Lexicon at H.C. Wainwright Conference: Strategic Pipeline Developments

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Published May 20, 2025 11:07AM ET

Lexicon at H.C. Wainwright Conference: Strategic Pipeline Developments

On Tuesday, 20 May 2025, Lexicon Pharmaceuticals (NASDAQ:LXRX) presented at the H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025. The company provided an insightful update on its cardiometabolic drug pipeline, highlighting both promising advancements and challenges. Key discussions included clinical trials, strategic partnerships, and financial metrics, reflecting Lexicon's commitment to innovation and market expansion.

Key Takeaways

  • Lexicon is shifting focus towards research and development, with a strong emphasis on partnerships.
  • The company has $195 million in cash and investments as of March.
  • A significant deal with Novo Nordisk for the obesity asset LX9851 could yield up to $1 billion in milestones.
  • Positive progress in clinical trials for pilovaparine and sotagliflozin, with plans to engage with the FDA.
  • Lexicon is maintaining sales for sotagliflozin despite a reduced commercial footprint in the US.

Financial Results

  • Cash and Investments: $195 million as of March.
  • Impefa (sotagliflozin) Sales: $1.4 million in Q1, consistent with previous quarters.
  • LX9851 Deal: $75 million upfront payment, with $45 million used to reduce debt, and potential for up to $1 billion in future milestones.

Operational Updates

  • Pilovaparine: Phase 2b PROGRESS trial completed; 10mg dose selected for Phase 3. Plans for data presentation at medical meetings and an end-of-Phase 2 meeting with the FDA.
  • Sotagliflozin (Impefa): Ongoing sales in the US, with regulatory submissions in UAE, Saudi Arabia, and Canada. The SONata HCM trial is actively enrolling patients globally.
  • LX9851: IND-enabling studies are in progress, with Novo Nordisk handling the IND submission.

Future Outlook

  • Pilovaparine: Seeking partnerships for commercialization and exploring additional indications.
  • Sotagliflozin (Impefa): Anticipating regulatory approvals outside the US and planning to file a supplemental NDA for HCM.
  • LX9851: Oral and combination therapies expected to play a significant role in obesity treatment.

Q&A Highlights

  • The SONata HCM trial's primary endpoint is the change in KCCQ score.
  • The trial includes both obstructive and nonobstructive HCM patients.
  • Lexicon's US commercial footprint has been scaled down, focusing on strategic market segments.

For a detailed understanding, readers are encouraged to refer to the full transcript below.

Full transcript - H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025:

Landon, Senior Research Associate, H. C. Wainwright: So good morning, everyone, and welcome to our next fireside chat. My name is Landon. I'm a senior research associate at H. C. Wainwright.

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And here with me are Mike Eckson, CEO, and Craig Renowitz, CMO of Lexicom Pharmaceuticals. Thank you both for joining us.

Mike Eckson, CEO, Lexicon Pharmaceuticals: Thanks so much for the invitation.

Landon, Senior Research Associate, H. C. Wainwright: Of course. Yeah. So before we start from a disclosure standpoint, we currently cover Lexicon with a buy rating. So maybe, Mike, you reported first quarter results last week. But for those who may not be familiar with the company, can you please provide an overview of Lexicon?

Mike Eckson, CEO, Lexicon Pharmaceuticals: Yeah. Pleasure. So Lexicon is a biopharmaceutical company that has a portfolio of preclinical, late late clinical stage and commercial assets that are oral medicines, predominantly in the cardiometabolic space in kind of hot areas right now of obesity, hypertrophic cardiomyopathy as well as neuropathic pain associated with diabetes. We have had some recent partnerships that have allowed us to continue to develop our medicines and get them in the hands of patients who desperately need them and have at the close of March about $195,000,000 in cash and investments. So the company really focused on addressing large unmet medical need in the cardiometabolic space.

Landon, Senior Research Associate, H. C. Wainwright: Perfect. Thank you for that. So maybe let me start with pilovaparine, the non opioid AKA1 inhibitor candidate for diabetic peripheral neuropathic pain. Can you please provide some insights about this indication and how it is different from recent approvals in acute pain?

