Zogenix Q3 2021 Earnings Call Transcript

Quartr
Provided by Quartr
November 3, 2021

There are 14 speakers on the call.

Operator

Thank you for standing by. This is the conference operator. Welcome to the Zogenix, Inc. Third Quarter 2021 Financial Results Conference Call. As a reminder, all participants are in listen only mode and the conference is being recorded.

Operator

After the presentation, there will be an opportunity to ask I would now like to turn the conference over to Brian Ritchie of Life Science Advisors. Please go ahead, Mr. Ritchie.

Speaker 1

Thank you, operator, and thank you all for joining the Zogenix management team this afternoon. On today's call are Chief Executive Officer, Doctor. Steven Farr Chief Operating Officer, Ashish Agrawalakar Chief Development Officer, Doctor. Gail Farfel Chief Medical Officer, Doctor. Brad Gaylor and Chief Financial Officer, Michael Smith.

Speaker 1

This afternoon, Zogenix issued a news release providing a business update and announcing financial results for the 3 9 months ended September 30, 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, Zogenix 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 ks and subsequent filings.

Speaker 1

This conference call also contains time sensitive information that is accurate only as of the date of this live broadcast, November 4, 2021. Sogenix 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'll turn the call over to Doctor. Steven Farr. Doctor.

Speaker 1

Farr?

Speaker 2

Thank you, Brian, and good afternoon to everyone and thank you for joining us. I'm delighted to be here with you today to provide updates on another strong and positive quarter for Zogenix as we continue to execute on the commercial launch FINTEPLA in Travet syndrome, prepared for potential launch in Lennox Gastaut syndrome and advanced our late stage development programs to both FINTEPLA and MT-sixteen twenty one. Positive momentum for the launches of Nutella in

Speaker 3

the U. S. And Europe continue

Speaker 2

And once again, we achieved solid quarter over quarter growth in the number of prescribers of, patients on and net sales of FINTEPLA in both regions. In a moment, Ashish will provide additional color and perspectives on the FINTEPLA market dynamics that are leading to the success and continued growth. We also remain excited with upcoming opportunities to potentially expand FINTEPLA as a treatment option in other severe refractory Childhood onset epilepsy syndromes. Towards this goal, in September, we submitted a supplemental NDA to the FDA seeking to expand the label indication of FINTEPLA for treatment of seizures associated with Lennox Gastaut syndrome or LGS. There are an estimated 30,000 to 50,000 patients suffering from LGS in the United States and notably the majority of patients have uncontrolled seizures despite taking existing approved anti seizure medications.

Speaker 2

Also, we are now getting close to starting our global Phase III trial of Intepid patients with CDKL5 deficiency disorder or CDD. Several sites in United States have recently been cleared to begin screening patients And we anticipate the 1st patient enrolling in the study within the next few weeks. Gary Farfel, our Chief Development Officer, will provide More detail on both of these programs later on the call. Last, during the quarter, we were pleased with the progress being made through regulatory interactions and ongoing development studies for the MT-sixteen twenty one program, our investigational substrate enhancement therapy for Thionine Kinase 2 Deficiency or TK2D. Brad Gaylor, our Chief Medical Officer, will walk you through this progress in more detail on this call.

Speaker 2

I also would like to remind you of our upcoming virtual investor on November 8 that will feature presentations by key opinion leaders on disease overview and natural history of TK2D and their experience with MT-sixteen twenty one as a treatment option for the TK2D patients. With that, I'll now hand the call over to Ashish. Ashish?

Speaker 4

Thank you, Steve. The continued solid growth for FINTEPLA in the 3rd quarter, Completing our 1st year of commercialization, all within a restrictive COVID environment and in single rare indication is reflective of the strong support we are seeing from patients, caregivers, healthcare providers and payers. That support, paired with potential in multiple new indications and geographies, provides us with confidence in FINTEPLA's commercial growth outlook in the short, medium and long term. Starting with the U. S, During the Q3, over 130 new patients were referred to the Ramps program and 133 new patients started reimbursed therapy with CintagLab.

Speaker 4

This progress reflects a steady cadence of new patient adds similar to the prior quarters from this year. By the end of the Q3, over 990 patients in the U. S. Have been prescribed FINTEPLA and referred to the REMS program. Adherence to the FINTEPLA therapy also remains strong and remarkably consistent with our experience in the long term clinical trial and expanded access program.

