UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 6-K
REPORT OF FOREIGN PRIVATE ISSUER
PURSUANT TO RULE 13a-16 OR 15d-16
UNDER THE SECURITIES EXCHANGE ACT OF 1934
For the month of January 2019
Commission File Number: 001-38722
ORCHARD THERAPEUTICS PLC
(Translation of registrant’s name into English)
108 Cannon Street
London EC4N 6EU
United Kingdom
(Address of principal executive offices)
Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.
Form 20-F ☒ Form 40-F ☐
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ☐
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ☐
On January 7, 2019, Orchard Therapeutics plc (the “Company”) issued a press release, a copy of which is attached hereto as Exhibit 99.1 and is incorporated herein by reference.
The Company will be conducting meetings with investors attending the 37th Annual J.P. Morgan Healthcare Conference in San Francisco beginning on January 7, 2019. As part of these meetings, the Company will deliver the slide presentation furnished to this report as Exhibit 99.2 and which is incorporated herein by reference.
The information in this report included as Exhibit 99.1 and Exhibit 99.2 and incorporated herein by reference shall not be deemed “filed” for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section. It may only be incorporated by reference in another filing under the Exchange Act or the Securities Act of 1933, as amended, if such subsequent filing specifically references the information furnished pursuant to this report.
EXHIBITS
| Exhibit |
Description | |
| 99.1 | Press Release dated January 7, 2019 | |
| 99.2 | Orchard Therapeutics plc Presentation at the 37th Annual J.P. Morgan Healthcare Conference, dated January 7, 2019. | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| ORCHARD THERAPEUTICS PLC | ||||||
| Date: January 7, 2019 | By: | /s/ Frank E. Thomas | ||||
| Frank E. Thomas Chief Financial Officer | ||||||
Exhibit 99.1
Orchard Therapeutics Highlights Recent Accomplishments and 2019 Strategic Priorities as a Global Leader in Gene Therapy
Preparing Three Lead Programs for MLD, ADA-SCID and WAS for Regulatory Filings Over the Next Three Years
Recently Announced Clinical Proof-of-Concept in X-CGD Demonstrates Platform’s Transformative Potential
Advancing Earlier Stage Pipeline with Potential Clinical Proof-of-Concept for TDBT and Clinical Trial Application for MPS-IIIA
Entering 2019 in a Strong Financial Position with $340M in Cash and Investments
BOSTON and LONDON, Jan. 7, 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 summarized recent accomplishments and 2019 strategic priorities in conjunction with its attendance at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco. Mark Rothera, president and chief executive officer, will present a business overview outlining the company’s progress as a global leader in gene therapy on Wednesday, January 9, 2019 at 3:00 p.m. PT that will be webcast at ir.orchard-tx.com.
“2018 was a momentous year for Orchard, marked by the success of our acquisition and integration of GSK’s rare disease ex-vivo gene therapy portfolio, initial scaling of our manufacturing capabilities and completion of our initial public offering,” said Mr. Rothera. “2019 will continue the company’s evolution as a leader in gene therapy, with multiple clinical milestones supporting three regulatory filings over the next three years and growing manufacturing capabilities. We have a bold vision and are well on our way to delivering gene therapies that have the potential to transform the lives of patients with rare, life-threatening diseases worldwide with a single treatment.”
