Regulus Therapeutics Reports First Quarter 2025 Financial Results and Recent Updates
Regulus Therapeutics (RGLS) has announced its Q1 2025 financial results and significant corporate updates. The company has entered into an acquisition agreement with Novartis for $7.00 per share in cash, plus a potential additional $7.00 per share through a CVR upon achieving regulatory milestones, valuing the deal at up to $1.7 billion. The transaction is expected to close in H2 2025.
The company reported positive topline data from the fourth cohort of its Phase 1b MAD study of farabursen (RGLS8429) for ADPKD treatment. The study demonstrated favorable safety profiles and efficacy in halting kidney volume growth. Regulus plans to initiate a Phase 3 pivotal trial in Q3 2025.
Q1 2025 financial results showed $65.4 million in cash and investments, with a net loss of $9.6 million ($0.15 per share). R&D expenses were $6.8 million, and G&A expenses were $3.7 million.
Regulus Therapeutics (RGLS) ha annunciato i risultati finanziari del primo trimestre 2025 e importanti aggiornamenti aziendali. La società ha stipulato un accordo di acquisizione con Novartis per 7,00 $ per azione in contanti, più un possibile ulteriore 7,00 $ per azione tramite un CVR al raggiungimento di determinate tappe regolatorie, valutando l'operazione fino a 1,7 miliardi di dollari. La transazione è prevista per la seconda metà del 2025.
La società ha riportato dati positivi preliminari dal quarto gruppo del suo studio MAD di Fase 1b su farabursen (RGLS8429) per il trattamento della ADPKD. Lo studio ha evidenziato un profilo di sicurezza favorevole e efficacia nel bloccare la crescita del volume renale. Regulus prevede di avviare uno studio pivotale di Fase 3 nel terzo trimestre 2025.
I risultati finanziari del primo trimestre 2025 mostrano 65,4 milioni di dollari in liquidità e investimenti, con una perdita netta di 9,6 milioni di dollari (0,15 $ per azione). Le spese per R&S sono state di 6,8 milioni di dollari, mentre quelle generali e amministrative di 3,7 milioni di dollari.
Regulus Therapeutics (RGLS) ha anunciado sus resultados financieros del primer trimestre de 2025 y actualizaciones corporativas importantes. La compañía ha firmado un acuerdo de adquisición con Novartis por 7,00 $ por acción en efectivo, más un posible adicional de 7,00 $ por acción a través de un CVR al alcanzar hitos regulatorios, valorando la operación en hasta 1,7 mil millones de dólares. Se espera que la transacción se cierre en la segunda mitad de 2025.
La empresa reportó datos positivos preliminares del cuarto grupo de su estudio MAD de Fase 1b de farabursen (RGLS8429) para el tratamiento de ADPKD. El estudio mostró perfiles de seguridad favorables y eficacia para detener el crecimiento del volumen renal. Regulus planea iniciar un ensayo pivotal de Fase 3 en el tercer trimestre de 2025.
Los resultados financieros del primer trimestre de 2025 mostraron 65,4 millones de dólares en efectivo e inversiones, con una pérdida neta de 9,6 millones de dólares (0,15 $ por acción). Los gastos en I+D fueron de 6,8 millones de dólares y los gastos generales y administrativos de 3,7 millones de dólares.
Regulus Therapeutics (RGLS)가 2025년 1분기 재무 결과와 주요 기업 업데이트를 발표했습니다. 회사는 Novartis와 주당 7.00달러 현금 인수 계약을 체결했으며, 규제 마일스톤 달성 시 주당 추가 7.00달러의 CVR을 통해 최대 17억 달러 규모의 거래로 평가됩니다. 거래는 2025년 하반기에 완료될 예정입니다.
회사는 ADPKD 치료를 위한 farabursen (RGLS8429)의 1b상 MAD 연구 4번째 코호트에서 긍정적인 초기 데이터를 보고했습니다. 연구는 안전성 프로필이 우수하고 신장 부피 증가를 억제하는 효능을 입증했습니다. Regulus는 2025년 3분기에 3상 주요 임상시험을 시작할 계획입니다.
