- Recent initiation of STAR phase 3 trial in
Choroideremia -
Nightstar Therapeutics plc (NASDAQ:NITE), a clinical-stage gene
therapy company developing treatments for rare inherited retinal
diseases, today reported financial results for the year ended
December 31, 2017 and provided an update on recent corporate and
clinical highlights.
“2017 was an exceptional year for the company,” said Dave
Fellows, Chief Executive Officer. “We set the stage for the recent
initiation of the first-ever Phase 3 trial in choroideremia and
today announced further data supporting the durability of the
treatment effect for NSR-REP1 in choroideremia. Elsewhere in our
pipeline, we initiated our Phase 1/2 gene therapy trial for
X-linked retinitis pigmentosa and in-licensed our program for
Stargardt disease. We also raised more than $130 million of gross
proceeds from our successful Series C financing and IPO to continue
our pioneering research and development activities.”
Business Highlights Include
- Initiated First-Ever Phase 3 Trial for Choroideremia
(NSR-REP1). The STAR Phase 3 registrational trial is
expected to enroll approximately 140 patients across 18 clinical
sites in the United States, Europe, Canada and South America, of
which six sites will be surgical centers. Eligible patients will be
randomized into one of three study arms: 56 patients receiving a
high-dose of NSR-REP1 in one-eye; 28 patients receiving a low-dose
of NSR-REP1 in one-eye; and 56 patients receiving no treatment
(no-sham, parallel control arm). The primary endpoint of the STAR
trial is the proportion of patients with an improvement of at least
15 ETDRS letters from baseline in visual acuity at 12 months
post-treatment. The primary endpoint will compare patients in the
high-dose treatment arm with patients in the control arm. We
anticipate that the STAR trial will be fully enrolled by the first
half of 2019.
- NSR-REP1 Data Supporting the Durability of Treatment
Effect to Be Presented at ARVO 2018
(NSR-REP1). After two years of follow-up in
the Oxford Trial, the median visual acuity (n=14) improved by 4.5
letters in the treated eyes compared with a loss of 1.5 letters in
the untreated eyes. At the last follow up (ranging from two to five
years), visual acuity had been maintained or had increased in all
12 treatment eyes that received gene therapy as per protocol,
compared to four of the 12 untreated eyes. (link to abstract)
- Natural History Data Increases Understanding of 2.5x
Greater Risk of Untreated Disease Progression in
Choroideremia. Preliminary top-line data from the
NIGHT natural history observational study at 20 months follow-up
(the final time point in the NIGHT study) indicated that 22% of
choroideremia eyes experienced a loss in visual acuity of five or
more ETDRS letters, compared to less than 8% for patients treated
with the high-dose of NSR-REP1. This data further supports
the importance of developing new treatments for choroideremia to
reverse or maintain the loss of visual acuity due to the natural
progression of disease.
- Initiated Phase 1/2 Gene Therapy Clinical Trial for
X-Linked Retinitis Pigmentosa (XLRP). Our second
retinal gene therapy product candidate, NSR-RPGR, is in a
dose-ranging Phase 1/2 clinical trial for the treatment of XLRP in
the United Kingdom and United States. We are
evaluating multiple doses and have completed dosing of the first
few cohorts of three patients each. We expect the initial data on
safety and tolerability from the dose escalation cohorts of this
trial to be available towards the end of 2018; this data will
determine the dose for study in an expansion cohort anticipated to
include up to 30 patients.
- Expanded Inherited Retinal Disease Pipeline with Novel
Gene Therapy for the Treatment of Stargardt Disease. We
recently in-licensed NSR-ABCA4 for the treatment of Stargardt
disease, utilizing a novel technology developed by the University
of Oxford. Preclinical proof-of-concept studies in the Abca4-/-
murine model of Stargardt disease have demonstrated the expression,
localization and function of the ABCA4 protein. Stargardt disease
is the most common form of inherited juvenile macular dystrophy
with a prevalence of one in 10,000 people. There are no treatments
currently available for Stargardt disease. This program will
leverage Nightstar’s capabilities, physician relationships and
clinical experience with NSR-REP1 and NSR-RPGR.
Financial Results
Research and development expenses for the year ended December
31, 2017 were $20.5 million versus $10.2 million for the year ended
December 31, 2016. The increase of $10.3 million resulted from
increases in program-related expenses of $3.4 million for NSR-REP1,
and $1.5 million for NSR-RPGR, as well as a $1.9 million increase
in personnel-related costs, and a $1.3 million expense to reduce
research and development tax relief claims receivable from HM
Revenue & Customs in the United Kingdom. Research and
development personnel costs increased due to additional employees
hired in 2017 to support our growing company and to assist in the
further development of our product candidates and pipeline.
This increase in research and development personnel-related costs
includes $0.5 million of additional non-cash stock-based
compensation compared to 2016.
General and administrative expenses for the year ended December
31, 2017 were $7.0 million, compared to $2.1 million for the
year ended December 31, 2016. The increase of $4.9 million is
mainly due to a $2.1 million increase in consulting and
professional fees, including increased legal, accounting and audit
fees associated with our corporate reorganization and
non-capitalizable costs incurred in connection with our IPO, and a
$1.8 million increase in personnel-related costs. General and
administrative personnel costs increased to support our increased
research and development activities and our status as a public
company. This increase in general and administrative
personnel-related costs includes $0.7 million of additional
non-cash stock-based compensation compared to 2016.
