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Orphazyme

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Orphazyme: First patient dosed in ALS Phase III study

Orphazyme announced late today that the first patient has been dosed in their Phase III ALS study with arimoclomol.

The study is a randomized, placebo-controlled Phase III trial in ALS patients, and will be conducted at 30 sites in North America and Europe. A total of 231 patients will be enrolled, randomized 2:1 to receive arimoclomol or placebo, and patients completing the study will be offered participation in an open-label extension trial. 

The primary endpoint is a combined assessment of function and survival (CAFS) at 18 months, with secondary endpoints measuring survival, change in ALSFRS-R, and slow vital capacity (SVC). Orphazyme estimates that results are expected by H1 2021. 

Orphazyme has an aggressive development schedule in the coming years, where they are investigating arimoclomol in four rare diseases: the two lysosomal storage diseases Niemann-Pick disease type C (NPC) and Gaucher's disease; and the two protein aggregation diseases sporadic inclusion body myositis (sIBM) and amyotrophic lateral sclerosis (ALS). In our view, ALS represents the largest commercial potential at around USD 500 million in top sales. The company has done solid pre-clinical work to back up their hypothesis, and also have patient data from around 100 ALS patients in early Phase II studies. However, ALS is a relentlessly progressing disease that has shown very hard to treat, and we set the probability of success at 35%. 

The ALS field is lacking effective treatments, and only two drugs with a modest survival benefit has been approved. The latest approval, Mitsubishi Tanabe's Radicava, was approved despite a questionable data package, which makes us believe that the FDA could be quite lenient to approve arimoclomol if data is positive. We also believe that Orphazyme's study is designed to show a real benefit and distinguish arimoclomol from the competition, given the data is positive. 

In the near-term, we expect data from the Phase II/III NPC trial during Q3-18. The data is the first for arimoclomol in protein-misfolding diseases and we see the event as a potential key catalyst that could change the investor perception of the stock going forward. We see an interesting case in Orphazyme and a good risk-reward at current levels, with a fair value at DKK 110 per share, and Bull and Bear Case at 200 and 35 per share, respectively. We go into more detail in our recent initiation report (english) (swedish).

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