IDO 8 / Idogen - LARVOL DELTA

Factor VIII regulates extracellular matrix proteins to stimulate angiogenesis and vessel stability (ISTH 2024) - "Background: Hemophilia A (HA) is an X-linked bleeding condition resulting from mutations in the coagulation factor VIII (FVIII)... HA BOECs showed alteration of vessel-formation, endothelial cell migration, and vessel permeability. This impaired phenotype was reverted by the re-introduction of FVIII gene or by acute treatment with recombinant FVIII. The endothelial function of FVIII was confirmed in vivo in NSG-HA mice which showed that an altered angiogenesis and vessel permeability could be corrected by exogenous FVIII."

Hematological Disorders • Hemophilia • Rare Diseases • NID2

Trial in Progress: An Open-Label, Multi-Center, First in Human, Phase 1/2a Trial to Evaluate the Safety and Preliminary Efficacy of Autologous Tolerogenic Dendritic Cells Ex Vivo Loaded with Recombinant Factor VIII (FVIII) in Adults with Congenital Hemophilia a (HA) with Neutralizing Antibodies to FVIII and Having Failed Immune Tolerance Induction (ITI) (ASH 2022) - "Concomitant emicizumab is allowed, but FEIBA and other FVIII-containing treatments are excluded except in the case of an urgent need to treat bleeding.The primary objective is safety/tolerability of ItolDC-028, evaluated as the number of adverse events (AEs), changes in vital signs, physical examination findings and laboratory assessments including titers of neutralizing antibodies (BU) and ECG recordings from administration of ItolDC-028 up to the week 26 visit.The secondary objective is to evaluate the preliminary therapeutic efficacy of three dose levels of ItolDC-028 through evaluation of immune response, titer of neutralizing antibodies (BU), FVIII recovery and half-life as response to FVIII challenge at the week 20 visit.A minimum of nine subjects will be included in a classic 3+3 trial design, with single incremental dose levels of 36x106, 72x106 and 144x106 cells...Figure 2.Twenty weeks after ItolDC-028 administration, the subject will be challenged by an..."

P1/2 data • Preclinical • Hematological Disorders • Hemophilia • Immunology • Mood Disorders • Rare Diseases

Idogen announces outcome of rights issue (Cision) - "The board of directors of Idogen AB...today announces the outcome of the rights issue of units that was announced on December 17, 2021....In total 7,856,837 units, corresponding to approximately 47.7 percent of the Rights Issue, were subscribed for by exercise of unit rights, and 157,092 units...With the outcome of the Rights Issue and the expected outcome from the exercise of warrants series TO 5, we get financial resources to start the clinical phase I/IIa study on patients with severe hemophilia, hemophilia A, and who have developed antibodies against their vital treatment with coagulation factor VIII (FVIII)."

Financing • New P1/2 trial • Genetic Disorders • Hemophilia

BioStock: Emission will take Idogen's IDO 8 to phase I / IIa [Google translation] (Cision) - "Idogen is on track to reach one of its most important milestones to date - to test cell therapy IDO 8 for the first time on patients with haemophilia....To finance the study with IDO 8, Idogen begins the year by carrying out a rights issue of SEK 50.4 million."

Financing • Genetic Disorders • Hemophilia

Idogen adds Norway for its clinical phase 1/2a study and submits an application for approval for its cell therapy treatment IDO 8 to the Norwegian Medicines Agency NoMA (Market Screener) - Idogen adds Norway for its clinical phase 1/2a study and submits an application for approval for its cell therapy treatment IDO 8 to the Norwegian Medicines Agency NoMA "Idogen AB announces today that in addition to its Swedish application, it has also sub-mitted an application to the Norwegian Medicines Agency, NoMA, to be able to begin its clinical phase 1/2a study of the company's most advanced cell therapy treatment IDO 8 in Norway....Given that the application is approved by NoMA without adjustments, the study is planned to start during the second quarter of 2022."