Speaker 4

Since launch, the discontinuation rate is around 14% overall and 11% within Dravet syndrome patients. We believe these rates are substantially lower than typically observed with other anti seizure medications commonly used to treat refractory epilepsies. We estimate that in the U. S. Our market penetration among eligible Dravet syndrome patients is currently about 50% to 20%.

Speaker 4

Our internal research indicates that we should be able to increase the market penetration by at least threefold with time. For example, brand awareness for FINTEPLA among U. S. Neurologists and epileptologists has almost doubled since late 2020 and is now higher than 80%. To take advantage of this momentum, we recently completed the expansion of our field based sales and medical teams by adding experienced professionals with deep understanding of the epilepsy market.

Speaker 4

The field sales team is now configured to educate and support an expanded list of healthcare providers about FINTEPLA in Dravet syndrome. While it's still early, we have already seen some positive trends with respect to increased physician engagement leading to new prescribers in the Q4. We continue to be very pleased with the payer coverage for FINTEPLA in both private and government sector. Typically, it takes 2 to 4 weeks for a new patient to receive FINTEPLA once they are referred to the REMS program and payer coverage continues to be strong with effectively all patients receiving positive insurance determinations. During the Q3, We supported the Dravet community through various local and virtual events, including a unique Fintepla photo diary contest through social media.

Speaker 4

More than 700 caregivers participated in that event. As you all probably know, November is Epilepsy Awareness Month. In this month, our team is participating in more than 30 community events, both virtually and in person to support and educate Dravet patients and caregivers. In Europe, the launch of FINTEPLA in Dravet syndrome in Germany We've continued to progress very well and our patient and prescriber base continues to grow steadily. We continue to add new patients to reimburse therapy in France through the temporary authorization of use program and currently in 8 additional countries through our Zogenix access program.

Speaker 4

We have made significant progress and executing commercial launches next year. Following the submission of our sNDA for LGS, We are now ramping our launch preparations for this new and potentially significant indication for FINTEPLA. We are conducting market research, advisory boards and 1 on 1 discussions with healthcare providers, caregivers and payers to best understand the need of the LGS community. These preparations are already helping us to refine our go to market strategies and along with our expanded commercial team will position us to launch FINTEPLA immediately upon potential approval in 2022. In summary, we are pleased with our strong sales growth in the United States, continued positive momentum in Europe and preparations for the potential commercial expansion of FINTEPLA into LGS.

Speaker 4

We anticipate continued growth in all key geographies and look forward to updating you in the coming months. Now I will turn the call over to Gail to share exciting new data on FINTEPLA.

Operator

Gail? Thank you, Ashish. I'll echo the enthusiasm expressed by Steve and Ashish around our submission of the for the SDA for LGS, which was submitted on September 27 with a request for priority review, which if granted which set an FDA goal to take action on the submission in 6 months. A standard review would establish a 10 month review timeline. The submission consisted of safety and efficacy data from 263 LGS patients who participated in our positive Phase 3 trial, which met the primary and key secondary endpoints.

Operator

The sNDA submission also contained Non clinical chronic toxicology and carcinogenicity studies and no new safety concerns were observed. Furthermore, Prospective assessment of cardiac valves in these studies did not reveal any microscopic changes at any dose level. Next, we recently presented results from the interim analysis of our ongoing 12 month Phase 3 open label extension study of FINTEPLA in LGS patients at the 2021 Annual Meeting of the Child Neurology Society. The results demonstrated that FINTEPLA produced highly statistically significant improvements in the frequency of drop seizures in the 2 47 patients enrolled in the extension. The median reduction in drop seizure frequency was 39.4% at 3 months and at 51.8 percent for patients who were assessed over months 10 to 12 and the reductions at both time points had p values less than 0.001 compared with Phase 1.

Operator

Of the 170 patients evaluated in the interim analysis at months 10 to 12. 51.2 percent reached the 50% improvement threshold and 25.3% reached a 75% improvement threshold, which is considered a profound level of improvement. These results highlight the durable and positive effects of long term FINTEPLA treatment in LGS patients. To date, The safety profile in the LGS studies is comparable with results seen in the Dravet syndrome trials and our experience in the post marketing setting. This profile in conjunction with FINTEPLA's unique mechanism of action highlights the potential of FINTEPLA to become a meaningful treatment option for LGS patients and their families, particularly considering the highly refractory nature of this condition.

