For the first time, the PRINCE phase 3 data on pegcetacoplan in adults with PNH who are treatment naïve - meaning they had not received a complement inhibitor within three months before entering the study - will be presented.

"We look forward to presenting data from the PRINCE study for the first time, evaluating the efficacy and safety of pegcetacoplan in people living with PNH who are treatment naïve," said Ravi Rao, Head of Research & Development and Chief Medical Officer at Sobi. "We are also excited to be able to collaborate and connect in person at this year's annual meeting."

In addition to the PRINCE data, a new post-hoc analysis on pegcetacoplan in patients who were either treatment naïve or still experiencing anaemia on treatment with the C5 inhibitor eculizumab will be presented.

In haemophilia A, new data on efanesoctocog alfa, previously known as BIVV001, will be presented. Efanesoctocog alfa, an investigational once-weekly factor therapy for people with haemophilia A, has the potential to provide high sustained factor VIII activity and near-normal factor levels for the majority of the week. Efanesoctocog alfa represents a potential new class of factor replacement treatment. Currently in phase 3 development in partnership with Sanofi, the new data from a post-hoc analysis of phase 1/2 studies will add more clarity on the new mechanism of half-life extension of efanesoctocog alfa by evaluating the independency of its pharmacokinetics from von Willebrand factor.

Sobi will also present new data from the pilot study of Gamifant® (emapalumab), a fully human, anti-interferon gamma (IFNγ) monoclonal antibody, in patients with systemic juvenile idiopathic arthritis (sJIA) developing MAS. These data demonstrate the pathogenic role of IFNγ in MAS/sJIA and the therapeutic value of IFNγ neutralisation in patients with MAS who have failed standard therapy with high-dose glucocorticoids. Additionally, new exposure-safety analyses of data obtained from the pivotal trial of Gamifant in patients with primary HLH will be presented.

