UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
PURSUANT TO SECTION 13 OR 15(d) OF
THE SECURITIES EXCHANGE ACT OF 1934
Date of Report (Date of Earliest Event Reported): January 3, 2014
CATALYST PHARMACEUTICAL PARTNERS, INC.
(Exact Name Of Registrant As Specified In Its Charter)
| Delaware | 001-33057 | 76-0837053 | ||
| (State or other jurisdiction of incorporation) |
(Commission File Number) |
(I.R.S. Employer Identification No.) | ||
| 355 Alhambra Circle Suite 1500 Coral Gables, Florida |
33134 | |||
| (Address of principal executive offices) | (Zip Code) | |||
Registrant’s telephone number, including area code: (305) 529-2522
Not Applicable
Former Name or Former address, if changed since last report
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ¨ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
| ¨ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| ¨ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR240.14d-2(b)) |
| ¨ | 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 January 6, 2014, the Company issued a press release announcing the Company’s successful completion of a Type B meeting with the U.S. Food and Drug Administration (FDA) about Firdapse™ tablets (Amifampridine), its lead product being evaluated for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS), and updating the progress of its Phase 3 clinical trial evaluating Firdapse™ for the treatment of LEMS. The press release is attached to this Current Report on Form 8-K as Exhibit 99.1 and is incorporated herein by reference.
On January 7, 2014, the Company issued a press release announcing that on January 3, 2014, the previously reported stockholder class action lawsuit that had been filed against the Company and certain of its officers and directors was dismissed without prejudice. The press release is attached to this Current Report on Form 8-K as Exhibit 99.2 and is incorporated herein by reference.
On January 8, 2014, the Company issued a press release announcing positive results from a study jointly funded with, and conducted by BioMarin Pharmaceuticals to assess the cardiac study of Firdapse™ tablets (Amifampridine). The study met the primary endpoint, demonstrating that at and above therapeutic levels, there was no effect of Firdapse™ (administered as phosphate salt) on heart rate or cardiac depolarization. The press release is attached to this Current Report on Form 8-K as Exhibit 99.3 and is incorporated herein by reference.
| Item 9.01 | Financial Statements and Exhibits. |
| (c) | Exhibits |
| 99.1 | Press Release issued by the Company on January 6, 2014 | |
| 99.2 | Press Release issued by the Company on January 7, 2014 | |
| 99.3 | Press Release issued by the Company on January 8, 2014 | |
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| Catalyst Pharmaceutical Partners, Inc. | ||
| By: | /s/ Alicia Grande | |
| Alicia Grande | ||
| Vice President, Treasurer and CFO | ||
Dated: January 8, 2014
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Exhibit 99.1
FOR IMMEDIATE RELEASE
Catalyst Pharmaceutical Partners Reports Successful Completion of Type B
Breakthrough Therapy Meeting with FDA on Firdapse Tablets Development Program
First meeting with FDA since receiving Breakthrough Therapy Designation for Firdapse
Anticipated Timeline for NDA Submission Remains on Track
CORAL GABLES, Fla., Jan. 6, 2014 — Catalyst Pharmaceutical Partners, Inc. (Catalyst) (Nasdaq: CPRX), a specialty pharmaceutical company focused on developing safe and effective, approved medicines targeting orphan neuromuscular and neurological diseases, today announced the successful completion of a Type B meeting with the U.S. Food and Drug Administration (FDA) about Firdapse™ tablets (Amifampridine), its lead product being evaluated for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS).
Catalyst provided FDA with an update on the development program for Firdapse, which received Breakthrough Therapy designation from the FDA in August 2013. The Company also confirmed with FDA the clinical, nonclinical, and chemistry and manufacturing controls requirements that FDA will require to approve a New Drug Application (NDA) for Firdapse.
“Catalyst remains committed to completing our late-stage clinical trial of Firdapse as expeditiously as possible and working with the FDA to provide a complete NDA filing,” said Patrick McEnany, President and CEO of Catalyst. “We believe that data obtained to date demonstrate the clinical value and benefit of Firdapse for LEMS patients, who today have no safe and effective, approved drug to treat their disease.”
This Type B meeting with the FDA was Catalyst’s first meeting as the sponsor of the IND for Firdapse. Catalyst will file rolling submissions of the NDA modules as completed in anticipation of receiving a priority review of its NDA for Firdapse.
