Primary Hyperoxaluria Type 2

Talicia® - World Gastro 2022 congress (August 17-18): RedHill invited to give prestigious oral presentation of important data detailing high eradication rates across body mass index (BMI) groups with Talicia, the U.S.'s leading brand for Helicobacter pylori (H. pylori) eradication treatment - invitation sent to researchers with significant recently published clinical findings -- Opaganib for COVID-19 – Suggested host-directed mechanism of action described in Drug Design, Development and Therapy journal : Multi-faceted potential to: Inhibit spike protein-ACE2 binding, Akt signaling and endocytosis, induce autophagy and apoptosis, and disrupt the viral replication-transcription complex (RTC) through simultaneous inhibition of three sphingolipid-metabolizing enzymes in human cells (SK2, DES1 and GCS) - supports hypothesis of broad antiviral effect and attenuation of multi-organ dysfunction in COVID-19 patients -- Opaganib for COVID-19 - 2nd ARDS Drug Development Summit (July 13-15): Presenting new COVID-19 biomarker methodology utilizing baseline Fraction of Inspired Oxygen (FiO2) as a new disease severity classification paradigm - Phase 2/3 data demonstrating a 62% reduction in mortality in opaganib-treated patients requiring FiO2 up to 60% to be presented -- Opaganib and RHB-107 (upamostat) for COVID-19 - International Conference on Emerging Infectious Diseases (ICEID, August 7-10): Presenting promising efficacy and safety data for RedHill's novel, oral, variant-agnostic investigational COVID-19 therapies, at ICEID, the premier infectious disease conference hosted by the CDC and the Global Task Force for Health RALEIGH, N.C. and TEL AVIV, Israel, July 12, 2022 /PRNewswire/ -- RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, announced the upcoming presentation of new Talicia® H. pylori eradication data as well as publication and presentation of data from the opaganib and RHB-107 (upamostat) late clinical-stage COVID-19 programs at important medical congresses in July and August, 2022. Talicia [1]: Publication of a study entitled "Helicobacter pylori Eradication by Low-Dose Rifabutin Triple Therapy (Talicia®) is Unaffected by High Body Mass Index" in the journal GastroHep has been selected by reviewers for oral presentation at the World Gastro 2022 congress, August 17-18. Such invitations to present are reserved for researchers with significant clinical findings published over the previous year. This post hoc analysis of 269 patients from the ERADICATE Hp and ERADICATE Hp2 Phase 3 clinical trials, demonstrated that Talicia is highly effective in eradicating H. pylori irrespective of patient BMI, including in obese and severely obese patients, compared to the active comparator (P<0.0001). Patients with a BMI between 30-40 kg/m2 and those with BMI >40kg/m2 treated with Talicia achieved eradication rates of approximately 90% (88.1% and 90.9% respectively) versus active comparator rates of 62.9% and 31.8% respectively - an approximately 50% lower eradication rate in the severely obese group for the active comparator. Talicia is the leading U.S. branded prescription medicine for H. pylori eradication. Dr. June Almenoff, MD, Ph.D., RedHill's Chief Medical Officer, said: "Because more than 70% of Americans are either overweight or obese[2], and because increased BMI has been linked to reduced eradication outcomes of many commonly used H. pylori therapies, this important work supports Talicia as a rational first line option regardless of patient BMI." RedHill's novel, oral, variant-agnostic late clinical-stage COVID-19 drug candidates, opaganib and RHB-107, have had data selected for publication or presentation as follows: Opaganib [3]: Suggested host-directed mechanism of action described in a manuscript entitled "Recent Progress in the Development of Opaganib for the Treatment of COVID-19" accepted for publication in the journal Drug Design, Development and Therapy: The paper outlines opaganib's multi-faceted potential to: inhibit spike protein-ACE2 binding, Akt signaling and endocytosis, induce autophagy and apoptosis, and disrupt the viral RTC (replication-transcription complex) through simultaneous inhibition of three sphingolipid-metabolizing enzymes in human cells (SK2, DES1 and GCS). These mechanisms support the hypothesis of broad antiviral effect and attenuation of multi-organ dysfunction in COVID-19 patients. Moreover, because of this tripartite targeting, viral resistance to opaganib is unlikely to be encountered through adaptive mutation during therapy or random mutation to generate additional viral variants. 