Xenon Pharmaceuticals Inc. (XENE) – Transcription de l'appel des résultats du deuxième trimestre 2019

Xenon Pharmaceuticals Inc. (XENE) – Transcription de l'appel des résultats du deuxième trimestre 2019

août 22, 2019 0 Par admin

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Xenon Pharmaceuticals Inc. (NASDAQ:XENE)
Q2 2019 Earnings Call
Aug. 6, 2019, 4:30 p.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good day ladies and gentlemen, and welcome to the 2nd Quarter 2019 Xenon Pharmaceuticals earnings conference call. At this time, all participants are in a listen-only mode. Later we will conduct a question and answer session and instructions will be given at that time. Should anyone require assistance during the conference, you may press * then 0 on your touchtone phone. As a reminder, this conference is being recorded. I would now like to turn the call over to your host, Jodi Regts. Please begin.

Jodi Regts — Independent — Investor Relations and Corporate Communications Consultant

Thank you. Good afternoon. Thank you for joining us on our call and webcast to discuss our financial and operating results from the 2nd Quarter of 2019. Joining me on today’s call are Dr. Simon Pimstone, Xenon’s Chief Executive Officer, and Ian Mortimer, Xenon’s President and Chief Financial Officer.

Following this introduction, Simon will give an overview of Xenon’s clinical programs, and then Ian will review our financial results. After that, we will open up the call to your questions.

Please be advised that during this call we will make a number of statements that are forward-looking, including statements about the sufficiency of our cash to fund operations into 2021, the timing of IND or IND equivalent submissions with regulatory agencies, the initiation of future clinical trials, the efficacy of our clinical trial designs and anticipated enrollment, the potential efficacy safety profile, future development plans, addressable market, regulatory success, and commercial potential of our and our collaborator’s product candidates, the timing of and results from ongoing clinical trials and preclinical development activities, our ability to achieve certain milestones in our propriety development programs, the result of our research and development efforts, the status and timing of the potential addition of new programs to our pipeline in related development activities, the timing of our public presentation and potential publication of future clinical data, and the potential to advance XEN496 to a Phase 3 clinical trial, XEN901 to a Phase 2 or 3 clinical trial, and XE007 into a Phase 2 clinical trial.

Forward-looking statements are subject to numerous risks and uncertainties, many of which are beyond our control, including the risks and uncertainties described from time to time in our FCC filings. Our results may differ materially from those projected on today’s calls. We undertake no obligation to publicly update any forward listing statement.

Today’s press release summarizing our 2nd Quarter 2019 results and the accompanying quarterly report on Form 10Q will be available under the investor’s section of our website at www.xenon-pharma.com and filed with the FCC and on SEDER.

Now I’d like to turn the call over to Simon.

Dr Simon Pimstone — Chief Executive Officer

Thank you, Jodi, and good afternoon, everyone. We’ve now passed the mid-point of the year, and as I reflect on our progress, we remain on track to reach our stated goals for 2019. Advancing our robust pipeline of neurology focused product candidates, including XEN496, XEN1101, XEN901, and XEN007, with multiple products in Phase 2 or later stage development by the end of this year.

Today, I’ll provide an update on each of the clinical-stage programs and highlight some of the important milestone events anticipated this year before opening up the call for questions at the end of Ian’s financial commentary.

Let me begin with XEN496, a CB-7 potassium channel modulator we’re developing for a rare pediatric neuro-developmental disorder called KCNQ2 developmental and epileptic encephalopathy or KCNQ2-DEE and is also sometimes referred to as EIEE7. XEN496 active ingredient is ezogabine, which was previously approved by the FDA as a treatment for adult focal epilepsy but was withdrawn from the market for commercial reasons.

However, published non-controlled clinical studies as well as anecdotal, parental, and physician feedback, suggest that ezogabine may be an efficacious and generally well-tolerated therapy for KCNQ2-DEE, with potential to improve development and cognition, as well as to reduce seizure burden.

We recognize that in order to develop XEN496 for this pediatric population, we would need to take a number of important steps. We obtained orphan drug designation or ODD from the FDA for XEN496 as a treatment for KCNQ2-DEE. We submitted a pre-IND briefing package with our proposal to study XEN496 in infants and children with KCNQ2-DEE with appropriate safety monitoring. In response, the FDA supported our proposed safety monitoring plans, including long-term follow up to monitor potential side effects, and indicated that a single small pivotal trial could be sufficient, provided we see substantial evidence of effectiveness in KCNQ2-DEE.

