Last update 21 Jun 2024

Eftrenonacog alfa

Last update 21 Jun 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Fusion protein

Synonyms

Coagulation factor IX recombinant immunoglubulin g1 fusion protein, Coagulation factor IX recombinant immunoglubulin G1 fusion protein;, Coagulation Factor IX(Long-Acting, Recombinant Fc Fusion Protein)

+ [17]

Target

factor IX

Mechanism

factor IX stimulants(Coagulation factor IX stimulants), Blood coagulation pathway activation, Coagulation factor replacements

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Hemophilia BHemorrhage

Inactive Indication-

Originator Organization

Sobi Analytics Ltd.

Active Organization

Bioverativ, Inc.Sanofi-Aventis Canada, Inc.

Swedish Orphan Biovitrum AB

+ [3]

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

US (28 Mar 2014),

Hemorrhage

RegulationOrphan Drug (US), Overseas New Drugs Urgently Needed in Clinical Settings (CN)

Gene Sequence

Sequence Code 169138

Source: *****

Sequence Code 616723820

Source: *****

Clinical Trials associated with Eftrenonacog alfa

NCT05856266 / TerminatedPhase 4

An 18-month Low-interventional Prospective, Multicentre Study to Assess Joint Outcomes in Patients With Haemophilia A or B on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa

The goal of this low-interventional study is to describe the overall joint health in patients with haemophilia A or haemophilia B prophylactically treated with rFVIIIFc or rFIXFc.
The main question it aims to answer is the:
• Evaluation of the overall joint status as detected by ultrasound in haemophilia A and B patients treated with rFVIIIFc or rFIXFc prophylaxis over the 18-month study period.
Participants will come to 6-monthly visits during the 18-month long study period and will perform an ultrasound with the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) protocol at each visit. At baseline and end of study visits, the patients will be assessed with the clinical scoring system Haemophilia Joint Health Score (HJHS) and complete patient questionnaires. Retrospective data from patient medical records will also be collected for at least 6 months before enrolment in the study.

Start Date24 Aug 2023

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

NCT05662319 / RecruitingPhase 3

A Phase 3, Single-arm, Multicenter, Multinational, Open-label, One-way Crossover Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged ≥ 12 Years With Severe Hemophilia A or B With or Without Inhibitory Antibodies to Factor VIII or IX

This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia.
The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in male adult and adolescent (≥12 years old) participants with hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX who have switched from their prior standard of care treatment.
The total study duration will be up to approximately 50 months (200 weeks, 1 study month is equivalent to 4 weeks) and will include:
A screening period up to approximately 60 days,
A standard of care (SOC) period of approximately 6 study months (24 weeks),
A fitusiran treatment period of approximately 36 study months (144 weeks),
An antithrombin (AT) follow-up period of approximately 6 study months (24 weeks) but may be shorter or longer depending on individual participants AT recovery.
The frequency for telephone visits will be approximately every 2 weeks. For site visits the frequency will be approximately every 8 weeks during the SOC period and approximately every 4 weeks during the fitusiran treatment period. If applicable and if allowed by local regulation, home and/or remote visits may be conducted during the study

Start Date01 Feb 2023

Sponsor / Collaborator

Sanofi

NCT06158334 / RecruitingNot Applicable

Study of Surgical Practices in Patient With Haemophilia A or B Treated With an Extended Half-life Recombinant Factor VIII-Fc or IX-Fc (ELOCTA®, ALPROLIX®)

Haemophilia A and haemophilia B are inherited bleeding disorders resulting from the absence or deficiency of coagulation factors VIII and IX, respectively. The peri-operative period of people with haemophilia is commonly managed with replacement therapy.
In phase 3 studies of Elocta® (extended half-life recombinant factor VIII-Fc) and Alprolix® (extended half-life recombinant factor IX-Fc), haemostatic efficacy was demonstrated to be good or excellent, close to the haemostatic efficacy usually seen in people without haemophilia, with maintenance and stability of circulating FVIII and FIX levels compatible with the surgical procedure, while reducing the frequency of infusions and consumption of therapeutic units. In 2019, the National Protocol for Diagnosis and Care in haemophilia recommended 2 methods for managing patients with haemophilia in the peri-operative period, either discontinuous injections of standard or extended half-life factor VIII/IX or a continuous infusion of FVIII/IX.
Many countries, including France, have adopted these rFVIII/IXFc therapeuitic products and recommended their use in the surgical management of patients. However, the use of these two products in real life during surgery in haemophilic A and B patients has not been described in detail. It seems therefore relevant to better document their use in order to progressively specify their use during surgeries with varied bleeding risks.

