Homology Medicines Reports Second Quarter 2021 Financial Results and Recent Highlights
August 12 2021 - 4:05PM
Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic
medicines company, announced today financial results for the second
quarter ended June 30, 2021, and highlighted recent
accomplishments.
“We continue to demonstrate the breadth and depth of our genetic
medicines platform with three clinical programs expected this year,
which span in vivo gene therapy and gene editing trials for rare
liver and CNS-based diseases,” stated Arthur Tzianabos, Ph.D.,
President and CEO of Homology Medicines. “With additional data from
our gene therapy trial and the start of our first gene editing
trial, both of which are focused on developing one-time treatments
for PKU and are expected this year, we will be closer to our goal
of helping adults and children living with this challenging
inherited metabolic disorder. In addition, we remain on track to
initiate our clinical trial for Hunter syndrome and we believe our
single I.V. delivery may address both peripheral and CNS aspects of
this disease.”
Dr. Tzianabos added, “Our AAVHSC technology was expanded during
the second quarter to include the launch of our GTx-mAb platform, a
new approach using gene therapy to deliver, produce and distribute
antibodies throughout the body. Preclinical proof-of-concept data
gave us great confidence in selecting our C5 antibody development
candidate for PNH, HMI-104, which uses another capsid from our
AAVHSC portfolio. In conjunction with our C5 program, we developed
a novel transfection system that has yielded up to 230 percent more
vector than the current industry standard transfection system.
Additional advances in our internal manufacturing platform were the
subject of scientific presentations, including one describing our
new formulation that will allow for a more convenient storage
condition than frozen storage, which is a clear advantage for our
clinical programs as well as potential commercial products.”
Second Quarter 2021 and Recent
Accomplishments
- Progressed Phase 2 of the pheNIX clinical trial evaluating a
single intravenous (I.V.) administration of HMI-102 gene therapy in
adults with phenylketonuria (PKU), with initial results expected by
year end.
- Phase 2 is designed with the potential to be converted to a
registrational trial.
- Homology is working closely with pheNIX sites to mitigate the
potential impact of a COVID-19 resurgence.
- On track to initiate a gene therapy clinical trial for Hunter
syndrome (MPS II), which is designed to evaluate a one-time I.V.
administration of HMI-203 in adults, by year end. Data from
IND-enabling studies were presented at the American Society of Gene
& Cell Therapy (ASGCT) Annual Meeting and showed:
- Long-term transduction and expression in multiple organs and
sustained secretion of I2S enzyme in the serum;
- Reduction of glycosaminoglycan heparan sulfate (GAG-HS),
including in the cerebrospinal fluid; and
- Phenotypic correction of joints and skeletal features.
- Shared first-ever data from Homology’s new GTx-mAb platform,
which is designed to deliver one-time in vivo gene therapy to
produce antibodies from the liver and secrete them throughout the
body. Building off the preclinical data presented at ASGCT,
Homology named a development candidate, HMI-104, for paroxysmal
nocturnal hemoglobinuria (PNH).
- Concluded IND-enabling studies with HMI-103, Homology’s in
vivo, nuclease-free gene editing candidate for PKU, which is on
track to enter the clinic by the end of 2021. Preclinical data at
ASGCT demonstrated HMI-103 integrated into the human PAH locus of a
humanized liver model with integration rates similar to those that
have resulted in sustained reduction of serum phenylalanine (Phe)
in the Pahenu2 PKU murine model.
- Presented data at ASGCT demonstrating the stability, at high
concentrations, of Homology’s novel formulation at cold storage
temperatures, which simplifies the supply chain by eliminating the
need for -80C frozen storage.
- Appointed gene therapy pioneer and ASGCT president Beverly
Davidson, Ph.D., as Chair of Homology’s Scientific Advisory Board,
on which Dr. Davidson has served since 2018.
- Spoke alongside gene therapy industry leaders during a panel at
the 2021 BIO International Convention and participated in national
and regional PKU patient advocacy-focused events.
- Completed a follow-on underwritten public offering of common
stock with gross proceeds of $50 million.
Second Quarter 2021 Financial Results
- Net loss for the quarter ended June 30, 2021 was $(30.5)
million or $(0.54) 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 June 30, 2021 were
$2.2 million, compared to $0.6 million for the quarter ended June
30, 2020. Collaboration revenues for the second quarter 2021
included the recognition of deferred revenue and reimbursement of
R&D expenses under the Company’s collaboration with Novartis,
in addition to revenue recognized under Homology’s stock purchase
agreement with Pfizer.
- Total operating expenses for the quarter ended June 30, 2021
were $32.7 million, compared to $36.3 million for the quarter ended
June 30, 2020, and consisted of research and development expenses
and general and administrative expenses.
- Research and development expenses for the quarter ended June
30, 2021 were $23.7 million, compared to $27.5 million for the
quarter ended June 30, 2020. Research and development expenses
decreased due to the completion of manufacturing of drug product in
the prior year for the Phase 1/2 pheNIX clinical trial.
