CPRX Catalyst Pharmaceuticals Inc.

3.16
+0.09  (+3%)
Previous Close 3.08
Open 3.06
52 Week Low 2.55
52 Week High 5.83
Market Cap $326,944,534
Shares 103,463,460
Float 96,191,469
Enterprise Value $212,090,181
Volume 901,965
Av. Daily Volume 2,014,564
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Upcoming Catalysts

Drug Stage Catalyst Date
Firdapse
Spinal Muscular Atrophy (SMA) Type 3
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
Firdapse
MuSK-antibody positive myasthenia gravis (MuSK-MG)
Phase 3
Phase 3
Phase 3 trial did not meet primary endpoint - August 10, 2020.
Firdapse
Congenital Myasthenic Syndromes (CMS)
Phase 3
Phase 3
Phase 3 trial did not meet primary endpoint - October 30, 2019.
Firdapse
Lambert-Easton Myasthenic Syndrome (LEMS)
Approved
Approved
FDA approval announced November 28, 2018.

Latest News

  1. CORAL GABLES, Fla., Aug. 26, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, and KYE Pharmaceuticals Inc. ("KYE"), a private company headquartered in Mississauga, Ontario and focused on bringing medicines that fulfill clinically significant unmet needs to the Canadian market, today announced the filing of a proceeding in Canadian Federal Court challenging Health Canada's issuance of a Notice of Compliance (NOC) on August 10, 2020 for Medunik's New Drug Submission (NDS) for the drug Ruzurgi® (amifampridine) for the treatment…

    CORAL GABLES, Fla., Aug. 26, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, and KYE Pharmaceuticals Inc. ("KYE"), a private company headquartered in Mississauga, Ontario and focused on bringing medicines that fulfill clinically significant unmet needs to the Canadian market, today announced the filing of a proceeding in Canadian Federal Court challenging Health Canada's issuance of a Notice of Compliance (NOC) on August 10, 2020 for Medunik's New Drug Submission (NDS) for the drug Ruzurgi® (amifampridine) for the treatment of Lambert-Eaton Myasthenic Syndrome despite the fact that Catalyst's Firdapse® (amifampridine phosphate) was issued an NOC on July 31, 2020 and was granted data protection as an "innovative drug" as it was the first amifampridine product to be approved in Canada.

    The proceeding seeks judicial review of Health Canada's decision to issue an NOC for Ruzurgi® as incorrect and unreasonable. Data protection, per Health Canada regulations, is supposed to prevent Health Canada from issuing an NOC to a drug that directly or indirectly references an innovative drug's data, for eight years from the date of the innovative drug's approval. The Ruzurgi® Product Monograph clearly references pivotal nonclinical carcinogenicity and reproductive toxicity data for amifampridine phosphate developed by Catalyst. As such, the Catalyst data was relied upon to establish the nonclinical safety profile of Ruzurgi® needed to meet the standards of the Canadian Food and Drugs Act. Data protection had, however, been granted to Firdapse® at the time that the NOC was issued for Ruzurgi®. Contrary to the regulations, Health Canada approved Ruzurgi® even though its drug submission included carcinogenicity and reproductive toxicity data with Firdapse®. Firdapse®'s status as an innovative drug should have prevented the marketing authorization of Ruzurgi®.

    "Innovative drugs, not previously approved in Canada, go through a rigorous drug approval process in Canada. A sponsor must submit clinical and nonclinical safety, tolerability, and efficacy data, to demonstrate the safety and efficacy of the drug," said Patrick J. McEnany, Chairman and Chief Executive Officer of Catalyst Pharmaceuticals, Inc. "In exchange for that effort, and as an incentive to do that, Health Canada regulations are supposed to prevent other pharmaceutical companies from being able to use the innovator's (in this case Catalyst's) data as a basis for approval for eight years from the date of the Notice of Compliance. In Firdapse®'s case, Health Canada only did so for ten days. We are compelled to bring this application to preserve the specialized regulatory framework for new chemical entities, and the future of drug innovation in Canada."

