Driving drug development with precision and expertise
Toxicology & Safety Evaluation
Overview
General Toxicology Studies
Medicilon offers a full suite of GLP and non-GLP toxicology studies to support both regulatory submissions and exploratory research. Our advanced platforms, experienced toxicologists, and global regulatory alignment ensure that your preclinical safety data meets the highest international standards.
Acute, Subchronic, and Chronic Toxicology in both rodent and non-rodent species. |
Single and Multiple Dose Studies to determine threshold and duration effects. |
Local Tolerance Assessments for subcutaneous, intramuscular, ophthalmic, and inhalation routes. |
Toxicokinetics (TK) to study absorption, distribution, metabolism, and excretion (ADME). |
Safety Pharmacology targeting CNS, cardiovascular, and respiratory systems. |
Core services
Specialized Toxicology Services
Beyond general tox, Medicilon’s specialized testing capabilities support complex biologics, advanced modalities, and long-term indications
Genotoxicity
Ames test, chromosomal aberration, micronucleus assays
Reproductive & Developmental Toxicity
Fertility, embryofetal development (EFD), peri/postnatal studies
Carcinogenicity
Long-term studies across full animal lifespans
Immunogenicity studies
Immune profiling, complement testing, ADA analysis for biologics and cell therapies
Advanced Pathology
Histopathology, IHC, tissue cross-reactivity (TCR), specialized staining
Special Routes
Safety evaluation for inhalation and ophthalmic drugs
Bioanalytical & Clinical Pathology
Biomarkers, systemic exposure, toxicodynamic assessments
Case Study
The flagship example of Medicilon’s specialized toxicology services.
Representative IHC images show specific detection of CD3, CD4, CD163, and PDL1 immune markers in mouse tissues, demonstrating Medicilon’s expertise in advanced pathology and biomarker validation for complex drug modalities.
International Quality & Regulatory Compliance
Our GLP-compliant studies adhere to international regulatory requirements
Our experienced study directors and project managers coordinate all aspects of toxicology studies, from in-life testing to data analysis. This reduces complexity, minimizes delays, and accelerates IND filing timelines.
- GLP-certified and AAALAC-accredited facilities
- Successful track record with FDA and NMPA GLP inspections
- SEND-compliant data management using Instem's submit™ platform
- OECD and ICH regulatory compliance
IND-Ready: eCTD Modules & SEND Compliance
The electronic Common Technical Document (eCTD) is the standard format for IND filings required by the FDA (Reference Link) and global regulators. It consists of five key modules:
Regional Information
Administrative documents specific to the region, including forms, cover letters, and product labeling.
Quality Information (CMC)
Detailed data on drug substance and product manufacturing, controls, and quality.
Clinical Study Reports
Comprehensive results and data from human clinical trials.
CTD Summaries
High-level overviews of nonclinical and clinical data to support regulatory review.
Nonclinical Study Reports
Full toxicology and pharmacology study reports from animal testing.
SEND Compliance Matters
The FDA mandates nonclinical studies to be submitted in SEND format, which:
- Standardizes data structure for easier regulatory review
- Reduces errors in large datasets
- Speeds up the IND assessment process
Our bioinformatics team leverages Instem’s submit™ platform to generate SEND-compliant datasets while compiling all documentation into an eCTD-ready format, ensuring:
- Organized and FDA-compliant submissions
- Comprehensive Safety Profile
- Faster and smoother IND approval
Study Design & Protocol Development
Strategic Approach: Non-GLP and GLP Toxicology for IND Success
Medicilon provides customized study designs aligned with ICH, OECD, FDA and NMPA guidelines, including:
- Dose selection strategies, animal models, and study endpoints
- Specialized assessments such as inhalation, ophthalmic, and immunogenicity studies
Toxicology Execution
- Non-GLP range-finding studies to determine safety thresholds
- Definitive GLP toxicology studies for pivotal assessments
- Integrated toxicokinetic analysis for comprehensive safety profiling
Data Analysis & Regulatory Reporting
- Detailed, regulatory-compliant study reports
- Robust quality assurance with on-site inspections and audits
- SEND-compatible datasets for seamless IND submission
IND Submission Support
- Compilation of pivotal study reports and regulatory documentation
- Expert regulatory consultation to address FDA and NMPA queries
Proven Success
Medicilon’s IND-Focused Toxicology Case Studies
Explore how Medicilon’s IND-focused toxicology studies have helped clients overcome regulatory hurdles, accelerate timelines, and advance drug candidates with confidence. Each case highlights our scientific expertise and strategic approach to IND success.
