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Case Studies

Project Background and Strategic Question

Successfully Waived a Thorough QT/QTc Study

The client was developing a small molecule drug for treatment of multiple different types of seizures. Phase 3 registration trials were ongoing, but the need for a thorough QT/QTc (TQT) study, as listed in the Food and Drug Administration (FDA) guidance for industry (https://www.fda.gov/media/71372/download), was not known. Because a TQT study is expensive and time-consuming, the question was whether the study could be waived based on the available phases 1 and 2 clinical data.

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Project Background and Strategic Question

Determination of Dose Range for the First-in-Human Study

The client was developing a molecule for the potential treatment of a pancreatic cancer and solid tumors with multiple mutations. A series of nonclinical studies were conducted to support an Investigational New Drug Application (IND). For the IND application, the client needed an investigational plan for a first-in-human study (FIH). However, the selection of starting dose and dose range in the FIH study was a challenge: choosing a narrow dose range might not identify a recommended phase-2 dose (RP2D), whereas selecting a wider dose range would be an expensive and time-consuming addition to the study objectives for the RP2D.

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Project Background and Strategic Question

Assessment of Drug-Drug Interaction Potential

The client was developing a molecule for the potential treatment of a pancreatic cancer and solid tumors with multiple mutations. A series of nonclinical studies were conducted to support an Investigational New Drug Application (IND). For the IND application, the client needed an investigational plan for a first-in-human study (FIH). However, the selection of starting dose and dose range in the FIH study was a challenge: choosing a narrow dose range might not identify a recommended phase-2 dose (RP2D), whereas selecting a wider dose range would be an expensive and time-consuming addition to the study objectives for the RP2D.

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Project Background and Strategic Question

PK Exposure-Response Analyses for New Drug Application

Patisiran (ONPATTRO®) is a member of a new class of drugs known as small interfering ribonucleic acid (siRNA) treatment. Patisiran is comprised of a novel siRNA, formulated in a lipid nanoparticle (LNP), and developed for treating hereditary transthyretin-mediated (hATTR) amyloidosis. The patisiran dose in the phase-3 APOLLO trial (patients with hereditary transthyretin amyloidosis) was 0.3 mg/kg, given intravenously every 3 weeks.

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Project Background and Strategic Question

Modeling and Simulation for Subcutaneous Dose Selection

Tocilizumab is a humanized monoclonal antibody that inhibits interleukin-6 (IL-6) from binding to soluble interleukin-6 (sIL-6R) and membrane-expressed forms of its receptor, thus inhibiting the IL-6–mediated signaling cascade that brings about the proinflammatory activity of the cytokine. Tocilizumab was approved in many countries for the treatment of patients with moderate to severe active rheumatoid

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