Bristol-Myers Squibb Company (BMY) Earnings Call Transcript & Summary

Ladies and gentlemen, thank you for standing by, and welcome to the Bristol-Myers Squibb Investor Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. [Operator Instructions] I would now like to hand the conference over to your speaker today, Mr. Tim Power, Vice President, Investor Relations. Thank you. Please go ahead, sir.

Thank you, Daniel, and good morning, everyone. Thanks for joining us for our call this morning. I'm joined today for today's call by Giovanni Caforio, our Chairman and Chief Executive Officer; by Samit Hirawat our Chief Medical Officer and Head of Global Drug Development. Giovanni will briefly open the call in a minute and then hand over to Samit for opening remarks. Also joining me today along with Giovanni and Samit for the Q&A section is Nadim Ahmed, President of Hematology. Before we get started, let me read our forward-looking statements. During this call, we make statements about the company's future plans and prospects that constitute forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the company's SEC filings. These forward-looking statements represent our estimates as of today and should not be relied upon as representing our estimates as of any future date. We specifically disclaim any obligation to update forward-looking statements even if our estimates change. With that, I'll hand over to Giovanni.

Thank you, Tim, and good morning, everyone. In light of this morning's announcement, we believe it is important to get on a call with all of you to describe what's happened and to provide you with our next steps. Let me just say that we are surprised and disappointed by the receipt of the refusal to file letter from the FDA for ide-cel. What I can tell you is that we are very confident in the profile of ide-cel. We believe it is an important potential medicine. We continue to work closely with bluebird, and we have a clear path to resubmit by the end of July at the latest. We are committed to bringing this medicine to patients as soon as possible and to meeting our CVR milestones. Samit will open with some details surrounding this morning's announcement, and then we will move quickly to the Q&A. Samit?

Thank you, Giovanni, and good morning, everyone. Let me walk you through the situation surrounding our announcements this morning. As we previously communicated, we submitted the BLA for ide-cel to the FDA at the end of March. Per the normal practice, the submission was informed by discussions we had with the FDA during our pre-BLA meeting. Based on those discussions, we believe we provided a complete dossier for a review by the FDA. However, as we've announced this morning, the FDA has notified the company of its view that the CMC component of the submission required further details to complete the BLA review, and the FDA issued a refusal to file letter. We are surprised and disappointed by FDA's decision. The company was not aware that the FDA had questions about the level of detail in the CMC module until we received the refusal to file letter. Nonetheless, following the FDA's decision, we are now focused on preparing the CMC module according to what the FDA has requested. We expect to resubmit the package by the end of July at latest, and we plan to request a priority review. As a reminder, ide-cel has breakthrough therapy designation. We plan to resubmit quickly because per the refusal to file letter, there is no request for more clinical or nonclinical data beyond the request for additional information in the CMC module. Despite today's news, what has not changed in our -- is our confidence in the profile of ide-cel. We believe the clinical data for ide-cel are very robust and believe this is a very important potential option for patients with highly refractory multiple myeloma. Later this afternoon, you'll see the strength of the clinical profile will be further supported by the updated data from the KarMMa study to be presented at ASCO. We are looking forward to making this treatment option available to patients with multiple myeloma as soon as possible. As mentioned, our next step is focusing on advancing this important application quickly, bringing this medicine to patients as soon as possible. With that, let me hand it over to Tim for the Q&A session. Thank you.

Thanks, Samit. Daniel, could we go to our first question, please?

[Operator Instructions] Our first question comes from Chris Schott with JPMorgan.

I'm just trying to get a little bit more color on this additional data the FDA is requesting. I guess, was this data that wasn't previously available? Or is it different than what's been requested from a manufacturing standpoint from some of the other CAR-Ts? It just seems like you got a quick turnaround you're targeting here. So I'm just trying to better understand why, I guess, whatever you're having to pull together wasn't part of any original submission package.

Well, thank you for your question. So thank you. As we just narrated and what we have learned, first of all, we are surprised and disappointed by this development. Now from the letter that we have received from a refusal to file perspective, what the FDA has really requested is to get further details of the protocols and processes that we have conducted. And it is not related to any new experiments or new clinical studies to be conducted. It is primarily in the CMC section that we have talked about.

Our next question comes from Seamus Fernandez with Guggenheim.

