Taysha Gene Therapies Reports Second Quarter 2021 Financial Results and Provides Corporate Update
Taysha Gene Therapies, Inc. (Nasdaq: TSHA) reported its Q2 2021 financial results, showcasing significant advancements in developing gene therapies for CNS diseases. A non-dilutive financing of up to $100 million was secured, enhancing cash reserves to $197.4 million. Positive feedback from regulators for multiple programs allows for anticipated IND filings later this year. R&D expenses increased to $30.6 million, and net loss was $40.9 million. Upcoming milestones include clinical trial data releases and regulatory updates, reflecting a robust pipeline.
- Secured $100 million non-dilutive financing to strengthen cash position.
- Positive pre-IND/CTA feedback from regulatory agencies for multiple programs.
- Completion of successful GMP runs to support five planned Phase 1/2 trials.
- Publication of positive preclinical data for multiple therapies.
- R&D expenses rose significantly to $30.6 million, indicating high operational costs.
- Net loss increased to $40.9 million, reflecting challenges in achieving profitability.
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today reported financial results for the second quarter ended June 30, 2021 and provided a corporate update.
“We continue to make significant progress on several key clinical, manufacturing and strategic corporate initiatives which were highlighted in our R&D and Manufacturing investor days and in our recent press release announcing our non-dilutive financing with SVB,” noted RA Session II, President, Founder and CEO of Taysha. “We have received positive pre-IND/CTA feedback from several key regulatory agencies for our Rett syndrome, GM2 gangliosidosis and CLN1 disease programs, which pave the way for multiple expected IND/CTA filings and clinical trial initiations in the second half of the year. Our success relies on robust, sustainable and high-quality manufacturing to support our portfolio, and we have completed numerous successful GMP runs to support five planned Phase 1/2 trials and several programs in IND/CTA-enabling studies. With ambitious corporate objectives planned over the next 12 to 18 months, we are pleased to have recently entered into a loan agreement for up to
Recent Corporate Highlights
- Positive pre-IND/CTA feedback obtained from multiple key regulatory agencies for TSHA-102 in Rett syndrome, TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1 disease, paving the way for multiple expected IND/CTA filings in the second half of 2021
-
Secured up to
$100 million non-dilutive term loan financing, with$40 million available at closing from Silicon Valley Bank (SVB), of which Taysha has drawn$30 million . The company has the option to draw down the remaining tranches, subject to certain conditions. The interest rate is the greater of7.0% or the WSJ Prime Rate plus3.75% and there are no financial covenants or warrants - Multiple successful GMP runs provided drug products to support five planned Phase 1/2 trials and multiple programs in IND/CTA-enabling studies in the second half of 2021
- Publication of GAN natural history data for TSHA-120 in peer-reviewed journal, Brain, that included robust assessment across clinical outcomes for GAN, including motor, sensory, respiratory, neurophysiologic, MRI and biopsy data. All GAN natural history data was generated and supported by the Intramural Program of the National Institute of Neurological Disorders and Stroke (NINDS), U.S. National Institutes of Health (NIH) under umbrella protocol NCT01568658 and under the leadership of the Principal Investigator, Carsten Bönneman
- Publication of positive preclinical data for TSHA-102 in Rett syndrome in Brain that provided quantitative evidence of miRARE’s ability to exhibit genotype-dependent regulation of MECP2 gene expression on a cell-by-cell basis across different regions of the brain in both wild type and knockout mouse models of Rett syndrome
-
Announcement of positive data from multiple programs at R&D Day on June 28-29
- Phase 1/2 visual acuity data for TSHA-120 in patients with GAN demonstrated a dose-dependent trend towards stabilization of visual acuity, one of the most challenging symptoms to treat. Recently obtained preclinical data demonstrated no impact on dorsal root ganglia in non-human primates when TSHA-120 was delivered intrathecally
- Preclinical data for TSHA-102 in Rett syndrome demonstrated that miRARE reduced overall expression of MECP2 transgene expression compared to unregulated MECP2 in wild type mice. miRARE regulated genotype-dependent MECP2 expression across different brain regions in wild type and Rett knockout mouse models on a cell-by-cell basis
- An in-depth look into natural history provided incremental data on disease progression and motor development delays in patients with GM2 gangliosidosis
- Preclinical data for TSHA-118 demonstrated sustained preservation of motor function and rescue of survival with higher doses of TSHA-118 and earlier intervention in CLN1 knockout mice
- Positive findings indicated a small increase in COX1 activity can significantly improve the clinical phenotype of patients with SURF1-associated Leigh syndrome; similarly, reduction in COX activity correlated with disease worsening in patient fibroblast data
- In SLC13A5 knockout mice, TSHA-105 decreased plasma citrate levels and reduced seizures and associated deaths
- In SLC6A1 knockout and heterozygous mouse models, CNS administration of TSHA-103 rescued abnormal seizure activity. Recently obtained data demonstrated rescue of functional measures such as nesting, open field activity, hind limb clasping and latency to fall from the rotarod
