Earlier this week, BioPharma Dive asked its readers which of these four companies — Pfizer Inc., Shire plc, Amgen Inc., or Sanofi SA — would be most likely to acquire gene therapy drugmaker Spark Therapeutics, Inc.
The thought behind the poll was driven by the recent decision by a Food and Drug Administration advisory committee to unanimously recommend approval of Spark’s gene therapy for a rare form of pediatric blindness, making approval of the drug by its user fee action date in January highly likely. An approval undoubtedly would also make Spark a takeout target for a number of companies.
The four companies were all chosen because a case could be made for each of them for buying out the biotech.
While the results from readers were split, Pfizer led the way — garnering 35.4% of the votes. Amgen came in a close second with 26.5% of the vote, Shire followed with 21.8%, and Sanofi rounded out the votes with 16.3%.
In Pfizer's blood
While we didn’t ask readers to explain their votes, we have a pretty good idea why Pfizer was the top pick. Not only does Spark have a history-making gene therapy set up for approval, but several more are in the pipeline. It currently has three other drugs in early-to-mid stages, including two gene therapies for hemophilia, and the hemophilia B asset is already partnered with Pfizer.
The big pharma got an amazing deal for the drug, signing up for the collaboration in 2014 when gene therapies were still considered a risky bet. Pfizer paid only $20 million upfront and promised $260 million in milestone payments. But the worldwide hemophilia market was worth about $15 billion in 2015, and is expected to grow to $25 billion by 2024, according to a report by Transparency Market Research.
Rare diseases are one of the six key spaces that Pfizer operates in currently. The big pharma has been building up its arsenal in the space. It recently inked another hemophilia A deal with Spark competitor Sangamo Therapeutics Inc., likely hedging its bets that one of the biotechs could have a gene therapy approach that works.
Pfizer also has some early prospects in the bleeding disorder in very early development and in 2016 picked up gene therapy development company Bamboo Therapeutics, giving it a small slate of rare disease therapies as well as a gene therapy manufacturing plant.
The might of Amgen
The original big biotech has been the butt of plenty of jokes about its litigious behavior, and with the entrance of biosimilars, it seems Amgen has been engaged in even more lawsuits than usual. The company not only has developed biosimilars of its own that it is fighting to bring to market, but also has a slew of products that are facing biosimilar competition.
Beyond that, its cholesterol drug Repatha (evolocumab) continues to flounder. While Amgan has a few promising late-stage prospects and seems to have a full early-stage pipeline, it could use a few acquisitions that could help it fill the gap.
But litigious behavior isn’t the only thing Amgen is known for; the big biotech has always been a pioneer of hard-to-develop therapies (being one of the first companies to start developing biologics en masse). Gene therapies would be a good fit for a company that is used to taking on daunting development prospects and hard-to-manufacture products.
Genetic disease pioneer
Spark could be an excellent fit for Shire. The company has long been a pioneer of genetic disease drug development. It successfully developed and commercialized rare genetic disease drugs Cinryze (C1 esterase inhibitor) and Firazyr (icatibant), as well as others.
It also added ViroPharma to its portfolio in 2013 to beef up its rare disease offerings. But it's most recent acquisition, the $32 billion pick up of Baxalta, further bolstered those offerings with hemophilia candidates. Shire recently chose to scrap Baxalta's pipeline candidate in hemophilia B, indicating inconsistent results for the product — that leaves an open slot that Spark could fill.
The company is also working to build up its presence in ophthalmics. It got approval of Xiidra (lifitegrast) last year and has been trying to make a name for itself in the space. A potential approval in rare blindness that Spark has coming up would be a nice complement.
The French drugmaker has been trying — and failing — to make a solid acquisition that could bolster its pipeline. First it lost out to Pfizer on Medivation Inc., and then Actelion Pharmaceuticals Ltd. slipped through its fingers and right into Johnson & Johnson's hands. M&A has not been its strong suit of late.
And even though its diabetes franchise has been flailing, Sanofi's Genzyme unit has been a bright spot for the company. The unit is the rare disease powerhouse at the company, and recent partnerships with Regeneron Pharmaceuticals have been fruitful in helping the French drugmaker gain approval of a couple of potential blockbusters. A gene therapy company would be a nice addition.
Not just Spark
High tide raises all boats, so to speak. The potential success of the gene therapy biotech means that several other gene therapy companies are also acquisition targets. UniQure N.V., BioMarin Pharmaceutical Inc., Bluebird Bio Inc. and Sangamo are now also worth the due diligence. Keep an eye on the American Society of Hematology meeting in early December for some more data from several of these companies that could further bolster their prospects.