Belinostat (licensed from TopoTarget)
In our PTCL study, we were able to enroll patients with platelet counts above 50,000. And despite enrolling a sicker group of patients, our efficacy profile was exactly where it should we, in the high-20s, which is consistent with all other drugs in this category. But more importantly, when you look at the myelosuppression, Grade 3, Grade 4 anemia, neutropenia, thrombocytopenia, our numbers compare favorably to all the other HDACs. So the value of this product is, yes, and for the first indication. But the true value is, can you combine it with the other drugs and truly improve the care of patients in other settings? And that's where we're going with belinostat.
So when belinostat -- you launched that product, do you expect it to cannibalize FOLOTYN sales?
So that's a great question, thank you for that. So today, FOLOTYN is the market leader in relapsed refractory peripheral T-cell lymphoma. The second drug in that category is a drug called romidepsin from Celgene. Unfortunately, both of these medicines have an overall response rate of 25% to 30%. So there's a great unmet need in this marketplace. What will happen with belinostat is doctors will start with FOLOTYN most likely. And then after FOLOTYN, they'll rotate through the other 2 drugs. So patients will typically get first-line treatment, second-line treatment, third-line treatment initially, belinostat will be adopted as a third-line treatment
Captisol-enabled Melphalan (licensed from Ligand Pharma LGND)
- The market in the U.S. for melphalan is $130 million a year.
- There are 2 issues with the current formulations of melphalan. First, all versions contain propylene glycol. With propylene glycol comes the increased risk of cardiac and kidney toxicity.
- Second issue with current versions of melphalan is that once the product is mixed, you must inject it, get it into the patient within 30 minutes. It's a very unstable product.
- But longer term, our key opinion leaders are very interested, and we've already gotten a number of requests to explore the ability to do 1-hour infusions, 2-hour infusions, 3-hour infusions of Captisol-enabled melphalan and then extend its use from just an ablative therapy and transplant to the treatment of multiple myeloma and other diseases.
- Our pivotal study is enrolling right now. This is a small study. It's only 60 patients. This study has been approved by the FDA. We have more than half the patients enrolled in this study today. We expect this study to be fully enrolled this year, and we anticipate filing the NDA next year.
Apaziquone (for bladder cancer, rights returned by Allergan AGN)
- We've conducted 2 Phase III studies in this setting. Both studies demonstrated a 22% reduction in relapse. However, both studies came up just short. The p-value was 0.07 in the individual studies.
- When you combine the 2 nearly identical studies, the p-value goes to 0.017, highly statistically significant.
- We met with the FDA. Based on our discussions with the FDA, we're starting another study, a Phase III study. That study is with the FDA right now. We'll shortly hear back from them. And in addition to that, we intend to file the new drug application. So that's where we are with apaziquone. The beauty of this product is the average patient with -- diagnosed with non-muscle invasive bladder cancer, is 67 years old. And so the paradigm of treatment goes as follows: patients typically get diagnosed, they go in for surgery. Today, some get intravascular chemotherapy, some don't. Unfortunately, most patients relapse. Not once, not twice, but 3, 4, 5 times. So the value here is immediately after surgery, doctors would instill apaziquone into the bladder, and that would reduce the rate of relapse significantly
Longer term catalysts:
1) SPI-2012 (long-acting GCSF) Right now, we're conducting a Phase II study. It's a dose-finding study and a study of chemotherapy-induced neutropenia. This study is enrolling very quickly, and our goal is to complete the study as soon as possible and then quickly start a Phase III study... "toward the end of next year"...phase 3 would be in breast cancer or NSCLC, two studies anticipate it would be vs pegfilgrastim Amgen competitor (noninferiority)
2) ZEVALIN for diffuse large b cell lymphoma
So we have started a Phase III study, this Phase III study. The primary endpoint is overall survival at 2 years. There's a great need in this marketplace. ZEVALIN is a perfect choice. The Phase III is going very, very well, and we expect that to read out over the next few years.
3) SPI-1620 (endothelial B receptor agonist.) So this is an early program that we're conducting Phase II studies right now in cholangiocarcinoma and in non-small cell lung cancer. Give it 30 minutes before chemotherapy
So FUSILEV today, 75% of our business with FUSILEV resides in the clinic segment. Right? that's our strong point, that's where we're, that's where we want to live today and tomorrow. And that market is very, very stable for us. It's very stable. The other part of the business, the 25%, is split. It's all hospitals, but it's split between 340B hospitals and non-340B hospitals. Non-340B hospitals, that is the price sensitive piece of the market. As generic leucovorin comes and goes, that market will come and go. Right? So today, the availability of generic leucovorin is consistent, so that business has been sliding. If it goes away, we'll get that business back. But for us, our core, what we really focus on, is the Clinic business, and that business is doing very well right now.