Sarepta Therapeutics Inc To Discuss Global Licensing and Collaboration Agreement with Arrowhead Pharmaceuticals Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Andrew Tsai - Jefferies LLC - Analyst : Congrats on the deal. Maybe the question we have as we think about time to approval and launch is that which of these programs have the potential for you to pursue an expedited accelerated approval pathway? Said another way, which of these biomarkers have been validated? Thanks.

Question: Gil Blum - Needham & Company, LLC - Analyst : And also, allow me to add my congratulations on an interesting deal. So maybe a more strategic question here. So does the investment in RNA technologies here suggest that you guys have a lower interest in gene therapy overall? Or maybe this is a repeat of your prior strategy that you did with DMD, where you started out with chronic and then moved to gene therapy? Thanks.

Question: Ritu Baral - TD Cowen - Analyst : Louise, could you review for us what data that you have in hand on the muscle penetrant and muscle-directed qualities of the TRiM platform? And if you could cover CNS as well, just because you made a very interesting comment about the CNS manifestations of DM1, which at least in my reading, are less prevalent in the literature. So if you could review those two things. Thank you.

Question: Tazeen Ahmad - BofA Securities, Inc. - Analyst : I wanted to maybe ask about FSHD and the comment that Doug made about being excited about next-gen technology. So FSH is one of those diseases where a few companies have been spending time trying to develop therapies. Can you just give us an idea of what about the technology for Arrowhead impresses you and makes -- in your mind, it differentiated from others? And in particular, a biomarker called DUX4 is something that companies pay close attention to. It's been kind of a noisy biomarker to date, but I was wondering if, Louise, you could give us your view on the reliability of using DUX4 given that it's still early in development. Thanks.

Question: Eliana Merle - UBS Securities LLC - Analyst : Congratulations on the deal. Just a follow-up on DUX4. You mentioned you're seeing some exciting data. Maybe just both with DUX4 and DM1, can you elaborate specifically on what you've seen so far? Are you referring to the preclinical work or also the clinical data? And then just a second question on the Huntington's program. Is this program targeting exon 1? And I guess, how do you see this asset as differentiated versus other assets in the Huntington's space such as Alnylam's Huntington's program? Thanks.

Question: Kostas Biliouris - BMO Capital Markets Corp. - Analyst : Congrats on the partnership here. A two-part question from us. It seems that the MMP7 is the first program of your pipeline outside of the neuromuscular space. So can you talk a little bit about your decision to choose this as the first program? And how do you prioritize the Arrowhead partner programs over your limb-girdle programs in the pipeline? Thank you.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Just for the four clinical programs that are under the collaboration, just curious, should we be expecting updates for all those programs next year? Or it will only be certain ones? Thanks.

Question: Danielle Brill - Raymond James & Associates, Inc. - Analyst : Congrats on the interesting deal here. So I feel like the pushback is going to be why not get a couple of strong quarters of the Elevidys launch under your belt before doing this deal? So Doug, maybe you can help us understand why now was the right time? Was this to get out in front of the IPF data? Or yes, however you can elaborate would be really helpful. Thank you.

Question: Uy Ear - Mizuho Securities USA LLC - Analyst : Congratulations on the deal. So maybe just a question on the potential changing dynamics at the FDA in general. Like I guess what gives you comfort in doing the deal now versus either waiting to see what the new administration would do? Thanks.

Question: Mitchell Kapoor - H.C. Wainwright & Co., LLC - Analyst : I wanted to ask about what possibilities for additional business development diversification you might have. I think you indicated this might not be the last deal, although this is the big one you were hoping for. And then second, if you could just comment on your expectations for R&D to change in the coming quarters and years. Thank you.

Question: Gavin Clark-Gartner - Evercore Group L.L.C. - Analyst : Congrats on the deal. So big picture, I was just wondering what makes you comfortable on the long-term safety, specifically for the alphaV beta 6 muscle focused platform? And more specifically also, what duration of in vivo chronic tox coverage do you have at this point? Thank you.

Question: Biren Amin - Piper Sandler & Co. - Analyst : Congrats on the deal. For the MMP7 program, what led you to focus on this target given Arrowhead has two other pulmonary programs also in Phase I? And I guess a second part of the question for MMP7 is what read-throughs gave you confidence on the safety profile from animal studies given there was an earlier pulmonary program from the TRiM platform targeting ENaC that had paused due to animal tox back in 2021? Thank you.

Question: Kristen Kluska - Cantor Fitzgerald & Co. - Analyst : Congrats on the deal. I know you're a very patient-centric company. So I was hoping you can comment on, for DM1 and FSHD in particular, what aspects of the disease that you think ultimately are going to matter most for these patients, especially considering there are no treatment options available for them today. Thank you.

Question: Joe Schwartz - Leerink Partners LLC - Analyst : Congrats on the deal. I was wondering if you could talk about the extent of preclinical or clinical data that you were able to evaluate in order to get comfortable that the Arrowhead programs, which are behind others in development for conditions like DM1, FSHD, et cetera, can have competitive efficacy and safety profiles. For instance, were there particular splicing or knockdown models that showed that at certain concentrations that you think could be achieved safely that drove things that were encouraging preclinically that you might be able to achieve in the clinic? Can you just give us a sense of the diligence and what you're able to see that built your confidence? Thank you.

Question: Gena Wang - Barclays Capital Inc. - Analyst : I know you've done a lot of work on TRiM platform. Just wondering how much have you done with other platform? There are quite a few RNA therapies or technology out there. How thorough have you done the due diligence process before selecting Arrowhead as a partner? And then second question, have you seen any initial clinical data from these programs?