TG Therapeutics, Inc. (TGTX) Earnings Call Transcript & Summary

Greetings, and welcome to the TG Therapeutics FDA Approval Conference Call and Webcast. [Operator Instructions] As a reminder, this conference is being recorded. [Operator Instructions] I would now like to turn the call over to Jenna Bosco, Senior Vice President of Corporate Communications. Please go ahead.

Welcome, everyone, and thank you all for joining us. I'm Jenna Bosco, and with me today to discuss the U.S. Food and Drug Administration's approval of umbralisib, now called UKONIQ, are Michael Weiss, our Executive Chairman and Chief Executive Officer; Dr. Owen O'Connor, our Chief Scientific Officer; and Adam Waldman, our Chief Commercialization Officer. Mike will begin today's call by providing some introductory comments and context around Friday's FDA approval of UKONIQ for patients with relapsed/refractory marginal zone lymphoma and follicular lymphoma. Owen will review the label as well as the clinical data supporting the approval, and Adam will discuss our launch and commercialization plans. We will then open the call up for Q&A. [Operator Instructions] On Friday, we issued a press release detailing the accelerated approval of UKONIQ by the FDA. This release as well as today's webcast presentation are available in the Investors section of our website at www.tgtherapeutics.com. For those of you that are dialed in, please note an associated slide deck for today's call can be found on our website on the Events page under the Investors section. Before we begin, I would like to remind everyone that various remarks that we make about our future expectations, plans and prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. TG cautions that these forward-looking statements are subject to risks that may cause our actual results to differ materially from those indicated. Factors that may affect TG Therapeutics' operations include various risk factors that can be found in our most recent Form 10-Q and other filings with the Securities and Exchange Commission. In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements. This conference call is being recorded for audio rebroadcast on TG's website where it will be available for the next 30 days. [Operator Instructions] Now I would like to turn the call over to our -- to Mike Weiss, our CEO.

Thanks, Jenna, and thanks to everyone for joining us today. I'm thrilled to be with you to formally announce the accelerated approval of umbralisib, which now has the trade name UKONIQ. As you have for sure heard, UKONIQ has been granted accelerated approval as a monotherapy for the treatment of adult patients with relapsed or refractory marginal zone lymphoma who have received at least 1 prior anti-CD20-based regimen and patients with follicular lymphoma who have received at least 3 prior systemic therapies. We are honored to have the opportunity now to provide to eligible patients UKONIQ, a treatment option, distinct from all others currently available in these indications. Developing a drug, like raising a child, takes a village. And we have a lot of folks to thank who have made this approval possible. First and foremost, I'd like to thank the patients and their caregivers as well as the physicians, nurses and their teams who are participating in our clinical trials and trusted us; our advisers and partners who have helped us discover, develop and manufacture umbralisib; the TG team, all of whom who have helped along the way to make this achievement possible; and of course, the dedicated team at the FDA that worked tirelessly through COVID to approve both marginal zone indication ahead of its February 15 PDUFA date and follicular 4 months ahead of its June 15 PDUFA date. The accelerated approval of UKONIQ in marginal zone and follicular lymphoma is based on overall response rate, and continued approval in these indications may be contingent upon verification and description of clinical benefit in a confirmatory trial. Accordingly, we are working closely with the FDA regarding the confirmation trial to convert the accelerated approval into a full approval. As we celebrate UKONIQ's approval, I am more confident than ever that this is just the beginning for TG. Since we founded our company over 9 years ago, we've had 1 mission: to develop novel treatment options for patients in need with the goal of developing combination therapies that can bring us, hopefully, closer and closer to functional cures. We have assembled a robust pipeline of medicines with complementary mechanisms of action, and we remain steadfast in our development mission. Today marks a truly transformational day for TG as we expand the scope of our business from a purely R&D company to a fully integrated, commercial-stage biopharmaceutical company. And while, as I mentioned above, we still have a lot more to achieve on the R&D side, we are excited to be able to offer our first drug commercially under the leadership of our Chief Commercialization Officer, Adam Waldman. Adam has built a talented and experienced commercial team who helped bring UKONIQ to those patients with marginal zone and follicular lymphoma who are in need of additional treatment options. Adam will share our commercialization plans in just a few minutes, but before he does, I would like to hand the call over to Dr. Owen O'Connor, our Chief Scientific Officer, to briefly review UKONIQ's prescribing information as well as the data supporting today's approval. Owen, please go ahead.

