- Phase 2 trial enrollment on schedule in PNH
combo, interim data expected in Q2:2019 -- Phase 2 trial enrollment
on schedule in C3G, 24 sites recruiting -- Next-generation factor D
inhibitor ACH-5228 US IND anticipated in Q4:2019 -- Reduced 2018
net cash spend to $60 million, projecting $80 million in 2019 --
Well funded with cash and securities of $271 million at December
31, 2018 -
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 financial results for the three and twelve months
ended December 31, 2018 and provided a corporate update.
“We made significant progress advancing our
portfolio of oral factor D inhibitors in the fourth quarter of
2018,” said Joe Truitt, President and Chief Executive Officer at
Achillion. “ACH-4471 has demonstrated preliminary proof-of-concept
in patients with paroxysmal nocturnal hemoglobinuria (PNH) and C3
glomerulopathy (C3G). We look forward to reporting updated interim
results for our Phase 2 trial of ACH-4471 for PNH, in combination
with a C5 inhibitor, in the second quarter of 2019. We plan to
present data for the PNH and C3G trials to the U.S. Food and Drug
Administration (FDA) in the fourth quarter of 2019. We also remain
on track to complete the ongoing ex-US Phase 1 multiple ascending
dose (MAD) study for our more potent next-generation oral factor D
inhibitor, ACH-5228. We anticipate submitting an Investigational
New Drug (IND) Application in the US for ACH-5228 in the fourth
quarter of 2019.” Mr. Truitt continued, “We took several
important steps to strengthen our leadership team and expand the
Company’s capabilities from a discovery engine to a clinical-stage
organization in 2018. Additionally, we were pleased to announce
that the Board of Directors recently elected Nicole Vitullo as
Chair and we added Brian Di Donato as Senior Vice President and
Chief Financial Officer to our executive management team. We also
expanded our operations into the Philadelphia area which adds
access to a strong talent pool for future growth. We remain well
positioned to advance our first- and next-generation oral factor D
inhibitors in patients with serious medical conditions. In 2019, we
will continue to focus on expanding our clinical operations for PNH
and C3G. We now have 24 trial sites recruiting patients for C3G. We
also plan to evaluate the potential of our oral small molecule
complement inhibitors in additional indications to deliver on the
promise of complement inhibition across a wide spectrum of
diseases.”
Key 2019 Planned Milestones
- ACH-4471, Complement Factor D Inhibitor for PNH and
C3G
- The Company expects to report
updated interim results for its Phase 2 open-label combination
trial of ACH-4471 for PNH, with C5 inhibitor (eculizumab), in the
second quarter of 2019 and is targeting an FDA meeting in the
fourth quarter of 2019.
- The Company plans to present data
from its Phase 2 clinical trials of ACH-4471 for C3G and immune
complex membranoproliferative glomerulonephritis (IC-MPGN) to the
FDA in the fourth quarter of 2019.
- ACH-5228, Next Generation Complement Factor D
Inhibitor
- The Company recently initiated a
Phase 1 MAD study of ACH-5228 and expects to complete the study and
submit a US IND Application in the fourth quarter of 2019.
Fourth Quarter 2018 Financial
Results
For the three months ended December 31, 2018,
the Company reported a net loss of $16.6 million, compared to a net
loss of $23.2 million in the three months ended December 31,
2017.
Research and development expenses were $12.2
million for the three months ended December 31, 2018, compared to
$15.7 million for the same period of 2017. The decrease was
primarily due to decreased personnel costs due to fewer employees
combined with decreased discovery research costs related to
ACH-5228. These amounts were partially offset by increased
manufacturing expenses for ACH-5228.
For the three months ended December 31, 2018,
general and administrative expenses totaled $6.0 million, compared
to $8.7 million for the same period of 2017. The decrease was
primarily the result of the Company’s payment of the underwriting
fees in connection with the public resale by the Johnson and
Johnson Development Corporation (JJDC) in November 2017 of all the
shares of common stock it acquired from the Company in
2015.
Year-end 2018 Financial
ResultsFor the year ended December 31, 2018, the Company's
net loss was $70.3 million, or $0.51 per share, compared to a net
loss of $85.2 million, or $0.62 per share, for the year ended
December 31, 2017.
For the year ended December 31, 2018, research and development
expenses totaled $50.1 million, compared to $63.6 million for the
year ended December 31, 2017. The decrease was primarily due to
manufacturing and formulation costs related to ACH-5228, discovery
research costs, and personnel costs and non-cash stock compensation
due to fewer employees. These amounts were partially offset by
increased clinical trial costs related to ACH-4471 and increased
pre-clinical costs related to ACH-5548.
General and administrative expenses were $23.9 million for the
year ended December 31, 2018, compared to $26.0 million for the
year ended December 31, 2017. The decrease was primarily due to
decreased corporate legal fees and consulting fees combined with
the Company’s payment of $2.9 million in underwriting fees in
connection with the sale by JJDC in November 2017 of all of the
shares of the Company’s common stock it acquired pursuant to the
JJDC Stock Purchase Agreement. These amounts were partially offset
by an increase in personnel and non-cash stock-based compensation
charges related to the transition of the Company’s former chief
executive officer and former chief financial officer.
Cash, cash equivalents, and marketable
securities at December 31, 2018 were $271.0 million.
