StemCells, Inc. (NASDAQ:STEM) (the “Company”) today announced its
decision to terminate the Company’s Phase II Pathway Study in
spinal cord injury following an in-depth review of data from the
study and after obtaining the concurrence of the study’s Interim
Analysis Data Monitoring Committee (the “IA-DMC”). While the
results showed overall improvement in patients treated with the
Company’s proprietary cells, the magnitude of the effect and the
perceived trend of the effect over time did not justify continuing
the study or exploring the variability in the initial patient
observations, given the financial resources available to the
Company.
Seventeen patients have already been dosed in
the Pathway Study. The first cohort of the Pathway Study,
consisting of six patients, was designed to assess the safety, and
preliminary signs of efficacy, of cell administration into the
cervical cord and select the dose level for the 40-patient second
cohort. The second cohort of the Pathway Study was a randomized,
controlled and single-blinded arm of the trial in 40 motor complete
patients.
The six-, nine- and twelve-month results from
the first cohort of the Pathway Study revealed encouraging patterns
of improvements from baseline, especially in the first six months
of the study. This was confirmed separately by a review of the data
by independent experts in spinal cord injury, who agreed that the
overall results indicated evidence of biological activity. However,
the Company observed in this cohort a declining trend in the
magnitude of the effect in both strength and function at the twelve
month time point. While the results at twelve months were still
improved from baseline, this late variability led the Company to
conduct an earlier‑than‑planned interim analysis of the Cohort II
data. The results of this interim analysis, which were
reviewed by the Company as well as by the IA-DMC, showed
differences in motor strength that favored the treatment group, but
the magnitude of the effect led both the Company and the IA-DMC to
conclude that achieving the primary endpoint objective of the
Pathway Study would be unlikely. Based upon these findings, the
Company has decided to terminate the study and close out
operations.
“Despite the outcome of the Pathway Study
reported above, the Company is proud of our team’s numerous
accomplishments and successes to date,” said Dr. Stephen Huhn,
Chief Medical Officer and VP of Clinical Research. “Data from
earlier clinical trials involving the Company’s proprietary
HuCNS-SC® human neural stem cells have demonstrated an early signal
of biological activity in multiple disease indications. Our earlier
Phase I/II clinical trial in chronic thoracic spinal cord injury
showed measureable gains, while the Phase I/II clinical trial in
geographic atrophy showed a positive safety profile and favorable
preliminary efficacy. Additionally, a Phase I study in children
with Batten’s disease showed that transplantation of the cells into
the brain was safe and resulted in long term survival of the
cells.”
Dr. Huhn continued, “Even in the Pathway Study,
we believe we see a biological signal in many of the
patients. Equally important, the first cohort of the study
also confirmed the safety of cell administration into the cervical
cord. The collective human data we have generated across all of our
studies reinforce our belief that our cells have an excellent
safety profile and that there are neurological and retinal
disorders with unmet need that may be helped by cell transplant.
Unfortunately, the Company does not have the resources to implement
changes in our development program to permit further
investigation.”
“That we did not see significant recovery of
motor functions in the Pathway Study is disappointing given the
Company’s nearly complete restoration of motor and sensory
functions with HuCNS-SC cells in spinal cord injured
immunodeficient mice, the recovery of sensory responses in patients
with thoracic spinal cord injury, and the many other encouraging
clinical and preclinical studies with these cells,” added
Dr. Irv Weissman, a Director and co-founder. “Given the
collective strength of past data with these cells, we sincerely
hope others will pick up the many questions we have about the
variability of results seen in the Pathway Study. Naturally,
over the next few weeks, we will endeavor to find a party able to
continue the development of this very promising
technology, which is so important not only for current and
future patients with these devastating diseases, but also for the
field of brain stem and progenitor cell therapies.”
“We would like to thank the patients who
participated in the Pathway Study as well as their families and
caregivers. Spinal cord injury is one of the most devastating
injuries to the central nervous system and any effort to help
advance or investigate a potential therapy is worthwhile. The
Company would also like to thank our clinical trial investigators
and their teams, who have shared our mission in helping these
patients. The Company will work with the clinical sites to suspend
the trial activities and detailed information will be provided to
patients in the trial regarding their participation,” concluded Dr.
Huhn.
The Pathway Study
The Pathway Study was a single blind,
randomized, controlled clinical trial investigating the use of the
Company’s proprietary HuCNS-SC human neural stem cells for the
treatment of chronic spinal cord injuries (SCI). Patients eligible
for the study had complete loss of motor control below the level of
injury, the most severe degree of SCI as defined by the American
Spinal Injury Association Impairment Scale (AIS). Clinicians used
both ISNCSCI (International Standards for Neurological
Classification of Spinal Cord Injury) and GRASSP (Graded Assessment
of Strength Sensibility and Prehension) measures to establish a
pre-transplant baseline for each patient and to assess
post-transplant progress. The goal of the Pathway Study was to
demonstrate improved upper extremity motor function and fine motor
skills.
