Timelines., page-3

Morning FP. And Fancy seeing you here away from the coalers (you might still get that boat trip after all)! Below is today's update from Bells. Will try and post the full thing but my IT skills are pretty poor. Enormous promise here. Absolutely enormous. Am heading off on my grand tour shortly so a very early merry Christmas to you all and a big hello to Wasa. A most civilized thread this!
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Neuren Pharmaceuticals (NEU)
Trofinetide shines in Phase II Fragile X trial, licensing prospects improved

Trofinetide successful in Phase II again
NEU has reported positive results from its Phase II Fragile X Syndrome (FXS) trial. The trial met its primary end-point of safety and tolerability with strong efficacy signals. A consistent pattern of clinical benefit of trofinetide (high dose) in improving core symptoms of the disease was observed, similar to that seen in the Phase II Rett trial last year. Importantly, the improvement was in both the clinician as well as caregiver assessments. NEU has Fast Track and orphan drug designation from the FDA for FXS and orphan drug designation from the EMA. NEU will hold end-of Phase II meeting with the FDA to discuss results and development path forward for FXS in 1H16. The company envisages following a similar development path to Rett for FXS.

It’s time to start paying attention to Neuren
Licensing and M&A activity has heated up in the orphan drug space in recent years, with large companies paying multi-billion dollars to acquire smaller companies with one or more orphan drugs nearing commercialization. NEU is particularly well positioned to benefit from this. In our view, the Phase II results provide proof-of-concept (POC) for trofinetide in FXS and more broadly in neurodevelopmental disorders with benefits seen across two different indications now, which significantly de-risks the company. FXS represents one end of the autism spectrum disorders (ASDs) while Rett represents the other. Success in both of these indications now positions trofinetide to be tried across other ASDs and therefore strengthens its value proposition and increases its licensing prospects. We believe that the POC in FXS, coupled with the positive results seen in Rett, will inject additional impetus into NEU’s partnering discussions. We assume trofinetide gets licensed for US$680m in 2HCY16.

Valuation lifted to $0.28/sh, Maintain Buy rating
We have increased the probability of success assigned to FXS (25% vs. 14.5%) and trofinetide’s market penetration for FXS, partially offset by revised launch timeline for it (FY20 vs. FY19). Our DCF valuation has lifted by 12% to $0.28/sh (was $0.25/sh). We retain Buy on NEU. Key catalyst: results from the Phase II trial for TBI in early 2016.

Disclosure: Bell Potter Securities acted as lead manager in the July 2011 and October 2013 placement and received fees for that service.

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0	Recommendation:	Buy, Speculative	Previous Close:	$0.12	Valuation:	$0.28 (was $0.25)