Translate Bio Announces Fourth Quarter and Full Year 2019 Financial Results and Highlights Recent Progress
March 12 2020 - 4:45PM
Translate Bio (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA)
therapeutics company developing a new class of potentially
transformative medicines to treat diseases caused by protein or
gene dysfunction, today announced financial results and business
highlights for the fourth quarter and full year ended December
31, 2019.
“2019 was a year of important progress for Translate Bio as we
reported positive interim data from the single-ascending dose (SAD)
portion of the Phase 1/2 clinical trial of MRT5005 in patients with
cystic fibrosis (CF). We look forward to sharing data from the
additional SAD dose group and the multiple-ascending dose (MAD)
portion of the trial in the third quarter of this year,” said
Ronald Renaud, chief executive officer of Translate Bio. “As the
first inhaled mRNA therapeutic designed to deliver mRNA coding for
the cystic fibrosis transmembrane conductance regulator (CFTR)
protein, MRT5005 has the potential to treat all patients with CF,
including those considered non-amenable to currently available CFTR
modulator treatments. This first-in-human clinical trial is a
critical step towards our goal of addressing that unmet need.”
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
Cystic Fibrosis
- The Company presented data at the North American Cystic
Fibrosis Conference from 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.
- The 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
Translate Bio is leveraging its lung delivery expertise towards
research in additional pulmonary diseases including idiopathic
pulmonary fibrosis (IPF), primary ciliary dyskinesia (PCD) and
pulmonary arterial hypertension (PAH).
- 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 Bio
intends 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 Bio
intends 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 Bio continues to work with collaborators at Sanofi
Pasteur to 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 Bio is 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
Translate Bio ended the fourth quarter of 2019 with $188.7
million in cash, cash equivalents and short-term investments and
60,022,067 shares of common stock outstanding. The Company expects
that its existing cash, cash equivalents and short-term investments
will enable it to fund its operating expenses and capital
expenditure requirements into the second quarter of 2021.
Translate Bio reported a net loss of $31.0 million and $6.0
million for the three months ended December 31, 2019 and 2018,
respectively, and a net loss of $113.3 million and $97.4 million
for the years ended December 31, 2019 and 2018, respectively.
Collaboration revenue was $3.9 million and $1.2 million for the
three months ended December 31, 2019 and 2018, respectively, and
$7.8 million and $1.4 million for the years ended December 31, 2019
and 2018, respectively. The collaboration revenue was derived from
the collaboration and license agreement that the Company entered
into with Sanofi Pasteur in 2018. The increase was related to the
advancement of the vaccine program during the year ended December
31, 2019 compared to the same period in 2018.
Operating expenses for the three months ended December 31, 2019
were $35.6 million, compared to $8.5 million for the same period in
2018, and were comprised of the following:
- Research and development expenses of $25.0 million during the
fourth quarter of 2019, compared to $17.2 million for 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 million during the
fourth quarter of 2019, compared to $5.9 million for 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 December 31, 2019 were
$123.6 million, compared to $105.7 million for the same period in
2018, and were comprised of the following:
- Research and development expenses of $76.4 million during the
year ended December 31, 2019, compared to $58.0 million for 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 million during the
year ended December 31, 2019, compared to $22.6 million for the
same period in 2018. The increase is primarily due to an increase
in personnel-related costs.
- Operating expense of less than $0.1 million for changes in
the fair value of contingent consideration related to future
potential milestone and earnout payment obligations, compared to
$25.0 million for 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.
About Translate BioTranslate Bio is a
clinical-stage mRNA therapeutics company developing a new class of
potentially transformative medicines to treat diseases caused by
protein or gene dysfunction. Translate Bio is primarily
focused on applying its technology to treat pulmonary diseases
caused by insufficient protein production or where the reduction of
proteins can modify disease. Translate Bio’s lead program is
being developed as a treatment for cystic fibrosis (CF) and is in
an ongoing Phase 1/2 clinical trial. The Company also believes its
technology is applicable to a broad range of diseases, including
diseases that affect the liver. Additionally, the platform may be
applied to various classes of treatments, such as therapeutic
antibodies or vaccines in areas such as infectious disease and
oncology. For more information about the Company, please visit
www.translate.bio or on Twitter at @TranslateBio.
Cautionary Note Regarding Forward-Looking
StatementsThis 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 Translate Bio expects that its existing
cash, cash equivalents and short-term investments 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,” “forward,” “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: Translate Bio’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 FDA, other regulatory authorities
and investigational review boards at clinical trial sites,
including decisions as it relates to ongoing and planned clinical
trials; Translate Bio’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 set forth under the caption “Risk
Factors” in Translate Bio’s Annual Report on Form 10-K for
the fiscal year ended December 31, 2019 filed with the Securities
and Exchange Commission on March 12, 2020 and in any other
subsequent filings made by Translate Bio. Any forward-looking
statements contained in this press release speak only as of the
date hereof, and Translate Bio specifically disclaims any
obligation to update any forward-looking statement, whether as a
result of new information, future events or otherwise.
