Bepirovirsen

Engitix CEO Giuseppe Mazza, Ph.D., said the deal \"highlights the unique capability of our ECM platform.\"\n GSK’s latest hepatology partnership is posing a less-asked question—how do you encourage the regression of patients’ liver fibrosis?To find the answer, the British Big Pharma is working with Engitix to identify targets involved in this process. Engitix—which, like GSK, is based in London—will use its extracellular matrix platform and disease models to generate multi-omics data sets that could be taken into development.Under the collaboration, GSK will have the option to license anything from those specific data sets to assays and targets that result from the work, with the pharma then taking the lead on further R&D and commercialization.In return, GSK will transfer 44.5 million pounds ($59.5 million) in upfront and near-term payments to Engitix, with potentially 118 million pounds ($157.7 million) in milestone payments tied to each target.GSK already has an asset in its pipeline that’s shown potential against liver fibrosis in the form of efimosfermin. The Big Pharma licensed the once-monthly FGF21 analog from Boston Therapeutics for $1.2 billion upfront a year ago. Meanwhile, GSK’s wider hepatology portfolio could soon be boosted if bepirovirsen, its “functional cure” for hepatitis B, bags an FDA approval in October.Kaivan Khavandi, M.D., Ph.D., GSK’s head of respiratory, immunology and inflammation R&D, said in this morning’s release that the pharma is “excited to collaborate with Engitix to access their differentiated human biology platform. “The ability to combine functional assays with rich multi-omics datasets complements our internal capabilities and network of collaborations in this space, supporting our work to develop differentiated, fibrosis-targeted medicines for patients,” Khavandi added.Engitix is focused on the extracellular matrix (ECM), a non-cellular network in tissues and organs that both provides physical scaffolding for the cellular parts and plays a role starting processes like tissue morphogenesis and homeostasis. While once seen as merely a physical barrier to chemotherapy and immune cells, more recent studies have pointed to a potential role for the ECM in driving fibrosis and solid tumors.The biotech touts its platform as a way to “integrate multi-omics and imaging data to decode the complexity of the extracellular matrix and its impact on disease.” This has been enough to convince Takeda to pen deals with the biotech in 2020 and 2022 for liver disease and inflammatory bowel disease, respectively.Engitix raised a $54 million series A in 2022 and followed that up with a $25 million extension round at the start of this year. The company’s internal pipeline features programs for solid tumors, inflammation and advanced fibrosis.Engitix CEO Giuseppe Mazza, Ph.D., said today’s deal with GSK is “highlights the unique capability of our ECM platform to capture the biology of fibrosis regression in human tissue.“By combining our human-centric disease models with GSK’s development capabilities, we aim to identify and validate high-confidence targets with the potential to transform treatment for liver fibrosis,” Mazza added.

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