Regulus Therapeutics Reports Third Quarter 2024 Financial Results and Recent Updates
Regulus Therapeutics has completed enrollment in the fourth cohort of its Phase 1b MAD clinical trial of RGLS8429 for treating ADPKD. The company presented promising data at ASN Kidney Week and is on track for an End-of-Phase 1 meeting with the FDA by year-end. Financially, Regulus ended Q3 2024 with $87.3 million in cash, ensuring a runway into H1 2026. R&D expenses increased to $11.3 million for Q3 2024, up from $5.5 million in Q3 2023. G&A expenses rose to $3.9 million, and the net loss widened to $14.1 million, or $0.21 per share, compared to $7.8 million, or $0.40 per share, in Q3 2023. Additionally, Regulus entered a licensing agreement with The University of Texas System, acquiring exclusive rights to patents targeting miR-17 for ADPKD treatment.
Regulus Therapeutics ha completato l'arruolamento nella quarta coorte del suo studio clinico di Fase 1b MAD per RGLS8429 nel trattamento dell'ADPKD. L'azienda ha presentato dati promettenti durante l'ASN Kidney Week ed è in linea per un incontro End-of-Phase 1 con la FDA entro la fine dell'anno. Dal punto di vista finanziario, Regulus ha chiuso il Q3 2024 con 87,3 milioni di dollari in cassa, garantendo una copertura fino alla prima metà del 2026. Le spese per R&S sono aumentate a 11,3 milioni di dollari per il Q3 2024, rispetto ai 5,5 milioni di dollari del Q3 2023. Le spese generali e amministrative sono aumentate a 3,9 milioni di dollari, e la perdita netta è aumentata a 14,1 milioni di dollari, equivalenti a 0,21 dollari per azione, rispetto ai 7,8 milioni di dollari, o 0,40 dollari per azione, del Q3 2023. Inoltre, Regulus ha stipulato un accordo di licenza con il Sistema Universitario del Texas, acquisendo diritti esclusivi su brevetti mirati al miR-17 per il trattamento dell'ADPKD.
Regulus Therapeutics ha completado el reclutamiento en la cuarta cohorte de su ensayo clínico de fase 1b MAD para RGLS8429 en el tratamiento de la ADPKD. La empresa presentó datos prometedores en la ASN Kidney Week y está en camino de tener una reunión de fin de fase 1 con la FDA a finales de año. Desde el punto de vista financiero, Regulus cerró el Q3 2024 con 87,3 millones de dólares en efectivo, asegurando recursos hasta la primera mitad de 2026. Los gastos de I+D aumentaron a 11,3 millones de dólares en el Q3 2024, en comparación con 5,5 millones de dólares en el Q3 2023. Los gastos generales y administrativos subieron a 3,9 millones de dólares, y la pérdida neta se amplió a 14,1 millones de dólares, o 0,21 dólares por acción, en comparación con 7,8 millones de dólares, o 0,40 dólares por acción, en el Q3 2023. Además, Regulus firmó un acuerdo de licencia con el Sistema Universitario de Texas, adquiriendo derechos exclusivos sobre patentes que apuntan al miR-17 para el tratamiento de la ADPKD.
레귤러스 치료제(Regulus Therapeutics)는 ADPKD 치료를 위한 RGLS8429의 1상 b MAD 임상 시험의 네 번째 코호트 등록을 완료했습니다. 이 회사는 ASN 신장 주간에서 유망한 데이터를 발표했으며, 연말까지 FDA와의 1상 종료 회의 일정을 맞추고 있습니다. 재무적으로, 레귤러스는 2024년 3분기에 8730만 달러의 현금을 보유하며, 2026년 상반기까지 자금을 확보했습니다. 연구 개발(R&D) 비용은 2024년 3분기 1,130만 달러로 증가했으며, 2023년 3분기에는 550만 달러였습니다. 일반 관리(G&A) 비용은 390만 달러로 증가하였고, 순손실은 1,410만 달러, 즉 주당 0.21달러로 확대되었으며, 이는 2023년 3분기 780만 달러, 즉 주당 0.40달러와 비교됩니다. 또한, 레귤러스는 텍사스 대학교 시스템과 라이선스 계약을 체결하여 ADPKD 치료를 위한 miR-17을 타겟으로 한 특허에 대한 독점 권리를 획득했습니다.
Regulus Therapeutics a terminé le recrutement de la quatrième cohorte de son essai clinique de phase 1b MAD sur le RGLS8429 pour le traitement de l'ADPKD. L'entreprise a présenté des données prometteuses lors de l'ASN Kidney Week et est en voie de planifier une réunion de fin de phase 1 avec la FDA d'ici la fin de l'année. D'un point de vue financier, Regulus a clôturé le T3 2024 avec 87,3 millions de dollars de liquidités, garantissant ainsi un financement jusqu'au premier semestre 2026. Les dépenses de R&D ont augmenté à 11,3 millions de dollars pour le T3 2024, par rapport à 5,5 millions de dollars au T3 2023. Les frais généraux et administratifs ont augmenté à 3,9 millions de dollars, et la perte nette s'est élargie à 14,1 millions de dollars, soit 0,21 dollar par action, contre 7,8 millions de dollars, soit 0,40 dollar par action, au T3 2023. De plus, Regulus a signé un accord de licence avec le système universitaire du Texas, acquérant des droits exclusifs sur des brevets ciblant le miR-17 pour le traitement de l'ADPKD.
