MiNA Therapeutics Ltd.

Tadekinig alfa is designed to bind and block IL-18. Credit: TheCorgi/Shutterstock. AB2 Bio has made an option and licencing agreement with Nippon Shinyaku , granting the latter the option to obtain exclusive rights to commercialise Tadekinig alfa in the US. This therapy treats a rare paediatric condition, primary monogenic interleukin-18 (IL-18)-driven hyperinflammatory syndrome, which can lead to rapid multiple organ failure and mortality if not treated. AB2 Bio will receive up to $36m in early payments, including a $6m payment upon signing. The company could also receive up to $150m in development milestone payments and commercial milestone and royalty payments potentially amounting to $500m. AB2 Bio is set to continue its preparations for a US biologics licence application (BLA) approval for the therapy for this indication. Upon Nippon Shinyaku exercising its option rights and following AB2 Bio’s receipt of the BLA approval in the US, NS Pharma, Nippon Shinyaku’s wholly owned subsidiary, will undertake the marketing of the therapy. Nippon Shinyaku will have exclusive rights to commercialise Tadekinig alfa for its primary indication in individuals with NLRC4 mutation and X-linked inhibitor of apoptosis protein (XIAP) deficiency within the US. AB2 Bio retains the rights to the therapy for all other indications within the country, including the US Virgin Islands, Guam and Puerto Rico, as well as for all indications in other parts of the world. AB2 Bio CEO Djordje Filipovic stated: “This partnership will accelerate bringing Tadekinig alfa to young patients suffering from this rare and devastating disease and potentially provide a significant improvement in treatment.” Tadekinig alfa is a recombinant human IL-18 binding protein. In April 2024, MiNA Therapeutics entered a research partnership and licensing agreement with Nippon Shinyaku. The collaboration is focused on developing small activating RNA therapeutics for rare neurodegenerative conditions.

LONDON--(BUSINESS WIRE)--MiNA Therapeutics Limited, the pioneer in small activating RNA (RNAa) therapeutics, today presented new pre-clinical data for its lead RNA activation program for the treatment of sickle cell disease, which demonstrated best-in-class activity and safety. The data shared at the 2024 American Society of Hematology (ASH) Annual Meeting in San Diego will also be highlighted at a poster walk session on December 9, 2024, at 12:30 pm PST. MTL-HBG is an RNAa medicine designed to increase transcription of the gamma globin gene (HBG), enabling people with sickle cell disease to produce enhanced levels of fetal hemoglobin (HbF). HbF is a compensatory form of hemoglobin which, when induced to sufficient levels, protects people with sickle cell disease from a range of symptoms including recurrent vaso-occlusive crises and progressive organ damage. “The pre-clinical data establish MTL-HBG as a promising drug candidate in sickle cell disease. The levels of fetal hemoglobin safely induced by MTL-HBG underscore the potential to protect from severe symptoms without the need for gene editing,” said Robert Habib, CEO of MiNA Therapeutics. “We are excited by this compelling evidence, which strongly supports advancement of MTL‑HBG into IND-enabling studies.” MTL-HBG is administered in vivo without the need for harmful pre-conditioning or complex cell engineering. MTL-HBG comprises an RNAa payload, which directly targets the HBG gene and is encapsulated in NOV340 liposomes. A previous RNAa medicine formulated in NOV340 liposomes has demonstrated biodistribution to greater than 60% of erythroid progenitor cells in bone marrow in non-human primates, and preliminary safety and activity in clinical trials. MiNA anticipates advancing MTL-HBG into IND-enabling studies in 2025. The poster entitled, “Small Activating RNA-Mediated Induction of HBG Via Liposome Delivery for In Vivo Treatment of Sickle Cell Disease and Beta-Thalassemia,” highlights how MTL-HBG induced HbF by 3.6-fold as a proportion of total hemoglobin (% HbF) in erythroid progenitor cells derived from human bone marrow tissue. HbF levels induced by MTL-HBG exceeded 20%, a threshold which is widely recognized to protect people with sickle cell disease from vaso-occlusive crises. The activity in erythroid progenitor cells was demonstrated to be durable, pancellular and highly specific. In vivo delivery of MTL-HBG was confirmed in non-human primates as evidenced by an increase in red blood cells containing HbF (F-cells). Monthly administration of MTL-HBG was predicted to achieve 20%-30% HbF in sickle cell patients in an industry-standard model simulation. MTL-HBG is the first drug candidate to emerge from MiNA’s genetic medicine portfolio. The poster presentation is available on the MiNA website: www.minatx.com. About MiNA Therapeutics MiNA Therapeutics is the leader in small activating RNA therapeutics. Harnessing innate mechanisms of gene activation, small activating RNA therapeutics are a revolutionary new class of medicines that can restore normal function to patients’ cells. We are advancing a proprietary pipeline of new medicines with an initial focus on cancer and genetic diseases, while collaborating with leading pharmaceutical companies to apply our technology platform across a broad range of therapeutic areas. Based on our unique know-how in RNA activation we are expanding the possibilities of RNA-based medicine for patients. For more information, visit www.minatx.com.

Boehringer Ingelheim has notched some success in the metabolic-associated steatohepatitis space so far this year. Boehringer Ingelheim is making yet another bet that RNA therapies hold the key to treating metabolic-associated steatohepatitis (MASH). The German drugmaker is paying British biotech Ochre Bio $35 million in upfront and near-term research-based milestone payments to investigate “multiple targets” for chronic liver disease. Top of the list of indications will be MASH, previously known as nonalcoholic steatohepatitis (NASH). Should any of the targets in the “multi-year” collaboration prove fruitful, Ochre is also in line for various clinical, regulatory and commercial milestones as well as tiered royalties, which add up to a deal value north of $1 billion. Ochre’s remit is developing RNA therapies that enhance the liver’s regenerative capabilities in order to halt or reverse chronic liver diseases. It’s far from the first time Boehringer has played in this space—the pharma kicked off the year signing a $2 billion biobucks deal with Suzhou Ribo Life Science and Ribocure Pharmaceuticals to see whether their RNAi drug platform that targets disease-causing genes specifically in liver cells could complement Boehringer’s own MASH expertise. Six years earlier, Boehringer penned back-to-back partnerships with Dicerna Pharmaceuticals and MiNA Therapeutics covering the use of siRNA and small activating RNA to treat MASH. Boehringer put this morning’s deal with Oxford, U.K.-based Ochre in the context of its “commitment to improving the lives of people living with interconnected cardiovascular, renal and metabolic diseases, including MASH cirrhosis.” “Our partnership with Ochre Bio is driven by a shared goal to accelerate the development of new treatments for chronic liver diseases including MASH cirrhosis,” Søren Tullin, Ph.D., global head of cardiometabolic diseases research at Boehringer, said in the release. “Ochre Bio brings to the table unique and exciting capabilities in liver disease research,” Tullin added. “We believe their application of advanced genomics and machine learning coupled with human-centric translational models holds the potential to uncover novel regenerative pathways that will make a meaningful difference in the lives of those affected by chronic liver disease.” Boehringer has already notched some success in the MASH space so far this year, with the company’s Zealand Pharma-partnered glucagon/GLP-1 agonist survodutide demonstrating improvements in disease activity and liver scarring in a phase 2 trial in February.