Translate Bio Announces First Quarter 2021 Financial Results and Provides Corporate Update
-- Advanced infectious disease vaccine programs with
-- Reported interim data from Phase 1/2 clinical trial of MRT5005 for the treatment of cystic fibrosis (CF) --
-- Enhanced internal capabilities across pulmonary programs, manufacturing and financial operations with key leadership additions and promotions --
“This quarter, we continued to make important progress in growing our team and advancing our pipeline of mRNA therapeutics and vaccines. With interim data from our ongoing trial of MRT5005 in CF reported in March, we’ve taken critical steps toward developing the first inhaled mRNA therapeutic to treat a genetic lung disease,” said
Renaud continued, “We are continuing to execute on our 5-year strategic plan, TBIO 2025, with the goal of further advancing platform innovation and driving multiple programs into the clinic. We believe our recent leadership appointments bring expertise in pulmonary science, manufacturing, and operations that will be critical to our continued growth.”
First Quarter 2021 and Recent Updates
- Initiated COVID-19 mRNA vaccine clinical trial (MRT5500) with Sanofi: In
March 2021, the Company announced the start of the Phase 1/2 clinical trial for MRT5500, an mRNA vaccine candidate against SARS-CoV-2, the virus that causes COVID-19. Translate Bioreceived a $25 millionpayment from Sanofi Pasteurbased on achievement of this milestone. Interim results from this trial are expected in the third quarter of 2021. Additionally, there is ongoing preclinical work evaluating MRT5500 as well as other constructs against the emerging SARS-CoV-2 variants to inform the incorporation of variant strains into current or future development plans.
- Published COVID-19 mRNA vaccine preclinical results: In
April 2021, the Company announced the peer-reviewed publication of preclinical results of its COVID-19 vaccine candidate, MRT5500, in the journal npj Vaccines. Preclinical evaluation of MRT5500 demonstrated a favorable immune response profile against SARS-CoV-2 and protection against the disease in an infection challenge model.
- Reported results from second interim data analysis from ongoing Phase 1/2 clinical trial of MRT5005 in patients with CF: In evaluating safety and tolerability, the primary outcome measure, data to date from the ongoing Phase 1/2 clinical trial suggest that repeat dosing of MRT5005 was generally safe and well tolerated. Percent predicted forced expiratory volume in 1 second (ppFEV1), a measure of lung function, was assessed as a safety measure at pre-defined timepoints throughout the trial; no significant change in ppFEV1 was observed. The Phase 1/2 clinical trial is ongoing and the Company anticipates reporting the findings from the clinical trial, including an additional multiple-ascending dose (MAD) dose group (20 mg) and a daily dosing cohort (4 mg once-daily for 5 days), at a future medical meeting. The Company plans to continue with ongoing and additional translational studies with MRT5005 and its next-generation CF candidate to support and optimize future clinical development, including research into dosing, formulation and nebulization.
- Advanced preclinical pulmonary programs: The Company advanced its next-generation CF and primary ciliary dyskinesia (PCD) programs with positive preclinical data generated to support planned initiation of investigational new drug application (IND)-enabling studies in the second half of 2021.
Expansion of Leadership Team:
- Appointed Dr.
Rand Sutherlandas President: In March 2021, the Company announced Dr. Rand Sutherlandjoined the team as President. Dr. Sutherlandbrings 20 years of combined industry, medical and academic expertise in pulmonary and rare diseases to this role. As President of Translate Bio, Dr. Sutherlandis responsible for research and development, corporate development and program management, including portfolio and pipeline strategy with a focus on prioritizing and optimizing the Company’s R&D opportunities while evaluating corporate and business development prospects.
- Strengthened leadership team with key additions and promotions: In
April 2021, the Company announced several key leadership appointments, including Brendan Smithas Chief Financial Officer, Greg Troianoas Chief Manufacturing Officer, and Jim Sullivanas Senior Vice President, Pulmonary Discovery. These roles support the Company’s long-term strategic plan, TBIO 2025, that emphasizes several areas for value creation across the Company including adding key in-house quality and manufacturing capabilities, deepening pulmonary expertise and increasing R&D investments with a goal of advancing platform innovation and driving multiple programs into the clinic.
