Zogenix, Inc.

Please stand by. The conference call will begin momentarily. We thank you for your patience and ask that you please remain on the line. Once again, please stand by. The conference call will begin momentarily. Greetings, and welcome to the Zojenix, Inc. First Quarter 2021 Financial Results Conference Call. During the presentation, all participants will be in a listen-only mode. Afterwards, we will conduct a question and answer session. At that time, if you have a question, please press the 1 followed by the 4 on your telephone. If at any time during the conference you need to reach an operator, please press star 0. As a reminder, this conference is being recorded today. Thursday, May 6th, 2021. I'd now like to turn the conference over to Brian Ritchie of LifeSci Advisors. Please go ahead.

Thank you, Operator, and thank you all for joining us this afternoon. With me on today's call are Chief Executive Officer, Dr. Stephen Farr, Chief Commercial Officer, Ashish Sagrolikar, and Chief Financial Officer, Michael Smith. This afternoon, Zogenix issued a news release providing a business update and announcing financial results for the first quarter ended March 31st, 2021. Please note that certain information discussed on the call today is covered under the safe harbor provision of the Private Securities Litigation Reform Act. We caution listeners that during this call, Sogenix management will be making forward-looking statements. Actual results could differ materially from those stated or implied by these forward-looking statements due to risks and uncertainties associated with the company's business. These forward-looking statements are qualified by the cautionary statements contained in Zogenix's press release issued today and the company's SEC filings, including in the annual report on Form 10-K and subsequent filings. This conference call also contains time-sensitive information that is accurate only as of the date of this live broadcast, May 6, 2021. Zigenix undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date of this conference call. Now, I'd like to turn the call over to Steve.

Thank you, Brian, and good afternoon to everyone. I'm very pleased to be here with you today to provide updates on what has been another exciting and productive quarter for Zigenix. Despite the operational challenges brought on by the ongoing pandemic, our commercial launches of Fintepla for Dravet syndrome in the United States and Europe continue to progress well. As Ashish will describe shortly, we are very encouraged by the steady growth in adoption by both existing and new prescribing physicians and patients, and by the continuing positive dialogue with commercial and government payers. With more physicians gaining experience with Fintepla and more patients reaching that effective dose, we see a marked increase in the type of prescribing base and a beneficial impact on dosing trends. We are now beginning to see an increase in the ability of our field-based teams to travel and meet with physicians and other healthcare professionals. As a result, we are looking forward to the positive impact this will have on our commercial activities and other aspects of our business. I'll now turn the call over to Ashish for further details on how our launches are progressing in the US and in Europe. Ashish, over to you.