2025년 1분기 재무 결과는 현금 및 투자 자산이 6540만 달러이며, 순손실은 960만 달러 (주당 0.15달러)였습니다. 연구개발 비용은 680만 달러, 일반관리비는 370만 달러였습니다.
Regulus Therapeutics (RGLS) a annoncé ses résultats financiers du premier trimestre 2025 ainsi que des mises à jour importantes concernant l'entreprise. La société a conclu un accord d'acquisition avec Novartis pour 7,00 $ par action en espèces, plus un complément potentiel de 7,00 $ par action via un CVR en cas d'atteinte de jalons réglementaires, valorisant l'opération jusqu'à 1,7 milliard de dollars. La transaction devrait être finalisée au second semestre 2025.
La société a communiqué des données préliminaires positives issues de la quatrième cohorte de son étude MAD de phase 1b sur farabursen (RGLS8429) pour le traitement de la polykystose rénale autosomique dominante (ADPKD). L'étude a montré un profil de sécurité favorable et une efficacité pour stopper la croissance du volume rénal. Regulus prévoit de lancer un essai pivot de phase 3 au troisième trimestre 2025.
Les résultats financiers du premier trimestre 2025 indiquent 65,4 millions de dollars en liquidités et investissements, avec une perte nette de 9,6 millions de dollars (0,15 $ par action). Les dépenses en R&D se sont élevées à 6,8 millions de dollars, tandis que les frais généraux et administratifs ont atteint 3,7 millions de dollars.
Regulus Therapeutics (RGLS) hat seine Finanzergebnisse für das erste Quartal 2025 und wichtige Unternehmensupdates bekanntgegeben. Das Unternehmen hat eine Übernahmevereinbarung mit Novartis getroffen, die einen Barpreis von 7,00 $ pro Aktie sowie eine mögliche zusätzliche Zahlung von 7,00 $ pro Aktie über einen CVR bei Erreichen regulatorischer Meilensteine vorsieht, wodurch der Deal mit bis zu 1,7 Milliarden Dollar bewertet wird. Der Abschluss der Transaktion wird für die zweite Hälfte 2025 erwartet.
Das Unternehmen berichtete positive vorläufige Daten aus der vierten Kohorte seiner Phase-1b-MAD-Studie zu farabursen (RGLS8429) zur Behandlung von ADPKD. Die Studie zeigte günstige Sicherheitsprofile und Wirksamkeit bei der Hemmung des Nierenvolumenwachstums. Regulus plant, im dritten Quartal 2025 eine Phase-3-Pivotalstudie zu starten.
Die Finanzergebnisse für das erste Quartal 2025 zeigten 65,4 Millionen Dollar an liquiden Mitteln und Investitionen sowie einen Nettoverlust von 9,6 Millionen Dollar (0,15 $ pro Aktie). Die F&E-Ausgaben betrugen 6,8 Millionen Dollar, die allgemeinen Verwaltungsaufwendungen 3,7 Millionen Dollar.
- Acquisition by Novartis at $7.00 per share with potential additional $7.00 through CVR, valuing the deal up to $1.7 billion
- Positive Phase 1b trial results showing halting of kidney volume growth
- FDA alignment on Phase 3 trial design with potential Accelerated Approval pathway
- Strong cash position of $65.4 million with runway into early 2026
- Increased net loss to $9.6 million in Q1 2025 from $8.5 million in Q1 2024
- Higher operating expenses with R&D costs up to $6.8M and G&A expenses up to $3.7M
Insights
Novartis acquisition offers Regulus shareholders substantial upfront cash with additional upside potential through regulatory milestone-based CVR.
The acquisition agreement between Regulus and Novartis represents a significant liquidity event for Regulus shareholders. The deal structure provides $7.00 per share in cash upfront plus a Contingent Value Right (CVR) that could deliver an additional $7.00 per share upon achievement of a regulatory milestone for farabursen. This creates a total potential value of approximately $1.7 billion, with $0.8 billion guaranteed at closing.
The transaction's structure is strategically balanced - providing immediate guaranteed value while allowing shareholders to maintain exposure to farabursen's future regulatory success. This approach effectively transfers late-stage development risk to Novartis, whose global development and commercial infrastructure significantly enhances the probability of successful commercialization.