    "As the marketing partner for Firdapse® in Canada," added Douglas Reynolds, President of KYE, "it is essential that we seek to enforce the data protection for Firdapse® as the regulations have been designed to do. Without that incentive, the motivation to bring new chemical entities to Canada is significantly diminished."

    About Lambert-Eaton Myasthenic Syndrome (LEMS)

    Lambert-Eaton myasthenic syndrome, or LEMS, is a rare autoimmune disorder, most often characterized by muscle weakness of the limbs. The disease is caused by an autoimmune reaction where antibodies are formed against voltage gated potassium channels in the connection between nerves and the muscles they communicate with. In approximately 50% of cases, LEMS is associated with an underlying malignancy, most commonly small-cell lung cancer, and in some individuals, LEMS is the first symptom of such malignancy. LEMS generally affects the extremities, especially the legs. As the disease most affects the parts of limbs closest to the trunk, difficulties with climbing stairs or rising from a sitting position are commonly noted. Physical exercise and high temperatures tend to worsen the symptoms. Other symptoms occasionally seen include weakness of the muscles of the mouth, throat, and eyes. Individuals affected with LEMS also may have a disruption of the autonomic nervous system, including dry mouth, constipation, blurred vision, impaired sweating, and/or hypotension.

    About Catalyst Pharmaceuticals

    Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton myasthenic syndrome (LEMS), anti-MuSK antibody positive myasthenia gravis (MuSK-MG), and spinal muscular atrophy (SMA) Type 3. Catalyst's new drug application for Firdapse® (amifampridine) 10 mg tablets for the treatment of adults with LEMS was approved in November 2018 by the U.S. Food & Drug Administration ("FDA"), and Firdapse® is now commercially available in the United States. Further, Canada's national healthcare regulatory agency, Health Canada, recently approved the use of Firdapse® (amifampridine) for the treatment of patients in Canada with LEMS.

    Firdapse® is currently being evaluated in clinical trials for the treatment of MuSK-MG and SMA Type 3 and has received Orphan Drug Designation from the FDA for myasthenia gravis.

    About KYE Pharmaceuticals

    KYE Pharmaceuticals is a private company headquartered in Canada focused on bringing medications to the Canadian market which fulfill clinically significant and unmet needs. KYE has licensed many innovative products and was founded on an entrepreneurial spirit that optimizes our team's strengths and brings unique value to our partners, Canadian healthcare professionals, and most importantly, our patients. For more information please visit www.kyepharma.com

    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 (i) whether KYE can successfully commercialize Firdapse® in Canada, (ii) whether any such commercialization of Firdapse® in Canada will be on a profitable basis, (iii) the impact of competition from Ruzurgi® on sales of Firdapse® in Canada, (iv) whether Catalyst's action to overturn the approval of Ruzurgi® in Canada will be successful, (v) the impact in the United States if an amifampridine product is purchased in Canada for use in the United States, and (vi) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2019 and Catalyst's 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.

    Investor Contact
    Brian Korb
    Solebury Trout 
    (646) 378-2923
    
    
    Company Contact
    Patrick J. McEnany
    Catalyst Pharmaceuticals
    Chief Executive Officer
    (305) 420-3200
    
    
    Media Contact
    David Schull
    Russo Partners
    (212) 845-4271
    

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  2. CORAL GABLES, Fla., Aug. 18, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, and KYE Pharmaceuticals Inc. ("KYE"), a private company headquartered in Mississauga, Ontario and focused on bringing medicines that fulfill clinically significant unmet needs to the Canadian market, today announced that the companies have entered into an exclusive license agreement under which KYE will commercialize Firdapse® in Canada. Firdapse® is indicated for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults…

    CORAL GABLES, Fla., Aug. 18, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, and KYE Pharmaceuticals Inc. ("KYE"), a private company headquartered in Mississauga, Ontario and focused on bringing medicines that fulfill clinically significant unmet needs to the Canadian market, today announced that the companies have entered into an exclusive license agreement under which KYE will commercialize Firdapse® in Canada. Firdapse® is indicated for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. Under the terms of the agreement, Catalyst will supply Firdapse® to KYE and KYE will be responsible for promotion, sales, advertisement, marketing, product importation and distribution. KYE will also be responsible for the ongoing maintenance of the regulatory file and future communications with Health Canada.

