Sio Gene Therapies Announces Prioritization of Lead Gene Therapy Programs in GM1 and GM2 Gangliosidosis, Extension of Cash Runway, and CEO Transition
January 31 2022 - 4:01PM
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 announcing the prioritization of AXO-AAV-GM1 and
AXO-AAV-GM2, its clinical stage AAV gene therapy programs for GM1
and GM2 gangliosidosis (Tay-Sachs/Sandhoff disease). The portfolio
prioritization extends the Company’s estimated cash runway into the
second half of 2023, beyond multiple key clinical milestones for
both gene therapy programs. With this prioritization, the company
intends to terminate its licensing agreement with Oxford Biomedica
for AXO-Lenti-PD, its lentiviral gene therapy program for
Parkinson’s disease.
In addition, the Company has appointed David Nassif, J.D., its
Chief Financial Officer and General Counsel, as interim Chief
Executive Officer (CEO) and a member of the Board of Directors with
the resignation of its CEO, Dr. Pavan Cheruvu, M.D., who is leaving
the company to pursue new opportunities. A search committee of the
Sio Board will consider both internal and external candidates to
identify a permanent successor CEO with the assistance of a leading
executive search firm.
Mr. Nassif added, “I’m honored to take the helm of Sio as we
solidify the focus of the organization on rare genetic diseases and
extend our cash runway into the second half of 2023. I believe our
programs in GM1 and GM2 have the ability to transform the care for
patients and deserve this heightened focus. We will continue to
actively consider a variety of initiatives and options to further
improve our financial position in order to provide the necessary
capital to continue to pursue our key priorities for the longer
term. We believe that pragmatic measures to grow our business in a
well-managed process are aligned with the interests of patients in
need, our employees and our shareholders, and we look forward to
updating our progress on these efforts in the future.”
“The Board is confident that Sio is in good hands with David’s
deep expertise in finance, law and operations, which will ensure
that the company continues operating seamlessly to successfully
advance our priority programs targeting GM1 and GM2 gangliosidosis.
The Board is grateful for Pavan’s service to the company and wishes
him all the best moving forward,” said Frank Torti, Chairman of the
Board.
Dr. Cheruvu added, “Serving as the CEO of Sio for the last four
years has been an immense privilege. I’m proud of our efforts to
transform Sio into a leader in CNS gene therapies. I’m grateful to
have had the opportunity to lead the company and am optimistic that
its rare disease programs will have the potential to improve the
lives of many children with GM1 and GM2 gangliosidosis.”
Updated Financial PositionThe Company provided
an update on its financial resources, including having cash and
cash equivalents of approximately $82 million as of December 31,
2021, and expects its estimated cash runway to fund current
operations into the second half of 2023. The Company intends to
report fiscal Q3 2021 results on February 11, 2022.
Program Status and Strategic Priorities
- AXO-AAV-GM1
- Ten patients across all pediatric subtypes of GM1
gangliosidosis have received AXO-AAV-GM1 gene therapy to-date. The
data 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
- 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
- AXO-Lenti-PD
- After a thorough analysis of the Parkinson’s landscape, the
company is deprioritizing its Parkinson’s disease program due to
several factors, including resource requirements and development
timelines to reach meaningful value inflection for the program and
an increasingly challenging market and regulatory environment for
Parkinson’s disease
- The Company expects the rights to the program to revert back to
Oxford Biomedica by March 31, 2022.
David Nassif, J.D., has served as Sio’s Chief Financial Officer
and General Counsel since July 2019, and has more than 25 years of
life sciences industry experience in executive financial management
roles in development-stage, commercial-stage, public and private
companies. Prior to joining Sio, Mr. Nassif was Executive Vice
President, Chief Financial Officer and General Counsel of SteadyMed
Therapeutics, a specialty pharmaceutical company, where he was
instrumental in its initial public offering in 2015 and in its
acquisition by United Therapeutics in August 2018. From 2011 to
2014, Mr. Nassif served as the President and Chief Financial
Officer of Histogen, a regenerative medicine company, where he also
oversaw manufacturing and quality systems. Previously, he was
Executive Vice President, General Counsel and Chief Financial
Officer of Zogenix, a CNS-focused specialty pharmaceutical company
and the Executive Vice President, Chief Financial Officer, General
Counsel and Head of Business Development at Amphastar
Pharmaceuticals, a leading generic and specialty pharmaceutical
company, where he originated three company-defining strategic
transactions. Earlier in his career, Mr. Nassif held various
positions with Cypros Pharmaceuticals, where he was instrumental in
leading its merger with Ribogene, Inc. to form Questcor
Pharmaceuticals, Inc. He holds a BS in finance and management
information systems with honors from the University of Virginia and
a J.D. from the University of Virginia School of Law.
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. 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 November 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 AnalystsParag V. Meswani,
Pharm.D.Sio Gene Therapies Inc.Chief Commercial
OfficerParag.Meswani@siogtx.com
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