Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company
focused on developing gene therapies to radically transform the
lives of patients with neurodegenerative diseases, today provided a
corporate update and financial results for its fiscal first quarter
ended June 30, 2021.
“We are enormously grateful for the support of the patient
community and our clinical investigators as we mark a milestone in
the GM1 gangliosidosis gene therapy program with the enrollment of
the targeted eight Type II children in the dose-escalation stage of
our clinical trial of AXO-AAV-GM1,” said Pavan Cheruvu, M.D., Chief
Executive Officer of Sio Gene Therapies. “We are focused on
continued clinical execution and look forward to presenting
12-month data from the low-dose cohort of this study in the second
half of this year. With this additional data in hand, as well as
emerging data from the high-dose cohort, we expect to meet with the
FDA in the first half of 2022 to discuss the registrational pathway
in GM1 gangliosidosis.”
Dr. Cheruvu continued, “The deep expertise and commitment of the
Sio team, paired with our strong balance sheet, leaves us well
positioned for continued execution to advance our pipeline of
transformative gene therapies that address rare pediatric and adult
neurodegenerative disease.”
Key Highlights and Development Updates
AXO-AAV-GM1 gene therapy for GM1 gangliosidosis
- Completed enrollment and dosing of eight late infantile and
juvenile-onset (Type II) patients across the low-dose (n=5) and
high-dose (n=3) cohorts, achieving the targeted enrollment of Type
II patients in Stage 1 (dose-escalation) of the trial. The Company
continues to collect information from additional Type II patients
for potential enrollment in Stage 2 of the trial.
- Enrollment and screening of Type I (infantile-onset) patients
for the low- and high-dose cohorts has been initiated.
- Cerebrospinal fluid (CSF) biomarker data presented at the
American Society of Gene and Cell Therapy (ASGCT) 24th Annual
Meeting demonstrated reductions in CSF GM1 ganglioside in 4 out of
5 children treated with the lowest dose of AXO-AAV-GM1 at 6-months
follow up, providing the first evidence of a biochemical effect in
the CNS following intravenous delivery.
- Upcoming milestones:
- 12-month topline safety, biomarker, and efficacy data from the
low-dose cohort in late Q3 or early Q4 2021.
- Meeting with the U.S. Food and Drug Administration (FDA) to
discuss the registrational pathway for AXO-AAV-GM1 in the first
half of 2022.
AXO-AAV-GM2 gene therapy for Tay-Sachs and Sandhoff diseases
- Dosed first two patients in the Phase 1/2 trial investigating
AXO-AAV-GM2 in Tay-Sachs and Sandhoff diseases, including one
patient at the starting dose and one patient at the low dose.
- Expect continued patient identification, screening, and
enrollment in Stage 1 of the dose-ranging trial throughout
2021.
AXO-Lenti-PD gene therapy for Parkinson’s disease
- Two GMP batches have been manufactured using an updated
suspension-based process and have now completed fill and finish.
Sio is awaiting final testing of these batches to support
certification of at least one batch for use as clinical trial
material by a Qualified Person in Q4 2021.
- Pending review and approval of an IMPD amendment by the MHRA in
the U.K., we expect to resume enrollment of patients in the
AXO-Lenti-PD clinical program in 2022.
Fiscal First Quarter Financial Summary
For the fiscal first quarter ended June 30, 2021, research and
development expenses were $8.1 million, an increase of $2.9 million
compared to the fiscal first quarter ended June 30, 2020. The
current period increase was primarily related to increases in:
(i) AXO-AAV-GM1 clinical trial material manufacturing expenses
for the planned enrollment of infantile patients in the high dose
cohort, as well as clinical trial expenses due to the ongoing
enrollment of juvenile patients in the high dose cohort and for the
planned enrollment of infantile patients in the low dose
cohort;
(ii) AXO-AAV-GM2 clinical trial expenses associated with the
ongoing enrollment of juvenile patients in the low dose cohort and
for the planned enrollment of patients in the low-to-mid dose
cohorts, as well as clinical trial material manufacturing expenses
for the planned enrollment of patients in the mid-to-high dose
cohorts (versus the prior year period, when this program was on
clinical hold); and
(iii) personnel-related costs primarily due to increased
headcount.