Net loss for the year ended December 31, 2017 was $29.7 million,
or ($1.63) basic and diluted net loss per ordinary share, as
compared to $12.2 million for the year ended December 31, 2016, or
($1.36) basic and diluted net loss per ordinary share.
As of December 31, 2017, our cash and cash equivalents totaled
$129.4 million, compared to $10.1 million at December 31,
2016. In October 2017, we sold 6,164,000 American Depositary
Shares (representing the same number of ordinary shares), including
the full exercise by the underwriters of their option to purchase
additional ADSs, at $14.00 per ADS in our IPO for net proceeds of
approximately $77.4 million. In June 2017, we completed a $45
million Series C financing with Syncona, NEA, Wellington and
Redmile. As of December 31, 2017, we had 28.9 million
ordinary shares outstanding.
Upcoming Conference Participation
Nightstar will participate in the Bloomberg Biotech Innovations
Conference on Monday, April 9th, 2018 at 3:30pm ET in New York
City. Additional information is available at
http://b.bloomberg.com/reg-BiotechInnovations. The link to the live
audio webcast and replay of the presentation will be available
through Bloomberg terminals at Live <go>.
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 trial 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 Phase 1/2 clinical 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
full-year 2017, cash position and sufficiency of capital resources
to fund our operating requirements, our planned clinical trials for
NSR-REP1 and NSR-RPGR, including our STAR Phase 3 trial in
choroideremia and the expansion of future clinical trials, 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 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 the
"Risk Factors" section of our prospectus filed pursuant to Rule
424(b)(4) under the U.S. Securities Act of 1933, as amended, on
September 28, 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
Media: Alicia Davis, THRUST IR
910-620-3302 alicia@thrustir.com
NIGHTSTAR THERAPEUTICS
PLC |
|
|
|
|
|
Consolidated Statements of Operations and
Comprehensive Loss |
(In thousands, except share and per share
amounts) |
|
|
|
|
|
|
|
Year Ended December
31, |
|
|
2017 |
|
2016 |
Operating expenses: |
|
|
|
|
Research
and development |
|
$ |
20,502 |
|
|
$ |
10,165 |
|
General
and administrative |
|
|
7,001 |
|
|
|
2,055 |
|
Total
operating expenses |
|
|
27,503 |
|
|
|
12,220 |
|
Other
income (expense): |
|
|
|
|
Interest
and other income |
|
|
709 |
|
|
|
22 |
|
Other
expense, net |
|
|
(2,855 |
) |
|
|
— |
|
Total
other income (expense), net |
|
|
(2,146 |
) |
|
|
22 |
|
Loss
before provision for income taxes |
|
|
(29,649 |
) |
|
|
(12,198 |
) |
Provision
for income taxes |
|
|
37 |
|
|
|
— |
|
Net
loss |
|
|
(29,686 |
) |
|
|
(12,198 |
) |
|
|
|
|
|
Other
comprehensive loss: |
|
|
|
|
Foreign
exchange translation adjustment |
|
|
3,988 |
|
|
|
(1,385 |
) |
Total
comprehensive loss |
|
$ |
(25,698 |
) |
|
$ |
(13,583 |
) |
Basic
and diluted net loss per ordinary share |
|
$ |
(1.63 |
) |
|
$ |
(1.36 |
) |
Weighted
average basic and diluted ordinary shares |
|
|
18,186,752 |
|
|
|
8,954,388 |
|
|
|
|
|
|
NIGHTSTAR THERAPEUTICS
PLC |
|
|
|
|
|
|
|
Consolidated Balance Sheets |
|
(In thousands) |
|
|
|
|
|
|
|
|
|
December 31, |
|
|
|
2017 |
|
2016 |
|
Assets |
|
|
|
|
|
Current
assets: |
|
|
|
|
|
Cash and
cash equivalents |
|
$ |
129,404 |
|
$ |
10,122 |
|
Prepaid
expenses and other assets |
|
|
5,438 |
|
|
4,110 |
|
Total
current assets |
|
|
134,842 |
|
|
14,232 |
|
Property
and equipment, net |
|
|
355 |
|
|
363 |
|
Total
assets |
|
$ |
135,197 |
|
$ |
14,595 |
|
Liabilities and shareholders’ equity |
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
Accounts
payable |
|
$ |
3,196 |
|
$ |
1,229 |
|
Accrued
expenses and other liabilities |
|
|
6,189 |
|
|
4,322 |
|
Total
current liabilities |
|
|
9,385 |
|
|
5,551 |
|
Total
liabilities |
|
|
9,385 |
|
|
5,551 |
|
|
|
|
|
|
|
Total
shareholders’ equity |
|
|
125,812 |
|
|
9,044 |
|
Total
liabilities and shareholders’ equity |
|
$ |
135,197 |
|
$ |
14,595 |
|
|
|
|
|
|
|
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