Pfizer doubles down on gene therapy pipeline with $70M Sangamo buy-in

After deals and acquisitions with Spark Therapeutics and Bamboo, Pfizer is once again looking to bolster its rare and gene therapy pipeline as it outlines a new drug pact with Sangamo.

The collaboration license agreement focuses on the development and eventual sale of the biotech’s gene therapy programs for hemophilia A, including SB-525, one of Sangamo’s four lead product candidates.

This early candidate is slated to enter the clinic this quarter, centering on testing safety as well as blood levels of Factor VIII protein, and other efficacy endpoints.

Sangamo gets $70 million upfront from the Big Pharma, and could gain $475 million in biobucks and sales royalties on any medications from the collaboration that gain approval.

Under the deal, Sangamo will take the lead on the SB-525 phase 1/2 test as well as unspecified manufacturing activities.

Pfizer, meanwhile, will be “operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities” for the therapy, as well as any additional products, if any.

Sangamo will also work with Pfizer on manufacturing and technical ops using viral delivery vectors.

SB-525 works as a AAV vector carrying a Factor VIII gene construct driven by a synthetic, liver-specific promoter. The FDA has already cleared the start of human trials for SB-525, and given it an orphan drug tag.

The deal has proved powerful for Sangamo, with its shares jumping 44% after hours on the news last night.

This marks another step into the new world of gene therapies for Pfizer, coming less than a year after its $700 million buy of Bamboo Therapeutics, adding advanced recombinant adeno-associated virus (rAAV)-based gene therapies to its pipeline.

It also has a long-standing deal with Spark Therapeutics, in hemophilia, penned in 2014. Back in January, Pfizer in fact paid a $15 million milestone bonus to Spark for hitting its marks in the ongoing hemophilia B phase 1/2 trial FDA breakthrough-tagged SPK-9001.

Pfizer also has a series of preclinical gene therapies, including a neuromuscular candidate for Duchenne muscular dystrophy (DMD), as well as preclinical candidates to treat Friedreich’s ataxia and Canavan disease, and a phase I candidate for giant axonal neuropathy.

Pfizer also gained an operating gene therapy manufacturing facility that Bamboo bought from the University of North Carolina last year.

The pharma also has several academic research agreements, including one with King’s College London to develop a series of rAAV gene therapy vectors and another with the University of Iowa Research Foundation to develop a potential gene therapy for cystic fibrosis.

And its partnered with Emeryville, CA-based Molecular Therapeutics (4DMT) to discover and develop targeted next-generation rAAV vectors for cardiac disease; it made an investment in the company a few years back.

Once seen as the next big thing in research, gene therapies have however come under pressure in recent months about just how viable they are on the market. After struggling for years to make a commercial success out of Glybera, the world’s first approved gene therapy, uniQure recently called it quits on the treatment.

The drugmaker said it wouldn’t bother asking European authorities to renew the $1-million-plus gene therapy’s marketing authorization when it expires in October, and comes after it abandoned plans to gain an approval in the U.S. Reports from MIT Technology Review suggest only one patient ever used the med.

GlaxoSmithKline has also been struggling in Europe with its “bubble boy syndrome” gene therapy Strimvelis. Mindful of Glybera’s cost, GSK put its price tag at half that of Glybera, at $665,000, and also offered a money-back guarantee.

It’s been approved in Europe for nearly a year, but it only treated its first patient this month, according to Business Insider.

Treatment is tough as the drug is not so much manufactured as it is “created” for each individual patient, with a site in Italy currently the only approved site in the world for this type of manufacture, and thus the only place where patients can be treated. Only around 15 patients in Europe are believed to have the condition.

Other biotechs are however working on the manufacturing side in order to try and make these therapies more available for patients, and thus open up their viability.

There are already a number of medications on the market for hemophilia, such as from Biogen spin-off Bioverativ and Sobi, with gene therapy predicted by some also working in the space, including uniQure and BioMarin, to be the next class for treating the blood disorder.

But speaking to FierceBiotech at the start of the year, Bioverativ’s new chief and former Biogen exec John Cox told me that while they are to working on gene therapy approaches to hemophilia, there are reasons to be cautious: “There are of course question marks over gene therapy: The obvious one is safety, because of the history here, and this is a risk-averse population, for good reason, and the other question is naturally over efficacy, and how long does it last, as well as manufacturing, scale and so on.

“We’re all hoping for a cure, and of course we’re doing work on gene therapy now, but I don’t think people are looking at these now as a permanent cure; the questions are over durability, rather than cure.”

He said that investors and even doctors talk a lot about gene therapy in the hemophilia space, but that if you talk to hemophilia A patients about what they really want, “being able to dose, once a week [which is the target with its candidate, or even just less frequently, is what they want.”