Press Release
Oct 1, 2025 AliveGen Presents Preclinical Data Showing ALG-801 Outperforms Standard Therapies in Pulmonary Arterial Hypertension Model
-- ALG-801 demonstrates superior therapeutic efficacy in a gold standard PAH disease model
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--AliveGen USA Inc. (AliveGen), a clinical-stage biopharmaceutical company, today announced new preclinical data demonstrating that its investigational therapy ALG-801 delivered superior efficacy compared to current standard-of-care agents in a widely accepted animal model of pulmonary arterial hypertension (PAH). The findings were presented at the European Respiratory Society (ERS) Congress, held September 27–October 1, 2025, in Amsterdam.
PAH is a progressive and life-threatening disease characterized by increased pulmonary vascular resistance, leading to elevated pulmonary arterial pressure, right heart failure, and premature death. Despite multiple approved therapies, median transplant-free survival remains only about six years, underscoring the urgent need for new treatments.
To assess ALG-801’s potential in PAH, researchers conducted head-to-head studies in sugen-hypoxia (SuHx) rats, a gold standard model for pulmonary hypertension, comparing ALG-801 with Sildenafil and ActRIIA-Fc (Sotatercept), both approved therapies for PAH.
Data Highlights
- During disease development: ALG-801 prevented pulmonary hypertension, right ventricular hypertrophy and pulmonary vascular remodeling more effectively than Sildenafil.
- In established PAH: ALG-801 was significantly more effective than ActRIIA-Fc, reversing elevated pulmonary arterial pressure, right ventricular hypertrophy, and pulmonary vascular remodeling. ALG-801 treatment also normalized circulating BNP levels and extended survival.
“These results demonstrate ALG-801’s ability to outperform approved PAH therapies in rigorous preclinical testing,” said Dr. HQ Han, CEO of AliveGen. “The data suggest that ALG-801 has potential to transform treatment outcomes for patients with PAH, where survival remains unacceptably poor despite recent advances.”
The findings position ALG-801 as a promising candidate for clinical development in PAH and related conditions.
About ALG-801
ALG-801 is a next-generation ActRIIA/IIB hybrid ligand trap designed to selectively block Smad2/3 pathway-activating ligands implicated in the pathogenesis of PAH and other diseases. ALG-801 has completed Phase 1a and 1b trials in healthy volunteers and is ready to advance into Phase 2 clinical studies. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ALG-801 for the treatment of PAH.
About AliveGen
AliveGen is a privately held, clinical-stage biotechnology company based in Thousand Oaks, California. The company is dedicated to developing first-in-class and best-in-class biotherapeutics for diseases with high unmet medical needs, including cardiometabolic disorders, obesity, neuromuscular diseases, age-related osteosarcopenia, and various wasting disorders. AliveGen’s mission is to improve care and quality of life for patients through innovative therapies that are both safe and highly effective. For more information, visit www.alivegen.com.
Jan 7, 2025 AliveGen Receives FDA Orphan Drug Designation for ALG-801 for Pulmonary Arterial Hypertension
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--AliveGen USA Inc. (AliveGen), a clinical stage biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ALG-801 for the treatment of pulmonary arterial hypertension (PAH), a rare, serious, and life-threatening condition characterized by high blood pressure in the lung arteries that can lead to right-sided heart failure.
“FDA’s Orphan Drug Designation for ALG-801 underscores the need for identifying better treatment options for patients suffering from PAH,” said Dr. HQ Han, CEO of AliveGen. “The designation represents another milestone in AliveGen’s efforts to discover and develop first-in-class/best-in-class therapeutics for high unmet medical needs. We believe that ALG-801, which has demonstrated superior efficacy and improved safety profile in preclinical and clinical Phase 1 studies, holds a remarkable potential to become a best-in-class medicine for PAH. We look forward to progressing ALG-801 clinical development.”