Start Date01 Jun 2022

Sponsor / Collaborator

Hospices Civils de Lyon

100 Clinical Results associated with Eftrenonacog alfa

100 Translational Medicine associated with Eftrenonacog alfa

100 Patents (Medical) associated with Eftrenonacog alfa

135

Literatures (Medical) associated with Eftrenonacog alfa

01 Mar 2024·Biosensors & bioelectronics

A ferrocene-based hydrogel as flexible electrochemical biosensor for oxidative stress detection and antioxidation treatment

Article

Author: Zhang, Haiqi ; Ma, Lie ; Li, Yan ; Hu, Hongtao ; Wang, Jinze

Real-time sensing of reactive oxygen species (ROS) and timely scavenging of excessive ROS in physiological environments are critically important in the diagnosis and prevention of ROS-related diseases. To solve the mismatch problem between conventional rigid ROS biosensors and biological tissues in terms of both modulus and composition, here, we present a flexible ferrocene-based hydrogel biosensor designed for oxidative stress detection and antioxidation treatment. The hydrogel was fabricated through a supramolecular assembly of ferrocene-grafted polyethylenimine (PEI-Fc), sodium alginate (SA), and polyvinyl alcohol (PVA). Multiple non-covalent interactions, including electrostatic interactions between PEI-Fc and SA, hydrophobic interactions and π-π stacking among ferrocene groups, and the PVA crystalline domain, synergistically improve the mechanical properties of the PVA/SA/PEI-Fc hydrogel. The flexible PVA/SA/PEI-Fc hydrogel biosensor exhibited a broad detection range for hydrogen peroxide (H₂O₂), from 0 to 120 μM, using the differential pulse voltammetry method. Furthermore, the hydrogel demonstrated effective ROS scavenging and oxygen generation performance, desirable biocompatibility, and satisfactory antibacterial activity, making it suitable for biological interfaces. In vitro studies revealed that the PVA/SA/PEI-Fc hydrogel could monitor H₂O₂ concentration in the proximity of inflammatory cells, and effectively scavenge ROS to protect cells from oxidative stress damage. This all-in-one multifunctional hydrogel, integrating both sensing and treatment functions, holds great promise for clinical applications in the diagnosis and management of ROS-related diseases.

01 Jan 2024·British journal of clinical pharmacology

A new population pharmacokinetic model for recombinant factor IX‐Fc fusion concentrate including young children with haemophilia B

Article

Author: Cnossen, Marjon H ; Bukkems, Laura H ; Payne, Jeanette ; Mathôt, Ron A A ; Chowdary, Pratima ; Tait, R Campbell ; Goedhart, Tine M H J ; Leebeek, Frank W G ; Bagot, Catherine N ; Fijnvandraat, Karin ; Coppens, Michiel ; Curry, Nicola ; Koopman, Sjoerd F ; Collins, Peter W ; Mathias, Mary ; Mulders, Trevor M

Abstract:

Aims:

Recombinant factor IX Fc fusion protein (rFIX‐Fc) is an extended half‐life factor concentrate administered to haemophilia B patients. So far, a population pharmacokinetic (PK) model has only been published for patients aged ≥12 years. The aim was to externally evaluate the predictive performance of the published rFIX‐Fc population PK model for patients of all ages and develop a model that describes rFIX‐Fc PK using real‐world data.

Methods:

We collected prospective and retrospective data from patients with haemophilia B treated with rFIX‐Fc and included in the OPTI‐CLOT TARGET study (NTR7523) or United Kindom (UK)‐EHL Outcomes Registry (NCT02938156). Predictive performance was assessed by comparing predicted with observed FIX activity levels. A new population PK model was constructed using nonlinear mixed‐effects modelling.

Results:

Real‐world data were obtained from 37 patients (median age: 16 years, range 2–71) of whom 14 were aged <12 years. Observed FIX activity levels were significantly higher than levels predicted using the published model, with a median prediction error of −48.8%. The new model showed a lower median prediction error (3.4%) and better described rFIX‐Fc PK, especially for children aged <12 years. In the new model, an increase in age was correlated with a decrease in clearance (P < .01).

