Connexa Pharmaceuticals at Jefferies Conference: Strategic Growth and Innovation

On Thursday, 05 June 2025, Connexa Pharmaceuticals (NASDAQ:KNSA) shared its strategic vision at the Jefferies Global Healthcare Conference 2025. The company highlighted significant revenue growth and ongoing clinical advancements, while addressing challenges in market penetration. Connexa’s efforts to expand its treatment reach and develop new therapies were underscored by a strong financial performance and future plans.

Key Takeaways

• Connexa reported a 75% year-over-year increase in Q1 2025 revenue, reaching $137.8 million.
• The company raised its 2025 net revenue guidance to between $590 million and $605 million.
• Clinical trials for KPL-387 are progressing, with a potential market launch between 2028-2029.
• Connexa aims to increase its penetration in the multi-recurrence patient population for Arcalist.
• The company maintains a robust cash reserve of $268 million.

Financial Results

• Q1 2025 Net Revenue: $137.8 million, a 75% increase from the previous year.
• 2025 Net Revenue Guidance: Adjusted to $590 million-$605 million.
• Profitability: Achieved in Q1 2025, driven by Arcalist sales.
• Cash Reserves: $268 million at the end of Q1 2025.

Operational Updates

• Arcalist Revenue: Nearly $1 billion since launch.
• Prescriber Base: Over 3,150 unique prescribers, with 26% prescribing to multiple patients.
• Treatment Duration: Average increased from 27 to 30 months.
• Payer Approval Rates: Exceed 90%.

Future Outlook

• Market Penetration: Focus on the 14,000 multi-recurrence patient population.
• Growth Strategy: Promote Arcalist for all recurrence levels and ensure positive prescriber experiences.
• Clinical Development: Advance KPL-387 with a Phase 2/3 trial starting mid-2025 and data readout in 2026.

Q&A Highlights

• Market Expansion: Connexa targets broader patient education and awareness.
• Arcalist Eligibility: No restrictions based on the number of recurrences.
• Cash Flow: Management aims for annual positive cash flow.

Readers are encouraged to refer to the full transcript for more detailed insights.

Full transcript - Jefferies Global Healthcare Conference 2025:

Roger Sung, Senior Analyst, Jefferies: Alright. Okay. Good afternoon. Welcome to twenty twenty five Jefferies Global Healthcare Conference. My name is Roger Sung, one of the seniors cover SimiCA Biotech in The US.

It is my pleasure to introduce our next printing company, Connexa, and then we have the chief commercial chief commercial officer Ross and then chief medical officer John. So, they will do a normal corporate presentation and then we’ll save some q and a toward the end. Welcome.

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: Okay. Thank you very much, Roger, and thanks to the Jefferies team for hosting us here today. It’s a pleasure to be here. My name is Ross Motes. I’m the Chief Commercial Officer at Knixa Pharmaceuticals.

And today, I will be providing both a corporate and a commercial update on Kynixa. I’m joined here by my good colleague, Doctor. John Paolini, our Chief Medical Officer, who will be providing a review of our clinical trial design for KPL-three eighty seven, an exciting potential new development and a treatment option for recurrent pericarditis. Today, we will be providing forward looking statements that are subject to risks and uncertainties, a full copy of which can be found either on this slide or under SEC filings on our corporate website. Kynixa is a well capitalized, growth orientated company, putting patients at the center of everything that we do.

We are aiming to address unmet needs by advancing novel therapies, particularly within cardiovascular indications. With Arcelis, we have established our leadership in the recurrent pericarditis market. And in Q1, at our earnings call, we were pleased to provide an increase to our net revenue guidance for 2025 of between $590,000,000 and $6.00 $5,000,000 We are also focused on advancing our clinical portfolio by developing KPL-three eighty seven in recurrent pericarditis. And to that regard, our Phase twothree clinical design is expected to initiate in the mid of twenty twenty five, with data readout from the Phase II portion of the study in the second half of twenty twenty six. Additionally, we are focused on maintaining a strong financial position.

In Q1 of twenty twenty five, with our Arcelis revenue, we achieved profitability for the organization and that added to our cash reserves, taking us to $268,000,000 which is important as it provides us optionality to invest in further value creation activities across the organization. Kynixa has an innovative portfolio of both commercial and clinical stage assets. You may know that Arclis has been approved since 2021 in recurrent pericarditis and is also approved in two other rare conditions, in caps and in an ultra rare condition called dera. And today, John will talk us through the KPL-three 87 clinical trial design in recurrent pericarditis. But before he does, I’ll provide an update on the commercial side of the business with ArcList.