Operator

I'd also add that we remain on track to submit a JNDA in Japan to Japan's Pharmaceutical and Medical Devices Agency by the end of 2021. Now let's move on to our newest FINTEPLA program in CDKL5 deficiency disorder or CBD. CBD is a rare, severe and highly refractory childhood onset epilepsy in which patients experience multiple symptoms such as gastrointestinal, visual and sleep disturbances in addition to treatment resistant seizures. Zogenix has joined the Lulu Foundation, The University of Pennsylvania and other industry collaborators to support a 3 year non interventional study of patients with CBD to characterize developmental trajectories and clinical features of this disorder. In a small open label conducted by Doctor.

Operator

Oren Dubinsky of NYU Langone Medical Center and presented at the American Epilepsy Society Annual Meeting in 2020. Treatment with fecloramine produced clinically meaningful reductions in seizure frequency with a 90% reduction in generalized tonic product seizures and a 55% reduction in toic seizures in a case series of 6 patients with CDD with a median treatment duration of 5.3 months. These initial compelling results form the basis of our decision to launch a study of FINTEPLA in CBD patients, and we anticipate the 1st patient will enroll shortly. The placebo controlled fixed dose 2 arm trial We'll enroll 80 patients randomized to either 0.7 milligrams per kilogram per day FINTEPLA or placebo. FDA confirmed earlier this year in a pre IND meeting that a single positive randomized controlled trial could form the basis of the supplemental NDA in this indication.

Operator

With that, I'll turn it over to Brad for an overview of our MT-sixteen twenty one program.

Speaker 3

Thank you, Gail. We made meaningful progress on our MT-sixteen twenty one program throughout the quarter, expanding and enhancing our planned data set that will be included in our NDA submission in 2022. Among these additional data was an updated analysis of our primary efficacy trial, Study 101, formally termed the RETRO study for the final CSR that will be included in the NDA. The study data continued to be highly compelling and consistent with The primary efficacy endpoint for Study 101, an updated survival analysis using a time dependent comps regression model shows that the difference in probability of survival between treated patients in untreated natural history control patients was consistent with previously reported results, highly Typically significant at p=0.0007. In addition to this compelling survival benefit, The majority of MT-sixteen twenty one treated patients also demonstrated clinically meaningful improvements in motor, Respiratory and or feeding abilities with some patients reacquiring previously lost motor milestones.

Speaker 3

As the literature describes and our clinical data is confirming, TK2D is a progressive disease in all patients, regardless of age of onset and thus spontaneous improvements in motor milestones are rarely seen in this patient population. We are continuing to make good progress on the clinical and non clinical studies for the NDA submission. The timing for completing Study 107, which is a retrospective chart review study to collect the vital status of any untreated and treated patients in the U. S. And Europe not involved in our sponsored studies has been impacted by the pandemic and has moved into the Q1 of 2022 from the end of this year.

Speaker 3

We're now planning for a pre NDA meeting in Q2 of next year and an NDA submission in the second half of twenty twenty two. In addition to our regulatory progress in the U. S, we are preparing for a scientific advice meeting with EMA Q1 of 2022. With that, let me hand it over to Mike. Mike?

Speaker 5

Thanks, Brad, and good afternoon, everyone. Today, we issued a press release announcing our business and financial results for the Q3 ended September 30, 2021. In the Q3 of 2021, we achieved $21,400,000 in FINTEPLA net product sales globally, representing 20% growth over the Q2 of 2021. This includes SYNTEPLA net sales of $18,400,000 in the U. S.

Speaker 5

And $3,000,000 in Europe. The sustained sales growth reflects significant interest to initiate FINTEPLA therapy we continue to observe across the Dravet communities. We recognized $22,600,000 in total revenue during the Q3 of 2021, which was an increase of 22% compared to $18,800,000 recorded in

Speaker 6

in Q2

Operator

of 2021.

Speaker 5

Of the $22,600,000 in total revenue, again $21,400,000 was in The form of product sales of FINTEPLA and $1,200,000 was related to our collaboration with Nivanshinayaku for FINTEPLA and Dravet syndrome in LGS in Japan. Our total revenue for the corresponding prior year period was $2,900,000 R and D expenses for the Q3 were $33,300,000 a decrease as compared to the $34,400,000 recorded in the corresponding period of 2020. The decrease was a result of overall R and D operating costs decreasing 24 percent to $26,300,000 as some operating costs and activities related to Teva, Dravet and LGS late stage development programs have concluded. This decrease was offset by a $1,700,000 charge associated with an agreement to amend our original FEMTEQUA license and collaboration agreement with regards to revenue sharing on our past and future collaboration with Japan in Japan, sorry, with Nifansha and Yako as well as an increased investment in MP1621 development. SG and A expenses for the Q3 ended September 30, 2021 totaled $39,500,000 and this compares to $24,600,000 for the Q3 of prior year.