Key Sobi data to be presented at ASH 2021

Haemophilia
Alprolix® Prophylaxis with #498. Oral presentationSession:
(eftrenonacog alfa) rFIXFc Reduces the 322. Sunday, 12 December, Session
Frequency and Delays Time: 4:30 PM - 6:00
Time to First PM/Presentation Time: 5:45 PM
Spontaneous Bleed ET.Joint with Sanofi.
Event in Previously
Untreated Patients
with Hemophilia B: A
Post Hoc Analysis of
the PUPs B-LONG
Trial.
Efanesoctocog alfa Efanesoctocog Alfa #1035. Poster
(investigational) Half-Life and presentationSaturday, 11 December,
Clearance Are 5:30 PM - 7:30 PM. Joint with
Independent of von Sanofi.
Willebrand Factor in
Severe Hemophilia A:
A Post Hoc Analysis
from Phase 1/2a
Studies.
Elocta®/Eloctate® Retrospective #3031. Poster presentationSession:
(efmoroctocog alfa) Observational 904. Sunday, 12 December, 6:00 PM
Descriptive Study on - 8:00 PM ET.  Presented by
the Effectiveness and Sanofi.
Usage of Emicizumab
and Antihemophilic
Factor (recombinant),
Fc Fusion Protein in
Patients with
Hemophilia A in the
US.
Paroxysmal
nocturnal
haemoglobinuria
Aspaveli®/Empaveli™ Efficacy and Safety #606. Oral presentationMonday, 13
(pegcetacoplan) of Pegcetacoplan December, 11:45 AM ET, Georgia
Treatment in World Congress Center, Georgia
Complement-Inhibitor Ballroom 1-3Joint with Apellis.
Naïve Patients with
Paroxysmal Nocturnal
Hemoglobinuria:
Results from the
Phase 3 PRINCE Study.
Changes in #1112. Poster
Hemoglobin Measures presentationSaturday,
Observed in PNH 11 December, 5:30 PM
Patients Treated -7:30 PM ET.Joint
with Both C5 with Apellis.
Inhibitors
Ravulizumab and
Eculizumab: Real
-World Evidence
from a US-Based EMR
Network.
Post Hoc Analysis #2194. Poster
of the Effect of presentationSunday,
Pegcetacoplan 12 December, 6:00 PM
Treatment of -8:00 PM ET.Joint
Patients with with Apellis.
Paroxysmal
Nocturnal
Hemoglobinuria and
Baseline Hemoglobin
Levels Greater Than
10 Grams per
Deciliter.
Evaluation of the #2175. Poster
Long-Term Safety presentationSunday,
and Efficacy of 12 December, 6:00 PM
Pegcetacoplan -8:00 PM ET.Joint
Treatment for with Apellis.
Paroxysmal
Nocturnal
Hemoglobinuria
Patients: An
Extension Study. 
Categorized #1104. Poster
Hematologic presentationSaturday,
Response to 11 December, 5:30 PM
Pegcetacoplan and -7:30 PM ET.Presented
Correlations with by Apellis.
Quality of Life in
Patients with
Paroxysmal
Nocturnal
Hemoglobinuria:
Post Hoc Analysis
of Data from Phase
1b, Phase 2a, and
Phase 3 Trials.
Chronic immune
thrombocytopenia
Doptelet® Durability of #1015. Poster presentationSession:
(avatrombopag) Platelet Response 311. Saturday, 11 December, 5:30
When Switching from PM - 7:30 PM ET.
Eltrombopag or
Romiplostim to
Avatrombopag in
Immune
Thrombocytopenia
(ITP): A Multicenter
Study.
Further #2086. Poster
Characterization of presentationSession:
Thromboembolic 311. Sunday, 12
Events and December, 6:00 PM -
Association with 8:00 PM ET.
Platelet Count
Occurring during
the Avatrombopag
Immune
Thrombocytopenia
(ITP) Clinical
Development
Program.
Rationale and #4210. Online
Design of an publication in
Observational November issue of
Multicenter Study Blood.
to Evaluate the Use
and Effectiveness
of Avatrombopag in
Adult Patients with
Immune
Thrombocytopenia:
The Adopt Study. 
Rationale and #4211. Online
Design of a Phase publication in
3b Multicenter, November issue of
Randomized, Double Blood.
-Blind Placebo
-Controlled,
Parallel-Group
Trial with an Open
-Label Extension
Phase to Evaluate
the Efficacy and
Safety of
Avatrombopag for
the Treatment of
Pediatric Patients
with Immune
Thrombocytopenia.
Hemophagocytic
lymphohistiocytosis
Gamifant® Safety of Emapalumab #2061. Poster
(emapalumab) in Pediatric Patients presentation.Session: 201Sunday,
with Primary 12 December, 6:00 PM - 8:00 PM ET.
Hemophagocytic
Lymphohistiocytosis
(HLH): Relationship
to Treatment
Exposure.
Real-World #4991. Online
Treatment Patterns publication in
and Outcomes in November issue of
Patients with Blood.
Hemophagocytic
Lymphohistiocytosis
(HLH) and Other
Clinical Conditions
Treated with
Emapalumab: The
Real-HLH Study
Design
Macrophage
activation syndrome
Gamifant® Macrophage Activation #2058. Poster
(emapalumab) Syndrome (MAS) in presentation.Session:  201 Sunday,
Systemic Juvenile 12 December, 6:00 PM - 8:00 PM ET.
Idiopathic Arthritis
(sJIA): Treatment
with Emapalumab, an
Anti-Interferon Gamma
(IFNγ) Monoclonal
Antibody.
Trials in Progress: #4195. Online
A Two-Cohort, Open publication in
-Label, Single-Arm November issue of
Study of Blood.
Emapalumab, an Anti
-Interferon Gamma
(IFNγ) Monoclonal
Antibody, in
Patients with
Macrophage
Activation Syndrome
(MAS) in Rheumatic
Diseases

All abstracts can be accessed via the official ASH website. (https://www.hematology.org/meetings/annual-meeting)

