DRNA Dicerna Pharmaceuticals Inc.

21.61
+0.22  (+1%)
Previous Close 21.39
Open 21.44
52 Week Low 17.76
52 Week High 40.14
Market Cap $1,679,835,457
Shares 77,734,172
Float 74,503,276
Enterprise Value $1,134,917,939
Volume 2,182,931
Av. Daily Volume 1,127,780
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Upcoming Catalysts

Drug Stage Catalyst Date
Nedosiran (PHYOX4)
Primary Hyperoxaluria Type 3
Phase 1
Phase 1
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Belcesiran
Alpha 1-Antitrypsin deficiency-associated liver disease
Phase 1
Phase 1
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Nedosiran - PHYOX2
Primary hyperoxaluria (PH) - Type 1
NDA Filing
NDA Filing
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Drug Pipeline

Drug Stage Notes
DCR-AUD
Alcohol Use Disorder
Phase 1
Phase 1
Phase 1 trial to commence 3Q 2021.
LY3819469
Cardiometabolic diseases
Phase 1
Phase 1
Phase 1 IND acceptance announced May 27, 2021.
Nedosiran - PHYOX3
Primary hyperoxaluria (PH)
Phase 1
Phase 1
Phase 1 multi-dose data presented March 31, 2020.
RG6346
Hepatitis B virus (HBV)
Phase 2
Phase 2
Phase 2 trial initiation announced March 4, 2021.
LY3561774
Undisclosed cardiometabolic disease.
Phase 1
Phase 1
Phase 1 IND accepted November 2020.
DCR-MYC
Hepatocellular carcinoma (HCC) - cancer
Phase 1/2
Phase 1/2
Development discontinued September 2016.

Latest News

  1. Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that Douglas M. Fambrough, Ph.D., President and Chief Executive Officer, will participate in a virtual fireside chat at the 16th Annual Citi Biopharma Conference on Sept. 9, 2021 at 7:55 a.m. ET.

    An audio webcast of the fireside chat will be accessible from the "Events and Presentations" page in the "Investors and Media" section of the Dicerna website at www.dicerna.com. An archived replay will be available on the Company's website following the event.

    About Dicerna Pharmaceuticals, Inc.

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) is a biopharmaceutical company…

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that Douglas M. Fambrough, Ph.D., President and Chief Executive Officer, will participate in a virtual fireside chat at the 16th Annual Citi Biopharma Conference on Sept. 9, 2021 at 7:55 a.m. ET.

    An audio webcast of the fireside chat will be accessible from the "Events and Presentations" page in the "Investors and Media" section of the Dicerna website at www.dicerna.com. An archived replay will be available on the Company's website following the event.

    About Dicerna Pharmaceuticals, Inc.

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) is a biopharmaceutical company focused on discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to silence selectively genes that cause or contribute to disease. Using our proprietary GalXC™ and GalXC-Plus™ RNAi technologies, Dicerna is committed to developing RNAi-based therapies with the potential to treat both rare and more prevalent diseases. By silencing disease-causing genes, Dicerna's GalXC platform has the potential to address conditions that are difficult to treat with other modalities. Initially focused on disease-causing genes in the liver, Dicerna has continued to innovate and is exploring new applications of its RNAi technology with GalXC-Plus, which expands the functionality and application of our flagship liver-targeted GalXC technology to tissues and cell types outside the liver, and has the potential to treat diseases across multiple therapeutic areas. In addition to our own pipeline of core discovery and clinical candidates, Dicerna has established collaborative relationships with some of the world's leading pharmaceutical companies, including Novo Nordisk A/S, Roche, Eli Lilly and Company, Alexion Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH and Alnylam Pharmaceuticals, Inc. Between Dicerna and our collaborative partners, we currently have more than 20 active discovery, preclinical or clinical programs focused on cardiometabolic, viral, chronic liver and complement-mediated diseases, as well as neurodegenerative diseases and pain. At Dicerna, our mission is to interfere – to silence genes, to fight disease, to restore health. For more information, visit www.dicerna.com.

    Cautionary Note on Forward-Looking Statements

    This press release includes forward-looking statements pertaining to the Company's planned participation at an investor conference, which may include discussion of the Company's business and operations, including the discovery, development and commercialization of our product candidates and technologies, and the therapeutic potential thereof, the success of our collaborations with partners and any potential future collaborations. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those relating to our preclinical research and clinical programs and other risks identified under the heading "Risk Factors" included in our most recent Form 10-Q and Form 10-K filings and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.

    GalXC™ and GalXC-Plus™ are trademarks of Dicerna Pharmaceuticals, Inc.

    View Full Article Hide Full Article
  2. – Reported Positive Top-Line Data From Pivotal PHYOX2 Clinical Trial of Nedosiran Investigational GalXC RNAi Therapy for Treatment of Primary Hyperoxaluria –

    – Announced U.S. Food and Drug Administration (FDA) Clearance of Investigational New Drug (IND) Application for DCR-AUD for Alcohol Use Disorder –

    – Announced Interim Phase 1 Results and Initiated Patient Dosing in ESTRELLA Phase 2 Study of Belcesiran for the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease (AATLD) –

    – Received $10.0 Million Milestone Payment Following FDA Acceptance of Lilly's Second GalXC RNAi IND Application Under Companies' Global Research Collaboration and Licensing Agreement –

    – Company Reported $709.6 Million in Cash, Cash Equivalents and

    – Reported Positive Top-Line Data From Pivotal PHYOX2 Clinical Trial of Nedosiran Investigational GalXC RNAi Therapy for Treatment of Primary Hyperoxaluria –

    – Announced U.S. Food and Drug Administration (FDA) Clearance of Investigational New Drug (IND) Application for DCR-AUD for Alcohol Use Disorder –

    – Announced Interim Phase 1 Results and Initiated Patient Dosing in ESTRELLA Phase 2 Study of Belcesiran for the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease (AATLD) –

    – Received $10.0 Million Milestone Payment Following FDA Acceptance of Lilly's Second GalXC RNAi IND Application Under Companies' Global Research Collaboration and Licensing Agreement –

    – Company Reported $709.6 Million in Cash, Cash Equivalents and Held-to-Maturity Investments as of June 30, 2021 –

    – Management to Host Conference Call Today at 8:00 a.m. ET –

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today reported its financial results for the quarter ended June 30, 2021 and provided a corporate update.