Conclusions:

The published population PK model significantly underpredicted FIX activity levels. The new model better describes rFIX‐Fc PK, especially for children aged <12 years. This study underlines the necessity to strive for representative population PK models, thereby avoiding extrapolation outside the studied population.

01 Mar 2024·International journal of biological macromolecules

Ferrocene crosslinked and functionalized chitosan microspheres towards bio-based Fenton-like system for the removal of organic pollutants

Article

Author: Lu, Guoqiang ; Zhang, Yongmin ; Mu, Meng ; Xie, Huan

Dye-containing wastewater treatment has been a major long-term global challenge. For this purpose, a novel bio-based microspheres (CS-FC) with high specific surface area (63.24 m²·g^-1) and nano-channels (17.95 nm) was prepared using chitosan as the framework and ferrocene as a crosslinking active group. CS-FC not only has the ability to rapidly enrich methyl orange (MO) through hydrogen-bonding and electrostatic attraction, but also almost completely degrades it in the presence of H₂O₂/K₂S₂O₈ through a synergistic radical/non-radical mechanism under the activating effect of ferrocene. Without H₂O₂/K₂S₂O₈, the maximum MO adsorption capacity of CS-FC is in the range 871-1050 mg·g^-1, and conforms to a Langmuir isothermal model with pseudo-second-order kinetics. In the presence of H₂O₂/K₂S₂O₈, the removal of MO dramatically increased from 32 % to nearly 100 % after incubation for 60 min, due to the simultaneous formation of highly reactive ¹O₂ and ·OH. The significant contribution from ¹O₂ endowed CS-FC/H₂O₂/K₂S₂O₈ with high universality for degrading various organic pollutants (including azo dyes and antibiotics), a wide pH window (2-8), and low sensitivity to co-existing ions. Such cost-effective, recyclable porous bio-based microspheres are suitable for heterogeneous Fenton-like catalysis in organic wastewater treatment that rely on synergistic radical/non-radical reaction pathways.

News (Medical) associated with Eftrenonacog alfa

19 Jun 2024

·www.prnewswire.com

European Commission grants Sobi® Marketing Authorisation for ALTUVOCT™ for treatment of haemophilia A

