Pharmacology Guide

In drug discovery research, the success rate from compound screening (exploratory stage) to approval is said to be 1 in 30,000. During the period from 1991 to 2000, 30% of compounds were dropped (rejected) due to efficacy issues. Therefore, entrusting the work to highly specialized contract research organizations (CROs) with advanced technical capabilities can be the key to overcoming this challenging situation.

The importance of pharmacological and efficacy testing in drug discovery business

In the drug discovery business, pharmacological studies are extremely important. This is because approximately 30% of projects are dropped due to "efficacy," meaning the expected therapeutic effect is not observed, making it the top reason for project delays since the 2000s.

How to choose a nonclinical drug efficacy and pharmacology
contract testing company

In the drug discovery stage,Choosing a CRO as a strategic approach to eliminate the "translational gap"It is important. Here we summarize the key points for choosing a CRO, so please use it as a reference.

Whether the disease phenotype is reproduced comparably to human clinical settings

Many diseases are not caused by a single gene or molecule. Therefore, especially for complex chronic diseases such as MASH/NASH, fibrosis, and cancer,how well the phenotypes (appearance/behavior of a disease) resulting from multiple interacting factors in human patients matchThis point is important.

The key is not just whether there are biomarkers specific to the disease, but whether there is a model that can be evaluated in parallel with histopathological features and clinical diagnostic criteria. Another point to check when choosing a CRO is whether they can present scientific evidence showing a high correlation between gene expression profiles and signal transduction pathways with human clinical patient data.

Are there alternative testing systems to overcome species differences in drug discovery targets?

In cases where candidate compounds target "human-specific receptors or molecules," there's an aspect where drug efficacy cannot be correctly evaluated in normal wild-type animals, even with sophisticated disease model animals.

From the above,Whether contract research organizations (CROs) develop and maintain humanized models.In addition to,Is there plenty of validation data?I will confirm. Another point is whether we can propose a study design that seamlessly combines in vitro efficacy evaluation systems using "patient-derived cells (PDCs)" and "human 3D organoids" to complement the limitations of animal models.

Is there a "quantification and digital pathology technology" that can capture the gradation of medicinal effects?

Relying solely on visual scoring by pathologists carries the risk of overlooking subtle gradations (changes) in drug efficacy. To satisfy regulatory authorities such as PMDA and FDA, objective and rigorous quantification is necessary.

Therefore,The introduction of digital pathology utilizing AI and image analysis software enables the acquisition of quantitative data that eliminates observer subjectivity.This is raised as a selection point. Furthermore, regarding the correlation between drug dosage and target occupancy in tissues (PD) and actual efficacy (dose-response curve),Do you possess experimental techniques and know-how that can output clear graphs with little variation?I will also check.

Can a "reverse translational" study design be done with clinical protocols in mind?

not only administering to the mice according to the stated protocol,Consulting capabilities to optimize non-clinical study designs by working backward from planned clinical trialsWhether they have this is also a good indicator of an excellent CRO.

It is also important that we can discuss together an appropriate study design that considers the actual routes and frequencies of administration in clinical trials, and matches the kinetics in animal models.

It is also important to scientifically propose the selection of positive controls and the calculation of appropriate sample sizes for each stage, from screening tests in the exploration phase to rigorous efficacy tests for clinical trials.

Five highly specialized pharmacology testing CROs

Here, the effical editorial team independently researched contract service providers and selected companies that offer a broad range of disease models and study examples. These companies were classified according to the target area of the new drug under development.

Efficacy and pharmacology studies in organ fibrosis
SMC Laboratories

Why Choose SMC Laboratories, Inc.
for Efficacy and Pharmacology Studies

• In-house–developed STAM™ mice support the development of challenging therapeutics
  

  The STAM™ model has become widely used as a tool for drug development in oncology. However, in liver cancer, development has reportedly been delayed because immune checkpoint inhibitors demonstrate limited efficacy. In response,

  SMC Laboratories has developed a new model that enables the evaluation of drug efficacy using cancer immunotherapy approaches. This system can be used to evaluate liver diseases, an area that has historically been difficult to assess, and supports more reliable efficacy evaluations in the liver.

