Catalyst Pharmaceuticals, Inc. (CPRX) Q3 2018 Earnings Report, Transcript and Summary

Greetings and welcome to the Catalyst Pharmaceuticals Third Quarter 2018 Financial Results Call. At this time, all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Ms. Aly 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; Dr. Steven Miller, Chief Operating Officer and Chief Scientific Officer; Dr. Gary Ingenito, Chief Medical Officer and Head of Regulatory Affairs; and Dan Brennan, Chief Commercial Officer. 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 purposes of the Federal Securities Laws. These statements relate to 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 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, Aly, and good morning everybody. Thank you for joining us today. And welcome to our third quarter 2018 results call. On today's call Steve Miller will provide a status report on our clinical development activities for Firdapse as well as our supply chain logistics and inventory build in preparation of a potential launch for Firdapse in early 2019. Dan Brennan, our recently hired Chief Commercial Officer, will provide an update and highlights regarding our commercial launch readiness plans for Firdapse. And lastly, Aly will review our financial results for the third quarter. We will then take your questions. Let me start by saying, I'm pleased to report that the third quarter of 2018 reflects successful execution across all of our key strategic priorities and metrics that we have set for 2018. Let me reiterate our key strategic initiatives near-term and long-term. First is being prepared provided commercially available, FDA approved evidence-based therapy to treat patients with Lambert-Eaton Myasthenic Syndrome or LEMS, and as part of that to do everything within compliance to ensure that all patients will have affordable access to Firdapse and further that objectives. We have developed catalyst Pathways, our branded patient services program. It is intended that pathways will be the best in-class program to assist LEMS patients and physicians with disease education, clinical diagnosis, and assistance in navigating the reimbursement landscape. This will include ensuring that all patients that are currently on Firdapse or three, Firdapse therapy for LEMS will be seamlessly transitioned to commercially available Firdapse. Our second key initiative is to accelerate our clinical studies evaluating Firdapse for the potential treatment of MuSK antibody positive myasthenia gravis, congenital myasthenic syndromes and spinal muscular atrophy type 3. So that in, we have recently hired Dr. Stanley Iyadurai is our Vice President of Clinical Development to accelerate and manage these various clinical programs. Dr. Iyadurai is a licensed neuromuscular specialist with extensive clinical experience in treating congenital myasthenic syndromes, as well as other neuromuscular diseases. Our third key strategic initiative is assuming an FDA approval later this month for us to begin taking required steps to get Firdapse approved in Canada. So the Canadian residents will also have access to an approved therapy to treat LEMS. Lastly, and more long-term, we began an effort to develop a longer acting formulation of Firdapse. As you know, Firdapse or amifampridine phosphate is dosed three to four times a day. Because of the short half-life of the drug and discussions with both physicians and patients we've heard that there would be a great clinical benefit and having a once or twice a day dosing, not to mention that it would likely lead to better medication compliance and adherence. In the second quarter of this year, we announced that the FDA had accepted for priority review our new drug application or NDA for treatment of LEMS. We have been working closely with the agency throughout the review process. And they have set a PDUFA goal date of November 28 2018, which is now just weeks away. We continue to be hopeful that our NDA will be approved and are about the PDUFA goal date. With our goal of developing a world class fully integrated rare neuromuscular disease company. In June of this year, we hired Dan Brennan as our Chief Commercial Officer. Dan is a seasoned and accomplish senior commercial leader in the rare disease field. His most relevant experience was a Lundbeck U.S., where he was VP and Group General Manager of the neurology Division. During his six years of Lundbeck, Dan was responsible for the successful commercial launch of four drugs to treat rare neurological diseases. Dan has rounded out the commercial senior leadership team in the past few months, but the hiring of two of his colleagues from the Lundbeck case. Jason James, who is now our Senior VP of Operations and Analytics, and Jeff Del Carmen, who is our senior VP of Sales and Marketing, we are confident that we're taking all the necessary steps to prepare for a successful commercial launch in early 2019. So with that as an introduction now, now I'll turn the call over the Dan Brennan, who will provide an overview of our commercial launch readiness preparations. Dan.