Our team has driven robust growth And we’ve delivered almost $1,000,000,000 in the four years since our launch as net revenue. In fact, in Q1 of twenty twenty five, we delivered $137,800,000 which is a 75% year over year increase. The Q1 growth was driven by increases to both the active commercial patients on therapy as well as longer durations while on therapy. And the active commercial patients was driven by an increase to the prescriber base particularly from new prescribers where in Q1 we had around 300 new prescribers of Arcanist in recurrent pericarditis. In fact, that’s one of the largest growths that we’ve had in any quarter since our launch.

And that took our total prescribers to more than 3,150 unique individual prescribers. Of that group, around twenty six percent or eight twenty physicians have prescribed for two or more patients. As I mentioned, the average total duration of therapy has also continued to grow since the time of our launch, most recently having gone from around twenty seven months to now around thirty months of an average treatment duration, which underscores a growing understanding that recurrent pericarditis is a chronic multi year disease in most patients. Additionally, the other underlying commercial fundamentals also remain strong, whether that’s the payer approval rates or patients compliance to therapy. Our strong commercial performance in Q1 enabled us to increase our net revenue guidance for 2025 to between $590,000,000 and $6.00 $5,000,000 And our strategy is focused on continuing to drive future growth, whether that be through promoting to the breadth of the label, which is agnostic to the number of recurrences a patient has to have suffered before being treated with Arcalist.

And in fact, around fifteen percent of all the patients prescribed Arcalist are treated when they’re on their first recurrence of disease with around eighty five percent being on two or more recurrences. It’s also crucial for us to continue to ensure physicians have a positive prescribing experience when prescribing ArcLIST. That means everything from knowing how to prescribe the drug, how to go through the prior authorization request, seeing their patient get access to therapy, and then also witnessing the effects while being on therapy aligned with what we saw in our clinical trials, which is a highly efficacious and well tolerated drug. So we’re pleased with what we’ve achieved so far since our launch over the last four years. However, our Kynixa team is even more excited about the future.

In fact, at the end of twenty twenty four, we had reached around thirteen percent penetration into the multiple recurrence target population. That’s the patients that have two or more recurrences in any given year. So that speaks to the opportunity that we have ahead and our team are very excited about reaching more and more patients and helping them during the suffering from this debilitating disease. With that, I’d like to hand over to Doctor. John Paolini to provide an update on KPL-three eighty seven and the exciting potential new treatment option in recurrent perikilitis.

John?

John Paolini, Chief Medical Officer, Kynixa Pharmaceuticals: Thanks, Ross. And as you can see, Ross and the entire commercial team have really performed exceptionally, placing the company on a robust trajectory and expanding our reach to help ever increasing numbers of patients with recurrent pericarditis. Of course, we intend to maintain and extend our leadership in this space, building on that success. And so our commitment to patients with this debilitating disease extends also to our pipeline development plans. Earlier this year, we announced a new program, KPL-three 87, our independently developed monoclonal antibody targeting the IL-one signaling pathway.

Today, we would like to share additional details of this program as it continues in its progress. On this slide, you can see some of the top line data from the single ascending dose study, the single subcutaneous administration portion of that phase one study. This is a randomized, double blind, placebo controlled study, SAD and MED, in approximately one hundred and twelve healthy participants. And it evaluated the safety, tolerability, PK, PD, and immunogenicity of KPL-three eighty seven. These data show that a single dose of KPL-three eighty seven at the three hundred milligram sub q dose level, which is shown in red, demonstrated sustained serum concentrations above the target concentration.

And that supports the profile of a monthly dosing paradigm. Data from this study were utilized to support the design of the upcoming phase twothree clinical trial in recurrent pericarditis. And that’s the slide that’s shown here. Next slide. There we go.

The phase twothree clinical trial will consist of three overlapping parts, which have been combined into a single protocol. The phase two dose focusing portion, the phase three pivotal portion, and two long term extensions. The dose focusing portion of the study will enroll up to 80 patients randomized equally across four blinded dose levels to receive subcutaneously administered KPL-three eighty seven for twenty four weeks. Concomitant treatment with conventional oral therapies will then be weaned off to attain KPL-three eighty seven monotherapy. The primary efficacy endpoint is time to treatment response.

Patients completing the dose focusing portion would then be eligible to enter a two year long term extension to provide for additional exposures and additional treatment duration. Following the dose focusing portion, the pivotal trial will begin. Up to 85 participants would be enrolled in a single blind run-in period, during which KPL-three eighty seven would be initiated and conventional oral therapies will again be weaned. Participants will be blinded to the duration of the run-in period. Subsequently, in the randomized withdrawal period, participants who respond to KPL-three eighty seven in the run-in period will then receive either continued KPL-three eighty seven or a placebo.

