Greetings and welcome to the Catalyst Pharmaceuticals First Quarter 2020 Results Conference. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. As a reminder, this conference is being recorded. It’s now my pleasure to introduce your host, Ms. Ali Grande, CFO. Thank you. You may begin.

Good morning, everyone, and welcome to today's conference call. Joining me on today's call are members of the Catalyst team, including Patrick McEnany, Chairman and Chief Executive Officer; Dr. Steven Miller, Chief Operating Officer and Chief Scientific Officer; Dr. Gary Ingenito, Chief Medical Officer and Head of Regulatory Affairs; and Dan Brennan, our Chief Commercial Officer.

Thank you, Ali, and thanks everyone for joining us this morning for our first quarter results and business update call. I hope that everyone is staying safe and healthy during these unprecedented and challenging times. I'd like to start by saying that our priorities as a company are ensuring the safety and well being of our Catalyst team members, safeguarding an uninterrupted supply of Firdapse to an adult LEMS population, and supporting healthcare providers and patient communities. In addition, we have catalysts who are working diligently to complete clinical studies, evaluating Firdapse for other rare neuromuscular diseases, including MuSK antibody positive Myasthenia Gravis. I'm proud of how quickly our team has adapted to the COVID-19 business environment. On March 16, we announced the suspension of all travel and face-to-face interactions with physicians, patients, or other contractors or vendors, including clinical trial sites. All employees are required to work remotely from home as we've all expanded the use of digital communications, as well as other enabling technology to conduct our daily tasks. During this first quarter, we demonstrated solid execution across all functional areas of the company. Our first quarter revenues were minimally impacted by COVID-19 as we recorded 29.1 million in net revenues, achieving our internal revenue forecast versus $12.4 million for the first quarter of 2019. This represents a 134% increase quarter-to-quarter, recognizing that the first quarter of last year was first quarter of the launch of Firdapse. During the first quarter of 2020, we generated an after-tax operating profit of $10.4 million or $0.10 per share basic and fully diluted. We also ended the quarter with almost $102 million in cash and investments and no funded debt. The current patient days that is on reimburse Firdapse is fairly stable. Remember, the LEMS is a chronic disease, and Firdapse is an oral medication administered at home and delivered monthly to the patient’s residence.

Thanks Pat, and good morning, everyone. We are happy with our continued success helping adult LEMS patients with Firdapse now that we have officially moved out of our launch year. With 46 new patients prescribed Firdapse in the first quarter, over 575 patients have now received a Firdapse prescription from initial launch to the end of the first quarter of 2020. We were excited to see a noticeable uptick in new patient enrollments in January and February, compared to the last few months in 2019 as a direct result of our updated commercial strategies, field force expansion, and initiation of a well run inside sales lead generation and communication effort. March new patient enrollments were ahead of forecast and it was even looking like we might have our first month of 20 plus enrollments since mid-last year, but as Pat mentioned, on March 16, we pulled our Salesforce from the field and began to see the impact of a greatly reduced number of LEMS patient office visits. As you may know, LEMS patients have two separate key risk factors, making them susceptible to severe COVID-19 disease and that they are often over 60 years old, and have weakened immune systems. They have been rightfully worried about venturing out to hospitals or physician offices, for anything other than emergency care. As Pat already indicated, our first quarter net sales of 29.1 million was at our target, but I will explain how we arrived there for a few different reasons than expected. We felt the beginning of the year headwinds normally experienced with medication in an ultra rare disease area, with patients changing insurance carriers, the reset of deductibles and and the payer reauthorization process In our favor was the increased number of new enrollments mentioned earlier, as well as an unforeseen slight increase in forward purchasing of prescriptions that patients requested in order to ensure that they had enough medication on hand to address their anxiety about medical supply shortages in-light of the coronavirus pandemic.