Thank you, Steve. I'm very pleased to update you on our team's continued strong execution of the Fintepla launch in the US and to share with you details of the early momentum we are gaining in Europe. In the first quarter of 2021, we achieved $12.3 million in Fintepla net product sales. Fintepla net sales in the US were 11.3 million and 1 million in Germany. This strong performance reflects the significant desire within the Dravet community to initiate and continue treatment with FintechLab. We have been really pleased with our ability to sustain this moment despite significant COVID-related headwinds and in the U.S., weather-related disruptions that impacted most of the country in the beginning of the year. We continue to expect higher adoption and long-term use of Fentepla to increase as more physicians gain positive experience treating patients. And as Steve noted, the impacts of COVID begin to diminish. In the U.S., growth in the number of healthcare professionals enrolling into the REMS program, the initial step and indicator of physician's interest in prescribing Fentepla remains robust. By the end of Q1, approximately 570 HCPs were certified in our REMS program with a majority already prescribing Fentepla. More than 90% of these prescribers are new, that is, they did not participate in our clinical trial or expanded access program, reflecting the broadening interest in prescribing Fentepla. During the first quarter, 151 new patients were prescribed Frentecla, which brought the total number of REMS-referred and prescribed patients in the U.S. to just over 700. This is a 27% increase since the end of 2020. Importantly, we saw a consistently increasing rate of new patient referrals month to month from January through March. We are extremely pleased by this trend, and expect to see continued growth throughout the year. By the quarter end March 31st, the number of patients receiving reimbursed Phentepla in the US grew by 35% to more than 560 patients. More than 90% of this growth in Q1 was driven by patients who were new to Phentepla and the rest consisted of the final participants transitioning to commercial therapy from our open-label studies and expanded access program. We continue to be extremely pleased with the payer coverage and access for Phenteplath. By the end of Q1, payers covering more than 75% of U.S. lives had published a formal policy to cover Phenteplath as per label and on parity with other AEDs. With the rest of plans, covering it through prior authorization and medical necessity process due to the rare prevalence of Dravet syndrome in their covered population. Additionally, we successfully completed the coverage re-verification process for all patients during the month of January, reinforcing Peirce's belief in Fentoplast therapeutic values. Since launch, the majority of Fentepla prescriptions in the US have been written for patients diagnosed with Dravet syndrome, but we are also seeing some patients prescribed and enrolled in the REMS program for other forms of severe rare epilepsy. By the end of Q1, the percentage of prescriptions written for seizure disorders other than Dravet syndrome was in the mid teens. We also see increased physician confidence in titrating patients to the effective dose relative to patient's weight, seizure reduction, and tolerance. Current prescribing patterns indicate that approximately 55% of patients reach their effective dose by the fourth month of the treatment, with the remainder reaching by six. The average daily dose of Finteplat following titration is similar to what we saw in the expanded access program, namely, approximately 0.5 milligram per kilogram per day. Although it is still very early in our launch, our retention rate continues to be very high at 93%, consistent with our experience in the expanded access program and clinical studies. As vaccination rates in the U.S. increase and restrictions ease, more of our customer-facing teams have been able to schedule in-person interactions with HCPs and their staff. We believe this positive trend will continue into the second half of the year, making it an easier environment for us to interact with physicians, patients, and families who are now considering treatment with Fentecla to improve seizure control. Transitioning to Europe, our first European launch in Germany is progressing very well. In the first few weeks after the mid-February launch, more than 50 clinicians had enrolled in the controlled access program to become eligible to initiate Fentepla therapy for their patients. In Germany, approximately 100 Dravet patients are participating in our open-label extension studies and expanded access program. These patients are now in the process of transitioning to commercial therapy a progression we expect to be completed by the end of June. In France, patients are now being prescribed Fentepla under the Temporary Use Authorization, or ATU, as we discussed earlier in this year. Access to Fentepla in France will be under this ATU program until reimbursement negotiations are finalized. Pricing and reimbursement negotiations across major European markets, such as United Kingdom, France, and Italy are progressing well. In April, we launched the Zergenics Access Program to further expand the availability of Fentep Lab to physicians in the other parts of the world where local regulations allow. This also includes some European countries where reimbursement has not yet been established. We look forward to supporting more HCPs and patients in need through this new program. Further reflecting a strong global interest in Fentepla, in Q1, we entered into an agreement with Medicine Pharma to make Fentepla available for patients in Israel. We are now working with our partners to prepare and file the regulatory and reimbursement documents for approval and covered use in Israel. To conclude, we are very pleased that more patients are now benefiting from treatment with Fentepla due to our strong growth in the United States, progress in Europe, and addition of new programs and partnerships in other countries. We anticipate continued momentum, and we hope the worst impacts of pandemic pass, and we receive pricing and reimbursement approvals in additional European countries. Now I will turn it back to Steve. Steve?