Thank you, Pat. I'm thrilled to have joined the Catalyst team. It's such an exciting time and I look towards bringing leadership and experience as we prepare for a potential commercial launch of Firdapse in early 2019, our new commercial leadership team has deep product launch experience for drugs that treat rare neurological diseases. And we have already made strong progress with launch readiness plan and developing a strong commercial infrastructure. In fact, progresses being made now as I'm actually dialing in from Chicago as we are completing a three-day interviewing and hiring event that culminates many weeks of efforts to attract the best field based commercial and patient support people to fill out the remainder of our commercial team. We're currently focusing our integrated Firdapse launch readiness plan, which involves both building a commercial infrastructure and actively implementing plans and processes to facilitate a swift and efficient process to ensure that patients and our expanded access program are able to access commercial product in a timely manner without any lapse in medication for them. Our focus is to ensure patient therapy is not disrupted during an expected transition period to an FDA approved product. And we likewise welcome the opportunity to ensure that all three Firdapse experience patients would be able to gain access to treatment without lapses to their available medication during this important transition time. Our commercial infrastructure development includes several functions including patient advocacy teams, marketing teams, market access teams, operations and analytics team, and an experienced and well-prepared Salesforce along with a field-based patient provider liaison group for insurance navigation, co-pay assistance, information support, and patient education. All this includes appropriate coordination with our medical team. In addition to building this home office and field based commercial infrastructure. We are working to ensure a comprehensive patient and provide a support program in play for launch appropriately named Catalyst Pathways, Catalyst Pathways, one food services to coordinate all efforts between patients, caregivers, healthcare providers, payers, and specialty pharmacy. Within that program we're also exploring and developing innovative programs based on our discussions with physicians and LEMS patients about their needs, and concerns that include dosing guidance, diagnostic supports, educational materials, and other programs to help patients get to their therapeutic doses safely and quickly as possible during this hydration process. Payers have also remarked that they appreciate the careful attention to appropriate and effective use of the medicine provided by our programs. Our overarching goal will be to ensure that patients who desire Firdapse have the ability to access it for their treatment and can safely achieve therapeutic effects similar to that demonstrated so clearly in the catalyst clinical studies. Having launched several specialty orphan drugs in my career, all of which were within the neurology arena, I'm confident in our team's ability to develop the necessary foundation ahead of our impending launch that will address challenges to successfully launch Firdapse in the U.S. to patients who desperately desire relief from what I am now seeing as the often-debilitating aspects of LEMS. I look forward to collaborating with the rest of the Catalyst management team as we work to bring Firdapse to even more patients suffering from rare debilitating diseases. I'll now turn the call over to Steve Miller, our Chief Operating Officer and Chief Scientific Officer who will provide more information about our current development programs. Steve?

