Drug Discovery and Development Solutions for Sarcopenia

Solve Research and Development Problems Through Our Professional Services

Accelerating Sarcopenia Drug Development

Against the backdrop of accelerating global population aging and a sharp rise in sarcopenia prevalence, sarcopenia has become a serious public health and economic burden. However, due to its complex pathophysiology and the lack of standardized preclinical models, the development of sarcopenia treatments remains a significant challenge. Protheragen is a professional partner for the development of innovative sarcopenia therapies, providing comprehensive preclinical drug development services. We offer a fully customized drug discovery and development solution, from target identification and lead compound optimization to IND submission, designed to accelerate the commercialization of your sarcopenia treatment.

Introduction to Sarcopenia

Sarcopenia is a progressive and generalized skeletal muscle disorder characterized by the accelerated loss of muscle mass, strength, and function. It is strongly associated with aging and is a major contributor to frailty, mobility impairment, increased risk of falls and fractures, loss of independence, and higher mortality. Primary causes include:

Neurological Decline and Hormonal Changes
Chronic Inflammation and Mitochondrial Dysfunction
Nutritional Deficiencies and Lack of Exercise

Therapeutic Targets for Sarcopenia

Currently, there is no globally approved pharmacological therapy specifically for sarcopenia, with management primarily reliant on resistance exercise and nutritional support. Consequently, drug development is actively targeting specific molecular pathways involved in muscle loss. Key therapeutic targets are as follows:

Targets	Molecular Pathways	Related Drugs	Core Mechanism
Myostatin (GDF-8)	TGF-β signaling pathway	Stamulumab, Landogrozumab (Monoclonal antibodies)	Neutralizes myostatin to block its inhibition of muscle growth.
Activin A / ActRIIB	TGF-β signaling pathway	Bimagrumab (Anti-ActRII antibody), ACE-031 (Fusion protein)	Blocks ActRIIB receptor, inhibiting signaling of myostatin, activin, and other ligands.
Androgen Receptor (AR)	Androgen signaling pathway	Enobosarm, LGD-4033 (SARMs)	Selectively activates AR in skeletal muscle to promote protein synthesis with reduced side effects.
Ghrelin Receptor (GHSR-1a)	Growth hormone secretagogue pathway	Anamorelin, MK-0677 (Ghrelin mimetics)	Stimulates appetite and GH secretion to indirectly promote anabolism.
AMPK	Cellular energy sensor pathway	AICAR, Metformin (under investigation)	Activates AMPK to improve mitochondrial function and cellular energy homeostasis.
PPARδ	Fatty acid oxidation & metabolism pathway	GW501516 (under investigation)	Activates PPARδ to enhance oxidative metabolism and promote slow-twitch muscle fibers.
SIRT1	Mitochondrial biogenesis & senescence pathway	Resveratrol, other SIRT1 activators (under investigation)	Activates SIRT1 to promote mitochondrial health and delay cellular aging processes.
TNF-α / IL-6	Inflammatory signaling pathway	Various anti-inflammatory agents (under investigation)	Inhibits chronic low-grade inflammation ("inflammaging") to reduce muscle proteolysis.

Drug Discovery and Development Services

In Vitro Model Development Services
Animal Model Development Services
Pharmacodynamic (PD) Evaluation Services
Pharmacokinetic (PK) Research Services
Toxicology Research Services
Biomarker Discovery and Validation Services

Protheragen is dedicated to designing, establishing, and validating highly physiologically relevant cellular systems that accurately recapitulate key pathological features of sarcopenia. We provide customized, robust models to serve as predictive platforms for early-stage drug efficacy and mechanism-of-action studies. Key models we offer include:

Types	Models
Cell-Based Models	Primary Muscle Cell Lines: Primary human myoblasts/myotubes, primary mouse myoblasts/myotubes, etc. Immortalized Muscle Cell Lines: C2C12 mouse myoblasts, LHCN-M2 human myoblasts, etc. Co-culture Models: Neuromuscular co-culture models, muscle-fibroblast/adipocyte co-culture models, etc. Senescence-Induced Models: Replicative senescence models, genotoxic stress-induced senescence models, oxidative stress-induced senescence models, etc.
Organoid & 3D Tissue-Engineered Models	Myospheres & 3D Bioprinted Muscle Constructs Muscle-on-a-Chip (Microphysiological Systems) Induced Pluripotent Stem Cell (iPSC)-Derived Muscle Organoids Vascularized or Innervated Muscle Organoids

Types

Models

Cell-Based Models

Primary Muscle Cell Lines: Primary human myoblasts/myotubes, primary mouse myoblasts/myotubes, etc.
Immortalized Muscle Cell Lines: C2C12 mouse myoblasts, LHCN-M2 human myoblasts, etc.
Co-culture Models: Neuromuscular co-culture models, muscle-fibroblast/adipocyte co-culture models, etc.
Senescence-Induced Models: Replicative senescence models, genotoxic stress-induced senescence models, oxidative stress-induced senescence models, etc.

