LISCure Bioscience Co. Ltd.

Alexion is exploring the potential of Voydeya as a monotherapy. Credit: Sai Thaw Kyar/Shutterstock.com. Alexion, AstraZeneca Rare Disease has received approval from the US Food and Drug Administration (FDA) for Voydeya (danicopan) as an add-on therapy to treat extravascular haemolysis (EVH) in adults with paroxysmal nocturnal haemoglobinuria (PNH). A first-in-class, oral Factor D inhibitor, Voydeya has been developed for patients who continue to suffer from EVH despite treatment with C5 inhibitors such as Ultomiris (ravulizumab) or Soliris (eculizumab). The FDA’s decision is grounded in the positive outcomes of the ALPHA Phase III trial, which demonstrated that Voydeya met its primary endpoint of haemoglobin change from baseline to week 12, alongside all key secondary endpoints. The drug was generally well-tolerated with no new safety concerns. As part of the ALPHA double-blind, placebo-controlled, multiple-dose trial, patients were enrolled and randomised to receive Voydeya or placebo apart from their ongoing Soliris or Ultomiris therapy over 12 weeks. See Also: US FDA grants fast track status to LISCure’s LB-P8 drug for PSC FDA extends review period for Applied Therapeutics’ govorestat NDA It aimed to assess the superiority of Voydeya as an adjunct to C5 inhibitor therapy. The trial included a prespecified interim analysis and an option for a two-year extension period for those completing the initial 24 weeks. Japan already approved Voydeya and the European Union is poised to follow suit. The drug has also achieved breakthrough therapy designation in the US and PRIority MEdicines (PRIME) status in the EU. Alexion CEO Marc Dunoyer stated: “The approval of first-in-class, Factor D inhibitor Voydeya marks an important advancement in the treatment of PNH and builds on our leadership and commitment to bring forward innovation in complement science. “As the ALPHA trial suggests, dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for this subset of patients with EVH, enabling them to continue with proven standard-of-care therapy.” Alexion is also exploring Voydeya’s potential as a monotherapy for geographic atrophy in a Phase II clinical trial. The FDA recently approved Alexion’s Ultomiris to treat adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder.

LISCure expects Phase II study preliminary results in early 2025. Credit: BlurryMe/Shutterstock.com. The US Food and Drug Administration (FDA) has granted fast track designation to LISCure Biosciences’ investigational drug LB-P8, for the treatment of primary sclerosing cholangitis (PSC). With the designation, the company will fast track the development of the drug to fulfill the unmet medical needs of people affected by PSC. LB-P8 is designed to treat inflammation and fibrosis as well as to modulate bile acids. LISCure said the LB-P8 live biotherapeutic product (LBP) is the only one of its kind reported to be in clinical development for PSC, a rare and chronic liver disease with no approved treatment options. The drug previously received orphan drug designation for PSC in 2022. See Also: Morris & Dickson will distribute mpox vaccine Jynneos in the US FDA accepts AstraZeneca-Daiichi Sankyo’s Datopotamab deruxtecan BLA Its safety and key biomarkers have been confirmed in a Phase I study. LISCure plans to conduct a Phase II study across multiple sites in the US and Europe, with preliminary results expected in early 2025. It is also under development for metabolic dysfunction-associated steatohepatitis (MASH). LISCure clinical development head Jiyoung Ahn said: “Receiving fast track designation is a significant milestone in addressing the high unmet medical need for PSC, and it will facilitate the efficient development of LB-P8 by enabling close communication with the regulatory authority. “By having enhanced communication with the regulatory authority, we will expeditiously bring forward a novel therapy option in an area with limited available treatments.” Earlier this year, LISCure secured FDA’s clearance of its Investigational New Drug (IND) application for LB-P8. In the upcoming months, the company plans to treat the first patient and expects topline results for Part 1 in the first half of 2025.

Phase 2 study is underway and LB-P8 is the only live biotherapeutic product currently reported to be in clinical development for the treatment of PSC FDA's Fast Track designation for LB-P8 underlines the urgent need for treatment options to fulfill the unmet medical needs of people affected by PSC SEONGNAM, South Korea, March 29, 2024 /PRNewswire/ -- LISCure Biosciences, a leading clinical-stage biopharmaceutical company focused on discovering and developing innovative microbiome-based therapies, announces the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for LB-P8, LISCure's investigational drug for the treatment of primary sclerosing cholangitis (PSC). PSC is a rare, chronic, cholestatic liver disease with significant unmet medical needs as there are no approved drugs available to treat it. Fast Track designation is designed to facilitate the development and expedite the review of new drugs intended to treat serious conditions and address unmet medical need. Fast Track designation allows for enhanced communication between the FDA and sponsors, with the goal of accelerating the delivery of new therapeutics to patients. LB-P8 is currently undergoing evaluation in a Phase 2 study in patients with PSC. LB-P8 is the only live biotherapeutic product currently reported to be in clinical development to address the needs of individuals with PSC. LB-P8 was granted Orphan Drug Designation for PSC in 2022, and the safety and key biomarkers of LB-P8 have been confirmed in Phase 1 study. LISCure will conduct Phase 2 study in multiple sites across the US and Europe. The preliminary results are expected in early 2025. Based on these results, LISCure will make maximum use of expedited programs to bring LB-P8 to market as quickly as possible. LB-P8 is also being developed for metabolic dysfunction-associated steatohepatitis (MASH). "Receiving Fast Track designation is a significant milestone in addressing the high unmet medical need for PSC, and it will facilitate the efficient development of LB-P8 by enabling close communication with the regulatory authority," said Jiyoung Ahn, Head of Clinical Development, LISCure. "By having enhanced communication with the regulatory authority, we will expeditiously bring forward a novel therapy option in an area with limited available treatments." About LISCure Biosciences LISCure Biosciences Inc. is a clinical-stage company developing microbe-oriented new drugs and drug delivery platforms based on innovative modalities for the areas with high unmet medical needs such as oncology, metabolic diseases, neurosciences, and immunology. LISCure uses single strain, microbial-derived metabolites, and extracellular vesicles (EVs) to create "Next Generation Microbial Therapeutics" and "EV-based Drug Delivery Platforms". LISCure has its own screening platform technology based on a proprietary in-house microorganism bank and is dedicated to developing first-in-class new drugs. For more information on LISCure or LISCure's clinical trials, please visit , or follow us on LinkedIn. SOURCE LISCure Biosciences Inc.