Life science news 7 September 2026: two US approvals, a UK suspension, a Phase 3 failure and four global deals.
PharmaEssentia won FDA approval for Besremi in essential thrombocythemia, a label expansion for the interferon therapy. The MHRA suspended Avacopan Vifor for new patients after concluding its pivotal trial data can no longer be relied upon. Novartis reported that its Phase 3 Lp(a)HORIZON cardiovascular outcomes trial missed its primary endpoint. Eli Lilly agreed to buy Merida Biosciences for up to $2.875 billion to expand its autoimmune pipeline.
PharmaEssentia has won US Food and Drug Administration approval for Besremi (ropeginterferon alfa-2b-njft) in essential thrombocythemia. The regulator cleared the interferon therapy for adults with the rare blood cancer on 31 August 2026. The decision expands a label under which Besremi was already approved for polycythemia vera. PharmaEssentia describes Besremi as the first new FDA-approved treatment for essential thrombocythemia in nearly three decades. Approval rests on the Phase 3 SURPASS-ET trial, which compared Besremi against anagrelide. Ruben Mesa is principal investigator on the trial and president of Advocate Health’s Cancer National Service Line. He said people living with the disease have had limited treatment innovation for nearly 30 years.
PharmaEssentia said Besremi achieved superior durable response rates compared with anagrelide. It also reduced thromboembolic events over twelve months of treatment, without publishing specific percentages. Besremi carries a boxed warning covering fatal or life-threatening neuropsychiatric, autoimmune, ischaemic and infectious disorders linked to interferon alfa products. Mesa said patients need options that control blood counts while also addressing the underlying disease. Ko-Chung Lin is founder and chief executive of PharmaEssentia. He called the approval a significant advancement reflecting the company’s commitment to myeloproliferative neoplasms.
Eli Lilly has agreed to acquire Merida Biosciences for up to $2.875 billion in cash. The total includes an upfront payment and contingent milestones. The companies announced the deal on 31 August 2026. Merida’s lead candidate, MER511, is a Phase 1 antibody designed to selectively degrade pathogenic autoantibodies while preserving normal immune function. Lilly plans to develop it in Graves’ disease and thyroid eye disease. Francisco Ramírez-Valle is senior vice president of Lilly Immunology Research and Early Clinical Development. He said Lilly is building its pipeline around therapies that meaningfully change the course of disease.
Lilly did not disclose the individual upfront and milestone components that make up the $2.875 billion total. The transaction remains subject to customary closing conditions and is expected to close in the fourth quarter. Lilly said initial Phase 1 data showed robust reductions in pathogenic thyroid-stimulating antibodies. It reported a favourable initial safety profile but gave no specific figures. Adam Townsend, chief executive of Merida, said the company was founded to change how autoimmune and allergic diseases are treated. MER511 remains in early-stage development, so its promise currently rests on Phase 1 data rather than confirmed outcomes.
The Medicines and Healthcare products Regulatory Agency has concluded that Avacopan Vifor no longer has a positive benefit-risk balance. The regulator announced the decision on 1 September 2026. It has suspended the use, supply and sale of the medicine to new patients from that date. Avacopan Vifor, previously known as Tavneos, was authorised for adults with severe, active granulomatosis with polyangiitis or microscopic polyangiitis. It was given alongside rituximab or cyclophosphamide. The MHRA said questions over the integrity of the pivotal ADVOCATE study prompted its review. It concluded that the study can no longer be relied upon to demonstrate the medicine’s efficacy.
Healthcare professionals have been told not to start new patients on the treatment. Existing patients may continue during a six-month managed withdrawal period, ending on 1 March 2027. The MHRA intends to revoke the marketing authorisation on that date. Patients must speak to their specialist before stopping. Julian Beach, MHRA executive director for healthcare quality and access, said patient safety is the agency’s highest priority. He added that the efficacy data is no longer sufficiently reliable to support a positive benefit-risk balance. The UK action follows the European Commission’s revocation of the EU licence in August 2026. The FDA proposed withdrawing US approval of Tavneos in April 2026.
Roche has agreed to license SIM0660, a tri-specific antibody, from Simcere Zaiming in a deal worth up to $1.53 billion. The companies announced the agreement on 1 September 2026. SIM0660 combines a CD3-engaging arm with binding domains targeting the B-cell antigens CD79a and CD19. Roche gains exclusive global rights to develop, manufacture and commercialise the candidate for B-cell-mediated diseases. SIM0660 is in preclinical development, according to trade coverage of the agreement. Renhong Tang is chairman and chief executive of Simcere Zaiming. He said the company is pleased to collaborate with Roche to accelerate the candidate’s clinical development.
The $1.53 billion total includes a $75 million upfront payment. The remaining milestones were not disclosed individually. Tiered royalties of up to double digits on future net sales sit outside that headline figure. Simcere Zaiming designed SIM0660 to induce potent T-cell-mediated cytotoxicity while limiting cytokine release, a common side effect of T-cell engagers. Boris Zaïtra, head of corporate business development at Roche, said SIM0660 represents a potential advancement for patients with B-cell-mediated diseases. The release describes the dual CD79a and CD19 targeting design as a potential first-in-class approach.
In further life science news 7 September 2026, the week also brought an MHRA and NHS sandbox partnership. Two more deals and a Phase 3 setback followed.