We also needed to address the formulation challenges of ezogabine, since the original hard-coated tablet was unsuitable for administration to children. It is difficult to cut the coated tablet precisely and obtain an accurate dose. In addition, ezogabine may be unstable in solution when compounding and parents had reported the compounded ezogabine would discolor.

Xenon has developed XEN496 with a focus on addressing these challenges. We have made excellent progress over the past few months and now have a final pediatric-friendly formulation to advance into clinical testing.

The XEN496 final drug product is a granular formulation, packaged as single-dose sachets. Parents or caregivers would tear open one sachet and disperse the granules into the chose semi-solid or liquid food carrier. We designed the formulation with a goal of making certain that XEN496 does not have an objectionable taste or mouthfeel, it’s compatible with standard carrier systems, including plastics, and that XEN496 does not discolor as a granulated form.

We believe our formulation has major product advances over the prior ezogabine tablets, and in addition, may provide us with commercial exclusivity well beyond the orphan exclusive seven-and-a-half-year term.

Standard in-vitro testing has shown that XEN496 acts as an immediate release drug, like ezogabine, with essentially overlapping dissolution curves. We have almost completed animal in vivo pharmacokinetics or PK studies, and again, XEN496 is performing as expected, with PK similar to what was observed with ezogabine. The testing done to date gives us a high degree of confidence to move this final formulation into clinical development and long-term stability studies, which are now under way.

In order to ensure XEN496 is providing adequate drug exposure in humans, we will test this granular formulation in a PK study of healthy adult volunteers, which is expected to be initiated later this quarter. We thereafter intend to file an investigation new drug or IND application in the 4th Quarter, to support the initiation of Phase 3 clinical trial in KCNQ2-DEE.

We continue to support initiatives that may help us in our development, and that may help patients with rare forms of epilepsy such as KCNQ2-DEE. As noted previously, we are a sponsor of the behind the seizures program in the U.S. and Canada, to provide no-cost testing of children up to 60 months of age with seizures. This access to genetic testing allows for the identification of the cause of seizures and the implementation of specific treatment in many treatments. This information helps to build a database of physicians treating these rare disorders, and additionally, patients may be identified for relevant clinical studies.

Recently we were invited to participate in an educational webinar hosted by a patient advocacy group called the KCNQ2 Cure Alliance. We, along with a well-known neurologist in this field, Dr. John Millichap, provided some background on drug development for rare pediatric epilepsies, delivered a status update on XEN496, answered questions submitted by parents in a panel discussion, and discussed an upcoming Xenon sponsored patient survey.

Data from the survey, which will gather feedback directly from caregivers of children with KCNQ2-DEE will be extremely useful and will help inform the clinical trial design for a planned Phase 3 trial in KCNQ2-DEE.

We have also identified other diagnostic companies and academic consortia, to expand our reach, to identify as many KCNQ2-DEE cases as we can, both for our upcoming clinical trial as well as for future commercial reasons. To date, through our interactions with diagnostic companies, we are aware of approximately 800 clinically diagnosed cases. In addition, a recent epidemiological study from Europe reports that the KCNQ2-DEE birthrate of approximately 1 in 17,000, not far off Dravet syndrome, which has been reported as approximately 1 in 12,000 to 1 in 15,000 births. Based on this information, we think that KCNQ2-DEE may be more common than initially thought.

We expect to put more diagnostic partnerships in place to support our plan Phase 3 trial and beyond. We believe given our precision approach as well as our planned administration in the very young, XEN496 in its new pediatric formulation offers children with KCNQ2-DEE a promising chance of seizure control, as well as a chance at disease modification, something not yet observed with any ADE.

Next up in our pipeline is XEN1101, a differentiated, next-generation KV7 potassium channel modulator, being developed for the treatment of epilepsy and potentially other neurological disorders. Site selection and patient enrollment for our XEN1101 Phase 2B clinical trial are currently under way in the United States, Canada, and Europe. The trial is designed as a randomized, double-blind, placebo-controlled, multi-center study to evaluate the clinical efficacy, safety, and tolerability of XEN1101 administered as adjunctive treatment in adult patients with focal epilepsy.

