Translate Bio Announces First Quarter 2019 Financial Results and Provides Corporate Update
-- Completed dosing in single-ascending dose portion of Phase 1/2 clinical trial in cystic fibrosis; data expected 3Q19 --
“This quarter, we have continued to focus on progressing our programs for rare genetic diseases while also advancing our mRNA therapeutic (MRT) platform to fuel future pipeline programs,” said Ronald Renaud, chief executive officer of
First Quarter 2019 and Recent Updates
- Completed dosing in single-ascending dose (SAD) portion of Phase 1/2 clinical trial of MRT5005 for the treatment of cystic fibrosis(CF):
Translate Biocompleted dosing of all patients in the three dose cohorts of the SAD 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. The Company now expects to report interim data from this trial in the third quarter of 2019. Patient dosing continues in the ongoing multiple-ascending dose portion of this trial. Details related to this clinical trial can be found at www.clinicaltrials.gov (NCT03375047).
$47.5Mprivate placement: In May 2019, Translate Bioentered into a securities purchase agreement with several institutional accredited investors for the private placement of 5,582,940 shares of common stock at a purchase price of $8.50per share, yielding gross proceeds of $47.5 million.
- Presented preclinical data at ASGCT demonstrating successful delivery of lipid-nanoparticle encapsulated mRNA in relevant metabolic disease models: In
April 2019at the American Society of Gene and Cell Therapy(ASGCT) Annual Meeting, Translate Biopresented preclinical results that demonstrate the potential of mRNA therapeutics as treatments for urea cycle disorders and organic acidemias using the Company’s proprietary liver delivery technology. The presentation summarized findings from experiments in three mouse models of metabolic disorders including ornithine transcarbamylase (OTC) deficiency, argininosuccinate synthetase (ASS1) deficiency (citrullinemia) and methylmalonic acidemia (MMA), which are each caused by a deficiency of a single enzyme specific for each disease.
- Strengthened leadership and board of directors with recent appointments: In April, the Company announced the appointments of Dr.
Richard Woosteras chief scientific officer and Pat Saccoas senior vice president of technical operations. The Company also announced the appointments of Dr. Robert Meyerand Dr. Robert Plengeto its board of directors. These appointments strengthen Translate Bio’s leadership team and highlight the Company’s commitment to innovative mRNA research and development.
- MRT5005 (CF): Interim data from Phase 1/2 clinical trial in the third quarter of 2019
- MRT5201 (OTC Deficiency): Complete additional preclinical studies and submit data from these studies to the
FDAin fourth quarter of 2019
- Identify lead preclinical candidates for additional lung and liver disease targets
Frank DeRosa, Translate Bio’s senior vice president of Research and Development, will give a presentation entitled “Advancing the Delivery of LNP-encapsulated mRNA Therapeutics” in The State of the Art in mRNA Delivery session at the TIDES 2019 Conference on Monday, May 20, 2019at 10:15 a.m. PTin San Diego, California.
- The Company will give a corporate presentation and host one-on-one meetings at the Jefferies 2019 Global Healthcare Conference taking place
June 4-7at the Grand Hyattin New York, New York.
First Quarter 2019 Financial Results and Financial Guidance
Collaboration revenue was
Operating expenses for the three months ended
- Research and development expenses of
$17.4 millionduring the first quarter of 2019, compared to $12.7 millionfor the same period in 2018. The increase is primarily due to an increase in costs associated with the continued advancement of the Company’s CF program and MRT discovery program.
- General and administrative expenses of
$6.6 millionduring the first quarter of 2019, compared to $4.8 millionfor the same period in 2018. The increase is primarily due to an increase in personnel-related costs.
- In the three months ended
March 31, 2019and 2018, the Company recognized non-cash operating expenses of $11.7 millionand $4.9 million, respectively, for changes in the fair value of contingent consideration liabilities, related to future potential milestone and earnout payment obligations and, prior to the IPO, anti-dilution rights with respect to common stock issued to Shire. The increase is attributed primarily to the progress of the Company’s CF and OTC deficiency programs, the time value of money due to the passage of time, as well as an adjustment in the discount rate.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the potential for MRT5005 to address the underlying cause of CF; Translate Bio’s plans to report interim data from the Phase 1/2 clinical trial of MRT5005 in the third quarter of 2019; Translate Bio’s plans to conduct additional preclinical studies for MRT5201 and its plan to submit data from these studies to the
|TRANSLATE BIO, INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|Three Months Ended March 31,|
|Research and development||17,423||12,702|
|General and administrative||6,554||4,779|
|Change in fair value of contingent consideration||11,702||4,908|
|Total operating expenses||35,679||22,389|
|Loss from operations||(34,205||)||(22,389||)|
|Other income (expense):|
|Total other income (expense), net||521||77|
|Loss before benefit from income taxes||(33,684||)||(22,312||)|
|Benefit from income taxes||486||1,103|
|TRANSLATE BIO, INC.|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|March 31,||December 31,|
|Cash and cash equivalents||$||62,431||$||55,199|
|Prepaid expenses and other current assets||5,247||4,474|
|Total current assets||128,857||149,602|
|Property and equipment, net||10,771||10,245|
|Right-of-use assets, net||10,769||—|
|Intangible assets, net||105,980||106,445|
|Liabilities and Stockholders' Equity|
|Current portion of deferred revenue||5,708||2,572|
|Current portion of operating lease liability||411||—|
|Total current liabilities||16,763||14,287|
|Long-term portion of contingent consideration||115,344||103,642|
|Deferred revenue, net of current portion||38,017||41,841|
|Deferred tax liabilities||—||481|
|Operating lease liability, net of current portion||12,504||—|
|Additional paid-in capital||374,113||371,257|
|Accumulated other comprehensive income||351||196|
|Total stockholders' equity||95,108||125,295|
|Total liabilities and stockholders' equity||$||277,736||$||287,651|
|Teri Dahlman||Maura Gavaghan|
Source: Translate Bio, Inc.