STOCKHOLM, June 18, 2019 /PRNewswire/ -- Swedish Orphan
Biovitrum AB (publ) (Sobi™) (STO:SOBI) announces that new research
demonstrating the effects of emapalumab in patients with macrophage
activation syndrome (MAS), a form of secondary haemophagocytic
lymphohistiocytosis (HLH) complicating systemic juvenile idiopathic
arthritis (sJIA), was presented at the European League Against
Rheumatism (EULAR)/ Paediatric Rheumatology European Society (PReS)
Scientific Congress in Madrid.
The study showed that treatment with emapalumab led to rapid
neutralisation of interferon gamma (IFNγ) and a complete response
in all patients who participated in the study. Furthermore,
emapalumab demonstrated a favourable safety profile[1]. Emapalumab
has previously been shown to induce rapid and sustained responses
in patients with primary HLH.
Emapalumab is FDA-approved for paediatric (newborn and older)
and adult patients with primary HLH with refractory, recurrent or
progressive disease or intolerance to conventional HLH therapy.
The data was also recognised with the Gold PReS KOURIR Award
during the conference.
MAS is a rare, life-threatening condition characterised by
uncontrolled hyperinflammation which may develop on a background of
rheumatic diseases such as sJIA. It is classified as a secondary
form of HLH and is caused by excessive activation and expansion of
T cells and macrophages. A vast body of evidence has been
accumulated which points to uncontrolled overproduction of IFNγ as
a major driver of hyperinflammation and hypercytokinaemia in
diseases such as MAS and HLH.
"It is extremely encouraging to see a complete response in the
first six patients treated with emapalumab, particularly
considering that MAS is a serious and potentially fatal
complication, and that all of these patients had failed other
treatments," said Dr. Fabrizio De
Benedetti, Head of the Division of Paediatric Rheumatology
and Head of the Laboratory of ImmunoRheumatology, Ospedale
Pediatrico Bambino Gesù, Rome,
Italy.
The new data represents the results of an on-going international
pilot study, in which six MAS patients with a background of sJIA
and inadequate response to high-dose intravenous glucocorticoids
received emapalumab according to the prescribed dose regimen
required per protocol.
Emapalumab is a monoclonal antibody that neutralises interferon
gamma (IFNγ), a key cytokine which contributes to the inflammation
and tissue damage seen in MAS. The purpose of the study is to
assess the drug's pharmacokinetics, efficacy and safety for
treatment of MAS and to confirm the proposed dose regimen.
The data was presented in a session entitled "Adults are just
grown up children! Discuss" on Thursday, 13 June, by Dr. De
Benedetti. Details of the abstract can be accessed at the EULAR
website:
http://scientific.sparx-ip.net/archiveeular/?c=a&view=3&item=2019OP0204
About macrophage activation syndrome (MAS)
Macrophage activation syndrome (MAS) is a severe complication of
rheumatic diseases, most frequently systemic juvenile idiopathic
arthritis (sJIA) – a rare systemic disorder of auto-inflammatory
nature with common clinical manifestations such as daily spiking
fever, typical transient cutaneous rash, arthritis,
lymphadenopathy, hepatosplenomegaly and serositis. MAS is
characterised by fever, hepatosplenomegaly, liver dysfunction,
cytopenias, coagulation abnormalities and hyperferritinaemia,
possibly progressing to multiple organ failure and death. MAS is
classified as a secondary form of haemophagocytic
lymphohistiocytosis (HLH).
About emapalumab
Emapalumab is a monoclonal antibody (mAb) that binds to and
neutralises interferon gamma (IFNγ). In the US, emapalumab is
indicated for paediatric (newborn and older) and adult primary
haemophagocytic lymphohistiocytosis (HLH) patients with refractory,
recurrent or progressive disease, or intolerance to
standard-of-care HLH therapy, and is marketed under the name
Gamifant. Emapalumab is the first and only medicine approved in the
US for primary HLH, a rare syndrome of hyperinflammation that
usually occurs within the first year of life and can rapidly become
fatal unless diagnosed and treated. The FDA approval is based on
data from the phase 2/3 studies (NCT01818492 and NCT02069899).
Emapalumab is indicated to be administered through intravenous (IV)
infusion over one hour twice per week until haematopoietic stem
cell transplant (HSCT). Visit www.gamifant.com for more
information, including full US prescribing Information.
Emapalumab was developed and submitted for approval to the FDA
by Novimmune, a privately held Swiss biopharmaceutical company
focused on discovering and developing antibody-based drugs targeted
for the treatment of inflammatory diseases, immune-related
disorders and cancer. Sobi acquired the global rights to emapalumab
from Novimmune through an exclusive licensing agreement announced
in July 2018, which is now superseded
by the acquisition of emapalumab and related assets announced on
12 June 2019, subject to customary
closing conditions.
About Sobi™
At Sobi, we are transforming the lives of people affected by
rare diseases. As a specialised international biopharmaceutical
company, we provide sustainable access to innovative therapies in
the areas of haematology, immunology and specialty care. We bring
something rare to rare diseases – a belief in the strength of
focus, the power of agility and the potential of the people we are
dedicated to serving. The hard work and dedication of our
approximately 1050 employees around the globe has been instrumental
in our success across Europe,
North America, the Middle East, Russia and North
Africa, leading to total revenues of SEK 9.1 billion in 2018. Sobi's share (STO:SOBI)
is listed on Nasdaq Stockholm. You can find more information about
Sobi at www.sobi.com.
For more information please contact
Sobi
Paula Treutiger
Head of Communication & Investor
Relations
+46-733-666-599
paula.treutiger@sobi.com
Linda Holmström
Corporate Communication & Investor
Relations
+46-708-734-095
linda.holmstrom@sobi.com
[1] Emapalumab, an interferon gamma (IFN-Y)-blocking
monoclonal antibody, in patients with macrophage activation
syndrome (MAS) complicating systemic juvenile idiopathic arthritis
(sJIA)
Fabrizio De Benedetti,
Paul Brogan, Alexei Grom, Pierre Quartier Dit Maire, Rayfel
Schneider, Kathy De Graaf,
Philippe Jacqmin, Maria Ballabio, Cristina
de Min
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SOURCE Swedish Orphan Biovitrum AB