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Thoughts from Panno

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    Author’s reply »Regulus was expecting that RG-101 would be a single shot treatment for HCV, much the same as Benitec’s TT-034. The cure rate for the patients in the current RG-101 trial don’t match up to those for drugs from Gilead or AbbVie. In fact, the rate falls a long way short. This brings into question the commercial viability of RG-101.

    There may be ways that Regulus can improve the efficacy, such as increasing the dosage, or using the drug in combination with oral treatments. I think the talk of using RG-101 in conjunction with oral drugs is speculation on the Internet and not statement of direction from the company.

    Peter French at Benitec has also indicated that a combination of TT-034 with oral drugs could be on the cards if TT-034 does not match the cure rate of the market leaders. However, my personal view is that if either TT-034 or RG-101 require other drugs to make them effective, then they will not be commercially viable. All the advantages of a single shot would be gone and so what would be a reason for clinicians to prescribe them?

    I think Regulus will take a long hard look at the current results before determining which direction they go in from here.

    The mode of action of RG-101 is completely different to TT-034. RG-101 is an anti-mir (similar to antisense) targeting microRNA 122 (miR-122), which is a “host” miRNA and not a viral miRNA. miR-122 is reported to account for about 70% of all hepatic miRNA. TT-034 uses three shRNA’s to target “viral” RNA in order to prevent viral replication. TT-034 integrates with the host’s genome and constantly produces the therapeutic shRNAs but RG-101 does not integrate. TT-034 is delivered by a viral vector. RG-101 is delivered using Regulus’s GalNAc-conjugated (see Alnylam).

    As TT-034 targets viral RNA and uses a tried and tested viral vector, I would be surprised if any unexpected side-effects are reported in the current trial, but one can never be 100% certain. Regulus as reported limited side-effects of RG-101 but, as miR-122 represents 70% of all liver miRNA and as its effect on the liver is still being discovered, I would like to see a longer term analysis of side-effects before ticking that box.
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