Denali Therapeutics Announces First-In-Human Dosing Of Its RIPK1 Inhibitor Clinical Program And The Appointment Of Peter Klei...
March 19 2018 - 7:00AM
Denali Therapeutics Inc. (NASDAQ:DNLI), a biopharmaceutical company
developing a broad portfolio of therapeutic candidates for
neurodegenerative diseases, today announced that it has commenced
dosing of its small molecule inhibitor of RIPK1 in a Phase 1
clinical trial in healthy volunteers and achieved proof of concept
of its large molecule blood-brain barrier delivery platform
technology in nonhuman primates. In addition, the Company reported
financial results for the fourth quarter and full year ended
December 31, 2017 and appointed Peter Klein to the Board of
Directors.
"The recent clinical progress with our RIPK1 inhibitor and LRRK2
inhibitor programs, along with achieving proof of concept for our
blood-brain barrier delivery platform for biologics in nonhuman
primates, are important milestones towards our goal of developing
medicines for patients suffering from neurodegenerative diseases,"
said Ryan Watts, Ph.D., CEO. "Furthermore, we are very excited
about initiating our collaboration with Takeda, which has allowed
us to expand efforts on our blood-brain barrier delivery technology
platform."
Fourth Quarter 2017 and Recent Corporate
Highlights
- Achieved first-in-human dosing in Phase 1 clinical
trial of RIPK1 inhibitor program - In March 2018, Denali
commenced dosing of its small molecule inhibitor of RIPK1, DNL747,
in healthy volunteers in the Netherlands. DNL747 is a
potent, selective and brain-penetrant small molecule inhibitor of
RIPK1 in development for Alzheimer’s disease and ALS.
- Advanced and expanded LRRK2 inhibitor program
- In December 2017, Denali announced that it had achieved
robust target engagement in humans with DNL201, a LRRK2 inhibitor,
in a Phase 1 clinical trial, and that the FDA removed the partial
clinical hold on DNL201. This Phase 1 clinical trial in healthy
volunteers for DNL201 is continuing. Denali also announced
that it commenced dosing of its second LRRK2 inhibitor, DNL151, in
a Phase 1 clinical trial in healthy volunteers in the Netherlands.
DNL201 and DNL151 are both potent, selective
and brain-penetrant small molecule inhibitors of LRRK2 in
development for Parkinson’s disease.
- Achieved proof of concept of the blood-brain barrier
delivery platform technology in nonhuman primates - In
January 2018, Denali completed a 28-day study demonstrating
sustained brain activity of its proprietary Antibody Transport
Vehicle (ATV) technology, as measured by reduction of cerebral
spinal fluid amyloid beta in cynomolgus monkeys after dosing of an
anti-BACE1 antibody that is enabled by Denali’s proprietary ATV
technology, compared to a standard anti-BACE1 antibody. These data
are consistent with initial 7-day study data previously disclosed
and establish preclinical proof of concept for future human
studies.
- Entered into Option and Collaboration Agreement with
Takeda - In January 2018, Denali entered into an Option
and Collaboration Agreement with Takeda pursuant to which Denali
granted Takeda an option in respect of three named Denali programs
to develop and commercialize, jointly with Denali, certain biologic
products that are enabled by Denali's blood-brain barrier delivery
platform technology and intended for the treatment of
neurodegenerative disorders. Denali received $155.0 million of cash
associated with this transaction in February 2018, including $110.0
million for Takeda’s purchase of 4,214,559 shares of Denali’s
common stock.
- Appointed Peter Klein to the Board of Directors
- On March 16, 2018, Peter Klein joined the Board of
Directors as an independent director. Mr. Klein has 25 years
of experience as a senior finance executive. He served as Chief
Financial Officer of Microsoft Corporation from November 2009 until
May 2013 and spent over 11 years at Microsoft. Most recently, he
served as Chief Financial Officer of WME, a global leader in sports
and entertainment marketing, from January 2014 until June 2014.
Mr. Klein holds a B.A. from Yale University and an M.B.A from
University of Washington. He currently serves on the board of
directors of two publicly traded companies: Apptio Inc. and F5
Networks, Inc.
- Raised net proceeds of $294.2 million during the fourth
quarter of 2017 through the sale of convertible preferred stock and
a subsequent initial public offering and listing on the Nasdaq
Global Select Market.
Fourth Quarter and Full Year 2017 Financial
Results
For the fourth quarter of 2017, Denali reported a net loss of
$22.9 million, compared with a net loss for the fourth quarter of
2016 of $19.4 million. For the year ended December 31, 2017, net
loss was $88.2 million, compared with a net loss for the same
period in 2016 of $86.7 million.
Total operating expenses for the fourth quarter of 2017 were
$23.5 million compared with $19.8 million for the same period in
2016 including non-cash stock-based compensation of $1.5 million
and $0.7 million in the fourth quarter of 2017 and 2016,
respectively. Total operating expenses for the year ended
December 31, 2017 were $90.1 million compared with $87.4 million
for the same period in 2016, including non-cash stock-based
compensation of $4.4 million and $3.0 million in 2017 and 2016,
respectively. The increase in total operating expenses is due to
the increase in research, development, and general and
administrative costs as Denali's pipeline expanded and
advanced.
Cash, cash equivalents, and marketable securities were $467.0
million as of December 31, 2017.
About Denali Therapeutics
Denali is a biopharmaceutical company developing a broad
portfolio of therapeutic candidates for neurodegenerative diseases.
