Levacetylleucine

New data for Genmab and AbbVie’s bispecific antibody Epkinly wowed analysts and could set the partners up for an expanded approval in diffuse large B cell lymphoma; deals have flowed steadily this week, including Novartis’ nearly $8 billion bet on mRNA drugs with China’s Abogen Biosciences; and could Sarepta Therapeutics turn things around from last year’s annus horribilis? > Listen on Spotify > Listen on Apple Products > Listen on Amazon Music > Listen on iHeart > Watch on YouTube Genmab and AbbVie’s bispecific antibody Epkinly could set a new standard of care in diffuse large B cell lymphoma. Approved in May 2023 for patients who have undergone at least two prior lines of systemic treatment, new late-stage data speak to the drug’s use in frontline treatment. In the Phase 3 study, Epkinly plus R-CHOP— a five-drug regimen based on Roche’s rituximab—generated a 51% reduction in risk of disease progression or death, leading to effusive analyst commentary about the “highly differentiated” results, comparing the drug to Roche’s Polivy and Incyte’s Monjuvi. On the deals side of biopharma, this past week has seen considerable action. Just weeks after securing expanded approval for the rare disease drug Aqneursa, IntraBio has exited in a $2 billion buyout by Japan’s Shionogi. First cleared by the FDA in September 2024 for Niemann-Pick disease type C, Aqneursa is now also approved as the first ever treatment for ataxia-telangiectasia, a fatal disease that involves the progressive degeneration of the cerebellum. Shionogi saw the value in the drug, for which Jefferies analysts project global peak sales of $1 billion. Meanwhile, Novartis has bet up to $7.8 billion on Abogen Biosciences and an mRNA-based in vivo T cell engager designed to deplete disease-causing B cells. Novartis was attracted to Abogen because of its differentiated proprietary RNA platform and pioneering work in mRNA-encoded T cell engagers and other RNA-encoded therapeutic modalities, a spokesperson told BioSpace on Monday. In addition, Eli Lilly is expanding its relationship with Gate Bioscience, putting some $870 million more on the line after first striking an alliance last year worth up to $856 million, and CSL is risking up to around $1.6 billion to partner with Alentis Therapeutics on drugs for rare and progressive diseases affecting the kidneys and liver. Finally, Sanofi and Regeneron have renewed their longstanding collaboration with a $1 billion upfront payment in an effort to develop a successor to their blockbuster Dupixent. Regeneron will have the chance to collect up to $7 billion more in biobucks as the two work on four next-gen Regeneron antibodies for immune-mediated conditions. Over at Sarepta Therapeutics, things are looking up —at least according to Jefferies analysts. Last year, Sarepta faced a very public saga over its Duchenne muscular dystrophy gene therapy Elevidys after multiple deaths were associated with the vector technology that underlies the treatment. Last week, the biotech shared new data at the World Muscle Society’s annual meeting in Japan, highlighting Elevidys’ durable and clinically meaningful benefits in older patients with Duchenne muscular dystrophy. Jefferies also pointed to upcoming catalysts in the form of mid-stage readouts in myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy and an anticipated FDA decision on traditional approval of Sarepta’s exon-skippers, Amondys 45 and Vyondys 53. Finally, check out BioSpace ’s Q3 recaps on M&A and VC activity, and read about who’s hot and who’s not in biotech and pharma , as well as our annual 40 under 40 list of young biopharma leaders who demonstrate the industry’s values of innovation, impact and commitment to improving outcomes for patients.

Shionogi’s potential acquisition of IntraBio follows on from its purchase of Tanabe Pharma’s ALS med, Radicava. Credit: agustin.photo / Shutterstock.com. Japanese pharma company, Shionogi, is set to acquire IntraBio in a multibillion-dollar deal, adding neurological and movement disorder drug, Aqneursa (levacetylleucine), to its portfolio and marking its fortification in the rare disease space. As per the transaction, Shionogi will pay $2bn to acquire all of IntraBio’s outstanding shares, which will see the company secure the worldwide IP and commercialisation rights to Aqneursa – a small molecule drug that acts by addressing metabolic dysfunction in the central nervous system (CNS). IntraBio will become a wholly owned subsidiary of Shionogi Inc., a New Jersey-based subsidiary of Shionogi. Aqneursa was first approved by US regulators to treat neurological manifestations of a rare lysosomal disorder called Niemann Pick disease type C (NPC), which is characterised by the gradual buildup of harmful lipids in the cells, in 2024. Since then, the drug has also secured the EU regulatory greenlight in NPC, as well as the US Food and Drug Administration (FDA) go-ahead in rare genetic movement disorder, ataxia-telangiectasia (A-T). EU regulators are currently reviewing an approval application for Aqneursa in A-T. By adding Aqneursa to its portfolio of medicines, Shionogi would double down on its new-found intentions to break into the rare disease space. This build on its $2.5bn acquisition of Tanabe Pharma’s amyotrophic lateral sclerosis (ALS) medication, Radicava (edaravone) , which came earlier this year. The Japanese drugmaker also hopes to leverage IntraBio’s expertise in the rare disease space to fortify its current pipeline, which includes drugs for conditions with high unmet needs such as Fragile X syndrome, Pompe disease and Jordan’s syndrome, alongside its early-stage programmes for neurodegenerative diseases. According to Nathan McCutcheon, President and CEO of Shionogi Inc, bringing Aqneursa to the company would help it to continue its expansion into the rare disease market across Europe, the US and further afield, building on the “meaningful foundation IntraBio has established with the NPC community,” while delivering the first and only therapy for A-T. “In addition to Aqneursa, the capabilities and resources gained through this acquisition would help to accelerate the development of new treatment options. With this transaction, we deepen our commitment to serving rare disease communities today and delivering innovation tomorrow,” McCutcheon concluded.