Homology Medicines Reports First Quarter 2021 Financial Results and Recent Highlights
May 06 2021 - 4:05PM
Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic
medicines company, announced today financial results for the first
quarter ended March 31, 2021, and highlighted recent
accomplishments.
“We are in the midst of a transformational time at Homology as
we continue to mature and expand our genetic medicines platform and
we are on track to report initial Phase 2 data from our pheNIX
clinical trial and move two additional programs into the clinic
this year,” stated Arthur Tzianabos, Ph.D., President and Chief
Executive Officer of Homology Medicines. “We were excited to unveil
our new GTx-mAb platform last week, from which we will feature data
that demonstrated proof of principle targeting complement protein 5
with a one-time treatment in a humanized murine model, alongside
data from our multiple gene therapy and gene editing programs at
the ASGCT Annual Meeting next week. The GTx-mAb platform expands
our capability to address diseases with unmet needs, including
those with larger patient populations.”
Dr. Tzianabos continued, “With multiple catalysts ahead of us
this year and next, we recently completed a modest financing to
ensure that we are able to keep pace with our advancing programs,
and importantly, remain focused on continuing to achieve our goals.
We plan to provide an update from our pipeline and showcase
highlights from our presentations at ASGCT next week in a
conference call and webcast.”
First Quarter 2021 and Recent
Accomplishments
- Progressed the dose expansion phase of the Phase 1/2 pheNIX
trial evaluating HMI-102 gene therapy for the treatment of adults
with phenylketonuria (PKU), which has the potential to be converted
to a registrational trial.
- Following encouraging clinical data from the dose-escalation
phase of pheNIX, Homology continued to engage additional sites to
enroll patients.
- Unveiled the Company's new GTx-mAb platform, which leverages
its AAVHSCs to deliver vectors at a high efficiency to the liver
and secrete antibodies throughout the body through a one-time
administration of in vivo gene therapy, as shown in a humanized
murine model. Proof of concept data targeting complement protein 5
(C5) that support this platform, in addition to new data from
across the Company’s programs, including PKU, MPS II (Hunter
syndrome) and metachromatic leukodystrophy (MLD), and advancements
from its internal GMP manufacturing process and platform, are the
subject of seven presentations, including two oral presentations,
at the American Society of Gene & Cell Therapy (ASGCT) Annual
Meeting May 11-14. The Company plans to name a development
candidate in a new therapeutic area from the GTx-mAb platform in
2021.
- Shared the first-ever data from the Company’s nuclease-free
gene editing technology platform that demonstrated the ability of
AAVHSCs to transduce relevant cell types in both non-human primate
(NHP) and human cells and nuclease-free gene editing in two
ophthalmic targets in human retinal explants in a co-presentation
with Novartis at the Association for Research in Vision and
Ophthalmology (ARVO) Annual Meeting. An evaluation of 11 AAVHSC
capsids from in vivo studies with NHPs and ex vivo studies with
human retinal cells following a single intravenous (I.V.) or
subretinal dose, respectively, found that:
- All capsids crossed the blood-retinal and blood-brain barriers
in NHPs.
- AAVHSC15 achieved cross-species transduction of human and NHP
photoreceptor cells.
- Molecular methods confirmed seamless editing in two independent
loci and detection of hybrid transcript in human retinal
cells.
- Highlighted data from Homology’s PKU and Hunter syndrome gene
therapy programs in presentations at the American College of
Medical Genetics and Genomics (ACMG) Annual Clinical Genetics
Meeting, including presentations on the pheNIX trial and the
Company’s development candidate for Hunter syndrome, HMI-203. The
Company expects to initiate Phase 1/2 dose-escalation clinical
trials with HMI-203 and its in vivo gene editing candidate for PKU,
HMI-103, in 2021.
- Showcased new and long-term data from IND-enabling studies for
Homology’s central nervous system platform, including HMI-203 and
the gene therapy development candidate HMI-202 for MLD,
respectively, in presentations at the 17th Annual WORLDSymposium™
Meeting.
- Participated in patient advocacy-focused events, including Cure
MLD’s Standards of Care Conference and Project Alive’s Hunter
syndrome Community Conference.
- Completed a $50.0 million underwritten public offering of
shares of common stock in April, strengthening Homology’s financial
position in support of key development milestones anticipated in
each of the Company’s programs.
- Promoted Tim Kelly to Chief Operating Officer.
First Quarter 2021 Financial Results
- Net loss for the quarter ended March 31, 2021 was $(1.1)
million or $(0.02) per share, compared to a net loss of $(35.3)
million or $(0.78) per share for the same period in 2020.
- Collaboration revenues for the quarter ended March 31, 2021
were $29.3 million, compared to $0.6 million for the quarter ended
March 31, 2020. Collaboration revenues for the three months ended
March 31, 2021 includes the recognition of approximately $28.5
million of deferred revenue and reimbursement of R&D expenses
under the Company’s strategic collaboration with Novartis, which
Novartis decided to conclude in February 2021 following a portfolio
review. Also included in collaboration revenues is revenue
recognized under the Company’s stock purchase agreement with
Pfizer.
- Total operating expenses for the quarter ended March 31, 2021
were $30.4 million, compared to $37.1 million for the quarter ended
March 31, 2020, and consisted of research and development expenses
and general and administrative expenses.
- Research and development expenses for the quarter ended March
31, 2021 were $21.8 million, compared to $29.3 million for the
quarter ended March 31, 2020. Research and development expenses
decreased due to higher expenses in the prior year related to the
accelerated procurement of raw materials for manufacturing drug
product for the Phase 1/2 pheNIX clinical trial and other
development programs as part of Homology’s risk mitigation efforts
in response to the COVID-19 pandemic. Additionally, the continued
optimization of the Company’s ‘plug and play’ manufacturing process
and platform has created greater than 50% efficiencies in
subsequent programs that directly reduced spend for clinical trial
materials. Costs incurred with Homology’s contract research
organization (CRO) to conduct and manage the pheNIX trial with
HMI-102 continued to increase, as well as personnel costs to
support ongoing development programs, research initiatives,
technology platforms and manufacturing capabilities.
