Hemogenyx Pharmaceuticals PLC on Thursday said the US Food & Drug Administration has accepted its plan to address concerns which sparked a clinical hold on its chimeric antigen receptor T-cell or Hemo-Car-T therapy.
The London-based biopharmaceutical group, which aims to develop new therapies and treatments for deadly blood diseases, submitted an investigational new drug application to the FDA for Hemo-Car-T in May. It was seeking authorisation to begin a phase 1 clinical trial of the therapy for acute myeloid leukemia.
Car-T therapy involves the patient’s T-cells, a type of immune cell, being modified to recognise and kill cancer cells. Hemogenyx said the treatment is ‘more benign and effective’ than chemotherapy.
However in June the regulator placed Hemo-Car-T on clinical hold, due to a splicing deficiency observed during the manufacture of the lentivirus used to produce Car-T cells.
Hemogenyx received a review letter confirming the clinical hold in July, but said it was re-maufacturing the lentivirus having identified the source of the deficiency and developed a method to eliminate it.
The FDA’s review letter included suggestions for improving the treatment’s safety, although this had not contributed to the clinical hold.
Hemogenyx on Thursday said it had replied to the FDA in August with a ‘detailed plan, supported by laboratory tests’ to allow for the clinical hold to be lifted. It has now received confirmation from the FDA that its plan satisfactorily addresses the regulator’s concerns.
‘We are pleased that the FDA has agreed to our plan and preliminary test results to address their concerns regarding our Hemo-Car-T IND application,’ said Chief Executive Officer Vladislav Sandler. ‘We are now working hard to complete the schedule of work set out in the plan and to re-submit the IND as expeditiously as possible in order to move forward with clinical trials of Hemo-Car-T.’
Hemogenyx shares were up 2.0% at 1.68 pence in London on Thursday afternoon.
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