Thank you, Dan, and good morning, everyone. As Pat mentioned, we are excited as we anticipated FDA decision on our NDA for Firdapse for the treatment of LEMS with a PDUFA goal date of November 28, we have been working closely with the FDA throughout the review process and we remain hopeful that the NDA will be approved on or about fiscal date. Our Firdapse NDA was submitted only for the treatment of LEMS as we decided at the time of submission not to include an indication for the limited types of CMS that are mechanistically similar to LEMS. However, we remain focused on our clinical program for CMS which is currently in Phase III development with enrolment expected to complete in the first half of 2019 and top line results expected in the second half of 2019, assuming positive top line results from this trial, we will work closely with the FDA to evaluate the suitability of Firdapse for all available CMS types and hope to file a supplemental NDA to a potentially approved Firdapse NDA and the supplemental NDA submission we intend to seek approval to market Firdapse for the treatment of one or more types of CMS. We have seen evidence of efficacy of Firdapse in CMS through numerous published reports as well as a small blinded study that was completed some years ago, there are currently no proof therapies or effective off label alternatives for the treatment of CMS and we believe that obtaining approval of Firdapse will be for the benefit of the CMS patients and their families. As Pat mentioned in addition to Firdapse to CMS, we are currently evaluating Firdapse for the treatment of MuSK, antibody positive myasthenia gravis or MuSK MG, our Phase III trial of Firdapse for MuSK MG is underway following our successful proof of concept trial showing clinical improvement in multiple assessments. This trial is being conducted under an FDA special protocol assessment and we anticipate reporting top line results from this trial in the second half of 2019. Pending successful top line results from this trial next year, we hope to work with the FDA to include MuSK MG on the label of Firdapse by way of a future supplement to an already approved NDA. Patients at our Catalyst clinical trial for the treatment of MuSK MG with Firdapse are eligible to continue to receive Firdapse at no cost in a long-term safety follow-up trial if the patient and their physician feel that the patient benefited from the treatment of Firdapse. Lastly, we are currently evaluating Firdapse for the treatment of spinal muscular atrophy or SMA Type 3, we have initiated a proof of concept trials with a target of 12 patients who have a genetically and clinically confirmed diagnosis of SMA Type 3. Based on input from several neuromuscular medical experts, we believe that Firdapse has the potential to improve neuromuscular function in these patients, increase motor neuron use of activity and perhaps slow the progression of nerve degeneration seen in SMA patients. We also continue to support our expanded access program which currently provides patients with LEMS and CMS access to amifampridine phosphate tablets. For these eligible patients, therapy is provided free of charge if the patients have no other treatment option and the physicians believe that this treatment could provide relief to their patients. Catalyst has also begun the production of Firdapse 10 milligram tablets in its commercial presentation. We anticipate having adequate inventory of Firdapse to supply all known expanded access patients and other patients who can benefit from this treatment upon launch. Firdapse will be provided in 10 tablet chart resistant poster package and in bottled pharmacy shops. I will now turn the call over to Aly Grande, our Chief Financial Officer to review our financial results.

Thanks, Steve. All reference to per share in this call refers to basic and diluted shares. For the quarter ended September 30, 2018 Catalyst reported a GAAP net loss of $7.8 million or $0.08 per share compared to a GAAP net loss of $4.2 million or $0.05 per share for the same period in 2017. For the third quarter of 2018 and 2017, non-GAAP net loss was the same as GAAP net loss as there were no non-GAAP adjustments. Research and development expenses were $4.5 million for the third quarter of 2018 compared to $2.7 million for the same period in 2017. The increase in R&D was compared to the same period in 2017 was primarily due to increased income closing expense, milestone expense in connection with the acceptance of our NDA submission in May 2018, expenses from our medical affairs showroom and compensation on related personnel costs as we expand our headcount to support our currently ongoing clinical trials and programs. We expect that research and development costs will continue to be substantial during the balance of 2018 and we were source completing trials, collaborating Firdapse for the treatment of CMS, MuSK MG and SMA type 3, continue expanded access program for Firdapse and our other development programs and prosecute our accepted NDA submission for Firdapse. General and administrative expenses for the third quarter of 2018 totaled $3.6 million compared to $1.6 million in the third quarter of 2017, the increase when compared to the same period in 2017 is primarily due to increases in pre-commercialization expense, headcounts and corporate expenses as we've build up our infrastructure in commercial programs in preparation for the potential for Firdapse launch in 2019. We expect G&A cost including pre-commercialization expenses will continue to increase in 2018 compared to the G&A cost incurred in 2017 as we continue to expand our workforce and operations in preparation for a potential launch of Firdapse in 2019. Catalyst had no revenues in the third quarter 2018 or 2017, at September 30, 2018 Catalyst had cash and investments of $66.7 million and no debt. Although there is going to be no assurance based on current available information, we believe that these resources will be sufficient to support our plan operations through 2019 without considering revenues and cash receipts that we might receive in 2018 if we are successful in obtaining an approval for Firdapse on launch of the product in 2019. More detailed information and analysis maybe found in the company's quarterly report and Form 10-K which was filed with the Securities and Exchange Commission yesterday November 7, 2018 and can be found on the Investor Relations page of our website at www.catalystpharma.com I will now turn the floor back to Pat.

