TSHA-120 program demonstrated clear arrest of
disease progression and long-term durability at therapeutic dose
levels in patients with giant axonal neuropathy (GAN); Expects
clinical data from highest dose cohort in second half of 2021 and
regulatory feedback from agencies by year-end 2021
Expects to have five programs in Phase 1/2
trials in the second half of 2021, including GAN, GM2
gangliosidosis, CLN1 disease, Rett syndrome and SURF1-associated
Leigh syndrome
Positive preclinical data for TSHA-102 in Rett
syndrome provided quantitative evidence of miRARE’s ability to
exhibit genotype-dependent regulation of MECP2 gene expression
across different brain regions in both wild type and knockout mouse
models of Rett syndrome; Results published in Brain
Treatment with TSHA-102 resulted in a
statistically significant survival extension by 56% in 4-5 week-old
knockout Rett mice with meaningful accumulated disease, a more
translatable model of the disorder in humans
New data for multiple preclinical programs,
including tauopathies, SLC13A5 deficiency, SLC6A1
haploinsufficiency, Angelman disease, Adult Polyglucosan Body
Disease (APBD), Lafora disease, and GM2 AB variant, highlighted
Taysha’s next wave of novel gene therapies that have the potential
to impact meaningful patient populations
Plans for IND/CTA submission from one of the
following programs by year-end 2021: SLC13A5 deficiency, APBD,
Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency
Advancing development of multiple preclinical
programs, including tauopathies and Angelman syndrome
Virtual Research and Development Day on June
28-29, 2021 to feature Key Opinion Leaders and highlight progress
across pipeline
Conference call and webcast today at 8:00 AM
Eastern Time
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 first quarter ended March 31, 2021 and provided a corporate
update.
“Our team has ushered in the new year with a continued focus on
achieving our corporate objectives and creating value for patients
and shareholders,” said RA Session II, President, Founder and CEO
of Taysha. “Our recent acquisition of TSHA-120 for GAN immediately
transformed Taysha into a pivotal-stage gene therapy company. Based
on the compelling clinical and preclinical data package generated
to date for this promising product candidate, we intend to engage
with regulatory agencies to discuss a pathway to approval and look
forward to providing clinical and regulatory updates in the second
half of 2021 and by year-end 2021, respectively. We are also
extremely pleased to share newly published preclinical data for
TSHA-102 in Rett syndrome that provided, for the first time,
quantitative evidence of miRARE’s ability to show
genotype-dependent regulation of MECP2 gene expression across
different regions of the brain in wild type and knockout mouse
models. Rett syndrome is an incredibly difficult disease to treat
with gene replacement therapy given the challenges of safely
regulating the degree of MECP2 expression from the MECP2 gene and
we are encouraged that miRARE has achieved this regulation on a
cell-by-cell basis without associated toxicities. The built-in
self-regulatory feedback loop mechanism is a culmination of
approximately 14 years of research and holds great potential for
the treatment of what we now consider a reversible disease.
Importantly, TSHA-102-treated knockout Rett mice with meaningful
disease accumulation experienced a statistically significant
survival extension by 56%, which we believe is a more translatable
model of the disorder in humans. We believe these data validate our
novel approach to treating Rett syndrome, help de-risk the clinical
program and support advancement of TSHA-102 into a Phase 1/2 trial
by year-end. With these and other recent value-creating
achievements, we are even more confident in our outlook for the
full year.”
Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical
Officer and Head of Research and Development of Taysha, said, “We
expect a steady flow of near-term clinical, regulatory and
preclinical catalysts for the remainder of 2021. We anticipate
having five programs in Phase 1/2 trials and an additional six
programs in IND/CTA-enabling studies by year-end 2021. For the
remainder of 2021, we expect data from the highest dose cohort from
the ongoing TSHA-120 trial for GAN, a regulatory update on the GAN
program, first-in-human Phase 1/2 clinical data from the Queen’s
University trial of TSHA-101 in GM2 gangliosidosis and the
initiation of Phase 1/2 trials in GM2 gangliosidosis in the U.S.,
CLN1 disease, Rett syndrome and SURF1-associated Leigh syndrome.
For our preclinical programs, we expect to submit an IND/CTA for
one of six programs in IND/CTA-enabling studies by year-end 2021.
Moreover, we will continue to make advancements on payload design
and on the miRARE, mini-gene and vagus nerve redosing platforms,
which are expected to drive further innovation. We look forward to
providing additional updates at our two-day R&D Day event in
June and throughout the year.”
