Fate Therapeutics, Inc.
Q3 2018 Earnings Call Transcript

Published: 2 Nov 2018

[Operator:]

Welcome to the Fate Therapeutics Third Quarter 2018 Financial Results Conference Call. At this time, all participants are in listen-only mode. This call is being webcast live on the Investors & Media section of Fate's website at fatetherapeutics.com. As a reminder, today's call is being recorded. I would now like to introduce Scott Wolchko, President and CEO of Fate Therapeutics.

[Scott Wolchko:]

Thank you. Good afternoon and thanks everyone for joining us for the Fate Therapeutics third quarter 2018 financial results call. Shortly after 4

[Operator:]

[Operator Instructions]. Our first question comes from Robyn Karnauskas.

[Nicole Germino:]

This is Nicole on the line for Robyn. For FT500 as we received FDA warrants for the IND, 1 global study initially and how many patients are you planning to enroll?

[Scott Wolchko:]

Sure. Hi, Nicole. How are you? Happy to turn that call over to Chris Storgard, our CMO, who can speak to the FT500 studies.

[Chris Storgard:]

Thanks. Chris Storgard here. So we are working very closely with our sites and we anticipate to be ready for dosing by the end of the year from a site perspective.

[Nicole Germino:]

And to start on FT500, it sounds like likely it's going to initiate around year-end during 2019, are there other studies that we should expect to be initiated in 2019?

[Scott Wolchko:]

So the other studies that we expect to initiate in 2019 include FT516, which is the second product emerging from our iPSC platform, that is the product that expresses the high affinity non-cleavable CD16 receptor. We expect to file the IND for that product candidate by the end of this year. So certainly expect that those clinical trials will be running in 2019. In 2019, we fully expect to file at least one, if not two INDs, additional INDs, certainly one of them will be with respect to our first iPSC-derived CAR product candidate.

[Nicole Germino:]

And for NK100, can you remind us how many patients are in each of these programs and the range of the duration of patients on treatment?

[Scott Wolchko:]

Sure, I can start that and I'll let Chris follow-up. So we've treated in total approximately 20 patients, we provide -- we plan to give an update at SITC next week at an investor event, where we will discuss progress across all three studies.

[Operator:]

Thank you. Our next question comes from Ted Tenthoff of Piper Jaffray. Your line is now open.

[Ted Tenthoff:]

Wanted to follow-up, in term -- maybe a little bit higher level question. And I'm willing to understand sort of how NK100 will fit into the treatment landscape use of the iPSC result; because it always seems to me like NK100 second gene and we're getting into third gene with iPSC. So, it's just something where you go after different indication, what ultimately do you see as the primary product for Fate?

[Scott Wolchko:]

Sure. And I'm happy to discuss this more at our SITC event. I think it's a little bit early for us to comment on this with specificity and certainty. I will say generally, and I have said this in the past, I'd fundamentally believe in iPSC-derived approach where you're creating master cell lines, resulting in the production of homogeneous products that can be given in multi-doses to patients is completely disruptive of autologous approaches as well as donor-derived approaches. So Fate Therapeutics is absolutely committed to advancing NK100. We are learning a tremendous amount for our NK 100 studies. That said if our iPSC-derived platform is successful, I fully expect that an iPS-derived approach can absolutely replace any autologous and any donor-derived cell therapy and do that with significantly more safety and efficacy.

[Ted Tenthoff:]

And then just going back to that and I apologize if this was answered in earlier, but what remains to be done with this cell line characterization for FT516? Is this pretty standard -- or I'm sorry, for FT500, is this pretty standard stuff? Were there any surprises in its request for increased characterization and so on?

[Scott Wolchko:]

This is -- so, thanks. So FT500, the additional characterization of the master cell line. So yes, we believe this is very standard, we believe this is significantly de-risked. The testing for instance, AVANT tissues agents is a common tests that's done on cells in order to characterize them. In fact, we characterize indeed, AVANT tissues agent testing on the original cell used to make and reprogram to create our master cell bank. So, as you know Ted, we source and for instance, we source neonatal foreskin fibroblasts in order to make our iPS cells and create our master cell bank. We did full testing and characterization of the initial neonatal foreskin fibroblasts that was used to derive the iPS master cell line. And so we did testing most proximal to where materials were used to create our cell line. We were very pleased with all outcomes of our testing. In discussions with the FDA, the FDA recognizes though, we have created a quote unquote master cell line. There is specific guidance around qualifying a quote unquote master cell line. And so, we are testing the master cell line at their request the FDA, I believe, understands, our objective is to never recreate the master cell line and continue to use it throughout the lifecycle of the product. And so, they asked us to test it for AVANT tissues agents and we're complying with that and we feel very comfortable in the outcomes of those tests.

[Ted Tenthoff:]

Very cool update. Very cool progress. Good. Thank you.

[Operator:]

Thank you. Our next question comes from Do Kim of BMO Capital Markets. Your line is now open.

[Unidentified Analyst:]

This is Neil, I'm filling in for Do. I had two questions. The first one regarding ProTmune Phase 2 enrollment. It sounds like you had 14 patients enrolled in the second quarter and now you're saying you're over 30, I was wondering if you could give a little bit more granularity as to the numbers and what do you see enrollment sort of plateauing? And then the second question is just a little bit more of a higher level question about NK100 and the multi-dosing regimen. And sort of what you're thinking about and what you're learning about in terms of how you're assessing success in the multi-dosing regimen, if it's just primarily safety or what kind of efficacy measures are you kind of focusing on in the interim?