Thanks, Aly. We've a very exciting upcoming couple of months and we look forward to hearing from the FDA regarding the review of our NDA, we're confident that we are taking all the steps necessary for a successful launch pending FDA approval and most importantly are excited for the potential to bring a commercially available FDA approved evidence based therapy to LEMS community. We have a great commercial leadership team and we are grateful and confident and your experience regarding commercialization for therapies for rare diseases such as LEMS. In addition to the upcoming PDUFA goal date, we are very pleased with all of the progress that we've made on our clinical development programs namely in CMS and MuSK MG. We look forward to providing you updates on each of these programs as well as any business development activities that may occur in the near future. This will end the formal presentation and will turn the call over to the operator for questions.

Thank you. We will now be conducting a question-and-answer session. [Operator Instructions] Our first question comes from the line of Joe Catanzaro with Piper Jaffray. Please proceed with your question.

Hey guys, thanks for taking my questions, just a couple ones for me, you noted your ongoing interactions with the FDA, and I appreciate that you probably can't provide so many specifics. So I was wondering if you maybe could just provide some high level comments about how those interactions over the last few months have gone. And then can you just remind us how many LEMS patients you guys currently have in the expanded access program, and how quickly they could convert to commercial product, this is a matter of weeks, a few months, just thoughts around that? Thanks.

So Joe, yes, we don't talk publicly about the number of patients we have under extended access program. We just did not have to be in a position to have to update that on a quarterly basis. And so, we're not going to start now, we have a fair amount of LEMS patients that are in our expanded access program, and we expect that we will have a bridge to get those patients on the commercially available drug as soon as possible. And as you can imagine it'll probably take 30 to 90 days to get reimbursement approved by payers. So and then, maybe Gary, Our Chief Medical Officer, can give you a little bit of color about our interactions with the agency over the last few months, during the review process.

Yes. So we have received several requests for information, all were answered promptly without any delays. There are no outstanding requests right now and we will be continuing to work through the final product labeling over the next week.

Okay, got it. That's very helpful. May be just one last quick follow up, so it seems like CMS data have been pushed out from first half 2019 to the second half of the year to just provide the some insight around that.

Sure. Gary will advise you on that as well.

So CMS is a rare disease even more rare than LEMs and in order to accumulate the necessary pediatric safety data that means we are already sub-setting a small population to focus on children, as you know pediatric trials are historically difficult to conduct and in addition in CMS today there are now over 50 mutations that cause various levels of symptoms, so we look through those in addition to find ones that will meet the inclusion, exclusion criteria of the protocol.

Okay. As a result, as you can imagine it's taking longer than we expected, we identified patients through physicians. Primarily at children's hospitals and getting parents to agree to submit your kids to a clinical trial is also a challenge and so we're getting there but it's just taking longer than we think we have the patients identified, we just need to go through the logistics to get them enrolled and through the study.

Okay, very helpful. Thanks for taking my questions.

Thank you.

Thank you. Our next question comes from the line of Leland Gershell with Oppenheimer & Company. Please proceed with your question.

Hey, good morning thanks for taking my question.

Good morning.

Hi, I just wanted to ask a bit further on the mention of the possibility that the CMS eventual CMS legal extension could be for all patients at CMS versus perhaps on this specific mutations given that in the past we've only seen, certainly patients be eligible for that, just want to further about that if you've seen any observations that would you believe that there may be potential for us to have use in the overall see if U.S. population and other mutation that has not been previously include?

So we are testing out most of the genetic defects, there are few which are known to actually get worse with a product like for Firdapse. Those are excluded, however the study is ongoing and still blinded, so at this point I can't say which ones may be responsible and which ones will not be until we unblind the data.

Okay, thanks and then question on the commercial side just with regard to the, that the feel of course build if you can remind us what the goal is in terms of the size of that infrastructure will be and where you are it sounds like directly building that as we speak but where you are now in the process of building that out.

Sure. Sure Dan Brennan will answer that call for you, Leland.

