Bepirovirsen

GSK taps Engitix for clues to liver fibrosis regressionSangamo hunts for a lifelineSanofi wins EU nod for Sarclisa SC in myelomaGNI to acquire Ayumi for $280M GSK taps Engitix for clues to liver fibrosis regressionGSK is teaming up with UK biotech Engitix in a bid to identify and validate new targets linked to the regression of liver fibrosis. The deal centres on Engitix's human extracellular matrix (ECM) platform, which integrates tissue-derived models with multi-omics datasets to better capture disease biology.Unlike many fibrosis programmes that focus on halting or slowing progression, the partnership is specifically meant to uncover mechanisms that drive fibrosis reversal. Engitix will build ECM-based disease models and generate high-resolution translational datasets, while GSK has the option to license assays, datasets and targets for further R&D and perhaps eventual commercialisation down the line. Financial terms include up to £44.5 million ($59.4 million) in upfront and near-term payments to Engitix, with potential milestones reaching up to £118 million ($157 million) per target, alongside tiered low-single-digit royalties on future sales.GSK's liver disease pipeline includes the FGF21 analogue efimosfermin acquired from Boston Pharmaceuticals in a deal last year worth up to $2 billion. It is currently in Phase III for metabolic dysfunction-associated steatohepatitis (MASH). The pharma also recently delivered a pair of historic Phase III wins for its hepatitis B candidate bepirovirsen (see – KOL Views Q&A: GSK's bepirovirsen is one small step for HBV patients, one giant leap for HBV therapy).-Anna BratulicSangamo hunts for a lifelineSangamo Therapeutics is weighing its future amid mounting financial strain, despite seeming regulatory progress for its Fabry disease gene therapy isaralgagene civaparvovec (isa-vec). The decision comes "after extensive consideration of the company's pipeline and financial resources." Company shares — listed for over-the-counter trading after being delisted from NASDAQ last month — were down about 20% on Monday."With our best-in-class BLA-ready Fabry gene therapy programme, differentiated zinc finger epigenetic regulation and capsid delivery platforms, and innovative MINT platform for large-scale genomic engineering, we have made the decision to explore alternatives," said CEO Sandy Macrae.Sangamo recently reported a steep loss, with first-quarter revenue of just $1.4 million and a net loss of $31 million. Cash on hand was $27.6 million at the end of March and the company estimated that its runway would only take it into the third quarter of 2026.That strain has been compounded by strategic setbacks over the last few years, including the collapse of high-profile collaborations with Sanofi, Biogen and Novartis, as well as a headquarters closure in Brisbane, California accompanied by layoffs. The company was looking to chart a comeback towards the end of 2024 and last year, the FDA allowed it to file a rolling submission for isa-vec; on Monday, the company said the first two modules have been submitted.-Anna BratulicSanofi wins EU nod for Sarclisa SC in myelomaThe European Commission approved a subcutaneous (SC) formulation of Sarclisa (isatuximab) for multiple myeloma (MM) across all indications currently covered by the anti-CD38 mAb's intravenous (IV) counterpart — making the product the first oncology therapy in the EU administered via an on-body injector for use in outpatient or home settings.The latest approval is backed by the Phase III IRAKLIA study, with results showing that Sarclisa SC achieved an objective response rate of 71.1% compared with 70.5% for Sarclisa IV in patients with relapsed/refractory MM. Meanwhile, in April, the FDA pushed back its decision on Sarclisa SC by three months — assigning a new target action date of July 23.-Pavan KamatGNI to acquire Ayumi for $280MGNI Group agreed to acquire a 100% stake in Ayumi Pharmaceutical from Blackstone-led shareholders, valuing the latter and its pain management portfolio at approximately JPY 44.8 billion ($280 million). The buyout will make the Japanese specialty pharma, which generated sales of JPY 38.5 billion ($240 million) in its latest fiscal year, a wholly owned subsidiary of GNI.Ying Luo, chief executive of GNI, said the deal "marks a monumental milestone in the execution of our long-term strategy to establish ourselves as a cross-Pacific biopharmaceutical powerhouse."The transaction is set to expand GNI's commercial footprint in Japan, with combined fiscal 2026 revenue expected to reach approximately JPY 65.2 billion ($407 million). The integrated entity will focus on five therapeutic areas — fibrosis, pain management, rheumatology, oncology and orthopaedics — and aims to accelerate the introduction of innovative therapies, biosimilars, and internally developed products into the Japanese market.-Pavan Kamat

GSK has announced positive pivotal data for bepirovirsen, its investigational antisense oligonucleotide (ASO) for the treatment of chronic hepatitis B (CHB). Results from the two phase 3 trials, B-Well 1 and B-Well 2, were simultaneously published in the New England Journal of Medicine and presented at the European Association for the Study of the Liver (EASL) congress. Pooled data from both trials showed that six-month treatment with bepirovirsen achieved a statistically significant and clinically meaningful 19% functional cure response rate (233 of 1,220 vs. 0 of 614 in the placebo group) in the overall study population (adults with ≤3000 IU/ml hepatitis B surface antigen (HBsAg) level), meeting the primary endpoint. In a key secondary endpoint, a functional cure rate of 26% (200 of 768 vs. 0 of 393 in the placebo group) was achieved in participants with ≤1000 IU/ml HBsAg level, a group that represents approximately 45% of diagnosed CHB cases globally. The current standard of care typically requires lifelong therapy, with functional cure rates achieved in less than 1% of patients. Functional cure occurs when the hepatitis B virus (HBV) DNA and HBsAg are undetectable in the blood for at least six months after stopping all treatment. This indicates the disease is controlled by the immune system without medication. A loss in HBsAg is also associated with an 89% reduction in risk of liver cancer and a 62% reduction in risk of all-cause mortality. Notably, in an exploratory analysis, 49% of bepirovirsen recipients achieved a quantitative hepatitis B surface antigen (qHBsAg) of ≤100 IU/mL one year after the end of treatment. Medical literature has linked this level of low surface antigen with increased immune control and improved patient outcomes. Moreover, 23% of all bepirovirsen recipients (283 of 1220 vs 0 of 614 in the placebo group) and 31% of bepirovirsen recipients with baseline HBsAg ≤1000 IU/mL (237 of 768 vs 0 of 393 in the placebo group) achieved a sustained HBV DNA lower limit of quantification (