Clinical Trial Updates (SCD)

exa-cel (gene editing)

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  • Product Information

    Product Information

    Scientific name: exa-cel (exagamglogene autotemcel)
    Brand name: N/A
    RESPONSIBLE: Vertex and CRISPR Therapeutics collaboration

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Last update: 22/6/2023

    CLIMB-121

    Trial Name: CLIMB-121
    Code: NCT03745287 
    Phase: 1/2/3
    Eligible patient diagnosis: SCD (adult & paediatric/adolescent; ages 12 – 35)
    No. of Patients enrolled: 35 (Last update: 10/6/2023)
    Study Sites: 17  Sites per country

    Anticipated completion date: October 2024
    Scope of the Study / Aim: No VOCs for at least 12 consecutive months

    15 Years Follow-up Study

    Trial Name: Long-term follow-up study (CLIMB-131)
    Code: NCT04208529
    Phase: Long-term follow-up study of CLIMB-111 (TDT) & CLIMB-121 (SCD)
    Eligible patient diagnosis: TDT patients who participated in CLIMB-121 (ages 2 and older)
    No. of Patients enrolled: 114 [total anticipated] (Last update: 30/6/2023)
    Study Sites: 15 Sites per country

    Anticipated completion date: September 2039
    Scope of the Study / Aim: After completing the parent clinical study, patients will be followed for 15 years post infusion. Monitoring for malignancies, new or worsening haematologic disorders, serious adverse events, mortality.

    CLIMB-161

    Trial Name: CLIMB-161
    Code: NCT05477563
    Phase: 3
    Eligible patient diagnosis: TDT – all genotypes & SCD (adult & paediatric/adolescent; ages 12 – 35)
    No. of Patients enrolled: 18 [total anticipated] (Last update: 7/7/2023)
    Study Sites: 5 Sites per country

    Completion date: February 2025
    Scope of the Study / Aim: Concentration of Total (Hb) and Fetal (HbF) for up to 12 months after infusion

    VX21-CTX001-151

    Trial Name: VX21-CTX001-151
    Code: NCT05329649
    Phase: 3
    Eligible patient diagnosis: : SCD (paediatric; ages 2 -11)
    No. of Patients enrolled: 15 [anticipated] (Last update: 31/5/2023)
    Study Sites: 7 Sites Sites per country

    Completion date: May 2026
    Scope of the Study / Aim: Safety & efficacy in children with SCD (no VOCs for at least 12 consecutive months)

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

    Additional notable points:

    • EMA: Orphan Drug Designation (2019), Priority Medicines (PRIME) designation (2020 – SCD) ; Marketing Authorisation Application for TDT & SCD submitted in Dec. 2022 – Under review
    • FDA: Fast Track Designation (2019), Regenerative Medicine Advanced Therapy (RMAT) designation (2020), Orphan Drug Designation (2020) ; BLA submission accepted for TDT & SCD (Jun 2023) – – Priority Review PDUFA Target Date: 8 December 2023
    • MHRA: Granted Innovation Passport (2023), Marketing Authorisation Application submitted in Jan. 2023 – Under review

Update: 30 September 2023

  • FDA plans to hold an advisory committee meeting for exa-cel (date to be confirmed).
  • Investigation in gentler conditioning for exa-cel is underway.
  • The Institute for Clinical and Economic Review (ICER) determined that lovo-cel will be cost-effective if priced between $1.35M to $2.05M. ICER has made recommendations to encourage companies to set prices toward lower end of this range to facilitate access and affordability across all insurance systems (in the USA).

Sources: https://investors.vrtx.com/news-releases/news-release-details/vertex-reports-second-quarter-2023-financial-results 
https://icer.org/news-insights/press-releases/icer-publishes-final-evidence-report-on-gene-therapies-for-sickle-cell-disease/


Update: 30 June 2023

FDA Submission
• BLA application submitted to FDA (3 April 2023) for exa-cel for patients with SCD and TDT. The submission is based on the results of the ongoing Phase 3 studies, CLIMB-111 and CLIMB-121, as well as an ongoing long-term follow-up study, CLIMB-131.
• FDA has accepted Priority Review for BLA submission and set a PDUFA (the date by which the FDA must respond to the application) target date of 8 December 2023.

Trial Progress
• Dosing in the Phase 1/2/3 CLIMB-111 and CLIMB-121 studies continues, as does the CLIMB-131 long-term follow-up study in patients 12 years of age and older.
• Two additional Phase 3 studies of exa-cel continue to enroll patients 5 to 11 years of age with TDT or SCD.

New data
Data presented at the 28th EHA Annual Congress (9 – 11 June 2023) in Frankfurt (Germany) showed that:

• Of the 35 patients with SCD who had received exa-cel at the time of the analysis, 17 patients were evaluable for the primary and key secondary endpoint at the time of the data cut.
• 16/17 (94.1%) were VOC-free for at least 12 consecutive months
• Mean duration of VOC-free was 18.7 months, with a maximum of 36.5 months.
• 17/17 (100%) did not need hospitalisation for VOCs for at least 12 consecutive months
• All patients who received exa-cel, mean fetal hemoglobin was more than 30% of total hemoglobin by Month 3 and was then maintained at approximately 40.0% through follow-up, with pancellular distribution.

Sources: https://www.businesswire.com/news/home/20230402005036/en/
https://investors.vrtx.com/news-releases/news-release-details/vertex-and-crisprtherapeutics-complete-submission-rolling
https://investors.vrtx.com/news-releases/news-release-details/fda-accepts-biologicslicense-applications-exagamglogene
https://investors.vrtx.com/news-releases/news-release-details/vertex-reports-firstquarter-2023-financial-results
https://investors.vrtx.com/news-releases/news-release-details/positive-resultspivotal-trials-exa-cel-transfusion-dependent


Update: 31 March 2023

  • The Phase 1/2/3 CLIMB-111 and CLIMB-121 studies and the CLIMB-131 long-term follow-up study are ongoing in patients 12 years of age and older.
  • Two additional Phase 3 studies of exa-cel in pediatric patients with TDT and SCD continue to enroll patients.

Source: https://news.vrtx.com/news-releases/news-release-details/vertex-reports-fourthquarter-and-full-year-financial-2022

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