Talphera (TLPH) 2026年第二季度业绩电话会：NEPHRO CRRT入组率达到75%

核心要点

• Talphera (TLPH) 表示，注册性 NEPHRO CRRT 研究的患者入组率已达到 75%，且所有最终临床中心均已启动。管理层仍按计划将于 2026 年晚些时候完成入组。
• 预计在入组完成后约四周至五周公布顶线数据。公司的目标是在 2027 年上半年提交上市前批准（PMA）申请。
• 截至 2026 年 6 月 30 日，现金总额为 1710 万美元。Talphera 表示，这一余额加上未来的条件性分期融资，应能支持运营直至至少预计于 2027 年获得潜在批准。
• 如果满足相关条件，两笔条件性分期融资可提供约 1600 万美元的追加资金。
• 最新的市场调研预计，2027 年美国连续性肾脏替代治疗（CRRT）例数约为 20 万例，比此前预估的 16.5 万例高出 21%。约 70 家机构可能占到这些治疗例数的一半以上。
• KDIGO 2026 年指南草案提到了萘莫司他（nafamostat）可作为枸橼酸盐的局部抗凝替代方案。在 5 月份公众征求意见结束后，最终版本的发布仍在等待中。

关键财务数据

业务与运营表现

NEPHRO CRRT 研究旨在评估萘莫司他在连续性肾脏替代治疗中是否为安全有效的抗凝剂。Talphera 已完成了试验中心的调整，现由肾脏科医生担任主要研究者，招募工作主要集中在内科重症监护室。

管理层表示，研究人员认为给药、剂量滴定及监测流程简单明了。然而，该试验仍处于双盲状态，管理层强调最终结论将取决于揭盲后的结果。

根据电话会议上讨论的双盲运营观察数据，在方案规定的起始输注速率下，约 80% 至 85% 的患者处于治疗有效范围内。几乎所有患者都在约 30 分钟内达到该范围，利用床旁 ACT 检测每 15 分钟可进行一次剂量滴定。据报道，大多数患者的治疗剂量为每小时 20 至 25 毫克，低于许可的最大剂量每小时 50 毫克。

该公司还强调了《KDIGO 2026年急性肾损伤和急性肾脏病临床实践指南（草案）》。管理层表示，如果产品获得批准，萘莫司他的纳入将有助于支持医生教育和医疗机构临床方案的采纳。

Talphera 更新后的调研预测，2027 年美国 CRRT 例数约为 20 万例，而此前预估为 16.5 万例。管理层表示市场呈现集中态势，约 70 家机构占治疗例数的 50% 以上。这种集中度支持以客户经理、教育人员、培训人员和同行教育为核心的针对性商业化模式，而非传统的广泛销售团队模式。

管理层指引

管理层预计将于 2026 年晚些时候完成 NEPHRO CRRT 的入组，并在约四至五周后公布顶线数据。随后，Talphera 预计还需要大约三个月的准备工作，然后提交 PMA，目标是在 2027 年上半年提交。

公司计划在提交 PMA 后开始有限度的战略性招聘和上市前医学教育。如果获得批准，管理层目前的目标是在获批时立即推向市场，无实质性延误。

Talphera 还指出，CorMedix 在数据公布后拥有为期 60 天的首要谈判权。公司正在同步为独立上市做准备，并评估潜在的战略替代方案。

风险与关注焦点

• NEPHRO CRRT 仍处于双盲状态，因此关于给药、剂量滴定和研究者经验的观察并不能确立不同治疗组的疗效或安全性。
• PMA 时间表及 2027 年的潜在批准仍取决于研究结果、申报准备和监管审查。
• 剩余的两笔分期融资为条件性融资，只有在满足条件的情况下，才可能在顶线数据发布和研究完成前后完成交割。
• KDIGO 2026 年指南仍等待最终发布，管理层承认此类指南在美国医疗机构和临床医生之间的采纳程度存在差异。
• 市场预测和机构集中度数据基于更新后的研究，公司计划在未来的投资者活动中对其进行验证并呈报更多细节。

