RE: RNS: Annual IND report14 Apr 2026 07:55
My understanding is that Relapsed/Refractory Acute Myeloid Leukaemia (R/R AML) is, in general, fatal within months. So, in Phase I oncology trials, it is sadly common for patients to pass away from the underlying cancer itself.
The FDA's primary concern in Phase I is whether the therapy harmed the patient (e.g., severe neurotoxicity or Cytokine Release Syndrome). Even if a patient from Cohort 1 ultimately succumbed to their leukaemia after several months, it does not mean the CAR-T therapy failed its safety mandate, nor does it derail the trial.
If we want proof that the safety data in that report is sound, read the RNS from March 31. Hemogenyx announced that they are officially initiating adult dose escalation and opening a pediatric trial. The FDA and the independent Data Safety Monitoring Board (DSMB) are incredibly strict. If the first three patients had experienced severe, fatal toxicities caused by HG-CT-1, the FDA would have slapped a "Clinical Hold" on the trial immediately. They absolutely would not have permitted Hemogenyx to give the drug to children or double the dose in adults.