TIDMOXB
RNS Number : 0120U
Oxford Biomedica PLC
15 December 2011
Oxford BioMedica Announces Interim Update on ProSavin(R) Phase
I/II Study in Parkinson's Disease
-- Positive review of all patient cohorts by Data Monitoring
Committee --
-- ProSavin(R) provides long-term improvement of motor function
--
Oxford, UK - 15 December 2011: Oxford BioMedica plc ("Oxford
BioMedica" or "the Company") (LSE: OXB), the leading gene-based
biopharmaceutical company, today announces new data from its Phase
I/II study to assess the safety, efficacy and dose evaluation of
ProSavin(R) in patients with mid-stage Parkinson's disease (PD). As
part of an interim review, Oxford BioMedica and its clinical
experts have analysed the current data set for all 15 patients and
there is a pre-recorded webcast available via:
http://cache.cantos.com/webcast/static/ec2/4000/5275/9523/9863/Lobby/default.htm.
Summary of Oxford BioMedica's efficacy analysis of all 15
patients treated:
-- ProSavin(R) mediates improvement in UPDRS Part III "OFF"
scores(1) in all cohorts at six-month primary efficacy
endpoint;
-- Population analysis of cohorts 1-3 indicates that ProSavin(R)
significantly improves motor function relative to baseline, with
statistically significant improvements up to 12 months;
-- ProSavin(R) demonstrates efficacy across range of disease severity; and
-- Treatment with ProSavin(R) supports average reduction in
L-DOPA "equivalent" therapy in all cohorts with signs of a
dose-related response - cohort 4 (5x dose) indicates highest level
of dopamine provision vs. earlier cohorts
1. Motor function is assessed according to the Unified
Parkinson's Disease Rating Scale (UPDRS) in patients' "OFF" state
(i.e. after withdrawal of PD medication).
ProSavin(R) has been evaluated in four ascending dose cohorts
(1x, 2x and 5x) in a total of 15 patients. Six patients received
the 2x dose, the latter three of which were treated using an
enhanced administration procedure which facilitates higher dosing,
and six patients received the 5x dose. The primary efficacy
end-point of the Phase I/II trial is the six-month UPDRS assessment
and results from all six patients in the fourth (5x dose) cohort
are expected in H1 2012. Results from the latest assessment of all
four cohorts have now been verified and reviewed by the study's
independent Data Monitoring Committee (DMC).
DMC highlights of all four patient cohorts (n=15):
-- Favourable safety profile with no serious adverse events
related to ProSavin(R) or the enhanced administration
technique;
-- Cohort 4 (5x dose) shows most promising signs of efficacy to
date including improved UPDRS Part III "OFF" scores, improved UPDRS
Part III "ON" scores(2) despite a reduction in L-DOPA "equivalent"
therapy and a reduced Positron Emission Tomography (PET) scan
signal implying a higher level of dopamine provision;
-- Average L-DOPA "equivalent" therapy has either reduced or
remained stable in all four cohorts, in what is usually a
progressively degenerative disease requiring an increase in dose;
and
-- Long-term safety profile 36 months post-treatment (1x dose)
2. UPDRS in patients' "ON" state (i.e. with medication and when PD symptoms are not present).
Professor Olivier Rascol, Chair of the independent DMC, said:
"We are pleased that ProSavin(R) and the enhanced administration
technique continue to demonstrate a long-term safety profile. The
signals of improvements in motor function with decreased oral
dopaminergic therapy observed to date are encouraging, particularly
at the 5x dose. The functional imaging data also suggest possible
dopamine provision attributable to ProSavin(R) at this dose. We
would support investigation of a further dose escalation for this
novel product as part of a Phase II development programme."
Oxford BioMedica and its clinical experts believe that the
interim ProSavin(R) data set continues to support planning for a
sham-controlled Phase II study. During 2011, the Company has
identified and developed an enhanced product construct based on the
ProSavin(R) dopaminergic enzymes. The new construct can potentially
provide more than a 10-fold increase in dopamine production
capacity, allowing further dose escalation without impacting volume
or rate of administration. In addition, the new construct offers
extended patent protection and a relative reduction in cost of
goods, thus increasing the commercial opportunity for ProSavin(R) .
The Company looks forward to six-month results from all six
patients in the fourth (5x dose) cohort in H1 2012 and, given the
potential of the new construct, will evaluate a strategy to move it
into development as part of a Phase II programme.
John Dawson, Chief Executive Officer of Oxford BioMedica, said:
"Our pioneering Phase I/II trial is designed to assess the safety
of ProSavin(R) and provide an indication of efficacy. We are
therefore pleased to see such encouraging improvements across
several measures of clinical benefit in patients with an inexorably
degenerative disease at this stage of development.
