AXGT Axovant Gene Therapies Ltd.

3.05
+0.07  (+2%)
Previous Close 2.98
Open 3
52 Week Low 1.41
52 Week High 8.08
Market Cap $127,967,258
Shares 41,956,478
Float 23,339,339
Enterprise Value $79,734,294
Volume 251,743
Av. Daily Volume 331,005
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Upcoming Catalysts

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

Drug Stage Notes
AXO-AAV-GM2
GM2 gangliosidosis
Phase 1
Phase 1
Phase 1 data presented October 23, 2019.

Latest News

    • Data readouts for AXO-Lenti-PD and AXO-AAV-GM1 remain on-track for Q4 2020
    • Completed 3-year manufacturing and supply agreement with Oxford BioMedica for AXO-Lenti-PD
    • Company had $55.5 million of cash and cash equivalents as of June 30, 2020

    NEW YORK and BASEL, Switzerland, Aug. 11, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (NASDAQ:AXGT), a clinical-stage gene therapy company developing innovative gene therapies for neurodegenerative diseases, today provided financial results for its first fiscal quarter ended June 30, 2020.

    "During our first fiscal quarter, we continued to make significant progress across our three clinical-stage gene therapy development programs in Parkinson's disease, GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases…

    • Data readouts for AXO-Lenti-PD and AXO-AAV-GM1 remain on-track for Q4 2020
    • Completed 3-year manufacturing and supply agreement with Oxford BioMedica for AXO-Lenti-PD
    • Company had $55.5 million of cash and cash equivalents as of June 30, 2020

    NEW YORK and BASEL, Switzerland, Aug. 11, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (NASDAQ:AXGT), a clinical-stage gene therapy company developing innovative gene therapies for neurodegenerative diseases, today provided financial results for its first fiscal quarter ended June 30, 2020.

    "During our first fiscal quarter, we continued to make significant progress across our three clinical-stage gene therapy development programs in Parkinson's disease, GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases and remain on-track for key data readouts later this year," said Pavan Cheruvu, M.D., Chief Executive Officer of Axovant. "Notably this quarter, we completed Type II (Juvenile) patient enrollment in the low dose cohort of our dose-escalation study of AXO-AAV-GM1 and have begun preparations to initiate treatment of Type I (infantile) GM1 later this year. Additionally, our gene therapy manufacturing strategy has meaningfully progressed as we extended our supply collaboration with Oxford BioMedica around a new scalable, suspension-based manufacturing process to support our clinical studies and future commercialization of AXO-Lenti-PD. In the coming months, we look forward to presenting program updates across each of our three clinical-stage programs and will work tirelessly to execute our strategy on behalf of patients, families, caregivers and our shareholders."

    Key Highlights and Development Updates

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

    • Completed enrollment of the second dose cohort with the enrollment of four patients and remain on-track to deliver 6-month safety and efficacy data in Q4 2020.
    • Entered into a clinical supply and manufacturing agreement with Oxford BioMedica to produce clinical trial material at-scale to support randomized, controlled Phase 2 and Phase 3 clinical studies and eventual commercialization.
    • Anticipated completion of the first batch of AXO-Lenti-PD manufactured using a suspension-based process by year-end 2020.
    • Enrollment of the first subject in a randomized, sham-controlled Phase 2 study of AXO-Lenti-PD anticipated in 2021.
    • Presented an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in May 2020 summarizing the mechanistic rationale and development status of AXO-Lenti-PD by Gregory Stewart, Ph.D., SVP, Scientific Affairs.

    AXO-AAV-GM1 gene therapy for GM1 gangliosidosis

    • Enrolled five Type II (juvenile) patients in the low-dose cohort of Stage 1 of the registrational study with 6-month safety and efficacy data expected in Q4 2020.
    • Expanded protocol through successful IND amendment to enable dosing of Type I (infantile) patients and to explore a higher dose of 4.5 x 1013 vg/kg in both infantile and juvenile patients.
    • Enrollment of both Type I and Type II patients at the low and high-dose cohorts expected to continue throughout the second half of 2020.
    • Presented an oral presentation at the ASGCT Annual Meeting in May 2020 demonstrating the superior efficacy of intravenous GM1 gene therapy administration in the feline model from collaborator Amanda Gross from Auburn University (Poster #495). 

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

    • Company-sponsored investigational new drug (IND) application clearance is expected in Q4 2020.
    • Presented clinical and preclinical data at the ASGCT Annual Meeting in May 2020 highlighting the mechanistic rationale for gene replacement in Tay-Sachs/Sandhoff disease and preliminary evidence of safety and efficacy from a first-in-human gene therapy study in Tay-Sachs disease. 

