Axovant Gene Therapies Ltd (NASDAQ: AXGT), a clinical-stage company
developing innovative gene therapies, today provided financial
results and corporate updates for its second fiscal quarter ended
September 30, 2019.
“Presenting early evidence of disease
stabilization in children with Tay-Sachs was a significant
accomplishment for Axovant and our academic collaborators, and
suggests an opportunity for disease modification with gene
replacement therapy in monogenic pediatric lysosomal storage
disorders such as GM1 and GM2 gangliosidoses,” said Pavan Cheruvu
M.D., Chief Executive Officer of Axovant. “This year, we achieved
our goal of commencing dosing across all three gene therapy
programs in our portfolio. We remain committed to delivering
transformative gene therapies to patients with devastating
neurologic diseases, and look forward to a productive 2020 during
which we expect to provide several meaningful updates across our
programs, including providing safety and efficacy data from the
second dose cohort in Parkinson’s disease and Part A data at
6-months from our ongoing registrational study in GM1
gangliosidosis.”
Key Highlights and Development
Updates
- AXO-AAV-GM2: Presented first evidence of
clinical disease stabilization in two patients dosed with
AXO-AAV-GM2 at the European Society of Gene and Cell Therapy
(ESGCT) 27th Annual Congress in October 2019. Initial 3-month
follow-up data from a child with early symptomatic Tay-Sachs
disease treated at 6-months of age suggests a pattern of stable
CHOP-INTEND scores, an improvement from baseline in myelination on
brain MRI, and an increase in enzyme activity in CSF. The treatment
was generally observed to be well-tolerated in both patients with
no serious adverse events related to therapy.
- AXO-AAV-GM1: The first two patients in the
AXO-AAV-GM1 Phase 1/2 program were dosed intravenously earlier this
year, and the therapy has been generally well tolerated to date
with no serious adverse events attributed to the therapy. Axovant
expects to present an update from the first child dosed with
AXO-AAV-GM1 gene therapy in Q4 2019, and data from Part A (n=5),
which is expected to focus on safety and tolerability as well as
exploratory measures of efficacy after 6 months of follow-up, is
expected in mid-2020. Axovant also announced plans to expand the
clinical development plan for AXO-AAV-GM1 to include infantile GM1
gangliosidosis subjects (Type I), the most severely affected
population with the disease.
- AXO-Lenti-PD: Axovant expects to present
3-month data from the second dose cohort (n=4) of the SUNRISE-PD
Phase 2 study of AXO-Lenti-PD in the first half of calendar year
2020, allowing for an evaluation of safety, tolerability, and
various measures of clinical efficacy in Parkinson’s disease.
Axovant is actively initiating new clinical sites to expedite
patient enrollment by expanding neurosurgical capacity for current
and future studies. Based on the outcome of dose-escalation
studies, Axovant expects to initiate a randomized, sham-controlled
study of AXO-Lenti-PD by the end of calendar year 2020.
Fiscal Second Quarter Financial
Summary
Research and development expenses were $6.8 million for the
three months ended September 30, 2019 compared to $21.5
million for the three months ended September 30, 2018. Excluding a
$10.0 million upfront license fee paid in the prior year period to
Benitec Biopharma Limited ("Benitec"), research and development
expenses decreased by $4.7 million in the current year period,
primarily due to the discontinuation of our small molecule drug
programs.
General and administrative expenses decreased by $5.5 million
from $10.6 million for the three months ended September 30,
2018 to $5.1 million in the three months ended September 30, 2019,
primarily due to reductions in (i) share-based compensation expense
of $3.1 million, attributable to reduced headcount, (ii) legal and
accounting fees of $0.8 million, (iii) costs allocated under our
services agreements with Roivant Sciences, Inc. ("RSI") and Roivant
Sciences GmbH ("RSG") of $0.7 million as a result of the
decentralization of the services provided to us, and (iv) a
reduction in marketing costs of $0.6 million. Going forward, the
costs allocated to us under our services agreements with RSI and
RSG are expected to continue to be insignificant.
Net loss for the three months ended September 30, 2019 was
$13.9 million, or $0.61 per share, based on a weighted-average of
22.8 million common shares outstanding, compared to a net loss of
$33.8 million, or $2.24 per share, based on a weighted-average of
15.1 million common shares outstanding for the three months ended
September 30, 2018.
Fiscal First-Half Financial
Summary
Research and development expenses were $27.9 million for the six
months ended September 30, 2019 compared to $58.9 million for
the six months ended September 30, 2018. Excluding a net
amount of $13.0 million due to Oxford BioMedica (UK) Ltd.
