UNITED STATES SECURITIES
AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM6-K
REPORT OF FOREIGN PRIVATE ISSUER
PURSUANT TO RULE13a-16 OR15d-16
UNDER THE SECURITIES EXCHANGE ACT OF 1934
For the Month of November 2018
Commission File Number:001-38217
Nightstar Therapeutics plc
(Translation of registrant’s name into English)
10 Midford Place, 2nd Floor
London W1T 5BJ United Kingdom
(Address of principal executive office)
Indicate by check mark whether the registrant files or will file annual reports under cover of Form20-F or Form40-F:
☒ Form 20-F ☐ Form40-F
Indicate by check mark if the registrant is submitting the Form6-K in paper as permitted by RegulationS-T Rule 101(b)(1): ☐
Indicate by check mark if the registrant is submitting the Form6-K in paper as permitted by RegulationS-T Rule 101(b)(7): ☐
Other Events
On November 13, 2018, Nightstar Therapeutics plc issued a press release announcing financial results for the three and nine months ended September 30, 2018. The press release is attached as Exhibit 99.1 hereto and is incorporated by reference herein.
Exhibit | Description | |
| 99.1 | Press Release issued on November 13, 2018 | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
NIGHTSTAR THERAPEUTICS PLC | ||||||
Date: November 13, 2018 | By: | /s/ Bryan Yoon | ||||
| Name: Bryan Yoon | ||||||
| Title: General Counsel and Secretary | ||||||
Exhibit 99.1
Nightstar Announces Planned Initiation of Phase 2/3 Expansion Study in XIRIUS Trial forNSR-RPGR in XLRP and Reports Third Quarter 2018 Financial Results
Phase 2/3 Expansion Study to IncludeNo-Sham Control Arm in Alignment with FDA Draft Guidance on Development of Gene Therapy Products for Retinal Disorders
Preliminary Efficacy Data from Phase 2/3 Expansion Study Expected inMid-2019
WALTHAM, Mass. and LONDON, UK – November 13, 2018(GLOBE NEWSWIRE)– Nightstar Therapeutics plc (NASDAQ: NITE), a clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, today provided an update on recent achievements and upcoming clinical milestones and reported financial results for the quarter ended September 30, 2018.
“We are pleased with the upcoming initiation of the Phase 2/3 expansion study in line with our previous guidance after being the first sponsor to establishproof-of-concept inX-linked retinitis pigmentosa,” said Dave Fellows, Chief Executive Officer. “The modified Phase 2/3 expansion study is designed to be consistent with recommendations in FDA’s draft guidance on the development of gene therapy products for retinal disorders, which we believe will allow us to expedite the development ofNSR-RPGR.”
XIRIUS is a clinical trial consisting of an open-label, dose escalation study followed by an expansion study. Enrollment of the Phase 1 dose escalation study in the XIRIUS trial was completed in August 2018, consisting of six cohorts of three patients each for a total of 18 adult patients. On September 22, 2018, Nightstarannounced positive preliminary safety and efficacy data from the XIRIUS trial for the first five cohorts (combined n=15) of the dose escalation study at the EURETINA medical meeting.Six-monthfollow-up data on all 18 patients in the dose escalation study is expected to be available in the second quarter of 2019, withone-yearfollow-up data expected to be available in the fourth quarter of 2019.
The Phase 2/3 expansion study is designed to evaluate the safety and efficacy ofNSR-RPGR in patients with a diagnosis of XLRP due to RPGR mutations, as confirmed by genetic testing. The primary efficacy endpoint will evaluate changes in retinal sensitivity following treatment withNSR-RPGR. Secondary endpoints include both anatomical and functional endpoints of efficacy and safety similar to those evaluated in the dose escalation study as well as exploratory efficacy endpoints such as mobility maze assessments. Approximately 45 patients across six surgical centers in both the United States and the United Kingdom will be enrolled. The eligibility criteria for the expansion study will include patients with functional impairment as measured by microperimetry and the presence of viable photoreceptors as indicated by ellipsoid zone measurements on optical coherence tomography. Patients will be randomized on a masked basis into one of three study arms: approximately 15 patients receiving a high-dose of NSR-RPGR inone-eye (2.5x10^11 genome particles, or gp); approximately 15 patients receiving alow-dose ofNSR-RPGR inone-eye (5x10^10 gp); and approximately 15 patients receiving no treatment(no-sham, parallel control arm). The two treatment groups correspond to doses used in cohorts 5 and 3 of the dose escalation study, respectively. A standardized eight-week steroid
regimen will be included to maximize any potential treatment benefit. The Phase 2/3 expansion study is expected to begin by the end of 2018. Preliminary efficacy data is expected to be available inmid-2019, which would serve as the basis for discussions with regulatory agencies on potential Phase 3 requirements.One-yearfollow-up data from the expansion study is expected to be available in 2020.
