Full Press Release Details
Commercial Launch of Firdapse
Confirmation #13685524
PHARMACEUTICALS, INC.
Commercial Launch of Firdapse
Confirmation #13685524
Greetings and welcome to the Catalyst Pharmaceuticals conference call to discuss the commercial launch of Firdapse. At this time, all participants are in a listen-only mode. A question and answer session will follow the formal presentation. If
anyone should require operator assistance during the conference, please press star, zero on your telephone keypad. As a reminder, this conference is being recorded.
It is now my pleasure to introduce your host Ali Grande, CFO. Please go ahead.
Alicia Grande: Thank you, Operator. Good morning everyone and thank you for joining us today to discuss the commercial launch of Firdapse. Before we begin, I
would like to remind you that in the following comments and the question and answer session will include forward-looking statements based on current expectations. Such statements represent our judgment as of today and may involve risks and
uncertainties. Please refer to our filings with the SEC which are available from the SEC or on our website for information concerning the risk factors that could affect our company and the launch of Firdapse.
Commercial Launch of Firdapse
Joining me on today s call are Pat McEnany, our Chairman and CEO; Dan Brennan, our Chief Operating
Officer; Dr. Steve Miller, our Chief Commercial Officer and CSO; Dr. Gary Ingenito, our Chief Medical Officer; and Brian Elsbernd, our Senior Vice President of Legal and Compliance.
As we always do on our calls, we will take questions from analysts after our prepared remarks. I will now turn the call over to Pat.
Patrick McEnany: Thank you, Ali, and good morning and thanks to everyone for joining us on today s call. As you can imagine, this has been a very
exciting couple of weeks for the Catalyst team, and we were thrilled to have ended November with the FDA approval for Firdapse. This represents the culmination of many years of hard work and investment. After completing the comprehensive clinical
and nonclinical development programs that were required for us to obtain FDA approval of Firdapse as a treatment for LEMS, we are gratified to finally be able to deliver a much-needed evidence-based therapy to all LEMS patients in the U.S.
Commercial Launch of Firdapse
For nearly 40 years, LEMS patients have relied on investigational drugs with very limited access or off-label, unapproved therapies with no proven safety or efficacy to treat this debilitating disease. With the approval of Firdapse, we believe that there is new hope for the lives of these patients to change for
Firdapse is the first and only FDA approved evidence-based therapy that addresses the dysfunction in the neuromuscular junction, affecting
muscle contraction and strength of patients suffering from LEMS.
The most common symptoms of LEMS are proximal muscle weakness and abnormally rapid
fatigue. Currently, more than half the people with LEMS receive an initial misdiagnosis and they often wait years for a proper confirmed LEMS diagnosis. Symptoms can be life-threatening when the weakness involves respiratory muscles. Approximately
50% of LEMS patients have an underlying malignancy, typically small cell lung cancer.
Firdapse, or amifampridine, has been approved as a new molecular
entity, meaning that the molecule has never previously been approved by the FDA for any indication. That also means that Firdapse is not a repurposed drug or one that has previously been approved for another indication.
Commercial Launch of Firdapse
Catalyst and BioMarin have spent more than nine years developing Firdapse. Combined, we have conducted more
than 70 clinical and nonclinical studies, including two positive Phase III studies at a cost of tens of millions of dollars.
By treating LEMS patients
with Firdapse, doctors will be able to help these patients to improve their ability to conduct the simple daily activities that many of us take for granted. LEMS patients have a limited ability to care for themselves and often cannot hold a job
requiring standing, moving or walking and it is difficult for them to participate in everyday activities such as drying their hair, cooking dinner, going up and down stairs or walking down a long hallway. We believe that our efforts to study this
medicine and bring it through the FDA review and approval process will make a meaningful difference in the lives of LEMS patients and the lives of their caregivers and loved ones.
