Adapting and Evolving in a Volatile Market: Cell Therapy and Rare Disease Developments

If there’s one thing that remains constant in biotech, it’s the ability of companies to adapt and evolve relative to available resources and current limitations. Recent shifts occurring industry-wide have given the life sciences executive search consultants at BayBridge an opportunity to reflect on reverberations of a market downturn, and other developments constantly occurring in the always fascinating world of biotech!

Rare Diseases

 Although the number of treatable patients in the space of rare disease is relatively small, rare disorders have attracted attention in recent years due to less market competition, and their ability to garner funding from big pharma companies looking to keep pipelines progressive and relevant.

Additionally, effective drugs can receive regulatory and commercial incentives, and may be met with less pushback on price given the lack of alternative treatments (Bell, 2022). Several rare disease therapies from multinational biopharma companies have already been fast-tracked so far in 2022. Just a few days ago, the FDA granted Dizal Pharmaceutical (a China-based biotech) a Fast-Track designation for golidocitinib, a treatment for patients with refractory or relapsed peripheral T-cell lymphoma. The treatment is an orally available, potent, JAK1-specific inhibitor. And California-based ImmixBio has been granted a rare pediatric disease designation for the treatment of rhabdomyosarcoma, a life-threatening form of cancer in children.

In a down market, being part of an emerging biotech company focused on rare diseases can be reassuring. Some of the biggest deals in biotech to date have been acquisitions of rare disease companies. In 2018, Takeda paid $62B for Shire, working on multiple rare disease targets in hematology, neuroscience, and ophthalmology, amongst others. And in 2021, AstraZeneca negotiated a $40B acquisition of Alexion Pharmaceuticals, a rare disease company researching the complement system, which helps the body fight infection (Bell, 2022).

The global market for orphan drugs is expected to grow at a CAGR of 11% annually, jumping from $138B in 2018 to $262B by 2024, which is a staggering 22% of the overall prescription drug market. With over 7,000 known rare diseases, and treatment existing for only a few hundred of these, the scale of potential is huge (Ruzicic et al., 2018).

NK cells

 NK cell therapy has been garnering plenty of attention from drug makers recently, as they are thought to be potentially safer than T cells, cheaper to produce, and better able to distinguish between cancerous cells and healthy ones (Fidler, 2022). Several companies are exploring using NK cells in combination with CARs, as well as combining CRISPR gene editing with NK cells to develop more targeted treatments for cancer.

Alliances between biotech companies working in cell therapy are growing, particularly when it comes to production of next-generation NK cells. The creative world of partnerships – through the likes of licensing, co-development, capability and risk sharing, technology collaborations, and joint ventures – has been responsible for pushing forward the advancement of next-generation cell therapies in recent years (Novasecta, 2022). Companies that have been pioneers of CRISPR – such as Editas, Intellia, and CRISPR Therapeutics – have been making progress to combine CRISPR technology with NK cell therapy. Most of these companies are also focusing on the production of allogeneic NK cells, which in recent studies have shown less susceptibility to GVHD than T cells. Of course, big pharma has also gotten in on the action, with Gilead, Merck & Co., and Johnson & Johnson having invested in NK cell therapies (Fidler, 2022).

There are still numerous limitations to NK cell therapies, such as their durability, limited cell numbers often requiring ex vivo expansion and activation, and susceptibility to the immunosuppressive tumor microenvironment (Basar et al., 2020). However, as cell engineering techniques become more advanced, researchers are finding ways to overcome these obstacles.

Executive Search Insights

 With increasing strategic partnerships in the biopharma industry, life sciences executive search consultants must continue to build on their knowledge of rare diseases and the treatments being developed to address them. Working knowledge of advances in C&GT is essential to build working relationships with clients and candidates when utilizing a talent management approach.

References 

Bell, J. (16 February 2022). Star Therapeutics emerges with plans to treat rare diseases through a solar system of drug companies. Biopharma Dive. https://www.biopharmadive.com/news/star-electra-biotech-startup-launch/618921/

 Fidler, B. (15 February 2022). Intellia follows CRISPR peers with deal to explore ‘natural killer’ cell therapy. Biopharma Dive. https://www.biopharmadive.com/news/intellia-onk-gene-editing-nk-cell-therapy/618874/

 Novasecta. (n.d.) Better Partnerships: The alternative to M&A? https://novasecta.com/better-partnerships-the-alternative-to-ma/

 Ruzicic, A., Gardiner, P. & Hall, C. (November 2018). Rare diseases: The seamless go-to-market model. https://www.executiveinsight.ch/application/files/6815/4330/6737/PME_Seamless_GTM.pdf