Good day and welcome to Akebia's Second Quarter 2023 Financial Results Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session and instructions will be given at that time. As a reminder this call is being recorded.

I would now like to introduce your host for today, Mercedes Carrasco, Senior Director of Corporate Communications and Investor Relations. You may begin..

Thank you. Thank you and welcome to Akebia's second quarter 2023 financial results and business updates conference call. Please note that a press release was issued earlier today, Friday, August 25th, detailing our second quarter financial results and that release is available on the Investors section of our website.

For your convenience, a replay of today's call will also be available on our website after we conclude. Joining me for today's call, we have John Butler, Chief Executive Officer; Dr. Steve Burke, Chief Medical Officer and Ellen Snow, Chief Financial Officer. I'd like to remind everyone that this call includes forward-looking statements.

Each forward-looking statement on this call is subject to risks and uncertainties that could cause actual results to differ materially from those described in these statements.

Additional information describing these risks is included in the financial results press release that we issued on August 25th as well as in the Risk Factors and Management Discussion and Analysis section of our most recent annual and quarterly reports filed with the SEC.

The forward-looking statements on this call speak only as to the original date of this call, and except as required by law, we do not undertake any obligation to update or revise any of these statements.

As described in the press release, we intend to file an amendment to our 2022 annual report on Form 10-K to reflect corrections to our previously issued financial statements due to recently identified accounting errors related to the recording and reporting of return reserves for Auryxia as well as our second quarter Form 10-Q.

Please note that comments made during this call regarding the company's financials reflect revised financial statements for the years ended December 31st, 2022, 2021 and 2020 and the first quarter of 2023. As outlined in the press release and to be described in the amendment to the 2022 Form 10-K and the second quarter Form 10-Q.

With that, I'd like to introduce our CEO, John Butler..

Thanks, Mercedes, and thanks, everyone, for joining us. It's a pleasure to be here with you today. While this is a little later than we expected to be together, the extra time spent was necessary.

We're very pleased that the revisions to our financial statements that we had to work through are not material and they don't impact the value of the company.

You'll hear from Ellen shortly, but I want to thank her and her team for the work done over the past several weeks that helped to ensure we are best positioned to maximize value as we approach several important catalysts.

First, let's focus on the operational progress that we've made over the past few months, working through towards the potential approval of our second product for patients with kidney disease, vadadustat.

We are now on a clear path to potentially gain US approval for vadadustat as a treatment for anemia due to chronic kidney disease or CKD in adult patients on dialysis.

In fact, as we shared earlier, we've received the minutes from our recent end of dispute Type A meeting with the FDA, which reflect our productive discussion with the agency and support our optimism that we will resubmit our new drug application or NDA for vadadustat by the end of this quarter.

With that timing for resubmission, we would expect a decision from the FDA on the vadadustat NDA in March of 2024. We have Dr. Steve Burke here to answer any questions about our interactions with the FDA or our time line for NDA resubmission at the end of the call. And I'll also discuss initial plans for an anticipated launch next year, if approved.

What's especially encouraging for our team at Akebia is that the momentum in the US regulatory discussions follows tangible progress in markets around the globe. Vadadustat is approved in 34 countries.

In the past quarter alone, the European Commission, the United Kingdom Medicines and Healthcare Products Regulatory Agency and Swiss Agency for Therapeutic products approved Vafseo vadadustat for the treatment of symptomatic anemia associated with CKD in adults on chronic maintenance dialysis in the EU, United Kingdom and Switzerland, respectively.

Further, Akebia expects a regulatory opinion on vadadustat in Australia this year. I want to credit our team who assumes responsibility for the regulatory processes outside the US mid last year and worked efficiently to secure local approvals to help to enable Akebia to deliver a new therapeutic option to patients in need.

To that end, we had previously discussed our priority was to secure a partner in Europe who would bring Vafseo to market. Here again, we met our objective and signed a license agreement with Medice. Medice is a fully integrated pharmaceutical company based in Germany with a strategic focus on the dialysis market.

The license agreement grants Medice the exclusive rights to market and sell Vafseo in the European economic area, the United Kingdom, Switzerland and Australia for the treatment of anemia in patients with CKD. The agreement included an upfront payment of $10 million.

And in addition, we're eligible for commercial milestone payments up to an aggregate of $100 million and tiered royalty payments ranging from 10% to 30% of Medice's net sales. We believe Medice is situated to maximize the European market potential for vadadustat.

They understand the unique country-by-country pricing and access dynamics related to dialysis. They are committed and driven to succeed, and we continue to support their efforts. We expect Medice to launch as soon as possible in 2024.

