PKD1

Mechanistic dose response observed at a 2mg/kg dose level based on urinary biomarker analyses Encouraging exploratory results of imaging-based biomarkers with greatest reductions in total kidney volume seen in patients with the highest increases in PC1 and PC2 Cohort 3 fully enrolled with data readout anticipated in mid-2024; Cohort 4 screening to start in 2Q 2024 Company to hold conference call at 8:30am ET today SAN DIEGO, March 12, 2024 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced positive topline results from the second cohort of patients in its Phase 1b MAD study of RGLS8429 for the treatment of ADPKD. The Phase 1b MAD study is a double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of RGLS8429 in adult patients with ADPKD. The study is evaluating RGLS8429 treatment across three different weight-based dose levels, including measuring changes in urinary polycystins 1 and 2 (PC1 and PC2), height-adjusted total kidney volume (htTKV), cyst architecture, and overall kidney function. PC1 and PC2 are the protein products of the PKD1 and PKD2 genes and have been shown to inversely correlate with disease severity. The third cohort is being dosed at 3mg/kg of RGLS8429 or placebo every other week for three months. The Company recently added a fourth cohort for an open label fixed dose of 300mg of RGLS8429 dosed every other week for three months. Based on the results from this second cohort, the Company plans to amend the protocol to increase the sample size to up to 30 patients. In addition to PC1 and PC2 and safety, imaging biomarkers will also be evaluated. 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. Clear evidence of a mechanistic dose response at a 2mg/kg dose level based on urinary PC1 and PC2 polycystin levels was observed. Urinary measurements of PC1 and PC2 demonstrated greater biological activity of RGLS8429 at 2mg/kg compared to 1mg/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 reductions in htTKV >4%, with corresponding reductions in total kidney cyst volume (TKCV). These data suggest potential impact on htTKV and TKCV, which the Company will further explore at higher doses in cohorts 3 and 4. The ongoing Phase 1b study is evaluating RGLS8429 dosing at 3 mg/kg in cohort 3 and will explore a 300 mg fixed dose in cohort 4 that will provide higher exposure in a larger number of patients based on anticipated body weight. "These results from cohort 2 are encouraging, with biomarker data that demonstrate increased activity of RGLS8429 at 2 mg/kg," said Alan Yu, M.B., B.Chir, from the University of Kansas Medical Center. "Noting that several patients in this cohort who had the greatest reductions in kidney volume also had the greatest increases in PC1 and PC2 is certainly intriguing, and I look forward to seeing results from the higher doses planned in the study. As a physician managing patients with ADPKD, the continued development of a potential novel therapeutic is both important and welcome." "We are pleased with the data we have seen in the second cohort, in particular, the mechanistic dose response, as it continues to validate RGLS8429's potential efficacy in ADPKD," said Preston Klassen, M.D., President and Head of Research & Development of Regulus. "Based on these exciting results from cohort 2, we plan to increase the sample size of the cohort 4 fixed-dose open label enrollment up to 30 patients in order to further examine potential impact on cystic volume in patients with APDKD. The clear increases in polycystin levels we saw in the second cohort were encouraging, in particular their potential correlation with improvements in kidney volume and function. We look forward to seeing the topline data from the third cohort in the middle of this year." "These data represent another important step on our way to bringing a potential treatment to those living with ADPKD," said Jay Hagan, CEO of Regulus. "RGLS8429 holds the potential to be able to address the underlying genetic causes of the disease, and we look forward to the data from our third and fourth cohorts to inform our potentially pivotal Phase 2 trial design. As always, I would like to thank the patients and investigators participating in our past and ongoing studies as well as our team who has worked so hard to bring us these data." In addition, the Company recently held a Type D meeting with FDA to discuss the accelerated approval pathway. The meeting was constructive and confirms the potential for an accelerated approval pathway based on a single Phase 2 study of RGLS8429 for the treatment of ADPKD. As of December 31, 2023, the Company had cash and cash equivalents of approximately $23.8 million. More information about the MAD clinical trial is available at clinicaltrials.gov (NCT05521191). Conference Call Information The Company will host a conference call and live audio webcast on March 12, 2024, at 8:30 am Eastern Time. To access the call, please dial (833) 816-1394 (domestic) or (855) 669-9657 (international). The live webcast can be found under "Events and Presentations" through the investor relations section of the Company's website. To access the telephone replay of the call, dial (877) 344-7529 (domestic) or (412) 317-0088 (international), passcode ID 6852735. The webcast and telephone replay will be archived on the Company's website at following the call. 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 50% of ADPKD patients by age 60. Approximately 160,000 individuals are diagnosed with the disease in the United States alone, with an estimated global prevalence of 4 to 7 million. 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 robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity have been demonstrated along with a superior pharmacologic profile in preclinical studies compared to Regulus' first-generation compound, RGLS4326. 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 and is similar to the PK data from the first-generation compound. In the Phase 1b MAD study Regulus announced both top line data from the first cohort of patients, and completion of enrollment for the second cohort of patients in September 2023. After review of all available safety data, Regulus has advanced to the third cohort where dosing has begun, and patients will receive 3 mg/kg of RGLS8429 or placebo every other week for three months. Regulus announced completion of enrollment in the third cohort in January 2024. Regulus announced top line data from the second cohort of patients in March 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 San Diego, CA. 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 potential accelerated approval pathway, 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, 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' quarterly report on Form 10-Q for the quarter ended September 30, 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. SOURCE Regulus Therapeutics Inc.