This is an event driven randomized withdrawal study, with a primary efficacy endpoint of time to first adjudicated pericarditis recurrence. And upon closure of the randomized withdrawal period, participants may be eligible to participate in the long term extension. And as always, clinicaltrials.gov is a helpful resource for additional information and updates regarding our clinical programs. Now, as Sandra mentioned in our previous meetings, our goal is to put this product into the hands of our commercial team as quickly as humanly possible, and to bring this potential new treatment option to patients as soon as the twenty twenty eight-twenty twenty nine timeframe. And so with that, I’ll turn it over to Ross for closing remarks.

Ross?

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: Thank you, John. So in summary, Kniksa is well positioned for both future success and value generation. You’ve heard from us today that we continue to be acutely focused on the Arcelis commercial performance, and we were pleased to provide the net revenue guidance for this year of between $590,000,000 and $6.00 $5,000,000 We are also advancing at pace with our clinical portfolio, as you heard from John, for KPL-three eighty seven expected to start in mid-twenty twenty five. And we are also incredibly focused on maintaining a strong financial profile with a growing cash balance, but optionality to invest in future value creation activities, whether that be via internal development or external business development. The team at Knixa could not be more excited about the future that we have ahead.

We’d like to thank you very much for those of you in the room today and listening online and hand back to Roger to moderate the Q and A session. Thank you.

Roger Sung, Senior Analyst, Jefferies: Thank you, Ross. Thank you, John. Yeah. That’s a very comprehensive overview of the company. And then, yes, it’s very exciting journey for Connexa for the RP and then moving forward.

So maybe we stick on the the commercial side, understanding the right now, the key focus is the multi recurrence targeted population, and then the current penetration rate is about 13%. And then this is not like a real time, but, you know, kind of based on your market research. And then how should we think about the overall you know, what what is the the real potential for alkalis to penetrate into that targeted population? And another interesting question is those are the multi recurrent at any given year. If they are not using alkalis, what they are using?

And then what they are waiting for not to use the acrylics?

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: Yes. Great. Thank you for those questions, Roger. Yes. I think the best way of summing up is that we’re very excited and focused on the future.

We think we have a huge potential left ahead, having been only thirteen percent penetrated into the 14,000 patient population. And you’re right that we are focused on that fourteen thousand population, the two or more recurrences, but we are also promoting to the breadth of the label, which is the whole 40,000 patient population. And I think as physicians become more and more ingrained with Arcalist and understand how to prescribe the drug and they witness the benefits that their patient sees by being on treatment, We think that creates a positive halo effect whether that’s for individual prescribers to prescribe more when they identify their next patients or through peer to peer education and encouraging other prescribers to diagnose and prescribe Arcalis for their recurrent pericarditis patients. So we think all those things are going very well. A lot of it is just down to education and awareness and the fact that there are many patients out there broadly spread around The US, being looked after by many different cardiologists, and everyone having a different experience.

I think what you’ve seen is one by one we’re switching on more and more physicians to this new wave, a new way of how to treat the disease. People are becoming more aware that recurrent pericarditis is driven by interleukin-one alpha and beta, and that Arclis is a targeted therapy for those cytokines and a targeted immunomodulator for them with the tremendous results that we’ve seen under the clinical trials. So I think that all bodes well for the future. To your point about what are they waiting for, we think it’s more education, more knowledge, more experiences in the community. There are a growing number of centres that are taking more and more of an interest now that there is a treatment available in pericardial diseases per se and kind of build in more centers of excellence across the country.

And we think that’s very positive for the patient community to try to get a timely accurate diagnosis and access through to targeted therapy when that’s appropriate for the patients. Got it.

Roger Sung, Senior Analyst, Jefferies: Yeah. I know you are you don’t want to guide to the peak penetration rate or what’s the future look like. Just out of curiosity, who are the patient population you think is not going to be addressed by the alkalist in terms of the multi recurrent population? We’re gonna talk about the first recurrent in a minute. But just, you know, based on the profile and based on the feedback you are getting from physician, do you think it really have a certain patient with multiple recurrence did not eligible or should not, you know, they’re not going to be using the alkalis there anyway?

Now, even with more education.

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: Yeah. It’s a great question. I mean, I think the short answer is really not that we’ve seen so far. Obviously it’s up to individual physicians to decide what the right treatment path is for their patients and having a robust understanding of the patient’s history and any underlying etiologies or complications that the patient may have. But the ArcLIS label is very broad.

