Orchard Therapeutics Reports 2019 Financial Results and Reviews Key Strategic Priorities for 2020
Commercial Preparations on Track for Potential Second Half 2020 EU Launch of OTL-200 for MLD with
Initiation of Rolling
Recent WORLD Presentations Showcased Data from Neurometabolic Franchise; Additional Clinical Data from MPS-I and MPS-IIIA Proof of Concept Trials Expected in 2020
“We are inspired by the possibilities ahead for Orchard in 2020 and beyond to bring the benefits of our gene therapy approach to patients worldwide,” said
Recent 2020 Achievements
- Presented data from the company’s neurometabolic franchise at the 16th Annual WORLD Symposium in
Orlando, Florida including:- MLD:
- Data from a total of 33 patients treated with OTL-200, a gene therapy under an accelerated assessment review by the
European Medicines Agency (EMA) for the treatment of metachromatic leukodystrophy (MLD). - Emerging data from a separate study quantifying caregiver-reported quality of life experiences was shown and describes the immense burden on their financial, professional, psychosocial and physical well-being.
- Data from a total of 33 patients treated with OTL-200, a gene therapy under an accelerated assessment review by the
- MPS-I: Data from the ongoing proof-of-concept trial of OTL-203 for the treatment of mucopolysaccharidosis type I (MPS-I). Evidence of engraftment and overexpression of the alpha-l-iduronidase (IDUA) enzyme was seen in all evaluable patients, with a median follow-up of six months (range of 1 - 12 months).
- MPS-IIIA: A case report of the first patient treated with ex-vivo, autologous hematopoietic stem cell (HSC) gene therapy for mucopolysaccharidosis type IIIA (MPS-IIIA) under compassionate use was presented by Royal Manchester Children’s Hospital1.
- MLD:
- Appointed industry veteran
Steven Altschuler , M.D. to its board of directors.Dr. Altschuler brings nearly 20 years of experience in the healthcare industry and currently serves as managing director ofHealthcare Ventures atZiff Capital Partners . He was previously chair of the board of directors at Spark Therapeutics (now part of Roche), a commercial-stage gene therapy company. - Announced that the FDA has granted orphan drug designation for OTL-102 for the treatment of X-linked chronic granulomatous disease (X-CGD).
2020 / 2021 Corporate Priorities and Expected Key Milestones
- Obtain approval for and launch OTL-200 for the treatment of MLD in
Europe in the second half of 2020. - Submit a biologics license application (BLA) filing in the
U.S. for OTL-200 for the treatment of MLD in late 2020 or early 2021. - Initiate a rolling BLA filing in the
U.S. for OTL-101 in adenosine deaminase severe combined immunodeficiency (ADA-SCID) in the first half of 2020 with anticipated completion of the filing within 12 months of initiation. - Prepare for BLA and marketing authorization application (MAA) regulatory filings for OTL-103 in Wiskott-Aldrich Syndrome (WAS) in the
U.S. and EU in 2021. - Release interim data from the OTL-203 proof-of-concept clinical trial in MPS-I in 2020. One-year follow-up results for the first eight patients, including the primary endpoints, are anticipated in 2021.
- Enroll five patients and release interim data on the first patient from the recently initiated proof-of-concept clinical trial for OTL-201 in MPS-IIIA in 2020.
- Initiate construction of an in-house manufacturing facility in
Fremont, California in 2020 (design of the facility is complete).
1 Patient was treated by the Royal Manchester Children’s Hospital (RMCH) under a “Specials” license, granted by the
Fourth Quarter 2019 Financial Results
Cash, cash equivalents and investments as of
During the three months ended
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, cash equivalents 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
About Orchard
Orchard has its global headquarters in
Availability of Other Information About Orchard
Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (twitter.com/orchard_tx and www.linkedin.com/company/orchard-therapeutics), including but not limited to investor presentations and investor fact sheets,
Forward-Looking Statements
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,” “plans,” “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, the company’s business strategy and goals, the therapeutic potential of Orchard’s product candidates, including the product candidate or candidates referred to in this release, Orchard’s expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, 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, the likelihood the company will initiate construction of an in-house manufacturing facility in 2020, 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 risk that any one or more of Orchard’s product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized, the risk of cessation or delay of any of Orchard’s ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchard’s product candidates, 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, and the risk of 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
Consolidated Statements of Operations Data
(in thousands, except share and per share data)
(Unaudited)
| Three Months Ended |
Twelve Months Ended |
||||||||||||||||
| 2019 | 2018 | 2019 | 2018 | ||||||||||||||
| Product sales, net | $ | 595 | $ | 689 | $ | 2,513 | $ | 2,076 | |||||||||
| Costs and operating expenses: | |||||||||||||||||
| Cost of product sales | 191 | 142 | 805 | 422 | |||||||||||||
| Research and development | 30,899 | 17,426 | 117,363 | 205,319 | |||||||||||||
| Selling, general and administrative | 18,531 | 11,952 | 57,218 | 31,366 | |||||||||||||
| Total costs and operating expenses | 49,621 | 29,520 | 175,386 | 237,107 | |||||||||||||
| Loss from operations | (49,026 | ) | (28,831 | ) | (172,873 | ) | (235,031 | ) | |||||||||
| Other income (expense): | |||||||||||||||||
| Interest income | 1,843 | 1,114 | 7,362 | 1,116 | |||||||||||||
| Interest expense | (633 | ) | — | (1,538 | ) | — | |||||||||||
| Other income (expense), net | 2,533 | 3,056 | 1,387 | 4,390 | |||||||||||||
| Total other income (expense), net | 3,743 | 4,170 | 7,211 | 5,506 | |||||||||||||
| Net loss before income tax | (45,283 | ) | (24,661 | ) | (165,662 | ) | (229,525 | ) | |||||||||
| Income tax (expense) benefit | (133 | ) | (402 | ) | 2,240 | (970 | ) | ||||||||||
| Net loss attributable to ordinary shareholders | (45,416 | ) | (25,063 | ) | (163,422 | ) | (230,495 | ) | |||||||||
| Net loss per share attributable to ordinary shareholders, basic and diluted | $ | (0.46 | ) | $ | (0.42 | ) | $ | (1.75 | ) | $ | (10.22 | ) | |||||
| Weighted average number of ordinary shares outstanding, basic and diluted | 98,243,915 | 59,435,325 | 93,240,355 | 22,559,389 | |||||||||||||
Consolidated Balance Sheet Data
(in thousands)
(Unaudited)
| 2019 | 2018 | |||||
| Assets | ||||||
| Current assets: | ||||||
| Cash and cash equivalents | $ | 19,053 | $ | 335,844 | ||
| Marketable securities | 305,937 | — | ||||
| Trade receivables | 1,442 | 2,103 | ||||
| Prepaid expenses and other assets | 8,530 | 6,985 | ||||
| Research and development tax credit receivable, current | 14,934 | 10,585 | ||||
| Total current assets | $ | 349,896 | $ | 355,517 | ||
| Non-current assets: | ||||||
| Operating lease right-of-use assets | 19,415 | — | ||||
| Property and equipment, net | 7,596 | 5,476 | ||||
| Research and development tax credit receivable | 13,710 | — | ||||
| Other long-term assets | 8,664 | 5,049 | ||||
| Total non-current assets | 49,385 | 10,525 | ||||
| Total assets | $ | 399,281 | $ | 366,042 | ||
| Liabilities and shareholders' equity | ||||||
| Current liabilities: | ||||||
| Accounts payable | $ | 11,984 | $ | 18,125 | ||
| Accrued expenses and other current liabilities | 37,980 | 29,780 | ||||
| Operating lease liabilities | 5,892 | — | ||||
| Total current liabilities | 55,856 | 47,905 | ||||
| Long-term debt, net | 24,699 | — | ||||
| Operating lease liabilities, non-current | 15,320 | — | ||||
| Other long-term liabilities | 4,213 | 6,799 | ||||
| Total liabilities | 100,088 | 54,704 | ||||
| Shareholders’ equity: | 299,193 | 311,338 | ||||
| Total liabilities and shareholders’ equity | $ | 399,281 | $ | 366,042 | ||
Contacts
Investors
Director, Investor Relations
+1 862-242-0764
Renee.Leck@orchard-tx.com
Media
Manager, Corporate Communications
+1 978-339-3378
media@orchard-tx.com
Source: Orchard Therapeutics (Europe) Limited