ARM Releases its End-of-Year Cell Therapy Data Read-Out for 2017

Luke Thorstenson, Market Strategy Analyst | February 9, 2018

The Alliance for Regenerative Medicine (ARM) is the preeminent global advocate for regenerative and advanced therapies, such as cell and gene therapy. ARM is chaired by Robert Preti, PhD, the Chief Executive Officer and President of Hitachi Chemical Advanced Therapeutics Solutions, and General Manager of Hitachi Chemical’s Regenerative Medicine Business Sector.

Each quarter, ARM releases a report detailing the state of the cell and gene therapy industry, including the global landscape, financial information, major clinical trials, key data events, and some regulatory and legislative priorities.

Last month, ARM presented their end-of-year data read-out for 2017. Below please find a few key takeaways from their data.

The State of the Industry

The most significant news from 2017 was the three U.S. Food and Drug Administration (FDA) approvals (Novartis – Kymriah, Kite/Gilead – Yescarta, Spark Therapeutics – Luxturna). Both the Novartis and Kite/Gilead products are ex-vivo gene therapies (gene-modified cell therapy), and the Spark product is in-vivo gene therapy

In Europe, the European Medicines Agency (EMA, the FDA's counterpart), endorsed an allogeneic product for the first time ever—TiGenix’s Cx601 product. Full marketing approval is expected in Q1 2018.

These landmark approvals by the FDA and the EMA’s endorsement of an allogeneic treatment mark a significant step forward for the regenerative medicine industry. This could be an indication that the industry is approaching a more stable phase where approvals for gene-modified cell therapies become much more common.

North America still tops the list for most regenerative medicine companies in the world. According to the end-of-year report, 460 of the 854+ regenerative medicine companies in the world reside there—more than the rest of the world combined.

Regulatory Success Appears to Facilitate High Profile Deals

In August, shortly after Yescarta received a positive indication from the FDA, Gilead Sciences announced they planned to acquire Kite Pharma for $11.9 billion. The acquisition completed in October. Later in the year, Kite/Gilead added to their capabilities by acquiring Cell Design Labs for roughly $567 million ($175 million up front).

So far in 2018, the strong market for mergers and acquisitions has continued with Celgene announcing their intention to acquire Juno Therapeutics for around $9 billion. 

Bioverativ, which has a partnership with Sangamo Therapeutics to develop and commercialize gene-edited cell therapies for beta-thalassemia and sickle cell disease, is preparing to be acquired by global biopharmaceutical company Sanofi for $11.6 billon. 

In early January, shortly after receiving its positive EMA endorsement, Takeda announced their intention to acquire TiGenix for around $630 million.

Spark Therapeutics also announced entering into a $105 million up-front licensing agreement with Novartis to commercialize Luxturna outside the U.S. 

Financial Facts from 2017

According to the ARM end-of-year report, the gene and gene-modified cell therapy industry raised roughly $4.5 billion in 2017 —an increase of over 264% over 2016. For all cell therapy, the industry also increased fundraisings over the previous year—raising $4 billion in 2017, representing an increase in financings of 222% over 2016.

Much of the growth in financings can be attributed to follow-on stock offerings, mergers and acquisitions in the industry. Financings from follow-ons topped $4.0 billion in 2017—up 348% over 2016. Mergers and acquisitions accounted for $13.5 billion of financings in 2017—surpassing the total for 2016 of $1.1 billion. The Kite/Gilead acquisition ($11.9 billion) accounts for the bulk of the overall mergers and acquisitions number.

Where in the World Are Cell Therapy Clinical Trials?

The most recent cell therapy-specific ARM data (as of Q3 2017) states that there are a total of 607 active cell therapy clinical trials. The majority of these trials (370) were in phase 2. A further 192 trials were in phase 1. Less than 7.5% (45) of the 607 trials were in phase 3.

The relatively low ratio of phase 3 trials to phase 1 or 2 trials may demonstrate just how difficult it can be to advance a cell therapy product beyond phase 2. Keep in mind, however, that due to advantageous regulatory pathways, there are cases where a phase 1/2 trial is the pivotal and final trial of a product’s development.

The ARM report shows that there were 946 total current regenerative medicine clinical trials of various therapeutic categories “as of end 2017.”

One significant statistic is that of all the clinical trials currently in progress across all categories, 53% were listed as being “in oncology, including leukemia, lymphoma, and cancers of the brain, breast, bladder, cervix, colon, esophagus, ovaries, pancreas and others”. The runner-up category of “cardiovascular” therapies consisted of just 81 clinical trials at the end of the year—less than one-sixth the amount of oncology indication clinical trials.

This shows how much interest there is in gene and cell therapy as a way to treat various cancers compared to other indications. This interest in oncological indications may be fueled, in part, by the large number of new cancer cases occurring in the U.S. each year. Estimates from the American Cancer Society put the number of new cancer cases (across all sites) at 1,688,780 in 2017.

The large total number of different oncological diseases and subsets mean that many are eligible for the “orphan” designation from the FDA. Orphan designation has historically meant smaller and shorter clinical trials as well as providing extra incentives—such as 7 years of marketing exclusivity, tax credit for 50% of clinical trial costs (the new tax bill changes this to 25%), protocol assistance, and a FDA fee waiver.

Regulatory Environment

In December 2016, the 21st Century Cures Act established a new expedited pathway in the FDA for regenerative medicine products known officially as a Regenerative Medicine Advanced Therapy (RMAT) designation. In 2017 the FDA issued at least 12 RMAT designations, and signaled it plans to modernize the regulatory process to better fit novel therapies, like regenerative medicine. (Please see the FDA announcement for detailed information, like guidance documents)

This is further evidence that the regulatory bodies overseeing the cell and gene therapy industry are committed to helping improve and streamline the regulatory process for these novel therapies.

2017 was a big year for cell therapy, and the PCT services team is excited for the future of cell therapy manufacturing. If you are interested in learning more about cell therapy manufacturing, good manufacturing practices, and how to optimize the path from clinical trials to commercial viability, please contact us today!

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Luke Thorstenson, Market Strategy Analyst

Luke Thorstenson, Market Strategy Analyst

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