Axovant Gene Therapies Ltd (NASDAQ: AXGT), a clinical-stage company
developing innovative gene therapies, today provided financial
results and corporate updates for its first quarter ended June 30,
2019.
“I’m proud of the continued progress across
Axovant’s pipeline as we prepare to report data from all three of
our clinical-stage programs in the fourth quarter of 2019,
including results from the second cohort of our AXO-LENTI-PD study
and data from additional children dosed with AXO-AAV-GM1 and
AXO-AAV-GM2,” said Pavan Cheruvu, M.D., Chief Executive Officer of
Axovant. “Our reduced expenses this quarter reflect ongoing efforts
to streamline operations and drive long-term value for our
shareholders. Additionally, our newly forged partnership with
Yposkesi, a leading manufacturing group, provides us with
sufficient manufacturing capacity for our AAV-based gene therapy
pipeline. Finally, I’m pleased to welcome David Nassif and Senthil
Sundaram, seasoned leaders with decades of biopharmaceutical
industry experience, to help guide the company through an exciting
period ahead.”
Key Highlights and Development
Updates
- Initiated second dose cohort of the SUNRISE-PD study of
AXO-LENTI-PD in April, and we expect initial data from up to six
patients in this cohort in the fourth quarter of calendar 2019. In
June, we provided a six-month update on the first cohort of
AXO-LENTI-PD in Parkinson’s disease patients. AXO-LENTI-PD was
observed to be generally well tolerated at six months and continued
to demonstrate benefits in both patients across multiple measures
after a single administration. At month six, the patients
experienced an average improvement from baseline in UPDRS III
(motor) OFF score of 17 points, representing an average improvement
of 29%, and the Parkinson’s Disease Questionnaire-39 (“PDQ-39”)
Summary Index score showed a 32-point reduction from baseline, an
approximate 65% improvement.
- Preclinical data of AXO-LENTI-PD was published in Molecular
Therapy: Methods and Clinical Development. The publication titled
“Gene Therapy for Parkinson’s Disease: preclinical evaluation of an
optimally configured TH:CH1 fusion for maximal Dopamine synthesis”
reports safety and efficacy of AXO-LENTI-PD in the MPTP macaque
model of Parkinson’s disease.
- Investigators recently dosed a second patient with AXO-AAV-GM2.
The patient was a 7-month old with early symptomatic infantile
Tay-Sachs disease, and AXO-AAV-GM2 was delivered into the thalamus
bilaterally as well as into the cisterna magna and lumbar
intrathecal space, the planned route of administration for patients
in the program. We expect to enroll patients in a multi-subject
clinical trial in the second half of calendar 2019 and into 2020
and expect to provide additional data in fourth quarter of calendar
2019.
- Dosed first patient in the AXO-AAV-GM1 clinical program, with
initial data expected in the fourth quarter of calendar 2019.
Enrollment of patients will continue throughout calendar 2019 and
2020.
- Entered into a strategic partnership with Yposkesi, a leading
Contract Development and Manufacturing Organization (CDMO) spinout
from Genethon, for preferred access and reserved capacity for cGMP
grade viral vector production. Yposkesi will support our gene
therapy programs as they proceed through development and
commercialization.
- Appointed Mr. David Nassif as Chief Financial Officer and
expanded our Board of Directors with the addition of Mr. Senthil
Sundaram.
- Data from the AXO-AAV-GM2 and AXO-LENTI-PD programs were
presented at the 22nd Annual Meeting of the American Society of
Gene & Cell Therapy (ASGCT) and 5th Congress of the European
Academy of Neurology (EAN).
Fiscal First Quarter Financial
Summary
Research and development expenses were $21.1 million for the
first fiscal quarter ended June 30, 2019, including a net
payment of $13.0 million due to Oxford BioMedica (UK) Ltd.
("Oxford") for a development milestone achieved for our
AXO-LENTI-PD program. Research and development expenses were $37.4
million for the first fiscal quarter ended June 30, 2018,
including the $25.0 million upfront license fee paid to Oxford.
Excluding the net development milestone achieved and upfront
license fee paid, research and development expenses decreased by
$4.3 million from $12.4 million in the first fiscal quarter ended
June 30, 2018 to $8.1 million in the first fiscal quarter
ended June 30, 2019, which was primarily due to the
discontinuation of our intepirdine and nelotanserin small molecule
drug programs.
General and administrative expenses decreased by $5.3 million
from $11.8 million for the three months ended June 30, 2018 to
$6.5 million in the three months ended June 30, 2019. The
decrease was primarily related to reductions in i) professional
fees of $2.3 million supporting business development and financing
activities in the prior year, ii) share-based compensation expense
of $1.9 million attributable to reduced headcount, and iii) costs
allocated under the Roivant services agreements of $1.2 million as
a result of the decentralization of the services provided to us.
Going forward, the costs allocated to us under the services
agreements are expected to be insignificant.
