We are reiterating our OUTPERFORM rating on shares of Sangamo
Therapeutics (SGMO) following data update for its hemophilia A gene
therapy, SB-525, with partner Pfizer (PFE). Overall data suggest
stable Factor VIII activity in the normal range between weeks 19-24
for the highest dose cohort and well above the 68% level mean level
from competitor Biomarin (BMRN) at 24 weeks. With Biomarin data
suggesting waning efficacy from 64% to 36% between 12 months
and 24 months, additional follow up will be important but at this
stage data appear best-in-class, in our view, and supportive of
significant upside potential given Pfizer investment in late stage
development and manufacturing.
? Sangamo Therapeutics (SGMO) and partner Pfizer (PFE) reported
over the weekend updated data from the phase 1/2 Alta study of
their gene therapy product candidate SB-525 in hemophilia A at the
International Society on Thrombosis and Haemostasis (ISTH)
meeting, and also announced that FDA has granted SB-525 the
RMAT (regenerative medicine advanced therapy) designation.
? The Alta study treated 10 patients at four dose levels (9E11, 2E12,
1E13, and 3E13 vg/kg). The highest dose cohort has treated 4
patients, including 2 patients (P7 and P8) for whom initial data were
reported in April 2019, and two new patients (P9 and P10). The
factor VIII (FVIII) levels in P7 and P8 remained in normal range
(measured by a chromogenic assay) at 24 and 19 weeks of follow-
up, respectively. The two new patients, P9 and P10, had 6 and 4
weeks of follow-up, respectively, and they demonstrated rapid FVIII
increase kinetics that appear consistent with P7 and P8 at similar
time points (though with slightly lower starting levels). SGMO noted
that, after the data cut-off for the ISTH presentation, P9 reached
normal FVIII levels at week 7. Overall, it was noted that patients in
the highest dose cohort reached normal FVIII levels (50-150%)
within 5-7 weeks of treatment, with sustained FVIII levels and no
bleeding episodes and no factor replacement use. SGMO also
reported that the two patients treated in the second highest dose
cohort (1E13 vg/kg) have demonstrated durability of FVIII levels
through weeks 52 and 32, respectively.
? SB-525 was described as generally well tolerated. Patients in the
Alta study were not treated with prophylactic steroids. There was
one case of treatment-related SAE as previously reported
(hypotension and fever 6 hours after SB-525 infusion), and no
similar hypotension event was observed in the three subsequent
patients dosed. ALT and AST elevation occurred in 30% and 10% of
patients, respectively, though no patients experienced ALT elevation
associated with loss of FVIII expression. In the highest dose cohort,
2 patients experienced Grade 1 ALT elevation (above 1.5x baseline)
managed with a tapering course of oral steroids.
? SGMO expects to treat the 5th patient (P11) in the 3E13 vg/kg dose cohort soon, and SGMO and PFE are working on plans to advance SB-525 to a registrational study. PFE is to assume responsibility for SB-525 late-stage development and manufacturing, and transfer of the SB-525 manufacturing process from SGMO to PFE has been initiated.
? SGMO announced that, based on data from the Alta study, the FDA has granted RMAT designation for SB-525 to treat severe hemophilia A. RMAT designation, granted to regenerative medicine therapies with preliminary clinical evidence indicating the potential to address an unmet medical need with a serious condition, includes all the benefits of fast track and breakthrough therapy designation programs, including early interactions with FDA.”
Recent SGMO News
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 06/13/2024 08:42:13 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 06/06/2024 08:26:31 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 06/06/2024 08:25:56 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 06/06/2024 08:25:28 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 06/06/2024 08:24:55 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 06/06/2024 08:24:27 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 06/06/2024 08:23:57 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 06/06/2024 08:23:31 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 06/06/2024 08:23:04 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 06/05/2024 09:10:53 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 05/29/2024 09:18:02 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 05/29/2024 09:15:56 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 05/29/2024 09:00:17 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 05/29/2024 08:57:40 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 05/29/2024 08:54:22 PM
- Form 10-Q - Quarterly report [Sections 13 or 15(d)] • Edgar (US Regulatory) • 05/09/2024 08:04:01 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 05/09/2024 08:01:30 PM
- Sangamo Therapeutics Reports Recent Business Highlights and First Quarter 2024 Financial Results • Business Wire • 05/09/2024 08:01:00 PM
- Sangamo Therapeutics Presents Next-Generation Modular Integrase Technology Engineered to Enable Large-Scale Genome Editing • Business Wire • 05/09/2024 12:05:00 PM
- Sangamo Therapeutics Announces First Quarter 2024 Conference Call and Webcast • Business Wire • 05/02/2024 08:15:00 PM
- Sangamo Therapeutics to Present Neurology-Focused Pre-Clinical Data From Its Epigenetic Regulation, Capsid Delivery and Genome Engineering Platforms at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) • Business Wire • 04/22/2024 08:35:00 PM
- Sangamo Therapeutics Announces Pricing of $24.0 Million Registered Direct Offering • Business Wire • 03/22/2024 12:05:00 PM
- Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results • Business Wire • 03/13/2024 12:05:00 PM
- Sangamo Therapeutics Announces Data From Novel Proprietary Neurotropic AAV Capsid Demonstrating Industry-leading Blood-brain Barrier Penetration and Brain Transduction in NHPs • Business Wire • 03/13/2024 12:01:00 PM
- Sangamo Therapeutics Announces Fourth Quarter and Full Year 2023 Conference Call and Webcast • Business Wire • 03/12/2024 10:30:00 PM
Glidelogic Corp. Becomes TikTok Shop Partner, Opening a New Chapter in E-commerce Services • GDLG • Jul 5, 2024 7:09 AM
Freedom Holdings Corporate Update; Announces Management Has Signed Letter of Intent • FHLD • Jul 3, 2024 9:00 AM
EWRC's 21 Moves Gaming Studios Moves to SONY Pictures Studios and Green Lights Development of a Third Upcoming Game • EWRC • Jul 2, 2024 8:00 AM
BNCM and DELEX Healthcare Group Announce Strategic Merger to Drive Expansion and Growth • BNCM • Jul 2, 2024 7:19 AM
NUBURU Announces Upcoming TV Interview Featuring CEO Brian Knaley on Fox Business, Bloomberg TV, and Newsmax TV as Sponsored Programming • BURU • Jul 1, 2024 1:57 PM
Mass Megawatts Announces $220,500 Debt Cancellation Agreement to Improve Financing and Sales of a New Product to be Announced on July 11 • MMMW • Jun 28, 2024 7:30 AM