Regulus Therapeutics hat die Rekrutierung in der vierten Kohorte seiner Phase 1b MAD-Studie für RGLS8429 zur Behandlung von ADPKD abgeschlossen. Das Unternehmen präsentierte vielversprechende Daten während der ASN Kidney Week und ist auf Kurs für ein End-of-Phase-1-Treffen mit der FDA bis zum Jahresende. Finanztechnisch hat Regulus das 3. Quartal 2024 mit 87,3 Millionen Dollar an Barmitteln abgeschlossen, wodurch die Finanzierung bis zur ersten Hälfte 2026 gesichert ist. Die F&E-Ausgaben stiegen im 3. Quartal 2024 auf 11,3 Millionen Dollar, gegenüber 5,5 Millionen Dollar im 3. Quartal 2023. Die allgemeinen und administrativen Ausgaben erhöhten sich auf 3,9 Millionen Dollar, während der Nettoverlust auf 14,1 Millionen Dollar (0,21 Dollar pro Aktie) ausgeweitet wurde, im Vergleich zu 7,8 Millionen Dollar (0,40 Dollar pro Aktie) im 3. Quartal 2023. Darüber hinaus hat Regulus eine Lizenzvereinbarung mit dem Universitätssystem von Texas unterzeichnet und exklusive Rechte an Patenten erworben, die auf miR-17 zur Behandlung von ADPKD abzielen.
- Completed enrollment in the fourth cohort of Phase 1b MAD trial of RGLS8429.
- Presented promising clinical data at ASN Kidney Week.
- On track for an End-of-Phase 1 meeting with the FDA by year-end.
- Ended Q3 2024 with $87.3 million in cash, ensuring a runway into H1 2026.
- Increased R&D expenses to $11.3 million in Q3 2024, up from $5.5 million in Q3 2023.
- G&A expenses rose to $3.9 million in Q3 2024, compared to $2.6 million in Q3 2023.
- Net loss widened to $14.1 million, or $0.21 per share, in Q3 2024, compared to $7.8 million, or $0.40 per share, in Q3 2023.
Insights
The Q3 results reveal significant financial dynamics for RGLS. With
The UTSW licensing agreement represents strategic IP expansion with manageable upfront costs of
The completion of enrollment in the fourth cohort of RGLS8429's Phase 1b MAD trial marks a important milestone. Previous cohorts demonstrated promising mechanistic dose response through increased urinary polycystins and potential reductions in htTKV, a critical endpoint for ADPKD treatment evaluation. The transition from weight-based dosing to a fixed 300mg bi-weekly regimen in the fourth cohort could optimize the therapeutic approach.
The successful engineering of RGLS8429 to eliminate CNS toxicity while maintaining miR-17 targeting efficacy demonstrates sophisticated drug development. With an End-of-Phase 1 FDA meeting scheduled and preliminary topline data expected in early 2025, the program is advancing systematically toward potential Phase 3 trials.
Completed enrollment in the fourth cohort of the Phase 1b multiple-ascending dose (MAD) clinical trial of RGLS8429 for the treatment of autosomal dominant polycystic kidney disease (ADPKD)
Presented a poster highlighting data from the ongoing MAD study of RGLS8429 in ADPKD at American Society of Nephrology (ASN) Kidney Week
On track for an End-of-Phase 1 meeting by year-end
Ended third quarter 2024 with cash, cash equivalents, and investments of
"We have remained focused on rapidly progressing RGLS8429 for the potential treatment of ADPKD and were pleased to announce the completion of enrollment in our fourth and final cohort of the Phase 1b MAD study last month. Our team continues to be energized not only by the positive results we've seen so far in the clinic but also by the recent opportunities we've had to interact with the broader nephrology community and present on RGLS8429 at recent meetings," said Jay Hagan, CEO of Regulus. "We are in a strong position as we move towards the end of the year and into 2025. We expect to share preliminary topline data from a substantial number of patients from the fourth cohort in the Phase 1b MAD study of RGLS8429 in ADPKD in early 2025."
Program Updates
RGLS8429 for ADPKD: In October 2024, the Company announced completion of enrollment in the fourth cohort of patients in the Phase 1b MAD study of RGLS8429 in ADPKD. Patients in the fourth cohort are receiving an open-label 300 mg fixed dose of RGLS8429 administered every other week for three months, and the Company plans to share topline data from a substantial number of these patients in early 2025.
