AZ strikes Alderley cancer pact, Cerenis mulls IPO, 4D to start microbiome trials in 2015

Welcome to the latest edition of our weekly EuroBiotech Report. Having spent much of 2014 touting the potential of its clinical-stage oncology candidates, AstraZeneca ($AZN) struck a deal with Redx Pharma to work on the next generation of cancer drugs. The partners will work alongside each other at Alderley Park, the R&D campus AstraZeneca is vacating in its relocation to Cambridge. Down the road from Alderley Park in Manchester, 4D Pharma (AIM:DDDD) continued its strong performance in 2014 by committing to starting clinical trials of two of its microbiome therapies next year. Ipsen also provided an update on its clinical pipeline, with the French biopharma expecting a quick regulatory decision on Somatuline after FDA granted it priority review status. Fellow French biotech Cerenis Therapeutics received a boost from regulators, too. The European Medicines Agency awarded its lead candidate CER-001--a once-hot cholesterol drug that failed Phase II in January--orphan drug status in two indications. ThromboGenics (EBR:THR) is another company plotting a recovery. The Belgian biotech is slimming down and focusing on diabetic eye disease as it seeks to bounce back from weak sales of Jetrea. And more. Nick Taylor (email | Twitter)

1. AstraZeneca strikes cancer R&D pact with Alderley Park tenant
2. Cerenis bags orphan drug nods and eyes IPO as it plots a recovery
3. Green light for live bacteria trials triggers stock uptick at 4D Pharma
4. Ipsen nabs priority review for cancer drug to speed FDA decision
5. ThromboGenics narrows R&D focus to diabetic eye disease

And more >>

AstraZeneca strikes cancer R&D pact with Alderley Park tenant

AstraZeneca ($AZN) has inked a cancer drug discovery collaboration with Redx Pharma, a British biotech that was among the first tenants of the Alderley Park R&D hub created by the Big Pharma's move to Cambridge. And the Alderley Park site will play a central role in the collaboration, with scientists from Redx and AstraZeneca working together at the campus.

Redx CEO Dr. Neil Murray

Redx moved into Alderley Park last year, building a 50-person anti-infective team to complement the 100-person oncology unit it runs in Liverpool. The Liverpool unit will handle most of the AstraZeneca work, which is focused on discovering new drugs targeting a genetic cause of solid tumor growth. AstraZeneca has made an undisclosed upfront payment to collaborate with Redx on the target and secure the sole rights to any resulting drugs. Redx is also in line for "significant" R&D and commercial milestones, plus tiered royalties.

"This is a big deal for Redx. While we have done other commercial deals before, this is our first with a blue chip pharmaceutical company," Redx CEO Dr. Neil Murray told TheBusinessDesk. The deal comes 6 months after Redx formed a skin cancer pact with France's Pierre Fabre. Both deals tap into Redx's drug discovery capabilities, like the proprietary Redox Switch approach it uses to find improved versions of existing commercial and late-phase drugs. Redx has used the capabilities to build an in-house pipeline of 5 drugs--four anticancer and one anti-infective--at the lead optimization stage.

As candidates discovered in the AstraZeneca pact advance up to this stage and beyond, the Big Pharma's Oncology Innovative Medicines Unit will collaborate on their progress. If successful, the collaboration will provide a good example of the sort of Big Pharma-biotech crossover the new owner of the Alderley Park site hopes to facilitate. "The co-location of our scientists will facilitate our collaboration," AstraZeneca's Susan Galbraith said in a statement. - read the release, BusinessDesk article and MEN's take

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Cerenis bags orphan drug nods and eyes IPO as it plots a recovery

Cerenis Therapeutics has received two orphan drug designations in Europe for its lead candidate, an HDL-like drug that was a hot property before a Phase II failure dampened enthusiasm. And having secured an alternative path forward as a rare disease drug--and looked for positives in the failed trial data--the French biotech is planning to raise cash through an IPO or financing round.

The orphan drug nods from the European Medicines Agency cover the use of CER-001 in two rare diseases that cause HDL deficiency. CER-001 is designed to work in the same way as HDL, potentially giving people with apoA-I or ABCA1 deficiencies a way to clear plaque from their arteries. The potential market for such a treatment extends far beyond the rare disease space, but Cerenis' pitch for the mainstream took a hit in January when CER-001 failed to beat placebo in a Phase II trial.

