Snippet roundup: Aerie stumbles on safety, and Stada is sold at last

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

This week, April 10 to 13, 2017, we had thoughts on the following: Aerie’s safety data fail to reassure; activist pressure succeeds in achieving Stada sale; Geron still waiting for J&J to pull trigger on imetelstat; Roche gears up for Alk domination; some relief for companies trying the CD123 approach.

These snippets were previously published daily via twitter.

Aerie’s safety data fail to reassure

April 13, 2017

Aerie Pharmaceuticals has crossed one bridge, getting the US FDA to accept its filing for Rhopressa, but a bigger problem is its inability to allay safety concerns associated with the glaucoma project. In the six-month cut of safety data – released yesterday from the Rocket-4 trial at the request of European regulators – adverse events including eye redness, opaque deposits on the cornea, and conjunctival haemorrhage were all higher in the Rhopressa arm. Aerie shares fell 5% in the post-market, before recovering this morning. Rhopressa, a Rho kinase (Rock) and norepinephrine transporter (Net) inhibitor, maintained non-inferiority to timolol, but the safety findings did nothing to allay fears about how regulators and payers would view the project, and how restrictive its label might be. The FDA has accepted the Rhopressa filing, and a decision is due by February 28, 2018. EvaluatePharma’s consensus of sellside analysts has shrunk in the past two years, from 2020 Rhopressa revenue expectations of $239m in 2020 to $118m today.

Activist pressure succeeds in achieving Stada sale

April 10, 2017

The private equity firms Bain Capital and Cinven won the bidding war for the German generics maker Stada Arzneimittel with a €5.3bn ($5.6bn) bid. The 23rd-biggest generics company had been the target of three separate approaches, and the combined offer from the two investment companies values it at €66 a share, representing a 49% premium over its price on December 9, 2016, when the first reports of a takeout attempt emerged. Stada had been able to repel takeover offers by virtue of a type of share that loses voting rights if sold without the consent of management, but an investor, Active Ownership Capital, persuaded shareholders to scrap this structure. The buyers have agreed to honour union contracts and say they will not engage in any unplanned business-related layoffs for four years.

Geron still waiting for J&J to pull trigger on imetelstat

April 10, 2017

Any investors hoping that Geron’s short-notice conference call this morning signalled a decision from its partner Johnson & Johnson to move forward with imetelstat will be disappointed. Two mid-stage trials of the project have now passed a second interim review, but it seems that J&J is taking its time committing. However, the continued limbo is preferable to the studies being declared futile, and Geron’s stock opened up 17% this morning. The company did not give many details on the analyses of the studies, IMerge in lower-risk myelodysplastic syndromes and IMbark in relapsed or refractory myelofibrosis. But it did say it proposed amendments to the second phase III portion of IMerge and, if J&J opts to continue, enrolment should start in the fourth quarter. Meanwhile, IMbark will carry on unchanged, and J&J will assess its results as they emerge over the next year before making a call. Geron had already dropped the lower 4.7mg/kg dosing arm in IMbark after a previous interim analysis. Chief executive officer John Scarlett believes that the unmet need in myelofibrosis could work in its favour, noting that nothing is approved after progression on Incyte’s Jak inhibitor Jakafi. However, it is now up to J&J which, if it proceeds with imetelstat, will have to do so for all indications, Mr Scarlett said during the call.

Roche gears up for Alk domination

April 10, 2017

Roche’s Alecensa is coming for the Alk inhibitor crown after besting Pfizer’s rival agent Xalkori in the first-line setting. Roche did not give more detailed data from the Alex trial, in Alk-driven non-small cell lung cancer, but the win should cement Alecensa’s position as the best seller in this class. It had already been expected to overtake Xalkori by 2018, according to EvaluatePharma sellside consensus; forecasts for Pfizer’s product have been declining, and could now fall further still. The Alk inhibitor space has become crowded, but Alecensa looks unlikely to be troubled by Novartis’s Zykadia, which has succeeded in a first-line study, but only versus chemotherapy. Meanwhile, brigatinib, developed by Ariad and now owned by Takeda, is due an approval decision in Xalkori failures by the end of April, and is also in a first-line trial, Alta-1L, versus Xalkori. But Alecensa has now become the product to beat, and Roche has some time to make its advantage count.

Some relief for companies trying the CD123 approach

April 10, 2017

Targeting the CD123 antigen was thrown into doubt by problems at Stemline Therapeutics and Johnson & Johnson, so the resumption of a J&J study should have others trying this approach breathing a little more easily. Chief among these might be Cellectis, whose lead wholly owned asset, UCART123, is an allogeneic CAR versus CD123 – a popular antigen of interest in acute myelogenous leukaemia (AML). The US FDA in February cleared UCART123 to begin a phase I study, though this has yet to get under way. The J&J trial that just resumed concerns the anti-CD123 bispecific JNJ-63709178; last year this study was marked on Clinicaltrials.gov as suspended owing to a serious adverse event, but has now resumed enrolling AML patients. The case of Stemline is curious: two deaths have been associated with its lead asset, a CD123-targeting protein/toxin conjugate coded SL-401, despite a protocol change, yet the FDA has allowed its trial to continue. This suggests that the agency is comfortable with the risks around this target, at least in a setting as intractable as AML, though Cellectis bulls should be cautious: off-tumour effects are likely to be more pronounced with CAR-T than with an antibody.

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