Living Cell Technologies : Treatment completed for all patients in Parkinson’s trial

Treatment completed for all patients in Parkinson's trial

1 May 2017 - Sydney, Australia & Auckland, New Zealand - Living Cell Technologies Limited has completed treatment of all six patients in the third and final group of patients in the Phase IIb clinical trial of NTCELL® for Parkinson's disease, at Auckland City Hospital.

Four patients had 120 NTCELL microcapsules implanted into the putamen on each side of their brain, and two patients had sham surgery with no NTCELL implanted. To date there are no safety issues in any of the six patients.

The company is blind to the results until 26 weeks after the completion of group 3 of the trial. The results will then be analysed in accordance with the statistical plan and the conclusions announced. This is anticipated to occur in November 2017. Thereafter the patients who received the placebo will receive the optimal treatment.

The Phase IIb trial aims to confirm the most effective dose of NTCELL, define any placebo component of the response and further identify the initial target Parkinson's disease patient sub group. Providing the trial is successful, the company will apply for provisional consent in Q4 2017 with a view to treating paying patients in New Zealand in 2018.

"The completion of treatment for the patients in group 3 brings us a step closer to our goals of obtaining provisional consent and launching NTCELL as the first disease modifying treatment for Parkinson's disease," says Dr Ken Taylor, CEO of Living Cell Technologies.

NTCELL, a unique cell therapy, is an alginate coated capsule containing clusters of neonatal porcine choroid plexus cells that are sourced from a unique herd of designated pathogen-free pigs bred from stock originally discovered in the remote sub-Antarctic Auckland Islands. Choroid plexus cells are naturally occurring "support" cells for the brain and secrete cerebrospinal fluid (CSF), which contains

a range of factors that support nerve cell functions and protective enzymes that are crucial for nerve growth and healthy functioning. In NTCELL, the porcine choroid plexus cells are coated with LCT's propriety technology IMMUPEL™ to protect them from attack by the immune system. Therefore, no immunosuppressive regimen is required for treatment.

Following implantation into a damaged site within the brain, NTCELL functions as a neurochemical factory producing CSF and secreting multiple nerve growth factors that promote new central nervous system (CNS) growth and repair disease-induced nerve degeneration while potentially removing waste products such as amyloids and proteins.

LCT has global patents pending entitled "Treatment of CNS disease with encapsulated inducible choroid plexus cells". LCT also has gene chip analysis of NTCELL identifying multiple growth and trophic factors, antioxidants, chaperone molecules and other bioactive components.

NTCELL has the potential to treat neurodegenerative diseases because choroid plexus cells help produce CSF as well as a range of neurotrophins (nerve growth factors) that have been shown to protect against neuron (nerve) cell death in animal models of disease. NTCELL has been shown in preclinical studies to regenerate damaged tissue and restore function in animal models of Parkinson's disease, stroke, Huntington's disease, hearing loss and other non-neurological conditions, such as wound healing. In addition to Parkinson's disease, NTCELL has the potential to be used in a number of other CNS indications, including Huntington's, Alzheimer's and motor neurone diseases including amyotrophic lateral sclerosis (ALS).

Parkinson's disease is a progressive neurological condition characterised by a loss of brain cells that produce dopamine (a neurotransmitter that conveys messages between brain cells to ensure effective movement and planning of movement) and many other types of neurons. People with Parkinson's disease experience reduced and slow movement (hypokinesia and bradykinesia), rigidity and tremors.

Parkinson's disease is the second most common neurodegenerative disorder after Alzheimer's disease, affecting approximately 7 million people worldwide. The average age of onset is 60 years, and the incidence increases with age. Men are one and a half times more likely to have Parkinson's disease than women.

Current treatments for Parkinson's disease are symptomatic and do not reverse or slow the degeneration of neurons in the brain. Most existing pharmaceutical treatment options focus on restoring the balance of dopamine and other neurotransmitters. The effectiveness of dopamine replacement therapy declines as the disease progresses. When dopamine treatments are no longer useful, some patients are treated with Deep Brain Stimulation (DBS), in which a medical device is surgically implanted in the brain in order to send electrical impulses to regions of the brain involved in the control of movement. While DBS leads to short-term symptomatic improvement, it does not impact disease progression and is not curative or neuroprotective.

Living Cell Technologies Limited (LCT) is an Australasian biotechnology company improving the wellbeing of people with serious diseases worldwide by discovering, developing and commercialising regenerative treatments which restore function using naturally occurring cells.

LCT's lead product, NTCELL®, is an alginate coated capsule containing clusters of neonatal porcine choroid plexus cells. After transplantation NTCELL functions as a biological factory, producing factors to promote new central nervous system growth and repair disease-induced nerve degeneration.

The Phase I/IIa NTCELL clinical trial in New Zealand for the treatment of Parkinson's disease met the primary endpoint of safety and reversed progression of the disease two years after implant. Results from this trial were used to design a larger Phase IIb trial to confirm the most effective dose of NTCELL, define any placebo component of the response and further identify the initial target Parkinson's disease patient sub group. This trial commenced in March 2016. If the trial is successful,

the company will apply in late 2017 for provisional consent to treat paying patients in New Zealand and launch NTCELL as the first disease modifying treatment for Parkinson's disease.

In addition to Parkinson's disease, NTCELL has the potential to be used in a number of other central nervous system indications, including Huntington's, Alzheimer's and motor neurone diseases including amyotrophic lateral sclerosis (ALS).

LCT's proprietary encapsulation technology, IMMUPEL™, allows cell therapies to be used without the need for co-treatment with drugs that suppress the immune system.

LCT is listed on the Australian (ASX: LCT) and US (OTCQX: LVCLY) stock exchanges. The company is incorporated in Australia, with its operations based in New Zealand.

For more information visit www.lctglobal.com or follow @lctglobal on Twitter.

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