Translate Bio Announces Third Quarter 2020 Financial Results and Reviews Recent Progress
-- Advances Phase 1/2 clinical trial of MRT5005 for the treatment of cystic fibrosis (CF) with resumption of COVID-impacted enrollment and dosing --
-- Presented promising preclinical data at NACFC from next-generation CF program that leverages advances in mRNA technology --
-- Reported positive preclinical results from COVID-19 mRNA vaccine program with collaborator
“We continue to leverage our mRNA and lung delivery capabilities to advance our technology with a focus on addressing unmet needs in CF and other pulmonary diseases. With the resumption of enrollment and dosing in the Phase 1/2 clinical trial of MRT5005, and promising preclinical data from our next-generation CF program, we continue to demonstrate our commitment to developing a treatment for all patients with CF, particularly those non-amenable to modulator treatments,” said
Third Quarter 2020 and Recent Updates
- Expanding research and development footprint: In
November 2020, the Company entered into a lease agreement for new office and laboratory space in order to expand its research and development footprint and build upon internal capabilities with a continued focus on advancing platform innovation and driving multiple programs towards clinical development.
- Reported positive preclinical data for MRT5500, lead COVID-19 mRNA vaccine candidate, now anticipated to begin Phase 1/2 clinical trial in first quarter 2021: In
October 2020, Translate Bioand Sanofi Pasteurannounced preclinical results for MRT5500, an mRNA-based vaccine candidate against SARS-CoV-2, the virus that causes COVID-19. In preclinical studies, MRT5500 induced potent neutralizing antibodies against SARS-CoV-2 at levels significantly higher than those observed in a panel of samples from COVID-19 patients. These data supported the selection of MRT5500 for clinical development. A Phase 1/2 clinical trial was anticipated to begin in the fourth quarter of 2020, and is now expected to begin in the first quarter of 2021 due to a delay in the manufacturing of clinical trial material.
- Presented data from mRNA clinical and preclinical product candidates for treatment of CF at NACFC: In
October 2020, Translate Biogave oral and poster presentations at the 34th Annual North American Cystic Fibrosis Conference(NACFC) which underscored the remaining unmet need in the treatment of CF, reviewed encouraging clinical data shared to date for the Company’s lead program, MRT5005, and described preclinical data for a next-generation CF program which applies advances in mRNA science, lipid nanoparticle (LNP) chemistry, and investigation of the cystic fibrosis transmembrane conductance regulator (CFTR) biology. Preclinical data presented from the next-generation CF program demonstrated that the design of novel mRNA sequences encoding wild-type and rationally engineered CFTR protein produced active ion channels and enhanced protein expression when compared to reference experiments in vitro. Preclinical studies are ongoing to select the optimal mRNA sequence and LNP for the Company’s next-generation CFTR product candidate.
- Resumed dosing and enrollment in Phase 1/2 clinical trial for MRT5005 for the treatment of CF: In
September 2020, Translate Bioannounced that enrollment and dosing in its Phase 1/2 clinical trial for MRT5005 in CF resumed, with multiple clinical sites open for enrollment. In March 2020, the Company had announced a pause to enrollment and dosing in the clinical trial as a consequence of the COVID-19 pandemic. The Company plans to provide updated timing on the expected interim data readout of the additional single-ascending dose (SAD) group and the multiple-ascending dose (MAD) portion of the clinical trial at a later date.
- MRT5500 (COVID-19 vaccine): Initiate Phase 1/2 clinical trial in Q1 2021 (
- Flu vaccine: Advance lead LNP/mRNA formulation to clinical proof of technology trial anticipated to begin mid-year 2021 (
- MRT5005 (CF): Report results from additional SAD dose group and MAD portion of the ongoing Phase 1/2 clinical trial
- Preclinical pulmonary programs: Advance next-generation CF, primary ciliary dyskinesia, idiopathic pulmonary fibrosis and pulmonary arterial hypertension programs toward selection of development candidates
- Platform: Identify next-generation LNPs to support liver, lung and additional disease program development
- The Company will give presentations on its infectious disease vaccine and therapeutic pulmonary programs at the 8th International mRNA
Health Conferencetaking place virtually November 9-10, 2020.