Thank you, Ashish, for the great progress. Next, I'd like to update you on our pipeline, including Fintepla for our second and larger indication opportunity, Lennox-Gastaut syndrome, or LGS, as well as Fintepla for Dravet and LGS in Japan. Our community and patient advocacy discussions reinforce that LGS remains one of the most challenging rare epilepsies to manage, and many LGS patients still desperately need new treatments for this severe disease. Therefore, we're excited to be on track for a planned third quarter 2021 submission of a supplemental NDA in the United States for Fintech and FDS, followed by an MAA filing in Europe in the fourth quarter. Both are high priorities and significant milestones for us in 2021. As part of our preparations, we intend to meet with the FDA during this quarter for a pre-SNDA meeting to confirm discussions and agreements from our earlier end of phase three meeting and to align on the final electronic submission structure. In parallel, we were excited to report new clinical data for PIN-TEPA in LTS in a podium presentation at the recent AAN 2021 meeting. These results from our pivotal clinical trial in LGS show the positive impact of FinTAPLA on patients' abilities to self-regulate behavior, emotions, and cognitions. These non-seizure findings complement earlier Phase III data analyses demonstrating that FinTAPLA significantly reduced drop-seizing frequency, and notably the frequency of generalized tonic-clonic seizures that are a primary risk factor for sudden unexpected death in epilepsy or SUDEP. We believe these data, combined with a novel mechanism of action relative to other approved therapies, position Fintepla as a potentially important new treatment option for LGS patients. The clinicians we have engaged in advisory panels reinforce that seizure control is the primary treatment goal for LGS, and that any patient whose seizures are not well controlled could be candidates for Fintepla treatment. While clinicians indicate they would likely try Fintepra for all of their refractory LGS patients, they also stated that they may place it higher in the treatment algorithm for patients who experience a high number of generalized chronic chronic seizures and who may, in other ways, be at risk of SUDEP. Now, turning to Fintepra in Japan, we remain on track for our planned JNDA submission for Duray syndrome later this year. Separately, the last of 33 patients has been randomized into our LGS study in Japan, which the PMDA requested to enable the comparison responses in Japanese patients to the multinational data from study 1601, our broader phase three trial. In addition to dribbling LGS, we are also making great progress around the planning of a phase three trial of Fintepa in CDKL5 deficiency disorder, or CDD. As a reminder, Dr. Oren Davinsky at the NYU Langone Medical Center last year shared compelling data from his investigator-initiated study of several patients with CDD. We plan to discuss the final protocol with the FDA during this quarter and expect to begin enrollment of CDD patients into the study in the second half of the year. As you can see, we have significant growth plans for Fintepa. To complement these objectives, our intellectual property position for Fintepla was further strengthened last quarter by the addition of two further U.S. patents in the Orange Book. This brings the total listed patents for Fintepla to 10, with patent expiries ranging from 2033 to 2039. Now I'd like to discuss MT1621, our late-stage investigational therapy for thymidine kinase 2 deficiency, or TK2D. I'm excited to report our continued progress as we bring this therapy forward to a patient population with no approved treatments. Leveraging our data set, demonstrating benefit in both morbidity and mortality in TK2D, we will pursue an indication encompassing all patients in our FDA filing. Based on our discussions with the EMA in Europe, we will pursue an initial indication of patients with early onset disease with a goal of making this drug available as soon as possible. The late onset group represents a minority of the population, an estimated 20% of all TK2D patients, which has only recently been recognized and is less studied. Young onset patients represent the largest group of patients and those with a clear high unmet need. Untreated disease in infant and childhood onset patients often results in loss of developmental milestones and may progress rapidly to respiratory failure and death. All our studies required for the record submissions are advancing as planned. and we remain on track to submit an NDA for MT1621 in the first half of 2022, and an MAA for European approval shortly thereafter. In parallel to our development work, we are pursuing a range of activities to identify patients and better characterize TK2D. As with many mitochondrial diseases, TK2D is significantly underdiagnosed. In the setting of clinical suspicion of the disease, TK2D is easily and definitively diagnosed by genetic testing. We are therefore engaged in three important areas of work. Development of disease awareness and medical education programs to drive urgency to get a genetic test, laboratory partnerships to improve access to non-cost testing and to ensure testing is present on testing panels, and scientific and market research to understand and characterize impact of this disease on patients, caregivers, and families. To conclude, this has been a very productive and exciting cohort with Zigenix as we ramp up commercial activities for Fintepla and continue to advance clinical and regular programs for MT1621 and the new Fintepla indications. So with that, let me hand over the call to Mike for the financial review. Mike?

