Ascendis Pharma A/S (NASDAQ:ASND) Q2 2023 Earnings Call Transcript

Ascendis Pharma A/S (NASDAQ:ASND) Q2 2023 Earnings Call Transcript September 5, 2023

Ascendis Pharma A/S beats earnings expectations. Reported EPS is $2.16, expectations were $-2.68.

Operator: Hello, and welcome to Ascendis Pharma Q2 2023 Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. [Operator Instructions] I would now like to hand the conference over to Tim Lee, Senior Director, Investor Relations, Ascendis Pharma A/S. Sir, you may begin.

Tim Lee: Thank you, operator, and thank you, everyone, for joining our second quarter 2023 financial results conference call. I’m Tim Lee, Senior Director, Investor Relations of Ascendis Pharma. Joining me on the call today is Jan Mikkelsen, President and Chief Executive Officer; Scott Smith, Executive Vice President and Chief Financial Officer; and Dr. Stina Singel, Executive Vice President and Head of Clinical Development Oncology. Before we begin, I’d like to remind you that this conference 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, our U.S. commercialization and continued development of SKYTROFA for the U.S. market, our revenue projections for SKYTROFA, the commercialization of TransCon hGH for the EU market and our planned of SKYTROFA in Germany, statements regarding our NDA for TransCon PTH and expected timing of the potential approval and launch of TransCon PTH in the U.S. market, statements regarding the expected timing of the potential approval and launch of TransCon PTH in Europe, statements regarding the potential approval of TransCon CNP, our expectations regarding our new TransCon technology, and our progress on our pipeline candidates and our expectations with respect to their continued progress, statements regarding our strategic plans, our goals regarding our clinical pipeline, including the timing of clinical results, statements regarding our pipeline product candidates, statements regarding our ongoing and planned regulatory filings, and our expectations regarding the timing and the results of regulatory decisions, our expansion into new therapeutic areas and statements regarding our progress towards Vision 3×3 and our ability to create a sustainable, profitable and leading global pharma company.

These statements are based on information that is available to us today. Actual results and events could differ materially from those in our forward-looking statements, and we may not be able to achieve our goals, carry out our plans, our intentions, our 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 as circumstances change except as required by law. For additional information concerning the factors that could cause actual results to differ materially, please see our forward-looking statements section in today’s press release and the risk factor sections of our most annual report on Form 20-F, filed February 16, 2023.

TransCon Human Growth Hormone, or TransCon hGH, is approved by the FDA in the U.S. under the brand name SKYTROFA for the treatment of pediatric patients one year and older weighing at least 11.5 kilograms and having growth failure due to inadequate secretion of endogenous growth hormones. In addition, the European Commission has granted a marketing authorization for SKYTROFA to Ascendis Pharma developed under the name TransCon hGH as a once weekly subcutaneous injection for the treatment of children and adolescents aged 3 to 18 for growth failure due to insufficient secretion of endogenous growth hormone. In general, we refer to this product as TransCon growth hormone, unless we’re referring to the product in the context of particular jurisdictions such as the United States or the European Union.

Otherwise, please note that our product candidates are investigational and are not approved for commercial use. As investigational products, the safety and effectiveness of the product candidates have not been reviewed or approved by any regulatory agency. None of the statements made on the conference call regarding our product candidates shall be viewed as promotional. On the call today, we’ll discuss our second quarter 2023 financial results and we’ll provide further business updates. Following some prepared remarks, we will then open up the call for questions. I’ll now turn the call over to Jan Mikkelsen, President and Chief Executive Officer. Jan?

Jan Mikkelsen: Thanks, Tim. Good afternoon, everyone. In 2019, at the JP Morgan Conference, we announced our Vision 3×3 for building a leading sustainable global biopharma company by 2025. Today, I’m pleased to share with you update how close we are to achieving our vision. As outlined in our Vision 3×3, using our TransCon technology platform and our algorithm for product innovation, we are on the pathway to achieve the regulatory approval of three independent endocrinology rare disease product, TransCon Growth Hormone, TransCon PTH and TransCon CNP, by 2025. In addition, we are building global commercialization capabilities to bring this growing portfolio of highly differentiated product to patients. In the U.S., for the second quarter, we reported SKYTROFA revenue of €36 million.

