16 February 2015

R &D FO C US dr ugne w s

in urinary GAG excretion of 60% at a 4 mg/kg dose of UX

003. An improvement in pulmonary function was observed
in one patient; the other two patients were unable to be
evaluated for this measurement. Improvements were also
observed in the MPS Health Assessment Questionnaire,
measuring functional capabilities, and on the Physician
Global Impression of Change scale, assessing overall
health status. The reduction in liver size in two patients
with enlarged livers at baseline, initially reported at
week 12, was sustained. No serious adverse events or
infusion-associated reactions were reported through week
36. The most common adverse events included respiratory
disorders, infections and arthralgia, and were related to the
iv administration route or consistent with the symptoms of
MPS 7. A phase III trial of UX 003 is ongoing, for which
Ultragenyx expects to release data in first half 2016.

and it has also efficacy in the intra-arterial, intratumoral,

sc or ip delivery of therapeutic cells. The technology can be
applied to deliver a range of cell types in different disease
areas, including diabetes, Parkinson's disease, hemophilia,
hypercholesterolemia, peripheral artery disease, and
metabolic disease (in biomolecule augmentation over
extended time periods), and in immunotherapy. Cell-in-aBox technology is available for partnering.
For further information on the opportunities available,
contact:
Rahul Pawa
Regional Implementation Manager
Austrianova Co. Ltd
Thailand Science Park
Paholyothin road

Conferences

Klong 1

BioAsia, 2-4 February

2015, Hyderabad, India

Pathumthani 12120

Klong Luang
Thailand
Tel: +66 874019128
Email: pawa@austrianova.com

Opportunity with Austrianova

Cell-in-a-Box
Austrianova partnering opportunity,
Worldwide
At BioAsia, 2-4 February 2015, Hyderabad, India, Rahul
Pawa, Regional Implementation Manager at Austrianova
informed R&D Focus that the company has developed
Cell-in-a-Box, an encapsulation technology for delivery
of therapeutic cells. The technology comprises an
artificial, semi-permeable capsule, made from the natural
biopolymer cellulose, that is permeable to nutrients and
oxygen and allows the release of cellular products into
the bloodstream or adjacent tissues. The capsular material
excludes immune cells and antibodies that would cause

Updates from Aurigene

AUNP 12
Aurigene provides update on IND filing
At BioAsia, 2-4 February 2015, Hyderabad, India, R&D
Focus was informed by CSN Murthy, CEO of Aurigene, that
the company expects to file an IND for the initiation of
a phase I trial of AUNP 12 in India for the treatment of
cancer in March 2015.
AUNP 12, a peptide immune checkpoint regulator that
antagonizes the programmed cell death-1 (PD-1) signaling
pathway, is being developed for the treatment of multiple
types of cancer. AUNP 12 is a branched 29-amino acid

rejection and destroy the implant of therapeutic cells,

thus eliminating the need for immunosuppressive drugs.
The encapsulation technology has demonstrated safety and
efficacy in clinical trials for pancreatic and breast cancer

peptide sequence engineered from the PD-L1/L2 binding

domain of PD-1; the peptide blocks the PD-1/PD-L1, PD-1/
PD-L2 and PD-L1/CD80 pathways. Preclinical evaluation is
ongoing.

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