Thanks for the commercial update Dan, and now I'll provide an update on our clinical pipeline to develop Firdapse for additional neuromuscular indications. First, I will turn to our Phase 3 trial and MuSK MG to evaluate the safety, tolerability, and potential efficacy of Firdapse for the symptomatic treatment of MuSK Myasthenia Gravis. As we reported in March, we completed and exceeded our enrollment goals for this Phase 3 trial, and I'm pleased to report that we successfully completed all subject visits by mid-April for the clinical portion of the trial and collected all necessary clinical endpoint and safety data. When the status of this clinical trial was reported in March, we indicated the top line results would be reported in the first half of 2020. This was based on statements by public health authorities and government officials at that point in time regarding when the COVID-19 related restrictions and mitigation strategies would be eased, and our assessment of what impact the timing of those COVID-19 restrictions would have on the trial. By the time of our April 8 COVID-19 update press release, only a few subjects needed to complete their last visits for the sites in Italy, Serbia, and the U.S.; about this point in time Serbia also announced restrictions on the conduct of clinical trials that fortunately did not materially affect the completion of the last MuSK MG patient there, but did affect the SMA proof of concept trial, which I'll discuss in a moment. Since that time, it has become clear that COVID-19 restrictions and mitigation strategies will be eased later than we previously anticipated, and that the easing of restrictions as was recently described by the White House will be a more gradual three phase process. These prolonged restrictions are delaying the completion of required regulatory,…

Thanks Steve. Yesterday we filed our first quarter Form 10-Q and reported GAAP net income of 10.4 million or $0.10 per basic and diluted share, compared to a GAAP net loss of 645,000, or $0.01 per basic and diluted share in the same period of 2019. For the first quarter of 2020, non-GAAP net income, excluding 1.5 million of expenses related to non-cash stock based compensation was 11.9 million or $0.12 per basic and $0.11 per diluted share. In comparison, first quarter 2019 non-GAAP net income excluding 933,000 of expenses related to non-cash stock based compensation was 289,000 or $0.00 for basic and diluted share. Net product revenue for Firdapse, which was launched during January 2019 was 29.1 million for the first quarter of 2020 with the related cost of sales of 4.2 million. For the first quarter of 2019, net product revenue Firdapse was 12.4 million with related cost of sales in the quarter of 1.7 million. It's important to remember that our gross margins continue to benefit from the inventory manufacturer and expense prior to the FDA approval of Firdapse. Research and development expenses were 4.2 million for the first quarter of 2020, as compared to 3.3 million for the first quarter of 2019. Research and development expenses for the first quarter of 2020, and the first quarter of 2019, primarily consisted of expenses for medical and regulatory affairs, a quality assurance programs, expenses from our ongoing Firdapse clinical trials and studies and cost for our Expanded Access Program. We expect that research and development costs will continue to be substantial in 2020 as we complete our ongoing clinical trials and studies in MuSK MG and SMA Type 3 continue our Expanded Access Program and our sustained release formulation program for Firdapse. Began to evaluate Firdapse as a treatment for other neuromuscular diseases, and assuming positive results from the trial, supplemental NDA for Firdapse for the treatment of MuSK MG. SG&A expenses for the first quarter of 2020 total 10.1 million, compared to 8.4 million in the first quarter of 2019. The increase when compared to the same period in 2019 is primarily attributable to increases in headcount due to the expansion of the sales force or contracting with a rare disease experienced inside sales agency and an increase in non-cash compensation expense. We expect that SG&A will continue to increase in 2020 as we continue to build our infrastructure and commercial and patient programs in support of our sales activities for Firdapse and continue to pursue our lawsuit against the FDA. On March 31, 2020 Catalyst had cash in investments of 101.8 million and no funded debt. More detailed 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 yesterday, May 11, and can be found on the investor relations page of our website at www.catalystpharma.com. Now, I’ll turn the call over to Pat.

Thank you, Ali. Again, I'm very proud of the entire Catalyst team and the way in which our organization has come together during this global pandemic. We are committed to bringing life saving treatments to patients in need and in these challenging times we will continue to fully support our patients, healthcare providers, and the broader community. We started of 2020 with a strong execution and expect to continue their momentum through the balance of this year. Despite the difficult times that we face today, I have never been more optimistic about the prospects for our company in the second half of this year and beyond. Thank you again to everyone who joined us this morning. And with that, we'll open the lines for questions. Operator?

Thank you. Our first question is coming from Joe Catanzaro of Piper Sandler. Please go ahead.

Hi, guys, thanks so much for taking my questions here. Maybe first a couple on the commercial launch year. I'm wondering if maybe you'd be able to provide some metrics that you previously provided, namely, how many patients who are actively receiving insurance reimburse Firdapse at the end of 1Q. And, Dan, you noted that 90 day discontinuation rates continue to improve, just wondering at this point, if you have a good sense of how many new patient starts you need per quarter to outpace the rate of existing patients coming off therapy? Thanks.

