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 second fiscal quarter ended September 30,
2020.
“In recent months, we have taken significant strides forward
with our AAV-based gene therapy programs in GM1 gangliosidosis and
Tay-Sachs/Sandhoff diseases. We obtained rare pediatric disease
designation for both programs, and following IND clearance of
AXO-AAV-GM2 from the FDA, we’re delivering on our goal of advancing
the first potentially curative gene therapy clinical development
programs for both GM1 and GM2 gangliosidosis,” said Pavan Cheruvu,
M.D., Chief Executive Officer of Sio Gene Therapies. “We also
advanced AXO-Lenti-PD in the SUNRISE-PD dose-escalation study and
presented detailed patient-level data from the mid-dose cohort at
our Parkinson’s disease focused R&D Day last month. Our
rebranding as Sio Gene Therapies signifies a new beginning for the
company – with a scientific focus, management team, Board of
Directors, and portfolio strategy that is wholly committed to
developing disease-modifying and curative genetic medicines on
behalf of patients in need.”
Key Highlights and Development
Updates
AXO-AAV-GM1 gene therapy for GM1
gangliosidosis
- On-track to report 6-month topline
data, with a focus on safety and tolerability, from 5 children in
the low-dose juvenile cohort (Type II) by year-end 2020.
- The U.S. Food and Drug
Administration (FDA) granted Rare Pediatric Disease Designation for
AXO-AAV-GM1 in GM1 gangliosidosis.
- Expect to complete dosing of
juvenile (Type II) patients in the high-dose cohort of the ongoing
AXO-AAV-GM1 clinical study before year-end 2020.
AXO-AAV-GM2 gene therapy for Tay-Sachs/Sandhoff
disease
- FDA cleared Company-sponsored
Investigational New Drug (IND) application for AXO-AAV-GM2 in
Tay-Sachs and Sandhoff diseases.
- FDA granted Rare Pediatric Disease
Designation for AXO-AAV-GM2.
AXO-Lenti-PD gene therapy for Parkinson’s
disease (PD)
- Hosted an R&D Day on October
30, 2020, during which individual patient-level 6-month follow up
data were presented from the second cohort of the SUNRISE-PD dose
escalation trial. In addition, key opinion leaders in Parkinson’s
disease clinical research and the Michael J. Fox Foundation
discussed the treatment landscape and the potential role of
AXO-Lenti-PD gene therapy in the treatment paradigm.
- Reported positive 6-month follow-up
data from the second cohort of the SUNRISE-PD Phase 2 trial
- 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
- Reported a
21-point mean improvement in UPDRS Part III
“OFF” score in the two patients with evaluable data, a 40%
improvement from baseline
- Totality of individual patient
outcomes across cohort demonstrate consistency of treatment
benefit
- Based on new information received from our manufacturing
partner, Oxford Biomedica, in mid-October regarding delays in CMC
data and third-party fill/finish issues, the development of a
suspension-based manufacturing process for AXO-Lenti-PD will take
longer than expected. As a result, the Company believes that it is
unlikely that its planned randomized, sham-controlled trial of
AXO-Lenti-PD will enroll patients by the end of calendar year 2021.
Manufacturing of several GMP batches is now underway and planned at
Oxford Biomedica with a goal of generating material for use in
future clinical trials as soon as possible. The Company expects to
provide an update in the first quarter of 2021 or as program
timelines are clarified.
Corporate Updates
- Continued corporate transformation
activities, including:
- Company name change to Sio Gene
Therapies. In connection with the name change, the Company’s ticker
on the NASDAQ exchange will change to “SIOX” and will be effective
at market open on November 13, 2020
- Appointment of Kristiina Vuori,
M.D., Ph.D, as a new director, establishing a majority independent
Board of Directors
- No longer being a majority-owned
and controlled public company
- Completed redomiciliation to
Delaware
- Signed strategic gene therapy development and manufacturing
partnership with Viralgen, an AskBio subsidiary, securing access to
cGMP capacity and resources to support the development and
commercialization of AAV gene therapy programs in GM1
gangliosidosis and Tay-Sachs/Sandhoff diseases.
- Opening of new laboratory space in Research Triangle Park,
North Carolina, focused on in-house preclinical and analytical
development activities.
