Translate Bio Announces Fourth Quarter and Full Year 2019 Financial Results and Highlights Recent Progress
“2019 was a year of important progress for
Renaud continued, “On the preclinical side, we have ongoing discovery efforts focused on a next-generation CF program, and beyond CF we continue our work to identify lead product candidates in additional pulmonary diseases, including primary ciliary dyskinesia, idiopathic pulmonary fibrosis and pulmonary arterial hypertension. We are excited about the advancements we’ve made in our pipeline programs and look forward to sharing more details throughout this year.”
Development Program Progress and Updates
- The Company presented data at the
North American Cystic Fibrosis Conferencefrom the SAD portion of the Phase 1/2 clinical trial of MRT5005 in 12 patients with CF. MRT5005 was generally well-tolerated at low and mid-dose levels with no serious adverse events reported at any dose level. Marked increases in percent predicted forced expiratory volume in one second (ppFEV1) were observed after a single dose of MRT5005, primarily at the mid-dose level.
- The Company continues to enroll and dose patients in the ongoing Phase 1/2 clinical trial of MRT5005 in patients with CF and anticipates reporting data for the additional SAD dose group and the MAD portion of the trial in the third quarter of 2020.
U.S. Food and Drug Administration(FDA) has granted Fast Track designation for MRT5005 for the treatment of CF. This designation facilitates the expedited review of drugs that are intended to treat serious or life-threatening conditions and demonstrates the potential to address unmet medical needs.
- To support expansion of the Company’s pipeline opportunities in CF, discovery activities are underway to identify a next-generation CF product candidate with efforts focused on novel lipid nanoparticles (LNPs), protein engineering approaches and manufacturing process enhancements. In vivo studies are ongoing to evaluate preclinical safety, protein expression and duration of expression to support product candidate selection.
Additional Pulmonary Programs
- IPF is a chronic lung disorder characterized by thickening, stiffening and scarring, or fibrosis, of tissue within the lungs. There are approximately 83,000 diagnosed cases of IPF in
the United States. Translate Bio’s preclinical discovery efforts in IPF are primarily focused on delivering siRNA to the lung to knock down the target protein to potentially provide clinical benefit. Preclinical studies are ongoing to demonstrate proof-of-concept to support product candidate selection.
- PCD is an autosomal recessive genetic condition in which clearance of mucus from the respiratory tract is impaired due to defects in ciliary function. Cilia are tiny, hair-like structures on the cells that line the airways. Mutations in more than 30 genes are known to cause PCD and there are approximately 16,000 diagnosed cases of PCD in
the United States. Translate Biointends to use its mRNA platform to potentially restore ciliary function in the lungs of patients with PCD. The Company is conducting preclinical studies in multiple PCD genes to demonstrate proof-of-concept and support the selection of a lead PCD program.
- PAH is a rare, progressive disorder characterized by narrowing of the small arteries of the lungs resulting in increased blood pressure through the lungs, which can damage the heart. There are approximately 53,000 diagnosed cases of PAH in
the United States. In developing an mRNA product candidate for PAH, Translate Biointends to use its MRT platform to produce a number of protein targets that could potentially slow the progression of the disease. Preclinical studies are underway to evaluate and validate target proteins for this disease.
Sanofi Pasteur Collaboration
Translate Biocontinues to work with collaborators at Sanofi Pasteurto develop mRNA vaccines in infectious diseases and has advanced its preclinical development programs including screening, optimization and production of mRNA and LNP formulations across multiple targets. Preclinical studies are being conducted with lead candidates to support an anticipated investigational new drug (IND) filing in 2021.
Lipid Nanoparticle Delivery Discovery
- A robust effort to discover proprietary next-generation delivery technologies is ongoing. Using in-house, high-throughput LNP production, as well as working with external collaborators,
Translate Biois generating novel LNPs and optimizing delivery formulations, resulting in an extensive library of LNPs. In vivo studies are being conducted to identify lead next-generation LNPs to support lung, liver and additional disease program development.
