November 27, 2022

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Dicerna Announces Next Quarter 2021 Monetary Final results and Provides a Small business Update

– Noted Constructive Major-Line Data From Pivotal PHYOX2 Scientific Trial of Nedosiran Investigational GalXC RNAi Treatment for Therapy of Most important Hyperoxaluria –

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

– Announced Interim Stage 1 Effects and Initiated Client Dosing in ESTRELLA Period 2 Research of Belcesiran for the Procedure of Alpha-1 Antitrypsin Deficiency-Linked Liver Ailment (AATLD) –

– Obtained $10. Million Milestone Payment Adhering to Fda Acceptance of Lilly’s Second GalXC RNAi IND Software Beneath Companies’ International Investigation Collaboration and Licensing Arrangement –

– Organization Reported $709.6 Million in Dollars, Income Equivalents and Held-to-Maturity Investments as of June 30, 2021 –

– Management to Host Conference Connect with These days at 8:00 a.m. ET –

LEXINGTON, Mass., August 09, 2021–(Business WIRE)–Dicerna Prescribed drugs, Inc. (Nasdaq: DRNA) (the “Firm” or “Dicerna”), a main developer of investigational ribonucleic acid interference (RNAi) therapeutics, nowadays noted its economical final results for the quarter finished June 30, 2021 and delivered a company update.

“We have noticed sizeable development across our main and collaborative pipelines more than the earlier couple of months, which include the accomplishment of a big milestone in our maturation as a corporation,” stated Douglas Fambrough, Ph.D., President and Chief Executive Officer at Dicerna. “Particularly, our not too long ago declared PHYOX™2 results symbolize our initially optimistic pivotal trial readout. PHYOX2 generated robust data, assembly the most important and vital secondary efficacy endpoints, and nedosiran was commonly well tolerated in the demo. Centered on these effects, we think nedosiran has important prospective as a procedure for clients with PH1, and we are on program to post a New Drug Application to the Fda for the treatment method of PH1 in the fourth quarter. Notably, these optimistic effects mirror broadly on our GalXC™ RNAi system and bode nicely for our additional proprietary and partnered courses.

“As positive as these effects are for PH1, the inconsistent data noticed specially in contributors with PH2 have led us to make the strategic final decision not to transfer forward with our system to construct Dicerna into a absolutely built-in business enterprise to help nedosiran,” Dr. Fambrough ongoing. “Instead, we intend to pursue commercial out-licensing alternatives to support ensure world accessibility to nedosiran, subject matter to essential approvals. This tactic will allow us to deploy our cash and talent on our discovery and growth pipeline endeavours with our GalXC and GalXC-Plus™ RNAi investigational therapeutics for ourselves and our associates. With these strategic changes centered on our core strengths, we can lengthen our income runway into 2025.”

The latest Updates

  • Good Top rated-Line Info From Pivotal PHYOX2 Clinical Demo of Nedosiran for Main Hyperoxaluria (PH). Dicerna announced in August good best-line effects from the pivotal PHYOX2 scientific trial of nedosiran. Nedosiran achieved the key endpoint in the PHYOX2 trial, demonstrating a statistically significant reduction from baseline in urinary oxalate (Uox) excretion as opposed 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

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 source version on businesswire.com: https://www.businesswire.com/news/home/20210809005221/en/

Contacts

Media:
Amy Trevvett
+1 617-612-6253
[email protected]

Investors:
Janhavi Mohite
+1 212-362-1200
[email protected]