The MHRA and Manchester University NHS Foundation Trust have partnered to launch a health innovation sandbox. The regulator and the trust announced the partnership on 2 September 2026. The Manchester Sandbox will deploy and test emerging healthcare technologies in real NHS settings. Its initial focus covers AI-enabled medical devices that identify patients at higher risk of complications from chronic conditions. The sandbox is designed to generate evidence on safety, effectiveness and clinical impact before wider rollout. Participation does not guarantee regulatory approval or NHS adoption. Lawrence Tallon, MHRA chief executive, said AI and other emerging technologies have enormous potential to improve patient care.
Mark Cubbon, the trust’s chief executive, said patients should never have to choose between access and safety. No financial terms were disclosed. The partnership forms part of a wider MHRA effort to modernise how medical technology is evaluated before NHS adoption. It follows separate amendments the government tabled to the Health Bill on 1 September 2026. These cover information sharing, regulatory reform and a future medical device licensing regime. The MHRA said the amendments introduce no immediate changes and would require further consultation. The agency has not yet said which specific AI devices will enter the sandbox first.
Novartis has signed an option and license agreement with Alteogen for its Hybrozyme subcutaneous delivery platform. The deal is worth up to $3.22 billion if all options are exercised and all milestones are achieved. The companies announced it on 2 September 2026. Hybrozyme is built around ALT-B4, or berahyaluronidase alfa, a recombinant human hyaluronidase. It temporarily breaks down hyaluronan in tissue, converting biologics normally given by intravenous infusion into faster subcutaneous injections. The agreement gives Novartis multiple options to obtain exclusive rights across several of its products. Alteogen did not name which medicines are covered.
Alteogen did not disclose a separate upfront figure. It said only that option fees, development and commercial milestones and royalties could together reach $3.22 billion. Tae-Yon Chun, chief executive of Alteogen, said the deal demonstrates the technological strength of the Hybrozyme platform. Alteogen described Novartis as a world-leading pharmaceutical company. Alteogen said the agreement was its fourth licensing deal signed this year, according to its own announcement. The deal adds to a wider run of subcutaneous reformulation agreements across the industry this year.
GSK has licensed HMPL-A830, an antibody-targeted therapy conjugate, from HUTCHMED in a deal worth up to $1.295 billion. HUTCHMED announced the agreement on 3 September 2026. The companies describe HMPL-A830 as a first-in-class conjugate pairing a KRAS small-molecule inhibitor payload with an anti-EGFR antibody. It targets KRAS-altered colorectal, pancreatic and lung cancers. The candidate has not yet entered clinical trials. A Phase 1 study, registered as NCT07718581, is expected to begin in the second half of 2026. HUTCHMED keeps development and commercial rights across mainland China, Hong Kong, Macau and Taiwan.
GSK’s subsidiary takes worldwide rights everywhere else and becomes responsible for later development and commercialisation. The deal includes a $110 million upfront payment and up to $1.185 billion in milestones. Tiered royalties on net sales sit outside that $1.295 billion total. The agreement remains subject to customary closing conditions, including antitrust clearance. Hesham Abdullah is senior vice president and global head of oncology research and development at GSK. He said the dual KRAS-EGFR mechanism has the potential to significantly improve on current standard of care. Johnny Cheng, acting chief executive of HUTCHMED, called the collaboration a significant step for the candidate.
Ionis Pharmaceuticals has won FDA approval for Zanvastro (zilganersen) in Alexander disease. The approval, announced on 3 September 2026, covers paediatric and adult patients. It arrived ahead of a 22 September action date. Ionis describes Zanvastro as the first and only disease-modifying treatment for the rare, often fatal neurological condition. Approval rests on a global Phase 1 to 3 study measuring gait speed with the 10-Meter Walk Test. The pivotal 50 mg dose cohort met the primary endpoint in patients aged five and over. Treated patients held gait speed stable while the control group declined, a least-squares mean difference of 33.3 per cent (p=0.041).
The study also showed improved gross motor function in children aged two to four. This was measured with the Gross Motor Function Measure-88. The most common adverse reactions were vomiting, back pain, cough, headache and post-lumbar puncture syndrome. The prescribing information advises standard-of-care workup if symptoms consistent with aseptic meningitis develop. Amy Waldman is a paediatric neurologist and lead investigator for the study at Children’s Hospital of Philadelphia. She said the approval represents a significant advancement in care. Brett Monia, chief executive of Ionis, called it the company’s first independent launch from its neurology pipeline. Recordati holds rights outside the US, with European and Japanese submissions expected in 2027.
Novartis has reported that the Phase 3 Lp(a)HORIZON trial missed its primary endpoint. The company announced the topline results on 4 September 2026. The cardiovascular outcomes study tested pelacarsen in patients with elevated lipoprotein(a) and established cardiovascular disease. It did not reduce the risk of cardiovascular events compared with placebo. The composite endpoint covered cardiovascular death, non-fatal myocardial infarction, non-fatal stroke and urgent coronary revascularisation requiring hospitalisation. Participants were already receiving guideline-directed treatments, including lipid-lowering and antihypertensive therapies.
Novartis said pelacarsen still achieved lower Lp(a) levels in the study population. The trial enrolled 8,323 patients and missed the primary endpoint in the overall population. Shreeram Aradhye is president of development and chief medical officer at Novartis. He said the results were not what the company hoped for, but that they advance scientific understanding. Elevated Lp(a) is an inherited cardiovascular risk factor with no approved targeted treatment. Novartis holds global rights to pelacarsen under a licence and collaboration agreement with Ionis Pharmaceuticals. Ionis also secured the Zanvastro approval covered above. Novartis intends to present the full trial data at a forthcoming medical congress.
Catch up on the 31 August news roundup.
