Orchard Therapeutics Reports 2018 Financial Results and Reviews Recent Business Highlights
Three Lead Programs for MLD, ADA-SCID and WAS on Track for Regulatory Filings Over the Next Three Years, with MLD and ADA-SCID Expected in 2020
Registrational Data Showing 100% Overall Survival and 100% Event-Free Survival at Two Years in ADA-SCID Patients Recently Presented at ASBMT; Two and Three Year Data in MLD Patients to be Presented Next Week at EBMT
Recent CTO Appointment Enhances Global Commercial Manufacturing Capabilities
Strong Financial Position with Approximately
BOSTON and LONDON,
“In 2018 we expanded and advanced our portfolio of autologous, ex vivo, hematopoietic stem cell gene therapies and established the capabilities to scale a global, fully integrated biopharmaceutical company serving patients. This year, we plan to build on these successes and further establish Orchard as a leader in the gene therapy field,” said
Recent Highlights
- Presented two-year follow-up data in 20 patients from the fresh formulation registrational trial of OTL-101 in patients with severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) at the 2019 Transplantation and Cellular Therapy Meetings of ASBMT and CIBMTR. Patients treated with OTL-101 in this trial showed 100% overall survival and 100% event free survival at 24 months, demonstrating favorable outcomes compared to a historical control group of patients who received a hematopoietic stem cell transplant (link to full data here).
- Presented clinical proof of concept data for OTL-102 for the treatment of X-linked chronic granulomatous disease (X-CGD), also at ASBMT. Six patients receiving OTL-102 continue to show sustained levels of functioning neutrophils 12 months after treatment with gene therapy and no longer receive CGD-related prophylactic antibiotics (link to full data here). This represents the company’s fourth program to reach clinical proof of concept.
- Recognized the publication in Nature Medicine by the
San Raffaele-Telethon Institute for Gene Therapy of OTL-300 interim clinical data in seven patients with transfusion-dependent beta-thalassemia (TDBT). - Appointed Ran Zheng as chief technical officer. In this role Ms. Zheng will be responsible for global manufacturing and technical operations. Ms. Zheng spent the last 16 years in roles of increasing responsibility within operations at
Amgen , most recently as a vice president overseeing end-to-end supply chain and manufacturing operations to enable global clinical development, product launch and commercial supply.
Upcoming Data Presentations & Remaining 2019 Milestones
Neurometabolic Disorders
- Present two and three-year follow-up data in 20 patients from the fresh formulation registrational trial of OTL-200 for metachromatic leukodystrophy (MLD) at the 45th Annual Meeting of the
European Society for Blood and Marrow Transplantation (EBMT ) onMarch 26 inFrankfurt, Germany - Release engraftment data in the first three patients from the cryopreserved formulation clinical trial of OTL-200 for MLD
- 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
- Release engraftment data in 10 patients from a cryopreserved formulation clinical trial of OTL-101 for ADA-SCID
- Publish three-year follow-up data in eight patients from the fresh formulation registrational trial of OTL-103 for Wiskott-Aldrich syndrome (WAS)
- Initiate enrollment in a cryopreservation formulation clinical trial for OTL-103 for patients with WAS
- Design and engage regulators on the registrational trial for OTL-102 for patients with X-CGD
Hemoglobinopathies
- Report clinical proof-of-concept data in nine patients at 12 months for OTL-300 in TDBT
Full Year 2018 Financial Results
Cash and investments as of
During 2018, the company recognized
Research and development expenses were
Selling, general and administrative expenses were
Net loss attributable to ordinary shareholders was
The company expects that its cash and investments as of
Conference Call & Webcast Information
Orchard will host a conference call and live webcast with slides today at
About Orchard
Orchard’s portfolio of autologous, ex vivo, hematopoietic stem cell gene therapies includes Strimvelis, the first such treatment approved by the
Orchard currently has offices in the
Forward-Looking Statements
This press release contains certain forward-looking statements 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,” “anticipates,” 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 through the second half of 2020. These statements are neither promises nor guarantees but 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. For additional disclosure regarding these and other risks faced by Orchard, see the disclosure contained in Orchard’s public filings with the Securities and Exchange Commission, including in the final prospectus related to Orchard’s initial public offering filed with the
| Consolidated Statements of Operations Data (in thousands, except share and per share data) (Unaudited) |
||||||||||||
| Six Months Ended December 31, |
Twelve Months Ended December 31, |
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| 2018 | 2017 | 2018 | 2017 | |||||||||
| Product sales, net | $ | 2,076 | $ | — | $ | 2,076 | $ | — | ||||
| Costs and operating expenses | ||||||||||||
| Cost of product sales | 422 | — | 422 | — | ||||||||
| Research and development | 45,157 | 21,981 | 205,319 | 32,527 | ||||||||
| Selling, general and administrative | 19,418 | 3,715 | 31,366 | 5,985 | ||||||||
| Total costs and operating expenses | 64,997 | 25,696 | 237,107 | 38,512 | ||||||||
| Loss from operations | (62,921) | (25,696) | (235,031) | (38,512) | ||||||||
| Other income (expense): | ||||||||||||
| Total other income (expense), net | 5,105 | (779) | 5,506 | (1,179) | ||||||||
| Net loss before income tax | (57,816) | (26,475) | (229,525) | (39,691) | ||||||||
| Income tax expense | (1,135) | (95) | (970) | (53) | ||||||||
| Net loss attributable to ordinary shareholders | $ | (58,951) | $ | (26,570) | $ | (230,495) | $ | (39,744) | ||||
| Net loss per share attributable to ordinary shareholders, basic and diluted | $ | (1.69) | $ | (2.89) | $ | (10.22) | $ | (4.48) | ||||
| Weighted average number of ordinary shares outstanding, basic and diluted | 34,864,911 | 9,180,759 | 22,559,389 | 8,872,768 | ||||||||
| Consolidated Balance Sheet Data (in thousands) (Unaudited) |
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| December 31, |
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| 2018 | 2017 | |||||
| Assets | ||||||
| Current assets: | ||||||
| Cash | $ | 335,844 | $ | 89,856 | ||
| Trade and other receivables | 2,153 | 1,247 | ||||
| Prepaid expenses and other current assets | 6,935 | 2,247 | ||||
| Research and development tax credit receivable | 10,585 | 871 | ||||
| Total current assets | 355,517 | 94,221 | ||||
| Non-current assets: | ||||||
| Property and equipment, net | 5,476 | 2,713 | ||||
| Restricted cash | 3,837 | — | ||||
| Other long-term assets | 1,212 | 360 | ||||
| Total non-current assets | 10,525 | 3,073 | ||||
| Total assets | $ | 366,042 | $ | 97,294 | ||
| Liabilities and shareholders' equity | ||||||
| Current liabilities: | ||||||
| Accounts payable | $ | 18,125 | $ | 3,891 | ||
| Accrued expenses and other current liabilities | 29,780 | 6,864 | ||||
| Total current liabilities | 47,905 | 10,755 | ||||
| Other long-term liabilities | 6,799 | 134 | ||||
| Total liabilities | 54,704 | 10,889 | ||||
| Shareholders’ equity: | 311,338 | 86,405 | ||||
| Total liabilities and shareholders’ equity | $ | 366,042 | $ | 97,294 | ||
Contacts
Corporate & Investor contact
+1 862-242-0764
Renee.Leck@orchard-tx.com
Media contact
LifeSci Public Relations
+1 646-627-8383
Allison@lifescipublicrelations.com
Source: Orchard Therapeutics Limited