• Development of New Models When No Suitable Disease Model Exists
  

  Models that can represent human pathology are essential for drug development. HoweverMany diseases for which there is no model yet.and, if it already was, it is unbearable for current clinical practice.There are many models with challenges

  SMC Laboratories identifies the issues in models that cannot support meaningful evaluation and addresses these challenges by re-establishing disease pathologies that overcome the identified limitations. When no suitable model can be found in the existing literature or market, SMC Laboratories can also develop a new model from scratch.

Representative Disease Models
at SMC Laboratories

An example of a pathological model

SMC Laboratories, Inc. offers a diverse lineup of 25 proprietary disease mouse models, covering key research areas in inflammation, fibrosis, metabolic, and immuno oncology—including their patented STAM™ mouse model.Lineup of 25 disease model mice

The STAM™ model, widely recognized in MASH/NASH research, and the UUO model, a well-established renal fibrosis model, are two representative examples we will show below.

Pharmacology & Efficacy Case Studies – SMC Laboratories, Inc.

SMC Laboratories serves over 1,000 clients* worldwide and has extensive experience across multiple therapeutic areas, including the use of STAM™ mice in nonclinical studies targeting fibrosis, inflammation, metabolism, and cancer immunology.

Below are examples of pharmacology and efficacy studies that successfully supported the transition to Phase 2 clinical trials.

• Phase 2 study in NASH-related liver cirrhosis
  Galectin Therapeutics Inc. (NASDAQ: GALT) conducted a drug evaluation study targeting fibrosis in STAM™ mice, supporting a Phase 2 study in NASH-related liver cirrhosis.
  *Reference: https://www.smccro-lab.com/jp/quality/
• Ongoing Phase 2 study in NASH at the hepatic fibrosis stage
  Can-Fite BioPharma Ltd. (NYSE American: CANF, TASE: CFBI) conducted a drug evaluation study targeting NASH using the STAM™ model. A Phase 2 study targeting NASH at the hepatic fibrosis stage is currently ongoing.
  *Reference: https://www.smccro-lab.com/jp/quality/

Company Information

Retina / Neuro-ophthalmology Efficacy and Pharmacology Studies: spanDrug Safety Testing Center

Why Drug Safety Testing Center is recommended
for outsourced efficacy and pharmacology studies

• Strong track record in ophthalmology, including high intraocular pressure and dry eye
  

  Drug Safety Testing Center specializes in ocular disease drug discovery using rabbits and rats and maintains a wide range of ocular disease models. In particular, the organization has developed numerous models for glaucoma and retina-related studies and is characterized by a high likelihood of generating reliable data in these areas.Developed many models in glaucoma and retina-related trialsand are characterized by a high likelihood of obtaining credible data.

• Faster reporting to accelerate drug discovery
  

  Drawing on many years of experience, the Drug Safety Testing Center has improved the speed from data extraction to reporting, enabling an increase in the number of drug discovery trials that can be conducted. The organization also has highly responsive specialist staff who can quickly address testing inquiries and requests.

Example disease models at Drug Safety Testing Center
An example of a pathological model

Drug Safety Testing Center conducts a wide range of evaluations in ophthalmology, including high intraocular pressure, retinal, and dry eye indications. A key strength is the ability to perform studies in multiple animal species, such as dogs, rabbits, and rats, which facilitates identification of the potential utility of drug candidates.

Pharmacodynamic and Pharmacological Studies Case Studies
- Drug Safety Testing Center

No case studies were listed on the official website.

Company Information

Metabolic disease efficacy and pharmacology studies: Bozo Research Center

Why Bozo Research Center is recommended
for outsourced efficacy and pharmacology studies

• Applicable data for lifestyle-related disease therapeutics
  

  Bozo Research Center specializes in the development of therapeutics for lifestyle-related diseases and provides data with high applicability to metabolic drug development. The organization conducts a wide range of in vivo pharmacology studies using endpoints such as food intake, body weight, blood glucose, and lipid parameters, as well as in vitro pharmacology studies using cultured cells. A key strength is the ability to efficiently perform studies in metabolic disease systems.

• Credible testing by experts in toxicologic pathology
  

  It employs toxicologic pathologists and other specialists and, drawing on extensive experience and a strong track record, designs and conducts studies aimed at generating valuable data. All studies can be performed as tests compliant with reliability standards, and test systems can be proposed by staff with direct experience in drug discovery research.

Example disease models
at Bozo Research Center

In the metabolic disease area, Bozo Research Center performs the tests and evaluations required for the development of therapeutics for lifestyle-related diseases. The core of its service offering consists of in vivo efficacy and pharmacology studies using both normal animals and disease model animals.

