Greetings, and welcome to the Catalyst Pharmaceuticals Incorporated Second Quarter 2017 Financial Results Conference. [Operator Instructions] As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Ms. Alli Grande, Chief Financial Officer. Thank you. You may begin.

Good morning, everyone, and thanks for joining our conference call. On today's call, we have Pat McEnany, Chairman and Chief Executive Officer; Mr. Steven Miller, Chief Operating Officer and Chief Scientific Officer; and Dr. Gary Ingenito, Catalyst's Chief Medical Officer and Head of Regulatory Affairs. Before we begin, I would like to remind you that in the following comments and in the Q&A session, we will make statements about expected future results, which may be forward-looking statements for the purpose of the federal securities law. These statements relate our current expectations, estimates and projections, and are not guarantees of future performance. They involve risks, uncertainties and assumptions that are difficult to predict and which may prove not to be accurate. Actual results may vary. These forward-looking statements should be considered only in conjunction with the detailed information contained in our SEC filings, including the risk factors discussed in our annual report on Form 10-K. At this time, it is my pleasure to turn the call over to Pat McEnany, our Chief Executive Officer.

Thank you, Alli, and good morning, everybody. Thanks for joining us today and welcome to our second quarter 2017 results call. We look forward today to providing you with a report on our recent activities and progress. Steve Miller will provide a status report on our pipeline and Alli will review our financial results for the second quarter. We will then take your questions at the end of the call. During the second quarter, we continued to work towards completing enrollment of our clinical trials evaluating Firdapse to treat patients suffering from Lambert-Eaton Myasthenic Syndrome or LEMS and congenital myasthenic syndromes CMS. We remained focused on submitting an NDA for Firdapse by year-end as well as developing a plan to pursue a path forward for Firdapse as a potential therapy for patients suffering from muscular antibody-positive myasthenia gravis or MuSK-MG. Last quarter, we received positive top line results for the use of Firdapse as a potential treatment for MuSK-MG, which was very encouraging. As there is currently no FDA-approved therapy for this neuromuscular disease. We are also pleased to see that a poster highlighting the results of the stage IIb study for Firdapse and the treatment of MuSK-MG was presented at the 13th International Conference on Myasthenia Gravis and Related Disorders in New York in May. As a result, we are now in discussions with the FDA regarding the design of our registration trial. We remained focused on our second Phase III trial that began in December of 2016, known as LMS-003, which is evaluating the safety and efficacy of Firdapse in patients with LEMS. This trial is progressing and we're looking forward to completing this trial during the second half of this year. Assuming positive results, we plan to resubmit our NDA for Firdapse later this year. In May,…

Thanks, Pat. And good morning, everyone. As Pat mentioned, we are continuing to enroll subjects in our second Phase III trial of amifampridine phosphate for the symptomatic treatment of LEMS and are nearing completion of enrollment. We reiterate our expectation, that we will complete this trial in the second half of 2017. We are also making progress in enrolling patients in our CMS trial, evaluating amifampridine phosphate for the symptomatic treatment of CMS. Due to the rarity of this condition, enrollment in the CMS clinical trial has proceeded slower than we originally expected and we now anticipate completion of this trial during the first half of 2018. We do not expect a delay in the completion of enrollment for the CMS clinical trial to delay the resubmission of our NDA for Firdapse for the treatment of LEMS. We also anticipate submitting in any NDA that we file for Firdapse, information regarding certain forms of CMS that are mechanistically similar to LEMS. While this approach has been discussed with the FDA, there could be no assurance that the information provided will be sufficient to justify the granting of an indication for CMS on this basis. Our Expanded Access Program continues to provide access to amifampridine phosphate tablets free of charge for patients, who have no other treatment options. And for which the patients' treating physicians feels, this could improve their disease condition. The program is currently designed only for patients diagnosed with LEMS, CMS or Downbeat Nystagmus. As a reminder, last quarter, we announced the positive results of a Catalyst supported investigator sponsored clinical trial of amifampridine phosphate for the treatment of myasthenia gravis patients, diagnosed with the MuSK antibody subtype of this disease. Patients diagnosed with MuSK-MG constitute approximately 5% to 8% of the overall myasthenia gravis population. And we estimate that there are about 3,000 to 4,800 patients in the United States, with this type of myasthenia gravis. The patients in this trial tolerated amifampridine phosphate very well and exhibited significant clinical benefit from the treatment. In spite of the small sample of only 7 patients, statistical significance was reached for all endpoint measures. The results of this proof for constant trial were presented in a poster at the 13th International Conference on Myasthenia Gravis and Related Disorders in New York City on May 15, by Dr. Silvia Banano and we anticipate that the full results of this clinical study will be published in the first half of 2018. These results are particularly promising because there are currently no generally recognized effective treatments for MuSK-MG. We look to further pursuing this indication and hopefully providing some much needed relief to this population of patients. Catalyst is currently in discussions with the FDA on the design of an appropriate trial for this indication. And subject to availability of funding, we anticipate initiating a multicenter Phase III trial in the second half of 2017. I will now turn the call over the Alli to review our financial results.