Thank you, Mike, and thank you to all of you for joining us this morning. I am the Chief Scientific Officer at TG Therapeutics. While I officially joined TG -- the TG team last year, I have been closely involved with the development of umbralisib since the company in-licensed the drug back in 2012. On Slide 8 -- we'll advance the slide. So in over 20 years of treating patients with marginal zone and follicular lymphoma, I have been treating patients with umbralisib in the early Phase I and Phase II clinical trials. It was clear to me that umbralisib was different from other drugs I've used for these patients in the past. In addition, during my tenure at Columbia University, my team spent a lot of time researching umbralisib's mechanism of action, including its unique inhibition of casein kinase 1 epsilon, or CK1-epsilon. While pharmacologically distinct from other kinase inhibitors, the clinical data has really highlighted some of these mechanistic differences, including its differentiated safety profile, it's once-a-day dosing and its impressive clinical activity as a monotherapy in heavily treated patients. We believe that these features of umbralisib open up the potential for the drug to be safely combined with other agents active across lymphoid malignancies, for which there are now numerous studies ongoing evaluating this potential. I want to echo Mike's sentiments. The approval of UKONIQ is an enormous accomplishment, thanks to the hard work of many. But as a physician who treats these complex diseases, I'm especially happy for our patients whose physicians now have a new treatment option in their armamentarium for patients with relapsed or refractory marginal zone or follicular lymphoma. Umbralisib, or UKONIQ, as we will now refer to it, brings significant clinical value to patients based on data from the UNITY-NHL study, the details of which I will share with you in a moment. As you've already heard, UKONIQ is the first and only oral once-daily inhibitor of both phosphoinositide 3 kinase delta, a mouthful, more easily referred to as PI3K-delta and casein kinase 1 epsilon, which we'll refer to as CK1-epsilon, now approved for the treatment of patients with relapsed or refractory marginal zone and follicular lymphoma. Next slide. UKONIQ is highly selective for the delta isoform of PI3K, which is known to play an important role in cancer cell proliferation and survival, cell differentiation, intracellular trafficking and host immune response. PI3K is well-established to be grossly dysfunctional in malignant B-cells. And as you can see on the slide, in contrast to other PI3 kinase inhibitors, UKONIQ selectively inhibits the PI3K-delta isoform and does not inhibit the alpha, beta or gamma isoforms. As I mentioned earlier, UKONIQ also uniquely inhibits CK1-epsilon, a regulator of protein translation, which controls the synthesis of critical oncoproteins like MYC, Bcl-2, cyclin D1, all of which have been implicated in the pathogenesis of lymphoid malignancies. Slide 10. The efficacy of UKONIQ was evaluated in an open-label, international, multicenter, multicohort clinical trial, evaluating 69 patients with relapsed or refractory marginal zone lymphoma who had received at least 1 prior therapy, including an anti-CD20 monoclonal antibody, and 117 patients with relapsed or refractory follicular lymphoma who are treated with at least 2 prior systemic therapies, including an anti-CD20 and an alkylating agent. Patients in the trial received 800 milligrams of UKONIQ administered orally once daily. Patients with relapsed or refractory marginal zone lymphoma achieved an overall response rate of 49% and a complete response rate of 16%. While patients with relapsed or refractory follicular lymphoma achieved an overall response rate of 43% and a complete response rate of 4%. Importantly, with a median follow-up of over 20 months for both diseases, the median duration of response has not been reached for the marginal zone lymphoma cohort and it was found to be 11 months for the follicular lymphoma patients. Next slide. Additionally, as you can see on this slide, with a median follow-up of 27 months, no patients with relapsed or refractory marginal zone lymphoma who achieved a complete remission has relapsed to date. For patients with this type of chronic indolent malignancy, this durability of response, coupled to a favorable safety profile, really represents an exciting opportunity for our patients. Next slide. As a clinician who has been treating these patients now for 2 decades, I know in order for us to optimally manage patients with chronic forms of indolent lymphoma, we need drugs with manageable toxicity profiles that can be clinically -- that can produce clinically meaningful and durable responses. We believe that the safety profile of UKONIQ is one of the drug's key attributes. The safety of UKONIQ was evaluated based on pooled population, which included 221 patients with marginal zone and follicular lymphoma who received the recommended dose of UKONIQ of 800 milligrams once daily. Low rates of severe immune-mediated toxicity were reported, and AEs led to drug discontinuation in 14% of patients. It is also noteworthy that there's no box warning nor are there any recommended dose adjustments for concomitant medications. That said, the label does include warnings and precautions for infections, neutropenia, diarrhea, noninfectious colitis, hepatotoxicity and severe cutaneous reactions. In summary, we are very pleased with the approval of UKONIQ for patients with relapsed or refractory marginal zone and follicular lymphoma. We believe UKONIQ offers a treatment option that is distinct from other drugs presently available for these challenging diseases and fills an important unmet medical need. This marks only the beginning of UKONIQ's journey as we continue to explore UKONIQ in a variety of combination regimens, all with an ambition to improve the management of patients with all forms of B-cell lymphoma. Thank you, everyone, for your time this morning. Now I'll turn the call over to Adam Waldman, our Chief Commercialization Officer.

