Regulus Therapeutics has reported positive clinical results and a regulatory update for its antisense therapy, farabursen, in ADPKD.
The positive results came from an interim analysis of data from 14 patients in a fourth and final cohort in the company’s Phase 1 b MAD trial. These patients received a fixed 300 mg dose every 2 weeks for 3 months.
Continuing excellent safety and tolerability of farabursen was seen as well as a statistically significant increase in PC1 and PC2 protein levels and a suggested reduction in TKV. Results seen were similar to those seen in the previous cohort which received a 3mg/kg dose.
Regulus has already reached alignment with the FDA for a pivotal 2 year global trial in 336 patients, aged 18-70, with moderate to severe ADPKD. Patients will be randomized 2:1 and receive a subcutaneous 300mg dose bi-weekly.
The FDA has agreed that a 12-month htTKV endpoint is acceptable for accelerated approval, while a 24-month eGFR endpoint is required for full approval.
Regulus is aiming to at least match, but hopefully improve on the efficacy of the currently approved therapy, tolvaptan, but without the poor safety and tolerability profile which rules out the drug for most ADPKD patients.
Regulus has indicated that there are 160,000 ADPKD diagnosed patients in the United States, with two thirds considered to be at high risk to ESRD. While only 7% of patients are currently prescribed tolvaptan, Regulus’ surveying of physicians has shown that 82% would be willing to prescribe farabursen if it could match the efficacy of tolvaptan without the side effects.
In response to a Leerink analyst question about partnering intentions, the CEO of Regulus said that there had been growing interest with the company receiving many inbound calls. The company does not intend to outlicense in the United States but will consider licensing ex-US rights.
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