Latest news with #pharmacokinetic
Business Wire
29-05-2025
- Health
- Business Wire
Drug Farm Reports Data on Safety and Pharmacokinetics from Phase 1 First-in-Human Trial of DF-003 in Healthy Volunteers
ALBANY, N.Y. & SHANGHAI--(BUSINESS WIRE)--Drug Farm, a private biotechnology company utilizing genetics and artificial intelligence technologies to discover and develop innovative, immune-modulating therapies, today announced positive results from a recently completed Phase 1 study (NCT05997641) of DF-003 in healthy volunteers. The data were presented at the American Society for Clinical Pharmacology & Therapeutics (ASCPT) meeting in Washington, DC. DF-003 is a first-in-class, immune-modulating alpha-kinase 1 (ALPK1) inhibitor that targets the root cause of the rare genetic disease, ROSAH syndrome. This randomized, placebo-controlled, double-blinded study evaluated the safety, tolerability and pharmacokinetics of DF-003 in forty-eight healthy volunteers. In the first portion of the trial, single ascending doses were orally administered in five cohorts (randomized 3:1; DF-003: placebo) ranging from 3 mg up to 150 mg. In the second portion of the trial, 50 mg of DF-003 was orally administered daily for fourteen consecutive days in one cohort (randomized 3:1; DF-003: placebo) in eight healthy volunteers. The primary objective of the study was to evaluate the safety and tolerability of DF-003, as well as its pharmacokinetic properties. Key Results Safety: DF-003 was safe at all doses tested with no serious adverse events described. Rates of treatment emergent adverse events (TEAEs) were similar between active and placebo-treated participants, and no TEAEs required dose modification or interruption. Pharmacokinetics: The pharmacokinetic profile of DF-003 was largely dose proportional and the data support additional clinical trials as a once-daily, orally administered drug. 'We are happy to share the results from our Phase 1 study that demonstrate the excellent safety of DF-003,' said Neil Solomons, MD, Head, Clinical Development at Drug Farm. 'We are also pleased that the pharmacokinetic profile of DF-003 leads to trough concentrations in blood that are consistent with efficacy in preclinical models of ROSAH syndrome and cardio-renal disease. The dose ranges explored in this Phase 1 trial will be used in our upcoming proof of concept trials in ROSAH syndrome and cardio-renal disease patient populations.' 'This is a major inflection point for Drug Farm in our quest to advance DF-003, a first-in-class, immune-modulating drug that precision targets the disease-causing mutations of ROSAH syndrome, as well as the excessive activation of the ALPK1 pathway found in cardio-renal disease,' said Henri Lichenstein, PhD, Chief Executive Officer at Drug Farm. 'There are no approved drugs for ROSAH syndrome, and we are excited about the potential of DF-003 to save sight and to ameliorate other debilitating inflammatory symptoms associated with this disease.' About DF-003 DF-003 is a proprietary, first-in-class drug developed by Drug Farm that inhibits the activity of ALPK1 and variants of ALPK1 which cause ROSAH syndrome. DF-003 has therapeutic potential for ROSAH syndrome and cardio-renal disease as the drug has shown efficacy in preclinical models of these disease indications. DF-003 has completed a Phase 1 clinical trial (NCT05997641) in normal healthy volunteers and is now accruing patients with ROSAH syndrome in a Phase 1b trial (NCT06395285). About ROSAH Syndrome ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome is a rare, autosomal dominant autoinflammatory genetic disease named according to the characteristic symptoms exhibited by affected patients (1, 2). Disease-causing mutations in ALPK1 lead to ROSAH syndrome. The most common presenting symptom is a progressive decline in visual acuity that typically begins before 20 years of age, with ophthalmologic examination often revealing optic disc elevation, uveitis, and retinal nerve degeneration (2, 3). Most ROSAH patients also exhibit inflammatory features such as non-infectious low-grade fevers, arthralgia, headaches, and persistently elevated levels of inflammatory cytokines including tumor necrosis factor-α (TNFα), interleukin-6 (IL-6), and IL-1β (3). 1. Tantravahi SK, et al. An inherited disorder with splenomegaly, cytopenias, and vision loss. Am J Med Genet A. 2012;158(3):475-81. 2. Williams LB, et al. ALPK1 missense pathogenic variant in five families leads to ROSAH syndrome, an ocular multisystem autosomal dominant disorder. Genet Med. 2019;21(9):2103-15. 3. Kozycki CT, et al. Gain-of-function mutations in ALPK1 cause an NF-κB-mediated autoinflammatory disease: functional assessment, clinical phenotyping and disease course of patients with ROSAH syndrome. Ann Rheum Dis. 2022;81(10):1453-64. About Drug Farm Drug Farm is a private biotechnology Company developing innovative treatments targeting innate immunity for hepatitis B, heart and kidney diseases, and ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome. Drug Farm's unique IDInVivo platform combines breakthrough technologies in genetics and AI to discover new treatments. IDInVivo technology allows the direct assessment of gene targets in living animals with intact immune systems. Using the IDInVivo platform, Drug Farm has identified novel innate immunity pathways and targets and is now rapidly advancing multiple first-in-class drug candidates into clinical development. For more information please visit:
Yahoo
05-05-2025
- Business
- Yahoo
FDA clears OBI Pharma's IND to begin Phase I/II solid tumour trial
OBI Pharma has received clearance from the US Food and Drug Administration (FDA) for an investigational new drug (IND) application for OBI-902 to commence a Phase I/II trial for treating individuals with advanced solid tumours. The clinical trial is set to commence subject enrolment in the second half of this year. The trop-2 antibody-drug conjugate (ADC), OBI-902, employs the company's GlycOBI ADC enabling technology, offering hydrophilicity and stability. Trop-2 is known to be highly expressed in numerous solid tumour types, including ovarian, breast, and gastric cancers. The ADC is said to carry a topoisomerase I inhibitor payload with a drug-antibody ratio (DAR) of four, designed to target and destroy tumour cells. With its enhanced pharmacokinetic characteristics, this site-specific glycan-conjugated ADC is claimed to have shown antitumor efficacy and a favourable safety profile in several animal models. OBI Pharma CEO Heidi Wang said: 'The impending OBI-902-001 clinical trial intends to evaluate the safety, pharmacokinetics, and preliminary efficacy of OBI-902 in patients with advanced solid tumours. We are very excited to begin dosing patients in our Phase I/II clinical study of OBI-902 later this year.' Since December 2021, the company has licenced Trop-2 targeting antibody from the biotech company Biosion, securing the exclusive global rights, with the exception of China. OBI has global commercial rights to OBI-902, except for the rights pertaining to the antibody in China. The company noted that its GlycOBI platform is compatible with various antibodies, payloads, and linkers. Leveraging EndoSymeOBI and HYPrOBI, the company's enzymatic and linker technologies, respectively, the GlycOBI platform produces homogenous ADCs that are site-specific, under good manufacturing practice (GMP) conditions. The GlycOBI conjugation process preserves the antibody structure, ensuring that the ADC maintains biophysical characteristics akin to the native antibody. OBI's linker technology is claimed to have enhanced the payload conjugation efficiency and minimised aggregation propensity, offering manufacturing advantages for ADC products. Additionally, the company has further developed the ThiOBI platform to enable irreversible cysteine-based conjugation. "FDA clears OBI Pharma's IND to begin Phase I/II solid tumour trial" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
24-03-2025
- Business
- Yahoo
Celcuity Inc. Schedules Release of Fourth Quarter and Full Year 2024 Financial Results and Webcast/Conference Call
MINNEAPOLIS, March 24, 2025 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, today announced that it will release its financial results for the fourth quarter and full year 2024 after the market closes on Monday, March 31, 2025. Management will host a webcast/teleconference the same day at 4:30 p.m. Eastern Time to discuss the results and provide a corporate update. Webcast and Conference Call Information To participate in the teleconference, domestic callers should dial 1-800-717-1738 and international callers should dial 1-646-307-1865. A live webcast presentation can also be accessed using this weblink: A replay of the webcast will be available on the Celcuity website following the live event. About Celcuity Celcuity is a clinical-stage biotechnology company focused on development of targeted therapies for treatment of multiple solid tumor indications. The company's lead therapeutic candidate is gedatolisib, a potent, pan-PI3K and mTOR inhibitor. Its mechanism of action and pharmacokinetic properties are differentiated from other currently approved and investigational therapies that target PI3K or mTOR alone or together. A Phase 3 clinical trial, VIKTORIA-1, evaluating gedatolisib in combination with fulvestrant with or without palbociclib in patients with HR+/HER2- advanced breast cancer is currently enrolling patients. More detailed information about the VIKTORIA-1 study can be found at A Phase 1b/2 clinical trial, CELC-G-201, evaluating gedatolisib in combination with darolutamide in patients with metastatic castration resistant prostate cancer, is ongoing. A Phase 3 clinical trial, VIKTORIA-2, evaluating gedatolisib plus a CDK4/6 inhibitor and fulvestrant as first-line treatment for patients with HR+/HER2- advanced breast cancer is expected to begin enrolling patients in the second quarter of 2025. Celcuity is headquartered in Minneapolis. Further information about Celcuity can be found at Follow us on LinkedIn and Twitter. View source version of release on Contacts: Celcuity Inc. Brian Sullivan, bsullivan@ Vicky Hahne, vhahne@ (763) 392-0123 ICR HealthcarePatti Bank, 513-1284