Anticipated 2021 Milestones
- COVID-19 Vaccine (MRT5500): Report interim results from the ongoing Phase 1/2 clinical trial in the third quarter of 2021 (Sanofi Pasteur collaboration)
- Influenza Vaccine: Advance lead lipid nanoparticle (LNP)/mRNA formulations to begin clinical proof-of-technology trial mid-year 2021 (Sanofi Pasteur collaboration)
- Additional Vaccine Programs: Advance ongoing preclinical studies for targets against additional viral and bacterial pathogens
- CF Clinical Program (MRT5005): Complete enrollment and dosing for the ongoing Phase 1/2 clinical trial and report data at a future medical meeting, including the 20 mg MAD group and a daily dosing cohort assessing 4 mg once-daily for 5 days
- CF Discovery Program (Next-generation CF):
- Advance lead candidate into IND-enabling studies in the second half of 2021
- Continue ongoing and additional translational studies with MRT5005 and a next-generation CF candidate to support and optimize future clinical development, including research into dosing, formulation and nebulization
- Primary Ciliary Dyskinesia (PCD): Advance lead candidate into IND-enabling studies in the second half of 2021
- Additional Pulmonary: Conduct preclinical studies to validate targets, including pulmonary arterial hypertension and respiratory infectious diseases
- Liver: Progress efforts to identify next-generation LNPs to enable advancement of Company's liver-directed therapeutic portfolio
- mRNA Platform:
- Delivery: Identify next-generation LNPs to support additional disease program development
- Next Wave Areas: Explore new areas for application of mRNA technology including therapeutic antibodies and protein degradation
Upcoming Scientific Presentations
- Preclinical Presentation in Primary Ciliary Dyskinesia at the
American Thoracic Society(ATS) 2021 International Conference: A Messenger RNA (mRNA)-Based Therapeutic Designed to Treat Primary Ciliary Dyskinesia (Abstract #A1138), Mini Symposium, May 18from 10:00– 11:30 am ET
Upcoming Investor Events
Jefferies Virtual Healthcare Conference, June 1–3
- 42nd Annual
Goldman Sachs Global Healthcare Conference, June 8–10
- 2nd Annual SVB Leerink CyberRx Series:
Vaccine Forum, June 21–22 William Blair Biotech Focus Conference2021, July 14–15
First Quarter 2021 Financial Results and Financial Guidance
Collaboration revenue was
Operating expenses for the three months ended
- Research and development expenses of
$41.1 millionduring the first quarter of 2021, compared to $21.4 millionfor the same period in 2020. The increase is primarily due to continued development of the Company’s discovery and vaccine programs as well as increases in occupancy and personnel-related costs.
- General and administrative expenses of
$10.8 millionduring the first quarter of 2021, compared to $7.5 millionfor the same period in 2020. The increase is primarily due to increases in recruiting fees, consulting and legal costs and an increase in personnel-related costs.
- Change in fair value of contingent consideration of
$44.0 millionrelated to future potential milestone and earnout payment obligations. This decrease was primarily driven by revisions to the clinical development plan for MRT5005.
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: Translate Bio’s plans to complete and report data from its Phase 1/2 clinical trial of MRT5005 for CF at a future medical meeting; plans to report interim data from its Phase 1/2 clinical trial of MRT5500 for COVID-19 in the third quarter of 2021; plans to commence clinical trials for an influenza vaccine mid-year 2021; plans to advance lead candidates for next-generation CF and PCD programs in the second half of 2021; plans to further advance platform innovation and drive multiple programs into the clinic; the goals and objectives of the TBIO 2025 long-term strategic plan; Translate Bio’s expectations with respect to its recent leadership appointments; the potential for mRNA-based therapeutics to apply to the treatment of many diseases caused by protein or gene dysfunction; Translate Bio’s beliefs regarding the broad applicability of its technology; Translate Bio’s plans to advance its pipeline of mRNA therapeutics and validate targets for additional pulmonary diseases and advance its liver-directed therapeutic portfolio; and the period in which
|Investor Relations||Media Relations|
|Tel.: +1 (617) 817-8655||Tel: +1 (617) 233-1154|
|CONSOLIDATED STATEMENTS OF OPERATIONS|
|Three Months Ended
|Research and development||41,140||21,439|
|General and administrative||10,817||7,458|
|Change in fair value of contingent consideration||(43,979||)||(9,452||)|
|Total operating expenses||7,978||19,445|
|Income (loss) from operations||26,622||(14,791||)|
|Other income, net||154||509|
|Income (loss) before income tax provision||26,776||(14,282||)|
|Income tax provision||(254||)||—|
|Net income (loss)||$||26,522||$||(14,282||)|
|CONSOLIDATED BALANCE SHEETS|
|Cash and cash equivalents||$||155,746||$||342,027|
|Prepaid expenses and other current assets||16,721||11,741|
|Total current assets||699,540||697,193|
|Property and equipment, net||16,563||15,372|
|Right-of-use assets, net||71,154||72,957|
|Intangible assets, net||77,106||79,127|
|Liabilities and Stockholders' Equity|
|Current portion of deferred revenue||72,373||67,563|
|Current portion of operating lease liability||12,084||11,733|
|Income tax liability||254||—|
|Total current liabilities||118,019||101,337|
|Deferred revenue, net of current portion||226,876||228,659|
|Operating lease liability, net of current portion||48,604||50,953|
|Additional paid-in capital||775,499||769,965|
|Accumulated other comprehensive income||77||—|
|Total stockholders' equity||388,890||356,757|
|Total liabilities and stockholders' equity||$||890,640||$||889,936|
Source: Translate Bio, Inc.