- General and administrative expenses for the quarter ended March
31, 2021 were $8.7 million, compared to $7.8 million for the
quarter ended March 31, 2020. General and administrative expenses
increased due to personnel costs as a result of new hires,
increased audit and legal costs and increased costs associated with
expanded operations.
- As of March 31, 2021, Homology had approximately $188.6 million
in cash, cash equivalents and short-term investments. Based on
current projections, Homology expects cash resources, together with
the approximately $49.4 million in net proceeds received from a
follow-on offering of its common stock in April 2021, to fund
operations into the first quarter of 2023.
Upcoming Events
- American Society of Gene & Cell Therapy (ASGCT) 24th Annual
Meeting: May 11-14
- Homology Medicines Corporate & Pipeline Update Conference
Call & Webcast: May 13
- RBC Capital Markets Healthcare Conference: May 18-19
- Bank of America Securities 2021 Napa Biopharma Virtual
Conference: June 14-16
- National PKU Alliance 2021 Virtual Mini-Conference: July
9-10
About Homology Medicines, Inc. Homology
Medicines, Inc. is a clinical-stage genetic medicines company
dedicated to transforming the lives of patients suffering from rare
genetic diseases with significant unmet medical needs by curing the
underlying cause of the disease. Homology’s proprietary platform is
designed to utilize its human hematopoietic stem cell-derived
adeno-associated virus vectors (AAVHSCs) to precisely and
efficiently deliver genetic medicines in vivo either through a gene
therapy or nuclease-free gene editing modality across a broad range
of genetic disorders. Homology has a management team with a
successful track record of discovering, developing and
commercializing therapeutics with a particular focus on rare
diseases. The Company’s intellectual property covers its family of
15 AAVHSCs. Homology believes that its compelling preclinical data,
scientific expertise, product development strategy, manufacturing
capabilities and intellectual property position it as a leader in
the development of genetic medicines. For more information, please
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. 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 new GTx-mAb
platform; our plans to name a development candidate in a new
therapeutic area and potential thereof; plans and timing for the
release of additional preclinical and clinical data, including
initial Phase 2 data from the pheNIX clinical trial; our beliefs
regarding our manufacturing capabilities; our position as a leader
in the development of genetic medicines; the sufficiency of our
cash, cash equivalents and short-term investments to fund our
operations; and our participation in upcoming presentations and
conferences. 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 capabilities of our
manufacturing facility; 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;
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; failure to attract, retain and motivate qualified
personnel; the possibility of system failures or security breaches;
risks relating to intellectual property and significant costs 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 March 31, 2021 and our
other filings with the SEC 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 STATEMENTS OF OPERATIONS |
(in
thousands, except share and per share amounts) |
(Unaudited) |
|
|
|
|
|
|
|
Three months ended March 31, |
|
|
|
2021 |
|
|
|
2020 |
|
Collaboration revenue |
|
$ |
29,305 |
|
|
$ |
588 |
|
Operating
expenses: |
|
|
|
|
Research and development |
|
|
21,755 |
|
|
|
29,310 |
|
General and administrative |
|
|
8,661 |
|
|
|
7,770 |
|
Total operating expenses |
|
|
30,416 |
|
|
|
37,080 |
|
Loss from
operations |
|
|
(1,111 |
) |
|
|
(36,492 |
) |
Other
income: |
|
|
|
|
Interest income |
|
|
38 |
|
|
|
1,161 |
|
Total other
income |
|
|
38 |
|
|
|
1,161 |
|
Net
loss |
|
$ |
(1,073 |
) |
|
$ |
(35,331 |
) |
Net loss per
share-basic and diluted |
|
$ |
(0.02 |
) |
|
$ |
(0.78 |
) |
Weighted-average common shares outstanding-basic and diluted |
|
|
50,363,579 |
|
|
|
45,151,265 |
|
|
|
|
|
|
HOMOLOGY
MEDICINES, INC. |
|
CONDENSED
CONSOLIDATED BALANCE SHEETS |
|
(in
thousands) |
|
(Unaudited) |
|
|
|
|
|
|
|
|
|
As of |
|
|
|
March 31, 2021 |
|
December 31, 2020 |
|
Cash, cash equivalents and short-term investments |
|
$ |
188,649 |
|
$ |
217,431 |
|
Property and
equipment, net |
|
|
36,153 |
|
|
37,002 |
|
Right-of-use
assets |
|
|
5,618 |
|
|
5,897 |
|
Other
assets |
|
|
4,277 |
|
|
3,407 |
|
Total assets |
|
$ |
234,697 |
|
$ |
263,737 |
|
|
|
|
|
|
|
Accounts
payable, accrued expenses and other liabilities |
|
$ |
10,301 |
|
$ |
14,525 |
|
Operating
lease liabilities |
|
|
14,821 |
|
|
15,442 |
|
Deferred
revenue |
|
|
8,746 |
|
|
37,775 |
|
Stockholders' equity |
|
|
200,829 |
|
|
195,995 |
|
Total liabilities and stockholders' equity |
|
$ |
234,697 |
|
$ |
263,737 |
|
|
|
|
|
|
|
Company ContactsTheresa McNeelyChief
Communications Officerand Patient
Advocatetmcneely@homologymedicines.com781-301-7277
Media Contact:Marisa CitranoSenior Corporate
Communications
Associatemcitrano@homologymedicines.com617-335-2841
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