Ladies and gentlemen, thank you for standing by and welcome to the Legend Biotech Reports Fourth Quarter 2020 Financial Results. At this time, all participant lines are in a listen-only mode. After the speaker presentation, there will be a question-and-answer session. [Operator Instructions] Please be advised that today's conference is being recorded.

[Operator Instructions] I would now like to hand the conference to your speaker today, Jessie Yeung. Please, go ahead, ma'am..

Good morning, everyone. I hope everyone is safe and healthy. Welcome to Legend Biotech's fourth quarter and full year 2020 earnings conference call. This is Jessie Yeung, Head of Corporate Finance and Investor Relations for Legend Biotech. Joining me on today's call is Ying Huang, Chief Executive and Chief Financial Officer for Legend Biotech.

As usual, just before we get into the details, this review is being made available via webcast, accessible through the Investor Relations section of the Legend Biotech website. Please note that today's presentation includes forward-looking statements.

We encourage you to review the cautionary statement regarding such statements in today's presentation, as well as the company's prospectus filed with the SEC on June 8, which identifies certain factors that may cause the company's actual results to differ materially from those projected, should one more or more of these risks or uncertainties materialize, or should underline assumptions prove incorrect, actual results may vary materially from those described in this presentation.

Legend Biotech specifically disclaims any obligation to update any forward-looking statements. Moving to today's agenda. We will be starting with opening remarks followed by the financial results. Then we will do a quick review of the ASH 2020 data before we end today's call with a Q&A session. I would now like to turn the conference call to Ying..

Thank you, Jessie, and good morning, everyone. Welcome to our fourth quarter and 2020 results earnings call. So before I start, I would like to thank everyone and hope everyone is safe and healthy. Let me begin today's call by reminding what we do here and why we do what we do here at Legend, it is to serve the patients.

Our team at Legend Biotech works tirelessly to bring innovative therapies to patients living with debilitating diseases. I am extremely proud to be a part of this team and grateful for this effort. Within the fourth quarter of 2020, we achieved critical milestones.

At the recent ASH 2020 conference, our collaboration partner J&J and Legend shared exciting results from combined Phase 1b and Phase 2 CARTITUDE-1 study of cilta-cel and investigation of BCMA-targeted CAR-T therapy. The data continued to show a very high overall response rate. Specifically, 97% of patients achieved a response.

More importantly, 67% of patients achieved a stringent complete response at a median follow-up of 12.4 months. Also in December 2020, we initiated rolling submission of BLA or Biologics License Application to the U.S. FDA for cilta-cel.

This submission is based on results from the pivotal Phase 1b, Phase 2 CARTITUDE-1 study that evaluated the efficacy and safety of cilta-cel in the treatment of patients with relapsed and/or refractory multiple myeloma. We also announced the U.S.

FDA cleared the IND application for LB1901, which is Legend Biotech's investigational autologous CAR-T targeting CD4 for the treatment of T-cell lymphoma.

Given that a substantial portion of patients with peripheral T-cell lymphoma and also cutaneous T-cell lymphoma experience relapse, even with the current treatment options, there remains a high unmet medical need for these patients. With this IND clearance, we're planning to initiate a Phase 1 first-in-human study for LB1901 in United States.

Lastly, we also achieved the designation of accelerated assessment in Europe for the treatment of relapsed and refractory myeloma for cilta-cel.

The CHMP or Committee for Medicinal Products for Human Use in European Medicine Agency accepted the request from Janssen, our collaboration partner for this assessment, which means instead of the 210 evaluation days for the evaluation process by EMA, this accelerated assessment usually takes 150 evaluation days.

Now let's turn over to the financial results from 2020 and also fourth quarter 2020. Our net loss under IFRS accounting standards in 2020 was about $303 million. This compared to a loss of $133 million in 2019. Loss per share for 2020 was $1.28, compared to $0.66 loss per share for 2019.

And these results were driven mostly by larger team and also a higher number of clinical trials, as well as a higher number of clinical outpatients in our CARTITUDE program as well as the CARTIFAN program in China.

In terms of quarter-over-quarter comparison, in the fourth quarter of 2020, our net loss was about $58 million and that compared to $64 million loss in the fourth quarter of 2019. Loss per share for the fourth quarter of 2020 was $0.22, compares to $0.32 loss per share in the Q4 of 2019.

In the next slide, as you can see, we continue to push forward with a robust pipeline of next-generation cell therapies. Beside the BCMA program for which we're conducting the CARTITUDE-1 in the U.S., the CARTITUDE-2 five cohort Phase 2 trial globally and also the ongoing Phase 3 randomized active controlled CARTITUDE-4 Phase 3 trials.

In China, we're conducting the pivotal Phase 2 trial CARTIFAN-1. Besides the BCMA program, we're continuing to conduct the Phase 1 first-in-human studies, IP studies in China. This includes a dual targeting CD19, CD22 auto CAR-T for non-Hodgkin's lymphoma.

