Catalyst Pharmaceuticals Q3 2021 Earnings Call Transcript

Quartr
Provided by Quartr
November 10, 2021

Key Takeaways

  • Positive Sentiment: Record Q3 net Firdapse revenues: Generated $35.9 million in the third quarter, a 23% year-over-year increase, contributed to a 20% rise in pre-tax income, and ended the period with $174.8 million in cash and short-term investments.
  • Positive Sentiment: Orphan drug exclusivity affirmed: The Eleventh Circuit Court of Appeals upheld Firdapse’s orphan drug exclusivity for the 10 mg tablets, bolstering U.S. market protection and reducing generic competition risks.
  • Positive Sentiment: Pediatric label expansion planned: Catalyst will file a supplemental NDA for pediatric LEMS in Q1 2022, aiming to provide an approved treatment option for the estimated 10–15 children with the condition.
  • Positive Sentiment: Japan commercialization effort: Partner Diido Pharma is preparing to launch a small Phase III trial for Firdapse in 2022, marking a key step in the product’s global expansion beyond North America.
  • Negative Sentiment: MuSK MG program discontinued: After thorough review of trial endpoints, regulatory feedback and market potential, Catalyst elected to end its MuSK myasthenia gravis development to reallocate R&D resources.
AI Generated. May Contain Errors.
Earnings Conference Call
Catalyst Pharmaceuticals Q3 2021
00:00 / 00:00

There are 8 speakers on the call.

Operator

and welcome to the Catalyst Pharmaceuticals Third Quarter twenty twenty one Earnings Call and Webcast. At this time, 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 turn the call over to Ali Grande, CFO.

Operator

Please go ahead.

Speaker 1

Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's third quarter twenty twenty one financial results and corporate highlights. Leading the call today, we have Patrick McEnany, Chairman and Chief Executive Officer We are also joined by Doctor. Steven Miller, Chief Operating Officer and Chief Scientific Officer and Jeffrey Del Carmen, Chief Commercial Officer. For the Q and A session, we'll also have Doctor. Gary Intonido, Chief Medical and Regulatory Officer.

Speaker 1

Before we begin, I would like to remind you that in the following comments and in the Q and A session, we will make statements about expected future results, which may be forward looking statements for purposes of 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 may prove not to be accurate, especially in light of the effects of COVID-nineteen. 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 described in our 2020 annual report on Form 10 ks.

Speaker 1

At this time, I'd like to turn the call over to Simon.

Speaker 2

Thanks, Allie. Good morning, everyone, and thank you for joining us today for Catalyst's third quarter results and update call. I'm very pleased to report that we delivered record Firdapse revenue results for the third quarter ending September 30. We generated net Firdapse revenues of $35,900,000 an increase of 23% versus the third quarter of twenty twenty. Firdapse revenues for the nine month period were $99,700,000 as compared to $87,900,000 for the nine months last year, which represents a 13% increase year over year.

Speaker 2

At this time, I anticipate continued organic growth through the end of this year and for the foreseeable future. Net income before income taxes for Q3 twenty twenty one was $14,100,000 a 20% increase when compared to $11,700,000 for the third quarter of last year. We reported GAAP net income of $10,300,000 for the third quarter of this year or $0.10 per basic and diluted share. We ended the quarter with $174,800,000 in cash and short term investments, further supporting our long term growth strategy to diversify through reinvestments in our business or external growth opportunities. These results also highlight the strength of our commercial capabilities and resilience as we continue to execute throughout the current environment.

Speaker 2

Allie

Speaker 3

will

Speaker 2

have more to provide during her financial presentation. Our share purchase program continues with 949,746 shares purchased in the open market during the third quarter. And since inception in March of this year, through the end of the third quarter, we have purchased 1,000,007 and 49,746 shares at an average price of $5.15 per share. Notwithstanding our success in treating many LEMS patients, as of now, we have only reached about twenty five percent of the estimated three thousand U. S.

Speaker 2

LEMS patients. We remain confident of the progress of our commercial team is making in evolving the market for the Firdapse brand. We continue to make substantial investments in Firdapse, focusing on expanding reach to targeted healthcare specialists, most recently to oncologists and general neurologists, providing resources to help shorten the diagnostic journey for LEMS patients and support through patient and healthcare provider education programs. Our focus on commercial execution excellence remains a top priority to us as well as to the patients we serve. Jeff will provide additional information on our commercial progress shortly.

Speaker 2

Importantly, we've made substantial investments related to Firdapse exclusivity and commercial potential in The U. S. Most notable was the receipt of a positive decision from the eleventh Circuit Court of Appeals supporting the orphan drug exclusivity for Firdapse tablets ten milligram for treating LEMS patients in The U. S. We are currently extremely pleased with the court's decision.

