“We made significant progress in 2018 across our portfolio as we focus on our mission to become a fully-integrated company developing and commercializing RNA medicines for patients with inherited retinal diseases (IRDs). The positive interim data for sepofarsen in LCA10 has resulted in our ability to move forward with a pivotal trial and further increased our confidence in our portfolio of programs targeting severe IRDs, including Usher syndrome and adRP,” said
Corporate Highlights and Business Update
Sepofarsen (formerly QR-110) for LCA10
January 2019, agreement was reached with the U.S. Food and Drug Administration( FDA) on the design of the pivotal Phase 2/3 ILLUMINATE trial. The trial is expected to begin in the first half of 2019 with interim data expected around the end of 2020.
- In December, ProQR was awarded an innovation credit by the Dutch government for the clinical development and efforts to obtain marketing approval for the sepofarsen program.
- In September, positive interim results of a clinical trial of sepofarsen for the treatment of Leber’s congenital amaurosis 10 (LCA10) were presented at the Retinal Degeneration 2018 meeting (RD2018). The landmark analysis presented three-month results from eight subjects who received a single dose treatment. The results were published in the journal Nature Medicine in December.
- In August, preclinical data for sepofarsen were published in Molecular Therapy – Nucleic Acids, an official journal of the
American Society of Gene & Cell Therapy.
QR-421a for Usher syndrome type 2
January 2019, the FDAgranted Fast Track designation for QR-421a for Usher syndrome type 2 and non-syndromic RP due to mutations in exon 13 of the USH2A gene.
- In December, the
FDAcleared the Investigational New Drug (IND) application for QR-421a for Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. The Phase 1/2 STELLAR trial is expected to initiate in the first half of 2019, with interim data expected in mid-2019.
- In February, ProQR entered into a partnership with the Foundation Fighting Blindness, FFB, through which ProQR will receive up to
$7.5 millionin funding from FFB for the preclinical and clinical development of QR-421a for Usher syndrome type 2 targeting mutations in exon 13.
QR-1123 for autosomal dominant retinitis pigmentosa (adRP)
- In October, ProQR in-licensed exclusive worldwide rights for IONIS-RHO-2.5Rx, renamed QR-1123, from
Ionis Pharmaceuticals. A first in human Phase 1/2 clinical trial in adRP patients is expected to start in 2019.
QR-313 for dystrophic epidermolysis bullosa
- In June, WINGS, the first clinical trial to evaluate the safety and efficacy of QR-313 in patients that have recessive dystrophic epidermolysis bullosa (RDEB) due to mutations in exon 73 of the COL7A1 gene, was initiated. Interim data are expected in Q1 2019 with full results in 2019.
- Also in June, ProQR entered into a partnership with the
EB Research Partnership(EBRP) and the EB Medical Research Foundation(EBMRF). ProQR will receive up to $5 millionin funding for the clinical development of QR-313.
- In December, ProQR was added to the NASDAQ Biotechnology Index (NASDAQ: NBI).
- Also in December, ProQR added key functions to the leadership team:
Aniz Girach, MD was hired as the Chief Medical Officer and Tiffany Burtas Vice President, Head of Commercial.
- In September, ProQR closed an underwritten public offering of 6,612,500 ordinary shares at a price of
$15.75per share including full exercise of underwriters’ option to purchase 862,500 additional ordinary shares. Gross proceeds totaled approximately $104.1 million, which extended the Company’s cash runway into 2021.
- In May,
Yi-Tao Yu, Ph.D., a professor of biochemistry and biophysics at the University of Rochester Medical Center, was appointed to the scientific advisory board. Dr. Yu’s research focuses on RNA editing and his expertise will help to develop the Company’s novel RNA editing technologies.
- In April, Dr.
Peter A. Bealwas appointed to the scientific advisory board. Dr. Beal is an expert in the field of RNA-based therapeutics, particularly in the area of RNA editing.
Research and development costs increased to €9.5 million for the quarter ended
General and administrative costs increased to €4.6 million for the quarter ended
Net loss for the three month period ended
2018 Annual Reports
The consolidated statement of financial position of
About sepofarsen (formerly named QR-110)
Sepofarsen is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to restore normal (wild-type) CEP290 mRNA leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in
QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional usherin protein that causes the disease. QR-421a is designed to exclude the genetic defect from the RNA in the eye, such that it leads to the expression of a shortened but functional usherin protein, thereby modifying the underlying disease. QR-421a has received orphan drug designation in
QR-1123 is a first-in-class investigational oligonucleotide (gapmer) that was developed by
QR-313 is a potential first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils. The clinical development of QR-313 is supported with funding from
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such statements include those relating to the development and therapeutic potential of our product candidates, including sepofarsen, QR-421a, QR-1123 and QR-313, our plans and timing of initiating and obtaining results from our ongoing and planned clinical trials, our plans for launching our product candidates, if approved, the expansion of our portfolio of product candidates, our financial resources and cash runway, and our plans for and intended benefits of strategic collaborations and alliances for our programs. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the