Net loss for the fiscal first quarter ended June 30, 2019
was $28.1 million, or $1.23 per share, based on a weighted-average
of 22.8 million common shares outstanding, compared to a net loss
of $51.9 million, or $3.85 per share, based on a weighted-average
of 13.5 million common shares outstanding for the quarter ended
June 30, 2018. Net cash used in operating activities for the fiscal
first quarter ended June 30, 2019 was $17.7 million, or $16.7
million excluding $1.0 million paid to the University of
Massachusetts Medical School for a development milestone achieved.
Excluding milestone payments, we expect our net cash used in
operations to be lower during the fiscal year ending March 31, 2020
than in the prior year due to a reduced operating cost structure,
the discontinuation of our legacy small molecule drug programs and
the termination of the license and collaboration agreement with
Benitec Biopharma Limited.
As of June 30, 2019, we had $84.2 million of cash and cash
equivalents, working capital of $37.7 million, total net debt of
$39.4 million and long-term debt of $17.5 million. As of
June 30, 2019, we had 22.8 million common shares
outstanding.
About Axovant
Axovant, part of the Roivant family of companies, is a
clinical-stage gene therapy company focused on developing a
pipeline of innovative product candidates for debilitating
neurological and neuromuscular diseases. Our 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 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 August 9,
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
Tricia Truehart(631)
892-7014media@axovant.cominvestors@axovant.com
AXOVANT GENE THERAPIES LTD. |
Condensed Consolidated Statements of
Operations |
(Unaudited, in thousands, except share and per share amounts) |
|
|
|
Three Months Ended June 30, |
|
2019 |
|
2018 |
Operating expenses: |
|
|
|
Research and development
expenses(1) |
|
|
|
(includes $721 and $2,517 of share-based compensation expense for
the three months ended June 30, 2019 and 2018, respectively) |
$ |
21,090 |
|
|
$ |
37,418 |
|
General and administrative
expenses(2) |
|
|
|
(includes $1,414 and $3,342 of share-based compensation expense for
the three months ended June 30, 2019 and 2018, respectively) |
6,468 |
|
|
11,754 |
|
Total operating expenses |
27,558 |
|
|
49,172 |
|
Other expenses: |
|
|
|
Interest expense |
1,558 |
|
|
1,970 |
|
Other (income) expense |
(1,097 |
) |
|
668 |
|
Loss before income tax
expense |
(28,019 |
) |
|
(51,810 |
) |
Income tax expense |
38 |
|
|
78 |
|
Net loss |
$ |
(28,057 |
) |
|
$ |
(51,888 |
) |
Net loss per common share — basic
and diluted |
$ |
(1.23 |
) |
|
$ |
(3.85 |
) |
Weighted-average common shares
outstanding — basic and diluted |
22,780,114 |
|
|
13,473,740 |
|
(1) Includes total costs allocated from our parent company,
Roivant Sciences Ltd. ("RSL") and certain of its wholly owned
subsidiaries of $0 and $2,619 for the three months ended June 30,
2019 and 2018, respectively.
(2) Includes total costs allocated from RSL and certain of its
wholly owned subsidiaries of $28 and $1,302 for the three months
ended June 30, 2019 and 2018, respectively.
AXOVANT GENE THERAPIES LTD. |
Condensed Consolidated Balance Sheets |
(Unaudited, in thousands, except share and per share amounts) |
|
|
June 30, 2019 |
|
March 31, 2019 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
84,176 |
|
|
$ |
106,999 |
|
Prepaid expenses and other current assets |
5,657 |
|
|
5,859 |
|
Income tax receivable |
1,916 |
|
|
1,726 |
|
Total current assets |
91,749 |
|
|
114,584 |
|
Long-term investment |
5,871 |
|
|
5,871 |
|
Other non-current assets |
46 |
|
|
973 |
|
Operating lease right-of-use
assets |
2,664 |
|
|
— |
|
Property and equipment, net |
1,115 |
|
|
1,278 |
|
Total assets |
$ |
101,445 |
|
|
$ |
122,706 |
|
|
|
|
|
Liabilities and
Shareholders’ Equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable |
$ |
2,896 |
|
|
$ |
1,698 |
|
Accrued expenses |
27,698 |
|
|
20,619 |
|
Current portion of operating lease liabilities |
1,586 |
|
|
— |
|
Current portion of long-term debt |
21,855 |
|
|
21,182 |
|
Total current liabilities |
54,035 |
|
|
43,499 |
|
Operating lease liabilities, net
of current portion |
525 |
|
|
— |
|
Long-term debt |
17,533 |
|
|
22,994 |
|
Total liabilities |
72,093 |
|
|
66,493 |
|
|
|
|
|
Shareholders’ equity: |
|
|
|
Common shares, par value $0.00001 per share, 1,000,000,000 shares
authorized, 22,780,672 and 22,779,891 issued and outstanding at
June 30, 2019 and March 31, 2019, respectively |
— |
|
|
— |
|
Additional paid-in capital |
743,486 |
|
|
741,318 |
|
Accumulated deficit |
(714,073 |
) |
|
(686,016 |
) |
Accumulated other comprehensive (loss) income |
(61 |
) |
|
911 |
|
Total shareholders’ equity |
29,352 |
|
|
56,213 |
|
Total liabilities and
shareholders’ equity |
$ |
101,445 |
|
|
$ |
122,706 |
|
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