Latest news with #MRI-PDFF
Business Wire
16 hours ago
- Health
- Business Wire
PRD Therapeutics宣布啟動PRD001的首例人體研究
東京--(BUSINESS WIRE)--(美國商業資訊)-- PRD Therapeutics, Inc.是一家臨床階段公司，專注于開發靶向純合子家族性高膽固醇血症(HoFH)和代謝功能障礙相關脂肪性肝病(MASH/MASLD)的新型脂質代謝調節劑。該公司今日宣布，近期已啟動PRD001的首例人體(FIH)臨床試驗給藥。PRD001是一款同類首創的SOAT2（原稱ACAT2）選擇性抑制劑。 PRD Therapeutics執行長兼共同創辦人Kanji Hosoda博士表示：「我們很高興啟動PRD001臨床試驗的給藥。此前已展開過多項針對SOAT1/2雙重抑制劑或SOAT1選擇性抑制劑的臨床試驗，但這是第一個針對SOAT2選擇性抑制劑的臨床試驗。多項關於SOAT1或2基因敲除小鼠的研究結果已發表，顯示僅敲除或抑制SOAT2對證明安全性和有效性至關重要。PRD001是全球第一個且唯一的SOAT2選擇性抑制劑，可望也在人體中展現安全性和有效性。我們的臨床前動物模型（LDL-R KO小鼠；HoFH模型，以及高脂飲食誘導的MASH模型小鼠）顯示，PRD001可降低血液和肝臟脂質水準，抑制脂肪肝和動脈粥樣硬化的進展，且無不良事件。對於LDL受體活性缺失或極低的HoFH病患，PRD001可望成為同類第一個有效且安全的口服療法。」 該FIH 1期研究旨在評估PRD001在成年健康志願者中的安全性、耐受性、藥物動力學及早期療效跡象（LDL-C降低效應，以及使用MRI-PDFF檢測的肝臟脂肪量化值）。更多資訊可在 NCT07034183 查詢。 關於PRD001 PRD001是同類首創的口服小分子SOAT2選擇性抑制劑。它透過單一藥物獨特控制脂質代謝的三大關鍵途徑：肝臟膽固醇合成、小腸膽固醇吸收以及血液LDL-C攝取，從而強效降低血液LDL-C水準，且不依賴LDL受體。 本研發專案由日本醫療研究開發機構(AMED)透過 製藥初創企業生態系統強化計畫 提供支援（專案名稱：「同類首創口服脂質代謝調節劑PRD001的開發及脂質代謝紊亂的POC驗證」）。 免責聲明：本公告之原文版本乃官方授權版本。譯文僅供方便瞭解之用，煩請參照原文，原文版本乃唯一具法律效力之版本。
Business Wire
20 hours ago
- Health
- Business Wire
PRD Therapeutics Announces Initiation of First-in-Human Study for PRD001
TOKYO--(BUSINESS WIRE)--PRD Therapeutics, Inc., a clinical stage company focused on the development of novel lipid metabolism regulators targeting homozygous familial hypercholesterolemia (HoFH) and metabolic dysfunction associated fatty liver disease (MASH/MASLD), today announced that the company recently initiated dosing in a First-in-Human (FIH) clinical trial of PRD001, a first-in-class SOAT2 (formerly known as ACAT2) selective inhibitor. 'We are excited to initiate dosing in this clinical trial of PRD001. Many clinical trials have been conducted on SOAT1/2 dual or SOAT1 selective inhibitors, but this is the first clinical trial of an SOAT2 selective inhibitor' said Kanji Hosoda, Ph.D., CEO and co-founder of PRD Therapeutics. 'Several results with SOAT1 or 2 knockout mice have been published, suggesting that knocking out or inhibiting only SOAT2 is crucial to demonstrate safety and efficacy. PRD001 is the world's first and only SOAT2-selective inhibitor and is expected to exhibit safety and efficacy in humans as well. Our preclinical animal models (LDL-R KO mice; HoFH model, and high-fat diets induced MASH model mice) have shown that PRD001 lowers blood and liver lipids and suppresses the progression of fatty liver and atherosclerosis with no adverse events. PRD001 has the potential to be the first-in-class effective and safe oral therapy for HoFH patients suffering from no or extremely low LDL receptor activity.' This FIH Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, and the signs of early efficacy (LDL-C lowing effect, and liver fat quantitative value using MRI-PDFF) of PRD001 in adult healthy volunteers. More information can be found at NCT07034183. About PRD001 PRD001 is a first-in-class, orally available small molecule SOAT2-selective inhibitor. It uniquely controls three key pathways of lipid metabolism; cholesterol synthesis in the liver, cholesterol absorption in the small intestine, and uptake of blood LDL-C with a single drug, leading to a potent reduction in a blood LDL-C level independent of the LDL receptor. This research and development is supported by Japan Agency for Medical Research and Development (AMED) under Strengthening Program for Pharmaceutical Startup Ecosystem (Project title: " Development of First-in-class oral lipid metabolism regulator PRD001 and POC obtained for lipid metabolism disorder ").