Thank you, Owen, and good morning, everybody. First, let me start by thanking all of my TG colleagues who have worked so hard for so many years to make this day possible. Today marks an incredible milestone for TG, achieving our first FDA approval, but also represents a great day for patients with relapsed indolent lymphoma who are in need of new treatment options. We are extremely excited to work with the lymphoma community to launch this novel treatment and have built an incredible team of experienced commercialization experts who are ready to bring UKONIQ to patients. Next slide. Thank you. Despite the advances in treating indolent lymphoma over the last 20 years, there remains a significant unmet need for effective, and importantly, well-tolerated treatment options. Most patients will be diagnosed in their late 60s and early 70s, and therefore, the tolerability of their treatment is critical. Unfortunately, indolent lymphoma remains an incurable disease, and most patients will relapse and become refractory to existing treatment options. After initial first-line treatment, there is no recognized standard of care and as available options are largely viewed as suboptimum. Each year, there are approximately 18,000 to 20,000 patients in the U.S. who are in need of a new treatment for their relapsed disease in marginal zone and follicular lymphoma. And we estimate that of those, there are approximately 8,000 to 10,000 patients that fall within our FDA-approved indications. UKONIQ is the first and only multikinase inhibitor of PI3K-delta and CK1-epsilon for the treatment of relapsed/refractory marginal zone and follicular lymphoma. Based on feedback from our advisers and extensive market research over the last 12 months, we are confident that UKONIQ offers an attractive profile for patients in our approved indications. Physicians have been impressed with the combination of tolerability, including the low rate of discontinuations due to adverse events and the consistent efficacy across both marginal zone and follicular in a once-daily oral dosing regimen. The absence of a box warning as well as the unique mechanism of action of UKONIQ has been viewed as important points of differentiation by physicians. We believe this overall profile helps address many of the unmet needs in this market. As a team, we are laser-focused on ensuring that patients who can benefit from UKONIQ have the opportunity to do so. We understand the urgency needed to educate and drive awareness of this important new treatment option and therefore are focused on 3 simple priorities to drive our launch: one, accelerating the awareness of UKONIQ's differentiated profile with our target customers; two, proactively setting expectations on patient management to ensure a positive first experience; and three, minimizing patient access barriers. Next slide, please. We have built a truly best-in-class team to support the launch of UKONIQ. The team is hired, fully trained and prepared to execute. Our vision was to create a nimble, flexible customer-centric model that can scale over time to support future launches as TG continues to grow. We were meticulous in hiring top-tier talent from premier oncology companies with extensive experience launching and commercializing products in the hematology space. Our sales team has an average of 20 years hematology experience and 14 years of specific experience in lymphoma. Knowing we are launching during a pandemic, we only hired those with strong relationships, experienced with key oncology centers and demonstrated ability to execute both virtually and in live engagements. Everyone on the team has recently launched a product in COVID or has been successfully selling in a virtual environment. We will have a targeted approach to launch with highly coordinated execution plans focusing on large community practices and major academic centers where we know there are high volumes of patients and a large percentage of physicians are already aware of UKONIQ. We're also well prepared to ensure that patients and health care providers have a positive first experience with the necessary education, support and resources. All in, including our sales, medical, marketing and access teams, we have a field footprint of approximately 70 highly experienced professionals focused on providing education about UKONIQ's compelling efficacy and safety profile and facilitating the integration of UKONIQ into the relapsed, indolent, non-Hodgkin lymphoma treatment paradigm. We are already off to a strong start. Since announcing our approval on Friday, we have already reached over 1,200 health care providers with personal e-mails or calls. We've conducted a national broadcast promotional program, and we have scheduled engagements starting later today. COVID, of course, does present some challenges to driving an awareness, but we have had almost a year to prepare and have invested in the technology platforms and multichannel and digital capabilities to complement our field force that will enable the teams to effectively engage right out of the gate. We are also seeing live interactions in some areas of the country as more health care providers are becoming vaccinated and have established appropriate safety protocols. Simply stated, we have invested in the right areas with the right people so that we can flexibly meet our customers where they need us to be. At TG -- next slide, please. At TG, our commitment and dedication to the patient goes beyond the science. We believe that access is essential to ensuring that the advances are available to patients who are in need of treatment options. We are committed to supporting patients in accessing UKONIQ, which is why we built a comprehensive patient services program called TG Patient Support. The program includes a dedicated team to provide patients with access and reimbursement support, financial assistance programs for those who are eligible and nursing support for patient education. Additionally, our highly experienced account management team has already been engaging with payers over the last several months to help ensure patients who can benefit will have access to UKONIQ. We will now work urgently to ensure UKONIQ is added to formularies as soon as possible. The wholesale acquisition cost for a 30-day supply of UKONIQ is $15,900. Statutory government discounts such as Medicaid rebate sales to 340B covered entities and [ VA ] facilities, among others, will reduce the net price that TG will receive. We've also designed an effective distribution model to support our patients and health care providers as part of our ongoing commitment to patient access. UKONIQ will be made available in the next week through a specialty pharmacy and specialty distributor network. In summary, we are ready to launch UKONIQ. We have the right people in the right positions. We know the market. We have built an incredible patient focus and high-performance culture. And UKONIQ is a differentiated product that will provide value to patients and providers. We are excited to take this big step forward in our mission to bring novel treatments in combinations for B-cell malignancies and autoimmune diseases to patients. Thank you for your time. And now I'll hand the call back over to Mike.

