Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage
biopharmaceutical company dedicated to transforming the lives of
patients and families affected by complement-mediated diseases,
today reported its financial results for the three and six months
ended June 30, 2019 and provided a corporate update.
“We continued to advance our complement factor D
inhibitors in the second quarter of 2019 by efficiently executing
on our global development and regulatory strategies, while
continuing to strengthen our intellectual property position. As we
maintain discipline on our spending, we continue to increase our
chemistry, manufacturing, and controls (CMC) capabilities in
preparation for our expanding portfolio,” said Joe Truitt,
President and Chief Executive Officer at Achillion. “We believe our
portfolio of oral factor D inhibitors has potential clinical
benefits in multiple indications, and we look forward to initiating
a clinical trial in a new indication in 2020, in addition to our
Phase 3 clinical trial of danicopan for paroxysmal nocturnal
hemoglobinuria (PNH) in early 2020.”
Key Highlights and Development
Updates:
Danicopan
- The ongoing Phase 2 PNH clinical
trial assessing the safety and effectiveness of danicopan
(ACH-4471) in combination with intravenous eculizumab is expected
to be completed in the third quarter of 2019. Interim data,
presented at The New Era of Aplastic Anemia and PNH Meeting in May
2019, demonstrated clinically meaningful improvements in laboratory
parameters of PNH including hemoglobin, reticulocyte count, and
bilirubin, as well as meaningful improvements in health-related
quality of life FACIT-Fatigue scores relative to baseline scores on
eculizumab. The addition of danicopan nearly eliminated the
patients’ needs for blood transfusions in this study. The full data
set is expected in the fourth quarter of 2019. Dosing with
ULTOMIRIS™ (ravulizumab) has been initiated in the extension study
in combination with danicopan.
- The Company completed enrollment in
the danicopan Phase 2 clinical trials for C3 glomerulopathy (C3G)
and C3G/immune complex-mediated membrane glomerulonephritis
(IC-MPGN). A total of 32 patients have been enrolled in the two
trials; 13 C3G patients in the 6-month double-blind,
placebo-controlled trial and 19 C3G/IC-MPGN patients in the
12-month single-arm open-label trial. Pending an analysis of the
C3G data, the Company plans to meet with the U.S. Food and Drug
Administration (FDA) for an End-of-Phase 2 meeting and the European
Medicines Agency (EMA) for scientific advice, in 2020.
Next-generation Factor D Inhibitors
- Results from the completed ACH-5228
Phase 1 multiple ascending dose study demonstrated that ACH-5228,
when dosed 120 mg twice a day (BID) or higher, achieved near
complete and sustained alternative pathway (AP) inhibition with a
mean value of >95% at steady state concentrations as measured by
AP Hemolysis and AP Wieslab assays. In the study, ACH-5228 was
generally well-tolerated over the dose ranges tested, which
included the doses expected to be evaluated in Phase 2 clinical
trials. The Company expects to submit an investigational new drug
(IND) application to the FDA for ACH-5228 in the fourth quarter of
2019.
- The Company has identified a series
of third-generation factor D inhibitors, with unique
pharmacokinetic attributes, which are believed to provide
additional optionality and durability for our factor D development
program. The Company plans to nominate one of the third-generation
factor D inhibitors for clinical development in 2020.
Second Quarter 2019 Financial
Results
For the three months ended June 30, 2019,
Achillion reported a net loss of $19.4 million compared with a net
loss of $17.2 million during the same period of 2018. Research and
development expenses were $15.9 million for the three months ended
June 30, 2019, compared with $11.0 million for the same period of
2018. The increase was primarily due to increased clinical trial
costs related to danicopan and ACH-5228, combined with increased
manufacturing and formulation costs related to ACH-5228 and
ACH-5548. These amounts were partially offset by decreased
discovery research costs.
For the three months ended June 30, 2019,
general and administrative expenses were $5.1 million, compared
with $7.5 million incurred during the same period in 2018. The
decrease was primarily due to decreased personnel costs and
non-cash stock-based compensation charges related to the transition
of our former chief executive officer in the second quarter of
2018.
Non-cash stock compensation expense totaled $1.9
million for the second quarter of 2019 as compared with $3.5
million for the second quarter of 2018 and is included in research
and development expenses and general and administrative
expenses.
Cash, cash equivalents and marketable securities
as of June 30, 2019 was $241.3 million. The company expects
net-cash spend for the full year of 2019 to be approximately $85
million, resulting in year-end 2019 cash, cash equivalents and
marketable securities of approximately $185 million.