    Patrick J. McEnany, Catalyst's Chairman and CEO, said, "Catalyst remains focused on making a meaningful impact in the lives of those suffering from rare diseases. We are excited to partner with the experienced team at KYE in making Firdapse® available to LEMS patients throughout Canada."

    Doug Reynolds, KYE Co-Founder and President, said, "Partnering with Catalyst to make Firdapse® available to LEMS patients in Canada allows us to continue fulfilling our goal to bring critically needed medicines to Canadian patients and the healthcare community. We look forward to working with the experienced and talented Catalyst team in the years ahead."

    About Lambert-Eaton Myasthenic Syndrome (LEMS)

    Lambert-Eaton myasthenic syndrome, or LEMS, is a rare autoimmune disorder, most often characterized by muscle weakness of the limbs. The disease is caused by an autoimmune reaction where antibodies are formed against voltage gated potassium channels in the connection between nerves and the muscles they communicate with. In approximately 50% of cases, LEMS is associated with an underlying malignancy, most commonly small-cell lung cancer, and in some individuals, LEMS is the first symptom of such malignancy. LEMS generally affects the extremities, especially the legs. As the disease most affects the parts of limbs closest to the trunk, difficulties with climbing stairs or rising from a sitting position are commonly noted. Physical exercise and high temperatures tend to worsen the symptoms. Other symptoms occasionally seen include weakness of the muscles of the mouth, throat, and eyes. Individuals affected with LEMS also may have a disruption of the autonomic nervous system, including dry mouth, constipation, blurred vision, impaired sweating, and/or hypotension.

    About Catalyst Pharmaceuticals

    Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton myasthenic syndrome (LEMS), anti-MuSK antibody positive myasthenia gravis (MuSK-MG), and spinal muscular atrophy (SMA) Type 3. Catalyst's new drug application for Firdapse® (amifampridine) 10 mg tablets for the treatment of adults with LEMS was approved in November 2018 by the U.S. Food & Drug Administration ("FDA"), and Firdapse® is now commercially available in the United States. Further, Canada's national healthcare regulatory agency, Health Canada, recently approved the use of Firdapse® (amifampridine) for the treatment of patients in Canada with LEMS.

    Firdapse® is currently being evaluated in clinical trials for the treatment of MuSK-MG and SMA Type 3 and has received Orphan Drug Designation from the FDA for myasthenia gravis.

    About KYE Pharmaceuticals

    KYE Pharmaceuticals is a private company headquartered in Canada focused on bringing medications to the Canadian market which fulfill clinically significant and unmet needs. KYE has licensed many innovative products and was founded on an entrepreneurial spirit that optimizes our team's strengths and brings unique value to our partners, Canadian healthcare professionals, and most importantly, our patients. For more information please visit www.kyepharma.com.

    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 (i) whether KYE can successfully commercialize Firdapse® in Canada, (ii) whether any such commercialization of Firdapse® in Canada will be on a profitable basis, (iii) the impact of competition from Ruzurgi® on sales of Firdapse® in Canada, (iv) the impact in the United States if an amifampridine product is purchased in Canada for use in the United States, and (v) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2019 and Catalyst's 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.

    Investor Contact
    Brian Korb
    Solebury Trout 
    (646) 378-2923
    
    
    Company Contact
    Patrick J. McEnany
    Catalyst Pharmaceuticals
    Chief Executive Officer
    (305) 420-3200
    
    
    Media Contact
    David Schull
    Russo Partners
    (212) 845-4271
    

    Primary Logo

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  3. CORAL GABLES, Fla., Aug. 11, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for U.S. Patent Application Serial Number 14/128,672.  The allowed application, "Methods of Administering 3,4-Diaminopyridine", claims a method of treating a human patient diagnosed with a 3,4-DAP sensitive disease by administering 3,4-DAP (or salts thereof) to slow acetylating patients having certain mutations in each allele of the NAT2…

    CORAL GABLES, Fla., Aug. 11, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for U.S. Patent Application Serial Number 14/128,672.  The allowed application, "Methods of Administering 3,4-Diaminopyridine", claims a method of treating a human patient diagnosed with a 3,4-DAP sensitive disease by administering 3,4-DAP (or salts thereof) to slow acetylating patients having certain mutations in each allele of the NAT2 gene.

