Neuralstem, Inc. (NASDAQ:CUR), a biopharmaceutical company focused on the development of central nervous system therapies based on its neural stem cell technology, announced that the first patient has been enrolled in the double-blind, placebo-controlled multi-center Phase 2 study of NSI-189 for the treatment of major depressive disorder (MDD).
NSI-189, the lead compound in Neuralstem’s neurogenic small molecule program, is a proprietary, new chemical entity that stimulates neurogenesis, synaptogenesis and increases hippocampal volume, all of which are believed to be effective in reversing depression, enhancing cognition, and promoting neuroregeneration. The Phase 2 trial will randomize 220 patients, in three cohorts (two doses plus placebo), at 12 select MDD trial sites, under the direction of principal investigator (PI) Maurizio Fava, MD, Executive Vice Chair, Department of Psychiatry and Executive Director, Clinical Trials Network and Institute, Massachusetts General Hospital.. The primary efficacy endpoint is a reduction in depression symptoms, as measured by the Montgomery-Asberg Depression Rating Scale (MADRS). Secondary endpoints encompass additional clinical outcomes, including cognition improvement measures. The trial, designed to reduce the placebo effect and site variability, will last for 12 weeks, with an observational follow-up period of six months to assess NSI-189’s long-lasting durability of benefits. Neuralstem expects to report results in the second half of 2017.
“A new class of treatment is needed in major depression, where existing compounds are not effective for all patients and have a high side effect profile, so patients discontinue treatment,” said Maurizio Fava, MD, Executive Vice Chair, Department of Psychiatry and Executive Director, Clinical Trials Network and Institute, Massachusetts General Hospital. “We were encouraged by the signs of improvement in the depression and cognitive symptoms of MDD patients, as witnessed in Phase 1 with NSI-189, and look forward to validating in Phase 2.”
“NSI-189 works via a new pathway that is different from current antidepressants. It appears to create long-lasting, positive structural changes in the brain with long-lasting clinical benefits,” commented Karl Johe, Ph.D., Chairman and Chief Scientific Officer, Neuralstem. “In animal models, we saw significant increases in neurogenesis, synaptogenesis, and hippocampal volume. In humans, the therapeutic effects seen in Phase 1b patients after completion of the 28- day dosing, persisted for additional 56 days without the drug, which supports the hypothesis of a new mechanism of action that induces long-lasting structural changes in key areas of the brain.”
In Phase 1b safety trial data in patients with MDD, published in Molecular Psychiatry. NSI-189 was shown to be safe and demonstrated large treatment effects in two key depression outcome measures. These were Symptoms of Depression Questionnaire (SDQ) and MADRS, with large effect sizes (d=0.90, p=0.02; d=0.95, p=0.09 respectively), compared to control. Effect sizes of all four measured outcomes were consistent with a medium-to- large effect size. The study also showed significant improvement in cognitive symptoms, as measured by the Cognitive and Physical Functioning Questionnaire (CPFQ), compared to placebo. Brain imaging (Quantitative EEG) showed increase in alpha brain waves in two parts of the brain (left posterior temporal and left parietal region), both involved in depression and cognition, compared to placebo. Pharmacologically, a steady state was reached after 96-120 hours across all doses. Mean time to maximal peak concentration ranged from 1 to 2 hours at day one and at day 28 of dosing. There were no significant adverse effects. (Original Source)
Shares of Neuralstem are currently trading at $0.325, down $0.007 or 2.20%. CUR has a 1-year high of $2.37 and a 1-year low of $0.30. The stock’s 50-day moving average is $0.64 and its 200-day moving average is $0.89.
On the ratings front, Brean Murray Carret analyst Jonathan Aschoff reiterated a Buy rating on CUR, with a price target of $8, in a report issued on February 17. The current price target represents a potential upside of 2324.2% from where the stock is currently trading. According to TipRanks.com, Aschoff has a yearly average return of -12.4%, a 32.2% success rate, and is ranked #3824 out of 3845 analysts.
Neuralstem, Inc. is a biopharmaceutical company, which is focused on the research, development and commercialization of central nervous system therapies based on human neuronal stem cells and small molecule compounds. Its patented technology enables the commercial-scale production of multiple types of central nervous system stem cells, which are under development for the potential treatment of central nervous system diseases and conditions. The company was founded by I. Richard Garr, Karl Y. Johe and Merrill Solomon in 1996 and is headquartered in Germantown, MD.