Speaker 2

While there is a forty five day period for which an en bloc hearing request can be submitted to the court related to the court's decision, we remain confident in our position and look forward to when we can fully recognize our exclusivity. Our priority has always been and will continue to be on addressing patients' needs first. We are well prepared to do everything we can to ensure that all LEMS patients will have uninterrupted access to amifampridine for treating their condition. We also continue to make important progress in bolstering our intellectual property for Firdapse. Currently, two patents have been issued that extend the life of our intellectual property portfolio to 2,034, both of which are listed in the Arch book.

Speaker 2

We have additional patent spending to further strengthen the IP portfolio, adding to our commitment to protect the innovation and the product's commercial potential. As we continue to execute our commercial related initiatives, we are taking a very targeted approach towards our clinical development programs. We are working on expanding the label for Firdapse as we plan to see U. S. Approval to include pediatric LEMS patients, which we believe numbers our 10 to 15 children.

Speaker 2

This program further underscores our continued commitment to providing an important treatment option for LEMS patients of all ages. We are also continuing our efforts to expand the global footprint for Firdapse beyond The U. S, Canada and Japan. Steve will have more to add shortly regarding activities outside The U. S.

Speaker 2

In addition, having objectively and thoroughly evaluated the clinical development time line, regulatory path and commercial viability for MuSK MG. We have elected to end the program and concentrate our R and D efforts in other areas. We believe having a more focused approach towards our development programs will will drive the greatest value for the company and enable us to better utilize our resources to pursue value creating opportunities to expand our focus beyond Firdapse. During the quarter, we made considerable progress in our efforts to build a more diversified portfolio that aligns our growth strategy and priorities, including the potential to expand the breadth and depth of our pipeline. With a robust process in place during the third quarter of twenty twenty one, we engage in extensive due diligence activities on a potential acquisition that fit our strategic objectives.

Speaker 2

However, after a thorough and extensive assessment of the acquisition candidate, we elected not to further pursue this opportunity. Coupled with our strong balance sheet, we are in an excellent position to take advantage of opportunities that meet our specified criteria and commitment to pursue rare disease opportunities and are very encouraged about projects that are currently under review. Our quarterly achievements demonstrate continued performance excellence, supported by several key strategic decisions that have positioned Catalyst firmly for the future. In saying that, we are excited about the path ahead as we continue to execute it across all priorities to drive sustainable growth for the long term. Finally, there are a couple of other points worth noting.

Speaker 2

First, we achieved these results during a period when Catalyst and most of our peers were affected to some degree by the Delta variant of COVID-nineteen. As this variant is abating, we are beginning to see a more normalized pattern of practices with healthcare providers and patients. Lastly, while addressing the COVID-nineteen effects, I want to reiterate that as we have previously stated, our supply chain is rock solid with qualified redundancies throughout our supply chain. Now I will turn the call over to Jeff Del Carmen, our Chief Commercial Officer, who will provide further highlights execution for the quarter.

Speaker 4

Thanks, Pat, and good morning, everyone. As Pat mentioned, Q3 net Firdapse sales were $35,900,000 which represents 23.1% growth quarter versus the same quarter last year, 6.7% growth versus Q2 twenty twenty one. We are extremely proud of the continued strong performance by the entire Catalyst organization. Strong net revenue in Q3 was driven primarily by steady new patient enrollments, continued favorable reimbursement dynamics and stable discontinuation rates. While we continue to see some sporadic COVID-nineteen related slowdowns in new patient starts due to delays at the point of care between diagnostic testing and initiation of treatment, Q3 new patient enrollments were still 26% higher this quarter versus same quarter last year and slightly higher than Q2 twenty twenty one.

Speaker 4

Our strong patient persistency resulted in continued low ninety day discontinuation rates of less than fifteen percent. In October, net new patients were the highest monthly total in 2021, a result of consistent naive deferred apps new enrollments, adult LEMS patients restarting Firdapse and low discontinuation rates. We are proud that around eight hundred adult LEMS patients have received the Firdapse prescription since launch. However, Catalyst is focused on the significant remaining opportunity to help all adult LEMS patients. We are confident that our continued heavy investment in patient resources will shorten the diagnostic journey for LEMS patients and enable Catalyst to serve the significant number of remaining LEMS patients sooner.

Speaker 4

Thus far, approximately 3,000 unique undiagnosed potential patients have opted in to receive useful information regarding LEMS, an increase of nearly 1,000 since our last earnings call. Furthermore, we continue to meet key milestones in the development and execution of our oncology LEMS strategy. Approximately fifty percent of LEMS patients are thought to be tumor LEMS patients with the vast majority having small cell lung cancer. Therefore, we believe that oncology is a key element of our future growth. After completing a thorough assessment of the market, conducting ad boards with oncology thought leaders and ongoing market research, we are excited to launch our non personal promotion to thoracic oncologists in Q4.

