Friday Five – Your weekly pharma review

Trumped – No end in sight to drug pricing debate

As FirstWord predicted last month – and Allergan CEO Brent Saunders warned last week – Donald Trump has, for the first time since winning the Presidential election, cited a reduction in US drug prices as a goal of his administration. In familiar Trump fashion the details are lacking, but in an interview with Time magazine the President-elect said "I'm going to bring down drug prices. I don't like what's happened with drug prices."

There had initially been some relief among investors when Hilary Clinton fell to a surprise election defeat; in the preceding months pharmaceutical and biotech shares had been hurt by a series of tweets from Clinton and fellow democrat Bernie Sanders. Trump, of course, has embraced Twitter as a key medium both pre- and post-election; speaking at the Forbes Healthcare Summit last week, Saunders had suggested that Trump's future Twitter activity could potentially have an even greater impact on investor sentiment than Clinton's did in the past.

To this end, Saunders has called on drug manufacturers to seize initiative in the drug pricing debate by embracing self-regulation. Allergan has previously announced a 'social contract' with patients, which both limits net price increases and provides free drug access to those meeting certain criteria. Perhaps marking the beginning of a trend, Novo Nordisk publicised its own 'perspective on affordability' this week, which limits annual net price increases to single-digit percentages, seeks to reduce out-of-pocket patient costs and will support efforts to make the US pricing system more transparent. 

ViewPoints: Allergan and Express Scripts applaud Novo Nordisk's stance on drug pricing

Jardiance's CV label claim – will physician adoption continue to increase?

Having recently written off its most advanced Alzheimer's disease asset solanezumab – see KOL Views: Neurology expert cautions against over-interpreting results from EXPEDITION3 of Eli Lilly’s solanezumab – Eli Lilly was in need of a fillip. It received one late last week when the FDA approved its type 2 diabetes treatment Jardiance (an SGLT-2 inhibitor co-marketed with Boehringer Ingelheim) for the prevention of cardiovascular (CV) death in patients with type 2 diabetes and established CV disease.

Jardiance became the first diabetes drug to demonstrate a CV benefit in a dedicated outcomes study last year. FDA approval means these data can now be promoted to US physicians. According to key opinion leader interviews conducted by FirstWord, adoption of Jardiance has already increased as a result of positive study data with this trend reportedly causing some consternation among sales representatives for competing SGLT-2 inhibitors; particularly as there is a widely held view that any CV benefit will be shown as a class-effect when data from subsequent outcomes studies are reported.

To gauge the impact of this FDA approval on usage of Jardiance, FirstWord is polling US-based endocrinologists and primary care practitioners. Results from this survey will be published for FirstWord Pharma PLUS clients in the next few days – see Physician Views: Jardiance approved by FDA for reducing cardiovascular death in diabetes patients – what impact on treatment trends?

Sanofi said to be interested in Actelion, opens up on Medivation pursuit

Reports this week suggest that Sanofi could emerge as a rival bidder to Johnson & Johnson for the Swiss biotech Actelion. Given the difficulties that Johnson & Johnson is said to have encountered during its negotiations with Actelion, this makes for intriguing speculation, while some may question the authenticity of these rumours altogether given Johnson & Johnson's significantly greater financial firepower.

Should a move by Sanofi materialise it would suggest a new found preference for revenue and earnings stability – provided by Actelion's market-leading pulmonary arterial hypertension portfolio – over M&A discipline. Any near-term M&A on Sanofi's part also needs to be viewed through the lens of its failed attempt to acquire Medivation earlier this year, where it was aggressively out-bid by Pfizer.

In a note to investors published by Credit Suisse this week, research analysts from the investment bank provided details of a recent conversation with Sanofi CEO Oliver Brandicourt, where he described Medivation as a unique oncology acquisition target because of its marketed product (Xtandi for prostate cancer) and an R&D pipeline.

Brandicourt conceded that Sanofi, in its bidding strategy, made a mistake in assuming that US rivals would not compete aggressively for a marketed asset "fully encumbered" by Medivation's Japanese marketing partner Astellas. Sanofi is unlikely to find another oncology asset that fits each of its M&A requirements, the CEO added. These 'three boxes' comprise a positive impact on earnings, R&D pipeline and strategic rational. 