TRANSLATE
BIO, INC. |
|
CONSOLIDATED
STATEMENTS OF OPERATIONS |
|
(IN
THOUSANDS) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended December 31, |
|
Years Ended December 31, |
|
|
|
|
2019 |
|
|
|
2018 |
|
|
|
2019 |
|
|
|
2018 |
|
|
Collaboration revenue |
|
$ |
3,890 |
|
|
$ |
1,182 |
|
|
$ |
7,804 |
|
|
$ |
1,420 |
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
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): |
|
|
|
|
|
|
|
|
|
Interest income |
|
|
703 |
|
|
|
825 |
|
|
|
2,010 |
|
|
|
1,323 |
|
|
Other expense |
|
|
— |
|
|
|
(2 |
) |
|
|
(20 |
) |
|
|
(53 |
) |
|
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 |
|
|
Net
loss |
|
$ |
(31,036 |
) |
|
$ |
(6,037 |
) |
|
$ |
(113,293 |
) |
|
$ |
(97,395 |
) |
|
|
|
|
|
|
|
|
|
|
|
TRANSLATE
BIO, INC. |
|
|
CONSOLIDATED
BALANCE SHEETS |
|
|
(IN
THOUSANDS) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December
31, |
December
31, |
|
|
|
|
|
|
|
|
|
2019 |
|
|
|
2018 |
|
|
|
Assets |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
Cash and cash equivalents |
$ |
84,580 |
|
|
$ |
55,199 |
|
|
|
|
|
Short-term investments |
|
104,098 |
|
|
|
88,904 |
|
|
|
|
|
Short-term collaboration receivables |
|
4,596 |
|
|
|
833 |
|
|
|
|
|
Prepaid expenses and other current assets |
|
9,391 |
|
|
|
3,641 |
|
|
|
|
|
Restricted cash |
|
950 |
|
|
|
1,025 |
|
|
|
|
|
|
|
Total current assets |
|
203,615 |
|
|
|
149,602 |
|
|
|
Property and equipment, net |
|
12,539 |
|
|
|
10,245 |
|
|
|
Right-of-use assets, net |
|
10,400 |
|
|
|
— |
|
|
|
Goodwill |
|
|
|
21,359 |
|
|
|
21,359 |
|
|
|
Intangible assets, net |
|
85,536 |
|
|
|
106,445 |
|
|
|
Other assets |
|
|
2,752 |
|
|
|
— |
|
|
|
|
|
|
|
Total assets |
$ |
336,201 |
|
|
$ |
287,651 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Liabilities and Stockholders' Equity |
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
Accounts payable |
$ |
15,968 |
|
|
$ |
5,168 |
|
|
|
|
|
Accrued expenses |
|
7,072 |
|
|
|
6,547 |
|
|
|
|
|
Current portion of deferred revenue |
|
18,100 |
|
|
|
2,572 |
|
|
|
|
|
Current portion of operating lease liability |
|
530 |
|
|
|
— |
|
|
|
|
|
|
|
Total current liabilities |
|
41,670 |
|
|
|
14,287 |
|
|
|
Contingent consideration |
|
103,655 |
|
|
|
103,642 |
|
|
|
Deferred revenue, net of current portion |
|
25,256 |
|
|
|
41,841 |
|
|
|
Deferred tax liabilities |
|
— |
|
|
|
481 |
|
|
|
Deferred rent |
|
|
— |
|
|
|
2,105 |
|
|
|
Operating lease liability, net of current portion |
|
12,084 |
|
|
|
— |
|
|
|
|
|
|
|
Total liabilities |
|
182,665 |
|
|
|
162,356 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Stockholders’ equity: |
|
|
|
|
|
|
|
Common stock |
|
60 |
|
|
|
45 |
|
|
|
|
|
Additional paid-in capital |
|
512,231 |
|
|
|
371,257 |
|
|
|
|
|
Accumulated deficit |
|
(359,496 |
) |
|
|
(246,203 |
) |
|
|
|
|
Accumulated other comprehensive income |
|
741 |
|
|
|
196 |
|
|
|
|
|
|
|
Total stockholders' equity |
|
153,536 |
|
|
|
125,295 |
|
|
|
|
|
|
|
Total liabilities and stockholders' equity |
$ |
336,201 |
|
|
$ |
287,651 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Contacts for Translate Bio |
Investors Teri Dahlman
tdahlman@translate.bio 857-242-7792 |
MediaMaura
Gavaghanmgavaghan@translate.bio 857-242-7789 |
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