Survival Guide to FDA Inspections: Part 3, Responding to Observations

In the first part of a two-part interview, Paz Vellanki of Precision for Medicine explains what sponsors should bring to early FDA meetings and how manufacturing and assay readiness can delay rare disease trials.

FDA clears the IND for Biolojic Design’s BD200, an AI-designed Trop2/Nectin-4 multibody ADC; Blackstone Life Sciences agrees to provide Pfizer up to $1.5 billion to develop its monthly GLP-1 receptor agonist berobenatide; and enGene Therapeutics plans a year-end BLA for detalimogene voraplasmid in BCG-unresponsive non-muscle invasive bladder cancer.

Detalimogene voraplasmid produced a 56% complete response rate in BCG-unresponsive bladder cancer, with an estimated 43% of responses lasting a year.

FDA has cleared Biolojic Design’s IND for BD200, a bispecific antibody-drug conjugate targeting Trop2 and Nectin-4 whose arms can each bind either target. A global Phase 1 study in solid tumors is expected to begin in the coming weeks.

Blackstone Life Sciences will fund up to $1.5 billion of development costs through 2028 for Pfizer’s monthly GLP-1 receptor agonist berobenatide, in exchange for milestone payments tied to approval and sales. Pfizer keeps full control of the program.

Gene-corrected nula-cel freed all 3 RESTORE patients from transfusions, with sickle hemoglobin below 20% in the 2 followed for over 6 months.

Beam Therapeutics’ base-edited cell therapy risto-cel meets its primary endpoint in the BEACON sickle cell disease trial, Amgen’s CD40L antagonist dazodalibep reduces systemic disease activity in the phase 3 OASIZ-301 Sjögren’s disease trial, and ImvivaBio’s allogeneic CD19/BCMA CAR-T CTA313 sustains lupus remission through 12 months.

Matt Truppo, Richard Jaenisch, and Brian Drapeau examine AI's impact on drug success, human oversight, data governance, and biopharma's future.