Regulus has previously shared positive topline data from the first three weight-based cohorts in the Phase 1b MAD study showing evidence of a mechanistic dose response based on increases in urinary polycystins (PC1 and PC2) and exploratory imaging analysis showing mean reductions in height-adjusted total kidney volume (htTKV), a key measurement that will be evaluated in the fourth cohort and in the potentially pivotal Phase 3 trial. The Company remains on track for an End-of-Phase 1 meeting with the
Corporate Highlights
Presented a late-breaking poster on the Company's ongoing study of RGLS8429 in ADPKD at ASN Kidney Week: The poster, "RGLS8429 increases urinary PC1 and PC2 and may reduce height-adjusted total kidney volume (htTKV) in patients with ADPKD," featured data from the first three weight-based cohorts in the Company's ongoing clinical study of RGLS8429 in ADPKD. Data shared in the poster provide clinical proof of dose-responsive RGLS8429 mechanistic activity on PC1 and PC2 and highlights the exploratory analysis which suggests a reduction of htTKV at 2 and 3 mg/kg doses over a relatively short treatment period. These findings validate miR-17, which RGLS8429 has been designed to inhibit, as a potential therapeutic target for ADPKD.
Presented a poster on the path to creating RGLS8429 at the Oligonucleotide Therapeutics Society Annual Meeting: The poster, "Deciphering and overcoming off-target AMPAR inhibition of anti-miR oligonucleotide RGLS4326," outlined the Company's transition from the previous generation molecule RGLS4326 to current compound RGLS8429 after the nonclinical observation of dose-limiting central nervous system (CNS)-related toxicity during chronic toxicity studies. By switching the 3'terminus guanine with adenine, Regulus' next-generation anti-miR-17, RGLS8429, was devoid of off-target AMPA-receptor interaction and CNS toxicity while preserving the potency against the on-target miR-17. These findings show the effect certain nucleobase changes can have on the overall profile of an oligonucleotide and support the clinical development of RGLS8429.
Entered into a Patent & Technology License Agreement (the "UTSW Agreement") with The Board of Regents of The University of Texas System on behalf of The University of
Financial Results
Cash, Cash Equivalents and Marketable Securities: As of September 30, 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 topline data from the first cohort of patients in September 2023, from the second cohort of patients in March 2024 and from the third cohort of patients in June 2024. Regulus completed enrollment in the fourth cohort of patients in October 2024. Patients in the fourth cohort are receiving an open-label 300 mg fixed dose of RGLS8429 administered every other week for three months.
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 San Diego, CA.
Forward-Looking Statements
Statements contained in this press release 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 and preclinical pipeline, the potential that RGLS8429 may be eligible for an Accelerated Approval pathway, potentially achieving therapeutic efficacy and clinical translation for patients, the expected timing for reporting interim or topline data, and the timing and future occurrence of other preclinical and clinical activities. 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 risk that the approach we are taking to discover and develop drugs is novel and may never lead to marketable products, that preliminary or topline results are based on a preliminary analysis of key efficacy and safety data, and such data may change following a more comprehensive review of the data related to the clinical trial and may not be indicative of future results, the FDA has not designated RGLS8429 for an Accelerated Approval pathway and such designation may not lead to a faster development, regulatory review or approval process and does not increase the likelihood that RGLS8429 will receive marketing approval, the risk that 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, the risk that additional toxicology data may be negative, and risks related to our ability to successfully secure and deploy 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' quarterly report on Form 10-Q available on the Company's website or at www.sec.gov. 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)
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Three months ended September 30, | Nine months ended September 30, | ||||||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||||||
Operating expenses: | |||||||||||||||
Research and development | 11,347 | 5,488 | 25,625 | 15,389 | |||||||||||
General and administrative | 3,863 | 2,637 | 10,599 | 7,420 | |||||||||||
Total operating expenses | 15,210 | 8,125 | 36,294 | 22,809 | |||||||||||
Loss from operations | (15,210) | (8,125) | (36,294) | (22,809) | |||||||||||
Other income, net | 1,148 | 301 | 2,728 | 834 | |||||||||||
Loss before income taxes | (14,062) | (7,824) | (33,566) | (21,975) | |||||||||||
Income tax expense | - | - | (1) | (1) | |||||||||||
Net loss | $ | (14,062) | $ | (7,824) | $ | (33,567) | $ | (21,976) | |||||||
Other comprehensive loss: | |||||||||||||||
Unrealized gain on short-term investments, net | 207 | - | 120 | - | |||||||||||
Comprehensive loss | (13,855) | (7,824) | (33,447) | (21,976) | |||||||||||
Net loss per share, basic and diluted | $ | (0.21) | $ | (0.40) | $ | (0.63) | $ | (1.19) | |||||||
Weighted average shares used to compute basic and diluted net loss per share: | 65,471,132 | 19,628,815 | 53,272,582 | 18,535,209 | |||||||||||
September 30, | December 31, | |||||||
Cash, cash equivalents and short-term investments | $ | 87,309 | $ | 23,767 | ||||
Total assets | 93,763 | 30,750 | ||||||
Term loan, less debt issuance costs | - | 1,334 | ||||||
Stockholders' equity | 86,805 | 21,187 | ||||||
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SOURCE Regulus Therapeutics Inc.
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