UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of
the Securities and Exchange Act of 1934
| Date of Report (Date of earliest event reported): June 10, 2010 (June 9, 2010) |
| REGENERON PHARMACEUTICALS, INC. |
| (Exact name of registrant as specified in its charter) |
| New York | 000-19034 | 13-3444607 | ||||||||
| (State or other jurisdiction of | (Commission File Number) | (I.R.S. Employer | ||||||||
| incorporation) | Identification Number) | |||||||||
| 777 Old Saw Mill River Road, Tarrytown, New York 10591-6707 |
| (Address of principal executive offices) (Zip Code) |
| (914) 347-7000 | ||
| (Registrant's telephone number, including area code) | ||
Check the appropriate
box below if the Form 8-K filing is intended to simultaneously satisfy the
filing obligation of registrant under any of the following
provisions:
| c | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) | |
| c | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) | |
| c | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) | |
| c | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
| Item 8.01 | Other Events | |
|
On June 9, 2010,
Regeneron Pharmaceuticals, Inc. issued a press release announcing results
of two Phase 3 studies evaluating the efficacy and safety of ARCALYST®
(rilonacept) in two different gout settings. A copy of this press release
is attached as Exhibit 99(a) to this Form 8-K and is incorporated herein
by reference.
On June 9, 2010,
Regeneron’s President and Chief Executive Officer, Dr. Leonard Schleifer,
and other members of senior management of Regeneron hosted a webcast
conference call to discuss the findings of the Phase 3 studies evaluating
the efficacy and safety of ARCALYST® (rilonacept) in two different gout
settings. The slides for this webcast are furnished as Exhibit 99(b) to
this Form 8-K.
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|
Item 9.01
|
Financial Statements and
Exhibits
|
|
|
(c) Exhibits
99(a) Press release dated June
9, 2010 announcing the results of two Phase 3 studies evaluating the
efficacy and safety of ARCALYST® (rilonacept) in two different gout
settings.
99(b) Slides for June 9, 2010
webcast to discuss the findings of two Phase 3 studies evaluating the
efficacy and safety of ARCALYST® (rilonacept) in two different gout
settings.
|
Pursuant to the requirements of the Securities and Exchange Act of 1934,
the registrant has duly caused this report to be signed on its behalf by the
undersigned hereunto duly authorized.
| REGENERON PHARMACEUTICALS, INC. | ||||
| Dated: June 10, 2010 | By: | /s/ Stuart Kolinski | ||
| Stuart Kolinski | ||||
| Senior Vice President and General Counsel | ||||
2
Exhibit Index
| Number | Description | ||
|
99(a)
|
Press release
dated June 9, 2010 announcing results of two Phase 3 studies evaluating
the efficacy and safety of ARCALYST® (rilonacept) in two different gout
settings.
|
||
|
99(b)
|
Slides for June 9,
2010 webcast to discuss the findings of two Phase 3 studies evaluating the
efficacy and safety of ARCALYST® (rilonacept) in two different gout
settings.
|
||
3
Exhibit 99(a)
| Press Release |
ARCALYST® (rilonacept) Meets Primary and All Secondary Endpoints in Phase 3 Trial
of Prevention of Gout Flares in Patients Initiating Allopurinol
Therapy
- Regeneron plans to file by
mid-2011 for regulatory approval for ARCALYST in this setting assuming
positive results from two ongoing studies
- Another Phase 3 trial in a different gout setting, where patients were in the midst of an acute gout attack, showed that ARCALYST did not significantly improve pain relief when added to indomethacin
Tarrytown, NY (June 9, 2010)
-- Regeneron
Pharmaceuticals, Inc. (Nasdaq: REGN) today
announced that a Phase 3 study in gout patients initiating allopurinol therapy
to lower their uric acid levels showed that ARCALYST (rilonacept), also known as
IL-1 Trap, prevented gout attacks, as measured by the primary endpoint of the
number of gout flares per patient over the 16 week treatment
period.
- Primary Endpoint:
- Patients who received ARCALYST at a weekly, self-administered, subcutaneous dose of 160 milligrams (mg) had an 80% decrease in mean number of gout flares compared to the placebo group over the 16 week treatment period (0.21 flares vs. 1.06 flares, p<0.0001).
- Patients who received ARCALYST at a weekly dose of 80 mg had a 73% decrease compared to the placebo group (0.29 flares vs. 1.06 flares, p<0.0001).
