Homology Medicines Reports Fourth Quarter and Full Year 2021 Financial Results and Recent Highlights
March 23 2022 - 4:10PM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today financial results for the fourth quarter
and full year ended December 31, 2021, and highlighted recent
accomplishments.
“We recently closed our deal with Oxford Biomedica to form a new
company that incorporates Homology’s expertise in AAV process
development and manufacturing capabilities, team and GMP facility,”
stated Arthur Tzianabos, Ph.D., President and Chief Executive
Officer of Homology Medicines. “Months ago, we made a strategic
decision to find the right opportunity to leverage these internal
capabilities that supported three successfully cleared INDs and our
pipeline. We believe the structure of this partnership with a
global leader in viral vector manufacturing is unique in allowing
us to retain an ownership position in what we have built over the
years. It also enables us to benefit from preferred customer access
to the same high quality, innovative manufacturing capabilities and
team, as well as any future value creation. In addition, the $130
million in non-dilutive capital and the resulting cost-savings from
the deal significantly extends our cash runway in support of our
three clinical programs and pipeline.”
Albert Seymour, Ph.D., Chief Scientific Officer of Homology
Medicines, added, “Initiating the pheEDIT gene editing trial was a
major accomplishment as this is a novel approach to treat PKU that
utilizes a different arm of our AAVHSC technology platform.
Launching our juMPStart gene therapy trial for Hunter syndrome
represents the first program to enter the clinic with a one-time
systemic administration that is designed to address peripheral and
CNS components of the disease, which is a highly differentiated
approach. We will continue to work with our clinical sites and the
patient communities to execute on both trials, and plan to provide
program updates by year-end 2022.”
Consistent with Homology’s prior announcement of a clinical hold
on its pheNIX gene therapy trial for phenylketonuria (PKU), the
Company received the anticipated letter from the U.S. Food and Drug
Administration (FDA) detailing the information requested for
elevated liver function tests (LFTs) observed in the trial and
modified clinical-mitigation measures with nothing related to
Homology’s other two clinical programs or its CMC/manufacturing
capability. In patients who experienced elevated LFTs, all have
resolved and no hospitalizations were required. Among the
risk-mitigation methods that Homology intends to propose is a new,
more targeted immunosuppressive regimen that is shorter in duration
and includes a T-cell inhibitor used in combination with a
steroid-sparing regimen that may improve patient compliance. The
Company has already incorporated this regimen into its pheEDIT gene
editing trial for adults with PKU. The use of T-cell inhibitors has
been shown to be effective in dampening the anticipated immune
response to AAV capsids, which are commonly employed to deliver
genetic medicines. Homology also noted that interest in the pheEDIT
study is steadily increasing with 15 clinical sites already
selected to participate, several pending site initiation visits
expected shortly, and more clinical trial sites on the horizon. A
program update on pheEDIT is still expected by year-end 2022.
With the additional information requested by FDA on the pheNIX
trial and the planned conversion to a more specific steroid-sparing
immunosuppressive regimen, Homology estimates that it will require
more time to submit and receive feedback on its proposed clinical
risk-mitigation strategy. This also includes time needed to amend
the pheNIX study protocol. As a result, the Company now expects to
provide a program update when the path forward is established with
FDA.
Fourth Quarter 2021 and Recent
Accomplishments
- Formed a new Manufacturing and Innovation Business with
UK-based, global viral vector manufacturer Oxford Biomedica (OXB),
which incorporates Homology’s leading internal technical and
manufacturing operations and is led by Homology’s former Chief
Operating Officer, Tim Kelly, as Chief Executive Officer and Board
Chair. Key benefits to Homology include:
- Received $130 million from OXB, which will support the
advancement of the three clinical programs and genetic medicines
platform;
- Non-dilutive capital and significant reduction in manufacturing
expenses that extends cash runway into 2H 2024;
- 20% ownership and a Board seat in the new company;
- Access to the AAV ‘plug and play’ process and platform, which
met CMC requirements for Homology’s three INDs, as a preferred
customer;
- Continuing to work with AAVHSC experts within the
state-of-the-art manufacturing facility built by Homology; and
- $50 million investment from OXB in the new company that aims to
leverage OXB’s existing client base and commercial expertise.