Thanks, Steve, and good afternoon, everyone. Today we issued a press release announcing our business and financial results for the first quarter ended March 31, 2021, which I'll now review. We recognized $13.7 million in total revenue for the first quarter of 2021, which was an increase of 61% as compared to the $8.5 million recorded in the fourth quarter of 2020. This increase was the result of $11.3 million in product sales of Fintepla in the United States, $1 million in product sales of Fintepla in Germany, and $1.3 million related to our exclusive distribution collaboration with Nippon Shin-Yaku for Fintepla for Jure Syndrome and LGS in Japan. Total net product sales of Fintepla of $12.3 million were an increase of 53% as compared to the $8.1 million reported in the fourth quarter of 2020. We recognize 1.2 million in total revenue for the three months ended March 31st, 2020, a year ago. R&D expenses for the first quarter were $31 million, a decrease as compared to $33.2 million recorded in the corresponding Q1 period of 2020. SG&A expenses for the first quarter ended March 31st, 2021, totaled 31.2 million, compared with 21.3 million for the first quarter of 2020. The increase of approximately 9.9 million was primarily driven by the investment related to the launch of Fintepla in the United States and in Europe and the preparations related to the commercial launch in other countries forthcoming in Europe in the coming year. Net loss for the first quarter ended March 31st, 2021 with $55.6 million or a dollar per share. This compares to a net loss of 25.8 million or 54 cents per share in the first quarter ended March 31st, 2020. We ended the first quarter with a strong balance sheet cash, cash equivalents, marketable securities totaling $435 million. With that, I'll now turn the call over to the operator to start our Q&A session. Operator, can you please open up the line for questions?

Thank you. If you'd like to register a question, please press the 1 followed by the 4 on your telephone. You will hear a three-tone prompt to acknowledge your request. If your question has been answered and you'd like to withdraw your registration, please press the 1 followed by the 3. One moment, please, for the first question. Our first question comes from the line of Paul Matias from Stifel. Please go ahead.

Hey, thanks so much as always for taking the questions. Always appreciate it. So a couple, a couple. On the commercial side, it's encouraging to hear that you've had more in-person interaction as the pandemic wanes. Is there any way you can quantify that? What percent of your face-to-face meetings have been virtual versus in-person over the past month or two? And are you seeing any increase in volumes as a byproduct of it? And then separately on LGS, just ahead of this pre-SMDA meeting, I was wondering, Steve, could you just set the stage a little bit? Is this more administrative or do you feel like you still need to kind of get, you know, reaffirmation that a single pivotal can serve as the basis for a filing? Thanks so much.

Thank you, Paul. Let me take the LGS question first, and Ashish, if you could get ready to address Paul's commercial question. So, regarding the pre-SNDA meeting, this is a standard meeting as part of a development programme towards an NDA, and I think we, like all other sponsors, really take advantage of as much time as we can to be in front of the FDA, in order to ensure there's absolute clarity for to ensure that the review remains hopefully on track. So as we've mentioned in the past, we had really good interaction with the FDA at the end of last year around the clinical package and the non-clinical package and really got agreements on what those would be. So this is more administrative and more reaffirmation and confirming of our previous agreements. And as I mentioned, it's standard. It's helpful for both them and for us. It also gives us the opportunity to get initial feedback on what would be our proposed label for LGS. So there's a lot of upside going forward in the meeting, and it's not going to impact the timeline.

Great.

Thanks. Over to you, Ashish.

