Homology Medicines Reports Third Quarter 2022 Financial Results and Recent Highlights
November 10 2022 - 4:07PM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today financial results for the third quarter
ended September 30, 2022, and highlighted recent accomplishments.
“We remain on track to provide an update from our pheEDIT and
juMPStart clinical trials, including site initiations and
enrollment status, by the end of this year,” said Albert Seymour,
Ph.D., President and Chief Executive Officer of Homology Medicines.
“With our recent pipeline prioritization, which included devoting
resources to our ongoing clinical trials and select preclinical
programs, Homology extended its cash runway into the fourth quarter
of 2024 through key milestones across all programs. We continue to
progress HMI-104, our lead GTx-mAb development candidate, through
IND-enabling studies and believe that our optimized in vivo gene
therapy development candidate for MLD, which has demonstrated the
ability to cross the blood-brain-barrier following a single I.V.
administration in preclinical studies, is differentiated from other
approaches and makes it an attractive program for us to partner.
Our work to bring first-in-class genetic medicine candidates
forward for patients is ongoing, and we look forward to sharing our
continued progress.”
Third Quarter 2022 and Recent Highlights
- Announced a pipeline prioritization, which included shifting
resources from the pheNIX gene therapy trial evaluating HMI-102 for
phenylketonuria (PKU) and pausing enrollment to focus on generating
data from the pheEDIT clinical trial that is evaluating in vivo
gene editing candidate HMI-103 for PKU. The mechanism of action of
this gene editing candidate has the potential to treat adults and
ultimately pediatric patients.
- Continued to advance the juMPStart clinical trial evaluating in
vivo gene therapy candidate HMI-203 for MPS II, or Hunter syndrome.
Homology remains on track to provide an update on enrollment and
site status for this trial and the pheEDIT trial by the end of this
year.
- Continued to advance IND-enabling studies of HMI-104, a C5
antibody development candidate for paroxysmal nocturnal
hemoglobinuria (PNH), which utilizes the Company’s GTx-mAb platform
that has the potential to address larger market indications.
- Announced intention to partner the optimized in vivo gene
therapy candidate HMI-204 for metachromatic leukodystrophy (MLD),
which demonstrated the ability to cross the blood-brain-barrier as
well as reach the central nervous system and key peripheral organs
involved in MLD following a single I.V. administration.
- Presented design of the pheEDIT gene editing trial for HMI-103
and supporting preclinical data, as well as data that demonstrated
the use of next-generation sequencing methods that can lead to
improved AAVHSC vector design, which were featured at the American
Society of Human Genetics Meeting.
- Promoted Albert Seymour, Ph.D., to Chief Executive Officer of
Homology and to the Board of Directors, succeeding Arthur
Tzianabos, Ph.D., who served as CEO and Board member since 2016 and
who was appointed Chair of the Board in conjunction with this
transition.
- Announced peer-reviewed publication in Scientific Reports, a
Nature journal, on the use of long-read sequencing methods that
could be applied to further characterize Homology’s AAVHSCs and
optimize vector design.
- Spoke alongside industry leaders on panels focused on
innovations in gene editing and AAV manufacturing during the Cell
& Gene Meeting on the Mesa.
Third Quarter 2022 Financial Results
- As of September 30, 2022, Homology had approximately $201.1
million in cash, cash equivalents and short-term investments. Based
on current projections, Homology expects cash resources to fund
operations into the fourth quarter of 2024.
- Net loss for the quarter ended September 30, 2022 was $(33.7)
million or $(0.59) per share, compared to a net loss of $(30.6)
million or $(0.54) per share for the quarter ended September 30,
2021.
- Collaboration revenues for the quarter ended September 30, 2022
were $0.8 million, compared to $1.7 million for the quarter ended
September 30, 2021. Collaboration revenue in each period included
deferred revenue recognized under Homology’s stock purchase
agreement with Pfizer. Collaboration revenues for the third quarter
2021 also included the recognition of deferred revenue and
reimbursement of R&D expenses under the Company’s former
collaboration with Novartis.
- Total operating expenses for the quarter ended September 30,
2022 were $33.7 million, compared to $32.3 million for the quarter
ended September 30, 2021, and consisted of research and development
expenses and general and administrative expenses.
- Research and development expenses for the quarter ended
September 30, 2022 were $25.9 million, compared to $24.0 million
for the quarter ended September 30, 2021. Research and development
expenses increased primarily due to increased development costs for
our ongoing HMI-103 and HMI-203 clinical-stage programs and
earlier-stage programs. Partially offsetting these increases were
lower employee-related costs as a result of transferring employees
to Oxford Biomedica Solutions, the AAV Innovation and Manufacturing
Business that Homology established with Oxford Biomedica in the
first quarter of 2022 in order to leverage its in-house
manufacturing capabilities while establishing a 20% stake and
preferred customer status in the new business.
