SIOX Sio Gene Therapies Inc.

1.58
-0.06  -4%
Previous Close 1.64
Open 1.59
52 Week Low 1.49
52 Week High 4.15
Market Cap $116,112,179
Shares 73,488,721
Float 54,628,010
Enterprise Value $19,960,502
Volume 463,139
Av. Daily Volume 1,382,399
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Upcoming Catalysts

Drug Stage Catalyst Date
AXO-Lenti-PD - SUNRISE-PD
Parkinson's disease
Phase 1/2
Phase 1/2
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AXO-AAV-GM1
GM1 gangliosidosis
Phase 1/2
Phase 1/2
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Drug Pipeline

Drug Stage Notes
AXO-AAV-GM2
GM2 gangliosidosis
Phase 1/2
Phase 1/2
Phase 1/2 initiation announced February 3, 2021. Enrollment on-going, November 12, 2021.

Latest News

  1. –   Data from AXO-AAV-GM1 Phase 1/2 study demonstrated consistent dose-dependent biomarker improvement including normalization of beta-galactosidase activity in serum and GM1 ganglioside in CSF
    –   Granted Fast Track Designation for both AXO-AAV-GM1 and AXO-AAV-GM2 for the treatment of GM1 gangliosidosis and Tay-Sachs/Sandhoff disease
    –   Three AXO-Lenti-PD GMP campaigns successfully completed fill and finish, achieving target titers using suspension-based manufacturing process; on-track to complete certification of clinical trial material in Q4 2021
    –   $101.7 million of cash and cash equivalents as of September 30, 2021, providing cash runway into calendar Q4 2022

    NEW YORK and DURHAM, N.C., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies…

    –   Data from AXO-AAV-GM1 Phase 1/2 study demonstrated consistent dose-dependent biomarker improvement including normalization of beta-galactosidase activity in serum and GM1 ganglioside in CSF

    –   Granted Fast Track Designation for both AXO-AAV-GM1 and AXO-AAV-GM2 for the treatment of GM1 gangliosidosis and Tay-Sachs/Sandhoff disease

    –   Three AXO-Lenti-PD GMP campaigns successfully completed fill and finish, achieving target titers using suspension-based manufacturing process; on-track to complete certification of clinical trial material in Q4 2021

    –   $101.7 million of cash and cash equivalents as of September 30, 2021, providing cash runway into calendar Q4 2022

    NEW YORK and DURHAM, N.C., Nov. 12, 2021 (GLOBE NEWSWIRE) -- 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 second quarter ended September 30, 2021.

    "This quarter we advanced our mission of developing innovative gene therapies for patients in desperate need of new treatment options. Importantly, in October we shared the most comprehensive dataset yet from our lead program in GM1 gangliosidosis, demonstrating clear and consistent dose-dependent biomarker improvements paired with encouraging safety, neuroimaging, and clinical outcomes. These exciting results give us great confidence in the therapeutic potential of AXO-AAV-GM1, which we hope makes a difference in the lives of many patients and families living with this debilitating disease," said Pavan Cheruvu, M.D., Chief Executive Officer of Sio Gene Therapies.

    Dr. Cheruvu continued, "I'm also excited by the progress this year on the development of a reliable, suspension-based manufacturing process that has generated clinical trial material for the future development of AXO-Lenti-PD. This represents an inflection point for the program that clarifies our path forward in Parkinson's disease. We are pleased to end our second fiscal quarter of 2021 with cash runway into calendar Q4 2022 and a capable team that looks forward to continued execution."