Sure. So, the first question again was on the – can you repeat the first part, I got the second part on the 90-day discontinuation?

Yeah. So the first part is with regards to a metric I think you guys have previously provided around how many patients were actively receiving insurance reimbursed Firdapse at the end of 1Q?

Yeah, I mean, we're trying to – now that we're through the launch year, and we were able to give you a lot of metrics to get into detail, we're trying to just, you know, rely on the sales levels to give you – from here on out, you know, clear understanding of our performance. We did have somewhere in the neighborhood of 335 patients to 340 patients that were reimbursed, but some of these metrics, again, we gave them last year, so you had an idea of where things were going, but they're variable and it gets complex. Like, for example, in the first quarter, that number jumps around because a lot of patients are changing insurance. They're getting reauthorizations done and such. So, that's why we’ve stuck with the revenue level. As far as the 90-day discontinuation rate, yeah, we're very happy with that. I think I mentioned last year in the middle of the year, it was around 35%. By the end of the year, it got down to 25%. We're actually now looking like it's lower than 20%, and we hope that holds, did a lot of the programs that we have in place. And so that is an important metric for us. And in order to get the growth that we are hoping and ultimately with the previous guidance we need to be in the neighborhood of 15 patients to 20 patients, new patient enrollments per month in order to get that type of growth.

Okay, got it. That's very helpful. If I could just asked one follow-up, Dan, I think you also mentioned that there was maybe an uptick in long term, longer-term prescriptions towards the end of the quarter, just wondering if you could elaborate a little bit on that and how that could potentially impact 2Q revenues? Thanks.

Right. We were first very interested in making sure that we were – making sure that the patients had coverage and that their anxieties were at ease with regards to being able to get continued supply. And so we did see in the middle and end of March, a little bit of additional prescribing. So, perhaps a few advanced scripts coming in normally would have waited until April coming in at the end of the month, and some small percentage of patients that went up to 60 days or 90 days, but overall, it didn't have a very large impact. And we think it's because we've provided such good service to them, and they have good confidence with us. We communicated in advance. And so the impact really is somewhere in the one-to-one and a $0.5 million range, which will ultimately resolve throughout the rest of the year.

Okay, got it. That's very helpful. Thanks so much for taking my questions.

Thanks, Joe.

Thank you. Our next question is coming from Charles Duncan of Cantor Fitzgerald. Please go ahead.

Good morning, Charles.

Yeah. Good morning Pat and team. Thanks for all the information and appreciate the challenges that you have as there's a lot of folks in terms of managing this current pandemic. I wanted to ask you a question and then a couple for Dan and maybe one for Steve, with regard to you, as you think about whether or not to withdraw versus widened guidance, I'm wondering if withdrawing is in abundance of caution or are you seeing anything new? Just kind of let us know your philosophy on why now you would withdraw versus widened guidance?

Yeah. We gave a lot of thought to that Charles, and we talked a great deal about it. At the board level as well as with the audit committee, and we just felt if we were not comfortable with in this environment and leaving that guidance out at , which was already a fairly wide range. We didn't think it'd be appropriate to lower the guidance or to widen it, and then perhaps have to come back in a month or two or three, and do it again. So until we have a better handle on what things look like for us and for the healthcare community at large, we felt it was the prudent thing to do. It's nothing more than that. It's there is no trend. In fact, you know, we're extremely optimistic about, you know, the qualitative direction of the company, and what we're seeing out there on the commercial side. So, I hope that answers your question.

Yeah, it does. And I appreciate the challenge and I appreciate the conservatism. Hopefully it proves to be the case. Maybe if I could ask Dan, a couple of questions related to that, which is he mentioned increase use of virtualized platforms in reaching out with two potential prescribers. And I'm wondering, Dan, if you could provide any additional, I guess, information, what those platforms may be not specific names, but really the strategy behind them?

Well, I think, it mainly gets to staying in contact with physicians and continuing to remind them that patients with rare disease have significant issues. And so, we're doing that and quickly move to approve a lot of our materials that we had in hand to be able to be emailed that we have increased our digital presence and reminders to physicians online with banners and such, that drives them to our websites and ask them to request for a phone consultation and such. We're having virtual launches. And so we have all of those in place and we still are seeing new patient starts, but it is very evident that patients and physicians just aren't seeing each other, and to some extent with telemedicine that helps, but we can really tell that physicians and patients just aren't seeing each other during this time.

