111 / 0.078 |
48 weeks |
12.0 / 12.4 |
6.6 / 5.9 |
0.069 / 0.058 |
HAQ-DI - mean improvement |
48 weeks |
0.20 / NA |
0.00 / NA |
0.044 / NA |
† Analysis
of co variance using ANCOVA mean changes from baseline. |
|
|
‡ Mixed
model repeated measure analysis, MMRM mean changes from
baseline. |
|
|
NA Data not
available. |
|
|
|
|
“The safety and efficacy results from this trial in the diffuse
subset of patients with scleroderma are impressive and represent
important new information for the field,” said Dinesh Khanna, MD,
Frederick G.L. Huetwell Professor of Medicine and Director of the
University of Michigan Scleroderma Program. “The STAR trial
suggests that Habeo may provide clinically meaningful improvements
in the hand function and functional disability to patients with
diffuse form of the disease who have no other treatment options.
The diffuse subset has a more severe disease burden with
significant hand dysfunction and internal organ involvement as well
as the highest mortality rate among all rheumatic diseases. It is
important for these patients that this innovative technology moves
forward in the clinical and regulatory process.”
“We are disappointed that the study missed the primary and
secondary endpoints. However, we are very encouraged by the trends
toward improved hand function and scleroderma-related health status
in patients with diffuse cutaneous scleroderma,” said Marc H.
Hedrick, MD, President and Chief Executive Officer of Cytori
Therapeutics, Inc. “We thank the STAR investigators and patients
for participating in this trial, which has yielded new insights
into scleroderma and shows yet again that discrete patient
populations may respond differently to investigational therapies –
an important consideration for individuals currently lacking
treatment options for rare diseases. After we review the complete
data set, we will work collaboratively with our team, trial
investigators, patient advocates and the regulatory bodies in our
key markets, to chart the next steps for this therapy.”
In general, the adverse events were rated as mild to moderate in
the majority of cases and there were no significant safety issues
identified for Habeo or the procedure itself (including liposuction
and finger injection in the placebo group) during the trial.
“We are pleased with both the efficacy trends as well as the
safety profile of Habeo,” said Dr. Mark Marino, MD, Senior Vice
President and Chief Medical Officer of Cytori Therapeutics, Inc.
“Following the evaluation of the full STAR data set, Cytori, in
conjunction with the investigators, intends to submit the full data
set, as a late-breaking abstract, to the American College of
Rheumatology meeting in November 3-8, 2017. Simultaneously, we
anticipate seeking a post-trial meeting with FDA as soon as
possible to define next steps. We further plan to continue to
support the investigator-initiated SCLERADEC-II trial in France,
which is over 50 percent enrolled.”
Management Conference Call Webcast Cytori
will host a management conference call at 8:30 a.m. Eastern Time
today to further discuss the preliminary top-line results from the
STAR trial. The webcast will be available live and by replay two
hours after the call and may be accessed under "Webcasts" in the
Investor Relations section of Cytori's website. If you are unable
to access the webcast, you may dial in to the call at
+1.877.402.3914, Conference ID: 60408495.
About STAR The STAR trial was a
prospective, double-blind, randomized, multicenter, parallel-group
Phase III pivotal study assessing the safety and efficacy of a
single, subcutaneous administration of Habeo Cell Therapy (40
million cells per subject) into the fingers of patients with hand
dysfunction due to scleroderma. The subjects were randomized 1:1 to
receive either Habeo Cell Therapy or placebo. Investigators
conducted final assessments at 48 weeks.
The primary study endpoint was improvement in the Cochin Hand
Function Score, a self-reported measure of hand function, which was
assessed at 24 and 48 weeks. The Cochin score is based on 18
questions relating to hand function; each question is graded on a
0–5 scale, with a total score of 90 points reflecting maximal
disability.