2019 Strategic Priorities
Neurometabolic Disorders
| • | Release two and three-year follow-up data in 20 patients from the fresh formulation registrational trial of OTL-200 for metachromatic leukodystrophy (MLD) |
| • | Release engraftment data in the first three patients from the cryopreserved formulation clinical trial of OTL-200 for MLD |
| • | Submit clinical trial application (CTA) for OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA) and support initiation of a clinical trial |
Primary Immune Deficiencies
| • | Release two-year follow-up data in 20 patients from the fresh formulation registrational trial of OTL-101 in adenosine deaminase severe combined immune deficiency (ADA-SCID) |
| • | Release engraftment data in 10 patients from a cryopreserved formulation clinical trial of OTL-101 in ADA-SCID |
| • | Release three-year follow-up data in eight patients from the fresh formulation registrational trial of OTL-103 in Wiskott-Aldrich syndrome (WAS) |
| • | Initiate cryopreservation formulation clinical trial for OTL-103 in WAS |
| • | Design and engage regulators on registrational trial for OTL-102 in X-linked chronic granulomatous disease (X-CGD), which recently achieved clinical proof-of-concept (link to full release here) |
Hemoglobinopathies
| • | Report clinical proof-of-concept data for OLT-300 in transfusion-dependent beta-thalassemia (TDBT) |
Major 2018 Accomplishments
Pipeline Expansion and Advancement
| • | Completed the strategic acquisition and subsequent integration of GSK’s rare disease ex-vivo gene therapy portfolio, including Strimvelis®, the only treatment for patients with ADA-SCID approved in the EU, along with clinical programs in MLD, WAS and TDBT |
| • | Completed pre-biologics license application (BLA) and CMC specific meetings with the U.S. Food and Drug Administration (FDA) for OTL-101 for ADA-SCID, following which the program remains on track for a BLA filing in the U.S. in 2020 |
| • | Achieved clinical proof of concept for OTL-102 in X-CGD, demonstrating sustained levels of functioning neutrophils in patients after 12 months |
| • | Obtained Rare Pediatric Disease Designations from the FDA for OTL-200 for the treatment of MLD and OTL-201 for the treatment of MPS-IIIA |
| • | Obtained priority medicines (PRIME) designation from the European Medicines Agency (EMA) for OTL-300 for the treatment of TDBT |
Corporate & Manufacturing Developments
| • | Raised approximately $375 million in gross proceeds in 2018 from a Series C financing and underwritten initial public offering |
| • | Leased a manufacturing site in Fremont, CA and opened a Boston, MA corporate office. The manufacturing facility will enhance the company’s capacity to develop and deliver ex-vivo lentiviral vector and gene-corrected hematopoietic stem cells for a wide range of rare diseases on a global scale and will complement the existing network of partner CMOs that will underpin the launches for the first three programs. (Link to full release here) |
Cash Guidance
The company ended 2018 with approximately $340 million of cash and investments. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2018 will enable the company to fund its currently anticipated operating expenses and capital expenditure requirements into the second half of 2020.
Presentation at 37th Annual J.P. Morgan Healthcare Conference
Orchard will webcast its corporate presentation from the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 9, 2019 at 3:00 p.m. PT. 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. A replay of the webcast 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.
Orchard’s portfolio of autologous ex vivo gene therapies includes Strimvelis®, the first autologous ex vivo gene therapy approved by the European Medicines Agency for adenosine deaminase severe combined immunodeficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies 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) and transfusion-dependent beta-thalassemia (TDBT), as well as an extensive preclinical pipeline.
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 which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. 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 Orchard’s guidance that its existing cash, cash equivalents and marketable securities as of December 31, 2018 will enable the company to fund its anticipated operating expenses and
capital expenditure requirements into 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 Securities and Exchange Commission pursuant to Rule 424(b) of the Securities Act of 1933, as amended, as well as subsequent filings and reports filed by Orchard with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of publication of this document. Orchard undertakes no 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.
Contacts
Corporate & Investor contact
Renee Leck
Orchard Therapeutics
+1 862-242-0764
Renee.Leck@orchard-tx.com
Media contact
Allison Blum, Ph.D.
LifeSci Public Relations
+1 646-627-8383
Allison@lifescipublicrelations.com
Mark Rothera President & Chief Executive Officer January 9, 2019 Exhibit 99.2
Forward Looking Statements Certain information set forth in this presentation and in statements made orally during this presentation contains “forward-looking statements”. Except for statements of historical fact, information contained herein constitutes forward-looking statements and includes, but is not limited to, the Company’s expectations regarding: (i) the safety and efficacy of its product candidates; (ii) the expected development of the Company’s business and product candidates; (iii) the timing of regulatory submissions for approval of its product candidates; (iv) the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates; (v) 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; (vi) the likelihood of approval of such product candidates by the applicable regulatory authorities; (vii) execution of the Company’s vision and growth strategy, including with respect to global growth; and (viii) projected financial performance and financial condition, including the sufficiency of the Company’s cash and cash equivalents to fund operations in future periods and future liquidity, working capital and capital requirements. The words “may,” “should,” “expects,” “intends," “plans,” “anticipates,” “believes,” “estimates,” “predicts," “potential,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements are provided to allow investors the opportunity to understand management’s beliefs and opinions in respect of the future so that they may use such beliefs and opinions as one factor in evaluating an investment. These statements are neither promises nor guarantees of future performance. Such forward-looking statements necessarily involve known and unknown risks and uncertainties, which may cause actual performance and financial results in future periods to differ materially from any projections of future performance or result expressed or implied by such forward-looking statements. You are cautioned not to place undue reliance on forward-looking statements. These statements are subject to a variety of risks and uncertainties, many of which are beyond the Company’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. For additional disclosure regarding these and other risks faced by the Company, see the disclosure contained in the Company’s public filings with the Securities and Exchange Commission, including in the final prospectus related to the Company’s initial public offering filed with the Securities and Exchange Commission pursuant to Rule 424(b) of the Securities Act of 1933, as amended, as well as subsequent filings and reports filed with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this presentation. The Company undertakes no 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.