- Key Secondary Endpoints: All secondary endpoints of the study were highly positive (p<0.001 vs. placebo). These include:
- Treatment with ARCALYST reduced the proportion of patients who experienced two or more flares during the study period by up to 88% (3.7% with ARCALYST 160 mg, 5.0% with ARCALYST 80 mg, and 31.6% with placebo, p<0.0001).
- Treatment with ARCALYST reduced the proportion of patients who experienced at least one gout flare during the study period by up to 65% (16.3% with ARCALYST 160 mg, 18.8% with, ARCALYST 80 mg, and 46.8% with placebo, p<0.001).
ARCALYST was generally
well tolerated with no reported drug-related serious adverse events. Injection
site reaction, generally considered mild, was the most commonly reported adverse
event with ARCALYST.
“Gout is a very painful
and common form of arthritis that results from high levels of uric acid. Uric
acid-lowering therapy, most commonly with allopurinol, is a mainstay of
treatment to reduce gout flares over the long term. Paradoxically, the
initiation of uric acid-lowering therapy often triggers an increase in frequency
of gout attacks in the first several months of treatment that may lead to discontinuation of therapy”,
stated George D. Yancopoulos, M.D., Ph.D., President of Regeneron Research
Laboratories. “This positive pivotal study showed that ARCALYST® (rilonacept) markedly reduced the occurrence
of painful gout attacks in patients initiating uric acid-lowering therapy. We
look forward to data from our second efficacy study in this setting and our
larger safety study. If these additional studies are successful, we plan to file
for regulatory approval by mid-2011.”
"Chronic urate-lowering
therapy is critical to control the symptoms and the resulting consequences of
gout in the joints. We in the rheumatology community have long recognized the
challenge of adherence to uric acid-lowering therapies. Data suggest that many
patients discontinue allopurinol within the first few months of therapy, in part
due to increased flares," said H. Ralph Schumacher, M.D., Professor of Medicine,
University of Pennsylvania, Philadelphia, PA. "Current therapies recommended to
reduce the risk of gout flares in patients taking uric acid-lowering therapy are
under-prescribed, especially outside of rheumatology practice. While additional
Phase 3 data are needed, the results from this study suggest that concomitant
use of rilonacept during the first several months of uric acid-lowering therapy
may help avoid gout flares, which could, in turn, improve patient outcomes."
Results of a Phase 3
study in patients presenting with an ongoing acute gout flare showed that
compared to indomethacin, a non-steroidal anti-inflammatory drug considered a
standard of care, there was no significant benefit from combining indomethacin
with ARCALYST, as measured by the primary endpoint of the average intensity of
gout pain from 24 to 72 hours after initiation of treatment. Patients treated
with indomethacin alone experienced an average reduction in patient-reported
pain scores (0 to 4 Likert scale where 0 represents no pain and 4 represents
extreme pain) of 1.40 points from baseline compared to an average reduction of
1.55 points from baseline in patients treated with both indomethacin and
ARCALYST (p=0.33). Patients who received ARCALYST alone experienced an average
pain reduction of 0.69 points. Treatment with ARCALYST was generally well
tolerated with no reported drug-related serious adverse events. The most
commonly reported adverse event with ARCALYST was headache.
