“BREAKING NEWS - Sangamo (SGMO) - SB-525 Looks Highly Competitive At ISTH; FDA Grants RMAT & Pfizer Planning Registration Trials - With the updated ALTA study presented at ISTH, Sangamo has firmly made its case for SB-525 as a leading hemophilia A gene therapy treatment. The FDA appears to agree - it has granted the Company RMAT status - the first one given for a hem A gene therapy. Partner Pfizer (included in the press release headline) will be taking over the program as it heads into Phase III clinical trials. REITERATE BUY........
Efficacy - Fast Acting, Consistent & Durable Responses To Date - The two original 3E13 patients have been treated for 19 and 24 weeks, respectively (as of 5/30/19). Two more patients have since been treated at the higher dose (as per the extension cohort) and are showing the same rapid response in Factor VIII activity to SB-525 as the first two - hence 4 subjects have now been treated at the “3e13” dose. Since the cutoff date, one of those two (subject 9) has also since attained normal Factor VIII levels by week 7. As the chart above shows, Factor VIII levels of the first two have stayed well in the “normal” range with no diminution, while the other two appear on their way, too. As a comparison, it took ~16 weeks for BMRN’s ValRox subjects to reach normal FVIII levels versus 7 weeks for SB-525. And the ValRox normal levels reached were below that of SB-525, too.
No Spontaneous Bleeds - In two additional tables below, at the high dose there has been no bleeding episodes in any of the 4 patients after 3 weeks post-treatment (the fourth had yet to reach the >3 weeks period at the cutoff).
No Need For FVIII Infusions - Prophylactic Factor VIII usage in the 3e13 dose, also went to zero after the >3 week treatment period. The prophylactic FVIII regimen of the four subject prior to SB-525 treatment was an infusion either every other day, every 3 days or every 4 days.........
Safety - Pretty Clean - SB-525 was generally well tolerated. Unlike other gene therapies for hem A, patients in the Alta study were not treated with prophylactic steroids. One treatment-related serious adverse event (SAE) was reported. This patient experienced hypotension and fever six hours after completion of SB-525 infusion and this fully resolved with treatment and the patient was discharged as planned within 24 hours. No similar hypotension event was observed in the three subsequent patients dosed. Adverse events observed in 10% (n=1) or more patients included: increased alanine aminotransferase (30%) and aspartate aminotransferase (10%), pyrexia (30%), fatigue (10%), hypotension (10%), myalgia (10%), and tachycardia (10%). No patients treated with SB-525 have experienced an alanine aminotransferase (ALT) elevation associated with a loss of Factor VIII expression. In the 3e13 vg/kg cohort, two subjects experienced a transient grade 1 ALT elevation (>1.5 x baseline) managed with a tapering course of oral steroids. This safety profile suggests that SB-525 could be the cleanest of the three (ValRox, SPK-800, SB-525).....
FDA Grants SB-525 RMAT Status - Based on the accumulating results from the Alta study, the FDA has granted regenerative medicine advanced therapy (RMAT) designation for SB-525 gene therapy to treat severe hemophilia A. RMAT designation is granted to regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition, for which preliminary clinical evidence indicates that the medicine has the potential to address an unmet medical need. The RMAT designation includes all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with FDA. In our view, the RMAT designation will accelerate the clinical and approval timelines for SB-525.
PFE Is About To Take Over - Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Diseases Research Unit said, “We are encouraged by the initial clinical data suggesting safety, tolerability, and efficacy of SB-525 and are beginning preparations, including manufacturing, to potentially advance into a registrational study. We are also encouraged by our interactions with regulators and by the FDA’s recent RMAT designation. If FVIII levels are sustained, and patients continue to have no bleeding episodes and remain off factor replacement therapy, we believe that this gene therapy may potentially represent a transformative treatment paradigm for severe hemophilia A.” The PR also mentions that Pfizer will soon assume responsibility for SB-525 late-stage development and manufacturing.Transfer of the SB-525 manufacturing process from Sangamo to Pfizer has been initiated.
SGMO - Now A Bonafide HEM A GT Player - Before ISTH, SGMO was off of investors’ radar screens for hem A gene therapy. While the number of patients is small and time on SB-525 is still relatively short compared with BMRN and ONCE (acquired by Roche recently for $5 billion), the new updated data, along with the RMAT designation and PFE’s increased participation in the program has no doubt placed the Company among the leaders in the race for blockbuster hemophilia A gene therapy treatments.
SMGO is a BUY under 20 with a TARGET PRICE of 30”