2nd ARDS Drug Development Summit, Boston, July 13-15: Data to be presented from the opaganib Phase 2/3 study showing the potential for a new methodology for selecting COVID-19 patients for treatment with opaganib, based on a new paradigm for classification of disease severity utilizing baseline Fraction of Inspired Oxygen (FiO2), in which opaganib demonstrated a 62% reduction in mortality in patients requiring FiO2 up to 60%. Opaganib and RHB-107 (upamostat) [4]: International Conference on Emerging Infectious Diseases (ICEID), Atlanta, August 7-10: Data to be presented at ICEID, the premier infectious disease conference hosted by the CDC and the Global Task Force for Health, will include: Prespecified analyses from opaganib's Phase 2/3 study (NCT04467840), showing improved viral RNA clearance, faster time to recovery and reduced mortality in key subpopulations of opaganib treated moderate to severe hospitalized patients with COVID-19. Data for RHB-107, from Part A of its two-part Phase 2/3 study in a non-hospitalized setting, includes demonstration of a 100% reduction in hospitalization due to COVID-19 and an approximately 88% reduction in reported new severe COVID-19 symptoms after treatment initiation. Dr. Mark Levitt, RedHill's Chief Scientific Officer said: "Acceptance for publication and presentation of these important data is testament to the quality and strength of RedHill's R&D capability. Oral opaganib and oral RHB-107, both of which have novel host-targeting antiviral mechanisms, have shown effect across multiple variants and virus models, and could serve as important tools in responding to the current and future pandemic waves, whether caused by SARS-COV-2 variants or by other viruses, and of particular concern, as Fall/Winter approach, is the specter of both COVID-19 and influenza circulating in abundance. We are seeing a shift in focus of government funding sources, public health experts, institutions and industry towards looking for broad host-directed antiviral mechanisms of action that will not be subject to resistance by viral mutation and that could address emerging new variants of SARS-CoV-2 and also combat other viruses that might create future pandemic waves - a more sustainable long-term approach than having to rediscover and reinvent very specific antiviral therapeutics which quickly become obsolete in the face of rapidly mutating viruses." About H. pylori infection H. pylori is a bacterial infection that affects approximately 35%[5] of the U.S. population, with an estimated two million patients treated annually[6]. Worldwide, more than 50% of the population has H. pylori infection, which is classified by the WHO as a Group 1 carcinogen. It remains the strongest known risk factor for gastric cancer[7] and a major risk factor for peptic ulcer disease[8] and gastric mucosa-associated lymphoid tissue (MALT) lymphoma[9]. More than 27,000 Americans are diagnosed with gastric cancer annually[10]. Eradication of H. pylori is becoming increasingly difficult, with current therapies failing in approximately 25-40% of patients who remain H. pylori-positive due to high resistance of H. pylori to antibiotics – especially clarithromycin – which is still commonly used in standard combination therapies[11]. About Talicia Talicia is the only low-dose rifabutin-based therapy approved for the treatment of H. pylori infection and is designed to address H. pylori's high resistance to other antibiotics. The high rates of H. pylori resistance to clarithromycin have led to significant rates of treatment failure with clarithromycin-based therapies and are a strong public health concern, as highlighted by the ACG, FDA and the World Health Organization (WHO) in recent years. Talicia is a novel, fixed-dose, all-in-one oral capsule combination of two antibiotics (amoxicillin and rifabutin) and a proton pump inhibitor (PPI) (omeprazole). In November 2019, Talicia was approved by the U.S. FDA for the treatment of H. pylori infection in adults. In the pivotal Phase 3 study, Talicia demonstrated 84% eradication of H. pylori infection in the intent-to-treat (ITT) group vs. 58% in the active comparator arm (p<0.0001). Minimal to zero resistance to rifabutin, a key component of Talicia, was detected in RedHill's pivotal Phase 3 study. Further, in an analysis of data from this study, it was observed that subjects who were confirmed adherent[12] to their therapy had response rates of 90.3% in the Talicia arm vs. 64.7% in the active