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Pfizer pares back early-stage research in rare disease, cancer

Dive Brief:

  • Pfizer is trimming early-stage research in rare diseases and cancer, seeking to “externalize” development by licensing out programs, including viral vector gene therapies, a spokesperson confirmed Thursday. Late-stage gene therapy programs in hemophilia and muscular dystrophy won’t be affected.


  • The shift in strategy, first reported by Barron’s, may also involve selling off a gene therapy manufacturing facility in Durham, N.C., the newspaper reported. Pfizer plans on continuing early genetic medicine research that involves non-viral vector delivery tools, the spokesperson said.


  • Big drugmakers often sell off or license assets to cut costs or try to improve R&D success when their pipelines get too large. Last year, Pfizer partnered with Roivant to form two separate companies to develop Pfizer-originated drugs in autoimmune and skin conditions, and earlier ceased work in neurological diseases.

Dive Insight:

Pfizer’s moves will allow it to “focus on programs where our innovation and investments are differentiated and where we are best positioned to generate high-impact medicines and vaccines,” the spokesperson said in an email.

Those programs include the viral vector gene therapies in hemophilia A and B and Duchenne muscular dystrophy, which are approaching some key milestones. The hemophilia B gene therapy has shown it can control bleeding in a late stage trial, while Pfizer’s hemophilia A and Duchenne muscular dystrophy programs are also in late-stage trials, with results expected in the next two years.

As for the “highly innovative, niche programs” that will be licensed to biotechs, the company is seeking “to maximize the success for these potential medicines,” the spokesperson said. “Pfizer may maintain ties to some of these programs through strategic investments.” The partnerships with Roivant involved forming separate companies to shepherd the experimental drugs through development phases, for example.

Barron’s reported that some of the oncology assets that will be sold or licensed are those being developed by an R&D center formed around the former Array BioPharma, which Pfizer bought for $11.4 billion in 2019. That acquisition gave Pfizer two targeted cancer drugs called Braftovi and Mektovi that had combined sales of $285 million through the first nine months of 2022.

Pfizer may seek to invest more into acquisitions, too. In the past four years, Pfizer has spent tens of billions of dollars to acquire companies with already approved drugs, such as Array, BioHaven Pharmaceutical and Global Blood Therapeutics, as well as others with products still in development, like Arena Pharmaceuticals, Reviral and Trillium Therapeutics.

Externalization is a common strategy employed by companies looking to cut R&D costs or shed shrinking products to boost profits. AstraZeneca became a case study over the last decade as it ditched some prized gastrointestinal and mental health medicines in a bit to reshape itself as an oncology and respiratory powerhouse.

Pfizer is in a different position as a company, however. It is still earning blockbuster sales from its COVID-19 vaccine and antiviral drug, while some of its key assets in infectious disease and cancer are still growing.

The company is preparing for a wave of launches resulting from its current research and development work — as many as 19 over the next 18 months, the spokesperson said.