- Setbacks have resulted in delays across all three of Pfizer Inc’s PFE gene therapy programs, the Company revealed in its Q3 conference call.
- Pfizer slammed breaks on its Sangamo-partnered Phase 3 hemophilia A study after some patients reported high Factor VIII levels, an essential blood-clotting protein.
- Multiple patients in Pfizer’s hemophilia A trial had Factor VIII levels over 150%, though the Company has not said how many.
- To date, no patients have experienced thrombotic events, and some are being treated with oral anticoagulants to reduce the risk.
- Pfizer halted dosing until it could amend the protocol to provide guidelines for managing elevated Factor VIII levels.
- Separately, Pfizer said, “recent interactions with the FDA” have led it to decide against conducting interim analyses for both its hemophilia A and B studies.
- Instead, the Company will wait for complete analyses based on at least 50 participants in the hemophilia A study and 40 participants in the hemophilia B study, pushing readout timings.
- While Pfizer anticipated readouts in 2022 for its hemophilia B and A programs, it is now looking at Q1 2023 readout for hemophilia B.
- Pfizer is also expecting a delay in its pivotal DMD study, which is expected to read out in 2023.
- Three cases of muscle weakness, two of which included heart inflammation, forced the trial amendment.
- Pfizer believes that an immune response against the mini-dystrophin protein caused these cases.
- All patients were treated with higher doses of steroids.
- Related: Pfizer More Than Doubles Earnings, Sales In Q3; Forecasts $36B From COVID-19 Vax Sales In FY21.
- Price Action: PFE shares closed higher by 4.15% at $45.45 on Tuesday.
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