Catalyst Pharmaceuticals Pre-Announces Estimated Firdapse® Revenues and Provides Updates on Advancement of Clinical Developm...
January 06 2020 - 8:03AM
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 provided a corporate update including preliminary net revenue
for 2019, the status of Catalyst’s clinical development programs
and other corporate matters.
“Catalyst has completed its first year as a
commercial-stage company following the successful U.S. launch of
Firdapse® (amifampridine) for the treatment of adult LEMS patients
last January,” said Patrick J. McEnany, Chairman and Chief
Executive Officer of Catalyst Pharmaceuticals. “In reviewing the
launch metrics that we established, we are pleased that Catalyst
has exceeded all expectations for product revenues and the number
of patients who are currently being treated with Firdapse,
including the many individuals who for the first time have received
a medicine to treat Lambert-Eaton myasthenic syndrome (LEMS). In
2020 we will roll out additional programs to assist LEMS patients
and their healthcare providers. Furthermore, we are on track for
completing the MuSK-MG trial and reporting top-line data in the
first half of this year as we focus on expanding the possible use
of Firdapse as a treatment for other indications, while at the same
time we aim to broaden our entire product pipeline.”
Corporate Highlights
Preliminary Unaudited 2019 Financial
Results
- Catalyst expects to report
Firdapse® net product revenues for the treatment of LEMS of
approximately $30 million for the fourth quarter of 2019 and
Firdapse net product revenues of approximately $102 million for the
year 2019.
- Catalyst expects to report year-end
cash and investments of approximately $95 million and no funded
debt.
- No near-term need to raise cash for
operations through an equity offering.
The above information is based on preliminary
unaudited information and management estimates for the full year
2019, and is subject to the completion of Catalyst’s financial
closing procedures. Catalyst expects to report its 2019 results of
operations on or before March 16, 2020.
2020 Financial Guidance
- Catalyst anticipates full year 2020
Firdapse net product revenues for treating adult LEMS patients to
be in the range of $135 to $155 million.
- Catalyst anticipates GAAP R&D
and SG&A expense for the full year 2020 to be approximately
$65 million, which includes approximately $4 million in
non-cash compensation.
Advancing Neuromuscular Portfolio in
2020
- Anti-MuSK antibody positive
myasthenia gravis (MuSK-MG)
- 750 Myasthenia Gravis patients were screened to identify the 60
plus MuSK-MG patients who are participating in the trial. Note that
only about 5-8% of the estimated 60,000 myasthenia gravis patients
are MuSK antibody positive.
- Enrolled more than 60 MuSK antibody positive patients in 2019,
and anticipate enrolling a few more patients in the next few weeks.
- Enrollment beyond 60 patients is for ethical reasons due to
qualified MuSK-MG diagnosed patients already in the queue for
enrollment that have a strong desire to participate in the
trial.
- Remain on track to report top line results in the first half of
2020.
- Assuming positive data we would expect to file a supplemental
new drug application (sNDA) with the FDA about year end.
- Congenital Myasthenic Syndromes
(CMS)
- Extensive briefing package submitted to FDA for review
including our results from first and only double-blind,
placebo-controlled study of amifampridine in genetically confirmed
CMS subjects.
- FDA has advised us that the results of the study do not support
any types of CMS and amifampridine does not appear to have a
clinically meaningful benefit in the CMS patient population. They
further stated that controlled clinical data demonstrating efficacy
would need to be provided to support review of any indication for
CMS.
- Catalyst will continue to provide Firdapse to CMS patients who
are already enrolled in the CMS expanded access program and wish to
remain on therapy.
- Spinal Muscular Atrophy Type 3
(SMA)
- SMA-001 study (n=12) is ongoing, with five subjects having
already completed the study.
- Additional site has been opened for the study and on-target to
report top-line data in the first half of 2020.
- Long-acting formulation of
amifampridine
- Primary formulation development contractor retained, and
formulation development is ongoing.
- Gastro-intestinal tract drug absorption characterization in
human beings is expected to be completed in 2020.
- Initial formulation candidates are expected to be completed,
and their drug release and absorption properties are expected to be
determined in 2020.
- Additional neuromuscular conditions to be studied in 2020
- Kennedy's disease.
- Hereditary neuropathy with
liability to pressure palsies (HNPP).
Commercial status
- Nearly completed commercial
expansion that:
- Almost doubles the field sales team,
- Adds a newly established partnership with a rare-disease
experienced inside sales agency to generate interest among a much
larger group of over 9,000 potential prescribers and institution
providers targets, and
- Increases digital non-personal promotional activities to
supplement awareness generation created by the field sales and
inside sales teams.
- Added experienced digital and
social media focused marketer to enhance all Catalyst on-line
communication with LEMS patient community.
- Catalyst Pathways patient/caregiver
satisfaction levels remain high at 4.8 to 5.0 stars (n=290
respondents), resulting in significantly fewer transitions to
competitive therapies in November and December.
Portfolio Expansion through Business
Development
- Board of Directors’ direction to
develop a formal process for evaluating additional rare disease
opportunities
- Ideally acquisitions or in licensing opportunities in
neuromuscular or neurology therapeutic areas.
- Currently interviewing candidates
for the executive level business development position who will
oversee this program and bring further focus and formality to these
efforts.
- Strong balance sheet and expected
availability of conventional loans would support a range
of various opportunities.
Litigation Update on Challenge to FDA Approval of
Ruzurgi®
- Catalyst filed its Motion for
Summary Judgement on December 18, 2019; Defendants’ response is due
on January 17, 2020 and our Reply is due by February 7, 2020.
- If the briefing schedule is not
delayed, we would expect a decision mid-year 2020.
About Catalyst
PharmaceuticalsCatalyst 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 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 StatementsThis
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's forecast of expected net revenues for 2019
and 2020, and its forecast of year end 2019 cash and investment,
will prove accurate, (ii) whether Catalyst's forecast of 2020
SG&A and R&D expenses will prove accurate, (iii) whether
Catalyst will report top line results from its MuSK-MG clinical
trial and its SMA Type 3 proof of concept study during the first
half of 2020, and whether those trials will be successful, (iv)
whether Catalyst will ever be approved to commercialize Firdapse
for the treatment of MuSK-MG and SMA Type 3, (v) whether Catalyst
can develop and obtain the right to commercialize a long acting
formulation of Firdapse, (vi) whether Catalyst can successfully
increase its sales of Firdapse through the expansion of its
commercial team, (vii) whether Catalyst can successfully source
future acquisitions or in licensing opportunities and obtain
non-dilutive financing for such opportunities, (viii) whether
Catalyst will be successful in its lawsuit to overturn the FDA's
approval of Ruzurgi; (ix) whether any proof-of-concept pilot
studies that Catalyst undertakes evaluating Firdapse for the
treatment of additional neuromuscular diseases will be successful,
and (x) those factors described in Catalyst's Annual Report on Form
10-K for the fiscal year 2018 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
bkorb@troutgroup.com
Media Contact
David Schull
Russo Partners
(212) 845-4271
david.schull@russopartnersllc.com
Company Contact
Patrick J. McEnany
Catalyst Pharmaceuticals
Chief Executive Officer
(305) 420-3200
pmcenany@catalystpharma.com
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