Selected, preliminary data from the MERIT |
research abstract stated,“ This current study |
complete remission( CR) or a complete remission |
trial is expected to be presented at the ALK company- |
investigates whether mesencephalic astrocyte-derived |
with incomplete blood count recovery( Cri). |
sponsored symposium on June 23 during the EAACI- |
neurotrophic factor( MANF) inhibits oxygen-glucose |
“ The initiation of this randomized phase II |
WAO( European Academy of Allergy and Clinical |
deprivation( OGD)-induced cell damage and |
trial is an important milestone in the clinical |
Immunology – World Allergy Organization) |
inflammatory cytokine secretion by suppressing |
development of Pracinostat,” said Robert D Mass, |
Congress. This announcement does not impact ALK ' s |
endoplasmic reticulum stress in rat primary |
MD, chief medical officer of MEIPharma.“ The |
financial guidance for 2013. ALK is a research-driven |
astrocytes. We found that MANF alleviated OGD- |
results from the pilot study reported at ASH were |
global pharmaceutical company focusing on allergy |
induced astrocyte damage and rescued the cell |
very exciting and helped to inform the design of this |
prevention, diagnosis and treatment. |
viability, and the upregulation of GRP78 |
more robust, placebo-controlled trial. Now, our goal |
POSITIVE RESULTS ON MANF |
( endoplasmic reticulum( ER) stress marker) and NF- |
is to build on these preliminary data and get a more |
NEUROPROTECTIVE PROPERTIES |
? B p65( one of the central mediators of |
precise estimate of the clinical benefit of Pracinostat |
DEMONSTRATED IN PRECLINICAL STUDY: |
proinflammatory pathways) induced by OGD were |
in combination with standard-of-care.” |
Amarantus BioScience Holdings, Inc., a |
significantly reduced by preincubation of MANF. In |
The multi-centre phase II trial is expected to |
biotechnology company discovering and developing |
addition, the increases of secretion and mRNA |
enroll 100 patients with a one-to-one randomization. |
treatments and diagnostics for diseases associated |
expression levels of the proinflammatory cytokines |
Completion of enrollment is anticipated by June 2014 |
with neurodegeneration and apoptosis, has reported |
IL-1ß, IL-6, and TNF-a in astrocytes induced by |
with topline data in December 2014. The primary |
positive neuroprotective properties for its MANF |
OGD were significantly suppressed by MANF.” |
endpoint of the study is complete remission( CR). |
therapeutic were demonstrated in a preclinical |
MANF( Mesencephalic-Astrocyte-derived |
Secondary endpoints include overall response rate |
research study conducted by the Department of |
Neurotrophic Factor) is believed to have broad |
( CR + CRi + PR), haematologic improvement, duration |
Orthopaedics at Qilu Hospital of Shandong |
potential because it is a naturally-occurring protein |
of response, progression-free survival, rate of |
University, in Jinan, China. |
produced by the body for the purpose of reducing and |
leukaemic transformation, overall survival and safety. |
Specifically, a published abstract of the study |
preventing apoptosis( cell death) in response to injury |
The trial is being conducted in collaboration with the |
concluded,“ These findings demonstrate that MANF |
or disease, via the unfolded protein response. By |
Sarah Cannon Research Institute; Dr Guillermo |
shows the potential to alleviate cell damage and |
manufacturing MANF and administering it to the |
Garcia-Manero of the MD Anderson Cancer Centre is |
inflammation in rat primary astrocytes by suppressing |
body, Amarantus is seeking to use a regenerative |
the principal investigator. |
ER( endoplasmic reticulum) stress, indicating that |
medicine approach to assist the body with higher |
“ This represents the first in a series of phase II |
MANF plays an important role in astrocyte |
quantities of MANF when needed. Amarantus is the |
studies we have planned for Pracinostat in the months |
inflammation and functioning and may suggest a |
front-runner and primary holder of intellectual |
ahead,” said Daniel P Gold, Ph. D., president and |
promising strategy for neuroprotection in the central |
property( IP) around MANF, and is initially focusing |
chief executive officer of MEI Pharma.“ We believe |
nervous system. |
on the development of MANF-based protein |
that Pracinostat truly has the potential to become a |
“ The findings from the study at Shandong |
therapeutics. MANF ' s current lead indication is |
best-in-class drug. We intend to execute a |
University are consistent with the results we obtained |
Parkinson ' s disease with additional focus on |
