January 17, 2019
BIOINVEST NEWS: Sangamo (SGMO)
JPM Update – WORLD, 5 Trials Underway, 4 More To Start, Etc.
Sangamo presented an overview of its clinical and technological gene therapy/editing leadership. With 5 trials underway and data from all five clinical results due this year, to us 2019 will be the year when SGMO gaps ahead of the gene editing crowd. The upcoming WORLD meeting (2/7) will begin to show that. REITERATE BUY.
WORLD To Include IDS Levels – The upcoming WORLDSymposium on February 7 in Orlando (https://www.worldsymposia.org) will demonstrate the most up-to-date CHAMPIONS Phase I/II trial of SB-913 in MPSII. The data will include 24-week safety and changes in GAG /IDS levels on 6 patients of data. The company will also be using a much higher-sensitivity IDS assay. This is rather important, as SMGO shares dropped sharply after the first look at CHAMPIONS data last September showed a dose-dependent drop in GAG but not a material and corresponding rise in IDS levels. IDS levels are the study’s primary endpoint and what the Company believes is being produced – and that will lead to patient’s withdrawing from Enzyme Replacement Therapy (ERTs).
SGMO shares fell after the breakout session when someone asked what is the main way to know if SB-913 is working? McCrae said that is when a patient comes off ERTs. Although that is not the primary endpoint and knowing that it is just too early for patient’s to have begun to withdraw from ERTs (or maybe they have started), that data point will not be at WORLD – even though it was never supposed to be there in the first place. This was a major over-reaction in our view. Sometime in 2019, liver biopsy and ERT withdrawal data will be released/updated which should prove the efficacy. That is and has always been the plan.
The key event for us is the IDS levels – and learning that the new assay will be presented at WORLD is a major positive, to prove that SB-913 is indeed producing therapeutic IDS levels (that will eventually lead to ERT withdrawal). Producing and measuring therapeutic IDS are the next logical step in the clinical program of SB-913.
MPS I Update Also At WORLD – The first look at the second SGMO in-vivo gene editing trial will include 4-week safety on 3 patients.
Hemophilia A Gene Therapy – SB-525 with Pfizer – The next SMC meeting is due soon and a further recommendation to begin dosing the second patient in the fourth cohort is likely. SGMO believes this is probably the last dose for SB-525. Updated SB-525 data are anticipated sometime in 2019.
New Zinc Finger 2.0 Publication – An upcoming zinc finger publication is due in early 2019, describing ZNF 2.0 and how the nuclease technology can be optimized to generate higher specificity/lower off target cutting.
INDs/Updates For 2019 – Include Fabry’s disease, Beta-thalassemia (BIVV/SNY), sickle cell disease and the first TReg trial from GILD (renal transplants). Further partnerships may include CNS (tau/Alzheimer’s)/Immunology.
In our view, if any one of the above studies is successful, both the SGMO market cap and takeover potential will skyrocket. With the current update, we remain confident that it will.