LAVA Therapeutics gets FDA orphan drug status for LAVA-051 in CLL

LAVA Therapeutics has been granted orphan drug designation (ODD) in chronic lymphocytic leukemia (CLL) from the US Food and Drug Administration (FDA) for LAVA-051, its CD1d-targeted Gammabody.

According to the US-based biotech company, LAVA-051 is a bispecific antibody that triggers the immune system to target cancer cells that express CD1d through the selective activation of Gamma Delta T cells as well as type 1 natural killer (NKT) cells.

Currently, LAVA Therapeutics is in the process of enrolling participants in a multi-center, open-label phase 1/2a clinical trial in relapsed and/or irreparable CLL, multiple myeloma (MM), and subsequently, in acute myeloid lymphoma (AML).

The NCT04887259 clinical trial was launched in July 2021. It is intended to test the safety, tolerability, pharmacokinetics, and pharmacodynamics, as well as preliminary antitumor activity and immunogenicity of LAVA-051.

Results from the phase 1 dose escalation part of the study are targeted to be released in the first half of 2022, with the top line clinical data from the phase 2a expansion cohorts scheduled for the second half of next year.

Stephen Hurly — president and CEO of LAVA Therapeutics said: “We are excited to receive our first orphan drug designation from the FDA for LAVA-051, our most advanced product candidate from our off-the-shelf Gammabody platform that is designed to unlock the full anti-cancer potential of this specialized effector cell population.

“This designation will be helpful in enhancing our communication with the FDA on our development of LAVA-051. We are grateful to the FDA for highlighting the need for new and improved therapies to address the unmet needs in CLL.”