Since Regulus’ (RGLS) lead candidate drug RG101 for HCV was put on clinical hold by the FDA in 2016, the stock has enormously suffered and with it the firm’s value. Finally, we are experiencing a tremendous change as we started hearing from the firm, not about its pipeline products’ clinical updates this time, but about the firm’s made decisions about changes.
On June 12, 2017, Regulus issued a press release announcing pipeline updates and advancements. Regulus decided to focus its resources and capital on its most promising discovery and development programs. Jay Hagan, President and Chief Executive Officer of Regulus acknowledged that MicroRNA therapeutics have the potential to become an important new class of drugs with broad therapeutic application in case the firm focuses on diseases with significant unmet medical need and in organs where the delivered oligonucleotide therapeutics is effectively made, such as in the liver and kidney.
Here is how the Rugulus product pipeline will be handled from now on:
RG-012 for Alport syndrome: This program is on track as planned and the firm will continue to advance it with important changes made to its Phase 2 study. The changes are made to accelerate patient enrollment, improve statistical power, and potentially achieve proof of mechanism with data by the end of 2017.
HERA, the Phase 2 randomized (1:1), double-blinded, placebo-controlled study evaluating the safety and efficacy of RG-012 in Alport syndrome patients, has been modified as follows:
– Enrollment: increasing enrollment to 40 patients to improve the trial’s statistical power.
– Dose frequency: Has been adjusted to once every other week.
– A separate renal biopsy study to evaluate RG-012 renal tissue pharmacokinetics, target engagement and downstream effects on genomic disease biomarkers.
The Data from the renal biopsy study is anticipated by year-end and interim data from HERA is anticipated mid-2018.
RG-101 (anti-miR122) for HCV: The clinical development of this product will be discontinued upon the completion of the one remaining clinical study, which is expected to be in July 2017.
RGLS4326 (anti-miR-17): The IND for this product intended for autosomal dominant polycystic kidney disease (ADPKD) is on track for filing by year end 2017.
IND enabling toxicology, repeat pharmacology and manufacturing work have all been completed as scheduled to support the regulatory submissions. Data from the preclinical program have been recently published in Nature Communications and support the rationale for targeting miR-17 for the treatment of ADPKD, an orphan indication with no treatment options affecting approximately 600,000 people in the United States.
RGLS5040 (anti-miR-27): This drug has been discontinued.
Separately, AstraZeneca informed the Company that it intends to terminate the clinical development program for AZD4076(RG-125) for the treatment of NASH in Type 2 Diabetes/Pre-diabetes. Pursuant to the terms of the licensing agreement, AstraZeneca’s rights with respect to AZD4076(RG-125) will revert to Regulus when the termination becomes effective in twelve months. AZD4076(RG-125) was jointly identified and selected as a clinical candidate in April 2015 by AstraZeneca under the companies’ strategic alliance to discover, develop and commercialize microRNA therapeutics.
One day after these changes have been made, Regulus announced the promotion of Dr. Mark Deeg to Chief Medical Officer. Dr. Deeg joined Regulus in April 2017 as Vice President of Translational Medicine. In his new role, he will lead the Company’s clinical development teams in addition to his oversight of translational medicine.
Prior to joining Regulus, Dr. Deeg was the Chief Medical Officer of the Chorus Group, an autonomous early drug development division of Eli Lilly focused on innovative approaches to drive rapid and efficient Proof-of-Concept decisions. Before joining Eli Lilly, Dr. Deeg served on the faculty at Indiana University for 11 years conducting translational research.
A Strategic Agreement
With the Contract Research Organization Aptuit Holding
A few days before the firm announced its new decisions, Aptuit Holding LLC issued a press release stating that Dr. Jonathan Goldman, the CEO of Aptuit LLC, announced a strategic provider relationship between Aptuit, a leading Partnership Research Organization and Regulus. The agreement includes a broad range of discovery and development services to support Regulus programs targeting microRNAs.
Aptuit provides high quality standalone and integrated solutions in the target to candidate, and candidate to IND phases of development. Aptuit is expert in chemistry and biology, which contributes to improving the probability of the drug developing firms’ success. It reduces timeline and costs, and is an excellent fit with Regulus’ focus on microRNA.
Dr. Jonathan Goldman, Aptuit CEO has expressed his firm’s conviction that its support for Regulus will allow it to rapidly progress more programs.
Regulus seemed determined to succeed and patients who suffer the horror of some genetic diseases should be happy with the firm’s determination and changes. A very few people, mostly experts, know how torturing is Alport syndrome and how frightening it is for parents whose children are diagnosed with this disease, not to forget about the young patients themselves and those who know them and care about them. The successful outcome of Regulus product RG-012 will be a gift of life for these people. Of course there are no other treatments approved for this disease.
As we said in the beginning of this text, the firm’s stock has plummeted following the FDA decision to halt the Company’s HCV product. The good news is that the firm’s scientists know by now the reason of the adverse effects of the HCV product RG101 and decided to discontinue its development.
RGLS is trading now at $0.94 with a market CAP less than $50 million. The question is: Could the changes made in the firm’s pipeline and Regulus’ newly promoted Chief Medical Officer Dr. Mark Deeg and the CRO firm Aptuit save this firm and the patients inheriting the life-threatening Alport syndrome and other genetic life-threatening disease patients? Could the changes provide near-term catalysts that rejuvenate the stock?
Let’s observe this firm instead of distancing ourselves from it. Miracles always happen at the hands of highly motivated, experienced and determined scientists like those working at Regulus and at the hands of highly specialized chemists and biologists who work for the CRO firm Aptuit.
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