分析师问答亮点

管理层表示，商业化准备初期仅需招聘少量人员，专注于医学教育、萘莫司他的临床背景及 KDIGO 指南。产品上市策略预计将优先考虑 CRRT 治疗量较大的中心。

首席医疗官 Shakil Aslam 表示，研究人员未发现意外的运营问题。独立数据安全监测会议在未发现新的患者风险或异常情况后，建议研究无需修改继续进行。

在定位方面，管理层表示，KDIGO 指南草案将萘莫司他视为枸橼酸盐不可用或禁忌时的替代方案。公司认为，易用性和更可预测的剂量滴定可能会促进其采纳，若获批，甚至有可能从补救或二线用药向一线用药拓展。

Talphera 计划在未来举办一场投资者与分析师活动，重点探讨市场规模、增长、细分及商业化。公司表示，其研究包括数据分析、30 多位医生的反馈以及护士关于 CRRT 相关 ICU 工作量的定性意见。

业绩电话会议完整转录

完整财报电话会议逐字稿

管理层陈述

Unknown Speaker

Thank you.

Operator

Welcome to the Telfero Second Quarter 2026 Financial Results Conference Call. This call is being broadcast live via the events page of the Investors Section of Telferra's website at www.telferra.com. listen to a replay of this webcast by going to the investor section of telpara's website would now like to turn a call over to Rafi Asfordian, El Perro's Chief Financial Officer.

Raffi Asadorian

Thank you for joining us on the call today. Today we announced our second quarter, 2026 financial results and associated business updates in a press release. With me today are Vince Angotti, our Chief Executive Officer, and Dr. Shaquille Aslam, Telferra's Chief Medical Officer. Before we begin, I want to remind listeners that during this call, we will likely make forward-looking statements within the meaning of the federal securities laws. These forward-looking statements involve risks and uncertainties regarding the operations and future results of Telfera. Please refer to our press release in addition to the company's periodic, current, and annual reports filed with the SEC for a discussion of the risks associated with such forward-looking statements.

These documents can also be found on our website within the Investors section.

Vincent Angotti

I'll now hand the call over to Vince. Thanks, Rafi. Good afternoon, and thank you to everyone joining our call today. We remain on track to complete enrollment in the NEFRO CRRT study later this year. This registrational trial is designed to evaluate whether Nefamistat is a safe and effective anticoagulant for use during continuous renal replacement therapy, or CRRT. The study is being conducted in hospital ICUs with nephrologists leading as principal investigators. With all of our final clinical sites now activated, we have reached 75% enrollment in the NEFRO CRRT study. This progress allows us to increasingly turn our attention to the commercial aspects of the famostat.

We remain especially pleased with the high level of engagement from our principal investigators and study site personnel. Today, we'll share some details on these areas, along with some initial insights from our updated market research on the FAMISTAT and the CRRT space. On the commercial front, clinical guidelines are an important part of the treatment landscape and were encouraged by recent developments. Specifically, the KDGO 2026 Clinical Practice Guideline for Acute Kidney Injury and Acute Kidney Disease is current. pending final publication following the close of its public comment period in May. KDGO, which stands for Kidney Disease Improving Global Outcomes, is the global nonprofit organization that develops and implements evidence-based clinical practice guidelines in kidney disease. Of note, the guideline now references Nifamistat as an acceptable regional anticoagulant. The only available regional anticoagulant being used in the U.S. today is Citrate, yet regional Citrate is not FDA approved for CRRT, and it's also complex to use and has other As a result, most U.S. sites simply don't incorporate it into their CRRT protocols.

This is the first time the FAMISTAT has been recommended in the CADIGO guidelines, supported by historical studies and publications, a positive change from prior published recommendations. KDGO guidelines are widely referenced by healthcare providers internationally. We believe this guideline will provide momentum for the commercialization of Nefamistat if approved. This new guidance should further highlight to healthcare professionals that Nafamistat is already standard medical practice in Japan and South Korea, where it is is the most widely used method of anticoagulation during CRRT. A 2018 national survey in Japan found that Nefamistat was used in about 80% of cases, making it the most common method of circuit anticoagulation. In South Korea, the famistat was approved for this use in 2005 and remains one of the most widely used anticoagulation methods today. In addition, our updated market research on the CRRT space points to a larger market opportunity for the FAMSTAT than initially estimated.

