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Accelerating Pulmonary Arterial Hypertension Drug Development

Hypertension, particularly in the context of pulmonary arterial disease, presents a significant and complex therapeutic challenge, demanding innovative approaches to drug development. Protheragen is a specialized partner in the preclinical development of therapeutics targeting hypertension and pulmonary arterial conditions. Leveraging a comprehensive suite of preclinical services—from target validation through IND-enabling studies—Protheragen delivers end-to-end solutions tailored to the unique requirements of this therapeutic area. With a team of experienced scientists and access to advanced technology platforms, Protheragen ensures the highest standards of scientific rigor and data integrity. The company’s operations are fully aligned with global regulatory expectations, supporting clients in meeting critical milestones efficiently and with confidence. Protheragen’s integrated approach streamlines the transition from discovery to clinical readiness, minimizing risk and optimizing candidate selection. Driven by a commitment to scientific excellence and innovation, Protheragen accelerates the development of next-generation therapies for hypertension and pulmonary arterial disease. Through strategic collaboration and a focus on quality, Protheragen empowers its partners to achieve therapeutic breakthroughs that address unmet medical needs.

What is Pulmonary Arterial HypertensionTargets for Pulmonary Arterial HypertensionDrug Discovery and Development ServicesWhy Choose Us

What is Pulmonary Arterial Hypertension

Pulmonary arterial hypertension (PAH) is a progressive and life-threatening disorder characterized by elevated pressure in the pulmonary arteries due to narrowing, remodeling, and obstruction of small pulmonary vessels. The disease arises from a combination of endothelial dysfunction, smooth muscle proliferation, inflammation, thrombosis, and genetic predispositions—most notably mutations in the BMPR2 gene in heritable cases. PAH may be idiopathic, heritable, drug- or toxin-induced, or associated with other conditions such as connective tissue diseases, HIV infection, or congenital heart defects. These pathophysiological changes increase pulmonary vascular resistance, leading to right ventricular hypertrophy and, ultimately, right heart failure. Clinically, PAH presents with nonspecific symptoms such as exertional dyspnea, fatigue, chest pain, syncope, and signs of right heart dysfunction. Diagnosis involves a comprehensive assessment including clinical evaluation, echocardiography, and definitive confirmation by right heart catheterization, which demonstrates elevated mean pulmonary arterial pressure and increased vascular resistance. Additional testing helps exclude secondary causes and identifies candidates for specific therapies. Treatment strategies target key molecular pathways and include endothelin receptor antagonists (e.g., macitentan, ambrisentan, bosentan), phosphodiesterase-5 inhibitors (e.g., tadalafil), prostacyclin analogs (e.g., treprostinil, selexipag), and novel agents like sotatercept. Early diagnosis and targeted therapy have improved outcomes, but PAH remains associated with significant morbidity and mortality.

Launched Drugs

Structure Generic Name CAS Registry Number Molecular Formula Molecular Weight
sotatercept (Prop INN; USAN); sotatercept-csrk 1001080-50-7
macitentan/tadalafil
img-475086-01-2-selexipag-prop-inn-usan selexipag (Prop INN; USAN) 475086-01-2 C26 H32 N4 O4 S 496.622
img-830354-48-8-treprostinil-diethanolamine-rec-innmtreprostinil-d treprostinil diethanolamine (Rec INNM); treprostinil diolamine (USAN) 830354-48-8 C23 H34 O5 . C4 H11 N O2 495.649
img-441798-33-0-macitentan-rec-inn-usan macitentan (Rec INN; USAN) 441798-33-0 C19 H20 Br2 N6 O4 S 588.273
img-625115-55-1-riociguat-rec-inn-usan riociguat (Rec INN; USAN) 625115-55-1 C20 H19 F N8 O2 422.416
img-177036-94-1-ambrisentan-rec-inn ambrisentan (Rec INN) 177036-94-1 C22 H22 N2 O4 378.421
img-171596-29-5-tadalafil-rec-inn-usan tadalafil (Rec INN; USAN) 171596-29-5 C22 H19 N3 O4 389.404
img-289480-64-4-treprostinil-sodium-rec-innm-usanuniprost treprostinil sodium (Rec INNM; USAN); uniprost 289480-64-4 C23 H33 O5 . Na 412.495
img-147536-97-8-bosentan-rec-inn-usan-jan bosentan (Rec INN; USAN; JAN) 147536-97-8 C27 H29 N5 O6 S 551.614

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Targets for Pulmonary Arterial Hypertension

Targets in Clinical or Later Phases of Development

Target Name Gene Symbol
AMP-activated protein kinase (AMPK)
endothelin receptor type A EDNRA
endothelin receptor type B EDNRB
insulin INS
phosphodiesterase 5A PDE5A
Transforming growth factor beta (nonspecified subtype)
Voltage-Gated Calcium Channel alpha-2/delta (nonspecified subtype)
prostaglandin I2 receptor PTGIR
Soluble guanylyl cyclase
KIT proto-oncogene, receptor tyrosine kinase KIT

Pulmonary arterial hypertension (PAH) is driven by complex molecular mechanisms involving dysregulated vasoconstriction, vascular remodeling, and oxidative stress. Key therapeutic targets include endothelin receptors (EDNRA, EDNRB), which mediate potent vasoconstrictive and proliferative signals, and the prostaglandin I2 receptor (PTGIR), responsible for vasodilation and antiproliferative effects. Phosphodiesterase 5A (PDE5A) modulates cGMP signaling, influencing vascular tone, while platelet-derived growth factor receptors (PDGFRA, PDGFRB) and KIT receptor tyrosine kinase are central to abnormal cell proliferation and vascular remodeling. Additionally, transcriptional regulators such as NFE2L2 (NRF2) and heme oxygenase 1 (HMOX1) orchestrate antioxidant defenses, mitigating oxidative injury in the pulmonary vasculature.