Business Highlights Include
| • | Appointment of Strategic Biotechnology Executive Paula Cobb to Board of Directors in September 2018.Ms. Cobb currently serves as the executive vice president of corporate development at Decibel Therapeutics, Inc. and brings strategic, worldwide commercialization experience in rare diseases, having led teams to three new drug approvals and played key roles on four product launches, which will be invaluable as Nightstar evolves into a commercial-stage company. |
| • | PositiveProof-of-Concept Data from Dose Escalation Study in XIRIUS Trial forNSR-RPGR in XLRP in September 2018.NSR-RPGR data presented at the EURETINA 2018 Congress demonstratedproof-of-concept with durable dose-related improvements seen as early as month 1 across multiple microperimetry analyses. Preliminary efficacy responses were observed in 5 out of 9 patients (56%) in cohorts 3, 4 and 5, including durable improvements in overall macula sensitivity, central 16 macula sensitivity and number of improved macula loci.NSR-RPGR was well-tolerated with no dose limiting toxicities or serious treatment-related adverse events. |
| • | Inaugural R&D Day in September 2018.The R&D day featured presentations on XLRP endpoints, positiveproof-of-concept data onNSR-RPGR from the XIRIUS dose escalation study, program updates onNSR-REP1 for the treatment of choroideremia andNSR-ABCA4 for the treatment of Stargardt disease, as well as an overview of Nightstar’s hybrid manufacturing model for gene therapy. |
| • | Received Orphan Drug Designation forNSR-RPGR in September 2018.NSR-RPGR was granted orphan drug designation for the treatment of inherited retinal dystrophies due to defects in the RPGR gene from the U.S. Food and Drug Administration, or FDA, in the United States. |
| • | Completion of Public Offering of 4,600,000 American Depository Shares, or ADSs, in October 2018.The primary offering of 4,600,000 ADSs, including the full exercise by the underwriters of their overallotment option, at $18.00 per ADS, resulted in estimated net proceeds to Nightstar of approximately $77.1 million. |
Anticipated Milestones for 2019 and 2020
| • | NSR-RPGR forX-Linked Retinitis Pigmentosa |
| • | 2Q 2019:Six-MonthFollow-up Data from Dose Escalation Study |
| • | Mid-2019: Preliminary Efficacy Data from Phase 2/3 Expansion Study |
| • | 4Q 2019:One-YearFollow-up Data from Dose Escalation Study |
| • | 2020:One-YearFollow-up Data from Phase 2/3 Expansion Study |
| • | NSR-REP1 for Choroideremia |
| • | 1H 2019: Completion of Enrollment for Phase 3 STAR Registrational Trial for Choroideremia |
| • | 2020:One-yearFollow-up Data from Phase 3 STAR Trial |
Third Quarter 2018 Financial Results
Three Months Ended September 30, 2018 and 2017
Research and development expenses were $7.8 million for the three months ended September 30, 2018, compared to $4.0 million for the three months ended September 30, 2017. The increase of $3.8 million resulted primarily from increases in program-related expenses of $1.1 million forNSR-REP1 and $1.7 million forNSR-RPGR, as well as a $1.0 million increase in personnel-related costs and a $1.0 million increase in indirect research and development expenses. The increased expenses were partially offset by an increase of $1.0 million of research and development tax credits from Her Majesty’s Revenue & Customs, or HMRC. Research and development personnel-related costs increased due to an increase in headcount 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 additionalnon-cash share-based compensation compared to the same period in 2017.
