Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader,
today reported financial results for the year ended December 31,
2020, as well as recent accomplishments, 2021 strategic priorities
and upcoming milestones, and related organizational leadership
updates.
Frank Thomas, president and chief operating officer said, “It is
gratifying to witness the positive momentum Orchard has already
established in early 2021 driven by solid execution. Our compelling
data in neurodegenerative disorders at the WORLDSymposium and
successful completion of the $150 million financing exemplify this
recent progress and showcase a growing appreciation for the
potential of HSC gene therapy. We look forward to continuing our
work in the year ahead and delivering further benefit for patients
and our shareholders."
Recent Accomplishments
February 2021
- Executed a $150 million private
investment in public equity (PIPE) financing at a price of $6.22
per share with several existing and new investors with expertise in
healthcare, including RA Capital Management, Avidity Partners,
Casdin Capital, Farallon Capital, and Surveyor Capital (a Citadel
company), among others. The link to the full release is available
here.
- Presented data from multiple
neurometabolic programs at the 2021 WORLDSymposium. The link to the
full release is available here.
- Interim data for OTL-203 showing
positive clinical results in multiple disease manifestations of
MPS-IH was highlighted. All eight patients treated with OTL-203
showed stable cognitive function, motor function and growth within
the normal range at multiple data points post-treatment. Treatment
with OTL-203 has been generally well-tolerated with a safety
profile consistent with the selected conditioning regimen.
- An oral presentation featuring
supportive biomarker data from the first three patients in the
ongoing OTL-201 POC study for mucopolysaccharidosis type IIIA
(MPS-IIIA, or Sanfilippo syndrome) was also featured. The treatment
has been generally well-tolerated in the first three patients with
no treatment-related serious adverse events (SAEs), and all
transplant-related SAEs and adverse events have resolved.
January 2021
- Appointed Braden Parker as chief commercial officer. Mr. Parker
most recently served as Orchard’s senior vice president and general
manager for North America and has more than 20 years of experience
in the healthcare industry, including commercial leadership roles
at Celgene, NPS Pharma (Shire) and PTC Therapeutics, where he led
the company’s U.S. product launch in Duchenne muscular dystrophy
and oversaw strategic planning for the gene therapy business.
- Received Regenerative Medicine
Advanced Therapy (RMAT) designation for OTL-200 from the U.S. Food
and Drug Administration (FDA) for early-onset MLD. This designation
has the potential to enhance regulatory interactions in the U.S.
and open an expedited path to a biologics license application (BLA)
filing.
- Secured partnerships with two leading
regional specialty pharmaceutical companies to broaden access to
Libmeldy (OTL-200) for eligible patients with MLD in the Middle
East & Turkey.
2021 Corporate Priorities and Upcoming
Milestones
Orchard previously outlined the following key corporate
objectives and upcoming expected milestones:
- Build a successful commercial
business in hematopoietic stem cell (HSC) gene therapy
- Launch Libmeldy (OTL-200) for the
treatment of eligible patients with early-onset MLD in Europe in
the first half of 2021
- Complete additional interactions
with the FDA by mid-2021 to determine the path to a BLA filing for
OTL-200
- File an Marketing Authorization
Application (MAA) for OTL-103 in Wiskott-Aldrich syndrome (WAS)
with the European Medicines Agency by year-end 2021; followed by a
BLA filing in the U.S. in 2022
- Continue to lead the development of
gene therapies for neurodegenerative disorders by advancing two POC
programs in MPS-IH and MPS-IIIA
- Initiate a registrational trial for
OTL-203 for MPS-IH by year-end 2021
- Complete enrollment in the
five-patient POC trial for OTL-201 for MPS-IIIA
- Present additional clinical data
from the OTL-203 and OTL-201 POC trials
- Investigate the potential of HSC
gene therapy in larger indications
- Announce new preclinical data from research programs in
frontotemporal dementia with progranulin mutations (GRN-FTD) and
Crohn’s disease with mutations in the nucleotide-binding
oligomerization domain-containing protein 2 (NOD2-CD) in the second
half of 2021
Organizational Leadership Updates
Given the progress on key development programs, Anne Dupraz has
been appointed to the expanded role of chief development officer.