Speaker 5

The increase of approximately $15,000,000 is primarily driven by the continued investment related to the launch of the FINTEPLA in the U. S. And Europe. Net loss for the Q3 ended September 30, 2021 was $58,000,000 or $1.04 per share. This compares to a net loss of 60,000,000 or $1.08 per share in the Q3 ended September 30, 2020.

Speaker 5

We ended the Q3 with a strong balance sheet with cash and cash Equivalence and marketable securities totaling $343,000,000 And with that, I'll turn the call over to the operator to start our Q and A session. Operator, can you please open up the line for questions?

Speaker 7

Thank you.

Operator

We will now begin the question and answer session. Our first question is from Paul Matteis with Stifel. Please go ahead.

Speaker 8

Hey, thanks so much for taking the question. Appreciate it. Good to see that new patient adds were relatively stable. I guess the one question I had is just it looks like patient adds to the REMS program were sequentially a little bit lower. Is there anything behind that?

Speaker 8

And maybe could you comment on just what you saw for demand throughout the quarter On a monthly basis and kind of how you feel so far, in October, if you're comfortable? And then just second, I would be curious if you wouldn't mind breaking out U. S. And Rest of World. Thanks again.

Speaker 2

Thanks for your question, Paul. I'm going to hand that over to Ashish

Speaker 3

to address it for you.

Speaker 2

Ashish, are you on mute?

Speaker 4

I'm sorry. So Paul for the Q3, despite the resurgence of COVID, the new patients had We're consistent with the prior quarter. And when you look at month by month, we have seen the consistent growth. We haven't broken that out, but what we have seen is, especially when you say towards the July August when we had a lot of closures Because of the resurgence of Delta variant, we did see little dip there and I think that contributed to where we are. But when you look at the overall numbers of 133 starts, which is a growth over last quarter, has been steady.

Speaker 4

In terms of U. S. And rest of the world, We haven't broken down the patient numbers because as you know, in Europe, number of finding number of patients actually is very difficult because of the Regulations. So every patient number that we are reporting is purely from the U. S.

Speaker 4

Perspective. Hope that answers your question.

Speaker 8

Yes. Thank you so much, Ashish. Can you break out U. S. And Europe in terms of FINTEPLA revenues?

Speaker 5

Yes. I can get that, Paul. This is Mike.

Speaker 6

Go ahead, Mike. Sorry, Gene.

Speaker 5

Yes, in the quarter, dollars 18,400,000 was in the U. S. And $3,000,000 was in Europe.

Speaker 8

Great. Thanks so much. Appreciate it.

Speaker 2

Yes. And Paul, I think you had a question about what are we seeing during this As much as we can say, I just want to remind you that we did move forward with an expanded sales Which became effective in the field at the beginning of the quarter. Obviously, it will take them a few weeks to get acclimated and to start to have conversation with physicians. But we've been, I think, very pleased so far this quarter with our expanded team to increase physician engagement. So that's a very positive sign for us.

Speaker 2

And we know that has led to new prescriptions. So obviously, it's early days still. I'm sure should say that, but we feel encouraged with what our expanded team is doing right now.

Speaker 8

Okay. Thanks, Steve. Appreciate it.

Operator

The next question is from Jason Butler with JMP Securities. Please go ahead.

Speaker 9

Hi. Thanks for taking the questions. Just first one, as you're preparing for the LGS launch, can you just speak to any work that you've done with payers or plan to do with Payers to get them ready for the label expansion. And then on MT-sixteen twenty one, can you just give us some context Any prior dialogue that you've had with regulatory consultants or KOLs in Europe that give insights on how the what the approval path could look like there and what you're looking to get out of a scientific advice meeting?

Speaker 2

Thanks, Jason. I'm going to ask Ashish to take your first question and Brad will get ready to respond to your second one.

Speaker 4

Ashish? Yes. Yes. Jason, from the LGS launch, as I said in the prepared remarks, we are doing 1 on 1 as well as for advisory reports with payers as well as every other stakeholder. What we are hearing consistently That LGS is a challenging and unpredictable disorder and payers do think that the data is meaningful And they are excited and they also are aware and appreciate that LGS is a pretty refractory condition.

