Ann: 2021 Results - a transformational year for Neuren, page-52

Trofinetidepoints from ACADIA’s Earnings Call earlier today as per Seeking Alpha’stranscript and in case you can’t get past the paywall if you’ve exhausted freearticles - sorry for length

StephenDavis

In preparing for our NDA submission, we scheduled 2 meetings withthe FDA. We've already met with the FDA to review the overall content andformat of the clinical data to be included in our NDA submission. The secondmeeting scheduled for March will be dedicated to covering similar ground on CMCaspects of the submission. We are planning for an NDA submission around midyearand expect a priority review with the PDUFA action date in the first quarter of2023.

BrendanTeehan

Now let's discuss the opportunity with trofinetide for thetreatment of Rett syndrome. With strong positive Phase III results in hand, weare now focused on preparing for a highly successful commercial launch. Today,we have developed partnerships with engaged and motivated patient advocacygroups within the Rett community and are working with the Rett Syndrome Centersof Excellence as we work to identify patients who can benefit from trofinetideat launch. We are evaluating opportunities to increase education and genetictesting to further support patient identification. And we will leverage ourexisting sales force and infrastructure while investing appropriately infurther dedicated customer-facing roles for trofinetide where necessary.

SrdjanStankovic

Let's turn to our trofinetide NDA, starting on Slide 18. Rettsyndrome is a very serious and rare disorder for which we estimate there arebetween 6,000 and 9,000 patients in the United States.

Importantly,trofinetide showed improvement across all 8 domains of the RSBQ. In addition,the study achieved statistical significance versus placebo on its key secondaryefficacy outcome, another caregiver assessment focused on the patient's abilityto communicate. This has the potential to address a significant challenge forparents and their children whose lack of ability to communicate fullyinterferes with many aspects of their daily living. Importantly, the efficacyresults were consistent across all age groups and severity of disease.

Werecently met with the FDA to review the overall content and format of theclinical data to be included in our NDA submission. We have a dedicated CMCreview meeting in March and plan to submit our NDA around midyear.

Q&A

RituBaral

It'son trofinetide for Rett. Have you guys discussed functional unblinding analysisas part of the FDA meeting that you had on the data? Or was it submitted aspart of the statistical analysis plan? And then further, can you just gothrough what the gating items are aside from the CMC link for that submission?

Srdjan Stankovic

Yes.Thanks, Ritu. As a part of our briefing documents for the meetings, we havesubmitted a detailed information on the results from the -- our pivotal trialand from the results of the trial. So a complete and full both efficacy andsafety information was available to the FDA in preparation for the meeting.

Toyour question whether this potential functional unblinding was discussed, theanswer is no. That question did not -- was not raised in the meetings and thecontacts that we had with the FDA. So we did not discuss that at all.

Stephen Davis

Justto recap the analysis that we've done on this point. Sorry, Ritu.

Yes, Serge, maybe you can just give a veryhigh-level recap of the analysis that we've done on that point.

Srdjan Stankovic

Yes.I mean we did a very detailed analysis, both evaluating the efficacy data forpatients with and without diarrhea either of the co-primary outcome measures.We also looked at the scatter plots. We looked at the responder analysis. So wedid look -- we did perform a very detailed analysis and all of them point inthe same consistent conclusion that there is no indication of any bias in termsof the assessment of efficacy in regard to diarrhea as adverse event. But as Isaid that question was not raised and there was no discussion on that questionat all in the meetings.

Ritu Baral

Gotit. Were those analysis -- Serge, were those analysis prespecified andsubmitted as part of the statistical analysis plan?

Srdjan Stankovic

Thoseanalysis will be part of our NDA submission. But obviously, statisticalanalysis plan is always submitted prior to unblinding the data. So there wasn't-- these analyses were not part of the statistical analysis plan because therewasn't any information or based on which you could anticipate those analysis.

Jeff Hung

Foryour collaboration with Stoke on Rett, can you just remind us of how that maybe differentiated from trofinetide and how different might the patientpopulation be than those that you could treat with trofinetide?

