RG6358 / Roche - LARVOL DELTA

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors (clinicaltrials.gov) - P1/2 | N=4 | Completed | Sponsor: Spark Therapeutics | Active, not recruiting ➔ Completed

Gene therapy • Trial completion • Gene Therapies • Genetic Disorders • Hematological Disorders • Hemophilia • Rare Diseases

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors (clinicaltrials.gov) - P1/2 | N=4 | Active, not recruiting | Sponsor: Spark Therapeutics | Recruiting ➔ Active, not recruiting | N=30 ➔ 4

Enrollment change • Enrollment closed • Gene therapy • Gene Therapies • Genetic Disorders • Hematological Disorders • Hemophilia • Rare Diseases

SAFETY AND EFFICACY OF GENE THERAPY FOR HEMOPHILIA IN HIV-POSITIVE PARTICIPANTS (EAHAD 2023) - "One serious AE was reported, unrelated to SPK-8011/8016.One of the four participants required prednisone for a presumed capsid immune response. Similar efficacy and safety profiles of SPK-8011 and SPK-8016 were observed in HIV-positive participants and previous reports of overall study populations. Data suggest carefully selected concomitant HAART is suitable for liver-directed AAV GT. In one case, IM therapy was complicated by a DDI involving HIV therapy."

Clinical • Gene therapy • Acne Vulgaris • Gene Therapies • Hematological Disorders • Hemophilia • Hepatitis C • Hepatology • Human Immunodeficiency Virus • Immune Modulation • Infectious Disease • Inflammation • Osteoporosis • Rare Diseases • Rheumatology • IFNG

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors (clinicaltrials.gov) - P1/2 | N=30 | Recruiting | Sponsor: Spark Therapeutics | Active, not recruiting ➔ Recruiting | Trial completion date: Dec 2022 ➔ Dec 2023 | Trial primary completion date: Dec 2021 ➔ Dec 2022

Enrollment open • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Hematological Disorders • Hemophilia • Rare Diseases

[VIRTUAL] SPK‐8016: PRELIMINARY RESULTS FROM A PHASE 1/2 CLINICAL TRIAL OF GENE THERAPY FOR HEMOPHILIA A (EAHAD 2021) - "These 3 subjects received courses of oral steroids for 43-48 weeks; azathioprine or tacrolimus was added for Subjects 1 & 2 to limit steroid exposure... Preliminary results from the ongoing Phase 1/2 trial of SPK-8016 demonstrate safety and sustained FVIII:C levels ranging from 6.2-21.8% at ≥ 52 weeks follow-up at the first dose tested of 5 x1011 vg/kg. The observed safety and phenotypic correction support ongoing clinical study of SPK-8016 including optimization of the immunomodulatory regimen, with the potential for clinically meaningful FVIII activity at low vector doses."

Clinical • P1/2 data • Gene Therapies • Hematological Disorders • Hemophilia • Human Immunodeficiency Virus • Immune Modulation • Immunology • Infectious Disease • Inflammation • Rare Diseases • CD4

Spark Therapeutics Announces Preliminary Data from Phase 1/2 Clinical Trial of SPK-8016 in Hemophilia A at EAHAD 2021 Virtual Congress (GlobeNewswire) - P1/2, N=4; "Spark Therapeutics...today announced preliminary data from part one of the ongoing Phase 1/2 open-label, non-randomized, dose-finding study of the investigational SPK-8016 in hemophilia A....These data were presented at the European Association for Haemophilia and Allied Disorders (EAHAD) 2021 Virtual Congress by investigator Spencer Sullivan, M.D., Mississippi Center for Advanced Medicine....Data show a 98-percent reduction in annualized infusion rate (AIR) and 85-percent reduction in annualized bleed rate (ABR) after a follow up of between 15 and 18 months or 5.5 total patient years."

P1/2 data • Hemophilia

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors (clinicaltrials.gov) - P1/2; N=30; Active, not recruiting; Sponsor: Spark Therapeutics; Trial completion date: Nov 2021 ➔ Dec 2022; Trial primary completion date: Nov 2020 ➔ Dec 2021

Clinical • Trial completion date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors (clinicaltrials.gov) - P1/2; N=30; Active, not recruiting; Sponsor: Spark Therapeutics; Trial completion date: May 2020 ➔ Nov 2021; Trial primary completion date: May 2020 ➔ Nov 2020

Clinical • Trial completion date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors (clinicaltrials.gov) - P1/2; N=30; Active, not recruiting; Sponsor: Spark Therapeutics; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Roche looks undervalued given the stability of the business (SeekingAlpha) - "Roche also announced that it was pushing back the Phase III study of SPK-8011 for hemophilia A into 2021 to do more work on optimizing dosing and immunosuppression; with a high bar for hemophilia drugs (due to the availability of drugs like Hemlibra), that makes sense, and Phase I/II data should be available in July."

P1/2 data • Trial initiation date • Genetic Disorders • Hemophilia

Hemophilia A-OK! FTC thumbs up effectively closes Roche’s $4.3bn Spark buy (BioProcess International) - "In February, Swiss Biopharma firm struck a deal to buy Philadelphia, Pennsylvania-based Spark Therapeutics for $4.3 billion (€3.8 billion). Now after months of investigation by the US Federal Trade Commission (FTC), Roche is on the verge of closing the deal; There is no risk of competitive harm between Roche’s approved hemophila A monoclonal antibody and Spark’s gene therapy candidates, say the US and UK competition bodies."

M&A