Orchard Therapeutics Reports Second Quarter 2019 Financial Results and Builds Momentum Ahead of Regulatory Filings
Metachromatic Leukodystrophy (MLD) MAA Submission and Initiation of ADA-SCID Rolling BLA Expected in the First Half of 2020; OTL-103 Recently Granted RMAT Designation for Wiskott-Aldrich Syndrome (WAS)
MLD and Mucopolysaccharidosis Type I (MPS-I) Abstracts Accepted for Oral Presentations at SSIEM 2019 Symposium
Ended the Second Quarter of 2019 with Approximately
Conference Call Scheduled for Today at
“In the first half of 2019, we made tremendous progress advancing our three lead programs toward regulatory filings, while pursuing a broader range of clinical opportunities for the hematopoietic stem cell-based gene therapy platform,” said
Second Quarter and Recent Business Achievements
- Received regenerative medicine advanced therapy (RMAT) designation from the
U.S. Food and Drug Administration( FDA) for OTL-103 for the treatment of Wiskott-Aldrich Syndrome (WAS). RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising advanced therapy products, including gene therapies. The OTL-103 program is continuing to enroll patients in a clinical trial using a cryopreserved formulation to support regulatory filings in the U.S. and Europein 2021. Link to the full RMAT release here.
- Completed an underwritten public offering of 9,725,268 American Depositary Shares in
June 2019resulting in proceeds, net of underwriting discounts and commissions, of approximately $129.7 million. Link to the full release here.
- Signed a collaboration agreement with Fondazione Telethon and Ospedale San Raffaele for a new clinical-stage gene therapy program in mucopolysaccharidosis type I (MPS-I) showing encouraging preliminary data in an ongoing proof-of-concept trial. The trial is expected to enroll up to eight patients by the first half of 2020. Link to the full release here.
- Held a positive Marketing Authorization Application (MAA) pre-submission meeting with the
European Medicines Agency(EMA) in early May 2019for OTL-200 for metachromatic leukodystrophy (MLD). The company has brought forward the timeline for the planned submission of an MAA to the first half of 2020 and also expects to submit a Biologics License Application (BLA) in the U.S. approximately one year after the MAA submission.
Upcoming Data Presentations & Remaining 2019 Milestones
- Present data from the ongoing proof-of-concept trial of OTL-203 for MPS-I at the
Society for the Study of Inborn Errorsof Metabolism (SSIEM) 2019 Symposium on September 4, 2019in Rotterdam, The Netherlands.
- Present an integrated data analysis of OTL-200 for MLD at the SSIEM 2019 Symposium on
September 4, 2019in Rotterdam, The Netherlands. The integrated analysis includes the 20 patient registrational data set in addition to data from expanded access patients.
- Report engraftment data in the first three patients from the ongoing clinical trial of OTL-200 for MLD using a cryopreserved formulation.
- Support the submission of a clinical trial application for OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA) in preparation of a clinical trial initiation.
Primary Immune Deficiencies
- Report engraftment data in 10 patients from a clinical trial of OTL-101 for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) using a cryopreserved formulation.
- Report three-year follow-up data in eight patients from a registrational trial of OTL-103 for WAS.
- Design and engage regulators on a planned registrational trial for OTL-102 for patients with X-linked chronic granulomatous disease (X-CGD).
Second Quarter 2019 Financial Results and Updated Cash Runway
Cash and investments as of
Research and development expenses were
Selling, general and administrative expenses were
Net loss attributable to ordinary shareholders was
The company expects that its existing cash and investments will enable funding of its anticipated operating and capital expenditure requirements into the second half of 2021.
Conference Call & Webcast Information
Orchard will host a conference call and live webcast with slides today at
Orchard’s portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis®, a gammaretroviral vector-based gene therapy and the first such treatment approved by the
Orchard currently has offices in the
This press release contains certain forward-looking statements about Orchard’s strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” and “future” or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchard’s expectations regarding the timing of regulatory submissions for approval of its product candidates, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, the likelihood of approval of such product candidates by the applicable regulatory authorities, and the company’s financial condition and cash runway into the second half of 2021. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchard’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the delay of any of Orchard’s regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchard’s product candidates, the receipt of restricted marketing approvals, or delays in Orchard’s ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.
Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchard’s annual report on Form 20-F for the year ended
Condensed Consolidated Statements of Operations
(In thousands, except share and per share amounts)
|Three Months Ended June 30,||Six Months Ended June 30,|
|Costs and operating expenses:|
|Research and development||$||40,478||$||150,991||$||57,971||$||160,162|
|Selling, general and administrative||13,674||7,421||24,464||11,948|
|Total costs and operating expenses||54,152||158,412||82,435||172,110|
|Loss from operations||(54,152||)||(158,412||)||(82,435||)||(172,110||)|
|Other income (expense), net||2,850||2,097||987||401|
|Net loss before income tax||(51,302||)||(156,315||)||(81,448||)||(171,709||)|
|Income tax (expense) benefit||772||82||179||165|
|Net loss attributable to ordinary shareholders||(50,530||)||(156,233||)||(81,269||)||(171,544||)|
|Net loss per share attributable to ordinary shareholders, basic and diluted||$||(0.56||)||$||(15.45||)||$||(0.92||)||$||(16.99||)|
|Weighted average number of ordinary shares outstanding, basic and diluted||89,712,916||10,115,335||88,369,311||10,095,863|
Condensed Consolidated Balance Sheets
|June 30,||December 31,|
|Cash and cash equivalents||$||132,783||$||335,844|
|Trade and other receivables||1,956||2,153|
|Prepaid expenses and other assets||5,143||6,935|
|Research and development tax credit receivable, current||10,594||10,585|
|Total current assets||437,224||355,517|
|Property and equipment, net||5,549||5,476|
|Research and development tax credit receivable||9,731||—|
|Other long-term assets||1,770||1,212|
|Liabilities and shareholders’ equity|
|Accrued expenses and other current liabilities||26,574||29,780|
|Total current liabilities||55,435||47,905|
|Long-term debt, net||24,501||—|
|Other long-term liabilities||7,024||6,799|
|Total shareholders’ equity||371,157||311,338|
|Total liabilities and shareholders’ equity||$||458,117||$||366,042|
Director, Investor Relations
Manager, Corporate Communications
Source: Orchard Therapeutics