- Company provides update on HCV
collaboration with Janssen; Phase 2b combination study evaluating
doublet and triplet regimens for six and eight weeks anticipated to
begin in Q3 2016 –
Achillion Pharmaceuticals, Inc.
(Nasdaq:ACHN) today reported financial results for the three months
ended March 31, 2016. For the first quarter of 2016, the Company
reported a net loss of $18.1 million or $0.13 per share, compared
with a net loss of $19.3 million or $0.17 per share for the first
quarter of 2015. Cash, cash equivalents, marketable securities and
interest receivable as of March 31, 2016 were $444 million.
“In the first quarter of 2016, we continued to
progress our complement pipeline and to work with Janssen as they
advance our combined HCV pipeline under our global collaboration,”
said Milind Deshpande, Ph.D., President and Chief Executive Officer
of Achillion. “By the end of 2016, Achillion expects to report a
number of data points from both the HCV program and our ongoing
phase 1 trial with ACH-4471, our first orally-administered,
small-molecule complement factor D inhibitor drug candidate being
developed for rare diseases, including PNH and C3G.”
First Quarter 2016 Results
For the first quarter of 2016, the Company
reported a net loss of $18.1 million, or $0.13 per share, compared
with a net loss of $19.3 million, or $0.17 per share for the first
quarter of 2015. Cash, cash equivalents, marketable securities, and
interest receivable as of March 31, 2016 were $444 million.
Research and development expenses were $13.3
million in the first quarter of 2016, compared with $15.2 million
for the same period of 2015. The decrease was primarily due to
decreased manufacturing, clinical trial and consulting costs
related to our HCV compounds which were licensed to Janssen in
2015, offset by increased manufacturing and clinical costs related
to ACH-4471. Additionally, personnel costs increased due to the
addition of personnel in our discovery and development groups.
For the three months ended March 31, 2016,
general and administrative expenses totaled $5.4 million, compared
to $4.2 million for the same period in 2015, with the increase
primarily due to increased salaries and non-cash stock compensation
charges due to the addition of personnel.
Non-cash stock compensation expense totaled $3.0
million for the first quarter of 2016 as compared with $2.9 million
for the first quarter of 2015, and is included in research and
development and general and administrative expenses.
Update on world-wide collaboration with
Janssen for HCV
In May 2015, Achillion announced an exclusive
worldwide collaboration with Janssen Pharmaceuticals, Inc.
(Janssen), one of the Janssen Pharmaceutical Companies of Johnson
& Johnson, for the treatment of chronic hepatitis C viral
infection (HCV).
“Our collaboration with Janssen creates a
combined pipeline that has the potential to provide more effective
and shorter duration regimens for the treatment of HCV,” said Dr.
Deshpande. “In the coming months, we anticipate release of
data on both doublet and triplet treatment regimens from Janssen’s
ongoing phase 2a clinical trial.”
Update of HCV Clinical
Program
- Phase 2a randomized, open-label study to evaluate the
safety, pharmacokinetics and efficacy of the combination of AL-335,
odalasvir (ACH-3102), and simeprevir in treatment-naïve subjects
with genotype 1 chronic hepatitis CThe Janssen sponsored
phase 2a clinical trial evaluating all-oral regimens for durations
of eight weeks and less remains ongoing. This trial is evaluating a
triplet regimen, consisting of odalasvir, AL-335, and simeprevir,
for durations of eight and six weeks, as well as a doublet regimen,
consisting of odalasvir and AL-335, for eight weeks. Top line
results from this study are expected to be released in the third
quarter of the year.
- Phase 2b, multicenter, randomized, open-label study to
investigate the efficacy, safety and pharmacokinetics of different
treatment regimens of AL-335, odalasvir, and simeprevir in
treatment-naive and treatment-experienced subjects with chronic
hepatitis C virus genotype 1, 2, 3, 4, 5, and 6 infection, with and
without cirrhosisJanssen’s planned global phase 2b
clinical trial is expected to begin enrolling patients in the third
quarter of 2016. This phase 2b clinical trial is designed to
evaluate two regimens, a triplet (odalasvir, AL-335, and
simeprevir) regimen and a doublet regimen (odalasvir and AL-335),
for treatment durations of six and eight weeks in treatment-naïve
and treatment-experienced patients with genotypes 1 - 6 HCV,
including those patients with or without cirrhosis This trial is
expected to enroll approximately 400 patients.