These increases were partially offset by a $1.1 million decrease
in AXO-Lenti-PD costs. The delays in the development of a reliable
suspension-based manufacturing process at Oxford Biomedica have
resulted in lower than expected manufacturing expenses and also
have delayed the initiation of further clinical studies of
AXO-Lenti-PD. Additionally, early development programs were
completed in the prior year period and as a result, development
expenses have also decreased in the current year period.
General and administrative expenses for the fiscal first quarter
ended June 30, 2021 were $3.9 million, a decrease of $0.7 million
compared to the fiscal first quarter ended June 30, 2020, primarily
related to decreased rent expense due to the downsizing of our New
York office footprint, as well as reductions in accounting,
auditing and tax fees, resulting, in part, from the simplification
of our corporate structure and the domestication of the parent
entity from Bermuda to Delaware that occurred in the prior year
period.
The net loss for the fiscal first quarter ended June 30, 2021
was $11.9 million, or $0.16 per share, compared to a net loss of
$8.6 million, or $0.20 per share, in the fiscal first quarter ended
June 30, 2020. The prior year period net loss was partially offset
by a gain of $2.2 million on our long-term investment in Arvelle
Therapeutics B.V. ("Arvelle"). For the fiscal first quarter ended
June 30, 2021, net cash used in operating activities was $12.7
million and net cash provided by investing activities of $4.2
million included $4.3 million of proceeds received from the sale of
our long-term investment in Arvelle.
As of June 30, 2021, we had $111.0 million of cash and cash
equivalents. We hold no short-term or long-term debt on the balance
sheet. We estimate that our current cash and cash equivalents will
sustain our operations into Q4 2022, beyond the expected dates of
major upcoming milestones for our AXO-AAV-GM1 gene therapy program
for the treatment of GM1 gangliosidosis.
About Sio Gene Therapies
Sio Gene Therapies combines cutting-edge science with bold
imagination to develop genetic medicines that aim to radically
improve the lives of patients. Our current pipeline of
clinical-stage candidates includes the first potentially curative
AAV-based gene therapies for GM1 gangliosidosis and
Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal
pediatric conditions caused by single gene deficiencies. We are
also expanding the reach of gene therapy to highly prevalent
conditions such as Parkinson’s disease, which affects millions of
patients globally. Led by an experienced team of gene therapy
development experts, and supported by collaborations with premier
academic, industry and patient advocacy organizations, Sio is
focused on accelerating its candidates through clinical trials to
liberate patients with debilitating diseases through the
transformational power of gene therapies. For more information,
visit www.siogtx.com.
Forward-Looking Statements
This press release contains forward-looking statements for the
purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995 and other federal securities laws.
The use of words such as "expect," "estimate," "may" and other
similar expressions are intended to identify forward-looking
statements. For example, all statements Sio makes regarding costs
associated with its operating activities, funding requirements
and/or runway to meet its upcoming clinical milestones, and timing
and outcome of its upcoming clinical and manufacturing milestones
are forward-looking. All forward-looking statements are based on
estimates and assumptions by Sio’s management that, although Sio
believes to be reasonable, are inherently uncertain. All
forward-looking statements are subject to risks and uncertainties
that may cause actual results to differ materially from those that
Sio expected. Such risks and uncertainties include, among others,
the impact of the Covid-19 pandemic on our operations; the actual
funds and/or runway required for our clinical and product
development activities and anticipated upcoming milestones; actual
costs related to our clinical and product development activities
and our need to access additional capital resources prior to
achieving any upcoming milestones; the initiation and conduct of
preclinical studies and clinical trials; the availability of data
from clinical trials; the development of a suspension-based
manufacturing process for AXO-Lenti-PD; the scaling up of
manufacturing; the expectations for regulatory submissions and
approvals; the continued development of our gene therapy product
candidates and platforms; Sio’s scientific approach and general
development progress; and the availability or commercial potential
of Sio’s product candidates. These statements are also subject to a
number of material risks and uncertainties that are described in
Sio’s most recent Annual Report on Form 10-Q filed with the
Securities and Exchange Commission on August 12, 2021, as updated
by its subsequent filings with the Securities and Exchange
Commission. Any forward-looking statement speaks only as of the
date on which it was made. Sio undertakes no obligation to publicly
update or revise any forward-looking statement, whether as a result
of new information, future events or otherwise, except as required
by law.