Recent Corporate Highlights
- Published new preclinical data for TSHA-102 in Rett syndrome in
Brain journal
- Preclinical data provided quantitative evidence of miRARE’s
ability to exhibit genotype-dependent regulation of MECP2 gene
expression across different brain regions in both wild type and
knockout mouse models of Rett syndrome
- TSHA-102 resulted in a statistically significant survival
extension by 56%, whereas unregulated constructs did not extend
survival significantly in the validated MECP2 knockout Rett mouse
model
- Benefit in 4-5 week-old TSHA-102-treated knockout Rett mice
with meaningful accumulated disease should be a more translatable
model to humans
- In the pons and midbrain, miRARE inhibited mean expression in a
genotype-dependent manner, as indicated by significantly fewer
myc(+) cells observed in wild type mice than knockout mice
(p<0.05), thereby demonstrating MECP2 levels within normal
physiological parameters
- These quantitative data, for the first time, demonstrated
miRARE’s ability to regulate gene expression on a cell-by-cell
basis, highlighting its potential application in numerous diseases
that require controlled gene expression
- Acquired exclusive worldwide rights to TSHA-120, a
clinical-stage AAV9 gene therapy program for the treatment of GAN
- Human proof-of-concept data for TSHA-120 demonstrated clear
arrest of disease progression and long-term durability at
therapeutic dose levels in patients with GAN
- To date, 14 patients have been dosed with one of four dose
levels of TSHA-120. TSHA-120 has demonstrated a dose-response
relationship with arrest of disease progression at the
second-highest dose level (1.8x1014 total vector genomes [vg]) at
one-year post-treatment, affecting a statistically significant
8-point improvement on the MFM32 score
- Six of these patients treated at therapeutic dose levels have
shown sustained dose-dependent improvements in MFM32 scores for
more than three years
- Bayesian analyses confirmed nearly 100% probability of
clinically meaningful slowing of disease in patients dosed at
1.8x1014 total vg compared to natural history
- Long-term results demonstrated that treatment with TSHA-120 at
multiple dose levels was well-tolerated with no severe drug-related
adverse events
- Reported new preclinical data for TSHA-113 for tauopathies,
TSHA-105 for SLC13A5 deficiency, TSHA-103 for SLC6A1
haploinsufficiency, TSHA-106 for Angelman syndrome, TSHA-112 for
APBD, TSHA-111-LAFORIN and TSHA-111-MALIN for Lafora disease, and
TSHA-119 for GM2 AB variant that support advancement into clinical
testing
- TSHA-113 significantly reduced tau mRNA and protein levels in
mouse models of human tauopathies via cerebral spinal fluid (CSF)
delivery
- TSHA-105 significantly reduced plasma citrate levels,
normalized EEG brain activity, and reduced the number of seizures
and seizure susceptibility in SLC13A5 knockout mice
- TSHA-103 improved nesting and EEG activity in the SLC6A1
knockout mouse model and reduced spike train activity in SLC6A1
knockout and heterozygous mouse models
- TSHA-106 increased UBE3A expression through shRNA-mediated
knockdown of UBE3A-ATS in in vitro cell lines across 26 distinct
shRNA candidates for the treatment of Angelman disease
- TSHA-112 generated significant reductions in GYS1 protein,
abnormal glycogen accumulation and polyglucosan bodies in the APBD
knockout mouse model
- TSHA-111-LAFORIN and TSHA-111-MALIN achieved effective
knockdown of GYS1 expression and insoluble glycogen and decreased
Lafora body formation in laforin and malin mouse models
- TSHA-119 caused a dose-dependent reduction of GM2 accumulation
at 20 weeks in GM2A knockout mice
- Announced presentations of preclinical data for TSHA-104 in
SURF1-associated Leigh syndrome and TSHA-105 in SLC13A5 deficiency
at the 24th Annual Meeting of the American Society of Gene &
Cell Therapy (ASGCT)
- TSHA-104 increased COX1 activity in brain and muscle and
restored elevation of blood lactate on exhaustive exercise in a
dose-dependent manner in SURF1 knockout mice
- TSHA-105 significantly reduced plasma citrate levels,
normalized EEG brain activity, and reduced the number of seizures
and seizure susceptibility in SLC13A5 knockout mice
- Initiated construction of its internal 187,000-square-foot
current Good Manufacturing Practices (cGMP) manufacturing facility
in Durham, North Carolina, that will include multiple production
suites designed to have a total capacity of 2,000 liters for
preclinical, clinical, and commercial production of Taysha’s gene
therapy pipeline; facility will include development, analytical,
manufacturing and quality control testing capability for its broad
portfolio of gene therapies
- Established collaboration with Yale University, a key addition
to partnerships with Cleveland Clinic and UTSW, to advance
next-generation mini-gene payloads for AAV gene therapies for the
treatment of genetic epilepsies and neurodevelopmental
disorders
- Grew company from 80 to approximately 120 employees between
February and April 2021
- Announced a two-day virtual R&D Day on June 28th and June
29th to feature Key Opinion Leaders and highlight progress across
the pipeline
First Quarter 2021 Financial Highlights
Research and Development (R&D) Expenses: R&D
expenses were $23.9 million for the first quarter ended March 31,
2021, compared to $5.5 million for the first quarter ended March
31, 2020. The increase was primarily related to the company’s
development programs, as a result of increased
manufacturing-related spend, clinical and preclinical activities,
and headcount.
