AbbVie’s strategy of growing in oncology by focusing on a particular type of targeted therapy is paying off with another FDA approval, this time for a drug developed for an ultra-rare and aggressive form of blood cancer with few treatment options.
The Wednesday regulatory decision for the drug, pivekimab sunirine, covers the treatment of adults who have this cancer, blastic plasmacytoid dendritic cell neoplasm (BPDCN). AbbVie will market its new intravenously infused medicine under the brand name Decnupaz.
BPDCN is a cancer that originates in bone marrow and blood and then progresses to organs, the central nervous system, and the skin. Deep purple skin lesions are common in BPDCN patients, who are typically age 60 or older. The disease typically affects more men than women.
Standard BPDCN has been chemotherapy. Research into this cancer found that BPDCN cells overexpress a protein called CD123, opening the door to targeted therapies addressing that protein. In 2018, Stemline Therapeutics (now a Menarini Group subsidiary) received FDA approval for Elzonris, a fusion protein that targets CD123. Targeted therapy can be followed by a stem cell transplant. But even with these treatments, relapse is common.
FDA approval of Decnupaz brings BPDCN patients a new targeted therapeutic option. This AbbVie drug is an antibody drug conjugate (ADC) designed to target CD123. The drug was evaluated in an open-label Phase 1/2 study that enrolled adults whose BPDCN was positive for the target protein. In the cohort of 33 patients who were treatment naïve, results showed that 69.7% (23 patients) achieved complete remission or clinical complete remission with a median follow-up of 21.5 months. The median duration of this response was 9.7 months. In the cohort of 51 patients with relapsed or refractory BPDCN, 15.7% (eight patients) achieved complete remission or clinical complete remission with a median follow-up of 24.1 months. The median duration of this response was 9.2 months.
“This approval delivers a new option for treating BPDCN and demonstrates our determination to drive meaningful advancements for patients affected by difficult-to-treat cancers,” AbbVie’s Roopal Thakkar, executive vice president, research and development, chief scientific officer, said in a prepared statement.
The most common adverse reactions reported in the BPDCN clinical trial included edema, fatigue, muscle pain, hemorrhage, and infusion-related reactions. Decnupaz’s label carries a black box warning for veno-occlusive disease, a severe form of liver toxicity that can become fatal. The label instructs clinicians to monitor for signs of this complication and to conduct tests to assess liver health before each dose of the intravenously infused drug.
Decnupaz came to AbbVie as part of the $10 billion acquisition of ADC specialist ImmunoGen. The centerpiece of that 2023 deal was Elahere, which under ImmunoGen became the first ADC approved for treating ovarian cancer. At the time of the buyout, Decnupaz had reached the Phase 2 portion of its Phase 1/2 study.
AbbVie’s own ADC research has also borne fruit. Last year, AbbVie’s Emrelis received accelerated FDA approval in non-small cell lung cancer. This ADC targets a protein called c-Met. AbbVie also has high hopes for telisotuzumab adizutecan, or Temab-A, which AbbVie describes as a next-generation c-Met-directed ADC. Temab-A’s clinical development program spans a range of solid tumors including gastroesophageal adenocarcinoma and non-small cell lung cancer. The most advanced clinical trial is a Phase 3 study in colorectal cancer. At the upcoming annual meeting of the American Society of Clinical Oncology in Chicago, AbbVie plans to present early clinical data for Temab-A in ovarian cancer and head and neck cancer.
Meanwhile, AbbVie may find additional applications for Decnupaz as a treatment for other types of blood cancer. A Phase 2 test of this ADC is ongoing in acute myeloid leukemia.
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