As I said earlier, agnostic to the number of flares and also agnostic to any other prior therapies that a patient has been on to try to control their disease. What we know is when patients have more and more flares, it precipitates longer disease and higher burden of the disease. So the earlier these appropriate patients can get diagnosed and treated appropriately, the better for the patients and for their outcome and for their overall quality of life.

Roger Sung, Senior Analyst, Jefferies: Got it. Okay. Yeah. That’s a very good way to frame this. And then, in terms of first recurrence, that’s still not the current focus, but seems you are getting more tractions than expected for the first recurrence population.

And just in terms of the the practice experience, do you see the difference between difference between the first recurrence patient versus multiple recurrence patients using alkalis? And then, if so, how you think alkalis can better serve to the first recurrent patient with some, you know, different strategy or anything?

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: Yeah. So, haven’t seen any differences that we’ve observed so far. And John can certainly speak around the resonance registry that we have to track real world experience of the disease, which has come out with multiple data points that are interesting for the future to really understand this disease in the real world setting. So other than that, no major differences between different patient populations what we’ve seen. But we think it makes sense for physicians to when a drug first comes to the market and when you’re really focusing on a paradigm shift and moving people away from previously having reached for corticosteroids because it was pretty much the only other available option after NSAIDs or colchicine have been utilized within these patients.

It takes time to just create that new way of treating the disease. And I think it stands to reason that physicians often wait for the ideal type of patient, maybe someone that’s had multiple recurrences, and they start there. And to your point, over time we’ve seen growth of the number of patients on the first recurrence of the disease, which I think speaks to that positive feedback loop, how physicians are getting on, how the treatment paradigm is really changing and Arclis is being seen as a steroid sparing therapy for patients. And I think that all bodes very well for the future. Got it.

Okay. And then, how do you

Roger Sung, Senior Analyst, Jefferies: think about the payer side? I know the label is covering the entire RP. Do you see the difference on the reimbursement side for the first recurrence versus multiple recurrence?

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: Yeah, generally not as a mainstay across the payer landscape. We have very good payer coverage and that’s greater than 90%. And in fact, that’s been the case ever since the time of launch, even when patients and physicians had to go through medical exceptions before policies were actually put into place. And now of course payers policies have been in place for multiple years and have been iterated upon over time. So, generally speaking, we’ve got a greater than 90% payer approval rate.

We feel strongly that payers really understand the value proposition that Arcelis brings to them and their patients and how it helps patients.

Roger Sung, Senior Analyst, Jefferies: And then, how about the duration on treatments? I know you probably have limited data for first line, first recurrent versus multiple recurrence. But, in terms of the patient, maybe they are less severe, they will be on treatment a bit shorter. So, how should we think about the duration of treatment between those two populations?

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: Yeah, think some of that is certainly borne out in the literature showing that the more recurrences a patient has had, the more likely they are to have longer duration of disease. In patients that have two or more recurrences, the median duration of the disease is around three years and around a third of the patients are still suffering from recurrent pericarditis five years after the original index episode of the disease and around a quarter of the patients still at eight years. So we’ve been pleased to see that Arclis’ duration of therapy has been growing since the time of launch, most recently as an average total duration of around thirty months. And I think that speaks to, one, how patients are getting on therapy two, how physicians are understanding that this is a long term, chronic, multiyear disease in the vast majority of patients. And ultimately that’s how Arcalis was also designed, to be used throughout the natural history of the disease to control recurrent pericarditis, reduce the symptoms associated with the disease, and ultimately to prevent future recurrences.

And I think that’s exactly what we’re seeing, whether that’s in the real world evidence through the resonance data sets or if you look back at the clinical trials.

Roger Sung, Senior Analyst, Jefferies: Just to clarify, for that duration of treatment, that’s include both the first recurrence and the multiple recurrence? That’s also just multiple?

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: That’s correct. So that’s the aggregated number across all recurrence of patients. And it’s the treatment time. So there are patients when they stop therapy. We see around forty five percent of patients who come back onto therapy generally within around an eight week time period for most patients and symptomology returns if the underlying auto information is not resolved.

And so if you add up all of the treatment periods from what we’ve seen so far, that’s what adds up to around thirty months on average across all the cohorts.

Roger Sung, Senior Analyst, Jefferies: Yeah, across all the cohort. Do you see the difference between the cohort in terms of the duration on treatment? That’s aggregated, you know, duration on treatment.

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: Yeah. And that we’ve got robust data to share or provide in that regard. And some of that comes down to the fact that actually recording the number of flares a patient has had is also very difficult. If you ask a patient, they will tell you one thing. If you ask a physician, they will tell you something else for that same patient.

