Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today reported financial results for the three months ended
September 30, 2017. For the third quarter of 2017, Achillion
reported a net loss of $19.3 million or $0.14 per share, compared
with a net loss of $20.7 million or $0.15 per share for the third
quarter of 2016. Cash, cash equivalents, marketable securities, and
interest receivable as of September 30, 2017 were $353.5 million.
"Throughout 2017, our focus has been on
executing on an aggressive global development program aimed at
establishing biologic activity and evidence of potential efficacy
of factor D inhibition as a novel approach to treating
complement-mediated diseases. With the positive interim data
reported in August from our phase II clinical trial of ACH-4471 for
patients with untreated PNH, we now look forward to reporting
initial data from our phase II trial for C3G before the end of the
year,” commented Milind Deshpande, Ph.D., President and Chief
Executive Officer of Achillion. “Over the coming months, the
Achillion team plans to launch additional phase II trials of
ACH-4471 for PNH, C3G, and IC-MPGN, as well as to evaluate
potential extended release formulations of ACH-4471 in phase I by
year-end. In parallel, we look forward to advancing ACH-5228, our
next-generation factor D inhibitor, into a phase I clinical trial
by the end of 2017.”
Third Quarter Results
For the three months ended September 30, 2017,
Achillion reported a net loss of $19.3 million compared with a net
loss of $20.7 million during the same period of 2016.
Research and development expenses were $15.6
million for the three months ended September 30, 2017, compared
with $16.7 million for the same period of 2016. The decrease for
the three months ended September 30, 2017 was primarily due to
decreased preclinical and manufacturing costs related to ACH-4471,
as well as decreased legal fees. These amounts were partially
offset by increased clinical trial costs related to ACH-4471 and
preclinical costs related to ACH-5228.
General and administrative expenses were $4.8
million for the three months ended September 30, 2017, and $4.8
million during the same period in 2016.
Non-cash stock compensation expense totaled $2.4
million for the third quarter of 2017 as compared with $2.7 million
for the third quarter of 2016 and is included in research and
development and general and administrative expenses.
Dr. Deshpande further commented, “In October,
Janssen reported that 98.9% of non-cirrhotic patients with HCV
genotypes 1, 2, 4, 5 and 6 achieved SVR12 after receiving just six
weeks of once daily treatment JNJ-4178, the triple combination of
odalasvir, simeprevir, and AL-335, in the OMEGA-1 phase IIb
clinical trial. In light of the efficacy demonstrated in OMEGA-1,
we remain disappointed that the JNJ-4178 regimen is no longer being
advanced and we continue to believe that odalasvir is a
best-in-class NS5A inhibitor that demonstrates the strength of
Achillion’s internal discovery capabilities.”
Nine Month Results
For the nine months ended September 30, 2017,
Achillion reported a net loss of $62.0 million, compared to a net
loss of $57.3 million in the same period in 2016.
Research and development expenses were $49.4
million and $44.1 million for the nine months ended
September 30, 2017 and 2016, respectively. The increase for
the nine months ended September 30, 2017 was primarily due to
increased clinical trial costs related to ACH-4471 combined with
increased preclinical and manufacturing costs for ACH-5228.
General and administrative expenses in the
period ending September 30, 2017 were $15.9 million, compared with
$15.4 million incurred during the same period in 2016. The increase
for the nine months ended September 30, 2017 was primarily due
to increased marketing-related consulting fees, corporate legal
fees, and software license fees, partially offset by a decrease in
corporate taxes.
Non-cash stock compensation expense totaled $8.4
million for the nine months ended September 30, 2017 as compared
with $8.3 million for the same period in 2016, and is included in
research and development and general and administrative
expenses.
Complement Factor D
Platform
“ACH-4471 is the first factor D inhibitor to
show evidence of complement alternative pathway inhibition in
humans after oral dosing,” said Dr. Deshpande. “Through pioneering
research in complement biology, and data emerging from the ACH-4471
clinical program, we are gaining important insights into PK/PD
relationships as well as in vivo biomarkers to guide development in
patients. Upcoming presentations at ASH, we believe, will highlight
important research with ACH-4471, complement biology, and the
potential of factor D inhibition.”
ACH-4471: Expanding Clinical Development
Program
“In the United States, we currently have open
INDs for PNH and C3G, and are actively working to expand our
clinical trial footprint beyond our ongoing trials in Australia and
New Zealand into Europe,” stated Dr. David Apelian, M.D., Ph.D.,
M.B.A., Executive Vice President and Chief Medical Officer at
Achillion. “As a first-in-class oral factor D inhibitor, we believe
we are making great strides in understanding the role of AP
modulation by ACH-4471 and the potential for this approach in
patients with PNH and for patients with either C3G or IC-MPGN.”
