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Sangamo Clinical Trials Update

|About: Sangamo Therapeutics, Inc. (SGMO), Includes: BLUE, BMRN, CRSP, ONCE, PFE, QURE, RGX, SHP, SNY

Sangamo is conducting five trials (HemA, HemB, MPS I, MPS II and Beta-Thalassemia.  A sixth approved trial will be run by collaboration partner Sanofi.

There will be significant updates in the next few months and readouts for all six trials as well as the sponsor initiated HIV trial funded by Case University in 2019.

This blog will be updated as additional information is disclosed.  The last update was based upon the London Jefferies presentation Nov 15th.

Sangamo (SGMO) is released 3Q18 earnings and with updates on their pipeline in on Nov 8th.  Five trials are being conducted by Sanagmo with a sixth approved trial to be conducted by collaboration partner Sanofi (SNY). 

The details for the Sangamo programs are:

Alta Study SB-525 

There have been six patients treated in three dose cohorts.  Sangamo and Pfizer decided not to present a late breaking abstract with data at ASH 2018.  Due to a data embargo from ASH, Sangamo disclosures  indicating the treatments are considered safe and well-tolerated, the second cohort has shown treatment benefits from reduced enzyme replacement therapy and the first patient in the third dose cohort has achieved therapeutic efficacy.  There was a safety review meeting in October which recommended dose escalation which will begin shortly.  The company has decided not up provide data updates until after this cohort is complete for competitive reasons.

  • Pfizer (PFE) collaboration
  • Hemophilia A gene therapy
  • There are now 12 clinical sites open in US.
  • Other related trials from BioMarin (BMRN), Spark (ONCE) and Shire (SHP).

FIXtendz Study SB-FIX

  • Not collaborated
  • Hemophilia B genome editing
  • There are 6 US clinical sites open.
  • There are 2 EU sites open in London.
  • The company has skipped the original first two cohort dose levels based upon the safety profile of MPS trials.
  • Other related trials from Spark and Uniqure (QURE).

Empowers Study SB-318

The initial trial protocol has been amended to eliminate the first low-dose cohort which was found to be safe and well-tolerated in the SB-913 study.  The first patient was treated in mid-July.  At the October SMC the decision was made to move to the third dose cohort for the 2nd patient based upon the safety profile of MPS II study.  This patient has now been treated. Sangamo hopes to provide preliminary data on these treatments in February at WORLDSymposium2019.  

  • Not collaborated
  • MPS I genome editing
  • There were 6 clinical sites open in June (last update).
  • Other related trial from Regenxbio (RGX).

Champions Study SB-913

There was a discussion of the preliminary results for this trial on September 5, 2018 for the first 5 patients.  The treatment was considered safe and well-tolerated.  Sangamo was unable to measure IDS in circulating plasma, indicating their assay could not measure precisely enough and was not validated for use.  The company believes the editing took place because the urinary GAG reduction was significant as compared to uGAG after enzyme replacement treatment.  The next update will occur in February at WORLDSymposium2019.  At the October SMC the decision was made to 1) expand this cohort, 2) begin ERT withdrawal and 3) begin adolescent recruitoing.

  • Not collaborated
  • MPS II genome editing
  • There are 7 clinical sites open. 
  • The safety and efficacy data from this trial was sufficient to dose escalate both SB-FIX and SB-318 studies.
  • Other related trial from Regenxbio.

Thales Study ST-400

The first patient was enrolled in September with a second expected by CYE.  Treatment is expected in 2H18.  Sangamo has indicated they hope to discuss further in early 2019.

  • Sanofi collaboration
  • Transfusion dependent Beta-Thalassemia or TDT
  • There are 4 clinical sites open.
  • Other related trials from Bluebird bio (BLUE) and CRISPR (CRSP).

Sanofi will conduct the sickle cell disease or SCD gene therapy trial.  This has been approved for the clinic.  No patients have been processed.  No additional information is expected until 2019. 

Disclosure: I am/we are long SGMO.