("Oxford") for a development milestone achieved in the current year
period for AXO-Lenti-PD, as well as upfront license fees of $35.0
million paid in the prior year period to Oxford and Benitec,
research and development expenses decreased by $9.0 million in the
current year period, primarily due to the discontinuation of our
small molecule drug programs.
General and administrative expenses decreased by $10.9 million
from $22.4 million for the six months ended September 30, 2018 to
$11.5 million in the six months ended September 30, 2019,
primarily due to reductions in (i) share-based compensation expense
of $5.0 million attributable to reduced headcount, (ii) legal and
accounting fees of $3.0 million, and (iii) costs allocated under
our services agreements with RSI and RSG of $1.9 million as a
result of the decentralization of the services provided to us.
Net loss for the six months ended September 30, 2019 was
$41.9 million, or $1.84 per share, based on a weighted-average of
22.8 million common shares outstanding, compared to a net loss of
$85.7 million, or $6.00 per share, based on a weighted-average of
14.3 million common shares outstanding for the six months ended
September 30, 2018. Net cash used in operating activities for the
six months ended September 30, 2019 was $36.5 million, or
$30.5 million excluding a total of $6.0 million paid to Oxford and
the University of Massachusetts Medical School for development
milestones achieved. Excluding milestone payments, we expect our
net cash used in operating activities to be lower during the fiscal
year ending March 31, 2020 than in the prior fiscal year due to a
streamlined operating structure, the discontinuation of our small
molecule drug programs and the termination of the license and
collaboration agreement with Benitec.
As of September 30, 2019, we had $60.3 million of cash and cash
equivalents, which exceeded the minimum cash balance currently
required by our loan and securities agreement with Hercules
Capital, Inc. by $30.3 million, working capital of $19.6 million,
total debt of $34.4 million, net of discount, of which $11.7
million is classified as long-term debt.
About Axovant
Axovant Gene Therapies, part of the Roivant family of companies,
is a clinical-stage company focused on developing a pipeline of
innovative gene therapy product candidates for debilitating
neurological diseases. The company's 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.
About Roivant
Roivant Sciences aims to improve health by rapidly delivering
innovative medicines and technologies to patients. It does this by
building Vants – nimble, entrepreneurial biotech and healthcare
technology companies with a unique approach to sourcing talent,
aligning incentives, and deploying technology to drive greater
efficiency in R&D and commercialization. For more information,
please visit www.roivant.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 the initiation, timing,
progress, and reporting of results of its preclinical programs,
clinical trials, and research and development programs; cash to be
used in operating activities; its ability to advance its gene
therapy product candidates into and successfully initiate, enroll,
and complete clinical trials; the potential clinical utility of its
product candidates; its ability to continue to develop its gene
therapy platforms; its ability to develop and manufacture its
products and successfully transition manufacturing processes; its
ability to perform under existing collaborations with, among
others, Oxford BioMedica and the University of Massachusetts
Medical School, and to add new programs to its pipeline; its
ability to enter into new partnerships or collaborations; its
ability to retain and successfully integrate its leadership and
personnel; and the timing or likelihood of its regulatory filings
and approvals 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 preclinical
studies and clinical trials; the availability of data from clinical
trials; the expectations for regulatory submissions and approvals;
the continued development of its small molecule and 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 November