Business Wire
16-06-2025
- Health
- Business Wire
D&D Pharmatech Announces Positive Phase 2 Results for DD01 in MASH with Robust Reductions in Liver Fat Accompanied by Improvements in Liver and Metabolic Health
GYEONGGI-DO, South Korea & GAITHERSBURG, Md.--(BUSINESS WIRE)--D&D Pharmatech, Inc. (D&D) (KOSDAQ: 347850), a clinical-stage biotechnology company developing breakthrough treatments for liver and metabolic diseases, today announced positive results from DD01-DN-02 study, an ongoing 48-week Phase 2 trial designed to evaluate the efficacy and safety of DD01 (a once-weekly dual GLP1/glucagon receptor agonist) in 67 overweight/obese subjects with MASH. DD01 treatment was initiated with two weeks of dosing at 20 mg, followed by the 40mg once-weekly maintenance dose. Results of a planned 12-week assessment of safety and efficacy revealed DD01 was well tolerated, and the study's primary endpoint was met. Following a 1:1 randomization of 40mg DD01 and placebo, 75.8% of subjects treated with DD01 achieved at least a 30% reduction in liver fat, 72.7% of subjects achieved greater than 50% reduction in liver fat, and 57.6% of subjects achieved greater than 70% liver fat reduction (in each case, with p < 0.0001). DD01 achieved a mean reduction of liver fat content of 62.3% vs 8.3% for placebo at 12 weeks of treatment, and 48.5% of DD01 subjects achieved normalization of liver fat fraction (defined as liver fat fraction of 5% or less by MRI-PDFF). No placebo subject achieved normalization. Treatment related reductions in Non-invasive markers of MASH progression were used to evaluate subjects at baseline and after 12 weeks of treatment. DD01 treatment was associated with statistically significant improvements in liver stiffness (MRE), pro-C3 levels, and ELF score. Additional Endpoints Twelve-week weight loss data were encouraging. While placebo-treated subjects had no significant weight loss, 42.4% of DD01-treated subjects achieving a greater than 5% reduction in weight. Also encouraging were the results of HbA1c testing. While the study population was not selected to be diabetic, following 12 weeks of DD01 treatment, HbA1c reduction was statistically significant compared to placebo. Safety Results DD01 was well tolerated. Gastrointestinal (GI) side effects were most common, generally mild to moderate, but transient and manageable. To date, only 3 subjects have discontinued treatment due to GI-related adverse events. Implications Seulki Lee, Ph.D., President and Chief Executive Officer of D&D Pharmatech, stated, 'We have remarkably positive results for DD01 after only 12 weeks of treatment in MASH. The magnitude of improvements is equivalent to what has been achieved only after longer-term treatment with FGF and GLP-1 based drugs already validated with histology data in MASH.' 'Considering the combination of liver and metabolic benefits and the favorable tolerability profile, DD01 has the potential to provide patients and physicians with a MASH treatment that is easy to manage, encourages weight loss and is diabetes friendly.' Mazen Noureddin, MD, MHSc, Professor of Medicine at Houston Methodist Hospital; Co-Chairman of the Board, Summit & Pinnacle Clinical Research; Director, Houston Research Institutes, commented, 'The degree of liver fat reduction with DD01 is striking, with nearly three-quarters of patients achieving at least a 30% reduction and almost half reaching normalization within just 12 weeks. The observed improvement in liver stiffness by MRE, though early, adds further support to the biological activity of this dual GLP-1/glucagon approach, which is designed to act directly on the liver.' 