Status of Complement Factor D Inhibitor
Program: Developing ACH-4471 for Rare Diseases
Dr. Deshpande further commented, “We have
synthesized more than 1,300 small molecule factor D inhibitor
compounds to date and continue to make significant progress with
our complement factor D program. This past February, we advanced
ACH-4471 into a phase 1 study in healthy volunteers, and are
working to advance ACH-4471 as a potential treatment for two
serious and rare diseases, PNH and C3G.”
Summary of ACH-4471 Clinical
Program
- Phase 1 Single Ascending Dose Study to Assess the
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of
ACH-4471 in Healthy VolunteersDuring the first quarter of
2016, Achillion initiated a first-in-human phase 1 clinical trial
to evaluate the safety, pharmacokinetics and pharmacodynamics of
ACH-4471, the Company’s first orally-administered, small molecule
inhibitor of complement factor D. Interim results from this study
are expected to be reported during the second quarter of 2016.
- Phase 1 Multiple Ascending Dose Study to Assess the
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of
ACH-4471 in Healthy VolunteersDuring the second quarter of
2016, Achillion anticipates starting a phase 1 multiple ascending
dose study with ACH-4471 evaluating up to 14 days of dosing in
healthy volunteers. Interim results from this study are expected to
be reported during the third quarter of 2016.
Developing ACH-4471 Complement-Based
Therapeutics for Rare Diseases
- PNH (Paroxysmal Nocturnal
Hemoglobinuria)Following the completion of phase 1 studies
in healthy volunteers, Achillion plans to initiate phase 2 trials
in patients with PNH evaluating both treatment-naïve patients and
patients who sub-optimally respond to current therapies. We
anticipate interim results in treatment-naïve patients by the end
of 2016.PNH is a rare, acquired, life-threatening disease
characterized by destruction of red blood cells (hemolytic anemia),
blood clots (thrombosis), impaired bone marrow function, and a risk
of developing leukemia. Preclinical studies suggest ACH-4471
inhibits factor D within the alternative pathway of the complement
cascade leading to blockade of C3 convertase
production. Furthermore, unlike C5 inhibitors, ACH-4471 is
also thought to prevent C3 fragment deposition on PNH cells and may
confer a pharmacological advantage by protecting PNH cells from
both intravascular and extravascular hemolysis.
- C3G (C3 Glomerulopathy)C3G is a rare renal
disease which is believed to be the result of over-activity of the
alternative pathway. As ACH-4471 has been shown in vitro to inhibit
alternative pathway activity, potentially decreasing the formation
of C3 protein fragments, the company plans to initiate a phase 2
study of ACH-4471 in C3G patients by the end of 2016. Achillion
also plans to support a natural history study of C3G with a leading
international research institution to add to the understanding of
this devastating disease.There is currently no cure available for
C3G, no approved treatment to prevent disease progression and a
poor prognosis for patients, of whom approximately 30-50% require
dialysis or transplant 10 years after diagnosis.
About HCV
The hepatitis C virus (HCV) is one of the most
common causes of viral hepatitis, which is an inflammation of the
liver. It is currently estimated that more than 150 million people
are infected with HCV worldwide including more than 5 million
people in the United States. Three-quarters of the HCV patient
population is undiagnosed; it is a silent epidemic and a major
global health threat. Chronic hepatitis, if left untreated, can
lead to permanent liver damage that can result in the development
of liver cancer, liver failure or death. Few therapeutic options
currently exist for the treatment of HCV infection.