Mike Eckson, CEO, Lexicon Pharmaceuticals: So maybe I'll lead off and ask Craig to speak more specifically in acute versus neuropathic pain, which are two very different disease states, not only in terms of how they present clinically and their signs and symptoms, but also in the pathophysiology. And you're right, there's been a lot of activity both from recent approvals as well as recent data in acute pain. And yet there remains really very little compelling data in neuropathic pain. And so we recently concluded the Phase 2b progress study in diabetic peripheral neuropathic pain, which is important because it's by far the largest market opportunity. There are about nine million people in The U.

S. Diagnosed with diabetic peripheral neuropathic pain. And it's a disease state where really there are very little suitable treatment. So there hasn't been any new oral medicine developed for two decades. And patients typically start in treatment with gabapentin, but they quickly cycle through other options either switching or add on really because they get very little pain relief and they're pretty intolerable some of these medicines.

So in the end, a lot of patients end up cycling through different medicines and give up on traditional Rx medicines and go to alternative acupuncture and all sorts of non prescription type medicines. And so there's a desperate need for new treatments that are non opioid because as we all know, there's a big opioid crisis in The U. S. In particular, but worldwide. And indeed in DPNP, about 20% of the market at the moment are oral opioids.

So it's really something where both politically and from the patient viewpoint and the physician viewpoint, there's desperate need for new medicines. Craig, your thoughts around acute versus neuropathic pain?

Craig Renowitz, CMO, Lexicon Pharmaceuticals: So the physiology is quite distinct between the two, and the impact on patient lives is remarkably different. So if think about acute pain, most of the time, it's some sort of surgical, post surgical pain, neuromuscular pain, or otherwise. By definition, acute pain resolves within a couple months. Chronic pain, by definition, again, is a consistent pain state for greater than six months. There you have not only a very different set of underlying disease conditions, but there's also rewiring of the central brain chemistry and circuitry around chronic pain.

So there is an element of anticipatory pain and really just a way of how it impacts patients' lives overall, which is quite different and distinct from acute pain. Perfect.

Landon, Senior Research Associate, H. C. Wainwright: So, yeah, as you said in March, you announced top line results from the phase two b progress clinical trial. Can you guide us through these results and maybe how they could inform the upcoming registrational studies?

Craig Renowitz, CMO, Lexicon Pharmaceuticals: So the phase two program, like most phase two programs, was really designed to definitively determine dose route and schedule for your phase three pivotal trials. And the PROGRESS trial was first and foremost designed to answer that question with two very important underlying, questions that were there. The first is what was the value of the loading dose? Because in the pilot study, the relief study, there was a day one loading dose of ten times the maintenance dose. And there was also a lot of early dizziness associated with the drug.

So the question is, could you separate the dizziness with the dose and the PK related to that? The second is we had two doses that demonstrated activity, a ten milligram daily dose and a twenty milligram daily dose. So the two objectives of the progress study is value and importance and impact of the daily loading dose on both efficacy and safety. And can we pick a single dose to go into phase three to have a streamline phase three registration program? The trial achieved both those objectives.

The ten milligram dose clearly support performed superiorly to the twenty, and the loading dose was associated with the dizziness, and it really added nothing to the efficacy over the long term. So we believe the study in that regard of what it's designed to do, which is the entry way into a streamlined phase three, was very important, and we believe the progress study derisked the development program.

Mike Eckson, CEO, Lexicon Pharmaceuticals: I think to pile on to that, you know, because we have to take the Phase two program in its totality now. And importantly, the ten milligram dose across both progress because there were two essentially two 10 milligram chronic dosing in progress and with relief. We've now shown three times that that ten milligram dose will separate from placebo. And that's very different any other program that is looking at neuropathic pain because when they've used placebo in those trials, in fact, they've shown no separation. So when Craig says we think we've got a derisked dose and asset to move into Phase three, I think that gives us the confidence that three shots on goal essentially that we do have an asset that truly does show meaningful separation from placebo and will continue to show separation in the Phase three trials, which will be extended to twelve weeks.

Landon, Senior Research Associate, H. C. Wainwright: Perfect. So, yeah, with those data in hand, what's the net what are the next regulatory steps and the phase three clinical plans for pilovaparin?