- Initiated the pheEDIT trial, a Phase 1 dose-escalation study in
adults with PKU evaluating HMI-103, a one-time, in vivo product
candidate that utilizes a nuclease-free gene editing approach for
PKU. A program update is expected by year-end 2022.
- Presented data supporting the recently launched juMPStart trial
evaluating a one-time, systemic administration of HMI-203
investigational gene therapy for Hunter syndrome. Presentations at
WORLDSymposium™ included:
- Clinical trial design, select eligibility criteria and planned
endpoints for the Phase 1 dose-escalation trial. A program update
is expected by year-end 2022;
- Incorporated patient, caregiver and key opinion leader feedback
into the clinical trial design, which related to unmet medical need
and expectations for a one-time gene therapy that has the potential
for enzyme replacement therapy (ERT)-independence; and
- Homology’s gene therapy approach to Hunter syndrome and other
lysosomal storage diseases, including metachromatic leukodystrophy
(MLD), which is developed to target both the peripheral as well as
the central nervous system (CNS) manifestations of these
multi-organ disorders.
- Received orphan medicinal product designation (OMPD) and
advanced therapy medicinal product (ATMP) classification from the
European Medicines Agency (EMA) for both HMI-203 for Hunter
syndrome and HMI-103 for PKU.
- Continued to advance HMI-104, a C5 antibody development
candidate for paroxysmal nocturnal hemoglobinuria (PNH), which is
currently in IND-enabling studies. The Company’s GTx-mAb platform
is designed to deliver one-time in vivo gene therapy to produce
antibodies from the liver and secrete them throughout the
body.
Fourth Quarter 2021 and Full Year Financial
Results
- Net loss for the quarter ended December 31, 2021 was $(33.6)
million or $(0.59) per share, compared to a net loss of $(29.8)
million or $(0.62) per share for the same period in 2020. Net loss
for the year ended December 31, 2021 was $(95.8) million or $(1.73)
per share, compared to a net loss of $(128.7) million or $(2.80)
per share for the same period in 2020.
- Collaboration revenues for the three and twelve months ended
December 31, 2021 were $0.8 million and $34.0 million,
respectively, as compared to $1.0 million and $2.7 million for the
comparable periods in 2020. Collaboration revenues consisted
primarily of revenue recognized as a result of concluding the
Company’s collaboration with Novartis. Also included in
collaboration revenues is revenue recognized under the Company’s
stock purchase agreement with Pfizer.
- Total operating expenses for the three and twelve months ended
December 31, 2021 were $34.4 million and $129.9 million,
respectively, as compared to $30.8 million and $133.0 million for
the comparable periods in 2021, and consisted of research and
development expenses and general and administrative expenses.
- Research and development expenses for the three and twelve
months ended December 31, 2021 were $23.6 million and $93.1
million, respectively, as compared to $23.2 million and $100.4
million for the comparable periods in 2020. Research and
development expenses decreased in 2021 due to a reduction of 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. Further, direct
research expenses related to our HMI-202 program decreased over the
prior year as the Company was applying the learnings from the
IND-enabling studies to further optimize a vector with a better
therapeutic profile. Partially offsetting these decreases were
increased direct research expenses for HMI-103 and HMI-203 as
Homology advanced both programs into the clinic in 2021, as well as
increased personnel costs to support ongoing development programs,
research initiatives, technology platform and manufacturing
capabilities.
- General and administrative expenses for the three and twelve
months ended December 31, 2021 were $10.8 million and $36.8
million, respectively, as compared to $7.6 million and $32.6
million for the comparable periods in 2020. General and
administrative expenses increased due to personnel costs as a
result of new hires and increased legal costs and other
professional fees.
- As of December 31, 2021, Homology had approximately $155.9
million in cash, cash equivalents and short-term investments. Based
on current projections, Homology expects current cash resources,
including the $130.0 million received from Oxford Biomedica in
March 2022, to fund operations into the second half of 2024.