comparator arm[13]. Talicia is eligible for a total of eight years of U.S. market exclusivity under its Qualified Infectious Disease Product (QIDP) designation and is also covered by U.S. patents which extend patent protection until 2034 with additional patents and applications pending and granted in various territories worldwide. About Opaganib (ABC294640) Opaganib, a new chemical entity, is a proprietary, first-in-class, orally-administered, investigational sphingosine kinase-2 (SK2) selective inhibitor, with suggested dual anti-inflammatory and antiviral activity. Opaganib is host-targeted and, based on data accumulated to date, is expected to maintain effect against emerging viral variants, having already shown in vitro inhibition against variants of concern, including Omicron and Delta. Opaganib has also shown anticancer activity and positive preclinical results in renal fibrosis, and has the potential to target multiple oncology, viral, inflammatory, and gastrointestinal indications. In prespecified analyses of Phase 2/3 clinical data, oral opaganib has demonstrated improved viral RNA clearance, faster time to recovery and significant mortality reduction in key patient subpopulations versus placebo on top of standard of care. Data from the opaganib global Phase 2/3 study has been submitted for peer review and recently published in medRxiv. Opaganib previously delivered promising U.S. Phase 2 data in patients with moderate to severe COVID-19, published in Open Forum Infectious Diseases. Opaganib has also received Orphan Drug designation from the U.S. FDA for the treatment of cholangiocarcinoma and is being evaluated in a Phase 2a study in advanced cholangiocarcinoma and in a Phase 2 study in prostate cancer. Patient accrual, treatment and analysis in the prostate cancer study are ongoing. Opaganib demonstrated potent antiviral activity against SARS-CoV-2, the virus that causes COVID-19, inhibiting viral replication of the original SARS-CoV-2 and variants tested to date in an in vitro model of human lung bronchial tissue. Additionally, preclinical in vivo studies have demonstrated opaganib's potential to decrease renal fibrosis, have shown decreased fatality rates from influenza virus infection, and amelioration of bacteria-induced pneumonia lung injury with reduced levels of IL-6 and TNF-alpha in bronchoalveolar lavage fluids[14]. The ongoing clinical studies with opaganib are registered on , a web-based service by the U.S. National Institute of Health, which provides public access to information on publicly and privately supported clinical studies. About RHB-107 (upamostat) RHB-107 is a proprietary, first-in-class, once-daily orally-administered investigational antiviral, that targets human serine proteases involved in preparing the spike protein for viral entry into target cells. Because it is host-cell targeted, RHB-107 is expected to also be effective against emerging viral variants with mutations in the spike protein. RHB-107 is being evaluated in a Phase 2/3 study for treatment of patients with symptomatic COVID-19 who do not require inpatient care. In addition, RHB-107 inhibits several proteases targeting cancer and inflammatory gastrointestinal disease. RHB-107 has undergone several Phase 1 studies and two Phase 2 studies, demonstrating its clinical safety profile in approximately 200 patients. RedHill acquired the exclusive worldwide rights to RHB-107, excluding China, Hong Kong, Taiwan and Macao, from Germany's Heidelberg Pharmaceuticals (FSE: HPHA) (formerly WILEX AG) for all indications. About RedHill Biopharma RedHill Biopharma Ltd. (Nasdaq: RDHL) is a specialty biopharmaceutical company primarily focused on gastrointestinal and infectious diseases. RedHill promotes the gastrointestinal drugs, Movantik® for opioid-induced constipation in adults[15], Talicia® for the treatment of Helicobacter pylori (H. pylori) infection in adults, and Aemcolo® for the treatment of travelers' diarrhea in adults[16]. RedHill's key clinical late-stage development programs include: (i) RHB-204, with an ongoing Phase 3 study for pulmonary nontuberculous mycobacteria (NTM) disease; (ii) opaganib (ABC294640), a first -in -class oral SK2 selective inhibitor targeting multiple indications with a Phase 2/3 program for hospitalized COVID-19 and Phase 2 studies for prostate cancer and cholangiocarcinoma ongoing; (iii) RHB-107 ( upamostat), an oral serine protease inhibitor in a Phase 3-stage study as treatment for non-hospitalized symptomatic COVID-19, and targeting multiple other cancer and