Rain Enhancement Technologies Holdco Inc (RAIN) 2022 Q2 法說會逐字稿

Good day and welcome to the Rain Therapeutics second-quarter 2022 earnings conference call. Today's conference is being recorded. At this time, I would like to turn the conference over to Bob Yedid. Please go ahead.

Thank you, operator, and good afternoon, everyone. With me today on the call are Avanish Vellanki, Chief Executive Officer of Rain Therapeutics; and Nelson Cabatuan, SVP of Finance. During today's call, Avanish will provide an overall business update including Rain's clinical programs and research efforts, and Nelson will review the financials.

Before we begin, I would like to remind you that statements made during this conference call that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based upon Rain's current expectations and involve assumptions that may never materialize or may prove to be incorrect.

Actual results may differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties as described in Rain's annual report on Form 10-K for the year ended December 31, 2021, and subsequent filings with the Securities and Exchange Commission. All forward-looking statements made during this conference call are based on management's assumptions and estimates as of today, August 4, 2022.

Rain undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after today, except as required by law. With that said, it's my pleasure to turn the call over to Avanish Vellanki, CEO of Rain Therapeutics. Avanish?

Thank you, Bob, and thanks to everyone for joining us for our second-quarter earnings call. We are pleased to share updates today on our two ongoing clinical trials: the MANTRA Phase 3 registrational trial in patients with liposarcoma and the MANTRA-2 tumor-agnostic basket trial in advanced solid tumor patients exhibiting MDM2 gene amplification.

We're going to have a different format on this call with prepared remarks from myself and Nelson Cabatuan, Rain's Senior Vice President of Finance, and then move more quickly to Q&A, where we'll be joined by our President and Chief Scientific Officer, Dr. Bob Doebele and our Chief Medical Officer, Dr. Richard Bryce.

First and foremost, we announced earlier this morning that we have completed enrollment in the Phase 3 MANTRA trial in patients with well-differentiated/dedifferentiated liposarcoma. As at the end of July, we completed enrollment with 175 patients, exceeding our 160-patient target and five months ahead of our prior year-end guidance.

We saw a clear acceleration of enrollment since our update call at the end of the first quarter of this year, at which point, we announced we were approximately half enrolled. We believe this speaks to the tremendous unmet need in patients with dedifferentiated liposarcoma for which milademetan offers a targeted therapeutic strategy relative to standard cytotoxic options.

We enrolled across 70 international sites in the US, Europe, and Asia. And as a reminder, this pivotal trial compares milademetan monotherapy to the approved agent, Yondelis or trabectedin. Milademetan was dosed at 260 milligrams daily for three days, followed by 11 days off therapy. We refer to this as the 3-out-of-14-day schedule.

We anticipate topline data from this trial in the first half of 2023. We also anticipate that if the MANTRA data are supportive, we would submit an NDA for milademetan in liposarcoma in the US with similar submissions in Europe and possibly, other regions as well.

Second, our Phase 2 tumor-agnostic MANTRA-2 trial is progressing as planned with 11 sites activated in the US to date and additional just-in-time sites, opening as necessary from referrals from the Tempus and Caris genomic testing services. To recap, this trial is evaluating milademetan in multiple solid tumor types in patients with p53 wildtype tumors that exhibit high MDM2 gene amplification at a copy number of 12 or greater.

We anticipate enrolling approximately 65 patients across a range of solid tumors and using the same dosing regimen as in the registrational MANTRA trial and continue to anticipate interim data of the first 10 available patients in the fourth quarter of this year.

We continue to anticipate commencing two additional trials before year-end, both the MANTRA-3 trial in Merkel cell carcinoma and the MANTRA-4 trial, evaluating the combination of milademetan with a checkpoint inhibitor, Roche's atezolizumab, in patients with p53 wildtype advanced solid tumors that exhibit loss of the gene CDKN2A.

