Nightstar Therapeutics Reports First Quarter 2018 Financial Results and Acceleration of Preliminary NSR-RPGR Readout to Third...
June 06 2018 - 5:35AM
Preliminary data from dose escalation study in
the Phase 1/2 XIRIUS trial for X-Linked Retinitis Pigmentosa
expected ahead of schedule in Q3 2018
Nightstar Therapeutics plc (NASDAQ:NITE), a clinical-stage gene
therapy company developing treatments for rare inherited retinal
diseases, today reported financial results for the quarter ended
March 31, 2018 and provided an update on recent achievements and
upcoming clinical milestones.
“We started 2018 with a steadfast commitment to execution,” said
Dave Fellows, Chief Executive Officer. “We’re pleased to announce
that our program for the treatment of X-Linked Retinitis Pigmentosa
(XLRP) is ahead of schedule. In the third quarter of this year, we
expect to announce preliminary data from the dose escalation study
of the Phase 1/2 XIRIUS trial and, in the fourth quarter of this
year, begin enrollment in the expansion study in XIRIUS. This
follows our achievement earlier this year of initiating our Phase 3
STAR trial in choroideremia ahead of schedule. We look forward to
continuing our mission to cure inherited retinal diseases.”
Anticipated Milestones for 2018 and 2019
- NSR-RPGR for X-Linked Retinitis Pigmentosa
- Q3 2018: Preliminary Data from Dose Escalation
Study. Preliminary efficacy and safety data of NSR-RPGR
from the dose escalation study in the XIRIUS trial is expected to
be presented at an upcoming medical meeting.
- Q4 2018: Initiation of Expansion Study. The
expansion study in the XIRIUS trial is intended to enroll
approximately 30 patients at a therapeutic dose informed by the
dose escalation study. It is anticipated that the expansion study
will include a low-dose control group of approximately 15
patients.
- Mid 2019: Preliminary Data from Expansion
Study
- 2H 2019: One-Year Follow-up Data from Dose Escalation
Study
- 2020: One-Year Follow-up Data from Expansion
Study
- NSR-REP1 for Choroideremia
- 1H 2019: Completion of Enrollment for Phase 3 STAR
Registrational Trial for Choroideremia
- 2020: One-year Follow-up Data from Phase 3 STAR
Trial
First Quarter 2018 Financial Results
Research and development expenses for the quarter ended March
31, 2018 were $6.1 million versus $2.8 million for the quarter
ended March 31, 2017. The increase of $3.3 million resulted from
increases in program-related expenses of $1.0 million for NSR-REP1
and $1.4 million for NSR-RPGR, as well as a $1.4 million increase
in personnel-related costs, partially offset by an increase of $0.7
million of research and development tax credit from HM Revenue
& Customs in the United Kingdom. Research and development
personnel-related costs increased due to an increase in headcount
in the first quarter of 2018 to support our growth and to assist in
the further development of our product candidates and pipeline. The
increase in research and development personnel-related costs
includes $0.2 million of additional non-cash share-based
compensation compared to the same period in 2017.
General and administrative expenses for the quarter ended March
31, 2018 were $2.8 million, compared to $0.7 million for the
quarter ended March 31, 2017. The increase of $2.1 million was
mainly due to a $0.7 million increase in consulting and
professional fees including increased legal, accounting and audit
fees, and a $1.1 million increase in personnel-related costs.
General and administrative personnel-related costs increased due to
an increase in employees to support our increased research and
development activities and our status as a public company.
The increase in general and administrative personnel-related costs
included $0.5 million of additional non-cash share-based
compensation compared to 2017.
Net loss for the quarter ended March 31, 2018 was $14.4 million,
or $0.52 basic and diluted net loss per ordinary share, as compared
to $3.5 million for the quarter ended March 31, 2017, or $0.30
basic and diluted net loss per ordinary share.
As of March 31, 2018, our cash and cash equivalents totaled
$122.3 million, compared to $129.4 million at December 31, 2017. As
of March 31, 2018, we had approximately 28.9 million ordinary
shares outstanding.
About Nightstar
Nightstar is a leading clinical-stage gene
therapy company focused on developing and commercializing novel
one-time treatments for patients suffering from rare inherited
retinal diseases that would otherwise progress to blindness.
Nightstar’s lead product candidate, NSR-REP1, is currently in Phase
3 development for the treatment of patients with choroideremia, a
rare, degenerative, genetic retinal disorder that has no current
treatments and affects approximately one in every 50,000 people.
Positive results from a Phase 1/2 trials of NSR-REP1 were published
in The Lancet in 2014 and in The New England Journal of Medicine in
2016. Nightstar’s second product candidate, NSR-RPGR, is
currently being evaluated in a clinical trial known as the XIRIUS
trial for the treatment of patients with X-linked retinitis
pigmentosa, an inherited X-linked recessive retinal disease that
affects approximately one in every 40,000 people.
For more information about Nightstar or its clinical trials,
please visit www.nightstartx.com.