About the Phase 3 Gout Flare Prevention
Study
The North American-based PRE-SURGE 1 (PREventative Study against URate-lowering drug-induced Gout Exacerbations) study was a double-blind, placebo-controlled study which evaluated the number of gout flares per patient over the first 16 weeks following initiation of allopurinol therapy. In the trial, a gout flare was defined as patient-reported acute articular pain typical of a gout attack that is deemed (by patient and/or investigator) to require treatment with an anti-inflammatory therapeutic; presence of at least three of the following four signs/symptoms: joint swelling, redness, tenderness and pain, and at least one of the following: rapid onset of pain, decreased range of motion, joint warmth or other symptoms similar to a prior gout flare. A total of 241 patients were randomized on a 1:1:1 basis to receive one of the following treatment regimens:
The North American-based PRE-SURGE 1 (PREventative Study against URate-lowering drug-induced Gout Exacerbations) study was a double-blind, placebo-controlled study which evaluated the number of gout flares per patient over the first 16 weeks following initiation of allopurinol therapy. In the trial, a gout flare was defined as patient-reported acute articular pain typical of a gout attack that is deemed (by patient and/or investigator) to require treatment with an anti-inflammatory therapeutic; presence of at least three of the following four signs/symptoms: joint swelling, redness, tenderness and pain, and at least one of the following: rapid onset of pain, decreased range of motion, joint warmth or other symptoms similar to a prior gout flare. A total of 241 patients were randomized on a 1:1:1 basis to receive one of the following treatment regimens:
- ARCALYST 160 mg as an initial subcutaneous loading dose, followed by weekly 80 mg subcutaneous injections (n=80)
- ARCALYST 320 mg as an initial subcutaneous loading dose, followed by weekly 160 mg subcutaneous injections (n=81)
- Subcutaneous weekly placebo injections (n=80)
Adverse event that
occurred at a frequency of at least 5% in any study group were: injection site
reaction (19.8 % with ARCALYST® (rilonacept) 160 mg, 8.8 % with ARCALYST 80
mg, and 1.3 % with placebo), upper respiratory tract infection (9.9% with
ARCALYST 160 mg, 8.8% with ARCALYST 80 mg, and 7.6% with placebo), lower
respiratory tract infection (0% with ARCALYST 160 mg, 5.0 % with ARCALYST 80 mg,
and 2.5% with placebo), musculoskeletal pain/ discomfort (6.2 % with ARCALYST
160 mg, 7.5 % with ARCALYST 80 mg, and 8.9 % with placebo), and headache, (3.7%
with ARCALYST 160 mg, 6.3% with ARCALYST 80 mg, and 1.3% with placebo).
Detailed data from this
study will be presented at future scientific conferences.
About the Acute Gout Flare Treatment
Study
The North-American-based
SURGE (Study Utilizing
Rilonacept in Gout Exacerbations)
study was a double blind, placebo-controlled, Phase 3 study that evaluated pain
during the initial 72 hours of treatment in patients experiencing an acute gout
attack. A total of 225 patients were randomized on a 1:1:1 basis to receive one
of the following treatment regimens:
- ARCALYST 320 mg administered by subcutaneous injection on day 1 plus oral placebo taken for 3 days or more (n=76)
- ARCALYST 320 mg administered by subcutaneous injection on day 1 plus oral indomethacin (an anti-inflammatory drug currently indicated for the treatment of gout) taken for 3 days or more (n=74)
- Placebo administered by subcutaneous injection on day 1 plus oral indomethacin taken for 3 days or more (n=75)
Adverse events reported
at an incidence of at least 5% in any group were headache (7.8% indomethacin
alone, 5.5% with indomethacin plus ARCALYST, and 10.8% with ARCALYST alone) and
neurological signs and symptoms (dizziness; 5.2% with indomethacin alone, 4.1%
with indomethacin plus ARCALYST, and 2.7% with ARCALYST alone).
Detailed data from this
study will be presented at future scientific conferences.
About the Additional Phase 3 Gout
Studies
The ongoing studies in
the Phase 3 program with ARCALYST in gout include:
- The global PRE-SURGE 2 (PREventative Study against URate-lowering drug-induced Gout Exacerbations) study evaluating the number of gout flares per patient over the first 16 weeks of initiation of allopurinol therapy. PRE-SURGE 2, which has a similar trial design as PRE-SURGE 1, is over 80% enrolled and data is expected in early 2011. A total of 240 patients will be randomized on a 1:1:1 basis to receive one of the following treatment regimens:
- ARCALYST 160 mg as an initial loading dose, followed by weekly 80 mg subcutaneous injections
- ARCALYST 320 mg as an initial loading dose, followed by weekly 160 mg subcutaneous injections
- Weekly placebo injections
- The global RE-SURGE (REview of Safety Using Rilonacept in preventing Gout Exacerbations) study, evaluating the safety of ARCALYST® (rilonacept) versus placebo over 16 weeks in patients who are at risk for gout flares because they are taking uric acid-lowering drug treatment. RE-SURGE is over 80% enrolled and data is expected in early 2011. Over 1000 patients will be randomly allocated in a 1:3 ratio to receive weekly placebo or ARCALYST dosed at 320 mg as an initial loading dose, followed by weekly 160 mg subcutaneous injections. Patients can be taking any of four uric acid-lowering drugs, allopurinol, febuxostat, probenecid, or sulfinpyrazone, with no requirements in the study design as to the total number of patients taking each.