Six Month 2019 Financial
Results
For the six months ended June 30, 2019,
Achillion reported a net loss of $38.4 million, compared to a net
loss of $37.8 million in the same period in 2018. For the six
months ended June 30, 2019, research and development expenses
totaled $30.8 million, compared with $25.1 million during the same
period in 2018. The increase was primarily due to increased
clinical trial costs related to danicopan and ACH-5228, combined
with increased manufacturing and formulation costs related to
ACH-5228 and ACH-5548. These amounts were partially offset by
decreased discovery research costs.
General and administrative expenses were $10.3
million for the six months ended June 30, 2019, compared to $13.5
million in the same period in 2018. The decrease was primarily due
to decreased personnel costs and non-cash stock-based compensation
charges related to the transition of our former chief executive
officer.
Non-cash stock compensation expense totaled $3.2
million for the six months ended June 30, 2019 as compared with
$5.9 million for the same period in 2018 and is included in both
research and development and general and administrative
expenses.
About the Achillion Complement Factor D
PortfolioAchillion has leveraged its internal discovery
capabilities and a novel complement-related platform to develop
oral small molecule drug candidates that are inhibitors of
complement factor D. Factor D is an essential serine protease
involved in the alternative pathway (AP) of the complement system,
a part of the innate immune system. Achillion's complement platform
is focused on seeking to advance oral small molecules that inhibit
the AP and can potentially be used in the treatment of
immune-related diseases in which complement AP plays a critical
role. Potential indications currently being evaluated for these
compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3
glomerulopathy (C3G), and immune complex-mediated
membranoproliferative glomerulonephritis (IC-MPGN).
About Achillion
PharmaceuticalsAchillion Pharmaceuticals, Inc. (Nasdaq:
ACHN) is a clinical-stage biopharmaceutical company focused on
advancing its oral small molecule complement inhibitors into
late-stage development and commercialization. Research has shown
that an overactive complement system plays a critical role in
multiple disease conditions including the therapeutic areas of
nephrology, hematology, ophthalmology and neurology. Achillion is
initially focusing its drug development activities on
complement-mediated diseases where there are no approved therapies
or significant unmet medical needs persist despite existing
therapies. Potential indications being evaluated for its compounds
include paroxysmal nocturnal hemoglobinuria (PNH), C3
glomerulopathy (C3G), and immune complex membranoproliferative
glomerulonephritis (IC-MPGN). Each of the product candidates in the
Company’s oral small molecule portfolio was discovered in its
laboratories and is wholly owned. In its efforts to advance its
investigational product candidates into registrational clinical
trials and commercialization, the Company plans to work closely
with key stakeholders including patients, payors, regulators, and
healthcare providers.
More information is available at
http://www.achillion.com.
Cautionary Note Regarding
Forward-Looking StatementsThis press release includes
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995 that are subject to risks,
uncertainties and other important factors that could cause actual
results to differ materially from those indicated by such
forward-looking statements. Achillion may use words such as
“expect,” “anticipate,” “project,” “target,” “intend,” “plan,”
“aim,” “believe,” “seek,” “estimate,” “can,” “could,” “focus,”
“will,” “look forward,” “continue,” “goal,” “strategy,”
“objective,” “may,” “potential,” and similar expressions to
identify such forward-looking statements. These forward-looking
statements include statements about: the potential benefits of
factor D inhibition as a treatment for complement-mediated
diseases, including danicopan (ACH-4471) for PNH; the potential
benefits of, and indications for, Achillion’s compounds that
inhibit factor D, including danicopan and ACH-5228; Achillion’s
belief that its portfolio of compounds could expand factor D
portfolio opportunities, provide strategic optionality or create
significant value; the status of enrollment in Achillion’s ongoing
clinical trials; Achillion’s expectations regarding the advancement
of, and timeline for reporting results from, clinical trials of its
product candidates (including danicopan and ACH-5228) as well as
its ability to advance additional compounds; Achillion’s
expectations regarding the timing of regulatory interactions and
filings; Achillion’s anticipated cash expenditures for 2019 and the
sufficiency of its existing cash resources; and other statements
concerning Achillion’s strategic goals, efforts, plans, and
prospects. Among the important factors that could cause actual
results to differ materially from those indicated by such
forward-looking statements are risks relating to, among other
things, Achillion’s ability to: demonstrate in any current and
future clinical trials the requisite safety, efficacy and
combinability of its product candidates, including danicopan and
ACH-5228; advance the preclinical and clinical development of its
complement factor D inhibitors under the timelines it projects in
current and future preclinical studies and clinical trials; whether
interim results from a clinical trial will be predictive of the
final results of that trial or whether results of early clinical
trials or preclinical studies will be indicative of the results of
later clinical trials; enroll patients in its clinical trials on
its projected timelines; obtain and maintain patent protection for
its product candidates and the freedom to operate under third party
intellectual property; obtain and maintain necessary regulatory
approvals, and the granting of orphan designation does not alter
the standard regulatory requirements and process for obtaining such
approval; establish commercial manufacturing arrangements;
identify, enter into and maintain collaboration and other
commercial agreements with third-parties; compete successfully in
the markets in which it seeks to develop and commercialize its
product candidates and future products; manage expenses; manage
litigation; raise the substantial additional capital needed to
achieve its business objectives; and successfully execute on its
business strategies. These and other risks are described in the
reports filed by Achillion with the U.S. Securities and Exchange
Commission, including its Quarterly Report on Form 10-Q for the
quarterly period ended March 31, 2019, and any other SEC filings
that Achillion makes from time to time.