'These consistent MRI-PDFF results provide strong confidence that DD01 has the potential to meet both key regulatory endpoints - MASH resolution and fibrosis improvement - as the program advances. Coupled with favorable metabolic effects and a very low treatment discontinuation rate, DD01 is emerging as a well-differentiated and promising therapy, both within its class and compared to other potent agents in development.' Additional data will be presented at upcoming medical meetings. About DD01 DD01 is a once-weekly dual GLP1/glucagon receptor agonist with a half-life of 7-8 days in obese/overweight patients with T2D and MASLD. Following a Phase 1 study that showed DD01 rapidly reduced liver steatosis, improved glucose tolerance, and encouraged weight loss in obese subjects with MASLD and type 2 diabetes, FDA granted DD01 Fast Track Designation for the treatment of MASH. Current Phase 2 results confirm and extend these findings revealing additional benefits in patients with MASH. The effect of DD01 is consistent with its dual-agonist pharmacology. Clinical results show the effects of DD01 are remarkably rapid. DD01 achieves robust results rapidly and with tolerability comparable to other validated MASH treatments. The GLP1:glucagon potency ratio for DD01 is 11:1. GLP-1R potency is likely an important driver in glucose management for diabetic patients and in weight loss. Importantly, with this ratio, DD01 is clearly also a potent glucagon receptor agonist. Substantial liver fat reduction was observed after only 4 weeks in obese subjects with MASLD (Phase 1). Liver fat reduction by DD01 is clearly not secondary to weight loss at these early timepoints, differentiating it mechanistically from the pure incretin approach. In terms of the balance between efficacy and tolerability, the pharmacokinetics of DD01 are also unique. Good tolerability at clinically active doses is likely aided by very slow absorption (tmax = 6 days) and a long half-life (7-8 days), features which make the onset and rise in DD01 exposure gradual. Peaks associated with poor tolerability and troughs associated with diminished efficacy are avoided. As a result, the need for a lengthy titration period is eliminated and therapeutic levels are reached rapidly. A key differentiator for DD01 is the balanced constellation of clinical benefits afforded by a unique pharmacologic and pharmacodynamic signature. Current data suggest DD01 has the necessary attributes to offer patients and physicians a treatment that is easy to use, supports both liver and metabolic health. About the DD01-DN-02 Trial With the support of staff at Summit Clinical Research, the study investigators, and the study participants, the DD01-DN-02 study is fully enrolled and currently ongoing at 12 centers in the U.S. Baseline characteristics were well balanced between both treatment groups with nearly identical mean liver fat fractions (overall mean of 20.7%) and similar body weight (overall mean 99.4 kg) at baseline. Treatment is ongoing and the study includes non-invasive and histologic assessments of MASH resolution and change in fibrosis at 48 weeks. More information about the study is available at under the identifier NCT06410924. About D&D Pharmatech D&D Pharmatech is a clinical-stage biopharmaceutical company focused on developing revolutionary medicines for patients with a number of metabolic, fibrotic, and neurodegenerative diseases. DD01 and TLY012 are in development for fibrotic liver disease (MASH and cirrhosis). TLY012 has completed IND-enabling studies and has been shown to slow and even reverse established fibrosis in models of liver cirrhosis, chronic pancreatitis, and systemic scleroderma. Two products are in development for neurodegenerative diseases. NLY02 is a small molecule BBB penetrating kinase inhibitor targeting glial activation. It is a potent inhibitor of neurodegeneration. NLY01 is also a potent inhibitor of glial activation. It acts through the incretin pathway to reduce neuronal loss and slow functional decline in models of Parkinson's, Alzheimer's, and multiple sclerosis. In a recently completed Phase 2 study conducted in >250 Parkinson's patients, 36-week of treatment with NLY01 resulted in a significant reduction in motor function decline (UPDRS III) in ~1/3 of trial subjects. NLY01 appeared to slow the progressive decline in motor function in newly diagnosed patients who were young (<60) and treatment naïve. D&D Pharmatech's ORALINK technology allows efficient oral uptake of therapeutic peptides. In partnership with Metsera, the company is developing orally active incretin-based peptide drugs for obesity.