    Key Highlights and Development Updates

    AXO-AAV-GM1 gene therapy for GM1 gangliosidosis

    • Presented positive interim safety and biomarker data from ongoing Phase 1/2 clinical study at the European Society of Gene and Cell Therapy (ESGCT) Virtual Congress in October 2021:
      • Ten patients have received gene therapy to date without serious adverse events (SAEs) attributable to AXO-AAV-GM1
      • AXO-AAV-GM1 was generally well-tolerated at both low and high doses, with the majority of adverse events considered mild to moderate
      • Data in Type II (late-infantile to juvenile) patients demonstrated a dose-dependent improvement in key biomarkers of disease activity: β-galactosidase enzyme activity in the serum and GM1 ganglioside activity in the CSF
        • Serum β-galactosidase activity achieved a normal range, increasing by 12× and 17× pre-treatment levels, respectively, in both patients in the high-dose cohort at six months
          • All five patients in the low-dose cohort saw a 1.3-2.3× increase in the same timeframe
        • Levels of CSF GM1 ganglioside, the toxic substrate which accumulates in patients with GM1 gangliosidosis, were normalized in both patients in the high-dose cohort with 42% and 72% reductions, respectively, at six months
          • GM1 ganglioside levels were below baseline in all five low-dose patients at 12 months
      • No overt disease progression in six out of seven patients treated across low- and high-dose cohorts
    • Granted Fast Track Designation by the U.S. Food and Drug Administration (FDA)
    • Upcoming milestones:
      • 1H 2022: Intend to present data update from Stage 1 of the study, including both Type I (early-infantile) and Type II patients, at future scientific conferences
      • 1H 2022: Intend to engage with the FDA to review Stage 1 data and discuss next steps for clinical development

    AXO-AAV-GM2 gene therapy for Tay-Sachs and Sandhoff diseases

    • Dosed first three 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 two patients at the low dose
    • Granted Fast Track Designation by the U.S. FDA
    • Expect continued patient identification, screening, and enrollment in Stage 1 of the dose-ranging trial throughout 2021

    AXO-Lenti-PD gene therapy for Parkinson's disease

    • Three GMP batches have successfully completed fill and finish, achieving target titers using the updated suspension-based process
    • On-track to complete final testing of these batches to support certification by a Qualified Person of at least one batch for use as clinical trial material in Q4 2021
    • Successfully completed a scientific advice meeting with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. regarding the AXO-Lenti-PD clinical development program. The MHRA provided guidance on the:
      • Appropriate development pathway for completion of the Phase 1 dose-ranging study
      • Acceptability of a comparability protocol between the prior adherent and new suspension-based manufacturing process
      • New device administration system to support bilateral simultaneous infusions
    • Expect to provide a program update in Q1 2022

    Fiscal Second Quarter Financial Summary

    Research and development expenses were $11.4 million for the three months ended September 30, 2021 and $5.1 million for the three months ended September 30, 2020. The $6.3 million increase was primarily related to increases in:

    (i) AXO-AAV-GM1 clinical trial material manufacturing expenses for the planned enrollment of infantile patients in the high-dose cohort, as well as clinical trial expenses due to the enrollment of juvenile patients in the high-dose cohort and for the enrollment of infantile patients in the low-dose cohort;

    (ii) AXO-AAV-GM2 clinical trial material manufacturing expenses for the planned enrollment of patients in the mid-to-high dose cohorts (versus the prior year period, when this program was on clinical hold), as well as clinical trial expenses associated with the ongoing enrollment of juvenile patients in the low-dose cohort and for the planned enrollment of patients in the low-to-mid dose cohorts; and

    (iii) personnel-related costs primarily due to increased headcount.

    These increases were partially offset by a $1.3 million decrease in AXO-Lenti-PD costs related to: (i) the delays at our partner, Oxford Biomedica (UK) Ltd. ("Oxford"), which have not only resulted in lower than expected manufacturing expenses, but have also delayed further clinical studies of AXO-Lenti-PD, and (ii) early development programs that were completed during the quarter ended December 31, 2020 and as a result, development expenses have also decreased in the current year period.

    General and administrative expenses were $9.7 million for the three months ended September 30, 2021 and $4.5 million for the three months ended September 30, 2020. The increase of $5.2 million was primarily related to $5.9 million of stock-based compensation expense associated with certain Roivant Sciences Ltd. ("RSL") equity instruments that are held by our Chief Executive Officer (the "RSL Equity Instruments"), for which expensing commenced upon the liquidity event vesting condition being met upon the closing of RSL's business combination with Montes Archimedes Acquisition Corp. ("MAAC") on September 30, 2021. Going forward, these charges are expected to decline significantly on a quarterly basis. Excluding the $5.9 million of stock-based compensation expense recorded for the RSL Equity Instruments during the three months ended September 30, 2021, general and administrative expenses decreased by $0.7 million primarily related to: (i) reductions in tax and legal 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, and (ii) decreased rent expense due to the downsizing of our New York office footprint.

