Lion the first beneficiary of NCI’s trial resumption

The suspension of 13 cell therapy studies at the US National Cancer Institute in April cast uncertainty on their corporate partners, but less than three months later, and with little fanfare, five have resumed patient enrolment.

This is good news for Lion Biotechnologies, as the five include all three of the tumour-infiltrating lymphocyte studies that are subject to a co-operative R&D agreement it has with the NCI. Four trials for which Kite Pharma has a separate CRADA remain suspended, but should reopen within three months, an NCI spokesperson told EP Vantage.

This means that the suspensions look to have been less serious than had been suspected at the time. In April the NIH said the move was due to follow-up from an earlier finding of serious problems with sterile manufacturing processes, and concerned an NCI cell therapy lab as well as a facility at the National Institute of Mental Health.

Of course, because CRADAs are arm’s-length arrangements that do not involve a formal licence with a corporate entity, Kite and Lion were able to shrug the suspensions off. Nevertheless, Kite stock was hit amid confusion over disclosure of the halts as well as underwhelming data presented at the AACR meeting (AACR – Mage-A3 double-whammy hits Kite, April 17, 2016).

Lion said the suspensions did not affect its own manufacturing capabilities for tumour-infiltrating lymphocyte (TIL) therapy, as these relied on agreements with Lonza and WuXi AppTec. However, this only told half the story since the suspension concerned the lab of Dr Steven Rosenberg and included the TIL project Lion had accessed under its CRADA.

This now becomes academic, as all three of Dr Rosenberg’s TIL trials reopened for patient enrolment on June 17, said Shannon Hatch, an NCI spokesperson. “We expect the remaining eight trials to reopen for enrolment within three months,” she added.

Lion investors might see this as another line drawn under various simmering problems. The company recently raised $100m in a private placement, and replaced its previous chief executive, Elma Hawkins, with Maria Fardis.

Meanwhile, Kite’s focus remains on filing its lead CAR-T asset, KTE-C19, with the US FDA. Irrespective of the ongoing suspension of the NCI’s Mage-A3 engineered TCR project, Kite’s own studies of this asset are to begin under an IND to be filed later this year.

The other two NCI projects to which Kite has access under a CRADA – a CAR against EGFRvIII and an engineered TCR targeting NY-ESO-1 – also remain suspended for now. Kite has had problems with these, and recently downgraded them both to preclinical status, citing the need for “additional engineering”.

If the NCI’s studies do resume within three months this will not solve such issues, but it should at least remove some of the negative sentiment.

The tables in this story were updated on July 6. To contact the writer of this story email Jacob Plieth in London at [email protected] or follow @JacobPlieth on Twitter