Translate Bio Announces Fourth Quarter and Full Year 2018 Financial Results and Reviews Recent Highlights

LEXINGTON, Mass., March 21, 2019 (GLOBE NEWSWIRE) -- Translate Bio (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today announced financial results for the fourth quarter and full year ended December 31, 2018, and provided a summary of recent updates and upcoming milestones.

“2018 was a transformational year for our company. We made progress in all aspects of our business through a number of pivotal milestones as we expanded our team, advanced our first clinical program, announced a strategic partnership, and became a publicly traded company,” said Ron Renaud, chief executive officer of Translate Bio. “In 2019, we are continuing to advance the Phase 1/2 clinical trial for MRT5005 in patients with cystic fibrosis for which we anticipate interim data in the second half of the year. We are also conducting the additional preclinical studies needed to proceed with the IND for our OTC deficiency candidate, MRT5201, and identifying additional disease targets to pursue as part of our discovery efforts.”

Renaud continued, “All of our achievements build upon the eleven years of expertise behind our mRNA therapeutic (MRT) platform, which we believe position us well to move closer to our goal of bringing first-in-class mRNA therapeutics to patients with genetic diseases.”

Fourth Quarter 2018 and Recent Updates

• Initiated dosing of patients in multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial of MRT5005 for the treatment of cystic fibrosis (CF): Translate Bio initiated dosing of patients in the MAD portion of the ongoing first-in-human clinical trial of MRT5005, an mRNA therapeutic product candidate that is designed to treat all patients with CF by addressing the underlying cause of the disease, including those with limited or no cystic fibrosis transmembrane conductance regulator (CFTR) protein.
  
  
• Received Positive Opinion from the European Medicines Agency Committee for Orphan Medicinal Products (COMP): The COMP recommended the granting of orphan drug designation (ODD) for MRT5005 for the treatment of cystic fibrosis. MRT5005 was previously granted ODD by the U.S Food and Drug Administration (FDA). The positive opinion issued by COMP will be sent to the European Commission, which will issue a decision within 30 days of receipt of the COMP’s recommendation. ODD in the European Union (EU) allows the Company to benefit from reduced regulatory fees, protocol assistance, access to centralized authorization procedures for the EU, and a prescribed period of market exclusivity, to develop a medicine for the treatment of a rare disease affecting not more than five in 10,000 people in the EU.
  
  
• Submitted an Investigational New Drug (IND) Application for MRT5201 for the treatment of ornithine transcarbamylase (OTC) Deficiency; conducting additional preclinical studies: Translate Bio submitted the IND in December 2018 to support the initiation of a Phase 1/2 clinical trial of MRT5201 in patients with OTC deficiency. MRT5201 is a first-in-class treatment designed to directly address the underlying cause of OTC deficiency by providing mRNA encoding the fully functional OTC enzyme in patients with the disease. In January 2019, the Company announced that the FDA placed the IND on clinical hold. The FDA is requiring additional preclinical data. The Company has identified the additional preclinical studies required, and plans to complete these studies and submit a response to the FDA in the fourth quarter of 2019.
  
  
• Published preclinical data demonstrating efficacy of a liver-targeted mRNA therapeutic in disease model of Fabry, a lysosomal storage disease: In March 2019, Translate Bio scientists published preclinical data showing that delivery of a nanoparticle-formulated mRNA produced sustained expression of human α-galactosidase protein in vivo, and demonstrated efficacy through clinically relevant biomarker reduction in an animal model of Fabry disease. Fabry disease is primarily caused by a deficient quantity of α-galactosidase A, an enzyme needed to break down a fatty substance in the body called globotriaosylceramide. When this substance accumulates in the body’s cells, the resulting cell damage can cause a range of mild to severe symptoms including life-threatening complications such as kidney failure, heart attacks and strokes, often at a young age.
  
  
• Strengthened intellectual property portfolio with liver-specific delivery patent: In December 2018, Translate Bio announced that the United States Patent and Trademark Office had issued U.S. Patent 10,143,758. This patent titled “Liver Specific Delivery of Messenger RNA” builds on the Company’s 2009 patent filing relating to liposomal delivery of therapeutic mRNA to the liver, enhancing the Company’s current intellectual property by providing additional coverage for core delivery capabilities. This patent is owned by Translate Bio and provides protection until March, 2032.

Anticipated Milestones

• MRT5005 (CF): Interim data from Phase 1/2 clinical trial in second half of 2019
• MRT5201 (OTC Deficiency): Complete additional preclinical studies and submit complete response to FDA in fourth quarter of 2019
• Identify lead preclinical candidates for additional lung and liver disease targets

Upcoming Events

• The Company will host one-on-one meetings at the Guggenheim Healthcare Talks Rare Disease and Genomic Medicines Day on April 2, 2019 in Cambridge, MA.
• A poster presentation entitled “Treatment of Ornithine Transcarbamylase Deficiency with a Targeted mRNA Therapeutic (MRT)” will be given at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting on April 7, 2019 in Bellevue, WA.
• A presentation will be given at the Keystone Symposia: Protein Replacement through Nucleic Acid Therapies during a session entitled “Protein Replacement with mRNA” on April 8, 2019 in Steamboat Springs, CO.
• The Company will give a corporate presentation and host one-on-one meetings at the 18^th Annual Needham Healthcare Conference on April 10, 2019 in New York, New York.
• The Company will participate in a presentation entitled “Successful case study 1: Sanofi – Translate Bio collaboration to develop mRNA vaccines for multiple infectious diseases” at the World Vaccine Congress on April 16, 2019 in Washington DC.
• An oral presentation entitled “Treatment of Metabolic Disorders Using Lipid Nanoparticle (LNP)-Encapsulated mRNA Therapeutics (MRT)” will be given at the 22^nd Annual Meeting of The American Society for Gene & Cell Therapy on Monday, April 29, 2019 in Washington DC.

