CTI Biopharma Corp (CTIC) 2021 Q1 法說會逐字稿

Good day, ladies and gentlemen. Thank you for standing by. Welcome to CTI BioPharma's First Quarter 2021 Earnings Call. (Operator Instructions) This conference is being recorded today, June 1, 2021.

I'd now like to turn the conference over to Dr. Adam Craig, CEO and President of CTI BioPharma. Please go ahead.

Thank you, Liz. Welcome to this afternoon's conference call. Joining me today are David Kirske, Chief Financial Officer; and Bruce Seeley, Chief Operating Officer. Following formal remarks, the conference will be opened for questions.

Before I begin, please note that during this call, we'll be making forward-looking statements based on current expectations. Such forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties. They may -- that may cause actual results to differ materially from those anticipated by the forward-looking statements. Additional information concerning these risks and uncertainties is contained in today's press release.

This quarter, CTI continued to execute on the critical activities that have further advanced pacritinib towards a potential U.S. approval and commercial launch later this year. Most notably, today, we announced that our NDA submission for the use of pacritinib in patients with cytopenic myelofibrosis, that is those with severe thrombocytopenia defined as platelet counts of less than 50 x 10^9 per liter has been accepted by the Food and Drug Administration and has been granted priority review.

Our PDUFA target action date is November 30, 2021, and the FDA has informed us that they are not currently planning to hold an advisory committee to discuss the application. The NDA was accepted based on data from the 2 Phase III PERSIST trials and a Phase II PAC203 trial, with a focus on the severely thrombocytopenic patients enrolled in these studies who received pacritinib 200 milligrams twice a day. A population includes frontline treatment-naive patients and patients with prior exposure to JAK2 inhibitors.

In the PERSIST-2 study, in patients with severe thrombocytopenia, who were treated with pacritinib 200 milligrams twice a day, 29% of patients had a reduction in spleen volume of at least 35% compared to 3% of patients receiving best available therapy. 23% of patients had a reduction in Total Symptom Score of at least 50% compared to 13% of patients receiving the best available therapy, which included ruxolitinib. In the same population of patients treated with pacritinib, adverse events were generally low grade, manageable with supportive care and rarely led to discontinuations. Platelet counts and hemoglobins were stabilized.

Following approval, the ongoing Phase III PACIFICA study is now expected to be completed as a post-approval confirmatory trial. Over the last few months, as the COVID pandemic has abated in the U.S., enrollment on PACIFICA has picked up significantly, and we now expect to report top line primary efficacy data in 2022.

With the NDA accepted and the PDUFA target date set, we are now even closer to being able to provide pacritinib to patients with cytopenic myelofibrosis, who are underserved by existing therapies. As a reminder, 1/3 of the existing myelofibrosis population has severe thrombocytopenia or approximately 7,000 patients. To address this unmet medical need and to prepare to deliver pacritinib to patients this year, our team has been working diligently on precommercialization activities, including market access, distribution, supply chain, disease education, and field force planning and deployments.

Moving on to an additional indication for pacritinib. Last year, we launched the study of pacritinib in severe COVID-19 patients in response to the pandemic. This study, PRE-VENT, is a randomized, double-blind, placebo-controlled multicenter Phase III clinical trial comparing the use of pacritinib plus standard of care versus placebo plus standard of care in hospitalized patients with severe COVID-19. The primary endpoint of the trial will assess the proportions of patients who've progress to invasive mechanical ventilation and/or extracorporeal membrane oxygenation or die by day 28. We expect to report on the outcome of the interim analysis from the study in the third quarter of 2021.

Finally, as we have previously reported, we are investigating the use of pacritinib in the prevention of acute graft-versus-host disease, or GvHD. At the American Society Hematology Meeting or ASH in December 2020, data was presented from an investigator-sponsored Phase I/II study, showing that adding pacritinib to the standard prophylaxis of sirolimus and low-dose tacrolimus resulted in a significant reduction in the expected acute graft-versus-host disease rates in patients within the first 100 days of therapy as compared to historical data, without compromising transplantation outcomes and without any new safety concerns. The Phase II component of this study continues to enroll, and we remain on track to provide an update on the progress of the Phase II trial and any potential regulatory interactions around this indication later in the year.

I'll now turn the call over to David to review our quarterly financials. David?

Thank you, Adam. As of March 31, 2021, cash, cash equivalents and short-term investments totaled $37.2 million as compared to $52.5 million as of December 31, 2020. On April 6 of this year, we completed an equity financing with net proceeds of $53.8 million, which extends our cash position to fund our operations into the fourth quarter of this year.

Operating loss was $17.1 million and $11.9 million for the 3 months ended March 31, 2021, and 2020, respectively. No revenues were reported for the first 3 months ended March 31, 2021, or for the 3 months ended December 31, 2020. Net loss for the 3 months ended March 31, 2021, was $17.3 million or $0.23 basic and diluted loss per share as compared to a net loss of $12.2 million or $0.20 basic and diluted loss per share for the same period in 2020.

So with that, Adam, I'll hand it back to you.

Thank you, David. So in summary, we are very pleased that our NDA has been accepted by the FDA with a PDUFA date of November 30, 2021. This represents a significant step forward in our efforts to deliver a treatment-paradigm-altering medicine to myelofibrosis patients in need.

We look forward to continuing to work collaboratively with the FDA over the coming months and to advance our pre-commercial activities in preparation for end of year product launch.

This now concludes our remarks. Liz, please open the call for questions.

(Operator Instructions) Our first question comes from Ben Burnett with Stifel.