- General and administrative expenses for the quarter ended
September 30, 2022 were $7.8 million, compared to $8.4 million for
the quarter ended September 30, 2021. General and administrative
expenses decreased primarily as a result of finance, human
resources, IT and legal services provided by the Company to OXB
Solutions under the transitional services agreement that provided
for these services to be reimbursed, as well as decreased
depreciation expense as the Company’s leasehold improvements were
transferred to Oxford Biomedica Solutions. Partially offsetting
these decreases was an increase in audit and legal fees.
Upcoming Events
- Stifel 2022 Healthcare Conference: November 15, 2022 at 4:10
p.m. ET – Fireside Chat
About Homology Medicines, Inc.Homology
Medicines, Inc. is a clinical-stage genetic medicines company
dedicated to transforming the lives of patients suffering from rare
diseases by addressing the underlying cause of the disease. The
Company’s clinical programs include HMI-102, an investigational
gene therapy for adults with phenylketonuria (PKU); HMI-103, a gene
editing candidate for PKU; and HMI-203, an investigational gene
therapy for Hunter syndrome. Additional programs focus on
metachromatic leukodystrophy (MLD), paroxysmal nocturnal
hemoglobinuria (PNH) and other diseases. Homology’s proprietary
platform is designed to utilize its family of 15 human
hematopoietic stem cell-derived adeno-associated virus (AAVHSCs)
vectors to precisely and efficiently deliver genetic medicines in
vivo through a gene therapy or nuclease-free gene editing modality,
as well as to deliver one-time gene therapy to produce antibodies
throughout the body through the GTx-mAb platform. Homology has a
management team with a successful track record of discovering,
developing and commercializing therapeutics with a focus on rare
diseases. Homology believes its initial clinical data and
compelling preclinical data, scientific and product development
expertise and broad intellectual property position the Company as a
leader in genetic medicines. For more information, visit
www.homologymedicines.com.
Forward-Looking Statements This press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. We intend such
forward-looking statements to be covered by the safe harbor
provisions for forward-looking statements contained in Section 27A
of the Securities Act of 1933, as amended and Section 21E of the
Securities Exchange Act of 1934, as amended. All statements
contained in this press release that do not relate to matters of
historical fact should be considered forward-looking statements,
including without limitation statements regarding: our expectations
surrounding the potential, safety, efficacy, and regulatory and
clinical progress of our product candidates; the potential of our
gene therapy and gene editing platforms, including our GTx-mAb
platform; our plans and timing for the release of additional
preclinical and clinical data; our position as a leader in the
development of genetic medicines; the sufficiency of our cash and
cash equivalents to fund our operations; our plans to engage in
future collaborations and strategic partnerships; and our
participation in upcoming presentations and conferences. The words
“believe,” “may,” “will,” “estimate,” “potential,” “continue,”
“anticipate,” “intend,” “expect,” “could,” “would,” “project,”
“plan,” “target,” and similar expressions are intended to identify
forward-looking statements, though not all forward-looking
statements use these words or expressions. These statements are
neither promises nor guarantees, but involve known and unknown
risks, uncertainties and other important factors that may cause our
actual results, performance or achievements to be materially
different from any future results, performance or achievements
expressed or implied by the forward-looking statements, including,
but not limited to, the following: the impact of the COVID-19
pandemic on our business and operations, including our preclinical
studies and clinical trials, and on general economic conditions; we
have and expect to continue to incur significant losses; our need
for additional funding, which may not be available; failure to
identify additional product candidates and develop or commercialize
marketable products; the early stage of our development efforts;
potential unforeseen events during clinical trials could cause
delays or other adverse consequences; risks relating to the
regulatory approval process; interim, topline and preliminary data
may change as more patient data become available, and are subject
to audit and verification procedures that could result in material
changes in the final data; our product candidates may cause serious
adverse side effects; inability to maintain our collaborations, or
the failure of these collaborations; our reliance on third parties,
including for the manufacture of materials for our research
programs, preclinical and clinical studies; failure to obtain U.S.
or international marketing approval; ongoing regulatory
obligations; effects of significant competition; unfavorable
pricing regulations, third-party reimbursement practices or
healthcare reform initiatives; product liability lawsuits;
securities class action litigation; failure to attract, retain and
motivate qualified personnel; the possibility of system failures or
security breaches; risks relating to intellectual property and
significant costs incurred as a result of operating as a public
company. These and other important factors discussed under the
caption “Risk Factors” in our Quarterly Report on Form 10-Q for the
quarter ended September 30, 2022 and our other filings with the
Securities and Exchange Commission could cause actual results to
differ materially from those indicated by the forward-looking
statements made in this press release. Any such forward-looking
statements represent management’s estimates as of the date of this
press release. While we may elect to update such forward-looking
statements at some point in the future, we disclaim any obligation
to do so, even if subsequent events cause our views to change.
