Pfizer dodges costs and scrutiny by springing loose rare disease assets

Pfizer’s decision to spin out four assets into a new venture-backed group raises the question of whether Springworks Therapeutics can avoid the developmental missteps of its big pharma patron.

With little to link the assets other than the banner of rare diseases, Springworks will be working in three distinct disease areas with projects that were apparently not compelling enough for Pfizer to commit resources to. Should any of these succeed, the tiny Springworks might be better placed to launch with a hefty orphan pricetag without drawing much political heat.

Not worth the time – for now 

Offloading stalled pipeline assets is a tried and true big pharma strategy, with Astrazeneca showing the way on “externalisation”.

Pfizer’s research & development arm does not appear to have the necessary focus to advance these agents, as demonstrated by the fact that it was willing to spend $56m for a failed sickle-cell drug when it acquired Senicapoc and then do absolutely nothing with it. Moreover, even with rich orphan pricing, there is always the question of whether the revenue gain, in the scheme of Pfizer’s annual income of more than $50bn, would be worth the R&D expenditures.

Moreover, with Pfizer often being cast as public enemy number one in the pricing and tax debates, the New York-based group may have no interest in trying to launch an orphan drug if any of these still long-shot bets turn out winners.

Rare cancer, blood and PTSD

Springworks will take two of Pfizer's four projects immediately into phase III with $103m from a series A round backed by Bain Capital Life Sciences, Bain Capital Double Impact, Orbimed, Pfizer and Lifearc. Those two projects are linked in some sense, in that they aim to treat rare cancers – PF-03084014 in desmoid tumour and PD-032590 in neurofibromatosis.

The former project is a gamma secretase inhibitor that has completed one 72-patient phase I trial looking at safety, pharmacokinetics and early signs of efficacy, and the latter a Mek inhibitor currently in investigator-led studies in solid tumours and colorectal cancer.

Springworks's not-so-new pipeline
Project New indication Last seen
PF-03084014  Desmoid tumour Terminated phase II trial in advanced breast cancer, 2015
PD-0325901 Neurofibromatosis Ongoing investigator led trials in advanced KRAS mutant malignancies and colorectal cancer
Senicapoc Hereditary xerocytosis  Phase II trial in exercise induced asthma, 2009
PF-0445784 PTSD Terminated phase II trial in PTSD, 2014

The other two projects are further afield. Senicapoc is being developed in hereditary xeroctosis, a condition in which red blood cells become dehydrated, and the Faah inhibitor PF-0445784 will be tested in post-traumatic distress disorder (PTSD).

Pfizer bought senicapoc for $56m when it acquired Icagen in 2011 – no clinical trial records exist since 2009, when it had been studied in asthma. Earlier, it had been tested in sickle cell disease in partnership with Johnson & Johnson, and the failure of that effort led to the big pharma pulling out.

Faah inhibition, meanwhile, has been linked with haemorrhagic and necrotic brain lesions in work done with the Bial project BIA 10-2474; J&J subsequently suspended a trial of its own Faah agent JNJ-42165279 in major depressive disorder shortly thereafter (Therapy focus – Why it might be FAAH better to take a break, January 22, 2016).

PTSD is of course not the orphan disorder that the previous three are, but it is recognised as an unmet medical need, with combat veterans, among others, suffering at significant rates.

There is no denying these conditions deserve better treatments, and Pfizer is probably too big to give these projects the attention they need to advance. It just so happens that letting a smaller player do the heavy lifting through launch would fit with a longer game Pfizer is playing to maximise revenue without drawing political heat.

To contact the writer of this story email Jonathan Gardner in Virginia at [email protected] or follow @ByJonGardner on Twitter

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