The Company provided a briefing package to the FDA that described all completed, in-progress, and planned preclinical studies, clinical studies, and drug manufacturing activities. This package included summaries of 54 preclinical studies, six clinical studies, and information related to drug manufacturing (the clinical supplies and the to-be marketed commercial product). The FDA concurred that the Company’s completed, in-progress, and planned development activities represented a nearly complete package of information that would be needed for a complete NDA.
Catalyst also will submit data from additional in-vitro preclinical studies. Based on the discussions at this meeting and based on past communications and meetings with the FDA about Firdapse, all of these studies and remaining development activities constitute information needed to file a complete NDA and seek approval for Firdapse. The Company does not anticipate that these additional studies will impact the NDA filing timeline or materially add to its forecast of the aggregate development costs for Firdapse.
The Company and FDA also discussed the acceptability of the primary and secondary endpoints specified in the protocol for the ongoing Phase 3 trial. FDA requested a slight modification in the analyses to be conducted for the endpoints, which the Company believes will not require any changes in the data being collected or the number of patients needed to complete enrollment.
Update on Phase 3 Clinical Trial Status
The Company anticipates meeting previously disclosed timelines for submission and approval of an NDA for Firdapse for the treatment of LEMS.
In order to accelerate the enrollment in this clinical trial, over the last few months the Company has expanded the on-going clinical trial that was initiated by BioMarin Pharmaceutical, Inc. (Nasdaq: BMRN), to include many more sites internationally. The initiation of these clinical trial sites in numerous different countries has presented challenging logistical, contractual and regulatory issues that are nearing closure. These issues included site identification followed by numerous IRB/ethics committee filings, contracts, clinical trial applications, translations, IRB approvals, multilingual drug labeling, and other multinational issues.
There are currently 22 active sites with a sufficient number of already identified LEMS patients to complete enrollment of the trial, based on the screening, enrollment, and randomization success metrics achieved to date. As a result, the Company expects to complete screening and enrollment of the clinical trial during this quarter and to report top line data from the trial in the third quarter of this year.
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette Syndrome. Catalyst’s lead candidate, Firdapse™ for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal Phase 3 trial and recently received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA). In 2012, Catalyst licensed Firdapse from BioMarin and Catalyst assumed management of the Phase 3 pivotal trial, initiated by BioMarin. Firdapse is the first and only European approved drug for symptomatic treatment in adults with LEMS.
Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as other neurological conditions
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associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette Syndrome. CPP-115 has been granted U.S. orphan drug designation for the treatment of infantile spasms by the FDA and has been granted E.U. orphan medicinal product designation for the treatment of West Syndrome by the European Commission.
Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst’s actual results in future periods to differ materially from forecasted results. A number of factors, including the timing of completion of Catalyst’s currently ongoing Phase 3 trial of Firdapse™, whether the Phase 3 trial will be successful, whether the receipt of breakthrough therapy designation for Firdapse will expedite the development and review of Firdapse by the FDA, whether FDA will give any NDA for 3,4-DAP priority review, or accept rolling submissions of the NDA modules as completed, whether the product will be found to be safe and effective, whether an NDA for Firdapse will ever be accepted for filing by the FDA, the timing of any such NDA filing or acceptance, whether Catalyst will be the first company to receive an approval for 3,4-DAP, giving it 7-year marketing exclusivity for its product, whether any of Catalyst’s product candidates will ever be approved for commercialization or successfully commercialized, and those other factors described in Catalyst’s Annual Report on Form 10-K for the fiscal year 2012 and other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst’s filings with the SEC are available from the SEC, may be found on Catalyst’s website or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.
