Regulus Therapeutics Reports First Quarter 2024 Financial Results and Recent Updates
Regulus Therapeutics Inc. reported financial results for Q1 2024, showing a cash position of $107.7 million with a cash runway into H1 2026. The company initiated the fourth cohort in the Phase 1b MAD study of RGLS8429 for ADPKD, with topline data expected in mid-2024. Positive topline data from the second cohort indicated promising results in reducing kidney volume in patients with ADPKD. The company also closed an oversubscribed $100 million private placement in March 2024.
Regulus Therapeutics reported a cash position of $107.7 million as of March 31, 2024, ensuring a cash runway into H1 2026, providing financial stability for future operations and milestones.
The initiation of the fourth cohort in the Phase 1b MAD study of RGLS8429 for ADPKD demonstrates the company's commitment to advancing its clinical pipeline and seeking innovative treatments for patients.
Positive topline data from the second cohort of patients in the Phase 1b MAD study of RGLS8429 showed promising results in reducing kidney volume in patients with ADPKD, indicating the potential efficacy of the treatment.
The oversubscribed $100 million private placement in March 2024 reflects investor confidence in Regulus Therapeutics, attracting participation from new and existing institutional investors.
Research and development expenses increased to $6.0 million for the three months ended March 31, 2024, compared to $4.9 million for the same period in 2023, potentially impacting the company's overall financial performance.
The net loss for the three months ended March 31, 2024, was $8.5 million, or $0.29 per share, showing a higher loss compared to $7.1 million, or $0.42 per share, for the same period in 2023, reflecting potential financial challenges.
Insights
Examining Regulus Therapeutics' financial health, the company's reported cash and cash equivalents position of
However, investors should note the year-on-year increase in both R&D and G&A expenses, indicative of the company's investment in advancing their clinical pipeline, which is typical for a biopharmaceutical firm in the clinical stage. The net loss widening from
Progress in Regulus Therapeutics' Phase 1b MAD study for RGLS8429 is noteworthy for stakeholders with interest in the ADPKD treatment space. Positive topline data from the second cohort is a pivotal development, as it indicates the potential efficacy of RGLS8429 for reducing htTKV and TKCV, important markers in ADPKD progression. The decision to increase the sample size for the fourth cohort suggests confidence from the company in RGLS8429's biological activity and its potential impact on disease markers, which may be a precursor to future clinical success.
However, it is critical for investors to understand that the drug is still in the early stages of clinical trials and many candidates fail to make it to market. While the preliminary results are promising, the long-term investment thesis will depend heavily on the outcomes of the pivotal trials and subsequent regulatory approvals.
Initiated the fourth cohort of patients in the Phase 1b multiple-ascending dose (MAD) clinical trial of RGLS8429 for the treatment of autosomal dominant polycystic kidney disease (ADPKD)
Topline data from the third cohort of patients in the Phase 1b MAD study of RGLS8429 expected in mid-2024
Ended first quarter 2024 with cash, cash equivalents, and investments of
"Our work in ADPKD continues to progress and most recently we announced the initiation of our fourth, and final, cohort in the Phase 1b MAD study of RGLS8429," said Jay Hagan, CEO of Regulus. "With the
Program Updates
RGLS8429 for ADPKD: In January, the Company announced completion of enrollment in the third cohort of patients in the ongoing RGLS8429 MAD study. Patients in the third cohort were dosed at 3 mg/kg of RGLS8429 or placebo every other week for three months, with topline data expected in mid-2024. On May 6, the Company announced the initiation of the fourth and final cohort in the Phase 1b MAD study. In this open label cohort, patients will receive a fixed dose of 300 mg of RGLS8429 every other week for three months.