In addition, any forward-looking statement in
this press release represents Achillion's views only as of the date
of this press release and should not be relied upon as representing
its views as of any subsequent date. Achillion disclaims any duty
to update any forward-looking statement, except as required by
applicable law.
Investor Relations: Clayton RobertsonAchillion
Pharmaceuticals, Inc.Tel. 215-709-3078 crobertson@achillion.com
Media: Susanne Heinzinger Senior VP, Corporate
CommunicationsAchillion Pharmaceuticals, Inc. Tel. 215-709-3032
sheinzinger@achillion.com
ACHILLION PHARMACEUTICALS INC. (ACHN) |
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Statements of
Operations |
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(Unaudited, in thousands, except per share
amounts) |
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Three Months
Ended |
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Six Months
Ended |
|
June
30, |
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June
30, |
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2019 |
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2018 |
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2019 |
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2018 |
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Revenue |
$ |
- |
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|
$ |
- |
|
|
$ |
- |
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|
$ |
- |
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|
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Operating expenses: |
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|
|
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|
Research and development |
|
15,932 |
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|
|
11,023 |
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|
|
30,750 |
|
|
|
25,072 |
|
General and administrative |
|
5,109 |
|
|
|
7,463 |
|
|
|
10,266 |
|
|
|
13,478 |
|
Restructuring charges |
|
- |
|
|
|
75 |
|
|
|
655 |
|
|
|
1,825 |
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Total operating expenses |
|
21,041 |
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|
|
18,561 |
|
|
|
41,671 |
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|
40,375 |
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Loss from operations |
|
(21,041 |
) |
|
|
(18,561 |
) |
|
|
(41,671 |
) |
|
|
(40,375 |
) |
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Other income (expense): |
|
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|
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Interest income |
|
1,621 |
|
|
|
1,370 |
|
|
|
3,302 |
|
|
|
2,609 |
|
Interest expense |
|
(6 |
) |
|
|
(8 |
) |
|
|
(16 |
) |
|
|
(21 |
) |
|
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Net loss |
|
(19,426 |
) |
|
|
(17,199 |
) |
|
|
(38,385 |
) |
|
|
(37,787 |
) |
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Net loss per share - basic and
diluted |
$ |
(0.14 |
) |
|
$ |
(0.12 |
) |
|
$ |
(0.28 |
) |
|
$ |
(0.27 |
) |
|
|
|
|
|
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|
Weighted average shares
outstanding - basic and diluted |
|
138,761 |
|
|
|
138,426 |
|
|
|
138,739 |
|
|
|
138,221 |
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Balance Sheets |
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(Unaudited, in thousands) |
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|
June
30, |
|
December
31, |
|
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2019 |
|
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2018 |
|
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|
Cash, cash equivalents and
marketable securities |
$ |
241,305 |
|
|
$ |
270,977 |
|
|
|
|
Working capital |
|
221,286 |
|
|
|
263,551 |
|
|
|
|
Total assets |
|
248,375 |
|
|
|
277,858 |
|
|
|
|
Long-term liabilities |
|
510 |
|
|
|
17 |
|
|
|
|
Total liabilities |
|
16,732 |
|
|
|
11,846 |
|
|
|
|
Total stockholders'
equity |
|
231,643 |
|
|
|
266,012 |
|
|
|
|
Source: Achillion Pharmaceuticals, Inc.
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