Yep. Hi, Paul. In terms of the in-person interaction, there has been a recent trend in last year, four weeks, and In terms of quantifying, I'll give you primarily a qualitative. I would say more than 60% or two-thirds of our key account managers have been able to go out in the field and call on their physicians. There are certain pockets in the country we still are not allowing the pharmaceutical industry reps to come in. So those areas kind of still stay kind of off limits. But we are hoping that they will start opening up because we are getting the appointments. The other thing I would add is we have been doing this. We got in starting towards the end of March. We are doing our dinner programs in person. We are doing in-services in person. We just did a huge webinar, which was attended in some places in person. So all of these things kind of going towards the positive trend. And we are expecting that that trend is continuing with patients coming and visiting the offices. and offices allowing more patients to come in. Because as you know, in some of these chronic conditions, the offices want to continue with the telemedicine because of the COVID risk. But as the vaccinations are increasing, we are seeing that in almost every part of the country. So more to come on that one.

Okay, great. And would you mind just finally just quickly quantifying what gross to net was in the quarter? Thank you, Jeff.

Uh, yeah, I can take that. It's, um, it's in the teens. Uh, so, you know, it's been, um, been in there since we started. So I think we're eventually, we're going to be, we think eventually once we get the full patient population in over a year post launch will be, you know, low twenties, but it, um, it currently has a little bit lower than that because commercial is coming up a little earlier than, um, in the public page.

All right. Thanks, Mike. Thanks, everyone. Thank you, Paul.

Our next question comes in line of Mark Goodman with SVB Learing. Please go ahead.

Yes, hi. I guess one can give us just a sense of the average price that the patients who are on the therapy, that's first question. Second question is, What kind of color can you give us about how April's been and just, you know, first week of May, just trying to get a sense of how things are going relative to last time. And then lastly, you mentioned that off-label was, I think you said 15% this quarter. What was it last quarter? Thank you.

Thanks, Mark. I'll provide a little bit of color on, you know, sort of what's going on this quarter. And then Ashish, if you could take a question on average price and also these non-derived patients that we're seeing as well. So just very simply, Mark, and I think it follows what Ashish said in the previous response, previous question. We've seen a nice increase in rates of patients month over month in Q1. So in other words, we had more patients coming to the REMS in March than we did in January. And we're seeing that trend in the early part of this course as well. So I think that just is a result of what Ashish talked about earlier. And with that, I'll hand over to Ashish.

Yeah, thank you. Hi, Mark. In terms of the average price for therapy, I would say it continues to be the same. because, again, we are in the first year. But we are having more and more patients now getting towards that 0.5. The average dose now is around 0.5. So if you translate that into annualized, it's around 109,000. So that would be going forward rate, it would be. In terms of the patients on the indications other than Dravet, That's not like the overall is since the launch. So just to clarify my comment, I think that mid-teens number is what we have said is the entire population that has come starting from the launch to now. And if you recall earlier, we hadn't really kind of separated that out. And when we did the analysis and everything, that number now looks in mid-teens. So that's why we are sharing it.

So the comment is that it's been pretty consistent, you're saying, since launch at 15% is what you're saying. And I guess if that's true, just one follow-up, which is are there LGF patients there or is this everything besides Gervais and LGF?

Yeah. So, yeah, it has been consistent that I can confirm. But in terms of it's all the refractory epilepsies or the rare epilepsies that you see. And as you can imagine, majority of that is classified as LGS. So you see up to two-thirds of them in LGS and rest in some of the other refractory epilepsies.

Thanks. Thank you, Mark.

Our next question comes on the line of Tazeen Ahmad with Bank of America. Please go ahead.

Hi. Good afternoon. Thank you so much for taking my questions, and congratulations on the quarter. As you prepare to file for the LGS application, I wanted to get your thoughts, Steve, on whether you think that this is likely to get an accelerated path to approval. In some other examples of some, you know, CNS and other therapeutic areas, there have been some recent filings where expectations might have been set to accelerate and an SEA has granted a standard review. So I wanted to get your thoughts on that, and then I have a follow-up.

