Promising Early Phase Trial Results Lead to Orphan Drug Designation for Efineptakin Alfa in Pancreatic Cancer

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The FDA has granted Orphan Drug Designation (ODD) to NeoImmuneTech, Inc’s efineptakin alfa (NT-17) for the treatment of pancreatic cancer. Efineptakin alfa is the only clinical stage long-acting human interleukin-7.¹

The ODD was based on early findings from an ongoing Phase I/II trial (NCT04332653) of the novel therapy in combination with pembrolizumab (Keytruda) for patients with advanced solid tumors, including pancreatic cancer.² Efineptakin alfa is also being evaluated in other Phase I and II trials, having demonstrated the potential to increase T cells and enhance treatment responses in patients with pancreatic cancer and other solid tumors, according to NeoImmuneTech.¹

“We are excited that the FDA granted NT-I7 an ODD in the treatment of pancreatic cancer. This decision adds further credibility to our existing evidence that NT-I7 has the potential to bring a much-needed therapy option to people suffering from pancreatic cancer,” said Luke Oh, PhD, president of NeoImmuneTech, in a press release. “We look forward to continuing our collaboration with FDA, as we explore the therapeutic benefits of combining NT-I7 with other anticancer treatments such as immunotherapies for patients with pancreatic cancer.”¹

Trial data demonstrated that at a median follow-up of 3.53 months among individuals with pancreatic ductal adenocarcinoma (PDAC), the objective response rate (ORR) per RECIST v1.1 criteria was 3.8%, disease control rate (DCR) was 30.8%, median duration of response was 8.7 months, and median time to response (TTR) was 2.8 months. Per iRECIST v1.1 criteria, ORR was 7.7%, DCR was 34.6%, median DOR was 6.1 months, and median TTR was 3.4 months, with a median progression-free survival (PFS) of 6.0 months.

For trial NCT04332653, investigators enrolled patients with relapsed or refractory microsatellite stable (MSS) colorectal cancer (CRC) and PDAC who are naive to treatment with checkpoint inhibitors (CPI); CPI-exposed patients with relapsed or refractory triple-negative breast cancer (TNBC); non–small cell lung cancer (NSCLC), and small cell lung cancer (SCLC).

Patients were administered efineptakin alfa at a dose of 1200 µg/kg intramuscularly every six weeks with 200 mg of Keytruda intravenously every three weeks until disease progression or intolerable toxicity.

The trial’s primary endpoints are analysis of preliminary antitumor activity for the combination as per ORRs by RECIST and iRECIST v1.1 criteria. Secondary endpoints include analysis of DOR, DCR, PFS, and overall survival per RECIST and iRECIST criteria.

Investigators enrolled 106 patients were as of the data cutoff date of April 15, 2022, of whom 81 were found to be evaluable for safety and efficacy.

Among all patients enrolled, median follow-up of 5.13 months, ORR per RECIST v1.1 criteria was 4.9%, DCR was 32.1%, median DOR was 4.4 months, and median TTR was 2.8 months; whereas per iRECIST v1.1 criteria, ORR was 9.9%, DCR was 43.2%, median DOR was 4.4 months, and median TTR was 3.3 months.

Among patients with TNBC in the trial, at a median follow-up of 6.08 months, no patients had responded to treatment. Per RECIST v1.1 criteria, DCR was 14.3% compared with 28.6% per iRECIST v1.1 criteria and median PFS was 5.1 months.

For patients with NSCLC, at a median follow-up of 4.85 months, ORR per RECIST v1.1 criteria was 5.6%, DCR was 33.3%, median DOR was 1.3 months, and median TTR was 3.6 months; whereas per iRECIST v1.1 criteria, ORR was 11.1%, DCR was 66.7%, median DOR was 3.1 months, and median TTR was 3.3 months, with median PFS at 6.0 months. In patients with SCLC, at a median follow-up of 6.08 months, ORR per RECIST v1.1 criteria was 33.3%, DCR was 33.3%, median DOR was 4.1 months, and median TTR was 1.2 months, which were the same as the iRECIST v1.1 criteria, with a median PFS of 9.6 months.

Among patients with MSS CRC, at a median follow-up of 5.13 months, ORR per RECIST v1.1 criteria was 3.7%, DCR was 37.0%, median DOR was 4.6 months, and median TTR was 2.8 months; whereas per iRECIST v1.1 criteria, ORR was 11.1%, DCR was 40.7%, median DOR was 4.6 months, and median TTR was 4.1 months, with a median PFS of 6.7 months.

In terms of safety, adverse events were reported by 74.5% of all patients.

References

1. FDA grants orphan drug designation (ODD) status to NeoImmuneTech’s NT-17 for advanced pancreatic cancer treatment. News release. NeoImmuneTech, Inc. January 30, 2024. Accessed February 5, 2024. https://neoimmunetech.com/news/page01_view.html?number=274

2. Naing A, Mamdani H, Barve MA, et al. Efficacy and safety of NT-17, long-acting interleukin-7 plus pembrolizumab in patients with advanced solid tumors: results from the phase 2a study. J Clin Oncol. 2022;40(suppl 16):2514. doi:10.1200/JCO.2022.40.16_suppl.2514