NP-G1-044 Receives FDA Orphan Drug Designation for Pancreatic Cancer

The FDA has granted orphan drug designation to NP-G2-044 (Prilukae), a novel oral fascin inhibitor, for the treatment of patients with pancreatic cancer.¹

NP-G2-044 is a small molecule inhibitor designed to block tumor metastasis and enhanced immune response.² The agent targets fascin, the primary actin-bundling protein that plays a critical role in tumor cell migration and metastasis. By inhibiting fascin, the drug blocks the motility and invasion of tumor cells and simultaneously activates intratumoral dendritic cells, ultimately leading to the proliferation of CD8-positive T cells.

Preclinical evidence has demonstrated synergistic activity between NP-G2-044 and anti–PD-1 therapy, which enabled the conversion of nonresponsive tumors into responsive tumors. These findings served as the rationale for investigating NP-G2-044 in combination regimens in addition to monotherapy.

“FDA orphan drug designation for our fascin inhibitor represents an important regulatory milestone…and validates [our] scientific and clinical approach in the fight against pancreatic cancer, as it remains one of the most lethal solid tumors with limited therapeutic progress over decades,” Stewart Campbell, chief executive officer of Novita Pharmaceuticals, stated in a news release.¹ “Fascin inhibition offers a novel approach with the potential to enhance antitumor immune activity and improve outcomes for patients with this devastating disease, and we look forward to continuing advancement of NP-G2-044 to address the high unmet need.”

Of note, NP-G1-044 is currently under investigation as both monotherapy and in combination with anti–PD-1 therapy in the phase 1/2 NP-G2-044-P2-01 trial (NCT05023486).^2,3

What is the design of the NP-G2-044-P2-01 trial?

This trial is a multicenter, open-label study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics, and preliminary antitumor activity of NP-G1-044 alone or in combination with an anti–PD-1 therapy in patients with advanced or metastatic solid tumors, including those with documented primary or acquired resistance to anti–PD-(L)1 therapy.³

An estimated total of 140 patients will be enrolled onto the study across 18 locations. Across both arms, eligibility criteria include being at least 18 years of age, having an ECOG performance status of 0 or 1, and having adequate organ and bone marrow function. In the monotherapy arm, patients must have a histopathologically confirmed advanced or metastatic solid tumor for which standard therapies are no longer effective, not tolerated, or for which they are ineligible; and have measurable disease per RECIST 1.1 criteria.

Eligible patients receive NP-G2-044 orally at a daily dose of 1600 mg, 2000 mg, or 2100 mg for each 28-day cycle. In the combination arm, NP-G2-044 is administered along with previously initiated standard-of-care anti–PD-1 therapy.

The study’s primary end point is overall response rate (ORR); secondary end points include progression-free survival, duration of response, disease-control rate, and safety.²

What prior data have read out from the NP-G2-044-P2-01 trial in solid tumors?

Findings from the phase 2 study were reported at the 2025 ASCO Annual Meeting. Patients treated with NP-G2-044 (n = 45) achieved an ORR of 21% (95% CI, 9.0%-38.9%). This included 4 complete responses (CR)—2 of which were pathologic CRs—and 3 partial responses. The disease control rate was 76%, and 55% of patients showed no new metastases during the study. At the time of data presentation, 4 patients were in ongoing treatment, 2 of whom had been receiving treatment for more than 80 weeks.

Regarding safety, no new safety signals were observed with the addition of NP-G2-044 to PD-L1 inhibitors, and there was a lack of cumulative toxicities. Adverse effects were deemed manageable and transient by study investigators, and no dose-limiting toxicities were reported.

References

1. Novita Pharmaceuticals announces FDA orphan drug designation granted to NP-G2-044 for the treatment of pancreatic cancer. News release. Novita Pharmaceuticals. January 12, 2026. Accessed January 26, 2026. https://www.novita-pharm.com/wp-content/uploads/2026/01/Novita-ODD-Press-Release-1.8.26-vUpload.pdf
2. Kasi A, Birrer MJ, Brown JR, et al. Durable responses in ICI-refractory or acquired resistance: Phase 2 study of NP-G2-044 combined with anti-PD-1 therapy. J Clin Oncol. 2025;43(16_suppl):2513-2513. doi:10.1200/jco.2025.43.16_suppl.2513
3. 5. NP-G2-044 as monotherapy and combination therapy in patients with advanced or metastatic solid tumor malignancies. ClincialTrials.gov. Updated January 14, 2026. Accessed January 26, 2026. https://clinicaltrials.gov/study/NCT05023486