Yeah, thanks, Dean. Appreciate the question. I think where we stand at the moment is that we're going to do our best to put forward rationale to allow the FDA to determine a priority review for the supplemental NDA. And that's based upon what we are observing is that LGS is a very notoriously hard syndrome to treat. despite the fact that the FDA have already approved 708 drugs for LTS, there's still an urgent medical need. And we hear that not only from physicians, but also, importantly, from the patient advocacy groups as well. So we feel that it's in everyone's best interest to try and move forward with a priority review. Having said that, the downside of that argument is that the FDA has approved drugs already in LTS. And that might push them to a standard review, which would be 10 months. But as you know, it's their decision to make. We can justify, but ultimately it's their decision to make. But we will do our best to move forward with a priority review.

Okay, great. Thanks for the color. And then as it relates to the launch, maybe Ashish can provide some color on how exactly, like how important is it to be, you know, on formulary for a doctor to feel comfortable writing a script for Fintupla? Is it the case that even if it's not on formulary, you know, doctors at this point feel like they understand what kind of paperwork they need to fill out and that's not a rate-limiting factor for them?

Yeah, definitely. It has not been a rate-limiting factor at all. Physicians are in this particular space, as you know, in rare diseases, prescribe what is best for their patients. And in every conversation and the interactions we have had, that particular hasn't really come up. The other thing to add there is we do have Zogenic Central, which is really geared up to support the office staff as well as the physician into the entire process. And the staff there is really experienced in managing this process for the rare diseases. And so far, in my prepared remarks, we have had really good success. And that becomes a reference point for the physicians to look at, but also be comfortable that this product will go through the entire process within two to four weeks and the patient will get the therapy.

Okay. And if I could, please, on a last question, for patients that are onboarding that are, you know, the profile of the patient, do you know what percent of them have already tried EpiDialect?

We haven't broken that out, but as you can imagine, most of them would have tried something else. What I can share with you is that we have seen On an average, it's around three to four different anti-epileptic drugs that they are on. And on a very worse case, we have seen up to 10. And as you know, Fintepla gets added to the therapy. So even if the patient is on one of the CBD products or any of the other products, Fintepla will get added to that. And once the patient is stabilized, then they will start looking at, okay, what else they can do in terms of reducing the overall product burden.

Okay. Got it. Thank you so much, Ashish. Thanks.

Thank you, Suzanne.

Our next question comes from the line of Danielle Brill with Raymond James. Please go ahead.

Hi, this is Daniel on for Danielle. Thank you for taking the question. Just very quickly, can you help us understand what percent of patients that are enrolled in RAMS end up on reimbursed drug? We're asking because it looks like last quarter you had 550 patients enrolled in RAMS. This quarter you have 560 patients on paid drug. Does that imply that 10 new patients enrolled in REMS actually have received reimbursed drug in the 1Q? Thank you.

Ashish, do you want to address that and explain the difference between the top of the funnel in the REMS and the prescribed drug?

Yes. In terms of when you look at the patients going in on the top of the funnel and then getting reimbursed therapy, you want to look at the absolute numbers. 151 at the top of the funnel and around 145 plus at the end of the funnel. Because if you recall at the end of Q4, we had around 415 patients on reimbursed therapy and 550 patients were just enrolled into the program. And it takes around two to four weeks. So let's just say if it is four weeks, you're going to have that lag as patients come on to the therapy, as coming to the REMS program, and then they end in receiving the reimbursed therapy. I hope that helps understand the equation there.

Yes, thank you very much. Thank you, Anil.

As a reminder, to register for a question, please press the one followed by the four on your telephone. Our next question comes from the line of Tim Lugo with William Blair. Please go ahead.