Approximately 300 patients will be randomized in a blinded manner to 1 of 3 active treatment groups or placebo in a 2 to 1 to 1 to 2 fashion, using doses of XEM1101 of 25, 20, and 10 milligrams, plus a placebo. The primary endpoint in this trial is the median percent change in monthly focal seizure frequency from baseline compared to a treatment period of active versus placebo.

To date, feedback from sites is promising. There is strong interest in the XEM1101 study, as investigators support this KB7 mechanism of action in epilepsy. And XEM1101 would represent an only drug in class if approved. Furthermore, there is not a significant burden of competing trials at the moment.

Long-term 6- and 9-month toxicology studies are under way and are expected to support the planned 12-month open-label extension for patients enrolled in the Phase 2B clinical trial. Depending on the rate of enrollment, top line results are anticipated in the 2nd half of 2020.

Turning to the third product in our pipeline, XEN901, a potent, highly selective, NAB1.6 sodium channel inhibitor, being developed for the treatment of epilepsy. We have completed a randomized, double-blind, placebo-controlled Phase 1 clinical trial to evaluate XEN901’s safety, tolerability, and PK in both single ascending and multiple ascending dose cohorts of healthy adult subjects and included a pilot TMS arm.

As with XEN1101, the observed changes in TMS, EMG, and TMS EEG parameters suggest activity of XEN901 in the target CNS tissue. We received important feedback from the FDA regarding the requirements to support advancing XEN901 directly into a pediatric clinical trial, examining its efficacy in pediatric treatments with SEN-8A epileptic encephalopathy, otherwise known as SEN8A-EE.

Similar to XEN496, we recognized the need to create a pediatric friendly formulation of XEN901, and we recently completed the development of a granular formulation with compelling invitro and in vivo characteristics.

We are also in the process of completing juvenile toxicology studies to support pediatric development activities. Again, mirroring the path of XEN496, we intend to run a PK study in healthy adult volunteers with the new XEN901 pediatric formulation beginning in the 3rd Quarter of this year, followed by an IND submission to start a proposed clinical trial in SEN8A-EE patients thereafter. We look forward to keeping you updated on our progress over the coming quarters.

The fourth clinical-stage product in our neurology pipeline is XEN007, with the active ingredient flunarizine. XEN007 is a CNS acting channel modulator that modulates CAV2.1 and T type calcium channels. Other reported mechanisms included dopamine, histamine, and serotonin inhibition.

Available in certain countries outside the United States, flunarizine has been reported to have clinical benefit in treating migraine and other neurological disorders, including hemiplegic migraine or HM, alternating hemiplegia of childhood or AHC, vertigo, and as an adjunctive treatment in certain epilepsy, including childhood absence epilepsy or CAE. The FDA granted us a rare pediatric disease designation for the treatment of AHC with XEN007 and previously granted orphan drug designation as XEN007 as a treatment of both AHC and HM.

In addition, we entered into key licensing and manufacturing agreements to support the advanced clinical development of XEN007.

Various development strategies for XEN007 are under consideration. Given the well-understood efficacy and safety profile of flunarizine, we continued to have discussions with a number of physicians who have expressed an interest in running investigator-sponsored clinical trials.

In the near-term, we anticipate the initiation of Phase 2, investigator-led, open-label clinical trial examining the potential clinical efficacy, safety, and tolerability of XEN007 as an adjunctive treatment of childhood absence epilepsy. Flunarizine has demonstrated efficacy in pre-clinical absence seizure models, which produce seizure characteristics that are consistent with human absence seizures. Both as mono therapy and as a combination therapy, flunarizine significantly reduced the number and duration of spike-wave discharges on EEG when compared to standard of care medicines, including valproic acid or ethosuximide, alone in these pre-clinical models.

With a mechanism of action that is differentiated from other calcium channel blockers, coupled with a vast amount of safety and clinical data generated with flunarizine, we believe XEM007 may potentially offer a better alternative for treating CAE than other anti-epileptic drugs currently available. We look forward to providing more details when this Phase 2 study is up and running.

My status update today reflects our excitement with the continued advancement of the clinical-stage therapeutic products within our neurology focused pipeline, including our progress advancing XEM1101 in our large adult focal epilepsy Phase 2 trial. We also look forward to conducting the important PK studies that I mentioned, so that we can test our new pediatric formulations prior to initiating clinical trials in rare pediatric disorders such as KCNQ2 or SEN-8A encephalopathic epilepsies.