In March 2024, the Company announced positive topline data from the second cohort of patients in the Phase 1b MAD study of RGLS8429 for the treatment of ADPKD. In the second cohort, 14 patients were randomized 3:1 to receive either 2 mg/kg of RGLS8429 or placebo every other week for three months. RGLS8429 was well tolerated with no safety findings of concern. Greater biological activity of RGLS8429 was observed at 2 mg/kg based on urinary polycystin levels compared to 1 mg/kg and placebo, which was most evident after 3 months of dosing. Exploratory results of imaging-based biomarkers were encouraging with 3 patients with the highest increases in PC1 and PC2 having the greatest reductions in height-adjusted total kidney volume (htTKV), with corresponding reductions in total kidney cyst volume (TKCV), which was observed after 3 months of treatment with RGLS8429. These data suggest that targeting miR-17 may have a potential impact on htTKV and TKCV in patients with ADPKD. Based on these topline data from cohort 2, the Company increased the sample size of the fourth cohort to up to 30 patients in order to further examine potential impact on total kidney volume in patients with APDKD.
Corporate Highlights
Closed Oversubscribed
Financial Results
Cash Position: As of March 31, 2024, Regulus had
Research and Development (R&D) Expenses: Research and development expenses were
General and Administrative (G&A) Expenses: General and administrative expenses were
Net Loss: Net loss was
About ADPKD
Autosomal dominant polycystic kidney disease (ADPKD), caused by 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
About RGLS8429
RGLS8429 is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown clear improvements in kidney function, size, and other measures of disease severity in preclinical models. Regulus announced completion of the Phase 1 SAD study in September 2022. The Phase 1 SAD study demonstrated that RGLS8429 has a favorable safety and PK profile. RGLS8429 was well-tolerated with no serious adverse events reported and plasma exposure was approximately linear across the four doses tested. In the Phase 1b MAD study Regulus announced both topline data from the first cohort of patients in September 2023 and from the second cohort of patients in March 2024. Regulus announced completion of enrollment in the third cohort in January 2024 with patients receiving 3 mg/kg of RGLS8429 or placebo every other week for three months. Topline data from the third cohort is anticipated in mid-2024. The fourth and final cohort of the MAD study was initiated in May 2024.
About Regulus
Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in
Forward-Looking Statements
Statements contained in this presentation regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the Company's RGLS8429 program, the expected timing for initiating clinical studies, potentially achieving therapeutic efficacy and the potential to address the underlying genetic causes of ADPKD, the expected timing for reporting data, the timing and future occurrence of other preclinical and clinical activities, the expected length of our cash runway and other statements relating to future events or conditions. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the approach we are taking to discover and develop drugs is novel and may never lead to marketable products, preliminary or initial results may not be indicative of future results, preclinical and clinical studies may not be successful, risks related to regulatory review and approval, risks related to our reliance on third-party collaborators and other third parties, risks related to intellectual property, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and in the endeavor of building a business around such drugs, and our need for additional capital. These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission, including under the "Risk Factors" heading of Regulus' most recently filed annual report on Form 10-K for the fourth quarter and year ending December 31, 2023. All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Regulus Therapeutics Inc. Selected Financial Information Condensed Statement of Operations (In thousands, except share and per share data)
| ||||||||||
Three months ended March 31, | ||||||||||
2024 | 2023 | |||||||||
Operating expenses: | ||||||||||
Research and development | 6,040 | 4,925 | ||||||||
General and administrative | 2,786 | 2,444 | ||||||||
Total operating expenses | 8,826 | 7,369 | ||||||||
Loss from operations | (8,826) | (7,369) | ||||||||
Other income (expense), net | 357 | 230 | ||||||||
Loss before income taxes | (8,469) | (7,139) | ||||||||
Income tax expense | - | - | ||||||||
Net loss | $ | (8,469) | $ | (7,139) | ||||||
Net loss per share, basic and diluted | $ | (0.29) | $ | (0.42) | ||||||
Weighted average shares used to compute basic and diluted net loss per share:
| 28,746,802 | 16,844,243 | ||||||||
March 31, | December 31, | |||||
Cash, cash equivalents and investments | $ | 107,725 | $ | 23,767 | ||
Total assets | 114,138 | 30,750 | ||||
Term loan, less debt issuance costs | 540 | 1,334 | ||||
Stockholders' equity | 107,588 | 21,187 | ||||
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SOURCE Regulus Therapeutics Inc.
FAQ
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