New P1/2 trial • Genetic Disorders • Hemophilia

Idogen carries out a fully guaranteed rights issue of units of approximately SEK 50.4 million and brings forward the publication of the year-end report for 2021 [Google translation] (Cision) - "The clinical phase I/IIa study for IDO 8 is intended to be based on hemophilia patients with severe haemophilia and the outcome of the study is expected at the end of 2023....Upon full subscription in the Rights Issue, Idogen will initially receive approximately SEK 50.4 million before issue costs, which are estimated to amount to approximately SEK 9.4 million. The net proceeds from the Rights Issue of approximately SEK 41.0 million are intended to be used for the following areas of use, in order of priority: 55% - Start of clinical study of IDO 8 - approximately SEK 22.6 million..."

Financing • P1/2 data • Genetic Disorders • Hemophilia

Idogen submits an application to the Swedish Medical Products Agency to initiate a clinical phase 1/2a study of the cell therapy treatment IDO 8 (Cision) - "Idogen AB announces today that it has submitted a clinical trial application to the Swedish Medical Products Agency (MPA) to begin its clinical phase 1/2a study of the company's most advanced cell therapy treatment, IDO 8....The aim of the planned phase 1/2a study, which has been designed in close dialogue with the regulatory authorities in the Nordic countries, is to evaluate the safety of IDO 8 and to monitor patients for signals of a clinical treatment effect. The planned start date of the trial is dependent on the MPA review process, but it is anticipated the study, once approved, will be initiated in the second quarter of 2022."

New P1/2 trial • Genetic Disorders • Hemophilia

Idogen has successfully completed the optimization of the GMP manufacturing process for the first clinical study with its tolerogenic cell therapy IDO 8 (Cision) - "Idogen AB announces today that it has successfully maximized the antigen levels in the cell therapy IDO 8 that the body should learn to recognize and tolerate. This is considered to be an important feature in order to obtain a good treatment effect....A successful tech transfer and scale-up of the manufacturing process for Idogen's cell therapy has been followed by extensive work aimed at optimizing, documenting and qualifying the manufacturing process for the planned first clinical study. The company's manufacturing partner, Radboud University Medical Center (RUMC) in the Netherlands...has now optimized and qualified the manufacturing process, achieving product properties equivalent to those generated when IDO 8 was manufactured in Idogen's own research laboratory. This has been achieved through an effective collaboration between RUMC and Idogen."

Commercial • Preclinical • Genetic Disorders • Hemophilia

Idogen AB updates on the company’s Q3 report and key activities [Google translation] (Cision) - "Idogen is now in the final phase of preparations for its first clinical study with the tolerogenic cell therapy IDO 8 that is being developed for severe haemophilia, hemophilia A."

New trial • Genetic Disorders • Hemophilia

Today begins the exercise period for warrants of series TO 4 in Idogen [Google translation] (Cision) - "In total, Idogen issued 9,121,654 units during the fourth quarter of 2020, which consisted of one (1) share and one (1) Warrant. The exercise period of the Warrants runs from today, September 21 , 2021, up to and including October 5, 2021. In the event that all Warrants are exercised for subscription of shares, the Company will receive approximately SEK 18 million before issue costs. The net proceeds of approximately SEK 17 million are intended to be used for the following purposes in the specified order of priority: Phase I / IIa study in IDO 8, approximately SEK 5 million..."

Financing • Genetic Disorders • Hemophilia

Idogen continues to optimize its production method prior to the start of the patient study with IDO 8 [Google translation] (Cision) - "Idogen AB announces today that it has initiated additional activities in order to maximize the effect of its tolerogenic cell therapy IDO 8 before the start of the first patient study. The company has already established a functioning and patent-pending process intended for a GMP production of individually adapted treatments....The company has conducted a customary dialogue with relevant drug authorities, completed study protocols and prepared their application for study start. This application is ready to be submitted as soon as the now initiated activities to optimize the production process have been completed....The start of studies, which was previously expected to take place at the turn of the year 2021/22, is now planned for the beginning of the second quarter of 2022."

New trial • Trial initiation date • Genetic Disorders • Hemophilia

Factor VIII exhibits chaperone-dependent and glucose-regulated reversible amyloid formation in the endoplasmic reticulum. (PubMed, Blood) - "A short aggregation motif in the FVIII A1 domain (termed Aggron) is necessary and sufficient to seed b-sheet polymerization and BiP binding to this Aggron prevents amyloidogenesis. Our findings provide novel insight into mechanisms that limit FVIII secretion and ER protein aggregation in general and have implication for ongoing hemophilia A gene therapy clinical trials."

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