    A Notice of Allowance is issued after the USPTO determines that the prosecution on the merits of a patent has been completed.  The patent can then be granted from an application upon payment of the patent issue fee.  Catalyst expects the patent to issue in the next few months.  Once issued, the patent would be expected to expire no earlier than June 29, 2032.  The expiration of this patent could also be extended based on delays in patent prosecution, if any.  This extension, if any, will be calculated after the patent has been issued.  The Notice of Allowance and the allowed claims for this application are posted on the USPTO public PAIR website.

    "The receipt of this Notice of Allowance from the USPTO represents an important milestone in expanding our Firdapse® intellectual property portfolio and adds to Catalyst's options for further developing the Firdapse® franchise," said Dr. Steven Miller, Chief Operating Officer and Chief Scientific Officer.  Dr. Miller continued, "Notably, we believe that the comprehensive nature of the allowed claims in the resulting patent will provide Catalyst with additional protection through at least June of 2032.  This will permit us to continue, and possibly expand, our research efforts for other indications, as well as improved formulations for Firdapse®."

    Upon issuance of this patent, Catalyst will seek to list this patent in the FDA's "Orange Book" (the FDA's list of all approved drugs in the United States).  When a patent is listed in the FDA's Orange Book for an approved drug, no generic equivalent of the drug may be approved by the FDA unless the generic drug applicant can prove that they do not infringe the listed patent or until the applicant successfully challenges the validity of the listed patent.

    About Catalyst Pharmaceuticals

    Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton myasthenic syndrome (LEMS), anti-MuSK antibody positive myasthenia gravis (MuSK-MG) and spinal muscular atrophy (SMA) Type 3. Catalyst's new drug application for Firdapse® (amifampridine) 10 mg tablets for the treatment of adults with LEMS was approved in November 2018 by the U.S. Food & Drug Administration ("FDA"), and Firdapse® is now commercially available in the United States. Prior to its approval, Firdapse® for LEMS had received breakthrough therapy designation and orphan drug designation from the FDA.

    Firdapse® is currently being evaluated in clinical trials for the treatment of MuSK-MG and SMA Type 3 and has received Orphan Drug Designation from the FDA for myasthenia gravis.

    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 (i) when the patent will officially be issued, (ii) when the patent, once officially issued, will expire, (iii) the scope of protection from competition provided by the patent, and (iv) those factors described in Catalyst's Annual Report on Form 10-K for fiscal year 2019 and its 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.

    Investor Contact
    Brian Korb
    Solebury Trout 
    (646) 378-2923
    
    
    Company Contact
    Patrick J. McEnany
    Catalyst Pharmaceuticals
    Chief Executive Officer
    (305) 420-3200
    
    
    Media Contact
    David Schull
    Russo Partners
    (212) 845-4271
    

    Primary Logo

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  4. - Firdapse® Second Quarter Net Revenues of $29.6 Million

    - Health Canada Approves Marketing Authorization for Firdapse® for Treating LEMS Patients

    - MuSK-MG Phase 3 Trial did not Achieve Statistical Significance for Primary or Secondary Endpoints

    - Company to Host Quarterly Conference Call at 8:30 am ET Tomorrow

    CORAL GABLES, Fla., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today reported financial results for the second quarter ended June 30, 2020 and provided a business update.

    "Despite operating…

    - Firdapse® Second Quarter Net Revenues of $29.6 Million

    - Health Canada Approves Marketing Authorization for Firdapse® for Treating LEMS Patients

    - MuSK-MG Phase 3 Trial did not Achieve Statistical Significance for Primary or Secondary Endpoints

    - Company to Host Quarterly Conference Call at 8:30 am ET Tomorrow

    CORAL GABLES, Fla., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today reported financial results for the second quarter ended June 30, 2020 and provided a business update.

