Ranok Therapeutics Co. Ltd.

-- Study follows ongoing Phase 1 trial in U.S. and will enroll patients in China to evaluate the first protein degrader developed using Ranok’s innovative CHAMP® technology BOSTON & HANGZHOU, China--(BUSINESS WIRE)-- Ranok Therapeutics, a clinical-stage biopharmaceutical company that is developing a novel approach to targeted protein degradation for the treatment of cancers, today announced the initiation of patient dosing in China for a Phase 1 study of RNK05047, its first protein degrader candidate. The study, which mirrors an ongoing Phase 1 study in the U.S., will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of RNK05047 in Chinese patients with advanced solid tumors or lymphomas. Ranok anticipates preliminary data from both the US and China Phase 1 studies by the end of 2024. “RNK05047, which has been developed using Ranok’s proprietary CHAMP® technology, is the first BRD4-selective protein degrader to have entered clinical testing in China. This extends our ongoing clinical study of RNK05047 in the U.S. to an Asian patient population, which we believe will provide important insights,” said Weiwen Ying, Ph.D, Founder and Chief Executive Officer of Ranok. “RNK05047 degrades BRD4 protein preferentially in tumors, which may potentially lead to improved safety and efficacy. We look forward to sharing initial results from both the US and China studies later this year.” “BRD4 is an epigenetic target of great interest for the treatment of many different cancer types,” said Ning Li, M.D., Professor and Vice President of the National Cancer Center/Cancer Hospital, Chinese Academy of Medical Sciences (CAMS) in Beijing China, as well as Lead Principal Investigator for this study. “The Cancer Hospital is the first and largest cancer hospital in China for the diagnosis and treatment of cancer. We are excited to be participating in this study and are hopeful that RNK05047 will provide an important new therapeutic option for cancer patients.” Additional information can be found at (CTR20233943) and (NCT05487170). About Ranok’s CHAMP® platform Ranok’s proprietary Chaperone-mediated Protein Degradation (CHAMP®) platform takes advantage of the cellular chaperone network, which regulates the folding and stability of proteins, distinguishing it from other targeted protein degradation approaches. CHAMPs have a number of unique advantages, such as the evasion of mechanisms of drug resistance, and are designed to improve safety and efficacy due to the selective targeting of disease tissues. About RNK05047 RNK05047 is a first-in-class, small-molecule, tumor- and BRD4-selective protein degrader that was discovered and developed using Ranok’s proprietary approach to targeted protein degradation, CHAMP®. The bromodomain transcription factor BRD4 is a key epigenetic regulator of oncogenes such as MYC and BCL2 and is involved in diverse cancer types. Phase 1 trials of RNK05047 are currently underway in both the U.S. and China to assess its safety, tolerability and pharmacokinetics, and also include measures of anti-tumor activity and pharmacodynamic readouts as secondary endpoints. Initial results are expected by the end of 2024. About Ranok Therapeutics Ranok is a privately held biopharmaceutical company that is pioneering CHAMP®, an innovative approach to targeted protein degradation for the discovery and development of novel therapeutics. Our R&D team brings deep biological insight and chemistry expertise to rapidly identify and advance CHAMP degraders for a variety of important disease targets, with the goal to create transformative medicines that benefit patients around the world suffering from cancer and other serious diseases. For more information, please visit the company website at or follow us on Twitter (@RanokTx) and LinkedIn.

