Operating loss for the first half a year amounted to DKK 164 million (-108), and with no reported revenues. For the full year 2019, the guidance from the company is an operating loss in the span of DKK 315-345. It is according to our estimates as well. The company reported a cash balance of DKK 225.6 million at the half year-end and the guidance is that the cash position will be DKK 110 million by year-end.
Regarding the cash position, Orphazyme announced last week that they have engaged in a loan facility of EUR 9 million. Although we are not too keen on that a non-profit making biotech company engage with a loan structure with a high-interest rate, it is important to stress that it will provide the company with some flexibility as arimoclomol move nearer filing in the US and Europe in Niemann-Pick disease type C (NPC). Regardless, additional funding is needed, and options are under evaluation, according to the company.
In Niemann-Pick disease (NPC), Orphazyme has initiated preparation for filing, in the US as well as in Europe (note and note). This could support filing and approval in 2020. In the second half of 2019, 12-months, open label-extension (OLE) data will be presented after which we will come back with a subsequent update.
Gaucher disease, as NPC, is a lysosomal storage disorder. Orphazyme is running a phase 2 trial in India in treatment naïve patients to enzyme replacement therapy. The trial is a 6-month, placebo-controlled trial in which 39 patients are randomized into four treatments arms, one of them is placebo. The primary endpoint is a change of levels in chitotriosidase, a biomarker, in the blood. Top-line results are expected in H1 20. This is a minor delay from previous communication.
In April, Orphazyme announced the completion of enrollment in the phase 2/3 trial in Sporadic Inclusion Body Myositis (sIBM) (our comment here). This trial comprises 150-patients and is a 20-month, randomized, double-blinded, and placebo-controlled trial that could support registration. The next, important milestone is 12-month interim-results, to be presented in H1 20.
In ALS, arimoclomol is in a phase 3 trial taking place at 30 centers in the US and Europe. A total of 213 patients have been randomized, the last patient was recruited in July this year (note). The successful recruitment means that top-line results will get presented in H1 21, there will be no interim-results from this trial.
We will come back with a full review and update later in the fall. We see an interesting news flow during the remaining 2019 and beginning of 2020, with results or interim results being presented in three of the four targeted indications. Next up is data from the OLE in NPC.
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