The successful suitor for Horizon Therapeutics plc is Amgen Inc., which will buy the Dublin-based company for $27.8 billion in cash in what is by far the year’s biggest acquisition. The deal brings successful biologic therapies into Amgen’s portfolio that include Tepezza (teprotumumab), a monoclonal antibody targeting insulin-like growth factor 1 for treating thyroid eye disease, Krystexxa (pegloticase), a pegylated uricase enzyme for treating chronic refractory gout, and Uplizna (inebilizumab), a humanized anti-CD19 B cell for treating neuromyelitis optica spectrum disorder. In late November, Dublin-based Horizon disclosed that it was in “highly preliminary” discussions about interest in a takeover that had been expressed by Amgen along with Janssen Global Services LLC and Sanofi SA. The year’s biggest deal so far had been CSL Ltd.’s acquisition of Vifor Pharma Ltd. for an equity value of $11.7 billion. Right behind that was May’s Pfizer Inc. takeover of Biohaven Pharmaceutical Holding Co. Ltd. for $11.6 billion.

Cytokinetics hoping for good adcom review of heart failure drug

What could be Cytokinetics Inc.’s first approved drug will take center stage tomorrow at a meeting of the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee. Judging from the FDA’s briefing document for the meeting, the spotlight on Cytokinetics’ heart failure drug, omecamtiv mecarbil, could be harsh. While the FDA is asking a solo voting question – the typical “do the drug’s benefits outweigh its risks?” – the agency has scripted several discussion questions ranging from the adequacy of the evidence from the second largest trial ever conducted for a heart failure drug to the potential need for an approved companion diagnostic.

ASH 2022: Affimed sinks on mixed data with innate cell engager in HL, PTCL

Shares of Affimed NV (NASDAQ:AFMD) were trading midday at $1.25, down 79 cents, or 38%, after the company disclosed phase I/II results with its lead innate cell engager, AFM-13, precomplexed with cord blood-derived natural killer (NK) cells in patients with CD30-positive relapsed or refractory Hodgkin and non-Hodgkin lymphoma (HL). AFM-13 plus NKs yielded 77% complete response (CR) and 97% objective response rates (ORR, n=31) in HL at the recommended phase II dose. Also at the American Society of Hematology meeting, Heidelberg, Germany-based Affimed offered monotherapy data with AFM-13 from a phase II trial in peripheral T-cell lymphoma (PTCL), showing 10% CR and 32% ORR (n=108), with a 2.3-month median duration of response.

ASH 2022: One-time infusion CSL’s Hemgenix gene therapy shows durable response for hemophilia B

After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding. Presented at the American Society of Hematology (ASH) annual meeting on Dec. 10, data showed 24-month results reinforced the safety of treatment, with no serious treatment-related adverse effects. The gene therapy for treatment of adults with hemophilia B was approved by the FDA in late November under a priority review pathway, making it the first-ever gene therapy treatment option for patients with hemophilia B.

ASH 2022: First patient treated with base-edited donor T cells

Doctors at Great Ormond Street Hospital London have reported the first successful use of base-edited donor T cells, in the treatment of refractory T-cell lymphoblastic lymphoma. The case of a 13-year-old girl named Alyssa, whose disease had not responded to chemotherapy and a bone marrow transplant, was presented at the American Society of Hematology meeting in New Orleans on Dec. 11, 2022.

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