Examining Regulus's financial position, the company reported $65.4 million in cash as of March 31, 2025, with a runway extending into early 2026. The quarterly financial results show R&D expenses of $6.8 million (up from $6.0 million year-over-year) and G&A expenses of $3.7 million (up from $2.8 million), resulting in a net loss of $9.6 million for Q1 2025.
The transaction is expected to close in H2 2025, subject to shareholder approval (requiring tender of at least majority shares) and regulatory clearances. For Regulus shareholders, this deal eliminates the binary regulatory risk while providing both immediate returns and potential additional value through the CVR mechanism.
Positive Phase 1b data, FDA alignment on pivotal trial design, and Novartis acquisition validate farabursen's potential as an ADPKD treatment.
The clinical data from farabursen's Phase 1b multiple-ascending dose study represents a significant development in ADPKD treatment. The completed fourth cohort (26 patients receiving 300mg fixed dose) demonstrated two critical outcomes: a mechanistic dose response based on urinary PC1 and PC2 levels and a mean halting of height-adjusted total kidney volume (htTKV) growth over the four-month study period.
These results are particularly meaningful because htTKV growth is a validated biomarker of ADPKD progression, and its stabilization suggests potential disease-modifying effects. The finding that the 300mg fixed dose showed comparable efficacy to the 3mg/kg weight-based dosing (while maintaining a favorable safety profile) provides clear direction for the upcoming pivotal trial.
The FDA alignment on the Phase 3 trial design is strategically significant, with a 12-month htTKV endpoint for Accelerated Approval and a 24-month eGFR endpoint for Full Approval. This dual-endpoint approach could substantially expedite market access while gathering definitive evidence of kidney function preservation.
Novartis's acquisition validates farabursen's potential in a therapeutic area with limited treatment options. The regulatory milestone-based CVR highlights the importance of the upcoming Phase 3 trial, scheduled to begin in Q3 2025. Novartis's established global development and commercial capabilities significantly enhance the probability of successfully bringing this potential therapy to ADPKD patients, addressing a substantial unmet medical need.
Entered into an agreement to be acquired by Novartis for
Positive topline data from the completed fourth cohort of patients in the Phase 1b multiple-ascending dose (MAD) study of farabursen (RGLS8429) for the treatment of autosomal dominant polycystic kidney disease (ADPKD)
Company on track for initiation of Phase 3 single pivotal trial in the third quarter 2025
"We recently announced that we have entered into an agreement to be acquired by Novartis, whose established global development and commercial capabilities will potentially bring farabursen to patients with ADPKD, who currently have limited treatment options," said Jay Hagan, CEO of Regulus Therapeutics. "We've made important progress with farabursen so far this year, having announced positive results from the completed fourth cohort of patients in March, reporting evidence of a mechanistic dose response based on urinary PC1 and PC2 levels, and results suggesting that kidney volume growth rate was halted after only a relatively short treatment period. We look forward to investigating further as we head towards initiation of the pivotal Phase 3 trial in the third quarter of this year."
Program Updates
Farabursen for ADPKD: In March 2025, the Company shared positive topline results from all patients in the fourth cohort of the Phase 1b MAD study of farabursen for the treatment of ADPKD. In the fourth cohort, 26 subjects received a fixed dose of 300 mg of farabursen every other week for three months. Consistent with the previously announced interim analysis of efficacy data from the first 14 subjects of this fixed-dose cohort in January 2025, in the full cohort of 26 patients we observed similar mechanistic response based on urinary polycystins 1 and 2 (PC1 and PC2) levels as well as a mean halting of height-adjusted total kidney volume (htTKV) growth over the four-month study period. These results were in line with data from the third cohort, dosed at 3 mg/kg, which is predicted to achieve optimal kidney exposure. Final efficacy data confirmed the appropriateness of a 300 mg fixed dose for the Phase 3 pivotal trial. Based on complete safety data from all 26 subjects we observed that farabursen dosed at 300 mg had a favorable safety and tolerability profile in this study, consistent with all earlier cohorts.
