Orchard Therapeutics Reports Third Quarter 2019 Financial Results and Achievement of Key 2019 Milestones for Neurometabolic and Primary Immune Deficiencies Franchises
ASH Abstracts Published Today Confirm Achievement of All Key Endpoints in Wiskott-Aldrich Syndrome Registrational Trial and Demonstrate Consistency of Data Between the Fresh and Cryopreserved Formulations of Investigational Gene Therapies
Marketing Authorization Application in EU for Metachromatic Leukodystrophy on Track for Near-Term Submission Following Recent Presentation of Cryopreservation Data
Ended the Third Quarter of 2019 with Approximately
Conference Call Scheduled for Today at
“We made important clinical and operational progress at Orchard over the past quarter, advancing our three lead programs toward regulatory submissions while moving additional programs through earlier stages of clinical development,” said
Recent Achievements for the Neurometabolic Franchise
Metachromatic Leukodystrophy (MLD):
- Presented engraftment data in four patients receiving the cryopreserved formulation of OTL-200 for the treatment of MLD at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress. The initial data showed that cellular engraftment using a cryopreserved formulation was similar to that observed using a fresh formulation, with the longest patient having 12 months of follow-up since treatment. Link to the full release here.
- In addition, an integrated analysis of 29 patients receiving the OTL-200 fresh formulation, which demonstrated positive clinical effects in the treatment of MLD and a consistent safety profile, was presented at the
Society for the Study of Inborn Errorsof Metabolism (SSIEM) 2019 Symposium. Link to the full release here.
- The company is on-track for a near-term Marketing Authorization Application (MAA) submission for OTL-200.
Mucopolysaccharidosis type I (MPS-I):
- Presented data from the ongoing proof-of-concept trial of OTL-203 for MPS-I at SSIEM. Evidence of engraftment and overexpression of the alpha-l-iduronidase (IDUA) enzyme in the peripheral blood and cerebrospinal fluid (CSF) was seen at the time of analysis in the six patients treated. Link to the full release here.
-- An abstract has been accepted with updated proof-of-concept data for an oral presentation at the 61st
American Society of Hematology(ASH) Annual Meeting in December.
-- The study is expected to enroll eight patients by the first half of 2020, with primary endpoint results expected after one year of follow-up.
Sanfilippo Syndrome type A (MPS-IIIA):
- The clinical trial application for a proof-of-concept trial at the Royal Manchester Children’s Hospital (RMCH) for Orchard’s MPS-IIIA program (OTL-201) was recently accepted and enrollment is expected to begin later this year.
- This trial utilizes the same technology and procedures that were used to treat the first MPS-IIIA patient with ex vivo hematopoietic stem cell-based (HSC) gene therapy at RMCH under a “Specials” license.
- The patient treated under the “Specials” license continues to do well approximately 10 months after treatment, according to a statement made this week by the
Manchester University NHS Foundation Trust.
-- Six-month results indicate early evidence of metabolic correction with supranormal N-sulfoglucosamine sulfohydrolase (SGSH) enzyme activity in peripheral leukocytes and reduction of glycosaminoglycans (GAGs) in the urine, CSF and plasma.
Upcoming Data Presentations for Primary Immune Deficiencies Franchise
- As announced in a separate release this morning, the company will present data from two registrational programs (OTL-103 for the treatment of Wiskott-Aldrich Syndrome (WAS) and OTL-101 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID)) at the ASH meeting in December. Highlights include:
-- OTL-103 for WAS: The registrational trial has met its key primary and secondary endpoints (n=8 at three years) with data from an integrated analysis (n=17) reinforcing the treatment benefits of gene therapy and durability of effect in additional patients. Successful engraftment was observed within three months, leading to an increase in WAS protein expression and a vector copy number that has been maintained for up to eight years.
-- OTL-101 for ADA-SCID: Similar profiles and engraftment have been observed between the cryopreserved and fresh formulations of OTL-101, which represents an important achievement toward the future regulatory filing and potential approval of this investigational gene therapy and a key step toward global patient availability.
Third 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.
“Orchard is well positioned to enter its next phase of growth as a company with potentially multiple commercial-stage gene therapies, building on a proven track record in identifying and treating patients with Strimvelis, the first ex vivo HSC gene therapy on the market,” said
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, 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, 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 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
Condensed Consolidated Statements of Operations
(In thousands, except share and per share amounts)
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Product sales, net||$||1,918||$||1,387||$||1,918||$||1,387|
|Costs and operating expenses:|
|Cost of product sales||614||280||614||280|
|Research and development||28,493||27,731||86,464||187,893|
|Selling, general and administrative||14,223||7,466||38,687||19,414|
|Total costs and operating expenses||43,330||35,477||125,765||207,587|
|Loss from operations||(41,412||)||(34,090||)||(123,847||)||(206,200||)|
|Total other income (expense), net||2,481||935||3,468||1,336|
|Net loss before income tax||(38,931||)||(33,155||)||(120,379||)||(204,864||)|
|Income tax (expense) benefit||2,194||(733||)||2,373||(568||)|
|Net loss attributable to ordinary shareholders||(36,737||)||(33,888||)||(118,006||)||(205,432||)|
|Net loss per share attributable to ordinary shareholders, basic and diluted||$||(0.38||)||$||(3.29||)||$||(1.29||)||$||(20.27||)|
|Weighted average number of ordinary shares outstanding, basic and diluted||97,817,847||10,294,498||91,553,803||10,132,334|
Condensed Consolidated Balance Sheets
|September 30,||December 31,|
|Cash and cash equivalents||$||35,478||$||335,844|
|Trade and other receivables||5,006||2,153|
|Prepaid expenses and other assets||8,326||6,935|
|Research and development tax credit receivable, current||13,988||10,585|
|Total current assets||393,475||355,517|
|Property and equipment, net||5,714||5,476|
|Research and development tax credit receivable||8,240||—|
|Liabilities and shareholders’ equity|
|Accrued expenses and other current liabilities||32,712||29,780|
|Total current liabilities||44,307||47,905|
|Long-term debt, net||24,609||—|
|Other long-term liabilities||6,079||6,799|
|Total shareholders’ equity||339,744||311,338|
|Total liabilities and shareholders’ equity||$||414,739||$||366,042|
Source: Orchard Therapeutics (Europe) Limited