General and administrative expenses were $3.0 million for the three months ended September 30, 2018, compared to $2.0 million for the three months ended September 30, 2017. The increase of $1.0 million is mainly due to an increase in personnel-related costs. General and administrative personnel-related costs increased due to an increase in headcount to support our increased research and development activities, growth of our company and our status as a public company. The increase in general and administrative personnel-related costs includes $0.3 million of additionalnon-cash share-based compensation compared to the same period in 2017.
Net loss for the three-month period ended September 30, 2018 was $7.6 million, or $0.27 basic and diluted net loss per ordinary share, as compared to $8.2 million, or $0.38 basic and diluted net loss per ordinary share, for the three-month period ended September 30, 2017.
Nine Months Ended September 30, 2018 and 2017
Research and development expenses were $22.0 million for the nine months ended September 30, 2018, compared to $10.3 million for the nine months ended September 30, 2017. The increase of $11.7 million resulted primarily from increases in program-related expenses of $4.5 million forNSR-REP1 and $4.2 million forNSR-RPGR, as well as a $3.7 million increase in personnel-related costs and a $1.9 million increase in indirect research and development expenses. The increased expenses were partially offset by an increase of $2.6 million of research and development tax credits from HMRC. Research and development personnel-related costs increased due to an increase in headcount 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 $1.0 million of additionalnon-cash share-based compensation compared to the same period in 2017.
General and administrative expenses were $9.1 million for the nine months ended September 30, 2018, compared to $3.4 million for the nine months ended September 30, 2017. The increase of $5.7 million is mainly due to a $4.4 million increase in personnel-related costs and a $1.3 million increase in consulting and professional fees, including increased legal, accounting and audit fees and insurance costs. General and administrative personnel-related costs increased due to an increase in headcount to support our increased research and development activities, growth of our company and our status as a public company. The increase in general and administrative personnel-related costs includes $1.4 million of additionalnon-cash share-based compensation compared to the same period in 2017.
Net loss for the nine-month period ended September 30, 2018 was $30.0 million, or $1.07 basic and diluted net loss per ordinary share, as compared to $15.9 million, or $0.84 basic and diluted net loss per ordinary share, for the nine-month period ended September 30, 2017.
As of September 30, 2018, our cash, cash equivalents and marketable securities totaled $100.8 million, compared to $129.4 million at December 31, 2017. As of September 30, 2018, there were approximately 28.9 million ordinary shares outstanding. On October 2, 2018, Nightstar completed an underwritten public offering of 4,600,000 ADSs (representing the same number of ordinary shares), including the full exercise by the underwriters of their option to purchase additional ADSs, at a public offering price of $18.00 per ADS, resulting in estimated net proceeds to Nightstar of approximately $77.1 million, after deducting underwriting discounts and commissions and estimated offering expenses.
About Nightstar
Nightstar is a leading clinical-stage gene therapy company focused on developing and commercializing novelone-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 treatments currently available and affects approximately one in every 50,000 people. Positive results from a Phase 1/2 trial ofNSR-REP1 were published in Nature Medicine in 2018, in The New England Journal of Medicine in 2016, and inThe Lancet in 2014. 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 withX-linked retinitis pigmentosa, an inheritedX-linked recessive retinal disease that affects approximately one in every 40,000 people. In September 2018, Nightstarannounced positive preliminary safety and efficacy data from the XIRIUS trial for the first five cohorts (combined n=15) of the dose escalation study at the EURETINA medical meeting.
For more information about Nightstar or its clinical trials, please visitwww.nightstartx.com.
AboutX-Linked Retinitis Pigmentosa (XLRP)
XLRP, a form of retinitis pigmentosa, is a rare inheritedX-linked recessive genetic retinal disorder primarily affecting males. Approximately 70% of XLRP cases are due to variants in the genes responsible for the production of RPGR. RPGR is involved in the transport of proteins necessary for the maintenance of photoreceptor cells. Loss of RPGR function in the retinal cells causes the progressive loss of rod and cone photoreceptors, leading to the loss of vision experienced by patients. The estimated worldwide prevalence of XLRP due to RPGR variants is approximately one in 40,000 people, which translates to approximately 17,000 patients in the United States and the five major European markets. There are no treatments currently available for XLRP. Nightstar is conducting a prospective, natural history observational study, referred to as the XOLARIS study, to better understand the progression of untreated XLRP in up to approximately 100 patients enrolled from approximately 23 centers in North America and Europe.