SKYTROFA achieved growth hormone market value leadership in the U.S. in the second quarter with a penetration of less than 10% of treated U.S. pediatric growth hormone deficiency patients, which represent about half of the growth hormone market in the U.S. today. For the full year of 2023, we now expect SKYTROFA U.S. revenues should be €165 million to €170 million. We believe three factors continue to drive demand. First, a growing number of physicians have patient with over a year of experience on SKYTROFA, and these physicians have observed the long-term benefit for both treatment naive and switch patients. Second, the consolidation of the daily growth hormone market continues and the recent approvals of two other long acting growth hormone products, which could accelerate this shift to long acting.

Third, Ascendis has become a trusted partner within the endocrinology community, as we continue to invest in our products, in science and in our support for patient and healthcare and provide them with a reliable supply chain. In our global commercial reach, we are ready to launch SKYTROFA this month in Germany. Our medical affairs and commercial teams are in place and have been active engaging endocrinologists across the country. We recently received FDA approval at Lonza for a high-capacity drug substance manufacturing site, for which we expect EU approval in the first half next year. This added drug substance manufacturing capacity supports our goal to commercialize in new markets and additional indications, and to achieve our goal of global market leadership and value in a growing global growth hormone market.

During the fourth quarter, we expect to see a topline result for our global Phase 3 foresiGHt trial, our TransCon growth hormone in adult growth hormone deficiency. We believe adult growth hormone deficiency is an underpenetrated indication. The recent study showed that less than 4% of adult patient suspected of having growth hormone deficiency are treated with growth hormone. Moving to TransCon PTH for adult hypothyroidism. In June, we requested a Type A meeting with FDA and submitted an updated control strategy. The Type A meeting was held with FDA in late August based on the agency’s availability. Following a constructing Type A meeting, we submitted additional information to FDA supporting the updated controlled strategy. I believe the materials submitted to FDA combined with that Type A meeting discussion will position us to resubmit the NDA for TransCon PTH for adults with hypoparathyroidism in October 2023.

If our NDA is accepted, which we expect within 30 days following resubmission, FDA will notify us whether the resubmission is Class 1 or Class 2 and provide a new PDUFA date which we estimate could be in December this year or April 2024. It’s important to note that we will not know the new PDUFA date until our resubmission is accepted and this classification is communicated to us. Besides this information, we will not comment further on the resubmission procedure while we are having ongoing communication with FDA. In the European Union, we received our Day 180 assessment report with feedback on our MAA for TransCon PTH and have submitted our response to the list of outstanding issues. We remain on track for a European Commission decision during the fourth quarter.

And if approved, we plan to launch TransCon PTH in Germany in early 2024. Finally, today, we released new positive data supporting the potential beneficial effect of TransCon PTH on kidney. Over the course of one year, patient in our Phase 3 trial demonstrated profound increases in eGFR, a key mark of kidney function, with increases of around plus 9 milliliter permitted across all patients. Importantly, in the subset of patient with eGFR less than 60 at baseline, the threshold for kidney dysfunction, TransCon PTH demonstrated increases of 11 to 12 milliliter per minute. Around half of the pathway for patient with a eGFR of less than 60 after [TransCon P] (ph) treatment for one year experienced a eGFR improving to about 60, meaning they went from having a diagnosis of [indiscernible] impairment to be within the normal range of kidney function.

For patient treated with TransCon PTH, same improvement in eGFR of this magnitude may reduce risk of progressing to chronic or late-stage kidney diseases. This is a major comorbidity in patient with hypoparathyroidism and a major contributor to medical costs. We plan to present detailed results at our upcoming medical conferences. The U.S. Expanded Access Program and German Compassionate Use Program for TransCon PTH continues to be open for enrollment of patients, and we expect to initiate comparable programs in additional countries. In the ongoing extension portion of our clinical trial, 145 out of the initial 154 patient from the original clinical trials continued treatment with TransCon PTH for over three years. We continue to prepare for the expected launch in U.S. and Europe.

