Bluebird sends blood disorder drug to FDA for approval; CG Oncology enters collaboration with Roche for Tecentriq
Bluebird bio announced it completed the rolling submission of its BLA to the FDA for betibeglogene autotemcel gene therapy.
The therapy, designed for patients with beta-thalassemia who require regular red blood cell transfusions, was previously granted breakthrough therapy designation for treating transfusion-dependent beta-thalassemia (TDT). If approved, beti-cel will be the first hematopoietic stem cell ex-vivo gene therapy for patients in the US.
“We look forward to working with the FDA on its review of this BLA as we realize the promise that one-time gene therapies hold for patients,” said new bluebird president Andrew Obenshain in a statement.
Bluebird was in the news recently as the previous CEO, Nick Leschly, branched off to lead a new company in 2seventy, splitting the gene therapy and rare disease and gene therapy pipeline from its efforts in oncology.
The BLA submission for beti-cel is based on data from two Phase III studies and Phase I/II studies with a combined 63 patients treated as of March 9 — the most recent update on the timeline. The data include two patients with up to seven years of follow-up, eight with at least six years of follow-up and 19 with at least five years of follow-up. — Paul Schloesser
CG Oncology enters a collaboration with Roche’s Tecentriq
California biopharma CG Oncology has entered into a clinical trial collaboration agreement with Roche to evaluate combining CG’s oncolytic immunotherapy CG0070 with Roche’s PD-(L)1 drug Tecentriq.
The Phase I/II trial will evaluate safety and efficacy of this combination in patients with various advanced solid tumors yet to be disclosed. CG Oncology will sponsor the study and Roche will provide Tecentriq.
CG0070, CG Oncology’s lead I/O candidate, is in an ongoing Phase III monotherapy study and has been administered in over 100 patients for non-muscle invasive bladder cancer. CG0070 is based on a modified adenovirus type 5 backbone and is designed to destroy various cancer cells, including bladder tumor cells, through a defective retinoblastoma pathway.
“We are pleased to enter into this collaboration agreement with Roche,” said CG CEO Arthur Kuan in a statement. “We are excited to initiate this important clinical trial with our lead oncolytic immunotherapy, CG0070, in combination with atezolizumab to evaluate the potential synergy of these two immunotherapies in cancer,” Kuan added.
Additional details of the collaboration were not disclosed. — Paul Schloesser
Months after its public debut, Celularity strikes a development deal with Oncternal
Oncternal Therapeutics is joining forces with Celularity and its placental-derived cells to develop cell therapies targeting ROR1, the companies said Monday.
Though they’re keeping the terms of the deal under wraps, the duo say they will also explore the use of Oncternal’s Phase II monoclonal antibody cirmtuzumab in combination with Celularity’s NK cells. Specifically, they’ll be looking at cirmtuzumab and CYNK-101, a placental-derived NK cell therapy that has been engineered to work together with therapeutic antibodies. Celularity will also evaluate ROR1-targeted CAR-NK and CAR-T cell therapies as extensions of its CYNK and CyCART programs, respectively.
“Our research studying ROR1 suggests the potential for a range of new targeted therapeutics, capable of addressing a wide variety of both solid tumors and hematological malignancies,” James Breitmeyer, founder and CEO of Oncternal, said in a statement.
Celularity CEO Bob Hariri steered the company to Wall Street a few months ago, merging with a SPAC called GX Acquisition Corp. The deal gave the company $138 million to work with as it pushes its NK cell and T cell therapies deeper into early-stage trials. Oncternal also jumped onto Nasdaq by way of a reverse merger, combining with GTx back in 2019. — Nicole DeFeudis