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Intellia announced in its fourth-quarter earnings report that Novartis had ended development of sickle cell treatment OTQ923/HIX763. (Getty Images)
Novartis will no longer develop an ex vivo sickle cell disease program that was part of an older deal with Intellia, and the gene editing biotech’s CEO John Leonard, M.D., thinks he knows why.
“We’ve always believed that the future lies with the in vivo approaches, and that’s been a focus of the work that we do,” Leonard said. “I’m sure they looked at the ex vivo space and may have had some of the same realizations that we had some years ago.”
Leonard, of course, said he wasn’t completely sure why Novartis opted to cut the program, but noted that the Big Pharma is undergoing a broad pipeline reorganization.
Novartis confirmed just that in an emailed statement to Fierce Biotech, saying that the program was discontinued for strategic reasons. The overall partnership with Intellia remains intact, however, the spokesperson said.
Intellia announced in its fourth-quarter earnings report Thursday that the Swiss pharma ended development of OTQ923/HIX763 this month.
The therapy uses autologous, ex vivo, CRISPR-edited hematopoietic stem cells to target fetal hemoglobin for treating sickle cell disease. Novartis initiated dosing on a phase 1/2 trial for the Intellia-partnered program in 2021.
Intellia has both types of candidate in its pipeline, but the in vivo list is longer and more advanced, with NTLA-2001 in transthyretin (ATTR) amyloidosis leading the pack.
Novartis and Intellia have had a cell therapy partnership since January 2015, which was three months after Intellia launched from Atlas Venture and Caribou Biosciences. The agreement was revised in 2018 to expand to ex vivo development of cell therapies using certain ocular stem cells. At that time, Intellia received a $10 million payment, but other financial details of the agreement have not been disclosed. Novartis gained the rights to opt in on one or more programs, while Intellia earned the right to use the pharma’s lipid nanoparticle technology for all genome editing applications in both in vivo and ex vivo settings.
Intellia, working with its partner Regeneron, has shown over the past year that CRISPR/Cas9 in vivo gene editing can cause high, seemingly durable levels of gene knockdown in humans. While questions about the Intellia data, and the concept more broadly, remain unanswered, there is now early evidence that the approach may be effective and, as importantly, safe. Precision is one of a clutch of companies barreling toward the clinic in the wake of Intellia, and the potential of its Arcus platform to provide greater precision and versatility than CRISPR/Cas9 and zinc finger nuclease has now attracted a suitor.
To add to its in vivo capabilities, Novartis is set to pay $50 million in cash to partner with Precision. The deal also features a $25 million equity investment priced at $2.01 per share, a 20% premium over the recent average for the stock, as well as up to $1.4 billion in milestones, research funding and royalties ranging from the mid-single-digit to low-double-digit percentages.
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