Global Fully Integrated Biotech Dedicated to Transforming the Lives of Patients with Rare Diseases Through Innovative Gene Therapies Singular focus on autologous ex-vivo gene therapy for rare diseases
Orchard’s Lead Programs Show Transformative Potential Source: UCLA study; n=20 Overall survival data Gross motor function measure (GMFM) GMFM total score in late infantile (LI) MLD at 24 months post OTL-200 vs. natural history Severe infection rate Pre Gene Therapy Post Gene Therapy OTL-101 for ADA SCID OTL-200 for MLD OTL-103 for WAS 100% overall survival 66% treatment difference vs. untreated Reductions in severe infections Source: clinical study report (CSR) of 05 Dec 2017 Source: interim clinical study report (CSR) of 10 Jan 2017 Threshold for primary endpoint (10 percentage points above natural history)
Orchard Now a Leading Global Gene Therapy Company 2 clinical stage programs 5 pre-clinical programs 55 employees ~$320M post Series B London, UK Single CA site Primarily investors 1 commercial product 5 clinical stage programs 10 pre-clinical programs ~180 employees Executive leadership team in place ~$1.3B market cap post IPO $340M cash on the balance sheet London, UK Boston, MA Multiple CA sites Primarily independent industry experts Entering 2018 Entering 2019 Pipeline People Value Locations Board of Directors
Global Leadership in Gene Therapy for Rare Diseases Deep pipeline of five clinical-stage gene therapies & potential to treat CNS disorders Over 150 patients treated, with promising clinical data and durable long-term effects Three submissions for product approvals anticipated over the next three years (MLD, ADA-SCID, WAS) Establishing manufacturing capabilities to deliver products globally Recently announced X-CGD clinical POC and TDBT clinical POC expected in 2019 Strong balance sheet entering 2019 with $340M in cash
Strimvelis® OTL-101 OTL-103 OTL-102 OTL-3003 OTL-200 OTL-201 OTL-202 Deep Pipeline of Gene Therapies with Transformative Potential Preclinical Clinical proof of concept Registrational trial Commercialization Designations Neurometabolic disorders Hemoglobinopathies Primary immune deficiencies MLD (metachromatic leukodystrophy) MPS-IIIA (Sanfilippo type A) TDBT (transfusion-dependent beta-thalassemia) X-CGD (X-linked chronic granulomatous disease) WAS (Wiskott–Aldrich syndrome) ADA-SCID (adenosine deaminase severe combined immunodeficiency) Several additional research and preclinical programs under development MPS-IIIB (Sanfilippo type B) ADA-SCID (adenosine deaminase severe combined immunodeficiency) PRIME RPD; BKT BKT PRIME RPD RPD RPD RPD RPD Program with Rare Pediatric Disease Designation; eligible for a Priority Review Voucher Breakthrough Therapy Designation; Priority Medicine (PRIME) Designation
Over 150 Patients Treated with Orchard’s Autologous Ex Vivo Gene Therapies 1 Patients treated in the development phase, including in clinical trials and under pre-approval access (defined as any form of pre-approval treatment outside of a company-sponsored clinical trial, including, but not limited to, compassionate use, early access, hospital exemption or special license). Data as of December 2018 Data include all patients treated with CD34+ hematopoietic stem cells transduced ex vivo with vector of interest. Franchise Program Patients treated1 Longest patient follow-up Primary immune deficiencies Strimvelis® (ADA-SCID) 24 18 years OTL-101 (ADA-SCID) 62 6 years OTL-103 (WAS) 16 8 years OTL-102 (X-CGD) 10 3 years Neurometabolic disorders OTL-200 (MLD) 32 8 years Hemoglobinopathies OTL-300 (TDBT) 9 3 years Total 153 patients Persistent, long-term effects across five indications
Delivering Therapeutic Genes to Multiple Physiological Systems Platelets Granulocyte MLD MPS-IIIA MPS-IIIB ADA-SCID WAS X-CGD CD34+ Hematopoietic stem cells (HSCs) TDBT WAS Viral vector carrying therapeutic gene Macrophage / Microglia Central nervous system Immune system Red blood cell and platelet lineage Progenitor Cells Potential for sustained disease correction after a single administration via gene-modified HSCs engraftment Erythrocyte B cells T cells NK cells Hematopoietic stem cells Mature blood cells Physiological systems Current Indications