Where we're at right now that is just the beautiful Chicago O'Hare helped in the hotel interviewing up to about 25 or 30 people for what looks to be a field force in total including of our director total sales reps. And that would cover the U.S. and in addition to that, we also plan on having five patient access or patients access managers or liaison that help once a prescription is made help those patients on board, go to titration and handle the insurance navigation and co-pay that goes along with the prescription.

Okay, great. Thanks for taking the questions.

Thank you. [Operator Instructions] Our next question comes from the line of Scott Henry with ROTH Capital. Please proceed with your question.

Thank you and good morning. Just a couple questions, thank you, Pat. First with regards to Firdapse in Canada could you just give a little color on kind of, how we should think about the time line there and the potential size of that market?

So once we have the U.S. approval, we will set up a meeting with Health Canada to determine again their requirements and format and I think that will really set the timeline and so will have a much better determination after we have meeting Health Canada.

Scott, we'll likely look to find a partner and we've had some discussions with a couple of Canadian pharmaceutical companies that market drugs in the neurology space that might be appropriate partners for us, so that's probably the route that we would travel any idea in the timeline here is. Not at this point. We will advise shortly and we of course estimated the market size and the market opportunity sort of on a pro-rata basis and it would equate to probably 10% of the U.S. market opportunity.

Okay, thank you for the color on that and then second you also mentioned the pursuit of a longer acting formulation, how should we think about it at least in general terms the timelines on that obviously probably wouldn't start until after approval at least in any significant way but how should we think about that?

Well, our plans are to start as you point out shortly after approval we have to not only develop the formulation but also do some clinical work in order to be eligible for a three year new product exclusivities and finally we will and we intend to pursue appropriate intellectual property for it as well and there is a patent prosecution time and has to be factored into all of this. As far as overall timing is concerned, we would ideally like to time the introduction and transition of patients to the sustained release product at approximately the time frame when the exclusivity is for the IR would be running out.

Okay, great. Thank you for that. Final question, I was just reading through the 10-Q. And I noticed on the SMA a proof of concept that you classified as a Phase 3 proof of concept. I was just curious why Phase 3 versus Phase 2 is the thought that perhaps you would only have to do one more trial after this proof of concept or just curious your thoughts on that?

Sure, yes you're correct in that we would try to pursue a strategy where we would only need to do one more trial after the proof of concept it should turn out to be positive.

Okay, great. Thank you for taking the questions.

Thank you, Scott.

Thank you. Our next question comes from the line of Charles Duncan with Cantor Fitzgerald. Please proceed with your question.

Hi guys, thanks for taking the questions. I had a couple the first one is perhaps for Dan Brenner. Regarding you've talked a fair amount about being ready in terms here go-to-market strategy and with the commercial infrastructure and I guess I'm wondering as you will be gauging effectiveness of your new sales force over the course of 2019, what are some of the metrics that you'll like to very likely keep track of to gauge whether or not these people are making getting traction in the market.

Sure, so thanks for the question certainly that the new patient starts, as it relates to the transition of people over from the AP. as well as other patients that we know are in the market through our claims data review that are diagnosed with LEMs and we target into those physician offices to have discussions with them. At the beginning that is our main target and probably through 2019 our primary target metrics and goals, certainly along with that is once people are prescribed that there's a high level of satisfaction from both the patients as well as the physicians in our entire process of getting patients on the drug and reimburse their insurance and such, so a high level of satisfaction with our Catalyst Pathways Program is also on the radar screen. Over time and probably at the end of next year and beyond we'll focus more, especially on the sales force side on the helping physicians diagnose more quickly and easily of these patients that are undiagnosed that are also floating around out there wondering what these symptoms mean and trying to get to the right type of neuromuscular specialist or neurologist that can do the right types of tests. So, all of those are the main metrics and goals that see for the sales force and our marketing team over the next six, 12, 18 months.

That's helpful. And hopefully you'll be willing to talk about some of those metrics over the course of the first few quarters. I guess I wanted to also ask you about Catalyst Pathways. As you valuate that and come it to some of your past experiences, are there any elements that you cite as being particularly interesting or how would Catalyst Pathways compare to other patient access programs that you've worked on?