Upcoming Events
- H.C. Wainwright Gene Therapy and Gene Editing Conference:
Available on-demand March 30 at 7:00 a.m. ET
- 21st Annual Needham Virtual Healthcare Conference: April 11 at
9:30 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 and broad intellectual property position the Company as a
leader in genetic medicines. For more information, visit
www.homologymedicines.com.
Forward-Looking StatementsThis 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, including timing and
expectations surrounding communications with the FDA regarding the
pheNIX trial clinical hold and related updates from the Company;
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; 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 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; 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 Annual Report on Form 10-K for the year ended
December 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.
HOMOLOGY
MEDICINES, INC. |
CONDENSED
CONSOLIDATED BALANCE SHEETS |
(in
thousands) |
|
|
|
|
|
|
|
December 31, |
|
|
|
2021 |
|
|
2020 |
|
|
|
|
|
Cash, cash
equivalents and short-term investments |
|
$ |
155,873 |
|
$ |
217,431 |
Assets held
for sale |
|
|
28,907 |
|
|
— |
Property and
equipment, net |
|
|
2,252 |
|
|
37,002 |
Right-of-use
assets |
|
|
15,607 |
|
|
5,897 |
Other
assets |
|
|
9,082 |
|
|
3,407 |
Total assets |
|
$ |
211,721 |
|
$ |
263,737 |
|
|
|
|
|
Accounts
payable, accrued expenses and other liabilities |
|
$ |
13,772 |
|
$ |
14,525 |
Operating
lease liabilities |
|
|
246 |
|
|
2,501 |
Operating
lease liabilities, net of current portion |
|
|
23,688 |
|
|
12,941 |
Deferred
revenue |
|
|
4,364 |
|
|
37,775 |
Stockholders' equity |
|
|
169,651 |
|
|
195,995 |
Total liabilities and stockholders' equity |
|
$ |
211,721 |
|
$ |
263,737 |
HOMOLOGY
MEDICINES, INC. |
CONSOLIDATED
STATEMENTS OF OPERATIONS |
(in
thousands, except share and per share amounts) |
|
|
|
|
|
|
|
|
|
|
|
For the Three Months Ended December 31, |
|
For the Year Ended December 31, |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
|
|
|
|
|
|
|
|
|
(unaudited) |
|
|
|
|
Collaboration revenue |
|
$ |
802 |
|
|
$ |
980 |
|
|
$ |
33,971 |
|
|
$ |
2,702 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
23,646 |
|
|
|
23,195 |
|
|
|
93,085 |
|
|
|
100,392 |
|
General and administrative |
|
|
10,781 |
|
|
|
7,587 |
|
|
|
36,835 |
|
|
|
32,573 |
|
Total operating expenses |
|
|
34,427 |
|
|
|
30,782 |
|
|
|
129,920 |
|
|
|
132,965 |
|
Loss from
operations |
|
|
(33,625 |
) |
|
|
(29,802 |
) |
|
|
(95,949 |
) |
|
|
(130,263 |
) |
Other
income: |
|
|
|
|
|
|
|
|
Interest income |
|
|
42 |
|
|
|
11 |
|
|
|
185 |
|
|
|
1,569 |
|
Total other
income |
|
|
42 |
|
|
|
11 |
|
|
|
185 |
|
|
|
1,569 |
|
Net
loss |
|
$ |
(33,583 |
) |
|
$ |
(29,791 |
) |
|
$ |
(95,764 |
) |
|
$ |
(128,694 |
) |
Net loss per
share-basic and diluted |
|
$ |
(0.59 |
) |
|
$ |
(0.62 |
) |
|
$ |
(1.73 |
) |
|
$ |
(2.80 |
) |
Weighted-average common shares outstanding-basic and diluted |
|
|
57,150,079 |
|
|
|
48,112,174 |
|
|
|
55,283,318 |
|
|
|
45,910,787 |
|
Company Contacts:Theresa 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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