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- Firdapse 2019 Net Product Revenues Expected to be Approximately $102 Million

- Anticipates Full Year 2020 Firdapse Net Product Revenues to be in the Range
of $135 million to $155 million

- MuSK-MG Clinical Trial Has Met Enrollment Goals and is on Schedule to Report
Top-Line Results in First Half of 2020

- Company is Accelerating Business Development Activities

CORAL GABLES, Fla., Jan. 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 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.