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
corporate updates and financial results for its fiscal third
quarter ended December 31, 2021.
“As we recently announced, I’m excited to lead Sio as we enhance
our focus on rare genetic diseases and implement measures to extend
our expected cash runway into the second half of 2023,” said David
Nassif, J.D., interim Chief Executive Officer of Sio. “While the
relentless nature of GM1 and GM2 gangliosidosis drives us to move
with urgency every day in order to potentially transform the lives
of these patients and their families, we believe our GM1 and GM2
programs also bring meaningful value for our shareholders. These
programs represent a critical opportunity, where the life-changing
value for families desperately in need and the market value for our
shareholders perfectly overlap and provide the motivation to keep
our team moving with a sense of purpose for all stakeholders. I
look forward to sharing continued updates across each of these
programs as they reach meaningful clinical milestones.”
Key Developments and Priorities
- AXO-AAV-GM1
- Presented updated safety data from the most advanced GM1 gene
therapy program in the industry, at the 18th Annual WORLDSymposium™
2022, held from February 7-11, 2022
- Ten patients across all pediatric subtypes (early infantile,
late infantile, and juvenile) of GM1 gangliosidosis have received
AXO-AAV-GM1 gene therapy to date
- AXO-AAV-GM1 remains generally well tolerated in all ten
patients at both low and high doses to date
- There have been no reported drug-related serious adverse
events
- The majority of adverse events were considered mild to
moderate, and no safety signals have been identified
- The totality of the data to date have demonstrated a favorable
risk: benefit profile and a dose-dependent improvement in key
biomarkers of disease activity (β-galactosidase enzyme activity in
the serum and GM1 ganglioside activity in the CSF) across the low-
and high-dose cohorts
- Based on developmental and mobility assessments, there was no
clinical evidence of disease progression in 4 out of 5 low-dose
subjects at 12 months or in the high-dose cohort at 6 months
- Strategic Priorities:
- Calendar 1H 2022: Present a data update from Stage 1 of the
Phase 1/2 study, including a first look at Type I (early infantile)
patients treated in the low-dose cohort and longer-term data from
the Type II (late infantile to juvenile) patient cohort at future
scientific conferences
- Calendar 2022: Intend to engage with the FDA to review Stage 1
data and discuss next steps for clinical development
- AXO-AAV-GM2
- Dosed first four 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 three patients at the low
dose
- Strategic Priorities:
- 2022: Expect continued patient identification, screening, and
enrollment in the mid-dose cohort (n= ~3) of the dose-ranging
trial
Fiscal Third Quarter Financial Summary
Research and development expenses were $21.3 million for the
three months ended December 31, 2021 and $6.4 million for the
three months ended December 31, 2020, increasing by $14.9
million. The $14.9 million increase was primarily related to:
(i) increased AXO-AAV-GM1 program
expenses primarily related to clinical trial material manufacturing
expenses for the planned enrollment of infantile patients in the
high-dose cohort, as well as a $1.5 million license fee milestone
due in December 2021 under the terms of our exclusive license
agreement with the University of Massachusetts Medical School
("UMMS" and collectively, the "UMMS Agreement");
(ii) increased AXO-AAV-GM2 program
expenses primarily related to a $1.5 million license fee milestone
due in December 2021 under the terms of the UMMS Agreement, as well
as non-GMP and GMP manufacturing expenses;
(iii) increased AXO-Lenti-PD program
expenses, primarily related to clinical trial material
manufacturing expenses resulting from Qualified Person
certification of GMP batches and a $2.0 million nonrecurring
development milestone achieved upon the successful completion of
the updated suspension-based manufacturing process for the
AXO-Lenti-PD program in the fourth calendar quarter of 2021;
and
(iv) increased personnel-related
costs primarily due to increased headcount.
General and administrative expenses were $4.1 million for the
three months ended December 31, 2021 and $4.2 million for the
three months ended December 31, 2020. The decrease of $0.1
million was primarily related to decreases totaling $1.0 million
for (i) rent, depreciation and overhead expenses due to the
downsizing of our New York office footprint, and (ii) tax, legal,
auditing and accounting fees resulting primarily from the
simplification of our corporate structure and the domestication of
Sio Gene Therapies Inc. from Bermuda to Delaware that was completed
in November 2020. These decreases were partially offset by an
increase of $0.7 million of stock-based compensation expense
primarily associated with certain equity instruments of our
affiliate, Roivant Sciences Ltd. ("RSL"), held by our former CEO
(the "RSL Equity Instruments"), who resigned as our CEO in January
2022. Expensing of the RSL Equity Instruments commenced upon the
liquidity event vesting condition being met upon the closing of
RSL's business combination with Montes Archimedes Acquisition Corp.
on September 30, 2021.
