Snippet roundup: Biomarin gets a double dose of doubt

Welcome to your weekly roundup of EP Vantage’s snippets – short takes on smaller news items.

This week, May 21-25, 2018, we had thoughts on the following: Biomarin expects slow start for Kuvan’s successor; haemophilia A gene therapy doubts hit Biomarin; Dova is as good as its word with FDA approval. 

These snippets were previously published daily via twitter.

Biomarin expects slow start for Kuvan’s successor

25 May, 2018

With yesterday’s US approval for pegvaliase, Biomarin should be able to combat incoming generic competition for its older phenylketonuria (PKU) drug, Kuvan. But a black box warning for pegvaliase – now tradenamed Palynziq – as well as recent cautious comments from management will have investors watching its launch closely. Palynziq’s net cost will be around $192,000 per year versus $150,000 for Kuvan. Kuvan leaves a lot to be desired: of the 11,000 adult PKU patients in the US, only around 1,400 are adequately controlled with the older drug. Still, Palynziq is not perfect either. The risk of anaphylaxis means that the new product will only be available through a REMs programme. And a strict titration schedule, in which patients will receive 2.5mg per week for four weeks before the dose is upped to 20-40mg per day, could lead to a lacklustre launch – management has admitted that revenues will be “fractional” during this titration phase. Biomarin still believes that Palynziq can eventually become a $1bn drug, while EvaluatePharma consensus forecasts 2024 sales of $370m. The company will provide initial launch numbers for Palynziq in the third quarter – which might give a better idea of whether it can live up to these expectations.

Haemophilia A gene therapy doubts hit Biomarin

23 May, 2018

What goes up must come down. Excitement over Biomarin’s haemophilia A gene therapy candidate valoctocogene roxaparvovec (valrox), spurred by last year’s Ash presentation, was tempered by new data from the same phase I/II trial reported yesterday at the World Federation of Hemophilia meeting in Glasgow, UK. Biomarin ended the day down 2% on two-year results with the highest dose of valrox, 6×1013vg/kg, which found a decline in factor VIII expression levels relative to those seen at Ash. While this raised questions about the durability of response to valrox, some investors argued that it could be positive for Biomarin – the Ash update revealed very high levels of FVIII activity, raising concerns about the risk of thrombosis. Indeed, a Biomarin spokesperson stressed to EP Vantage that no participant in the 201 study now had above-normal FVIII activity. While the company claims that activity with the high dose is “settling”, the apparent downward trend suggests that activity might fall further. Yesterday Biomarin also announced plans to expand its phase III Gener8-1 trial of high-dose valrox, from 40 to 130 patients, to assess superiority versus prophylactic FVIII therapy. That trial will now complete enrolment in the first quarter of 2019.

Dova is as good as its word with FDA approval

22 May, 2018

Dova is living the biotech dream, having gone from initial public offering to launch of its first product in less than a year. Now, with US FDA approval in the bag, it needs to set about changing the standard of care in surgical treatment of kidney disease patients with thrombocytopenia from platelet transfusions to avatrombopag. Dova executives have warned that the launch trajectory for avatrombopag, now sold as Doptelet, might not be straightforward, as payers and physicians must be persuaded of its advantages over platelet transfusions – analysts from Leerink recently trimmed their forecast for the pill from $46m to $20m because of this question. Doptelet has the potential to avert tens of thousands of dollars in transfusion costs for each of an estimated 70,000 annual procedures, but Dova will not announce a price until the drug’s June launch date. Shionogi’s lusutrombopag, a potential competitor, is due an FDA decision in August. Dova also expects to file Doptelet with the FDA later this year for idiopathic thrombocytopenic purpura, a space worth more than $1bn a year in which it would be pitted against Amgen’s Nplate and Novartis’s Promacta. The sNDA is based on studies conducted by licensor Eisai; should the regulator demand additional trials, it would add significant R&D costs to Dova’s outlook.

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