As the summer winds down, many biopharma companies are starting up new trials and reporting data from existing ones. Here’s a look at last week’s top clinical trial news. 89bio presented data from its Phase II ENTRIGUE proof-of-concept trial of pegozafermin in severe hypertriglyceridemia (SHTG) at the European Society of Cardiology (ESC) Congress 2022. The data, consistent with previous studies, demonstrated that the drug significantly decreased triglyceride levels, reduced atherogenic lipoproteins, and improved liver fat and glycemic control markers in select patients. It was generally well-tolerated with a favorable safety profile. The drug is a specifically engineered glycoPEGylated analog of fibroblast growth factor 21 (FGF21) being developed for the treatment of non-alcoholic steatohepatitis (NASH) and SHTG. Tonix Pharmaceuticals enrolled the first patient in the Phase II PREVAIL trial of TNX-102 SL for a subset of patients with Long COVID syndrome whose symptoms overlap with fibromyalgia. Long Covid, or Post-Acute Sequelae of COVID-19 (PASC), has a long list of symptoms, including multi-site pain, fatigue and sleep disturbance, all of which are associated with central sensitization syndromes like fibromyalgia. TNX-102 SL is a patented sublingual formulation of cyclobenzaprine hydrochloride that allows rapid transmucosal absorption and decreased production of a long half-life active metabolite, norcyclobenzaprine. It is being developed for Long Covid, fibromyalgia, PTSD, alcohol use disorder and agitation in Alzheimer’s disease. Belite Bio initiated enrollment for the U.S. Phase III trial of LBS-008 in patients with Stargardt Disease (STGD1), a progressive blinding disease. It is the most common inherited retinal dystrophy that causes blurring and/or loss of central vision in adults and children. LBS-008 is an oral, small molecule retinol binding protein 4 (RBP4) antagonist that selectively decreases the delivery of vitamin A (Retinol) to the eye, resulting in a decrease of toxic vitamin A byproducts implicated in the onset and progression of the disease. Immatics treated the first patient in its Phase Ib expansion cohort C of IMA203CD8 for hard-to-treat solid tumors. The drug is a second-generation TCR-T monotherapy where a proprietary CD8aß co-receptor is added to PRAME-specific IMA203 T cells. The CD8 co-receptor plays a key role during T cell antigen recognition and activation, allowing the engagement of CD8 and CD4 T cells in their attack against cancer cells. PRAME refers to a melanoma antigen preferentially expressed in tumors. Arcutis Biotherapeutics completed enrollment of its INTEGUMENT-2 pivotal Phase III trial of roflumilast cream 0.15% in adults and children with atopic dermatitis (AD). The cream is a once daily topical formulation of roflumilast, a potent and selective phosphodiesterase type 4 inhibitor (PDE4). The company also wrapped enrollment in an identically designed pivotal Phase III trial, INTEGUMENT-1, earlier in August. Topline data from both trials is expected by the end of this year. Apellis Pharmaceuticals announced topline data at 24 months, demonstrating increased effects over time with intravitreal pegcetacoplan. The results were from the Phase III DERBY and OAKS trials of the drug, a targeted C3 therapy, in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Both monthly and every-other-month pegcetacoplan demonstrated clinically meaningful reduction in GA lesion growth from baseline compared to sham. Between months 18-24, the treatment effect accelerated compared to the previous six months. Ranok Therapeutics initiated patient dosing in the U.S. for the Phase I/II CHAMP-1 trial of RNK05047 for advanced solid tumors or diffuse large B-cell lymphoma. The drug is the first based on the company’s CHAMP technology and the first BRD4 protein degrader to enter clinical testing. BRD4 is a master regulator of oncogenes found in multiple cancer types. The CHAMP, or Chaperone-mediated Protein Degrader/Degrader, platform leverages the cellular chaperone network, which regulates the folding and stability of proteins. Anthos Therapeutics initiated its second Phase III trial of abelacimab for patients at risk of thrombosis and are diagnosed with gastrointestinal/genitourinary (GI/GU) cancers. These cancers come with a higher risk of bleeding than other cancers. Abelacimab is a novel dual-acting human monoclonal antibody targeting both Factor XI and Factor XIa. In the MAGNOLIA trial, monthly treatment with abelacimab is being compared to once daily injection of dalteparin, the current standard of care anticoagulant for patients with GI/GU cancers. TG Therapeutics published the results from the ULTIMATE I and II Phase III trials of ublituximab in patients with relapsing forms of multiple sclerosis. The results were published in The New England Journal of Medicine. The drug is an anti-CD20 monoclonal antibody. The studies found that the treatment, compared to teriflunomide, resulted in significantly lower annualized relapse rates, a decrease in the total number of MRI-detectable lesions, and improved rates of patients achieving no evidence of disease activity (NEDA). Pfizer reported its Phase III trial for its respiratory syncytial virus (RSV) vaccine demonstrated efficacy in 85.7% of adults over 60 years of age with a more severe form of the infection. The RENOIR trial studied the company’s prefusion F vaccine candidate (RSVpreF). Approximately 37,000 patients participated globally.

