Funding over, focus now on pipeline
With a January 2015 IPO and December 2014 mezzanine funding raising a total c $170m, Ascendis Pharma (NASDAQ:ASND) now has breathing space to develop its pipeline. TransCon hGH, in Phase II in children, aims to be the first best-in-class weekly growth hormone injection, and has robust data to date. A key issue of whether the FDA will allow expedited filing via the 505(b)(2) route should be resolved in the next 12 months at the post-Phase II meeting.
TransCon hGH Phase II data expected mid-2015
The TransCon carrier-linker technology enables sustained drug release over a predictable timeframe (daily/weekly/half-yearly) without altering the parent drug. The $3bn human growth hormone (hGH) market is 90% paediatric and the burden of daily injections leads to poor compliance. Interim Phase II data for TransCon hGH showed comparable efficacy to daily injections of genotropin, with low side effects. We expect top-line full Phase II data in mid-2015 and, if successful, a 12-month Phase III paediatric trial could start in mid-2016. Ascendis hopes to file for several indications via the 505(b)(2) regulatory pathway based on a single Phase III trial (using the parent drug as a reference), but this requires FDA/EMA sign-off. The Bang & Olufsen Medicom delivery pen device will also need separate approval.
TransCon treprostinil reformulation delay
TransCon treprostinil once-daily injection would provide significant patient benefits over continuously infused remodulin. After United Therapeutics returned the rights in Q414, Ascendis' own initial Phase I in pulmonary arterial hypertension (PAH) demonstrated the required pharmacokinetics but revealed injection-site tolerability issues, and will require reformulating before Phase I can continue.
Collaborations validate and monetise the platform
Ascendis's collaborations with Sanofi (PARIS:SASY) in diabetes and Genentech in ophthalmology are at a preclinical stage. Ascendis suffered a setback in April 2015 when Sanofi handed back rights to insulin, although it continues with a TransCon Peptide in diabetes. Additional partnering would provide valuable validation of the technology.
Valuation: Pipeline supports c $240m EV
The January 2015 IPO raised c $111.5m at $18/share, leaving Ascendis well positioned to complete TransCon hGH Phase III and to develop much-needed new partnerships and programmes. Clinical data for TransCon hGH, expected in mid-2015, could provide near-term upside.
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