The Health Assessment Questionnaire-Disability Index (HAQ-DI), a
measure of functional disability and a secondary endpoint in the
STAR trial, consists of questions pertaining to activities of daily
living graded on a 0–3 scale (with 3 representing “unable to
perform”); the HAQ-DI also includes several visual analog scales
(VAS) for different body systems.
The Raynaud’s Phenomenon Condition Score, a secondary endpoint
in the STAR trial, is a patient reported outcome measure asking the
patients how much difficulty they had with their Raynaud’s symptoms
over the past 24 hours graded on a scale of 0 – 10 with 0 being no
difficulty and 10 being extreme difficulty.
Details of the STAR trial, including inclusion and exclusion
criteria, can be found at clinicaltrials.gov.
About Scleroderma Scleroderma is a rare
and chronic connective tissue disease generally classified as an
autoimmune rheumatic disorder. The word “scleroderma” is derived
from two Greek words: “sclera,” which means hard, and “derma,”
meaning skin, as hardening of the skin is one of the most visible
manifestations of the disease. An estimated 300,000 Americans have
scleroderma, about one-third of whom have the systemic form of the
disease, known as systemic sclerosis (SSc). SSc is further
sub-classified as diffuse cutaneous and limited cutaneous SSc.
Patients with diffuse cutaneous SSc have more severe disease with
significant hand dysfunction and internal organ involvement.
Diffuse scleroderma accounts for between one-third and one-half of
all cases of systemic sclerosis.3,4
SSc contributes to hand impairment through inflammatory
arthritis or inflammation of the joints, joint contractures,
Raynaud’s Phenomenon (RP, skin discoloration resulting from
narrowing of the blood vessels in response to cold, emotional
upset, or stress), digital ulcers, puffy hands and skin fibrosis
over the fingers and hands, and calcinosis (calcium deposits in the
soft tissues of the hand). These manifestations, which often
coexist, can contribute to difficulty with occupational activities
and activities of daily living, which can impair quality of life.
Whereas current treatment recommendations focus on management of
internal organ involvement, there is little treatment available for
hand impairment.
About SCLERADEC-II SCLERADEC-II is an
investigator initiated, multicenter, double blind, placebo
controlled trial of a single administration of Habeo Cell Therapy.
The trial contemplates enrollment of up to 40 patients randomized
in a 1:1 fashion to receive either active treatment or placebo
control. The primary endpoint is the Cochin Hand Function Score at
3 months following treatment. Key secondary endpoints include
Raynaud’s Condition Score, HAQ-DI, pain, the modified Rodnan Skin
Score, capillaroscopy and functional hand assessment. Patients
receiving placebo will be eligible for cross-over to the active arm
after 6 months with their respective cryopreserved cells.
About Habeo Cell Therapy Cytori is
developing cell therapies that harness the unique attributes of
adipose-derived regenerative cells (ADRCs), which are living cells
that are present in an adult human’s own adipose tissue.
Habeo Cell Therapy is a suspension of ADRCs that are
manufactured from a single lipoaspirate (material removed via
liposuction, a procedure in which fat is removed from under the
skin by suction). The process concentrates ADRCs intended for
autologous re-implantation subcutaneously into the digits. The
resultant cell suspension contains critical cells naturally
occurring in the patient’s own tissue. Preparation of autologous
ADRCs for subcutaneous delivery involves no cell culture and can be
prepared and re-implanted into the same patient within four
hours.
About Cytori
TherapeuticsCytori is a therapeutics company
developing regenerative and oncologic therapies from its
proprietary cell therapy and nanoparticle platforms for a variety
of medical conditions. Data from preclinical studies and clinical
trials suggest that Cytori Cell Therapy™ acts principally by
improving blood flow, modulating the immune system, and
facilitating wound repair. As a result, Cytori Cell Therapy™ may
provide benefits across multiple disease states and can be made
available to the physician and patient at the point-of-care through
Cytori’s proprietary technologies and products. Cytori
Nanomedicine™ is developing liposome encapsulated therapies for
regenerative medicine and oncologic indications. For more
information, visit www.cytori.com.