    The net loss for the fiscal second quarter ended September 30, 2021 was $21.2 million, or $0.29 per share, compared to a net loss of $10.0 million, or $0.21 per share, in the fiscal second quarter ended September 30, 2020.

    Fiscal First-Half Financial Summary

    Research and development expenses were $19.5 million for the six months ended September 30, 2021 and $10.3 million for the six months ended September 30, 2020. The $9.2 million increase was primarily related to increases in:

    (i) AXO-AAV-GM1 clinical trial material manufacturing expenses for the planned enrollment of infantile patients in the high-dose cohort, as well as clinical trial expenses due to the ongoing enrollment of juvenile patients in the high-dose cohort and for the planned enrollment of infantile patients in the low-dose cohort;

    (ii) AXO-AAV-GM2 clinical trial material manufacturing expenses for the planned enrollment of patients in the mid-to-high dose cohorts (versus the prior year period, when this program was on clinical hold), as well as clinical trial expenses associated with the ongoing enrollment of juvenile patients in the low-dose cohort and for the planned enrollment of patients in the low-to-mid dose cohorts; and

    (iii) personnel-related costs primarily due to increased headcount.

    These increases were partially offset by a $2.4 million decrease in AXO-Lenti-PD costs related to: (i) the delays at Oxford, which have not only resulted in lower than expected manufacturing expenses, but have also delayed further clinical studies of AXO-Lenti-PD, and (ii) early development programs that were completed during the quarter ended December 31, 2020 and as a result, development expenses have also decreased in the current year period.

    General and administrative expenses were $13.6 million for the six months ended September 30, 2021 and $9.1 million for the six months ended September 30, 2020. The increase of $4.5 million was primarily related to $5.9 million of stock-based compensation expense recorded for the RSL Equity Instruments during the six months ended September 30, 2021. Going forward, these charges are expected to decline significantly on a quarterly basis. Excluding the $5.9 million of stock-based compensation expense recorded for the RSL Equity Instruments during the six months ended September 30, 2021, general and administrative expenses decreased by $1.4 million primarily related to: (i) decreased rent expense due to the downsizing of our New York office footprint, and (ii) reductions in 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 six months ended September 30, 2021 was $33.1 million, or $0.45 per share, compared to a net loss of $18.6 million, or $0.41 per share, in the six months ended September 30, 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 six months ended September 30, 2021, net cash used in operating activities was $21.9 million and net cash provided by investing activities of $4.1 million included $4.3 million of proceeds received from the sale of our long-term investment in Arvelle.

    As of September 30, 2021, we had $101.7 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 calendar 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," "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, 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 occurrence of adverse safety events during our current and future trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; 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) 751-4361



    Investors and Analysts

    David W. Nassif

    Sio Gene Therapies Inc.

    Chief Financial Officer and General Counsel



    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,
     2021 2020 2021 2020
    Operating expenses:       
    Research and development expenses       
    (includes stock-based compensation expense of $489 and $458 for the three months ended September 30, 2021 and 2020 and $921 and $1,021 for the six months ended September 30, 2021 and 2020, respectively)$11,448  $5,058  $19,506  $10,252 
    General and administrative expenses       
    (includes stock-based compensation expense of $6,809 and $650 for the three months ended September 30, 2021 and 2020 and $7,698 and $1,677 for the six months ended September 30, 2021 and 2020, respectively)9,748  4,491  13,607  9,131 
    Total operating expenses21,196  9,549  33,113  19,383 
    Other (income) expenses:       
    Interest expense11  1  12  797 
    Other expense (income)30  580  10  (1,486)
    Loss before income tax benefit(21,237) (10,130) (33,135) (18,694)
    Income tax benefit  (146) (28) (116)
    Net loss$(21,237) $(9,984) $(33,107) $(18,578)
    Net loss per share of common stock — basic and diluted$(0.29) $(0.21) $(0.45) $(0.41)
    Weighted-average shares of common stock outstanding — basic and diluted72,941,507  46,731,666  72,901,906  45,018,855 



    SIO GENE THERAPIES INC.