We are confident that TransCon PTH, if approved, become an important new treatment option for adult patients living with this serious diseases. Switching now to TransCon CNP, following our end of Phase 2 meetings with U.S. and EU regulatory agencies, we have an agreed pathway to achieve regulatory approvals for TransCon CNP. First, FDA and EU regulatory agencies confirmed that absolute analyze growth velocity is acceptable as the primarily [indiscernible] pivotal Phase 3 trial ApproaCH trial. Second, these regulatory agencies agreed to our dose selection of 100 microgram per kilo per week dosed for ApproaCH. Third, based on this discussion, we expect that an indication for treatment of achondroplasia will be supported by emulating the beneficial impact on TransCon CNP on comorbidities and other important aspect of achondroplasia in addition to height.

Fourth, our pivotal Phase 3 trial ApproaCH is now fully enrolled with topline data expected in the second half of 2024. We believe that achondroplasia is a disease about skeletal growth and muscle disorder. Based on the rapid functional improvement observed in our ongoing Phase 2 ApproaCH trial and based on [indiscernible] review, there may be a primary muscular component to achondroplasia phenotype, besides the well described effect on skeletal growth. We believe it’s essential to have a continuous exposure to CNP to ultimately improve modest strength and endurance. In our pivotal trial, we will explore endpoint to measure how CNP might modulate the muscular weakness. In addition, this also means that there could be a treatment option for adults living with achondroplasia.

Our research in this area continues, and we expect to share more later this year, along with new data from ACcomplisH where all the initial 57 patient continue on treatment with TransCon CNP for over three years. Turning to oncology. We today announced that we had completed dose escalation for TransCon IL-2 beta/gamma in combination with pembro and a clear Recommended Phase 2 Dose at 120 microgram per kilo every 3 weeks. No dose-limiting toxicity, vascular leak syndrome, or grade 3 or 4 cytokine release syndrome were observed at any dose level evaluated. Finally, I’m excited to share with you some new development expected to drive Ascendis sustained growth. We had developed a new TransCon carrier platform, which integrate our reversible linkers and complements our two established care technologies, the soluble and hydrogel platforms.

Among the many applications, we believe this technology support high volume, low cost manufacturing, enabling product for new therapeutic areas. We have established proof of principle for once monthly dosing of the [GPL-1] (ph) analog, semaglutide, and on our website, you can see our preclinical data. In summary, Ascendis remains focused on building and maintaining a sustainable, profitable, leading biopharma company. With all programs making significant progress, we are nearing completion of our Vision 3×3 and have already begun the foundation for the next date of Ascendis. I will now turn the call over to Scott for our financial review before we open up for questions.

Scott Smith: Thank you, Jan. As Jan noted, we are making very strong progress at Ascendis. I will touch on key points surrounding our financial results. For further details, please refer to our Form 8-K filed today. Total revenue for the second quarter was EUR 47.4 million, including SKYTROFA revenue as well as licensing services provided to third parties, primarily VISEN Pharmaceuticals. SKYTROFA revenue for the second quarter of 2023 was EUR 35.9 million, compared to EUR 31.6 million reported in the first quarter. Second quarter revenue was negatively impacted by two items: a negative adjustment to provision for estimated sales rebates of EUR 2.1 million, which resulted from stronger-than-expected payer adoption related to prior periods; and a negative foreign currency impact of EUR 0.6 million as compared to the first quarter of 2023 due to a weaker U.S. dollar.

Based on reported results from other growth hormone manufacturers, SKYTROFA was the U.S. market leader in the second quarter of 2023 with less than 10% penetration into the U.S. pediatric GHD patient population. We see a large opportunity in front of us to grow our patient share in pediatric GHD, which we estimate to be only half of the addressable U.S. growth hormone market. Turning to expenses, R&D costs declined 1% sequentially, primarily driven by lower endocrinology related costs, partially offset by an increase in oncology related costs. SG&A expenses grew 6% sequentially, reflecting higher external commercial expenses for SKYTROFA in the U.S, pre-launch activities for SKYTROFA outside the U.S. global pre-launch activities for TransCon PTH, and higher employee-related expenses.

