Dicerna is no longer looking to move from being a clinical- to a commercial-stage biotech as it seeks out a new partner to do the heavy lifting for the sales work for its midstage RNAi asset nedosiran.
This drug, its second RNAi candidate for the rare metabolic disorder that impacts the kidneys called primary hyperoxaluria (PH) 1, saw mixed results from a pivotal study last week.
The test showed efficacy in one of PH type but not another, meaning Dicerna will not be able to seek approval in PH2, at least for now.
There are three forms of the disease, all of which are closely related and characterized by high urinary oxalate levels that cause frequent kidney stones and progression to end-stage renal disease. Dicerna designed nedosiran to silence a gene involved in what it believes to be the ultimate step in the oxalate production pathway, thereby enabling it to treat PH1, PH2 and PH3 with the drug.
The pivotal data raise significant doubts about the use of nedosiran in PH2. Across the mix of 34 PH1 and PH2 patients with at least one efficacy assessment, nedosiran drove a statistically significant drop in urinary oxalate. However, the overall result masks variation between PH1 and PH2 patients.
Given these data, and the sheer cost and energy needed to get over the finishing line and sell to the market, Dicerna will no longer follow the playbook of its rival Alnylam and move into being a commercial biotech for RNAi meds, but instead wants a new partner to help.
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“As positive as these results are for PH1, the inconsistent data seen specifically in participants with PH2 have led us to make the strategic decision not to move forward with our plan to build Dicerna into a fully integrated commercial enterprise to support nedosiran,” said Douglas Fambrough, Ph.D., president and CEO at Dicerna, in its second-quarter results Monday.
“Instead, we intend to pursue commercial out-licensing opportunities to help ensure global access to nedosiran, subject to necessary approvals. This approach will allow us to deploy our capital and talent on our discovery and development pipeline efforts with our GalXC and GalXC-Plus RNAi investigational therapeutics for ourselves and our partners. With these strategic adjustments focused on our core strengths, we can extend our cash runway into 2025.”
The biotech ended the day down more than 12% yesterday.
But analysts at Jefferies believe it was the right strategic decision, saying in a note to clients that “pragmatism has clearly prevailed,” giving it time to get more earlier shots on goal from its “rich pipeline.” On potential partners, “conversations are ongoing with both global pharma and regional players with rare disease expertise,” the firm’s analysts added.
“On timing, we do not think a deal is imminent as [Dicerna] plans to circle back in the “next couple of quarters.”