U.K. starts ambitious drug dev review, Onxeo to raise €41.6M, Galapagos mulls Nasdaq listing

Welcome to the latest edition of our weekly EuroBiotech Report. The United Kingdom government kicked off a review it hopes will deliver a faster, cheaper way of developing drugs. Data held in health records are expected to play a bigger role as the government tries to move away from the traditional model of trialling and assessing drugs. The cost of this traditional model forced several European biotechs to consider their finances this week. Onxeo (EPA:ONXEO) unveiled plans to raise €41.6 million ($52.2 million). Galapagos (AMS:GLPG) revealed it will consider listing on Nasdaq if AbbVie ($ABBV) backs out of their rheumatoid arthritis deal. And Efranat raised $4.5 million to trial a cancer drug with a controversial history. Finally, one of the researchers trialling GlaxoSmithKline's ($GSK) Ebola vaccine said the study has yielded "very satisfactory" safety data so far. And more. Nick Taylor (email | Twitter)

1. U.K. aims to slash drug development timelines and costs with regulatory rethink
2. Galapagos mulls Nasdaq listing in case AbbVie turns down RA option
3. Onxeo's plan to raise cash for R&D causes stock to drop
4. Efranat snags $4.5M for clinical trials of controversial cancer drug
5. GSK's Ebola vaccine delivers solid early safety data

And more >>

U.K. aims to slash drug development timelines and costs with regulatory rethink

The United Kingdom government has ordered a review into how regulators, the healthcare system and biopharma can collaborate to slash drug development costs and timelines--and the amount the state has to pay once a product is approved.

U.K. Minister for Life Sciences George Freeman

An independent, as-yet-unnamed organization will conduct the review and report its findings in the middle of next year, but the government already has a pretty clear idea of what it wants the review to achieve. The Times reports politicians want to bypass traditional clinical trials, turning instead to a model that makes greater use of health records to assess how effective drugs are in individual patients. The government would then reimburse pharma companies based on health outcomes.

At this stage the details are hazy, but the government has nonetheless made lofty claims about what the new approach will achieve. George Freeman, the U.K. minister for life sciences, said the overhaul will cut years off the time it takes for drugs to reach patients--The Times goes as far as saying it will halve the wait--and save hundreds of lives a year. Senior figures within the government reportedly view the current system as broken.

The failings of the current system--which contribute to the huge cost of drug development--ultimately affect the U.K. government in the form of high prices for therapeutics. By helping companies to lower the cost of developing drugs in the U.K. and moving toward a pay-for-performance reimbursement model, the government hopes to trim its tab while making cost less of a factor in patient access to new drugs. - read the release, FierceBiotech's take and The Times' article (sub. req.)

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Galapagos mulls Nasdaq listing in case AbbVie turns down RA option

Galapagos CEO Onno Van de Stolpe

Galapagos (AMS:GLPG) will consider listing on Nasdaq if AbbVie ($ABBV) turns down the option to take its JAK1 inhibitor into Phase III in rheumatoid arthritis. The listing would give Galapagos the cash to take GLPG0634 into Phase III unpartnered.

AbbVie can license the drug for $200 million (€160 million) once Galapagos delivers Phase IIb data, at which point the Big Pharma would take responsibility for running and financing a late-stage trial. Galapagos completed enrollment in one of its Phase IIb trials earlier this month and expects to have the complete, 24-week data package by the second quarter of 2015. AbbVie's decision--and the future of a big part of the $1.35 billion deal it struck in 2012--rests on the strength of the data.

Galapagos has formulated a contingency plan in case AbbVie decides not to license GLPG0634. Speaking at the Jefferies 2014 Global Healthcare Conference in London, CEO Onno Van de Stolpe said if Galapagos needs to finance the Phase III trial, he would consider listing the company on Nasdaq to raise the money. Galapagos already has a dual listing on the Euronext exchanges in Amsterdam and Brussels, but could tap into a new source of cash by trading on Nasdaq.