Make sense. And we've asked the question of many of our neurology and psychiatry focused revenue generators, but I guess I'm wondering if you could characterize at least qualitatively your discussions with prescribers in April versus May and what would you anticipate in June using those virtualized formats?

Well it really does depend upon the physician and the system that they're in, in the hospital to some extent and it's all over the board. Yes, physicians, perhaps in academic centers that are overrun with COVID, and they've completely shifted resources that don't want to talk to anyone. And you have others that are like yeah, I have some time to talk and I appreciate your willingness to provide this, you know a virtual launch and have a discussion with me about your programs that you still have in place and they're curious and so it does carry the gamut. Some physicians enjoy being able to communicate back and forth with e-mail and will reach out to us and talk to a sales representative if and when they have a patient that actually has called or come in to get a diagnosis, and we've seen some of those, just even in the last week, week and a half where patients are actually still getting diagnosed and whether that be to continuing to get antibody tests or . We're pleased to see that that's still happening. But Charles, it's really all – it’s all over the board.

Okay, appreciate that. One last question for Steve and that is related to the ongoing MuSK MG study. Steve, if you could remind us or update us on your kind of view on the conduct of this study, appreciate that, it's almost wrapped up, but as you look at, you know, measures of quality or heterogeneity, dispersion within the enrolled sample, how do you feel about that and what drives your cautious optimism on the outcome of that study?

Thanks Charles. As I mentioned in my opening remarks, the patient population is relatively homogeneous for MuSK. And by that I meant that all the MuSK Myasthenia Gravis patients’ disease is caused by a single antibody to a single protein in the neuromuscular junction. And so what happens is that neuromuscular junction and that protein in that junction has a very specific function, and so the expectation, when you have a disease that is so specific, is that there should be quite a bit of homogeneity and the type of damage that's done to the neuromuscular junction, as well as the clinical presentation of the patient. They all have similar symptoms and similar remedies related to the disease. Now, more importantly, that homogeneity and the fact that it's a single protein that's affected also results in homogeneity to response. We actually saw that in the Lambert-Eaton myasthenic – LEMS myasthenic disease, where the patients had an antibody to voltage gated calcium channels, and in that case, all of the patients had a very similar clinical presentation, a very similar response to treatment, not only in our two trials, but in other trials that were published in the literature as well. So, there's a lot of similarity in terms of homogeneity of the patient population, and so because of the fact that we had a very robust result in our proof of concept trial for the treatment of MuSK Myasthenia Gravis, coupled with the fact that the disease – the cause of the disease, as well as the clinical presentation appears to be very homogeneous. We believe that we should see similar kinds of findings for this ongoing trial and we remain cautiously optimistic about the outcome of the current trial.

Very good. Thanks for all the added color and look forward to gaining additional traction over the course of the year. Thank you.

Thanks Charles.

Excuse me. Thank you. Our next question is coming from Leland Gershell of Oppenheimer. Please go ahead.

Hey, good morning, Pat, thank you for taking my questions. Congratulations on navigating this, you know, this whole situation that we're all faced with so successfully. Wanted to ask your perspective on business development activities, you know, how those may have been impacted by COVID-19 itself, perhaps by maybe some more uncertainty around current year financials with regard to the Firdapse business, perhaps you can comment on your efforts there? And any focus that you may be directing your efforts towards in terms of, you know, particular projects or assets that may be of interest in the near future? Thanks.

Thanks for the question Leland. We stated earlier that we are really not looking for early stage opportunities in terms of product opportunities, looking for later stage beyond proof of concept. Ideally, we'd like to find projects that we could bolt-on with our sales reps meaning neuromuscular or at least in neurology in nature. And so, our criteria is fairly tight right now. We are seeing a fair amount of opportunities. And I think it's because of that we are now seeing all of these opportunities. I think also, the pandemic has probably caused us to see more opportunities than we normally would because companies are concerned about their next round of financing. And through the capital markets are open, I think the valuations are ground down pretty good. And so I think it's a very, very fertile environment for us to find the right opportunity of a product acquisition and or a company at this time.