STOCKHOLM, June 19, 2024 /PRNewswire/ -- Sobi® today announced the European Commission has granted Marketing Authorisation for ALTUVOCT™ (efanesoctocog alfa), for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A. ALTUVOCT is a high-sustained factor VIII replacement therapy for all ages and any disease severity. Children, adolescents, and adults can experience non-haemophilia factor VIII activity levels (above 40%) for a significant part of the week with once-weekly prophylaxis, reaching trough levels of 15% in adults and adolescents before the next dose. This results in significantly improved protection from bleeds compared to prior factor VIII prophylaxis. The European Commission also endorsed the European Medicines Agency (EMA) recommendation supporting ALTUVOCT's retention of orphan drug designation, granting a 10-year market exclusivity period. The EMA recommendation noted that even considering existing treatments, once-weekly ALTUVOCT prophylaxis demonstrated a significantly lowered annual bleeding rate compared to other factor VIII products, and this constitutes a clinically relevant advantage. "Despite advancements, haemophilia still limits the possibilities of patients' lives and this means there is still a need for treatments that offer elevated protection. ALTUVOCT's high-sustained factor VIII activity and once-weekly dosing schedule have the potential to significantly improve quality of life for people with haemophilia A. The trials demonstrated substantial improvements in the prevention and treatment of bleeds along with significant improvements in physical health, pain, and joint health," said Professor Robert Klamroth, MD, PhD, Head of the Department of Internal Medicine, Vascular Medicine and Coagulation Disorders at the Vivantes Klinikum Friedrichshain, Berlin, Germany. The granting of Marketing Authorisation is based on the results from the pivotal phase 3 studies: XTEND-1 in adults and adolescents and XTEND-Kids in children, which evaluated the efficacy and safety of ALTUVOCT in people with severe haemophilia A. These studies demonstrated that once-weekly ALTUVOCT prophylaxis (50 IU/kg) provided significant bleed protection for any age (mean ABR <1 and 80-88% of patients free from spontaneous bleeds). The outcomes also showed substantial improvement in joint health, physical health, pain and overall quality of life when comparing week 52 and baseline assessments.1,2 No factor VIII inhibitors were observed in the ALTUVOCT clinical program. "Today's announcement marks a major step forward in haemophilia care, offering the potential to significantly improve treatment outcomes and quality of life. For the first time, factor VIII activity levels can be sustained for a significant part of the week with simplified once-weekly dosing. We are proud to work alongside the haemophilia community, as we lead the paradigm shift towards normal haemostasis and create new possibilities together," said Lydia Abad-Franch, MBA, Head of Research, Development, and Medical Affairs, and Chief Medical Officer at Sobi. Haemophilia A is a rare, lifelong genetic condition in which the body does not produce enough, or makes dysfunctional, factor VIII – a protein that is essential for blood clotting. It occurs in about one in 5,000 male births annually, and more rarely in females. People with haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage, and life-threatening haemorrhages. Clinical outcomes have improved over time thanks to significant advances in the treatment options available, however important unmet clinical and social needs still exist for those living with the condition. ALTUVOCT was first approved in the US in February 2023 by the US Food and Drug Administration (FDA). The FDA previously granted efanesoctocog alfa Breakthrough Therapy designation in May 2022 — the first factor VIII therapy to receive this designation, Fast Track designation in February 2021, and Orphan Drug designation in 2017. References 1. von Drygalski. A., Chowdary. P., Kulkarni. R., et al. Efanesoctocog Alfa Prophylaxis for Patients with Severe Haemophilia A. N Engl J Med 2023; 388:310-318. 2.Malec, L., Dunn, A., Carcao, M., Zulfikar, B., Berrueco, R., Brown, S., Khan, U., Gunawardena, S., Neill, G., Abad-Franch, L., Bystrická, L., Santagostino, E., & Susen, S. (2023). Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children with Severe Hemophilia A in the XTEND-Kids Phase 3 Study. Blood, 142 (Supplement 1), 3993. About XTEND-1 XTEND-1 was an open-label, non-randomized interventional study with two parallel assignment arms. Participants in the prophylaxis arm received a weekly prophylactic dose of efanesoctocog alfa for 52 weeks. Participants in on-demand arm received efanesoctocog alfa on demand for 26 weeks followed by a switch to weekly prophylaxis for another 26 weeks. XTEND-1 evaluated efficacy, safety and pharmacokinetics in 159 previously treated patients ≥12 years of age with severe haemophilia A. About XTEND-Kids XTEND-Kids was an open-label, non-randomised interventional, single-arm study. Participants received a weekly prophylactic dose of efanesoctocog alfa for 52 weeks. XTEND-Kids evaluates efficacy, safety, and pharmacokinetics in 74 previously treated patients <12 years of age with severe haemophilia A. About ALTUVOCT™ ALTUVOCT (efanesoctocog alfa) [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is the first high-sustained FVIII replacement therapy with the potential to deliver near-normal factor activity levels for a significant part of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the established Fc fusion technology by innovatively adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. The European Commission granted Orphan Drug designation in June 2019 It is approved and marketed as ALTUVOCT™ by Sobi in Europe. It is approved and marketed as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the United States, Japan, and Taiwan. About the Sobi and Sanofi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of ALTUVOCT™, or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the centre of our ambitions. Sanofi is listed on EURONEXT: SAN andNASDAQ: SNY Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: SOURCE Swedish Orphan Biovitrum AB

Clinical ResultOrphan DrugPhase 3Drug ApprovalBreakthrough Therapy

26 Apr 2024

·www.prnewswire.com

Sobi® receives positive CHMP opinion recommending approval of efanesoctocog alfa for once-weekly treatment of haemophilia A

