Pfizer Unveils Delays Across Its Gene Therapy Programs

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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