AXO-Lenti-PD / Oxford Biomedica - LARVOL DELTA

Sio Gene Therapies Announces Fiscal Year 2021 Year-End Financial Results (GlobeNewswire) - "Sio Gene Therapies Inc...today provided financial results for its fiscal year ended March 31, 2022....$5.0 million in total milestone payments in the year ended March 31, 2022...AXO-Lenti-PD ($2.0 million) programs."

Commercial • CNS Disorders • Parkinson's Disease

SUNRISE-PD: Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease (clinicaltrials.gov) - P1/2 | N=6 | Terminated | Sponsor: Sio Gene Therapies | N=30 ➔ 6 | Trial completion date: Dec 2031 ➔ Apr 2022 | Active, not recruiting ➔ Terminated; As a result of termination of development of OXB-102-01, it is no longer possible for Sio to either commence or to continue any related clinical trials.

Enrollment change • Trial completion date • Trial termination • CNS Disorders • Movement Disorders • Parkinson's Disease

Sio Gene Therapies Announces Corporate Updates and Fiscal Third Quarter 2021 Financial Results (GlobeNewswire) - "Sio Gene Therapies Inc...today provided corporate updates and financial results for its fiscal third quarter ended December 31, 2021....Research and development expenses were $21.3 million for the three months ended December 31, 2021...increased AXO-Lenti-PD program expenses, primarily related to clinical trial material manufacturing expenses resulting from Qualified Person certification of GMP batches...As a result of our decision to terminate the AXO-Lenti-PD gene therapy program for the treatment of Parkinson's disease that is expected to become effective by March 31, 2022..."

Financing • Licensing / partnership • CNS Disorders • Parkinson's Disease

Sio Gene Therapies Announces Successful Manufacture of Three GMP Batches of AXO-Lenti-PD Gene Therapy for Parkinson’s Disease (GlobeNewswire) - "Sio Gene Therapies Inc...today provided a manufacturing and regulatory update for AXO-Lenti-PD, its clinical-stage gene therapy for Parkinson’s disease....Three GMP batches have successfully completed fill and finish, achieving target titers using the updated suspension-based process...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: Appropriate development pathway for completion of the Phase 1 dose-ranging study; Acceptability of a comparability protocol between the prior adherent and new suspension manufacturing process; New device administration system to support bilateral simultaneous infusions; Expect to provide a program update in Q1 2022"

Commercial • European regulatory • CNS Disorders • Gene Therapies • Parkinson's Disease

Sio Gene Therapies Announces Corporate Updates and Fiscal Second Quarter 2021 Financial Results (GlobeNewswire) - "Sio Gene Therapies Inc...today provided corporate updates and financial results for its fiscal second quarter ended September 30, 2021....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....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..."

Commercial • CNS Disorders • Gene Therapies • Parkinson's Disease

Sio Gene Therapies to Present New Data at the European Society of Gene and Cell Therapy Virtual Congress 2021 (GlobeNewswire) - "Sio Gene Therapies Inc...today announced that it will present new clinical and preclinical data in two oral presentations and one poster presentation at the upcoming European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021, to be held virtually from October 19-22, 2021....The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinson’s disease."

P1/2 data • CNS Disorders • Gene Therapies • Parkinson's Disease

[VIRTUAL] Phase 1/2 Open-label Dose Evaluation Study of AXO-Lenti-PD Gene Therapy for Parkinson's Disease: Efficacy, Safety, and Tolerability Data from the Second Cohort at 6 Months (MDS Congress 2021) - P1/2 | "Initial data of AXO-Lenti-PD gene therapy at 6M from cohort 2 in the SUNRISE-PD trial demonstrate that ALPD was generally well tolerated and suggest the potential for a clinically relevant effect. Further evaluation of ALPD is planned using a higher dose/volume open label cohort followed by a sham-controlled study."

Clinical • P1/2 data • CNS Disorders • Parkinson's Disease

Sio Gene Therapies Announces Corporate Updates and Fiscal First Quarter 2021 Financial Results (GlobeNewswire) - "AXO-Lenti-PD gene therapy for Parkinson’s disease: Two GMP batches have been manufactured using an updated suspension-based process and have now completed fill and finish. Sio is awaiting final testing of these batches to support certification of at least one batch for use as clinical trial material by a Qualified Person in Q4 2021. Pending review and approval of an IMPD amendment by the MHRA in the U.K., we expect to resume enrollment of patients in the AXO-Lenti-PD clinical program in 2022."

