8-K

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

 

 

FORM 8-K

 

 

CURRENT REPORT

Pursuant to Section 13 or 15(d)

of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): January 13, 2020

 

 

Translate Bio, Inc.

(Exact name of registrant as specified in its charter)

 

 

 

Delaware   001-38550   61-1807780

(State or other jurisdiction

of incorporation)

 

(Commission

File Number)

 

(IRS Employer

Identification No.)

29 Hartwell Avenue

Lexington, Massachusetts

  02421
(Address of principal executive offices)   (Zip Code)

Registrant’s telephone number, including area code: (617) 945-7361

Not applicable

(Former Name or Former Address, if Changed Since Last Report)

 

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

 

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

 

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

 

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

 

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

 

Title of each class

 

Trading

Symbol(s)

 

Name of exchange

on which registered

Common Stock, $0.001 par value   TBIO   Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company  ☒

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.  ☒

 

 

 


Forward-Looking Statements

This Current Report on Form 8-K 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 Company’s estimated cash, cash equivalents and short-term investments as of December 31, 2019; the Company’s belief that its cash, cash equivalents and short-term investments as of December 31, 2019 will enable it to fund its operating expenses and capital expenditure requirements into the second quarter of 2021; and the Company’s plans to report data in the third quarter of 2020 from the Phase 1/2 clinical trial of MRT5005. 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 Company’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; the content and timing of decisions made by the U.S. Food and Drug Administration, other regulatory authorities and investigational review boards at clinical trial sites, including as it relates to ongoing and planned clinical trials; the Company’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 discussed in the “Risk Factors” section in the Company’s Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2019 filed with the Securities and Exchange Commission (“SEC”) on November 6, 2019, and in any other subsequent filings made by the Company with the SEC. Any forward-looking statements contained in this Current Report on Form 8-K speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Item 2.02 Results of Operations and Financial Condition.

The information provided in Item 8.01 of this Current Report on Form 8-K regarding the Company’s estimated cash, cash equivalents and short-term investments as of December 31, 2019 and its ability to fund its operating expenses and capital expenditure requirements is incorporated by reference into this Item 2.02.

Item 7.01 Regulation FD Disclosure.

Translate Bio, Inc. (the “Company”) from time to time presents or distributes to the investment community, at various industry and other conferences, slide presentations to provide updates and summaries of its business. A copy of the Company’s current corporate slide presentation is attached to this Current Report on Form 8-K as Exhibit 99.1 and is incorporated by reference into this Item 7.01. The Company undertakes no obligation to update, supplement or amend the materials attached hereto as Exhibit 99.1.


The information in this Item 7.01 of this Current Report on Form 8-K, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933 or the Exchange Act, except as expressly set forth by specific reference in such filing.

Item 8.01 Other Events.

On January 13, 2020, the Company disclosed certain preliminary information related to the Company’s estimated cash, cash equivalents and short-term investments as of December 31, 2019 and its ability to fund its operating expenses and capital expenditure requirements. Although the Company has not yet completed its financial statements for the fourth quarter and fiscal year ended December 31, 2019, it expects to report cash, cash equivalents and short-term investments of $188.7 million as of December 31, 2019, and the Company believes that its cash, cash equivalents and short-investments as of December 31, 2019 will enable the Company to fund its operating expenses and capital expenditure requirements into the second quarter of 2021.

The Company’s estimates of its cash, cash equivalents and short-term investments as of December 31, 2019 and its ability to fund its operating expenses and capital expenditure requirements are preliminary and unaudited, represent management estimates as of the date of this Current Report on Form 8-K and are subject to completion of the Company’s financial closing procedures. The Company’s independent registered public accounting firm has not conducted an audit or review of, and does not express an opinion or any other form of assurance with respect to, the estimated cash, cash equivalents and short-term investments and its ability to fund its operating expenses and capital expenditure requirements.

On January 13, 2020, the Company also disclosed that it expects to report data in the third quarter of 2020 from the additional 20 mg single-ascending dose group and the multiple-ascending dose portion of the ongoing Phase 1/2 clinical trial which is evaluating the safety and tolerability of single and multiple ascending doses of MRT5005.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

 

Exhibit No.

