Celgene Doesn't Want To Be Left Out, Posts Promising JCAR017 Data

Summary

• Celgene announces positive preliminary data from its study treating patients with relapsed/refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) using JCAR017.
• A key risk is whether or not hospitals and insurers will pay the hefty price tag of CAR-T therapies, which may impact sales.
• Of the 81% of patients that got an ORR who were treated with JCAR017, 43% achieved a complete response.
• It is estimated that the global CAR-T cell therapy market could be worth $8 billion by 2028.
• Competition is strong in the lymphoma space using CAR-T. Such companies are, for instance, Novartis with Kymriah and Gilead Sciences with Yescarta.
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Recently, Celgene (NASDAQ:CELG) announced preliminary data from its study treating patients with relapsed/refractory (r/r) chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). The data shown was from the dose escalation part of an ongoing, open-label multicenter phase 1/2 study using JCAR017 to treat these patients. I think that the preliminary data is strong, especially when you consider that a large chunk of this population were given prior treatment with ibrutinib and had cytogenetic features of high-risk disease. This data is preliminary, but it is quite strong.

Phase 1/2 Monotherapy Data

The phase 1 portion of this program, known as TRANSCEND CLL-004, explored the monotherapy dose-escalation part of the main study. This study data included about 16 patients with at least a median of 4.5 lines of prior therapy. First of all, that is a lot of prior therapy attempts to get a patient to generate a response. Think about that, all these patients had gone through 4 or more therapies with no success. In addition, patients had high-risk cytogenetic features. In essence, cytogenetic features meaning erratic behavior of cells and how they divide. The problem with patients that have cytogenetic features are that they tend to respond differently depending upon what treatment they are given.

The final thing to note is that patients recruited into the study had already received and not responded to ibrutinib (Imbruvica) treatment. Imbruvica is a strong drug, but sometimes it may not generate a response for some patients in this refractory population. To showcase this item, it is important to look at what patients had taken as prior treatment with Imbruvica. At least 81% of the patients recruited had relapse/refractory disease on Imbruvica. It was also known that 50% of patients had received prior therapy with both Imbruvica and Venclexta (venetoclax). Having said all that, those treated with JCAR017 obtained an overall response rate (ORR) of 81%. Even more impressive is that 43% demonstrated a complete response. Complete response meaning that disease is no longer detected in the patient.

Competitors

Thus far, the data looks good for Celgene using its JCAR017 drug to treat r/r CLL. However, it won't be an easy ride because there are CAR-T pharmaceutical companies that have approvals in this space. They are Novartis (NVS) with Kymriah and Gilead Sciences (GILD) with Yescarta. The CAR-T space is starting to get a somewhat crowded, and that will especially hold true as more pharmaceutical companies enter the field.

It is estimated that the global CAR-T cell therapy market could be worth $8 billion by 2028. However, this highly depends upon one main issue for how it will play out in the coming years, which revolves around the pricing of these therapies. That's because CAR-T therapies are expensive, and that highly depends upon uptake of the drug in the market. The key issue being is whether or not insurers will pay the high cost of CAR-T therapy over other therapies. Kymriah and Yescarta cost around $373,000 for treatment of DLBCL. However, Kymriah still costs around $475,000 to treat patients with ALL. These costs are just to infuse the patients with the CAR-T therapy. Such costs don't include hospital stay, supportive care, or any doctor visits as well. Combining all those supportive costs, plus the cost of the CAR-T therapy itself, could mean a bill for the patient totaling $1 million. No doubt these treatments have the goods when it comes to efficacy such as ORR and CR rates. It's just important to track whether or not sales will start to grow at a substantial pace even with the high pricing.

Conclusion

The phase 1 portion of the phase 1/2 study achieved positive results using JCAR017 treating patients with r/r CLL and SLL. These were not very easy patients to treat, because they had gone through 4 or more therapies prior to entering this study. On top of that, they had even taken Imbruvica with no significant response. Despite all that, patients treated with JCAR017 showed an impressive ORR and a strong CR rate as well. Celgene is doing well in the CAR-T space just like the other pharmaceutical companies. They risk the difficult task of getting hospitals and insurers on board to pay for these expensive CAR-T therapies which run over $350,000. The good news is that there is no set price yet for JCAR017. In addition, it could become the 3rd drug to be approved for lymphoma as a CAR-T therapy. Another CAR-T approval therapy for lymphoma may be a good thing, because it possibly could drive prices lower as new competitors roll in.

Analyst’s Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.