Thanks, Steve. All references to per share in this call refer to basic and diluted shares. For the quarter ended June 30, 2017, Catalyst reported a GAAP net loss of $3.9 million or $0.05 per share compared to a GAAP net loss of $4.6 million or $0.06 per share for the same period in 2016. Excluding a noncash gain of $210,000 attributable to the change in fair value of liability-classified warrants, non-GAAP net loss was $4.1 million or $0.05 per share for the second quarter of 2017. In comparison, non-GAAP net loss for the second quarter of 2016 was $4.7 million, or $0.06 per share, which excludes non-cash gain of $153,000 for the change in fair value of liability-classified warrants. Research and development expenses were $2.5 million, for both the second quarter of 2017 and 2016. We expect that our R&D spend will continue to be substantial, throughout the balance of 2017 and into 2018. General and administrative expenses for the second quarter of 2017 totaled $1.7 million, compared to $2.3 million in the second quarter of '16. We expect G&A expenses, excluding pre-commercialization expenses, to remain consistent for the balance of 2017. We also expect pre-commercialization expenses, which are reported as part of G&A to increase in the second half of '17 as we begin to prepare for our potential 2018 launch of Firdapse. As a development-stage pharmaceutical company, Catalyst had no revenues in either the second quarter of 2017 or 2016. During the quarter ended June 30, 2017, Catalyst received proceeds from exercise of warrants of approximately $1.8 million. At June 30, Catalyst had cash, cash equivalents and investments of $35.1 million and no debt. Although there can be no assurance, we believe that these resources will be sufficient to support our planned operations through at least the next 12 months. 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 yesterday August 9, 2017, and can be found in the Investor Relations page of our website at www.catalystpharma.com. I'll now turn the call back to Pat.

Thanks, Alli. We are very excited about all of the progress that we have made on our Firdapse trials for LEMS and CMS, and look forward to completing enrollment and focusing on the path towards FDA approval of Firdapse. We look forward to sharing additional updates on our products and on the status of our development programs for all of our products over the coming quarters. This will be the end of our formal portion of the meeting and we'll turn it over to Donna for questions.

[Operator Instructions] Our first question is coming from Charles Duncan of Piper Jaffray.

Pat, wanted to ask you a question about LMS-003, I believe there were a couple of minor differences, seems minor differences between LMS-002 and LMS-003. And one of them, I just wanted to get clarification on and that is that in addition to enrolling patients from your Expanded Access Program for 003, you also had a primary endpoint assessment at day 4, versus in 002 that was day 14. I guess, I would like to hear your perspective on -- if that changes the probability of success for 003?

Sure, great question, Charles. I'll turn that over to Steve Miller.

Thank you, Charles and thank you Pat. The first trial, LMS-002, had a primary end-point on Day 14, which you pointed out, but also had a secondary endpoint of SGI and QMG at day 7. Furthermore, the LMS-002 trial had a dose taper for those patients that were randomized to placebo. As -- if you'll previously recall, the LMS-002 trial was a discontinuation design, where all patients entered the trial on the test medication. Half of those patients during the randomization period continued on the test medication and the other half of those patients were randomized to placebo. Well, because of those taper, what that means is that even though there was a measurement at day 7, the actual period of time for those patients, who were randomized to placebo, they were actually on no medication or a subtherapeutic amount of the medication was perhaps only 3 or 4 days. And we observed a very robust clinical response at day 7 in the LMS-002 trial and achieved very robust p-values for those endpoints as well. Therefore, we feel very confident that an exposure period of 4 days in the LMS-003 trial will be achievable and we remain very -- remain optimistic, that we will achieve statistical significance at the completion of this trial.