PNH: Phase II trials of ACH-4471 monotherapy for
untreated PNH
A phase II clinical trial evaluating ACH-4471
monotherapy for patients with untreated PNH remains open with a
target enrollment of 12 patients anticipated. To date, four PNH
patients have been treated, three of whom have completed the
initial three-month trial (-100 study) and have entered the
long-term extension trial (-103 study). As previously reported in
August 2017, a fourth patient voluntarily withdrew from the trial
on day 41 for reasons unrelated to safety or efficacy. Interim data
from this first-in-patient trial indicated that ACH-4471
mechanistically inhibited factor D, its intended target, and
meaningfully improved LDH, hemoglobin, fatigue score, and other
measures of response, including PNH clone size. Updated interim
results are expected to be reported before the end of the year.
(Clintrials.gov: NCT03053102/EudraCT:
2016-002652-25; and Clintrials.gov: NCT03181633/EudraCT:
2017-000665-79)
PNH: Phase II trial add-on trial of ACH-4471 for
partial responders
During the first half of 2018, Achillion expects
to begin enrolling up to 12 PNH patients in a phase II trial
evaluating ACH-4471 in PNH patients currently receiving eculizumab,
a therapy for PNH that is marketed by a third-party, and who remain
transfusion-dependent. This trial is designed to evaluate 6 months
of treatment with ACH-4471 plus eculizumab with the potential for
patients to transition to a long-term treatment extension.
C3G / IC-MPGN: Phase II 14-day
proof-of-mechanism trial of ACH-4471
In September 2017, Achillion initiated patient
enrollment in a phase II clinical trial of ACH-4471 for patients
with either C3G or IC-MPGN. This trial is designed to evaluate the
effect of factor D inhibition on the complement pathway and will
enroll up to 10 patients. Measurement of complement proteins,
including C3, a complement protein in blood plasma that is
typically low in C3G and IC-MPGN patients, and C3 fragments, which
are typically high in these patients, will be evaluated. Other
measures of kidney function, safety, and tolerability are also
being assessed. Per the protocol design, two patients have been
enrolled into Group 1 and treated with 100 mg three times daily
(TID) for 14 days. Up to eight additional patients are targeted for
enrollment into Group 2. Interim results from Group 1 are expected
to be reported by year-end 2017.
(Clintrials.gov: NCT03124368 /EudraCT:
2016-003525-42)
C3G / IC-MPGN: Phase II 12-month open-label
trial of ACH-4471
Achillion also plans to conduct a phase II
open-label, 12-month treatment trial for patients with
biopsy-confirmed C3G or IC-MPGN. All patients enrolled will receive
treatment with ACH-4471 with periodic assessment of clinical
endpoints including proteinuria and estimated glomerular filtration
rate (eGFR). Up to 20 patients are expected to be enrolled.
Enrollment is expected to be initiated in the first half of
2018.
C3G: Phase II 6-month randomized,
placebo-controlled trial of ACH-4471
During the first half of 2018, Achillion plans
to initiate a randomized, placebo-controlled, double-blinded phase
II trial evaluating the efficacy and safety of ACH-4471 in patients
with C3G. The trial is expected to assess renal biopsy findings and
clinical endpoints such as proteinuria and eGFR. Up to twenty
patients are expected to be enrolled.
Extended Release: Phase I bioavailability
trial
By the end of 2017, Achillion plans to initiate
a phase I trial evaluating potential extended release formulations
of ACH-4471 in healthy volunteers.
ACH-5228: Next-generation Oral Factor D
Inhibitor
ACH-5228 is a next-generation oral factor D
inhibitor that is expected to enter phase I clinical development by
the end of 2017. In preclinical studies, ACH-5228 had a two- to
three-fold increase in potency and improved pharmacokinetic
characteristics relative to ACH-4471.
Next-generation Factor D Inhibitors for
Ophthalmology
Achillion has selected several compounds from
its discovery library for properties that may be advantageous for
delivery of a factor D inhibitor to the back of the eye for the
potential treatment of geographic atrophy (GA) with the goal of
achieving treatment duration of three months or longer. Achillion
is advancing several of these compounds in preclinical studies, as
well as several delivery technologies, to optimize treatment
duration, and anticipates selecting one or more lead compounds and
delivery technologies during the first half of 2018.
About the Achillion Complement Factor D
Platform
Achillion has leveraged its internal discovery
capabilities and a novel complement-related platform to develop
small molecule drug candidates that are oral inhibitors of
complement factor D. Factor D is an essential serine protease
involved in the complement pathway, a part of the innate immune
system. Achillion's complement platform is focused on seeking to
advance small molecule compounds that inhibit factor D and can
potentially be used in the treatment of immune-related diseases in
which complement alternative pathway plays a critical role.