Organoid & 3D Tissue-Engineered Models

Myospheres & 3D Bioprinted Muscle Constructs
Muscle-on-a-Chip (Microphysiological Systems)
Induced Pluripotent Stem Cell (iPSC)-Derived Muscle Organoids
Vascularized or Innervated Muscle Organoids

Leveraging cutting-edge technological platforms and a professional team, Protheragen is committed to establishing robust in vivo model systems that faithfully recapitulate the complex and progressive pathology of sarcopenia, enabling reliable therapeutic efficacy evaluation. We offer a comprehensive portfolio of models, including natural aging, accelerated aging, and mechanistically induced aging systems, allowing you to select the most biologically and clinically relevant platform tailored to your specific drug candidate and its mechanism of action.

Types	Models
Aged Rodent Models	F344 Rats (>24 months) BN Rats (>32 months) Sprague Dawley Rats (>24 months)	Wistar Rats (>24 months) C57BL/6J Mice (>24 months) SAMP8 Mice (>9 months)
Mechanistic & Induced Atrophy Models	Hindlimb Unloading/Suspension Models Hindlimb Cast Immobilization Models Sciatic Nerve Denervation-Induced Models	Glucocorticoid-Induced Myopathy Models Dexamethasone-Induced Muscle Wasting Models Other Models

Protheragen's pharmacodynamic (PD) evaluation services provide comprehensive, multi-dimensional analysis to definitively characterize the biological effects and therapeutic efficacy of your sarcopenia drug candidate, both in vitro and in vivo. We deliver robust, quantitative data that links your compound's mechanism of action directly to functional improvements in muscle health, building a compelling efficacy package for preclinical development.

In Vitro Pharmacodynamic Evaluation

Target Binding and Pathway Activation Analysis: Evaluates the compound's binding affinity to specific targets and detects phosphorylation or nuclear translocation changes in downstream key signaling pathways.
Muscle Cell Phenotypic and Functional Evaluation: Quantifies anabolic effects (e.g., hypertrophy, protein synthesis), anti-atrophic effects, and metabolic/functional improvements (e.g., mitochondrial function, glucose uptake) in muscle cells.
Biomarker Quantification Analysis: Utilizes techniques such as ELISA, Western Blot, and qPCR for precise quantification of key biomarkers in cell culture supernatants or lysates.

In Vivo Pharmacodynamic Evaluation

Core Efficacy Endpoint Measurement: Quantifies key therapeutic outcomes including muscle mass, muscle strength and function, and body composition.
Molecular and Histological Analysis: Evaluates mechanisms and tissue-level changes through muscle histology (e.g., fiber cross-sectional area, fiber typing) and analysis of protein/RNA extracted from muscle tissue to validate target engagement and pathway modulation.
Exploratory Biomarker Discovery: Identifies and analyzes potential systemic biomarkers, such as myokines or circulating proteins in serum, to support clinical translatability and future monitoring strategies.

Pharmacokinetic research services provide comprehensive and critical data to characterize the absorption, distribution, metabolism, and excretion (ADME) profile of your therapeutic candidate for sarcopenia. We conduct rigorous studies to define "what the body does to the drug," generating essential insights into dosage, exposure, and safety that inform all stages of preclinical and clinical development.

Core Pharmacokinetic (PK) Services
Bioanalytical Method Development & Validation: Developing validated bioanalytical assays for precise drug and metabolite quantification. In Vitro ADME Profiling: Metabolic stability assay, cytochrome P450 (CYP) enzyme inhibition & induction screening, plasma protein binding determination, permeability assays, reaction phenotyping, etc. In Vivo Pharmacokinetic Studies: Single and repeat-dose PK studies, absolute oral bioavailability study, tissue distribution & penetration study, mass balance and excretion study, etc. Toxicokinetics (TK): Integrating pharmacokinetic analysis with toxicity studies for exposure-response correlation. PK/PD Modeling & Integration: Modeling exposure-response relationships to optimize dosing and predict human efficacy.