    "We have seen significant progress across our core and collaborative pipelines over the past few months, including the achievement of a major milestone in our maturation as a company," said Douglas Fambrough, Ph.D., President and Chief Executive Officer at Dicerna. "Specifically, our recently announced PHYOX™2 results represent our first positive pivotal trial readout. PHYOX2 generated robust data, meeting the primary and key secondary efficacy endpoints, and nedosiran was generally well tolerated in the trial. Based on these results, we believe nedosiran has significant potential as a treatment for patients with PH1, and we are on course to submit a New Drug Application to the FDA for the treatment of PH1 in the fourth quarter. Notably, these positive results reflect broadly on our GalXC™ RNAi platform and bode well for our additional proprietary and partnered programs.

    "As positive as these results are for PH1, the inconsistent data seen specifically in participants with PH2 have led us to make the strategic decision not to move forward with our plan to build Dicerna into a fully integrated commercial enterprise to support nedosiran," Dr. Fambrough continued. "Instead, we intend to pursue commercial out-licensing opportunities to help ensure global access to nedosiran, subject to necessary approvals. This approach will allow us to deploy our capital and talent on our discovery and development pipeline efforts with our GalXC and GalXC-Plus™ RNAi investigational therapeutics for ourselves and our partners. With these strategic adjustments focused on our core strengths, we can extend our cash runway into 2025."

    Recent Updates

    • Positive Top-Line Data From Pivotal PHYOX2 Clinical Trial of Nedosiran for Primary Hyperoxaluria (PH). Dicerna announced in August positive top-line results from the pivotal PHYOX2 clinical trial of nedosiran. Nedosiran achieved the primary endpoint in the PHYOX2 trial, demonstrating a statistically significant reduction from baseline in urinary oxalate (Uox) excretion compared to placebo (p<0.0001). The study also achieved the key secondary endpoint, with a significantly higher proportion of patients given nedosiran achieving and sustaining normal or near-normal Uox at two or more consecutive visits after Day 90 compared to placebo (p=0.0025). Uox reductions were significant in participants with PH1 while participants with PH2 (5 nedosiran and 1 placebo) showed inconsistent results in this trial. Nedosiran was generally well tolerated in the study with an overall adverse event (AE) profile consistent with previously reported data from PHYOX trials. The Company expects the results from the PHYOX2 trial to support marketing authorization applications for the treatment of PH1 in the U.S. and other major markets and intends to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2021.
    • Announced Clearance of Investigational New Drug (IND) Application for DCR-AUD for the Treatment of Alcohol Use Disorder (AUD). In July 2021, Dicerna announced U.S. Food and Drug Administration (FDA) clearance of the IND application for DCR-AUD, the Company's investigational GalXC RNAi candidate for the treatment of AUD. Dicerna plans to initiate a Phase 1 trial in the third quarter of 2021 to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single ascending doses of DCR-AUD in healthy volunteers.
    • Reported Interim Data From Phase 1 Trial of Belcesiran. In July 2021, Dicerna reported interim data from the Company's Phase 1 trial of belcesiran, a GalXC RNAi therapeutic candidate in development for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease (AATLD). The trial is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of belcesiran in healthy volunteers. Data from this interim analysis of the four completed active-treatment dose cohorts (0.1, 1.0, 3.0 and 6.0 mg/kg) showed dose-dependent reductions in serum AAT with administration of a single dose of belcesiran. In this analysis, belcesiran was found to have an acceptable safety profile and was generally well tolerated. The final 12.0 mg/kg dose cohort in this trial is ongoing, and the Company plans to present additional data from this study at a medical congress in 2021, subject to abstract acceptance.
    • Initiated ESTRELLA Phase 2 Clinical Trial Patient Dosing of Belcesiran for the Treatment of AATLD. In June 2021, Dicerna announced initiation of the Company's ESTRELLA Phase 2 trial for belcesiran, as part of the SHINE clinical development program for the treatment of AATLD. The ESTRELLA Phase 2 trial is a randomized, multidose, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of belcesiran in participants with AATLD.
    • Completed Nedosiran PHYOX4 Clinical Trial Dosing for Treatment of Primary Hyperoxaluria Type 3. In June 2021, Dicerna announced completion of patient dosing in its PHYOX4 trial, a randomized, placebo-controlled, double-blind, multicenter study evaluating safety and tolerability of nedosiran for the treatment of PH3. PHYOX4 is a part of the PHYOX clinical development program evaluating nedosiran in patients with all three known subtypes of PH. The Company expects to report top-line results from this study in October 2021.

    Collaboration Updates

    • Reported FDA Acceptance of IND for LY3819496 Filed by Eli Lilly and Company ("Lilly"). In May 2021, Dicerna announced FDA acceptance of Lilly's IND for LY3819469, the second clinical-stage investigational GalXC RNAi candidate to emerge from Dicerna's collaboration with Lilly. The IND milestone triggered a $10.0 million payment to Dicerna, and in June 2021, Lilly initiated dosing in a Phase 1 clinical trial of LY3819469, targeting the LPA gene, as a potential treatment of cardiometabolic diseases.
    • Announced Boehringer Ingelheim's Acceptance of DCR-LIV2 for Development Under RNAi Research Collaboration and License Agreement. In May 2021, Dicerna announced Boehringer Ingelheim's (BI) acceptance of DCR-LIV2, an investigational GalXC RNAi candidate for advancement under the existing agreement between the companies for the discovery and development of novel therapies for the treatment of chronic liver diseases. DCR-LIV2 will be evaluated for the treatment of nonalcoholic steatohepatitis (NASH), a chronic liver disease for which there are no approved therapeutic interventions. This candidate acceptance triggered a single-digit multimillion-dollar preclinical milestone payment to Dicerna in the second quarter of 2021.