comprehensive development programme in order to |
in our own research with MANF on rat models for |
Traumatic Brain Injury( TBI). Future indications may |
realize its full potential and determine the most |
Parkinson ' s disease, which were reported in March |
include myocardial infarction and certain rare and |
efficient registration path forward.” |
2013,” said John W. Commissiong, Ph. D., chief |
ultra-rare orphan diseases for which MANF is |
In addition to the randomized phase II clinical |
scientific officer of Amarantus.“ We are very pleased |
currently being evaluated. |
trial in frontline MDS, the Company is also preparing |
that independent research is contributing to the |
Amarantus is a development-stage |
for the initiation of two open-label phase II trials of |
further understanding of MANF. The protective |
biotechnology company and focussed on developing |
Pracinostat: one in combination with Vidaza in |
activity demonstrated by this independent work |
certain biologics surrounding the intellectual property |
frontline acute myeloid leukaemia( AML) in the fall |
clearly suggests MANF may have utility in the |
and proprietary technologies it owns to treat and / or |
of 2013 and the other in combination with Vidaza or |
treatment of conditions such as stroke, post-stroke |
diagnose Parkinson ' s disease, Traumatic Brain Injury, |
Dacogen( decitabine) in patients with refractory MDS |
recovery, Traumatic Brain Injury and concussion, as |
Ischemic Heart Disease and other human diseases. |
soon thereafter. |
well as having relevance to the use of MANF in |
PHASE II TRIAL OF PRACINOSTAT IN |
Pracinostat is an orally available histone |
Parkinson ' s disease. We continue to conduct |
COMBO WITH VIDAZA TO TREAT |
deacetylase( HDAC) inhibitor that has been tested in |
additional experiments with MANF in support of a |
MYELODYSPLASTIC SYNDROME:
MEI
|
a number of phase I and exploratory phase II clinical |
planned Investigational New Drug( IND) application |
Pharma, Inc., an oncology company focused on the |
trials in advanced haematologic disorders and solid |
to the FDA.” |
clinical development of novel therapies for cancer, |
tumour indications in both adult and paediatric |
An abstract of the research report titled |
has dosed its first patient in a phase II clinical trial of |
patients. Pracinostat has been generally well tolerated |
“ Mesencephalic Astrocyte-Derived Neurotrophic |
its lead drug candidate Pracinostat in combination |
in more than 200 patients to date, with readily |
Factor Inhibits Oxygen-Glucose Deprivation-Induced |
with Vidaza( azacitidine) in patients with previously |
manageable side effects that are often associated with |
Cell Damage and Inflammation by Suppressing |
untreated intermediate-2 or high-risk myelodysplastic |
drugs of this class, such as fatigue. Pracinostat has |
Endoplasmic Reticulum Stress in Rat Primary |
syndrome( MDS). The randomized, double-blind trial |
exhibited pharmacokinetic properties that compare |
Astrocytes,” was e-published in the Journal of |
is designed to evaluate the safety and efficacy of |
favorably to other oral HDAC inhibitors, including |
Molecular Neuroscience. Glucose and oxygen |
Pracinostat compared to placebo when combined |
Zolinza( vorinostat), which is approved by the FDA |
deprivation is a common factor in conditions |
with Vidaza, a drug approved by the US Food & |
for the treatment of cutaneous T-cell lymphoma. In |
involving reduced blood flow to the brain. This |
Drug Administration( FDA) for the treatment of |
addition to the evidence of clinical activity observed |
places energy stress on cells such as astrocytes, which |
MDS. |
in combination with Vidaza in patients with MDS, |
then become damaged, functionally impaired or even |
Results from an earlier pilot study of |
Pracinostat has demonstrated single-agent activity in |
die. Release of inflammatory mediators from stressed |
Pracinostat in combination with Vidaza in patients |
AML, including two CRs out of 14 patients( 14 per |
astrocytes causes further damage to neurons, with |
with intermediate-2 or high-risk MDS were presented |
cent) in a dose-escalation trial, with durable |
resulting neurological impairment. The work from |
at the American Society of Haematology( ASH) |
responses persisting up to 362 days. MEIPharma |
Shandong University suggests MANF may protect |
Annual Meeting in December 2012 showed an |
owns exclusive worldwide rights to Pracinostat. |
against such neurological damage by suppressing |
overall response rate of 89 per cent( eight out of |
|
these pro-inflammatory changes in astrocytes. The |
nine), including seven patients who achieved either a |