      -  Oral presentation on clinical data from two children treated in an expanded access clinical trial for the first-in-human gene therapy trial for Tay-Sachs Disease reported by Terence Flotte, M.D., Provost & Executive Deputy Chancellor, University of Massachusetts Medical School and Principal Investigator (Poster #1299).

      -  Poster presentation on the surgical technique for bilateral intrathalamic infusion of rAAVrh8-HEXA/HEXB gene therapy in an infant with Tay-Sachs Disease from collaborator Oguz Cataltepe, M.D., Professor, University of Massachusetts Medical School and Director of Pediatric Neurosurgery, UMass Memorial Medical Center (Poster #666).

      -  Oral presentation on preclinical data from intravenous AAV gene therapy demonstrating improved lifespan and clinical metrics in feline Sandhoff Disease from collaborator Anne Maguire from Auburn University (Poster #493).

    Corporate Updates

    • Axovant continues its corporate transformation to align corporate structure and governance with current and future business activity. Specific changes intended to occur by March 31, 2021 include:

      -  Redomiciliation to Delaware from Bermuda

      -  Plans to initiate a corporate name change of Axovant Gene Therapies Ltd.

      -  Transition of the Board of Directors to a majority of independent members

    First Fiscal Quarter Financial Summary

    For the first fiscal quarter ended June 30, 2020, research and development expenses were $5.2 million, a decrease of $15.9 million compared to the prior year quarter. Excluding a development milestone of $13.0 million achieved and due to our partner, Oxford BioMedica (UK) Ltd., in the prior year quarter, research and development expenses decreased by $2.9 million, primarily related to the termination of our legacy AXO-AAV-OPMD program in September 2019.

    General and administrative expenses for the first fiscal quarter ended June 30, 2020 were $4.6 million, a decrease of $1.9 million compared to the prior year quarter, primarily due to reductions in (i) personnel costs of $0.8 million and share-based compensation expense of $0.4 million attributable to reduced headcount and (ii) pharmaceutical market research costs of $0.6 million.

    Net loss for the first fiscal quarter ended June 30, 2020 was $8.6 million, or $0.20 per share, compared to a net loss of $28.1 million, or $1.23 per share, in the prior year quarter. Net cash used in operating activities was $13.5 million for the first fiscal quarter ended June 30, 2020.

    As of June 30, 2020, we had $55.5 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 second calendar quarter of 2021.

    About Axovant

    Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinson's disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit www.axovant.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 "intended", "may," "might," "will," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovant's management that, although Axovant 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 Axovant 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 scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Axovant's scientific approach and general development progress; and the availability or commercial potential of Axovant's product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovant's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 11, 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. Axovant 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

    Parag Meswani

    Axovant Gene Therapies Ltd.

    (212) 547-2523

    Investors and Analysts

    David Nassif

    Axovant Gene Therapies Ltd.

    Chief Financial Officer and General Counsel

    (646) 677-6770





    AXOVANT GENE THERAPIES LTD.

    Condensed Consolidated Statements of Operations

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

     Three Months Ended June 30,
     2020 2019
    Operating expenses:   
    Research and development expenses   
    (includes $563 and $721 of share-based compensation expense for the three months ended June 30, 2020 and 2019, respectively)$5,194   $21,090  
    General and administrative expenses   
    (includes $1,027 and $1,414 of share-based compensation expense for the three months ended June 30, 2020 and 2019, respectively)4,640   6,468  
    Total operating expenses9,834   27,558  
    Other (income) expenses:   
    Interest expense796   1,558  
    Other income(2,066) (1,097)
    Loss before income tax expense(8,564) (28,019)
    Income tax expense30   38  
    Net loss$(8,594) $(28,057)
    Net loss per common share — basic and diluted$(0.20) $(1.23)
    Weighted-average common shares outstanding — basic and diluted43,287,222   22,780,114  





    AXOVANT GENE THERAPIES LTD.

    Condensed Consolidated Balance Sheets

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

     June 30, 2020 March 31, 2020
    Assets   
    Current assets:   
    Cash and cash equivalents$55,482  $80,752 
    Prepaid expenses and other current assets3,998  2,971 
    Income tax receivable1,717  1,707 
    Total current assets61,197  85,430 
    Long-term investment8,055  5,871 
    Other non-current assets46  46 
    Operating lease right-of-use assets1,105  1,532 
    Property and equipment, net633  801 
    Total assets$71,036  $93,680 
        
    Liabilities and Shareholders' Equity    
    Current liabilities:   
    Accounts payable$2,970  $4,412 
    Accrued expenses8,949  11,319 
    Current portion of operating lease liabilities465  889 
    Current portion of long-term debt  15,423 
    Total current liabilities12,384  32,043 
    Operating lease liabilities, net of current portion71  79 
    Total liabilities12,455  32,122 
        
