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Dicerna™ Receives Orphan Drug Designation From European Commission for DCR-A1AT for Treatment of Congenital Alpha-1 Antitrypsin Deficiency

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Dicerna™ Pharmaceuticals, Inc. (NASDAQ:DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the European Commission (EC) has granted orphan drug designation to Dicerna's DCR-A1AT for the treatment of congenital alpha-1 antitrypsin (A1AT) deficiency based on a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). A1AT deficiency is a genetic disorder that can cause liver disease in children and adults, leading to complications such as fatigue, jaundice or unintended weight loss. Patients with A1AT deficiency are also at risk of serious or life-threatening conditions such as cirrhosis, liver failure and hepatocellular carcinoma, and may develop lung disease.1

"We are gratified to see regulators recognize the urgent need for the development of a safe and effective therapy for treatment of alpha-1 antitrypsin deficiency," said Ralf Rosskamp, M.D., chief medical officer of Dicerna. "This orphan drug designation from the European Commission acknowledges the needs of this underserved patient population, and we look forward to continuing to investigate DCR-A1AT's potential in our recently initiated DCR-A1AT development program that includes a Phase 1/2 study now underway in healthy volunteers."

In June 2019, the Company submitted a clinical trial application to the Swedish Medical Products Agency for DCR-A1AT for the treatment of patients with A1AT deficiency-associated liver disease and began enrolling healthy volunteers in the Phase 1/2 trial of DCR-A1AT (EudraCT number 2019-001999-11) in the fourth quarter of 2019.

The EMA grants orphan medicinal product designation to investigational drugs intended to treat, prevent or diagnose a life-threatening or chronically debilitating disease affecting fewer than five in 10,000 people in the EU and for which no satisfactory treatment is available. Orphan medicinal product designation provides regulatory and financial incentives for companies to develop and market therapies, including market exclusivity, protocol assistance, fee reductions and EU-funded research.

About DCR-A1AT

DCR-A1AT is a subcutaneously administered ribonucleic acid interference (RNAi) therapeutic that is being investigated for the treatment of liver disease in patients with alpha-1 antitrypsin (A1AT) deficiency. A1AT is a protein primarily produced in and released from the liver. A1AT plays a role in several biological functions, including regulating the activity of neutrophil elastase, which helps the body fight infection. In A1AT deficiency, the SERPINA1 gene encodes a mutated form of A1AT, the majority of which cannot be released from the liver. As a result, insufficient levels of A1AT are available to protect the lungs from harmful effects of neutrophil elastase. Additionally, the accumulation of mutated A1AT in the liver can lead to liver disease. There are currently no approved therapies to treat A1AT deficiency-associated liver disease. DCR-A1AT, which incorporates Dicerna's proprietary GalXC™ technology, is designed to target SERPINA1, reducing production of abnormal A1AT in the liver. A clinical trial program investigating the safety and efficacy of DCR-A1AT is currently underway.

About Alpha-1 Antitrypsin (A1AT) Deficiency

Alpha-1 antitrypsin (A1AT) deficiency is an inherited disorder that can lead to liver disease in children and adults, and lung disease in adults. The disorder is caused by mutations in a gene called SERPINA1. When functioning normally, this gene provides instructions for making a protein called A1AT, which protects the body from an enzyme called neutrophil elastase. This enzyme is released from white blood cells to fight infection, but it can attack normal tissues if not tightly controlled by A1AT. Mutations in the SERPINA1 gene can result in a deficiency, or shortage, of functional A1AT and an abnormal form of the protein that cannot control neutrophil elastase. Uncontrolled neutrophil elastase can destroy alveoli (small air sacs in the lungs) and cause lung disease.1 In the liver, the accumulation of abnormal A1AT can trigger an injury cascade, which can lead to liver injury.2

About 10% of adults with A1AT deficiency develop cirrhosis, or liver damage, due to formation of scar tissue in the liver.3 Individuals affected by A1AT deficiency are also at risk of developing hepatocellular carcinoma, a type of liver cancer. People with A1AT deficiency may develop the first symptoms of lung disease between the ages of 20 and 50 years. Symptoms can include shortness of breath following mild activity, reduced ability to exercise, wheezing, unintended weight loss, recurring respiratory infections, fatigue and rapid heartbeat upon standing. Some individuals with A1AT deficiency develop emphysema, a lung disease caused by damage to the alveoli. 1