In the January release, Cerenis latched on to the success of CER-001 at one dose level as evidence it still had a future. Since then the company has continued to dig through the data, a process CEO Jean-Louis Dasseux said has uncovered reasons to be optimistic. "It took some time for us to understand what was happening. After discussing with a lot of experts, we believe those data are compelling," Dasseux told Bloomberg.

Cerenis--a 2011 Fierce 15 company--is continuing with development of CER-001 as a way to prevent repeat heart attacks. What comes next depends on Cerenis' ability to raise cash. Having already raised €117 million ($153 million) from Sofinnova Partners, OrbiMed and others, Dasseux is weighing an IPO. The process of preparing for public or private fundraising will begin next month, with the start of the next trial "conditioned to the round of financing or the IPO," Dasseux said. - read Bloomberg's article and the press release

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Green light for live bacteria trials triggers stock uptick at 4D Pharma

The good news keeps coming for 4D Pharma (AIM:DDDD). Since listing on AIM in February, the British biotech has lured investment from Neil Woodford, seen its stock price triple and bagged an orphan drug designation. Now 4D is preparing to move its two lead candidates into clinical trials, sparking another jump in its stock price.

4D Pharma CEO Duncan Peyton

Phase I trials of the treatments are due to start in the second quarter of 2015, providing an early test for 4D and the live bacteria therapeutics developed by its Scottish subsidiary GT Biologics. While both treatments are set to enter the clinic in the same quarter, GT calls Thetanix its lead candidate. Thetanix has already secured an orphan drug designation, with FDA granting it the status for its potential as a pediatric Crohn's disease candidate in October.

The second drug, Blautix, is being trialed as a treatment for irritable bowel syndrome (IBS). Both candidates address the microbiome, the hotly tipped field that deals with the 100 trillion microorganisms in the body and has attracted interest from Johnson & Johnson ($JNJ) and Pfizer ($PFE). Lead candidate Thetanix is designed to address the lower level of bacterial diversity seen in people with Crohn's to lessen the major symptoms.

Clinical evidence to support this hypothesis is still years away, with next year's trials focusing mainly on assessing safety and tolerability. 4D hopes to gather some early efficacy data too though. The trial of Thetanix will enroll some patients with Crohn's and the study of Blautix will recruit a small number of people with symptomatic IBS. - read the release and the Financial Times' take

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Ipsen nabs priority review for cancer drug to speed FDA decision

The U.S. FDA has granted priority review status to Ipsen's marketing application for Somatuline in patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs). The status means Ipsen may be just months away from approval, with the French drugmaker expecting a decision in the first quarter of 2015.

Ipsen CEO Marc de Garidel

Decisions by European regulators are due to start coming in during the second quarter, setting Ipsen up for a big first half of 2015. The filings follow the publication of data from a 204-person Phase III trial of Somatuline, in which almost two-thirds of GEP-NET patients in the treatment arm achieved progression-free survival after 96 weeks. One-third of patients in the placebo arm hadn't progressed or died after 96 weeks. The data have led to analysts tipping Somatuline to rack up significant sales.

"Impressive Phase III CLARINET results for Somatuline in asymptomatic NETs, together with the positive pivotal U.S. ELECT trial in functioning NETs, underscore our belief in peak Somatuline sales of $950 million (€725 million)," Jefferies analyst Peter Welford wrote in a note to investors. Welford forecasts Somatuline sales of $675 million in NETs, with the remaining $275 million coming from the treatment of acromegaly, an indication for which it is already approved in the U.S. and other markets.

Revenue from Somatuline experienced a double-digit rise in the first half of 2014, a trend driven by upticks in the volume of sales in Germany, the United Kingdom and the U.S. Approval in GEP-NETs could further accelerate growth. Last year drug sales at Ipsen totalled €1.2 billion, a little more than double what Welford thinks the GEP-NET indication could generate. - read the filing news and financial results

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ThromboGenics narrows R&D focus to diabetic eye disease

With sales of Jetrea to patients with symptomatic vitreomacular adhesion (sVMA) slumping over the first half of 2014, ThromboGenics (EBR:THR) is slimming down and focusing its R&D budget on diabetic eye disease. The first milestone for the restructured organization is the start of a Phase II trial of Jetrea in patients with proliferative diabetic retinopathy.

ThromboGenics' CEO Dr. Patrik De Haes

ThromboGenics expects to start the U.S. trial in the first half of next year. With the drug struggling to make an impact with U.S. physicians in sVMA--Jefferies analyst Peter Welford called the Jetrea brand "somewhat tarnished"--the Phase II trial gives ThromboGenics another possible way to extract value from the faltering eye disease drug. ThromboGenics laid out the thinking behind the indication expansion on a conference call with investors to discuss the financial results.

"We have decided to move ahead with this potential indication as creating a total posterior vitreous detachment--which can be achieved with Jetrea--is accepted as an important step in preventing further revascularisation in proliferative diabetic retinopathy," ThromboGenics CEO Dr. Patrik De Haes said. Total posterior vitreous detachment was the secondary endpoint in the pivotal Phase III trials of Jetrea in patients with sVMA.

The trials met the secondary endpoint. After 28 days, 13.4% of eyes injected with Jetrea showed total posterior vitreous detachment, compared with 3.7% in the placebo arm. With Jetrea having failed in another mid-stage study last year, the proposed diabetic retinopathy trial is the shortest path to a new source of sales. ThromboGenics also has preclinical-stage diabetic eye disease partnerships with British biotech Bicycle Therapeutics and Eleven Biotherapeutics but plans to spin out its cancer R&D. - read the transcript and FierceBiotech's take

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Of Note:

A clutch of European drugmakers shared important cardiology data. Novartis ($NVS) and Sanofi ($SNY) were the big winners after impressing with late-stage data on their respective heart failure and cholesterol drugs. France's Servier had a tougher time, with its heart rate-reducing drug failing to beat placebo. Novartis | Sanofi | Servier

Dutch biotech Dezima took a step closer to taking a piece of the cardiovascular market from its giant rivals by posting Phase IIb data on its CETP inhibitor, TA-8995. But ex-Pfizer ($PFE) exec John LaMattina is sceptical, writing in Forbes that "the TA-8995 story is a major longshot." FierceBiotech | Forbes

Ablynx (EBR:ABLX) licensed its anti-TNFα candidate ozoralizumab to Eddingpharm in China. Eddingpharm will pay €2 million upfront plus milestones and royalties of up to 20% on sales of the drug, which has been through a Phase IIa trial in patients with rheumatoid arthritis. Release

French state health institute Inserm began talks to run trials of Ebola drugs in Guinea. Inserm wants to run 15-person trials of drugs from Fujifilm and Tekmira Pharmaceuticals. The start date is at least two months away. Bloomberg

Sanofi ($SNY) edged towards a payout on its billion-dollar dengue vaccine bet by posting positive data from its final clinical trial. The company plans to start filing for approval of the vaccine early next year. Reuters | FierceVaccines

Birken reported three Phase III trials of its wound healing candidate met their primary endpoints, putting the German biopharma in a position to submit a filing to the European Medicines Agency. The plan is to have the drug--a birch bark-based ointment called Oleogel-S10--on the market in 2016. Release

Galmed Pharmaceuticals ($GLMD) laid the groundwork for a Phase IIa trial of its nonalcoholic steatohepatitis (NASH) drug aramchol by completing two 6-month chronic toxicology studies. No drug-related deaths or effects on body weight were seen. The tests will continue for a further three months to satisfy the FDA. Release

UniQure said it expects a delay in gathering the clinical data it needs to support its FDA filing for gene therapy Glybera. The Dutch biotech now expects to start a Phase IV trial--which will try to meet the requirements of U.S. and European regulators--in the middle of next year. Release

Novo Nordisk ($NVO) tightened its focus on diabetes by dropping its anti-inflammatory program. The decision will affect around 400 employees, around half of whom could lose their jobs. FierceBiotech

Bavarian Nordic (OMX:BAVA), Johnson & Johnson ($JNJ) and the National Institutes of Health committed to accelerating their Ebola collaboration. A clinical trial of a combination of vaccines from Bavarian Nordic and J&J is due to start next year. FierceBiotech

Affimed Therapeutics closed a $15.7 million (€11.7 million) Series E round led by Aeris Capital, BioMedInvest, LSP Life Sciences Partners, Novo Nordisk A/S and OrbiMed. The German immuno-oncology company also secured a $14 million loan to give it the funds for clinical development. Release

Foamix Pharmaceuticals set the terms for its proposed Nasdaq IPO. The Israeli developer of topical foam-based therapies hopes to raise $65 million (€49 million), around one-third of which will go towards taking its treatment for moderate-to-severe acne through Phase III. F-1

Kamada ($KMDA) maintained a positive front after its lead candidate missed its primary and secondary endpoints. But while the Israeli biotech thinks it can continue uninterrupted towards approval in Europe, investors are less confident. FierceBiotech

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Read previous editions of the EuroBiotech Report here.