A dual targeting CD33 CLL-1 auto CAR-T for acute leukemia and also CD4 targeting T-cell lymphoma program. In the field of allo or allogeneic CAR-T, we're conducting two active Phase 1 studies. The first one is a CD20 targeting allogeneic CAR-T for non-Hodgkin lymphoma.

The second one is gamma delta BCMA targeting T-cell program for the treatment of myeloma. In a solid tumor field, we're conducting a study for Claudin 18.2, targeting auto CAR-T for gastric cancer and pancreatic cancer. In addition, we recently started a Phase 1 study for a mesothelin targeting auto CAR-T for ovarian cancer.

Now I'd like to take a moment to highlight some of the clinical data from the CARTITUDE-1 study that was presented recently at the ASH 2020 conference. As you can see from this slide, the data continues to show a very high overall response rate, specifically 94 out of the 97 patients enrolled achieved a response.

More importantly, 67% of those patients achieved a stringent complete response and the median follow-up of 12.4 months with the data cut. The median progression-free survival has not been reached yet.

This study also demonstrated a manageable safety profile for cilta-cel at the recommended Phase 2 dose, which is 0.75 million cells per kilogram body weight. Given that these patients were heavily pre-treated, in fact, the medium life of prior therapy was six. We believe these results are really excellent.

As we look into the rest of 2021 and also into 2022, I would like to discuss the near-term target for the company. In the first half of 2021, we're working with our collaboration partner Janssen Pharmaceuticals to potentially file the MA Application with EMA for cilta-cel.

As I just previously mentioned, recently the EMA accepted a request from Janssen on accelerated assessment for the marketing application for cilta-cel. We're also planning to file BLA in China, pending discussion with CDE for cilta-cel in the second half of this year.

In the United States, which is the most important market for cilta-cel, we're targeting FDA approval by end of 2021. Lastly, we're planning to initiate the Phase 1 first-in-human study for LB1901 for the T-cell lymphoma in the United States.

Our collaboration partner Janssen is also working to file an NDA to the Japan Ministry of Health, Labor and Welfare for cilta-cel in the second half of this year. In 2022, we're expecting the EMA approval for cilta-cel in European Union. We're also expecting the CDE approval of cilta-cel in China.

In terms of the data update for cilta-cel, I'm pleased to announce that Legend Biotech in collaboration with Janssen intend to present updated data from the CARTITUDE-1 and CARTITUDE-2 Phase 2 studies at major medical conferences in 2021.

In addition, Legend Biotech along with Janssen anticipates to publish the data from the Legend-2 Phase 1 first-in-human studies in 2021. As a reminder, this Phase 1 study enrolled a total of 74 patients in the Phase 1 study. And every patient has completed the required three-year follow-up as of November of 2020.

In the next slide, as you can see, we have a comprehensive clinical development program for cilta-cel in patients with multiple myeloma. In 2020, we initiated a global Phase 3 study called CARTITUDE-4. We also expanded the multi-cohort Phase 2 study called CARTITUDE-2.

The most recent cohort, cohort-e [ph] is enrolling patients with newly diagnosed multiple myeloma or first-line myeloma. We intend to present data from CARTITUDE-1 and also CARTITUDE-2 studies at major conference in 2021. And then, as I mentioned, we also anticipate to publish in a paper format, the Phase 1 Legend to long-term fallout.

With that operator, can you please open the Q&A?.

Of course. [Operator Instructions] Our first question will come from the line of Matthew Harrison from Morgan Stanley. Your line is open..

Great. Good morning. Thanks for taking the questions. I guess at two for me. Ying, I believe you said, your plan is to have the BLA approved in the U.S. by the end of 2021.

Can you maybe just comment, where and the progress of the rolling submission you guys are now? And are you able to meet that time line? I assume, you're assuming an accelerated assessment. So if you could comment on that that would be great.

And then second, maybe you could just -- we haven't talked about this before, but maybe you could just comment broadly on your expectations around the commercial opportunity in China. And what, sort of, work you're doing to prepare for that, given you're planning to submit sometime later this year? Thanks..

Thank you Matthew for the question. So let me address your first question about the BLA in the United States. So, we did say that both J&J and Legend expect the cilta-cel to be approved by FDA by end of this year. So we're expecting to ask FDA for a priority review, given our breakthrough definition by the FDA.

And as, you know, typically, after a BLA package has been completed, there's a two-month acceptance period, following which there is a six-month review period. So the total is eight months after we complete the BLA package. So, broadly speaking, J&J and Legend have stated that we expect the drug to be approved by end of 2021.

Therefore, if you do the math, that means, we should expect the BLA package to be completed by end of April. So we're not providing any guidance or provide any clarity on exactly when this is completed. But what we can tell you today is that, we're completely on track in terms of the BLA submission here.

And again, we stand by the expectation that we expect cilta-cel to be approved and launched by 2021 end of this year. So that's what we'll say about the BLA submission time-line. And as a reminder for -- yeah. Go ahead. .

