WOUDSCHOTEN, The Netherlands, Sept. 24 /PRNewswire/ --
Oxford GlycoSciences Plc (Nasdaq: OGSI; LSE: OGS.) announced that Professor
Ari Zimran, Director of the Gaucher Clinic at the Shaare Zedek Medical Center,
Jerusalem presented results from clinical studies on OGT 918 in type 1 Gaucher
disease at the 13th Workshop of the European Study Group on Lysosomal Diseases
in Woudschoten on 22 September 2001. Professor Zimran presented long term
data from 18 patients who continued into an extension protocol at the end of
the first 12 month clinical trial of OGT 918 in type 1 Gaucher disease.
For the extension cohort, (study 001 ext)(1) the data at 24 months show a
progressive improvement on the results previously described after 12 and 18
months of treatment. Mean liver and spleen organ volumes were significantly
reduced from baseline by 14.5% and 26.4% respectively. Mean haemoglobin
concentration and platelet count were significantly increased from baseline by
0.91g/dl and 13.6 x 10(9) [10 to the 9th power]/1 respectively. The
prevalence of adverse effects on OGT 918 continued to decline and 14 patients
reached the 24 month timepoint and have continued into their third year of
therapy.
Results were also presented from 18 patients enrolled in a six month study
of OGT 918 monotherapy conducted in patients with type 1 Gaucher disease in
Johannesburg and Jerusalem, (study 003)(2). This study examined a lower dose
of OGT 918 of 50mg taken three times daily (TID), and a similar study design
was employed to allow comparison with the original 12 month study, which was
based on a dose of 100mg TID. After six months, there were significant
reductions in mean liver and spleen volume of 5.9% and 4.5%, respectively.
Chitotriosidase activity (a biochemical marker related to the disease) was
significantly reduced by 4.6%. For all these parameters, on average, patients
in the 100mg TID dose group had responded more favourably than those in the
50mg TID dose group. Changes in haemoglobin concentration and platelet count
were small and not significant. As with the original study, the most common
adverse events were initial gastrointestinal effects and one patient withdrew
because of these. Seventeen patients completed the study and 16 entered an
optional extended use study in which patients were titrated to an increased
dose. This comparison addresses a dose of 100mg TID as a starting dose for
OGT 918 in type 1 Gaucher disease.
It is anticipated that full data from these studies will be submitted for
publication in peer-reviewed journals in the coming months.
References
1. D. Elstein, C. Hollak, S. van Weely, M. Maas, J. Aerts, T. Cox, R.
Lachmann, M. Hrebicek, A. Zimran. Efficacy and tolerability of the oral
glucosyltransferase inhibitor N-butyl-deoxynojirimycin (OGT 918) in adult
patients with type 1 Gaucher disease. Abstract 39:- European Study Group
on Lysosomal Diseases (ESGLD) meeting 20-23 September 2001, Woudschoten,
The Netherlands.
2. R. Heitner, D. Elstein, A. Zimran. A dose comparison of the efficacy
and safety of oral N-butyl-deoxynojirimycin (OGT 918) in two clinical
studies in type 1 Gaucher disease. Abstract 48:- European Study Group on
Lysosomal Diseases (ESGLD) meeting 20-23 September 2001, Woudschoten, The
Netherlands.
Notes to Editors
OGS has developed a proprietary technology platform in the emerging field
of proteomics, the comprehensive study of proteins, integrating proteomics
with genomics to create an innovative drug discovery platform. OGS'
proteomics collaborations with major pharmaceutical and biotechnology
companies include Bayer, Pioneer Hi-Bred/DuPont, Medarex, GlaxoSmithKline, and
Pfizer. OGS has technology development collaborations with Applera, Cambridge
Antibody Technology, Packard BioScience and the Institute for Systems Biology.
OGS has also entered into an IT/database joint venture, Confirmant
Limited, with Marconi plc. and a high throughput "targets to leads"
collaboration with NeoGenesis, of Cambridge, Massachusetts.
OGS has drug research discovery programmes in cancer, infectious disease
and glycosphingolipid ('GSL') storage disorders.