Now I’d like to turn the call over to Ian who will briefly recap our financial position and who will provide some summary commentary before opening up the call to your questions. Ian?

Ian Mortimer President and Chief Financial Officer

Thanks, Simon. Good afternoon, everyone. The specific details within our financial statements are covered in today’s press release and our 10Q filing, so I’ll focus on providing an overview of our cash position, cash runway guidance, and then I’ll conclude with a review of our upcoming milestones.

Cash and cash equivalents in marketable securities as of June 30th, 2019, were $101.8 million. This compares to $119.3 million as of December 31st, 2018. As of June 30th, 2019, we had approximately 25.8 million common shares outstanding, and approximately 1 million Series 1 preferred share outstanding. And these are convertible into common shares on a 1 for 1 basis, at the option of a holder subject to certain limitations.

So based on our current assumptions, and this includes fully supporting the planned clinical development of XEN496, XEN1101, XEN901, and XEN007 as outlined today, we anticipate having sufficient cash to fund operations into 2021. And this excludes any revenue-generating from existing partnerships or potential new partnering arrangements.

And as outlined by Simon today, we look forward to a number of anticipated milestone events including testing our new pediatric formulations of XEN496 and XEN901 in adult PK studies, followed by IND submissions to initiate pediatric clinical trials in KCNQ2 and SCN8A epilepsies. We believe these PK studies will provide important de-risking data, increasing our confidence as we look to move these programs directly into late-stage clinical development.

We look forward to advancing our ongoing XEN1101 Phase 2B clinical trial in adult focal epilepsy and supporting the initiation of an investigator-led, Phase 2 open-label trial of XEN007 as a treatment for CAE in the near term.

Although we didn’t spend time on our discovery research efforts in today’s call, we’ve also made excellent progress on dual inhibitors of both NAV1.6 and NAV1.2. This is another key sodium channel involved in neuro excitation, and we’re now ready to move molecule forward into GLP tox studies. We’ve also made good progress in a number of our other programs within our discovery portfolio.

So before we turn to your questions, I would like to recognize the dedication and determination of our team here at Xenon, who are committed to developing new innovative therapies for patients in need. As you know, 2019 is a year of critical execution across the entire pipeline, and we’ve made good progress across the portfolio in the first half of the year. And we look forward to keeping you updated on progress over the coming months.

Operator, we can now open the call up for questions.

Questions and Answers:

Operator

Thank you. Ladies and gentlemen, at this time, if you have a question, please press * then 1 on your touchtone phone. If your question has been answered or you would like to remove yourself from the queue, you may press the # key. To prevent any background noise, we ask that you place your line on mute once your question has been stated.

Our first question comes from Paul Matteis at Stifel. Your line is open.

Alex Puthumana — Stifel Investment Corp. — Analyst

Hi, this is Alex on for Paul. Just a couple questions. Thanks for the update. So first one, just a quick question on the 007 trial. Just kind of a little bit more color on what you’re expecting to see would be really helpful. Thanks.

Dr Simon Pimstone Chief Executive Officer

Yeah, Alex, it’s Simon. We’re going to provide, I think, a lot more color in the very near term. You know, we expect we’ll have a separate release once the study kicks off. As noted in this update, we expect that to happen very soon.

So, you know, I think what we’d like to do is provide a lot more detail in the coming weeks. I think we’re certainly looking to update on what we see as the medical need, the opportunity in CAE. It’s a very interesting orphan childhood epilepsy. Similar to other epilepsies, 30-plus percent remain refractory. And of course, tolerability concerns with existing drugs.

And you know, this will be an open-label trial, and we’ll certainly go through more around the study design and endpoints on a subsequent call or at least on a subsequent release in the near term.

Alex Puthumana Stifel Investment Corp. — Analyst

Great. That’s helpful. And then one more 1101, I know you’ve guided before that depending on how enrollment’s progressing, you’d expect a readout in 2020. Just wondering if that’s still going as planned. Or if you could provide a little bit more information there. How is that going?

Dr Simon Pimstone — Chief Executive Officer

No, absolutely, guidance remains top line, second off of 2020. We’re still on track. So the study is where it is at the stage a few months in. A number of sites are up. A number are being opened. Patients are being screened, pre-screened. A number of patients have been randomized. So all I is on track at this point.