# # #
| Media/Investor Contacts Donna LaVoie or David Connolly LaVoie Group (617) 374-8800 dlavoie@lavoiegroup.com dconnolly@lavoiegroup.com |
Company Contact Patrick J. McEnany Catalyst Pharmaceutical Partners, Inc. Chief Executive Officer (305) 529-2522 pmcenany@catalystpharma.com |
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Exhibit 99.2
FOR IMMEDIATE RELEASE
Catalyst Pharmaceutical Partners Reports Dismissal of Stockholder Class Action Lawsuit
CORAL GABLES, Fla., Jan. 7, 2014 — Catalyst Pharmaceutical Partners, Inc. (Catalyst) (Nasdaq: CPRX), a specialty pharmaceutical company focused on developing safe and effective, approved medicines targeting orphan neuromuscular and neurological diseases, today announced that on January 3, 2014, the previously reported stockholder class action lawsuit that had been filed against Catalyst and certain of its officers and directors was dismissed without prejudice. In the Court’s order, the plaintiffs were granted leave to file an amended complaint within 20 days. Catalyst has no information as to whether any such amended complaint is planned by the plaintiffs. If an amended complaint is filed in the case, Catalyst intends to vigorously defend the amended lawsuit.
In October and November 2013, three securities class action lawsuits were filed against Catalyst and certain of its officers and directors in the U.S. District Court for the Southern District of Florida. The complaints, which were substantially identical, purported to state a claim for violation of federal securities laws on behalf of a class of those who purchased Catalyst’s common stock between October 31, 2012 and October 18, 2013. Two of the lawsuits were voluntarily dismissed by the plaintiffs, and the last remaining case was the case dismissed on Friday.
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette Syndrome. Catalyst’s lead candidate, Firdapse™ for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal Phase 3 trial and recently received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA). In 2012, Catalyst licensed Firdapse from BioMarin and Catalyst assumed management of the Phase 3 pivotal trial initiated by BioMarin. Firdapse is the first and only European approved drug for symptomatic treatment in adults with LEMS.
Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette Syndrome. CPP-115 has been granted U.S. orphan drug designation for the treatment of infantile spasms by the FDA and has been granted E.U. orphan medicinal product designation for the treatment of West Syndrome by the European Commission.
Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst’s actual results in future periods to differ materially from forecasted results. A number of factors, including the timing of completion of Catalyst’s currently ongoing Phase 3 trial of Firdapse™, whether the Phase 3 trial will be successful, whether the receipt of breakthrough therapy designation for Firdapse will expedite the development and review of Firdapse by the FDA, whether an NDA for Firdapse will ever be accepted for filing by the FDA, the timing of any such NDA filing or acceptance, whether Catalyst will be the first company to receive an approval for 3,4-DAP, giving it 7-year marketing exclusivity for its product, whether any of Catalyst’s product candidates will ever be approved for commercialization or successfully commercialized, whether the class action lawsuit will be refiled and the ultimate outcome of that lawsuit, and those other factors described in Catalyst’s Annual Report on Form 10-K for the fiscal year 2012 and other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst’s filings with the SEC are available from the SEC, may be found on Catalyst’s website or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.
# # #
| Media/Investor Contacts Donna LaVoie or David Connolly LaVoie Group (617) 374-8800 dlavoie@lavoiegroup.com dconnolly@lavoiegroup.com |
Company Contact Patrick J. McEnany Catalyst Pharmaceutical Partners, Inc. Chief Executive Officer (305) 529-2522 pmcenany@catalystpharma.com |
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Exhibit 99.3
FOR IMMEDIATE RELEASE
Catalyst Pharmaceutical Partners Reports Positive Cardiac Safety Results
of Its Phase 3 Product, Firdapse Tablets
CORAL GABLES, Fla., Jan. 8, 2014 — Catalyst Pharmaceutical Partners, Inc. (Nasdaq: CPRX), a specialty pharmaceutical company focused on developing safe and effective, approved medicines targeting orphan neuromuscular and neurological diseases, today announced positive results from a study jointly funded with, and conducted by BioMarin Pharmaceuticals (Nasdaq:BMRN) to assess the cardiac safety of Firdapse™ tablets (amifampridine also known as 3,4-DAP). Firdapse is approved in the E.U., where it is marketed by BioMarin. Firdapse is also currently in Phase 3 development in the U.S. for Lambert-Eaton Myasthenic Syndrome (LEMS) by Catalyst.
The study met the pre-specified primary endpoint, demonstrating that at and above therapeutic levels, there was no effect of Firdapse (administered as phosphate salt) on heart rate or cardiac depolarization. None of the study subjects developed new, clinically relevant electrocardiographic/morphological changes following administration of Firdapse. Additionally, there was no significant effect of Firdapse on cardiac repolarization as assessed using the QT interval.
