Dicerna Pharmaceuticals, Inc. has announced that the company will focus its resources on its proprietary GalXCTM technology platform to advance development of product candidates in its core therapeutic areas of rare diseases, chronic liver diseases, cardiovascular disease and viral infectious diseases. Under this plan, Dicerna will transition its primary hyperoxaluria (PH) development program to focus on DCR-PHXC, a subcutaneously delivered GalXC clinical candidate, which was announced earlier this year. The Company also announced that it will discontinue clinical development of DCR-MYC, a DsiRNA-based therapeutic formulated as an EnCoreTM lipid nanoparticle (LNP) for delivery to solid tumors, because preliminary results do not meet the company’s expectations for further development.
“Based on the performance of the GalXC platform, the strength of the preclinical data and the broad therapeutic opportunities for RNAi in liver-targeted diseases, we are prioritizing resources to advance the product candidates emerging from this platform,” said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. “Discontinuing our DCR-PH1 and DCR-MYC lipid nanoparticle programs allows us to focus our resources on efficiently developing our GalXC product candidates and building on the solid scientific foundation in RNAi that Dicerna has developed over the past decade. We greatly appreciate the participation of all of the patients, families and clinical investigators in the DCR-PH1 development program, as their contributions provided important insights into the primary hyperoxaluria disease state, which will guide the development of DCR-PHXC.”
The GalXC platform is a fully enabled RNAi drug discovery engine with potentially powerful capabilities that the Company believes could result in potency that is on par with or better than comparable platforms. As Dicerna reported during its recent Investor Day, subcutaneously delivered GalXC compounds silenced 12 different disease targets in animal models, highlighting the long duration of action, infrequent dosing and tolerability of GalXC-based compounds. Use of the GalXC platform yielded gene silencing of greater than 90% for multiple genes in non-human primates (NHPs) after a single dose. In an NHP model of an undisclosed rare disease gene target, a single 3 mg/kg dose achieved a maximum gene silencing of 94%, with an average gene silencing of approximately 88%. Another single 3 mg/kg dose NHP study resulted in an average of 97% silencing of an undisclosed rare disease gene target. Based on this evidence, Dicerna believes that DCR-PHXC has the potential to be a new treatment option for patients with PH.
In addition to DCR-PHXC, which is in preclinical development, Dicerna expects to launch two more GalXC programs in 2016: one will focus on cardiovascular disease targeting PCSK9; the other is an undisclosed rare disease program. Dicerna expects to launch three additional programs annually, with the intent to advance five programs into the clinic by 2019.