CSL Behring LLC

In a notice with implications for the broader Huntington’s disease development landscape, uniQure N.V. (NASDAQ: QURE) announced that it received final meeting minutes from the US FDA following a Type A meeting held on January 30, 2026, to discuss AMT-130, an investigational AAV5-based gene therapy for Huntington’s disease. The agency stated that it cannot agree that data from the Phase I/II studies of AMT-130 Huntington’s disease program, compared against an external control arm, are sufficient to provide the primary evidence of effectiveness required to support a marketing application. The FDA strongly recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study before seeking approval. FDA’s stated rationale and practical implications The FDA’s position centered on the adequacy of evidence of effectiveness. The agency determined that data generated from the company’s Phase I/II clinical trials, when benchmarked against an external comparator rather than a concurrent randomized control group, did not meet the evidentiary standard required for a Biologics License Application. The FDA’s strong recommendation for a prospective, randomized, double-blind, sham surgery-controlled trial indicates that the agency views a concurrent control arm — including a sham surgical procedure — as necessary to account for potential placebo effects and confounding variables inherent in open-label neurosurgical studies. For uniQure, a Phase III study of the design recommended by the FDA would require substantial additional time and capital. Enrollment of patients willing to undergo randomization to a sham neurosurgical procedure in a progressive, fatal neurodegenerative disease presents its own operational and ethical complexities. The timeline to a potential marketing application is now extended by several years at minimum. Matt Kapusta, CEO of uniQure, stated that while the company did not reach alignment on a submission pathway based on Phase I/II data, the company believes the totality and durability of its data warrant continued dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be applied. The company said it intends to continue engaging with the FDA regarding Phase III development considerations and plans to request a Type B meeting in the second quarter of 2026 to discuss potential study design approaches. The AMT-130 gene therapy Huntington’s disease is a progressive, hereditary neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the HTT gene. The disease leads to motor dysfunction, cognitive decline, and psychiatric disturbances, and is uniformly fatal. AMT-130 is a one-time, adeno-associated virus serotype 5-based gene therapy that delivers an engineered microRNA designed to silence expression of the huntingtin gene through RNA interference. The therapy is administered directly to the striatum via MRI-guided stereotactic neurosurgery. By reducing levels of the toxic mutant huntingtin protein, AMT-130 aims to address the underlying genetic cause of Huntington’s disease rather than merely managing symptoms. The only FDA-approved treatments for Huntington’s disease — tetrabenazine (Xenazine, approved 2008) and deutetrabenazine (Austedo, approved 2017) — are VMAT2 inhibitors that address chorea symptoms only. The AMT-130 clinical trial program therefore targets an area of profound unmet medical need. The miRNA technology underlying AMT-130 originated in academic research on RNA interference approaches to huntingtin gene silencing. uniQure subsequently developed the AAV5-vectored construct internally using its proprietary adeno-associated virus platform. CSL Behring completed its acquisition of uniQure in 2024, which transferred stewardship of the entire uniQure pipeline, including AMT-130, to CSL. The regulatory and development strategy for the program now falls under CSL’s broader organizational structure, though the company continues to operate under the uniQure name for this program.

CSL will continue to explore clazakizumab for preventing cardiovascular events in kidney disease patients, while Lilly can develop and the therapy in “additional indications.”\n Eli Lilly is paying $100 million upfront (PDF) for the right to develop CSL’s phase 3-stage IL-6 antibody that had previously been explored for organ transplant rejection.Australia-based CSL picked up the anti-interleukin-6 monoclonal antibody, called clazakizumab, as part of its acquisition of Vitaeris in 2020. Clazakizumab was designed to treat chronic antibody-mediated organ rejection (AMR), and CSL oversaw a phase 3 trial for the drug as a treatment for AMR in kidney transplant patients.But that trial was terminated early for futility during a planned interim analysis, CSL explained to Fierce.Despite the setback in AMR, the company has continued to assess clazakizumab in a phase 3 study for the prevention of cardiovascular events in patients on dialysis with end-stage kidney disease (ESKD). That trial kicked off in 2022 and is due to wrap up in 2029, according to the trial database. CSL will be hoping this study has better luck than the AMR trial, and the company highlighted to Fierce that “trials evaluating the role of IL-6 in cardiovascular risk are supported by genetic and pharmacological evidence.”“Cardiovascular outcome trials ... have a very distinct rationale from the Imagine study in chronic antibody-mediated rejection,” a spokesperson added.This morning’s deal allows CSL to continue to develop clazakizumab for the specific indication of prevention of cardiovascular events in ESKD patients. Meanwhile, Lilly will be able to develop and commercialize the therapy in “additional indications,” according to a Feb. 18 release.Beyond the $100 million upfront payment, CSL will be eligible for clinical, regulatory and commercial milestones as well as royalties on global net sales—although the companies didn’t offer a ballpark figure for these potential payouts. “This agreement marks a significant step forward in our mission to bring innovative therapies to patients worldwide,” CSL’s head of R&D Bill Mezzanotte said in the release.“Clazakizumab is a promising therapeutic candidate with the potential to significantly impact the treatment landscape for various immuno-inflammatory and cardiovascular conditions,” Mezzanotte added. “Lilly is another patient-focused organization, and we look forward to working with them to maximise the potential of this important medicine.”Buoyed with cash from its blockbuster diabetes and obesity franchise, Lilly has been on a dealmaking spree over recent months, including buying gene editing partner Verve Therapeutics for $1 billion, inflammation biotech Ventyx Biosciences for $1.2 billion and in vivo CAR-T player Orna Therapeutics for up to $2.4 billion.This story was amended Thursday Feb. 19, 2026 to include additional information provided by CSL

CSL will need more than $100 million to recover from its current difficulties , but it’s a start. Eli Lilly has paid the nine-figure sum upfront for rights to the Australian company’s anti-IL-6 monoclonal antibody in unspecified indications. The drug is called clazakizumab, and CSL is developing it for patients with end-stage kidney disease who are on dialysis and at risk of major cardiovascular events. A placebo-controlled Phase 2/3 trial in more than 2,000 patients is ongoing, but will not generate data until 2029. CSL is keeping exclusive rights to develop and sell clazakizumab in this indication. What Lilly is planning for the molecule is less clear. CSL’s head of R&D Bill Mezzanotte said in a Wednesday press release that the drug has potential in “various immuno-inflammatory and cardiovascular conditions,” but the US drugmaker has not set out its development plans. Lilly could make further payments to CSL if the drug hits clinical, regulatory and commercial milestones, as well as royalties on global sales. These amounts were not specified, but CSL’s investors will have to hope the payments are frequent and generous. A week ago, the company released its financials for the six months ended Dec. 31, and they made for tough reading. Its net profit collapsed 81% from the prior year and it took $1.1 billion in write-downs, including for its $200 million upfront licensing agreement with Arcturus Therapeutics for Covid-19 and other vaccines. CSL’s CEO Paul McKenzie had left the day before , with the company’s chair saying McKenzie “didn’t have the skills that we wanted for the future.” IL-6-targeting antibodies have been the subject of dealmaking elsewhere. Last fall, Novartis bought Tourmaline Bio to get a hold of pacibekitug, which had shown promise in atherosclerotic cardiovascular disease. Pacibekitug is also in a mid-stage trial in thyroid eye disease, so perhaps this could be an avenue for Lilly to explore. Novo Nordisk also has an IL-6 candidate, which is called ziltivekimab. CSL obtained clazakizumab through its acquisition of Vitaeris for an unspecified amount in 2020.