Great. So I guess what I'm also trying to understand a little bit, if you guys could help us, can you just walk us through who of the 2 companies had direct responsibility for submitting the file? And also, I'm sure it was a coordinated effort on your part at bluebird -- on your part and bluebird's. But can you just help us understand whose responsibility it was to specifically file and also to kind of structure the BLA submission? Did bluebird have specific responsibilities here versus Bristol's? Was it a joint effort? And I guess the other question is, can you just help us, with a little bit of additional information, from a CMC perspective, what are some of the more complicated parts of the process here? Is it the vector development? Or is it sort of the next steps in the process that one would say is really unique to the manufacturing approach here.

Seamus, this is Giovanni. Let me just start, and then I'll ask Samit to jump in. So first of all, as we mentioned, the submission we made in March was informed by discussions we had with the FDA during the pre-BLA meeting. And based on those discussions, we believe that we submitted a complete dossier to the FDA. What the FDA is requesting includes some specific details regarding the CMC module of the application, which is typically required during a regulatory review process. And to answer your question about how we are working. We are working very collaboratively with bluebird. And from a regulatory perspective, Bristol-Myers Squibb has the lead responsibility for the BLA. Samit, any further comment?

Yes. Thank you, Giovanni, and thanks, Seamus, for the question. Two-part question, of course. So as Giovanni mentioned, it is the BMS team that is responsible for the submission. And just remember that this is a team that includes people from both heritage companies, BMS as well as Celgene, and has delivered some important regulatory milestones across therapeutic areas, such as ZEPOSIA approval as well as Reblozyl, as you recently saw, priority reviews for 227, 9LA, CC-486 that we are now awaiting reviews and approvals for. We do plan to submit quickly because per the refusal to file letter, there's no request for more clinical or nonclinical data, as I mentioned earlier. Now in terms of the specificity, obviously, we will not get into the specifics of what exactly the FDA is asking for. But as a general point, as we've said, what the FDA is requesting includes specific details regarding the CMC module of the application, which is typically required in the regulatory review process rather than in the initial application, from our understanding. So that's what we're working on, and that's why we believe that we can turn it around quickly.

Our next question comes from Geoff Meacham with Bank of America.

It looks like from the bluebird 8-K, the validation and control processes, using lentiviral in the vector and the drug product manufacturing are the main issues. But I wanted to ask you, what percent of product manufacturing and clinical trials had met specifications? And then how, if any, has your ide-cel manufacturing processes changed over time?

Geoff, thanks for the question. Let me just start. Let me just repeat, we're confident that we are able to provide the product to patients. The question here from the FDA is really about the information that they requested to complete the review. It's really not about the ability to manufacture the product.

Our next question comes from Tim Anderson with Wolfe Research.

Daniel, maybe we'll go to the next one, and we can see if Tim is available in a minute.

Our next question comes from Matt Phipps with William Blair.

Are there any FDA meetings that need to happen here to clarify anything in the refuse to file letter? Or is this something where it's just a matter of you guys putting together the additional information to submit by July? Just wondering if there's any other gating issues to get to that July submission time frame.

Thank you for the question. As you can imagine, that as we looked through the letter, there are questions that the FDA has put to us. We do intend to have mutual conversations with the FDA to ensure that we are going to be able to answer the questions and provide a complete package from a CMC perspective as well in the submission that we've talked about before the end of July.

And our next question comes from Steve Scala with Cowen.

It seems there's 4 possible areas FDA could be scrutinizing, stability, scalability, inspection deficiencies or missing data. On stability, it would seem unlikely, given the nature of the product. On scalability, Giovanni, I just think you said there was no issue with manufacturing. On inspection deficiencies, it would seem unlikely because the plant probably has not yet been inspected. So that leaves missing data. If that is kind of the perception that perhaps investors have, do you wish to modify that perception at all? And then secondly, are there specific examples in the past where a refuse to file application was refiled within 10 to 11 weeks?

Thanks, Steve. As always, a very thoughtful question and very specific question. As we've just pointed out, it is not necessarily deficiency of additional experimentation or deficiency of additional data from that perspective, but rather details of the experiments and details of the data. And that is why we do believe that in collaboration and conversation with the FDA, we will be able to answer the questions with the data that are available. And we will be able to comply with the submission before the timing that we have announced before the end of June. And we are really looking forward to bringing this therapy to patients as soon as possible following that.