About Elocta®/Eloctate®
Elocta®/Eloctate® (efmoroctocog alfa) is a recombinant clotting factor therapy developed for haemophilia A using Fc fusion technology (rFVIIIFc) to prolong circulation in the body. It is engineered by fusing factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling Elocta/Eloctate to use a naturally occurring pathway to extend the time the therapy remains in the body (half-life). Elocta/Eloctate is approved and marketed by Sobi for the treatment of haemophilia A in the EU including Russia, UK, Iceland, Norway, Liechtenstein, Switzerland, Kuwait and Saudi Arabia. It is approved and marketed as ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] by Sanofi in the United States, Japan and Canada. It is also approved in Australia, New Zealand, Brazil and other countries, where Sanofi has the marketing rights.

About Alprolix®
Alprolix® (eftrenonacog alfa) is a recombinant clotting factor therapy developed for haemophilia B using Fc fusion technology to prolong circulation in the body. It is engineered by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling Alprolix to use a naturally occurring pathway to extend the time the therapy remains in the body (half-life). Sobi and Sanofi collaborate on the development and commercialisation of Alprolix. Alprolix is approved and marketed by Sobi for the treatment of haemophilia B in the EU, Iceland, Kuwait, Liechtenstein, Norway, Saudi Arabia and Switzerland. It is also approved in the United States, Canada, Japan, Australia, New Zealand, Brazil and other countries where Sanofi has the marketing rights.

About efanesoctocog alfa (BIVV001)
Efanesoctocog alfa (rFVIIIFc-VWF-XTEN) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to potentially extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies. Efanesoctocog alfa was granted orphan drug designation by the US Food & Drug Administration in August 2017 and the European Commission in June 2019. Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been reviewed by any regulatory authority.

About Aspaveli®/Empaveli™
Aspaveli®/Empaveli™ (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Empaveli is approved in the United States for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for Aspaveli for the treatment of adults with PNH who are anaemic after treatment with a C5 inhibitor for at least three months. The positive opinion from the CHMP is now referred to the European Commission for an approval decision. The therapy is also under investigation for several other rare diseases across haematology, nephrology, and neurology.

About the PRINCE study
The PRINCE study (NCT04085601) is a 2:1 (pegcetacoplan: standard of care) randomised, multi-centre, open-label, controlled phase 3 study in 53 treatment-naïve adults with paroxysmal nocturnal haemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan in patients who have not received treatment with any complement inhibitor within three months prior to screening. During the 26-week randomised, controlled period, patients received either 1080 mg of pegcetacoplan twice weekly or standard of care therapy, which did not include complement inhibitors. Patients in the standard of care group had the option to escape to the pegcetacoplan group if their haemoglobin decreased 2 g/dL or more from their baseline value.

About the Sobi and Apellis Collaboration
Sobi and Apellis collaborate to develop and commercialize systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan. The companies have global co-development rights for systemic pegcetacoplan.

About Doptelet®
Doptelet® is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics the biologic effects of TPO in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count. It is approved by the European Medicines Agency and the US Food & Drug Administration for the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure, and for the treatment of thrombocytopenia in adult patients with primary chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Chronic ITP is a rare autoimmune bleeding disorder characterised by low number of platelets. The incidence of primary ITP in adults is 3.3/100 000 adults per year with a prevalence of 9.5 per 100 000 adults[1].

About Gamifant®
Gamifant® (emapalumab) is a monoclonal antibody that binds to and neutralises interferon gamma (IFNγ). In the US, emapalumab is indicated for the treatment of adult and paediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is 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 for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). In September 2020, emapalumab received Orphan Drug Designation (ODD) by the FDA for prevention of graft failure following haematopoietic stem cell transplantation.

About Sobi
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, the Middle East, Russia and North Africa. In 2020, Sobi's revenue amounted to SEK 15.3 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at www.sobi.com.

Contacts
For details on how to contact the Sobi Investor Relations Team, please click here (https://www.sobi.com/en/investors). For Sobi Media contacts, click here (https://www.sobi.com/en/media). 

References

1. Lambert et al. Blood 2017.

XTEN® is a registered trademark of Amu