So I’ve just come back, actually, from Europe. Met with — we have probably about 18 or 19 sites across Europe. And there’s a very, very significant amount of excitement for the study. As I mentioned in the update, one of the good news elements, which is obviously out of our control, is the competing landscape. And so there’s actually a very limited burden of studies in focal epilepsy at the moment, which we’re fortunate to be launching a trial into that.

And of course, because this is the only drug of its mechanism, certainly lots of interest. You know, the feedback, I would say, pretty much universal from the investigators I’ve talked to was an improved ezogabine would be a very, very interesting product for focal epilepsy. Again, significant feedback on people’s experience with ezogabine in focal epilepsy. In many patients, the drug worked extremely well, was limited by its PK, its pigmentation. They feel that this mechanism is a very well-validated mechanism, and it has shown very good promise with a prior drug.

So, you know, look, we’re not obviously updating recruitment numbers on a weekly, monthly, quarterly basis. We are where we expect to be, and we look like we’ll be on track for top line, as we said, back off of 2020 at this point.

Alex Puthumana Stifel Investment Corp. — Analyst

Great. Thanks for the update.

Dr Simon Pimstone Chief Executive Officer

Yeah.

Operator

Thank you. And our next question comes from Maury Raycroft from Jeffries. Your line is open.

Saul Nerron Jefferies Financial Group — Analyst

Yeah, hi. This is Saul Nerron for Maury. The first question I have is for the mind’s sake. So you are like planning the final PK study in 3Q ’19, so moving from there into the pediatric population, how do they think about that? Like do you go like at some level take doses, and then migrate it up? Or like how well is the physiology between pediatric and adult correlation?

Dr Simon Pimstone Chief Executive Officer

Good question. The correlation is good, and that’s been shown with ezogabine in its previous drug product form when it was on the market. There was some off-label use of ezogabine tablets which were compounded in children. And in fact, they published on the PK and showed that sort of dose for dose exposures in the children were similar to dose for dose for adults. So there doesn’t appear to be any significant difference between children, infants, and adults with respect to the ADME properties of this active ingredient.

We’ve now shown how in-vitro and in vivo PK in animals, the performance of the new formulation looks pretty much identical to what the previous ezogabine tablets were reported on, which is also very important. So we do predict good absorption and ADME properties of the drug, similar to the previous ezogabine with this new formulation.

That all being said, even though we’ve got sort of a target exposure range based on previous literature for the drug, we will, as you point out, run a dose titration model. The final design hasn’t been completely settled. We’ve got still a few months to finalize this, and this is an active discussion. Obviously, knowing the PK in the healthy volunteer study is going to be important. But we do expect we’ll start at a low milligram per kilogram dose, with dose titration every few days, up to a maximum tolerated dose or a target dose of in and around 20 milligrams per kilogram. Which was, again, the experience that was published in children who took ezogabine.

So, I think that really covers your question. What exactly that dose titration schedule for 486 looks like at this point, we haven’t finalized. But as I said, will be dose titrated starting at a low daily milligram per kilogram dose for a few days.

Saul Nerron Jefferies Investment Group — Analyst

Okay, yeah, cool. Thank you very much. And one more question on 1101. So you’re running the 6 to 9-month tox studies that are under way. So is there any plans for data disclosures from these studies?

Dr Simon Pimstone Chief Executive Officer

We haven’t made plans at this point, so probably not. But clearly, the tox studies will have to support the open-label extension. We expect that we’ll have patients who would be looking to move into the open-label extension this year, in 2019. And so, the tox studies clearly would have to support that.

So we don’t expect at this point to be publishing or presenting on the tox findings. But certainly, the market would be updated as to the ability for those tox studies to support the open-label extension. And that would be sufficient for our clinical program.

Saul Nerron — Jefferies Investment Group

Okay. Thanks for taking my questions.

Dr Simon Pimstone Chief Executive Officer

Not at all.

Operator

Thank you. As a reminder, ladies, and gentlemen, that’s *1 to ask a question. Our next question comes from Antonia Bulverthena from BloombergSen. Your line is open.

Antonia Bulverthena — Analyst — BloombergSen

Hi, Antonia on the line for Dave. My first question is related to XEN007. Just wondering, your choice to pursue childhood absence epilepsy over hemopoietic migraine and AHC, just wondering if that is primarily because you believe the mechanistic rationale is stronger in this indication? Or is it more due to commercial indication?