Gaucher disease
Gaucher disease is a GSL storage disorder in which individuals have a
specific genetic abnormality (enzyme defect) in their metabolism that leads to
accumulation of un-metabolised material in various tissues, within structures
known as lysosomes. This disease is sometimes referred to as a lysosomal
storage disease. Gaucher disease causes a range of debilitating symptoms and
in some cases premature death.
Treating GSL storage diseases with OGT 918
OGT 918 has a novel mode of action as an oral inhibitor of
glucosylceramide synthase, a key enzyme involved in GSL biosynthesis. The
rationale for the use of OGT 918 is to help balance the overall level of GSLs
by inhibiting the production or synthesis of GSLs. A wide variety of
preclinical studies have demonstrated that OGT 918 provides an effective
control over the rate of GSL synthesis.
OGT 918: Orphan Drug Designation granted in the US and EU
In the US, OGS has been granted Orphan Drug Designation for OGT 918 in
both Gaucher and Fabry diseases. The 1983 Orphan Drug Act guarantees the
developer of an Orphan Drug product seven years of market exclusivity in the
US following the approval of the product by the US Food and Drug
Administration ('FDA').
In the EU, OGT 918 has been designated as an Orphan Medicinal Product for
the treatment of Gaucher disease by the European Commission. Designation of
OGT 918 as an Orphan product in the EU allows direct access into the European
Agency for Evaluation of Medicinal Products ('EMEA') Centralised Procedure for
Marketing Authorisation Application and may provide for a partial or total
application fees waiver. Also, the designation allows for a 10 year marketing
exclusivity period, in the EU, following regulatory approval.
OGT 918: Fast Track Designation received in the US
Fast Track designation is granted by the FDA to potential new drugs in
order to facilitate their development and expedite subsequent regulatory
review of compounds that are intended to treat serious or life threatening
conditions and that demonstrate the potential to address unmet medical needs.
OGT 918: Regulatory Status
In July 2001, OGS announced the acceptance of its Marketing Authorisation
Application ('MAA') for OGT 918 for the oral treatment of type 1 Gaucher
disease by the EMEA and, in August 2001, it announced the completion of the
step-wise (rolling) submission of its New Drug Application ('NDA') for OGT 918
to the FDA.
This release contains forward-looking statements, such as OGT 918
providing effective control over the rate of GSL synthesis and the commercial
potential and success of OGS' collaborations and drug candidates. Factors
that could cause actual results to vary significantly from those expressed or
implied by these and other forward-looking statements include the success of
other trials and research involving OGT 918, acceptance by the scientific and
medical communities of OGS' conclusions as to the results of trials, the
validity of the technologies and medical conclusions on which OGT 918 is
based, uncertainties related to the regulatory process and the success of OGS'
research and development strategies. OGT 918 is an investigational drug and
has not received approval for marketing in any country.
For further information please contact:
Oxford GlycoSciences Plc
Michael Kranda, Chief Executive Officer
Dr Chris Moyses, Clinical and Development Director
Dr Stephen Parker, Chief Financial Officer
Tel: +44 (0) 1235 208000
WorldWide Web: http://www.ogs.com
UK: Financial Dynamics
David Yates/Melanie Toyne-Sewell
Tel: +44 (0) 20 7831 3113
USA: Feinstein Kean
Michelle Linn/Michael Lawson
Tel: +1 (617) 761 6765
+1 (617) 577 8110
SOURCE Oxford GlycoSciences Plc
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Related links:
http://www.ogs.com
CONTACT:
Michael Kranda, Chief Executive Officer, or
Dr Chris Moyses, Clinical and Development Director, or Dr Stephen
Parker, Chief Financial Officer, +44 (0) 1235 208000, all of
Oxford GlycoSciences Plc; or David Yates, or Melanie Toyne-Sewell
of Financial Dynamics, +44 (0) 20 7831 3113; or Michelle Linn,
+1-617-761-6765, or Michael Lawson, +1-617-577 8110, both of
Feinstein Kean
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