Craig Renowitz, CMO, Lexicon Pharmaceuticals: Yeah. The goal now is it's many of the listeners might know, is that there were two closes of the database. The first was a primary endpoint at eight weeks. The second was a four week blinded runoff period where we're collecting the data right now. We plan on presenting that data at major medical meetings medical meetings later this year.

When we have that data plus a couple other smaller studies we're running is to then have an end of phase two meeting with the FDA to finalize our three phase three design. Prior to starting the progress study, we did meet with the FDA. We sort of laid out in broad brush strokes what a phase three program would be, and we've publicly communicated that we're anticipating two parallel phase three trials in a similar patient population Based on the statistics, were reaffirmed by the PROGRESS trial, we believe each of those trials, about 600 patients in total with a one to one randomization between a single dose of pilavapitan, now ten milligrams, and placebo.

Landon, Senior Research Associate, H. C. Wainwright: And just maybe out of curiosity, as a side note, based on your comments during the earnings call, can you talk about pilavaparin's opportunities for additional indications and potential for partnerships?

Mike Eckson, CEO, Lexicon Pharmaceuticals: Yeah. It actually goes hand in hand because one of the reasons that we think it's important to look for a partner of this particular asset is like all of our medicines actually, we consider them a pipeline and a pill because there are multiple potential indications for them. And having a partner gives us potentially two options or two benefits. One, commercially providing some scale and strength commercially to launch this medicine in the importantly, really dive deep into the potential indications not only within neuropathic pain because we have Phase two data as well as a lot of preclinical data on a number of different types of neuropathic pain. So it looks to be a pan neuropathic pain asset, but also spasticity in particular related with MS and spinal cord injury.

So there really is a breadth of indications that we can explore with a partner and those discussions have been very productive and are ongoing as we await the full dataset and move towards the Phase two meeting. So we think while it's important to have a partner in this program unlike 09/1951, the obesity program where we completely out licensed it, we granted exclusive license. We feel that we have capabilities that are important to developing and commercializing this asset. So we see a partnership here being quite different, one in which Lexicon continues to have a significant stake in the development and commercialization moving forward.

Landon, Senior Research Associate, H. C. Wainwright: Right. So maybe changing gears into cardiac indications, your dual SGLT two, SGLT one inhibitor, soragliflozin, is currently approved in The US for heart failure. Brand name is Impefa. Can you tell us about soragliflozin's commercial progress, not only in The U. S.

But also globally?

Mike Eckson, CEO, Lexicon Pharmaceuticals: Yes. Great. So commercially, as you know, towards the end of last year, we pivoted to become an R and D focused company and we've seen great success this last quarter from achieving a number of our objectives in doing that. With doing that, we scaled down the commercial footprint in The U. S.

To essentially not much. And what I'm pleased to tell you is actually in Q1, we've been able to maintain our sales that we saw in Q3, Q4. So we generated about $1,400,000 in MPEFA sales with very little resourcing. And I think that really shows that when you do get access and you have prescribers who see the benefit and patients who see the benefit of this medicine, they truly are loyalists. So we expect no sales growth in Impefa this year, but we expect to continue to see sales come out of The U.

S. In Ex U. S, as we've mentioned in the earnings call, Beatrice have now submitted their dossier to The UAE and to Saudi. Canada will come soon as will a number of other countries. So as we go through the regulatory approvals worldwide, we will start to see sales meaningful sales come out of these countries and we have a pretty significant both milestone and royalty agreement with Viatris for that.

And in The U. S. Commercially, our aim is to continue to keep Impefra in the market, continue to drive differentiation as you've seen with the recent MACE publication in the Lancet to show that this SGLT12 dual inhibition truly makes meaningful difference and continue to build that evidence as we look towards the HCM data coming at the end of twenty twenty six with well finishing the study at the end of twenty twenty six with data coming in early twenty twenty seven.

Landon, Senior Research Associate, H. C. Wainwright: Okay. Yes. So as you said, now you're looking to expand growth opportunities for sotagliflozin. So my question would be why hypertrophic cardiomyopathy? And what's the rationale behind evaluating sotagliflozin in both forms of the disease, obstructive and nonobstructive HCM?