Fourth Quarter and Full Year 2019 Financial Results and Financial Guidance
Collaboration revenue was
Operating expenses for the three months ended
- Research and development expenses of
$25.0 millionduring the fourth quarter of 2019, compared to $17.2 millionfor the same period in 2018. The increase is primarily due to an increase in costs associated with the continued advancement of the Phase 1/2 trial of MRT5005 for the treatment of patients with CF and continued development of the vaccine programs as well as an increase in personnel-related costs.
- General and administrative expenses of
$7.3 millionduring the fourth quarter of 2019, compared to $5.9 millionfor the same period in 2018. The increase is primarily due to an increase in personnel-related costs and professional fees.
- Operating expense of $3.3 million for changes in the fair value of contingent consideration related to future potential milestone and earnout payment obligations. The operating expense was attributed to an increase in the fair value of the contingent consideration liability related to the CF program due to its continued progress and the time value of money due to passage of time.
Operating expenses for the year ended
- Research and development expenses of
$76.4 millionduring the year ended December 31, 2019, compared to $58.0 millionfor the same period in 2018. The increase is primarily due to an increase in costs associated with the continued advancement of the Phase 1/2 trial of MRT5005 for the treatment of patients with CF and continued development of MRT discovery and vaccine programs as well as an increase in personnel-related costs.
- General and administrative expenses of
$28.6 millionduring the year ended December 31, 2019, compared to $22.6 millionfor the same period in 2018. The increase is primarily due to an increase in personnel-related costs.
- Operating expense of less than
$0.1million for changes in the fair value of contingent consideration related to future potential milestone and earnout payment obligations, compared to $25.0 millionfor the same period in 2018. The operating expense in 2019 was attributed primarily to an increase in the fair value of the contingent consideration liability related to the CF program due to its continued progress and the time value of money due to passage of time offset by the decision to discontinue the ornithine transcarbamylase (OTC) deficiency program, which the Company discontinued in the third quarter of 2019 and which resulted in the removal of $23.2 million in contingent consideration liability related to the OTC deficiency program.
- An impairment charge of $18.6 million representing the value of the indefinite-lived in-process research and development intangible asset related to the discontinuation of the OTC deficiency program.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the potential for MRT5005 to address the underlying cause of CF and benefit patients; Translate Bio’s plans to report data from the additional SAD dose group and MAD portion of the Phase 1/2 clinical trial of MRT5005 in the third quarter of 2020; Translate Bio’s plans to advance its pipeline of mRNA therapeutics and validate targets for additional pulmonary diseases; Translate Bio’s expectations with respect to its collaboration with Sanofi and the anticipated IND filing in 2021; the period in which
|CONSOLIDATED STATEMENTS OF OPERATIONS|
|Three Months Ended
|Research and development||25,025||17,170||76,369||58,024|
|General and administrative||7,348||5,879||28,632||22,606|
|Change in fair value of contingent consideration||3,256||(14,569||)||13||25,020|
|Impairment of intangible asset||—||—||18,559||—|
|Total operating expenses||35,629||8,480||123,573||105,650|
|Loss from operations||(31,739||)||(7,298||)||(115,769||)||(104,230||)|
|Other income (expense):|
|Total other income (expense), net||703||823||1,990||1,270|
|Loss before benefit from income taxes||(31,036||)||(6,475||)||(113,779||)||(102,960||)|
|Benefit from income taxes||—||438||486||5,565|
|CONSOLIDATED BALANCE SHEETS|
|Cash and cash equivalents||$||84,580||$||55,199|
|Short-term collaboration receivables||4,596||833|
|Prepaid expenses and other current assets||9,391||3,641|
|Total current assets||203,615||149,602|
|Property and equipment, net||12,539||10,245|
|Right-of-use assets, net||10,400||—|
|Intangible assets, net||85,536||106,445|
|Liabilities and Stockholders' Equity|
|Current portion of deferred revenue||18,100||2,572|
|Current portion of operating lease liability||530||—|
|Total current liabilities||41,670||14,287|
|Deferred revenue, net of current portion||25,256||41,841|
|Deferred tax liabilities||—||481|
|Operating lease liability, net of current portion||12,084||—|
|Additional paid-in capital||512,231||371,257|
|Accumulated other comprehensive income||741||196|
|Total stockholders' equity||153,536||125,295|
|Total liabilities and stockholders' equity||$||336,201||$||287,651|
Source: Translate Bio, Inc.