Niiki Pharma Inc. announced interim results from the ongoing phase I clinical trial of its lead product, NKP-1339. NKP-1339 is a first-in-class transferrin targeted small molecule that down-regulates GRP78, a key regulator of mis-folded protein processing and a tumour survival factor.
NKP-1339 phase I interim data.

The phase I trial is conducted in patients with metastatic solid tumours resistant to standard therapies. Previous to enrollment in the trial, all patients had received multiple standard therapies and exhibited disease progression on their last treatment. Of the first 24 patients enrolled, six patients (25%) exhibited anti-tumour activity, demonstrated by disease stability and/or tumour regression for at least 12 weeks.
Of the responding six patients, one patient with carcinoid tumour, a non-pancreatic neuroendocrine tumour (non-pNET), has tumour regression and continues on NKP-1339 therapy for more than 70 weeks. The five other patients have experienced stable disease of up to 24 weeks with NKP-1339 treatment. These patients have the following tumour types: one gastrinoma non-pNET, one colorectal cancer, two non-small cell lung cancers and one cancer of unknown primary.

NKP-1339 treatment has been well tolerated to date with mild manageable side effects. The most common drug-related side effects are grade 1-2 fever and mild flu-like symptoms, which can be prevented with standard medications. The maximum tolerated dose has not been reached and NKP-1339 dose escalation continues.

The trial is being led by Dr Daniel Von Hoff, Virginia G. Piper Cancer Centre Clinical Trials at Scottsdale Healthcare in partnership with Translational Genomics Institute (TGen), and Dr Howard Burris, director of Drug Development at the Sarah Cannon Research Institute (SCRI).

“We need new treatments for patients with non-pNET. The NKP-1339 anti-tumour activity observed in these two non-pNET patients is significant. This activity, together with mild-side effect profile, makes NKP-1339 a potentially promising new treatment for this disease," said Dr. Daniel Von Hoff.

“It is gratifying to see someone whose tumour has been resistant to other therapies do well with this promising investigational therapy. The patient has received NKP-1339 for more than a year and continues to benefit,” added Dr Howard Burris.

“We are pleased by these interim results demonstrating the single agent anti-tumour activity of NKP-1339 in patients with advanced cancers refractory to standard treatments,” said Dr Angela Ogden, chief medical officer for Niiki Pharma Inc.

“The preliminary results of this ongoing trial, showing the anti-tumour activity and the safety profile of NKP-1339, support our preclinical studies that the drug targets tumours selectively and is active against different tumours. We are delighted to see years of research come to fruition in collaboration with Niiki Pharma's team,” said Professor Keppler, University of Vienna, inventor of NKP-1339.

NKP-1339 is a first-in-class transferrin targeted ruthenium-based anti-cancer compound. Transferrin receptor is significantly over-expressed in many tumour types. The intracellular target pathways of NKP-1339 include down-regulation of GRP78, a key regulator of mis-folded protein processing and a tumour survival factor.  GRP78 is the cause of resistance in many tumour types. In preclinical studies, NKP-1339 has demonstrated activity against multiple tumour types, including those resistant to other anti-cancer agents, including platinum-containing agents, anthracyclines and anti-tubulins.

NKP-1339 was invented by Professor Bernard K. Keppler, currently the Dean of the Faculty of Chemistry at University of Vienna, Austria, and Head of the Research Platform "Translational Cancer Therapy Research", a multi-disciplinary collaboration between University of Vienna and the Medical University of Vienna.

Niiki Pharma Inc. is an oncology therapeutics development company, which is focused on development of targeted first-in-class treatments for cancer.