The QT interval represents the amount of time the heart’s electrical system takes to repolarize, or recharge, after each beat. As prolongation of the QT interval may increase the risk for cardiac arrhythmias and sudden death, the U.S. Food and Drug Administration (FDA) requires a thorough QT (TQT) study for most new drugs in development. A TQT study is a specialized clinical trial designed to assess whether an investigational medication has the potential to prolong the QT interval.
“The findings of this cardiac safety study are clear. Firdapse did not lead to QT prolongation, even at high concentrations in the blood,” said Charles W. Gorodetzky, MD, PhD, Chief Medical Officer for Catalyst. Dr. Gorodetzky continued, “This study was designed in accordance with existing FDA guidelines. We are confident in these results and will continue to work toward making Firdapse available to patients in the U.S. if we are able to obtain marketing authorization from FDA.”
“This is the first formal human evaluation of cardiac safety of Firdapse and we are pleased with the outcome of the study,” said Steven Miller, Ph.D., Chief Scientific and Chief Operating Officer for Catalyst. Dr. Miller continued, “It is also important to caution the patient community that these findings are only relevant to Firdapse and do not necessarily indicate that other unapproved forms of 3,4-DAP would be safe, because the drug absorption and blood levels that result from other forms of 3,4-DAP may be different than Firdapse.”
At a pre-IND meeting in 2010, FDA requested TQT study results for Firdapse at exposures higher than typical therapeutic levels be included as part of the clinical safety package in any New Drug Application filed for 3,4-DAP.
Study Details
This randomized double-blind study, designed in accordance with the FDA’s published guidance on clinical evaluation of QT/QTc interval (ICH E14), compared the effects of Firdapse at or above therapeutic concentrations to placebo on the QT interval in 59 healthy human volunteers. The primary endpoint was to determine whether Firdapse administered at therapeutic and supratherapeutic concentrations differed from placebo in the mean change in the QTc interval.
All heart rate correction methodologies that satisfied the pre-specified selection criteria, including QTcF and QTcB (QTc calculated by Fridericia’s and Bazett’s formulas respectively), met the primary endpoint. Moxifloxacin, an antibiotic known to prolong the QT interval, was administered as a positive control.
Catalyst expects that BioMarin will present the full data set from this study at a major medical meeting at some time in the future and will submit the data for publication thereafter.
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette Syndrome. Catalyst’s lead candidate, Firdapse™ for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal Phase 3 trial and recently received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA). In 2012, Catalyst licensed Firdapse from BioMarin and Catalyst assumed management of the Phase 3 pivotal trial, initiated by BioMarin. Firdapse is the first and only European approved drug for symptomatic treatment in adults with LEMS.
Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette Syndrome. CPP-115 has been granted U.S. orphan drug designation for the treatment of infantile spasms by the FDA and has been granted E.U. orphan medicinal product designation for the treatment of West Syndrome by the European Commission.
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Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst’s actual results in future periods to differ materially from forecasted results. A number of factors, including the timing of completion of Catalyst’s currently ongoing Phase 3 trial of Firdapse™, whether the Phase 3 trial will be successful, whether the receipt of breakthrough therapy designation for Firdapse will expedite the development and review of Firdapse by the FDA, whether FDA will give any NDA for 3,4-DAP priority review, or accept rolling submissions of the NDA modules as completed, whether the product will be found to be safe and effective, whether an NDA for Firdapse will ever be accepted for filing by the FDA, the timing of any such NDA filing or acceptance, whether Catalyst will be the first company to receive an approval for 3,4-DAP, giving it 7-year marketing exclusivity for its product, whether any of Catalyst’s product candidates will ever be approved for commercialization or successfully commercialized, and those other factors described in Catalyst’s Annual Report on Form 10-K for the fiscal year 2012 and other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst’s filings with the SEC are available from the SEC, may be found on Catalyst’s website or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.
# # #
| Media/Investor Contacts Donna LaVoie or David Connolly LaVoie Group (617) 374-8800 dlavoie@lavoiegroup.com dconnolly@lavoiegroup.com |
Company Contact Patrick J. McEnany Catalyst Pharmaceutical Partners, Inc. Chief Executive Officer (305) 529-2522 pmcenany@catalystpharma.com |
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