    Shareholders' equity:   
    Common shares, par value $0.00001 per share, 1,000,000,000 shares authorized, 40,973,380 and 39,526,299 issued and outstanding at June 30, 2020 and March 31, 2020, respectively   
    Additional paid-in capital825,830  820,257 
    Accumulated deficit(767,238) (758,644)
    Accumulated other comprehensive loss(11) (55)
    Total shareholders' equity58,581  61,558 
    Total liabilities and shareholders' equity$71,036  $93,680 

     

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  1. Oxford Biomedica Signs Three Year Clinical Supply Agreement with Axovant Gene Therapies for Manufacture and Supply of AXO-Lenti-PD
             

    Oxford, UK – 31st July, 2020: Oxford Biomedica plc (LSE:OXB) ("Oxford Biomedica" or "the Group"), a leading gene and cell therapy group, announced today that it has signed a three year Clinical Supply Agreement ("CSA") with a wholly-owned subsidiary of Axovant Gene Therapies Ltd. ("Axovant") (NASDAQ:AXGT). The CSA builds on the worldwide license agreement signed between the two companies in June 2018 for the Parkinson's disease gene therapy program OXB-102, now called AXO-Lenti-PD.

    Under the terms of the CSA, Oxford Biomedica will manufacture GMP batches for Axovant to support the ongoing and future clinical…

    Oxford Biomedica Signs Three Year Clinical Supply Agreement with Axovant Gene Therapies for Manufacture and Supply of AXO-Lenti-PD

             

    Oxford, UK – 31st July, 2020: Oxford Biomedica plc (LSE:OXB) ("Oxford Biomedica" or "the Group"), a leading gene and cell therapy group, announced today that it has signed a three year Clinical Supply Agreement ("CSA") with a wholly-owned subsidiary of Axovant Gene Therapies Ltd. ("Axovant") (NASDAQ:AXGT). The CSA builds on the worldwide license agreement signed between the two companies in June 2018 for the Parkinson's disease gene therapy program OXB-102, now called AXO-Lenti-PD.

    Under the terms of the CSA, Oxford Biomedica will manufacture GMP batches for Axovant to support the ongoing and future clinical development of AXO-Lenti-PD, a clinical-stage gene therapy product to treat moderate to severe Parkinson's Disease based on Oxford Biomedica's LentiVector® platform. Axovant is currently conducting a Phase 2 SUNRISE-PD trial with AXO-Lenti-PD. Dosing of all patients in the second cohort is completed with 6-month safety and efficacy data expected in the fourth quarter of 2020. OXB expects to manufacture AXO-Lenti-PD in its commercial-scale GMP manufacturing facilities including Oxbox in the UK, and additionally in other OXB GMP facilities as required to ensure security of supply.

    John Dawson, Chief Executive Officer of Oxford Biomedica, said: "This new Agreement builds upon our existing worldwide licensing agreement with Axovant and highlights the strengths of Oxford Biomedica's commercial GMP manufacturing capabilities. We are pleased with how the partnership is progressing and excited by the clinical progress to date. The agreement today signals our commitment to the efficient ongoing development of this much needed product for patients with Parkinson's disease.

    "We are now at a stage in the partnership where we can determine the manufacturing activities and infrastructure required to support the mid and late-stage development of AXO-Lenti-PD in a way which is compatible with later commercialisation and we look forward to this next phase of our partnership."

    Pavan Cheruvu, Chief Executive Officer at Axovant Gene Therapies, said: "This Agreement with Oxford Biomedica means that together we can continue to advance the development of AXO-Lenti-PD in Parkinson's disease. We are pleased to extend our partnership with Oxford Biomedica, a world leader in lentiviral vector development and manufacturing, as we scale-up AXO-Lenti-PD production to support our Phase 2 and Phase 3 clinical studies and enable commercialization of the product. This marks another mutual accomplishment for our Parkinson's disease program where we expect to enroll the first subject in a randomized, sham-controlled trial in 2021."

    -Ends-

    Enquiries: 
     



    Oxford Biomedica plc



     



    John Dawson, Chief Executive Officer

    Stuart Paynter, Chief Financial Officer

    Catherine Isted, Head of Corporate Development & IR



     
     



     



     



    T: +44 (0)1865 783 000

    T: +44 (0)1865 783 000

    T: +44 (0)1865 954 161 / E:



     
     



    Consilium Strategic Communications



     



    Mary-Jane Elliott/Matthew Neal
     



     



     