The FDA Orphan Drug Designation program is granted to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides benefits to sponsors designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include tax credits for clinical costs, exemptions from certain FDA fees, and potential seven years of marketing exclusivity.
About ALG-801
ALG-801 is a novel next-generation ActRIIA/IIB hybrid ligand trap designed to selectively sequester a specific set of Smad2/3 pathway-activating ligands, which are critically involved in the pathogenesis of PAH as well as of many other diseases. ALG-801 has successfully completed its Phase 1a and 1b clinical trials in healthy volunteers and is ready for Phase 2 clinical development.
About AliveGen
AliveGen is a privately held clinical-stage biotechnology company located in Thousand Oaks, California. The company aims to discover first-in-class/best-in-class biotherapeutics for treating diseases with high unmet medical needs such as cardiometabolic disorders, neuromuscular diseases, age-related osteosarcopenia, and various wasting disorders. The company aspires to improve the care and quality of life of patients by developing innovative therapies that are safe and highly effective. Learn more about AliveGen on its website: www.alivegen.com.
Mar 18, 2024 AliveGen Announces Successful Completion of Phase 1b Multiple-Ascending Dose Clinical Trial for ALG-801
-- Profound effects on muscle gain, fat loss, and insulin sensitization, along with a dual anabolic/anti-resorptive action on bone, pave the way for Phase 2 clinical development
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--AliveGen USA Inc. (AliveGen), a clinical-stage biopharmaceutical company dedicated to developing first-in-class and best-in-class therapeutics for treating muscle wasting, metabolic disorders, and neuromuscular diseases, is delighted to announce the successful completion of its Phase 1b multiple-ascending dose (MAD) clinical trial for ALG-801.
ALG-801 is a novel, next-generation ligand trap, which selectively sequesters a subset of TGF-beta family, including growth differentiation factors and activins. The Phase 1b MAD clinical study was conducted in healthy volunteers. This achievement marks a significant milestone in AliveGen’s mission to discover and deliver innovative therapies that address unmet medical needs.
Key Highlights
- Study Design: The Phase 1b MAD study is a randomized double-blind placebo-controlled trial in postmenopausal women.
- Safety Profile: ALG-801 was found to be safe and well-tolerated across all dosing cohorts.
- Efficacy Signal: ALG-801 exhibited linear pharmacokinetic (PK) exposures, along with dose-dependent and statistically significant pharmacodynamic (PD) responses. These included muscle gain, fat loss, and increased insulin sensitivity, as well as a dual anabolic/anti-resorptive effect on bone. These findings highlight ALG-801’s therapeutic potential in treating a wide range of medical conditions, such as muscle wasting, obesity, cardiometabolic disorders, neuromuscular diseases, chronic kidney disease, liver steatosis and fibrosis, and osteosarcopenia in old age.
- Differentiated Mechanism: By selectively inhibiting multiple ligands of the Smad2/3 signaling pathway, ALG-801 demonstrates an outstanding efficacy and metabolic effect.
- Next Step: Following the successful Phase 1b MAD study, AliveGen plans to advance ALG-801 to Phase 2 clinical development.
“We are grateful to the healthy volunteers and healthcare professionals who actively participated in our Phase 1b clinical trial,” said HQ Han, MD, PhD, CEO of AliveGen. “We are thrilled with the excellent dose-response data on muscle gain, fat lose, and insulin sensitization from our Phase 1b MAD study. ALG-801, an innovative and powerful therapeutic approach, holds significant promise for addressing unmet needs across a diverse range of diseases. We look forward to advancing ALG-801 into Phase 2 clinical development.”
About AliveGen
AliveGen USA Inc. is a privately held biopharmaceutical company located in Thousand Oaks, California. The company is focused on the discovery and development of next-generation biotherapeutics to
treat the debilitating muscle wasting and loss of functional capacity as occurs in various metabolic disorders, neuromuscular diseases, and age-related frailty.
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