Vice President of Investor Relations and Corporate Communications
T: +1 202 360 4855
LifeSci Public Relations
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Unaudited Condensed Consolidated Statement of Financial Position
|December 31,||December 31,|
|€ 1,000||€ 1,000|
|Cash and cash equivalents||105,580||48,099|
|Prepayments and other receivables||1,544||2,064|
|Social security and other taxes||1,243||396|
|Total current assets||108,367||50,559|
|Property, plant and equipment||1,864||2,505|
|Equity and liabilities|
|Equity attributable to owners of the Company||92,915||39,363|
|Social security and other taxes||—||1,019|
|Other current liabilities||7,473||4,622|
|Total current liabilities||8,160||8,494|
|Total equity and liabilities||110,231||53,103|
Unaudited Condensed Consolidated Statement of Profit or Loss and OCI
(€ in thousands, except share and per share data)
|Three month period||Year|
|ended December 31,||ended December 31,|
|€ 1,000||€ 1,000||€ 1,000||€ 1,000|
|Research and development costs||(9,542||)||(8,345||)||(29,514||)||(31,153||)|
|General and administrative costs||(4,640||)||(2,891||)||(12,540||)||(10,840||)|
|Total operating costs||(14,182||)||(11,236||)||(42,054||)||(41,993||)|
|Finance income and expense||(128||)||(586||)||(792||)||(3,175||)|
|Result before corporate income taxes||(12,977||)||(11,311||)||(37,085||)||(43,673||)|
|Result for the period||(12,977||)||(11,311||)||(37,086||)||(43,675||)|
|Other comprehensive income||(13||)||37||(28||)||151|
|Total comprehensive income||(12,990||)||(11,274||)||(37,114||)||(43,524||)|
|Result attributable to|
|Owners of the Company||(12,944||)||(11,283||)||(36,894||)||(43,637||)|
|Weighted average number of shares outstanding1||38,809,784||28,695,362||34,052,520||25,374,807|
|Earnings per share attributable to the equity holders of the Company (expressed in Euro per share)|
|Basic loss per share1||(0.33||)||(0.39||)||(1.09||)||(1.72||)|
|Diluted loss per share1||(0.33||)||(0.39||)||(1.09||)||(1.72||)|
- For this period presented in these financial statements, the potential exercise of share options is not included in the diluted earnings per share calculation as the Company was loss-making in all periods. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal in this period.
Unaudited Condensed Consolidated Statement of Changes in Equity
|Attributable to owners of the Company|
|€ 1,000||€ 1,000||€ 1,000||€ 1,000||€ 1,000||€ 1,000||€ 1,000||€ 1,000|
|Balance at January 1, 2017||23,346,856||934||123,597||4,353||(15||)||(75,733||)||53,136||—||53,136|
|Result for the period||—||—||—||—||—||(43,637||)||(43,637||)||(38||)||(43,675||)|
|Other comprehensive income||—||—||—||—||151||—||151||—||151|
|Recognition of share-based payments||—||—||—||4,024||—||—||4,024||—||4,024|
|Issue of ordinary shares||8,573,975||343||25,342||—||—||—||25,685||—||25,685|
|Issue of treasury shares||4,503,149||180||(180||)|
|Share options exercised||1,034||0||4||—||—||—||4||—||4|
|Balance at December 31, 2017||36,425,014||1,457||148,763||8,377||136||(119,370||)||39,363||(38||)||39,325|
|Balance at January 1, 2018||36,425,014||1,457||148,763||8,377||136||(119,370||)||39,363||(38||)||39,325|
|Result for the period||—||—||—||—||—||(36,894||)||(36,894||)||(192||)||(37,086||)|
|Other comprehensive income||—||—||—||—||(28||)||—||(28||)||—||(28||)|
|Recognition of share-based payments||—||4||2,185||3,224||—||—||5,413||—||5,413|
|Issue of ordinary shares||6,724,973||265||83,926||—||—||—||84,191||84,191|
|Issue of treasury shares||(226,098||)|
|Share options lapsed||—||—||(97||)||—||97|
|Share options exercised||226,098||—||870||(724||)||—||724||870||—||870|
|Balance at December 31, 2018||43,149,987||1,726||235,744||10,780||108||(155,443||)||92,915||(230||)||92,685|
Unaudited Condensed Consolidated Statement of Cash Flows
|Three month period||Year|
|ended December 31,||ended December 31,|
|€ 1,000||€ 1,000||€ 1,000||€ 1,000|
|Cash flows from operating activities|
|— Share-based compensation||979||934||3,224||4,024|
|— Financial income and expenses||128||586||792||3,175|
|— Net foreign exchange gain / (loss)||(13||)||(28||)||151|
|Changes in working capital||220||703||1,294||164|
|Cash used in operations||(11,397||)||(8,793||)||(30,812||)||(35,096||)|
|Corporate income tax paid||—||—||—||(2||)|
|Net cash used in operating activities||(11,292||)||(8,715||)||(30,682||)||(34,951||)|
|Cash flow from investing activities|
|Purchases of intangible assets||—||—||—||—|
|Purchases of property, plant and equipment||(27||)||(10||)||(312||)||(121||)|
|Net cash used in investing activities||(27||)||(10||)||(312||)||(121||)|
|Cash flow from financing activities|
|Proceeds from issuance of shares, net of transaction costs||2,085||16,923||86,380||25,685|
|Proceeds from exercise of share options||210||3||870||4|
|Proceeds from borrowings||163||100||264||301|
|Proceeds from convertible loans||702||500||1,132||650|
|Net cash generated by financing activities||3,160||17,526||88,646||26,640|
|Net increase/(decrease) in cash and cash equivalents||(8,159||)||8,801||57,652||(8,432||)|
|Currency effect cash and cash equivalents||23||(444||)||(171||)||(2,669||)|
|Cash and cash equivalents, at beginning of the period||113,716||39,742||48,099||59,200|
|Cash and cash equivalents at the end of the period||105,580||48,099||105,580||48,099|
Source: ProQR Therapeutics N.V.