And sometimes there’s not a standard way of really understanding how many recurrences. So it’s a little bit tricky to get really robust data in that regard.

Roger Sung, Senior Analyst, Jefferies: Got it. Yeah. I know every time I’d put you on the spotlight that and I wanna talk you know, ask a couple questions to join the world. So thanks for for us. And then this is a, you know, a very, very important development for the next pipeline for the monthly dosing for I l r one three eighty seven.

So, the phase two, phase three seems they are very seamlessly, you know, switching or transition from phase two to phase three. But my question is, how are you gonna decide determine the dose from phase two, four doses to the phase three dose? And maybe if I’m reading this correctly, it’s one dose for phase three. And then are you powered to see the difference in phase two in order to make decision for phase three dose?

John Paolini, Chief Medical Officer, Kynixa Pharmaceuticals: Right. No. That’s a great question. And so, you know, kind of on first principles, it’s important to lay out that, you know, this part this is obviously a mechanism that, at Kinecta we know a lot about, and have a lot of experience with. And of course, we built upon our experience with, you know, with Rhapsody, you know, as a highly successful clinical trial, you know, in phase three, supporting you know, supporting ArcLIST.

And so we have a lot of knowledge of this pathway, and so that and how to kind of run these trials and extract useful information from them. So what we’ve put forward in terms of this package, if you will, is, you know, a phase two dose focusing portion of the study, and then a phase three pivotal portion of the study, two long term extensions, and they’re kind of interconnected, if you will, and overlapping, but they’re done as a single integrated protocol. And what that does is that allows us to move this program forward, you know, very quickly, which is why we say that this dose focusing portion starts in the mid year this year, in terms of how we then use that information. So these are all actively flaring patients who are coming in despite their background therapies, and KPL-three eighty seven is layered on, and the primary efficacy endpoint is certainly tied to treatment response. But there’s a wealth of information that you gather across these four different dose levels that help us understand if you anchor on the monthly dose, you know, is the month first principles, is the monthly dose sufficient?

Then if you give more drug, do you get additional efficacy, or in fact, is that sufficient? And then if you remove or give less drug, do you end up with certain weaknesses in certain of the endpoints that then help you understand or build the case to the agency that you have a minimally effective dose level? So once you’ve done that, you know, we will use the totality of that information going forward. I think that’s what we can say about that. What I will also say is that once we’ve kind of completed what we’ve needed to learn from that phase two portion, we can then start the phase three portion of the trial, even though, you know, there may be, you know, the phase two study will continue into the long term extension, you know, the dose focusing, you know, portion will continue into the long term extension.

And so, that concurrent activity is, again, a way to pick up speed. But the idea is to take forward one dose level into the pivotal program, and then that is what would move forward to registration.

Roger Sung, Senior Analyst, Jefferies: Got it. And then given this is, you know, based on totality of the data, we should not just expect you will have to see statistical difference among those four doses in order to make the phase three decision. Is that right?

John Paolini, Chief Medical Officer, Kynixa Pharmaceuticals: Yeah, no, that’s a great question. So, you by speaking to the totality of the data, if you will, the purpose of the study is to collect information at these different dose levels such that we can build our PKPD understanding and explain that to the agency. There’s no necessarily implied comparison across or explicit comparison across the different dose levels, but rather it’s more, as I mentioned, understanding that if you anchor on the concept of monthly dosing, then you ask the question, if you give more drug than, you know, what is on that anchor dose? If you do not see additional efficacy, and if you see less efficacy if you get underneath that’s the critical element. What you see in our PKPD modeling, which is on our corporate website, is that, you know, from that target concentration, we actually cover orders of magnitude above and below that target concentration, which allows us to generate an enormous amount of information.

Roger Sung, Senior Analyst, Jefferies: Excellent. Great. And then, just one last minute in terms of the financial position and then where you are in terms of the bottom line and then you give us the revenue guidance. I believe you have some operating OpEx guidance as well. So just what’s the financial position and then you can let us know.

Ross Motes, Chief Commercial Officer, Kynixa Pharmaceuticals: Yes. So we thank you, Roger. We’ve just shared that we aim to be cash flow positive on an annual basis. As I mentioned in Q1 of this year, we were profitable, thanks to the growing revenues of ArcList. We have cash reserves as of the end of Q1 of around $268,000,000 And we believe that that helps to provide us optionality for future investments.

We’re an organization that is very focused on growth and the ability to invest in future value generation.

Roger Sung, Senior Analyst, Jefferies: Excellent. All right. Thank you for being with us this afternoon and thank you everyone for listening. Thank you. Thank you so much.

Thank you.

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