IND-Enabling Studies

IND-enabling studies generate the nonclinical data required to support an Investigational New Drug (IND) application and the initiation of first-in-human clinical trials. These studies typically include pharmacology, pharmacokinetics (ADME), toxicology, and manufacturing assessments designed to demonstrate that a therapeutic candidate has an acceptable safety profile for initial clinical evaluation.

Our multidisciplinary scientific and regulatory teams support the design, execution, and documentation of IND programs to ensure alignment with regulatory expectations and efficient progression toward clinical development.

An Investigational New Drug (IND) application is a formal submission to the U.S. Food and Drug Administration (FDA) requesting authorization to begin clinical testing of a new therapeutic candidate in humans. The submission includes comprehensive nonclinical safety data, demonstrating a reasonable basis for expecting that the drug can be administered safely in initial clinical studies.

Key Development Steps

Nonclinical Development
Define the drug target and mechanism of action, design the nonclinical development strategy, and identify required safety studies.

IND-Enabling Studies
Develop analytical methods, manufacture study materials, and conduct pharmacology, pharmacokinetic, and toxicology evaluations.

Regulatory Submission
Compile nonclinical and manufacturing data and submit the IND application to the appropriate regulatory authority.

Early planning is essential, as preparation and execution of IND-enabling studies and supporting manufacturing activities may require 12–18 months. Establishing a clear development strategy early in the program helps streamline timelines and supports successful regulatory submission.

Preclinical GLP Toxicology Studies

Preclinical toxicology studies are a critical component of drug development, providing essential data on the safety profile of therapeutic candidates prior to clinical testing in humans. Conducted under Good Laboratory Practice (GLP) standards, these studies evaluate potential toxicity, identify target organs of toxicity, and help establish safe starting doses for first-in-human clinical trials.

Our toxicology programs are led by multidisciplinary teams of experienced study directors and scientists who work within a global network of AAALAC-accredited preclinical facilities operating under GxP regulations. These capabilities support both in vitro and in vivo toxicology studies designed to characterize the potential human health risks associated with pharmaceuticals, biologics, chemicals, and medical devices.

Successful toxicology studies require collaboration among specialists including toxicologists, pathologists, veterinarians, surgeons, regulatory experts, and technical support staff. By combining mechanistic toxicology expertise with regulatory insight, our teams are able to identify potential risk factors early in development and design studies that support regulatory submissions.

Many of our scientists actively contribute to regulatory and industry working groups, helping shape evolving standards and best practices in toxicology assessment. This involvement ensures that our study designs, methodologies, and regulatory strategies remain aligned with the latest scientific advances and regulatory expectations.

Safety Pharmacology Studies

Safety pharmacology studies are a critical component of IND-enabling programs, designed to identify potential acute or life-threatening effects of novel therapeutic candidates on vital physiological systems. These studies help characterize risks early in development and support informed decision-making prior to clinical testing.

Our safety pharmacology experts support programs beginning in discovery and lead optimization, helping to identify potential safety concerns and reduce development risk before entering the formal preclinical phase. Study design and execution are guided by extensive experience in both stand-alone safety pharmacology studies and integrated repeat-dose toxicology designs.

Core Safety Pharmacology Assessments

Standard safety pharmacology evaluations typically include the core battery required for small molecule therapeutics:

• Central Nervous System (CNS) assessments, including the Functional Observational Battery (FOB) or Irwin screen

• Respiratory function evaluation

• Cardiovascular assessment, including in vitro hERG assays to evaluate the potential risk of QT interval prolongation

When appropriate, these endpoints can also be integrated into repeat-dose toxicology studies to evaluate safety following chronic exposure and assess potential long-term effects. Integrated study designs can help meet regulatory expectations while supporting cost efficiency and the principles of the 3Rs (Replacement, Reduction, and Refinement) in animal research.

Regulatory Alignment

Our global safety pharmacology programs are designed in accordance with International Council for Harmonisation (ICH) guidelines S7A and S7B and provide expert interpretation to support regulatory submissions.

Our teams also incorporate the latest recommendations from ICH E14/S7B Question and Answer guidance, which emphasizes the integration of nonclinical cardiovascular data into clinical risk assessments. In addition, our cardiovascular safety experts actively participate in global industry working groups and contribute to best-practice publications related to in vitro QTc and cardiac safety evaluation.