Several other biotechs provided updates on their activities at the Jefferies Healthcare Conference. In a note to investors, Jefferies analyst Peter Welford listed Galapagos' Nasdaq plan alongside Bavarian Nordic's (CPH:BAVA) revelation that it expects to fully enrol its Phase III prostate cancer by next month as being among the biggest pieces of news to emerge from the first day of the conference. Galapagos also published data showing GLPG0634 is unlikely to interact with other rheumatoid arthritis therapies. - read Galapagos' GLPG0634 release

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Onxeo's plan to raise cash for R&D causes stock to drop

Onxeo CEO Judith Greciet

Onxeo (EPA:ONXEO) has turned to the public markets for cash to fuel its R&D ambitions, starting a rights issue in a bid to raise up to €41.6 million ($52.2 million). The Franco-Danish biotech plans to use the cash to expand a Phase III study of its liver cancer drug and prepare to advance its severe oral mucositis candidate into late-stage trials.

An expansion of the liver cancer trial topped Onxeo's list of uses for the money. Armed with the fresh funding, Onxeo plans to expand the trial to accelerate patient recruitment, a strategy that could cut the time it takes to generate pivotal data on the drug, Livatag. The rest of the cash will go toward readying Onxeo's next wave of late-phase hopefuls. Severe oral mucositis drug Validive is nearing the start of Phase III. And a late-phase trial of lymphoma drug Beleodaq is also in the pipeline.

Onxeo framed the fundraising as part of its strategy to leverage the assets it gained by merging BioAlliance Pharma and Topotarget to create a notable player in orphan drug development in Europe. "This rights issue will allow us to pursue this step-up, by accelerating and expanding development programs," Onxeo CEO Judith Greciet said in a statement.

The plan has the backing of major shareholders. Financière de la Montagne--which holds almost 9% of Onxeo's share capital--has committed to exercising all of its rights and placing an additional order of €13.5 million. Each investor will have the chance to get one new share for every four shares they hold already. The new shares cost €4.50, a 29% discount compared to Onxeo's closing price on November 14. In Paris, Onxeo closed down 6.5% on the day the rights issue was unveiled. - read the release

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Efranat snags $4.5M for clinical trials of controversial cancer drug

Efranat CEO Uri Yogev

Gc protein-derived macrophage activating factor (Gc-MAF) has a contentious history. Proponents of the drug touted it as a miracle cure on the back of early clinical data--fueling its unlicensed use--only for journals to retract several of the papers after cancer nonprofits tore holes in the research. Now, Efranat claims to want to bring some legitimacy to the field.

Rehovot, Israel-based Efranat has raised $4.5 million (€3.6 million) to fund its work, which includes a 40-person Phase I open-label trial in solid tumors that is already underway. Efranat was co-founded in 2009 by Nobuto Yamamoto, the professor who started the "wonder drug" claims and authored papers on its use in bowel and breast cancer that were later retracted. Yamamoto contributed patents and knowledge to Efranat but doesn't currently have an active role in the business.

Efranat wants to distance itself from the earlier controversies and particularly the operations that are selling Gc-MAF online. "One doctor set up a company in Guernsey Island that markets containers allegedly containing the material. We bought one of his containers, and couldn't find the molecule in it. We're keeping our distance from these people. We just want to produce and test the material according to the rules," Efranat CEO Uri Yogev told Globes.

The Phase I trial is testing Efranat's formulation of Gc-MAF at various doses to assess the safety, tolerability and preliminary efficacy of the drug. If Gc-MAF comes through the Phase I trial unscathed, it would then face its first real clinical test, assuming Efranat can finance the program. Advocates of the drug claim it activates macrophages to fight cancer, but to date, quality clinical data to support the hypothesis is lacking. - read Globes' article and Cancer Research UK's analysis

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GSK's Ebola vaccine delivers solid early safety data

Oxford University Professor Adrian Hill

The Ebola vaccine candidate GlaxoSmithKline ($GSK) acquired in its $325 million (€260 million) buyout of Swiss biotech Okairos has made a satisfactory start to clinical trials. Researchers have now dosed almost 200 people with the vaccine without encountering any safety concerns.

GSK is working with sites on three continents to trial the vaccine, a model that has enabled it to near 200 dosings within two months of starting the study. A site in Mali jointly run by the University of Maryland has led the way, recruiting 80 patients, while the National Institutes of Health and the University of Oxford have also contributed. Oxford dosed the last of the 60 healthy volunteers in its trial this week, prompting the leader of the study to provide an update on its progress.

"The safety data here have looked very satisfactory so far," Oxford University Professor Adrian Hill said in a statement. "The response we have seen from people coming forward to take part has been remarkable." The trial started in the U.S. in early September. Oxford University came on board two weeks later, followed by the site in Mali and one at the University Hospital of Lausanne in Switzerland last month. The Swiss hospital aims to enrol 120 people by December, but has dosed only 34 to date.