The net loss for the fiscal third quarter ended December 31,
2021 was $25.5 million, or $0.35 per share, compared to a net loss
of $10.5 million, or $0.20 per share, in the fiscal third quarter
ended December 31, 2020.
Nine-Months Financial Summary
Research and development expenses were $40.8 million for the
nine months ended December 31, 2021 and $16.7 million for the
nine months ended December 31, 2020, increasing by $24.1
million. The $24.1 million increase was primarily related to:
(i) increased AXO-AAV-GM1 program
expenses primarily related to clinical trial material manufacturing
expenses for the planned enrollment of infantile patients in the
high-dose cohort, as well as a $1.5 million license fee milestone
due in December 2021 under the terms of the UMMS Agreement;
(ii) increased AXO-AAV-GM2 program
expenses primarily related to non-GMP and GMP manufacturing
expenses, clinical trial expenses associated with the ongoing
enrollment of patients in the low-dose cohort and for the planned
enrollment of patients in the mid-dose cohort, as well as a $1.5
million license fee milestone due in December 2021 under the terms
of the UMMS Agreement;
(iii) increased AXO-Lenti-PD program
expenses, primarily related to clinical trial material
manufacturing expenses resulting from Qualified Person
certification of GMP batches and a $2.0 million nonrecurring
development milestone achieved upon the successful completion of
the updated suspension-based manufacturing process for the
AXO-Lenti-PD program in the fourth calendar quarter of 2021, which
were partially offset by decreased analytical development costs;
and
(iv) increased personnel-related
costs primarily due to increased headcount.
General and administrative expenses were $17.7 million for the
nine months ended December 31, 2021 and $13.3 million for the
nine months ended December 31, 2020. The increase of $4.4
million was primarily related to $6.7 million of stock-based
compensation expense primarily associated with the RSL Equity
Instruments recorded during the nine months ended December 31,
2021. This increase was partially offset by decreases totaling $2.7
million for (i) rent, depreciation and overhead expenses due to the
downsizing of our New York office footprint, and (ii) tax, legal,
auditing and accounting fees resulting primarily from the
simplification of our corporate structure and the domestication of
Sio Gene Therapies Inc. from Bermuda to Delaware that was completed
in November 2020.
The net loss for the nine months ended December 31, 2021 was
$58.6 million, or $0.80 per share, compared to a net loss of $29.1
million, or $0.61 per share, in the nine months ended December 31,
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 nine months ended December 31, 2021, net
cash used in operating activities was $42.5 million and net cash
provided by investing activities of $4.0 million included $4.3
million of proceeds received from the sale of our long-term
investment in Arvelle.
As of December 31, 2021, we had $81.9 million of cash and cash
equivalents. We hold no short-term or long-term debt on the balance
sheet. As a result of our decision to terminate the AXO-Lenti-PD
gene therapy program for the treatment of Parkinson's disease that
is expected to become effective by March 31, 2022, we estimate that
our current cash and cash equivalents are sufficient to support
operations into the second half of calendar year 2023, including
beyond the expected dates of major upcoming milestones for our
AXO-AAV-GM1 and AXO-AAV-GM2 gene therapy programs.
