Ascendis Pharma A/S (ASND) Earnings Call Transcript & Summary

Good day, ladies and gentlemen. Thank you for standing by. Welcome to Ascendis Pharma Regulatory Update Conference Call. [Operator Instructions] Please note that today's conference may be recorded. I will now hand the conference over to your speaker host, Tim Lee, Senior Director of Investor Relations. Please go ahead.

Thank you, operator, and thank you, everyone, for joining our regulatory update call this morning. I'm Tim Lee, Senior Director, Investor Relations. Joining me on the call today is Jan Mikkelsen, President and Chief Executive Officer. Before we begin, I'd like to remind you that this call will contain forward-looking statements that are intended to be covered under the safe harbor provided by the Private Securities Litigation Reform Act. Examples of such statements may include, but are not limited to statements regarding our plans to address and resolve concerns raised by the FDA and bring TransCon PTH to the market in the U.S. and statements regarding the potential approval of TransCon PTH in the EU. These statements are based on information that is available to us today. Actual results and events could differ materially from those in the forward-looking statements, and we may not be able to achieve our goals, carry out our plans or intentions or expectations or projections disclosed in our forward-looking statements, and you should not place undue reliance on these statements. Our forward-looking statements do not reflect the potential impact of any licensing agreements, acquisitions, mergers, dispositions, joint ventures or investments that we may enter into or terminate. We assume no obligation to update these statements for circumstances except as required by law. For additional information concerning the factors that can cause actual results to differ materially, please see our forward-looking statements section in today's press release and the Risk Factors sections of our most recent annual report on Form 20-F filed February 16, 2023. On today's call, Jan will make some prepared remarks, and then we'll open up the call for questions. With that, let me turn it over to Jan.

Thank you, Tim. Thanks to everyone for joining us. late Friday, we received a complete response letter from the FDA for our NDA for TransCon PTH in hypoparathyroidism. The CIL stated that FDA cannot approve the NDA in its current form based on deficiencies related to the manufacturing control strategy for variability of delivered dose in our drug device combination product. The FDA did not expect concerns about the clinical data submitted as part of the NDA package and no new pre clinic studies or Phase III clinical trials to evaluate safety or efficacy were requested in the letter. Today's news does not change the safety and efficacy profile observed to date in our clinical trial and our expanded access programs. We believe we are well prepared to address the FDA concerns related to control strategy. And we continue to have a high level of confidence that the concern raised by FDA can be resolved. We will request a type A meeting with the FDA to agree on the best pathway forward. We are committed to work with the FDA to resolve the issues in the CIL and submit the response as needed as soon as possible to bring TransCon PTH to the market in the U.S. In the EU, we remain on track, as laid out in our press release. With that, let us open the call for questions.

[Operator Instructions] Now first question coming from the line Jessica Fye with JPMorgan.

I have two, first, was manufacturing controls the only deficiency cited? And second, I think you mentioned no new preclinical nor Phase III studies were requested. Were any Phase I studies needed to back up the control strategy?

Let me answer your first question. Yes, there was only one deficiency cited in the letter. The second question related to the element how we are resolving the deficiency, we are working with FDA to find out exactly how we can resolve that. I'm convinced we can do it. And we will prefer to our interaction with FDA to exactly find out how we will solve it.

[indiscernible] your line is open.

I think that might be me. Thanks for the update. Just to clarify, are any type of clinical studies going to be requested as part of the path to get the drug approved? And secondly, Jan , do you have a sense of how long this takes to resolve? Is this something you think can be done in less than a year I know you have limited information that I think people are just trying to assess how long of a delay this could be.

The key question for me is that we now have a clarity related to with efficiency. That is the key question. The other key question for me is this deficiency is not really addressing the safety efficacy of anything what we have observed in our clinical trials don't demand new preclinical activities. What we're doing now is finding together with FDA, the pathway forward to -- exactly to resolve this issue. My belief is that we can resolve it, as I said before, in a fast manner because I believe this is element where we don't need to conduct additional clinical trials. So I feel we are in a position that we can address this efficiency in the fastest possible time. I think this clarity exactly about the timing, we will have more knowledge about having the confirmation of our pathway forward when we have our Type A meeting.

Our next question is coming from the line, David Lebowitz with Citi.

