Sobi to present new data at the ASH 2021 Annual Meeting – PRNewswire

STOCKHOLM, Nov. 4, 2021 /PRNewswire/ -- Sobi will present new data at the 63rd Annual Meeting of the American Society of Hematology (ASH) taking place from 11-14 December 2021. Sobi's commitment to developing innovative treatments for people living with rare diseases is highlighted in studies spanning several rare disorders, including haemophilia, paroxysmal nocturnal haemoglobinuria (PNH), chronic immune thrombocytopenia (ITP), macrophage activation syndrome (MAS) and haemophagocytic lymphohistiocytosis (HLH).

For the first time, the PRINCE phase 3 data on pegcetacoplan in adults with PNH who are treatment nave 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 nave," 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 nave 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 (IFN) monoclonal antibody, in patients with systemic juvenile idiopathic arthritis (sJIA) developing MAS. These data demonstrate the pathogenic role of IFN in MAS/sJIA and the therapeutic value of IFN 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 (eftrenonacog alfa)

Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial.

#498. Oral presentationSession: 322. Sunday, 12 December, Session Time: 4:30 PM - 6:00 PM/Presentation Time: 5:45 PM ET.Joint with Sanofi.

Efanesoctocog alfa (investigational)

Efanesoctocog Alfa Half-Life and Clearance Are Independent of von Willebrand Factor in Severe Hemophilia A: A Post Hoc Analysis from Phase 1/2a Studies.

#1035. Poster presentationSaturday, 11 December, 5:30 PM - 7:30 PM. Joint with Sanofi.

Elocta/Eloctate (efmoroctocog alfa)

Retrospective Observational Descriptive Study on the Effectiveness and Usage of Emicizumab and Antihemophilic Factor (recombinant), Fc Fusion Protein in Patients with Hemophilia A in the US.

#3031. Poster presentationSession: 904. Sunday, 12 December, 6:00 PM - 8:00 PM ET. Presented by Sanofi.

Paroxysmal nocturnal haemoglobinuria

Aspaveli/Empaveli (pegcetacoplan)

Efficacy and Safety of Pegcetacoplan Treatment in Complement-Inhibitor Nave Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from the Phase 3 PRINCE Study.

#606. Oral presentationMonday, 13 December, 11:45 AM ET, Georgia World Congress Center, Georgia Ballroom 1-3Joint with Apellis.

Changes in Hemoglobin Measures Observed in PNH Patients Treated with Both C5 Inhibitors Ravulizumab and Eculizumab: Real-World Evidence from a US-Based EMR Network.

#1112. Poster presentationSaturday, 11 December, 5:30 PM-7:30 PM ET.Joint with Apellis.

Post Hoc Analysis of the Effect of Pegcetacoplan Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Baseline Hemoglobin Levels Greater Than 10 Grams per Deciliter.

#2194. Poster presentationSunday, 12 December, 6:00 PM-8:00 PM ET.Joint with Apellis.

Evaluation of the Long-Term Safety and Efficacy of Pegcetacoplan Treatment for Paroxysmal Nocturnal Hemoglobinuria Patients: An Extension Study.

#2175. Poster presentationSunday, 12 December, 6:00 PM-8:00 PM ET.Joint with Apellis.

Categorized Hematologic Response to Pegcetacoplan and Correlations with Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from Phase 1b, Phase 2a, and Phase 3 Trials.

#1104. Poster presentationSaturday, 11 December, 5:30 PM-7:30 PM ET.Presented by Apellis.

Chronic immune thrombocytopenia

Doptelet (avatrombopag)

Durability of Platelet Response When Switching from Eltrombopag or Romiplostim to Avatrombopag in Immune Thrombocytopenia (ITP): A Multicenter Study.

#1015. Poster presentationSession: 311. Saturday, 11 December, 5:30 PM - 7:30 PM ET.

Further Characterization of Thromboembolic Events and Association with Platelet Count Occurring during the Avatrombopag Immune Thrombocytopenia (ITP) Clinical Development Program.

#2086. Poster presentationSession: 311. Sunday, 12 December, 6:00 PM - 8:00 PM ET.

Rationale and Design of an Observational Multicenter Study to Evaluate the Use and Effectiveness of Avatrombopag in Adult Patients with Immune Thrombocytopenia: The Adopt Study.

#4210.Online publication in November issue of Blood.

Rationale and Design of a Phase 3b Multicenter, Randomized, Double-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.

#4211.Online publication in November issue of Blood.

Hemophagocytic lymphohistiocytosis

Gamifant (emapalumab)

Safety of Emapalumab in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis (HLH): Relationship to Treatment Exposure.

#2061. Poster presentation.Session: 201Sunday, 12 December, 6:00 PM - 8:00 PM ET.

Real-World Treatment Patterns and Outcomes in Patients with Hemophagocytic Lymphohistiocytosis (HLH) and Other Clinical Conditions Treated with Emapalumab: The Real-HLH Study Design

#4991. Online publication in November issue of Blood.

Macrophage activation syndrome

Gamifant (emapalumab)

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Sobi to present new data at the ASH 2021 Annual Meeting - PRNewswire