Financial Guidance The company expects net cash
usage for 2019 will be approximately $80 million based on current
operating plans, anticipated timelines and the estimated costs of
clinical trials and product development programs. About
ACH-4471, Complement Factor D Inhibitor for PNH and
C3GAchillion’s first-generation oral complement factor D
inhibitor, ACH-4471, is being evaluated for safety and efficacy
with Phase 2 clinical programs in both PNH and C3G and has
demonstrated preliminary proof-of-concept in both indications. The
PNH program consists of a Phase 2 clinical trial evaluating
ACH-4471 in patients who are inadequately controlled or
sub-optimally responding to eculizumab. Additionally, the Company
continues to dose patients in its PNH monotherapy extension trial.
The C3G program consists of two Phase 2 clinical trials which are
currently recruiting, a six-month blinded, placebo-controlled study
and a 12-month open-label study. More information is available
at http://www.achillion.com/patients-and-clinicians/.
About ACH-5228 and ACH-5548, Next
Generation Complement Factor D InhibitorsACH-5228 and
ACH-5548 are the Company’s next-generation oral factor D inhibitors
currently in Phase 1 clinical trials. These compounds demonstrated
enhanced potency as well as improved pharmacokinetic properties
that should allow for higher alternative pathway inhibition along
with a reduced dosing frequency.
About Achillion
PharmaceuticalsAchillion Pharmaceuticals,
Inc. (NASDAQ:ACHN) is a clinical-stage biopharmaceutical
company focused on advancing its oral, small molecule,
complement system inhibitors into late-stage development and
commercialization. Each of the product candidates in the
Company’s portfolio was discovered in its laboratories and is
wholly owned. Achillion is initially focusing its drug
development activities on alternative pathway-mediated diseases
where there are no approved therapies or where existing therapies
are inadequate for patients. Initial indications being evaluated
for its compounds include paroxysmal nocturnal hemoglobinuria
(PNH), C3 glomerulopathy (C3G), and immune complex
membranoproliferative glomerulonephritis (IC-MPGN). To advance its
investigational drugs into phase 3 and commercialization, the
Company plans to work closely with key stakeholders including
patients, payors, regulators and healthcare professionals.
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 also include statements about: the potential benefits of
factor D inhibition as a treatment for complement-mediated
diseases; the potential benefits of, and indications for,
Achillion’s compounds that inhibit factor D, including ACH-4471,
ACH-5228 and ACH-5548; 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
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; 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; 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 fiscal quarter ended September 30, 2018, 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.
Investors: Brian Di Donato Senior VP, Chief
Financial OfficerTel. 215-709-3032 bdidonato@achillion.com
Media:Susanne HeinzingerSenior VP, Corporate
CommunicationsTel. 215-709-3055sheinzinger@achillion.com
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ACHILLION PHARMACEUTICALS INC. (ACHN) |
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Statements of
Operations |
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|
|
(in thousands, except per share amounts) |
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|
Three Months
Ended |
|
Year
Ended |
|
December
31, |
|
December
31, |
|
|
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|
|
|
|
|
|
|
2018 |
|
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|
2017 |
|
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|
2018 |
|
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|
2017 |
|
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|
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|
|
Revenue |
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
|
|
|
|
|
|
|
Operating
expenses: |
|
|
|
|
|
|
|
Research
and development |
|
12,202 |
|
|
|
15,411 |
|
|
|
50,118 |
|
|
|
63,607 |
|
General
and administrative |
|
5,972 |
|
|
|
8,944 |
|
|
|
23,896 |
|
|
|
25,969 |
|
Restructuring charges |
|
|
|
- |
|
|
|
1,900 |
|
|
|
- |
|
|
|
|
|
|
|
|
|
Total
operating expenses |
|
18,174 |
|
|
|
24,355 |
|
|
|
75,914 |
|
|
|
89,576 |
|
|
|
|
|
|
|
|
|
Loss from
operations |
|
(18,174 |
) |
|
|
(24,355 |
) |
|
|
(75,914 |
) |
|
|
(89,576 |
) |
|
|
|
|
|
|
|
|
Other income
(expense): |
|
|
|
|
|
|
|
Interest
income |
|
1,585 |
|
|
|
1,165 |
|
|
|
5,678 |
|
|
|
4,390 |
|
Interest
expense |
|
(12 |
) |
|
|
(13 |
) |
|
|
(36 |
) |
|
|
(50 |
) |
|
|
|
|
|
|
|
|
Net loss |
$ |
(16,601 |
) |
|
$ |
(23,203 |
) |
|
$ |
(70,272 |
) |
|
$ |
(85,236 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share -
basic and diluted |
$ |
(0.12 |
) |
|
$ |
(0.17 |
) |
|
$ |
(0.51 |
) |
|
$ |
(0.62 |
) |
|
|
|
|
|
|
|
|
Wtd avg shares
outstanding - basic and diluted |
|
138,638 |
|
|
|
137,870 |
|
|
|
138,418 |
|
|
|
137,180 |
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Balance Sheets |
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(Unaudited, in thousands) |
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|
December
31, |
|
December
31, |
|
|
|
|
|
|
2018 |
|
|
|
2017 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash, cash equivalents,
and marketable securities |
$ |
270,977 |
|
|
$ |
330,585 |
|
|
|
|
|
Working capital |
|
263,551 |
|
|
|
291,054 |
|
|
|
|
|
Total assets |
|
277,858 |
|
|
|
337,613 |
|
|
|
|
|
Long-term
liabilities |
|
17 |
|
|
|
214 |
|
|
|
|
|
Total liabilities |
|
11,846 |
|
|
|
13,098 |
|
|
|
|
|
Total stockholders'
equity |
|
266,012 |
|
|
|
324,515 |
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