STOCKHOLM, April 26, 2024 /PRNewswire/ -- Sobi® today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion recommending approval of efanesoctocog alfa, for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A. Efanesoctocog alfa is a once-weekly and high-sustained factor VIII replacement therapy for patients of all ages and any disease severity. Efanesoctocog alfa provides children, adolescents and adults with normal to near-normal factor VIII activity levels (above 40%) for a significant part of the week with once-weekly dosing, reaching trough levels of 15% in adults and adolescents at day 7. This results in significantly improved protection from bleeds compared to existing factor VIII prophylaxis. The CHMP positive opinion will now be submitted to the European Commission for a marketing authorisation decision. "Today's announcement marks a major milestone in haemophilia care and moves us one step closer to bringing efanesoctocog alfa to patients in the EU. Efanesoctocog alfa sustains high factor VIII activity levels throughout the week, giving patients confidence in the protection it can provide to prevent bleeds, manage surgery, and resolve bleeds. With the potential to significantly improve treatment outcomes and quality of life for people living with haemophilia A, we are excited about the positive impact this treatment could have around the world," said Lydia Abad-Franch, MBA, Head of Research, Development, and Medical Affairs, and Chief Medical Officer at Sobi. Haemophilia A is a rare, lifelong genetic condition in which the body does not produce enough, or makes dysfunctional, factor VIII – a protein that is essential for blood clotting. It occurs in about one in 5,000 male births annually, and more rarely in females. People with haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages. Clinical outcomes have improved over time thanks to significant advances in the treatment options available, however important unmet clinical and social needs still exist for those living with the condition. The recommendation from the CHMP is based on the results from the pivotal phase 3 studies: XTEND-1 in adults and adolescents and XTEND-Kids in children, which evaluated the efficacy and safety of efanesoctocog alfa. These studies demonstrated that once-weekly efanesoctocog alfa prophylaxis (50 IU/kg) provided significant bleed protection (mean ABR <1 and 80-88% of patients free from spontaneous bleeds) in people with severe haemophilia A of any age. The data also showed substantial improvement in joint health, physical health, pain and overall quality of life when comparing week 52 and baseline measurements.1 No factor VIII inhibitors were observed in the efanesoctocog alfa clinical program. Efanesoctocog alfa was first approved in the US in February 2023 by the US Food and Drug Administration (FDA). The FDA previously granted efanesoctocog alfa Breakthrough Therapy designation in May 2022 — the first factor VIII therapy to receive this designation, Fast Track designation in February 2021, and Orphan Drug designation in 2017. References von Drygalski. A., Chowdary. P., Kulkarni. R., et al. Efanesoctocog Alfa Prophylaxis for Patients with Severe Haemophilia A. N Engl J Med 2023; 388:310-318. About XTEND-1 XTEND-1 was an open-label, non-randomized interventional study with two parallel assignment arms. Participants in the prophylaxis arm received a weekly prophylactic dose of efanesoctocog alfa for 52 weeks. Participants in on-demand arm received efanesoctocog alfa on demand for 26 weeks followed by a switch to weekly prophylaxis for another 26 weeks. XTEND-1 evaluated efficacy, safety and pharmacokinetics in 159 previously treated patients ≥12 years of age with severe haemophilia A. About XTEND-Kids XTEND-Kids was an open-label, non-randomised interventional, single-arm study. Participants received a weekly prophylactic dose of efanesoctocog alfa for 52 weeks. XTEND-Kids evaluates efficacy, safety, and pharmacokinetics in 74 previously treated patients <12 years of age with severe haemophilia A. About efanesoctocog alfa Efanesoctocog alfa [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is a new class of recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for a significant part of the week, improving 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 extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. It is approved and marketed as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the United States, Japan, and Taiwan. The European Commission granted Orphan Drug designation in June 2019. About the Sanofi and Sobi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the centre of our ambitions. Sanofi is listed on (EURONEXT: SAN) and (NASDAQ: SNY). Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: SOURCE Swedish Orphan Biovitrum AB

