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|StemCells, Inc. Announces Termination of Phase II Pathway Study Following Review of Data|
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.
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.
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
As of May 31, 2016, the Company had cash and cash equivalents of approximately
“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.
ISSCR 2016 Annual Meeting
The Company will attend the 14th Annual Meeting of the
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
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