Numerous Data and Clinical Milestones Anticipated in 2019 OTL-200 (MLD) 2 & 3 year follow-up fresh formulation (n=20) Cryo formulation engraftment data (n=3) Clinical Trial Initiations & Other Milestones 3 Registrational Clinical Trial Data Sets OTL-101 (ADA-SCID) 2 year follow-up fresh formulation (n=20) Cryo formulation engraftment data (n=10) OTL-103 (WAS) 3 year follow-up fresh formulation (n=8) OTL-103 (WAS) Initiate cryo formulation trial OTL-102 (X-CGD) Design registrational trial & engage regulators OTL-300 (TDBT) Report data from POC trial (n=9) OTL-201 (MPS-IIIA) Submit CTA & support clinical trial initiation
Neurometabolic Disorders
Delivery of Proteins to the Brain Unlocks Potential to Treat Large Number of Neurometabolic Diseases Source: Capotondo et al. PNAS 2012;109:15018-15023; Brain of a wildtype mouse transplanted with GFP-LV transduced HSPCs after Busulfan conditioning Green = GFP (green fluorescent protein); blue = nuclei staining MLD MPS-IIIA MPS-IIIB Multiple potential additional neurometabolic indications Broad transgene distribution in brain of mouse after administration of HSCs transduced with GFP-encoding vector
Devastating Neurometabolic Diseases with No Approved Treatment Options 1 ARSA: arylsulfatase-A; 2 Mahmood (2010); 3 Sergijenko (2013) and Boelens (2010); 4 Buhrman (2013) SGSH: N-sulfoglycosamine sulfohydrolase; NAGLU: N-acetyl-alpha-glucosaminidase Disease Overview / Symptoms Deficiency in the ARSA1 enzyme Rapid & progressive neurodegeneration with loss of motor & cognitive function Incidence: 400-770 patients per year Deficiencies in the SGSH (MPS-IIIA) and NAGLU (MPS-IIIB) enzymes Progressive neurodegeneration, subsequent motor function decline; loss of language and mobility; seizures MPS-IIIA incidence: 250-480 patients per year Prognosis Severe form with high mortality rates: Infantile: 50% at 5 years (onset 0-3 years)2 Juvenile: 44% at 10 years (onset 3-16 years)2 Life expectancy: 10-25 years (MPA-IIIA) and 15-30 years (MPS-IIIB) Current Treatment Largely palliative addressing symptoms Very limited to no efficacy with allogeneic HSCT Largely palliative addressing symptoms Allogeneic HSCT not shown to be effective3 ERT not effective treating neurological manifestations4 Metachromatic Leukodystrophy (MLD) Sanfilippo Syndrome Type A and Type B (MPS-IIIA, MPS-IIIB)
OTL-200 for MLD: Significant Improvements in Motor Function MAA Submission Expected in 2020 (followed by BLA) Treatment difference (OTL-200 – untreated): 66.1% (LI) and 45% (EJ) respectively Source: clinical study report (CSR) of 05 December 2017 Late infantile MLD - GMFM Total Score at 24 months post OTL-200 vs. natural history Early juvenile MLD - GMFM Total Score at 24 months post OTL-200 vs. natural history 66% treatment difference vs natural history 45% treatment difference vs natural history Threshold for primary endpoint (10 percentage points above natural history) 32 patients treated (23 under clinical trials; 9 under compassionate use program) Evidence of normalized motor and cognitive function with early treatment Ongoing clinical trial with cryopreserved formulation (3 of 10 patients enrolled)
~80% decrease in heparan sulfate vs. MPS-IIIA wild type 11% enzyme expression vs. wild type OTL-201 and OTL-202 (MPS-IIIA And MPS-IIIB): Preclinical Proof of Concept CTA Submission for MPS-IIIA Expected in 2019 Sergijenko et al, Mol. Ther. 2013, 21(10), 1938-1949 Increased enzyme expression in the brain Decreased substrate accumulation in the brain Full behavioral correction to wild type levels WT MPS-IIIA LV-CD11b LAMP2 / NeuN Staining of neurons and lysosomes Percentage enzyme vs. wild type 100% 75% Frequency speed > 100 mm/second Reduced hyperactivity
Primary Immune Deficiencies (PIDs)
Life Threatening Inherited Immune Disorders: ADA-SCID, WAS and X-CGD 1 Oszahin (2008); Albert (2011); 2 Dupuis-Girod (2003); 3 van den Berg et. al, PLoS One. 2009;4(4):e5234. Disease Overview / Symptoms Deficiency in ADA enzyme T, B, and NK cell dysfunction Recurrent and life-threatening severe infections Incidence 80 – 180 patients per year Deficiency in WAS protein Thrombocytopenia causing severe bleeding and infections, eczema, autoimmunity and life-threatening malignancies1 Incidence 100 – 260 patients per year Deficiency in NADPH oxidase function Neutrophils / granulocytes unable to kill bacterial and fungal pathogens Life-threatening, repeated chronic fungal and bacterial infections Incidence 200 – 320 patients per year Prognosis Usually fatal within first two years of life without treatment Median survival ~15 years with conservative treatment2 ~40% mortality by age 353 Current Treatment Strimvelis (EU only) Allogenic HSCT Chronic ERT Conservative care Allogeneic HSCT Prophylactic antibiotics, antifungals and interferon Allogeneic HSCT Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Wiskott-Aldrich Syndrome (WAS) X-linked Chronic Granulomatous Disease (X-CGD)