    T: +44 (0)20 3709 5700

    About Oxford Biomedica

    Oxford Biomedica (LSE:OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford Biomedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector®), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology, CNS disorders, liver diseases and respiratory disease. The Group has also entered into a number of partnerships, including with Novartis, Bristol Myers Squibb, Sanofi, Axovant Gene Therapies, Orchard Therapeutics, Santen, Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium and Imperial Innovations, through which it has long-term economic interests in other potential gene and cell therapy products. Additionally the group has signed a Clinical and Commercial Supply Agreement with AstraZeneca for manufacture of the adeno based COVID-19 vaccine candidate, AZN1222. Oxford Biomedica is based across several locations in Oxfordshire, UK and employs more than 550 people. Further information is available at www.oxb.com

    About AXO-Lenti-PD

    AXO-Lenti-PD is an investigational gene therapy for the treatment of Parkinson's disease that is designed to deliver three genes (tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase) via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The investigational gene therapy aims to provide patient benefit for years following a single administration. The SUNRISE-PD Phase 2 trial is ongoing with dosing completed for all patients in cohort 2, with 6 month safety and efficacy data expected in Q4 2020. Axovant expects to dose the first patient in the Part B randomized, sham controlled study in 2021.

    About Axovant Gene Therapies

    Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinson's disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit www.axovant.com.

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  2. NEW YORK and BASEL, Switzerland, July 31, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (NASDAQ:AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, announced today that its subsidiary has signed a three-year Clinical Supply Agreement ("CSA") with Oxford Biomedica plc (LSE:OXB), a leading gene and cell therapy group. The CSA builds on the worldwide license agreement signed between the two companies in June 2018 for the Parkinson's disease gene therapy program OXB-102, now called AXO-Lenti-PD.

    Under the terms of the CSA, Oxford Biomedica will manufacture GMP batches for Axovant to support the ongoing and future clinical development of AXO-Lenti-PD, a clinical-stage gene therapy product to…

    NEW YORK and BASEL, Switzerland, July 31, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (NASDAQ:AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, announced today that its subsidiary has signed a three-year Clinical Supply Agreement ("CSA") with Oxford Biomedica plc (LSE:OXB), a leading gene and cell therapy group. The CSA builds on the worldwide license agreement signed between the two companies in June 2018 for the Parkinson's disease gene therapy program OXB-102, now called AXO-Lenti-PD.

    Under the terms of the CSA, Oxford Biomedica will manufacture GMP batches for Axovant to support the ongoing and future clinical development of AXO-Lenti-PD, a clinical-stage gene therapy product to treat moderate to severe Parkinson's Disease based on Oxford Biomedica's LentiVector® platform. Axovant is currently conducting the Phase 2 SUNRISE-PD trial with AXO-Lenti-PD. Dosing of all patients in the second cohort has been completed with 6-month safety and efficacy data expected in the fourth quarter of 2020. Oxford Biomedica expects to manufacture AXO-Lenti-PD in its commercial-scale GMP manufacturing facilities including Oxbox in the UK, and additionally in other OXB GMP facilities as required to ensure security of supply.

    "This Agreement with Oxford Biomedica means that together we can continue to advance the development of AXO-Lenti-PD in Parkinson's disease," said Pavan Cheruvu, Axovant Chief Executive Officer. "We are pleased to extend our partnership with Oxford Biomedica, a world leader in lentiviral vector development and manufacturing, as we scale-up AXO-Lenti-PD production to support our Phase 2 and Phase 3 clinical studies and enable commercialization of the product. This marks another mutual accomplishment for our Parkinson's disease program where we expect to enroll the first subject in a randomized, sham-controlled trial in 2021."

    John Dawson, CEO of Oxford Biomedica, added, "This new Agreement builds upon our existing worldwide licensing agreement with Axovant and highlights the strengths of Oxford Biomedica's commercial GMP manufacturing capabilities. We are pleased with how the partnership is progressing and excited by the clinical progress to date. The agreement today signals our commitment to the efficient ongoing development of this much needed product for patients with Parkinson's disease. We are now at a stage in the partnership where we can determine the manufacturing activities and infrastructure required to support the mid and late-stage development of AXO-Lenti-PD in a way which is compatible with later commercialization and we look forward to this next phase of our partnership."

    About Axovant Gene Therapies

    Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinson's disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit www.axovant.com.

    About AXO-Lenti-PD

    AXO-Lenti-PD is an investigational gene therapy for the treatment of Parkinson's disease that is designed to deliver three genes (tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase) via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The investigational gene therapy aims to provide patient benefit for years following a single administration. The SUNRISE-PD Phase 2 trial is ongoing with dosing completed for all patients in cohort 2, with 6-month safety and efficacy data expected in Q4 2020. Axovant expects to dose the first patient in the Part B randomized, sham controlled study in 2021.