  

Description

99.1    Corporate slide presentation of Translate Bio, Inc. dated January 13, 2020


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

    TRANSLATE BIO, INC.
Date: January 13, 2020     By:  

/s/ Paul Burgess

      Paul Burgess
      Chief Operating Officer, Chief Legal Officer and Secretary
EX-99.1

Slide 1

Transforming mRNA science into healthy expression Corporate Presentation January 2020 Exhibit 99.1


Slide 2

Cautionary Note Regarding Forward-Looking Statements and Legal Disclaimers This presentation contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such data and estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk. This presentation and various remarks we make during this presentation contain forward-looking statements of the Company within the meaning of the Private Securities Litigation Reform Act of 1995, including statements with respect to: our plans, prospects, strategies and expectations for our business; the possible achievement of research and clinical development goals and milestones; our estimated cash, cash equivalents and short-term investments as of December 31, 2019; our belief that our cash, cash equivalents and short-term investments as of December 31, 2019 will enable us to fund our operating expenses and capital expenditure requirements into the second quarter of 2021; our plan to report data from the Phase 1/2 clinical trial of MRT5005 in the third quarter of 2020; our future research and development efforts; the potential benefits of our product candidates; our collaboration plans; our key milestones and the estimated timing for potentially achieving such milestones; and our future operating results and financial position. We use words such as “anticipate,” “believe,” “estimate,” “expect,” “hope,” “intend,” “may,” “milestone,” “plan,” “predict,” “project,” “target,” “potential,” “would,” “can,” “could,” “should,” “continue,” and other words and terms of similar meaning to help identify forward-looking statements, although not all forward-looking statements contain these identifying words. There are a number of important risks and uncertainties that could cause actual results or events to differ materially from those indicated by such forward-looking statements. These risks and uncertainties include those inherent in pharmaceutical research and development, such as: adverse results in our drug discovery, preclinical and clinical development activities, our ability to enroll patients in our clinical trials, and the risk that any of our clinical trials may not commence, continue or be completed on time, or at all; decisions made by the U.S. FDA and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies with respect to our development candidates; our ability to obtain, maintain and enforce intellectual property rights for our platform and development candidates; our potential dependence on collaboration partners; competition; our ability to obtain necessary financing to conduct our planned activities and to manage unplanned cash requirements; and general economic and market conditions. These and other risk factors are described in greater detail under the caption “Risk Factors” and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 and in other filings that we may make with the SEC in the future. Any forward-looking statements contained in this presentation and various remarks we make during this presentation speak only as of the date hereof, and we expressly disclaim any obligation to update any forward looking statements, whether as a result of new information, future events or otherwise, except as required by law.


Slide 3

1st mRNA therapy in clinic for chronic disease Early encouraging clinical activity with ppFEV1 improvement Building off capabilities established for CF program 3 additional programs: primary ciliary dyskinesia idiopathic pulmonary fibrosis pulmonary arterial hypertension Preclinical data in multiple rare diseases LNPs to deliver mRNA to liver, as well as multiple other tissue types Industry-leading scale and process development to optimize purity and activity Potential to support commercial launch of CF Highly-Differentiated mRNA Therapeutics Company Cystic Fibrosis Pulmonary Focus Platform Manufacturing


Slide 4

Treating a monogenic disease with mRNA to replace a defective or missing protein MRT5005 Discovery program for additional CFTR protein/lipid approach Leveraging RNA and Delivery Expertise to Treat Pulmonary Diseases Treating a disease with mRNA and/or siRNA to address complex disease with multifaceted etiology Treating a disease with mRNA to produce a protein to activate signaling pathway Cystic Fibrosis Idiopathic Pulmonary Fibrosis (IPF) Pulmonary Ciliary Dyskinesia (PCD) Pulmonary Arterial Hypertension (PAH) Treating a monogenic disease with mRNA to replace a defective or missing protein


Slide 5

Unlocking the Power of RNA and Delivery to Expand Our Toolkit Delivery mRNA siRNA mRNA Engineered Proteins Delivery siRNA Foundational Technology Technology Expansion Wild Type Protein Wild Type Protein Protein Degradation