    Condensed Consolidated Balance Sheets

    (Unaudited, in thousands, except share and per share amounts)

     September 30, 2021 March 31, 2021
    Assets   
    Current assets:   
    Cash and cash equivalents$101,695  $118,986 
    Restricted cash1,184   
    Receivable from sale of long-term investment  4,343 
    Prepaid expenses and other current assets4,321  7,348 
    Income tax receivable1,733  1,656 
    Total current assets108,933  132,333 
    Long-term restricted cash  1,184 
    Operating lease right-of-use assets1,052  1,152 
    Property and equipment, net548  478 
    Total assets$110,533  $135,147 
    Liabilities and Stockholders' Equity    
    Current liabilities:   
    Accounts payable$2,673  $1,341 
    Accrued expenses7,366  9,196 
    Current portion of operating lease liabilities269  311 
    Total current liabilities10,308  10,848 
    Operating lease liabilities, net of current portion865  932 
    Total liabilities11,173  11,780 
    Stockholders' equity:   
    Common stock, par value $0.00001 per share, 1,000,000,000 shares authorized, 72,941,507 and 69,377,567 issued and outstanding at September 30, 2021 and March 31, 2021, respectively1  1 
    Additional paid-in capital923,198  914,100 
    Accumulated deficit(824,176) (791,069)
    Accumulated other comprehensive income337  335 
    Total stockholders' equity99,360  123,367 
    Total liabilities and stockholders' equity$110,533  $135,147 


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  2. - Completed three successful GMP campaigns using suspension-based manufacturing process
    - All batches achieved target titers and have successfully completed fill and finish
    - Qualified Person (QP) certification of at least one batch of clinical trial material on-track for Q4 2021 
    - Obtained scientific advice from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. regarding continued clinical development
    - Expect to provide program update in Q1 2022

    NEW YORK and DURHAM, N.C., Nov. 11, 2021 (GLOBE NEWSWIRE) -- 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 manufacturing and…

    - Completed three successful GMP campaigns using suspension-based manufacturing process

    - All batches achieved target titers and have successfully completed fill and finish

    - Qualified Person (QP) certification of at least one batch of clinical trial material on-track for Q4 2021 

    - Obtained scientific advice from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. regarding continued clinical development

    - Expect to provide program update in Q1 2022

    NEW YORK and DURHAM, N.C., Nov. 11, 2021 (GLOBE NEWSWIRE) -- 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 manufacturing and regulatory update for AXO-Lenti-PD, its clinical-stage gene therapy for Parkinson's disease.

    "Today's announcement is the culmination of several months of effort by the Sio and Oxford BioMedica teams focused on the development of a reliable, suspension-based manufacturing process to enable scale-up of production and advancement of the AXO-Lenti-PD program," said Pavan Cheruvu, M.D., Chief Executive Officer of Sio Gene Therapies. "With the successful manufacture of three batches, all of which achieved the target titer and have completed fill and finish, we now have a process that has generated sufficient clinical trial material for future clinical development. We believe these data, coupled with scientific advice from the MHRA, help clarify the path forward and collectively represent an inflection point for the AXO-Lenti-PD program."

    Key Highlights

    • Three GMP batches have successfully completed fill and finish, achieving target titers using the updated suspension-based process
    • Company is on-track to complete final testing of these three batches to support certification of at least one batch for use as clinical trial material by a Qualified Person in Q4 2021
    • Additionally, the Company successfully completed a scientific advice meeting with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. regarding the AXO-Lenti-PD clinical development program. The MHRA provided guidance on the:
      1. Appropriate development pathway for completion of the Phase 1 dose-ranging study
      2. Acceptability of a comparability protocol between the prior adherent and new suspension manufacturing process
      3. New device administration system to support bilateral simultaneous infusions
    • Expect to provide a program update in Q1 2022

    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," "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 occurrence of adverse safety events during our current and future trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; 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 August 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) 751-4361



    Investors and Analysts

    David W. Nassif

    Sio Gene Therapies Inc.