    About Oxford Biomedica

    Oxford Biomedica (LSE:OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford Biomedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector®), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology, CNS disorders, liver diseases and respiratory disease. The Group has also entered into a number of partnerships, including with Novartis, Bristol Myers Squibb, Sanofi, Axovant Gene Therapies, Orchard Therapeutics, Santen, Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium and Imperial Innovations, through which it has long-term economic interests in other potential gene and cell therapy products. Additionally the group has signed a Clinical and Commercial Supply Agreement with AstraZeneca for manufacture of the adeno based COVID-19 vaccine candidate, AZN1222. Oxford Biomedica is based across several locations in Oxfordshire, UK and employs more than 550 people. Further information is available at www.oxb.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 "may," "might," "will," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovant's management that, although Axovant 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 Axovant 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 scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of its gene therapy product candidates and platforms; Axovant's scientific approach and general development progress; and the availability or commercial potential of Axovant's product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovant's most recent Annual Report on Form 10- K filed with the Securities and Exchange Commission on June 10, 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. Axovant 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 & Investors

    Parag Meswani

    Axovant Gene Therapies Ltd.

    (212) 547-2523



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    • Completed enrollment of cohort 2 of SUNRISE-PD Phase 2 study of AXO-Lenti-PD in Parkinson's disease, with data from this cohort expected in Q4 2020
    • Completed enrollment of low-dose cohort of Phase 1/2 clinical study of AXO-AAV-GM1 for GM1 gangliosidosis, with data from this cohort expected in Q4 2020
    • Cash position of $80.8 million as of March 31, 2020 and expected runway into the second calendar quarter of 2021

    NEW YORK and BASEL, Switzerland, June 10, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (NASDAQ:AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, today provided financial results and corporate updates for its fiscal year ended March 31, 2020.

    "Over the last year, Axovant has progressed…

    • Completed enrollment of cohort 2 of SUNRISE-PD Phase 2 study of AXO-Lenti-PD in Parkinson's disease, with data from this cohort expected in Q4 2020

    • Completed enrollment of low-dose cohort of Phase 1/2 clinical study of AXO-AAV-GM1 for GM1 gangliosidosis, with data from this cohort expected in Q4 2020
    • Cash position of $80.8 million as of March 31, 2020 and expected runway into the second calendar quarter of 2021

    NEW YORK and BASEL, Switzerland, June 10, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (NASDAQ:AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, today provided financial results and corporate updates for its fiscal year ended March 31, 2020.

    "Over the last year, Axovant has progressed toward becoming a leading gene therapy company focused on delivering transformative benefit to patients with neurological diseases, while extending the reach of gene therapies to highly prevalent populations such as Parkinson's disease," said Pavan Cheruvu, M.D., Chief Executive Officer of Axovant. "We are encouraged by early findings across our programs, which now include evidence of durable motor improvement following administration of AXO-Lenti-PD in Parkinson's disease, and the first reported evidence of disease stabilization in infants with Tay-Sachs disease following administration of AXO-AAV-GM2. We have also completed enrollment in the low-dose cohort of our dose escalation study of AXO-AAV-GM1, and are expanding the ongoing registrational program to include patients with Type I (infantile) GM1 gangliosidosis. The dedication and resilience of our employees and scientific collaborators has fueled our progress, and we appreciate the continued trust of patients, families and our shareholders as we continue to advance Axovant's mission."

    Key Highlights and Development Updates

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

    • Dosing of all four patients in the second dose cohort (1.4 x 107 TU) of SUNRISE-PD dose-escalation study was completed in February 2020, with 6-month data from this cohort expected in Q4 2020.
    • At 12 months, 22-point mean improvement from baseline on UPDRS Part III "OFF" score observed for the first dose cohort treated with AXO-Lenti-PD, compared with 7-points, 11-points and 15-points of improvement from baseline for each of ProSavin cohorts 1, 2 and 3, respectively.
    • Expected completion of the first manufacturing batch of AXO-Lenti-PD using a suspension-based process by year-end 2020.
    • Expected enrollment of the first subject in a randomized, controlled trial of AXO-Lenti-PD in 2021.

    AXO-AAV-GM1 gene therapy for GM1 gangliosidosis

    • Completed enrollment of five late infantile and juvenile onset (Type II) patients in the low-dose cohort of Stage 1 of the registrational study with 6-month data expected in Q4 2020.
    • Reported an update from the first child dosed with AXO-AAV-GM1 under an investigator-initiated IND in the fourth quarter of calendar year 2019 indicating safety, tolerability, and evidence of clinical improvement following administration of gene therapy.
    • IND amendment cleared to expand Stage 2 of the registrational study protocol to include infantile (Type I) patients, the population most severely affected by the disease, and to evaluate a higher dose level for both Type I and Type II patients. 
    • Expect to initiate dosing in the high-dose cohort, which will include both Type I and Type II patients, in the second half of 2020.
    • Received U.S. Food and Drug Administration (FDA) orphan drug designation for AXO-AAV-GM1 for the treatment of GM1 gangliosidosis.