Thank you. That’s helpful. Also, just a quick question on the Firdapse business itself, now that you've been marketed for some time, I was wondering if you could design from your experience maybe harder to do from the numbers that we see, but any particular seasonality that you've determined from Firdapse as it's launched into LEMS, and any seasonality that may affect the LEMS population or the overall neuromuscular patient population? Thank you.

Dan, you want to take that question?

Yeah, I mean, we haven't to date seen any of that seasonality, mainly because you know, last year in the summer months, we were really in the launch growth phase. And – but we would expect that the summer months would have an increased level of prescriptions just because heat actually does affect the neuromuscular junction negatively. And so, you actually have an increased symptomatology with the summer months. So, we're encouraged by that concept, and we'll be ready looking for that, but we didn't – we haven't seen it thus far.

Okay, that's great. Thanks very much for the added information.

Thank you, Leland.

Thank you. Our next question is coming from Scott Henry of ROTH Capital. Please go ahead.

Thank you, and good morning. Just a couple questions around the launch, just trying to get my arms around it, you know, for clarity, it sounds like, you know if the churn rate is, you know, call it 15%, are you seeing really any new patients now at this point given COVID-19? I guess, what I'm getting at is we, it sounds like we should expect at least some decline in 2Q versus first quarter. Just want to make sure I am interpreting that correctly.

Scott, let me address it, and then I'll turn it over to Dan. You know, our reimbursed – number of reimburse patients on Firdapse is fairly stable and we expect that – and those that are on drug for longer than 90 days, I'm including in that – in a category are really stable and we view that as a pretty good base. Now, people do drop out, they cancel. They just continue. They unfortunately some pass away. But that's a fairly stable number. And albeit, you know, we've expanded double our field force, and as Dan mentioned, we've employed a lot of new technology to reach patients and to reach physicians. And despite the fact that in April, we saw a downtick as compared to January, February, and March where we were starting to get on a roll before the pandemic. Having said that, we weren't happy with the reduction, but we are enrolling new patients. And I don't want to get into numbers again. I think that over the past year, the launch year we've provided a lot of different launch metrics to investors into analysts. And we felt it was important to do so. We think now the most important thing and we'll rest on our revenues as we move forward. And I think that it's important to understand, as we talked about earlier, the qualitative direction of what is actually happening in the patient and physician community with Firdapse. So, again, we would all like to be sitting here with better numbers in Q2 than maybe we're seeing for the moment, but it's not falling off a cliff. And we are enrolling new patients. So Dan, you may have something you want to add to that.

I'm not sure if I have much more to add unless, Scott, you had some additional color you were looking for.

No, I think that was helpful, right there. But I guess in a related question, from a pricing standpoint, are we now at a time where the revenue per patient is relatively stable as well and you know, over time, you know, would you expect that to creep up for one reason or another? How should we think about that revenue per patient trend?

I don't see any significant changes to the revenue per patient trends.

Okay. And I guess further, any color on at least qualitatively, what you're thinking about how the Jacobus launch is preceding, and are you noticing any impact from that relative to the last quarter, or does it remain relatively modest?

It remains relatively modest. I mean, we did see again with the change of insurance and reauthorizations, a small uptick again at the very beginning of the year that has again subsided. So, I think it still becomes a minor impact, and really what matters for us and that we've seen was working and especially working with the expansion of our sales force and employing this inside sales is communicating to many physicians who, quite honestly are just seeing their first to second lens patient and are happy to hear that there's a medication available, and they're quickly getting on to Firdapse. And that's for us, getting back onto that pre-COVID trend of new patient enrollments is the name of the game.

Okay, great. And then final question, just for clarification, I think you said there were 46 new patients in the quarter, are you distinguishing between those naive to therapy, or you know, perhaps switches who have used it prior?

Yep, for the most part, you know all of those perhaps other than one or two are naive previously never on 3, 4 DAP and so these are truly patients that have either already been diagnosed and just hadn't had access to this type of great medicine or they were people that were newly diagnosed.

Okay, great. Thank you for taking the questions.

Thank you, Scott.

Thank you. At this time, I'd like to turn the floor back over to Pat for closing comments.

Thank you for joining us today. We look forward to further updates. Please be safe and have a great day.

Ladies and gentlemen, thank you for your participation. This concludes today's event. You may disconnect your lines at this time and have a wonderful day.