This research puts the estimated annual CRRT procedures in 2027, the projected timing of an ephemostat launch, at approximately 200,000 in the United States. That's a 21% increase from a prior estimate of 165,000, driven by new data from this research. We also expect a number of CRRT procedures to continue growing annually. We believe the Famistat, if approved, can address an unmet need in the market given the disadvantages of systemic heparin and regional citrate. Addressing that unmet need provides the famostat with an opportunity to gain a meaningful share of the CRRT anticoagulant market. These market insights are consistent with what we're hearing directly from principal investigators and other physicians about the currently available CRRT anticoagulants. We'll share the complete findings later this year at another investor and analyst event as we get closer to completing enrollment.

Now I'll turn the call over to Dr. Aslam, our Chief Medical Officer, for some comments on the study. Thank you. Shaquille?.

Shakil Aslam

Yes, thanks Vince and good afternoon everyone. I am very pleased with the progress we are seeing in the nephro CRRT study. as we move toward completing enrollment later this year. We have completed the realignment of sites to match our target profile. Now all sites have nephrologists as the principal investigators and are recruiting patients primarily from the medical ICUs. Finalizing our study sites coupled with protocol modifications granted by the FDA has us on track to complete enrollment later this year. As a reminder, nephroCRRT is a placebo-controlled study in which administration, titration, and monitoring of anticoagulation are identical in both treatment arms. With 75% of patients enrolled, our investigators have consistently reported that these procedures are straightforward to perform.

Investigators continue to express their excitement over having Nefamostat available for their use, if approved. and its inclusion in the public review draft of 2026 KDGO guideline as a regional anticoagulant alternative to citrate. The International Expert Panel's inclusion of nifamistat reflects decades of clinical experience and accumulated published evidence on the safety and efficacy of nifamistat as a regional anticoagulant during CRRT. and we look forward to sharing the results with you. With that, I'll hand the call over to Ravi to update you on the financial results for the second quarter.

Raffi Asadorian

Thanks, Shaquille. Our cash balance at June 30, 2026 was $17.1 million. We believe this cash, combined with future conditional financing tranches, will provide us sufficient capital through at least a potential NIAID PMA approval expected in 2027. Two conditional financing tranches remain, totaling approximately $16 million of additional capital. If the conditions are met, we expect these to close around the time we release our top-line data and announce the completion of the study. Our cash operating expenses, or combined R&D and SG&A expenses, for the second quarter of 2026 totaled $3.9 million compared to $3.7 million for the second quarter of 2025. excluding non-cash stock-based compensation expense, these amounts were $3.7 million for the second quarter of 2026 compared to $3.5 million for the second quarter of 2025. The $0.2 million increase in cash operating expenses in the second quarter of 2026 is primarily due to higher NIAID development expenses reflecting increased enrollment and certain SG&A expenses relating to initial market research activities. I'll now hand the call over to Vince.

Vincent Angotti

Thank you, Rafi. And thank you for joining our second quarter earnings call. With enrollment progressing well, we remain on track to complete enrollment in the NEPHRO Registrational Study later this year and to report top-line data soon thereafter. Our focus remains on bringing the Phamistat to the market as a new regional anticoagulant for CRRT, if approved. and I'd like to open the line up for any questions you might have. Operator?.

Operator

Ladies and gentlemen, we will now begin the question and answer session. If you have a question, please press the star followed by the one in the touchdown sound. Should you wish to cancel your request, please press the star followed by the two. using a speakerphone, please flip the answer before pressing any keys. Once again, that is star 1. Could you wish to ask a question? And your first question is from James Malloy from AGP Global Partners. Your line is now open.

分析师问答

Matthew Venezia

Hey guys, thanks for taking our questions. It's Matt on for Jim today. So as you guys are gearing up toward potential commercial launch here, When would we start to see maybe some strategic hires? And could you take us through a little bit of what the strategy might look like for a rollout, like centers of excellence that you guys may target, high enrolling sites that might want to come right on board, anything like that?.

Vincent Angotti

color would be helpful. Thanks. Sure. Thanks, Matt. Appreciate it. I think a couple of things that are important. I want to reiterate the fact that we do have a strategic potential with CoreMedix who has a right of first negotiation for 60 days post data readout. And as a reminder, the CEO of CoreMedix, Joe Tedisco, he is on our website. board and obviously knows everything going on with the company. So that's one consideration. With that as a backdrop, we are preparing for launch on our own, as well as any other strategic considerations that might happen. And more specific to the launch, we'll likely start the hiring once the PMA is submitted. We plan that for early next year.