Slide 6

Translate Bio’s Broadly Applicable Platform Fuels Our Pipeline Focus Area Indication Route of Administration Discovery IND-Enabling Phase 1 / 2 Clinical Direct Therapeutics LUNG Cystic Fibrosis (CF) Inhalation Cystic Fibrosis (CF) Inhalation Idiopathic Pulmonary Fibrosis (IPF) Inhalation Primary Ciliary Dyskinesia (PCD) Inhalation Pulmonary Arterial Hypertension (PAH) Inhalation LIVER Undisclosed Intravenous OTHER RARE Undisclosed Various Vaccines LYMPHATIC Infectious Disease Undisclosed Immuno-Oncology Various MRT5005


Slide 7

Pulmonary Delivery of CFTR mRNA Cystic Fibrosis MRT Program


Slide 8

Weekly self-administered nebulization Significant improvements in lung function Halt the progressive decline in lung function Substantially reduce the frequency of pulmonary exacerbations Deliver mRNA encoding fully functional CFTR protein to the lung Initial development focused on patients non-amenable to CFTR modulators MRT5005, nebulized CFTR mRNA in a lipid particle Granted U.S. and EU Orphan Drug Designation Goals MOA Dosing Target Patient Population Product MRT5005: Designed to Address the Underlying Cause of CF Regardless of Mutation


Slide 9

13.6% 9.4% Genotyped Patients Eligible for Trikafta™ 66,580 patients ~$9.0B1 (2030) Genotyped Patients Eligible for Kalydeco® 11,750 patients $1.0B (actual sales in 2018) Genotyped Patients Not Amenable to Modulators 8,100 patients 77.0% Cystic Fibrosis Patients WW 86,430 patients in U.S., EU, Canada, and Australia registries Sources: 2018 U.S. CFF Patient Registry; 2017 ECFS Patient Registry. 1. Peak sales estimate from SVB Leerink, 22 Oct 2019 and 19 Mar 2019. Significant Number of Patients Lack Treatment Despite Trikafta Approval


Slide 10

13.6% 9.4% Genotyped Patients Eligible for Trikafta™ 66,580 patients ~$9.0B1 (2030) Genotyped Patients Eligible for but Potentially Not Taking Trikafta™ 2 Cannot tolerate, contraindicated No FEV1 response Low (0-5%) FEV1 response 57.2% Cystic Fibrosis Patients WW 86,430 patients in U.S., EU, Canada, and Australia registries 0.4% 2.6% 16.9% Sources: 2018 U.S. CFF Patient Registry; 2017 ECFS Patient Registry. 1. Peak sales estimate from SVB Leerink, 22 Oct 2019 and 19 Mar 2019. 2. N Engl J Med 2019; 381:1809-1819; Lancet. 2019 Nov 23;394(10212):1940-1948. Genotyped Patients Eligible for Kalydeco® 11,750 patients $1.0B (actual sales in 2018) Genotyped Patients Not Amenable to Modulators 8,100 patients Significant Number of Patients Lack Treatment Despite Trikafta Approval


Slide 11

PART A: Single Ascending Dose (SAD) Groups 8 mg 16 mg 24 mg 12 Patients Total Phase 1/2 Clinical Trial Design Design Randomized, double-blind, placebo-controlled, staggered dosing Each cohort: N=4 (3:1) Objective Evaluate safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization Eligibility & Inclusion 18 years+ ppFEV1 ≥50% and ≤90% Patients with two Class I and/or Class II CFTR mutations 11 patients had at least one copy of the F508del mutation and 1 patient did not have a F508del mutation and was considered non-amenable to CFTR modulator treatment 7 of the 12 patients were taking an approved CFTR modulator through screening, dosing and follow-up PART B: Multiple Ascending Dose (MAD) Groups 5 weekly doses Ongoing