    Chief Financial Officer and General Counsel



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  3. -   Designation complements previously granted Rare Pediatric Disease and Orphan Drug designations
    -   Enrollment ongoing in the registration-enabling trial of AXO-AAV-GM2 in Tay-Sachs/Sandhoff diseases

    NEW YORK and DURHAM, N.C., Nov. 01, 2021 (GLOBE NEWSWIRE) -- 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 the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to AXO-AAV-GM2, an investigational gene therapy for the treatment of early infantile, late infantile, and juvenile-onset Tay-Sachs and Sandhoff disease. The Fast Track designation is intended to facilitate…

    -   Designation complements previously granted Rare Pediatric Disease and Orphan Drug designations

    -   Enrollment ongoing in the registration-enabling trial of AXO-AAV-GM2 in Tay-Sachs/Sandhoff diseases

    NEW YORK and DURHAM, N.C., Nov. 01, 2021 (GLOBE NEWSWIRE) -- 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 the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to AXO-AAV-GM2, an investigational gene therapy for the treatment of early infantile, late infantile, and juvenile-onset Tay-Sachs and Sandhoff disease. The Fast Track designation is intended to facilitate the development and review of drugs to treat serious conditions and fill an unmet medical need.

    "The FDA's decision to grant AXO-AAV-GM2 gene therapy Fast Track designation signifies an important milestone towards developing a safe and effective treatment for Tay-Sachs and Sandhoff diseases, both rare and fatal pediatric diseases with no approved treatments," said Pavan Cheruvu, M.D., Chief Executive Officer of Sio Gene Therapies. "This designation complements the previously granted Rare Pediatric Disease and Orphan Drug designations for AXO-AAV-GM2, along with similar designations for AXO-AAV-GM1 for GM1 gangliosidosis, and we look forward to working closely with the FDA as we continue enrollment in our ongoing, registration-enabling trial in Tay-Sachs and Sandhoff diseases to bring AXO-AAV-GM2 to patients and families."

    The current Phase 1/2 study (NCT04669535) is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of surgical delivery of AXO-AAV-GM2 directly to the brain and spinal cord of pediatric participants with both infantile and juvenile GM2 gangliosidosis (also known as Tay-Sachs or Sandhoff disease).

    GM2 gangliosidosis is a set of rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode the enzyme β-Hexosaminidase A. It can be categorized into two distinct diseases, Tay-Sachs disease, which results from a mutation in the gene encoding the alpha subunit of the β-Hexosaminidase A enzyme (HEXA), and Sandhoff disease, which results from a mutation in the gene encoding the beta subunit of the β-Hexosaminidase A enzyme (HEXB). Children affected by GM2 gangliosidosis suffer from a progressively debilitating disease course and reduced life expectancy. Currently, there are no FDA-approved treatment options for GM2 gangliosidosis.

    About AXO-AAV-GM2

    AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the β-hexosaminidase A (HexA) enzyme. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via delivery of two co-administered AAVrh8 vectors.

    AXO-AAV-GM2 has received Orphan Drug Designation, Rare Pediatric Disease Designation and Fast Track Designation from the Food and Drug Administration and is the only gene therapy in clinical development for all pediatric forms of GM2 gangliosidosis.

    In 2018, Sio licensed exclusive worldwide rights from UMass Chan Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

    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 occurrence of adverse safety events during our current and future trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; 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 August 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) 751-4361



     

    Investors and Analysts

    Parag V. Meswani, Pharm.D.

    Sio Gene Therapies Inc.

    Chief Commercial Officer



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  4. NEW YORK and DURHAM, N.C., Oct. 21, 2021 (GLOBE NEWSWIRE) -- 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, announced today that Gavin Corcoran, M.D., Chief Research and Development Officer, will be leaving the company effective November 12, 2021, to pursue an opportunity with a private healthcare technology company. Pavan Cheruvu, M.D., the Company's President and Chief Executive Officer (CEO), will assume Dr. Corcoran's responsibilities as Chief Research and Development Officer going forward. As part of a realignment of the Company's research and development organization, the Company has established two new…

    NEW YORK and DURHAM, N.C., Oct. 21, 2021 (GLOBE NEWSWIRE) -- 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, announced today that Gavin Corcoran, M.D., Chief Research and Development Officer, will be leaving the company effective November 12, 2021, to pursue an opportunity with a private healthcare technology company. Pavan Cheruvu, M.D., the Company's President and Chief Executive Officer (CEO), will assume Dr. Corcoran's responsibilities as Chief Research and Development Officer going forward. As part of a realignment of the Company's research and development organization, the Company has established two new executive positions, Chief Medical Officer (CMO) and Senior Vice President of Early Development and Scientific Affairs, both reporting directly to the CEO, and has commenced searches to fill these positions.

