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Sio Gene Therapies, Inc. (AXGT)

NasdaqGS - NasdaqGS Real-time price. Currency in USD
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2.1000-0.0500 (-2.33%)
At close: 4:00PM EST
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Previous close2.1500
Open2.1200
Bid0.0000 x 2200
Ask0.0000 x 4000
Day's range2.0700 - 2.1700
52-week range1.4100 - 6.1200
Volume490,080
Avg. volume1,805,595
Market cap88.109M
Beta (5Y monthly)N/A
PE ratio (TTM)N/A
EPS (TTM)N/A
Earnings date11 Aug 2020
Forward dividend & yieldN/A (N/A)
Ex-dividend dateN/A
1y target est8.50
  • Globe Newswire

    Sio Gene Therapies Appoints Gene Therapy Pioneer Guangping Gao, Ph.D., as Chief AAV Scientific Advisor

    A pioneering researcher, Dr. Gao has made foundational contributions to the discovery of novel AAV vectors for gene therapyDr. Gao recently served as President of the American Society for Gene and Cell Therapy (ASGCT) and has been ranked as one of the top 20 translational researchers by Nature Biotechnology NEW YORK and RESEARCH TRIANGLE PARK, N.C., Nov. 19, 2020 (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 the appointment of Guangping Gao, Ph.D., as Chief AAV Scientific Advisor. Dr. Gao, a world-recognized scientist and past President of the ASGCT, has played key roles in the discovery and characterization of adeno-associated virus (AAV) serotypes which were instrumental in the resurgence of gene therapy. In his advisory role, Dr. Gao will provide strategic guidance and scientific and technical input across Sio’s AAV-based gene therapy programs. “We are honored to welcome Dr. Gao, a gene therapy pioneer, to Sio Gene Therapies,” said Pavan Cheruvu, M.D., Chief Executive Officer of Sio. “Dr. Gao brings an incredible wealth of knowledge ranging from fundamental discoveries in viral vectors, preclinical and clinical gene therapy product development, to viral manufacturing for clinical research. We believe his experience and insight will be invaluable to our team as we continue to advance our pipeline and evaluate potential business development opportunities. We look forward to collaborating with Dr. Gao as we work toward our mission of providing transformative treatments to patients with severe genetic disease.” Dr. Gao said, “Sio’s strategic approach to gene therapy directly targets the underlying disease biology, which I believe has the potential to lead to transformative and life-saving treatments. I have been impressed by the team’s comprehensive execution in driving forward clinical programs while in parallel laying a strong manufacturing foundation to support their mission to deliver these treatments to patients as rapidly as possible. I am thrilled to begin my role at Sio and look forward to leveraging my diverse experiences to fully unlock the potential of their gene therapy portfolio." Dr. Gao is Co-Director of the Li Weibo Institute for Rare Disease Research, Director of the Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems and Penelope Booth Rockwell Professor in Biomedical Research at the University of Massachusetts Medical School. Dr Gao’s more than 30 years in scientific research in molecular genetics have made foundational contributions to the development of viral vector gene therapy for rare genetic diseases including the discovery, development and engineering of novel viral vectors for in vivo gene delivery as well as preclinical and clinical gene therapy product development. He has also made significant contributions to the development of viral vector manufacturing for gene therapy applications and the development of technology platforms for novel gene therapy approaches in humans. Dr. Gao has published nearly 300 research papers and serves as the Executive Editor-In-Chief of Human Gene Therapy, Senior Editor of the Gene and Cell Therapy book series and serves on the Editorial Boards of several other gene therapy and virology journals. In addition to previously serving as President of ASGCT, he is an elected fellow of the U.S. National Academy of Inventors, holding 174 patents and an additional 373 patent applications pending with over 10 licensed to pharmaceutical companies. Dr. Gao is co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. 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 and Investors Parag MeswaniSio Gene Therapies, Inc.Chief Commercial Officerinvestors@siogtx.com Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

  • Globe Newswire

    Sio Gene Therapies Announces Corporate Updates and Financial Results for Second Fiscal Quarter Ended September 30, 2020

    Continued progress across pipeline of gene therapy programs, including recent IND clearance for AXO-AAV-GM2 in Tay-Sachs/Sandhoff diseasesCompleted rebranding to Sio Gene Therapies as part of corporate transformation aligning corporate structure and governance with current and future business activityCompany had $63.2 million of cash and cash equivalents as of September 30, 2020, with sufficient cash runway into Q4 2021 NEW YORK and RESEARCH TRIANGLE PARK, N.C., Nov. 13, 2020 (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 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 monthsGreater than 2-hour improvement from baseline in both diary “good ON time” and diary OFF time assessments observed across all four patients in Cohort 2Reported 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, 2020Appointment of Kristiina Vuori, M.D., Ph.D, as a new director, establishing a majority independent Board of DirectorsNo longer being a majority-owned and controlled public companyCompleted 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 2019Operating 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 $1,896 for the six months ended September 30, 2020 and 2019, respectively)4,491  5,051  9,131  11,519 Total operating expenses9,549  11,884  19,383  39,442 Other (income) expenses:       Interest expense1  1,313  797  2,871 Other expense (income)580  560  (1,486) (537)Loss before income tax (benefit) expense(10,130) (13,757) (18,694) (41,776)Income tax (benefit) expense(146) 127  (116) 165 Net loss$(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 diluted46,731,666  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, 2020Assets   Current assets:   Cash and cash equivalents$63,171  $80,752 Prepaid expenses and other current assets5,406  2,971 Income tax receivable1,747  1,707 Total current assets70,324  85,430 Long-term investment8,055  5,871 Other non-current assets169  46 Operating lease right-of-use assets663  1,532 Property and equipment, net560  801 Total assets$79,771  $93,680 Liabilities and Shareholders’ Equity    Current liabilities:   Accounts payable$2,172  $4,412 Accrued expenses7,837  11,319 Current portion of operating lease liabilities34  889 Current portion of long-term debt—  15,423 Total current liabilities10,043  32,043 Operating lease liabilities, net of current portion55  79 Total liabilities10,098  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 capital846,558  820,257 Accumulated deficit(777,222) (758,644)Accumulated other comprehensive loss337  (55)Total stockholders’ equity69,673  61,558 Total liabilities and stockholders’ equity$79,771  $93,680

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