Price of UniQure gene therapy tops €1.1M, fund backs RET inhibitors, Promethera bags €25M

Welcome to the latest edition of our weekly EuroBiotech Report. Chiesi and UniQure ($QURE) have raised the bar for rare drug prices, setting the tone for the blossoming gene therapy sector in the process. The pair's gene therapy, Glybera, is to go on sale in Germany for €1.1 million ($1.4 million) per course. The rest of the European biotech scene was awash with big figure headlines, too. Neil Woodford and Cell Medica topped the bill with a £200 million fund and £50 million Series B round, respectively, but Promethera Biosciences, Nanobiotix and the Cancer Research UK (CRUK) Manchester Institute Drug Discovery Unit also got in on the action. Promethera bagged €25 million--and began looking for more--to finance the advance of its orphan drug pipeline into late-stage trials. Nanobiotix raised €10 million to fund its go-it-alone U.S. development strategy. And CRUK's Manchester Institute pocketed an undisclosed slice of a £50 million fund to move a RET inhibitor into Phase I. BiondVax became the latest Israeli biotech to be linked with a Nasdaq IPO. Ablynx (EBR:ABLX) detailed plans to seek conditional approval of its therapy for thrombotic thrombocytopenic purpura. And more. Nick Taylor (email | Twitter)

1. Chiesi slaps record €1.1M price tag on a course of UniQure's gene therapy
2. Promethera pockets €25M and begins hunting for more to fund PhII/III trial
3. Nanobiotix raises €10M to fund solo U.S. development strategy
4. Joint EU-CRUK VC unit commits cash to move RET inhibitors into Phase I
5. Ablynx sets sights on conditional approval of blood disorder drug
6. Report: BiondVax mulls following Israeli biotech peers to Nasdaq

And more >>

Chiesi slaps record €1.1M price tag on a course of UniQure's gene therapy

Chiesi and UniQure ($QURE) just set the benchmark for gene therapy prices sky high. Parma, Italy-based Chiesi has slapped a €1.1 million ($1.4 million) price tag on lipoprotein lipase deficiency (LPLD) treatment Glybera, making it the most expensive rare disease drug in the world. 

UniQure CEO Jörn Aldag

Glybera won approval in Europe two years ago but the need to generate 6-year follow-up data has delayed its launch. With that data now available, Chiesi--which is handling market access and paying UniQure net royalties of up to 30%--has filed a pricing document in Germany, Reuters reports. Chiesi is charging €53,000 per vial. And with 21 vials needed to treat the average patient, the cost of a full course of treatment is north of €1 million.

The price is valid in Germany for one year. In the longer term, the outcome of an assessment of the benefits by G-BA--the body that has infuriated Eisai and other drugmakers--and negotiations with statutory health insurance funds will dictate the figure. Germany has traditionally been used by other European countries as a benchmark for their own prices. As such, the effects of its increasingly tough line on which drugs it is willing to pay for have echoed across the continent.

Bluebird bio ($BLUE), Bayer, Novartis ($NVS), Sanofi ($SNY) and other drugmakers that think gene therapy's time has come--and have backed up their hunch with R&D investments--will be keeping tabs on events in Germany. Chiesi argues the price is justifiable given the long-term efficacy data. G-BA will be the first to assess whether the claims stack up, but other negotiations with HTAs and payers await. UniQure is aiming to win FDA approval for Glybera in 2018. - read Reuters' article

Back to top

Promethera pockets €25M and begins hunting for more to fund PhII/III trial

Having gone from founding the company to the cusp of Phase IIb/III in five years, the team behind Belgian biotech Promethera Biosciences has topped up its bank account with a €25.3 million ($31.6 million) Series C round. The money will go toward a prospective, open label trial of Promethera's lead drug in pediatric patients with urea cycle disorders.

Mont-Saint-Guibert, Belgium-based Promethera received clearance to start the trial last month and has now raised money to finance the work. The Series C round attracted two new backers--SFPI-FPIM and SMS Investments--that join Vesalius Biocapital, Boehringer Ingelheim Venture Fund, Pall-ATMI LifeSciences and Shire ($SHPG) on the list of investors in Promethera. The financiers are already on the hunt for fresh capital, too.

Vesalius partner Alain Parthoens

"We hope to raise additional funds in the coming months in order to maximize the potential of the product and develop HepaStem in large acquired adult liver indications," Alain Parthoens, partner at Vesalius Biocapital, said in a statement. The European Medicines Agency (EMA) and FDA have both granted HepaStem--a therapy based on allogeneic stem cells from healthy adult human livers--orphan status as a treatment for various forms of urea cycle disorder and Crigler-Najjar syndrome.