- TSHA-112 reduced GYS1 expression in the APBD knockout model which resulted in decreased polyglucosan body formation in mice brain
- TSHA-111-LAFORIN and TSHA-111-MALIN reduced GYS1 expression in the laforin and malin knockout models which resulted in decreased Lafora body formation in mice brain
- Preclinical data for TSHA-113 demonstrated significant reduction of tau mRNA and protein levels, validating the potential of using AAV-mediated gene silencing to achieve life-long reduction of tau protein levels and warranting further preclinical development
- Two novel approaches to treat Angelman syndrome disclosed, including knockdown of UBE3A-ATS to unsilenced the paternal allele and a gene replacement strategy on UBE3A to mimic maternal UBE3A allele expression
- Manufacturing Day on July 27th highlighted the company’s unique three-pillar approach to the manufacturing process, its manufacturing capabilities, the regulatory environment for gene therapy manufacturing, and the immunology of gene therapy with a focus on the practical aspects of initiating and monitoring immunosuppressive regimes
- Appointment of Chief Development Officer, Mary Newman, who previously served as Senior Vice President of Regulatory Affairs at Astellas Gene Therapies, formerly Audentes, and brings over 30 years of experience in translational development, program management and regulatory affairs in the biotech industry
- Founding member of the newly formed Rare Disease Company Coalition, a first-of-its-kind alignment of life sciences companies committed to discovering, developing and delivering rare disease treatments for the patients they collectively serve
- Continued progress on internal manufacturing facility with completion of the demolition phase
- Opened the Dallas headquarters in May 2021
- Expansion of employee base to 150 across all areas of the organization
Anticipated Milestones by Program
TSHA-120 for giant axonal neuropathy (GAN): an intrathecally dosed AAV9 gene therapy currently being evaluated in a clinical trial for the treatment of GAN, a rare inherited genetic disorder that affects both the central and peripheral nervous systems and is caused by loss-of-function mutations in the gene coding for gigaxonin
- Report clinical data for TSHA-120 from the high dose cohort of 3.5x1014 total vg in second half of 2021
- Engage with key regulatory agencies to discuss the approval pathway and provide a regulatory update by year-end 2021
TSHA-101 for GM2 gangliosidosis: the first bicistronic gene therapy in clinical development designed to deliver two genes – HEXA and HEXB, comprising the alpha and beta sub-units of Beta Hexoseaminidase A, intrathecally for the treatment of GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease
- Report preliminary Phase 1/2 safety and biomarker data (Queen’s University trial) in second half of 2021
- Submit an Investigational New Drug (IND) application in the U.S. in second half of 2021
- Initiate Phase 1/2 clinical trial in the U.S. in second half of 2021
TSHA-118 in CLN1: a self-complementary AAV9 viral vector designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1, a rapidly progressing rare lysosomal storage disease with no approved treatments
- CLN1 Investor Day on August 30, 2021
- Maintain current open IND
- Initiate a Phase 1/2 clinical trial in second half of 2021
- Report biomarker data in first half of 2022
TSHA-102 in Rett syndrome: a self-complementary AAV9 gene therapy in development for a severe neurodevelopmental disorder, designed to deliver MECP2, as well as a novel miRARE platform that regulates transgene expression on a cell-by-cell basis
- Rett syndrome Investor Day in September 2021
- Submit IND/CTA filing in second half of 2021
- Initiate Phase 1/2 clinical trial by year-end 2021
- Report clinical data by year-end 2022
TSHA-104 in SURF1-associated Leigh syndrome: a self-complementary AAV9 viral vector with a transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no approved treatments
- Submit IND/CTA filing in second half of 2021
- Initiate Phase 1/2 trial by year-end 2021
- Report biomarker data in first half of 2022
Pipeline programs in IND/CTA-enabling studies
- Submit an IND/CTA filing for one of six programs in 2021: TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN and TSHA-111-MALIN in two forms of Lafora disease, TSHA-112 in APBD, TSHA-119 in GM2 AB variant and TSHA-103 in SLC6A1 haploinsufficiency disorder
Discovery programs
- Advance four new undisclosed programs focused on neurodevelopmental disorders, genetic epilepsies and neurodegenerative diseases into preclinical development in 2021
Next-generation technology platform
- Continue development efforts focused on regulated transgene expression with expansion of miRARE platform into additional CNS diseases
- Initiate confirmatory preclinical studies for the vagus nerve redosing platform in canines
- Advance mini-gene discovery program in genetic forms of epilepsy and neurodevelopmental disorders
- Continue discovery and development efforts around next-generation capsids
Second Quarter 2021 Financial Highlights
Research and Development (R&D) Expenses: Research and development expenses were
General and Administrative (G&A) Expenses: General and administrative expenses were
Net loss: Net loss for the second quarter ended June 30, 2021 was
Cash and cash equivalents: As of June 30, 2021, Taysha had
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today at 8:00 a.m. ET / 7:00 a.m. CT to review its financial and operating results and to provide a corporate update. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13722197. The live webcast and replay may be accessed by visiting Taysha’s website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days.