- Promoted Parag V. Meswani, Pharm.D. to Chief Commercial Officer
to support Axovant’s commercialization efforts across the
clinical-stage pipeline.
Fiscal Second Quarter Financial Summary
For the second fiscal quarter ended September 30, 2020, research
and development expenses were $5.1 million, a decrease of $1.8
million compared to the prior year quarter, primarily due to (i)
lower AXO-Lenti-PD clinical expenses of approximately $0.9 million
as the enrollment of Cohort 2 was completed in February 2020, (ii)
reduced costs of $0.7 million while awaiting FDA clearance of the
IND for the AXO-AAV-GM2 program, and (iii) a $0.5 million reversal
of an accrual for manufacturing development services for our
AXO-AAV-GM1 and AXO-AAV-GM2 programs under an agreement that was
terminated.
General and administrative expenses for the second fiscal
quarter ended September 30, 2020 were $4.5 million, a decrease of
$0.6 million compared to the prior year quarter, primarily due to
reductions in personnel costs (including severance) attributable to
reduced headcount.
The net loss for the second fiscal quarter ended September 30,
2020 was $10.0 million, or $0.21 per share, compared to a net loss
of $13.9 million, or $0.61 per share, in the prior year
quarter.
Fiscal First-Half Financial Summary
For the six months ended September 30, 2020, research and
development expenses were $10.3 million, a decrease of $17.7
million compared to the six months ended September 30, 2019.
Excluding the net amount of $13.0 million due to Oxford for a
development milestone achieved in the prior year period as well as
a decrease of $2.1 million of expenses associated with our
discontinued legacy AXO-AAV-OPMD program that was terminated in
September 2019, research and development expenses decreased by $2.6
million in the current year period. The current period decrease was
primarily due to (i) reduced costs of $1.0 million while awaiting
FDA clearance of the IND for the AXO-AAV-GM2 program, (ii) a $0.8
million payment in the prior year period to our licensor,
University of Massachusetts Medical School, for reaching a
manufacturing milestone for the AXO-AAV-GM1 program, and (iii) a
$0.5 million reversal of an accrual for manufacturing development
services for our AXO-AAV-GM1 and AXO-AAV-GM2 programs under an
agreement that was terminated.
General and administrative expenses for the six months ended
September 30, 2020 were $9.1 million, a decrease of $2.4 million
compared to the six months ended September 30, 2019, primarily
related to reductions in (i) personnel costs (including severance)
of $1.3 million and stock-based compensation expense of $0.2
million attributable to reduced headcount, and (ii) pharmaceutical
market research expenses of $0.6 million.
The net loss for the six months ended September 30, 2020 was
$18.6 million, or $0.41 per share, compared to a net loss of $41.9
million, or $1.84 per share, in the six months ended September 30,
2019. Net cash used in operating activities was $25.3 million for
the six months ended September 30, 2020.
As of September 30, 2020, we had $63.2 million of cash and cash
equivalents. The Company holds no short-term or long-term debt on
the balance sheet. We expect the cash and cash equivalents to
sustain our operations into the fourth calendar quarter of
2021.
On October 2, we filed a prospectus supplement with the SEC
pertaining to a $50 million at-the-market equity financing
facility. No sales under the facility occurred prior to our press
release on October 6 and no sales have occurred since October 9.
Approximately 1.2 million shares for total proceeds of $5.1
million, net of brokerage fees, were sold under the facility during
this period.
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 “will,” “expect,” “believe,” “estimate,”
and other similar expressions are intended to identify
forward-looking statements. For example, all statements Sio makes
regarding costs associated with its operating activities 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
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 Quarterly Report on Form
10-Q filed with the Securities and Exchange Commission on November
13, 2020, 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.
