Myeloma treatment HDP-101 put on FDA fast track
Heidelberg Pharma drug currently in Phase 1/2 clinical trial
The U.S. Food and Drug Administration (FDA) has granted fast track designation to HDP-101 (pamlectabart tismanitin), a multiple myeloma therapy currently in early clinical testing.
The FDA gives this designation to promising investigational therapies that, based on early data, have the potential to address unmet medical needs in the management of serious diseases. The designation aims to speed the development of new medicines that are urgently needed. As part of the designation, the therapy’s developer Heidelberg Pharma will get access to perks like more frequent communication with the FDA during the drug development process.
“The FDA’s granting of Fast Track Designation is fantastic news for Heidelberg Pharma and underscores the potential of HDP-101 for the treatment of severely ill and heavily pretreated patients,” Andreas Pahl, PhD, CEO of Heidelberg, said in a company press release.
The designation was supported by preclinical experiments as well as data from an ongoing Phase 1/2 clinical trial (NCT04879043) testing the safety of HDP-101 in adults with myeloma whose disease has come back or failed to respond to prior treatment. Pahl said the designation “will support our efforts to advance [HDP-101] efficiently toward patients with multiple myeloma who continue to face significant unmet medical needs.”
Myeloma is a type of blood cancer marked by the uncontrolled growth of plasma cells, which are immune cells that produce antibodies. The cancer grows in bone marrow — the spongy tissue inside bones where new blood cells are made. Myeloma treatments are available, but they aren’t always successful at eradicating the disease.
Antibody therapy targets BCMA protein
HDP-101 belongs to a class of medicines called antibody-drug conjugates (ADCs). These medicines work by attaching a toxic molecule to an antibody that targets a protein on cancer cells. When the antibody sticks to its target on the cancer cell, it delivers the toxic molecule, killing the cell.
The therapy uses an antibody targeting BCMA, a protein that’s expressed by plasma cells. The antibody is attached to a toxic molecule called amanitin, which kills cells by blocking a protein called RNA polymerase II, a key part of the cellular machinery that reads DNA to make new proteins.
According to Heidelberg, other ADCs for myeloma use toxins that target DNA or the molecular machinery that cells use to divide, so targeting RNA polymerase II is a novel approach for this type of treatment for the disease.
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