Contacts:
Media
Josephine Belluardo, Ph.D. LifeSci Communications(646)
751-4361jo@lifescicomms.cominfo@siogtx.com
Investors and Analysts
David W. NassifSio Gene Therapies Inc.Chief Financial Officer
and General Counseldavid.nassif@siogtx.com
SIO GENE THERAPIES
INC.Condensed Consolidated Statements of
Operations(Unaudited, in thousands, except share and per
share amounts)
|
Three Months Ended June 30, |
|
2021 |
|
2020 |
Operating expenses: |
|
|
|
Research and development
expenses |
|
|
|
(includes stock-based compensation expense of $432 and $563
for the three months ended June 30, 2021 and
2020, respectively) |
$ |
8,058 |
|
|
$ |
5,194 |
|
General
and administrative expenses |
|
|
|
(includes stock-based compensation expense of $889 and $1,027
for the three months ended June 30, 2021 and
2020, respectively) |
3,859 |
|
|
4,640 |
|
Total operating expenses |
11,917 |
|
|
9,834 |
|
Other
(income) expenses: |
|
|
|
Interest expense |
1 |
|
|
796 |
|
Other income |
(20 |
) |
|
(2,066 |
) |
Loss
before income tax (benefit) expense |
(11,898 |
) |
|
(8,564 |
) |
Income
tax (benefit) expense |
(28 |
) |
|
30 |
|
Net
loss |
$ |
(11,870 |
) |
|
$ |
(8,594 |
) |
Net loss
per share of common stock — basic and diluted |
$ |
(0.16 |
) |
|
$ |
(0.20 |
) |
Weighted-average shares of common stock outstanding — basic
and diluted |
72,861,870 |
|
|
43,287,222 |
|
SIO GENE THERAPIES
INC.Condensed Consolidated Balance
Sheets(Unaudited, in thousands, except share and per share
amounts)
|
June 30, 2021 |
|
March 31, 2021 |
Assets |
|
|
|
Current
assets: |
|
|
|
Cash and cash equivalents |
$ |
110,976 |
|
|
$ |
118,986 |
|
Receivable from sale of long-term investment |
— |
|
|
4,343 |
|
Prepaid expenses and other current assets |
6,276 |
|
|
7,348 |
|
Income tax receivable |
1,681 |
|
|
1,656 |
|
Total current assets |
118,933 |
|
|
132,333 |
|
Long-term restricted cash |
1,184 |
|
|
1,184 |
|
Operating lease right-of-use assets |
1,103 |
|
|
1,152 |
|
Property
and equipment, net |
595 |
|
|
478 |
|
Total assets |
$ |
121,815 |
|
|
$ |
135,147 |
|
Liabilities and Stockholders’ Equity |
|
|
|
Current
liabilities: |
|
|
|
Accounts payable |
$ |
937 |
|
|
$ |
1,341 |
|
Accrued expenses |
6,389 |
|
|
9,196 |
|
Current portion of operating lease liabilities |
269 |
|
|
311 |
|
Total current liabilities |
7,595 |
|
|
10,848 |
|
Operating lease liabilities, net of current portion |
921 |
|
|
932 |
|
Total liabilities |
8,516 |
|
|
11,780 |
|
Stockholders’ equity: |
|
|
|
Common stock, par value $0.00001 per share, 1,000,000,000
shares authorized, 69,639,509 and 69,377,567 issued and
outstanding at June 30, 2021 and March 31, 2021,
respectively |
1 |
|
|
1 |
|
Additional paid-in capital |
915,900 |
|
|
914,100 |
|
Accumulated deficit |
(802,939 |
) |
|
(791,069 |
) |
Accumulated other comprehensive income |
337 |
|
|
335 |
|
Total stockholders’ equity |
113,299 |
|
|
123,367 |
|
Total
liabilities and stockholders’ equity |
$ |
121,815 |
|
|
$ |
135,147 |
|
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