Contacts:
Media
Josephine Belluardo, Ph.D. LifeSci Communications(646)
751-4361jo@lifescicomms.cominfo@siogtx.com
Investors and Analysts
David NassifSio Gene Therapies, Inc.Chief Financial Officer and
General Counsel(646) 677-6770investors@siogtx.com
SIO GENE THERAPIES
INC.Condensed Consolidated Statements of
Operations(Unaudited, in thousands, except share and per
share amounts)
|
Three Months Ended September 30, |
|
Six Months Ended September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Operating expenses: |
|
|
|
|
|
|
|
Research and development
expenses |
|
|
|
|
|
|
|
(includes stock-based compensation expense of $458 and $409 for the
three months ended September 30, 2020 and 2019 and $1,021 and
$1,130 for the six months ended September 30, 2020 and 2019,
respectively) |
$ |
5,058 |
|
|
$ |
6,833 |
|
|
$ |
10,252 |
|
|
$ |
27,923 |
|
General and administrative
expenses |
|
|
|
|
|
|
|
(includes stock-based compensation expense of $650 and $482 for the
three months ended September 30, 2020 and 2019 and $1,677 and
$1,896 for the six months ended September 30, 2020 and 2019,
respectively) |
4,491 |
|
|
5,051 |
|
|
9,131 |
|
|
11,519 |
|
Total operating expenses |
9,549 |
|
|
11,884 |
|
|
19,383 |
|
|
39,442 |
|
Other (income) expenses: |
|
|
|
|
|
|
|
Interest expense |
1 |
|
|
1,313 |
|
|
797 |
|
|
2,871 |
|
Other expense (income) |
580 |
|
|
560 |
|
|
(1,486 |
) |
|
(537 |
) |
Loss before income tax (benefit)
expense |
(10,130 |
) |
|
(13,757 |
) |
|
(18,694 |
) |
|
(41,776 |
) |
Income tax (benefit) expense |
(146 |
) |
|
127 |
|
|
(116 |
) |
|
165 |
|
Net loss |
$ |
(9,984 |
) |
|
$ |
(13,884 |
) |
|
$ |
(18,578 |
) |
|
$ |
(41,941 |
) |
Net loss per common share — basic
and diluted |
$ |
(0.21 |
) |
|
$ |
(0.61 |
) |
|
$ |
(0.41 |
) |
|
$ |
(1.84 |
) |
Weighted-average common shares
outstanding — basic and diluted |
46,731,666 |
|
|
22,783,182 |
|
|
45,018,855 |
|
|
22,781,657 |
|
SIO GENE THERAPIES
INC.Condensed Consolidated Balance
Sheets(Unaudited, in thousands, except share and per share
amounts)
|
September 30, 2020 |
|
March 31, 2020 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
63,171 |
|
|
$ |
80,752 |
|
Prepaid expenses and other current assets |
5,406 |
|
|
2,971 |
|
Income tax receivable |
1,747 |
|
|
1,707 |
|
Total current assets |
70,324 |
|
|
85,430 |
|
Long-term investment |
8,055 |
|
|
5,871 |
|
Other non-current assets |
169 |
|
|
46 |
|
Operating lease right-of-use
assets |
663 |
|
|
1,532 |
|
Property and equipment, net |
560 |
|
|
801 |
|
Total assets |
$ |
79,771 |
|
|
$ |
93,680 |
|
Liabilities and
Shareholders’ Equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable |
$ |
2,172 |
|
|
$ |
4,412 |
|
Accrued expenses |
7,837 |
|
|
11,319 |
|
Current portion of operating lease liabilities |
34 |
|
|
889 |
|
Current portion of long-term debt |
— |
|
|
15,423 |
|
Total current liabilities |
10,043 |
|
|
32,043 |
|
Operating lease liabilities, net
of current portion |
55 |
|
|
79 |
|
Total liabilities |
10,098 |
|
|
32,122 |
|
Stockholders’ equity: |
|
|
|
Common stock, par value $0.00001 per share, 1,000,000,000 shares
authorized, 47,249,729 and 39,526,299 issued and outstanding at
September 30, 2020 and March 31, 2020, respectively |
— |
|
|
— |
|
Additional paid-in capital |
846,558 |
|
|
820,257 |
|
Accumulated deficit |
(777,222 |
) |
|
(758,644 |
) |
Accumulated other comprehensive loss |
337 |
|
|
(55 |
) |
Total stockholders’ equity |
69,673 |
|
|
61,558 |
|
Total liabilities and
stockholders’ equity |
$ |
79,771 |
|
|
$ |
93,680 |
|
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