Dr Simon PimstoneChief Executive Officer

No, I think there’s a number of factors. The others still represent interesting indications. And in fact, we’ve just had a significant investigator meeting on AHC with regards to 007. Antonia, it was really certainly good mechanistic support, both pre-clinically as well as clinically. Secondly, it’s a sizable orphan population. Thirdly, epilepsy is obviously a strong focus for us, so there’s leverage off of some of the work we’re currently doing in terms of internal expertise. And lastly, it was just in terms of the ability to get the study we wanted to do set up, up and running. We could move quickly, swiftly. And we’ve got a top investigator who was very interested in running an investigator-led study.

So it was sort of alignment of all of these factors. Good size patient population. Doable study. Feasible. Endpoints well understood. Of course, coupled with good mechanistic support for the calcium channel mechanism in this condition, both at the pre-clinical level and at the clinical level. Which led us to at least kick off the program. That’s not to say that 007 won’t advance in other areas over time. Of course, we continue to explore those. Those are obviously more complex clinical studies, and this was just easy to get up and running.

Antonia Bulverthena — Analyst — BloombergSen

Okay, great. Thanks. And then, for XEN496, just a couple questions. Firstly, is there any potential to get IP for your new formulation? And also, how quickly does the blue color appear in patients in a clinical setting?

Dr Simon Pimstone Chief Executive Officer

Right. So I’ll answer both those questions. Absolutely we believe that this new formulation granulaire se prêtera à une nouvelle propriété intellectuelle. Cela nous donnera donc une vaste exclusivité commerciale bien au-delà du type d’orphelin actuel, orphelin de sept ans et demi, que nous espérons obtenir. Alors oui, nous pensons en fait que les probabilités de délivrance sont très élevées et je pense que nous obtiendrons une nouvelle propriété intellectuelle. Voilà donc le point n ° 1. P>

En termes de couleur bleue, il y a vraiment deux éléments. L’un est la couleur bleue de l’ingrédient actif pharmaceutique actuel, l’ezogabine, qui se manifestait rapidement avec l’ezogabine. En d’autres termes, lorsque les anciens comprimés étaient composés, les parents nous disaient souvent qu’ils verraient les comprimés virer au bleu. D’une couleur blanche à une décoloration bleue ou but. Et en fait, nous avons fait ces expériences. Lorsque vous mettez l’ancienne solution en ezogabine et que vous l’exposez à l’oxygène, elle devient violette. P>

En ce qui concerne la décoloration de la couleur chez l’homme, l’ézogabine a généralement pris quelques années avant la pigmentation. Ainsi, comme vous le savez sans doute probablement, le premier médicament a été approuvé en dehors de son programme pivot d’environ 1 500 patients sans problème de pigmentation au moment de l’approbation. Les premiers cas étaient une fois que les patients prenaient le médicament depuis 2 ans ou plus. La pigmentation chez l’homme prend donc plusieurs années, mais la pigmentation a été remarquée avec l’ingrédient actif. P>

Avec les nouvelles formulations 496, nous pouvons au moins affirmer qu’il n’ya pas de décoloration du produit pharmaceutique en solution par rapport à la performance de l’ezogabine. C’est donc une différence très significative. Si cela finit par pigmenter, le temps nous le dira. Mais je pense que nous avons considérablement réduit le risque de pigmentation, n ° 1. P>

Non. 2, pour l’indication que nous cherchons, il s’agit d’un problème purement esthétique. Nous ne pensons pas que le risque de pigmentation constitue un passif commercial. Il n’y a eu aucun cas, aucun, dans la littérature rapporté de toxicité visuelle avec l’ézogabine. Même lorsque la pigmentation a effectivement eu lieu, la vision est préservée, aucun cas de toxicologie autre que la pigmentation elle-même en termes de risque de pigmentation entraînant une responsabilité. P>

Donc, pour cette indication grave en pédiatrie, même si 496 se pigmentaient, et je vous ai expliqué pourquoi, selon nous, la probabilité est plus faible, je pense que le risque est, d’un point de vue commercial, négligeable, le cas échéant.