Core Pharmacokinetic (PK) Services

Bioanalytical Method Development & Validation: Developing validated bioanalytical assays for precise drug and metabolite quantification.
In Vitro ADME Profiling: Metabolic stability assay, cytochrome P450 (CYP) enzyme inhibition & induction screening, plasma protein binding determination, permeability assays, reaction phenotyping, etc.
In Vivo Pharmacokinetic Studies: Single and repeat-dose PK studies, absolute oral bioavailability study, tissue distribution & penetration study, mass balance and excretion study, etc.
Toxicokinetics (TK): Integrating pharmacokinetic analysis with toxicity studies for exposure-response correlation.
PK/PD Modeling & Integration: Modeling exposure-response relationships to optimize dosing and predict human efficacy.

Protheragen offers comprehensive toxicology research services designed to identify and characterize potential adverse reactions of your sarcopenia drug candidates, ensuring patient safety and supporting regulatory submissions for global clinical development and market approval.

Genetic Toxicology
General Cytotoxicity
Cardiotoxicity Screening
Organ-Specific Toxicity
Acute and Repeat-Dose Toxicity Studies
Safety Pharmacology Core Battery
Developmental and Reproductive Toxicology (DART)
Local Tolerance Studies

At Protheragen, we offer an integrated, end-to-end biomarker platform specifically tailored for sarcopenia drug development. We identify and measure biomarkers at multiple levels: in vitro (secreted factors from muscle cells), in vivo (serum/plasma myokines, imaging markers), and in tissue (histological and molecular signatures). We help you develop pharmacodynamic biomarkers to demonstrate target engagement and proof-of-mechanism in your studies.

Items	Description
Biomarker Discovery & Identification	Multi-omics profiling (transcriptomics, proteomics, metabolomics) of muscle tissue and biofluids from preclinical models. Secretome analysis of muscle cell cultures to identify myokines and other secreted factors. Exploration of imaging biomarkers (e.g., MRI for muscle quality, DXA for composition) correlated with molecular data.
Analytical Assay Development & Validation	Development of quantitative assays (LC-MS/MS, multiplex immunoassays, ELISA, dPCR) for candidate biomarkers. Full analytical validation for sensitivity, specificity, precision, accuracy, and stability in biological matrices.
Preclinical Biomarker Qualification	Pharmacodynamic (PD) biomarker studies in relevant animal models to link drug exposure to target modulation. Correlation of biomarker levels with functional efficacy endpoints (e.g., grip strength, muscle mass, endurance). Longitudinal biomarker monitoring to assess disease progression and treatment response.

Why Choose Us

As an ideal preclinical development partner for sarcopenia therapeutics, Protheragen offers unparalleled end-to-end solutions specifically designed for the complexities of muscle-wasting disorders. We integrate deep expertise in muscle biology with a full suite of GLP-compliant platforms, providing a single point of accountability, accelerated timelines, and meticulously curated data packages to de-risk your program and build confidence with investors and regulators. Partner with us to advance your scientific vision into a viable sarcopenia therapy.

Deep Sarcopenia Expertise
Cutting-Edge Technology
Customized Service Workflow
End-to-End Solutions

FAQs

Q: Can you support the development of different therapeutic modalities for sarcopenia (e.g., small molecules, biologics, cell therapies)? A: Yes, we provide end-to-end preclinical support for a broad range of therapeutic modalities targeting sarcopenia, including small molecules, antibodies, peptides, therapeutic proteins, cell therapies, and gene therapies. Our in vitro and in vivo platforms are tailored to the unique mechanism of each modality, with specialized assays for target engagement, functional validation, biodistribution, and safety assessment, ensuring translational relevance across diverse molecular formats.
Q: How do you ensure the translational relevance of your preclinical models? A: We prioritize models that replicate human disease pathophysiology. This includes using primary human muscle cells, naturally aged animals as the gold standard, and focusing on functional outcomes (strength, endurance) alongside molecular biomarkers. Our goal is to bridge preclinical findings directly to potential clinical trial endpoints.
Q: What does your "integrated, end-to-end" service actually look like for a typical project? A: We can act as your single partner from early discovery (target validation, in vitro screening) through candidate selection, comprehensive IND-enabling studies (efficacy, DMPK, toxicology), and final report compilation. This integrated approach ensures seamless data flow, consistent quality, and accelerated timelines by eliminating the need to coordinate multiple vendors.
Q: How flexible is your collaboration model? Can we outsource only part of our program? A: We offer full flexibility. You can engage us for a complete turnkey program or for discrete, modular services (e.g., just PK/PD studies or a specific efficacy model). Our collaboration model is customized to your needs, resources, and internal capabilities.
Q: How do you handle intellectual property (IP) and confidentiality in partnerships? A: Protecting your IP is our top priority. We operate under robust, client-focused confidentiality agreements (CDAs). All work is performed as "work for hire," and any novel IP generated during your project is exclusively assigned to you. Our systems and agreements are designed to ensure complete confidentiality and secure data management.