Commercial • Enrollment status • CNS Disorders • Parkinson's Disease

[VIRTUAL] A GLP Safety and Biodistribution Study of AXO-Lenti-PD Manufactured via Two Processes Delivered at a Higher Volume and Flow Rate (ASGCT 2021) - "There were no responses against the transgene components in any AXO-Lenti-PD-treated animal. Overall, the results of the study did not identify any LacZ- or AXO-Lenti-PD-related adverse effects associated with a higher volume and flow rate of infusion or any differences in the safety profile of adherent- or suspension-manufactured AXO-Lenti-PD material."

Clinical • CNS Disorders • Gene Therapies • Immunology • Inflammation • Mood Disorders • Movement Disorders • Ophthalmology • Parkinson's Disease

[VIRTUAL] AXO-Lenti-PD Gene Therapy for Parkinson’s Disease: Efficacy, Safety, and Tolerability Data from the Second Cohort in Open-Label Dose Evaluation Study SUNRISE-PD at 6 Months Post Administration (ASGCT 2021) - P1/2 | "The 6M data from the first two cohorts in SUNRISE-PD trial showed that ALPD gene therapy was generally well tolerated, and the initial efficacy data suggest the potential for a clinically relevant effect. Further evaluation of ALPD is planned using a higher dose/volume open label cohort followed by sham-controlled study."

Clinical • CNS Disorders • Depression • Gene Therapies • Immunology • Infectious Disease • Major Depressive Disorder • Mood Disorders • Movement Disorders • Novel Coronavirus Disease • Parkinson's Disease • Psychiatry

Sio Gene Therapies Announces Four Upcoming Oral Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy (GlobeNewswire) - "Sio Gene Therapies...today announced four upcoming oral presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), to be held virtually between May 11th to May 14th, 2021."

P2 data • CNS Disorders • Parkinson's Disease

Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease (clinicaltrials.gov) - P1/2; N=30; Active, not recruiting; Sponsor: Sio Gene Therapies; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • CNS Disorders • Movement Disorders • Parkinson's Disease • MRI

Sio Gene Therapies Announces Corporate Updates and Financial Results for Second Fiscal Quarter Ended September 30, 2020 (GlobeNewswire) - "For the second fiscal quarter ended September 30, 2020, research and development expenses were $5.1 million...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..."

Commercial • CNS Disorders • Parkinson's Disease

Axovant Gene Therapies Announces Name Change to Sio Gene Therapies (GlobeNewswire) - "Axovant Gene Therapies Ltd....today announced it will change its name to Sio Gene Therapies, Inc., effective November 13, 2020. In connection with the name change, the company’s ticker will change to 'SIOX' and will be effective at market open on November 13, 2020, along with the company’s new corporate website at www.siogtx.com. The former ticker 'AXGT' will remain effective through market close on November 12, 2020. SIOX will ring the Nasdaq Closing Bell at 4:00 pm ET on Monday, November 16, 2020."

Commercial • CNS Disorders • Parkinson's Disease

Axovant shares sink on manufacturing delay for Parkinson's gene therapy (BioPharma Dive) - "Axovant Gene Therapies doesn't expect to begin enrolling patients with Parkinson's disease in a planned Phase 2 study of the company's experimental gene therapy until 2022, a delay tied to a longer-than-expected timeline for developing a new manufacturing process....Shares in Axovant plunged by 40% Friday morning as investors and analysts reset their expectations for development progress for AXO-Lenti-PD. The company has so far treated six Parkinson's patients with two doses of its gene therapy."

Enrollment status • Stock price • CNS Disorders • Parkinson's Disease

Axovant Gene Therapies to Present AXO-Lenti-PD Program Update During Virtual Parkinson’s Disease R&D Day on October 30, 2020 (GlobeNewswire) - "Summary of available individual patient-data from the second dose cohort at the 6-month timepoint...Improvement in Hauser diary 'Good ON time' and 'OFF time' changes from baseline for all 4 patients; 23-point improvement from baseline to 12 months in UPDRS Part III 'OFF' score observed for the first patient in cohort 2 who has reached the 12-month evaluation...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....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....The Company expects to provide an update on program timelines in the first quarter of 2021 or as program timelines are clarified."