Clinical ResultOrphan DrugDrug ApprovalPhase 3Fast Track

02 Apr 2024

·www.biospace.com

Avalo Therapeutics Appoints Biotech Leaders to Board of Directors

Appointments include industry veterans Aaron Kantoff, Jonathan Goldman, and Samantha Truex as Independent Directors Board of Director appointments in conjunction with Avalo’s recent acquisition of AVTX-009, an anti-IL-1β mAb, and up to $185 million private placement financing WAYNE, Pa. and ROCKVILLE, Md., April 02, 2024 (GLOBE NEWSWIRE) -- Avalo Therapeutics, Inc. (Nasdaq: AVTX) today announced the addition of multiple biotech leaders to its Board of Directors. The Avalo Board brings together a team with expertise spanning finance, business operations, drug discovery and clinical development. “I am honored to announce these additions to our Board of Directors and welcome their operational expertise as we pursue the promise of rapidly developing AVTX-009 in hidradenitis suppurativa and other devastating autoimmune conditions," said Garry A. Neil, MD, CEO and Chairman of the Board at Avalo. The recent board appointments include Aaron Kantoff, Jonathan Goldman, and Samantha Truex as Independent Directors. Aaron Kantoff is a founder and Managing Partner of Scion Life Sciences. He has spent over a decade specializing in founding, building and investing in biotech companies. Aaron was a founding board member of Tourmaline Bio (Nasdaq: TRML), Centessa Pharmaceuticals, and RayzeBio, which was recently acquired by Bristol Myers Squibb. From 2011 until 2019, Aaron was a Partner at Apple Tree Partners (ATP), a life sciences venture capital firm. During his time at ATP, he was a board member of several companies, including Akero Therapeutics , Corvidia (acquired by Novo Nordisk), and Syntimmune (acquired by Alexion Pharmaceuticals, Inc.). Aaron received a B.S. in finance and international business from the New York University Leonard N. Stern School of Business. Jonathan Goldman, MD has 30 years of experience across life sciences as a CEO, Chief Medical Officer, investor, and senior executive. He currently serves as the CEO of Clinical ink, a global life science company that brings data, technology, and patient-centric research together. Prior to Clinical ink, Jonathan served as the CEO and a board member of Abzena and was previously the CEO of Aptuit. He has also held senior executive positions at ICON plc and Point Biomedical Corp. in addition to holding appointments as Associate Clinical Professor of Medicine in the division of Cardiology at the University of California San Francisco, and as an Attending Cardiologist at the San Francisco Veterans Administration Medical Center. Jonathan trained in medicine at St. Bartholomew’s Hospital Medical College, in London and in cardiology at St. George’s Hospital Medical School, London. He received a BSc in immunology, as well as MBBS and MD degrees in medicine from the University of London, UK. He was awarded MBAs by Columbia University in New York and the University of California at Berkeley. Samantha Truex is a seasoned biotech executive with almost 30 years of industry experience, including the last five years in CEO roles. Sam is on the board of Artios Pharma and has previously served on the boards of Hotspot Therapeutics, iPierian (acquired by Bristol Myers Squibb) and True North (acquired by Bioverativ Inc.). Sam was the founding CEO of Upstream Bio, the CEO of Quench Bio, COO of Synlogic and CBO for Padlock Therapeutics. Previously, Sam was Vice President of Corporate Development at Biogen Inc. where she led transactional business development activities and served as program executive for now-marketed products FAMPYRA,® ELOCTATE™ and ALPROLIX™. Sam also previously worked in Corporate Development at Genzyme, Chiron Diagnostics and in consulting for Health Advances. Sam earned a BA in biology from Dartmouth College, a BE in biomedical engineering from the Thayer School at Dartmouth and an MBA from the Tuck School at Dartmouth. Samantha also chairs the Board of Advisors for Thayer School of Engineering at Dartmouth and is a member of the Board of Advisors for Life Science Cares. About AVTX-009 AVTX-009 is a humanized monoclonal antibody (IgG4) that binds to interleukin-1β (IL-1β) with high affinity and neutralizes its activity. IL-1β is a central driver in the inflammatory process. Overproduction or dysregulation of IL-1β is implicated in many autoimmune and inflammatory diseases. IL-1β is a major, validated target for therapeutic intervention. There is evidence that inhibition of IL-1β could be effective in hidradenitis suppurativa and a variety of inflammatory diseases in dermatology, gastroenterology, and rheumatology. About Avalo Therapeutics Avalo Therapeutics is a clinical stage biotechnology company focused on the treatment of immune dysregulation. Avalo’s lead asset is AVTX-009, an anti-IL-1β mAb, targeting inflammatory diseases. Avalo’s pipeline also includes quisovalimab (anti-LIGHT mAb) and AVTX-008 (BTLA agonist fusion protein). For more information about Avalo, please visit Forward-LookingStatements This press release may include forward-looking statements made pursuant to the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts. Such forward-looking statements are subject to significant risks and uncertainties that are subject to change based on various factors (many of which are beyond Avalo’s control), which could cause actual results to differ from the forward-looking statements. Such statements may include, without limitation, statements with respect to Avalo’s plans, objectives, projections, expectations and intentions and other statements identified by words such as “projects,” “may,” “might,” “will,” “could,” “would,” “should,” “continue,” “seeks,” “aims,” “predicts,” “believes,” “expects,” “anticipates,” “estimates,” “intends,” “plans,” “potential,” or similar expressions (including their use in the negative), or by discussions of future matters such as: the intended use of the proceeds from the private placement; integration of AVTX-009 into our operations, including trial design and IND preparation and filing for the planned Phase 2 trial; drug development costs, timing of trials and trial results, and other risks, particularly for AVTX-009, including reliance on investigators and enrollment of patients in clinical trials; reliance on key personnel; regulatory risks; general economic and market risks and uncertainties, including those caused the war in Ukraine and the Middle East; and those other risks detailed in Avalo’s filings with the Securities and Exchange Commission, available at . Actual results may differ from those set forth in the forward-looking statements. Except as required by applicable law, Avalo expressly disclaims any obligations or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in Avalo’s expectations with respect thereto or any change in events, conditions or circumstances on which any statement is based. For media and investor inquiries: Christopher Sullivan, CFO Avalo Therapeutics, Inc. ir@avalotx.com 410-803-6793 or Chris Brinzey ICR Westwicke Chris.brinzey@westwicke.com 339-970-2843