Aug. 24, 2022 12:00 UTC Study will evaluate the first protein degrader developed using Ranok’s innovative CHAMP™ technology in patients with advanced solid tumors or diffuse large B-cell lymphoma (DLBCL) BOSTON & HANGZHOU, China--(BUSINESS WIRE)-- Ranok Therapeutics, a clinical-stage biopharmaceutical company that is developing a novel approach to targeted protein degradation for the treatment of cancer and other serious diseases, today announced the initiation of patient dosing in the U.S. for a Phase 1/2 study of RNK05047. The trial, entitled CHAMP-1, will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of RNK05047 in patients with advanced solid tumors or diffuse large B-cell lymphoma (DLBCL). Ranok anticipates preliminary data from the study in the second half of 2023. “RNK05047 is Ranok’s first therapy based on our proprietary CHAMP™ technology, as well as the first BRD4 protein degrader in the pharmaceutical industry to enter clinical testing,” said Weiwen Ying, Ph.D., Founder and Chief Executive Officer of Ranok. “RNK05047 is designed to selectively degrade BRD4 protein preferentially in tumors, thereby improving safety and efficacy, which differentiates it from other investigational therapies such as non-selective BET inhibitors.” “The BET bromodomain transcription factor BRD4 is a master regulator of oncogenes involved in diverse cancer types,” said Manuel Hidalgo Medina, M.D., Ph.D., Chief of the Division of Hematology and Medical Oncology at Weill Cornell Medicine and New York-Presbyterian/Weill Cornell Medical Center, Associate Director of Clinical Services at the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine and a site Principal Investigator in the trial. “We are pleased to be participating in this trial and are hopeful that RNK05047 will provide a beneficial new therapeutic option for patients.” Additional information on this clinical trial (NCT05487170) can be found at . About Ranok’s CHAMP™ platform Ranok’s proprietary Chaperone-mediated Protein Degradation/Degrader (CHAMP™) platform and Chaperone-Tether Library™ are based on our founders’ extensive backgrounds researching protein homeostasis. CHAMP™ technology takes advantage of the cellular chaperone network, which regulates the folding and stability of proteins, distinguishing it from other targeted protein degradation approaches. CHAMP™ has a number of unique advantages, such as the evasion of mechanisms of drug resistance, and is designed to improved safety and efficacy due to the selective targeting of disease tissues. About RNK05047 RNK05047 is a first-in-class, small-molecule, tumor- and BRD4-selective protein degrader that was discovered and developed using Ranok’s proprietary approach to targeted protein degradation, CHAMP™. The bromodomain transcription factor BRD4 is a key regulator of oncogenes such as MYC and BCL2 and is involved in diverse cancer types. CHAMP-1 is a Phase 1/2 trial of RNK05047 currently underway in the U.S. that will assess its safety, tolerability and pharmacokinetics, and also includes measures of anti-tumor activity and pharmacodynamic readouts as secondary endpoints. Preliminary data is expected from the trial in the second half of 2023. About Ranok Therapeutics Ranok is a privately held biopharmaceutical company that is pioneering CHAMP™, an innovative approach to targeted protein degradation for the discovery and development of novel therapeutics. Our R&D team brings deep biological insight and chemistry expertise to rapidly identify and advance CHAMP™ degraders for a variety of important disease targets, with the goal to create transformative medicines that benefit patients around the world suffering from cancer and other serious diseases. For more information, please visit the company website at or follow us on Twitter (@RanokTx) and LinkedIn.