About Taysha Gene Therapies
Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team’s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients’ lives. More information is available at www.tayshagtx.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, the potential market opportunity for these product candidates, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply and our anticipated cash runway assuming full access to the
Taysha Gene Therapies, Inc. Consolidated Statements of Operations (in thousands, except share and per share data) (Unaudited) |
||||||||||||||||
| For the Three Months Ended June 30, |
For the Six Months Ended June 30, |
|||||||||||||||
2021 |
2020 |
2021 |
2020 |
|||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ |
30,643 |
|
$ |
3,062 |
|
$ |
54,497 |
|
$ |
8,576 |
|
||||
| General and administrative |
|
10,129 |
|
|
948 |
|
|
18,365 |
|
|
1,018 |
|
||||
| Total operating expenses |
|
40,772 |
|
|
4,010 |
|
|
72,862 |
|
|
9,594 |
|
||||
| Loss from operations |
|
(40,772 |
) |
|
(4,010 |
) |
|
(72,862 |
) |
|
(9,594 |
) |
||||
| Other income (expense): | ||||||||||||||||
| Change in fair value of preferred stock tranche liability |
|
- |
|
|
(17,210 |
) |
|
- |
|
|
(17,030 |
) |
||||
| Interest income |
|
40 |
|
|
- |
|
|
106 |
|
|
- |
|
||||
| Interest expense |
|
(194 |
) |
|
- |
|
|
(194 |
) |
|
(27 |
) |
||||
| Total other expense, net |
|
(154 |
) |
|
(17,210 |
) |
|
(88 |
) |
|
(17,057 |
) |
||||
| Net loss | $ |
(40,926 |
) |
$ |
(21,220 |
) |
$ |
(72,950 |
) |
$ |
(26,651 |
) |
||||
| Net loss per common share, basic and diluted | $ |
(1.09 |
) |
$ |
(1.95 |
) |
$ |
(1.96 |
) |
$ |
(2.45 |
) |
||||
| Weighted average common shares outstanding, basic and diluted |
|
37,479,164 |
|
|
10,894,999 |
|
|
37,237,115 |
|
|
10,894,999 |
|
||||
Taysha Gene Therapies, Inc. Consolidated Balance Sheet Data (in thousands, except share and per share data) (Unaudited) |
||||||||
| June 30, 2021 |
December 31, 2020 |
|||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ |
197,370 |
|
$ |
251,253 |
|
||
| Prepaid expenses and other current assets |
|
9,932 |
|
|
6,626 |
|
||
| Total current assets |
|
207,302 |
|
|
257,879 |
|
||
| Deferred lease asset |
|
691 |
|
|
715 |
|
||
| Property, plant and equipment, net |
|
33,867 |
|
|
287 |
|
||
| Total assets | $ |
241,860 |
|
$ |
258,881 |
|
||
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ |
6,355 |
|
$ |
1,994 |
|
||
| Accrued expenses and other current liabilities |
|
22,036 |
|
|
5,135 |
|
||
| Total current liabilities |
|
28,391 |
|
|
7,129 |
|
||
| Build-to-suit lease liability |
|
26,209 |
|
|
- |
|
||
| Other non-current liabilities |
|
765 |
|
|
450 |
|
||
| Total liabilities |
|
55,365 |
|
|
7,579 |
|
||
| Stockholders' equity | ||||||||
| Preferred stock, authorized and no shares issued and outstanding as of June 30, 2021 and December 31, 2020 |
|
- |
|
|
- |
|
||
| Common stock, authorized and 38,391,165 and 37,761,435 issued and outstanding as of June 30, 2021 and December 31, 2020, respectively |
|
- |
|
|
- |
|
||
| Additional paid-in capital |
|
320,571 |
|
|
312,428 |
|
||
| Accumulated deficit |
|
(134,076 |
) |
|
(61,126 |
) |
||
| Total stockholders’ equity |
|
186,495 |
|
|
251,302 |
|
||
| Total liabilities and stockholders' equity | $ |
241,860 |
|
$ |
258,881 |
|
||
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FAQ
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