General and Administrative (G&A) Expenses: G&A
expenses were $8.2 million for the first quarter ended March 31,
2021, compared to less than $0.1 million for the first quarter
ended March 31, 2020. The increase was primarily due to an increase
in personnel costs resulting from increased headcount, professional
services fees, and other corporate-related expenses.
Net loss: Net loss for the first quarter ended March 31,
2021 was $32.0 million, or $0.87 per share, as compared to a net
loss of $5.4 million, or $0.50 per share, for the first quarter
ended March 31, 2020.
Cash and cash equivalents: As of March 31, 2021, Taysha
had $228.7 million in cash and cash equivalents, which is expected
to support planned operations into 2023.
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 3.5x1014 total vg
dose cohort in the second half of 2021
- Engage with major 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 the second half of 2021
- Submit an Investigational New Drug (IND) application in the
U.S. in the second half of 2021
- Initiate Phase 1/2 clinical trial in the U.S. in the 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
- Maintain current open IND
- Initiate a Phase 1/2 clinical trial in the second half of
2021
- Report biomarker data in the 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
- Submit IND/CTA filing in the 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 the second half of 2021
- Initiate Phase 1/2 trial by year-end 2021
- Report biomarker data in the 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
Anticipated Corporate Milestones in 2021
- Continue construction of internal cGMP facility in 2021
- Complete buildout of Dallas headquarters in Q2 2021
- Expand employee base from approximately 120 (as of April 30,
2021) to approximately 150 by year-end 2021
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today
at 8:00 am ET / 7:00 am CT to review its financial and operating
results and to provide a corporate update. The dial-in number for
the conference call is 855-327-6837 (U.S./Canada) or 631-891-4304
(international). The conference ID for all callers is 10014460. 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,” 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. Forward-looking
statements are based on management’s current expectations and are
subject to various risks and uncertainties that could cause actual
results to differ materially and adversely from those expressed or
implied by such forward-looking statements. Accordingly, these
forward-looking statements do not constitute guarantees of future
performance, and you are cautioned not to place undue reliance on
these forward-looking statements. Risks regarding our business are
described in detail in our Securities and Exchange Commission
(“SEC”) filings, including in our Annual Report on Form 10-K for
the full-year ended December 31, 2020, which is available on the
SEC’s website at www.sec.gov. Additional information will be made
available in other filings that we make from time to time with the
SEC. Such risks may be amplified by the impacts of the COVID-19
pandemic. These forward-looking statements speak only as of the
date hereof, and we disclaim any obligation to update these
statements except as may be required by law.
Taysha Gene Therapies,
Inc.
Consolidated Statements of
Operations
(in thousands, except share and
per share data)
(Unaudited)
For the Three Months Ended
March 31, 2021
For the Three Months Ended
March 31, 2020
Operating expenses: Research and development
$
23,854
$
5,514
General and administrative
8,236
70
Total operating expenses
32,090
5,584
Loss from operations
(32,090
)
(5,584
)
Other income (expense): Change in fair value of preferred
stock tranche liability
-
180
Interest income
66
-
Interest expense
-
(27
)
Total other income, net
66
153
Net loss
$
(32,024
)
$
(5,431
)
Net loss per common share, basic and diluted
$
(0.87
)
$
(0.50
)
Weighted average common shares outstanding, basic and diluted
36,992,377
10,894,999
Taysha Gene Therapies,
Inc.
Consolidated Balance Sheet
Data
(in thousands)
(Unaudited)
March 31,
December 31,
2021
2020
Cash and cash equivalents
$
228,684
$
251,253
Total assets
$
242,829
$
258,881
Total liabilities
$
19,957
$
7,579
Total stockholders’ equity
$
222,872
$
251,302
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210511005453/en/
Company Contact: Kimberly Lee, D.O. SVP, Corporate
Communications and Investor Relations Taysha Gene Therapies
klee@tayshagtx.com Media Contact: Carolyn Hawley Canale
Communications carolyn.hawley@canalecomm.com
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