First quarter is our goal. And with that PMA submission, we would likely start to hire and launch our pre-launch planning in our medical education. So it won't be significant number of hires, just right now as a preliminary plan, just a couple of hires really about education on the FAMAS status background, the KDGO guidelines, et cetera. As a strategy for rollout in general, as you mentioned, if you recall, this market is highly concentrated. At least that's what the data suggests to date, and we'll be doing some additional targeting and segmentation work moving forward. But those 200,000 procedures that we mentioned in today's call based off of the most recent data we have and will validate moving further, about 70 institutions in the country, as far as we can tell, represent over 50% of those procedures. So from a rollout standpoint, we believe it will be highly targeted and concentrated and likely, at least today, not your traditional CISO. sales representative rollout. It'd be more account managers and educators and trainers. and a lot of peer-to-peer education moving forward.

So again, we believe it will be highly concentrated. I think a big focus for us will be on the pre-launch activities in medical education. And that'll coincide once the PMA submitted next year. And Matt, just to correct you, just to...

Matthew Venezia

be clear on the PMA filing, not first quarter, first half is what it's supposed to be. First half, yes. Yes. Got you. All right. Thanks, guys. Thanks for the callers. And thanks for taking our questions today.

Operator

Of course. Thank you and your next question is from Ed Arcee from West Park Capital. Your line is now open.

Unknown Speaker

Hi guys, thanks for taking my questions and congrats on the continued progress with the trial. A couple of questions for me. I just wanted to ask as we are approaching full enrollment here. I wanted to ask if you could run through again, just to be clear on the timeline enrollment to the data collection to the extent that there is much after that and then top line readout and the interim between that and what's necessary to submit your PMA, as you mentioned, in the first half of next year. And then separately from the timeline, I also wanted to ask if you have heard any information know, anything of note, anything particularly interesting in terms of feedback from these nephrologist PIs? You mentioned that the... The feeling has been that the procedure remains quite straightforward. You know, that's one aspect of this that's been repeated, I think, before.

But are there any other aspects of this as, you know, as all the sites now are up and running? Thanks.

Vincent Angotti

Thanks, Ed, for the question. Raf, I'll refer to you for the timelines, and then we'll move to Shaquille for the nephrology feedback.

Raffi Asadorian

relative to our PIs? Yes, so to get the data, it's going to be about four weeks after we complete enrollment. around that time, four or five weeks, something like that to get to the top line data. We're working to get that on the sooner end of that, but that's probably around the timing. And then to get to a PMA filing, we may be requesting an interim meeting just to make sure everything is clear and everything, but we're probably... probably talking another couple months or so, probably another three months or so to get to that PMA filing, which will put us inside the, should put us inside that first half of now. next year. Does that answer your question? Yes. I guess related to that. Obviously, there's the PMA approval.

Unknown Speaker

that's expected sometime around the middle of the year. But is there any... necessary activities between approval and full launch. any color on the timeline there as well. Yes, I would suggest. Okay.

Vincent Angotti

I would suggest likely not, Ed. If you're asking between approval and launch, our goal would be to launch at approval. And part of that would be what I'll call market conditioning and sensitization to nifamistatin, its availability in the medical education. I don't suggest there be much of a delay between approval and a launch, at least how we're planning it today. I think the second portion of your question was feedback from the nephrology PIs. Shaquille, you want to comment on that? And I think one other aspect outside of what we said today, Shaquille, if you wouldn't mind mentioning how, at least based off of the early feedback as well, they're using other agents off in the second line. They might move the FAMISTAT the first line when and if it's approved.

Shakil Aslam

Right. So, just remember that this is a blinded study, so there is no feedback that really pertains to one treatment arm versus the other. But overall, there have not been any surprises in the study. And their experience is exactly as they were expecting. Monitoring is extremely straightforward, simple, and very predictable. I think that's two important things in this patient population that your response should be consistent and predictable. And so far, that's the feedback we have gotten. We have had our first independent data safety monitoring meeting a couple of months ago, and we They did not identify any risks to the patient or any new findings.

