MDRNA Selects Lead Candidate in Hypercholesterolemia Program
MDRNA announced this week that it has selected a lead candidate in its hypercholesterolemia program.
The molecule, called MDR-04227, is a Dicer-substrate targeting apolipoprotein B and is formulated with MDRNA’s proprietary lipid-based delivery technology.
“In a preclinical animal model of hypercholesterolemia, MDR-04227 was shown to be extremely potent with an IC50 of less than 100 pM,” the company said. “When formulated for systemic administration … MDR-04227 demonstrated approximately 85 percent knockdown in target messenger RNA of ApoB and a similar level of reduction of serum cholesterol following a single 1 mg/kg dose of siRNA.”
MDRNA first announced its hypercholesterolemia program in August (see RNAi News, 9/7/2008).
Alnylam Receives $20M Under Takeda Alliance
Alnylam said this week that it has received a $20 million technology-transfer payment from partner Takeda Pharmaceutical as part of the companies’ strategic alliance (see RNAi News, 5/29/2008).
Under that arrangement, Takeda acquired a worldwide, non-exclusive access to Alnylam’s RNAi intellectual property and technology to help develop drugs for cancer and metabolic diseases. In exchange, Alnylam is to receive $150 million upfront, $50 million in near-term technology-transfer payments, and up to $171 million in development and commercial milestones, as well as royalties.
With the most recent payment, Alnylam has already received $120 million of its upfront payment.
Calando Issued Patent Covering Lead RNAi Drug Candidate
Calando Pharmaceuticals said this week that it has been issued a US patent covering the active ingredient of its phase I siRNA-based cancer drug candidate CALAA-01.
The patent, No. 7,427,605, is entitled, "Inhibitors of Ribonucleotide Reductase Subunit 2 and Uses Thereof." According to Calando, it contains claims directed to inhibitory nucleic acid sequences targeting the Ribonucleotide Reductase Subunit 2 gene, as well as pharmaceutical compositions and methods for inhibiting tumor growth utilizing the sequences.
This summer, Calando began dosing patients in a phase I trial of CALAA-01, making it the first RNAi drug developer to begin testing a formulated siRNA therapeutic in humans (see RNAi News, 6/5/2008).