Ann: 2021 Results - a transformational year for Neuren, page-53

* Trofinetide NDA submission is key priority for Acadia this year: Re-confirmed NDA submission timelines + First FDA meeting following Phase 3 results done - Acadia has met up with the FDA once since the Lavender results were released on content and format of clinical data required for their NDA submission. They now comfortable on what FDA wants to see on clinical side in the NDA application. They have another meeting this month (in March) on CMC (chemistry, manufacturing and controls) part of the submission. Company re-confirmed their expectation of mid-CY22 NDA submission, eligibility for priority review and expectation of 1QCY23 for PDUFA date (FDA decision on approval). I expect an end May/ early June timing for NDA submission and a Jan PDUFA date followed by commercial launch in 1QCY23 in US.
* Preparations and investment for a successful commercial launch of Trofinetide is ongoing: Company will leverage existing sales force but also where necessary add dedicated Trofinetide personnel. They have a three pronged approach to commercial readiness which makes sense and gives me confidence that the initial uptake post launch will be rapid:

1. Partnering with and having strong relationships with patient advocacy groups and Rett foundations such as IRSF which provide support to patients, caregivers and the medical community on rett. This gives Acadia access to databases of diagnosed rett patients and networks of physicians who treat rett which will be their initial targets post launch
2. Increase disease awareness which should increase diagnosis rate and also make MeCP2 genetic testing readily available at HCPs (Healthcare providers) for testing of patients suspected of having rett
3. Implement a patient getting started'' support program which will make the treatment a positive experience for patients and their caregivers. This would also include assistance with prior authorisation with co-pay etc.
In addition the company will be engaging with payors to ensure they understand the unmet need in rett for the target patient population (~6k-9k patients in US). They also called out investing in pre-launch infrastructure in CY22 for trofinetide as part of their planned SG&A spend guidance this year.

* Interest and Enthusiasm in rett Community post Lavender results is very pleasing: Acadia mentioned that the enthusiasm following the Lavender results among patients, their caregivers, scientific and medical community and rett support groups has been huge which is a function of Lavender being the first late stage trial in this setting to be successful and now there is hope of having the first drug approved for Rett to treat patients in the not too distant future.

All in all a very positive update from Acadia which shows their commitment and laser focus on getting Trofinetide approved and launch in the US market. First pre-NDA FDA meeting this quarter has been positive and CMC meeting this month should also progress smoothly. Looking at early CY23 launch. As a reminder NEU stands to get US$10m in 1H22 on filing and US$40m in CY23 following launch of the product in US, plus addition share of PRV, sales milestones and escalating double digit royalties. Company is undervalued given the size of the opportunity (orphan drug pricing, close knit community making patients and treating doctors easier to identify) and makes a compelling investment opportunity ahead of upcoming catalysts and what we view as a very high likelihood of FDA approval and a successful commercial launch with rapid uptake. Their second asset NNZ-2591 which is soon to enter Phase 2 and admittedly has a addressable market larger than Trofinetide adds to the appeal.