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
financial results for its fiscal year ended March 31, 2021.
“At Sio we remain committed to bringing urgently needed
treatments to patients with rare pediatric disorders and adult
neurodegenerative diseases. We are grateful for the steadfast
dedication of patients, caregivers, investigators, and patient
advocacy groups who have supported our mission in the midst of a
global pandemic, and we are proud of our team’s efforts in
advancing the development of Sio’s three clinical-stage product
candidates through an extraordinary year,” said Pavan Cheruvu,
M.D., Chief Executive Officer of Sio Gene Therapies.
“The positive data recently shared from our Phase 1/2 study of
AXO-AAV-GM1, together with our industry-leading position in
clinical enrollment and investment in earlier diagnosis, give us
renewed momentum as we aim to advance this investigational therapy
towards commercialization. In the second half of 2021, we will
present 12-month follow-up data on safety, biomarkers, and clinical
efficacy from the low-dose cohort of the GM1 gangliosidosis
program. We expect these data, as well as a planned regulatory
meeting with the FDA expected in the first half of 2022, will help
define our registrational pathway in GM1 gangliosidosis. We have
confidence in AXO-AAV-GM1 as well as our two other clinical-stage
candidates in Tay-Sachs/Sandhoff disease and Parkinson’s disease,
and look forward to several major milestones across our three
programs during this fiscal year.”
Dr. Cheruvu continued, “In parallel, we are continuing to build
our preclinical and analytical development capabilities in our
growing laboratory in Research Triangle Park, and are conducting a
search and evaluation process for promising genetic medicines that
may expand our pipeline with new product candidates in the years
ahead. Our strengthened balance sheet leaves us well-positioned to
develop first-in-class and best-in-class gene therapy product
candidates in our current pipeline, while also investing in
exciting new, biology-driven opportunities in areas of significant
unmet patient need.”
Key Highlights and Development Updates
AXO-AAV-GM1 gene therapy for GM1 gangliosidosis
- Reductions in CSF GM1 ganglioside were observed in 4 out of 5
children treated with the lowest dose of AXO-AAV-GM1 at 6-months
follow up, providing the first clear evidence of a biochemical
effect in the CNS following intravenous delivery (data presented at
the American Society of Cell and Gene Therapy conference in May
2021)
- Announced six-month
follow-up data from 5 patients in the low-dose cohort (1.5×1013
vg/kg) of AXO-AAV-GM1, demonstrating that the investigational gene
therapy had a favorable safety profile with evidence of clinical
disease stability and biomarker improvement
- Based on the
favorable safety profile at the low dose, the independent Data
Safety Monitoring Committee (DSMC) concurred with progressing to
the high-dose cohort (4.5×1013 vg/kg). Thus far two patients have
received the high dose without complications.
- Upcoming milestones:
- 12-month topline
safety, biomarker, and efficacy data from the low-dose cohort in
the second half of 2021
- 12-month topline
data from the first two patients dosed in the high-dose cohort
(4.5×1013 vg/kg) in Q1 2022
- 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 disease
- Dosed the first patient in the Phase 1/2 trial investigating
AXO-AAV-GM2 in Tay-Sachs and Sandhoff diseases
- Based on the favorable safety profile in the starting dose, the
independent Data Safety Monitoring Committee (DSMC) concurred with
progressing to the planned enrollment in the low-dose cohort (total
dose 1.95×1014 vg delivered directly to the CNS). Thus far one
patient has received the starting dose and a second patient has
received the low dose without complications.
- FDA granted Rare Pediatric Disease Designation for
AXO-AAV-GM2
- Upcoming milestones:
- Continued patient identification, screening, and enrollment in
the ongoing dose-escalation study expected throughout FY2021
AXO-Lenti-PD gene therapy for Parkinson’s disease
- Reported positive 6-month follow-up data from the second cohort
of the SUNRISE-PD Phase 2 trial in October 2020
- AXO-Lenti-PD was observed to be well-tolerated with no
treatment related serious adverse events at 6 months
- Greater than 2-hour improvement from baseline in both diary
“good ON time” and diary OFF time assessments observed across all
four patients in Cohort 2
- 21-point mean improvement in UPDRS Part III
“OFF” score in the two patients with evaluable data, representing
a 40% improvement from baseline
- Upcoming milestones:
- Continued development of a scaled-up suspension-based
manufacturing process at Oxford Biomedica to enable production of
clinical trial material, with certification of at least one batch
of clinical trial material by a Qualified Person expected in Q4
2021, following the successful manufacturing of both drug substance
and drug product.