    "Gavin has been instrumental in leading our R&D organization through a period of growth as we evolved our business model towards the development of CNS gene therapies," said Pavan Cheruvu, M.D., Chief Executive Officer. "I'm proud of the team he has attracted who will carry on our patient-focused mission, and on behalf of the company, I want to thank Gavin for his many contributions to Sio."

    Dr. Cheruvu continued, "With the continued progress of our gene therapy programs in the clinic, including positive data announced earlier today in GM1 gangliosidosis, and plans to expand our pipeline by investing in next-generation genetic medicines, the addition of a CMO and SVP of Early Development and Scientific Affairs will strategically position us for the advancement and expansion of our pipeline."

    "I have enjoyed the time that I have spent at Sio and I am extremely proud of the R&D team that we have built. This is one of the best teams in the industry and they are well positioned to successfully move these programs to approval. It has been a real honor to work with our academic collaborators and patient organizations, and I know that the families and patients that need these medicines will be in good hands," said Dr. Corcoran. "We have come a long way in my three plus years since I joined Pavan at Sio, and now with a strong leadership team, a promising clinical-stage gene therapy pipeline, and one of the most experienced teams in gene therapy development, the future for Sio is bright. I'm looking forward to seeing Sio achieve continued success."

    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 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 August 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) 751-4361



    Investors and Analysts

    Parag V. Meswani

    Sio Gene Therapies Inc.

    Chief Commercial Officer

     



    Primary Logo

    View Full Article Hide Full Article
  5. NEW YORK and DURHAM, N.C., Oct. 21, 2021 (GLOBE NEWSWIRE) -- 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 the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of Type I (early infantile-onset) and Type II (late infantile-onset and juvenile-onset) GM1 gangliosidosis. The Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

    "Receiving Fast Track Designation is a…

    NEW YORK and DURHAM, N.C., Oct. 21, 2021 (GLOBE NEWSWIRE) -- 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 the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of Type I (early infantile-onset) and Type II (late infantile-onset and juvenile-onset) GM1 gangliosidosis. The Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

    "Receiving Fast Track Designation is a critical step in our mission to develop the first potential treatment for all pediatric forms of this rare, terminal disease. This designation joins both the Orphan Drug Designation and Rare Pediatric Disease Designation assigned to AXO-AAV-GM1 by the FDA, which we believe further demonstrates the potential impact of this work on the patient community," said Pavan Cheruvu, M.D., Chief Executive Officer of Sio Gene Therapies. "Building on the recently presented data at ESGCT demonstrating normalization of key disease biomarkers in the high-dose cohort with no serious adverse events attributed to AXO-AAV-GM1, this designation will help us accelerate clinical development of this promising investigational therapy for children and families."

    The current Phase 1/2 study (NCT03952637) is designed to evaluate the safety, tolerability, and potential efficacy of AXO-AAV-GM1 gene therapy delivered intravenously in children with early infantile, or Type I, and late infantile and juvenile, or Type II, GM1 gangliosidosis. Stage 1 of the study is a dose-escalation study in which the low-dose cohort is evaluating 1.5x1013 vg/kg and the high-dose cohort is evaluating a dose of 4.5x1013 vg/kg. Stage 2 of the trial will then evaluate the efficacy and safety of the optimal dose identified in Stage 1.

    GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene that cause impaired production of the β-galactosidase enzyme. Currently, there are no FDA-approved treatment options for GM1 gangliosidosis.

    About AXO-AAV-GM1

    AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring β-galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to achieve a broad central and peripheral biodistribution. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1's ability to increase β-galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

    AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA and is the only gene therapy in clinical development for all pediatric forms of GM1 gangliosidosis.

    In 2018, Sio licensed exclusive worldwide rights from UMass Chan Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

    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 occurrence of adverse safety events during our current and future trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; 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 August 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) 751-4361

     

    Investors and Analysts

    Parag V. Meswani

    Sio Gene Therapies Inc.

    Chief Commercial Officer



    Primary Logo

    View Full Article Hide Full Article
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