Very similar to many of the programs that we had in place at Lundbeck for a number of the different products that I worked on there in Huntington's disease, and infantile spasms, and Lennox-Gastaut, so many of the programs are similar, however on this end with it being such a high-touch small ultra-rare group, we have built up the infrastructure at our patient services center with people that have done this before, both from a hub perspective, but also people that had worked with the liaisons that are in the field. I think that that's -- as we're talking with the physicians right now that have patients that are on Firdapse [ph] whether they're in the EAP or not, they're very impressed with the level of comprehensive support that we are providing. And it includes education on dosing and titration. I mean they are -- many of the programs that you would expect to see from these programs, we have staffed it up especially at the beginning to handle what could be an influx in bolus of patients at the very beginning, and then to carry them through throughout.

Okay, that's helpful. And then one perhaps for Pat or whoever, I wondered if you could provide us some additional color on really your view of the pharmacoeconomic value of Firdapse and logic that may provide input for pricing. I'm not asking you about pricing because I doubt you'll answer that, but some of the features or the way that you think about that at this time.

Yes, Charles, good question. We've spent a lot of time reviewing and studying the value proposition that we provide with Firdapse. And we think that, obviously, one of the major keys is an FDA-approved therapy. And what's involved for a company to get a therapy FDA approved. And the key there is with an FDA-approved therapy all patients will have affordable access to Firdapse, which is not the case today. There, we estimate and based on anecdotal information as well as what some of the other participants are saying in the marketplace that there are anywhere from 250 to 300 patients that are currently on, what I'll call, early access to Firdapse or 3,4-DAPP therapy. And so the issue is what are the other 90% doing. And the other 90% don't have access because their doctor doesn't want to participate in an investigator IND program because of all of the required paperwork. Patients don't want to have to travel to referral sites 300, 400, 500 miles to see a doctor to get drugs. So I think that the main factor or the main feature is that one, we have an FDA approved therapy, where docs can simply write a script and patients will have drug delivered to their house. Two, our Catalyst Pathways program we hope will ensure that all patients have affordable access to the drug, minimal copays, minimal out-of-pocket expenses. And then of course the clinical benefits of changing their lives, life-changing events with the drug, changing their quality of life and their activities of daily living. And so I think those are sort of the cornerstones for which we use to build our value proposition regardless of where we price the product.

Okay, that's helpful, Pat. I appreciate the different inputs into that thinking. And we'll look forward to updates in the future. Last question, perhaps for Steve, the long-acting version that you mentioned earlier, I'm wondering if beyond the length of the franchise, if you will, the lifecycle of the franchise, do you believe that that could have a different clinical profile in terms of efficacy? Surely it may do so in terms of compliance. But do you think there are any opportunities for even further improving the efficacy or safety of Firdapse with the long-acting?

You know, Charles, that's actually a very good question. And the short answer to your question is, yes, there definitely will be some improvements in terms of the medical utility of Firdapse as an SR product. One of the most common concerns that patients have is that the drug wears off while they're sleeping and they have tremendous difficulty getting up out of bed in the morning. A sustained release product would maintain blood levels to therapeutic levels throughout the night so that they would be able to get up in the morning and immediately start their day. And we think it would have a tremendous benefit for them. With regard to potential changes in efficacy and safety, I'm just going to give you a nebulous answer of, probably. I don't want to go into any significant detail for intellectual property reasons, because any benefits that we do see might be involved or perhaps be the basis of future intellectual property.

Yes, it makes sense to me. Thanks for taking my questions. We look forward to some updates in the near-term.

Thank you, Charles.

Thank you. There are no further questions. At this time, I would like to turn the call back over to Mr. McEnany for any closing remarks.

Thank you, Michelle. Thank you all for your participation on this morning's call. And we want to thank all of our various stakeholders for their continued support, especially our employees that have worked tirelessly to help bring Firdapse to patients in need. We look forward to achieving and reporting on several key milestones in the upcoming weeks and months. And we're excited about the potential launch of Firdapse in the beginning of 2019. We will keep you advised as matters progress. Thank you.

Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation. Have a wonderful day.