Promethera has picked urea cycle disorders as its indication for the next phase of development. The disorders are caused by eight metabolic glitches, each of which stops the normal transfer of nitrogen into urea. All eight of the disorders share some common characteristics, which Promethera thinks it can address by processing and administering adult liver cells. The idea is being tested in 20 patients with urea disorders. Promethera is looking at changes in urea formation in the year after treatment as an indicator of efficacy. - read the release

Back to top

Nanobiotix raises €10M to fund solo U.S. development strategy

Nanobiotix CEO Laurent Levy

Nanobiotix (EPA:NANO) has outlined a new development strategy, in which it will push into pivotal trials in the U.S. unpartnered and test its radiotherapy-boosting nanomedicine in a broader range of indications. The Paris, France-based biotech is financing its dialled-up ambitions with a €10.4 million ($13.0 million) private placement from Capital Ventures International (CVI).

Further funding is in the pipeline. Warrants could generate an additional €14 million, while Nanobiotix also has the option to return to CVI for another €10 million. The money will allow Nanobiotix to shift gears, moving from an R&D strategy in which it was looking for a partner for the U.S. market to one that will see it wrap up pivotal trials solo. If all goes to plan, Nanobiotix will look for a partner in the U.S. to help with commercialization once it has pivotal trial data.

"We believe that it is important to develop our product ourselves for the U.S. market, thereby retaining the value in the company," Nanobiotix CEO Laurent Levy said in a statement. Nanobiotix's near-term focus is Europe and Asia, where it is testing its lead candidate, NBTXR3, in 156 patients with soft tissue sarcoma. Depending on how the data look in 2016, Nanobiotix may run a bridging study of NBTXR3--which is classed as a medical device--to gather evidence for a submission to FDA.

NBTXR3 is made up of hafnium oxide nanoparticles that are small enough to enter cancer cells. When exposed to radiotherapy, the nanoparticles effectively increase the dose, Nanobiotix claims, allowing the administration of a blast strong enough to destroy cancer cells without damaging healthy tissues. - read the release

Back to top

Joint EU-CRUK VC unit commits cash to move RET inhibitors into Phase I

The £50 million ($79 million) Cancer Research Technology Pioneer Fund (CPF) has made its latest investment in early-stage oncology R&D in the United Kingdom. CPF selected a program to develop RET inhibitors at the Cancer Research UK (CRUK) Manchester Institute Drug Discovery Unit as the recipient of its fourth investment.

CRUK researchers will use the cash to identify compounds that inhibit RET, a gene linked to medullary thyroid cancer and a tiny proportion of lung tumors. CPF has committed an undisclosed slice of its £50 million investment fund to take a RET inhibitor as far as Phase I, at which point the team will look for industry partners to help move the candidate into mid-stage trials and beyond.

The program is following what CRUK hopes will become a well-established development pathway. Manchester Cancer Research Centre is responsible for the basic biological discoveries, which are then turned into early-phase compounds by the CRUK Manchester Institute and offloaded to an industry partner for further development.

CPF was set up in 2012 by the commercial wing of nonprofit CRUK and the European Investment Fund to finance the work needed to turn basic biological discoveries into early-phase clinical candidates. "It's absolutely essential that we take steps to bridge the innovation gap in U.K. drug discovery," Keith Blundy, chief executive of CRUK's commercial wing, said in a statement. - read the release

Back to top

Ablynx sets sights on conditional approval of blood disorder drug

Ablynx CEO Edwin Moses

Ablynx (EBR:ABLX) has set its sights on securing an accelerated approval pathway for its thrombotic thrombocytopenic purpura (TTP) drug, caplacizumab. Ghent, Belgium-based Ablynx plans to hold talks with regulators in Europe and the U.S. early next year about a development plan that could see its drug come to market in 2018.

A lot of things have to go Ablynx's way for that 2018 goal to become a reality. Ablynx plans to get the process underway at the start of 2015 by asking officials at FDA and the European Medicines Agency (EMA) whether its treatment for TTP is a suitable candidate for their respective fast-track pathways, Bloomberg reports. "We will know after those meetings whether an application like that will be successful," Ablynx CEO Edwin Moses said.

Under the conditional approval pathway now in place in Europe, Ablynx could bring caplacizumab to market around the time it expects to wrap up its Phase III trial in late 2017. If Ablynx fails to win the conditional approval designation, it will have to wait until after it has gathered all of the Phase III data to submit an application to EMA, potentially adding two years to the process. EMA conditionally approved PTC Therapeutics' ($PTCT) Duchenne muscular dystrophy drug Translarna in August.