Fiscal Fourth Quarter Financial
Summary
For the fourth fiscal quarter ended March 31, 2021, research and
development expenses were $8.2 million, a decrease of $2.7 million
compared to the prior year quarter. The current period decrease was
primarily related to reduced AXO-Lenti-PD program clinical expenses
as the enrollment of Cohort 2 was completed in the prior year
quarter, as well as lower manufacturing expenses due to the delays
in the development of a suspension-based manufacturing process at
our manufacturing partner, Oxford Biomedica.
General and administrative expenses for the fourth fiscal
quarter ended March 31, 2021 were $4.0 million, a decrease of $1.1
million compared to the prior year quarter primarily related to
reductions in stock-based compensation expense.
The net loss for the fourth fiscal quarter ended March 31, 2021
was $3.3 million, or $0.05 per share, compared to a net loss of
$16.6 million, or $0.54 per share, in the prior year quarter. The
net loss for the fourth fiscal quarter ended March 31, 2021 was net
of $9.1 million of gains on our long-term investment in Arvelle
Therapeutics B.V. ("Arvelle") that was sold in February 2021.
Fiscal Year Financial Summary
For the fiscal year ended March 31, 2021, research and
development expenses were $24.9 million, a decrease of $22.2
million compared to the fiscal year ended March 31, 2020. The
current period decrease was primarily related to $14.0 million in
certain nonrecurring development and regulatory milestones achieved
in the prior year for the AXO-Lenti-PD ($13.0 million) and
AXO-AAV-GM2 programs. In addition, there were reduced
program-specific research and development costs of $7.2 million due
to (i) lower AXO-Lenti-PD clinical expenses as the enrollment of
Cohort 2 was completed in the prior year, as well as lower
manufacturing expenses due to the delays at Oxford, (ii) reduced
clinical and manufacturing expenses while awaiting FDA clearance of
the IND for the AXO-AAV-GM2 program, and (iii) the discontinuation
of the AXO-AAV-OPMD program during the prior year.
General and administrative expenses for the fiscal year ended
March 31, 2021 were $17.3 million, a decrease of $4.8 million
compared to the fiscal year ended March 31, 2020, primarily related
to reductions in (i) stock-based compensation expense of $2.2
million primarily attributable to lower grant date fair values per
share for equity awards and lower headcount, and (ii) personnel
costs (including severance) of $1.3 million attributable to lower
headcount.
The net loss for the fiscal year ended March 31, 2021 was $32.4
million, or $0.62 per share, compared to a net loss of $72.6
million, or $2.93 per share, in the fiscal year ended March 31,
2020. The net loss for the fiscal year ended March 31, 2021 was net
of $11.3 million of gains on our long-term investment in Arvelle
that was sold in February 2021. For the fiscal year ended March 31,
2021, net cash used in operating activities was $46.6 million and
net cash provided by investing activities included $11.6 million of
proceeds received from the sale of our long-term investment in
Arvelle.
