Friday Five – This week’s key news stories

More CAR-T choice = more confusion?

US oncologists now have the choice of two pioneering CAR-T therapies with which to treat lymphoma patients.

So which product - Gilead Sciences' Yescarta, approved in this indication last year, or Novartis' Kymriah, approved by the FDA this week, is likely to be used more frequently?

It's hard to tell. Yescarta benefits from more favourable labelling, which shows superior 'best' overall response and complete remission (CR) rates over Kymriah, but key opinion leaders believe six-month CR rate data will be the key determiner. Based on currently available data, they think it's hard to single one of the two products out.

ViewPoints: A CAR-T showdown

Novartis told FirstWord this week the two CAR-T therapies cannot be compared against one another as they are "significantly different" in terms of construct and manufacturing process, but also as they have been evaluated in studies with differing designs and patient populations.

Other factors such as toxicity and whether treatment can be provided in an outpatient setting will likely come to the fore - Physician Views Poll Results: Kymriah’s toxicity and outpatient use could prove critical in showdown with Yescarta.

See also ViewPoints: CAR-Ts make baby steps on securing reimbursement

Right drug, wrong price?

Regeneron Pharmaceuticals and Sanofi previously disclosed plans to improve access to their PCSK9 inhibitor in March. This week they announced a deal with Express Scripts to secure exclusive drug class status for Praluent on the pharmacy benefit manager's (PBM) national preferred formulary.

At what cost? - Regeneron and Sanofi have agreed to significantly reduce the list price of Praluent.

They will be hoping that increased volumes drive up Praluent revenues, which have lagged those of Amgen's competitor drug Repatha; but make no mistake, both these drugs have struggled to gain share since coming to market a number of years ago despite their potential to be practice changing in certain patients.

One view is that by aggressively lowering Praluent's price, Sanofi and Regeneron have conceded defeat to payers and spoken for Amgen too with this decision; the biotech will presumably have to follow suit in its own negotiations with PBMs.

ViewPoints: Regeneron/Sanofi steal a march on Amgen with Express Scripts deal – but at what cost?

Issues mount for Esperion

Reduced pricing for the PCSK9s is also bad news for Esperion, which is developing bempedoic acid; an experimental cardiovascular disease therapy that is being studied primarily in combination with existing oral treatments such as statins. Esperion was hoping to pitch bempedoic acid as an easy to prescribe therapy that may lower LDL-cholesterol less aggressively than the PCSK9s, but at a significantly cheaper cost.

This potential strategy looks to have been undermined by Sanofi and Regeneron's pricing pledge and Esperion may have more to worry about closer to home.

Results from a first longer-term Phase III study were announced this week showing an LDL-C reduction versus placebo that disappointed some investors and a higher rate of fatalities among patients prescribed bempedoic acid.

Esperion did its best to contextualise both the efficacy and safety data by highlighting the high-risk population enrolled into its study, but results raise the question of whether the FDA will approve a drug that remains mechanistically misunderstood without outcomes data.

ViewPoints: Events conspire to put the heat on Esperion

Results from an ongoing outcomes study are expected in 2022, but Esperion hopes to file bempedoic acid next year. If it can't, its bid to take the PCSK9 inhibitors on my have failed before it has even started.  

Big Pharma plays down large-scale M&A

With Takeda and Shire thrashing out a takeover agreement, it has been suggested this deal could trigger a wave of consolidation across the pharmaceutical sector. 

But executives at Pfizer and Merck & Co. both played down the prospect of large acquisitions during their first-quarter earnings calls this week.

See ViewPoints: Investors rattled over Pfizer's lack of M&A interest and ViewPoints: Merck & Co. urged to look beyond Keytruda.

Bankers and lawyers may be particularly disconcerted to have heard Pfizer talking up its internal R&D pipeline, given that large-scale M&A is embedded in the company's recent history; and some would say its corporate DNA.

The flipside, of course, is that no company - least of all an old hand like Pfizer - will talk up its willingness to go on a spending spree, but smaller 'bolt on' deals appear to be the new vogue.     

Johnson & Johnson goes viral with immuno-oncology aspirations

And if there is a model to follow for the use of externalisation to bolster in-house R&D productivity, competitors could do worse than look at Johnson & Johnson's deal making over the past decade.

The company - more specifically its Janssen division - announced its latest deal this week; the acquisition of BeneVir Biopharm for $140 million upfront and up to $900 million in milestone payments.

This particular 'bolt on' will give Johnson & Johnson a preclinical oncolytic viruses platform and potentially allow it to play catch up in the immuno-oncology market (an area where in spite of its strong R&D showing, the company is conspicuously absent from).

ViewPoints: Better late than never? Johnson & Johnson playing catch-up in I/O

The deal follows Merck's acquisition of Viralytics - another deal focused on the potential of oncolytic viruses - a few months ago - see ViewPoints: Merck & Co. hits on a viral strategy to stay in the I/O game.

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