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

    • Reported the first evidence for potential disease modification in Tay-Sachs disease from an expanded access study administering investigational AXO-AAV-GM2 gene therapy in two patients with infantile (Type I) Tay-Sachs disease.
    • AXO-AAV-GM2 was successfully administered in both patients and has been generally well-tolerated to date, with no serious adverse events or clinically relevant laboratory abnormalities related to therapy.
    • Clearance of a Company-sponsored investigational new drug (IND) application is expected in calendar year 2020.

    Corporate Updates

    • Announced collaboration with Invitae, a leading medical genetics company, in the Detect Lysosomal Storage Diseases program to facilitate faster diagnoses for children with lysosomal storage disorders including GM1 gangliosidosis and GM2 gangliosidosis, also known as Tay-Sachs/Sandhoff disease.
    • Announced in February 2020 the closing of an underwritten public offering resulting in net proceeds of $70.8 million.
    • Announced in April 2020 the prepayment in full of $15.7 million outstanding loan from Hercules Capital.

    Fiscal Fourth Quarter Financial Summary

    For the fiscal fourth quarter ended March 31, 2020, research and development expenses were $10.9 million, an increase of $3.8 million over the prior year quarter. The current period increase was principally due to an increase in costs related to the Company's gene therapy programs, while the prior year period reflected a winddown of the small-molecule programs and the beginning of the increase in spending on the gene therapy programs. Within the current period research and development expense, $0.8 million was attributable to non-cash, share-based compensation expense compared to $1.5 million in the prior-year quarter. The current period decrease in share-based compensation expense is due to reduced headcount.

    General and administrative expenses for the fiscal fourth quarter ended March 31, 2020 were $5.1 million, a decrease of $1.0 million compared to the prior-year quarter. The current decrease was principally due to reductions of $0.8 million in personnel costs and $0.3 million in share-based compensation expense related to reduced headcount. Within the current period expense, $1.8 million was attributable to non-cash, share-based compensation expense compared to $2.1 million in the prior-year quarter.

    Net loss for the fiscal fourth quarter ended March 31, 2020 was $16.6 million, or $0.54 per share, compared to a net loss of $9.0 million, or $0.45 per share, for the prior-year quarter. Net cash used in operating activities for the fiscal fourth quarter ended March 31, 2020 was $17.9 million.

    Fiscal Year Financial Summary

    For the fiscal year ended March 31, 2020, research and development expenses were $47.1 million, a decrease of $40.5 million compared to the prior year. Excluding upfront license fees and development and regulatory milestones achieved and due to our partners, Oxford, Benitec and UMMS of $14.0 million in the current year and $46.0 million in the prior year, research and development expenses decreased by $8.5 million. Program-specific costs increased by $12.6 million in the current year for the gene therapy programs currently under development, which were offset by decreases of (i) $9.5 million related to the wind-down of the small molecule programs, (ii) $4.9 million in share-based compensation and personnel-related costs primarily associated with a decrease in headcount, and (iii) $2.4 million in costs allocated under our service agreements with Roivant Sciences, Inc. ("RSI") and Roivant Sciences GmbH ("RSG"), which are wholly owned subsidiaries of our affiliate, Roivant Sciences Ltd., as a result of the decentralization of the services formerly provided to us. Within the current period expense, $2.8 million was attributable to non-cash, share-based compensation expense compared to $4.8 million in the prior-year period.

    General and administrative expenses for the fiscal year ended March 31, 2020 were $22.1 million, a decrease of $17.4 million compared to the prior year, primarily due to reductions in (i) share-based compensation expense of $6.6 million and personnel costs of $2.3 million attributable to reduced headcount, (ii) legal fees of $4.5 million, and (iii) costs allocated under our services agreements with RSI and RSG of $2.6 million as a result of the decentralization of the services provided to us. Within the current period, $5.1 million was attributable to non-cash, share-based compensation expense compared to $11.7 million in the prior year period.

    Net loss for the year ended March 31, 2020 was $72.6 million, or $2.93 per share, compared to a net loss of $129.1 million or $8.02 per share, in the prior-year period. Net cash used in operating activities was $67.5 million for the fiscal year ended March 31, 2020.

    As of March 31, 2020, the Company had $80.8 million of cash and cash equivalents, working capital of $53.4 million, and gross indebtedness of $15.7 million, which was fully prepaid in April 2020. In February 2020, the Company issued and sold 16.6 million common shares and pre-funded warrants to purchase up to 3.3 million common shares in a follow-on public offering resulting in net proceeds to the Company of approximately $70.8 million. In April 2020, the Company prepaid the $16.0 million outstanding balance due under the Loan Agreement with Hercules. The Company expects the cash and cash equivalents to sustain its operations into the second calendar quarter of 2021.