With rare diseases beyond Sanofi's current focus of genetic conditions, noted by Brandicourt as one area of M&A focus, interest in Actelion cannot be ruled out.

CAR-Ts – too effective?

CAR-T therapies took centre stage at this year's annual meeting of the American Society of Hematology (ASH) in San Diego, with bluebird bio and Celgene serving up a pre-congress appetiser.

Early-stage data for bb2121 presented at the EORTC-NCI-AACR Molecular Targets and Cancer Therapies symposium, held in Munich just days ahead of ASH, provides the most compelling evidence to date that CAR-T therapies could be used to treat multiple myeloma.

ViewPoints: bluebird validates Celgene’s big bet on BCMA

At ASH, Kite Pharma and Novartis both reiterated their intention to file CAR-T therapies (KTE-C19 for relapsed/refractory aggressive B-cell non-Hodgkin lymphoma and CTL019 for paediatric and young-adult patients with relapsed/refractory B-cell acute lymphoblastic leukaemia, respectively) with the FDA during the first quarter of 2017, with Kite also confirming that it has initiated a rolling submission. Updated data for both products was also presented.

With safety concerns over lead asset JCAR015 persisting, Juno Therapeutics appears to be losing ground on its rivals. The company was able to present encouraging data for what appears to be a safer CAR-T product in JCAR017, but switching primary focus to this asset would likely push regulatory submission back to 2018 at the earliest.

Spotlight On: Juno gets lapped in CAR-T race as Kite and Novartis vie for finish line

Although questions have recently been raised about the true potential of CAR-T therapy, in light of Juno's toxicity issues, in an interview with FirstWord Robert Nelsen of ARCH Venture Partners, an early backer of some of the field’s heavy hitters, dismissed the naysayers and suggested the future has never been brighter. An unprecedented level of activity has been both a blessing and a curse for CAR-T programmes, says Nelsen.

"When these things go after cancer they really go after it, and they can cause really toxic events. However, these seem mostly to be related to the dose being used and the amount of disease burden in late-stage patients," he said, implying that with time and experience clinicians will figure out how best to use these agents and the better ones that are already being devised. "We have spent years worrying about how to kill tumours and the biggest problem of these new CAR-T cells is that they are killing too much tumour and doing it too quickly," Nelson remarked

Read the rest of the interview here.

All change at the FDA

It was confirmed this week that director of the FDA's Office of New Drugs (OND) – John Jenkins – is to retire in January and will temporarily be replaced by Janet Woodcock, director of the Centre for Drug Evaluation and Research. One of Jenkins' last public actions in his role at the FDA was to challenge the development programme used by Sarepta Therapeutics for its Duchenne muscular dystrophy treatment Exondys 51. The drug was controversially approved by the FDA in September with this decision championed by Woodcock.

KOL Views: Regulatory expert sounds off on the FDA’s controversial decision to approve Sarepta’s Exondys 51

Jenkins has played down any impact from the Sarepta approval and suggested his retirement has been a few years in the making. A regulatory insider – speaking to FirstWord anonymously – said that his departure "may well create greater uncertainty and loss of predictability," particularly given Jenkin's 15-year tenure as head of the OND and his 25-year career at the FDA.

"There will be no lower bar, of that I'm sure," they added, "although one fear, post-election, is that this could start a cascade of senior people leaving.  This, coupled with no obvious replacement for Jenkins, could be very unsettling for staff." There is a sense of uncertainty within the FDA – not to mention other government agencies – as staff try to work out what the goals of the incoming administration are, the source added.

On Wednesday, Bloomberg reported that Jim O'Neill, a venture capitalist and libertarian, has been short-listed by Donald Trump to head the FDA. Quotes made by O'Neill at a biotech conference in 2014, where he argued that new drugs should be approved once safety is established, and efficacy thereafter, have quickly resurfaced; as Matthew Herper at Forbes points out, pharma has reason to be concerned. 

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