Fourth Quarter and Full Year 2018 Financial Results and Financial Guidance

Translate Bio ended the fourth quarter of 2018 with $144.1 million in cash, cash equivalents and investments. The Company expects that its existing cash, cash equivalents and investments will enable it to fund its operations into the second quarter of 2020.

Translate Bio reported a net loss of $6.0 million and $16.1 million for the three months ended December 31, 2018 and 2017, respectively. The Company reported a net loss of $97.4 million and $66.4 million for the years ended December 31, 2018 and 2017, respectively.

Collaboration revenue was $1.2 million in the three months ended December 31, 2018 and $1.4 million for the year ended December 31, 2018, which was derived from the collaboration and license agreement that the Company entered into with Sanofi Pasteur, which became effective in July 2018. There was no collaboration revenue in the three months ended December 31, 2017 or in the year ended December 31, 2017.

Operating expenses for the three months ended December 31, 2018 were $8.5 million, compared to $24.6 million for the same period in 2017, and were comprised of the following:

• Research and development expenses of $17.2 million during the fourth quarter of 2018, compared to $12.8 million for the same period in 2017. The increase is primarily due to an increase in costs associated with the continued advancement of the Company’s MRT discovery program and CF program.
   
• General and administrative expenses of $5.9 million during the fourth quarter of 2018, compared to $4.6 million for the same period in 2017. The increase is primarily due to an increase in professional fees and insurance costs.
   
• The Company recognized an adjustment in the fair value of contingent consideration liabilities of ($14.6) million and $7.2 million in the three months ended December 31, 2018 and 2017, respectively, related to future potential milestone and earnout payment obligations and anti-dilution rights with respect to common stock issued to Shire that the Company recorded in connection with its acquisition of the MRT Program in December 2016. The decrease is attributed primarily to a fair value adjustment and to the full satisfaction of the Company’s obligation to issue shares of common stock to Shire upon closing of the Company’s initial public offering in the third quarter of 2018.

Operating expenses for the year ended December 31, 2018 were $105.7 million, compared to $79.2 million for the same period in 2017, and were comprised of the following:

• Research and development expenses of $58.0 million for the year ended December 31, 2018, compared to $47.0 million for the same period in 2017. The increase is primarily due to an increase in costs associated with the continued advancement of the Company’s CF and OTC deficiency programs and MRT discovery program as well as an increase in personnel-related costs.
   
• General and administrative expenses of $22.6 million during the year ended December 31, 2018, compared to $14.3 million for the same period in 2017. The increase is primarily due to increases in personnel-related costs, professional fees and insurance costs.
   
• The Company recognized an adjustment in the fair value of contingent consideration liabilities of $25.0 million and $17.9 million in the years ended December 31, 2018 and 2017, respectively, related to the Shire transaction as described above. The increase is attributed primarily to the progress of the Company’s CF and OTC deficiency programs, the time value of money due to the passage of time, as well as an adjustment in the discount rate.

About Translate Bio
Translate Bio is a clinical-stage mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. The Company’s MRT platform is designed to develop product candidates that deliver mRNA carrying instructions to produce intracellular, transmembrane and secreted proteins for therapeutic benefit. Translate Bio believes that its MRT platform is applicable to a broad range of diseases caused by insufficient protein production or where production of proteins can modify disease, including diseases that affect the lung, liver, eye, central nervous system and lymphatic system. The Company also believes its MRT platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as infectious disease and oncology. Translate Bio’s two lead programs are being developed as treatments for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency. For more information about the Company, please visit www.translate.bio or on Twitter at @TranslateBio.

Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding:  the potential for MRT5005 to address the underlying cause of CF; Translate Bio’s plans to report interim data from the Phase 1/2 clinical trial of MRT5005 in the second half of 2019; Translate Bio’s plans to conduct additional preclinical studies for MRT5201 and its plan to submit a response to FDA in the fourth quarter of 2019; Translate Bio’s plans to provide a complete response to the FDA; Translate Bio’s plans to identify lead preclinical candidates for additional disease targets; Translate Bio’s expectations with respect to the granting of ODD for MRT5005 in the EU; the period in which Translate Bio expects that its existing cash, cash equivalents and investments will enable it to fund its operations; Translate Bio’s expectations regarding the potential for its patent portfolio to protect its intellectual property; Translate Bio’s beliefs regarding the broad applicability of its MRT platform; and Translate Bio’s plans, strategies and prospects for its business, including its lead development programs. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to: the risk that Translate Bio may not resolve the clinical hold on MRT5201 or any other clinical hold in the near term or at all, or that the FDA could make decisions that adversely impact the ability of Translate Bio to advance its programs in development; Translate Bio’s ability to advance the development of its platform and programs under the timelines it projects, demonstrate the requisite safety and efficacy of its product candidates and replicate in clinical trials any positive findings from preclinical studies; Translate Bio’s ability to obtain additional preclinical data to support its IND application for MRT5201; the content and timing of decisions made by the FDA, other regulatory authorities and investigational review boards at clinical trial sites, including decisions that may arise as a result of the clinical hold on MRT5201 and as it relates to ongoing and planned clinical trials; Translate Bio’s ability to obtain, maintain and enforce necessary patent and other intellectual property protection; the availability of significant cash required to fund operations; competitive factors; general economic and market conditions and other important risk factors set forth under the caption “Risk Factors” in Translate Bio’s Annual Report on Form 10-K for the quarterly period ended December 31, 2018 filed with the Securities and Exchange Commission on March 21, 2019 and in any other subsequent filings made by Translate Bio. Any forward-looking statements contained in this press release speak only as of the date hereof, and Translate Bio specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.