Welcome to your weekly roundup of EP Vantage’s snippets – short takes on smaller news items.
This week, June 4-8, 2018, we had thoughts on the following: Vertex cost analysis could put triplet market on ice; Biogen doubles down on stroke; “Positive” Proteostasis data taken otherwise by investors; Philips pays $300m for EPD and hopes for clearance; Reshape hit harder by one death than Apollo is by four.
These snippets were previously published daily via twitter.
Vertex cost analysis could put triplet market on ice
8 June, 2018
It is a truth universally acknowledged that Vertex’s cystic fibrosis drugs are pricey. But a final report by the Institute for Clinical and Economic Research (Icer) has shown just how expensive the therapies are – and contends that their price tags would need to be slashed by 77% to make them cost-effective. Icer calculated that Vertex’s products cost nearly $1m per quality of life year (QALY) gained versus best supportive care – the figures have come down since the organisation put out draft guidance in March, but only slightly. The organisation recommended that Vertex’s newest CF drug, Symdeko, be priced at around $68,000 per year to achieve a benchmark of $100,000 per QALY. Its actual wholesale cost is around $292,000. The issue of how to pay for CF drugs will only intensify if and when three-drug combinations make it to market. Vertex is at the front of this race, having started phase III trials of two different triplets earlier this year.
Biogen doubles down on stroke
7 June, 2018
Biogen’s failure with Tysabri in stroke earlier this year has not put it off this tricky indication – the company has just acquired a new candidate from the Japanese company TMS. But at just $4m up front, it is only making a tiny bet on TMS-007, a plasminogen activator. Tissue plasminogen activator is the gold-standard therapy for ischaemic stroke, but needs to be given within three hours of onset, which has limited its use. Biogen believes that TMS-007 could extend this treatment window – the project is currently in a phase II Japanese trial evaluating its administration up to 12 hours after stroke onset. As well as breaking down blood clots, TMS-007 could also inhibit local inflammation. It is now the second stroke candidate in Biogen’s pipeline – BIIB093, which is set to go into phase III soon, works differently, targeting the Sur1-Trpm4 channel that mediates brain swelling in stroke. It is unclear whether Biogen will try combining the two agents, but it seems possible. Neither project is currently forecast to be a big seller, with the sellside seemingly more excited about stem cell-based approaches. But with few options on the market, this will change if positive data come through.
“Positive” Proteostasis data taken otherwise by investors
7 June, 2018
Perhaps Proteostasis hoped that releasing data on its cystic fibrosis therapy PTI-801 as the European Cystic Fibrosis Society meeting kicked off would generate positive buzz, but it was not to be. Three different doses of PT-801, a CFTR corrector, in combination with Vertex’s Orkambi, all failed to generate a significant improvement in forced expiratory volume (FEV) over Orkambi plus placebo after two weeks of treatment. The best result came with the middle dose, 200mg, which resulted in a 2.5% increase in FEV. The combination did produce a significant positive change in sweat chloride levels, a biomarker for chloride channel activity. Proteostasis shares fell 36% in early trading today. The Orkambi data were to serve as a stand-in for its own doublet combination of PTI-801 and PTI-808, and triplet with the addition of PTI-428, but absent a benefit over Orkambi it looks like ’801 will struggle.
Philips pays $300m for EPD and hopes for clearance
6 June, 2018
Philips bought ten companies last year, but so far in 2018 the total is just two. The second of these chimes perfectly with the company’s stated aim of moving from imaging to intervention in the cardiovascular field. Philips has paid €250m ($294m) cash upfront for EPD Solutions, whose cardiac imaging and navigation system produces a 3D cardiac image and displays the location and orientation of catheters during diagnostic and therapeutic procedures for cardiac arrhythmias, including cardiac ablation. The tech, CE marked in February, will fit neatly into Philips’ image-guided therapy business, next to the devices it obtained when it spent more than $2bn on Spectranetics. There are other parallels with the much larger Spectranetics deal: that company’s flagship device, the drug-coated balloon Stellarex, was unapproved in the US when the deal was agreed, but came through just a month later. EPD’s imaging system is under 510(k) clearance review by the FDA at the moment, and Philips will be hoping that this is granted in a similarly swift manner.
Reshape hit harder by one death than Apollo is by four
5 June, 2018
Five more obesity patients implanted with fluid-filled gastric balloons have died, prompting the US FDA to issue an alert to doctors, advising them to monitor patients closely for complications and explain to patients which symptoms could be signs of serious problems – and what to do if they occur. The warning follows a similar alert issued last August, which concerned seven deaths among recipients of the same two products: Orbera, developed by Apollo Endosurgery, and Reshape Lifesciences’ ReShape. This time, the single death of a ReShape recipient and three of the four deaths in patients treated with Orbera were attributed to gastric perforation, a known risk of the balloons’ implantation procedure, occurring between one day and three and a half weeks of implantation. Apollo is still investigating the death of the fourth Orbera patient. The FDA said it had approved labelling changes last week “to reflect this information about deaths possibly associated with the devices”. Apollo countered by stating that global rates among Orbera recipients of death, gastric perforation and oesophageal perforation were all 0.01% or lower – and that death rates for gastric banding and bypass procedures are higher than this. Perhaps this was why its shares closed down 1% yesterday, whereas Reshape’s slid 10%.