- Financial Tables Follow -
HOMOLOGY
MEDICINES, INC. |
CONDENSED
CONSOLIDATED BALANCE SHEETS |
(in
thousands) |
(Unaudited) |
|
|
|
|
|
As of |
|
September 30, 2022 |
|
December 31, 2021 |
Cash, cash equivalents and short-term investments |
$ |
201,074 |
|
$ |
155,873 |
Assets held
for sale |
|
— |
|
|
28,907 |
Equity
method investment |
|
27,132 |
|
|
— |
Property and
equipment, net |
|
1,415 |
|
|
2,252 |
Right-of-use
assets |
|
20,900 |
|
|
15,607 |
Other
assets |
|
12,130 |
|
|
9,082 |
Total assets |
$ |
262,651 |
|
$ |
211,721 |
|
|
|
|
Accounts
payable, accrued expenses and other liabilities |
$ |
21,805 |
|
$ |
13,772 |
Operating
lease liabilities |
|
29,827 |
|
|
23,934 |
Deferred
revenue |
|
1,958 |
|
|
4,364 |
Stockholders' equity |
|
209,061 |
|
|
169,651 |
Total liabilities and stockholders' equity |
$ |
262,651 |
|
$ |
211,721 |
HOMOLOGY
MEDICINES, INC. |
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS |
(in
thousands, except share and per share amounts) |
(Unaudited) |
|
|
|
|
|
|
|
|
|
Three months ended September 30, |
|
Nine months ended September 30, |
|
2022 |
|
2021 |
|
2022 |
|
2021 |
Collaboration revenue |
$ |
802 |
|
|
$ |
1,677 |
|
|
$ |
2,406 |
|
|
$ |
33,169 |
|
Operating
expenses: |
|
|
|
|
|
|
|
Research and development |
|
25,854 |
|
|
|
23,987 |
|
|
|
71,202 |
|
|
|
69,439 |
|
General and administrative |
|
7,810 |
|
|
|
8,351 |
|
|
|
29,991 |
|
|
|
26,054 |
|
Total operating expenses |
|
33,664 |
|
|
|
32,338 |
|
|
|
101,193 |
|
|
|
95,493 |
|
Loss from
operations |
|
(32,862 |
) |
|
|
(30,661 |
) |
|
|
(98,787 |
) |
|
|
(62,324 |
) |
Other
income: |
|
|
|
|
|
|
|
Gain on sale of business |
|
- |
|
|
|
— |
|
|
|
131,249 |
|
|
|
— |
|
Interest income |
|
1,269 |
|
|
|
53 |
|
|
|
1,775 |
|
|
|
143 |
|
Total other
income |
|
1,269 |
|
|
|
53 |
|
|
|
133,024 |
|
|
|
143 |
|
Income
(loss) before income taxes |
|
(31,593 |
) |
|
|
(30,608 |
) |
|
|
34,237 |
|
|
|
(62,181 |
) |
Benefit from
(provision for) income taxes |
|
46 |
|
|
|
— |
|
|
|
(816 |
) |
|
|
— |
|
Loss from
equity method investment |
|
(2,179 |
) |
|
|
— |
|
|
|
(4,131 |
) |
|
|
— |
|
Net income
(loss) |
$ |
(33,726 |
) |
|
$ |
(30,608 |
) |
|
$ |
29,290 |
|
|
$ |
(62,181 |
) |
Net income
(loss) per share-basic |
$ |
(0.59 |
) |
|
$ |
(0.54 |
) |
|
$ |
0.51 |
|
|
$ |
(1.14 |
) |
Net income
(loss) per share-diluted |
$ |
(0.59 |
) |
|
$ |
(0.54 |
) |
|
$ |
0.51 |
|
|
$ |
(1.14 |
) |
Weighted-average common shares outstanding-basic |
|
57,447,192 |
|
|
|
57,106,639 |
|
|
|
57,372,399 |
|
|
|
54,704,410 |
|
Weighted-average common shares outstanding-diluted |
|
57,447,192 |
|
|
|
57,106,639 |
|
|
|
57,901,298 |
|
|
|
54,704,410 |
|
Company ContactsTheresa McNeelyChief
Communications Officer and Patient
Advocatetmcneely@homologymedicines.com781-301-7277 |
Media Contact:Cara Mayfield Vice President,
Patient Advocacy and Corporate Communications
cmayfield@homologymedicines.com 781-691-3510 |
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