Antonia Bulverthena – strong> Analyste – BloombergSen em> p>

Et je n’ai plus qu’une dernière question à poser sur 496. Vous avez mentionné une approbation potentielle si vous constatiez une amélioration substantielle des saisies. Je me demandais simplement quelle était votre cible. P>

Dr Simon Pimstone strong> – Directeur général em> p>

Nous n’avons pas encore dit cela. C’est une bonne question. Je veux dire, cela va vraiment, en fin de compte, avoir besoin d’une sorte de participation de la FDA. Mais vous savez, je pense que la question que vous posez est exactement la bonne et que nous passons pas mal de temps là-dessus, comme vous pouvez l’imaginer, c’est si, en raison de la rareté du désordre, si nous sommes réellement capables de courir essai sera limité à un, appelons-le une petite étude pour le moment, en termes de nombre de patients. Probablement quelques dizaines de patients. P>

Nous devrons être en mesure de nous convaincre ainsi que les régulateurs de l’activité du médicament dans un petit échantillon. Nous devons créer une sorte d’effet et de taille d’effet qui, à notre avis, et la FDA, et KOL le croira, auront un effet significatif sur ces enfants. P>

Maintenant, quand vous regardez les études sur l’épilepsie focale générale, ce nombre est souvent compris entre 15% et 20%. La différence entre un placebo et un actif dans l’épilepsie générale, appelons-le un essai d’épilepsie focale est souvent plus ou moins, vous savez, un placebo à 20%, une activité active de 35% à 40%. P>

Maintenant, avec les épilepsies pédiatriques plus graves, ce delta peut changer et devrait. Je pense donc qu’il suffise de dire que 15 à 20% ne suffiront probablement pas. Nous ne le savons pas. Mais je ne pense pas que vous ayez nécessairement besoin d’une différence de 60% ou de 70% par rapport au placebo. P>

Donc, c’est probablement quelque part entre ce que l’on observe habituellement et ce qui est maintenant attendu dans les épilepsies plus graves. Ce qui est probablement autour de 30% à 40% du delta. Quelque part dans cette gamme, Antonia, mais je n’ai pas de numéro fixe pour vous aujourd’hui. P>

Antonia Bulverthena – strong> Analyste – BloombergSen em> p>

d’accord. Merci beaucoup. P>

Dr Simon Pimstone strong> – Directeur général p>

Oui. p>

Opérateur strong> p>

Merci. Et à ce moment-là, je ne montre plus aucune question de la file d’attente. Je vais devoir retourner l’appel à Jodi Regts. P>

Jodi Regts strong> – Indépendant – Consultant en relations avec les investisseurs et en communication d’entreprise em> p>

Merci à tous d’être venus nous rejoindre aujourd’hui. Nous sommes impatients de vous tenir au courant de nos progrès. P>

… p>

Opérateur, nous allons maintenant mettre fin à l’appel. p>

Opérateur strong> p>

Mesdames et messieurs, merci de votre participation à la conférence de ce jour. Vous pouvez maintenant vous déconnecter. Tout le monde passe une merveilleuse journée. P>

Durée: 40 minutes strong> p>

Appelez les participants: h2>

Jodi Regts strong> – Indépendant – Consultant en relations avec les investisseurs et en communication d’entreprise em> p>

Dr Simon Pimstone strong> – Directeur général em> p>

Ian Mortimer strong> – président et chef de la direction financière em> p>

Alex strong> em> Puthumana strong> – Stifel Investment Corp. – Analyste em> p>

Saul Nerron strong> – Groupe financier Jefferies – Analyste em> p>

Antonia Bulverthena – strong> Analyste – BloombergSen em> p>

Plus d’analyses XENE p>

Transcriptions d’appels de toutes les recettes p>

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br> Lorsque les génies investissent David et Tom Gardner ont un indice de stock, il peut être rentable d’écouter . Après tout, le bulletin d’information qu’ils utilisent depuis plus de dix ans, Motley Fool Stock Advisor em>, a quadruplé le marché. * P>

David et Tom viennent de révéler ce qu’ils considéraient être les

* Le conseiller en actions est revenu à compter du 1er juin 2019 span> em> em> p> div>                                                                                                                                                                             span>                                      Transcription de Motley Fool

ne occupe aucune place dans Motley Fool n’a pas de position sur un des titres mentionnés, mais possède une politique de divulgation de l’information. . em> p> « > span>                                                                                                                                                                                                                                       section>                                                                                            div>


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