Cautionary Statement Regarding Forward-Looking
StatementsThis press release includes forward-looking
statements regarding events, trends and business prospects, which
may affect Cytori’s future operating results and financial
position. Such statements, include, but are not limited to,
statements regarding, Cytori’s plans to evaluate the STAR data in
consultation with various internal and external resources, experts
and the FDA; Cytori’s conduct of its STAR trial, and possible
future clinical trials of Habeo Cell Therapy; Cytori’s
intention to continue its support of the investigator-initiated
SCLERADEC II trial; the ability to fully characterize the efficacy
and safety profile of Habeo Cell Therapy through further study and
the potential to yield additional insights into its clinical
utility; and the ability of Cytori Cell Therapy™ to provide
benefits across multiple disease states and be made available to
the physician and patient at the point-of-care through Cytori’s
proprietary technologies and products. These statements are subject
to risks and uncertainties that could cause Cytori’s actual results
and financial position to differ materially. Some of these risks
and uncertainties include: risks in the conduct of Cytori’s STAR
trial and future clinical trials (including risks in the collection
and results of clinical data); risks associated with the conduct of
investigator-initiated trials using our cellular therapeutics,
including the French, investigator-initiated SCLERADEC II trial;
risks associated with potential benefits of Cytori’s products
(including any potential benefits of Habeo Cell Therapy identified
in the STAR trial); risks associated with development of Cytori’s
clinical pipeline, including the possibility that Cytori may
determine that there may not be a viable continued development path
for Habeo Cell Therapy; final clinical outcomes (including the risk
that top-line data may not accurately reflect the complete results
of a particular study or trial); regulatory risks and
uncertainties, including the risk that FDA and other
regulatory authorities may not approve Habeo Cell Therapy, or that
any marketing approvals, if granted, may have significant
limitations on their use; risks related to reimbursement (including
failure to achieve desired pricing for Habeo Cell Therapy); risks
related to dependence on third party performance; the risk that
Habeo Cell Therapy may never be successfully commercialized, or
receive anticipated levels of commercial acceptance; Cytori’s
ability to raise additional funding that it may need to continue to
pursue its commercial and business development plans; and other
risks and uncertainties described under the "Risk Factors" section
in Cytori's Securities and Exchange Commission filings on Form 10-K
and Form 10-Q. Cytori assumes no responsibility to update or revise
any forward-looking statements contained in this press release to
reflect events, trends or circumstances after the date of this
communication.
1 Nguyen C, Bérezné A, Mestre-Stanislas, et al. Changes over
time and responsiveness of the Cochin Hand Function Scale and Mouth
Handicap in Systemic Sclerosis Scale in patients with systemic
sclerosis: a prospective observational Study. American Journal of
Physical Medicine & Rehabilitation. 2016;95(12):e189-e197.
2 Pope J. Measures of systemic sclerosis (scleroderma): Health
Assessment Questionnaire (HAQ) and Scleroderma HAQ (SHAQ),
Physician- and Patient-Rated Global Assessments, Symptom Burden
Index (SBI), University of California, Los Angeles Scleroderma
Clinical Trials Consortium Gastrointestinal Scale (UCLA SCTC GIT)
2.0, Baseline Dyspnea Index (BDI) and Transition Dyspnea Index
(TDI) (Mahler’s Index), Cambridge Pulmonary Hypertension Outcome
Review (CAMPHOR), and Raynaud’s Condition Score (RCS). Arthritis
Care & Research. ol. 2011;63(S11):S98 –S111. doi
10.1002/acr.20598.
3 http://www.scleroderma.org/site/PageNavigator/patients_whatis.html#.WWZdOYjyvGh
4 Arthritis Rheumatism Vol 48, (8), August 2003, pp 2246–2255
DOI 10.1002/art.11073
Investor Contact:
Tiago Girao
Cytori Therapeutics, Inc.
1-858-458-0900
Media Contact:
Rob Murphy
SmithSolve LLC
1-973-442-1555 ext. 116
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