A cardiovascular cell therapy player grabs $54M for a new beginning — where next-gen CAR-T approach features prominently
Before the advent of CAR-T, the term cell therapy conjured up very different images. Sure, there were cancer immunotherapies like Dendreon’s Provenge, but more common were the various flavors of stem cell therapies and cell transplantation.
None of that has gone away, even if they’ve been nudged out of the spotlight — and a little biotech has garnered $54 million (£40 million) to show that both the old and new ideas of cell (and gene) therapy can mesh together to form a special breed of platform company.
Ixaka is launching with quite a bit of history. Formerly Rexgenero, it had licensed a technology known as concentrated multi-cell therapies from the Andalusian Regional Ministry of Health in Seville, Spain to get started in 2015 and last June expanded its focus by bagging France’s aratinga.bio and its targeted nanoparticles — which puts a twist to off-the-shelf CAR-T therapies. Through it all, the company’s headquarters had remained in London.
While the two initial programs — a Phase III multi-cell therapy designed to treat the serious cardiovascular condition chronic limb-threatening ischemia, and a preclinical CD19 cancer drug — may seem unrelated to each other, CEO Joe Dupere believes that they require the same underlying expertise to deliver. That spans legislative knowledge, manufacturing knowhow, regulatory deftness, logistical experience and more.
“So there are a number of factors which are very, very common between cell and gene therapies, together with some specific factors around individual indications,” he told Endpoints News. “So we have capabilities and a good network in the cardiovascular area, in the oncology area, but we very much view that the capabilities we have from a scientific, medical, clinical perspective enable us to design solutions that we can apply to a whole range of different indications but utilizing our fundamental understanding of the biology, medicine, and how do you bring these products to market.”
The new funding, which comes from existing shareholders, will see Ixaka to the end of its ongoing Phase III for REX-001, the cardiovascular product.
To date, more than 150 people had been treated with the autologous therapy, Dupere noted. Clinicians first extract bone marrow from patients, then transported to a facility where the most active cells (stem cells, T cells, monocytes, granulocytes, and so on) are picked out and packaged into a syringe that then gets infused back, targeting the vessels in the lower leg.
The idea is that the cells would then restore the imbalance of cells at those vessels and, according to Ixaka, it had looked promising in Phase II trials. Up to 80% of those smaller groups had responded as hoped, with blood flow restored, “ulcers healed, pain relieved.”
With an interim analysis slated for later this year, Dupere said Ixaka will also plan for a second Phase III — this time not just in Europe but also in the US — as well as scaling up manufacturing and getting ready for commercialization and market access, even though approval isn’t expected until 2024 or 2025.
For the second lead program, dubbed Celtic, the Series A should be sufficient to fuel animal proof of concept. Somewhat like Umoja, a fellow upstart based out of Seattle, Ixaka is aiming to produce a biologic that lets cancer patients make their own CAR-T in vivo.
The tools they’re using are polymer-based nanoparticles that contain a modified, inert lentivirus and a cell-specific promoter as the genetic cargo. Ixaka will start by leveraging the well-known CD19 target to see if it can prove faster, cheaper and even more effective than the first (and, by the time it gets further, likely second) wave of CAR-T products. In both programs, Dupere added, the cost of goods is a fraction of what people currently associate with CAR-T cell therapies.
“That gives you much more scope to go for different indications which wouldn’t potentially support such high levels,” he said — indications that Ixaka is now in the process of “triaging.”