you are successful
in discovery?
Congrats!
then starts the cmc obstacle course
Bringing a new drug from the lab to the clinic is a completely different
process from identifying the target and molecule in the first place.
CMC is the most underappreciated aspect of the whole drug development process, but it can make or break development.
Grounded in science and regulated by governments, health care is a challenging sector. In the life of any startup, there are plenty of opportunities for fatal missteps.
But that doesn’t mean your company has to make one.
How to maintain pharmaceutical industry’s high-level standards ?
Each stage in drug development is key, not only demonstrating
proof of concept : 3biotech will support you day after day.
We are your first crash-test:
challenging, then supporting and accelerating
A stronger valuation of your innovation : #challenging to avoid drug development failures and get a clear Chemistry Manufacturing and Control strategy.
Define the best path from beginning to end : # supporting step by step from scientific ideas to innovative drugs.
Go faster from discovering drug candidate until clinical proof of concept : # accelerating the entire drug development
value chain
3Biotech development strategy
relies on 3 major pillars
SAFETY
EFFICACY
manufacturability
Safety
Before administering the drug to human subjects for the first time (First in Human), it is crucial to evaluate the safe dosing limits and potential adverse effects of the drug candidate. This assessment is essential in minimizing risks to clinical subjects and program development.
We assist in the design and execution of pharmacology, drug metabolism and pharmacokinetics (DMPK), and toxicology studies to evaluate the potential adverse effects of the drug candidates related to the intended use of the product.
The prudent selection of a drug candidate relies not only on the expected therapeutic efficacy of the product but also on the toxicology and pharmacology assessments that contribute to the safety package.
Safety
The lack of therapeutic efficacy is a leading cause of drug development failure, especially during the clinical phases. However, a significant reduction in this figure can be achieved by emphasizing nonclinical efficacy studies.
in vitro assessments, including target engagement, tissue cross-reactivity, immune-mediated cell killing, and other specific ex-vivo and in-vivo assays related to the Mechanism of Action (MoA). These studies are referred to as "translational studies" as they translate potential drug candidates' on- and off-target nonclinical properties to clinical consequences.
By conducting translational studies, the best drug candidates for early clinical testing can be identified based on their efficacy and safety profile. This approach can help to minimize the risk of failure during clinical development and optimize the drug development process.
Safety
In drug development, safety and efficacy are not the only important factors to consider. It is also essential to develop a robust manufacturing process that can consistently produce high-quality drugs.
The Chemistry, Manufacturing
and Controls (CMC) section
is a critical aspect of any pharmaceutical clinical trial or marketing application. Lack of a comprehensive CMC strategy is a common reason for market authorization denials, accounting for 25% of such cases in the US. This can result in processes and products that fail to meet the regulatory standards.
Regulatory authorities require assurance that there is consistency between the product tested during clinical trials and the batches produced for commercialization years later. Therefore, it is crucial to establish an exhaustive CMC strategy to ensure that the drug product is not only safe and effective but also consistent between batches.
Frequently Asked Questions
What is lead optimization in drug development?
Lead optimization is the process of refining drug candidates, whether small molecules or biologics, to improve their efficacy, safety, and manufacturability before advancing to clinical development. For small molecules, this involves structure-activity relationship (SAR) studies, absorption, distribution, metabolism, excretion, and toxicity (ADME-Tox) profiling.
For biologics, optimization includes protein engineering, immunogenicity assessment, expression system refinement, and stability evaluation. Comprehensive preclinical assessments are performed to ensure the highest likelihood of success in human trials.
What is translational research in drug development?
Translational research bridges preclinical discoveries to human trials by optimizing drug candidates for clinical evaluation. It includes dose selection, biomarker identification, and first-in-human study design to ensure successful clinical translation.
What is Chemistry, Manufacturing, and Control (CMC) in pharma?
Chemistry, Manufacturing, and Controls (CMC) encompasses all activities related to the development of drug substances and products. This includes formulation, process optimization, analytical testing, and ensuring regulatory compliance.
CMC ensures the physical and chemical characteristics of drug products are assessed to maintain quality and consistency throughout manufacturing.
What is continued process verification (CPV) and why is it required?
Continued Process Verification (CPV) is the ongoing monitoring of a manufacturing process to ensure it consistently produces products with the desired quality attributes throughout the product lifecycle.
CPV is required for regulatory compliance and helps maintain product quality and consistency during commercial production.
What are accelerated regulatory pathways (e.g., Fast Track, Priority Medicines [PRIME])?
Accelerated regulatory pathways, such as Fast Track (FDA) and Priority Medicines (PRIME, European Medicines Agency), are programs designed to speed up the development and approval of drugs for serious conditions or those addressing unmet medical needs.
These pathways aim to provide patients with faster access to potentially life-saving therapies.