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced multiple program updates ahead of upcoming investor meetings in January, including the company’s scheduled webcast from the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 11:15 a.m. ET/8:15 a.m. PT. “2023 was marked by continued strong performance in the cystic fibrosis business and acceleration of our development stage pipeline, propelling the company into 2024 with tremendous momentum," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “We continue to reach more patients than ever before with our CF medicines; we’ve launched CASGEVY™ in the U.S., UK and Bahrain following the historic approvals of this gene editing therapy late last year, and we are well positioned to advance our mission of bringing additional transformative medicines to people with serious diseases.” Disease Areas with Approved Medicines Cystic Fibrosis Next-in-class triple combination: Completed three pivotal studies evaluating the next-in-class, triple combination CFTR modulator therapy vanzacaftor/tezacaftor/deutivacaftor compared to TRIKAFTA® in patients with CF ages 6 years of age and older (ages 12+ in SKYLINE 102 and 103 studies; ages 6-11 in the RIDGELINE study). Vertex expects to share the results of all three studies in early 2024. VX-522: Completed dosing in the single ascending dose (SAD) portion of the Phase 1/2 study of VX-522 in patients with CF and initiated the multiple ascending dose (MAD) portion of the study. VX-522 is a CFTR mRNA therapeutic that Vertex is developing in collaboration with Moderna for the >5,000 patients with CF who do not make any CFTR protein and cannot benefit from CFTR modulators. Epidemiology update: Vertex revised its estimates for the number of patients living with cystic fibrosis from ~88,000 to ~92,000 in the U.S., Europe, Australia, and Canada. Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) – CASGEVY Received regulatory approvals for CASGEVY in the fourth quarter of 2023 in the U.S. for sickle cell disease, and in Great Britain and Bahrain for the treatment of both SCD and TDT; also received positive CHMP opinion for CASGEVY for both SCD and TDT from the European Medicines Agency (EMA). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of March 30, 2024, for CASGEVY in TDT. Additional regulatory submissions for CASGEVY are currently under review in Switzerland and the Kingdom of Saudi Arabia, with submission in Canada planned for the first half of 2024. Completed enrollment in two global Phase 3 studies of CASGEVY in patients 5 to 11 years of age with SCD or TDT. Signed an agreement with Synergie Medication Collective, a medication contracting organization founded by a group of Blue Cross and Blue Shield affiliated companies covering approximately 100 million people, to provide access to CASGEVY. Activated 9 authorized treatment centers (ATCs) in the U.S. and 3 in Europe, with an ultimate goal of activating approximately 50 ATCs in the U.S. and 25 in Europe. Epidemiology update: Vertex revised its estimates for the number of patients living with severe sickle cell disease, from ~25,000 patients to ~30,000 patients in the U.S. and Europe, with additional patients in Bahrain and the Kingdom of Saudi Arabia. Vertex’s updated estimate for patients with transfusion-dependent beta thalassemia is revised from ~7,000 to ~5,000 patients in the U.S. and Europe, with additional patients in Bahrain and the Kingdom of Saudi Arabia. Pipeline Disease Areas Acute Pain VX-548: Completed three Phase 3 studies of VX-548 for the treatment of moderate to severe acute pain, including two randomized placebo-controlled studies: one following abdominoplasty and one following bunionectomy surgery, as well as a third single-arm safety and effectiveness study, which enrolled both surgical and non-surgical patients with moderate to severe acute pain. Vertex expects to share the results of all three studies in early 2024. VX-993: Completed a Phase 1 study of an oral formulation of VX-993, a next-generation NaV1.8 inhibitor, and anticipates initiating a Phase 2 study for the treatment of moderate to severe acute pain in 2024. Vertex also anticipates initiating a Phase 1 study of an intravenous formulation of VX-993 for the treatment of moderate to severe acute pain in 2024. Peripheral Neuropathic Pain (PNP) VX-548: Following the December 2023 release of positive Phase 2 results with VX-548 in diabetic peripheral neuropathy, which represents ~20% of peripheral neuropathic pain patients, Vertex plans to meet with regulators in the first quarter of 2024 and anticipates