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 is comprised of 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. 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 “believe”, “expect”, “intend”, “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, expected cash burn runway, expectations regarding
licensing and commercial agreements, 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 Sio’s operations; the actual funds and/or
runway required for Sio’s clinical and product development
activities and anticipated upcoming milestones; actual costs
related to Sio’s clinical and product development activities and
Sio’s 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 occurrence of adverse safety events
during our current and future trials; the scaling up of
manufacturing; the outcome of interactions with regulatory agencies
and 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 Quarterly Report on Form 10-Q filed with the Securities
and Exchange Commission on February 11, 2022, 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.Interim Chief Executive
Officer; 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 December 31, |
|
Nine Months Ended December 31, |
|
2021 |
|
2020 |
|
2021 |
|
2020 |
Operating expenses: |
|
|
|
|
|
|
|
Research and development
expenses |
|
|
|
|
|
|
|
(includes stock-based compensation expense of $130 and $259 for the
three months ended December 31, 2021 and 2020, respectively, and
$1,051 and $1,280 for the nine months ended December 31, 2021 and
2020, respectively) |
$ |
21,287 |
|
|
$ |
6,407 |
|
|
$ |
40,793 |
|
|
$ |
16,659 |
|
General and administrative
expenses |
|
|
|
|
|
|
|
(includes stock-based compensation expense of $1,268 and $617 for
the three months ended December 31, 2021 and 2020, respectively,
and $8,966 and $2,294 for the nine months ended December 31, 2021
and 2020, respectively) |
|
4,086 |
|
|
|
4,198 |
|
|
|
17,693 |
|
|
|
13,329 |
|
Total operating expenses |
|
25,373 |
|
|
|
10,605 |
|
|
|
58,486 |
|
|
|
29,988 |
|
Other expenses (income): |
|
|
|
|
|
|
|
Interest expense |
|
7 |
|
|
|
1 |
|
|
|
19 |
|
|
|
798 |
|
Other expense (income) |
|
76 |
|
|
|
98 |
|
|
|
86 |
|
|
|
(1,388 |
) |
Loss before income tax
benefit |
|
(25,456 |
) |
|
|
(10,704 |
) |
|
|
(58,591 |
) |
|
|
(29,398 |
) |
Income tax benefit |
|
— |
|
|
|
(188 |
) |
|
|
(28 |
) |
|
|
(304 |
) |
Net loss |
$ |
(25,456 |
) |
|
$ |
(10,516 |
) |
|
$ |
(58,563 |
) |
|
$ |
(29,094 |
) |
Net loss per share of common
stock — basic and diluted |
$ |
(0.35 |
) |
|
$ |
(0.20 |
) |
|
$ |
(0.80 |
) |
|
$ |
(0.61 |
) |
Weighted-average shares of common stock outstanding — basic and
diluted |
|
73,335,279 |
|
|
|
52,679,816 |
|
|
|
73,046,889 |
|
|
|
47,581,795 |
|
SIO GENE THERAPIES INC. |
Condensed Consolidated Balance Sheets |
(Unaudited, in thousands, except share and per share amounts) |
|
|
December 31, 2021 |
|
March 31, 2021 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
81,910 |
|
|
$ |
118,986 |
|
Restricted cash |
|
1,184 |
|
|
|
— |
|
Receivable from sale of long-term investment |
|
— |
|
|
|
4,343 |
|
Prepaid expenses and other current assets |
|
5,244 |
|
|
|
7,348 |
|
Income tax receivable |
|
1,732 |
|
|
|
1,656 |
|
Total current assets |
|
90,070 |
|
|
|
132,333 |
|
Long-term restricted cash |
|
— |
|
|
|
1,184 |
|
Operating lease right-of-use
assets |
|
2,613 |
|
|
|
1,152 |
|
Property and equipment,
net |
|
623 |
|
|
|
478 |
|
Total assets |
$ |
93,306 |
|
|
$ |
135,147 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable |
$ |
6,611 |
|
|
$ |
1,341 |
|
Accrued expenses |
|
7,762 |
|
|
|
9,196 |
|
Current portion of operating lease liabilities |
|
765 |
|
|
|
311 |
|
Total current liabilities |
|
15,138 |
|
|
|
10,848 |
|
Operating lease liabilities,
net of current portion |
|
1,903 |
|
|
|
932 |
|
Total liabilities |
|
17,041 |
|
|
|
11,780 |
|
Stockholders’ equity: |
|
|
|
Common stock, par value $0.00001 per share, 1,000,000,000 shares
authorized, 73,697,110 and 69,377,567 issued and outstanding at
December 31, 2021 and March 31, 2021, respectively |
|
1 |
|
|
|
1 |
|
Additional paid-in capital |
|
925,558 |
|
|
|
914,100 |
|
Accumulated deficit |
|
(849,632 |
) |
|
|
(791,069 |
) |
Accumulated other comprehensive income |
|
338 |
|
|
|
335 |
|
Total stockholders’ equity |
|
76,265 |
|
|
|
123,367 |
|
Total liabilities and
stockholders’ equity |
$ |
93,306 |
|
|
$ |
135,147 |
|
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