4. Pfizer

Office lobby
The Big Pharma is still one of the biggest players in the world when it comes to medical R&D spend.

R&D budget: $7.87 billion
Change from 2015: Up 3%
Total 2016 revenue: $52.82 billion
R&D budget as percentage of revenue: 14.8%

For many years before the loss of its Lipitor (atorvastatin) patents, Pfizer was hit by waves of criticism for its failure to develop new blockbusters to replace the big drugs that had bulked up its revenue. Those critics wanted to see more outsourcing, more partnerships and smaller in-house research empires. Pfizer has in many ways steered toward that course, and 2016 saw more evidence of this.

The Big Pharma is still one of the biggest players in the world when it comes to medical R&D spend. Although it no longer tops the list, it still spent big on buying up other companies’ research last year with its $14 billion buyout of cancer biotech Medivation and its blockbuster prostate cancer med Xtandi (enzalutamide).

Its newly expanded pipeline includes the late-stage PARP candidate talazoparib, as well as blood cancer drug pidilizumab, which is in midstage testing. A candidate for bladder cancer and a multiple myeloma therapy are also now in its early-stage pipeline.

This deal came, however, after it failed to buy Allergan in a $160 billion-plus megamerger that was dropped a year ago, after new tax rules made a pact less appealing, and likely prompted Pfizer’s desire to steal the in-demand company away from the likes of Sanofi.

Pfizer did in fact spend much time and money in 2016 on R&D outside of its pipeline, with a big focus on its near-$1 billion checkpoint inhibitor deal with German Merck for avelumab (Bavencio), homing in on assessing its efficacy when combined with a host of other drugs from across a series of classes.

The two are testing the drug in many different cancers, including lung cancers, renal cell carcinoma and ovarian, gastric and bladder cancer, and Merkel cell carcinoma, which in March this year became its first approval.

Pfizer is playing catch-up in this new cancer class space and is some years behind leaders Bristol-Myers Squibb, Merck & Co. and Roche, but still managed to beat out AstraZeneca to be the fourth to market.

RELATED: Pfizer, Merck KGaA score watershed FDA nod on cancer immunotherapy Bavencio

A little more quietly than its Medivation deal, Pfizer also bought up gene therapy player Bamboo Therapeutics, adding advanced recombinant adeno-associated virus-based gene therapies, which it expects will complement its existing rare disease and gene therapy portfolios.

These include a preclinical neuromuscular candidate for Duchenne muscular dystrophy, a rare disease that has seen a number of recent clinical disappointments (but also two recent FDA approvals), as well as preclinical candidates to treat Friedreich’s ataxia and Canavan disease and a phase 1 candidate for giant axonal neuropathy.

But Pfizer was also hit last year when its 13-year veteran senior principal scientist Min-Jean Yin appeared to be cut from the company, coming after the U.S. Big Pharma giant initiated a series of research article retractions after allegations of data manipulation.

It also saw a major pipeline failure, saying in November that it was to dump all work on its proprotein convertase subtilisin/kexin type 9 inhibitor (PCSK9i) bococizumab due to weak data and an “evolving treatment and market landscape for lipid-lowering agents.”

The shock announcement came as the Big Pharma found that bococizumab was “not likely to provide value to patients, physicians, or shareholders” after its ability to lower LDL-C weakened over time and increased the risk of some adverse events. In short, it didn’t really work, and it caused side effects.

This was a big knock for the company, coming as Amgen and the Medicines Company/Alnylam look to carve out a new, and potentially lucrative, market in the PCSK9i space.

>> Check out Pfizer’s latest product pipeline.

4. Pfizer

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