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,” “could,” “intend,”
“estimate,” “will,” “would,” “may,” “should,” “project,” “target,” “track,” “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 cash position and sufficiency of capital resources to fund our operating requirements, trends and other factors that may affect our financial results; our planned and ongoing clinical trials forNSR-REP1 andNSR-RPGR, including our Phase 3 STAR trial in choroideremia and our XIRIUS trial in X-linked retinitis pigmentosa; potential results and timelines relating to the dose escalation study and the Phase 2/3 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; timelines, pathway and prerequisite activities, including additional trials or studies, to filing for and receiving marketing authorization for any of our product candidates from any regulatory agency; the prevalence of patient populations for our targeted indications; and the utility of the endpoints, including any exploratory endpoints, in our clinical trials and the prior preclinical and clinical data in determining future clinical results. These forward-looking statements are based on management’s current expectations of future events as of the date of this release and are subject to a number of 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 those related to the timing and costs involved in commercializing any product candidate that receives regulatory approval; the initiation, timing and conduct of clinical trials; the availability of data from clinical trials and expectations for regulatory submissions and approvals; whether interim results of a clinical trial will be predictive of the final results of the trial; whether results of small or early stage clinical trials will be predictive of the results of later-stage trials; our scientific approach and general development progress; the availability or commercial potential of the our product candidates; the sufficiency of our cash resources; and other risks and uncertainties set forth in our Report of Foreign Private Issuer on Form6-K furnished to the U.S. Securities and Exchange Commission on September 25, 2018 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. Any reference to our website address in this press release is intended to be an inactive textual reference only and not an active hyperlink.
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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 per share amounts)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2018 | 2017 | 2018 | 2017 | |||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 7,845 | $ | 3,983 | $ | 21,961 | $ | 10,275 | ||||||||
General and administrative | 3,019 | 2,025 | 9,119 | 3,432 | ||||||||||||
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Total operating expenses | 10,864 | 6,008 | 31,080 | 13,707 | ||||||||||||
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Other income (expense): | ||||||||||||||||
Interest and other income | 798 | 55 | 1,887 | 61 | ||||||||||||
Other Income (expense), net | 2,564 | (2,258 | ) | (1,097 | ) | (2,258 | ) | |||||||||
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Total other income (expense), net | 3,362 | (2,203 | ) | 790 | (2,197 | ) | ||||||||||
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Loss before benefit for income taxes | (7,502 | ) | (8,211 | ) | (30,290 | ) | (15,904 | ) | ||||||||
Provision for (benefit from) income taxes | 83 | — | (251 | ) | — | |||||||||||
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Net loss | (7,585 | ) | (8,211 | ) | (30,039 | ) | (15,904 | ) | ||||||||
Other comprehensive income (loss) | (2,733 | ) | 1,848 | 932 | 3,570 | |||||||||||
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Total comprehensive loss | $ | (10,318 | ) | $ | (6,363 | ) | $ | (29,107 | ) | $ | (12,334 | ) | ||||
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Basic and diluted net loss per ordinary share | $ | (0.27 | ) | $ | (0.38 | ) | $ | (1.07 | ) | $ | (0.84 | ) | ||||
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Weighted average basic and diluted ordinary shares | 28,139 | 21,514 | 27,975 | 18,858 | ||||||||||||
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NIGHTSTAR THERAPEUTICS PLC
Consolidated Balance Sheets
(In thousands)
| September 30, 2018 | December 31, 2017 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 45,974 | $ | 129,404 | ||||
Marketable securities | 54,800 | — | ||||||
Prepaid expenses and other assets | 12,531 | 5,438 | ||||||
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Total current assets | 113,305 | 134,842 | ||||||
Property and equipment, net | 534 | 355 | ||||||
Other assets | 632 | — | ||||||
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Total assets | $ | 114,471 | $ | 135,197 | ||||
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Liabilities and shareholders’ equity | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 4,423 | $ | 3,196 | ||||
Accrued expenses and other liabilities | 10,340 | 6,189 | ||||||
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Total current liabilities | 14,763 | 9,385 | ||||||
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Total liabilities | 14,763 | 9,385 | ||||||
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Total shareholders’ equity | 99,708 | 125,812 | ||||||
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Total liabilities and shareholders’ equity | $ | 114,471 | $ | 135,197 | ||||
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