This is Carolina Ibanez-Ventoso on for Ben Burnett. And congrats on all the progress. Adam, you mentioned in your prepared remarks, you are launching different initiatives to support the commercialization of pacritinib, including the space allocation, customer engagement, et cetera. Could you provide additional context and a time frame for these different activities?

Yes. I'll hand the question over to Bruce Seeley.

Carolina, so the activities for commercialization are well underway. The teams have been following a very detailed launch planning playbook to prepare for launch. And the teams are already working towards that. Hiring is on time, execution of the commercialization activities are on time. As Adam mentioned in his opening remarks, we are spending a lot of time right now working on market access activities.

Clearly, for an oral compound that's going to be very important for us, we've hired a very good team to be able to help us with that, and those activities are well underway. I'm very confident that we will be able to meet the time lines to be prepared for launch later on.

If I may, as a follow-up, when do you plan to complete the sales force and the other commercial teams? And then do you have a headcount target for the sales force that you could share? And then how long is the training process they have to go through?

So the -- so we've not made a specific time line as to the hiring of the sales force, in particular. We're focused on bringing in sales leadership right now. The field force is going to be later on this year, a little bit closer to launch.

As it relates to training, we've already completed the development of all our training materials for the sales organization. The total training time line will be about 4 to 6 weeks for the sales representatives to be field ready at that time.

Our next question comes from Reni Benjamin with JMP Securities.

Let me add my congratulations as well. Adam, can you just maybe take us through in terms of how much drug is available? What the kind of status of manufacturing is? And does the manufacturing plant needs to be inspected as well? Is that -- or is that kind of already done? Maybe just a sense of how that's progressing.

Yes. Well, we are -- just give you brief, manufacturing as well is under control. We, for some time, had a substantial amount of drug products available. So we do have commercial lots and we were ready to -- we're ready to launch if the approval comes early. Towards the latter part of this year, we will be manufacturing some new material. But at the moment, we are ready and able to move forward as and when the drug is approved.

Got it. And then I guess just following up on the COVID-19 study, I believe it was -- you had mentioned in the prepared remarks, third quarter. I just wanted to get a sense, is that still -- should we still be considering that quite material for the company? Or now that you have the acceptance and so really a lot more about launch preparations, should we really be focused on that?

Well, we -- we're still going to -- thank you, Ren, we're still going to conduct the interim correctly and assess the position of the drug once we have -- this trial rather, once we have the interim results. It's certainly not the main driver for the company at the moment. The majority of our focus -- the vast majority of our focus is on the successful commercialization and launch of the drug at the end of this year. But if there is an opportunity to move forward with COVID, based on the interim analysis, then we'll discuss it and we'll let The Street know.

Our next question comes from Thomas Flaten with Lake Street Capital Markets.

Congrats as well. Just curious, Adam, it looks like the MPN guidelines were updated in mid-April. Do you have a sense if that's -- on what time schedule there might be for another update there? I don't know how closely your team has been interacting with the NCCN Guideline Committee for MPNs.

Bruce will take this question, Thomas.

The -- we've been thinking a lot about our NCCN strategy. There's an opportunity for NCCN -- for us to inform NCCN of the data. And so we have plans to be able to do that at the -- in the middle of the year. And then towards the end of the year is generally when they meet, although it's -- it is our expectation that time of approval that the NCCN committee can meet at the time of any new important approval in the marketplace. So that's what we expect.

Got it. And Adam, could you remind us, did you submit the indication with or without the restriction for the platelet counts?

Our top line indication was for primary and secondary myelofibrosis.

Got it. And then just one final one for me. Is there anything you can share with us around your thoughts on pricing, given the disparity in pricing between JAK and pacritinib?

Not at this time, Thomas. We're not commenting on pricing.

Our next question comes from Chad Messer with Needham.

Congratulations on the NDA acceptance. Can you just talk a little bit about how patients who have low platelet counts, how they're treated in the system in terms of -- are there any specialists they see? Or how are you going to target them, given that this is your label, I guess, is my question?

I'll hand over to Bruce.

So our market research suggests that community physicians are also quite comfortable treating myelofibrosis patients with low platelet counts. When the patients are referred to the academic centers, they generally have constitutional symptoms, which would include spleen volume increases, that the community oncologist is no longer comfortable treating in their offices. So at that point, they get referred.

From a deployment perspective, it's going to be important for us not to just focus on the academic centers, but also have outreach to the community centers as well. They may not treat on an individual basis that many patients in general. But as an -- in aggregate, they do represent a very large portion of the treating population.

Our next question comes from Bert Hazlett with BTIG.

Yes. Let me add my congratulations on the priority review as well. Just a quick one on GvHD, could you remind of any differences between the Phase II design that's ongoing and the Phase I study...

Sorry, Bert. We can't hear you properly. It's very muffled.

Sorry. Is that better?

Yes.

Could you just remind us -- first of all, congratulations again on priority review. And then with GvHD, could you remind us of the -- any differences between the Phase II design that the ongoing study and the Phase I/II IST study? Just any changes or tweaks in design or patient population or endpoints or anything of the like?

No, not really. They're substantially very similar. The purpose of the Phase I was to identify a safe and effective dose to go into Phase II. And then at Phase II, the number of patients was expanded.

But so having tested 2 dose levels at Phase II, 100 milligrams twice at Phase I, 100 milligrams twice a day was selected, and that will be given in combination with sirolimus and tacrolimus to all Phase II patients.

That concludes today's question-and-answer session. I'd like to turn the call back to Dr. Craig for closing remarks.

Thank you, Liz, and thank you, everyone, for joining the call today. We look forward to further conversations over the coming months.

This concludes today's conference call. Thank you for participating. You may now disconnect.