8, 2019, 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 and Investors
Parag MeswaniAxovant Gene Therapies(212)
547-2523investors@axovant.commedia@axovant.com
|
AXOVANT GENE THERAPIES LTD. |
|
Condensed Consolidated Statements of
Operations |
|
(Unaudited, in thousands, except share and per share amounts) |
| |
| |
Three Months Ended September 30, |
|
Six Months Ended September 30, |
| |
2019 |
|
2018 |
|
2019 |
|
2018 |
| Operating expenses: |
|
|
|
|
|
|
|
| Research and development
expenses(1) |
|
|
|
|
|
|
|
|
(includes share-based compensation expense (benefit) of $409 and
$(1,128) for the three months ended September 30, 2019 and 2018 and
$1,130 and $1,389 for the six months ended September 30, 2019 and
2018, respectively) |
$ |
6,833 |
|
|
$ |
21,502 |
|
|
$ |
27,923 |
|
|
$ |
58,920 |
|
| General and administrative
expenses(2) |
|
|
|
|
|
|
|
|
(includes share-based compensation expense of $482 and $3,585 for
the three months ended September 30, 2019 and 2018 and $1,896 and
$6,927 for the six months ended September 30, 2019 and 2018,
respectively) |
5,051 |
|
|
10,622 |
|
|
11,519 |
|
|
22,376 |
|
|
Total operating expenses |
11,884 |
|
|
32,124 |
|
|
39,442 |
|
|
81,296 |
|
| Other (income) expenses: |
|
|
|
|
|
|
|
|
Interest expense |
1,313 |
|
|
1,932 |
|
|
2,871 |
|
|
3,902 |
|
|
Other (income) expense |
560 |
|
|
(315 |
) |
|
(537 |
) |
|
353 |
|
| Loss before income tax
expense |
(13,757 |
) |
|
(33,741 |
) |
|
(41,776 |
) |
|
(85,551 |
) |
| Income tax expense |
127 |
|
|
94 |
|
|
165 |
|
|
172 |
|
| Net loss |
$ |
(13,884 |
) |
|
$ |
(33,835 |
) |
|
$ |
(41,941 |
) |
|
$ |
(85,723 |
) |
| Net loss per common share — basic
and diluted |
$ |
(0.61 |
) |
|
$ |
(2.24 |
) |
|
$ |
(1.84 |
) |
|
$ |
(6.00 |
) |
| Weighted-average common shares
outstanding — basic and diluted |
22,783,182 |
|
|
15,107,932 |
|
|
22,781,657 |
|
|
14,295,301 |
|
(1) Includes total costs (benefit) allocated from certain wholly
owned subsidiaries of RSL of $0 and $(3,069) for the three months
ended September 30, 2019 and 2018, respectively, and $0 and $(450)
for the six months ended September 30, 2019 and 2018,
respectively.
(2) Includes total costs allocated from certain wholly owned
subsidiaries of RSL of $48 and $772 for the three months ended
September 30, 2019 and 2018, respectively, and $76 and $2,074 for
the six months ended September 30, 2019 and 2018, respectively.
|
AXOVANT GENE THERAPIES LTD. |
|
Condensed Consolidated Balance Sheets |
|
(Unaudited, in thousands, except share and per share amounts) |
| |
| |
September 30, 2019 |
|
March 31, 2019 |
| Assets |
|
|
|
| Current assets: |
|
|
|
|
Cash and cash equivalents |
$ |
60,255 |
|
|
$ |
106,999 |
|
|
Prepaid expenses and other current assets |
4,503 |
|
|
5,859 |
|
|
Income tax receivable |
2,071 |
|
|
1,726 |
|
|
Total current assets |
66,829 |
|
|
114,584 |
|
| Long-term investment |
5,871 |
|
|
5,871 |
|
| Other non-current assets |
46 |
|
|
973 |
|
| Operating lease right-of-use
assets |
2,237 |
|
|
— |
|
| Property and equipment, net |
1,081 |
|
|
1,278 |
|
|
Total assets |
$ |
76,064 |
|
|
$ |
122,706 |
|
| |
|
|
|
| Liabilities and
Shareholders’ Equity |
|
|
|
| Current liabilities: |
|
|
|
|
Accounts payable |
$ |
1,567 |
|
|
$ |
1,698 |
|
|
Accrued expenses |
21,427 |
|
|
20,619 |
|
|
Current portion of operating lease liabilities |
1,672 |
|
|
— |
|
|
Current portion of long-term debt |
22,613 |
|
|
21,182 |
|
|
Total current liabilities |
47,279 |
|
|
43,499 |
|
| Operating lease liabilities, net
of current portion |
3 |
|
|
— |
|
| Long-term debt |
11,742 |
|
|
22,994 |
|
|
Total liabilities |
59,024 |
|
|
66,493 |
|
| |
|
|
|
| Shareholders’ equity: |
|
|
|
|
Common shares, par value $0.00001 per share, 1,000,000,000 shares
authorized, 22,791,669 and 22,779,891 issued and outstanding at
September 30, 2019 and March 31, 2019, respectively |
— |
|
|
— |
|
|
Additional paid-in capital |
744,506 |
|
|
741,318 |
|
|
Accumulated deficit |
(727,957 |
) |
|
(686,016 |
) |
|
Accumulated other comprehensive income |
491 |
|
|
911 |
|
|
Total shareholders’ equity |
17,040 |
|
|
56,213 |
|
| Total liabilities and
shareholders’ equity |
$ |
76,064 |
|
|
$ |
122,706 |
|
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