On August 13, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the second quarter ended June 30, 2020 and provided a corporate update (Press release, Regulus, AUG 13, 2020, View Source [SID1234563591]).
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"We are pleased with the progress of our ADPKD program and the completion of dosing of the MAD clinical study for RGLS4326 in healthy volunteers," said Jay Hagan, CEO of Regulus. "We plan to initiate a Phase 1b study in patients with ADPKD to evaluate short-term treatment of RGLS4326 for safety, tolerability, pharmacokinetics, and changes in biomarkers of the disease. We have also recently completed additional non-clinical studies needed to address some of the FDA requirements to support studies of extended duration."
Second Quarter 2020 Corporate Highlights and Recent Updates
Appointment of Chief Scientific Officer: In August, Dr. Denis Drygin joined Regulus as its Chief Scientific Officer.
Program Updates
RGLS4326 for Autosomal Dominant Polycystic Kidney Disease: In July 2020, the Company completed dosing in the MAD clinical study for RGLS4326 in healthy volunteers. The Phase 1 MAD study is a randomized, double blind, placebo-controlled clinical trial evaluating three different dose levels of RGLS4326 for safety, tolerability, and pharmacokinetic properties. Top-line results showed that RGLS4326 is well-tolerated with no serious adverse events reported. Preliminary results suggest plasma exposure is dose proportional. In July 2020, the FDA granted Orphan Drug Designation to RGLS4326 for ADPKD.
The Company plans to initiate a Phase 1b open-label, short-term multiple dose study in patients with ADPKD in the second half of 2020. The study will evaluate RGLS4326 for safety, pharmacokinetics, and changes in levels of polycystin 1 (PC1) and polycystin 2 (PC2). Patients with ADPKD, due to the mutation in the PKD gene, have been reported to have low levels of PC1 and PC2. This study is designed to evaluate whether different dose levels of RGLS4326 can increase levels of PC1 and PC2 in ADPKD patients.
Advancement of Hepatitis B virus (HBV) Program: The Company has identified several microRNA targets that serve as host factors for the hepatitis B virus (HBV). Our lead compound directed to one of the host microRNAs has demonstrated nanomolar potency against HBV DNA replication and more than 95% reduction in Hepatitis B surface antigen in in vitro studies. Additionally, we have demonstrated reduction of HBV DNA, surface antigen and pgRNA in an in vivo efficacy model. We believe that targeting a host factor in the liver represents a unique mechanism of action for treatment of the virus compared to other programs in development and holds the potential for achieving a functional cure. We have nominated a development candidate and plan to commence IND-enabling activities.
Additional Research Updates: Cell therapies have been approved to treat a variety of hematological malignancies. Targeting solid tumors, however, has proven challenging for cell therapies due to the immune-suppressive effect of the tumor microenvironment (TME). The Company believes that ex vivo modulation of microRNA may enable cell therapy approaches to overcome the effects of the TME and address other challenges faced by cell therapies. The Company has demonstrated that targeting microRNA ex vivo can improve certain characteristics of engineered cells including an improved in vitro expansion, effector function, cytokine production, as well as resistance to exhaustion induced by tonic signaling. The Company is pursuing multiple applications of microRNA technology in a variety of cell therapies.
Second Quarter 2020 Financial Results
Cash Position: As of June 30, 2020, Regulus had $23.4 million in cash and cash equivalents.
Research and Development (R&D) Expenses: Research and development expenses were $4.2 million and $7.4 million for the three and six months ended June 30, 2020, respectively, compared to $1.8 million and $7.8 million for the three and six months ended June 30, 2019, respectively. The aggregate increase for the three months ended June 30, 2020, as compared to the three months ended June 30, 2019, was driven by an increase in external development expenses, primarily attributable to the FDA lifting the partial clinical hold on the MAD study in December 2019 and completion of the dosing for the Phase 1 of the MAD study in July 2020. The aggregate decrease for the six months ended June 30, 2020, as compared to the six months ended June 30, 2019, was driven by a reduction in personnel and internal expenses, partially offset by an increase in external development expenses attributable to recommencement and completion of the dosing of the MAD study in July 2020.
General and Administrative (G&A) Expenses: General and administrative expenses were $2.3 million and $4.7 million for the three and six months ended June 30, 2020, respectively, compared to $2.9 million and $6.4 million for the three and six months ended June 30, 2019, respectively. These amounts reflect personnel-related and ongoing general business operating costs. The decreases for the three and six months ended June 30, 2020, as compared to the three and six months ended June 30, 2019, are primarily attributable to continued cost reduction efforts subsequent to our corporate restructurings.
Revenue: Revenue was zero and less than $0.1 million for the three and six months ended June 30, 2020, respectively, compared to less than $0.1 million and $6.8 million for the three and six months ended June 30, 2019, respectively. Revenue recognized for the six months ended June 30, 2019 was attributable to the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.
Net Loss: Net loss was $6.9 million, or $0.23 per share (basic and diluted), and $12.9 million, or $0.48 per share (basic and diluted), for the three and six months ended June 30, 2020, respectively, compared to $5.0 million, or $0.30 per share (basic and diluted), and $8.3 million, or $0.61 per share (basic and diluted), for the same periods in 2019.
About ADPKD
ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.
About RGLS4326
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD. The Company recently completed the multiple ascending dose study of RGLS4326 in healthy volunteers. Administration of RGLS4326 was well-tolerated with no serious adverse events reported. The Company is initiating a Phase 1b study in patients with ADPKD to evaluate short-term treatment of RGLS4326 for safety, tolerability, pharmacokinetics, and changes in biomarkers of the disease. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration beyond the current Phase 1b study until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from the recently completed additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.