Thank you, Samit. Let me just add, as we mentioned, it is by no later than July that we will be able to resubmit.

Our next question comes from Terence Flynn with Goldman Sachs.

Apologies, I got on a little bit late. But just wondering, obviously, one of the major components of the Celgene deal was the push into the cell therapy space. And given the novelty of the technology, that offers a lot of opportunity, but also potential challenges, as now we've seen with both the 2121 and JCAR regulatory filings. So maybe you could just help frame for us the remaining challenges here on the forward? And then also how you see the opportunity? Are you still confident in the long term outlook?

Thank you, Terence. Let me start. I'll ask Samit first and then Nadim to both give you their perspective. So first of all, let me say, we have significant experience on cell therapy in the company. That's true across the board, from the regulatory team to the product development and manufacturing team and the commercial team. And our confidence in ide-cel and liso-cel has not changed. The data that supports the importance of these 2 really critical assets has not changed. In fact, as Samit mentioned, we look forward to presenting the KarMMa data at ASCO. So we remain committed to cell therapy. Importantly, we remain committed to both of the important assets that you've mentioned. Samit?

Yes. Thank you, Giovanni. And the only thing I would add beyond the fact that these are important medicines with transformational data certainly changing the landscape for treatment of the future in terms of potentially providing these treatments for patients with lymphoma for JCAR as well as for multiple myeloma for ide-cel. In general, from a therapeutic area perspective and from modalities perspective, certainly, cell therapies are a bit more complicated, and that's why there is a lot of question-and-answers that we go back and forth with, with health authorities. So it is usual to have that many questions. But as we have just shared, that these are -- we are in a place where we do have the data, we will be able to compile it and then provide it to the FDA so that we can move forward in terms of potentially bringing it to the patients as soon as possible. Nadim, maybe you want to add from a future perspective and a potential from commercialization?

Sure. I mean, obviously, the delays are disappointing. But they are delays that we feel do not impact the long-term potential of both of these therapies. So we still feel very confident about the clinical profile of liso-cel as a best-in-class treatment for that third line diffuse large B-cell indication. And ide-cel is the first CAR-T for multiple myeloma for highly refractory patients. So we feel very good about the long-term potential. And in fact, just this week, CMS issued their draft rule as reimbursement has been somewhat of a barrier in which they've now created a new DRG for cell therapy specifically. So we thought even better about the long-term potential of both of these assets and look forward to bringing them to patients.

Thanks, Nadim. I think, obviously, our partner, bluebird, has a call coming up, so we want to make sure we leave enough time for people who need to connect to that as well. So I think we might have time for one more question, if you don't mind, Daniel.

And our final question comes from David Risinger with Morgan Stanley.

So I was hoping that you could just comment on the lessons learned. So I guess what I'm thinking is, for example, regarding future filings, whether Bristol now needs to request input from different FDA leadership than you had engaged with in your previous discussions on this candidate and who you met with when you had your pre-BLA meeting. Could you provide some higher level perspective in the wake of this major surprise to the company?

Thank you, David. Let me just make a couple of comments here. So first of all, let me say, as Samit answered just a couple of minutes ago, cell therapy is a complex area. It is clearly one that is developing very rapidly. And there is a significant degree of complexity, particularly in the CMC section module of any application, which is really what we are discussing today. As we said at the beginning, we had discussions with the FDA during pre-BLA meeting. We believe we submitted a complete dossier to the FDA. So what we are really discussing here is the level of detail and information that the FDA has requested, which typically is part of the review process rather than the submission. And of course, we always learn from any resubmission. At the same time, now what we are focused on, we're focused on quickly assembling the data, and no later than July, the end of July, refiling the application. As we also said, as part of this process, we will meet again with the FDA. So we're comfortable that we're going to be working together with bluebird beginning immediately, and our focus now becomes the resubmission of the BLA in line with the expectation the FDA has communicated and the data that we have in-house. So thanks for the question, and thanks to all of you for your time today. We know that today's news was unexpected, and hopefully, we were able to provide you with additional color on the background and, importantly, where we're headed. Let me just echo, again, what Samit said earlier. Despite today's news, our focus now turns to resubmitting the application by the end of July at the latest. We are committed to getting this medicine to patients as soon as possible. Thank you for your time and attention, and have a good day.

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