So they essentially recommended to continue the study without any changes. So that's also very reassuring that everything is going as planned. I can tell you that people who have used citrate or clinicians who were looking into using citrate, many of them have informed us, gee, I should just wait for this to become available. Why invest all these resources? Because this is just going to be so much simpler and easier to use citrate. without any special training required, is essentially the use is just very, very similar to haprine, except that it's just more predictable and more consistent in terms of the way the anticoagulation goes in these patients. So I hope that answers your question. There have been no surprises and everybody remains. I talk to PIs almost on a weekly basis, and they all remain very excited.

And many of them have told me that they cannot wait to get their hands on it. So that's, I think, it's all very encouraging and quite as expected. Yes, that's great.

Unknown Speaker

Thanks, Dr. Asim. I appreciate that. Uh, if I, if I may, I just want to ask one quick, uh, question about the investor event planned. Is there any further details at this point that you could share? Thanks.

Raffi Asadorian

No, not yet. I mean, it's going to be focused on the market, the market opportunity, and our commercial approach to the market. And we'll be providing more details on all the updated market research that we've been going through. that's going to be a little closer to the time where we're imminently completed with the study.

Vincent Angotti

Just to give you a little more color, Ed, on Rob's comment, so we completed this in-depth market research with a data analysis provider on market sizing, growth rates. and stratification within it, meaning what are some of the most related to CRT. An example would be sepsis and then some others. Beyond that, what I'll call secondary market research, we have just recently completed primary market research with over 30 physicians on both the quant and qual subjects. format. It was fully chaperoned as opposed to more of a survey, really getting insightful. readouts from them on their feeling about today's current products, the target product profile of a new entrant, for instance, the FAMISTAT, how that might affect their use of products moving forward. Does it shift them from second-line therapies as rescue with coronary anticoagulants to potentially a proactive first-line therapy use with a new entrant like nifamistat based off that TPP, etc.? We've also done some recent research with some nurses to add some more qualitative feedback on just the burden that they have in the ICU. in the often one-to-one ratios of workload once a patient goes on CRRT, especially involved with citrate. So we're looking to finish the synthesis of all that data, package it properly, and that'll allow us to think about the proper mechanisms for launch moving forward, all of which we'll communicate with you Kate to you in that investor event. Fantastic. Thank you so much.

Operator

You're welcome. Thank you. And your next question is from Brad and folks from H.C. Wainwright. The line is now open.

Unknown Speaker

Hi, thanks for taking my questions and congrats on all the progress. from me, I apologize if I've been answered, just hopping between a few calls here. But firstly, do you have any blinded insight into the titration that's happening in the nephroclinical trial? Even if this is just through way of drug supply, and if so, is there anything you can say whether that is tracking in line with expectations? Secondly, maybe just on the, I guess, Kadeo side, guidelines. I'm assuming this is based on ex-US usage and history. Is there anything in those draft guidance that is different to the way the FAMISTAT would be used in the US? I guess, are they recommending it in any different way than you envision it being used in the US? That's it for me. Thank you.

Vincent Angotti

Shaquille, I think that's right down your alley. So any insights on, in particular, the titration or anything you can comment on, realizing it's a blinded study?.

Shakil Aslam

Absolutely. So the titration has been, again, it's a blinded study. and with only limited information available. But overall, it's behaving actually quite well. exactly as we were expecting. So we had a maximum dose that we can give. There's a ceiling on that dose. So most of patients in this study never really get even anywhere close to that dose. So most are titrated below half of the dose of the maximally allowed dose. So that's very, very good news.

So it's exactly in the window where we wanted it. So that's very important that we only use the minimum amount of agent to nifamustat to get a therapeutic effect. So from that point of view, it's quite aligned with it. And second thing that investigators point out is that there's hardly any need to make multiple adjustments. So when patients feel it gets titrated, they remain at the same level. For some of these patients, the study had gone up to one week, and they did not need to go back up and down to retitrate to stay in the therapeutic range. So I think that's a huge, huge advantage when you compare it to haptrin, for example, where titration can be all over the place.

And it's actually so frustrating that many physicians actually just, you know, do not even titrate with haptrin when they use for CRRT.

Vincent Angotti

Can you also remind them on the titration, how quickly it occurs as it relates to the protocol in the study?.

Shakil Aslam

So roughly about 80% of the patients, they are within therapeutic range at the starting dose, that infusion rate that we have in our protocol. So within half an hour. almost 100% of the patients are within therapeutic range.