Hey, this is Lachlan. I'm with Tim. Thanks for taking the question. So I was wondering, how should we think about the change in productivity of the sales reps as they get more and more in-person interactions compared to the sort of virtual or digital world that we've been in? And second question, if I may, within the REN certified doctors you have, how many of their potentially eligible patients do they have on therapy? So are most of them sort of trialing with one or two patients before expanding into the rest of their population? Or do they only have a few patients each?

Lachlan Banks. Yep. Lachlan Banks. I'll start with the second question first. What we are seeing is, in terms of physicians who are prescribing, they are not only your thought leaders, but also the community physicians. And as you can imagine, if you have thought leaders who have a lot more patients, they are writing a lot. And we have, if not majority, many, most of their patients have been written on for a FinTech lab. And when you have community physicians, and I can tell you that I said in my prepared remarks, almost 90% of our prescribers are coming from the community physicians who are not part of our clinical trial, the expanded access program, and where we are seeing multiple prescriptions. So I think it's both. Most of it is, and again, some of the places where they may not have written for all their patients or offered to all their patients because they haven't seen them. because of the COVID or because of the restrictions or the families who are not feeling comfortable going into a healthcare setting or even trying something else because of the COVID and any challenges that they may have with that. But that kind of leads into your first question. I don't have a benchmark to talk about what change will be because we launched within the COVID. I think the only benchmark I can go back is to what we saw for the entire anti-epileptic class, where the new-to-brand prescriptions in the COVID environment went down almost up to 35% to 40%. And we will be doing every effort to go back and go to every one of the physicians who have Dravet patients to educate them and make sure that they offer the product to their patients.

Thanks for the call.

Our next question comes from the line of Yatin Sunaya with Guga9 Partners. Please go ahead.

Hey, guys. Thank you for taking my question. I didn't know I had to press 1-4. I was pressing 1-5-star, so sorry about that. Okay, a couple questions for me. In terms of the total sale, is there any European component to it? Can you tell us how much was the sale? What was the number in Europe? So that's number one. The second question is just trying to get a sense of cadence of new patients going forward and also trying to understand the dynamic in Q1. Because I think Ashish did mention that there were some leftover EAP patients that were added in Q1 or that got the drug in Q1. So it seems like the de novo patients are less than 150. So it seems to be a little bit less than last quarter. So just trying to get a sense what the exact number was for de novo patient and how should we think about cadence going forward. Thank you.

Yeah, I can take these for you, Yatin. Just on your second question, first with respect to the cadence of the new patients, it was actually 150 de novo patients, or 151 de novo patients. So the early access patients were already in the REMS program, and around 80, 85% of them were already on reimbursed treatment by the end of last year, and the remainder went on to reimbursed treatment in this last quarter. However, they're already in the REMS, so they don't count against the new patients coming into the REMS. And with respect to cadence, I think I'd just reiterate what I said to Mark earlier. Of the overall number of patients we saw in Q1, we saw month over month increase in the rate. And we expect that will continue as we go through this quarter. On the total sales of 12.3 million net sales, 11.3 of that was in the United States and a million was in Europe.

Got it. And I understand it's very early in the launch. Any color on the compliance or the discontinuation rate? And then maybe if you can comment on what the average rate per patient that you're seeing for Dravet patients.

Ashish, I'll ask you to address that question.

Yeah, I'll start with the average weight. So the average weight we are seeing is consistent in what we saw in the expanded access program, around 35 kilograms. So you get the range there, but the average right now is 35. And the compliance, as I said, or the persistence in the, as I said in my prepared remarks, that rate is around 93% at this point in time. So 93% of the patients who started there are still under therapy. And again, it's very early, but as you know, in the early expanded access as well as clinical trial, the discontinuations were around 10%. So we expect that we will get there. But right now, I think we are seeing great results. Patients are seeing great results, and they are benefiting, and then they are staying on the product.

Great. Thank you very much.

Thank you, Yash. Thanks for the questions.

The last question comes on the line of Nina with Ritoguard with Citi. Please go ahead.