"Based on our positive analyses to date, independent advice from
our Data Monitoring Committee and our discussions with potential
partners, we believe that this data set supports exploration of a
higher dose as part of a Phase II programme to ensure the greatest
chance of success in randomised studies. Partnering discussions
continue and we look forward to reporting further progress in
2012."
Historic analysis of the Phase I/II study and top-line motor
function has focused on individual patient cohorts (as seen in
table 1 below). When treating small numbers of patients, results
can vary considerably - particularly in a heterogeneous disease
such as Parkinson's disease. For a more comprehensive and
informative analysis of the current data set, please refer to the
pre-recorded webcast.
Table 1: Summary of independently verified improvements in motor
function to date
Cohort(3) Dose Admin. 3 months 6 months 1 year 2 years
method (UPDRS) (UPDRS) (UPDRS) (UPDRS)
---------- ----- --------- ------------ --------------- --------- ---------
1, 1x Original Mean 27% Mean 30% Mean 29% Mean 20%
n=3 Max. up to Max. up to Max. up Max. up
30% 48% to 44% to 30%
---------- ----- --------- ------------ --------------- --------- ---------
2, 2x Original Mean 28% Mean 34% Mean 29% Mean 27%
n=3 Max. up to Max. up to Max. up Max. up
53% 53% to 56% to 47%
---------- ----- --------- ------------ --------------- --------- ---------
3, 2x Enhanced Mean 26% Mean 43% Mean 28% -
n=3 Max. up to Max. up to Max. up
52% 61% to 48%
---------- ----- --------- ------------ --------------- --------- ---------
4, 5x Enhanced Mean 26% Mean 24% n=3 - -
n=6 Max. up to Max. up of
49% to 31% 6
---------- ----- --------- ------------ --------- ---- --------- ---------
3. Cohorts 3 and 4 had more severe mean baseline UPDRS scores
than previous cohorts, therefore some patients may also have to
overcome other features of PD e.g. muscle weakness, rigidity
etc.
- Ends -
For further information,
please contact:
Oxford BioMedica plc: Tel: +44 (0)1865
Lara Mott, Head of Corporate 783 000
Communications
Media Enquiries: Tel: +44 (0)20 7920
Mary Clark/Emma Thompson/Claire 2342
Dickinson
M:Communications
Notes to editors
1. Oxford BioMedica
Oxford BioMedica plc (LSE: OXB) is a biopharmaceutical company
developing innovative gene-based medicines and therapeutic vaccines
that aim to improve the lives of patients with high unmet medical
needs. The Company's technology platform includes a highly
efficient LentiVector(R) gene delivery system, which has specific
advantages for targeting diseases of the central nervous system and
the eye; and a unique tumour antigen (5T4), which is an ideal
target for anti-cancer therapy. Through in-house and collaborative
research, Oxford BioMedica has a broad pipeline with current
partners and licensees including Sanofi, Pfizer, GlaxoSmithKline,
MolMed, Sigma-Aldrich, Biogen Idec, VIRxSYS, Emergent BioSolutions
and ImaginAb. Further information is available at
www.oxfordbiomedica.co.uk.
2. LentiVector(R) gene delivery technology
Oxford BioMedica's LentiVector(R) gene delivery technology is
one of the most advanced gene delivery systems currently available,
which has many applications in product development and discovery
research. It is the system of choice for gene-based treatments
addressing chronic and inherited diseases. Oxford BioMedica has
established a dominant intellectual property estate in the field of
lentiviral-vector mediated gene delivery through its in-house
research and from work conducted by the Company's co-founders at
Oxford University.
3. Parkinson's disease
Parkinson's disease affects approximately 1.5 million patients
in the seven major markets (US, Japan, UK, France, Germany, Italy
and Spain) which is projected to rise to 1.7 million by 2019. None
of the current treatments provide long-term relief from symptoms,
yet, by 2019, sales of these treatments could exceed US$2.8 billion
in the seven major markets (source: Datamonitor, Dec-2010).
ProSavin(R) has the potential to address a major unmet medical need
in Parkinson's disease, offering long-lasting benefit from a single
administration with an excellent safety profile. The product could
therefore also significantly reduce the social care burden that is
associated with the mid to late-stage of disease.
4. ProSavin(R)
ProSavin(R) uses the Company's LentiVector(R) gene delivery
technology to deliver the genes for three enzymes - AADC (aromatic
amino acid decarboxylase), TH (tyrosine hydroxylase) and CH1
(GTP-cyclohydrolase 1) - that are required for the synthesis of
dopamine. These genes re-programme transduced cells to manufacture
and secrete dopamine. The product is administered locally to the
region of the brain called the striatum, converting cells into a
replacement dopamine factory within the brain, thus replacing the
patient's own lost source of the neurotransmitter. ProSavin(R) has
the potential to address an unmet medical need in Parkinson's
disease, offering long-lasting benefit from a single administration
with an excellent safety profile.
This information is provided by RNS
The company news service from the London Stock Exchange
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