inflammatory gastrointestinal diseases; (iv) RHB-104, with positive results from a first Phase 3 study for Crohn's disease; (v) RHB-102 , with positive results from a Phase 3 study for acute gastroenteritis and gastritis and positive results from a Phase 2 study for IBS-D; and (vi) RHB- 106, an encapsulated bowel preparation. More information about the Company is available at twitter.com/RedHillBio. TALICIA: INDICATION AND IMPORTANT SAFETY INFORMATION Talicia is a three-drug combination of omeprazole, a proton pump inhibitor, amoxicillin, a penicillin-class antibacterial, and rifabutin, a rifamycin antibacterial­­, indicated for the treatment of Helicobacter pylori infection in adults. To reduce the development of drug-resistant bacteria and maintain the effectiveness of Talicia and other antibacterial drugs, Talicia should be used only to treat or prevent infections that are proven or strongly suspected to be caused by bacteria. IMPORTANT SAFETY INFORMATION Talicia contains omeprazole, a proton pump inhibitor (PPI), amoxicillin, a penicillin-class antibacterial and rifabutin, a rifamycin antibacterial. It is contraindicated in patients with known hypersensitivity to any of these medications, any other components of the formulation, any other beta-lactams or any other rifamycin. Talicia is contraindicated in patients receiving rilpivirine-containing products. Talicia is contraindicated in patients receiving delavirdine or voriconazole. Serious and occasionally fatal hypersensitivity reactions have been reported with omeprazole, amoxicillin and rifabutin. Severe cutaneous adverse reactions (SCAR) (e.g., Stevens-Johnson syndrome (SJS), Toxic epidermal necrolysis (TEN)) have been reported with rifabutin, amoxicillin, and omeprazole. Additionally, drug reaction with eosinophilia and systemic symptoms (DRESS) has been reported with rifabutin. Acute Tubulointerstitial Nephritis has been observed in patients taking PPIs and penicillins. Clostridioides difficile-associated diarrhea (CDAD) has been reported with use of nearly all antibacterial agents and may range from mild diarrhea to fatal colitis. Talicia may cause fetal harm. Talicia is not recommended for use in pregnancy. Talicia may reduce the efficacy of hormonal contraceptives. An additional non-hormonal method of contraception is recommended when taking Talicia. Talicia should not be used in patients with hepatic impairment or severe renal impairment. Cutaneous lupus erythematosus (CLE) and systemic lupus erythematosus (SLE) have been reported in patients taking PPIs. These events have occurred as both new onset and exacerbation of existing autoimmune disease. The most common adverse reactions (≥1%) were diarrhea, headache, nausea, abdominal pain, chromaturia, rash, dyspepsia, oropharyngeal pain, vomiting, and vulvovaginal candidiasis. To report SUSPECTED ADVERSE REACTIONS, contact RedHill Biopharma INC. at 1-833-ADRHILL (1-833-237-4455) or FDA at 1-800-FDA-1088 or . Full prescribing information for Talicia is available at This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements may be preceded by the words "intends," "may," "will," "plans," "expects," "anticipates," "projects," "predicts," "estimates," "aims," "believes," "hopes," "potential" or similar words. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company's control and cannot be predicted or quantified, and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties, including without limitation risks regarding the treatment effectiveness of Talicia® and the risk that the Company will not succeed to expand Talicia's reach to additional ex-U.S. territories; as well as other risk and uncertainties associated with (i) the initiation, timing, progress and results of the Company's research, manufacturing, pre-clinical studies, clinical trials, and other therapeutic candidate development efforts, and the timing of the commercial launch of its commercial products and ones it may acquire or develop in the future; (ii) the Company's ability to advance its therapeutic candidates into clinical trials or to successfully complete its pre-clinical studies or clinical trials; (iii) the extent and number and type of additional studies that the Company may be required to conduct and the Company's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings, approvals and feedback; (iv) the manufacturing, clinical development, commercialization, and market acceptance of the Company's therapeutic candidates and Talicia®; (v) the Company's ability to successfully commercialize and