Speaker 4

We are going to be doing these conversations. We started them doing last week at AMCP and we will be continuing that at this quarter as well as next quarter. And as soon as the indication is approved, our plan is to reengage them with the label and ensure that the product gets covered.

Speaker 3

Great.

Speaker 5

Steve?

Speaker 2

Hi, over to you.

Speaker 3

Hi, Jason. With regards to MT-sixteen 21 in Europe. We definitely are engaging with multiple regulatory consultant experts and KOLs to help us with our path there. The KOLs are very excited, especially the ones that have utilized MT-sixteen twenty one. As a matter of fact, one of which We'll be on the call on Monday, and she'll be discussing her experience as well.

Speaker 3

I'm very excited about Treating with this medication as they are seeing tremendous benefit as I mentioned in survival, but also other non survival benefit. The regulatory path as I will outline on Monday, maybe a bit different with a different indication, whereas In U. S, we are looking for an indication for all patients. In Europe, the EMA has been a little We're difficult in requesting a little bit more data, especially in the adult onset patients. So it may be a different indication with the younger age of onset But I'll be providing more information on Monday.

Speaker 2

And just to add to that, the majority of the patients that suffer from CK2D are in fact early onset, So children as opposed to later onset. And just reflects the fact, Jason, that we have more data on young onset or early onset we do for later And that's really, I think, the issue that the EMA is addressing right now.

Speaker 9

Okay. That's helpful. Thanks. And I look forward to the call on Monday.

Speaker 2

Great. Thank you.

Operator

The next question is from Yatin Zaneja with Guggenheim Securities. Please go ahead. Yes.

Speaker 10

Hi, this is Eddie on for Yatin. Just a couple for me. Can you talk about what the potential risks are to the LGS filing? Like why is there even a debate around potential Is there anything in the non clinical work that could create a risk? And then assuming approval in LGS, can you talk about the cadence of patient adds next year?

Speaker 10

And do you think that will be affected by the level of off label use you're seeing? And can you remind us what the off label use you're seeing for FINTEPLA in 3Q? Thanks.

Speaker 6

Thank you. Gail, would you like to take the first question?

Operator

Sure. Happy to. Thanks for the question. We are unaware of anything in the filing That would pose such a risk. Certainly, the nonclinical data are straightforward and those studies are complete.

Operator

In terms of the clinical data, the same has happened. We had comprehensive and fruitful discussions with the agency In the pre SMA meeting as well as in the Phase III meeting, we're able to come to good agreement about what should be included in the filing. And of course, we've done diligence and checked our datasets for the submission. So we believe that the submission will be accepted on time.

Speaker 2

And Ashish, could you address the second question?

Speaker 4

Sure. Hi, Eric. In terms of cadence of patient adds after the LGS, I think I'll give you a perspective in terms of number of patients. As you know, in LGS, it's at least 5 times or 6 times larger than Dravet. So you will consequently see the number of patients coming in is going to be higher than what we have for Dravet syndrome.

Speaker 4

As for your second question, will that have any impact in terms of how it is being used right now? One thing I would just I'd like to remind you and everyone is that we have only indication for Dravet syndrome and we promote only in Dravet. However, the data tells us that And as we said previously, it's around 20% of our patients who are being prescribed FINTRALA are for indications other than Dravet. And LGS is the majority of that particular cohort and that stayed true in Q3. Given the significant number of LGS patients and the feedback that we are hearing from the HCPs in the sense that the data is meaningful for them.

Speaker 4

They are excited to have a novel treatment options and they are looking forward to have FINTEPLA in their toolbox Because of these reasons, as and also the current treatments are somewhat inadequate to have a control to have seizure control. We expect that that will not be a huge factor. Thank you so much.

Operator

The next question is from Rohit Bassan with Needham and Company. Please go ahead.

Speaker 11

Hi, this is Rohit on for Serge, thanks for taking my question. Are you noticing any particular prescriber trends for FINTEPLA thus far? And then in terms of payer coverage, should we expect any changes for 2022? Is for 2022.

Speaker 2

Shisho, I'll hand back to you again.

Speaker 4

Sounds good. So in terms of prescriber trend, Rohit, we see consistent increase month after month. And as we said previously, what we are seeing is that physicians as we are connecting with them, they are As we educate them on the FINTEPLA and the transformational efficacy in Dravet syndrome, as they get comfortable with the process of the REMS as well as the entire Zogenix Central, they start using the product. And once they start using the product, it kind of snowballs on that. So we are getting good response from them and we are not only getting prescriptions from the epileptologist, but also from the community neurologists and neurologists at a large.