Building on the regulatory success we've had in other markets, we're eager to advance the regulatory approval process for vadadustat in the US. An approval in the US would represent our most significant commercial market opportunity and allow us to target the 550,000 US patients with anemia due to CKD who are receiving dialysis treatment.

I'll note that the resubmission is a very focused filing. As part of the resubmission, we'll submit safety data collected since our original submission in 2021, including data from two alternate dosing studies FO2CUS and MODIFY.

We'll also submit post-marketing data from Japan, where tens of thousands of patients have received the drug over the past two years. Now working through a potential launch time line, again, we expect to resubmit the NDA by the end of the quarter.

Since we're submitting data that was not in the original NDA, our expectation is that our resubmission would undergo a six-month review. And this review period would commence upon submission. As such, we anticipate a new PDUFA date likely in March of 2024.

Upon a potential approval, we'll quickly file for TDAPA reimbursement, which is a six-month process. After receiving TDAPA designation, we'd expect the product to be widely available for patients. We're actively preparing for a potential launch of vadadustat and are reengaging around the commercial opportunity. Let me summarize.

We estimate that approximately 88% of the nearly 550,000 patients on dialysis are treated with an erythropoiesis-stimulating agent or an ESA for anemia, an injectable that is currently the standard of care. Medications used to treat dialysis patients in the US are paid for as part of a bundled payment made to dialysis providers.

Now to promote innovative drug use for Medicare patients on dialysis, CMS created a transitional add-on payment adjustment over TDAPA. The add-on payment would cover the cost of vadadustat when a physician prescribes the drug for two years after receiving the TDAPA designation on top of the regular bundled payment.

The TDAPA payment would allow providers to incorporate innovative products like vadadustat into their treatment protocols while still receiving the full bundled payment for treatment of their PPS or prospective payment system, Medicare patients.

And we believe that we've created a favorable environment for adoption of the product as we prepare for a potential launch. We have a collaboration with Vifor CSL that provides access to up to 60% of the dialysis market through existing Vifor CSL relationships, which includes Fresenius Kidney Care and most small to midsized providers.

Vifor CSL has a relationship with Fresenius for the procurement of therapeutic products used in their network and vadadustat would be made available through that collaboration. We believe Vifor's unique relationship and our experience in the dialysis market will create a favorable environment for pull-through within the dialysis centers.

Together, we believe we are well positioned for a successful launch if vadadustat is approved. Our collaboration with Vifor CSL is a profit share, where we retain approximately two-thirds of the profit from vadadustat, net of certain pre-specified costs with Vifor CSL keeping the remaining one-third.

For the remaining 40% of the dialysis market that Vifor CSL does not have rights to sell to, we retain 100% of the economics. So one of the most common questions I get these days is on launch costs. I'll note that we are also in a strong position here. The most significant launch expense is related to people and product.

But we have the commercial organization in place today with only incremental additions needed and we already have the product on the shelf to launch as soon as we receive approval. We believe our additional costs will be truly incremental.

Now to speak more about our financial position, I'm pleased to introduce Ellen Snow, who joined our team as CFO and Treasurer last month.

Ellen's impact has been near immediate as she brought vast accounting and financial management expertise into the process to close the quarter and put the organization on the right footing by strengthening our product return reserves accounting process that was described in the press release filed this morning.

This financial discipline will be especially important as we prepare to launch vadadustat in the US if approved.

Ellen?.

Thanks, John. First, let me say that I'm extremely pleased to have joined Akebia last month.

I saw significant potential and upside for the organization associated with the pending potential vadadustat FDA approval as well as opportunities to optimize the company's cost structure and better leverage the cash flows from Auryxia towards improving the balance sheet and creating shareholder value.

I'm grateful to have a talented, hard-working and dedicated accounting team who have worked tirelessly over the last few weeks as we identified and corrected errors in the accounting. As previously announced during the second quarter close process, we identified errors related to the accruals for product returns.

We concluded the errors represent a material weakness in internal controls over financial reporting and deficiencies we are working to remediate.

We also intend to revise our financial statements in our 2022 annual report on Form 10-K to correct the impact of the errors which will primarily impact the balance sheet and the affected years, including liabilities for product returns, accounts receivable and goodwill as well as immaterial changes to the prior year revenues.

We expect to file our amended Form 10-K and second quarter Form 10-Q on Monday. Now let's move to our current financial position. Importantly, we believe our cash runway will fund our current operating plan for at least 12 months. Cash and cash equivalents as of June 30th, 2023, were $53.6 million.