Commercial Launch of Firdapse
Our mission does not end with the approval of Firdapse for LEMS. As you know, we are
conducting continuing to evaluate Firdapse in clinical trials for other rare neuromuscular diseases such as congenital myasthenic syndromes, MuSK antibody positive myasthenia gravis, and spinal muscular atrophy type 3. Additionally, we are in
the early stages of our work to develop a longer-acting formulation of Firdapse that would require once or twice per day dosing versus currently three to four times per day. We hope that in addition to being a more patient-friendly dosage form,
leading to better compliance, it would also ensure an optimum level of efficacy throughout the day as well as throughout the night.
address the commercial opportunity that Firdapse represents. We previously reported from the literature in our third-party initiated research studies that there are approximately 3,000 adult LEMS patients in the U.S. However, based on precedence in
the orphan space, we have also noted on many occasions that there is a high degree of inherent variability with these numbers when using top-down prevalence estimates for rare diseases. This is due to the
small number of patients and the even smaller sample size used in prevalence studies. Please note that this is not unusual, especially with ultra-orphan diseases for which there is no approved therapies.
Commercial Launch of Firdapse
Recently, our Commercial Analytics Team initiated a study with a
zero-based bottom-up approach of the overall LEMS prevalence, as well as today s addressable diagnosed market to better quantify estimates. This process involved a
thorough review of published epidemiology studies, healthcare claims transactions as well as hospital discharge data and other triangulation methodologies. This process has led us to continue to believe that there are approximately 3,000 patients in
the U.S. who suffer from LEMS of which approximately half, or 1,500, have been definitively identified in claims data over the past two years. This estimate of the diagnosed patient population compares well with the epidemiology studies in the
literature, and we have defined the approximate 1,500 patients as our immediate addressable market. The other half have low visibility in the claims data or are misdiagnosed or undiagnosed. We are also planning steps to help the neurology community
identify the other half for further diagnosis and treatment.
As you probably know, another company has been providing a different form of 3,4
diaminopyridine to patients at no cost for a number of years as an investigational drug under INDs for patients with LEMS and CMS. We at Catalyst commend them for this effort. At the same time, Catalyst has also been providing Firdapse for several
years at no cost to patients as part of our Expanded Access Program. We believe that between the two programs approximately 10% of all LEMS patients in the patient community currently are on therapy. The reason for this low percentage is that until
now there has not been an FDA approved drug that provided the necessary ease of use, pharmacovigilance, FDA approved dosing administration and safety information. Physicians have expressed great concern with the difficult, cumbersome process
required to file and maintain their own investigator INDs for compassionate use. Also, many patients were required to travel hundreds of miles to have access to a participating physician for diagnosis and the receipt of drug. This presented a
challenging situation for many physicians and patients who were either not aware of or unable to access this potentially beneficial treatment.
Commercial Launch of Firdapse
Our rationale for pursing FDA approval for Firdapse was to ensure that all, all patients would have access to
an evidence-based treatment with safety and efficacy properly studied and characterized, helping more than just the 10% of patients who are currently in an early access investigational program.
With the FDA approval of Firdapse, any physician will soon be able to write a prescription for their LEMS patients diagnosed and in a few days have Firdapse
delivered to his or her door from a rare disease experienced specialty pharmacy. So no matter where they live in the U.S. they will have the drug delivered to their doorstep.
We recognize that the price for medicines and the insurance coverage barriers of significant concern to patients, physicians, healthcare providers, payers and
policy makers. We have a comprehensive array of services under the program we are calling Catalyst Pathways, which we designed to be a single source for personalized treatment support, education and guidance, including a comprehensive patient
insurance navigation and financial assistance program designed so that a patient is out of pocket a minimal amount of money. We will be further describing the scope and breadth of our Catalyst Pathways program in detail in a few moments.