OTL-101 for ADA-SCID: Registrational Trial Supports Transformative Potential BLA Submission Expected in 2020 (followed by MAA) Data from registrational 2-year fresh cell product; n=20 Overall Survival Event-free Survival 100% overall survival (n=20) 100% event-free survival (n=20) 62 patients treated in total as of December 2018 Up to 6.5 years follow-up 100% overall survival; ~95% event-free survival
OTL-103 for WAS: Evidence of Consistent and Durable Efficacy Cryo Trial to Initiate 2019; BLA/MAA submission in 2021 Data based on interim clinical study report of 10 Jan 2017; figures reflect data as of the cut-off date of 29 April 2016 Bleedings per patient per year Severe infections per patient per year Pre-gene therapy Post-gene therapy Pre-gene therapy Post-gene therapy Reduction in the rate of severe infections, bleeding events and hospitalizations Well-tolerated among 16 patients treated (8 under clinical trials; 8 under compassionate use program)
OTL-102 for X-CGD: Evidence of Sustained Neutrophil Activity in Patients Proof of Concept Established in December 2018 Data provided by Great Ormond Street Hospital, Boston Children Hospital, NIH and UCLA; unaudited data as of 07-May-2018; † patient deceased from advanced disease Excludes data from 1 patient treated with drug product deemed by the investigator as different from the OTL-102 drug product Oxidase activity – % of DHR-positive peripheral mononuclear cells Functional neutrophils above 10% at 12 months in 6 patients providing clinical benefit 10% † † † †
CMO Infrastructure Established for Launch of First Three Cryopreserved Gene Therapy Products Isolate hematopoietic stem cells (HSCs) Gene-modify HSCs with lentiviral vector Cryopreserve HSC product Manufacture disease-specific lentiviral vector Collect blood or bone marrow Thaw and infuse product Ship cryopreserved product to hospital Ship tissue to manufacturing facility Conditioning Local treatment of patients in hospital Recently announced build-out of Orchard manufacturing facility to provide capacity and long-term security of supply Orchard Therapeutics supply chain Lentiviral vector manufacturing Drug product manufacturing (cell processing) Well positioned to supply market given focus on pediatric diseases
New facility will provide significant additional CGMP manufacturing capacity for lentiviral vector and cryopreserved cell therapy products Enhancing Our Ability to Supply Ex Vivo Gene Therapy Programs Drives efficiencies and scalability in terms of lentiviral vector and drug product development Complements existing vector and drug product manufacturing partner capabilities
Lead Indications Represent Potential >$2B Market Opportunity Orchard Retains Full Commercial Rights to All Indications in All Markets Data based on Company estimates derived from published literature. Prevalence Incidence 2,200 – 6,300 2,900 – 4,700 > $2B market opportunity based predominantly on incidence prevalent population represents considerable upside 80 – 180 400 – 770 100 – 260 200 – 320 250 – 480 1,030 – 2,010 Significant population of both incident and prevalent patients
Numerous Data and Clinical Milestones Anticipated in 2019 OTL-200 (MLD) 2 & 3 year follow-up fresh formulation (n=20) Cryo formulation engraftment data (n=3) Clinical Trial Initiations & Other Milestones 3 Registrational Clinical Trial Data Sets OTL-101 (ADA-SCID) 2 year follow-up fresh formulation (n=20) Cryo formulation engraftment data (n=10) OTL-103 (WAS) 3 year follow-up fresh formulation (n=8) OTL-103 (WAS) Initiate cryo formulation trial OTL-102 (X-CGD) Design registrational trial & engage regulators OTL-300 (TDBT) Report data from POC trial (n=9) OTL-201 (MPS-IIIA) Submit CTA & support clinical trial initiation
Transforming the lives of patients through innovative gene therapies www.orchard-tx.com