    "Despite operating under COVID-19 conditions, I am quite pleased with the resiliency of our team and the operating results that were achieved during the second quarter. Additionally, as physicians' practices begin to open again to in-person meetings with patients and sales representatives, we are beginning to see an improvement in new LEMS patient enrollments in Catalyst Pathways and are hopeful that this is a start of a trend," said Patrick J. McEnany, Chairman and Chief Executive Officer of Catalyst Pharmaceuticals. "On a separate note, we are disappointed that the top-line results of our Phase 3 study for Firdapse® in anti-MuSK antibody positive myasthenia gravis patients did not replicate the robust positive results that were observed in the 2017 proof-of-concept study. We intend to continue to analyze the data and meet with our neuromuscular key opinion leaders to decide on our path forward for this program."

    Q2-20 Financial Results

    • Product revenue, net in the second quarter 2020 was $29.6 million compared to $28.8 for the second quarter 2019.
    • Reported net income of $9.8 million, or $0.09 per basic and diluted share, in the second quarter of 2020, compared with net income of $11.0 million, or $0.11 per basic and $0.10 per diluted share, for the second quarter of 2019.
    • Research and development expenses for the second quarter of 2020 were $4.3 million as compared to $4.6 million for the second quarter of 2019.
    • Selling, general and administrative expenses for the second quarter of 2020 totaled $10.8 million as compared to $9.0 million in the second quarter of 2019.
    • The Company's total operating expenses for the second quarter 2020 were $19.3 million.
    • Ended June 30, 2020 with $115.1 million in cash and investments and no funded debt. 

    Corporate Highlights and Milestones

    • Announced receipt of marketing authorization from Health Canada for Firdapse® for the treatment of patients in Canada with LEMS.
    • Announced Jeffrey Del Carmen promotion to Chief Commercial Officer.
    • Recently met with the Japanese PMDA and believe we have reached a tentative agreement for an acceptable and efficient regulatory plan.
    • Continued on-going evaluation of potential acquisition of products or companies.

    MuSK-MG Phase 3 Study Results

    • Firdapse® was safe and well tolerated during the MSK-002 study and demonstrated a safety profile similar to that seen for Firdapse® in the treatment of LEMS.
    • The primary endpoint, the Myasthenia Gravis Activities of Daily Living (MG-ADL) assessment, did not achieve statistical significance (p=0.2196).
    • The secondary endpoint, the Quantitative Myasthenia Gravis (QMG) assessment, did not achieve statistical significance (p=0.3736).
    • We plan to complete the full analysis of data and findings and to meet with our neuromuscular advisors to decide our path forward for the MuSK-MG indication.
    • Detailed results of this study will be made available in a future scientific forum.

    Other Firdapse® Development Programs

    • Enrollment in proof-of-concept spinal muscular atrophy type-3 study has been completed and we expect to report top-line data before year-end.
    • Firdapse® long-acting formulation development program continues on schedule.
    • Investigator studies for two additional neuromuscular indications are expected to commence later this year.

    COVID-19 Impact

    • Issued a no travel and remote work policy for all Catalyst employees on March 16th.
    • Diligently working to reduce COVID-19 impact on new enrollments and revenues.
    • We believe that our current base of LEMS patients on reimbursed Firdapse® is fairly stable and very compliant to their medication regimen.
    • Have not experienced any disruptions in the supply chain or production of Firdapse® and believe the safety stock of Firdapse® is more than adequate for current anticipated needs.
    • Proudly partnered with First Responder's Children's Foundation/COVID-19 Emergency Response Fund, which provides emergency grants to support frontline emergency and healthcare workers and their families enduring financial hardship during this COVID-19 pandemic.

    Balance Sheet and Key Activities in 2020

    At June 30, 2020, Catalyst had cash and cash equivalents and investments of $115.1 million and no funded debt. 

    The Company plans to continue investing in the following key activities in 2020:

    • Expansion of U.S. commercialization of Firdapse®.
    • On-going development programs evaluating Firdapse® for the treatment of MuSK-MG and SMA Type 3, and our Expanded Access Program for Firdapse®.
    • Continue support for our Firdapse® long acting formulation and other development programs.
    • Support Canada pre-commercialization activities for Firdapse®.
    • Continue Japan regulatory activities to seek marketing authorization for Firdapse®.

