Graphite Bio begins SCD cell therapy trial; Synthetic Biologics' future rests on VCN acquisition

11 Aug 2022
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Graphite Bio has dosed its first sickle cell disease patient with its cell therapy, the Bay Area biotech announced this morning. In its proof-of-concept CEDAR trial, the biotech plans to enroll 15 patients and share early data mid-2023. Graphite joins late on the hunt for a long-term treatment and potential cure for sickle cell disease. Vertex Pharmaceuticals and CRISPR Therapeutics could file for approval for their therapy, dubbed exa-cel, later this year, and bluebird bio said it plans to file a BLA in the first quarter of next year, though its program is under partial clinical hold for pediatric patients. Last month, Editas also dosed its first patient following a clinical hold. Unlike Vertex/CRISPR and Editas, which activate a fetal hemoglobin gene in patients, Graphite hopes to correct the mutated adult hemoglobin. Graphite Bio’s stock $GRPH went up around 8% today, though it is down over 75% since it went public. — Lei Lei Wu Five months after completing its acquisition of VCN Biosciences, Synthetic Biologics’ clinical development path largely rests on the biotech it snapped up for less than $10 million upfront. Instead of focusing on its microbiome and gastrointestinal pipeline, which included the mid-stage drug ribaxamase, the Maryland biotech will move forward with the oncolytic adenovirus platform from its Barcelona-based merger partner, which is centered around oncology. “We have made the strategic decision to rationalize our spend and prioritize our oncology program, which is expected to extend cash runway into the first quarter of 2024,” Synthetic CEO Steven Shallcross said in the company’s quarter earnings update Thursday morning. By the end of this year, Synthetic expects to start a Phase II study of VCN-01 in combo with chemo as a first-line therapy in newly diagnosed metastatic PDAC patients and, in the second half of next year, kick off a Phase II/III clinical trial of the drug as an adjunct to chemo in kids with advanced retinoblastoma. The University of Leeds will also help study the drug in brain tumors this year. For now, the company still expects data from one of its in-house programs, SYN-004, to read out in the second half of this year and then initiate a second cohort of the drug in allo-HCT patients. — Kyle LaHucik Affimed withdrew an IND that it submitted to the FDA just two months ago. After the agency gave feedback on the dose escalation study design, the Heidelberg, Germany-based biotech decided it was better to voluntarily withdraw and instead focus the drug’s clinical development outside the US. The bispecific, known as AFM28, targets CD16A on NK cells and macrophages and CD123 on leukemic blasts and leukemic stem cells. The drug will now enter human trials in the first half of 2023, based on the US withdrawal, the biotech said in its quarterly earnings update Thursday. The original plan was to start a Phase I trial of the drug, tested as a monotherapy, in patients with relapsed/refractory acute myeloid leukemia, the biotech said in June. The goal was to also test it in combination with allogeneic NK cell therapy once a starting dose had been determined. — Kyle LaHucik
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