Regulus plans to discontinue micro-RNA candidate for HCV

Regulus Therapeutics announced its plans to discontinue development of RG-101 for hepatitis C, according to a press release. Additionally, the company is initiating a phase 2 study for an Alport syndrome treatment, an investigational new drug for autosomal dominant polycystic kidney disease and discontinuing two other drug candidates.

“We are squarely focused on taking the steps necessary to advance our pipeline and continue building shareholder value. To that end, we recognize that we must be disciplined in our investment choices and focus our resources and capital on our most promising discovery and development programs, including the application of important development, regulatory and commercial consideration,” Jay Hagan, president and CEO of Regulus, said in the release.

Regulus plans to discontinue the development of RG-101 for HCV, expected to occur in July 2017 following one remaining clinical trial. The researchers suspected that a bilirubin transport mechanism is responsible for cases of hyperbilirubinemia in participants of the trial.

“We believe that a combination of factors including inhibition of conjugated bilirubin transport by RG-101, impaired baseline bilirubin transport in HCV patients and the preferential uptake of RG-101 by hepatocytes contributed to this mechanism,” Regulus reported. “Additional patient-specific contributing factors cannot be excluded.”

However, the researchers were optimistic about alternative compounds targeting miR-122 discovered during the trial and plan to consider these for further development pending an updated global commercial market assessment for HCV.

Additional announcements included the modified design to accelerate patient enrollment of a phase 2 safety and efficacy trial for RG-012, the candidate to treat Alport syndrome. The researchers increased enrollment to 40 patients for improved statistical power. Additionally, there will be a separate renal biopsy study to evaluate RG-012 renal tissue pharmacokinetics, target engagement and downstream effects on genomic disease.

The investigational new drug RGLS4326 for dominant polycystic kidney disease is on track for filing by the end of 2017. Pre-clinical program data were recently published and provide support for targeting miR-17 for the treatment of autosomal dominant polycystic kidney disease.

AstraZeneca plans to terminate the license agreement with Regulus for the development of AZD4076, a candidate for the treatment of nonalcoholic steatohepatitis in patients with type 2 diabetes or conditions of pre-diabetes. AZD4076 will revert to Regulus once the termination becomes effective in 12 months.

Regulus has also discontinued RGLS5040 for cholestatic disease based on a review of its standing compared with the competitive landscape and results of repeat studies.

“MicroRNA therapeutics have the potential to become an important new class of drugs with broad therapeutic application,” Hagan continued in the release. “Regulus’ focus will be in diseases with significant unmet medical need in organs to which we have been able to preferentially deliver oligonucleotide therapeutics effectively, such as liver and kidney.”

Reference: www.regulusrx.com