    Anticipated Upcoming 2021 Milestones

    • Nedosiran:
      • Top-line data from PHYOX4 trial in patients with PH3 in October 2021
      • Initiate PHYOX8 trial, an open-label study in patients aged 0-5 years with PH1 or PH2, in the third quarter of 2021
      • NDA submission in the fourth quarter of 2021
    • Belcesiran: Present Phase 1 data at a medical congress, subject to abstract acceptance
    • DCR-AUD: Initiate Phase 1 study in healthy volunteers in the third quarter of 2021

    Financial Results for the Second Quarter Ended June 30, 2021

    • Cash Position – As of June 30, 2021, Dicerna had $709.6 million in cash, cash equivalents and held-to-maturity investments, compared to $568.8 million as of Dec. 31, 2020.
    • Revenue – Dicerna recognized $41.3 million of revenue for the second quarter 2021, compared to $40.4 million for the same period in 2020. Revenue was relatively flat for the second quarter 2021, compared to the same period in 2020, as increases in Lilly, Novo, and BI revenues were largely offset by decreases in Roche and Alexion revenues.
    • Research and Development (R&D) Expenses – R&D expenses were $56.1 million for the second quarter 2021, compared to $53.4 million for the same period in 2020. The increase was primarily due to increases in facilities, depreciation, and other expenses and employee-related expenses as a result of an increase in R&D headcount necessary to support the Company's expanding pipeline and collaboration agreements. These increases were largely offset by a decrease in direct R&D expenses, primarily due to decreases in drug substance expense.
    • General and Administrative (G&A) Expenses – G&A expenses were $25.5 million for the second quarter 2021, compared to $20.6 million for the same period in 2020. The increase was primarily due to an increase in professional consulting fees.
    • Net Loss – Net loss was $40.8 million, or $0.53 per share, for the second quarter ended June 30, 2021, compared to a net loss of $31.8 million, or $0.43 per share, for the same period in 2020.

    Guidance

    Dicerna believes that its cash, cash equivalents, held-to-maturity investments, and anticipated milestone and other payments from existing collaborations will be sufficient to fund the execution of its current clinical and operating plan into 2025, which includes supporting all R&D activities for current internal and collaboration pipeline programs. This estimate assumes no funding from new collaboration agreements or from external financing events and no significant unanticipated changes in costs and expenses.

    Dicerna expects its research and development expenses to continue to increase for the foreseeable future, largely due to clinical manufacturing activities, continued clinical activities associated with its core product candidates and continued activities under its existing collaboration agreements. The Company continues to forecast receiving $83.0 million in cash from its current collaboration agreements during full-year 2021, of which $74.5 million has been received in the first six months of 2021.

    Conference Call

    Management will host a conference call at 8:00 a.m. ET today to review Dicerna's second quarter 2021 financial results and provide a general business update. The conference call can be accessed by dialing (855) 453-3834 or +1 (484) 756-4306 (international) and referencing conference ID 5418088 prior to the start of the call. The call will also be webcast and will be available under the "Investors & Media" section of the Dicerna website, www.dicerna.com. A replay of the call will be available approximately two hours after the completion of the call and will remain available for seven days. To access the replay, please dial (855) 859-2056 or +1 (404) 537-3406 and refer to conference ID 5418088. The webcast will also be archived on Dicerna's website.

    About Dicerna Pharmaceuticals, Inc.

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) is a biopharmaceutical company focused on discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to silence selectively genes that cause or contribute to disease. Using our proprietary GalXC™ and GalXC-Plus™ RNAi technologies, Dicerna is committed to developing RNAi-based therapies with the potential to treat both rare and more prevalent diseases. By silencing disease-causing genes, Dicerna's GalXC platform has the potential to address conditions that are difficult to treat with other modalities. Initially focused on disease-causing genes in the liver, Dicerna has continued to innovate and is exploring new applications of its RNAi technology with GalXC-Plus, which expands the functionality and application of our flagship liver-targeted GalXC technology to tissues and cell types outside the liver, and has the potential to treat diseases across multiple therapeutic areas. In addition to our own pipeline of core discovery and clinical candidates, Dicerna has established collaborative relationships with some of the world's leading pharmaceutical companies, including Novo Nordisk A/S, Roche, Eli Lilly and Company, Alexion Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH and Alnylam Pharmaceuticals, Inc. Between Dicerna and our collaborative partners, we currently have more than 20 active discovery, preclinical or clinical programs focused on cardiometabolic, viral, chronic liver and complement-mediated diseases, as well as neurodegenerative diseases and pain. At Dicerna, our mission is to interfere – to silence genes, to fight disease, to restore health. For more information, visit www.dicerna.com.

    Cautionary Note on Forward-Looking Statements

    This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements we make regarding our and our collaborative partners' product candidates and the development thereof, including nedosiran, belcesiran, DCR-AUD, DCR-LIV2 and LY3819469; the progress of and anticipated milestones for the Company's ongoing and planned trials, including those from its PHYOX program as well as other trials of nedosiran; results from ongoing and planned trials of the Company's PHYOX clinical development program; the initiation of trials for product candidates in our pipeline, including nedosiran and DCR-AUD; the filing of INDs of our and our partners' product candidates; the therapeutic potential of our product candidates, including nedosiran; the planned submission of the New Drug Application (NDA) for nedosiran and our commercialization strategy for nedosiran, if approved; our collaborations and other strategic arrangements, including the intended benefits thereof; our business and operations, including the discovery, development and commercialization of our product candidates and technology platform, and the therapeutic potential thereof; our collaborations with partners, including the pace and progress of development by our collaboration partners, the receipt of anticipated milestone payments therefrom, any potential future collaborations; and our financial position and cash runway.