    About Axovant Gene Therapies

    Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinson's disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit www.axovant.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 "may," "might," "will," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovant's management that, although Axovant 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 Axovant 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 scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of its gene therapy product candidates and platforms; Axovant's scientific approach and general development progress; and the availability or commercial potential of Axovant's product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovant's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission on June 10, 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. Axovant 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

    Parag Meswani

    Axovant Gene Therapies Ltd.

    (212) 547-2523

    Investors and Analysts

    David Nassif

    Axovant Gene Therapies Ltd.

    Chief Financial Officer and General Counsel

    (646) 677-6770



    AXOVANT GENE THERAPIES LTD.

     Consolidated Statements of Operations

    (in thousands, except share and per share amounts)

     Three Months Ended March 31, Years Ended March 31,
     2020 2019 2020 2019
     (unaudited) (unaudited)    
    Operating expenses:       
    Research and development expenses(1)       
    (includes share-based compensation of $766 and $1,459 for the three months ended March 31, 2020 and 2019 and $2,772 and $4,758 for the years ended March 31, 2020 and 2019, respectively)$10,920  $7,149  $47,110  $87,552 
    General and administrative expenses(2)       
    (includes share-based compensation of $1,791 and $2,096 for the three months ended March 31, 2020 and 2019 and $5,123 and $11,671 for the years ended March 31, 2020 and 2019, respectively)5,133  6,157  22,061  39,466 
    Total operating expenses16,053  13,306  69,171  127,018 
    Interest expense440  1,722  4,377  7,530 
    Other income(127) (5,891) (1,358) (5,616)
    Loss before income tax expense(16,366) (9,137) (72,190) (128,932)
    Income tax expense (benefit)282  (91) 438  133 
    Net loss$(16,648) $(9,046) $(72,628) $(129,065)
    Net loss per common share — basic and diluted$(0.54) $(0.45) $(2.93) $(8.02)
    Weighted average common shares outstanding — basic and diluted30,939,688  19,962,170  24,812,536  16,100,686 

    (1) Includes total costs (benefit) allocated from certain wholly owned subsidiaries of our affiliate, Roivant Sciences Ltd., of $0 for the three months ended March 31, 2020 and 2019 and $0 and $(450) for the years ended March 31, 2020 and 2019, respectively.

     (2) Includes total costs allocated from certain wholly owned subsidiaries of our affiliate, Roivant Sciences Ltd., of $44 and $126 for the three months ended March 31, 2020 and 2019, respectively, and $147 and $2,898 for the years ended March 31, 2020 and 2019, respectively.





    AXOVANT GENE THERAPIES LTD.

    Consolidated Balance Sheets

    (in thousands, except share and per share amounts)

     

     March 31, 2020
     March 31, 2019
        
    Assets   
    Current assets:   
    Cash and cash equivalents$80,752  $106,999 
    Prepaid expenses and other current assets2,971  5,859 
    Income tax receivable1,707  1,726 
    Total current assets85,430  114,584 
    Long-term investment5,871  5,871 
    Other non-current assets46  973 
    Operating lease right-of-use assets1,532   
    Property and equipment, net801  1,278 
    Total assets$93,680  $122,706 
    Liabilities and Shareholders' Equity   
    Current liabilities:   
    Accounts payable$4,412  $1,698 
    Accrued expenses11,319  20,619 
    Current portion of operating lease liabilities889   
    Current portion of long-term debt15,423  21,182 
    Total current liabilities32,043  43,499 
    Operating lease liabilities, net of current portion79   
    Long-term debt  22,994 
    Total liabilities32,122  66,493 
    Shareholders' equity:   
    Common shares, par value $0.00001 per share, 1,000,000,000 shares authorized, 39,526,299 and 22,779,891 issued and outstanding at March 31, 2020 and March 31, 2019, respectively   
    Accumulated other comprehensive (loss) income(55) 911 
    Additional paid-in capital820,257  741,318 
    Accumulated deficit(758,644) (686,016)
    Total shareholders' equity61,558  56,213 
    Total liabilities and shareholders' equity$93,680  $122,706 

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  3. -  Six-month data from low-dose cohort expected in Q4 2020

    -  IND amended to include Type I (infantile onset) patients and to evaluate a higher dose

    -  Expect to initiate high-dose cohort in 2H 2020

    NEW YORK and BASEL, Switzerland, June 08, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (NASDAQ:AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, today announced that it has completed enrollment in the low-dose cohort of the Phase 1/2 ("Stage 1") study for Type II (late infantile and juvenile onset) GM1 patients evaluating safety, tolerability, and exploratory measures of efficacy at a dose of 1.5x1013 vg/kg delivered intravenously. Currently, the study is on track to report 6-month data (n=5…

