Snippet roundup: Icell's CAR-T approach, PCSK9 pricing and Tioma's VC gains

Welcome to your weekly roundup of EP Vantage’s snippets – short takes on smaller news items.

This week, 15-19 August 2016, we had thoughts on the following: Icell turns CAR-T fratricide to its advantage; Pharma executives: Want fair cost-effectiveness analyses? Publish your real-world price; Tioma joins growing CD47 pipeline.

These snippets were previously published daily via twitter.

Icell turns CAR-T fratricide to its advantage

16 August 2016

Delays and the occasional safety scare notwithstanding, CAR-T continues to generate investor interest. One of the latest corporate entrants is Icell Gene Therapeutics, a private company that last week obtained US orphan drug designation for its lead asset, ICG122, an autologous CAR targeting CD4 and intended for use in T-cell lymphomas. Since CD4 is a co-receptor present on T helper cells this raises the issue of cell fratricide – the fact that the CAR-T cells will target each other because some will themselves display CD4. But Wyle Solomon, Icell’s chief executive, wants to turn this to Icell’s advantage. “Because we do not avoid T-cell fratricide we end up with a highly enriched CD8+ population during expansion,” he tells EP Vantage. “ICG122 will be indicated for CD4+ malignancies.” Still, tackling a malignancy with a CAR-T therapy comprising only CD8+ (T cytotoxic) cells could be problematic: Juno’s current thinking is that a 50/50 mix of CD4+ and CD8+ cells is an ideal defined composition CAR-T product. In contrast, Cellectis plans to target T-cell lymphomas with CD38 and CS1-directed CARs that have these antigens deleted on the CAR-T cells via genome editing to avoid fratricide.

For more on CAR-T see our free report.

Pharma executives: Want fair cost-effectiveness analyses? Publish your real-world prices.

17 August 2016

If pharma executives want their drugs to be judged more fairly by independent cost-effectiveness analyses, they would do well to release their real-world US prices. After all, the wholesale acquisition cost (WAC), the number executives are more than happy to publicise when a new drug launches, is never the price given to big payers. Yet that is the fundamental criticism of an analysis of cholesterol-lowering PCSK9 antibodies published in the Journal of the American Medical Association (JAMA). At more than $14,000 a year WAC, the Sanofi/Regeneron drug Praluent and Amgen’s Repatha were estimated to cost more than $400,000 per quality adjusted life year gained in atherosclerotic cardiovascular disease, according to JAMA. That latter number would obviously shrink if the drug companies disclosed their average US discounted price for the drugs.

Tioma joins growing CD47 pipeline

17 August 2016

The $86m series A round landed by Tioma Therapeutics yesterday points to growing interest in CD47 as a new checkpoint inhibition target. Tioma’s antibody programme joins projects from Trillium Therapeutics, Celgene/Inhibrx, Forty Seven, and Symphogen in the research pipeline, although only three have so far been tested in humans. Tioma’s fundraising also ranks as the fourth-biggest in biotech so far this year – and as if to underscore the interest in CD47, it pushes Forty Seven’s $75m series A in February into fifth place.

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