Conference Call
Regeneron will host a
webcast conference call to discuss these results today, June 9, 2010, at 8:30 a.m., Eastern Time. The dial-in information is:
Domestic Dial-in Number:
(877) 390-5538
International Dial-in Number: (408) 940-3843
Participant Passcode: 80129194
International Dial-in Number: (408) 940-3843
Participant Passcode: 80129194
The live conference call
is being webcast and it, and slides for the conference call, can be accessed on
the “Newsroom” page of the Company’s website, www.regeneron.com. The webcast
will be available for 30 days following the call.
About Gout
Gout is a condition that occurs when the bodily waste product, uric acid, is deposited in the joints and/or soft tissues. In the joints, these uric acid crystals cause inflammation, which leads to pain, swelling, redness, heat, and stiffness in the joints. Treatment guidelines recommend that patients with elevated uric acid levels who experience multiple gout attacks each year should receive chronic uric acid-lowering therapy, such as allopurinol. Allopurinol reduces the production of uric acid in the body to prevent the occurrence of gout attacks with long-term use. Approximately 750,000 gout patients initiate allopurinol therapy each year. During the first months of allopurinol therapy while uric acid blood levels are being reduced, the break up of the uric acid crystals can result in stimulation of inflammatory mediators, including interleukin-1 (IL-1), resulting in acute flares of joint pain and inflammation. Anti-inflammatory therapy with colchicine is sometimes used to help prevent these flares. However, the side effects associated with colchicine, which include diarrhea, abdominal cramps, nausea, and vomiting, can limit patients’ adherence to both colchicine and allopurinol treatment.
Gout is a condition that occurs when the bodily waste product, uric acid, is deposited in the joints and/or soft tissues. In the joints, these uric acid crystals cause inflammation, which leads to pain, swelling, redness, heat, and stiffness in the joints. Treatment guidelines recommend that patients with elevated uric acid levels who experience multiple gout attacks each year should receive chronic uric acid-lowering therapy, such as allopurinol. Allopurinol reduces the production of uric acid in the body to prevent the occurrence of gout attacks with long-term use. Approximately 750,000 gout patients initiate allopurinol therapy each year. During the first months of allopurinol therapy while uric acid blood levels are being reduced, the break up of the uric acid crystals can result in stimulation of inflammatory mediators, including interleukin-1 (IL-1), resulting in acute flares of joint pain and inflammation. Anti-inflammatory therapy with colchicine is sometimes used to help prevent these flares. However, the side effects associated with colchicine, which include diarrhea, abdominal cramps, nausea, and vomiting, can limit patients’ adherence to both colchicine and allopurinol treatment.
Rationale for the Clinical Exploration of Use
of ARCALYST in the Treatment of Gout
Interleukin-1 (IL-1) is a protein secreted by infection-fighting cells in the blood and tissues. In many cases, IL-1 acts as a messenger to help regulate immune and inflammatory responses by attaching to cell-surface receptors in cells that participate in the body’s immune system. In excess, it can be harmful and has been shown to be a key driver of inflammation in a variety of diseases, including gout. In gout, uric acid crystals stimulate the production of IL-1, which causes an inflammatory response in the joints and surrounding tissues.
Interleukin-1 (IL-1) is a protein secreted by infection-fighting cells in the blood and tissues. In many cases, IL-1 acts as a messenger to help regulate immune and inflammatory responses by attaching to cell-surface receptors in cells that participate in the body’s immune system. In excess, it can be harmful and has been shown to be a key driver of inflammation in a variety of diseases, including gout. In gout, uric acid crystals stimulate the production of IL-1, which causes an inflammatory response in the joints and surrounding tissues.
Rilonacept is an agent
that inhibits IL-1. It is designed to attach to and neutralize IL-1 in the blood
stream before the IL-1 can attach to cell-surface receptors and generate signals
that can trigger disease activity in body tissue. Once attached to rilonacept,
IL-1 cannot bind to the cell-surface receptors and is eventually eliminated from
the body.
Important Information About ARCALYST® (rilonacept)
Rilonacept, marketed as ARCALYST, is currently indicated in the U.S. for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 and older. Rilonacept is also approved, but not marketed, in the E.U. for the same patient population. The safety and efficacy of ARCALYST in the gout setting have not been evaluated by the Food and Drug Administration. ARCALYST is not approved for use in gout.
Rilonacept, marketed as ARCALYST, is currently indicated in the U.S. for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 and older. Rilonacept is also approved, but not marketed, in the E.U. for the same patient population. The safety and efficacy of ARCALYST in the gout setting have not been evaluated by the Food and Drug Administration. ARCALYST is not approved for use in gout.