A1AT deficiency occurs all over the world, though its prevalence varies by population. The disorder affects roughly one in 1,500 to 3,500 individuals with European ancestry and is uncommon in people of Asian descent.1 Congenital A1AT deficiency is estimated to affect 2.4 people out of every 10,000 in the EU.4 Many individuals with A1AT deficiency are thought to be undiagnosed, particularly those who also have chronic obstructive pulmonary disease (COPD). Some people with A1AT deficiency are misdiagnosed with asthma.1

About Dicerna's GalXC™ RNAi Technology Platform

Dicerna's proprietary ribonucleic acid interference (RNAi) technology platform, called GalXC™, aims to advance the development of next-generation RNAi-based therapies designed to silence disease-driving genes in the liver and other tissues. Liver-targeted GalXC-based compounds enable subcutaneous delivery of RNAi therapies that are designed to bind specifically to receptors on liver cells, leading to internalization and access to the RNAi machinery within the cells. The GalXC approach seeks to optimize the activity of the RNAi pathway so that it operates in the most specific and potent fashion. Compounds produced via GalXC are intended to be broadly applicable across multiple therapeutic areas, including both liver and non-liver indications.

About Dicerna™ Pharmaceuticals, Inc.

Dicerna™ Pharmaceuticals, Inc., is a biopharmaceutical company using ribonucleic acid interference (RNAi) to develop medicines that silence genes that cause disease. The Company's proprietary GalXC™ technology is being applied to develop potent, selective and safe RNAi therapies to treat diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases and viral infectious diseases. Dicerna aims to treat disease by addressing the underlying causes of illness with capabilities that extend beyond the liver to address a broad range of diseases, focusing on target genes where connections between gene and disease are well understood and documented. Dicerna intends to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners. Dicerna has strategic collaborations with Novo Nordisk A/S*, Roche*, Eli Lilly and Company, Alexion Pharmaceuticals, Inc. and Boehringer Ingelheim International GmbH. For more information, please visit www.dicerna.com.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements. 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: (i) research and development plans and timelines related to DCR-A1AT and the potential of DCR-A1AT to treat congenital A1AT or liver disease; and (ii) the potential of Dicerna's technology and drug candidates in the Company's research and development pipeline. The process by which an early-stage investigational therapy such as DCR-A1AT and an early-stage platform such as GalXC™ could potentially lead to an approved product or have a major impact on liver disease is a long-term effort 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 Form 10-Q filing 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; the likelihood of Dicerna's clinical programs being executed within timelines provided and reliance on the Company's contract research organizations and predictability of timely screening and enrollment of subjects and patients to advance Dicerna's clinical trials; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of Investigational New Drug Applications (NDAs) and clinical trial applications that are necessary to continue to advance and progress the Company's clinical programs and the regulatory review of NDAs; market acceptance for approved products and innovative therapeutic treatments; competition; the possible impairment of, inability to obtain and costs of obtaining intellectual property rights; and possible safety or efficacy concerns that could emerge as new data are generated in research and development, 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.

* Transactions are subject to clearance under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary closing conditions.

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

References

1.

Genetics Home Reference. Alpha-1 antitrypsin deficiency. Bethesda, Md.: U.S. Department of Health and Human Services, National Institutes of Health, National Library of Medicine; 2013. Available at: https://ghr.nlm.nih.gov/condition/alpha-1-antitrypsin-deficiency#genes. Accessed Dec. 16, 2019.
 

2.

Patel D, Teckman JH. Alpha-1-antitrypsin deficiency liver disease. Clinical Liver Disease. 2018;22(4):643-655.
 

3.

Townsend SA, Edgar RG, Ellis PR, Kantas D, Newsome PN, Turner AM. Systematic review: the natural history of alpha-1 antitrypsin deficiency, and associated liver disease. Aliment Pharmacol Ther. 2018;00:1–9. https://doi.org/10.1111/apt.14537.
 

4.

Based on Dicerna internal estimates.

 

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