A look back at 2019
The FDA approved 48 novel drugs last year and a handful of new biological products. We took a closer look at some of the key trends behind 2019's roster of new approvals, including perception of the regulator's relative leniency and willingness to process new drug applications faster than scheduled.
Read it here.
Next week we will publish our annual preview looking at which drugs are set to shape the pharmaceutical industry in 2020.
Merck & Co. makes more KRAS moves
Merck & Co. entered an agreement with two subsidiaries of Otsuka to develop small-molecule inhibitors against several cancer drug targets, including the KRAS oncogene, the companies announced Monday.
KRAS inhibition is poised to remain a red-hot area in 2020, building on progress made last year by the likes of Amgen and Mirati Therapeutics. More clinical data is expected to emerge over the next 12 months including provisional results for KRAS inhibitors used in combination with cancer immunotherapies.
Previewing the year ahead, analysts at Baird noted that it will be particularly interesting to see if other large cap pharma companies quickly enter the fray, given that "this is rather straightforward small-molecule chemistry after all."
Other key themes for 2020
What other biopharma trends will shape the year ahead? Taking place next week in San Francisco, the annual JP Morgan Healthcare Conference will help set the scene for 2020; FirstWord will be in attendance.
In the meantime…there was notable progress in the fields of gene therapy and RNAi last year, with momentum expected to continue in 2020. The next 12 months should also provide a clearer view on the clinical and commercial potential of bi-specific monoclonal antibodies and allogeneic (or 'off the shelf') CAR-T products.
With it being an election year in the US, there will likely be considerable chatter around drug pricing reform, though whether material changes occur is another matter.
Perception of the FDA's leniency could also be tested in 2020 by a number of high-profile regulatory decisions and potential AdCom meetings, with most focus poised to be sharpened on Biogen's bid to secure approval of aducanumab, its controversial Alzheimer's disease treatment.
More competition for complement inhibitors
Apellis Pharmaceuticals announced top-line results from a Phase III study showing that its experimental C3 inhibitor pegcetacoplan (also known as APL-2) demonstrated superiority over Alexion Pharmaceuticals' Soliris in adults with paroxysmal nocturnal haemoglobinuria (PNH) this week. Apellis' shares rose as much as 58% on the news, before paring some of the gains to end the day up about 20%.
Investor reaction likely reflects some uncertainty as to how large the commercial opportunity for pegcetacoplan may be (see ViewPoints: PEGASUS gives Apellis inside track – to what?). Nevertheless, Apellis' success provides further evidence of increased competition in the market for drugs that target the complement system. Alexion and UCB both announced notable acquisitions in this space late last year and Novartis and Regeneron Pharmaceuticals unveiled early-stage data for their own complement inhibitors.
Is this a PD-L1 comeback against Keytruda?
While most of the checkpoint inhibitor world has become comfortable with the concept of commoditisation for the PD-(L)1 drug class, fresh data this week for Keytruda (Merck & Co.) and Bavencio (Merck KGaA/Pfizer) could add some new inklings of differentiation.
Keytruda suffered a rare Phase III trial miss with Merck reporting that the PD-1 inhibitor plus chemotherapy missed the overall survival (OS) endpoint of the Keynote-604 trial evaluating first line treatment of small-cell lung cancer. However, the combo did improve progression-free survival.
Separately, Pfizer and Merck KGaA said that Bavencio met the primary endpoint of the Javelin Bladder 100 trial, improving OS when used as a maintenance therapy in first-line bladder cancer patients, the first immunotherapy to do so.
More analysis of the potential implications of these study readouts here.
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