Total revenue was $56.4 million for the second quarter of 2023. Of that, $42.2 million was Auryxia net product revenue and $14.1 million was other revenue. Other revenue consists primarily of the $10 million upfront payment in connection with the Medice license agreement. Auryxia net product revenue increased 21.6% over the previous quarter.

The growth was due to the timing of purchases in certain Auryxia customers and expected cyclical demand from the first quarter to the second quarter. Auryxia revenue decreased 2.5% compared to the same quarter of 2022.

The decrease compared to the prior year period is due in part to the impact of the shifting payer mix and a volume decrease partially caused by the contracting dynamics and phosphate binder market erosion. We expected quarterly swings in the Auryxia revenue but had also expected the binder market to rebound.

Based on our analysis, we believe the binder market is leveling, but there's evidence of a long tail impact of COVID in the dialysis community. We are in a position to affirm our 2023 net product revenue guidance of $175 million to $180 million.

Our team continues a high-touch engagement with key accounts, and we continue to carefully review the trends. We remain focused on maximizing Auryxia revenue through its loss of exclusivity in March of 2025. Regarding expenses, we reported a decrease in cost of goods sold and R&D expenses compared to the second quarter of 2022.

Cost of goods sold decreased slightly to $17.3 million in the second quarter of 2023 from $18.6 million in the second quarter of 2022. R&D expenses decreased from $26 million to $20.2 million, a more than 20% decrease as a result of reduced spending on vadadustat development, R&D headcount and outsourced contract services.

We have curtailed non-headcount-related expenses growth by continuing to find ways of operating more efficiently, placing an increased scrutiny on all areas of operating expenses and taking measures such as reducing our office footprint with the assignment of the Seaport office lease, which was completed at the end of the second quarter.

We are deliberate about managing our expenses in an effort to extend our runway which will be achieved in part by keeping 2023 headcount relatively flat with current levels. In the time I've spent with Akebia, it's clear that everyone on the team is dedicated to managing resources while continuing to deliver on their individual and group objectives.

Revenue from Auryxia continues to provide cash for operations, and our teams are motivated by the progress we've made across the business this quarter as we all work to prepare for the launch of vadadustat in the US next year, if approved. With that, let's open the line for questions..

Thank you. [Operator Instructions] Our first question comes from Allison Bratzel with Piper Sandler. Your line is open..

Hey, good morning, and thank you for taking the questions.

So first just hoping you could frame for us from a high-level topics that were addressed at the Type A meeting, any remaining gating factors to resubmission for vadadustat and just your confidence that FDA will find the resubmission approvable and dialysis without the generation of additional clinical data? And then I have a follow-up..

Sure, Ally. Thanks for the question. I mean it was I think the Type A meeting was pretty technical meeting a lot of ways. Steve is here and attended the meeting.

Steve, maybe give a little color?.

Sure. This is Steve Burke, the Chief Medical Officer. The meeting was -- it's like a pre-NDA meeting where you meet with the agency to describe how you're going to present the requested data and is that acceptable to them. So for instance like when we finish an NDA review, we have a 120-day safety update. So we have to do another safety update.

And so we just clarified, for instance, the window in which any new data would be collected and reported.

So John alluded earlier to the MODIFY FO2CUS studies, what we were going to present, what we're going to provide to them and was that acceptable, how we were going to present Japanese post-marketing surveillance data, the actual components and how would be analyzed and things of this nature.

They also had asked some questions about some additional analysis they wanted of the Phase III data. But all of this is pretty modest in scope. But as John indicated is new data and therefore we expect they will get a six-month review clock because of that.

I think we're satisfying all the requests and have really excellent clarity on what they want and expect from us and that we're able to deliver that. And there's no need for us to generate new data, clinical data..

Got it..

Yes. I would say that it was an amazingly collaborative meeting kind of given that the CRL from last year, it was very focused on what we need to put through. And even going into it, most of the questions that we ask, we pose to them, they found our approach reasonable. So there's really more clarification than anything else.

So we feel pretty good about where things are and very much on track for our filing..

That's helpful.

So now just hoping you could also walk us through your expectations for the vadadustat launch will look like kind of between the March PDUFA and the receipt of TDAPA designation during that time frame? And then separately, if you could just kind of frame or compare contrast for us whether KORSUVA's launch experience under TDAPA, is it good analog for vadadustat? Any color or perspective there would be helpful.

Thanks..