Craig Renowitz, CMO, Lexicon Pharmaceuticals: The underlying pathophysiology of the disease is similar whether it's obstructive or nonobstructive. There's really two components to what defines, HCM physiologically. The first is that there's an overdynamic myocardium that uses too much energy. The second is that you have insufficient relaxation during diastole, which leads to pressure overload, both of which ultimately lead to a thickened scarred heart that can't pump blood effectively. What you see with HCM, even in obstructive HCM, when you do a reduction surgery, a significant percentage of those patients do go on to develop heart failure.

So it's not just that there's the outflow of tract obstruction as the physiology. It is the outcome of the physiology, but not the underlying physiology. And in that regard, we believe that sotagliflozyl with its dual mechanism of action, and there are SGLT one receptors directly on the myocardium, provides something that is different from the CMIs, something that's different from beta blockers, calcium channel blockers, but also something that is differentiated compared to SGLT two inhibitors.

Landon, Senior Research Associate, H. C. Wainwright: Perfect. So, now sotagliflozin is getting ready for late stage clinical development in HCM in the SONata HCM clinical trial. If you can tell us a little bit about the current status of the program and the overall design of the trial.

Craig Renowitz, CMO, Lexicon Pharmaceuticals: Yeah. The design, we believe, is both broad and pragmatic. So it's including both obstructive and nonobstructive. And, again, this study before we started it, we gained alignment with the FDA on the design and the endpoints on what would be adequate for approval. And as a reminder, this is an approved drug that reduces the risk of heart failure.

The others are drugs that are associated with an increased risk of heart failure as a class. So we believe that that is a, you know, a very appealing, perspective to provide to to cardiologists. Also, we have a long established safety record both in terms of a large clinical trial program of 12,000 patients as well as post marketing experience with well over 3,000 patients in the post marketing, experience with a class of medications that cardiologists are familiar. The trial includes, as I said, both obstructive and nonobstructive. We're allowing the use of underlying medications, including CMI, and the major entry criteria is symptomatic HCM, whether obstructive or non obstructive, defined by a minimum KCCQ score.

The primary endpoint of the trial is change in KCCQ score. We've grounded that expected difference based on, again, our extensive HFpEF program because, again, HCM really is essentially HFpEF. And we feel quite confident that based on that experience and the other clinical data we have that we're gonna have a drug that has a high probability of achieving its endpoint.

Mike Eckson, CEO, Lexicon Pharmaceuticals: If you don't mind, let me let me pile on top here a little bit and just give a sort of a commercial perspective as we look in HCM because there's been a lot of data in q one quite quite revealing, I think, in HCM and the various mechanisms that are being studied. It's always been my position and our position that CMIs are going to be somewhat restricted in their availability commercially because of the complexity of the REMS. And we've seen that with BMS. We've now seen that with africanctin and the need to go back to the FDA with the REMS because those protocols are pretty laborious both from an echo frequency, from the need to screen for DDIs each time you give the medicine. So what that does is it limits the prescriber base to those academic centers that really can manage the complexity of just the logistics.

It's very complex to start and maintain those drugs. And you see that borne out in the fact that there's about one million HCM patients in The U. S. And currently treated with CMIs is about ten thousand, so less than one percent or one percent or thereabouts. The other factor that you need to consider with the CMIs is that they do potentially contribute to heart failure and now there's also an AFib signal.

And here you have a drug, an SGLT1 and two dual inhibitor that has potential not only in obstructive HCM, but we have good reason to believe that it's going to be effective in non obstructive HCM because it targets the underlying cause of the disease, not the hemodynamic obstruction that we see with obstructive HCM.

Landon, Senior Research Associate, H. C. Wainwright: Okay. And this is a global phase phase three trial. Right?

Craig Renowitz, CMO, Lexicon Pharmaceuticals: Yes. It's running in 20 countries. Trials posted on clin trials, and all the countries are listed. But we've now have regulatory approvals in all the countries. We've enrolled significant numbers of patients in The US and are now actively enrolling patients outside The United States.

Landon, Senior Research Associate, H. C. Wainwright: Okay. And the plan would be to file a supplemental NDA for HCM

Craig Renowitz, CMO, Lexicon Pharmaceuticals: Absolutely.