Pharmacology and efficacy case studies
– Bozo Research Center

No case studies were listed on the official website.

Company Information

Neurodegenerative disease efficacy and pharmacology studies: Mediford

Why Mediford is recommended
for outsourced efficacy and pharmacology studies

• Broad portfolio of central nervous system disease models with high applicability
  

  Mediford offers a wide range of disease models for central nervous system disorders, including Alzheimer’s disease, depression, pain, memory impairment, and epilepsy. Even within depression alone, Mediford maintains multiple model types, enabling pharmacology studies that can be tailored to detailed disease profiles.

• Surgical efficacy studies using genetically engineered and other specialized animals
  

  Mediford has extensive experience conducting studies with conventional animals, genetically modified animals, and mutant strains, and is particularly strong in drug efficacy studies that require surgical procedures. The organization also has substantial expertise in in vitro drug efficacy studies using PCR, flow cytometry, and various cell types, providing a high likelihood of obtaining useful data.

Example disease models
at Mediford

Mediford conducts contracted studies that apply a variety of disease models for efficacy and pharmacology testing. With a primary focus on the central nervous system, the company performs a broad range of studies, including in vitro drug efficacy tests in areas such as pain, infectious diseases, and the cardiovascular system.

Medford's.
Pharmacology and efficacy case studies – Mediford

No case studies were listed on the official website.

Mediford Company Information

Atopic dermatitis efficacy and pharmacology studies: Anpyo Center

Why Anpyo Center is recommended
for outsourced efficacy and pharmacology studies

Why we recommend it

• Testing with mouse models that reproduce human atopic dermatitis symptoms
  

  Atopic dermatitis is sometimes referred to as a “national disease,” and Anpyo Center can conduct studies using mouse models that develop symptoms similar to those seen in human atopic dermatitis. These models can support the development of new drugs targeting atopic dermatitis, whose underlying mechanisms are not yet fully understood.

• Quality assurance through reliability frameworks and in-house peer review
  

  In addition to a reliability assurance framework that verifies whether studies are being conducted in accordance with standard operating procedures and study plans, Anpyo Center safeguards study design and interpretation of study results through an in-house peer review system. Through these measures, the center continually works to enhance the quality of its studies and data.

Example disease models
at Anpyo Center

An example of a pathological model

Anpyo Center conducts efficacy and pharmacology studies with a particular focus on psychiatric disease systems such as Alzheimer’s disease, as well as on atopic dermatitis, and it holds especially well-regarded models in the atopic dermatitis area.

Pharmacology and efficacy case studies
– Anpyo Center

No case studies were listed on the official website.

Company Information

Typical disease and animal models used in pharmacological studies

In preclinical pharmacodynamic studies to evaluate the efficacy of new drugs, it is important to select models that accurately reproduce human pathological conditions. This section introduces representative disease models and animal models in various disease areas.

Chronic kidney disease model

A chronic kidney disease (CKD) model artificially reproduces chronic kidney dysfunction and its associated complications (such as fibrosis and renal anemia) in experimental animals like mice and rats. This model is an essential testing system for obtaining proof of concept, such as determining "whether a compound under development truly suppresses the decline in kidney function," "reduces proteinuria," and "prevents the progression of renal fibrosis."

Pulmonary function test

Pulmonary function testing is an indispensable method for quantitatively evaluating drug efficacy in the drug discovery of respiratory diseases. It is important to appropriately select indicators such as airway resistance (Raw) and dynamic compliance (Cdyn) depending on disease models such as asthma, COPD, and pulmonary fibrosis. Improving measurement accuracy is key to stabilizing anesthesia management and intubation techniques, as well as introducing P-V curve analysis and non-invasive longitudinal evaluation to enhance clinical translatability. By integrating these, it becomes possible to generate high-quality data with high clinical predictability.

Drug Discovery and Efficacy Evaluation Using Cancer Immunological Assays

Cancer immunoassays are essential for drug discovery in cancer immunotherapy.In addition to conventionally used methods, various new technologies such as patient-derived organoids (PDOs) that reproduce the tumor microenvironment and AI-based image analysis have been introduced in recent years, improving the accuracy of drug efficacy prediction. However, constructing advanced evaluation systems requires high technical skills and faces significant cost barriers.Outsourcing to a CRO specializing in tumor immunology is key to accelerating drug discovery.It can be said that