    The process by which investigational therapies, such as nedosiran and belcesiran, could potentially lead to an approved product is long and subject to highly significant risks. Applicable risks and uncertainties include those relating to Dicerna's clinical research and other risks identified under the heading "Risk Factors" included in the Company's most recent filings on Forms 10-K and 10-Q and in other future filings with the Securities and Exchange Commission. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners; the likelihood of Dicerna's clinical programs being executed on timelines provided; reliance on the Company's contract research organizations and predictability of timely enrollment of subjects and patients to advance Dicerna's clinical trials; the reliance of Dicerna on contract manufacturers to supply its products for research, development and commercialization and the risk of supply interruption from any contract manufacturer; the potential for future data to alter initial and preliminary results of preclinical studies, models and earlier-stage clinical trials; the impact of the ongoing COVID-19 pandemic and its variants on our business operations, including the conduct of our research and development activities; the regulatory review and unpredictability of the duration and results of the regulatory review of Investigational New Drug (IND) applications and Clinical Trial Applications (CTAs) that are necessary to continue to advance and progress the Company's clinical programs; the timing, plans and reviews by regulatory authorities of marketing applications such as NDAs and comparable foreign applications for one or more of Dicerna's product candidates, including for nedosiran; alignment with the FDA on the regulatory pathway to approval for our product candidates, including nedosiran; the ability to secure out-licensing opportunities to commercialize nedosiran, if approved, in the U.S. and abroad on acceptable terms, if at all; the ability to secure, maintain and realize the intended benefits of collaborations with partners; market acceptance for approved products and innovative therapeutic treatments; competition; the possible impairment of, inability to obtain and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in R&D and following commercialization; changes in our current clinical and operating plan; and general business, financial and accounting risks and litigation. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.

    GalXC™, GalXC-Plus™ and PHYOX™ are trademarks of Dicerna Pharmaceuticals, Inc.

    (tables follow)

     

    DICERNA PHARMACEUTICALS, INC.

    SELECTED FINANCIAL INFORMATION (UNAUDITED)

     

    CONDENSED CONSOLIDATED BALANCE SHEETS

     

    June 30,

    2021

     

    December 31,

    2020

    (In thousands)

     

     

    Cash and cash equivalents

     

    $

    221,210

     

     

    $

    126,023

     

    Held-to-maturity investments

     

    488,354

     

     

    442,820

     

    Restricted cash equivalents

     

    5,618

     

     

    6,362

     

    Contract receivables

     

    3,147

     

     

    34,713

     

    Prepaid expenses and other current assets

     

    20,380

     

     

    14,403

     

    Property and equipment, net

     

    23,593

     

     

    17,546

     

    Right-of-use operating assets, net

     

    74,400

     

     

    60,843

     

    Other noncurrent assets

     

    1,790

     

     

    5,136

     

    Total Assets

     

    $

    838,492

     

     

    $

    707,846

     

    Accounts payable

     

    $

    12,829

     

     

    $

    7,901

     

    Accrued expenses and other current liabilities

     

    33,523

     

     

    31,500

     

    Deferred revenue, current

     

    160,200

     

     

    138,537

     

    Deferred revenue, noncurrent

     

    268,606

     

     

    336,236

     

    Deferred income

     

    179,806

     

     

     

    Other noncurrent liabilities

     

    72,454

     

     

    55,918

     

    Total stockholders' equity

     

    111,074

     

     

    137,754

     

    Total Liabilities and Stockholders' Equity

     

    $

    838,492

     

     

    $

    707,846

     

     

     

     

     

     

     

     

    Common stock outstanding

     

    77,602

     

     

    75,757

     

     

     

    Three Months Ended

    June 30, 2021

     

    Three Months Ended

    June 30, 2020

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

     

     

    (In thousands, except per share data)

     

     

    Revenue

     

    $

    41,337

     

     

    $

    40,448

     

    Operating expenses:

     

     

     

     

    Research and development

     

    56,119

     

     

    53,376

     

    General and administrative

     

    25,462

     

     

    20,565

     

    Total operating expenses

     

    81,581

     

     

    73,941

     

    Loss from operations

     

    (40,244)

     

     

    (33,493)

     

    Other income (expense):

     

     

     

     

    Interest income, net

     

    107

     

     

    1,723

     

    Other expense

     

    (132)

     

     

    (50)

     

    Total other (expense) income

     

    (25)

     

     

    1,673

     

    Loss before income taxes

     

     

    (40,269)

     

     

     

    (31,820)

     

    Provision for income taxes

     

     

    (546)

     

     

     

     

    Net loss

     

    $

    (40,815)

     

     

    $

    (31,820)

     

    Net loss per share – basic and diluted

     

    $

    (0.53)

     

     

    $

    (0.43)

     

    Weighted average common shares outstanding

    – basic and diluted

     

    77,030

     

     

    74,001

     

     

    View Full Article Hide Full Article
  3. – Nedosiran Achieved Primary Endpoint, Demonstrating Statistically and Clinically Significant Sustained Reduction in Urinary Oxalate Excretion; Key Secondary Endpoint Also Achieved; Robust Efficacy Seen in PH1 Participants –

    – Nedosiran Was Generally Well Tolerated in PHYOX2 With a Safety Profile Similar to Previously Reported PHYOX Trial Results –

    – Results Further Validate GalXC™ RNAi Technology Platform and its Ability to Silence Disease-Driving Genes –

    – Management to Host Conference Call and Webcast Today at 4:30 p.m. ET –

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced positive top-line results from the…

    – Nedosiran Achieved Primary Endpoint, Demonstrating Statistically and Clinically Significant Sustained Reduction in Urinary Oxalate Excretion; Key Secondary Endpoint Also Achieved; Robust Efficacy Seen in PH1 Participants –

    – Nedosiran Was Generally Well Tolerated in PHYOX2 With a Safety Profile Similar to Previously Reported PHYOX Trial Results –

    – Results Further Validate GalXC™ RNAi Technology Platform and its Ability to Silence Disease-Driving Genes –

    – Management to Host Conference Call and Webcast Today at 4:30 p.m. ET –

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced positive top-line results from the Company's PHYOX™2 pivotal clinical trial of nedosiran, which is in development as a once-monthly treatment for primary hyperoxaluria (PH), a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys. Nedosiran is Dicerna's lead GalXC™ RNAi therapeutic candidate and is designed to inhibit the hepatic lactate dehydrogenase (LDH) enzyme – an enzyme that catalyzes the final step in the glyoxylate metabolism pathway that can lead to oxalate overproduction in patients with PH. The PHYOX2 clinical trial included participants with PH subtypes 1 and 2 (PH1 and PH2).

