Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) Q2 2023 Earnings Call Transcript

Brian Goff: Yes, it has been a great quarter and I’m happy to provide some comments around the deal that we announced this morning. Polycythemia Vera is a large market. We believe it is ripe for disruption and growth. And as I noted in my comments, you know, if you just think about the standard of care, it is phlebotomy and to us, that is just a great opportunity to do what we do best, which is reinvent the way that these rare diseases are treated and bring potential disease modifying therapies to these patients. So we are really excited about this particular ten per six assets from Alnylam. It fits really well with our discipline BD criteria, builds on our core capabilities and that is across our scientific expertise, our development capabilities, our commercial capabilities.

And if you think about the BD criteria that we have talked about so many times, it checks all the boxes. So this is a rare disease. It is transformational potential for patients. We see an opportunity for early de-risking, which we have talked about a lot as a sweet spot for us. We believe there is a clear regulatory pathway. And ultimately this will be value creating is our strong belief because it comes from the Alnylam, you know, world-class RNAi platform. We also have strong conviction in the probability of success from the data generated to date. So we are excited about this. I guess to your question of where we go from here for clinical development, I think, and I will ask Sarah if she wants to make a comment. But it is a little too early for us to define the target product profile specifically.

We have many options. We will be looking at efficacy dimensions, speed of action, for the product itself, for patients, safety profile, and of course convenience. And all of those are in play. But given the stage of development, we are just going to pause and bring the asset in, really look at it deeply for what, where we can put our development expertise in motion and then we will provide updates accordingly. And then the last thing I will just say to your point about is this a theme of the types of BD deals and so forth going forward. We retain optionality for a range of different BD deals. This one in particular really, as I mentioned, checked all the boxes and so we were eager to bring it in, but we will retain our disciplined BD criteria going forward.

Sarah, anything you wanted to add?

Sarah Gheuens: Only to say that, you know, we are very excited about this. Of course, once we move forward with the program and have more details on our clinical development program. We will always be shooting to deliver value for patients and make a meaningful change with the development program, and you can expect execution on the program as well.

Brian Goff: And we are excited to do that.

Operator: Our next question will come to the line of Gregory Renza from RBC Capital Markets.

Gregory Renza: Maybe just a couple quick ones for me, just following up on the in license on my own asset, maybe it would be helpful just to hear your thoughts and Sarah just, just on the preclinical data today certainly, others exploring manipulation of TMPRSS6. And just curious, how you think this asset can potentially differentiate or sets up well when you survey the available data to-date. And then secondly, great to see the dose selection for the Phase 3 and sickle. Just curious, just the rationale as you looked at the body of data over the last several weeks since the top line and made the step for 100 mg, thanks so much and congrats again.

Brian Goff: Thanks a lot Greg. And I’m going to send this over to Sarah then for both questions.

Sarah Gheuens: Yes, thank you. So around the preclinical data, it has demonstrated in Vevo proof-of-concept in non-human primates with a safety profile that was very favorable and very good knockdown of the target observed. So we feel very confident about that. And as we mentioned earlier, we are very eager to get going on the IND enabling studies and the CDP, and provide more detail on that as we progress with that. I think for us, yes, there are others that are also looking at this target. To Brian’s point, this is a very big market. There is going to be differentiation around efficacy, safety, mode of administration, and also frequency of administration. So we believe that our CDP will be able to deliver on what the market needs.