Anyone puzzled by Pfizer’s decision to scrap an alliance with Repligen covering a spinal muscular atrophy project need look no further than Roche’s takeover just 10 days earlier of Trophos, a private company active in the same disease.
With two projects in development, and a venture investment backing a third, the Swiss firm now has a grip on spinal muscular atrophy that makes trying to compete with an early project like Repligen’s pretty pointless. While Isis Pharmaceuticals and Novartis are still in the game here, their positions must now be seen as less secure too.
The more advanced of Novartis’s two SMA projects, BVS857, is in a phase II trial ending in December, while the two phase III studies of Isis’s ISIS-SMNRx are not due to finish until 2017. Trophos’s olesoxime, meanwhile, might be filed on its existing phase II/III data (Roche muscles into the lead in rare wasting disease, January 16, 2015).
Against such competition in a rare disease like SMA it made perfect sense for Pfizer to give up on Repligen’s RG3039, which had started phase I two years ago. No results from this study had been reported, so it seems a fair assumption that data were uninspiring compared with what Trophos’s olesoxime had generated.
With Repligen responsible for the phase I trial Pfizer’s outlay was limited to a $5m signing fee and a $1m milestone.
Repligen’s low-key announcement, through a brief SEC filing, of Pfizer’s pull-out befitted the unimportance of RG3039, and of drug development in general, to its business. The stock closed down just 3% on Friday.
The group had earlier tried to develop drugs for CNS disorders, taking RG2133 and RG1068 into the clinic in bipolar depression and schizophrenia respectively, as well as running a phase I multiple sclerosis study with RG2077, but these have all now been abandoned.
In the past few years Repligen has changed focus, and is now concentrating on selling consumables such as laboratory reagents for use in biological drug manufacturing; this bioprocessing division is a cash-generating business.
Apart from RG3039 its only current clinical asset is the HDAC inhibitor RG2833. Strangely enough, while other HDAC inhibitors like Merck & Co’s Zolinza and Celgene’s Istodax have been developed for cancer, RG2833 is in phase I for Friedreich’s ataxia.
Repligen’s work in HDAC inhibitors, including RG2833, was sold a year ago to BioMarin, with the originator company retaining only a milestone and royalty interest.
There might still be hope for the HDAC portfolio. For the SMA project RG3039, though, the future look bleak. Pfizer’s pull-out underlines Repligen’s lack of success in drug development, and makes the group’s change of focus look smart.