And you feel good about the quality at patients? I mean, it would seem that since most of them are coming from the expanded access that would be a nonissue for this trial?

We also feel very confident about the patient population. Everyone in our Expanded Access Program entered the Expanded Access Program because of a confirmed diagnosis with Lambert-Eaton Myasthenic Syndrome and has been under treatment for -- in some cases several years with Firdapse and remains on the test medication, because the medication is providing significant clinical benefit to them. Therefore, we believe that the patient population that we're drawing the LMS-003 patients from is a patient population that has confirmed Lambert-Eaton Myasthenic Syndrome and has shown benefit from LMS-003. And therefore we expect to see a robust clinical signal in LMS-003.

And post the trial due to the patients have the opportunity to go back on the EAP and if so, have they done so?

All of the patients have reentered the Expanded Access Program. In fact, the logistics with how this study works, is patients travel to the clinical trial site for LMS-003 with their expanded access medication, that is turned over to the site investigator for the period of treatment in LMS-003 and then their expanded access medication is returned to them at the conclusion of LMS-003. So they technically are essentially not leaving the Expanded Access Program, they're simply taking a short hiatus from it, while they participate in LMS-003.

And also they are just hopping over to the CMS trial, you mentioned the delay in terms of enrollment. I'm wondering if you have any thoughts on why that is happening and if it can be reconciled? And secondarily, when you consider the design of the Phase III for CMS versus that of LEMS, are there any differences that are notable, that you would like to point out that may impact the probability success in CMS versus LEMS?

Sure. Well let me answer your second question first. The design of the CMS trial is a crossover design. We anticipated due to the rarity of CMS, that there would be more difficulty in recruiting patients for the CMS clinical trial. And so we used a crossover design in order to maximize the power of the study. The study is designed to recruit about 20 patients, which is a smaller number than our previous -- than both the LMS-002 and LMS-003 trials. But because of the crossover design, we believe it will be adequately powered to achieve statistical significance for the primary endpoints of the CMS clinical trial. With regard to the recruiting of patients, due to the rarity of the condition, it's harder to find those patients. But there is also other logistical concerns, which are that, as a crossover design trial, the patients have to conduct more site visits over a longer period of time and as a result of that, it becomes increasingly difficult to get patients to want to participate in such a trial, simply for logistical reasons. Also CMS -- the CMS trial has more clinical trial sites and the difficulty -- there is some added difficulty in traveling to those sites again because of the increased number of subject visits. So all of those conditions combine with the fact that CMS is more rare than LEMS have resulted in a recruiting period, that's longer than we previously anticipated for that trial.

But has that reconciled or do you have confidence, that you'll be able to achieve the enrollment goals, now as defined?

We do have confidence based on how many patients have already been enrolled in the trial that we will achieve our enrollment goals in the first half of 2018.

And one more, I am just intrigued with regard to the regulatory strategy on CMS. Sounds like, in the initial NDA on LEMS, you'll be providing some information that suggests CMS is very similar to LEMS, at least in certain types of patients, and therefore seeking a broader label than just LEMS. But with the CMS Phase III, later reading out, would you see that as a major amendment? Have you discussed this with the agency and what has been their feedback on that?

We haven't discussed, how the CMS trial will be submitted to the FDA and the post approval, after the approval of the original NDA. The reason for that is simply because it's only recent been decided by us, based on the current enrollment rates, that this would -- this trial would complete after we submitted the NDA. However, we have had more than one discussion with the agency regarding a submission strategy, that involves a subgroup of CMS genetic defects, that are mechanistically similar to LEMS. And I'll remind you that LEMS is a condition in which there is a failure to release adequate amounts of acetylcholine at the neuromuscular junction. And that inadequate release is due to the fact that, there is an antibody that attacks the voltage gated calcium channels that initiate the biochemical cascade that ultimately results in the release of acetylcholine. There are certain genetic subtypes of CMS, that also are -- result in an inadequate release of acetylcholine at the neuromuscular junction. And those genetic defects are defects that affect the same biochemical machinery, that is triggered by the calcium, lack of calcium influx in the LEMS condition. And therefore, that is how the diseases are mechanistically similar. And so we have had discussions with the agency, about pointing out the mechanistic similarity between several CMS genetic conditions and LEMS and that is the approach that we intend to take in our initial NDA filing.