Denali is based in South San Francisco. For additional information,
please visit www.denalitherapeutics.com.
Forward-looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Forward-looking statements expressed or implied in this press
release include, but are not limited to, statements regarding
Denali’s progress and business plans; clinical trial plans,
timelines and expectations, including regarding proof of concept
for future human studies; Denali’s expectations regarding
collaborations; and statements made by Denali’s CEO. Actual
results are subject to risks and uncertainties and may differ
materially from those indicated by these forward-looking statements
as a result of these risks and uncertainties, including but not
limited to, risks related to: Denali’s early stage of clinical
drug development; Denali’s ability to complete the development of,
and if approved, commercialization of its product candidates;
Denali’s dependence on successful development of its BBB platform
technology and product candidates currently in its core program;
Denali’s ability to conduct or complete clinical trials on expected
timelines; the uncertainty that any of Denali’s product candidates
will receive regulatory approval necessary to be commercialized;
Denali’s ability to continue to create a pipeline of product
candidates or develop commercially successful products; Denali's
ability to obtain, maintain, or protect intellectual property
rights related to its product candidates; implementation of
Denali’s strategic plans for its business, product candidates and
BBB platform technology; and other risks, including those described
in Denali’s Annual Report on Form 10-K filed with
the SEC on March 19, 2018, the final prospectus
related to Denali’s initial public offering filed with the SEC on
December 8, 2017, and Denali’s future reports to be filed with the
SEC. The forward-looking statements in this press release are based
on information available to Denali as of the date
hereof. Denali disclaims any obligation to update any
forward-looking statements, except as required by law.
|
Denali Therapeutics Inc. |
Unaudited Consolidated Statements of
Operations |
(In
thousands, except per share amounts) |
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
2017 |
|
2016 |
|
2017 |
|
2016 |
Operating
expenses: |
|
|
|
|
|
|
|
Research
and development |
$ |
18,471 |
|
|
$ |
16,730 |
|
|
$ |
74,460 |
|
|
$ |
75,702 |
|
General
and administrative |
5,069 |
|
|
3,046 |
|
|
15,680 |
|
|
11,731 |
|
Total
operating expenses |
23,540 |
|
|
19,776 |
|
|
90,140 |
|
|
87,433 |
|
Loss from
operations |
(23,540 |
) |
|
(19,776 |
) |
|
(90,140 |
) |
|
(87,433 |
) |
Interest
income (expense), net |
653 |
|
|
422 |
|
|
1,955 |
|
|
781 |
|
Net
loss |
$ |
(22,887 |
) |
|
$ |
(19,354 |
) |
|
$ |
(88,185 |
) |
|
$ |
(86,652 |
) |
Net loss
per share, basic and diluted |
$ |
(0.74 |
) |
|
$ |
(2.41 |
) |
|
$ |
(5.89 |
) |
|
$ |
(13.49 |
) |
Weighted
average number of shares outstanding, basic and diluted1 |
30,743,977 |
|
|
8,022,066 |
|
|
14,964,144 |
|
|
6,424,720 |
|
1 Share numbers have been adjusted, as appropriate, for the
4-for-1 reverse stock split that occurred on November 28, 2017. The
increase in the weighted average number of common shares
outstanding from 2016 to 2017 is primarily due to the conversion of
preferred stock into common stock, and issuance of additional
shares of common stock, both of which occurred at the time of our
initial public offering in December 2017.
|
Denali Therapeutics Inc. |
Unaudited Consolidated Balance Sheets |
(In
thousands) |
|
|
December 31, |
|
2017 |
|
2016 |
|
|
|
|
Assets |
|
|
|
Current assets: |
|
|
|
Cash and
cash equivalents |
$ |
218,375 |
|
|
$ |
39,853 |
|
Short-term marketable securities |
187,851 |
|
|
138,478 |
|
Prepaid
expenses and other current assets |
3,381 |
|
|
3,624 |
|
Total
current assets |
409,607 |
|
|
181,955 |
|
Long-term marketable
securities |
60,750 |
|
|
72,580 |
|
Property and equipment,
net |
14,923 |
|
|
15,262 |
|
Other non-current assets |
1,441 |
|
|
1,270 |
|
Total assets |
$ |
486,721 |
|
|
$ |
271,067 |
|
Liabilities,
convertible preferred stock and stockholders’ equity
(deficit) |
|
|
|
Current
liabilities: |
|
|
|
Accounts
payable |
$ |
2,716 |
|
|
$ |
1,963 |
|
Accrued
liabilities |
5,364 |
|
|
3,850 |
|
Accrued
compensation |
5,166 |
|
|
2,592 |
|
Deferred
rent |
855 |
|
|
538 |
|
Other
current liabilities |
63 |
|
|
163 |
|
Total
current liabilities |
14,164 |
|
|
9,106 |
|
Deferred rent |
6,294 |
|
|
7,045 |
|
Other non-current liabilities |
467 |
|
|
397 |
|
Total liabilities |
20,925 |
|
|
16,548 |
|
Convertible preferred
stock |
— |
|
|
348,673 |
|
Total
stockholders’ equity (deficit) |
465,796 |
|
|
(94,154 |
) |
Total liabilities,
convertible preferred stock and stockholders’ equity (deficit) |
$ |
486,721 |
|
|
$ |
271,067 |
|
|
Contact
Morgan Warners (202) 337-0808 mwarners@gpg.com
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