promote Talicia®, Aemcolo® and Movantik®; (vi) the Company's ability to establish and maintain corporate collaborations; (vii) the Company's ability to acquire products approved for marketing in the U.S. that achieve commercial success and build its own marketing and commercialization capabilities; (viii) the interpretation of the properties and characteristics of the Company's therapeutic candidates and the results obtained with its therapeutic candidates in research, pre-clinical studies or clinical trials; (ix) the implementation of the Company's business model, strategic plans for its business and therapeutic candidates; (x) the scope of protection the Company is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; (xi) parties from whom the Company licenses its intellectual property defaulting in their obligations to the Company; (xii) estimates of the Company's expenses, future revenues, capital requirements and needs for additional financing; (xiii) the effect of patients suffering adverse experiences using investigative drugs under the Company's Expanded Access Program; and (xiv) competition from other companies and technologies within the Company's industry. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 20-F filed with the SEC on March 17, 2022. All forward-looking statements included in this press release are made only as of the date of this press release. The Company assumes no obligation to update any written or oral forward-looking statement, whether as a result of new information, future events or otherwise unless required by law. Category: R&D [1] Talicia® (omeprazole magnesium, amoxicillin and rifabutin) is approved in the U.S. and indicated for the treatment of H. pylori infection in adults. For full prescribing information see: [2] Adult Obesity Facts. Atlanta, GA: Centers for Disease Control and Prevention (CDC); 2020. . [3] Opaganib is an investigational new drug, not available for commercial distribution. [4] RHB-107 is an investigational new drug, not available for commercial distribution. [5] Hooi JKY et al. Global Prevalence of Helicobacter pylori Infection: Systematic Review and Meta-Analysis. Gastroenterology 2017; 153:420-429. [6] IQVIA Custom Study for RedHill Biopharma, 2019 [7] Lamb A et al. Role of the Helicobacter pylori–Induced inflammatory response in the development of gastric cancer. J Cell Biochem 2013;114.3:491-497. [8] NIH – Helicobacter pylori and Cancer, September 2013. [9] Hu Q et al. Gastric mucosa-associated lymphoid tissue lymphoma and Helicobacter pylori infection: a review of current diagnosis and management. Biomarker research 2016;4.1:15. [10] National Cancer Institute, Surveillance, Epidemiology, and End Results Program (SEER). [11] Malfertheiner P. et al. Management of Helicobacter pylori infection - the Maastricht IV/ Florence Consensus Report, Gut 2012;61:646-664; O'Connor A. et al. Treatment of Helicobacter pylori Infection 2015, Helicobacter 20 (S1) 54-61; Venerito M. et al. Meta-analysis of bismuth quadruple therapy versus clarithromycin triple therapy for empiric primary treatment of Helicobacter pylori infection. Digestion 2013;88(1):33-45. [12] Defined as the PK population which included those subjects in the ITT population who had demonstrated presence of any component of investigational drug at visit 3 (approx. day 13) or had undetected levels drawn >250 hours after the last dose. [13] The pivotal Phase 3 study with Talicia® demonstrated 84% eradication of H. pylori infection with Talicia® vs. 58% in the active comparator arm (ITT analysis, p<0.0001). [14] Xia C. et al. Transient inhibition of sphingosine kinases confers protection to influenza A virus infected mice. Antiviral Res. 2018 Oct; 158:171-177. Ebenezer DL et al. Pseudomonas aeruginosa stimulates nuclear sphingosine-1-phosphate generation and epigenetic regulation of lung inflammatory injury. Thorax. 2019 Jun;74(6):579-591. [15] Movantik® (naloxegol) is indicated for opioid-induced constipation (OIC). Full prescribing information see: . [16] Aemcolo® (rifamycin) is indicated for the treatment of travelers' diarrhea caused by noninvasive strains of Escherichia coli in adults. For full prescribing information see: . Logo - SOURCE RedHill Biopharma Ltd.