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Drug Discovery and Development Services

In Vitro Efficacy Testing ServicesIn Vivo Model DevelopmentPK/PD Study ServicesIn Vivo Toxicity Assessment ServicesBiomarker Analysis Services
In Vitro Efficacy Testing Services

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Our In Vitro Efficacy Testing Service accelerates pulmonary arterial hypertension (PAH) drug discovery by providing robust, sensitive platforms for compound screening and characterization. We offer comprehensive evaluation of candidate therapies targeting key PAH pathways, including endothelin, prostacyclin, phosphodiesterases, and growth factor receptors. Utilizing advanced biochemical and cell-based assays—such as luminescent, fluorescent, ELISA, and gene expression analyses—we deliver quantitative data on potency, efficacy, and mechanism of action. Our high-throughput methodologies ensure accurate assessment of pharmacological parameters, enabling data-driven lead optimization and prioritization, ultimately supporting the rapid development of effective, targeted therapies for PAH.

Abl Proto-Oncogene 1, Non-Receptor Tyrosine Kinase Colony Stimulating Factor 1 Receptor
Endothelin Receptor Type A Kit Proto-Oncogene, Receptor Tyrosine Kinase
Nfe2 Like Bzip Transcription Factor 2 Peroxisome Proliferator Activated Receptor Gamma
Phosphodiesterase 5A Platelet Derived Growth Factor Receptor Alpha
Platelet Derived Growth Factor Receptor Beta Prostaglandin I2 Receptor

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Why Choose Us

Choosing Protheragen means partnering with a team that possesses specialized expertise in the research and development of new therapeutics for Hypertension, including pulmonary arterial hypertension. At Protheragen, our professional teams are composed of experienced scientists and industry experts dedicated to advancing the field with cutting-edge knowledge and skills. We utilize advanced technology platforms that enable us to efficiently translate scientific discoveries into promising drug candidates. Protheragen’s proven track record in preclinical drug development services demonstrates our reliability and commitment to delivering high-quality results for our clients. We adhere to the highest quality standards and maintain strict regulatory compliance throughout every stage of the development process, ensuring that our work meets both industry and global expectations. Above all, Protheragen is deeply committed to advancing innovative therapeutics for Hypertension, pulmonary arterial, striving to improve patient outcomes and address unmet medical needs. By choosing Protheragen, you are selecting a trusted partner dedicated to excellence, innovation, and the future of cardiovascular medicine.

FAQs for Our Services

Q: What are the main preclinical research challenges specific to developing drugs for pulmonary arterial hypertension (PAH)?

A: Pulmonary arterial hypertension presents unique preclinical challenges, including the need for robust animal models that accurately recapitulate human disease pathology, such as vascular remodeling and right ventricular dysfunction. Additionally, the heterogeneity of the disease and complex pathophysiology require comprehensive pharmacodynamic and pharmacokinetic assessments. Our company addresses these challenges by utilizing validated PAH models, advanced imaging, and biomarker analysis to ensure translational relevance of preclinical findings.

Q: What are the key regulatory considerations for preclinical development of PAH therapeutics?

A: Regulatory agencies such as the FDA and EMA require extensive preclinical safety and efficacy data before advancing to clinical trials. For PAH, this includes demonstration of target engagement, dose-ranging studies, and evaluation of potential off-target cardiovascular effects. Our team is experienced in designing preclinical programs that meet or exceed regulatory expectations, including GLP-compliant toxicology studies, and can provide guidance on regulatory submissions specific to PAH drug development.

Q: What are the critical technical aspects to consider in preclinical PAH research?

A: Technical aspects include the selection of appropriate in vitro and in vivo models, precise measurement of hemodynamic parameters (e.g., right ventricular systolic pressure), and assessment of pulmonary vascular remodeling. We employ state-of-the-art technologies such as telemetry, echocardiography, and histopathological analysis to generate high-quality, reproducible data. Our expertise ensures that technical endpoints align with clinical outcomes, supporting a smooth transition to first-in-human studies.

Q: How do timeline and cost considerations impact preclinical PAH drug development?

A: Preclinical development for PAH therapeutics is often time-intensive due to the complexity of disease models and the need for thorough safety assessments. Timelines typically range from 12 to 24 months, with cost influenced by study duration, model selection, and regulatory requirements. Our company offers flexible project management and scalable services to optimize both timeline and budget, ensuring efficient progression from discovery to IND-enabling studies.

Q: What are the key success factors for preclinical development of PAH drugs?

A: Success in preclinical PAH drug development hinges on selecting clinically relevant models, robust study design, early identification of safety liabilities, and clear go/no-go criteria. Effective collaboration with regulatory authorities and integration of translational biomarkers further enhance the likelihood of success. Our multidisciplinary team provides end-to-end support, leveraging deep expertise in PAH biology and preclinical methodologies to maximize the probability of clinical advancement.

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