The Phase 1b MAD study was a double-blind, placebo-controlled trial which evaluated the safety, tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of farabursen in adult patients with ADPKD. The study evaluated farabursen treatment for three months followed by one month of follow up across three weight-based dose levels (1, 2 and 3 mg/kg) and one fixed dose level (300 mg). Primary endpoints were changes in urinary PC1 and PC2, with an exploratory examination of change in htTKV growth rate. PC1 and PC2 are the protein products of the PKD1 and PKD2 genes and their levels have been shown to inversely correlate with disease severity; htTKV has been shown to inversely correlate with kidney function.
A single, pivotal Phase 3 study is planned to be initiated in the third quarter of this year. In January, the Company announced that it held a meeting with the
Corporate Highlights
On April 30, 2025, the Company announced that it entered into an agreement and plan of merger with Novartis (the "Merger Agreement"). Pursuant to the Merger Agreement, Novartis, through an indirectly wholly owned subsidiary, will acquire Regulus for an initial payment of
Completion of the transaction is expected in the second half of 2025, subject to the satisfaction or waiver of customary closing conditions, including the tender of shares representing at least a majority of the total number of Regulus' outstanding shares of common stock and the receipt of required regulatory approvals. Until that time, Regulus will continue to operate as a separate and independent company.
Financial Results
Cash, Cash Equivalents and Short-Term Investments: As of March 31, 2025, 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 Farabursen (RGLS8429)
Farabursen is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney. Administration of farabursen has shown clear improvements in kidney function, size, and other measures of disease severity in preclinical models. Regulus announced the completion of the Phase 1 SAD study in September 2022. The Phase 1 SAD study demonstrated that farabursen has a favorable safety and PK profile. Farabursen was observed to be well-tolerated with no serious adverse events reported and plasma exposure was approximately linear across the four doses tested. In the now completed 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, from the third cohort of patients in June 2024, and from the fourth cohort in March 2025. Based on complete safety data from all cohorts, farabursen was observed to be well tolerated, and observed results showed anticipated biomarker response (PC1 & PC2) and impact on kidney volume that potentially derisks Phase 3 trial. Initiation of the Phase 3 trial is planned for the third quarter of 2025.
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
Important Information About the Tender Offer
The tender offer described above has not yet commenced. This communication is not an offer to buy nor a solicitation of an offer to sell any securities of the Company. The solicitation and the offer to buy shares of the Company common stock will only be made pursuant to a tender offer statement on Schedule TO, including an offer to purchase, a letter of transmittal and other related materials that Novartis and Redwood Merger Sub Inc. ("Merger Sub") intend to file with the Securities and Exchange Commission (the "SEC"). In addition, the Company will file with the SEC a solicitation/recommendation statement on Schedule 14D-9 with respect to the tender offer. Once filed, investors will be able to obtain a free copy of these materials and other documents filed by Novartis, Merger Sub and the Company with the SEC at the website maintained by the SEC at www.sec.gov or by directing a request for such materials and other documents to the information agent for the offer, which will be named in the tender offer statement. Investors may also obtain, at no charge, any such documents filed with or furnished to the SEC by the Company under the "Investors" section of the Company's website at https://ir.regulusrx.com/. Further risks and uncertainties that could cause actual results to differ materially from the results anticipated by the forward-looking statements are detailed from time to time in the Company's periodic reports filed with the SEC, including the Company's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. These filings, when available, are available on the investor relations section of the Company's website mentioned above or on the SEC's website at https://www.sec.gov.
INVESTORS AND SECURITY HOLDERS ARE ADVISED TO READ THESE DOCUMENTS WHEN THEY BECOME AVAILABLE, INCLUDING THE TENDER OFFER MATERIALS (INCLUDING AN OFFER TO PURCHASE, A LETTER OF TRANSMITTAL AND RELATED DOCUMENTS) AND ANY AMENDMENTS THERETO, THE SOLICITATION/RECOMMENDATION STATEMENT ON SCHEDULE 14D-9 OF THE COMPANY AND ANY AMENDMENTS THERETO, AS WELL AS ANY OTHER DOCUMENTS RELATING TO THE TENDER OFFER AND THE MERGER THAT ARE FILED WITH THE SEC, CAREFULLY AND IN THEIR ENTIRETY PRIOR TO MAKING ANY DECISIONS WITH RESPECT TO WHETHER TO TENDER THEIR SHARES INTO THE TENDER OFFER BECAUSE THEY CONTAIN IMPORTANT INFORMATION, INCLUDING THE TERMS AND CONDITIONS OF THE TENDER OFFER.