As of March 31, 2021, we had $119.0 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-K filed with the
Securities and Exchange Commission on June 9, 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.Consolidated Statements of
Operations(In thousands, except share and per share
amounts)
|
Three Months Ended March 31, |
|
Years Ended March 31 |
|
2021 |
|
2020 |
|
2021 |
|
2020 |
|
(unaudited) |
|
(unaudited) |
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development
expenses |
|
|
|
|
|
|
|
(includes stock-based compensation expense of $303 and $766 forthe
three months ended March 31, 2021 and 2020, respectively, and$1,583
and $2,772 for the years ended March 31, 2021 and
2020,respectively) |
$ |
8,244 |
|
|
$ |
10,920 |
|
|
$ |
24,903 |
|
|
$ |
47,110 |
|
General
and administrative expenses |
|
|
|
|
|
|
|
(includes stock-based compensation expense of $615 and $1,791
forthe three months ended March 31, 2021 and 2020, respectively,
and$2,909 and $5,123 for the years ended March 31, 2021 and
2020,respectively) |
3,965 |
|
|
5,133 |
|
|
17,294 |
|
|
22,061 |
|
Total operating expenses |
12,209 |
|
|
16,053 |
|
|
42,197 |
|
|
69,171 |
|
Other
(income) expenses: |
|
|
|
|
|
|
|
Interest expense |
1 |
|
|
440 |
|
|
799 |
|
|
4,377 |
|
Other income |
(8,971 |
) |
|
(127 |
) |
|
(10,359 |
) |
|
(1,358 |
) |
Loss
before income tax (benefit) expense |
(3,239 |
) |
|
(16,366 |
) |
|
(32,637 |
) |
|
(72,190 |
) |
Income
tax (benefit) expense |
92 |
|
|
282 |
|
|
(212 |
) |
|
438 |
|
Net
loss |
$ |
(3,331 |
) |
|
$ |
(16,648 |
) |
|
$ |
(32,425 |
) |
|
$ |
(72,628 |
) |
Net loss
per common share — basic and diluted |
$ |
(0.05 |
) |
|
$ |
(0.54 |
) |
|
$ |
(0.62 |
) |
|
$ |
(2.93 |
) |
Weighted-average common shares outstanding — basic and diluted |
66,251,597 |
|
|
30,939,688 |
|
|
52,181,398 |
|
|
24,812,536 |
|
SIO GENE THERAPIES
INC.Consolidated Balance Sheets(In
thousands, except share and per share amounts)
|
March 31, 2021 |
|
March 31, 2020 |
Assets |
|
|
|
Current
assets: |
|
|
|
Cash and cash equivalents |
$ |
118,986 |
|
|
$ |
80,752 |
|
Receivable from sale of long-term investment |
4,343 |
|
|
— |
|
Prepaid expenses and other current assets |
7,348 |
|
|
2,971 |
|
Income tax receivable |
1,656 |
|
|
1,707 |
|
Total current assets |
132,333 |
|
|
85,430 |
|
Long-term investment |
— |
|
|
5,871 |
|
Other non-current assets |
— |
|
|
46 |
|
Long-term restricted cash |
1,184 |
|
|
— |
|
Operating lease right-of-use assets |
1,152 |
|
|
1,532 |
|
Property and equipment, net |
478 |
|
|
801 |
|
Total
assets |
$ |
135,147 |
|
|
$ |
93,680 |
|
Liabilities and Shareholders’ Equity |
|
|
|
Current
liabilities: |
|
|
|
Accounts payable |
$ |
1,341 |
|
|
$ |
4,412 |
|
Accrued expenses |
9,196 |
|
|
11,319 |
|
Current portion of operating lease liabilities |
311 |
|
|
889 |
|
Current portion of long-term debt |
— |
|
|
15,423 |
|
Total current liabilities |
10,848 |
|
|
32,043 |
|
Operating lease liabilities, net of current portion |
932 |
|
|
79 |
|
Total liabilities |
11,780 |
|
|
32,122 |
|
Shareholders’ equity: |
|
|
|
Common stock, par value $0.00001 per share, 1,000,000,000 shares
authorized, 69,377,567 and 39,526,299 issued and
outstanding at March 31, 2021 and March 31, 2020,
respectively |
1 |
|
|
— |
|
Accumulated other comprehensive income (loss) |
335 |
|
|
(55 |
) |
Additional paid-in capital |
914,100 |
|
|
820,257 |
|
Accumulated deficit |
(791,069 |
) |
|
(758,644 |
) |
Total shareholders’ equity |
123,367 |
|
|
61,558 |
|
Total
liabilities and shareholders’ equity |
$ |
135,147 |
|
|
$ |
93,680 |
|
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