Interim Analysis
In performing the interim analysis of Cohort II,
an Interim Analysis Data Monitoring Committee consisting of three
leading clinicians in the spinal cord injury field, reviewed the
accrued data to date against specific clinically relevant criteria
linked to achieving the statistically significant result for
improving motor strength and function in treated patients.
Following this analysis, the IA-DMC concluded that the data failed
the futility criteria established for the interim analysis and
recommended cessation of the study. The Company took the
IA-DMC’s recommendation under advisement in making its decision to
terminate the Pathway Study.
Company to Initiate Orderly Wind
Down
The Company also announced that, in light of the
decision to terminate the Pathway Study, the Company’s available
strategic alternatives and its current cash position, the Board of
Directors approved a plan to wind down the Company. As part
of this process, the Company will evaluate opportunities to
monetize its intellectual property, including data collected in its
studies and trade secrets, as well as the transfer of its
proprietary HuCNS-SC cells and other assets through a potential
sale. The Company will not proceed with its earlier plans to
conduct a rights offering, for which it had filed a registration
statement with the SEC.
As of May 31, 2016, the Company had cash
and cash equivalents of approximately $5.5 million. The Company
cannot determine with certainty the amount of any liquidating
distribution to its stockholders and it is possible that there will
be no liquidating distribution to stockholders. The amount of any
cash distributed to its stockholders will depend upon, among other
things, the Company’s current liquid assets offset by its known and
unknown liabilities as well as operating expenses associated with
the wind down.
“We are extremely disappointed with the results
of our Pathway Study, which we had hoped to be the first clinical
program involving cellular transplantation to meaningfully improve
motor function in patients with chronic spinal cord injury,” said
Dr. Ian Massey, President and Chief Executive Officer. “However, we
continue to feel immense pride over the contributions we have made
to the stem cell research field, and we are confident that the
progress we made will be instrumental in future studies in this
area. The other directors and I are also very appreciative of
our employees for their hard work and dedication to our mission,
and thankful to the scientific community and our stockholders for
their support through the years.”
ISSCR 2016 Annual Meeting
The Company will attend the 14th Annual Meeting
of the International Society for Stem Cell Research (ISSCR) in San
Francisco, California, on June 23. The Company will present
Cohort I data from the Pathway Study and its interim analysis
outcome.
Further information about StemCells, Inc.,
including its preclinical and clinical studies, is available
at http://www.stemcellsinc.com.
Apart from statements of historical fact, the
text of this press release constitutes forward-looking statements
within the meaning of the U.S. securities laws, and is subject to
the safe harbors created therein. These statements include, but are
not limited to, statements regarding the future business operations
of StemCells, Inc. (the "Company"), the prospect for the
successful divestiture of any of the Company’s assets, the
possibility of a liquidating distribution to Company stockholders,
and the ability of the Company to pay its creditors and
successfully complete an orderly wind down. These forward-looking
statements speak only as of the date of this news release. The
Company does not undertake to update any of these forward-looking
statements to reflect events or circumstances that occur after the
date hereof. Such statements reflect management's current views and
are based on certain assumptions that may or may not ultimately
prove valid. The Company's actual results may vary materially from
those contemplated in such forward-looking statements due to risks
and uncertainties to which the Company is subject, including risks
whether the FDA and other applicable regulatory agencies
will support the Company’s plans to discontinue its currently
active clinical studies; uncertainty as to whether HuCNS-SC cells
will prove safe and not cause tumors or other adverse side effects
in the patients enrolled in the Company’s clinical studies;
uncertainties whether the Company’s stock will continue to be
listed on any securities exchange; litigation uncertainties;
uncertainties regarding the Company’s plans to wind down
operations; uncertainties regarding the validity, enforceability
and potential value of the Company's patents; uncertainties as to
whether the Company will be able to continue to pay its obligations
in the ordinary course of business as they come due; and other
factors that are described under the heading "Risk Factors" in the
Company's Annual Report on Form 10-K for the year
ended December 31, 2015.
CONTACT:
INVESTOR CONTACT:
Greg Schiffman
StemCells, Inc.
Chief Financial Officer
(510) 456-4128
Robert Haag
Managing Director
IRTH Communications
STEM@irthcommunications.com
1-866-976-4784
MEDIA CONTACT:
Lena Evans
Russo Partners
(212) 845-4262
Kelly Sullivan / Eric Brielmann
Joele Frank Wilkinson Brimmer Katcher
212-355-4449
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