    Nedosiran achieved the primary endpoint in the PHYOX2 trial, demonstrating a statistically significant reduction from baseline in urinary oxalate (Uox) excretion compared to placebo (p<0.0001). The study also achieved the key secondary endpoint, with a significantly higher proportion of patients given nedosiran achieving and sustaining normal or near-normal Uox at two or more consecutive visits after Day 90 compared to placebo (p=0.0025). Uox reductions were significant in participants with PH1 while participants with PH2 (5 nedosiran and 1 placebo) showed inconsistent results in this trial. Nedosiran was generally well tolerated in the study with an overall adverse event (AE) profile consistent with previously reported data from PHYOX trials.

    "We believe the reduction in Uox excretion seen in patients with PH1 showed that nedosiran knocks down LDHA in the liver and reconfirms the ability of Dicerna's GalXC RNAi technology to silence disease-driving genes, de-risking our growing pipeline of GalXC product candidates," said Shreeram Aradhye, M.D., Executive Vice President and Chief Medical Officer at Dicerna. "The heterogeneity of Uox response seen in participants with PH2, despite LDHA inhibition in the liver and in contrast to prior clinical experience, suggests more complexity in the PH2 disease biology than has been previously understood and will require further evaluation.

    "The results reinforce nedosiran's potential to be a therapeutic option for patients with PH1, if approved," Dr. Aradhye continued. "Our New Drug Application to the U.S. Food and Drug Administration, expected to be submitted in the fourth quarter of 2021, will reflect the results reported today and a strategy to pursue approval of nedosiran for the treatment of PH1 for the near-term. We are extremely grateful to the patients, caregivers, physicians and healthcare professionals who participated in our trial and look forward to continuing to work with the PH community."

    PHYOX2 Top-Line Results and PHYOX Development Program

    PHYOX2 (NCT03847909), a placebo-controlled, double-blind, multicenter, pivotal study, was designed to evaluate the efficacy, safety and tolerability of nedosiran over six months in participants aged six years and older across 11 countries, including the U.S., Japan and Europe, who have PH1 or PH2. Participants were randomized 2:1 to a fixed monthly dose of nedosiran or placebo administered once monthly by subcutaneous injection. Of the 35 patients randomized (23 nedosiran and 12 placebo; 29 with PH1 and 6 with PH2), 34 participants had at least one efficacy assessment after Day 90 (modified intent-to-treat population; mITT). Baseline mean estimated glomerular filtration rate (eGFR; a measure of kidney function) was 89.5 mL/min/1.73 m2 (SD=37.5) for participants given nedosiran and 82.0 mL/min/1.73 m2 (SD=30.0) for participants given placebo. Baseline mean Uox values were approximately 1.33 mmol/day (SD=0.47) and 1.96 mmol/day (SD=0.71) for the nedosiran and placebo groups, respectively.

    The primary endpoint of the study was the percent change from baseline in 24-hour urinary oxalate excretion, as assessed by area under the curve (AUC) from Day 90 to Day 180. The primary endpoint of the study was met, with nedosiran resulting in a statistically significant reduction in Uox (p<0.0001). In the overall trial, nedosiran resulted in a 57.5% greater daily average reduction over Day 90 to Day 180 compared to placebo.

    The key secondary endpoint was percentage of patients (PH1 and PH2) achieving normalization (defined as Uox level below 0.46 mmol adjusted per 1.73 m2 body surface area in participants younger than 18 years when collected over 24 hours) or near-normalization (defined as 1.3 times the upper limit of normal) on at least two consecutive visits from Day 90 to Day 180. Nedosiran achieved statistically significant results (p=0.0025) in the study, with 50% of nedosiran-treated patients reaching normal or near-normal Uox on at least two consecutive visits, compared to 0% for those receiving placebo.

    Nedosiran was generally well tolerated in this study with an AE profile consistent with that observed in the PHYOX1 Phase 1 study and from previous interim analyses from the ongoing PHYOX3 open-label trial. The most common AEs in the trial were mild, self-resolving injection-site reactions (2 patients given nedosiran and zero given placebo). There were three reported kidney stone AEs in participants given nedosiran (13%) and five in participants given placebo (42%). Of the 35 participants enrolled in the trial, two discontinued, with one withdrawing from the study due to declining renal function (a participant who was receiving placebo) and one discontinuing due to self-resolving, benign palpitations considered to be unrelated to study drug by external experts.

    Additional analyses of the Uox data for nedosiran-treated patients with PH1 demonstrated:

    • 59% greater reduction from baseline in 24-hour Uox averaged over Day 90 to Day 180 for nedosiran compared to placebo;
    • 68% (SD=14.6) mean maximum percent reduction in 24-hour Uox from baseline with nedosiran treatment at any time point;
    • 81% of participants achieved normal or near-normal Uox at Day 180;
    • 44% of participants had normal Uox at Day 180; and
    • 65% of participants had normal Uox on one or more visits during the 180-day period.

    We expect the results from the PHYOX2 trial to support marketing authorization applications in the U.S. and other major markets.

    Dicerna intends to present full results from PHYOX2 at an upcoming medical congress, subject to abstract acceptance.

    PHYOX2 is part of the broader PHYOX clinical trial program designed to evaluate nedosiran in participants with PH1, PH2 and PH3. Data from PHYOX1, a single-dose Phase 1 trial in healthy volunteers and participants with PH1 or PH2; PHYOX2; PHYOX4, a single-dose safety and tolerability study in participants with PH3; and the ongoing PHYOX3 open-label extension study, are expected to support the nedosiran New Drug Application (NDA) submission, which is planned for the fourth quarter of 2021.