Nov. 9, 2021 12:30 UTC – Reported Positive Top-Line Data From Pivotal PHYOX™2 Clinical Trial of Nedosiran Investigational GalXC™ RNAi Therapy for Treatment of Primary Hyperoxaluria (PH) – – Initiated Phase 1 Clinical Trial of DCR-AUD for the Treatment of Alcohol Use Disorder (AUD) – – Company Plans to Unveil First GalXC-Plus™ Extrahepatic Target in Early 2022 – – Cash Runway Extends Into 2025 – – Company to Host Conference Call Today at 8:30 a.m. ET – LEXINGTON, Mass.--(BUSINESS WIRE)-- Dicerna Pharmaceuticals (Nasdaq: DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today reported its financial results for the third quarter ended September 30, 2021 and provided a business update. "We had an exciting and productive third quarter as we continued to execute and advance key initiatives that we believe help position Dicerna for multiple value-creating milestones over the next 12 to 24 months," said Douglas Fambrough, Ph.D., President and Chief Executive Officer at Dicerna. "We look to build on this progress as we continue advancing core programs that include nedosiran for primary hyperoxaluria (PH), RG6346 for chronic hepatitis B virus (HBV) infection with Roche, belcesiran for alpha-1 antitrypsin deficiency-associated liver disease (AATLD) and DCR-AUD for alcohol use disorder (AUD). We also look forward to unveiling the next wave of innovations from Dicerna in early 2022 with the first of several wholly owned extrahepatic programs harnessing our GalXC-Plus technology." Dr. Fambrough continued, "Between Dicerna and our collaborative partners, we have 16 programs in preclinical or clinical development as well as more than 20 discovery-stage programs. Given the breadth of these activities, we expect new programs to be entering the clinic, on average, one per quarter over the next two years and potentially beyond that at a similar rate. We believe this creates the opportunity for Dicerna to dramatically expand in the future and meaningfully advance our mission to improve the lives of patients." Corporate Highlights Announced Positive Top-Line Data From Pivotal PHYOX2 Clinical Trial of Nedosiran for Primary Hyperoxaluria. In August 2021, Dicerna announced positive top-line results from the pivotal PHYOX2 clinical trial of nedosiran, Dicerna’s late-stage investigational GalXC RNAi therapeutic candidate in development for PH. 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 PH type 2 (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) pro with previously reported data from PHYOX trials. Presented PHYOX2 Data as Late-Breaker Poster Presentation at American Society of Nephrology (ASN) Kidney Week 2021. The data from Dicerna's pivotal PHYOX2 study were presented at ASN on November 4, 2021. Reported Top-Line Results for PHYOX4 Single-Dose Study of Nedosiran in Primary Hyperoxaluria Type 3 (PH3). In October 2021, Dicerna announced that its PHYOX4 study designed to evaluate the safety and tolerability of a single subcutaneous dose of nedosiran compared to placebo in patients with PH3 met its primary safety endpoint. Patients administered nedosiran also showed a trend in Uox reduction; however, these reductions did not meet prespecified secondary efficacy endpoint criteria. Initiated Phase 1 Clinical Trial of DCR-AUD for the Treatment of Alcohol Use Disorder. In September 2021, Dicerna announced that it dosed the first subjects in its Phase 1 clinical trial to assess DCR-AUD, Dicerna’s investigational GalXC RNAi therapeutic candidate in development for the treatment of AUD. The randomized, double-blind study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of single ascending doses of DCR-AUD in up to 36 healthy volunteers over a 24-week observation period. The trial is also assessing the interaction between DCR-AUD administration and alcohol consumption using standardized Ethanol Interaction Assessments (EIA) performed serially over the trial’s duration. 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 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) demonstrated 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 pro was generally well tolerated. Dosing in the final 12.0 mg/kg dose cohort in this trial has been completed, and the Company plans to present data from this trial at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting® taking place Nov. 12-15, 2021. Anticipated Upcoming Milestones Nedosiran: Subject to ongoing pre-NDA interactions with the U.S. Food and Drug Administration (FDA), submission of nedosiran New Drug Application (NDA) to FDA for the treatment of PH1 expected in the first quarter of 2022. Belcesiran: Phase 1 data to be presented at AASLD, Nov. 12-15, 2021. DCR-AUD: Interim Phase 1 data expected later in 2022. GalXC-Plus: First Dicerna GalXC-Plus extrahepatic clinical development program expected to be unveiled in early 2022. Financial Results for