Sorry, I was going to say, before you answer the second question, can I just ask a quick follow-up on that?.

Sure..

You announced -- will you announce when you complete the submission, or only when you have acceptance from the FDA?.

So J&J and Legend are actually discussing the disclosure around the BLA submission and also the BLA acceptance. Unfortunately, I am not at liberty to tell exactly today, what our plan is. Suffice to say that you will hear from the partners here about this. Probably the only thing I can say is that, stay tuned.

But you will hear something from J&J and Legend about this. So, to address your second question Matthew, in terms of commercial market potential in China, I guess if you look at the current landscape in China, there's no commercial CAR-T therapy yet.

If you look at the CDE process, so far two product, or two product candidates namely Yescarta from Fosun Kite which is JV from Fosun and Kite Pharma has been submitted. And right now, it's still being reviewed by CDE. The other one is the rights to sell product from Wuxi/Juno, which is a JV between Wuxi and the Juno Pharmaceutical.

And again, that is in the review process. The application has been submitted to CDE. So, given that there is no commercial CAR-T therapy yet in the Chinese market, it's difficult to comment on for example pricing and also the coverage from commercial insurance or reimbursement from the health care plans in China.

However, if you look at the sheer number of patients, who suffer from myeloma or foci [ph] and beyond myeloma, it's actually a similar or bigger number compared to what we have in the States.

As you know in the States every year about 14,000 patients unfortunately die from multiple myeloma and that's the minimal addressable market for us pending the FDA approval. And we believe the size of the patient population in China is actually larger.

However, since there's no commercial product that's been already approved in the market yet, we have no benchmark to assess that. In our opinion in the first launch phase, this drug is most likely going to be paid through self-pay mechanism. That is out-of-pocket from patients for this.

Because right now there's a ceiling for China national coverage for the drug cost. And we just don't think any CAR-T would come to the market immediately with the national health plan coverage..

Great. Thanks very much..

Thank you..

[Operator Instructions] Our next question will come from the line of Biren Amin from Jefferies. You may begin..

Yeah. Hi, guys. Thanks for taking my questions. Ying on CARTITUDE-2, I believe you're going to have some data this year.

Can you just maybe talk about which cohorts we should expect data in because I think there's about four or five cohorts in the trial that you guys are investigating? So, any color you could provide there would be helpful?.

Sure. Thanks Biren for your question. So, as you know, we have already opened five cohorts for the CARTITUDE-2 program so far, which is a global trial. And if you look at the patient population, we're enrolling because we opened the Cohort A first.

So, I just want to remind you that in the Cohort A, we are planning to enroll 20 to 30 patients, who were treated with one to three prior lines of therapy and also refractory to Revlimid. So, this is essentially the same patient population we're testing in the randomized global Phase 3 trial called CARTITUDE-4.

However, this is a single-arm Phase 2 study here. So, as you can imagine, if we do plan to publish or present any data from the CARTITUDE-2 trial, most likely it will start with Cohort A. That is the most likely patient population in which we will present data in 2021..

Great. And then, maybe on some of your Phase 1 programs, you've got several programs, the CD19, CD22, CD3, CLL and CD20 allogeneic.

Can you just maybe talk about when we could expect data from some of the Phase 1 studies? Should we expect it this year, or is it more likely next year?.

Absolutely. Happy to answer that question. So Biren, we do have six active Phase 1 ongoing studies in China for various indications. And depending on which program, we're in also different stage. In some programs, we're screening patients. In some programs, we’re open new sites getting through the ethical committee approvals.

In some programs, we're actually already dosing patients by now. So, given the pace of the enrollment, we're not providing any official guidance. However, I think the earliest possible time for us to report any data from these Phase 1 programs, would be second half towards the end of this year, we should be able to have some data in hand.

And that is we should have a reasonable number of patients in terms of data..

And would these data readout coincide with the IND filing here in the US for these programs?.

Not necessarily, Biren. For example, we're already working on an IND for the Claudin 18.2 targeting auto CAR-T for gastric cancer and pancreatic cancer. Right now, at the same time, we're also conducting a Phase 1 IIT study in China for this same program in gastric cancer patients.

Although, we don't have much clinical data for that program yet but, we have made a decision to move forward, given our belief in the scientific rationale for targeting Claudin 18.2 as a treatment for gastric and pancreatic cancer. And also, given the preclinical data we have observed in our lab, we have made a decision to move forward.

So, we're actually actively preparing for an IND application in the US for that program later this year..

Great, thank you..

Thank you..

Thank you. And I'm actually not showing any further questions in the queue. I'd like to turn the call over to Ying for any closing remarks..

Okay, great. Again, I just want to thank everyone for dialing in, and thank you very much for your interest in Legend Biotech. And Jessie and I will be standby. If you have any further questions, feel free to contact us through any means. And again, thank you very much, and we look forward to a very productive year in 2021..

Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect..