Friday Five – Your weekly pharma review

Pfizer spins

Like a number of its Big Pharma peers, Pfizer has decided that promising assets in its pipeline that don't meet the criteria of its core therapeutic focus are at risk of being lost altogether to de-prioritisation. The answer, it has decided, is to spin out a separate company to develop them – Spotlight On: Pfizer turns biotech – is the Big Pharma giant learning from its mistakes?

Pfizer being Pfizer, the new company – SpringWorks Therapeutics – has been spun-out on some scale and backed to the tune of $130 million in Series A funding, with additional investment from Bain, Orbimed and LifeArc.

In addition to developing drugs it inherits from Pfizer, SpringWorks will also seek out assets discarded by other pharma and biotech manufacturers – ViewPoints: Pfizer hands off rare disease candidates to SpringWorks- so what's in the bag?

It is not the first player to pursue this strategy, indeed, a number of other companies have been built largely around this model…

Axovant tumbles

…One of which, Axovant Sciences, reported eagerly anticipated data this week for its experimental Alzheimer's disease treatment intepirdine; an asset it acquired from GlaxoSmithKline for $5 million in 2014.

Alas, intepirdine failed to better placebo at treating patients with mild-to-moderate Alzheimer's disease in the Phase III MINDSET trial, Axovant revealed on Tuesday – ViewPoints: Another Alzheimer's setback – Axovant's name added to an unfortunate roll call.

Clinical precedent was not in Axovant's favour; since 2002, 99 percent of Alzheimer's disease drugs tested in pivotal-stage trials have failed. Nonetheless, Axovant's argument that intepirdine had not previously been tested with the right study design was compelling enough to attract hundreds of millions of dollars in investment; so too, was the high-profile hiring of CEO David Hung in April.

However, shares in Axovant slumped more than 70 percent when top-line results from MINDSET were announced.

EMA says Amsterdam move would avoid total chaos

Results from an internal staff survey run by the European Medicine Agency (EMA) indicate that re-location to Amsterdam would represent a best-case scenario if the regulator is required to leave its UK headquarters as a result of Brexit.

In comparison to 18 other cities that are vying to host a relocated EMA, it is likely that fewer employees would leave the agency if it moved to Amsterdam, the survey revealed. Results – based on feedback from 92 percent of the EMA's current workforce – suggest that 81 percent of staff could be retained if relocation to the Netherlands materialised, but retention rates would fall significantly below 30 percent for some locales.

With the EMA suggesting it would be unable to operate under these circumstances, some potential locations are presumably unviable. In a best-case scenario, nevertheless, the EMA has suggested that the approval rate for new drugs will slow for at least two years after any move.

A change of heart at the FDA?

At the time of writing, it looks incredibly unlikely that PTC Therapeutics' Duchene muscular dystrophy (DMD) treatment Translarna will be approved by the FDA. Briefing documents released by the agency ahead of an advisory committee (AdCom) meeting on Thursday (September 28) were critical of data supplied to support PTC's approval application – ViewPoints: FDA strikes back at a persistent PTC Therapeutics.

The FDA's assessment of Translarna raises further question marks over its conditional approval in Europe and is also reflective of the agency's continued evolution under the leadership of Commissioner Scott Gottlieb.

Last year, the FDA controversially approved Sarepta's DMD treatment Exondys 51, despite a negative vote from an AdCom panel and critical comments from agency reviewers. This was perceived as an unusual showing of leniency and one known to trigger significant debate within the FDA. It looks unlikely, however, to have set a precedent.

See also  ViewPoints: FDA moving forward in digital health as it seeks out a smoother path to real world data

FirstWord Physician Intelligence

FirstWord Pharma PLUS assesses various news events through the eyes of prescribers and leading clinical experts in the field, in the form of snap-polls and interviews…

There are promising signs for biosimilar Herceptin developers in Europe and encouraging noises from pulmonologists and primary care practitioners – in both the US and EU5 – towards GlaxoSmithKline's Trelegy Ellipta.

Less encouragingly, recent setbacks for two therapies aiming for regulatory passage into the NASH market may be indicative of commercial challenges that lie ahead, say gastroenterologists and hepatologists.

The competitive landscape between Xtandi and Zytiga is likely to remain fluid on the back of recent clinical trial disclosures, argued a leading oncologist, while another key opinion leader talked up the opportunity for AstraZeneca's Farxiga in type 1 diabetes.

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