Also, at the recent annual ASCO meeting, we presented preclinical data detailing the rationale for the MANTRA-4 trial. New preclinical data across multiple in vivo tumor models with CDKN2A loss and wildtype p53, supported potential synergy of milademetan and an immune checkpoint inhibitor in this genetic subset. We remain very excited about the potential for the MANTRA-4 trial and its strategy.

With that, I would like to turn it over to Nelson to review our financial results. Nelson?

Thank you, Avanish, and good afternoon, everyone. I'm pleased to provide an update of our financial results for the second quarter ended June 30, 2022. I would also like to invite you to review our Form 10-Q filed today for more details. For the second quarter of 2022, we reported a net loss of $17.6 million, compared to a net loss of $8.2 million in the second quarter of 2021.

Research and development expenses were $14.3 million in the second quarter of 2022 as compared to $5.5 million in the second quarter of 2021. The increase was primarily driven by R&D costs, mainly, for our lead candidate, milademetan, from the ongoing Phase 3 MANTRA clinical trial, our ongoing Phase 2 MANTRA-2 trial, as well as personnel costs.

As of June 30, 2022, Rain continues to have a strong balance sheet with $106 million in cash, cash equivalents, and short-term investments. We anticipate that our second-quarter cash position will provide runway into mid-2024, and we believe that the milademetan program is well funded.

With that, I'll now turn the call back over to Avanish.

Thanks, Nelson. Rain continues to focus on the clinical execution of our milademetan program. And we're thrilled with the rate of progress thus far, particularly with enrollment now completed in our registrational Phase 3 MANTRA trial five months earlier than previously guided.

Rain anticipates a broad clinical data set for milademetan with a steady cadence of updates over the next two-plus years. We look forward to Phase 3 MANTRA data in the first half of 2023. With that, we'll have Dr. Bob Doebele and Dr. Richard Bryce join us and available to answer questions. Operator?

(Operator Instructions) Michael Schmidt, Guggenheim.

Hey, good afternoon. This is Yige on for Michael. Thanks for taking our questions, and congrats on the earlier-than-expected enrollment for the MANTRA. I guess we'll start with the question on LPS. So one competitor has presented some early data at ASCO on its MDM2 inhibitor in LPS. So what do you think of the potential read-through from the presented data to milademetan's competitive positioning into the ongoing MANTRA study? And I have a follow-up. Thank you.

Hi, Yige. This is Avanish. Thanks for the question. Let me turn the question over to Bob.

Thanks for the question, Yige. Of course, we're not going to comment on (technical difficulty). However, we're aware that BI's entry into the MDM2 space validates our approach at Rain, using MDM2 as a validated cancer target in select patient populations.

Got it. And then I guess as a follow-up, can you elaborate your current thinking and consideration around the first-line strategy in LPS, either with mila monotherapy or maybe perhaps even in combination with other agents? Thanks.

Yeah. I'm happy to take that one, Yige. So obviously, we want to wait on MANTRA results before starting to articulate subsequent plans. So I think that's first and foremost. But I think we will comment that, if successful -- if MANTRA does have a successful outcome, our expectation would be to expand the opportunity across multiple indications inclusive of frontline liposarcoma. But we don't want to get into the details of what that looks like at this moment in time.

Okay. Great. Thank you very much.

Absolutely.

Joe Catanzaro, Piper Sandler.

Hi, this is Sam on for Joe. Thanks for taking our question. Could you just provide a little more detail on what you think drove this acceleration in enrollment? And then now with this rapid acceleration, do you think that the topline data can now come maybe in the early part of first half 2023?

Hi, Sam. Thanks for the question. Let me turn that over to Richard.

Sure. Happy to answer that. So I think this speaks to two things. First of all, I do want to take this opportunity to call out the excellence of our clinical team and our collaborative partners to -- with this achievement, this incredible enrollment into the study in the last six months. And I think that surpassed expectations both at our level and the physician level.