Vincent Angotti

And that titration schedule if they're not happens in the first hour, correct?.

Shakil Aslam

Right, right. So we titrate every 15 minutes and that's guided by bedside blood test of ACT. so every 15 minutes you can change the dose, go up or go down. As I said, 80-85% of the patients are already therapeutic at the starting dose and another 15% may need one up titration which is relatively small. As opposed to 50 mg which would be the highest dose, most patients.

Vincent Angotti

are therapeutic around 20 to 25 milligram per hour. I just want to reiterate that this is a blinded study. These are observations, but we'll see the final results when obviously it's unblinded. Shaquille, can you comment on the second question Brandon had relative to the KDGO guidelines and if those recommendations are of any treatment patterns or different use than how we're seeing at XUS?.

Shakil Aslam

Right, so the guidelines, the way they are phrased right now is that this is an alternative to citrate, so if there is contractual indication to use of citrate or if the citrate is not available, then this would be agents that could be used in that scenario. So in the U.S., Citrate use is around 25% of the patients or clinicians use citrate or the hospitals use citrate. So approximately 75% of the institutions do not have citrate available. And even within institutions where citrate is available, there are complexity to its use. So it's contraindicated in patients with liver disease, for example, and some other metabolic issues that can result from its use. And so there is a chunk of those patient institutions which do have access to citrate, they are not able to use citrate on every patient. Now, in Japan and South Korea, there's barely any use of citrate, so in those countries, this is the first-line agent.

So the way it's worded for the rest of the world right now, the KDU guideline is that you should use citrate first, but if it's not available or contraindicated, then you go to to Nefamostat as a use. But again, I think just because 75% of the U.S. facilities do not have access to Citroën, I think there's a pretty big chunk of market which will be a candidate for Nefamostat right now. out the gate and within even the 25 percent segment that does use it there's a lot of and a lot of labor-intense method that CITRUT uses, so there is a definite desire to move away from CITRUT if a better and easier to use agent becomes available.

Operator

All right, thank you very much. Sure. Thank you. And your next question is from Naz Rahman from Maxon Group. Here, Linus, I will open.

Nazibur Rahman

Hi, everyone. Congrats on the progress and thanks for taking my questions. Just a few. So, on the Cave-Deagle guidelines, could you comment on basically how long it takes or what are the next steps for this to become, I guess, confirmed or permanently approved guidelines? And following that, How quickly does this guideline get adopted by American institutions? And also, how closely is it followed by American institutions? And I guess not just in terms of anticoagulants for CRRT, but just in general.

Shakil Aslam

We're going to move to Shaquille again. So on confirmation, you know, once the commentary is done, Shaquille? Yes. So I unfortunately do not know the exact timeframe. They closed their public review comments on the in May of this year. So my guess would be somewhere between three to six months, they will have the final draft. More like three months, I would say. And in terms of its adoption within U.S. institutions, it's quite variable.

So these guidelines are well known and they are recognized by most health care professionals today. But there is institutional variability within the U.S. like any other place. Some clinicians follow them very closely. Others would have their own kind of versions of guidelines. So from our point of view, what the inclusion of Nufamistat does is, number one, it validates that this is actually an acceptable, safe, and efficacious alternative to other therapies and which has been used in other countries and they are recommending obviously wider use of nifamistat. And secondly, many institutions and clinicians when they want to develop their own protocols for anti-coagulation CRRT in their own institutions, having an international body of experts, basically giving you rationale and recommendation, I think it really makes things much easier for them to adopt those recommendations. recommendations at their own institutions. So I think it just supports that, okay, well, this data has been reviewed by international group of panels and therefore, You know, you can go to the pharmacy and other stakeholders in the hospital and tell them that this is what you want to do.

And so from that point of view, I think it's also positive news for us.

Operator

Got it. Thank you. Sure. Thank you. There are no further questions at this time. Please proceed with the closing remarks.

Vincent Angotti

Thank you, Operator. I just want to say thanks again to everyone on the call for your time with us today and your interest in Telferra. We're excited about the prospects moving forward and look forward to updating you on our progress.

Operator

Operator, that concludes our call. Thank you, ladies and gentlemen. The conference has now ended. Thank you all for joining. We now disconnect our lines.

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[Call has ended.]