    More detailed financial information and analysis may be found in the Company's Quarterly Report on Form 10-Q, which was filed with the Securities and Exchange Commission (SEC) on August 10, 2020.

    Non-GAAP Financial Measures

    Excluding expenses related to stock-based compensation of $1.8 million, non-GAAP1 net income for the second quarter of 2020 was $11.6 million, or $0.11 per basic and diluted share. This compares to a non-GAAP1 net income of $11.9 million, or $0.12 per basic share and $0.11 per diluted share, excluding stock-based compensation expense of $925 thousand, for the second quarter of 2019. Excluding expenses related to stock-based compensation of $3.3 million, non-GAAP1 net income for the six months ended June 30, 2020 was $23.5 million, or $0.23 per basic share and $0.22 per diluted share. This compares to a non-GAAP1 net income of $12.2 million, or $0.12 per basic and diluted share, excluding stock-based compensation expense of $1.9 million, for the six months ended June 30, 2019.

    Conference Call

    Catalyst management will host an investment-community conference call and webcast at 8:30 a.m. ET, tomorrow, Tuesday, August 11, 2020 to discuss the financial results and provide a corporate update. Investors who wish to participate in the conference call may do so by dialing (877) 407-8912 for domestic and Canadian callers or (201) 689-8059 for international callers. Those interested in listening to the conference call live via the internet may do so by visiting the Investors page of the company's website at www.catalystpharma.com and clicking on the webcast link on the Investors home page. A webcast replay will be available on the Catalyst website for 30 days following the call by visiting the Investor page of the company's website at www.catalystpharma.com.

    About Catalyst Pharmaceuticals

    Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing  and  commercializing innovative  therapies  for people with rare  debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton myasthenic syndrome (LEMS), anti-MuSK antibody positive myasthenia gravis (MuSK-MG) and spinal muscular atrophy (SMA) Type 3. Catalyst's new drug application for Firdapse® (amifampridine) 10 mg tablets for the treatment of adults with LEMS was approved in November 2018 by the U.S. Food & Drug Administration ("FDA"), and Firdapse® is now commercially available in the United States. Prior to its approval, Firdapse® for LEMS had received breakthrough therapy designation and orphan drug designation from the FDA.

    Firdapse® is being evaluated in clinical trials for the treatment of MuSK-MG and SMA Type 3 and has received Orphan Drug Designation from the FDA for myasthenia gravis and CMS. Firdapse® (amifampridine) 10 mg tablets is the first and only approved drug in Europe for the symptomatic treatment in adults with LEMS.

    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 (i) the impact of the effects of the COVID-19 pandemic on Catalyst's 2020 net product revenues and on the timeline for reporting the top-line results from Catalyst's SMA Type 3 proof-of-concept study; (ii) whether, even if Catalyst is successful in commercializing Firdapse®, Catalyst will achieve sustained profitability; (iii) the effect on Catalyst's business and future results of operations of the approval by the FDA of Ruzurgi® for the treatment of pediatric LEMS patients (ages 6 to under 17); (iv) whether Catalyst's suit against the FDA seeking to vacate the FDA's approval of Ruzurgi® will be successful; (v) whether Firdapse® will ever be approved for commercialization for the treatment of MuSK-MG, SMA Type 3, or any other disease; (vi) whether Catalyst can successfully commercialize Firdapse® in Canada; (vii) whether Catalyst is able to successfully complete the clinical trial required for Catalyst to seek approval to commercialize Firdapse® for sale in Japan; (viii) whether Catalyst will ever be approved for commercialization in Japan; and (ix) those other factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2019 and its 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.

    1 Statements made in this press release include a non-GAAP financial measure. Such information is provided as additional information and not as an alternative to Catalyst's financial statements presented in accordance with U.S. generally accepted accounting principles (GAAP). This non-GAAP financial measure is intended to enhance an overall understanding of Catalyst's current financial performance. Catalyst believes that the non-GAAP financial measure presented in this press release provides investors and prospective investors with an alternative method for assessing Catalyst's operating results in a manner that Catalyst believes is focused on the performance of ongoing operations and provides a more consistent basis for comparison between periods. The non-GAAP financial measure in this press release excludes from the calculation of net income (loss) the expense associated with non-cash stock-based compensation.  Non-GAAP income (loss) per share is calculated by dividing non-GAAP income (loss) by the weighted average common shares outstanding.



