We initiate coverage today on Prosensa with a $ 33 target price and Buy rating. Later today we publish our 64p initiating company note. Prosensa is a European (Dutch) biotechnology company focused on the discovery, development and commercialization of RNA-modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a focus on neuromuscular and neurodegenerative disorders.
By leveraging its RNA-modulating technology, Prosensa has become a frontrunner in the field of RNA-based therapies. Its most advanced product, drisapersen, could be the first marketed drug to stabilize the progression of a subset of patients with Duchene Muscular Dystrophy (DMD).
Drisapersen, together with other DMD pipeline products, is licensed to GSK, thereby not only validating the product’s potential (€ 900m by 2020), but also the remainder of the DMD pipeline. The FDA recently confirmed drisapersen’s innovative character by granting it the ‘breakthrough status’.
The product has shown statistically significant and clinically meaningful results in the 6-minute walk test versus placebo. Long-term follow-up studies confirmed stabilization in patient’s walking ability while fierce declines would be expected without treatment. Early October, GSK is due to report phase III results, which if positive, could allow for market entry by YE14 or early 2015. 3 other clinical products, targeting other DMD subpopulations could enter the market in the 2017/2018 timeframe.
Prosensa recently became public with a listing on NASDAQ, an unusual event for a Netherlands based biotech company, though at the same time a proof of the strong investor interest. The company raised $ 90m during its 10x oversubscribed IPO, resulting in an early July cash position of $ 122m or € 94m, versus a first half operational cash burn of € 10.5m.
Different valuation techniques point to an average Prosensa valuation of $ 31/sh. Given strong news flow and pipeline progress is expected in the coming months, we put our 6-month target price at $ 33/share, implying around 35% upside versus current trading levels. Hence, we initiate coverage with a BUY-rating.
25-27 September: Drisapersen 3.5year open label data
1-5 October (WMS meeting): Drisapersen phase II dystrophin details
1-5 October (WMS meeting): PRO044 phase I/II data
4Q13: Drisapersen: phase III (DMD114044 study) and new phase II (DMD114876) 24-week data trial (US patients)
October 2013: Feedback Sarepta’s FDA CMC meeting.