Yes. No, thanks for that question, Ally. So a couple of things that are really important to communicate. While we will make the product available during the post-launch pre-TDAPA is really available for dialysis providers to purchase the product and develop protocols or the like.

We don't expect widespread adoption until you have the TDAPA designation and they'll have the product reimbursed outside of the bundle. So that's -- I mean I think that should be a clear expectation for everyone. If we see any dollars, it will be very modest and you'll really see that uptake at TDAPA.

I think the KORSUVA question is a really important one as well, and I appreciate you asking that. The KORSUVA launch is a very different product and a very different opportunity than vadadustat. I think for a number of reasons.

When you look at KORSUVA, this is a product that was approved for pruritus, for severe itching, which is in a small number of patients. And the average pricing that's published now for KORSUVA is about $135 per dialysis session.

And there's nothing in the bundle to cover pruritus other than $0.25 for BENADRYL, right? So the $135, they'd be reimbursed on a cost basis as well.

But when you speak to physicians about that experience and that desire to use the product, they really worry about, well, when TDAPA ends, there's no mechanism for me to continue to use this product to benefit patients.

And think about bundled payments is about $280 per dialysis session, and you have $135 for KORSUVA is a real worry from a financial perspective about having to use that, continuing to use that product. So you're just not seeing patients who could benefit from the product benefit.

And so that's one of the reasons why we've been trying to push CMS to create continued add-on payment beyond TDAPA that follows the drug and haven't been successful yet. Now that do set very different, right, because there's money in the bundle for ESAs today, the pricing will be very different.

While it may be premium to ESAs, it's certainly not going to be $135 per dialysis session and that those dollars that are in the bundle will continue to be in the bundle post TDAPA. So now our pricing may have to change from a contracting perspective post TDAPA.

But it's not as if people are going to stop treating anemia or not treat anemia, right? They're going to do that, which they're not doing with pruritus. So TDAPA is very much a positive for us, it has been more of a challenge for KORSUVA..

That's super helpful. Thank you..

Thanks, Ally..

Thank you. Our next question comes from Ed Arce with H.C. Wainwright. Your line is open..

Great. Hello, everyone. Thanks for taking my questions. First, I just wanted to ask again on the Type A meeting. John, you and I have talked about this a number of times since the letter in May. Specifically about the two issues from the CRL, the thrombotic events and the risk of [indiscernible].

And wanted to get your thoughts on how those meeting minutes now that you have them in hand are fully aligned with the path that was offered in that letter from Dr. Stein back in May. And then I have a follow-up..

Yes. I think I can say unequivocally, that they're fully aligned. I mean I think we have -- as I said, and Steve, you kind of give more detail on, I mean, a lot of that meeting is kind of how you're going to present the data. It's more technical. But we had questions about our labeling, right, I want to submit that.

I mean that's always a good sign, right? So it was -- there was nothing to indicate in that meeting that there was anything that was misaligned with the letter that we received from Dr. Stein. As I've said all along, I think that letter describes kind of the guardrails in the path for us and for the division.

And I think that was reflected in the discussions. It was -- again, it was a very collaborative discussion. I mean it's fair to say you never are quite sure what you're going to -- when you get when you go into those meetings.

And I think sure Steve agrees, I mean, we were quite pleased with the quality of the focus on the issues and the collaborative nature of the discussion and makes us feel quite good about our path forward..

Great. And then I'd like to ask if you could remind us of the objective of having the MODIFY and FO2CUS studies included in the resubmission..

Yes. So for this resubmission, that's an important question. I mean, obviously, we think the MODIFY and FO2CUS studies will be quite helpful in securing three times weekly dosing in our label after our initial approval but it's clear they won't be looking at that three times weekly dosing regimen for this initial approval.

The reason for adding the MODIFY and FO2CUS data is to -- you basically have to add all of your safety data to your filing, right, which we have to do in '21 when we originally filed and now we simply have to update all of the safety data that we have.

So they won't be considering efficacy from those studies at all, while we know that they were successful. And we think we're expecting they'll support our 3 times weekly filing, and we're working to publish them quickly, it won't be in the label..

Okay. Great. Actually, if I could squeeze one more in, question on Auryxia, probably for Ms. Snow. Given that the half year net sales is now about $78 million and you're reaffirming your guidance of $175 million to $180 million for the full year.

I'm just wondering if you could help us understand what gives you the confidence to reach that guidance especially given what you described as a decline in the phosphate binder market. Thanks..

Yes. Thanks, Ed. I appreciate the question. So a number of things. There are -- if you look at the dynamics of sales over the past couple of years, it's kind of that September to December time frame really is kind of where we see a significant step-up.