    Conference Call and Webcast

    Management will host a conference call and webcast at 4:30 p.m. ET today to discuss the PHYOX2 top-line results. The conference call can be accessed by dialing (855) 453-3834 or +1 (484) 756-4306 (international) and referencing conference ID 2845518 prior to the start of the call. A webcast presentation will also be available under the "Investors & Media" section of the Dicerna website, www.dicerna.com. A replay of the call will be available approximately two hours after the completion of the call. To access the replay, please dial (855) 859-2056 or +1 (404) 537-3406 and refer to conference ID 2845518. The webcast will also be archived on Dicerna's website.

    About Primary Hyperoxaluria (PH)

    Primary hyperoxaluria (PH) is a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys. There are three known subtypes of PH (PH1, PH2 and PH3), each resulting from a mutation in one of three different genes. These genetic mutations cause enzyme deficiencies that result in the overproduction of an end-product of metabolism called oxalate. Abnormal production and accumulation of oxalate leads to recurrent kidney stones, nephrocalcinosis and chronic kidney disease that may progress to end-stage renal disease requiring intensive dialysis. Compromised renal function eventually results in the accumulation of oxalate in a wide range of organs including the skin, bones, eyes and heart. In the most severe cases, symptoms start in the first year of life. A combined liver-kidney transplant may be undertaken to resolve PH1 or PH2, but it is an invasive solution with limited availability and high morbidity that requires lifelong immune suppression to prevent organ rejection. Genetic studies suggest approximately 8,500 people in the U.S. are affected by PH, and researchers estimate that more than 80% of patients remain undiagnosed.1 There is currently only one approved therapy available that is limited to the treatment of patients with PH1.

    About Nedosiran

    Nedosiran is the only RNAi drug candidate in development for primary hyperoxaluria (PH) types 1, 2 and 3 and is Dicerna's most advanced product candidate utilizing the proprietary GalXC™ RNAi technology platform. Nedosiran is designed to inhibit production of the hepatic lactate dehydrogenase (LDH) enzyme – an enzyme that catalyzes the final step in the glyoxylate metabolism pathway that can lead to oxalate overproduction in patients with PH1, PH2 or PH3. Dicerna is evaluating the safety and efficacy of nedosiran in patients with all known subtypes of PH as part of its PHYOX™ clinical development program.

    About Dicerna Pharmaceuticals, Inc.

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) is a biopharmaceutical company focused on discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to silence selectively genes that cause or contribute to disease. Using our proprietary GalXC™ and GalXC-Plus™ RNAi technologies, Dicerna is committed to developing RNAi-based therapies with the potential to treat both rare and more prevalent diseases. By silencing disease-causing genes, Dicerna's GalXC platform has the potential to address conditions that are difficult to treat with other modalities. Initially focused on disease-causing genes in the liver, Dicerna has continued to innovate and is exploring new applications of its RNAi technology with GalXC-Plus, which expands on the functionality and application of our flagship liver-targeted GalXC technology to tissues and cell types outside the liver, and has the potential to treat diseases across multiple therapeutic areas. In addition to our own pipeline of core discovery and clinical candidates, Dicerna has established collaborative relationships with some of the world's leading pharmaceutical companies, including Novo Nordisk A/S, Roche, Eli Lilly and Company, Alexion Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH and Alnylam Pharmaceuticals, Inc. Between Dicerna and our collaborative partners, we currently have more than 20 active discovery, preclinical or clinical programs focused on cardiometabolic, viral, chronic liver and complement-mediated diseases, as well as neurodegenerative diseases and pain. At Dicerna, our mission is to interfere – to silence genes, to fight disease, to restore health. For more information, please visit www.dicerna.com.

    Cautionary Note on Forward-Looking Statements

    This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements we make regarding our product candidates and the development thereof, such as the Company's PHYOX clinical development program and potential impact of the results from the PHYOX2 trial of nedosiran as well as from the broader PHYOX clinical development program; the therapeutic potential of our product candidates, such as nedosiran; the Company's regulatory plans and timelines for nedosiran, including our planned submission of a New Drug Application (NDA) for nedosiran and the indication(s) expected to be pursued in the near term; our business and operations, including the discovery, development and commercialization of our product candidates and technology platform, and the therapeutic potential thereof; our collaboration with partners and any potential future collaborations. The process by which investigational therapies, such as nedosiran, could potentially lead to an approved product is long and subject to highly significant risks. Applicable risks and uncertainties include those relating to Dicerna's clinical research and other risks identified under the heading "Risk Factors" included in the Company's most recent filings on Forms 10-K and 10-Q and in other future filings with the Securities and Exchange Commission. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners; the likelihood of Dicerna's clinical programs being executed on timelines provided; our reliance on the Company's contract research organizations; the predictability of timely enrollment of subjects and patients to advance Dicerna's clinical trials; positive data from preclinical studies and earlier clinical trials may not be predictive of results from subsequent preclinical studies and clinical trials; the reliance of Dicerna on contract manufacturers to supply its products for research, development and commercialization and the risk of supply interruption from one or more such contract manufacturers; the potential for additional or future data to alter initial, interim and preliminary results of clinical trials; the results of clinical trials may produce negative, inconclusive or uncompetitive results; the impact of the ongoing COVID-19 pandemic on our business operations, including the conduct of our research and development activities; the regulatory review and unpredictability of the duration and results of the regulatory review of Investigational New Drug applications (INDs) and Clinical Trial Applications (CTAs) that are necessary to continue to advance and progress the Company's clinical programs; the timing, plans and reviews by regulatory authorities of marketing applications such as NDAs and comparable foreign applications for one or more of Dicerna's product candidates; alignment with the FDA on the regulatory pathway to approval for nedosiran; the ability to secure, maintain and realize the intended benefits of collaborations with partners; market acceptance for approved products and innovative therapeutic treatments; competition; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in R&D and following commercialization; and general business, financial, and accounting risks and litigation. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.

    GalXC™, GalXC-Plus™ and PHYOX™ are trademarks of Dicerna Pharmaceuticals, Inc.

    1 Hopp K, et al. J Am Soc Nephrol. 2015;26(10):2559-2570 and U.S. Census Bureau population on a date: February 20, 2020. United States Census Bureau website, 2020.