    CATALYST PHARMACEUTICALS, INC.

    CONSOLIDATED STATEMENTS OF OPERATIONS (unaudited)

     For the Three Months



     Ended June 30,
     For the Six Months



    Ended June 30,
     2020 2019 2020 2019
    Product revenue, net$29,604,764 $28,837,900 $58,741,236 $41,286,338
            
    Operating costs and expenses:       
    Cost of sales 4,139,873  4,261,625  8,290,739  5,973,413
    Research and development 4,349,643  4,629,364  8,572,454  7,937,323
    Selling, general and administrative 10,833,358  8,987,722  20,896,406  17,404,182
    Total operating costs and expenses 19,322,874  17,878,711  37,759,599  31,314,918
    Operating income (loss) 10,281,890  10,959,189  20,981,637  9,971,420
    Other income, net 111,269  450,410  447,502  793,676
    Net income (loss) before income taxes 10,393,159  11,409,599  21,429,139  10,765,096
    Provision for income taxes 613,172  449,651  1,223,137  449,651
    Net income (loss)$9,779,987 $10,959,948 $20,206,002 $10,315,445
            
    Net income (loss) per share:       
    Basic$0.09 $0.11 $0.20 $0.10
    Diluted$0.09 $0.10 $0.19 $0.10
            
    Weighted average shares outstanding:       
    Basic 103,414,523  102,869,202  103,410,881  102,808,897
    Diluted 106,730,423  105,928,970  106,433,862  105,098,930
     



    CATALYST PHARMACEUTICALS, INC.



    CONDENSED CONSOLIDATED BALANCE SHEETS

        
     June 30,



    2020
     December 31,



    2019
     (unaudited)  
    ASSETS   
    Current Assets:   
    Cash and cash equivalents$115,052,248 $89,511,710
    Short-term investments --  5,007,050
    Accounts receivable, net 6,762,262  10,536,997
    Inventory 1,827,924  1,956,792
    Prepaid expenses and other current assets 7,521,253  4,351,074
    Total current assets 131,163,687  111,363,623
    Operating lease right-of-use asset 71,711  793,252
    Property and equipment, net 167,514  210,467
    Deposits 8,888  8,888
    Total assets$131,411,800 $112,376,230
        
    LIABILITIES AND STOCKHOLDERS' EQUITY   
    Current Liabilities:   
    Accounts payable$5,804,778 $4,117,447
    Accrued expenses and other liabilities 14,405,597  19,981,295
    Total current liabilities 20,210,375  24,098,742
    Operating lease liability, net of current portion --  647,532
    Total liabilities 20,210,375  24,746,274
        
    Total stockholders' equity 111,201,425  87,629,956
    Total liabilities and stockholders' equity$131,411,800 $112,376,230
    Investor Contact
    Brian Korb
    Solebury Trout 
    (646) 378-2923
    
    
    Company Contact
    Patrick J. McEnany
    Catalyst Pharmaceuticals
    Chief Executive Officer
    (305) 420-3200
    
    
    Media Contact
    David Schull
    Russo Partners
    (212) 845-4271
    

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  5. CORAL GABLES, Fla., Aug. 06, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced that Canada's national healthcare regulatory agency, Health Canada, has approved the use of Firdapse® (amifampridine) for the treatment of patients in Canada with Lambert-Eaton myasthenic syndrome (LEMS), an ultra-rare, debilitating and potentially life-threatening neurodegenerative condition.

    Firdapse® was approved under Priority Review by Health Canada. The marketing application submitted to Health Canada included safety and…

    CORAL GABLES, Fla., Aug. 06, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (NASDAQ:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced that Canada's national healthcare regulatory agency, Health Canada, has approved the use of Firdapse® (amifampridine) for the treatment of patients in Canada with Lambert-Eaton myasthenic syndrome (LEMS), an ultra-rare, debilitating and potentially life-threatening neurodegenerative condition.

