CBMG hopes to bring China success to Maryland but is it late to the CAR-T party?

Cellular Biomedicine Group (CBMG) says it hopes to leverage R&D work done in China to expedite clinical trials in the US as it establishes facilities in Maryland.

Last month, Cellular Biomedicine Group, Inc. (NASDAQ: CBMG) announced plans to build a 22,000 square foot facility in Rockville, Maryland. The site will be the first outside China for the firm and is intended to initially support US clinical trials for its cell-based candidates targeting hematological indications.

Speaking on its Q3 2019 conference call, CBMG management told investors the Maryland facility will add global reach to its clinical endeavors, while harnessing the clinical confidence and investigator-initiated trials (IITs) approach developed at the firm’s site in Shanghai, China.

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“A big piece of our strategy that we’ve been very consistent about is about understanding and utilizing the IIT clinical process in China to dose and get early signs of efficacy and safety within our programs,” Derrick Li, Head of Strategy and Investor Relations, told stakeholders.

One of the reasons to bring operations to the US, he continued, is to translate positive results of proof-of-concept studies conducted in China and to prove efficacy of its candidates to the global market.

Tony Liu, CEO, added that having carried out IIT in China, the firm has greater confidence in doing R&D work in the US. “You’ve seen some indications from the efficacy and safety perspective, therefore you spend a bit more upfront [on those candidates] which in China have high efficacy from the efficiency perspective.”

This helps avoid a lengthy program in the US and while he was clear in pointing out that this does not guarantee any kind of success, he said it does help “to mitigate potential risks and provide better chances.”

CAR-T party

CBMG’s pipeline is made up of chimeric antigen receptor T cell (CAR-T) candidates targeting a range of liquid and solid tumors. Its lead candidate is an anti- B-cell maturation antigen (BCMA) program for multiple myeloma, but with patient enrolment only set to begin in January, the firm could be seen to be behind the pack of others in the industry.

Autologous cell-based candidates in the clinic for multiple myeloma include Bluebird and Celgene’s BB21217, Celgene’s JCARH125 and Janssen/Legend Biotech’s LCAR-B38M. Meanwhile, both Cellectis and Allogene have had Investigational New Drug (IND) applications approved for their respective allogeneic – or ‘off-the-shelf’ – anti-BCMA cellular therapies. And other firms are looking to other modalities to target this disease area, including GlaxoSmithKline which says it is planning to file for regulatory approval for its  antibody-drug conjugate (ADC) belantamab mafodotin by the end of this year.

“It may appear that we are slower than some of our peers in a crowded BCMA market, shown by others work in BCMA to obtain INDs ahead of us,” said Liu on the call. “Our approach has been to prioritize and institutionalize distinguishable manufacturing process that is repeatable and can help us ensure quality clinical data.

“Having seen our early clinical feedback, I remain cautiously optimistic that we can be one of the key companies in this indication. Our intention is still to show some early clinical feedback later this year and to continue to update everyone on our clinical progress.”

To support its growth, the firm has said it needs around $82 million. This will be used to develop its programs and to expand capacity and infrastructure. As such, the firm acknowledges it will require significant funding over the next 12 months. For the third quarter 2019, CBMG reported a net loss of $15.9 million (€14.5 million).

Swiss pharma giant Novartis owns a 9% stake in CBMG after investing $40 million in October 2018. Under terms of the deal, Chinese drugmaker is responsible for the production of local supply of Novartis’ CAR-T therapy Kymriah (tisagenlecleucel) from its Shanghai facility.