Intezyne Technologies Initiates First-In-Human Trials for Topoisomerase 1 Inhibitor IT-141
(NCT03096340)


Program overview

IT-141 leverages Intezyne's proprietary nanoparticle technology (IVECT) to deliver SN-38, an extremely potent topoisomerase-1 (TOP-1) inhibitor, directly to tumors.

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ABOUT Topoisomerase-1 (TOP-1) Inhibitors

Validated anti-cancer target in a wide variety of oncology indications:

Pfizer's Camptosar® (irinotecan)

  • FDA-approved for metastatic colorectal cancer (mCRC) in 1996
  • Irinotecan metabolization into SN-38 is unpredictable due to patient-to-patient variability
  • $969M peak sales in 2008
  • Remains on the WHO Model List of Essential Medicines
 

Merrimack's Onivyde® (liposomal irinotecan)

  • FDA-approved for gemcitabine-resistant advanced (metastatic) pancreatic cancer in 2015
  • Still suffers from irinotecan-based variability
  • Acquired January 2017 by Ipsen for $1B ($0.5B upfront)

 

Novartis's Hycamtin® (topotecan)

  • FDA-approved for ovarian and small cell lung cancer (SCLC)
  • $1.7B peak sales
 

ABOUT Irinotecan

SN-38 is a potent TOP-1 inhibitor

  • SN-38 is water-insoluble, meaning it cannot be directly administered

Irinotecan is a water-soluble produg that metabolizes into SN-38 once administered

  • Metabolizes into SN-38 once administered, but somewhat unpredictably
Mathijssen, R.H., et. al., Clin. Cancer Res., 2001, 7, 2182-2194.

Mathijssen, R.H., et. al., Clin. Cancer Res., 2001, 7, 2182-2194.

 

ABOUT Colorectal Cancer

8% of cancer (135,000), 8.4% (50,000) of cancer deaths

  •  4th most prevalent cancer; 2nd leading cause of cancer mortality
    • 5-year survival rate of 65% overall; 11% for metastatic (Stage IV) disease
  • Limited therapeutic innovation in past decade
  • Eligible for Breakthrough Therapy Designation
     

IT-141 Development Rationale

  • Camptosar® (irinotecan), which metabolizes inefficiently into SN-38, is one of the few effective treatments for colorectal cancer
    • Still widely used in the treatment of colorectal cancer, despite being off-patent
    • Used off-label in the treatment of breast, gastric, lung, and pancreatic cancers
  • IT-141 patients should be able to tolerate substantially higher doses of SN-38, resulting in significantly improved patient outcomes
    • Potential to treat irinotecan-refractory patients
 

preclinical Efficacy in hct116 (colorectal) xenograft model

Bakewell, S.J.; et. al., Nanomedicine: Nanotechnology, Biology, and Medicine, 2017, 13, 1353-1362.

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Phase 1 Clinical Trial

Safety and Pharmacokinetic Study of IT-141 in Monotherapy in Patients With Advanced Cancer
(NCT03096340)

Phase 1 open-label, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of IV-infused IT-141 in patients with recurrent or refractory solid tumors

Study Design


2Q17 through 3Q18

Study Period


Primary – Safety, tolerability, dose-limiting toxicity (DLT) and maximum tolerated dose (MTD)
Secondary – To determine tumor response by RECISTv1.1, estimate pharmacokinetic (PK) parameters, and explore imaging and pharmacodynamic endpoints which may be of use in the further development

Endpoints


Mary Crowley Cancer Center, Dallas, TX
Seattle Cancer Care Alliance, Seattle, WA

Trial Sites


John Nemunaitis, M.D., Mary Crowley Cancer Center
Kit Wong, M.D., Seattle Cancer Care Alliance

Lead Investigators


Up to 40

No. of Participants


Intravenous

Administration


 

Phase 1 Trial Design

Dose-Escalation

Accelerated '3+3' Design