Insights Into a New Anticancer Therapy and What Payers Should Know

Matthew R Price, MBA, executive vice president and chief operating officer, Phosplatin Therapeutics, discusses the current pipeline of treatments at Phosplatin—highlighting their lead candidate, PT-112—and explains how they will engage with payers in the future to lay out a case for PT-112 coverage and ways in which this treatment helps address challenges in oncology.

Please tell us about yourself.

Phosplatin was co-founded by our CEO Robert Fallon, and myself. I currently serve as Executive Vice President and Chief Operating Officer where I lead the strategy, finance and operations across the company’s activities. Once we formed the company, I helped negotiate the original license for the company's PT-112 development program, and currently oversee the clinical and translational research and development involving phase I / II studies in solid tumors and hematological malignancies.

The founding of Phosplatin was truly serendipitous. I never would have expected I could write what is above. I am a generalist by nature, and my background is fairly unusual in biotech (as an example, I studied medieval history in college). I came to know Robert through my time at Columbia Business School: he was on the adjunct faculty, and I was a recent MBA graduate working at a small investment firm with a focus on biotech. Robert was a very successful international banker, so I was seeking advice and mentorship from him. Little did I know that he was considering launching a company and having me join him. After spending time with the inventor of PT-112, Rathindra Bose, PhD, and some heavy due diligence on our part, we seized the opportunity and signed our formation papers in the Uris Deli at Columbia Business School. We did not fully realize what we were getting ourselves into, but I am very glad we did not let that stop us. We knew enough to know what we didn’t know, and worked to surround ourselves by the best people we could at each stage. My belief is that specialization and a broader sense of perspective must be married within a team to engender success. I may move from a call about patent filings or financial statements, or manufacturing filters, or cancer cell biology, or clinical eligibility criteria, to looking out the window and thinking about the gaps we need to fill, the inevitable pitfalls we face, and the horizon ahead. And each of these examples requires having the right people as collaborators at the right time.

Years later, we are proud to be working with renowned leaders within cancer drug development, and we have built a young and diverse internal team in New York City (and now remotely, of course), with terrific academic, medical, regulatory, manufacturing and pharma industry experience, and our R&D and manufacturing work has now spanned sixteen countries since we began.

Can you talk about your pipeline of cancer treatments? How do these compare with other agents that are available?

Our pipeline is currently focused on our lead candidate, PT-112. The first pyrophosphate conjugate in anti-cancer therapy, we have come to see that PT-112 offers a unique combination of advantages. It has an excellent safety profile, unlike conventional cytotoxic agents, and it offers a unique, pleiotropic mechanism of action that promotes immunogenic cell death (ICD). A complementary mechanism to existing cancer immunotherapy strategies, ICD is a form of cancer cell death that can activate the immune system in the tumor microenvironment and direct it to mount a tumor‐specific immune response. We believe ICD is an emerging field within cancer immunotherapy.

We have completed and reported on three successful Phase 1 studies with PT-112 and moved into the Phase 2 stage recently. We have seen confirmed clinical responses crossing non-small cell lung cancer, small cell lung cancer, in a rare disease called thymoma, and in multiple myeloma and late-stage prostate cancer. These are relatively small numbers of patients to date, but indicative of a promising early signal. Based on the data generated so far, we believe in PT-112’s promise of a pipeline within a product. We also believe that we can build our company over time via this concept of applying ICD in appropriate disease types, and adding future programs as well.

Can you talk about your current clinical collaborations? What do you hope to achieve? 

As I hinted, Phosplatin is built on high-quality external collaborations. Within the clinic, it’s essential to find like-minded investigators, who command the kind of infrastructure that can deliver high-quality care and data. Oncology Phase 1 studies treat patients without therapeutic alternatives, in most cases, and the nature of this situation requires experience and delicate care. We have worked with leading investigators and research sites, including the University of Texas MD Anderson Cancer Center, The Mayo Clinic, The University of Colorado Cancer Center, the Centre Hospitalier Universitaire Vaudois (CHUV), in Lausanne, Switzerland, Dana Farber Cancer Institute in Boston and Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College here in New York. We also currently run a formal clinical trial collaboration jointly with Pfizer and Merck KGaA, Darmstadt, Germany (EMD Serono), on a combination study of PT-112 and an immune checkpoint-inhibitor, and we launched an early sub-license collaboration with SciClone Pharmaceuticals for developing PT-112 in greater China.

At an early stage of development, we cleared the difficult (and by nature harrowing) hurdles of demonstrating safety and early evidence of efficacy of the drug, and have reported at the major medical forums, including the American Society of Clinical Oncology (ASCO), the European Society of Medical Oncology (ESMO), and the American Society of Hematology (ASH). At this point, we have now progressed toward defining target populations in specific diseases, with an eye of course to getting the drug approved and making it available to patients suffering from serious unmet needs. Our efforts in Phase 2 within late-stage, metastatic prostate cancer have been designed together with a wonderful clinical advisory group, with these needs – and with PT-112’s attributes – precisely in mind.

What should payers know about these treatment options and ongoing clinical trials? 

PT-112 is in clinical development, so it remains pre-commercial for now. In the future we plan to engage with payers to lay out the case for our therapy, and the ways in which it solves challenges in oncology. 

I can broadly describe how we believe PT-112’s attributes will come to make such a case, once trials are complete. The first is examining PT-112’s immunogenic cell death capabilities. We’re working on demonstrating that PT-112 can advance immunotherapy beyond the limitations seen with single agent resistance, and to address disease types (such as prostate cancer) that generally do not have immune involvement, and we are using very sophisticated immune-profiling strategies currently to do this. The other pathway we’re exploring relates to PT-112’s bone tropism. There aren’t many drugs available today that can address the terrible problem of bone metastatic sites of disease. This can be extremely painful for patients, and it has also recently been shown to be an immune-suppressive micro-environment, so any drug that can help address this for a number of different cancer types would be important.

How do you hope to see the future of care changing? 

There is no question that the degree of sophistication of care, and of therapeutic options in oncology, is rapidly evolving. There is a need to balance innovation and access, and much debate around how to do this. As a group that sees itself active on the innovation side, we hope that health care structures will continue to foster innovation in all forms. But we also see certain advances remaining almost inaccessible by the majority of patients, whether through high cost, limited access, or health equity problems. From a research perspective, we need to demonstrate PT-112’s effectiveness in a way that doesn’t have insurmountable regulatory challenges and will benefit many patients down the road. 

Today in health care we see a triangle where we seem to be unable have all three sides at the same time: quality, outcomes, and cost. This will have to change in US health care going forward, and we hope to do our part, beginning with well-defined studies within clear populations with evident unmet medical needs. Down the road, we believe PT-112 should be the kind of drug that is accessible broadly around the world. Staying abreast of policy changes, as a small company, is not trivial, and we expect continued evolution in health care policy in the US and beyond.

What else would you like to add to this conversation? 

It is an exciting time for our company: we are growing, hiring, taking on new funding, and facing new demands to scale and deepen the data we deliver from our trials. It is easy in oncology, which is moving so rapidly, to be swayed by the latest new thing as a biotech group. But to actually make any drug available to patients requires incredible focus and tenacity. We have a wonderful “little engine that could” in PT-112, and a team to fuel that engine with passion. The oncology field needs drugs like PT-112 for its unique set of properties, and emerging companies like ours that have a vision of cancer care making a real impact for patients, and a discipline of following the data. And we must not forget: it’s all about the people.