FDA News

T-cell therapy receives FDA’s orphan drug status for soft tissue sarcoma

Elliot Norry, MD, FACP
Elliot Norry

The FDA has granted orphan drug designation to ADP-A2M4, an investigational specific peptide-enhanced affinity receptor T-cell therapy, for the treatment of soft tissue sarcomas.

ADP-A2M4 (Adaptimmune) is an autologous, genetically modified SPEAR T-cell therapy that targets the MAGE-A4 antigen on the surface of cancer cells.

Certain soft tissue sarcomas express MAGE-A4 at high levels and uniformly across tumors — which makes them a good candidate for SPEAR T cells that are engineered to target MAGE-A4-positive cancer cells, according to Elliot Norry, MD, FACP, interim chief medical officer for Adaptimmune.

Norry explained that his company is currently conducting a phase 2 trial in two types of soft tissue sarcoma: synovial sarcoma and myxoid/round cell liposarcoma (MRCLS).

“We have seen success in treating patients with synovial sarcoma and MRCLS with our SPEAR T cells that by far outweighs any approved therapy for these indications,” he told Cell Therapy Next.

“The treatments available to these patients usually involve harsh chemotherapy, radiation and surgical amputations,” he added. “We have seen patients with sarcoma respond to SPEAR T cells with large bulky tumors disappearing and patients able to recover a quality of life that they did not have while receiving harsh treatments. We believe that our SPEAR T cells offer a viable and much-needed treatment for these patients with few other options.”

ADP-A2M4 is based on Adaptimmune’s proprietary SPEAR platform that enables it to engineer T cells to target multiple antigens that are expressed on the surface of cancer cells via T-cell receptors.

Norry said Adaptimmune has used its SPEAR platform to create a pipeline of T-cell receptors, or TCRs, that can target different types of cancers.

“When we find patients who are eligible for our trials, we take a patient’s own T cells and then put our specific engineered TCR into the patient’s T cells and then transfer these T cells back into the patient. Once these SPEAR T cells are back in the patient — they work to seek out and target cancer cells,” he explained.
“We are committed to bringing our ADP-A2M4 SPEAR T cells to market in 2022 to help patients living with rare and deadly sarcomas who have few other options,” Norry told Cell Therapy Next. “In addition, we are working diligently with teams of expert scientists to ensure we meet the needs of [patients with] cancer with a broad range of difficult-to-treat solid tumors, and we are currently enrolling trials with next-generation technologies to achieve this goal.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

Elliot Norry, MD, FACP
Elliot Norry

The FDA has granted orphan drug designation to ADP-A2M4, an investigational specific peptide-enhanced affinity receptor T-cell therapy, for the treatment of soft tissue sarcomas.

ADP-A2M4 (Adaptimmune) is an autologous, genetically modified SPEAR T-cell therapy that targets the MAGE-A4 antigen on the surface of cancer cells.

Certain soft tissue sarcomas express MAGE-A4 at high levels and uniformly across tumors — which makes them a good candidate for SPEAR T cells that are engineered to target MAGE-A4-positive cancer cells, according to Elliot Norry, MD, FACP, interim chief medical officer for Adaptimmune.

Norry explained that his company is currently conducting a phase 2 trial in two types of soft tissue sarcoma: synovial sarcoma and myxoid/round cell liposarcoma (MRCLS).

“We have seen success in treating patients with synovial sarcoma and MRCLS with our SPEAR T cells that by far outweighs any approved therapy for these indications,” he told Cell Therapy Next.

“The treatments available to these patients usually involve harsh chemotherapy, radiation and surgical amputations,” he added. “We have seen patients with sarcoma respond to SPEAR T cells with large bulky tumors disappearing and patients able to recover a quality of life that they did not have while receiving harsh treatments. We believe that our SPEAR T cells offer a viable and much-needed treatment for these patients with few other options.”

ADP-A2M4 is based on Adaptimmune’s proprietary SPEAR platform that enables it to engineer T cells to target multiple antigens that are expressed on the surface of cancer cells via T-cell receptors.

Norry said Adaptimmune has used its SPEAR platform to create a pipeline of T-cell receptors, or TCRs, that can target different types of cancers.

“When we find patients who are eligible for our trials, we take a patient’s own T cells and then put our specific engineered TCR into the patient’s T cells and then transfer these T cells back into the patient. Once these SPEAR T cells are back in the patient — they work to seek out and target cancer cells,” he explained.
“We are committed to bringing our ADP-A2M4 SPEAR T cells to market in 2022 to help patients living with rare and deadly sarcomas who have few other options,” Norry told Cell Therapy Next. “In addition, we are working diligently with teams of expert scientists to ensure we meet the needs of [patients with] cancer with a broad range of difficult-to-treat solid tumors, and we are currently enrolling trials with next-generation technologies to achieve this goal.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

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