Potential indications being evaluated for these compounds include
paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G),
immune complex-mediated membranoproliferative glomerulonephritis
(IC-MPGN), and geographic atrophy (GA).
About Achillion
Pharmaceuticals
Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is
a science-driven, patient-focused company seeking to leverage its
strengths across the continuum from discovery to commercialization
in its goal of providing better treatments for people with serious
diseases. The company employs a highly-disciplined discovery and
development approach that has allowed it to pursue best-in-class
oral antiviral therapy for chronic hepatitis C (HCV) and build a
platform of potent and specific complement inhibitors. Achillion is
rapidly advancing its efforts to become a fully-integrated
pharmaceutical company with a goal of bringing life-saving
medicines to patients with rare diseases. More information is
available at http://www.achillion.com.
Cautionary Note Regarding
Forward-Looking Statements
This 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,” “goal,” and “may”
and similar expressions to identify such forward-looking
statements. These forward-looking statements also include
statements about: Achillion’s expected plans, timing, data readouts
and results from ongoing and planned clinical trials of both
ACH-4471 and ACH-5228; the planned advancement of Achillion’s other
small molecule factor D inhibitors, including those for the
treatment of geographic atrophy; and statements concerning
Achillion’s strategic goals, milestone 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:
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; obtain and maintain
patent protection for its drug candidates and the freedom to
operate under third party intellectual property; demonstrate in any
current and future clinical trials the requisite safety, efficacy
and combinability of its drug candidates; obtain and maintain
necessary regulatory approvals; establish commercial manufacturing
arrangements; identify, enter into and maintain collaboration
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 June 30, 2017, and its subsequent SEC
filings.
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 & Media:Glenn
Schulman, PharmD, MPHExecutive Director, Investor
RelationsAchillion Pharmaceuticals, Inc.Tel. (203)
752-5510gschulman@achillion.com
-- Financial Tables Attached
--
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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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Nine Months
Ended |
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September
30, |
|
September
30, |
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2017 |
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2016 |
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2017 |
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2016 |
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Revenue |
$ |
- |
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|
$ |
- |
|
|
$ |
- |
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|
$ |
- |
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Operating
expenses: |
|
|
|
|
|
|
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Research
and development |
|
15,620 |
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|
|
16,701 |
|
|
|
49,368 |
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|
|
44,133 |
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|
General
and administrative |
|
4,843 |
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|
|
4,848 |
|
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|
15,853 |
|
|
|
15,443 |
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Total
operating expenses |
|
20,463 |
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|
21,549 |
|
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|
65,221 |
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|
59,576 |
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Loss from
operations |
|
(20,463 |
) |
|
|
(21,549 |
) |
|
|
(65,221 |
) |
|
|
(59,576 |
) |
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|
|
|
|
|
|
|
Other income
(expense): |
|
|
|
|
|
|
|
|
Interest
income |
|
1,133 |
|
|
|
846 |
|
|
|
3,225 |
|
|
|
2,353 |
|
|
Interest
expense |
|
(8 |
) |
|
|
(27 |
) |
|
|
(37 |
) |
|
|
(54 |
) |
|
Other
Income |
|
- |
|
|
|
- |
|
|
|
- |
|
|
|
- |
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|
Net loss |
$ |
(19,338 |
) |
|
$ |
(20,730 |
) |
|
$ |
(62,033 |
) |
|
$ |
(57,277 |
) |
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Net loss per share -
basic and diluted |
$ |
(0.14 |
) |
|
$ |
(0.15 |
) |
|
$ |
(0.45 |
) |
|
$ |
(0.42 |
) |
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|
Weighted average shares
outstanding - basic and diluted |
|
137,375 |
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|
136,681 |
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|
136,947 |
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|
136,647 |
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Balance Sheets |
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(Unaudited, in thousands) |
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|
September
30, |
|
December
31, |
|
|
|
|
|
2017 |
|
|
|
2016 |
|
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|
Cash, cash equivalents,
marketable securities and interest receivable |
$ |
353,499 |
|
|
$ |
392,486 |
|
|
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Working capital |
|
312,570 |
|
|
|
368,564 |
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Total assets |
|
360,442 |
|
|
|
413,875 |
|
|
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Long-term
liabilities |
|
269 |
|
|
|
450 |
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Total liabilities |
|
14,705 |
|
|
|
14,421 |
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Total stockholders'
equity |
|
345,737 |
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|
399,454 |
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