In addition to overseeing the company’s regulatory strategy, Ms.
Dupraz will lead product development with the goal of ensuring a
seamless approach to moving Orchard’s programs through to potential
regulatory approval. Ms. Dupraz possesses more than 20 years of
experience in the clinical and regulatory fields and has deep
expertise in advanced therapies, having been involved in more than
50 different tissue, cell and gene-based therapy development
programs in her career.
Ran Zheng, chief technical officer, and Andrea Spezzi, chief
medical officer, are stepping down from their respective leadership
positions with Orchard to pursue other opportunities. Orchard has
initiated a global search for permanent replacements for both of
these roles.
Fourth Quarter 2020 Financial Results
Research and development expenses were $22.6 million for the
three months ended December 31, 2020, compared to $30.9 million in
the same period in 2019. R&D expenses include the costs of
clinical trials and preclinical work on the company’s portfolio of
investigational gene therapies, as well as costs related to
regulatory, manufacturing, license fees and milestone payments
under the company’s agreements with third parties, and personnel
costs to support these activities. The company expects R&D
expenses to grow slightly in the upcoming periods as the company
continues to advance its programs through later stages of
development.
Selling, general and administrative expenses were $16.2 million
for the three months ended December 31, 2020, compared to $18.5
million in the same period in 2019. The decrease was primarily due
to realization of savings associated with an updated strategy and
corporate restructuring announced in May 2020.
Net loss attributable to ordinary shareholders was $33.6 million
for the three months ended December 31, 2020, compared to $45.4
million in the same period in 2019. The decline in net loss as
compared to the prior year was primarily due to savings realized in
our operating expenses as a result of the company’s updated
strategy and corporate restructuring. The company had 98.3 million
ordinary shares outstanding as of December 31, 2020.
Thomas continued, "Our burn rate has declined from prior periods
as we see the positive impact of our May 2020 corporate
restructuring take hold, providing a longer runway and greater
financial flexibility, aided by our recent financing. We are
investing to support execution for the highest value programs in
our portfolio while also dedicating capital to our longer-term
strategy to expand into larger indications.
Cash, cash equivalents and investments as of December 31, 2020,
were $191.9 million compared to $325.0 million as of December 31,
2019, with the decrease primarily driven by cash used to fund
operations in 2020. In the fourth quarter of 2020, the cash used to
fund operations was approximately $12.0 million after the receipt
of approximately $19.2 million from R&D tax credit refunds
related to 2019 qualifying activities under the tax code in the UK.
The company expects that its cash, cash equivalents and investments
as of December 31, 2020, along with gross proceeds of $150.0
million from the February 2021 private placement, will support its
currently anticipated operating expenses and capital expenditure
requirements into the first half of 2023. This cash runway excludes
the $50 million available under the company’s credit facility and
any non-dilutive capital received from potential future
partnerships or priority review vouchers granted by the FDA
following future potential U.S. approvals.
About Libmeldy / OTL-200Libmeldy (autologous
CD34+ cell enriched population that contains hematopoietic stem and
progenitor cells (HSPC) transduced ex vivo using a lentiviral
vector encoding the human arylsulfatase-A (ARSA) gene), also known
as OTL-200, has been approved by the European Commission for the
treatment of MLD in eligible early-onset patients characterized by
biallelic mutations in the ARSA gene leading to a reduction of the
ARSA enzymatic activity in children with i) late infantile or early
juvenile forms, without clinical manifestations of the disease, or
ii) the early juvenile form, with early clinical manifestations of
the disease, who still have the ability to walk independently and
before the onset of cognitive decline. Libmeldy is the first
therapy approved for eligible patients with early-onset MLD.The
most common adverse reaction attributed to treatment with Libmeldy
was the occurrence of anti-ARSA antibodies. In addition to the
risks associated with the gene therapy, treatment with Libmeldy is
preceded by other medical interventions, namely bone marrow harvest
or peripheral blood mobilization and apheresis, followed by
myeloablative conditioning, which carry their own risks. During the
clinical studies, the safety profiles of these interventions were
consistent with their known safety and tolerability.For more
information about Libmeldy, please see the Summary of Product
Characteristics (SmPC) available on the EMA website.Libmeldy is not
approved outside of the European Union, UK, Iceland, Liechtenstein
and Norway. OTL-200 is an investigational therapy in the US.
