Pharma spends big to access key cell and gene therapy assets
High-value cell therapy deals are driving investment in the cell and gene therapy field, but new approaches such as gene editing are also beginning to contribute. By Mike Ward 16 November 2023
In the ever-evolving landscape of biotechnology and pharmaceuticals, one sector has emerged as particularly promising in the quest to combat previously untreatable diseases: cell and gene therapy. Half a century after scientists proposed the idea of modifying defective genes to treat diseases with a genetic root, the approach is beginning to bear fruit.
To date, the United States Food and Drug Administration (FDA) has approved 31 cell and gene therapies, ten of which are targeting oncology indications. It has also approved multiple oligonucleotide-based drugs, while all eyes are currently on whether the agency will approve the first therapy developed using a gene-editing technology for sickle cell disease and beta thalassemia in December 2023.
Groundbreaking cell and gene therapy technologies have the potential to revolutionize medicine, offering new hope to patients facing previously insurmountable challenges and so have emerged as an attractive target for pharmaceutical companies. However, the journey from discovery to market is fraught with complexity, and therefore dealmaking could play a pivotal role in shaping the future of the field.
In the 12 months to the end of September 2023, according to BioWorld, 246 partnership deals focusing on cell and gene therapy programs and platforms were signed, of which 43 also disclosed financial details. During this 12-month period, the three highest valued deals were signed in 2022. In 2023, the leading ten transactions have a potential value of $16.8 billion.
Cell therapy-focused deals Many of the deals signed in the cell and gene therapy space involve cell therapies for cancer, and several deals in recent years have been penned for pharma companies to access next-generation cell therapy opportunities. For example, in August 2022, Roche signed a deal with Poseida Therapeutics to develop allogeneic CAR-T cell therapies for blood cancers including multiple myeloma, B cell lymphomas and other indications. Poseida will receive an upfront payment of $110 million, and if milestones are met Poseida could receive up to $6 billion, which makes this deal one of the most potentially valuable in the field in recent years. Also targeting multiple myeloma through a high-value deal, Arcellx and Kite Pharma joined forces in December 2022 to co-develop and co-commercialize Arcellx’s phase 2 cell therapy CART-ddBCMA (Table 1).
Big pharma Bristol Myers Squibb (BMS) also signed a couple of high-value oncology-focused deals earlier in 2022. In January 2022, BMS partnered with Century Therapeutics to use their induced pluripotent stem cell (iPSC)-based allogeneic cell therapy platforms to develop up to four iPSC-derived, natural killer cell and/or T cell programs to treat hematologic malignancies and solid tumors. BMS then expanded its relationship with Immatics to develop allogeneic TCR-T/CAR-T programs using Immatics’ gamma delta T cell-derived, adoptive cell therapy (ACT) platform, ACTallo . . . .
Freedom to operate One of the challenges for companies attempting to establish cell and gene therapy partnerships is to ensure that they have all the elements in place to have freedom to operate. In addition to the scientific challenges, issues around manufacturing and scale-up need to be addressed, often requiring access to the intellectual property estates of other organizations. Indeed, with CRISPR-Cas9 platforms, the challenge potential partners face is the fact that the United States and European Union have given primacy to different patent estates.
While the biggest headline-grabbing transactions have involved pharmaceutical companies getting access to promising therapeutic candidates, most of the deals are focused on tapping into the technologies that are essential to developing and manufacturing these new generations of therapies. Indeed, some of the transactions associated with manufacturing technologies can be very complex as pharma stack up the patents needed to have freedom to operate.
Clearly, the promise of cell and gene therapy is immense, with the potential to revolutionize the treatment of numerous diseases. So, as the industry evolves and technologies advance, the art of dealmaking will remain at the forefront of bringing these life-changing therapies from the laboratory to the clinic, offering hope to patients who have long awaited a cure.
One of my takeaways is that the the tide is turning, and Big Pharma’s former reluctance to consider a different operating model is changing. These companies are definitely aware of, and eager to partner with seemingly obscure gene and cell therapy companies, as noted by the sheer number of deals that were made last year, (~250) and that in the vast majority, (over 200) the financial details are actually unknown. So if anyone thinks that just because a deal hasn’t been struck with Northwest Bio yet, that Big Pharma companies aren’t interested, well nothing could be further from the truth.
Another, is that most of them are pre-clinical or early-phase developmental deals, to treat rare diseases with very small patient populations, while Northwest Bio is very late-stage, and targeting solid-tumor cancers, which is a massive potential patient population.
Additionally, many of the deals are focused on manufacturing technologies, which are complex and difficult to develop, and great importance is placed on having the patent protections to operate freely, and maintain superiority (and high pricing).
So, to sum it up: In comparison to virtually ALL of those companies, Northwest Bio is sitting in a very enviable partnership/buyout position; late-stage in their development, with marketing approval and automated manufacturing capability in hand, (or nearly, depending on timing) as well as a multi-patented manufacturing process, while addressing a massive, highly-desired market.
I’ve said this before, but I think it deserves repeating: Underestimate Linda Powers at your own peril. One of the many things she does clearly understand, is negotiating leverage.