What does it mean? Beyond that, I think it's important. It underlines or underscores the tremendous enthusiasm among physicians and patients for a new treatment option, particularly a targeted treatment option for patients with [dedifferentiated] liposarcoma, which as we know is MDM2 amplified by definition.

And so that's really reflected in the enormous enthusiasm to enroll into the study, and we would expect to see that play itself out in future trials and in future circumstances, hopefully, if the study is positive in the commercial setting as well.

Great. Thank you.

Jeff Jones, Oppenheimer.

Thank you and good afternoon, guys. I guess two questions from me. On the MANTRA-2 basket trial, any idea what kind of duration of response data you'll have in hand when that reads out topline end of the year? And then the second question, any updates on the RAD52 program?

Hi, Jeff. Thanks for the question. Let me turn that question over to Bob.

Yeah. So to answer the first part of the question regarding MANTRA-2, we've messaged in the past that we expect to have 10 patients with a meaningful operational follow-up. This would be at least four months or approximately two scans for those patients as well as a [mean] duration, and that expectation has not changed.

Regarding your second question on the RAD52 program, we are continuing to progress that project. And we've signaled in the past however that we have mitigated spend on that to focus on milademetan. And based on that, there's some progress in the RAD52 program.

Great. Thanks.

(Operator instructions) Soumit Roy, Jones Research.

Hi, everyone. Possibly, this is a question for Bob. I was wondering as we are saying in multiple Phase 3 trials, the standard of care or the control arm is outperforming the historical data -- anything that -- possibly due to better tumor monitoring or better supportive care. So curious to get your thoughts on -- if you think the control arm could outperform the seven-year or eight-year-old historic data or whether if there is any concern for that.

Thanks for the question, Soumit. Bob?

Yeah, no. I think this is -- we're looking at data, obviously, from the registrational trial of trabectedin, which is the control arm in our trial. That is a modern clinical trial with robust subset analysis, and we see no indication to expect that the trabectedin will outperform in the MANTRA study compared to its registrational (inaudible).

Yeah. Let me add there, Soumit, that it's, of course, difficult to predict. But as a reminder in terms of the clinical trial design for MANTRA, we actually did assume that the control arm would modestly be improved, its efficacy from the registrational study. So we did actually anticipate any potential improvement. It still anticipates an opportunity to succeed (inaudible).

Great. I have a quick second question, if I may. Are you allowing crossover from the control arm in the trial -- MANTRA trial?

No, we're not. There's no crossover in MANTRA.

Thank you so much for taking the questions, and congratulations on the fast enrollment.

Tony Butler, ROTH Capital.

Yes. Thanks very much. I just wanted to ask about MANTRA-4 since it's one of the things we haven't discussed this evening in detail, given the population size and the opportunity for the compound in that CDKN2A population.

And as you're looking forward to that trial, I would think that the number of sites that could or would like to participate would be quite a few. Could you just provide a brief discussion, if I may, on -- are you thinking about a small number or would this -- would you get in with both feet and perhaps, expand it including international -- thank you -- even if it is a Phase 1, I understand?

Let me -- thanks for the question, Tony. Let me turn that question over to Bob.

Yeah. Thanks for the question. Obviously, the MANTRA-4 study is very similar to the MANTRA-2 study, which is ongoing, in the fact that it is a basket design, all-comer solid tumors, genetically selected patients. So we hope to take the learnings from the MANTRA-2 trial into the MANTRA-4. And you could expect probably some overlap in terms of the sites that we'd use.

Obviously, as you mentioned, the population is far larger for the CDKN2A-loss population. And therefore, we would expect enrollment on a per-site basis to be much, much faster, given the select patient population [of those]. So hopefully, that answers your question.

Thank you, Bob. Appreciate it.

That concludes today's question-and-answer session. I would like to turn the conference back over to Mr. Vellanki for any additional closing remarks.

Thank you, operator. And we want to thank everyone for joining us on this quarter's earnings call, and we look forward to updating everyone on our progress over the next quarter. Thank you.

This concludes today's call. Thank you for your participation. You may now disconnect.