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Regulation

Nov. 5 Quick Takes: Setback for bluebird sinks shares; plus Cellectis, CRISPR, REMS, Novocure 

Nov 6, 2020 | 12:48 AM GMT

bluebird delays sickle cell BLA
bluebird bio Inc. (NASDAQ:BLUE) lost $9.72 (17%) to $48.83 Thursday after disclosing its BLA submission to FDA for LentiGlobin to treat sickle cell disease will be delayed to late 2022 from 2H21. The biotech cited COVID-19 as well as new CMC requirements from FDA to demonstrate analytical comparability between clinically and commercially produced lentiviral vectors and edited cells. The gene-modified cell therapy is approved in Europe as Zynteglo to treat transfusion-dependent β thalassemia.

Signs of efficacy for Cellectis’ off-the-shelf CAR T
Cellectis S.A. (Euronext:ALCLS; NASDAQ:CLLS) gained 10% to $18.13 on early evidence that its allogeneic CD22-targeted CAR T cell therapy UCART22 may help treat B cell acute lymphoblastic leukemia (ALL) without triggering graft-versus host disease (GvHD). In an abstract released ahead of December’s American Society of Hematology meeting, UCART22 led to an ORR of 40% in the first five patients treated with the off-the-shelf CAR T cell therapy, with two of three patients in the lowest dose arm achieving a CR with incomplete hematologic recovery. Two of the five patients experienced serious treatment-emergent serious adverse events.

CRISPR data at ASH point to durability for ex vivo-edited cell therapy
CRISPR Therapeutics AG (NASDAQ:CRSP) and Vertex Pharmaceuticals Inc. shared data in an ASH 2020 abstract suggesting ex vivo stem cell therapy CTX001 may have durable effects. Five transfusion-dependent β thalassemia patients in the CLIMB THAL-111 study remain transfusion-free for up to 15 months following CTX001 infusion, and two sickle cell disease patients in the CLIMB SCD-121 study remain free of vaso-occlusive crises for up to a year. One patient experienced four severe adverse events related to hemophagocytic lymphohistiocytosis. Shares of CRISPR have gained 19% to $103.34 in the two trading session since the data’s release. 

FDA to publish REMS reviews
FDA is responding to feedback from healthcare providers, researchers, industry members and other federal agencies by proposing to publish a summary of FDA’s review of risk evaluation and mitigation strategy (REMS) assessment reports. The agency is establishing a docket to solicit public comment on the proposal. Comments will be accepted for the next 60 days.

TB vaccine as potential COVID-19 treatment
A Phase II readout from a study by three major Indian institutions has found that Bacille Calmette-Guérin (BCG) raised the proportion of hospitalized COVID-19 patients requiring oxygen who recovered from pneumonia. In the 60-subject randomized, participant-blinded trial, none of the 30 patients who received the tuberculosis vaccine plus standard-of-care treatment died vs. two patients in the SOC alone cohort. The team from the Haffkine Institute for Training, Research and Testing; G. T. Hospital; and BJ Medical College and Sassoon General Hospital reported the data in medRxiv.

Novocure prices $500M note deal 
Oncology company Novocure Ltd. (NASDAQ:NVCR) said it priced $500 million in 0% convertible senior notes due 2025. Initial investors have an option to buy up to an additional $75 million in notes.

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