Speaker 4

So that's something has been very promising and very reassuring. In terms of payer coverage, as I said in my prepared remarks, almost every patient who is prescribed is able to get it and they have been able to get positive determination. And based on the feedback that we have from the payers, Not only for Jave, but also what we are doing on IGS, the conversations we are having. We do not see any roadblocks there and any concerns there.

Speaker 5

Great. Thank you.

Speaker 2

Thank you, Rokhan.

Operator

The next question is from Esther Rajavelu with UBS. Please go ahead.

Speaker 7

Hi. Thanks for taking my questions. I guess a couple for me. 2 on Zogenix and 1 on TK2B. The first one, You referred to increasing prescriptions from new docs.

Speaker 7

So can you give us a sense for whether these are community epileptologists or

Operator

part of larger comprehensive treatment centers.

Speaker 7

And then on the drop off rate, at what point in the therapy are patients dropping off? And if you can share the average duration of therapy, that would be helpful. And then lastly, on TK2D, If you can talk about what steps you may have to take next year as you're going through the regulatory process to identify patients? Or do you expect the majority of these patients to be readily available for treatment if the therapy is approved?

Speaker 2

Yes. Thanks for your questions. I'm going to ask Ashish again to take the first two for you.

Speaker 4

Yes. So as said, I think in terms of the prescriptions from NuDocs, as we add more physicians, Majority of them are community neurologists. As you can imagine, the all epileptologists who were part of our clinical trials and at the large centers Are already prescribing the product and are signed up for the REMS program. So every new prescription that or every new physician that we are bringing in, they are coming from this community neurologist group and also from centers because quite frankly, there are many centers where we did not have access to physicians for a long period of time because of COVID. I think slowly they are opening up And as we make enroll to them, we have seen prescriptions from that.

Speaker 4

As far as your second question on the drop off, Usually it takes around 5 months on average, the range is between 46. So the patients who are dropped off, they Try the product for at least 5 months between 4 6 and then they try and reach the maximum dose that they can tolerate and see the impact. One great thing about FINTEPLA is that as you escalate the dose, it will start working for you. But if it doesn't, you will know that pretty quickly. And I think that has been the range that we have seen.

Speaker 4

And usually they stay on the therapy for that long and then they decide to drop off. And majority of these drop offs are because the product did not work for them. And when you take that out, The patients who have started the therapy, they are still on the therapy. So we have patients who started last August When we launched the product, they are still on therapy and we also have majority of the patients who are part of our open label extension and the Expanded access program still on the therapy. So the average duration is much, much longer at this point in time.

Speaker 4

For TK2, I'll hand it over to Brad.

Speaker 2

I'll take that for Brad. Thanks for the question about trying to increase the number of identified patients. Obviously, when you're in the rare disease space, This becomes a very critical activity in conjunction the work you're doing in development to get the product approved. So we're working in a number of areas right now. I want to sort of give another advertisement for our Investor Day that's coming up next Monday where we'll talk a lot more about this.

Speaker 2

But we clearly have ongoing activities right now relating to increasing disease awareness Also to increase a diagnosis, so helping physicians to be able to identify TK2D earlier. And a key component of that is no surprise is genetic testing. We have a number of activities associated with improving the availability of genetic testing for TK2D. So we'll be thrilled to tell you all about that on Monday.

Operator

Thank you.

Speaker 2

Thank you.

Operator

Our next question is from Tim Lugo with William Blair. Please go ahead.

Speaker 6

Hey, this is Lachlan on for Tim. Thanks for taking the questions. So I was wondering with what sounds like potentially several launches across European markets next year, do you already have the commercial infrastructure you'll need in those markets in place? Or are you going to be building that out as you So we get closer to the reimbursement decisions there. And then secondly, I was wondering for the CDK05 study, are you comfortable sharing any details around the powering of that and maybe some of the assumptions you used there?

Speaker 2

Yes, I can take those for you. With respect to powering, we haven't publicly talked about that. But The type of powering that we use for Dravet syndrome is sort of where I would lead you as opposed to the powering that we needed for LGS. The reason for that is that CDKL5 is a monogenetic epilepsy very similar to Dravet syndrome and we feel comfortable with the N Equals 40 per group and we'll be able to hit significance. And just remind me of your first question again.