Thanks, Adam. And conference operator, I think we're ready to begin the Q&A session. If you could queue that up, please?

[Operator Instructions] Our first question today is coming from Alethia Young from Cantor Fitzgerald.

Let me be the first congratulate you on the approval for UKONIQ and the faster expected approval in follicular. So two for me. One, obviously, this space is one that's had a lot of history in some patients -- I mean some doctors have baggages and perhaps patients. Can you talk a little bit about like lessons learned? And you mentioned kind of really, I think, servicing, handholding patients and doctors. Like how do you really handhold some of these toxicities to get people to the other side, obviously, because the medicines are quite durable? And then the second question is just I was wanting you to maybe opine upon like how like maybe REV rituximab has changed the follicular landscape? And like where is that kind of place you guys? And how do you think about follicular as a percentage of the total 8,000 to 10,000 patients you discussed?

Sure. Adam, you want to take a crack at those?

Yes. So let's start with the first one about lessons learned. I think, first of all, we want to be proactive on patient management. We have a dedicated medical team that is there to help community and academic physicians and nurses become comfortable and know what to expect from UKONIQ right out of the gates. We want to make sure we do a really good job of that so they're ready to manage these. A lot of the toxicities that we see in this area are things that they are very familiar with and can handle quite easily if they're made aware upfront. So we have a team ready to do that, and we make sure that we do a really good job of that. And I think in previous launches around PI3K, that might not have been really well done. I think we do have a differentiated product. I think that, that may be another point of differentiation here from the previous launches. We think our safety profile is very different. And then as far as the landscape, like I mentioned in the prepared remarks, I think after first-line therapy, there really is no standard of care. Each of the therapies that exist today, including REVLIMID and R2, which I'm very familiar with, having worked on that for many years before I got here, they all have their pros and cons, right? And I think there is no standard because a lot of them are not viewed as optimal. And we've seen that in the market research. We've seen that physicians are eager to get another treatment that's oral once a day, well-tolerated and effective across both of these diseases. So we're feeling good about it. And we think that this will fit well into the treatment paradigm in the [ relapsing ].