Hey, guys. Thanks for taking my question. So I just have a question around the cardiac monitoring. And, you know, I guess at this point you have some patients who have been on commercial drug for six months, so they would essentially be going now for their six-month kind of echo, I guess. How has that been going? Have there been any kind of issues in terms of getting patients in for the six-month echo? Is that proving to be kind of, or is that kind of proving to be as smooth as the baseline echoes have been going? Thanks.

Go ahead, Ashish.

Yep. I think in terms of the cardiac monitoring and the second echoes, exactly as you said, it's been going smooth. Uh, we haven't had any, uh. Any challenges there and patients know that families know that and as, you know, we have a really robust central program, which really reminds individuals stays on top of it. We stay connected with the physicians. And we have many patients, a lot of patients and as, you know, some of the expanded access and the clinical trial patients who. went on to the commercial therapy, they already had the RECOS some time back in the past six months. So we have a lot of patients who have gone through their second RECOS again since even last year. And every one of them have been able to get it in time and without any challenges.

Gotcha. Perfect. Thanks. And I guess if I could just ask one more question quickly then on the just kind of going back to the pace of new starts. I think, you know, thinking about the number of patients that were yet to kind of start on commercial drug rather than kind of the number of patients that were in the REMS. I think I calculated, you know, in the fourth quarter, there were about 160 or so fintepli-naive new starts in fourth quarter. And this quarter, it seems like it's closer to 100, just given that there were some patients still starting drug from the EAP and the OLE. I guess, how should we think about the pace of new starts from here in terms of, you know, where that number could go, understanding that, you know, like you guys have said, there is this month-over-month increase, but, I mean, what's a reasonable number of new starts over the next couple of quarters? Thanks.

Chief, do you want to take that one?

Yep, I'll start. Yeah, so, you know, we are not giving any guidance, but as Steve said in an answer earlier, The 151 that we saw, we have seen a consistent increase month over month, and we continue to see that as we move into the second quarter. So I think that would be one guidance, if you will. And in terms of the Q4, as well as in Q1, in Q1, we did not have any patients from the expanded access program or from the clinical trial coming in. So everyone that you saw, that 151 that I said earlier, are all new to Fentepla. And every growth that's going to come for us is going to be from new patients to Fentepla.

Yeah, so I'll just add a little color to that. So I think with new starts being referred in the program and then coming through and getting drug, which is pretty close to 100% level at this point, it's just a function of them getting covered and set up. to get drugs through the initial REM screen and other elements that take, as she says, basically taking roughly three, four weeks. The new starts are the patients that are coming in referred to the program. We finished Q4, we had total around 100, between 150 and 160, and it's about the same, pretty much the same number in Q1, although we had the impact of severe impact of the COVID pandemic through the holidays and through the January year. And as I'm sure you're familiar with, there are the restarting or people holding off in January to set new payer programs and whatnot. So you had your typical January starting and we saw increasing throughout this quarter, month to month, and then also increasing and keeping up with increases Into this quarter as well, so you expect to be. You know, that increasing in the capacity to for it to. Be less challenged by cobit also for the balance of the year too. So it was great to see that we were able to come through this quarter and what is standardly a lower demand quarter, lower start quarter because of a January impact and to maintain the pace that we were on and increase it by the time getting through the end of the quarter.

Okay, thank you.

There are no further questions on the phone line. I'll turn the call back over to you.

Thank you very much, Operator, and thanks to all of you for joining our call today. This quarter, like the past quarter, has been incredibly active for us, and we're continuing to focus on advancing our commercial clinical programs. To bring tomorrow, there will be critical new treatment options for patients facing serious and medical needs. So really look forward to providing you with further updates on our activities throughout this year. Thank you all again for joining us on our call today and enjoy the rest of your day. Bye now.

That does conclude the conference call for today. We thank you for your participation and ask that you please disconnect your line.