Forward-Looking Statements
The statements included in this press release that are not a description of historical facts are forward-looking statements. Words or phrases such as such as "believe," "may," "could," "will," "estimate," "continue," "anticipate," "intend," "seek," "plan," "expect," "should," "would" or similar expressions are intended to identify forward-looking statements. The forward-looking statements are based on the Company's current beliefs and expectations and include, but are not limited to: statements regarding beliefs about the potential benefits of the transaction; the planned completion and timing of the transactions contemplated by the Merger Agreement or the CVR agreement governing the terms of the CVR; the prospective performance and outlook of the business, performance, and opportunities of the Company continuing as the surviving corporation; the Company's farabursen (RGLS8429) program; the potential that farabursen may be eligible for an Accelerated Approval pathway; predictions based on and future results and clinical outcomes suggested by the fourth cohort data; our Phase 3 trial design including whether the selected fixed dose will be efficacious or whether the FDA will ultimately determine its components and the overall design to be acceptable and sufficient to serve as a single pivotal study; planned data announcements; 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. Risks and uncertainties that could cause results to differ from expectations include: uncertainties as to the timing and completion of the tender offer and the merger; uncertainties as to the percentage of the Company stockholders tendering their shares in the tender offer; the possibility that competing offers will be made; the possibility that various closing conditions for the tender offer or the merger may not be satisfied or waived, including the failure to receive any required regulatory approvals from any applicable regulatory and/or governmental entities (or any conditions, limitations or restrictions placed on such approvals); risks relating to the Company's liquidity during the pendency of the tender offer and the merger or in the event of a termination of the Merger Agreement; risks that the milestone related to the CVR is not achieved; the effects of disruption caused by the transaction making it more difficult to maintain relationships with employees, collaborators, vendors and other business partners; risks related to diverting management's attention from the Company's ongoing business operations; the risk that stockholder litigation in connection with the transactions contemplated by the Merger Agreement may result in significant costs of defense, indemnification and liability; the risk that the approach the Company is taking to discover and develop drugs is novel and may never lead to marketable products; 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; an Accelerated Approval pathway designation may not be received, or even if it is received, lead to a faster development, regulatory review or approval process, and does not increase the likelihood that farabursen will receive marketing approval; the Company's clinical development strategy may change; the risk that preclinical and clinical studies may not be successful; risks related to regulatory review and approval; risks related to the Company's 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 data may be negative; and risks related to the Company's ability to successfully secure and deploy capital; and other risks and uncertainties pertaining to the Company's business, including the risks and uncertainties detailed in the Company's public periodic filings with the SEC, as well as the tender offer materials to be filed by Novartis and Merger Sub and the solicitation/recommendation statement on Schedule 14D-9 to be filed by the Company in connection with the tender offer. These and other risks are described in additional detail in Regulus' filings with the SEC, including under the "Risk Factors" heading of Regulus' most recently filed annual report on Form 10-K or subsequently filed 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.
You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and the Company undertakes no obligation to revise or update these statements to reflect events or circumstances after the date hereof, except as required by law.
Regulus Therapeutics Inc. | |||||||||
Three months ended | |||||||||
2025 | 2024 | ||||||||
Operating expenses: | |||||||||
Research and development | 6,819 | 6,040 | |||||||
General and administrative | 3,719 | 2,786 | |||||||
Total operating expenses | 10,538 | 8,826 | |||||||
Loss from operations | (10,538) | (8,826) | |||||||
Other income, net | 904 | 357 | |||||||
Loss before income taxes | (9,634) | (8,469) | |||||||
Net loss | $ | (9,634) | $ | (8,469) | |||||
Net loss per share, basic and diluted | $ | (0.15) | $ | (0.29) | |||||
Weighted average shares used to compute basic and diluted net loss per | 66,174,550 | 28,746,802 | |||||||
March 31, | December 31, | |||||
Cash, cash equivalents and short-term investments | $ | 65,368 | $ | 75,777 | ||
Total assets | 74,826 | 84,181 | ||||
Stockholders' equity | 69,028 | 76,408 | ||||
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SOURCE Regulus Therapeutics Inc.
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