Total operating expenses were EUR 175 million for the second quarter, up 2% sequentially from the first quarter of 2023. Overall, our operating loss declined sequentially by 2% to EUR 141 million for the second quarter from EUR 144 million in the first quarter of 2023. We ended the second quarter with cash, cash equivalents and marketable securities, totaling EUR 431 million. Finally, we have entered into a capped synthetic royalty funding agreement with Royalty Pharma for $150 million in exchange for a 9.15% royalty on net sales of SKYTROFA within the United States, with no royalty payments until 2025. The royalty payments are capped at 1.65 times the purchase price and fully paid prior to December 31, 2023, or 1.925 times the purchase price if not fully paid by December 31, 2023.

Further details are disclosed in a separate 6-K filed today. Looking forward, we expect continued momentum for SKYTROFA in the United States for the balance of this year and we are raising our expectations for SKYTROFA revenues to EUR 165 million to EUR 170 million for full year 2023. Let me now also provide a review of selected key program milestones. For TransCon Growth Hormone, we are on track to launch SKYTROFA in Germany this month, and we expect to report topline data from the global Phase 3 foresiGHt trial in adult GHD, our first potential label expansion in Q4. For TransCon PTH, we believe we will be in a position to resubmit the NDA for adult with hypoparathyroidism in October, and we expect a European Commission decision in Q4. If approved, we plan TransCon PTH as our second product launch in Germany in early 2024.

For TransCon CNP, we plan to share follow-up data from the open label extension of our Phase 2 ACcomplisH trial in Q4 this year, and we expect to report topline results from ApproaCH, our pivotal Phase 3 trial, in the second half of 2024. Within the oncology therapeutic area, during this quarter, we expect to dose the first patient with a combination of TransCon TLR7/8 agonist and TransCon IL-2 beta/gamma. And during Q4, we will be presenting dose escalation data supporting Recommended Phase 2 Dose at ESMO on TransCon IL-2 beta/gamma in combo with pembrolizumab and updated monotherapy dose escalation data. As Jan mentioned, we will not comment further on the NDA resubmission procedure for TransCon PTH while we are having ongoing communications with FDA.

With that, operator, we are now ready to take questions.

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Q&A Session

Operator: Thank you. [Operator Instructions] Our first question comes from the line of Li Watsek with Cantor. Your line is open.

Li Watsek: Hey, great. So, thank you for taking my question. So, I understand that there is a limited color that you can provide regarding the NDA resubmission, but just wondering if you can sort of elaborate on sort of the additional information that’s needed from the Type A meeting and what are the gating steps for you to reapply NDA?

Scott Smith: Yeah, Li, this is Scott. We cannot comment further on the resubmission procedure while we’re having ongoing communications with FDA. But we believe we’ll be in a position to resubmit in October as we stated.

Li Watsek: Okay, understood. So, my second question is maybe just comment on the timing behind doing a Royalty Pharma deal right now?

Scott Smith: Yeah, this is Scott again. I think that we looked at a transaction that had very attractive terms and allowed us to lower the cost to capital.

Li Watsek: Okay, thank you.

Operator: Thank you. Please stand by for our next question. Our next question comes from the line of Paul Choi with Goldman Sachs. Your line is open.

Paul Choi: Hi, good afternoon, and thank you for taking the question. Just on the commercial piece, can you maybe comment on where additional share opportunities remain for SKYTROFA? One thing we noticed was that there was a sequential decline in the gross margin. So, can you maybe just comment on whether some of these share gains and opportunities are coming at the expense of potentially higher rebating and/or discounting? And then, I have a follow-up.

Jan Mikkelsen: Let us just go back and give what was really the fundamentals for us in our commercial strategy in the U.S. market. And this is exactly the same strategy, we basically moving into each single market. We want to be the leading product in value and we want to do it in a growing growth hormone market. So, we basically building on the product strength of SKATROPA. And we have seen that is really happening in the U.S. We have seen where we basically come in and reset the market and this is basically what we’re doing now. What we see? We see a continued stable growth on patient coming both from switch patient, patient coming as naive patient, and we see it in a continued manual coming week by week, months by months.

And we also feel that we are now in a position, as we said in the script, the physician, the patient, everyone have seen really the full potential of the SKATROPA, because they have seen how we really are in position to give a better outcome related to analyze [indiscernible] that you were typical to see even in an highly compliant daily growth hormone setting. And that is basically why we see this. And it will only be enforced when we basically can get additional indication. This is why we really are thrilled for year — end of the year, Q4 come out with data from our adult growth hormone deficiency, which we really can show also that we really can provide an improved treatment option to this patient too.

