Homology Medicines Reports Third Quarter 2021 Financial Results and Recent Highlights
November 15 2021 - 7:10AM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today financial results for the third quarter
ended September 30, 2021, and highlighted recent accomplishments.
“We realized our goal to have three clinical programs underway
this year by moving our gene editing candidate for PKU and gene
therapy for Hunter syndrome into the clinic, both evaluating
first-of-a-kind approaches for these diseases,” stated Arthur
Tzianabos, Ph.D., President and CEO of Homology Medicines. “We have
always planned to develop two solutions for people living with PKU,
first with our ongoing pheNIX gene therapy trial in adults and now
with our gene editing trial, which is focused initially on adults
and then pediatric patients over time. In addition, our gene
therapy program for patients with Hunter syndrome plans to evaluate
a much needed one-time therapy designed to address both peripheral
organ and cognitive manifestations of this disease with an I.V.
infusion.”
Dr. Tzianabos added, “We continue to benefit from our internal
commercial GMP manufacturing platform, as this fully integrated
‘plug and play’ capability has now delivered product for three
successful INDs, including our first gene editing product
candidate. Confidence in our AAVHSC technology and the team’s
ability to develop genetic medicines builds as multiple programs
have entered the clinic and is reflected in our recent commitment
to expand our headquarters in support of continued growth and
success.”Third Quarter 2021 and Recent
Accomplishments
- Announced the pheEDIT clinical trial, a Phase 1 dose-escalation
study of one-time, in vivo product candidate HMI-103 that utilizes
a nuclease-free gene editing approach for phenylketonuria (PKU) and
leverages learnings from the Company’s ongoing pheNIX PKU trial
with the same AAVHSC vector. Once positive safety and efficacy
results are established in adults, Homology plans to enroll younger
patients in clinical trials.
- At the American Society of Human Genetics (ASHG) Meeting, data
supporting HMI-103 gene editing precision were presented, including
molecular methods that demonstrated on-target integration and no
evidence of integration into any other genomic location in a
humanized murine liver model.
- HMI-103 received Fast Track designation by the U.S. Food and
Drug Administration (FDA).
- Initiated the juMPStart clinical trial, a Phase 1
dose-escalation study of one-time, in vivo gene therapy candidate
HMI-203, the first I.V. gene therapy designed to address both
peripheral and central nervous system (CNS) effects, in adults with
mucopolysaccharidosis type II (MPS II), or Hunter syndrome.
- Data presented at ASHG showed adults with Hunter syndrome
reported unmet medical needs despite the use of enzyme replacement
therapy (ERT), particularly related to peripheral manifestations of
the disease (e.g., range of motion and mobility, pain, etc.) and
burden of chronic dosing. Preclinical data that supported the
HMI-203 IND were also shared at scientific conferences, including
systemic reduction of disease biomarkers in peripheral organs and
the CNS, and prevention of skeletal deformaties.
- Provided an update on the ongoing pheNIX clinical trial, a
Phase 2 dose expansion study evaluating HMI-102 gene therapy in
adults with PKU. Specifically:
- Both doses were generally well-tolerated and showed evidence of
biological activity, including clinically meaningful reductions in
phenylalanine (Phe) levels, increases in tyrosine (Tyr) and
reductions in the Phe-to-Tyr ratio;
- Added new clinical trial sites for a total of 13 with more
expected shortly, and expanded Medical Affairs, Clinical
Development and Operations teams to support ongoing trials;
and
- Plans to provide a detailed data update in mid-2022 when more
patients are expected to be enrolled in the trial.
- In addition to studies supporting three clinical programs,
Homology highlighted preclinical data from its GTx-mAb development
program for paroxysmal nocturnal hemoglobinuria (PNH) at the
European Society of Gene & Cell Therapy (ESGCT) Virtual
Conference.
- Continued to support the rare disease patient community through
educational efforts at national and regional patient
advocacy-focused events.
Third Quarter 2021 Financial Results
- Net loss for the quarter ended September 30, 2021 was $(30.6)
million or $(0.54) per share, compared to a net loss of $(28.2)
million or $(0.62) per share for the same period in 2020.
- Collaboration revenues for the quarter ended September 30, 2021
were $1.7 million, compared to $0.6 million for the quarter ended
September 30, 2020. Collaboration revenues for the third quarter
2021 included the recognition of all remaining deferred revenue and
final reimbursement of R&D expenses under the Company’s former
collaboration with Novartis, in addition to revenue recognized
under Homology’s stock purchase agreement with Pfizer.
- Total operating expenses for the quarter ended September 30,
2021 were $32.3 million, compared to $28.8 million for the quarter
ended September 30, 2020, and consisted of research and development
expenses and general and administrative expenses.