And final question, I apologize for all the questions, but regarding the MuSK-MG Phase III trial that you are hoping to commence in the second half yet this year. Wondering if you had any color that you could provide on the design or size or timelines or even the cost of that trial? And then the age range in the sample of this study that you -- the investigator sponsored study that you mentioned versus this age range in the patient population that is diagnosis respected of MuSK-MG?

I'll answer your second question, first again. The MuSK-MG population study -- for our study will be adults. As a reminder MuSK-MG is an autoimmune condition and autoimmune conditions typically develop later in life. So the vast majority of patients with MuSK-MG are adults. Although it is not impossible that there could be children and adolescents who also have the conditions, but that would be relatively rare. With regard to the design of the study, there is really nothing we can say about the design of the study, right now we are in discussions with the agency and we anticipate both announcing agreement on the design as well as initiating a multicenter Phase III trial in the second half of this year.

Our next question is coming from Edward Nash of SunTrust Robinson Humphrey.

This is Yun Zhong for SunTrust -- for Edward and thanks for taking the question. So first question is on CMS. And, I think the study was expanded to enroll in additional adult patients. I wonder when do the efficacy analysis will you be pulling both pediatric patient and adult patient for the point analysis?

I will let Gary go ahead and answer your question, about the modifications to the CMS trial as well as how we'll be analyzing the overall patient population.

Thank you, Steve. Yes, we have expanded the study to the adult population, realizing the fact that the genetic defect, while presenting at childhood or showing up at that point, persists through the entire life. So it's really the same disease carried through. So we would be pooling the results for analysis, but also do a sub-analysis, which would look at adults versus children.

Just to add one additional comment to what Gary just pointed out. The primary endpoint for the study is the motor function measure and that endpoint was selected in part, because it is one of a very small number of muscular function endpoints that accommodates both children and adults.

And the second question is a follow-up question on the MuSK antibody positive MG, sorry to ask that, but do you have any internal expectation, as to what kind of design you would like to have and if the FDA, if they don't agree with that. So what kind of design, might they ask for?

Well, I don't -- I simply can't comment on any specific design preferences or any progress on conversations with the agency regarding the overall design. The MuSK-MG population is only a small subset of the myasthenia gravis population. So one thing I can say, is that, we certainly would be seeking a design that focuses on the MuSK myasthenia gravis population. But again I can't comment on exactly where we will end up with the agency in that regard.

Yun this is Pat. We expect that by the end of this quarter, we should be in a position to talk more definitively about the design of the study and give a lot more color with regard to timeline, size and all of the logistics surrounding that study. So with a little patience, we will have a lot more information out there.

My last question is on LEMS and I think about 2 years ago you hosted a physician symposium, very well attended and I wonder during the past 2 years, have you done any additional market research and have you seen any changes in terms of the perception -- physician perception awareness?

Well, Dave Caponera, our VP of Patient Advocacy, is out there with the physician community as well as our RMS sells, and so I don't know that there is anything new to add at this point or any new findings over the last couple of years. We're in the process of reinitiation of our pre-commercialization activities. And we will get out there and start to refresh the primary and the secondary market research. And we will be doing that, of course, in advance of -- as we prepare to launch the product. So I don't think we have anything new to add or any new findings, at this point, but we'll certainly, as we do, we'll make them available to the public.

[Operator Instructions] Our next question is coming from Scott Henry of Roth Capital Partners.

Just a couple of questions. First, and I don't know if you want to give this much granularity, but I wanted to ask, you've mentioned expectations to file the NDA in the second half of '17. Would you expect an acceptance decision in 2017 as well? Just trying to gauge the timing?

Yes, Scott. I'm not sure that we can be that granular at this point. The FDA has 60 days to review it and accept it for filing. So I'm not sure that we at this point can be more definitive, but certainly as we get closer, we'll be in a position to update the status of that.

Next question. With regards to the CMS data, based on your prior discussions with the FDA, do you expect that they would want to see that data, before making a decision on the LEMS indication? Or are they comfortable strictly with the LEMS data?

Steve, Gary?

Can you repeat the question, Scott? I am not sure I understand it?

The question is, if the FDA would want to wait to see the CMS data, before making a decision on the LEMS?