Scott Smith: And, Paul, on the gross margin question, the SKYTROFA — remember, the gross margin is for all revenue, which includes some pass through revenue, as we mentioned. So I would say the SKYTROFA gross margins are largely unchanged, pretty stable over the last several quarters.

Paul Choi: Okay. Got it. Thanks for the additional color, Scott. And then, as my follow-up question, on TransCon CNP for the Phase 3 trial, can you comment on whether the regulators have asked for any additional clinical endpoints beside as a focal point for potential approval positions in addition to AGV or absolute high growth, and just sort of any other data you may be collecting to differentiate from the approved product on the market? Thank you.

Jan Mikkelsen: Yeah. First of all, the primary endpoint is analyzed high velocity over 12 months. And that is the primary endpoint. We are in discussion additional secondary endpoint in — related to the endpoint we already have, which really should go into the [biology] (ph), the idea how we really can address. As we said in the script, we believe that achondroplasia is not only a skeletal dysfunction, but also have a musculus impact. And we really have since benefitted there, which are really are being supported with our review of [indiscernible] where you really can show how achondroplasia has a muscle weakness. And we believe that this is one of the reasons why we see 100% attention in our trial and it was an immediately effect we observed in our Phase 2 trial.

And this is why we really is extremely thrilled to really take this product TransCon CNP. Not only we believe there is a clear benefit for pediatric, but potential that could also be in a proven for adults with achondroplasia.

Operator: Thank you. Please standby for our next question. Our next question comes from the line of [indiscernible] with Leerink Partners. Your line is open.

Unidentified Analyst: Great. Thanks so much and congrats on all the progress. I was wondering, how the gross to net for SKYTROFA has been evolving in the United States and what your pricing strategy will be in Germany and other European markets. Thank you.

Jan Mikkelsen: Let me take the last part of your question first. We know we believe we’re providing an improved treatment for the patients. We also believe that we’re following up to what we said before, a responsible premium pricing. This is how we really have launched our price structure in the U.S., and we will continue to implement that throughout all the different markets. This is where we want to be because we’re providing an improved treatment. Scott, will you take the last part?

Scott Smith: So, Joe, we don’t comment on GTN evolution. We just want you to focus on revenues ultimately, but I think we gave some interesting points that reported revenues. We were the market value leader in the quarter with less than 10% penetration into the pediatric GHD market, which itself is only half of the total growth hormone market in the U.S.

Jan Mikkelsen: Yes. And so, when you start to make this calculation, which you likely will do setting and calculate that, what does meaning mean? The basic mean is that we have the vision on being the value driver, the most value product, but in a growing growth hormone market. And that’s definitely what we are managed to do here because of the improvement in treatment we’re providing.

Unidentified Analyst: Thank you.

Operator: Thank you. Please stand by for our next question. Our next question comes from the line of Andreas Argyrides with Wedbush Securities. Your line is open.

Andreas Argyrides: Thanks for taking our questions. Congrats on all the progress. With the eGFR data, how should we interpret the 50% response rate? And how do you expect the eGFR analysis to be reflected on the potential label? And looking at the baseline characteristics of patients in [PaTHway 15] (ph) in the TransCon PTH group history of kidney stones, can you speak to the impact of TransCon PTH had on kidney stones? And if you plan on sharing this data in the future? And then, I have one follow-up.

Jan Mikkelsen: Okay. Thanks a lot. We are extremely thrilled with this data. Why we are thrilled? Because we really believe it’s providing a huge benefit to the patients. A huge benefit, because one of the element that the patient really fear is really on conventional therapy. Your basically are dividing [indiscernible] impairment, which at the end can be worst case scenario where you go into dialysis and really need to have a new kidney. So, out from that perspective, when we looked in the data, we basically selected 60 out from the perspective is that some kind of accepted definition of really where you have insufficient or not. And why we were thrilled with the data that we couldn’t take 50%, five-zero, of the patient that basically already worked classified of having [indiscernible] insufficient and basically move them up to what we call normality.