Publication of Clinical Data Demonstrating Low Immunogenicity and Excellent Efficacy of Nuwiq® in Previously Untreated Patients

LACHEN, Switzerland–(BUSINESS WIRE)–Octapharma is delighted to announce the publication of interim data from
the NuProtect study (GENA-05, NCT01712438), the first clinical
data on treatment with Nuwiq® in previously untreated
patients (PUPs), on August 16th 2017 as an Early View
article in the internationally renowned medical journal Haemophilia.

The NuProtect study was initiated in March 2013 to investigate the
immunogenicity, efficacy and safety of Nuwiq® in PUPs with
severe haemophilia A, who are at the greatest risk of developing
inhibitors. The ongoing study has enrolled 110 PUPs of any age or
ethnicity who will receive Nuwiq® for up to 100 exposure days
(EDs), making it one of the largest studies with a single FVIII product.
Patients who previously received any FVIII concentrates or blood
products containing FVIII are excluded from the study.

The recently published article describes interim results for 66 PUPs
treated for at least 20 EDs, the time by which most inhibitors arise.
The cumulative incidence (95% confidence interval) was 20.8% (10.7–31.0)
for all inhibitors and 12.8% (4.5–21.2) for high-titre inhibitors. The
study reported excellent efficacy of Nuwiq® in the prevention
of bleeds, with median annual bleeding rates of 0 for spontaneous bleeds
and 2.40 for all bleeds. Nuwiq® was also efficacious for the
treatment of bleeds (92.4% of bleeds were controlled with one or two
infusions) and as surgical prophylaxis (rated as “excellent” or “good”
for 89% of surgical procedures). These interim results confirm the
excellent haemostatic efficacy reported with Nuwiq® in
previously treated patients.

Larisa Belyanskaya, Head of IBU Haematology, said: “We are very
excited by the results of the NuProtect study and pleased that these
interim data are now available as a publication in the specialist
peer-reviewed journal Haemophilia. This is the first study to report
data from PUPs treated with a rFVIII produced in a human cell line, and
the results further differentiate Nuwiq
® from
other products on the market.”

Olaf Walter, Board Member at Octapharma, added that “These data
demonstrate low immunogenicity of Nuwiq
® in
PUPs, the patient group at the greatest risk of inhibitor development,
and confirm the excellent efficacy observed in previously treated
patients. This publication is a further important step towards
Octapharma’s goal of enabling patients with coagulation disorders to
live a normal life.”

Octapharma would like to thank everyone involved in the study, in
particular the patients and their families, without whom this research
would not be possible.

About the NuProtect (GENA-05) study

The NuProtect clinical study (NCT01712438) is a phase 3, open-label,
interventional clinical study being conducted across 38 centres, to
evaluate at least 100 previously untreated patients (PUPs) with severe
haemophilia A of all ages and ethnicities enrolled for study up to 100
exposure days (EDs) or 5 years maximum. Patients treated previously with
any FVIII concentrates/blood products containing FVIII are excluded. The
primary objective is to assess the immunogenicity of Nuwiq®
by determining inhibitor activity using the Nijmegen-modified Bethesda
assay at a central laboratory. Final data from the NuProtect study are
expected to become available in 2019. More information on this trial is
available at www.clinicaltrials.gov.
An extension study (GENA-15, NCT01992549) will further evaluate the
long-term immunogenicity, efficacy and safety of Nuwiq® in
PUPs.

About Nuwiq®

Nuwiq® is a 4th generation rFVIII protein1,
produced in a human cell line without chemical modification or fusion
with any other protein2. Nuwiq® is cultured
without additives of human or animal origin2, is devoid of
antigenic non-human protein epitopes3 and has a mean
half-life of 17.1 hours4,5 and a high affinity for the von
Willebrand coagulation factor6. Nuwiq® treatment
has been assessed in seven completed clinical trials which included 201
PTPs7,8 (190 individuals) with severe haemophilia A,
including 59 children9. Nuwiq® is approved for use
in the treatment and prophylaxis of bleeding across all age groups of
PTPs with haemophilia A in the EU, US, Canada, Australia, Latin America
and Russia. Further worldwide submissions for Nuwiq® are
planned.

References:
1. FDA Nuwiq® memorandum (STN 125555), 9
October 2014.
2. Casademunt E, et al. Eur J Haematol 2012;
89: 165-76.
3. Kannicht C, et al. Thromb Res. 2013;131:78-88.
4.
Nuwiq® European Public Assessment Report, 22 May 2014.
5.
NUWIQ® US Prescribing Information, September 2015.
6.
Sandberg H, et al. Thromb Res 2012; 130: 808-17.
7. Valentino LA,
et al. Haemophilia 2014; 20(Suppl. 1): 1-9
8. Lissitchkov T, et al.
Haemophilia 2017; e-pub ahead of print; doi: 10.1111/hae.13251
9.
Klukowska A, et al. Haemophilia 2016; 22, 232-39.

About Haemophilia A

Haemophilia A is an X-linked hereditary disorder caused by FVIII
deficiency which, if left untreated, leads to haemorrhages in muscles
and joints and consequently to arthropathy and severe morbidity. FVIII
replacement prophylactic treatment reduces the number of bleeding
episodes and the risk of permanent joint damage. This disorder affects
one in every 5,000 to 10,000 men worldwide. Globally, 75% of haemophilia
cases are left undiagnosed or untreated. The development of neutralising
FVIII antibodies (FVIII inhibitors) against infused FVIII represents the
most serious treatment complication. The cumulative risk of FVIII
inhibitor development is reported to be currently up to 39%.

About Octapharma

Headquartered in Lachen, Switzerland, Octapharma is one of the largest
human protein manufacturers in the world, developing and producing human
proteins from human plasma and human cell lines. As a family-owned
company, Octapharma believes in investing to make a difference in
people’s lives and has been doing so since 1983; because it’s in our
blood.

In 2016, the Group achieved €1.6 billion in revenue, an operating income
of €383 million and invested €249 million to ensure future prosperity.
Octapharma employs more than 7,100 people worldwide to support the
treatment of patients in 113 countries with products across three
therapeutic areas:

  • Haematology (coagulation disorders)
  • Immunotherapy (immune disorders)
  • Critical care

Octapharma owns six state-of-the-art production facilities in Austria,
France, Germany, Mexico and Sweden.

For more information visit www.octapharma.com

Contacts

Octapharma AG
International Business Unit – Haematology
Olaf
Walter
Olaf.Walter@octapharma.com
or
Larisa
Belyanskaya
Larisa.Belyanskaya@octapharma.com
Tel:
+41 55 4512121