IL-1 blockade may
interfere with immune response to infections. Serious, life-threatening
infections have been reported in patients taking rilonacept. Rilonacept should
be discontinued if a patient develops a serious infection. Taking rilonacept
with tumor necrosis factor inhibitors is not recommended because this may
increase the risk of serious infections. Treatment with rilonacept should not be
initiated in patients with active or chronic infections. Patients should not
receive a live vaccine while taking rilonacept. It is recommended that patients
receive all recommended vaccinations prior to initiation of treatment with
rilonacept. Patients should be monitored for changes in their lipid profiles and
provided with medical treatment if warranted. Hypersensitivity reactions
associated with rilonacept administration have been rare. Please see the full
Prescribing Information for ARCALYST, available online at
www.regeneron.com/ARCALYST-fpi.pdf.
About Regeneron
Pharmaceuticals
Regeneron is a fully integrated biopharmaceutical company that discovers, develops, and commercializes medicines for the treatment of serious medical conditions. In addition to ARCALYST® (rilonacept) Injection for Subcutaneous Use, its first commercialized product, Regeneron has therapeutic candidates in Phase 3 clinical trials for the potential treatment of gout, diseases of the eye (wet age-related macular degeneration and central retinal vein occlusion), and certain cancers. Additional therapeutic candidates are in earlier stage development programs in rheumatoid arthritis and other inflammatory conditions, pain, cholesterol reduction, allergic and immune conditions, and cancer. Additional information about Regeneron and recent news releases are available on Regeneron’s web site at www.regeneron.com.
Regeneron is a fully integrated biopharmaceutical company that discovers, develops, and commercializes medicines for the treatment of serious medical conditions. In addition to ARCALYST® (rilonacept) Injection for Subcutaneous Use, its first commercialized product, Regeneron has therapeutic candidates in Phase 3 clinical trials for the potential treatment of gout, diseases of the eye (wet age-related macular degeneration and central retinal vein occlusion), and certain cancers. Additional therapeutic candidates are in earlier stage development programs in rheumatoid arthritis and other inflammatory conditions, pain, cholesterol reduction, allergic and immune conditions, and cancer. Additional information about Regeneron and recent news releases are available on Regeneron’s web site at www.regeneron.com.
Forward Looking
Statement
This news release discusses historical information and includes forward-looking statements about Regeneron and its products, development programs, finances, and business, all of which involve a number of risks and uncertainties. These include, among others, risks and timing associated with preclinical and clinical development of ARCALYST®, determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize ARCALYST, competing drugs that are superior to ARCALYST, risks associated with the ability to market and sell ARCALYST, uncertainty of market acceptance of ARCALYST, uncertainty concerning the ability of Regeneron to obtain third party coverage and reimbursement for ARCALYST, unanticipated expenses, the availability and cost of capital, the costs of developing, producing, and selling products, and risks associated with third party intellectual property. A more complete description of these and other material risks can be found in Regeneron’s filings with the United States Securities and Exchange Commission (SEC), including its Form 10-K for the year ended December 31, 2009 and Form 10-Q for the quarter ended March 31, 2010. Regeneron does not undertake any obligation to update publicly any forward-looking statement, whether as a result of new information, future events, or otherwise, unless required by law.
This news release discusses historical information and includes forward-looking statements about Regeneron and its products, development programs, finances, and business, all of which involve a number of risks and uncertainties. These include, among others, risks and timing associated with preclinical and clinical development of ARCALYST®, determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize ARCALYST, competing drugs that are superior to ARCALYST, risks associated with the ability to market and sell ARCALYST, uncertainty of market acceptance of ARCALYST, uncertainty concerning the ability of Regeneron to obtain third party coverage and reimbursement for ARCALYST, unanticipated expenses, the availability and cost of capital, the costs of developing, producing, and selling products, and risks associated with third party intellectual property. A more complete description of these and other material risks can be found in Regeneron’s filings with the United States Securities and Exchange Commission (SEC), including its Form 10-K for the year ended December 31, 2009 and Form 10-Q for the quarter ended March 31, 2010. Regeneron does not undertake any obligation to update publicly any forward-looking statement, whether as a result of new information, future events, or otherwise, unless required by law.
###
| Contact Information: | |
| Michael Aberman, M.D. | Peter Dworkin |
| Investor Relations | Corporate Communications |
| 914.345.7799 | 914.345.7800 |
| michael.aberman@regeneron.com | peter.dworkin@regeneron.com |
Exhibit 99(b)
