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 $366.2M in Total Cash and Investments

Conference Call Scheduled for Today at 4:30 p.m. ET

BOSTON and LONDON, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced financial results and business highlights for the quarter ended September 30, 2019, as well as the achievement of certain corporate milestones for the primary immune deficiencies franchise.

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 Mark Rothera, president and chief executive officer of Orchard Therapeutics. The data presented at several major medical conferences throughout the quarter showcase the profound impact that our hematopoietic stem cell-based gene therapies may have across multiple life-threatening diseases. We are working with care and urgency to make these therapies broadly available to patients who need them.

Recent Achievements for the Neurometabolic Franchise

Metachromatic Leukodystrophy (MLD):

Mucopolysaccharidosis type I (MPS-I):

Sanfilippo Syndrome type A (MPS-IIIA):

Upcoming Data Presentations for Primary Immune Deficiencies Franchise

Third Quarter 2019 Financial Results

Cash, cash equivalents and investments as of September 30, 2019, were $366.2 million compared to $335.9 million as of December 31, 2018. The increase was primarily driven by proceeds from the companys public equity offering in June 2019 and a credit facility entered in May 2019, partially offset by the net cash used to fund operations in the nine months ended September 30, 2019. During the three months ended September 30, 2019, the companys cash used to fund operations included the paydown of accrued liabilities as of June 30, 2019, for the upfront payment and a milestone payment related to the license of a clinical-stage MPS-I program, or OTL-203.

During the three months ended September 30, 2019, the company recognized $1.9 million in revenue related to European sales of Strimvelis, the first gene therapy approved by the European Medicines Agency for ADA-SCID.

Research and development expenses were $28.5 million for the three months ended September 30, 2019 compared to $27.7 million in the same period in 2018. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and milestone payments under the companys agreements with third parties, and personnel costs to support these activities. The company expects R&D expenses to continue to increase as its programs advance through development.

Selling, general and administrative expenses were $14.2 million for the three months ended September 30, 2019, compared to $7.5 million in the same period in 2018. The increase was primarily due to increased investment to prepare for the potential commercialization of multiple late-stage programs, as well as higher costs to support public company operations.

Net loss attributable to ordinary shareholders was $36.7 million for the three months ended September 30, 2019, compared to $33.9 million in the same period in 2018. The increase in net loss as compared to the prior year was primarily due to higher costs related to pre-launch activities on the companys later-stage programs in development and expenses associated with being a public company, partially offset by higher interest income. The company had 97.8 million weighted average ordinary shares outstanding for the three months ended September 30, 2019.

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 Frank Thomas, Orchards chief financial officer and chief business officer. Having treated multiple patients with Strimvelis this quarter at a single center in Milan, Italy, we have deepened our understanding of the support required for patients and families along their treatment journey, successfully secured reimbursement for cross-border healthcare delivery within Europe and completed the manufacturing and release process scheduled and tailored for each individual patient. We look forward to expanding these capabilities and applying key learnings to future commercial gene therapies at multiple centers around the world using cryopreserved formulations of our future products, if approved.

Conference Call & Webcast Information

Orchard will host a conference call and live webcast with slides today at 4:30 p.m. ET to discuss the third quarter results and recent and upcoming business activities. To participate in the conference call, please dial 1-866-987-6504 (domestic) or +1-602-563-8620 (international) and refer to conference ID 8413109. A live webcast of the presentation will be available under "News & Events" in the Investors & Media section of the company's website at orchard-tx.com and a replay will be archived on the Orchard website following the presentation.

About Orchard

Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards 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 European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, initiated in 2010.

Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards 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 Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards 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 companys 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 Orchards 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 Orchards 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 Orchards 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 Orchards product candidates,the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards 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 Orchards annual report on Form 20-F for the year ended December 31, 2018, as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Condensed Consolidated Statements of Operations(In thousands, except share and per share amounts) (unaudited)

Condensed Consolidated Balance Sheets(In thousands) (unaudited)

Contact:Renee Leck, Director of Investor Relations Orchard Therapeutics+1 862-242-0764Renee.Leck@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2019 Financial Results and Achievement of Key 2019 Milestones for Neurometabolic and Primary Immune...