    -  Six-month data from low-dose cohort expected in Q4 2020



    -  IND amended to include Type I (infantile onset) patients and to evaluate a higher dose



    -  Expect to initiate high-dose cohort in 2H 2020

    NEW YORK and BASEL, Switzerland, June 08, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (NASDAQ:AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, today announced that it has completed enrollment in the low-dose cohort of the Phase 1/2 ("Stage 1") study for Type II (late infantile and juvenile onset) GM1 patients evaluating safety, tolerability, and exploratory measures of efficacy at a dose of 1.5x1013 vg/kg delivered intravenously. Currently, the study is on track to report 6-month data (n=5) from the low-dose cohort of the AXO-AAV-GM1 clinical trial by Q4 2020. Additionally, the investigational new drug (IND) filing has been amended to expand the program to include Type I (infantile) patients and to evaluate a 3-fold higher dose (4.5x1013 vg/kg). The Company expects to initiate dosing in the high-dose cohort, which will include both Type I and Type II patients, in the second half of 2020.

    "The successful enrollment of the low-dose cohort of the ongoing Phase 1/2 study amidst the COVID-19 pandemic speaks to the dedication of all involved and the significant unmet need that exists for these children," said Dr. Gavin Corcoran, chief R&D officer.  "With an expanded study protocol that now includes infantile-onset patients, AXO-AAV-GM1 is the only gene therapy in development to include both Type I and Type II GM1 patients, populations of children who suffer from a deficiency in the same enzyme, β-galactosidase. We are grateful for the collaboration and perseverance of the National Institutes of Health (NIH), patients, and their families to advance efforts towards finding a treatment for this devastating pediatric disease."

    This study is being conducted at the NIH under the direction of Dr. Cynthia Tifft, Deputy Clinical Director at the National Human Genome Research Institute (NHGRI) in collaboration with Axovant Gene Therapies. In late 2019, the Company presented an update from the first GM1 Type II child dosed with AXO-AAV-GM1 under an expanded access protocol who was observed to have clinically significant improvements from baseline gene transfer to six-month follow-up based on neurological exam, the Vineland-3 scale, Clinical Global Impression assessments, and nutritional status. In addition, AXO-AAV-GM1 was observed to be generally well-tolerated with no reports of serious adverse events related to the investigational gene therapy or intravenous administration of the vector.

    GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene leading to impaired production of the β-galactosidase enzyme. There are currently no approved treatments for GM1 gangliosidosis.

    AXO-AAV-GM1 was granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) in November 2019.

    About AXO-AAV-GM1

    AXO-AAV-GM1 is an investigational gene therapy that 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 broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1's ability to improve β-galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

    About Axovant Gene Therapies

    Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinson's disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit www.axovant.com.

    In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School (UMMS) for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases. A three-way Cooperative Research and Development Agreement (CRADA) among Axovant, the NHGRI, and the University of Massachusetts was established in 2019 to support the conduct of the clinical program.

    About the University of Massachusetts Medical School

    The mission of the University of Massachusetts Medical School is to advance the health and well-being of the people of the commonwealth and the world through pioneering education, research, public service and health care delivery.

    Research into potential therapies for lysosomal storage diseases such as Tay-Sachs, Sandhoff disease and GM1 gangliosidosis at UMass Medical School and Auburn University has led to significant advances in the field. Miguel Sena-Esteves, PhD, associate professor of neurology at UMass Medical School; Heather Gray-Edwards, PhD, DVM, formerly of Auburn and currently assistant professor of radiology at UMass Medical School; and Douglas Martin, PhD, professor of anatomy, physiology and pharmacology in the College of Veterinary Medicine and the Scott-Ritchey Research Center at Auburn University, have worked collaboratively for more than a decade on animal models and therapeutic approaches for these and similar disorders. For more information, visit www.umassmed.edu.                                                                  



    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 "may," "anticipate," "will," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the initiation, timing, progress, and reporting of clinical data for its clinical programs, are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovant's management that, although Axovant 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 Axovant expected. Such risks and uncertainties include, among others, the initiation and conduct of the AXO-AAV-GM1 program and the availability of data for disclosures; Axovant's scientific approach and general development, manufacturing and regulatory progress; Axovant's ability to perform under its existing clinical and business collaborations; and risks of unforeseen operational delays and other uncertainties caused by the COVID-19 pandemic. These statements are also subject to a number of material risks and uncertainties that are described in Axovant's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on February 10, 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. Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

    Media and Investor Contact:

    Parag Meswani

    Axovant Gene Therapies

    (212) 547-2523



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