Additionally, enhancements to the Company’s ‘plug and play’
internal manufacturing process and platform created greater than 50
percent efficiencies in subsequent programs that directly reduced
spend for clinical trial and other materials. These included:
- Leveraging the highly developed and optimized ‘plug and play’
platform;
- Developing a novel transfection system that yields up to 230
percent more vector; and
- Operational execution and expertise delivering a 100 percent
cGMP production run success rate.These decreases were partially
offset by increases in personnel costs to support ongoing
development programs and new clinical programs, research
initiatives, technology platform expansion and manufacturing
capabilities.
- General and administrative expenses for the quarter ended June
30, 2021 were $9.0 million, compared to $8.8 million for the
quarter ended June 30, 2020. General and administrative expenses
increased due to personnel costs as a result of new hires and
increased insurance expense, partially offset by decreased
consulting expense and legal fees.
- As of June 30, 2021, Homology had approximately $213.3 million
in cash, cash equivalents and short-term investments. Based on
current projections, Homology expects cash resources to fund
operations into the first quarter of 2023.
Upcoming Events
- PKU Northwest Family Camp: Presentation on HMI-102 gene therapy
program for PKU on August 21
- AGBT Precision Medicine Conference: September 9-11
- Georgia PKU Connect Summer Camp: Presentation on HMI-102 on
September 11
- H.C. Wainwright 23rd Annual Global Investment Conference:
Available on-demand September 13 at 7:00 a.m. ET
- Baird 2021 Global Healthcare Conference: September 14-15
- Intermountain PKU & Allied Disorders Lunch and Learn:
Presentation on HMI-102 on September 25
- Chardan’s 5th Annual Genetic Medicines Conference: October
4-5
- Cell & Gene Meeting on the Mesa: October 11-14
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 targeting the underlying cause of the disease. The
Company’s lead clinical program, HMI-102, is a gene therapy for
adults with phenylketonuria (PKU) and additional programs focus on
gene editing in PKU, lysosomal storage disorders including Hunter
syndrome, 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 and
believes that its data, 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; the potential of our
gene therapy and gene editing platforms, including our new GTx-mAb
platform; 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 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 June 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.
HOMOLOGY
MEDICINES, INC. |
|
CONDENSED
CONSOLIDATED BALANCE SHEETS |
|
(in
thousands) |
|
(Unaudited) |
|
|
|
|
|
|
|
As of |
|
|
June 30, 2021 |
|
December 31, 2020 |
|
Cash, cash equivalents and short-term investments |
$ |
213,293 |
|
$ |
217,431 |
|
Property and
equipment, net |
|
34,379 |
|
|
37,002 |
|
Right-of-use
assets |
|
5,328 |
|
|
5,897 |
|
Other
assets |
|
5,918 |
|
|
3,407 |
|
Total assets |
$ |
258,918 |
|
$ |
263,737 |
|
|
|
|
|
|
Accounts
payable, accrued expenses and other liabilities |
$ |
13,385 |
|
$ |
14,525 |
|
Operating
lease liabilities |
|
14,189 |
|
|
15,442 |
|
Deferred
revenue |
|
6,767 |
|
|
37,775 |
|
Stockholders' equity |
|
224,577 |
|
|
195,995 |
|
Total liabilities and stockholders' equity |
$ |
258,918 |
|
$ |
263,737 |
|
|
|
|
|
|
HOMOLOGY
MEDICINES, INC. |
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS |
(in
thousands, except share and per share amounts) |
(Unaudited) |
|
|
|
|
|
|
|
|
|
Three months ended June 30, |
|
Six months ended June 30, |
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
Collaboration revenue |
$ |
2,187 |
|
|
$ |
567 |
|
|
$ |
31,492 |
|
|
$ |
1,155 |
|
Operating
expenses: |
|
|
|
|
|
|
|
Research and development |
|
23,697 |
|
|
|
27,471 |
|
|
|
45,452 |
|
|
|
56,780 |
|
General and administrative |
|
9,042 |
|
|
|
8,793 |
|
|
|
17,703 |
|
|
|
16,563 |
|
Total operating expenses |
|
32,739 |
|
|
|
36,264 |
|
|
|
63,155 |
|
|
|
73,343 |
|
Loss from
operations |
|
(30,552 |
) |
|
|
(35,697 |
) |
|
|
(31,663 |
) |
|
|
(72,188 |
) |
Other
income: |
|
|
|
|
|
|
|
Interest income |
|
52 |
|
|
|
357 |
|
|
|
90 |
|
|
|
1,517 |
|
Total other
income |
|
52 |
|
|
|
357 |
|
|
|
90 |
|
|
|
1,517 |
|
Net
loss |
$ |
(30,500 |
) |
|
$ |
(35,340 |
) |
|
$ |
(31,573 |
) |
|
$ |
(70,671 |
) |
Net loss per
share-basic and diluted |
$ |
(0.54 |
) |
|
$ |
(0.78 |
) |
|
$ |
(0.59 |
) |
|
$ |
(1.56 |
) |
Weighted-average common shares outstanding-basic and diluted |
|
56,497,461 |
|
|
|
45,207,934 |
|
|
|
53,429,634 |
|
|
|
45,180,096 |
|
|
|
|
|
|
|
|
|
Company ContactsTheresa
McNeelyChief Communications Officerand 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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