[Scott Wolchko:]

Sure. I'll let Chris talk to ProTmune and enrollment with respect to that and Dan can talk a little bit about NK100 and the multi-dosing elements associated with that.

[Chris Storgard:]

So with respect to enrollment with ProTmune, we are definitely not seeing a plateauing enrollment, but we do see continued excitement by our investigators and everyone is very excited to see these patients. As Scott mentioned, the data from the Phase 1 will be reached at ASH, but it's very encouraging to date. So with that enrollment is progressing and going along very nicely, and we are on-track with our internal targets and it's not been a low in that enrollment.

[Daniel Shoemaker:]

Hi, Neil, this is Dan. So on your multi-dosing question for NK100. So we've been very interested in the biology behind how the patient will respond to multiple doses and a couple of the observations that we've actually done multi-dosing in two of our studies, the APOLLO and DIMENSION. And one of the observations is the persistence on the first dose and second dose, the persistence on the second dose has actually been better than the first dose. So one idea would be that the immune system would be keyed into the product now and that would immediately eliminate the second dose, so that has not happened, and so that was one of the learnings. The second thing is even as we assess blood samples from the patients, not only during the first dose and second dose, but we've recover the NK cells and do phenotypic testing, as well as functional assessments. And interestingly, on the second dose, we're very encouraged to see the phenotype of the NK100 persisting, so that the CD57 phenotype as well as some of the enhanced CD16 activity as well as persistence market. So we're seeing very favorable activity on both the first and second dose and we're pretty encouraged as this relates to multi-dosing in allogeneic settings.

[Scott Wolchko:]

We will -- this is Scott, I mean, we will spend more time talking about this at SITC in terms of the potential and the excitement around being able to safely administer multiple doses of NK cells and obviously do that with the intent of driving efficacy.

[Operator:]

Thank you. Our next question comes from Jim Birchenough of Wells Fargo. Your line is now open.

[Nick Abbott:]

It's Nick in for Jim this afternoon. First question is on ProTmune. Can you just remind us of the latest regulatory discussions regarding the primary endpoint and its suitability for registration? And am I right in assuming that you might get to say, 12-month follow-up endpoint by the middle of 2020?

[Scott Wolchko:]

Yes. So we originally received Fast Track designation with respect to the development of ProTmune around the Day 100 endpoint, which was incidence and severity of Grades 2-4 acute GvHD. That continues to remain our primary endpoint. As we've discussed though, the ultimate objective of hematopoietic cell transplantation is to drive curative outcomes. And so, the 1-year end point is fundamentally important as we think about developing a next-generation better graft for patients. And looking at for instance, the two key endpoints that we discussed disease-free survival as well as a live disease-free and free of severe GvHD. Those are two endpoints that we think are very important as we look at the durability of the efficacy effect associated with ProTmune. And your question with respect to the 1-year endpoint being looked at with respect to 2020. Yes, I mean absolutely, I think we're on track and feel very comfortable with the enrollment clips that we're seeing and would expect the one-year endpoints to be looked at in 2020.

[Nick Abbott:]

But to be clear, then you could file on the Day 100 endpoint?

[Scott Wolchko:]

So we are going to have discussions with the FDA on how we appropriately unblind the study, given it is a blinded study with respect to the Day 100 endpoint versus the 1-year secondary endpoints.

[Nick Abbott:]

And then just coming onto potential differences between FT819 and the iPSC products, CAR T products being development [indiscernible]. From what I can understand from the abstract FT819 starts with a CAR T that's engineered with the non-cleavable CD16 into differentiated to an iPSC and then it differentiated into a T-cell. Your comments on the ONO, it sounded like you start with an iPSC and then -- and so a CAR. Do I have that correct?

[Scott Wolchko:]

No. We're engineering iPS cells. We engineered an IPS cell level.

[Nick Abbott:]

So for 819 as well?

[Scott Wolchko:]

Yes.

[Nick Abbott:]

And then as you -- I think [indiscernible] very clear that you could get a CD8 T cell, you haven't quite figured out how to get a CD4 T cell yet. So where are you in terms of deriving CD4 T cell?

[Scott Wolchko:]

So, we will give an update on this at our investor event at ASH. We are absolutely differentiating and seeing 4s and 8s.

[Nick Abbott:]

And then maybe just last one from me, you have iNK CARs and IT CARs. So, with these two different products, do you see them being used in combination or do you see them being developed in different indications?

[Scott Wolchko:]

I wish I knew the answer to that. I don't know the answer to that question yet. I'm not -- it will be interesting to see quite frankly, when you start introducing multiple pieces of functionality into NK cells and T cells, how those cells behave differently if at all. And I think if I think of the world of allogeneic transplant, which is the only curative therapy today, as you know, both T cells and NK cells are given.

[Operator:]

Ladies and gentlemen, this concludes today's question-and-answer session. I would like to turn the call back over to Scott Wolchko for any closing remarks.

[Scott Wolchko:]

Thank you. Thank you all for participating in today's call. We look forward to speaking with you all very soon in the next several weeks, both at SITC and as well as ASH. Take care.

[Operator:]

Ladies and gentlemen, thank you for participating in today's conference. This concludes today's program and you may all disconnect. Have a great day.