International Stem Cell Corporation Announces Positive Top-Line Preliminary Results from Parkinson's Disease Clinical Trial
CARLSBAD, Calif., Nov. 05, 2018 (GLOBE NEWSWIRE) -- via NetworkWire - International Stem Cell Corporation (OTCQB: ISCO) (www.internationalstemcell.com) ("ISCO" or "the Company"), a California-based clinical stage biotechnology company developing stem cell-based therapies and biomedical products, announced today positive 12-month results of the first cohort and six-month interim results of the second cohort of its currently-ongoing, single-arm, open-label phase 1 clinical study (NCT02452723) evaluating the safety and tolerability of its lead candidate, ISC-hpNSC®, a cellular therapeutic consisting of human parthenogenetic neural stem cells, for the treatment of Parkinson's disease (PD).
Six of the 12 patients in the clinical trial of have now completed one year of follow-up observations and one patient in the low dose cohort has now been followed for 2 years. There have been no safety signals or serious adverse effects seen to date as related to the transplanted ISC-hpNSC® cells.
"We are excited about our clinical study progress and results. Patients followed for one year after cell transplantation have reported improvements in a Parkinson's specific quality of life measure when compared to baseline evaluations. In this context, the results are encouraging that the procedure of transplantation may be well tolerated," commented ISCO's Executive Vice President and Chief Scientific Officer Russell Kern, PhD.
The evaluation is based on more than 12 months of safety data from the first cohort (low dose), and 6 months of data from the second cohort (mid dose). Each cohort is comprised of 4 patients. Dosing of patients in Cohort 3, which is receiving the highest dose, is ongoing.
The Melbourne-based Data Safety Monitoring Board (DSMB) has overview responsibilities for evaluating adverse event data from the study and has determined that the favorable safety profile of ISC-hpNSC justified proceeding from Cohort 1 to Cohort 2 and Cohort 3.
In terms of preliminary efficacy, where scores are compared against baseline before transplantation, we observed a dose-pendent response. The % OFF-Time, which is the time of day when levodopa medication is not performing optimally, and PD symptoms return, decreased an average of 24% for the first cohort and 49% for the second cohort at six months post-transplantation. The same was true for % ON-Time without dyskinesia, which is the time of day when levodopa medication is performing optimally without dyskinesia. The % ON-Time increased an average of 19% for the first cohort and 33% for the second cohort during the same period. The quality of life of the patients as measured by the Parkinson's Disease Quality of Life Score-39 (PDQ-39) Summary Index, improved 15% for the first cohort and 47% for the second cohort at six months post-transplantation. These results should take into account that this Phase 1 safety study (due to its size), lacks the statistical power to generate efficacy conclusions. In addition, there was no placebo control group. Proper comparisons against a control group will be performed in the Phase 2 clinical trial that we are currently planning.
About the clinical study The Phase 1 clinical study is a dose escalation safety and preliminary efficacy study of ISC-hpNSC®, intracranially transplanted into patients with Parkinson's disease. The open-label, single center, uncontrolled clinical trial will evaluate three different dose regimens of 30 million to 70 million neural cells. A total of 12 participants with Parkinson's disease will be treated. Following transplantation, the patients will be monitored for 12 months at specified intervals to evaluate the safety and biologic activity of ISC-hpNSC®. A PET scan will be performed at baseline, as part of the screening assessment, and at 6 and 12 months after surgical intervention. Clinical responses compared to baseline after the administration of ISC-hpNSC® will be evaluated using various neurological assessments such as Unified Parkinson Disease Rating Scale (UPDRS), Hoehn and Yahr as well as other rating scales. An extension phase of the study will evaluate patients every 6 months for 5 additional years.
About Parkinson's disease Parkinson's disease is a degenerative disorder of the central nervous system mainly affecting the motor system. The motor symptoms of Parkinson's disease result from the death of dopamine-generating cells in the substantia nigra, a region of the midbrain. Early in the course of the disease, the most obvious symptoms are movement-related. These symptoms include shaking, rigidity, slowness of movement and difficulty with walking and gait. Later, thinking and behavioral problems may arise, with dementia commonly occurring in the advanced stages of the disease. Depression is the most common psychiatric symptom. Parkinson's disease is more common in people over the age of 50. There are no approved treatments that restore the damaged dopaminergic neurons. Medications typically used in the treatment of Parkinson's disease, levodopa and dopamine agonists, improve the early symptoms of the disease. As the disease progresses and dopaminergic neurons continue to be lost, the drugs eventually become ineffective, while at the same time frequently producing a complication marked by involuntary writhing movements. There are over 10 million people afflicted with Parkinson's disease, worldwide. In 2013 Parkinson's disease resulted in about 103,000 deaths, globally.In 1990, the death toll recorded was 44,000.
About ISC-hpNSC® International Stem Cell Corporation's proprietary ISC-hpNSC® consists of a highly pure population of neural stem cells derived from human parthenogenetic stem cells. ISC-hpNSC® is a suspension of clinical grade cells manufactured under cGMP conditions that have undergone stringent quality control measures and are free of any microbial and viral contaminants. Preclinical studies in rodents and non-human primates have shown improvement in Parkinson's disease symptoms and increase in brain dopamine levels following the intracranial administration of ISC-hpNSC®. ISC-hpNSC® provides neurotrophic support and neuroregeneration to the dying dopaminergic neurons of the recipient Parkinson's disease brain. Additionally, ISC-hpNSC® is safe, well tolerated and has shown not to cause adverse events such as dyskinesia, systemic toxicity or tumors in preclinical models. International Stem Cell Corporation believes that ISC-hpNSC® may have broad therapeutic applications for many neurological diseases affecting the brain, the spinal cord and the eye.
About International Stem Cell Corporation International Stem Cell Corporation (ISCO) is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs). hpSCs avoid ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell™. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide, through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at www.internationalstemcell.com.
Safe harbor statement Statements pertaining to anticipated developments, clinical studies expectations(including plans for future clinical trials), progress of research and development, and other opportunities for the Company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates,") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the Company's Securities and Exchange Commission filings. The Company disclaims any intent or obligation to update forward-looking statements.
International Stem Cell Corporation Russell Kern, PhD Executive Vice President, Chief Scientific Officer firstname.lastname@example.org