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  4. Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the Company will release its second quarter 2021 financial results before market open on Monday, Aug. 9, 2021.

    Management will host a conference call at 8:00 a.m. ET that day to discuss the Company's financial results and provide a general business update. The conference call will be webcast live and will be available from the "Investors & Media" section of the Dicerna website, www.dicerna.com. The webcast will also be archived on the Company's website.

    The conference call can be accessed by dialing (855) 453-3834 or +1 (484) 756-4306 (international) and referencing…

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the Company will release its second quarter 2021 financial results before market open on Monday, Aug. 9, 2021.

    Management will host a conference call at 8:00 a.m. ET that day to discuss the Company's financial results and provide a general business update. The conference call will be webcast live and will be available from the "Investors & Media" section of the Dicerna website, www.dicerna.com. The webcast will also be archived on the Company's website.

    The conference call can be accessed by dialing (855) 453-3834 or +1 (484) 756-4306 (international) and referencing conference ID 5418088 prior to the start of the call. A replay of the call will be available approximately two hours after the completion of the call and will remain available for seven days. To access the replay, please dial (855) 859-2056 or +1 (404) 537-3406 and refer to conference ID 5418088.

    About Dicerna Pharmaceuticals, Inc.

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) is a biopharmaceutical company focused on discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to silence selectively genes that cause or contribute to disease. Using our proprietary GalXC™ and GalXC-Plus™ RNAi technologies, Dicerna is committed to developing RNAi-based therapies with the potential to treat both rare and more prevalent diseases. By silencing disease-causing genes, Dicerna's GalXC platform has the potential to address conditions that are difficult to treat with other modalities. Initially focused on disease-causing genes in the liver, Dicerna has continued to innovate and is exploring new applications of its RNAi technology with GalXC-Plus, which expands on the functionality and application of our flagship liver-targeted GalXC technology to tissues and cell types outside the liver, and has the potential to treat diseases across multiple therapeutic areas. In addition to our own pipeline of core discovery and clinical candidates, Dicerna has established collaborative relationships with some of the world's leading pharmaceutical companies, including Novo Nordisk A/S, Roche, Eli Lilly and Company, Alexion Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH and Alnylam Pharmaceuticals, Inc. Between Dicerna and our collaborative partners, we currently have more than 20 active discovery, preclinical or clinical programs focused on cardiometabolic, viral, chronic liver and complement-mediated diseases, as well as neurodegenerative diseases and pain. At Dicerna, our mission is to interfere – to silence genes, to fight disease, to restore health. For more information, please visit www.dicerna.com.

    GalXC™ and GalXC-Plus™ are trademarks of Dicerna Pharmaceuticals, Inc.

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  5. – Novel ALDH2-Targeting GalXC™ RNAi Candidate Designed to Address a Highly Prevalent and Undertreated Disorder1

    – Dicerna Expects to Initiate Phase 1 Clinical Trial of DCR-AUD in the Third Quarter of 2021 –

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) ("Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced the U.S. Food and Drug Administration ("FDA") clearance of its Investigational New Drug (IND) application for DCR-AUD, the Company's investigational RNAi candidate for the treatment of alcohol use disorder (AUD).

    AUD is a medical condition characterized by the inability to stop or control alcohol use despite social, occupational or health consequences.2 Aldehyde…

    – Novel ALDH2-Targeting GalXC™ RNAi Candidate Designed to Address a Highly Prevalent and Undertreated Disorder1

    – Dicerna Expects to Initiate Phase 1 Clinical Trial of DCR-AUD in the Third Quarter of 2021 –

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) ("Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced the U.S. Food and Drug Administration ("FDA") clearance of its Investigational New Drug (IND) application for DCR-AUD, the Company's investigational RNAi candidate for the treatment of alcohol use disorder (AUD).

    AUD is a medical condition characterized by the inability to stop or control alcohol use despite social, occupational or health consequences.2 Aldehyde dehydrogenase 2, or ALDH2, is an enzyme that plays a key role in metabolizing alcohol. DCR-AUD has been shown to induce long-lasting liver-specific ALDH2 messenger RNA (mRNA) knockdown in nonclinical studies. By silencing ALDH2 in the liver and interrupting the alcohol metabolic pathway, DCR-AUD has the potential to induce real-time physiological feedback to help individuals seeking treatment for AUD regain control over harmful levels of alcohol use.

    "The key to developing a successful treatment for the more than 14 million people affected by AUD is finding a way to help individuals regain control over their alcohol use," said Bob D. Brown, Ph.D., Chief Scientific Officer and Executive Vice President of R&D at Dicerna. "We believe that RNAi may be ideally suited to do this. RNAi's long duration of effect and target specificity can achieve liver-specific reduction of ALDH2, which could be an effective strategy to reduce alcohol consumption. We've performed extensive screening and optimization of GalXC™ RNAi molecules targeting human ALDH2 to identify DCR-AUD and believe DCR-AUD has the potential to improve health outcomes for those with AUD."

    Dr. Brown continued, "We would also like to acknowledge the financial support we received from the National Institutes of Health in the form of a cooperative Small Business Innovation Research grant that has contributed to the development of DCR-AUD. The grant reviewers and the agency's votes of confidence in Dicerna's drug development capabilities and the liver-specific RNAi mechanism of action of GalXC have helped make our DCR-AUD program possible."

    Dicerna plans to initiate a 24-week, randomized, double-blind, placebo-controlled Phase 1 trial in the third quarter of 2021 to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics of single-ascending doses of DCR-AUD in healthy volunteers. The trial will also assess the interaction between DCR-AUD treatment and alcohol consumption using standardized Ethanol Interaction Assessments.