Genotoxicity IND-Enabling Case Study
Negative Ames, chromosomal aberration, and micronucleus assays enabled fast-track submission.
1）Executive Summary Table
This summary highlights Medicilon’s comprehensive genetic safety package for IND: in vitro and in vivo assays for regulatory compliance, with all results supporting safe first-in-human entry.
2. Enhanced Ames Test Details
Medicilon’s enhanced Ames test uses a broad panel of Salmonella strains and metabolic activation systems to rigorously evaluate mutagenic potential. Full dose ranges and controls establish robust assay sensitivity for regulatory review.
a) Strain Table – Genotype and Detection Profile
（Overview of Salmonella strains and their mutation detection capabilities used in Ames testing.）
b) Methodology, Dose, and Content – Assay Design and Controls (-S9)
（Assay setup including controls and dose levels for genotoxicity assessment in the absence of metabolic activation.）
c) Study Design with Metabolic Activation – Assay Design and Controls (+S9)
（Assay setup for genotoxicity screening with metabolic activation, highlighting tissue source and dose response.）
3. CA Test in CHL Cells
The chromosomal aberration (CA) assay evaluates structural chromosome damage in CHL cells under a range of dosing and control conditions, ensuring complete genetic risk characterization.
4. Bone Marrow MN Test in Rats
Medicilon’s in vivo micronucleus test in rats provides critical data on chromosomal damage potential. Dose-ranging, controls, and stringent sampling schedules ensure reliable genotoxicity assessment.
Small Molecule AAA:
Comprehensive IND-enabling GLP studies in rats and dogs, PK/PD assessment, and genetic toxicology supported Phase I entry.
AAA’s IND-enabling package covered PK, tissue distribution, binding assays, safety pharmacology, genotoxicity, and GLP repeat-dose studies in rodents and dogs. The package flow was designed to support regulatory submission and Phase I entry.
Study Design Flow
Results
Medicilon’s small molecule AAA toxicology package included acute, repeat-dose, and GLP-compliant studies in rats and dogs, integrating full clinical pathology, toxicokinetics, and advanced histopathology endpoints. The dog GLP study established NOAEL, characterized reversible metabolic and tissue changes, and enabled regulatory confidence for IND submission in liver disease indication.
Special Modalities
Bispecific mAbs, ADCs, siRNAs, and X-T cell therapies – all validated in IND-enabling studies.
Why work with Medicilon for your Safety Assessment Service needs
Medicilon delivers fast, reliable IND-focused toxicology services backed by global regulatory expertise, advanced facilities, and end-to-end support, from study design to SEND-ready eCTD submissions. Our proven track record with FDA and NMPA approvals helps pharmaceutical and biotech companies accelerate timelines and move drug candidates forward with confidence.
Proven Regulatory Success
Extensive experience with FDA, NMPA, and OECD inspections
Fast Turnaround
Large-scale facilities enable accelerated study timelines
Expert Consultation
Toxicologists and regulatory specialists guide study execution
End-to-End Services
From non-GLP pilot studies to final IND submission support
Frequently Asked Questions
Toxicology FAQ
What studies are essential before first-in-human trials?
Single/repeat-dose tox (rodent & non-rodent), genotoxicity (Ames, CA, MN), safety pharmacology (CNS, CVS, Resp), reproductive/developmental tox, local tolerance, and TK.
GLP vs. non-GLP - what's the difference?
GLP is mandatory for IND filings; non-GLP is for dose selection and early-phase insights.
How are animal species selected?
Based on pharmacological relevance – typically one rodent and one non-rodent species.
What’s the value of toxicokinetic studies?
TK studies correlate exposure to toxic response and inform risk assessment.
When is carcinogenicity required?
For chronic use drugs, as guided by regulatory risk analysis.
Are injection site reactions evaluated?
Yes, especially in SC/IM products; pathology findings often parallel clinical outcomes.
Partner with Medicilon for your next breakthrough
Medicilon’s comprehensive safety assessment services offer more than just data, they provide a pathway to innovation and compliance. Whether you are developing small molecules or biologics, we ensure safety, precision, and compliance at every step of your drug development process.