the Third Quarter Ended September 30, 2021 Cash Position – As of September 30, 2021, Dicerna had $646.6 million in cash, cash equivalents and held-to-maturity investments, compared to $568.8 million as of December 31, 2020. Revenue – Dicerna recognized $63.0 million of revenue for the third quarter 2021, compared to $48.9 million for the same period in 2020. The increase in revenue for the third quarter of 2021 primarily reflects an increase in revenue from the Company's collaboration with Novo Nordisk A/S. Research and Development (R&D) Expenses – R&D expenses were $61.2 million for the third quarter 2021, compared to $54.8 million for the same period in 2020. The increase was primarily due to higher facility-related expenses as well as increased depreciation and other expenses. General and Administrative (G&A) Expenses – G&A expenses were $22.0 million for the third quarter 2021, compared to $17.0 million for the same period in 2020. The increase was primarily due to increased rent expenses and an increase in software costs. Net Loss – Net loss was $17.1 million, or $0.22 per share, for the third quarter 2021, compared to a net loss of $21.8 million, or $0.29 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 R&D 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 $76.5 million has been received in the first nine months of 2021. Conference Call Management will host a conference call at 8:30 a.m. ET today to review Dicerna's third 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 7395414 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, . 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 7395414. 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 . 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 GalXC and GalXC-Plus RNAi technologies and the therapeutic potential thereof, including the advancement of our new, wholly owned GalXC-Plus extrahepatic development programs and timing to announce the first of them; our expected pace and duration of new programs entering into clinical trials and the potential impact thereof; our discovery and product candidates and those of our collaborative partners, including with respect to nedosiran, belcesiran and DCR-AUD, and the development thereof; the pace and progress of and anticipated milestones for the Company’s ongoing and planned trials, including from its Phase 1 clinical trial of DCR-AUD for the treatment of AUD; results and expectations from the Company's ongoing and completed trials, including from the Company’s PHYOX clinical development program; the initiation of trials for product candidates in our pipeline and our beliefs about our pipeline; the filing of Investigational New Drug Applications (INDs) for our product candidates and those of our collaboration partners, including the expected pace thereof; the therapeutic potential of our product candidates, including nedosiran; the planned submission of an NDA for nedosiran and the expected timing thereof, subject to ongoing interactions with the FDA; our commercialization strategy for nedosiran, if approved; our current and potential future collaborations and other strategic arrangements, including the intended benefits thereof, pace and progress of development by such partners and the receipt of anticipated milestone payments therefrom; our strategy, business and operations; and our financial position and expected 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 preclinical and 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 manufacturing organizations 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 process and unpredictability of the duration and results of the regulatory review of IND applications and clinical trial applications 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 for 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 our collaborations and other strategic partners; market acceptance for approved products and innovative therapeutic treatments; competition; the possible impairment of, inability to obtain or secure, and costs to obtain, secure and maintain 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 strategy, business or 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. (tables follow) DICERNA PHARMACEUTICALS, INC. SELECTED FINANCIAL INFORMATION (UNAUDITED) CONDENSED CONSOLIDATED BALANCE SHEETS SEPTEMBER 30, 2021 DECEMBER 31, 2020 (In thousands) Cash and cash equivalents $ 165,756 $ 126,023 Held-to-maturity investments 480,837 442,820 Restricted cash equivalents 5,618 6,362 Contract receivables 6,226 34,713 