Executive ChangeAcquisition

100 Deals associated with Eftrenonacog alfa

External Link

KEGG	Wiki	ATC	Drug Bank
D10522	-	B02BD04	DB11608

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia B	AU	Sanofi-Aventis Australia Pty Ltd.	01 May 2014
Hemorrhage	US	Bioverativ Therapeutics, Inc.	28 Mar 2014

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01027364 (B-LONG, ISTH2023)	Phase 3	Hemophilia B	-	Recombinant Factor IX Fc Fusion Protein	vafsherizl(cnnylkqlim) = glgfyzwwem ucyxkbxatc (afvglrlklw )	-	24 Jun 2023
NCT01027364 (B-LONG, ISTH2023)	Phase 3	Hemophilia B	-	Recombinant Factor IX Fc Fusion Protein	vafsherizl(cnnylkqlim) = uwhlhlavsk ucyxkbxatc (afvglrlklw )	-	24 Jun 2023
B-MORE (ISTH2022)	Not Applicable	Hemophilia B	117	Prophylactic rFIXFc treatment	auosefrsuf(uvcqogawjc) = kdcvnbhfyi zmzzduvciu (febmubrvoj )	-	09 Jul 2022
ISTH2022	Not Applicable	Hemophilia B	-	SHL FIX	fundobkpze(jcmziintgp) = jctjiftkcz txtrkudpvo (qgwsplfqle )	-	09 Jul 2022
ISTH2022	Not Applicable	Hemophilia B	-	rFIXFc	fundobkpze(jcmziintgp) = ugsbcendga txtrkudpvo (qgwsplfqle ) View more	-	09 Jul 2022
ISTH2021	Not Applicable	Hemorrhage	38	rFIXFc	tawiwxgeac(lrwnxydbwn) = jthetydrpl dxbvdvjndv (jgqvrddebk )	-	17 Jul 2021
ISTH2021	Not Applicable	Hemorrhage	38	SHL FIX	tawiwxgeac(lrwnxydbwn) = ndscmczokg dxbvdvjndv (jgqvrddebk )	-	17 Jul 2021
NCT02234310 (PUPs B-LONG, Pubmed \| Blood Adv) Manual	Phase 3	Hemophilia B	33	rFIXFc	yuotpbupmk(mjxkwpxxlg) = ucxvkbdyhi npdhlhqicf (cvlkgclfev, 0.08 - 15.76) View more	Positive	13 Jul 2021
ISTH2020	Not Applicable	Hemophilia B	40	N9-GP	zizglxfpjc(numxsttyws) = lmnaqjlsmh mogflkrsmi (ysojuzfpnf )	-	12 Jul 2020
ISTH2020	Not Applicable	-	-	rFIXFc	fimvyhnksc(kbuhfndkyz) = eepflwrwqp pnbumsttfi (xppohojdsx ) View more	-	12 Jul 2020
ISTH2020	Not Applicable	Hemophilia B	23	Recombinant Factor IX Fc Fusion Protein Concentrate	wgavjdgwno(eabgamivba) = zfystbgdod apjdvpodsf (ipdyonavij ) View more	-	12 Jul 2020
NCT01440946 (CTgov)	Phase 3	Hemorrhage \| Hemophilia B	30	FIX+rFIXFc (Participants < 6 Years Old)	lshimshvjl(tzaanowlyh) = egwnrnqlgw nryvmsmvdf (ldyesvxivl, nlwgqnvraj - phiixfxzxx) View more	-	18 Jun 2015
NCT01440946 (CTgov)	Phase 3	Hemorrhage \| Hemophilia B	30	FIX+rFIXFc (Participants 6 to < 12 Years Old)	lshimshvjl(tzaanowlyh) = xlqmpsvvhd nryvmsmvdf (ldyesvxivl, fycluvnhgi - uwkttawalh) View more	-	18 Jun 2015

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