Srdjan Stankovic

Yes.The Stoke technology addresses about 1/3 of the patient that have -- thatdisplay genetic hypermorphism. So that's not the entirety of the population,but rather as I said, about 35% to potentially 40% of the overall population ofthe patients. And the difference in technology is that based on thisintervention, the increase in the protein is actually on the gene that is not-- that is performing suboptimally. So from that perspective, it's a completelydifferent approach than what in general is approached with trofinetide.

Stephen Davis

Ithink just to -- maybe just to add a couple of indications there. One is withtrofinetide we have rights to North America and with Stoke collaboration ourrights are worldwide in that program. And then the short version is with theapproach that we're taking in collaboration with Stoke, there's a greaterpotential for disease modification. So potential for an even greater or moreprofound effect with that type of approach. So we're -- we think that programis a very, very nice complement to the franchise that we're building aroundtrofinetide.

MatthewNirenberg

Andthen separately, we were just curious about any physician feedback you mighthave gotten so far after the Phase III Lavender results for trofinetide and howyou're currently thinking about the market opportunity in terms of drivingdiagnosis rates. That would be really helpful.

Srdjan Stankovic

I can say that weare quite pleased with the level of enthusiasm and excitement that we areseeing with treating physicians and experts out there in regard to the data. Imean it's, to some extent, understandable, considering that so far, therehaven't been successful late-stage programs in Rett syndrome, for treatment ofsymptoms of Rett syndrome. So from that perspective, there is quite a bigexcitement not only in the medical community, in the scientific community butalso in the parents and caregivers and community.

Sobottom line, we are very enthusiastic to see the level of excitement andenthusiasm across and look forward to the NDA and approval.

Brendan Teehan

AndI just want to echo, Serge's comments. The Rett treatment community has givenus very encouraging feedback on what they've seen of the Lavender results. Butto address your question specifically, Matt, around Rett diagnosis. As with anumber of rare pediatric diseases, the pursuit of a diagnosis has increasedpretty dramatically over time. There is already a high diagnosis rate for Rett.So we have access to databases that will show us physicians and numbers ofpatients that they have that are already diagnosed with Rett. There's also beena significant increase in genetic testing in recent years. And we, as anorganization, will work diligently and are pursuing a number of avenues toeducate on MECP2 testing and then looking at potential partnerships to haveconfirmatory MECP2 testing for suspect Rett patients post launch.

JasonButler

Justwondering if you can speak to the commercial prep work, you'll be doing in Rettthis year. Obviously, I assume that the infrastructure outbuild will mainly betowards the end of the year and into next. But from a medical educationperspective, just can you walk us through the work you're going to be doing toget the market ready for the drug?

Brendan Teehan

Myhumble apologies, Jason, thanks again for the question. Yes, obviously, we'revery excited about trofinetide opportunity. And we have been preparing even asthe Lavender study was ongoing in Phase III for a favorable result. The initialwork, obviously, we want to work with the payer community to make sure thatthey're aware of the burden of illness, understand the unmet medical need andthe size of the patient population, roughly 6,000 to 9,000 patients. We alsohave developed over time strong relationships with the foundations that supportpatients, caregivers, and the health care community, which has helped us tounderstand where we're going to find our Rett patients and the volumes ofpatients that we're going to want to support.

Andthen broadly, another area that we'll focus on are our white glove supportservices to be prepared by the time of launch, to support families, not onlygetting started on trofinetide, but to have a successful clinical experience,all of the support that they need from a clinical perspective as well as priorauthorization assistance with co-pay and so on. So those are probably theprincipal areas where we'll focus as they relate to the product.

Forthe disease state, obviously, this is a tightly knit community. There's a highlevel of awareness of Rett syndrome. We will work with the community to makesure that where necessary confirmatory MECP2 tests will also be available forsuspected patients with Rett at any of their HCPs.

Events& Presentations | Acadia Pharmaceuticals Inc. (acadia-pharm.com)

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