Becoming the latest company to win conditional approval would bring forward the date on which Ablynx can begin generating sales from caplacizumab. Ablynx recently raised its sales expectations for the drug from €250 million ($312 million) to up to €400 million. And Moses thinks the drug could surpass the raised expectations if it fulfills early promise in preventing relapses. - read the Bloomberg article and Reuters' coverage

Back to top

Report: BiondVax mulls following Israeli biotech peers to Nasdaq

BiondVax CEO Ron Babecoff

The failure of many Israeli biotechs to set Nasdaq alight has seemingly done little to slow the flow of firms willing to put themselves at the mercy of investors. Universal flu vaccine developer BiondVax Pharmaceuticals (TASE:BNDX) is reportedly positioning itself to be one of the next to try its luck.

Ness Ziona, Israel-based BiondVax is still weighing up whether to file for a conventional IPO or list American Depositary Receipts, Globes reports, but knows it wants a way to access U.S. biotech investors. The company has traded publicly in Israel since 2007, but aside from a pandemic-driven surge in 2009 has done little to excite the investors. BiondVax currently has a market cap of NIS 42 million ($11 million) on the Tel Aviv Stock Exchange. A U.S. listing would have a similar value.

While the market cap means BiondVax is likely to be among the smaller Israeli Nasdaq IPOs, the firm is one of the more advanced of the universal flu vaccine hopefuls. BiondVax has already wrapped up a mid-stage trial of its universal flu vaccine--which targets the non-changing epitopes of the virus--and received a grant in September to help finance a Phase III study. However, it is yet to sign the deal with an established player in the flu vaccine market that could help validate the technology.

In October, the Times of Israel reported BiondVax planned to step up talks with potential vaccine development partners and the governments that may ultimately buy the product. The intensification of talks was triggered by the strengthening of BiondVax's intellectual property position in Europe and Japan. - read the Globes article and TOI piece

Back to top

Of Note:

Nordic Nanovector and Affibody entered into a collaboration to discover radio-immunotherapies for multiple myeloma. Oslo, Norway-based Nordic Nanovector shares a founder with Algeta, the biotech Bayer bought for $2.9 billion. Release

The National Institutes of Health (NIH) published data from its part of the Phase I trial of GlaxoSmithKline's ($GSK) Ebola vaccine. NIH reported the vaccine appears to be safe and trigger an immune response. Release

Helsinn revealed it is searching for licensing deals to expand its pipeline of cancer supportive care drugs. Treatments for Lupus and Crohn's disease--two side effects of immuno-oncology drugs--are on the Swiss company's radar. EP Vantage

Prosensa ($RNA) struck a deal with BioMarin ($BMRN) to sell the business for up to $840 million (€673 million). BioMarin will pay the first $680 million in cash, with the remaining $160 million tied to the approval of drisapersen in the U.S. and Europe. FierceBiotech

The FDA scheduled the advisory committee meeting on Basilea Pharmaceutica's (SIX:BSLN) isavuconazole for January 22. Isavuconazole is an antifungal Basilea is developing with Astellas Pharma. Release

Neil Woodford revealed further details of the biotech-focused fund he has talked up since leaving Invesco Perpetual. The plan is to create a £200 million ($314 million) fund focused on private biotechs. FierceBiotech | More

Genmab (CPH:GEN) and Janssen Biotech opened a new front in their development strategy for daratumumab. A Phase II trial of the monoclonal antibody in patients with smoldering multiple myeloma is set to start next year. Release

Cell Medica raised £50 million ($78 million) to finance its pipeline of oncology and infectious disease candidates. A who's who of British biotech investors backed the round, with Imperial Innovations (AIM:IVO), Invesco Perpetual and Woodford Investment Management all participating. FierceBiotech

Fosun Pharma's venture capital fund reportedly began looking for a partner with which to invest in Israeli biotechs and other businesses. China-based Fosun has made multiple forays into Israel in recent years. Globes

Lundbeck (CPH:LUN) CEO Ulf Wiinberg left the company after admitting to violating its code of conduct. The resignation was triggered by a gift from a service provider in which Lundbeck later invested. FierceBiotech

Midatech Pharma filed paperwork for an IPO on London's AIM. The nanomedicine firm is aiming to raise £30 million ($47 million) and buy Q Chip, a developer of a controlled-release technology. Schedule 1

Bankers and industry officials expressed doubts about the likelihood of Pfizer ($PFE) making another attempt to buy AstraZeneca ($AZN). FierceBiotech

Back to top

Brought to you by:

Suggested Articles

Preclinical-stage biotech Abpro Therapeutics wants to trial its two lead candidates for HER2-positive cancers and diabetic macular edema in 2019.

After a rough patch in 2017, the stars seem to be realigning for French CAR-T expert Cellectis, which just closed a $164 million U.S. public offering.

Investment firm Frazier Healthcare Partners has closed its 11th fund—worth $780 million—that will help established companies accelerate their growth.