advancing VX-548 into pivotal development. Initiated a Phase 2 study of VX-548 in lumbosacral radiculopathy, another type of peripheral neuropathic pain and the largest patient segment (over 40%) within the PNP category. VX-993: Vertex also anticipates initiating a Phase 2 study with an oral formulation of VX-993 for the treatment of PNP in 2024. APOL1-Mediated Kidney Disease (AMKD) – Inaxaplin (VX-147) Completed enrollment in the Phase 2B dose-ranging portion of the study of inaxaplin for the treatment of patients with AMKD. Vertex expects to select the inaxaplin dose for the Phase 3 portion of the Phase 2/3 pivotal trial and begin Phase 3 in the first quarter of 2024. Type 1 Diabetes (T1D) VX-880: Completed enrollment in Parts A, B, and C of the Phase 1/2 study of VX-880, an allogeneic, stem cell-derived, fully differentiated, insulin-producing islet cell therapy, used in conjunction with standard immunosuppression, in patients with T1D and impaired awareness of hypoglycemia and recurrent hypoglycemic events. Efficacy: As of the last data cut, all 14 patients dosed with VX-880 demonstrated islet cell engraftment and production of endogenous insulin. All Part A and B patients, except for one patient who withdrew consent from the study, demonstrated glycemic control to target ADA recommended levels with HbA1C <7% and no longer required exogenous insulin. Part C patients have trajectories similar to Part A and B patients. Safety: The safety pro VX-880 to date is consistent with immunosuppressives, the perioperative period and past medical history. Two patient deaths, both unrelated to VX-880, have occurred. Vertex has placed the study on a protocol-specified pause, pending review of the totality of the data by the independent data monitoring committee and global regulators. VX-264: The clinical trial for VX-264, which encapsulates the same VX-880 cells in a device that is designed to eliminate the need for immunosuppressants, is a multi-part, Phase 1/2 study. Part A has initiated, enrolled and dosed multiple patients. The study remains ongoing in multiple centers and countries as Vertex prepares for Part B initiation. Myotonic Dystrophy Type 1 (DM1) – VX-670 Received clearances from Health Canada and Medicines and Healthcare Products Regulatory Agency (MHRA – UK) for Clinical Trial Applications (CTA) for VX-670 for patients with DM1. Vertex initiated the Phase 1/2 clinical trial in patients with DM1 in Canada and will initiate the study in the UK near-term. Vertex also submitted an Investigational New Drug (IND) application to the FDA for VX-670. The FDA requested additional information, which resulted in a clinical hold. Vertex is working to address FDA comments and initiate the study in the U.S. VX-670 is an oligonucleotide that targets the underlying cause of DM1, linked to a cyclic peptide that promotes effective delivery into cells. DM1 is the most prevalent muscular dystrophy in adults with ~110,000 people living with the disease in the U.S. and Europe. Autosomal Dominant Polycystic Kidney Disease (ADPKD) – VX-407 Completed IND-enabling studies of VX-407 for the treatment of ADPKD and anticipates beginning a Phase 1 clinical trial in healthy volunteers this year. VX-407 is a first-in-class small molecule corrector that targets the underlying cause of ADPKD in a subset of patients with responsive PKD1 mutations, estimated at ~25,000 (or ~10%) of the overall ~250,000 ADPKD patient population. ADPKD is the most commonly inherited kidney disease. J.P. Morgan Healthcare Conference Presentation and Webcast Dr. Kewalramani will present at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 11:15 a.m. ET/8:15 a.m. PT. A live webcast of management's remarks will be available through the Vertex website, , in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes and alpha-1 antitrypsin deficiency. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 14 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and Twitter/X. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Reshma Kewalramani, M.D., in this press release, and statements about our expectations for our CF program, including plans to share results of our vanzacaftor/tezacaftor/deutivacaftor studies in early 2024, our expectations for CASGEVY™, including plans for additional regulatory submissions, and plans to activate approximately 50 ATCs in the U.S. and 25 in Europe, our expectations for our acute pain program, including plans to share results from the three Phase 3 studies in early 2024, and plans to initiate Phase 1 and Phase 2 studies evaluating VX-993 in 2024, our expectations for our PNP pain program, including plans for meetings with regulators, plans to advance VX-548 into pivotal development, and plans to initiate a Phase 2 study of VX-993 in 2024, our plans to select the inaxaplin dose and begin Phase 3 in the first quarter of 2024, expectations for our T1D program, including anticipated review of VX-880 data by the independent data monitoring committee and global regulators, and our plans to initiate Part B of the VX-264 study, expectations for our DM1 program, including plans to initiate a clinical trial for VX-670 in the UK and plans to work with the FDA on the VX-670 study in the U.S. and initiate the study in the U.S., and expectations for our ADPKD program, including plans to begin a Phase 1 clinical trial for VX-407 in 2024. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that clinical trial data might not be available on the expected timeline, that data from the company's research and development programs may not support registration or further development of its compounds due to safety, efficacy, and other risks, that our discussions with regulators may be delayed or cause delays in our pipeline programs, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at . You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on businesswire.com: Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com Media: mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607 Source: Vertex Pharmaceuticals Incorporated View this news release online at:

SAN DIEGO, Jan. 2, 2024 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced it has completed enrollment in the third cohort of patients in the Phase 1b MAD study of RGLS8429 for the treatment of ADPKD. "The completion of our third and final randomized placebo-controlled cohort in the Phase 1b MAD study is an exciting step for Regulus, and we are happy to reach this stage of development for RGLS8429," said Jay Hagan, CEO of Regulus. "Our team is motivated and gearing up for 2024 when we expect topline data from our second cohort in Q1 2024, topline data from our third cohort in mid-2024, and the initiation of the fourth cohort, an open label fixed dose, of RGLS8429." The Phase 1b MAD study is a double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of RGLS8429 in adult patients with ADPKD. The study will evaluate RGLS8429 treatment across three different weight-based dose levels, including measuring changes in polycystins, height-adjusted total kidney volume (htTKV), cyst architecture, and overall kidney function. The third cohort is being dosed at 3 mg/kg of RGLS8429 or placebo every other week for three months. The protocol was amended to include a fourth cohort of subjects who will receive an open label fixed dose of RGLS8429 to compare biomarker and safety data to the weight-based dosing. More information about the MAD clinical trial is available at clinicaltrials.gov (NCT05521191). 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 50% of ADPKD patients by age 60. Approximately 160,000 individuals are diagnosed with the disease in the United States alone, with an estimated global prevalence of 4 to 7 million. 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 robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity have been demonstrated along with a superior pharmacologic profile in preclinical studies compared to Regulus' first-generation compound, RGLS4326. 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 and is similar to the PK data from the first-generation compound. In the Phase 1b MAD study Regulus announced both top line data from the first cohort of patients, and completion of enrollment for the second cohort of patients in September 2023. After review of all available safety data, Regulus has advanced to the third cohort where dosing has begun, and patients will receive 3 mg/kg of RGLS8429 or placebo every other week for three months. Regulus announced completion of enrollment in the third cohort in January 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 San Diego, CA. 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 clinical translation for ADPKD patients, the expected timing for reporting topline data, the timing and future occurrence of other preclinical and clinical activities and the expected length of our cash runway. 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 the risk additional toxicology data may be negative 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 quarterly report on Form 10-Q. 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. SOURCE Regulus Therapeutics Inc.