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
Dose expansion
study in the XIRIUS trial expected to begin enrollment in Q4
2018
WALTHAM, Mass. and LONDON, June 06, 2018 (GLOBE
NEWSWIRE) -- 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
Officer
Brian Luque, Sr. Manager, Investor Relations
investors@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 |
|
|
|
|
|
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: Nightstar Therapeutics via Globenewswire
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