- The Company will participate in the following virtual investor conferences:
Jefferies Virtual London Healthcare Conference, November 17-19, 2020 Evercore ISI HealthCONx Conference, December 1-3, 2020
Third Quarter 2020 Financial Results and Financial Guidance
Collaboration revenue was
Operating expenses for the three months ended
- Research and development expenses of
$26.3 millionduring the third quarter of 2020, compared to $17.3 millionfor the same period in 2019. The increase is primarily due to continued development of the Company’s vaccine program associate with the Sanofi collaboration and discovery program as well as an increase in personnel-related costs, partially offset by a decrease in expenses related to the Company’s MRT5005 and MRT5201 programs.
- General and administrative expenses of
$9.2 millionduring the third quarter of 2020, compared to $6.9 millionfor the same period in 2019. The increase is primarily due to costs related to entering into the expanded Sanofi agreement.
- Operating expenses of
$14.2 millionfor change in the fair value of contingent consideration related to future potential milestone and earnout payment obligations. The expense recognized was primarily attributed to an increase in the fair value of the contingent consideration liability due to the time value of money due to the passage of time and a decrease in the discount rate.
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 and benefit patients; Translate Bio’s plans to provide updated timing on the expected interim data of the Phase 1/2 clinical trial of MRT5005; Translate Bio’s expectations with respect to its collaboration with Sanofi, including the anticipated initiation of a Phase 1/2 trial for MRT5500 in the first quarter of 2021 and the advancement of lead LNP/mRNA formulation for the flu vaccine to a clinical proof of technology trial to begin mid-year 2021; Translate Bio’s plans to advance its pipeline of mRNA therapeutics and validate targets for additional pulmonary diseases; Translate Bio’s plans to advance its additional disease program programs and platform; Translate Bio’s plans to expand its research and development footprint and build upon internal capabilities; the period in which Translate Bio expects that its existing cash and cash equivalents will enable it to fund its operating expenses and capital expenditure requirements; 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 of adverse current and potential future impacts of the COVID-19 pandemic on the Company’s business, financial condition, operations and liquidity; the risk that
|CONSOLIDATED STATEMENTS OF OPERATIONS|
|Three Months Ended
||Nine Months Ended
|Research and development||26,344||17,295||76,785||51,343|
|General and administrative||9,163||6,881||25,223||21,284|
|Change in fair value of contingent consideration||14,190||(19,834||)||20,085||(3,243||)|
|Impairment of intangible asset||—||18,559||—||18,559|
|Total operating expenses||49,697||22,901||122,093||87,943|
|Income (loss) from operations||16,749||(21,635||)||(34,673||)||(84,029||)|
|Other income, net||595||408||1,447||1,286|
|Income (loss) before benefit from income taxes||17,344||(21,227||)||(33,226||)||(82,743||)|
|Benefit from income taxes||—||—||—||486|
|Net income (loss)||$||17,344||$||(21,227||)||$||(33,226||)||$||(82,257||)|
|CONSOLIDATED BALANCE SHEETS|
|Cash and cash equivalents||$||674,050||$||84,580|
|Prepaid expenses and other current assets||10,822||9,391|
|Total current assets||710,961||203,615|
|Property and equipment, net||15,044||12,539|
|Right-of-use assets, net||75,650||10,400|
|Intangible assets, net||81,280||85,536|
|Liabilities and Stockholders' Equity|
|Current portion of deferred revenue||93,164||18,100|
|Current portion of operating lease liability||12,279||530|
|Total current liabilities||124,024||41,670|
|Deferred revenue, net of current portion||242,047||25,256|
|Operating lease liability, net of current portion||53,151||12,084|
|Additional paid-in capital||758,314||512,231|
|Accumulated other comprehensive income||—||741|
|Total stockholders' equity||365,666||153,536|
|Total liabilities and stockholders' equity||$||908,628||$||336,201|
Source: Translate Bio, Inc.