Landon, Senior Research Associate, H. C. Wainwright: For sotagliflozin.

Craig Renowitz, CMO, Lexicon Pharmaceuticals: With both obstructive and nonobstructive with a baseline EF down to 50 on top of, or in as monotherapy or on top of existing therapy, including CMIs.

Landon, Senior Research Associate, H. C. Wainwright: Perfect. Yeah. So I also wanna touch a little bit on your obesity asset, $98.51, and the recently announced deal with Novo Nordisk. Congratulations, by the way. So can you tell us about the the terms of the deal and the importance of it considering that $98.51 is a preclinical stage asset?

Right.

Mike Eckson, CEO, Lexicon Pharmaceuticals: Now look, we're delighted with doing that deal with a world leader in obesity and related disorders. And just to answer quickly the first question around the financial terms, we received $75,000,000 in upfront near term milestones, 45,000,000 of that was the upfront, which we actually used to down pay some of our debt, which is good for the balance sheet and up to $1,000,000,000 in regulatory and developmental milestones plus sales royalties. So really, if you look at that market, the long term potential of that particular asset, as we've said all along, you see it now more and more with both deals that are being done as well as scientific data that's coming out in this class or in this market that it really will turn to oral and combination therapies. I think that's classic. What happens in pretty much all of cardiovascular disease is you end up with oral combination therapies that are designed to suit that patient.

I think that's where 9,051 will fit in Novo's portfolio really well. The reason why we ended up and we had a number of folks interested in this particular asset and this particular mechanism is Novo, we believe really understands the science and the breadth of the potential of this asset not only in obesity, but in a number of other related disorders and they're committed to developing the breadth of that asset. So for the long term, although we're now finishing the preclinical IND enabling studies, Novo will be responsible for submitting the IND, but this has been already a very fruitful and collaborative partnership.

Landon, Senior Research Associate, H. C. Wainwright: And to the extent that you can share, how close is Novo involved in the IND enabling studies? And what's the anticipated time line for Novo for the

Mike Eckson, CEO, Lexicon Pharmaceuticals: clinical progress of the asset? Yes. Look, the IND enabling studies are pretty straightforward. So we're responsible for completing those. We're in touch with them, but they don't really have any involvement in running them.

That was how the deal was structured. And we expect to complete that this year. Submission of the IND is then fully Novo at Novo's discretion. But what I can say is their enthusiasm continues to be very, very strong. And so I wouldn't expect that there would be much or there would be a haste to get the IND submitted.

Landon, Senior Research Associate, H. C. Wainwright: So maybe for just for the sake of time, would you like to summarize the upcoming catalysts for the programs or clarify anything that investors should be aware of?

Mike Eckson, CEO, Lexicon Pharmaceuticals: Yes. Look, thanks for the discussion, first all and thanks for the invitation. We've got a number of exciting catalysts coming up ahead of us. We continue to close out and finish the rest of the secondary data for pilobapitan, working towards disclosing the full dataset at an upcoming medical meeting this year and then coordinating with the FDA for an end of Phase two meeting. We continue to get sites up and running now across both U.

S, EU and Latin America for Sonata HCM. We expect all those sites up and running by Q3 and now we have a sort of a clear runway to really push the enrollment of the Sonata HCM trial very strongly. As you know, we're continuing to look towards engaging with the FDA on some of our sotagliflozin data, particularly around MACE, where we see another one of another potential indication and another differentiation versus other SGLT2 inhibitors. We are completing the IND enabling studies for 09/1951. We'll conclude that this year.

And then finally, something that we don't talk about much, but we're wrapping up the process for the end of review meeting for Zynquista in Type one diabetes. As you know, the patient advocacy and demand for new treatments, particularly with sotagliflozin and Zynquista is still incredibly high. So we're committed to seeing through that process throughout this year and bring that to its natural conclusion. So a lot going on for us and a lot of positives as we continue to drive value for the development pipeline.

Landon, Senior Research Associate, H. C. Wainwright: Great. Well, thanks again, Mike and Craig. This was very informative. Thank you so much. And we look forward to upcoming company updates.

Mike Eckson, CEO, Lexicon Pharmaceuticals: Thanks so much. Thank you. You. Appreciate it.

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