Libmeldy was developed in partnership with the San
Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan,
Italy.About Orchard
Orchard Therapeutics is a global gene therapy leader
dedicated to transforming the lives of people affected by rare
diseases through the development of innovative, potentially
curative gene therapies. Our ex vivo autologous gene
therapy approach harnesses the power of genetically modified blood
stem cells and seeks to correct the underlying cause of disease in
a single administration. In 2018, Orchard acquired GSK’s rare
disease gene therapy portfolio, which originated from a pioneering
collaboration between GSK and the San Raffaele Telethon
Institute for Gene Therapy in Milan, Italy. Orchard now has
one of the deepest and most advanced gene therapy product candidate
pipelines in the industry spanning multiple therapeutic areas where
the disease burden on children, families and caregivers is immense
and current treatment options are limited or do not exist.
Orchard has its global headquarters
in London and U.S. headquarters in Boston.
For more information, please visit www.orchard-tx.com, and
follow us on Twitter and LinkedIn.
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 and LinkedIn), including but not limited to
investor presentations and investor fact sheets, U.S.
Securities and Exchange Commission filings, press releases,
public conference calls and webcasts. The information that Orchard
posts on these channels and websites could be deemed to be material
information. As a result, Orchard encourages investors, the media,
and others interested in Orchard to review the information that is
posted on these channels, including the investor relations website,
on a regular basis. This list of channels may be updated from time
to time on Orchard’s investor relations website and may include
additional social media channels. The contents of Orchard’s website
or these channels, or any other website that may be accessed from
its website or these channels, shall not be deemed incorporated by
reference in any filing under the Securities Act of 1933.
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. Forward-looking
statements include express or implied statements relating to, among
other things, Orchard’s business strategy and goals, including its
plans and expectations for the commercialization of Libmeldy, the
therapeutic potential of Libmeldy (OTL-200) and Orchard’s product
candidates, including the product candidates referred to in this
release, Orchard’s expectations regarding its ongoing preclinical
and clinical trials, including the timing of enrollment for
clinical trials and release of additional preclinical and clinical
data, the likelihood that data from clinical trials will be
positive and support further clinical development and regulatory
approval of Orchard's product candidates, and Orchard’s financial
condition and cash runway into the first half of 2023. 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, these risks and uncertainties
include, without limitation: the risk that prior results, such as
signals of safety, activity or durability of effect, observed from
clinical trials of Libmeldy will not continue or be repeated in our
ongoing or planned clinical trials of Libmeldy, will be
insufficient to support regulatory submissions or marketing
approval in the US or to maintain marketing approval in the EU, or
that long-term adverse safety findings may be discovered; the risk
that any one or more of Orchard’s product candidates, including the
product 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 Orchard may not successfully recruit or
enroll a sufficient number of patients for its 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 or the receipt
of restricted marketing approvals; the inability or risk of delays
in Orchard’s ability to commercialize its product candidates, if
approved, or Libmeldy, including the risk that Orchard may not
secure adequate pricing or reimbursement to support continued
development or commercialization of Libmeldy; the risk that the
market opportunity for Libmeldy, or any of Orchard’s product
candidates, may be lower than estimated; and the severity of the
impact of the COVID-19 pandemic on Orchard’s business, including on
clinical development, its supply chain and commercial
programs. 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 quarterly
report on Form 10-Q for the quarter ended September 30, 2020,
as filed with the U.S. Securities and Exchange
Commission (SEC), as well as subsequent filings and reports
filed with the SEC. The forward-looking statements contained
in this press release reflect Orchard’s 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.
