In 2010 Alnylam said it made “astounding” progress in developing RNAi delivery platforms and has now presented Phase I data to support its claim. The lipid nanoparticle (LNP) platform underpinned “major improvements in potency”, Alnylam said, and will be central to the company’s growth plans.
“We have what we believe is a very potent LNP platform. It really becomes the foundation for…the future of the company [and] speaks volumes to the investments we have made in delivery”, John Maraganore, CEO of Alnylam, told investors in a conference call.
Alnylam made investments to support its entire RNAi pipeline and views the positive ALN-PCS results as a boost for other products that use the platform, ALN-TTR02 in particular. ALN-TTR02 is due to enter Phase I soon and Maraganore has “great confidence” having seen the positive ALN-PCS data.
The two compounds are at the forefront of efforts at Alnylam to advance five RNAi programmes into late-stage development by the end of 2015. Achieving the goal would represent a resurgence of the RNAi sector after cutbacks by Roche, Abbott and Pfizer raised questions about its viability.
Alnylam data showing the potential of RNAi to cut low-density lipoprotein (LDL) cholesterol comes months after Amgen presented results for its LDL-targeting monoclonal antibody (mAb). Although both drugs aim to cut LDL their mechanisms are different and could be used in combination.
“We are turning off the faucet rather than mopping up the floor”, Akshay Vaishnaw, chief medical officer at Alnylam, said. “It’s certainly feasible [to combine the treatments]”, Vaishnaw said, but for now Alnylam is focused on increasing the dose of its own treatment to show how effective it can be.
By increasing the dose Alnylam believes once monthly dosing with ALN-PCS will be sufficient. Dosing every other month may also be possible, Vaishnaw said, and this would make the treatment more convenient for patients than statins that must be taken daily.
Alnylam is also considering use of ALN-PCS in conjunction with statins. Potential partners have shown “quite a bit of interest” in ALN-PCS, Maraganore said, and the potential of a statin combination could help Alnylam as discussions continue throughout the year.