About Complement Factor D
Platform
Achillion has leveraged its internal discovery
capabilities and a novel complement-related platform to develop
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 plays a critical
role. Potential indications being evaluated for these compounds
include paroxysmal nocturnal hemoglobinuria (PNH), C3
Glomerulopathy (C3G), dry age-related macular degeneration (dry
AMD), and chronic obstructive pulmonary disease (COPD). Achillion
anticipates that its platform could play a role in addressing the
needs of all PNH patients, including patients who have suboptimal
response to, or fail to respond to, the currently available
treatments, as well as for patients suffering from other
alternative pathway complement-mediated diseases. Achillion
nominated ACH-4471 for clinical development in December 2015, and
initiated clinical development in February 2016 with a phase 1
healthy volunteer trial assessing single-ascending doses of
ACH-4471.
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,”
“intend,” “plan,” “aim,” “believe,” “seek,” “ estimate,” “can,”
“focus,” “will,” “look forward,” “goal,” and “may” and similar
expressions to identify such forward-looking statements. These
forward-looking statements also include statements about: the
Company’s expected plans, timing, data readouts and results from
ongoing and planned clinical trials of both ACH-4471 and HCV
development candidates being advanced by Janssen under the
Company’s collaboration with Janssen; and statements concerning the
Company’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, including the current collaboration
with Janssen; 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. Furthermore,
because Janssen is solely responsible for the development and
commercialization of our HCV assets under the exclusive worldwide
license we granted to it and has the deciding vote on all
collaboration matters, Janssen generally has full discretion over
all development plans and strategies and may not advance the HCV
programs in the time frames Achillion or Janssen projects, or at
all, including with regard to the current and planned phase 2a and
phase 2b combination trials that include our licensed drug
candidates. These and other risks are described in the reports
filed by Achillion with the U.S. Securities and Exchange
Commission, including its Annual Report on Form 10-K for the
fiscal year ended December 31, 2015, 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.
-- Financial Tables Attached
--
|
|
|
|
ACHILLION PHARMACEUTICALS INC. (ACHN) |
|
|
|
Statements of
Operations |
|
|
|
(Unaudited, in thousands, except per share
amounts) |
|
|
|
|
|
|
|
|
Three Months
Ended |
|
March
31, |
|
|
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
|
Revenue |
$ |
- |
|
|
$ |
- |
|
|
|
|
|
Operating
expenses: |
|
|
|
Research and development |
|
13,278 |
|
|
|
15,156 |
|
General and administrative |
|
5,440 |
|
|
|
4,243 |
|
Total operating expenses |
|
18,718 |
|
|
|
19,399 |
|
|
|
|
|
Loss from
operations |
|
(18,718 |
) |
|
|
(19,399 |
) |
|
|
|
|
Other income
(expense): |
|
|
|
Interest income |
|
679 |
|
|
|
152 |
|
Interest expense |
|
(15 |
) |
|
|
(16 |
) |
|
|
|
|
Net loss |
$ |
(18,054 |
) |
|
$ |
(19,263 |
) |
|
|
|
|
|
|
|
|
Net loss per share -
basic and diluted |
$ |
(0.13 |
) |
|
$ |
(0.17 |
) |
|
|
|
|
Weighted average shares
outstanding - basic and diluted |
|
136,640 |
|
|
|
111,202 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance Sheets |
|
|
|
(Unaudited, in thousands) |
|
|
|
|
|
|
|
|
March
31, |
|
December
31, |
|
|
2016 |
|
|
|
2015 |
|
|
|
|
|
Cash, cash equivalents,
marketable securities and interest receivable |
$ |
444,033 |
|
|
$ |
460,540 |
|
Working capital |
|
429,578 |
|
|
|
447,930 |
|
Total assets |
|
448,656 |
|
|
|
464,525 |
|
Long-term
liabilities |
|
325 |
|
|
|
231 |
|
Total liabilities |
|
13,788 |
|
|
|
14,889 |
|
Total stockholders'
(deficit) equity |
|
434,868 |
|
|
|
449,636 |
|
|
|
|
|
Investors:
Glenn Schulman
Senior Director, Investor Relations
Achillion Pharmaceuticals, Inc.
Tel. (203) 624-7000
gschulman@achillion.com
Media:
Liz Power
Senior Director, Public Relations
Achillion Pharmaceuticals, Inc.
Tel: (203) 752-5509
lpower@achillion.com
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