Neuralstem, Inc. (NASDAQ:CUR), a bipharmaceutical company using neural stem cell technology to develop regenerative theroapies and small molecule pharmaceutical drugs for CNS diseases and disorders, announced that Karl Johe, Ph.D., Neuralstem’s Chairman and Chief Scientific Officer, provided an update on the company’s ongoing NSI-566 cell therapy clinical programs at the Phacilitate Cell & Gene Therapy World conference in Washington D.C.
The presentation reviewed the company’s NSI-566 human spinal cord-derived neural stem cells investigational trials for the treatment of amyotrophic lateral sclerosis (ALS), chronic spinal cord injury (cSCI), and motor deficits due to ischemic stroke. Dr. Johe highlighted the consistent biological activity and multiple modes of therapeutic actions, including the rescue of motor neurons, motor improvement, and neuronal integration in NSI-566 animal and human clinical data. He concluded that the collective trial data analysis showed the cells consistently demonstrated biological activity in all three indications.
Neuralstem’s NSI-566 cells have been safely administered to 40 patients, in dosing ranges of 1.2 million to 24 million cells per patient, in four investigational safety trials. The consistent safety profile continues to validate Neuralstem’s physiologically relevant stem cell technology platform. The company and its collaborators from leading research institutions conclude that the data supports the advancement of clinical development in each of the three indications.
“The consistent biological activity of motor improvement by NSI-566 across multiple disease conditions in humans supports our regenerative hypothesis and is consistent with our preclinical data,” said Dr. Johe. “Based on these encouraging results, we are preparing to conduct additional clinical trials in in each of these incurable neurodegenerative indications.”
NSI-566/ALS: A review of the Phase I and Phase II trials suggested a treatment-emergent improvement of function along multiple endpoints, including improved lung capacity, muscle strength, and a slowing of ALS progression. Phase I data shows long-term graft survival with transient immunosuppression. Additionally in Phase II, the data revealed that more than 50% of the patients experienced a reduction in the ALS Functional Rating Scale (ALSFRS) decline when compared to historical data sets. The remaining subjects, the majority of which had very low grip strength at entry, did not experience a change in their rate of decline. Based on these observations, the next trial will only include patients with sufficient muscle strength remaining for potential rescue.
NSI-566/cSCI: The Phase I feasibility study involved four AIS A thoracic-spinal cord injury patients (motor and sensory complete), one-to-two years post-injury at the time of stem cell treatment. They each received 1.2 million cells in six injections around the site of the spinal cord injury. The stem cell treatment demonstrated feasibility and safety; there were no serious adverse events. A self-reported ability to contract some muscles below the level of injury was confirmed via clinical and electrophysiological follow-up examinations in one of the four patients treated. There was no change in the clinical status of the three other patients. All patients will be followed for a total of five years. Dr. Johe commented that the investigators are planning to add a second cohort of four more patients to the study.
NSI-566/Ischemic Stroke: Dr. Johe presented encouraging preliminary results from the first cohort of patients in the Phase I NSI-566 feasibility study for treatment of paralysis from stroke. The Phase I trial is designed to treat three cohorts of three post-stroke patients each, with Cohort A receiving 5 injections of 40,000 cells each; Cohort B receiving 5 injections of 80,000; and Cohort C receiving 15 injections of 80,000 cells each. The clinicians have completed dosing the second cohort. Dr. Johe added that an innovative brain injection cannula, which can safely inject higher doses of cells, was introduced into the study. The trial is being conducted at BaYi Brain Hospital in Beijing, China. (Original Source)
Shares of Neuralstem closed yesterday at $0.66. CUR has a 1-year high of $3.92 and a 1-year low of $0.52. The stock’s 50-day moving average is $0.95 and its 200-day moving average is $1.28.
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 September 30. The current price target implies an upside of 1094.0% from current levels. According to TipRanks.com, Aschoff has a total average return of -11.2%, a 33.1% success rate, and is ranked #3568 out of 3607 analysts.
Neuralstem Inc is a biotechnology company. It is engaged in the development and commercialization of treatments for central nervous system disease based on transplanting human neural stem cells and the use of small molecule drugs.