    About Alcohol Use Disorder (AUD)

    Alcohol use disorder, or AUD, is a chronic disorder characterized by the inability to stop or control alcohol use despite social, occupational or health consequences. AUD presents as a problematic pattern of alcohol use leading to clinically significant impairment or distress. Symptoms can include compulsive drinking, loss of control over alcohol use and negative emotions when not drinking.2 AUD is one of the most common psychiatric disorders, affecting more than 14 million adults in the U.S. annually, and it is one of the leading causes of preventable death. Globally, AUD affects approximately 283 million people, according to the World Health Organization.3 AUD is often undiagnosed and untreated. Of the estimated 14 million individuals in the U.S. with AUD, fewer than 1.4 million received AUD treatment of any kind, including psychosocial support, and only a fraction of these 1.4 million received medication to treat this disorder.1,4

    About DCR-AUD

    DCR-AUD is Dicerna's GalXC™ RNAi investigational candidate designed to silence ALDH2 (aldehyde dehydrogenase 2) messenger RNA (mRNA) expression in the liver. DCR-AUD has been shown to induce long-lasting liver-specific ALDH2 mRNA knockdown in nonclinical studies. Some individuals are born with naturally occurring mutations in one or both gene copies that encode the ALDH2 enzyme. In these people with ALDH2 mutations, alcohol consumption can result in uncomfortable physiological effects that occur soon after drinking. These effects are thought to be the reason people with ALDH2 mutations are much less likely to be affected by AUD. Dicerna designed DCR-AUD based on human genetic data that suggest knocking down ALDH2 mRNA in individuals with AUD may provide similar physiological feedback that is protective against harmful levels of alcohol consumption. Preclinical research for DCR-AUD was supported by a grant from the National Institute on Alcohol Abuse and Alcoholism of the National Institutes of Health (NIH), under Award Number U44AA027404.

    About RNAi and Dicerna's GalXC™ RNAi Platform Technologies

    Ribonucleic acid interference, or RNAi, provides a unique advantage to other disease inhibitor technologies, like small-molecule pharmaceuticals or monoclonal antibodies. Instead of targeting proteins after they have been produced and released, RNAi silences the genes themselves via the specific destruction of the messenger RNA (mRNA) made from the gene. Rather than seeking to inhibit a protein, the RNAi approach can prevent a disease-causing protein's creation, directly impacting disease manifestation.

    Dicerna's proprietary GalXC™ RNAi platform aims to advance the development of next-generation RNAi-based therapies. Investigational therapeutics developed using our flagship GalXC technology utilize a proprietary N-acetyl-D-galactosamine (GalNAc)-mediated structure of double-stranded RNA molecules that are designed to bind specifically to receptors on liver cells, leading to selective hepatocyte internalization and access to the RNAi machinery within the cells. Dicerna is continuously innovating and exploring new applications of RNAi technology beyond GalNAc-mediated delivery to the liver, including alternative RNA structures and fully synthetic ligands that target other tissues and enable new therapeutic applications, referred to as GalXC-Plus™.

    About Dicerna Pharmaceuticals, Inc.

    Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) is a biopharmaceutical company focused on discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to silence selectively genes that cause or contribute to disease. Using our proprietary GalXC™ and GalXC-Plus™ RNAi technologies, Dicerna is committed to developing RNAi-based therapies with the potential to treat both rare and more prevalent diseases. By silencing disease-causing genes, Dicerna's GalXC platform has the potential to address conditions that are difficult to treat with other modalities. Initially focused on disease-causing genes in the liver, Dicerna has continued to innovate and is exploring new applications of its RNAi technology with GalXC-Plus, which expands the functionality and application of our flagship liver-targeted GalXC technology to tissues and cell types outside the liver, and has the potential to treat diseases across multiple therapeutic areas. In addition to our own pipeline of core discovery and clinical candidates, Dicerna has established collaborative relationships with some of the world's leading pharmaceutical companies, including Novo Nordisk A/S, Roche, Eli Lilly and Company, Alexion Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH and Alnylam Pharmaceuticals, Inc. Between Dicerna and our collaborative partners, we currently have more than 20 active discovery, preclinical or clinical programs focused on cardiometabolic, viral, chronic liver and complement-mediated diseases, as well as neurodegenerative diseases and pain. At Dicerna, our mission is to interfere – to silence genes, to fight disease, to restore health. For more information, visit www.dicerna.com.

    Cautionary Note on Forward-Looking Statements

    This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements we make regarding the potential of DCR-AUD for the treatment of alcohol use disorder; market size for people affected by AUD; and timing of initiating a Phase 1 clinical trial of DCR-AUD in healthy volunteers. The process by which investigational therapies could potentially lead to an approved product is long and subject to highly significant risks. Applicable risks and uncertainties include those relating to Dicerna's clinical research and other risks identified under the heading "Risk Factors" included in the Company's most recent filings on Forms 10-K and 10-Q and in other future filings with the Securities and Exchange Commission. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners; the likelihood of Dicerna's clinical programs being executed on timelines provided and reliance on the Company's contract research organizations and predictability of timely enrollment of subjects and patients to advance Dicerna's clinical trials; the reliance of Dicerna on contract manufacturers to supply its products for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the impact of the ongoing COVID-19 pandemic on our business operations, including the conduct of our research and development activities; the unpredictability of the duration and results of the regulatory review of Investigational New Drug applications (INDs) and Clinical Trial Applications (CTAs) that are necessary to continue to advance and progress the Company's clinical programs and the regulatory review of INDs and CTAs; the timing, plans and reviews by regulatory authorities of marketing applications such as New Drug Applications (NDAs) and comparable foreign applications for one or more of Dicerna's product candidates; the ability to secure, maintain and realize the intended benefits of collaborations with partners; market acceptance for approved products and innovative therapeutic treatments; competition; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in R&D; and general business, financial, and accounting risks and litigation. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.

    1. Substance Abuse and Mental Health Services Administration. Results from the 2019 National Survey on Drug Use and Health. Accessed on July 28, 2021.

    2. National Institute on Alcohol Abuse and Alcoholism. Understanding Alcohol Use Disorder. Alcohol Facts and Statistics. Accessed July 28, 2021.

    3.World Health Organization. Global Status Report on Alcohol and Health, 2018. Accessed July 27, 2021.

    4.Grant et al., JAMA Psychiatry 2015.

    GalXC™ and GalXC-Plus™ are trademarks of Dicerna Pharmaceuticals, Inc.

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