Slide 12

Positive Interim Results: First-in-Human Phase 1/2 Clinical Trial of MRT5005 in Patients with CF First mRNA therapeutic administered for a chronic disease and first to be administered via inhalation Generally well-tolerated at low- and mid-dose levels; No serious adverse events reported at any dose level Marked increases in ppFEV1* after single MRT5005 dose, primarily at mid-dose level Increases in ppFEV1 observed in patient with mutations not amenable to CFTR modulators; increases also observed in patients on stable background of CFTR modulators Findings support exploration of two additional dose cohorts * ppFEV1: percent predicted forced expiratory volume in one second


Slide 13

Marked Increases in ppFEV1 Observed After Single Dose of MRT5005, Primarily at Mid-Dose Mean Absolute Change from Baseline in ppFEV1 Patients in the 16 mg dose demonstrated: Mean maximum increase from baseline of 15.7% Individual maximal ppFEV1 increases of 11.1%, 13.6% and 22.2% 2 of the 3 had a stable CFTR modulator treatment regimen the third had genotype non-amenable to CFTR modulator treatments Increases in ppFEV1 were higher than expected based on known variability of ppFEV1 Time course of ppFEV1 improvements potentially support a CFTR-related mechanism Data suggest MRT5005 can enable production of functional protein In the 8-day post-dosing period: Mean (SD) Change from Baseline ppFEV1 %


Slide 14

Rapid FEV1 Increases Correlate with Time Course of CFTR Protein Expression Biosynthesis & Maturation of CFTR (Riordan JR 2008) 0 minutes CFTR mRNA transcription (t½=13-24 hours) 5-10 minutes Translation and core glycosylation, complete 30 minutes Final high molecular weight glycosylation 2 hours Cell surface functional CFTR (t½=12-24 hours) MRT5005 Preclinical Data 0 minutes Rodents nebulized 30 minutes mRNA detectable by qPCR (t½=61 hours) 2 hours mRNA detectable by ISH* 2 hours CFTR protein detectable by IHC** 48 hours CFTR protein peak staining intensity CFTR protein detectable out to 7-28 days MRT5005 Phase 1/2 SAD Data 0 minutes MRT5005 administered via nebulizer Maximal FEV1 increases seen at Day 1 (8h), Day 2, Day 3 or Day 8 Bronchial epithelium Lung tissue lysates *In situ hybridization (ISH) **Immunohistochemistry (IHC)


Slide 15

How Much CFTR Do You Need to be Effective? CFTR delivery to 25% of the lung surface epithelial cells restores normal mucus transport rates in vitro Zhang L et al. PLoS Biol 2009 No Symptoms 100% 50% CFTR Function % Obligate Heterozygotes CF-related Disease 0% Cystic Fibrosis Healthy Subjects CFTR expression of ~20% corrects CF chloride ion transport defect in vitro CFF, NACFC 2018; Linear regression for illustrative purposes only Farmen SL et al. Am J Physiol Lung Cel 2005


Slide 16

Approved CFTR Modulators Have Shown ppFEV1 Increases from 2-14% * Patients received Symdeko® during a 4-week run-in period and were then randomized to receive Trikafta™ or Symdeko® during a 4-week double-blind treatment period.


Slide 17

Safety: MRT5005 Generally Well-Tolerated at Low- and Mid-Dose Levels No SAEs at any dose Most common AEs – cough, headache Generally well-tolerated at low- and mid-dose levels Primarily at high dose, patients experienced transient, mild-moderate febrile reactions Occurred ~4-10 hours post dosing Symptoms resolved within 24 hours All patients discharged from study center on Day 2 as planned * Occurred at Day 25 and Day 27 Treatment Emergent Adverse Events (TEAEs) through Day 29 Pooled Placebo (N=3) MRT5005 8 mg (N=3) MRT5005 16 mg (N=3) MRT5005 24 mg (N=3) Overall TEAEs 11 28 25 33 Not Related 9 11 9 10 Related 2 17 16 23 TEAEs by Severity Mild 11 23 24 21 Moderate 0 5 1 12 Severe 0 0 0 0 Serious TEAEs 0 0 0 0 TEAEs Leading to Discontinuation 0 0 0 0 TEAEs Resulting in Death 0 0 0 0 TEAEs Classified as Pulmonary Exacerbation 0 0 0 2*


