Regulus Drug Candidate RG-012 to Treat Kidney Damage in Alport Patients Begins Phase 1 Clinical Trials

Alice Melão avatar

by Alice Melão |

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RG-012 therapy

RG-012, a drug to treat renal function decline in patients with Alport syndrome, is moving into Phase 1 clinical trial, says its developer, Regulus Therapeutics. The company, based in La Jolla, Calif., expects this new trial to overcome the setback announced last November on its RG-012 Phase 2 study, and give additional information supporting the drug’s clinical application.

Regulus focuses on drugs for renal, hepatic and central nervous system diseases. Targeting small non-coding RNA molecules,or microRNAs involved in disease development and progression, the company expects to supply new effective treatment possibilities for conditions with unmet medical needs.

A previous study showed that a microRNA called mir-21 was involved in kidney fibrosis and tissue damage — conditions also common in Alport patients.

Based on this, Regulus has developed RG-012 to inhibit mir-21 and its actions. In preclinical studies, RG-012 showed it was a potent mir-21 inhibitor, with promising results in Alport animal models. In combination with standard care therapies, RG-012 was improved the animals’ lifespan and kidney condition.

To overcome concerns over RG-012 Phase 2 study design, the company has initiated a Phase 1 multiple ascending dose (MAD) trial in healthy volunteers. This study will provide additional information on RG-012 efficacy and safety, and support the resume of the placebo-controlled Phase 2 HERA study (NCT02855268).

For now, Regulus has altered the HERA study and expects to accelerate patient enrollment in order to have initial results by mid-2018. Additionally, by late 2017 it expects to launch a Phase 1/2 renal biopsy study in Alport patients to address drug tissue specificity, and effects in other markers of the disease.

Both these clinical trials will begin enrollment protocols following completion of the MAD study.

Calling 2016 a “year of accomplishments and important key learnings,” Regulus President and CEO Paul Grint said in a press release he is “even more convinced that targeting microRNAs offer significant advancements in therapeutic potential for many diseases for which there are currently no or limited treatment options.”


Zvonimir Kodba avatar

Zvonimir Kodba

Well,I have not th comment but question. In articele is mention:''
In combination with standard care therapies, RG-012 was improved the animals’ lifespan and kidney condition.''
Coukd someon tell me more about 'standard care terapy for Alport syndrome? I have kids,heterozigotes twins, with diagnosys 'Alport syndrome or thin membrane desies'. The diagnosis is based on microhaematuria, and kidney biopsy (for one brother), for other is just suspected up to now but we are waiting for genetical conformation. Up to now (they are 16 y.o.), no other sympthoms occure. (Like hearing loss or decrease in kidney function). But I did not get any therapy recomanded or advice from nephrologist except '' we have to monitor the condittion''. Is there any recomanded standard therapy as it was mention in that sentence ?

Alice Melão avatar

Alice Melão

Although I am not a clinical doctor and so my knowledge on this matter is somewhat limited, from what I know there are no specific therapies for Alport patient's kidney symptoms. Patients are treated in agreement with the guidelines used to treat kidney diseases in general. So, when the researchers say "standard therapies" they are referring to those guidelines and potential therapies.

Zvonimir Kodba avatar

Zvonimir Kodba

Thanks a lot Alice.


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