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 diseases
- Completed rebranding to Sio Gene Therapies as part of corporate transformation aligning corporate structure and governance with current and future business activity
- Company had
$63.2 million of cash and cash equivalents as ofSeptember 30, 2020 , with sufficient cash runway into Q4 2021
“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
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 theMichael 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 months
- Greater than 2-hour improvement from baseline in both diary “good ON time” and diary OFF time assessments observed across all four patients in Cohort 2
- Reported 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, 2020 - Appointment of
Kristiina Vuori , M.D., Ph.D, as a new director, establishing a majority independent Board of Directors - No longer being a majority-owned and controlled public company
- Completed redomiciliation to
Delaware
- 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
- 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
General and administrative expenses for the second fiscal quarter ended
The net loss for the second fiscal quarter ended
Fiscal First-Half Financial Summary
For the six months ended
General and administrative expenses for the six months ended
The net loss for the six months ended
As of
On
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
Contacts:
Media
(646) 751-4361
jo@lifescicomms.com
info@siogtx.com
Investors and Analysts
Chief Financial Officer and General Counsel
(646) 677-6770
investors@siogtx.com
SIO GENE THERAPIES INC.
Condensed Consolidated Statements of Operations
(Unaudited, in thousands, except share and per share amounts)
Three Months Ended |
Six Months Ended |
||||||||||||||
2020 | 2019 | 2020 | 2019 | ||||||||||||
Operating expenses: | |||||||||||||||
Research and development expenses | |||||||||||||||
(includes stock-based compensation expense of |
$ | 5,058 | $ | 6,833 | $ | 10,252 | $ | 27,923 | |||||||
General and administrative expenses | |||||||||||||||
(includes stock-based compensation expense of |
4,491 | 5,051 | 9,131 | 11,519 | |||||||||||
Total operating expenses | 9,549 | 11,884 | 19,383 | 39,442 | |||||||||||
Other (income) expenses: | |||||||||||||||
Interest expense | 1 | 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 diluted | 46,731,666 | 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)
Assets | |||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 63,171 | $ | 80,752 | |||
Prepaid expenses and other current assets | 5,406 | 2,971 | |||||
Income tax receivable | 1,747 | 1,707 | |||||
Total current assets | 70,324 | 85,430 | |||||
Long-term investment | 8,055 | 5,871 | |||||
Other non-current assets | 169 | 46 | |||||
Operating lease right-of-use assets | 663 | 1,532 | |||||
Property and equipment, net | 560 | 801 | |||||
Total assets | $ | 79,771 | $ | 93,680 | |||
Liabilities and Shareholders’ Equity | |||||||
Current liabilities: | |||||||
Accounts payable | $ | 2,172 | $ | 4,412 | |||
Accrued expenses | 7,837 | 11,319 | |||||
Current portion of operating lease liabilities | 34 | 889 | |||||
Current portion of long-term debt | — | 15,423 | |||||
Total current liabilities | 10,043 | 32,043 | |||||
Operating lease liabilities, net of current portion | 55 | 79 | |||||
Total liabilities | 10,098 | 32,122 | |||||
Stockholders’ equity: | |||||||
Common stock, par value |
— | — | |||||
Additional paid-in capital | 846,558 | 820,257 | |||||
Accumulated deficit | (777,222 | ) | (758,644 | ) | |||
Accumulated other comprehensive loss | 337 | (55 | ) | ||||
Total stockholders’ equity | 69,673 | 61,558 | |||||
Total liabilities and stockholders’ equity | $ | 79,771 | $ | 93,680 |