|
Consolidated Statements of Operations Data(In
thousands, except share and per share data)(Unaudited) |
|
|
|
|
|
Three months ended December 31, |
|
|
2020 |
|
|
|
2019 |
|
Product sales, net |
$ |
— |
|
|
$ |
595 |
|
Costs and operating
expenses: |
|
|
|
Cost of product sales |
|
— |
|
|
|
191 |
|
Research and development |
|
22,648 |
|
|
|
30,899 |
|
Selling, general and administrative |
|
16,226 |
|
|
|
18,531 |
|
Total costs and operating expenses |
|
38,874 |
|
|
|
49,621 |
|
Loss from operations |
|
(38,874 |
) |
|
|
(49,026 |
) |
Other income (expense): |
|
|
|
Interest income |
|
279 |
|
|
|
1,843 |
|
Interest expense |
|
(575 |
) |
|
|
(633 |
) |
Other income, net |
|
5,635 |
|
|
|
2,533 |
|
Total other income, net |
|
5,339 |
|
|
|
3,743 |
|
Net loss before income tax |
|
(33,535 |
) |
|
|
(45,283 |
) |
Income tax expense |
|
(85 |
) |
|
|
(133 |
) |
Net loss attributable to ordinary
shareholders |
$ |
(33,620 |
) |
|
$ |
(45,416 |
) |
|
|
|
|
|
|
|
|
|
Consolidated Balance Sheet Data(in
thousands)(Unaudited) |
|
|
|
December 31, |
December 31, |
|
|
|
2020 |
|
2019 |
|
Assets |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
|
$ |
55,135 |
$ |
19,053 |
|
Marketable securities |
|
|
136,813 |
|
305,937 |
|
Trade receivables |
|
|
878 |
|
1,442 |
|
Prepaid expenses and other current assets |
|
|
13,365 |
|
8,530 |
|
Research and development tax credit receivable, current |
|
|
17,344 |
|
14,934 |
|
Total current assets |
|
|
223,535 |
|
349,896 |
|
Non-current assets: |
|
|
|
Operating lease right-of-use assets |
|
|
29,815 |
|
19,415 |
|
Property and equipment, net |
|
|
4,781 |
|
7,596 |
|
Research and development tax credit receivable |
|
|
— |
|
13,710 |
|
Other long-term assets |
|
|
22,806 |
|
8,664 |
|
Total non-current assets |
|
|
57,402 |
|
49,385 |
|
Total assets |
|
$ |
280,937 |
$ |
399,281 |
|
Liabilities and
shareholders' equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable |
|
$ |
8,823 |
$ |
11,984 |
|
Accrued expenses and other current liabilities |
|
|
28,943 |
|
37,980 |
|
Operating lease liabilities |
|
|
8,934 |
|
5,892 |
|
Notes payable, current |
|
|
4,861 |
|
— |
|
Total current liabilities |
|
|
51,561 |
|
55,856 |
|
Notes payable, long-term |
|
|
20,204 |
|
24,699 |
|
Operating lease liabilities,
non-current |
|
|
24,168 |
|
15,320 |
|
Other long-term liabilities |
|
|
6,570 |
|
4,213 |
|
Total liabilities |
|
|
102,503 |
|
100,088 |
|
Shareholders’ equity: |
|
|
178,434 |
|
299,193 |
|
Total liabilities and
shareholders’ equity |
|
$ |
280,937 |
$ |
399,281 |
|
|
|
|
|
|
|
|
Contacts
InvestorsRenee LeckDirector, Investor
Relations+1 862-242-0764Renee.Leck@orchard-tx.com
MediaChristine HarrisonVice President,
Corporate Affairs+1 202-415-0137media@orchard-tx.com
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