Prepaid expenses and other current assets 17,268 14,403 Property and equipment, net 22,096 17,546 Right-of-use operating assets, net 72,432 60,843 Other noncurrent assets 1,826 5,136 Total Assets $ 772,059 $ 707,846 Accounts payable $ 8,320 $ 7,901 Accrued expenses and other current liabilities 39,020 31,500 Deferred revenue, current 190,128 138,537 Deferred revenue, noncurrent 180,790 336,236 Deferred income 179,629 — Other noncurrent liabilities 66,509 55,918 Total stockholders’ equity 107,663 137,754 Total Liabilities and Stockholders' Equity $ 772,059 $ 707,846 Common stock outstanding 77,835 75,757 Three Months Ended September 30, 2021 Three Months Ended September 30, 2020 CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In thousands, except per share data) Revenue $ 62,954 $ 48,875 Operating expenses: Research and development 61,232 54,814 General and administrative 21,994 16,961 Total operating expenses 83,226 71,775 Loss from operations (20,272) (22,900) Other income: Interest income, net 124 1,050 Other income, net 3,886 1 Total other income, net 4,010 1,051 Loss before income taxes (16,262) (21,849) Provision for income taxes (810) — Net loss $ (17,072) $ (21,849) Net loss per share – basic and diluted $ (0.22) $ (0.29) Weighted average common shares outstanding – basic and diluted 77,747 74,523

- Presentation to be Webcast - MONTVALE, N.J. ,Nov. 10 /PRNewswire-FirstCall/ -- Synvista Therapeutics, Inc. announced today that Noah Berkowitz, M.D., Ph.D., President and Chief Executive Officer of Synvista will present at the upcoming 10th Annual Investor Forum on Tuesday, November 11, at 2:25 p.m. Eastern Time. The event will be held November 10th - 12th at the New York Palace Hotel in New York City. Dr. Berkowitz will discuss the company's planned commercialization of its haptoglobin test kit for diabetes and ongoing Phase 2 initiatives for alagebrium in heart failure. He will also provide an update of recent milestones. The presentation will be simultaneously webcast and can be viewed live on the investor relations section of the Company's website at Participants are encouraged to log on to the webcast approximately five to ten minutes prior to the start of the presentation. It will be archived there after the event. About Synvista Therapeutics Synvista Therapeutics is a biopharmaceutical company developing diagnostics and drugs to diagnose, treat and prevent cardiovascular disease in people with diabetes. The Company has developed a clinical diagnostic test for Hp2-2 Diabetes, a disease affecting almost 7 million patients in the United States. The genetic or protein form of this test can be used to identify diabetic patients at high risk for cardiovascular complications. These patients may benefit from a particular formulation of Vitamin E. The Company is also developing a kit to measure CML (carboxy-methyllysine), another potential cardiovascular risk marker. Synvista Therapeutics is developing oral antioxidant drugs to treat the HDL dysfunction seen in Hp2-2 Diabetes. The Company is also developing alagebrium, a proposed breaker of advanced glycation endproducts (AGEs) for the treatment of systolic and diastolic heart failure. For more information, please visit the Company's Web site at Any statements contained in this press release that relate to future plans, events or performance are forward-looking statements that involve risks and uncertainties including, but not limited to, the risks associated with the events described in this press release, future clinical development of Synvista Therapeutics' diagnostic tests and product candidates, and other risks identified in Synvista Therapeutics' filings with the Securities and Exchange Commission. Further information on risks faced by Synvista are detailed under the caption "Risk Factors" in Synvista Therapeutics' Annual Report on Form 10-K for the year ended December 31, 2007. These filings are available on a website maintained by the Securities and Exchange Commission at The information contained in this press release is accurate as of the date indicated. Actual results, events or performance may differ materially. Synvista Therapeutics undertakes no obligation to publicly release the result of any revision to these forward- looking statements that may be made to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. CONTACT: Synvista Therapeutics, Inc., +1-201-934-5000, ir@synvista.com; or Investor Relations Contact, Kim Sutton Golodetz of Lippert/Heilshorn & Associates, +1-212-838-3777, kgolodetz@lhai.com Web site: