Orchard Therapeutics Announces Expansion of Neurometabolic Disease Portfolio and Reports First Quarter 2019 Financial Results
New Collaboration with Fondazione Telethon and Ospedale San Raffaele for Clinical Program in Mucopolysaccharidosis Type I (MPS-I) Using Ex Vivo Autologous Hematopoietic Stem Cell Gene Therapy
Anticipated Marketing Authorization Application (MAA) Submission for the Treatment of Metachromatic Leukodystrophy (MLD) Brought Forward to the First Half of 2020
Clinical Trial in Sanfilippo Syndrome Type A (MPS-IIIA) Now Expected to Start Later This Year
Ended the First Quarter of 2019 with Approximately
Conference Call Scheduled for Today at
“The progress we’ve made advancing ex vivo autologous hematopoietic stem cell-based gene therapies aimed at correcting neurometabolic diseases furthers our confidence in the potential of our technology to treat additional serious, often fatal, rare diseases that to date have been difficult or impossible to treat,” said
Summary of Neurometabolic Franchise Updates
- Mucopolysaccharidosis Type I (MPS-I): Orchard has been granted an exclusive worldwide license from Fondazione Telethon and Ospedale San Raffaele to research, develop, manufacture and commercialize an ex vivo autologous HSC gene therapy program for the treatment of MPS-I, which will be referred to as OTL-203. The program has currently shown encouraging preliminary data with signs of metabolic correction in patients with the most severe subtype of MPS-I, known as Hurler syndrome, in the ongoing proof-of-concept clinical trial being conducted at
San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) inMilan, Italy . As of the data presented at theAmerican Society of Gene & Cell Therapy (ASGCT) annual meeting inApril 2019 , four patients have been enrolled in the trial with follow-up of up to nine months. The trial is expected to enroll up to eight patients by the first half of 2020. The terms of the license include an upfront cash payment, success-based milestones and royalties on net sales. - Metachromatic Leukodystrophy (MLD): Orchard held a positive Marketing Authorization Application (MAA) pre-submission meeting with the
European Medicines Agency (EMA) in early May. The company has brought forward the timeline for the planned submission of an MAA to the EMA for OTL-200 to the first half of 2020 and also expects to file a Biologics License Application (BLA) in the U.S. approximately one year after the MAA submission. - Sanfilippo Syndrome Type A (MPS-IIIA): Last week, the
Manchester University NHS Foundation Trust issued a statement that the Royal Manchester Children’s Hospital (RMCH) is the first in the world to treat an MPS-IIIA patient with an ex vivo HSC gene therapy. This was conducted under a “Specials” license, granted by theUK government for the use of an unlicensed pharmaceutical product in situations of high unmet need when there is no other treatment option available. Orchard holds the license to the MPS-IIIA program (OTL-201) and will support an upcoming proof-of-concept clinical trial, which will be conducted at RMCH, utilizing the same technology and procedures that were used to treat this first MPS-IIIA patient. The trial is expected to begin enrolling patients later this year.
First Quarter Business Milestone Achievements
- Achieved clinical proof-of-concept and presented data for OTL-300 for the treatment of transfusion-dependent beta-thalassemia (TDT). Data presented at ASGCT in
April 2019 demonstrated that eight of nine patients had a reduced or eliminated need for transfusions 12 months following treatment, with four of six pediatric patients achieving transfusion independence. - Presented two- and three-year follow-up data on 20 patients from the OTL-200 registrational trial for MLD, using the fresh product formulation, at the 45th Annual Meeting of the
European Society for Blood and Marrow Transplantation (EBMT). In the study, late infantile MLD patients achieved gross motor function scores of 65 and 72 percentage points higher at two and three years following gene therapy than MLD patients in a natural history patient cohort who did not receive gene therapy. - Dosed the first patient in a clinical trial using the cryopreserved formulation of OTL-103 for patients with Wiskott-Aldrich syndrome (WAS). This program remains on track for BLA and MAA filings in 2021.
First Quarter 2019 Financial Results
Cash, cash equivalents and restricted cash as of
Research and development expenses were
Selling, general and administrative expenses were
Net loss attributable to ordinary shareholders was
Credit Facility
In May, Orchard signed a five-year senior credit facility for up to
The company expects that its cash and investments as of
“The MPS-I program is a significant and important addition to our portfolio, enabling us to leverage our expertise in neurometabolic diseases and further extend the potential reach in addressing these conditions,” said
Conference Call & Webcast Information
Orchard will host a conference call and live webcast with slides today at
About Orchard
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
About MidCap Financial
MidCap Financial is a middle market-focused, specialty finance firm that provides senior debt solutions to companies across all industries. MidCap is headquartered in Bethesda, MD, with offices in Chicago and Los Angeles, and provides a broad array of products intended to finance growth and manage working capital. For more information, visit www.midcapfinancial.com.
MidCap Financial refers to
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,” “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 into 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 Data | ||||||||||
| (in thousands, except share and per share data) | ||||||||||
| (Unaudited) | ||||||||||
| Three Months Ended March 31, | ||||||||||
| 2019 | 2018 | |||||||||
| Costs and operating expenses | ||||||||||
| Research and development | $ | 17,493 | $ | 9,171 | ||||||
| Selling, general and administrative | 10,790 | 4,527 | ||||||||
| Total costs and operating expenses | 28,283 | 13,698 | ||||||||
| Loss from operations | (28,283 | ) | (13,698 | ) | ||||||
| Other income (expense), net | (1,863 | ) | (1,696 | ) | ||||||
| Net loss before income tax | (30,146 | ) | (15,394 | ) | ||||||
| Income tax (expense) benefit | (593 | ) | 83 | |||||||
| Net loss attributable to ordinary shareholders | (30,739 | ) | (15,311 | ) | ||||||
| Net loss per share attributable to ordinary shareholders, basic and diluted | $ | (0.35 | ) | $ | (1.53 | ) | ||||
| Weighted average number of ordinary shares outstanding, basic and diluted | 87,010,596 | 9,983,754 | ||||||||
| Condensed Consolidated Balance Sheet Data | ||||||||
| (in thousands) | ||||||||
| (Unaudited) | ||||||||
| March 31, | December 31, | |||||||
| 2019 | 2018 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 295,407 | $ | 335,844 | ||||
| Trade and other receivables | 354 | 2,153 | ||||||
| Prepaid expenses and other assets | 6,731 | 6,935 | ||||||
| Research and development tax credit receivable | 16,094 | 10,585 | ||||||
| Total current assets | 318,586 | 355,517 | ||||||
| Non-current assets: | ||||||||
| Property and equipment, net | 5,685 | 5,476 | ||||||
| Restricted cash | 3,840 | 3,837 | ||||||
| Other long-term assets | 1,208 | 1,212 | ||||||
| Total non-current assets | 10,733 | 10,525 | ||||||
| Total assets | $ | 329,319 | $ | 366,042 | ||||
| Liabilities and shareholders' equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 8,384 | $ | 18,125 | ||||
| Accrued expenses and other current liabilities | 26,316 | 29,780 | ||||||
| Total current liabilities | 34,700 | 47,905 | ||||||
| Other long-term liabilities | 7,143 | 6,799 | ||||||
| Total liabilities | 41,843 | 54,704 | ||||||
| Shareholders’ equity: | 287,476 | 311,338 | ||||||
| Total liabilities and shareholders’ equity | $ | 329,319 | $ | 366,042 | ||||
Contact:
+1 862-242-0764
Renee.Leck@orchard-tx.com
Source: Orchard Therapeutics