Heading into the new year, Alnylam Pharmaceuticals is bullish that its RNA interference platform will continue to churn out effective and commercially viable medicines.
But the company is also bluntly assessing where it feels like it needs to pull back, disclosing three program culls in its R&D update Thursday. The company said the culls were made to maintain its high-yield rate across its pipeline, funneling resources only to those medicines with a higher probability of success.
The most notable of the company’s reprioritization is its once-Regeneron-partnered IgA nephropathy med cemdisiran. Alnylam said despite encouraging phase 2 data, Regeneron elected not to participate in further development.
“As a result, we will now take some time to consider options for the best path forward for cemdisiran in this indication,” said Alnylam President Akshay Vaishnaw, M.D., Ph.D.
The company also announced that it was terminating development of ALN-XDH due to lukewarm preliminary data in addition to its recurrent renal stones treatment, lumasiran. A prescreening of patients enrolled in phase 2 trial for lumasiran found that urinary oxalate levels were not as high as expected, forcing the company to return to the drawing board.
The company acknowledged the clinical setbacks within the context of its higher-than-average probability of success rate in the clinic. Whereas the industrywide cumulative probability of success in clinical development is 10.3% (when including biomarker-driven programs), the success rate for Alnylam is 62%. The company noted that its success rate data include molecules that are being developed by other partners.
“The pipeline prioritization decisions I just shared with you illustrate the importance of exercising discipline when it comes to building a robust and high yielding pipeline,” said Vaishnaw.
No matter the spin, the company’s decision to part ways with three clinical-stage programs fits into a yearlong trend of pipeline reductions for pharmas large and small. In August, Alnylam said it was working expeditiously to advance cemdisiran into a phase 3 trial following positive secondary endpoint results. Now, it’s shelved. Regeneron is not disassociating from Alnylam altogether, however, with the two advancing an early stage nonalcoholic steatohepatitis product.