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In case it has not been published....

  1. mw03
    2,135 Posts.
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    14/03/23
    20:01
    Post #: 66764620
    In case it has not been published. https://trailtoatexastrial.wordpress.com/

    Daybue’s Debut Conference Call byAcadia

    Posted on March 13, 2023 by melelllan

    Disclaimer *Any comments on data aremine alone and are not confirmed nor authorized by Acadia*

    Acadia held an investor’s conferencecall today on the FDA approval of Daybue (trofinetide). While the call was forinvestors, there were several areas of interest for parents/caregivers of Rettchildren and adults. There was a summary of Rett symptoms as a review and thenthey hopped into the nitty gritty of approval, side effects, the key points onthe label and monetary issues, including cost, the awarded Priority ReviewVoucher. One of the most important parts of the call was the overview of AcadiaConnect and their financial assistance options.

    *One thing Acadia wants to reassurethe community is that the company is doing their best to respond to community questions. If you would like to ask them a question directly, please send your questions to [email protected]

    Dr. Kathie Bishop. who previouslyheld a webinar with IRSF, jumped into prescribing information and warnings,precautions, and adverse effects. You can see the slides yourselves and listento the call at DAYBUE FDA Approval Call (media-server.com). Some points I wantto highlight, the FDA placed no restriction on severity or gender; this shouldreassure those with an atypical diagnosis and parents/caregivers of boys; shealso noted that improvements were seen across all severities and ages, which isparticularly good news for those with older/adult children. And… QT SYNDROME ISNOT AN ISSUE!!!!!! Fantastic news.

    As for adverse reactions, if you lookat the slide, they are pretty self-explanatory- diarrhea/vomiting topped thelist (not unexpected) I’m surprised by the incident of seizures compared toplacebo as seizures were said to have been improved on average and so this willbe one of the questions I address. The other 3 of note were: Anxiety, fatigue, and decreased appetite. Katelin had increased anxiety at the beginning of her trial, and I surmised at that point since the neurons were now functioning at an increased level there was an influx of stimuli from the environment which most likely led to that, as she calmed down significantly after a short initial period of increased agitation.

    As far as fatigue and decreasedappetite, I can’t help but wonder if it is related to the GI upset anddiarrhea. It would be very tiring to have diarrhea at the rates being mentionedand add in other GI issues, such as gas and reflux, I can definitely see adecrease in appetite. I know that Katelin experienced a fairly severe decreasein appetite in the first couple of weeks of her trial. Weight loss is alsolisted, and I remember Katelin losing weight, but then she had the weight tolose and it was not a concern. I will be curious to see if those who try againat a lower dose and don’t have the severe diarrhea, if the weight loss issueresolves.

    Continuing onward, there are slidesthat discuss the trials, design and results. I’m skipping over this, I’vediscussed both at great length and parents are jumping in now to talk abouttheir experiences, which is better than any slide on results.

    There’s a section called“Post-marketing Commitments” The number one comment is “Renal Impairment PhaseI study- Completed”. This is something the community needs more information on.Daybue is processed through the kidneys and not the liver, I’ve read onDaybue.com that renal impairment is a concern and may be a contra-indication.This will be something parents/caregivers would definitely need to talk totheir children’s doctors about. The other big one here is that the FDA is notrequiring any more clinical studies-HUGE!!! Acadia, themselves, will befollowing patients to get a handle on long-term issues including improvementsand side effects.

    On to “Key Launch Initiatives”!Impressive, is the word that comes to mind. -Educate. Identify. Facilitate.They are off and running, having started this process months ago by contactingidentified hubs-Rett clinics, large hospitals, local neurology offices,insurance companies, Medicaid, Medicare etc. to EDUCATE them about Rettsyndrome and also to educate themselves on how the different agencies operateand their process for approving new meds. IDENTIFY- they have worked very hardto identify the Centers of Excellence (Rett Clinics), over 300 major hospitalsthat the majority of Rett patients are serviced by and continue to identifygeneral neurologists in the community that cover about 15% of patients. FACILITATE- Acadia Connect is a comprehensive program of assistance with insurance, prescriptions, pharmacy 24/7 assistance for questions, a dedicated pharmacy to handle shipping to patients and a care team of Nurse coordinators and Family Access Managers to assist patients and physicians throughout the process of getting and continuing on Daybue.

    Acadia Connect is so massive inscale, that it deserves its own blogpost which I will do next week. As well asDaybue.com which has information that we all need by way of prescribinginformation, side effects and FAQ’s. However, it is already active! AcadiaConnect 1-844-737-2223

    I know the big thing on EVERYONE’smind is how much is it going to cost??? Because DAYBUE is weight based and,taking into account other factors such as rarity, severity etc. of a disorder,the average cost to a Medicaid or Medicare insurer is ~ $ 375,000 per yearwhich is within the range of other rare disease specialty medications; forcommercial insurances the cost is ~$575,000. Importantly, this is NOT whatfamilies will pay. Acadia is committed to “cost should not be a barrier to accessing Daybue,” and Acadia Connect will have a number of patient support services that will, in most cases, keep the out-of-pocket costs very low. Those with commercial insurance will be automatically enrolled in the “Acadia Connect Commercial Co-pay program” and Acadia will pay the entire co-pay at no cost to you. For those without insurance, Acadia has financial programs to assist you, as well. Medicaid/Medicare patients are expected to have a possible nominal fee.

    What were some of the takeaways fromthe Q & A portion:

    *Those currently in trials willcontinue to receive Daybue until they transition to insurance coverage.

    *Doctors can/will/may adjust dosesbased on the individual needs of each patient, meaning they may start lower andmove up to a dose of clinical benefit in coordination with parents/caregivers.That means, those that discontinued the trial/s due to adverse effects of diarrheaand vomiting, if they are willing, can start off at smaller doses and move upas they feel comfortable and with input from their child’s doctor.

    *Other indications are beingconsidered. Right now, Fragile X is the only one being publicly acknowledged; however,there are no clinical trials currently in the works.

    I do have questions for Acadia, forwhich I hope to have a small follow-up.

    *Why was a Renal Impairment Studynecessary if renal insufficiency is not a contra-indication.

    *Why is there no warning on the labelto the effect of “do not take if allergic to any of the ingredients (Red dye 40jumps out)

    *Did any patient who did not alreadyhave seizures develop seizures while on Daybue.

    Thanks for getting this far. Pleasewatch and listen to the conference call yourselves and reach out to AcadiaConnect with questions, or I’m happy to help find your answers if I can.

    NEU Price at posting: $9.97 Sentiment: Hold Disclosure: Held
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