Slide 18

Updated Phase 1/2 Clinical Trial Protocol: Findings Support Exploration of Two Additional Dose Cohorts Up to 16 Patients 8 Patients Total Select dose level Select dose level PART B EXPANSION: Multiple Ascending Dose (MAD) Groups 5 weekly doses PART A: Single Ascending Dose (SAD) Groups 8 mg 16 mg 24 mg 20 mg 16 Patients Total PART B: Multiple Ascending Dose (MAD) Groups 5 weekly doses 8 mg 16 mg 12 mg Optional 20 mg Protocol Changes SAD: Add 20 mg dose MAD: Add 12 mg and 20 mg No 24 mg dose MAD Part B expansion: enroll additional patients at MAD doses of interest (in lieu of originally planned Part C) Protocol changes


Slide 19

Interim Data from Phase 1/2: First mRNA Therapeutic for Cystic Fibrosis ppFEV1 Safety Marked increases in ppFEV1 within the 8 days after treatment in 4 patients, including all 3 patients in 16 mg dose group Early improvement in ppFEV1 suggests MRT5005 crossing the mucus layer enabling production of functional CFTR protein ppFEV1 increases observed in “non-amenable patient” and in patients on modulator regimens No serious adverse events at any dose level Most common adverse events: cough and headache Generally well-tolerated at the low- and mid-dose levels Primarily at high dose: transient, mild to moderate febrile reactions


Slide 20

Discovery Efforts Driving Future Pipeline Programs


Slide 21

Focused Innovation with Pulmonary Delivery Inhalation may provide local efficacy while minimizing systemic toxicity 83,000 diagnosed cases of IPF in the U.S.2 Opportunity to address a range of protein targets to potentially reverse the disease 53,000 diagnosed cases of PAH in the U.S.2 Idiopathic Pulmonary Fibrosis (IPF) Pulmonary Ciliary Dyskinesia (PCD) Pulmonary Arterial Hypertension (PAH) Potential to restore ciliary function by providing instructions to make wildtype protein 16,000 diagnosed cases of PCD in the U.S.1 1DelveInsight, 2 Datamonitor PCD protein expression Control siRNA Target Expression


Slide 22

Progress in Sanofi Pasteur Vaccine Collaboration mRNA Production LNP Production Multiple Disease Programs Screened several infectious disease targets Multiple grams of mRNA produced for preclinical studies Large scale process development complete for lead mRNA constructs Over a dozen different structurally-unique lipid families produced and tested Hundreds of formulations tested with multiple leads applied towards disease targets Lead formulation optimization completed with large scale LNP process development underway Lead mRNA and LNP candidates tested across several infectious disease targets Comprehensive rodent and non-human primate studies complete across a number of programs Robust titers observed for all antigens tested


Slide 23

Robust Approach to Generating and Validating Next-generation Delivery Technologies High-throughput parallel synthesis for efficient library production with different structural cores and testing in vivo Prototype established for high-throughput LNP production Dan Anderson (MIT): Collaboration dating back to 2009 Additional university collaborations with high-profile chemists and biomedical engineers Formulation optimization: New processes Novel compositions Structure-activity relationship (SAR)-based approach for multiple ROAs In-House Lipid Production Collaborations Delivery Platform Development


Slide 24

MRT5005 (CF) Report data from MAD portion of Phase 1/2 clinical trial and additional 20 mg SAD dose group 3Q 2020 Anticipated Milestones Over Next 18 Months Pipeline Programs Sanofi Pasteur collaboration: advance lead candidate towards IND CF discovery: preclinical safety, protein expression, duration of expression for development candidate selection IPF: preclinical proof of concept to support development candidate selection PCD: preclinical proof of concept (restoration of cilia function) to select lead PCD program PAH: evaluate and validate target for PAH Platform Identify next-generation LNP to support liver, lung and additional disease program development Continue to evaluate promising liver targets Advance mRNA protein degradation technology $188.7 M Cash, cash equivalents and short-term investments Funding expected into 2Q 2021 as of 12/31/19 (unaudited) Expected


Slide 5

Thank you