Snippet roundup: Three approvals and more pricing headaches

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

This week, November 6 to 10, 2017, we had thoughts on the following: Merit tucks in once more; Roche fires first shot in haemophilia battle with Shire; Xeltis gets the biggest European haul this year; new device could stimulate the opioid addiction market; Ultragenyx bags approval but bigger priorities remain; ICER makes cost-effectiveness calculation on gene therapy Luxturna; Moderna clocks up another start in oncology.    

These snippets were previously published daily via twitter.

Merit tucks in once more

November 17, 2017

Merit Medical has built a reputation this year as a snapper-up of unconsidered trifles, and its latest deals are unusual only in that this time the price has hit nine figures. The group has spent $100m all told on various technologies from Becton Dickinson and C. R. Bard that the latter groups are selling to meet antitrust requirements for their pending $24bn merger. Specifically, Merit has obtained BD’s Achieve and Temno biopsy systems and Tru-Cut biopsy needles, and Bard’s Aspira pleural effusion drainage and peritoneal drainage systems. Merit has closed four other deals recently, buying: bone biopsy products from Laurane Medical; assets from Osseon including a steerable kyphoplasty balloon; IntelliMedical, an early-stage company developing a robotically steerable guidewire; and ITL Healthcare, which sells surgical and cath lab equipment. Merit paid about $35m for these four businesses, which have an annual run rate of about $15m in sales, Wells Fargo analysts say. Merit also bought Catheter Connections and the critical care division of Argon Medical Devices in January. Merit’s shares closed up 10% yesterday.

Pink Achieve biopsy device

Roche fires first shot in haemophilia battle with Shire

November 17, 2017

Roche’s attack on Shire’s haemophilia A franchise has begun in earnest with the FDA approval of Hemlibra for patients who have developed inhibitors – antibodies rendering front-line factor VIII clotting factors ineffective. Roche is already looking to the future, pricing Hemlibra in line with therapies for the broader non-inhibitor population. The product is still unproven here, but phase III data are due this quarter, and success could see Hemlibra give Shire’s market-leading factor replacement Advate/Adynovate franchise a run for its money. Shire investors will no doubt cling to the fact that Hemlibra, which contains the active ingredient emicizumab, has a black box warning for thrombotic events. However, this side effect had already been well publicised, and has only occurred in patients also receiving Shire’s bypassing agent Feiba. The safety warning might not limit uptake in the underserved inhibitor population where there are few other options, but could make stable non-inhibitor patients reluctant to switch, particularly given the conservative nature of the haemophilia market. Still, Bernstein analysts believe that it will not be an issue unless thrombotic events are also seen in non-inhibitor patients. This is all academic unless Hemlibra can show a benefit in this population – if it does, the real battle will begin.

Xeltis gets the biggest European haul this year

November 16, 2017

The €45m ($53m) series C round closed by the heart valve developer Xeltis yesterday is the largest to go to a European medtech company this year. The round was led by an unnamed “global strategic investor”, but VC Ysios Capital and existing institutional investors LSP, Kurma Partners and VI Partners also took part. The cash will fund clinical development of Xeltis’s bioresorbable aortic and pulmonary heart valve technologies. These are made from polymers that break down over time – but before they disappear the idea is that they are colonised by the patient’s own cells, leaving behind the tissue that has grown into the structure. The first clinical trial, Xplore-I, of the pulmonary valve is under way in Europe and Asia, and a feasibility study of the same valve, Xplore-II, is running in the US and is due to report next year. Preliminary data on the aortic valve programme were presented at a satellite session at the TCT meeting in October; the valve showed good haemodynamic performance, and fully functional valves were seen in vivo a year after implantation.

