Orchard Therapeutics Highlights 2020 Strategic Priorities
Commercial Preparations on Track for Potential 2020 EU Launch of OTL-200 for Metachromatic Leukodystrophy (MLD) with U.S. Regulatory Filing Expected Late 2020 / Early 2021
Initiation of Rolling U.S. Regulatory Filing for OTL-101 (ADA-SCID) Planned for 1H 2020;
U.S. and EU Regulatory Filings of OTL-103 (WAS) Expected in 2021
MPS-I and MPS-IIIA Proof-of-Concept Clinical Trials Ongoing with Additional Data Expected in 2020
BOSTON and LONDON,
Mark Rothera, Orchard’s president and chief executive officer, will present a business overview on
“2020 has the potential to be a watershed year for Orchard as we work to bring the benefits of our gene therapy approach and expertise to patients,” Rothera said. “We are preparing diligently for the anticipated EU regulatory approval of OTL-200 for MLD while building the commercial team and executing our go-to-market strategy that will help ensure broad patient access. At the same time, we are readying our first regulatory filings in the U.S. and advancing multiple clinical-stage programs through important milestones, while also exploring the potential for gene-corrected HSCs in a broader range of severe disorders, including non-rare indications. With an unrelenting focus on execution, our organization is highly motivated to bring these investigational therapies to patients around the world.”
2020 Corporate Priorities
Orchard has outlined the following three high-level corporate goals for 2020:
- Obtain approval for and launch OTL-200 for the treatment of MLD in
Europeand prepare for a biologics license application (BLA) filing in the U.S.;
- Advance two registrational programs in primary immune deficiencies toward regulatory filings; and
- Investigate the potential of our ex vivo HSC gene therapy platform approach in a broad set of neurodegenerative diseases and other new therapeutic areas, including ongoing proof-of-concept clinical trials in mucopolysaccharidosis type I (MPS-I) and mucopolysaccharidosis type IIIA (MPS-IIIA).
Obtain Approval for and Launch OTL-200 for MLD in
In preparation for a potential European approval in the second half of 2020, Orchard plans to establish the global infrastructure needed to support awareness and adoption of OTL-200. Orchard is putting in place a focused commercial team to serve as the backbone for the potential launch of OTL-200, as well as future product launches. A BLA filing for OTL-200 in the U.S. is planned for late 2020 or early 2021.
- Activities are underway to drive timely patient identification and access, including disease awareness, genetic testing and newborn screening pilots.
- The company continues to qualify treatment centers with specialized expertise in transplant and disease area knowledge across key geographies.
- The commercial supply chain for OTL-200 is being established, with the capacity and logistics to support an anticipated launch.
- The company has received stakeholder input on its value philosophy, as well as a set of guiding principles that will underpin Orchard’s pricing and market access strategy.
Advance Two Registrational Programs Toward Upcoming Regulatory Filings
In support of its lead primary immune deficiency programs:
- The company plans to initiate a rolling BLA filing in the U.S. for OTL-101 in adenosine deaminase severe combined immunodeficiency (ADA-SCID) in the first half of 2020 with anticipated completion of the filing within 12 months.
- The company anticipates BLA and MAA regulatory filings for OTL-103 in Wiskott-Aldrich Syndrome (WAS) in the U.S. and EU in 2021.
Investigate the Potential of ex vivo HSC Gene Therapy in Additional Neurodegenerative Diseases and New Therapeutic Areas
Orchard has entered into an agreement with Dr.
Dr. Biffi commented, “Developing gene therapies for diseases with central nervous system involvement, such as neurometabolic and other neurodegenerative diseases, has been a challenge in the field. I am thrilled to partner with Orchard to continue development of this innovative gene therapy technology and, together, look forward to advancing potentially transformative treatments for these devastating diseases.”
As part of the agreement, Dr. Biffi will serve as senior scientific advisor of neurometabolic diseases at Orchard. Dr. Biffi’s previous role at the
In addition, Orchard plans to continue to advance the following clinical-stage neurometabolic programs currently underway in MPS-I and MPS-IIIA:
- The OTL-203 proof-of-concept clinical trial in MPS-I, which is being conducted at SR-Tiget, has reached its initial enrollment target of eight study participants. One-year follow-up results for the first eight patients, including the primary endpoints, are anticipated in 2021, with interim data planned for presentations at medical conferences during 2020.
- Enrollment has initiated in a proof-of-concept clinical trial for OTL-201 in MPS-IIIA, conducted by the Royal Manchester Children’s Hospital. The trial is expected to enroll up to five patients in 2020 with interim data expected in 2020 and 2021.
Key 2019 Achievements
Orchard achieved each of its 2019 corporate milestones, with key achievements highlighted below.
- MLD European MAA submission: The Marketing Authorization Application (MAA) for OTL-200 for MLD was filed and accepted for review by the
European Medicines Agency(EMA) in November 2019, ahead of previous guidance.
- Cryopreserved gene therapy formulations: Similar engraftment profiles have been observed between the cryopreserved and fresh formulations of OTL-200 for MLD and OTL-101 for ADA-SCID, which represents an important achievement toward the potential approvals of these investigational gene therapies and a key step toward global patient availability.
- WAS registrational data set: The registrational trial for OTL-103 for WAS met its key primary and secondary endpoints (n=8 at three years), including the elimination of severe bleeding episodes and a significant reduction in the frequency of severe infections.
- MPS-I global license: Orchard signed an exclusive license with Fondazione Telethon and Ospedale San Raffaele in
Milan, Italy, for a clinical-stage HSC gene therapy program — OTL-203, a treatment for MPS-I that has shown promising early data in an ongoing proof-of-concept clinical trial.
The company ended 2019 with approximately
Presentation at 38th Annual
Orchard will webcast its corporate presentation from the 38th Annual
Availability of Other Information About Orchard
Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (twitter.com/orchard_tx and www.linkedin.com/company/10276396), including but not limited to investor presentations and investor fact sheets,
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,” “plans,” “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 company’s business strategy and goals, 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 approved, 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
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Source: Orchard Therapeutics (Europe) Limited