Speaker 6

I was wondering with I think you said you have 8 markets in Europe with Reinvestment expected. Yes. Do you already have the commercial infrastructure built out in those countries or are you going to be building that out between now and when you expect?

Speaker 2

Yes, we are Yes. We are playing very much a stage approach. So we have certain functions which are centralized and Important for Europe, based in Maidenhead in the UK. And then we are building our presence in various countries as and when the product So for example, today we have a presence in Germany where we've launched at the beginning of this year. As you know, we have about 10 employees in Germany And that's probably the sort of largest country office that we will have in Germany.

Speaker 2

So we will sort of build out these country presences Once we have reimbursement approved.

Speaker 6

Great. Thanks.

Speaker 2

Thanks for your questions.

Operator

The next question is from Daniel Brill with Raymond James. Please go ahead.

Speaker 5

Hey, this is Alex

Speaker 11

on for Danielle. Thanks for getting our question. So correct me if I'm wrong, my notes for MT-sixteen twenty one said that we'd be getting data by year end and that the NDA was looking to be submitted by the first half twenty twenty two. And today you're now guiding to second half twenty twenty two. Could you share The reason for this delay, and then was that informed by anything that came up in the FDA Type B meeting?

Speaker 11

Then additionally quickly, forgive me if I missed this, how many doctors are now enrolled in the REMS program?

Speaker 2

Question on Study 107 being the regulatory set to the NDA submission. Yes.

Speaker 3

So in our meeting, there are no our Type B meeting, there are no new studies. As I mentioned earlier. The delay is due to Study 107, which is identifying patients outside So Genex sponsored studies, both untreated and treated patients, and the delays due to the pandemic, the sites Having delays in the IRB meetings and they're signing clinical trial agreements. And that's, as you know, A problem across the industry, and that's why it's pushed that out 1 quarter.

Speaker 11

Okay, great. And then just on the RIMs registration, Do you have an updated number on those?

Speaker 2

Yes. At the end of last quarter, there were almost 720 As prescribers that were enrolled in the REMS program and approximately half of those were writing prescriptions for FINTEPLA.

Speaker 11

Okay, great.

Speaker 2

Great. Thank you for your questions.

Operator

Our next question is from Alvin Patel with Bank of America. Please go ahead.

Speaker 12

Hi, good evening. This is Bhavan Patel on for Jason Gerberry. Thanks for taking my questions. So first, on the LGS filing for FINTEPLA, with a few weeks Left ahead of the 60 day deadline to get priority review, I just want to hear your thoughts on what degree of confidence you have that you'll be granted the priority review? And then I also wanted to ask with regards to getting new physicians REMS certified, if you could quantify the number of docs certified at the end of 3Q.

Speaker 12

And so it looks like you had $720,000,000 you just mentioned at the end of 2Q. So how likely are you to meet or even exceed the goal of 1400 by year end, I think given you've mentioned identifying and adding more prescribers to your call list as well. And then finally on MT-sixteen twenty one, just wanted to get your thoughts on the path for market for that asset and What can you tell us about any data that we can expect at the event next Monday? Thank you.

Speaker 2

Thanks for your questions. Gail, would you like to

Speaker 6

take the LGS question? Sure.

Operator

As you know, granting a priority review is an FDA decision. And there are a number of other products that are approved for LGS. However, Looking to the patient community, there is still great unmet medical need among the community to have better treatment options. And FINTEPLA with the unique mechanism of action would provide a brand new alternative. Importantly, this study, the LGS Pivotal study meets the requirements of a pediatric written request from FDA and the standard review time for submission in response to a pediatric written request in 6 months.

Operator

So we believe that we would be granted or have a good chance of having granted the priority review timing because of this response characterization.

Speaker 2

Thank you, Ken. And just in case I misspoke, the number of certified doctors The REMS program was over $7,000,000 to $20,000,000 at the end of September, so at the end of Q3. Maybe Ashish, you can talk about what we're doing to increase the number of prescribers and to the REMS program? Yes.

Speaker 4

So one of the things I'll say is that our focus is on getting more patients Prescribed FINTABLAP at the top of the funnel. And in terms of getting number of physicians 35. And as we said in few quarters back, these physicians get certified once they have a patient and once they realize the value of So what we focus on, on a day to day basis is getting to all these physicians who have Dravet patients and we have just expanded that list from 1400 to almost 3,600. And the goal is to get to them, educate them on the transformational efficacy intravascular syndrome. And once they are educated and then they decide to prescribe, That's the time to get certified.

