A Look at Pharma’s Focus on CTLA-4, the Protein Researched by 2018 Nobel Prize Winners

The 2018 Nobel Prize in Medicine has been awarded to James P. Allison, the chair of the department of immunology at the University of Texas’s MD Anderson Cancer Center, and Professor Tasuku Honjo from the Graduate School of Medicine of Kyoto University.

Their work with the protein CTLA-4 has resulted in the development of innovative cancer treatments for advanced forms of melanoma. Currently, the drug Yervoy (BMS) treats melanoma and is being tested for its ability to treat colorectal and kidney cancers. 

Many healthcare companies are racing ahead with research into other immune checkpoint inhibitors with the hope of applications far beyond melanoma.  In the last 6 months, the protein “CTLA-4” has been mentioned 52 times on Earnings Transcripts and IR presentations. Who will be next to market with a life-saving CTLA-4 drug, and what indications will it target?  

Here’s a quick glimpse of 3 pharma companies that mentioned CTLA-4 on recent Earnings Transcripts.

Bristol-Myers Squibb

On July 26th, BMS mentioned CTLA-4 in their Earnings Transcripts. As the producer of Yervoy, BMS discusses CTLA-4 both in the context of Yervoy’s growth, as well as their goals to use CTLA-4 blockers to target other indications. They mention 2 “early stage, fairly large” Phase 1 trials.

In general, BMS uses tentative optimism when talking about applications of CTLA-4 beyond treating melanoma.

BMS Chief Scientific Officer Thomas Lynch says “For example, I’m very enthusiastic about CTLA-4 as a target” but that “we really don’t know what the promise of these drugs will be until we sort of see what the profile is coming out of Phase I. But I would imagine that you can look at this as potentially expanding the role of what CTLA-4 can do in cancer.


On an Earnings Transcript dated August 9th, 2018, Biotech company Agenus goes into many specifics about their proprietary CTLA-4 related-drugs, including naming the indications being targeted beyond melanoma. Specifically, they mention a trial involving angiosarcoma, a cancer of the inner lining of blood vessels that commonly occurs in the skin, breast, liver, spleen, and deep tissue, as well as a possible application for cervical cancer.

What’s interesting about this transcript is the high level of confidence suggested by the specificity of the information provided. For example,

  • Dates   “Our CTLA-4 and PD-1 programs are advancing in 3 active clinical trials today designed to take advantage of accelerated pathways for approval, BLA filings in as early as 2020.”
  • Results “We have reported exciting results from our CTLA-4 monotherapy trial at ASCO with 31% of treated patients with refractory cancers showing clinical benefit.”



On August 6th, 2018, Xencor discussed 2 different combination therapies involving CTLA-4:

  • PD-1 x CTLA-4 “PD-1 x by CTLA-4 bispecific antibody for the treatment of multiple oncology indications.”
  • “CTLA-4 x LAG-3 dual checkpoint inhibitor” also for the treatment of “multiple oncology indications”

Xencor also mentions expecting to be able to share some initial trial data for the above in the second half of 2018. They mention they’re “particularly excited” to be able to share that data.