Data on the immune responses of people enrolled at the Swiss and U.K. sites are expected to be available by late December, allowing GSK to compare the results to those gathered in Mali. - read the release and Reuters' take

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Of Note:

Xellia Pharmaceuticals opened an R&D facility in Oslo, Norway. The 1,900-square-meter site is focused on developing new active pharmaceutical ingredients. Anti-infectives are a particular focus of the R&D center. Release

The U.S. FDA awarded BerGenBio's acute myeloid leukaemia candidate orphan drug status. The Norwegian biotech recently started a Phase Ib clinical trial of the drug, BGB324. The FDA also granted the status to BioBlast Pharma's ($ORPN) spinocerebellar ataxia type 3 drug. Release | More

Basilea Pharmaceutica (SIX:BSLN) presented preclinical data on its experimental cancer drug, BAL101553. The data suggest BAL101553--which is in Phase IIa--has antitumor activity. Release

STADA Arzneimittel entered into negotiations to in-license a biosimilar copy of AbbVie's ($ABBV) Humira from mAbxience. The companies signed a letter of intent to reach a decision on the deal within 6 months. Release

The Swedish government awarded Beatica a SEK 3 million ($400,000) grant to work on epigenetic cancer drugs. Beatica will focus on inhibition of lysine-specific histone demethylases. Release

Alzheimer's Research UK joined a consortium that is supporting Stevenage Bioscience Catalyst's neurodegenerative diseases challenge. The consortium will review research proposals submitted to the center. Release

The European Union awarded a Midatech Pharma-led consortium a €7.9 million ($9.9 million) grant. Midatech will use the cash to scale up production of nanopharmaceuticals it is developing. Release

GlaxoSmithKline ($GSK) spinout Autifony Therapeutics moved its tinnitus drug into Phase IIa. The proof-of-concept trial will enrol patients at 12 sites in the United Kingdom. Release

Forward Pharma ($FWP) brought the total raised in its Nasdaq IPO up to $235.2 million (€187.5 million) through the sale of stock in an overallotment. Release

AstraZeneca ($AZN) touted the strength of its pipeline at an analyst day. The proximity of the event to the date on which Pfizer ($PFE) can make a second approach for AstraZeneca added to the focus on the presentations. FierceBiotech

Ipsen (EPA:IPN) renewed its collaboration with the Salk Institute for another three years. The French pharma began working with the Salk Institute in 2011 to better understand cancer and neurodegenerative disease. Release

Affimed ($AFMD) pushed back the expected date of first dosing in its Phase IIa trial of AFM13. The trial is now set to start in the first quarter of 2015, but Affimed still expects the trial to follow the same overall timeline. Release

Modern Biosciences, Domainex, Heptares Therapeutics, Ingenza and other British biotechs received funding in the latest round of Biomedical Catalyst awards. Release

NeuroDerm ($NDRM) listed on Nasdaq but fell well short of its goal. The Israeli biotech had hoped to raise as much as $72 million (€57 million) but ended up settling for $45 million. FierceBiotech

Zealand Pharma (CPH:ZEAL) moved its therapy for severe hypoglycemia in diabetes into the clinic. The Danish biotech expects to have data on the treatment, stable glucagon analogue, by the middle of next year. Release

Pfizer ($PFE) delivered a boost to Merck KGaA's beleaguered R&D operation by paying $850 million (€679 million) upfront for the right to co-develop immuno-oncology drugs. Release

Theravectys reported interim results from a Phase I/II trial of its therapeutic vaccine. The French biotech has yet to encounter safety concerns after administering the vaccine to 38 HIV-positive patients. Release

Neil Woodford invested $25 million (€20 million) in Northwest Biotherapeutics ($NWBO). The deal is a relatively rare foray outside of the European biopharma sector for Woodford's fund. FierceBiotech

Wilson Therapeutics started a Phase II trial of its lead candidate in patients with Wilson disease, a rare genetic disorder that renders the body unable to process copper. The trial will enrol 30 patients. Release

DBV Technologies ($DBVT) started a Phase I/II trial of its treatment for cow's milk allergy. The French biotech plans to enrol 150 patients to deliver data on dosing and efficacy after 12 months. Release

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