Sio Gene Therapies Provides Update on Cash Position and Major Upcoming GM1 Gangliosidosis Program Milestones
March 15 2021 - 7:00AM
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 announced
that it had $120.9 million in cash and cash equivalents as of March
12, 2021, augmented by a recent $15 million equity investment by
Suvretta Capital.
Since December 31, 2020, the Company has raised $49.1 million,
of which $37.1 million was received in gross proceeds from public
sales of its common stock (including the Suvretta Capital
investment) and $11.6 million was received from a non-dilutive
transaction (the sale of the Company’s shares in Arvelle
Therapeutics). The Company expects to receive another milestone
payment of approximately $4.8 million by mid-2021 upon the
marketing approval of cenobamate by the European Medicines Agency
(EMA).
“With a strengthened cash balance, we are well positioned to
fulfill our patient-oriented mission by advancing our
industry-leading pipeline programs well into the future. We believe
that the three products in our pipeline are the most advanced gene
therapies in clinical development for their respective indications,
and we look forward to progressing these programs and generating
additional data to support our leadership position,” said David
Nassif, Chief Financial Officer of Sio Gene Therapies.
The Company estimates that its current cash position extends
beyond the expected dates of major upcoming milestones for the
AXO-AAV-GM1 program:
- Updated 6-month data from the low-dose cohort of late-infantile
and juvenile (Type 2) children by mid-year 2021, to include
biomarker data from cerebrospinal fluid (CSF) as an indicator of
central nervous system activity
- 12-month topline data from the low-dose cohort in the second
half of 2021;
- 12-month data from the first two children dosed in the
high-dose cohort in Q1 2022;
- 24-month data from the low-dose cohort in mid-2022; and
- In the first half of 2022, meeting with the U.S. Food and Drug
Administration to discuss the registrational pathway for
AXO-AAV-GM1
“Thanks to the support of our clinical collaborators at the
N.I.H. and the GM1 patient community, our ongoing Phase 1/2 study
is currently leading the enrollment of gene therapy trials in GM1
gangliosidosis, with seven patients already enrolled across the low
and high-dose cohorts. We are grateful for this support and believe
our resources now extend beyond the dates of major upcoming
milestones for the AXO-AAV-GM1 program,” said Gavin Corcoran, MD,
Chief R&D Officer. “If the data collected at these key
milestones confirms, and builds upon, the preliminary results
reported in December 2020, AXO-AAV-GM1 may become the first
disease-modifying gene therapy to enter pivotal studies.”
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 “believe,” "estimate, " (may be” 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
of its upcoming clinical 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 Quarterly Report on Form
10-Q filed with the Securities and Exchange Commission on February
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 NassifSio Gene Therapies, Inc.Chief Financial Officer and
General Counsel(646) 677-6770david.nassif@siogtx.com
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