Enrollment status • P2 data • CNS Disorders • Parkinson's Disease

Axovant Gene Therapies to Host Virtual Parkinson’s Disease R&D Day on October 30, 2020 (GlobeNewswire) - P2, N=30; SUNRISE-PD (NCT03720418); Sponsor: Axovant Sciences Ltd; "Axovant Gene Therapies Ltd....today announced that it will host a virtual R&D Day on Friday, October 30, 2020...to discuss the Company's AXO-Lenti-PD gene therapy for Parkinson's disease. Axovant's Parkinson's disease R&D Day...will feature presentations on the current treatment landscape and unmet medical need for people living with Parkinson's disease from the following key opinion leaders: Charles Adler, M.D....The Company will present data from the second cohort of the Phase 2 SUNRISE-PD trial for AXO-Lenti-PD including: Summary of available data from the second dose cohort at the 6-month timepoint...12-month evaluation for the first patient treated in Cohort 2, who was assessed remotely."

P2 data • CNS Disorders • Parkinson's Disease

AXO-Lenti-PD: "AXO-Lenti-PD was well-tolerated in all patients dosed with no treatment-related serious adverse events (SAEs)"; Parkinson's disease (Axovant) - Corporate Presentation

P2 data • CNS Disorders • Parkinson's Disease

"It's actually n=19 (including ProSavin) with increasing responses with increasing dosage $AXGT" (@foreshadowd)

Axovant to Participate in Upcoming October Conferences and Provide AXO-Lenti-PD Program Update the Morning of October 6th (GlobeNewswire) - "Axovant Gene Therapies Ltd....today announced that the Company will present at upcoming conferences and will announce six-month safety and efficacy data from the second cohort of its AXO-Lenti-PD program the morning of Tuesday, October 6, 2020. Additionally, as previously announced the Company will hold a Parkinson’s Disease R&D Day on Friday, October 30."

P1/2 data • CNS Disorders • Parkinson's Disease

Axovant Announces Positive Six-Month Follow-Up Data From Second Cohort of SUNRISE-PD Phase 2 Trial of AXO-Lenti-PD Gene Therapy (GlobeNewswire) - P1/2, N=30; SUNRISE-PD (NCT03720418); Sponsor: Axovant Sciences Ltd; "AXO-Lenti-PD was well-tolerated with no treatment-related serious adverse events at 6 months; 21-point mean improvement in UPDRS Part III 'OFF' score, a 40% improvement from baseline....Greater than 2-hour improvement from baseline in both diary 'good ON time' and diary OFF time assessments....Company expects to investigate the safety and tolerability of a higher volume and flow rate to increase putaminal coverage and decrease operating room time (Cohort 3)....Axovant expects to dose the first patient in EXPLORE-PD in 2021. Additional program updates will be provided at Axovant’s Parkinson’s disease R&D Day on October 30, 2020."

New trial • P2 data • CNS Disorders • Parkinson's Disease

Axovant Announces First Parkinson's Disease Gene Therapy R&D Day and Participation in Upcoming September Conferences (GlobeNewswire) - "Axovant Gene Therapies Ltd...today announced that the company will provide a corporate overview and participate in 1:1 meetings at upcoming virtual investor and patient conferences in September. Additionally, the company announced plans for their 1st Parkinson’s Disease R&D Day with key opinion leaders."

Live event • CNS Disorders • Parkinson's Disease

Axovant Announces Corporate Updates and Financial Results for First Fiscal Quarter Ended June 30, 2020 (GlobeNewswire) - "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....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."

P2 data • Trial initiation date • CNS Disorders • Parkinson's Disease

Axovant signs gene therapy supply deal with Oxford Biomedica (SeekingAlpha) - "Axovant Gene Therapies Ltd. (NASDAQ:AXGT) inks a three-year agreement with Oxford Biomedica plc (OTCPK:OXBDF) to supply its AXO-Lenti-PD gene therapy, in phase 2 development for the treatment of Parkinson's disease."

Commercial • CNS Disorders • Parkinson's Disease

Axovant Signs Three-Year Clinical Supply Agreement With Oxford Biomedica for Manufacturing and Supply of AXO-Lenti-PD (GlobeNewswire) - "Axovant Gene Therapies Ltd...announced today that its subsidiary has signed a three-year Clinical Supply Agreement ('CSA') with Oxford Biomedica plc...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..."

Licensing / partnership • CNS Disorders • Parkinson's Disease