New device could stimulate the opioid addiction market

November 16, 2017

The desperation for new options to combat the US opioid crisis has led to the approval of a novel, device-based approach – but the product looks unlikely to dent sales of existing addiction drugs. The FDA has given the go-ahead to Innovative Health Solutions’ NSS-2 Bridge device, a noninvasive nerve stimulator placed behind the ear that sends electrical pulses to cranial nerves, designed to reduce withdrawal symptoms. Unlike long-term drug therapies, the NSS-2 Bridge is only recommended for up to five days during the acute physical withdrawal phase, so it is unlikely to be a threat to entrenched products. However, it could complement them. Alkermes's Vivitrol, for example, requires a seven to 10-day detox before treatment can begin, which can be difficult for patients to stick to. Indeed, this was given as a reason for Vivitrol’s inferior performance versus Suboxone in the intent-to-treat population of the head-to-head X-BOT study published in The Lancet this week – in patients who did manage to start therapy, the two drugs had similar relapse rates. However, the comparison might soon become less important, with new longer-acting options on the horizon in the form of Indivior’s RBP-6000 and Camurus and Braeburn’s CAM2038. As long as the opioid epidemic continues, it looks like there will be room for various approaches, including neurostimulation. 

Ultragenyx bags approval but bigger priorities remain

November 16, 2017

After a tough year, Ultragenyx has finally caught a break with the US approval of its Sly syndrome therapy vestronidase alpha, now trademarked Mepsevii. But with the product’s commercial potential modest, the company has bigger things on its mind, namely an approval decision on its most valuable asset, the X-linked hypophosphataemia candidate burosumab. An EU opinion is expected by the end of the year, and an FDA verdict is due by April, but pivotal data were mixed, giving Ultragenyx a nervous wait. In the meantime, at least the group has shown that it can get a project past the FDA, and it has received another boost in the form of a priority review voucher that could be worth nearly as much as Mepsevii. The product has a net present value of $155m, according to EvaluatePharma sellside consensus, while priority review vouchers have changed hands for up to $350m – although more recently their value has dropped to around $125m. This would still be a welcome boost to Ultragenyx, which had almost $400m in the bank at the end of September but is on the hook for $138m for its acquisition of Dimension Therapeutics. By early next year, Ultragenyx could have turned itself around with two approved products and two priority review vouchers – along with a pullback in the group’s valuation, this prospect could make the company a takeover target itself.

ICER makes cost-effectiveness calculation on gene therapy Luxturna

November 15, 2017

Luxturna looks like an odds-on favourite to become the first bona fide gene therapy to win US approval, meaning that the FDA could once again be kicking the can to payers. At the much-speculated $1m price, Spark’s hereditary blindness agent could cost as much as $741,000 per quality adjusted life year (QALY) gained, the cost-effectiveness organisation Institute for Clinical and Economic Review (ICER) stated in a draft evidence report published yesterday. The ICER report judged that improvement in visual acuity for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy treated with Luxturna at age 15 was worth an additional 1.3 QALYs per patient, and at age 3 an additional 3.3 QALYs. Luxturna looks more favourable if treatment takes place at 3 rather than 15 years of age, and if total societal costs of blindness are incorporated into the calculation. The analysis was limited in its discussion of quality of life (QoL) improvement, in part, ICER says, because improvement in visual acuity is usually used as a proxy for QoL in clinical trials, and because QoL has not been studied in this patient population. The FDA has until January 12 to make its decision after a unanimous advisory committee vote. ICER has given payers a new set of numbers to work with when the Spark medical affairs team comes calling.

Moderna clocks up another start in oncology

November 15, 2017

Moderna is continuing to break out of infectious diseases. Today the group said its oncology collaboration with Merck & Co was moving into the clinic. Some have questioned the group’s estimated $5bn valuation, chiefly based on early clinical products in the less than lucrative area of infectious diseases, so this trial news could help quiet those voices. The 90-patient phase I trial (Keynote-603) will study the cancer vaccine mRNA-4157 as a monotherapy and in combination with Keytruda in solid tumours. Today’s news also follows August’s announcement that the group had moved mRNA-2416, its Ox40 ligand project, into human trials. While the initiation of both studies is positive as these are both in the industry white hot space of immuno-oncology, the next and most important test for Moderna will be providing positive data for both studies. The history of cancer vaccines has, however, been littered with failure, and if Moderna puts one foot wrong the criticism that it has been very good at raising money but not so good at demonstrating meaningful progress with its pipeline will look justified.

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