This week: Severe spinal cord injury trial shows promise and first-in-class non-gene edited allogeneic CAR-T receives IND
The news highlights:
Clinical hold for Duchenne muscular dystrophy trial after out-of-specification product
Severe spinal cord injury trial demonstrates favorable safety and improved motor function
First-in-class non-gene edited allogeneic CAR-T receives IND
Trace levels of contaminant DNA in raw materials from a supplier has led to a clinical hold being placed Sarepta Therapeutics’ (MA, USA) Phase I/IIa trial for Duchenne muscular dystrophy by FDA. The trace amounts of DNA fragments in third party-supplied plasmid was found during preliminary in-vivo testing by the Research Institute at Nationwide Children’s Hospital (OH, USA), which is working with Sarepta.
“Patient safety is our top priority at Sarepta as we know it is for Nationwide Children’s Research Institute,” stated Doug Ingram, Sarepta’s president and chief executive officer. “We intend to rapidly respond to the FDA’s clinical hold letter, including a commitment to [FDA] to only use GMP-s plasmid. Independently, we will also request a meeting with the Agency to discuss the micro-dystrophin program with the goal of commencing a pivotal trial by year-end 2018.”
Asterias Biotherapeutics, Inc. (CA, USA) has released six month data from its SCiStar trial evaluating AST-OPC1, an oligodendrocyte progenitor cell-based therapy, for severe spinal cord injury. In the study, three escalating doses of AST-OPC1 are being tested in 25 subjects with subacute motor complete (AIS-A or AIS-B) cervical (C4 to C7) spinal cord injury.
“The results from the SCiStar study remain encouraging …and show that the AST-OPC1 cells are successfully engrafting in patients,” stated Ed Wirth, Chief Medical Officer. “We are pleased that 19 of the 22 subjects dosed in Cohorts 2 through 5 have recovered at least one motor level on at least one side through six months of follow-up and four of these subjects have recovered two motor levels during this same period.”
Michael Mulroy, Chief Executive Officer, commented: “There remain no serious or unexpected adverse events related to OPC1 and we believe the durable engraftment of the OPC1 cells, as evidenced in the earlier results as well, is an important prerequisite to seeing sustained clinical benefits. We expect to report the 12 month data on cohorts 3 and 4 later this quarter and the full trial results in early 2019.”
Celyad (Belgium) has announced that FDA has accepted the Investigational New Drug (IND) application for CYAD-01, a non-gene edited allogeneic CAR-T combined with a peptide, TIM, which inhibits TCR signaling. TCR signaling is responsible for Graft versus Host Disease. A trial to evaluate the safety and clinical activity of CYAD-01 in patients with unresectable colorectal cancer in combination with standard chemotherapy will now proceed.
Dr Christian Homsy, CEO of Celyad commented: “We are pleased to have achieved this important milestone. Celyad is the first company clinically evaluating a non-gene edited CAR-T candidate, which, we believe, offers significant advantages over gene edited approaches. Our non-gene edited program consists of a family of technologies aimed at reducing or eliminating T cell receptor (TCR) signaling without requiring genetic manipulation. CYAD-101 is part of a robust clinical development plan, establishing the foundations of next generation CAR-T products.”
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