    Firdapse® was approved under Priority Review by Health Canada. The marketing application submitted to Health Canada included safety and efficacy data from Catalyst's two previously reported multi-national clinical trials evaluating Firdapse® for the treatment of patients with LEMS, and the data from these trials was used to support the new drug submission (NDS) to Health Canada. Firdapse® was previously approved for marketing by the U. S. Food and Drug Administration and the European Commission in 2018 and 2009, respectively, and is currently being used to treat LEMS patients in the United States and in more than 15 additional countries.

    "At Catalyst, we are dedicated to changing the lives of patients suffering from rare neuromuscular diseases, and we are proud that we have brought forward an approved therapeutic option for patients in Canada suffering with LEMS. We anticipate continuing to interact with Health Canada as we try to expand the Firdapse® label to include other indications," said Gary Ingenito, M.D., Ph.D., Catalyst's Chief Medical and Regulatory Officer.

    "We are currently in discussions with a potential marketing and distribution partner in Canada to provide access for Firdapse®, patient-by-patient, as rapidly as possible," said David Ailinger, Vice President of Business Development at Catalyst.

    About Lambert-Eaton Myasthenic Syndrome (LEMS)

    Lambert-Eaton myasthenic syndrome, or LEMS, is a rare autoimmune disorder, most often characterized by muscle weakness of the limbs.  The disease is caused by an autoimmune reaction where antibodies are formed against voltage gated potassium channels in the connection between nerves and the muscles they communicate with.  In approximately 50% of cases, LEMS is associated with an underlying malignancy, most commonly small-cell lung cancer, and in some individuals, LEMS is the first symptom of such malignancy.  LEMS generally affects the extremities, especially the legs.  As the disease most affects the parts of limbs closest to the trunk, difficulties with climbing stairs or rising from a sitting position are commonly noted.  Physical exercise and high temperatures tend to worsen the symptoms.  Other symptoms occasionally seen include weakness of the muscles of the mouth, throat, and eyes.  Individuals affected with LEMS also may have a disruption of the autonomic nervous system, including dry mouth, constipation, blurred vision, impaired sweating, and/or hypotension.

    About Firdapse®

    Firdapse® (amifampridine) 10 mg tablets is an oral, nonspecific, voltage-dependent, potassium (K+) channel blocker that causes depolarization of the presynaptic membrane and slows or inhibits repolarization. This action results in the opening of slow voltage-dependent calcium (Ca2+) channels, allowing for a subsequent influx of Ca2+. In turn, it induces the exocytosis of synaptic vesicles containing Acetylcholine (ACh) to release more ACh into the synaptic cleft, enhancing neuromuscular transmission, and providing for improved muscle function.

    About Catalyst Pharmaceuticals

    Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton myasthenic syndrome (LEMS), anti-MuSK antibody positive myasthenia gravis (MuSK-MG), and spinal muscular atrophy (SMA) Type 3. Catalyst's new drug application for Firdapse® (amifampridine) 10 mg tablets for the treatment of adults with LEMS was approved in November 2018 by the U.S. Food & Drug Administration ("FDA"), and Firdapse® is now commercially available in the United States. Prior to its approval, Firdapse® for LEMS had received breakthrough therapy designation and orphan drug designation from the FDA. Firdapse® is currently being evaluated in clinical trials for the treatment of MuSK-MG and SMA Type 3 and has received Orphan Drug Designation from the FDA for myasthenia gravis.

    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 (i) whether Catalyst can successfully commercialize its product in Canada on a profitable basis, (ii) whether Catalyst will reach an agreement with a marketing and distribution partner to commercialize its product in Canada, (iii) whether Catalyst will have competition in Canada for its product, (iv) the impact in the United States if an amifampridine product is purchased in Canada for use in the United States, and (v) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2019 and Catalyst's 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.

    Investor Contact
    Brian Korb
    Solebury Trout 
    (646) 378-2923
    
    
    Company Contact
    Patrick J. McEnany
    Catalyst Pharmaceuticals
    Chief Executive Officer
    (305) 420-3200
    
    
    Media Contact
    David Schull
    Russo Partners
    (212) 845-4271
    
    

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