To be sure, I would say that our -- we had an expectation of our price being a bit higher, but our payer mix has skewed towards Part D a little bit more than we expected. So I would say my expectation is probably more on the lower end of our guidance range. But when you look at -- there's a number of qualitative things as well.

One of the large dialysis providers haven't let our sales reps in to see people at all and everything was being done virtually. That's just changed in the last month. And I think everyone could agree that sales reps can be much more effective when they're face-to-face with people. So we expect that to be helpful as well.

And a number of other -- those types of dynamics. And as Ellen said, we think that the binder market has still been under pressure, but it is flattening, if you will, we think.

This is obviously, we'll continue to monitor this, but as it flattens -- I mean patients still need to be treated, right? So as they come in the funnel, I think, there's opportunity there.

And we also expect competitive product to be launched in the back half of this year as well and that will come, I think, with a lot of focus on phosphate control, and that's kind of one of those rising tide lifts all boats type moments as well.

So I think that there's a number of those types of issues that allow us to maintain that guidance albeit I would say I would think more towards the lower end of the range where earlier in the year, I was expecting the higher end..

Great. That's very helpful. Thanks so much..

Thank you, Ed..

Thank you. Our next question comes from Julian Harrison with BTIG. Your line is open..

Hi. Good morning. Congrats on the progress and thank you for taking my questions. First, just on the three times weekly dosing regimen, again, you studied in FO2CUS.

I'm curious if you could talk more about your strategy there and how soon a potential label for vadadustat maybe be amended with this dosing regimen?.

Sure. So thanks, Julian, for the question. Welcome to the call. So three times weekly is very helpful for treating patients in the dialysis center, right? You give them the dose during your dialysis session, you ensure compliance from the patient, you don't have to send them at home with drug.

So we've always felt that, that was something that was important and that's something that we could potentially add to the label that we never expected to have it in the label at launch. We will publish the data. We expect it to be presented at the ASN meeting in October from FO2CUS.

FO2CUS was the large study that really looked at that three times weekly dosing. Then once we have our NDA approved in March, of course, as we've been talking about and expect, then we'll have a conversation with the FDA about supplemental NDA where we'll submit for three times weekly dosing.

Again, when you think about the low-hanging fruit for the product adoption initially, it's home patients where daily dosing works just fine. And I would say patients who are on the highest doses of ESAs where they're having the risk of exposure to high doses of ESAs and added cost as well to the dialysis provider.

And even though those might be in-center patients, I think, they'd be willing to dose them once a day and send them home a drug or rely on the publication and choose to use three times weekly dosing.

Of course, we wouldn't be promoting three times weekly dosing until it's in our label, but that's certainly something that physicians can choose to do with the data that supports it. And then as I said we will start to have the three times weekly dosing regimen added to our label as quickly as we can..

Okay. Got it. Thanks. That's helpful. And then earlier in the call, you gave a great overview on your current commercial preparations. Sorry if I missed it, but I was just curious if you're talking at all about how much you're manufacturing at risk ahead of potential approval of vadadustat..

Yes. Thanks for that question, Julian. As I mentioned, the two biggest costs are people and product and product is a really significant one. Remember, we had product on the shelf ready to launch back last year.

And our quality team has done a magnificent job of continuing to generate data and we're able to push out the expiry date on that product to four years. So that product that's on the shelf today will be able to support our launch. So we don't have to do any incremental manufacturing and take that incremental cost to be ready to launch the drug.

And of course, that drug is also going to support Medice and the European launch as well..

Excellent. Great to hear. Thanks again..

Thanks so much, Julian..

Thank you. There are no further questions. I'd like to turn the call over to John Butler for closing remarks..

Thanks, Michelle. We are extremely pleased with the progress we've made last quarter as we made significant strides towards our purpose to better the lives of people impacted by kidney disease. We're preparing for very near-term potential catalysts. Most immediately, the resubmission of the vadadustat NDA next month.

With this momentum, we believe we are now poised to disrupt the market for the anemia of CKD in dialysis patients if vadadustat is approved. Everyone at Akebia is excited to be in that position and remains motivated by our potential to help patients. The team is focused on the work we need to do in the very near future to get us there.

And I look forward to continuing to update you as we move through resubmission, acceptance and ultimately, potential approval and launch of vadadustat in the US. Thank you all for joining us today..

Thank you for your participation. This does conclude the program, and you may now disconnect. Everyone have a great day. Goodbye..