Understanding it's fairly early in the process. What is your expectation for the types of requests that you might have to like handle to be able to resolve the CRL. Is it an expectation of shifting process, additional testing? What -- do you have any inkling at this stage of what it will take to alleviate their concerns? Or does that really need to wait for the Type A meeting?

Yes. In the Type A meeting, we will discuss with FDA how we really address the issue in the Type A meeting, we are preparing with our accommodation related to how we believe we can solve this issue. And when we have got this alignment and both FDA and Ascendis feeling comfort, that is the way forward, I believe it's the time to come out and exactly disclose when we can have it.

Is there a typical timeline for how long a Type A meeting takes place?

Yes, that's pretty well regulated in less than three months from today or Friday, we will request a Type A meeting. And the Type A meeting will be granted in less than 30 days. So you can say that the clarification will happen pretty fast on how to resolve it. And after the Type A meeting, if there is an agreement on how to progress, there will be two way to move forward as one, if it's related to and you can read it yourself, there is a different element related to a type 1 on type 2 way to move forward.

Our next question coming from the line of Li Watsek with Cantor.

Jan, you mentioned before that TransCon PTH should built on established products. So in terms of the manufacturing control strategy, maybe help us understand how your process might differ from others in the market. And at this point, do you anticipate any potential modification to the device itself any possibility for additional human factors validation.

Yes, you're 100% right that when we look on the supply chain, it's built on what we call proven technology, proven side. Our [ Drug Skytrofa ] manufacturing is being done by Bachem that have a DMF for PTH 134 in the U.S. Our filling is happening by FECa, which are tubercle cartilage that fit into an insulin pen device instrument. And we basically have developed the instrument pen related to the specification that you typically use in the diabetes development. So from that perspective, I believe we are coming from what we call a supply chain that is proven. And I have not seen any kind of indication that we will change that supply chains.

And our next question coming from the line of Kyuwon Choi with Goldman Sachs.

Two from us, please. First, can you maybe comment on how your CDMOs may or may not already have some of the data you may require to satisfy questions or requests that may come up during the Type A meeting. And second, are you assuming that this is a Class II resubmission? And given your prior filing under a priority review, can you comment on whether you can expect a quicker review on the resubmission? Or would you assume a 6-month review cycle.

Yes. I think the clarification related to type 1 and type 2 with some may be determined on the -- when we have the Type A meeting. And so it's really a little bit hard for me to speculate about that. I think, as I said before, we are convinced to get this Type A meeting done as far as possible, we need to write it together our positioning, and then we will send it in as soon as possible. And as said before, FDA is what grant us the Type A meeting in the framework of at least 30 days after we have done our submission. So I think there is such a clear pathway forward on this year, and I'm really convinced that we can find a resolution to this issue here. So we basically can get TransCon PTH out to the patient here in the U.S.

And our next question coming from the line of Joshua Schimmer with Evercore.

I have a few of them. Maybe starting with the device itself. It's been used as you've noted in other settings such as diabetes. So why do you think this issue is coming up with TransCon PTH? Does it have to do with the unique viscosity? Or are there differentiating elements of this product?

I don't think it's reflecting anything on viscosity because it's a pretty low viscosity product we have developed here in this rate. And you're right, this is both a device that you will see in diabetes product. You will also see it in other rare diseases like [ batermis ] product is also built on [Indiscernible]. So it's a general used device system you have when I look also on the pathway forward the pathway EU, the pathway EU is really straightforward. And I believe this potential build on the development that we have done with our developed device the drug product and the combination out from the current guidelines that some way are fulfilling the requirement for the diabetes product and other product. That can always be requested from regulatory agent that is very, very specific to one single product. And I'm quite sure we can also be quite sure we can satisfied the specification in such a manner that we really can also bring this product out to the patients here in the U.S.

And what kind of variability has been seen with TransCon PTH dose delivered? Is it more to do with that variability? Or is it just more to do in tightening the release specs around what you have shown.

I think just as we stated before, we have not seen any safety or efficacy concerns in our ongoing clinical trial and be enrolling new patients every day in our ERP program. So we are in a position that we are convinced about the safety and efficacy that we have observed in our both clinical trial open label extension and our ERP program. We are taking this point here is to ensure that there is a controlled strategy that basically can take care of what we call extreme something that potentially can happen in the future in a [ slip ] cases. And I think that is where we're working with the FDA to ensure that we're getting sufficient control that we basically can ensure that we also can do this element in the future.

And our next question coming from the line of Leland Gershell with Oppenheimer.