Speaker 4

Certification is pretty easy process, means people can do it on the cell phone. But our focus is now more on getting physicians to write the product and getting more patients on the top of the funnel. And that's something we have been focused on in last few months. But one thing I can tell you is that between Q2 and Q3, we increased the number More than 75 physicians came on certified for the REMS. And in terms of number of physicians, We had more than 44 new physicians started prescribing in Q3 along with the physicians who are already prescribing.

Speaker 4

Hope that answers your question.

Speaker 12

Thank you. And then I just wanted finally on the MT-one hundred and to just what can you tell us about any data that we can expect at the event next Monday? Thank you.

Speaker 3

Yes. Thanks for allowing me to advertise. I won't give it all away, but the KOLs, one from U. S. And one Both who are investigators will be showing their own patient videos pre and post treatment, which are, you will see, I have one viewing, Quite impressive.

Speaker 3

In addition, I'll be presenting some non survival data. But in addition, As I mentioned, since Study 107 is delayed till next year and also we'll be analyzing the prospective Study 102. Next year, there will be a lot more non survival data as well. So a lot more to come, but I'll be presenting in the KOLs next Monday. There's a it will be an ongoing slew of great data coming out.

Speaker 6

Thanks, Brad. Thank you.

Operator

The next question is from Nina Vitruto Korg with Citi. Please go ahead.

Speaker 13

Hey guys. Thank you for taking my question. So I was just wondering if you could clarify how many patients We're actually on drug actively at the end of September. And then also if you could provide the number of total cumulative starts since launch as of the end of September? That will be great.

Speaker 13

Thank you.

Speaker 6

Vish, I'll

Speaker 2

hand that to you.

Speaker 4

Yes. I'll take that. So, Meenak, we didn't mention On the call, the number of patients, but we continue to add on a monthly basis patients And we will continue to do that in foreseeable future. The focus is for us to get them on the top of the funnel. And we know that once they are prescribed FINTEPLA, almost all of them, if not every one of them It's positive determination and we are able to ship the product in 2 to 4 weeks.

Speaker 4

In terms of the new patient starts, I think that was your second question. We had around 130 started in the Q3. And in terms of cumulative start, that number is well over 860.

Operator

We have a follow-up call a question from Yatin Sannaja with Guggenheim Securities. Please go ahead.

Speaker 10

Yes. Thanks for taking my follow-up here. Just quickly, you guided last quarter for about 50 patient adds per month. And given you I missed that slightly this quarter. I was wondering for 4Q if you're still sort of consistently thinking about 50 patients per month or more of a consistent to 3Q numbers?

Speaker 10

Thanks.

Speaker 4

I'll take that, Steve. So I think one thing I'll say is that In Q3, we are really happy with what we in number of adds because of the resurgence of COVID that we have experienced significantly in July as well as in August. And those new patients when you normalize for that have been consistent with the prior quarters. And in Q4, as you know, we have expanded our team. And the reason for that expansion was we wanted to reduce the geographies, which will allow for more Face to face interaction.

Speaker 4

This will also be we have expanded our call list and the list of prescribers because we are finding more prescribers are caring for Dravet patients and we want to call on them. And because of COVID, we couldn't call on them because the large geographies that expansion helped that. It's very early, but what we are seeing the impact that our ability to make this face to face call with HCP is increasing. We are educating them on FINTEPLA, especially through the speaker programs, but also our 1 on 1 visits. And We are very encouraged by the progress we have made, progress made by the team since start of this quarter.

Speaker 4

And we do see and we do expect to see A very meaningful impact of this expansion in the coming months in Q4 and beyond.

Operator

This concludes the question and answer session. I'd like to turn the conference back over to Doctor. Stephen Farr for closing remarks.

Speaker 2

Thank you for joining us on today's call and particularly appreciate the questions that you addressed that you asked us as well. I'm very happy to report on a strong quarter We're growing FINTEPLA sales, submission of the sNDA for our second indication and close to initiating a Phase 3 trial in the 3rd indication for FINTEPLA. I'm also very pleased to cover MT-sixteen twenty one today and especially excited to have our 1st mitochondrial disease focused investor event next Monday. I hope you're all able to attend this meeting virtually. And thank you all again and enjoy the rest of your day.

Speaker 2

Goodbye.

Operator

This concludes today's conference call. You may disconnect your lines. Thank you for participating and have a pleasant day.

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Earnings Conference Call
Zogenix Q3 2021
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