- Research and development expenses for the quarter ended
September 30, 2021 were $24.0 million, compared to $20.4 million
for the quarter ended September 30, 2020. Research and development
expenses increased due to higher external development costs as
HMI-103 and HMI-203 advanced into Phase 1 clinical trials during
the quarter, as well as increased personnel costs to support
ongoing development programs and new clinical programs, research
initiatives, technology platform expansion and manufacturing
capabilities. Partially offsetting these increases was a decrease
in direct research expenses for HMI-102 due to the completion of
manufacturing of drug product in the prior year for the Phase 1/2
pheNIX clinical trial. Additionally, the continued optimization of
Homology’s ‘plug and play’ manufacturing process and platform has
created efficiencies across all of programs that directly reduced
spend for clinical trial and other materials and limited reliance
on outside contract manufacturing organizations.
- General and administrative expenses for each of the quarters
ended September 30, 2021 and 2020 were $8.4 million.
- As of September 30, 2021, Homology had approximately $187.6
million in cash, cash equivalents and short-term investments. Based
on current projections, Homology continues to expect cash resources
to fund operations into the first quarter of 2023.
Upcoming Event
- Stifel 2021 Virtual Healthcare Conference: November 17 at 9:20
a.m. ET
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 vectors
(AAVHSCs) 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, internal manufacturing capabilities 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. 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; 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; our beliefs regarding our manufacturing
capabilities; our position as a leader in the development of
genetic medicines; the sufficiency of our cash and cash equivalents
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; 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 September 30, 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 BALANCE SHEETS |
|
(in
thousands) |
|
(Unaudited) |
|
|
|
|
|
|
|
|
|
As of |
|
|
|
September 30, 2021 |
|
December 31, 2020 |
|
Cash, cash equivalents and short-term investments |
|
$ |
187,551 |
|
$ |
217,431 |
|
Property and
equipment, net |
|
|
32,697 |
|
|
37,002 |
|
Right-of-use
assets |
|
|
5,028 |
|
|
5,897 |
|
Other
assets |
|
|
5,540 |
|
|
3,407 |
|
Total assets |
|
$ |
230,816 |
|
$ |
263,737 |
|
|
|
|
|
|
|
Accounts
payable, accrued expenses and other liabilities |
|
$ |
13,538 |
|
$ |
14,525 |
|
Operating
lease liabilities |
|
|
13,541 |
|
|
15,442 |
|
Deferred
revenue |
|
|
5,166 |
|
|
37,775 |
|
Stockholders' equity |
|
|
198,571 |
|
|
195,995 |
|
Total liabilities and stockholders' equity |
|
$ |
230,816 |
|
$ |
263,737 |
|
|
|
|
|
|
|
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, |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
Collaboration revenue |
|
$ |
1,677 |
|
|
$ |
567 |
|
|
$ |
33,169 |
|
|
$ |
1,722 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
23,987 |
|
|
|
20,417 |
|
|
|
69,439 |
|
|
|
77,197 |
|
General and administrative |
|
|
8,351 |
|
|
|
8,423 |
|
|
|
26,054 |
|
|
|
24,986 |
|
Total operating expenses |
|
|
32,338 |
|
|
|
28,840 |
|
|
|
95,493 |
|
|
|
102,183 |
|
Loss from
operations |
|
|
(30,661 |
) |
|
|
(28,273 |
) |
|
|
(62,324 |
) |
|
|
(100,461 |
) |
Other
income: |
|
|
|
|
|
|
|
|
Interest income |
|
|
53 |
|
|
|
41 |
|
|
|
143 |
|
|
|
1,558 |
|
Total other
income |
|
|
53 |
|
|
|
41 |
|
|
|
143 |
|
|
|
1,558 |
|
Net
loss |
|
$ |
(30,608 |
) |
|
$ |
(28,232 |
) |
|
$ |
(62,181 |
) |
|
$ |
(98,903 |
) |
Net loss per
share-basic and diluted |
|
$ |
(0.54 |
) |
|
$ |
(0.62 |
) |
|
$ |
(1.14 |
) |
|
$ |
(2.19 |
) |
Weighted-average common shares outstanding-basic and diluted |
|
|
57,106,639 |
|
|
|
45,227,231 |
|
|
|
54,704,410 |
|
|
|
45,196,459 |
|
|
|
|
|
|
|
|
|
|
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.com781-691-3510
Homology Medicines (NASDAQ:FIXX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Homology Medicines (NASDAQ:FIXX)
Historical Stock Chart
From Apr 2023 to Apr 2024