Our next question today is coming from Eric Joseph from JPMorgan.

Congrats on the approval. So just one surprise that we noticed later was the indicated line of therapy in follicular, essentially a fourth line label compared to the third line side of NDA filing. Do you have a sense of what FDA's conservatism is there for the later line indication? And how tightly do you expect payers to manage patient eligibility between sort of second to fourth line? Or how do you expect that payers [ manage the reimbursement line of therapy ]?

Thanks, Eric. So I'll take a crack and then maybe Adam can jump in. In terms of the line of therapy, look, since we conducted the trial and filed for approval, R2 was approved. So if you think logically about it, there was 2 prior lines. Now there are 3 prior lines approved with R2. So I think it was a sort of logical place for the FDA to go with the label. So that, I think, is just pretty straightforward. In terms of -- what was the second part of your question? I wrote it down, and I didn't write the whole thing down. How do you think -- sorry, Eric.

No problem. How have your payers sort of managed reimbursement line of therapy?

Yes. So I'll let Adam address that one. But just I will say anecdotally, you've got ibrutinib, which gets used quite frequently across follicular lymphoma, which has no label. And I think venetoclax is actually used at some level also in those patients without a label. But Adam, what are your thoughts on that question?

Yes. So I think payers will manage to believe -- I mean, one, this is not an area that is extensively managed. Payers will manage the label and [ compare your listings ]. But I don't -- we don't think it's tightly managed.

Okay. Maybe just one follow-up, if I could. You provided a little bit of color on sort of where you are in practitioner engagement. Can you just maybe give us a prelude to how you will be talking about a progress with the launch going forward, be it in terms of prescriptions, unique prescribers? And will you be making the prescription information available to third-party vendors?

Yes. So the last part about the third-party vendors, Adam, I'll let you tackle on the front end. I think we're figuring out our communication strategy, Eric. So I don't know that we have one as of yet. We'll look to lots of examples of companies doing early launch, and we'll try to model after that. Adam, the other part of your question?

Yes. I believe we are going to make the data available, Eric, and I'd double check on that. But I do think we are going to make that available, but let me double check on that.

Our next question is coming from Chris Howerton from Jefferies.

Great. Congratulations. And for what it's worth, both Roger and I really like the name UKONIQ.

Thanks, Chris.

All right. So I guess I think, Mike, you said -- you were saying that you're working with the FDA on the confirmatory trials. But is there any color on the what you would like to see or what you would hope to see for the trial designs and maybe some fence around what your time lines might be at this point?

Yes. So I don't want to get too far over my skis on the design of the trial while we're still having discussions. But in terms of timing, look, we'd like to start it as soon as we can. The path that we're going down in terms of the design, we were excited about. We think it will be a great study for us and a way for us to get into earlier lines of therapy in follicular lymphoma. So yes, so we're excited. We want to get it started as soon as we can. If all goes well, I'm assuming sometime in the second half of this year, we'll be able to start that trial. And again, we're working as hard as we can to get it done because as it stands right now, it's a design that we're pretty excited about.

Great. Okay. All right, that's fair. And then maybe as a follow-up to Adam and maybe to Owen as well. I don't know if you all were planning whether or not there would be a black box warning or not. But there was always, maybe at least in my mind, the potential there. So does that change the way that you address the physician community in terms of what the profile is? Or was that kind of the base case going in for you guys?

Adam, you want to go ahead?

Sure. Yes, I'm not going to say it was the base case. I think we knew it was a possibility. We were obviously pleased that it was not in our label. We do think it's a point of differentiation, although we want to make sure that we're focused on setting appropriate expectations with toxicity management. This drug is good. It's well tolerated. But there certainly are some things you need to look for, and we'll do a great job of that. So I don't think it changes our promotional strategy, although it is a differentiator, I think, in the minds of physicians and nurses.

Okay. Very good. Well, congrats again.