Commercial Launch of Firdapse
Over the past six months we have completed rigorous pricing and access study with a highly regarded and
recognized consulting firm. This firm has specific, relevant experience in pricing multiple orphan drug products with commercial success. As part of this process, our Market Access Team met with most of the top commercial and government payers in
the U.S. to describe LEMS and introduce our upcoming approval of Firdapse. In addition, we have held focus groups with general neurologists and a neuromuscular specialist in order to obtain feedback about potential plans to enable affordable market
access to all patients. This information and other factors were considered in our establishment of the annual list cost of therapy.
Based on the results
of this extensive work, we have set the annual list price or wholesale acquisition cost of Firdapse for a typical patient based on average dosing in our clinical trials and the Expanded Access Program at $375,000 before discounts and rebates. At
this price level, it gives the experienced patients adherence and compliance rates, our pricing strategy reflects our focus on ensuring broad and sustainable coverage from both private and public payers and assistance for patients in need. We have
also factored in continued clinical investment in the product to evaluate it for other neuromuscular diseases as well as future development of a new sustained-release formulation.
Commercial Launch of Firdapse
Catalyst has not spared any effort to be prepared for a successful launch of Firdapse, introducing a
transformative milestone in the lives of patients in the U.S. who are suffering from LEMS and giving us an expanded patient population access to a first-in-class and
At this time I d like to invite Dan Brennan, our Chief Commercial Officer, to provide greater detail of our launch plans. Dan?
Dan Brennan: Thank you, Pat, and good morning to everyone on today s call. I ve been excited to be part of this company and team since the early
summer. I believe at the time I was Employee number 25. One of the very first elements of ensuring the successful commercialization of Firdapse was to assemble a truly world class Commercial Team of passionate, experienced and patient-focused
healthcare industry veterans and partners. These individuals came to Catalyst from leadership roles at rare disease biopharmaceutical companies, non-profit patient organizations, rare disease patient support
service and specialty pharmacy organizations. Many bring specific expertise in successfully launching orphan neurology products. This is a tight group of highly experienced team members committed to making a difference in the lives of all LEMS
patients and their family members.
Commercial Launch of Firdapse
One of the first areas where we were able to leverage our rare disease experience was in our discussions with
payers to provide a disease state overview and the value proposition that Firdapse offers their LEMS patients. Our market research shows that most LEMS patients have insurance coverage and that breakdown is approximately 30% with commercial plans
versus 40% Medicare, 15% Medicaid, 10% dually eligible for both Medicare and Medicaid, and about 5% with other federal programs or cash pay. We have had several national account managers with rare orphan drug experience targeting and speaking with
over 70 key payer accounts that cover over 80% of the U.S. insured population.
In these discussions and extensive research with payers, we were able to
describe the difficulty that people suffering with LEMS experience in their daily lives and the overall medical and personal burden placed on the healthcare system without having any approved treatments. Not surprisingly, the payers were not
immediately aware of LEMS given the small number of patients, but after reviewing the disease and the impact it had on their patients, they realized that there is a clear, unmet need for treatment options with established safety and efficacy. They
also appreciated that this is a rare condition, currently affecting approximately 3,000 patients and fewer diagnosed patients.
Commercial Launch of Firdapse
Finally, they understand that as an orphan disease, the exclusivity and orphan pricing window is seven years
before generic price and competition is available.
As our team continues to further discussions now post approval, we expect our clinical study results
with sustained improvements and a relatively straightforward safety profile will establish the value Firdapse can bring to these LEMS patients who may be rare but are certainly in need of an effective therapy.
Now, by straightforward safety profile, I mean that due to the risk of seizures and allergic reactions, Firdapse is contraindicated in patients with a history
of seizures or allergic reactions to any aminopyridine product. The most common adverse reactions that occurred in more than 10% of trial subjects are paresthesia, which is a tingling or prickling sensation, upper respiratory tract infection,
abdominal pain, nausea, diarrhea, headache, elevated liver enzymes, back pain, hypertension or muscle spasms.
Commercial Launch of Firdapse
Based on the direct feedback we ve had from payers, we expect the majority of them to cover and