No. I don't believe so. We're doing 2 well controlled clinical trials for LEMS and so LEMS -- the LEMS indication will be approvable in and of itself. At least that's our expectations. With regard to the CMS, we will be using the LEMS clinical data along with a mechanistic argument that I had previously mentioned, and so as I said in earlier in our prepared statement, that there is no assurance that the FDA will ultimately decide, that the information -- the mechanistic information provided along with the link to the LEMS clinical data, would be sufficient to justify an indication for subset of CMS genetic defects. But we remain optimistic that it will. So in short, the answer to your question is that, we have 2 well controlled trials for LEMS and the NDA itself, of course, is approvable, we anticipate approval for the LEMS indication. It's the CMS indication that is less certain.

Scott, this is Pat. In addition to that, we'll have a pre-NDA meeting with the FDA and we'll get complete clarity on that to assure us that there is no conflict there. So you can bet we'll know exactly, what needs to be filed and what doesn't need to be filed, to have the NDA accepted for review and approved.

And I guess a related question. Is there a time point in the review of that NDA, where it becomes too late to submit that CMS data in the hopes of getting that included in the label? Is there a time where it's just too far along to bring something in that late or how does one think about that?

Let me just point out the logistics of an NDA. First, the NDA can't be amended by the applicant unilaterally. Basically any additional information that goes into the NDA has to be requested by the agency. Now we can prime the pump so to speak, by mentioning that there is clinical data that will be available in x number of months after filing. And the FDA may request that data. So to the degree that the timing of the completion of our CMS trial and the timing of the NDA submission permit, we may or may not mention, that there is CMS data from a clinical trial that is available, which would trigger the FDA to ask. But the bottom line is the agency has to ask for it. There is something called a 120 day update in an NDA , but typically that is usually just safety information, that is submitted to the agency prior to approval.

And then just a final question on the financials. Spending in Q2 particularly R&D. How would you expect that to trend in the remainder of the year?

Scott, I think that it will be fairly consistent between $2.5 million and $3 million a quarter, assuming that we initiate the Phase III study for MuSK-MG, you'll start to see that ramp up a little bit in the fourth quarter likely. And so we believe that, that will be a fairly static number with the -- plus the addition of the MuSK-MG cost, which at this point, we don't know what that's going to be. We have got a ballpark, though nothing definitive until we know exactly what the design and the logistics are going to be for that trial.

Our next question is coming from Charles Duncan of Piper Jaffray.

Just a quick one regarding the NDA and the CMC section. I'm wondering if -- it would seem with the Expanded Access Program you have, you have drug on board, having them manufactured and QC validated et cetera, but could you update that process and if you're set on the CMC. And then also regarding your form of the candidate versus others, that are out there. Have you done any additional analytical work to validate that?

Well with regard to the overall CMC, we definitely are prepared for it, not only have we made numerous batches as you point out for both clinical trial supplies, as well as medication for the Expanded Access Program. But while the original refuse to file, certainly was very disappointing. It has actually enabled us to collect a large amount of stability data at our current U.S. manufacturers, which would have been combined -- which is going to be combined with all the stability information, that was provided by BioMarin when we licensed it. So the bottom line is that, we have numerous exhibit batches for both the API and the product at our intended U.S. facilities, plenty of stability data. And we don't anticipate any significant issues with regard to CMC. Now can you remind me of what was your second question? It was related to the ...

That is helpful, Steve, that actually probably answers it, but in terms of the stability of your form of candidate versus others that may be out there. Wondering if you've done any additional work in terms of stability or other analytical work?

Well actually our product is on long-term stability and it has -- it is stable at room temperature for multiple years. And therefore has very good shelf-life and will be easy to manage in the distribution chain as well as very easy for patients to use. What you are referring to is the fact that the freebase form of this drug has very poor stability, typically a matter of months or less. And should the freebase form ever be commercialized, it would have to distributed in the cold chain as a refrigerated drug, although we don't anticipate that happening. And patients who do use the freebase form of this drug do actually have to keep their drug in the refrigerator. We have not gained access to any of that freebase form of the drug and have not been able to analyze the drug and determine what the degradation products are. But it clearly does lose potency and it just does not evaporate, it turns into something that shouldn't be there in the product.

[indiscernible]

Go ahead, sir.

Go ahead Donna, please.

At this time, we have no questions. Do you have any additional or closing comments?

Yes, thank you. I would like to thank everybody again for the participation on the call today. I'd like to thank all of our stakeholders for their support in helping Catalyst deliver on its promised advancing treatment for patients suffering from rare unmet needs. We look forward to seeing you at upcoming investor conferences and hope to provide more guidance as additional facts become available. We certainly appreciate your patience and promise you that we will work tirelessly to move forward with all of our shared values. Thank you.

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