Cautionary Language Concerning Forward-Looking
Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995. The words “believe,” “anticipate,” “intend,” “estimate,”
“will,” “may,” “should,” “expect” or other similar expressions are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. All
statements contained in this press release other than statements of
historical facts are forward-looking statements, including, without
limitation: statements about our results of operations for the
first quarter of 2018, cash position and sufficiency of capital
resources to fund our operating requirements, our planned and
ongoing clinical trials for NSR-REP1 and NSR-RPGR, including our
Phase 3 STAR trial in choroideremia, the dose escalation study in
the XIRIUS trial for X-linked Retinitis Pigmentosa and the planned
expansion study in the XIRIUS trial, the continued clinical
development of our pipeline, the timelines associated with our
research and development programs including the timing of patient
enrollment and the release of data from ongoing clinical trials and
studies, the prevalence of patient populations for our targeted
indications, and the utility of prior preclinical and clinical data
in determining future clinical results. These forward-looking
statements are based on management's current expectations of future
events and are subject to a number of involve substantial known and
unknown risks, uncertainties and other factors that may cause our
actual results, levels of activity, performance or achievements to
be materially different from the information expressed or implied
by these forward-looking statements, including the risks and
uncertainties set forth in Item 3.D. "Risk Factors" section of our
Annual Report on Form 20-F for the year ended December 31, 2017 and
subsequent reports that we file with the U.S. Securities and
Exchange Commission We may not actually achieve the plans,
intentions, estimates or expectations disclosed in our
forward-looking statements, and you should not place undue reliance
on our forward-looking statements. Actual results or events could
differ materially from the plans, intentions, estimates and
expectations disclosed in the forward-looking statements we make.
We anticipate that subsequent events and developments will cause
our views to change. We are under no duty to update any of these
forward-looking statements after the date of this press release to
conform these statements to actual results or revised expectations,
except as required by law. You should, therefore, not rely on
these forward-looking statements as representing our views as of
any date subsequent to the date of this press release.
Investors:Senthil Sundaram, Chief Financial
OfficerBrian Luque, Sr. Manager, Investor
Relationsinvestors@nightstartx.com
|
NIGHTSTAR THERAPEUTICS
PLC |
|
|
|
|
|
Consolidated Statements of Operations and
Comprehensive Loss |
(In thousands, except share and per share
amounts) |
|
|
|
|
|
|
|
Three Months Ended March
31, |
|
|
|
2018 |
|
|
|
2017 |
|
Operating expenses: |
|
|
|
|
Research
and development |
|
$ |
6,064 |
|
|
$ |
2,750 |
|
General
and administrative |
|
|
2,776 |
|
|
|
730 |
|
Total
operating expenses |
|
|
8,840 |
|
|
|
3,480 |
|
Other
income (expense): |
|
|
|
|
Interest
and other income |
|
|
367 |
|
|
|
4 |
|
Other
expense, net |
|
|
(5,885 |
) |
|
|
4 |
|
Total
other income (expense), net |
|
|
(5,518 |
) |
|
|
8 |
|
Net
loss |
|
|
(14,358 |
) |
|
|
(3,472 |
) |
|
|
|
|
|
Other
comprehensive loss: |
|
|
|
|
Foreign
exchange translation adjustment |
|
|
6,346 |
|
|
|
143 |
|
Total
comprehensive loss |
|
$ |
(8,012 |
) |
|
$ |
(3,329 |
) |
Basic
and diluted net loss per ordinary share |
|
$ |
(0.52 |
) |
|
$ |
(0.30 |
) |
Weighted
average basic and diluted ordinary shares |
|
|
27,861,709 |
|
|
|
11,679,707 |
|
|
|
|
|
|
|
NIGHTSTAR THERAPEUTICS
PLC |
|
|
|
|
|
Consolidated Balance Sheets |
(In thousands) |
|
|
|
|
|
|
|
March 31, |
|
December 31, |
|
|
|
2018 |
|
|
2017 |
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and
cash equivalents |
|
$ |
122,277 |
|
$ |
129,404 |
Prepaid
expenses and other assets |
|
|
6,135 |
|
|
5,438 |
Total
current assets |
|
|
128,412 |
|
|
134,842 |
Property
and equipment, net |
|
|
352 |
|
|
355 |
Total
assets |
|
$ |
128,764 |
|
$ |
135,197 |
Liabilities and shareholders’ equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts
payable |
|
$ |
5,148 |
|
$ |
3,196 |
Accrued
expenses and other liabilities |
|
|
4,980 |
|
|
6,189 |
Total
current liabilities |
|
|
10,128 |
|
|
9,385 |
Total
liabilities |
|
|
10,128 |
|
|
9,385 |
|
|
|
|
|
Total
shareholders’ equity |
|
|
118,636 |
|
|
125,812 |
Total
liabilities and shareholders’ equity |
|
$ |
128,764 |
|
$ |
135,197 |
|
|
|
|
|
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