Speaker 4

Engagement with this segment of the market has resulted in early positive indicators that the strategy put into motion will yield more patients with a proper diagnosis of LEMS and accelerate the opportunity to receive treatment for this disease. We continue to partner with key professional societies like AA and EM, which provide continuing education for all levels of neuromuscular healthcare providers. We had the opportunity at the recent annual meeting to engage with their constituency and educate clinicians about LEMS in new and innovative ways, helping to identify atypical LEMS adult patients. Through healthcare provider education, we expect more patients will benefit from a correct and timely LEMS diagnosis. Our LEMS I'm sorry, our Catalyst Pathways patient services team remains dedicated to supporting the needs of adult LEMS patients, caregivers and healthcare professionals as well as providing educational resources regarding LEMS and Firdapse.

Speaker 4

In addition, Catalyst Pathways has numerous types of financial assistance programs to help patients with their out of pocket costs. Patients enrolled in Catalyst Pathways, including those who are covered by Medicare and accessing foundation assistance, have an average co pay of less than $2 a month. Prescription approval rates remain over 90% across all payers, government or private commercial insurers. Catalyst Pathways patient services team also connects patients to community and advocacy resources such as patient support groups. In closing, we are pleased with our performance in Q3 and are excited about the significant opportunity ahead to help all adult LEMS patients.

Speaker 4

We are confident that the strategies and tactics we have put in place will deliver sustained growth in Q4 and beyond. I want to thank the entire team at Catalyst for their unwavering commitment to the LEMS community. I will now turn the call over to Doctor. Stephen Miller, our Chief Operating Officer and Chief Scientific Officer, for an update on R and D activities.

Speaker 3

Thanks for the commercial update, John. I'll now provide an update on our clinical pipeline and product development efforts. We continue to make important progress with our development programs for Firdapse, including advancing our plans for developing a long acting formulation of amifampridine phosphate in order to provide an improved dosing experience that is a more convenient option for patients and provides enhanced consistency in its therapeutic effect. Plans are underway to initiate an additional pharmacokinetic study in the first quarter of twenty twenty two to study the drug release and absorbance characteristics of the most recent long acting candidate formulations. Our design efforts have been guided by input from patient and physician panels regarding the desired performance characteristics of a long acting version of Firdapse.

Speaker 3

We believe the target characteristics of our long acting formulation of amifampridine phosphate will address the needs of the LEMS patient community as we continue to execute on our plans to advance this development program. In alignment with our commitment to provide a treatment for all LEMS patients, we are now preparing a supplementary NDA submission package for the treatment of pediatric LEMS with Firdapse. We anticipate filing this supplement with the FDA in the first quarter of twenty twenty two. Catalyst has all the required data, including all necessary safety data to file a complete submission for this label expansion. The preparation of this supplement is a high priority for Catalyst as we remain committed to advancing our efforts to expand the use of Firdapse so that we may be able to provide all LEMS patients with an approved treatment option.

Speaker 3

Now I would like to provide an update on the MuSK MG program. As previously announced, Catalyst submitted plans, including a protocol for a new clinical trial for the symptomatic treatment of MuSK at myasthenia gravis or MuSK MG to the agency for their review and comment. Having received the agency's comments, we convened an advisory board meeting during the third quarter consisting of key opinion leaders and experts for input and options for the MuSK MG development program. This included some physicians that had experience using Firdapse to treat MuSK MG in our most recent trial. After an in-depth evaluation of the feasibility and measurability of study endpoints for this indication as well as the input from the agency and advisers that was received, we have concluded that the universal use of Firdapse as a first line therapy for MuSK MG is unlikely, and therefore, we have decided to put off further development plans at this time.

Speaker 3

However, for those patients that currently receive Firdapse to treat their MuSK MG symptoms, will continue to support them through investigator initiated programs in accordance with agency regulations. We continue to make valuable progress in increasing health care provider awareness of LEMS through our medical affairs programs. An accredited continuing medical education or CME course about LEMS is now available through Medscape. To date, over 4,600, mostly healthcare provider learners have viewed the course. And as of this October, thirteen thirty eight licensed health care providers have taken the CME test.

Speaker 3

Our medical science liaisons also recently completed hosting several neuromuscular medicine fellows from various academic medical centers for another Catalyst sponsored hands on training session on LEMS given by neuromuscular experts earlier this month. Previous sessions like this one have received strong positive feedback on their ability to raise awareness among fellows of the diagnostic challenges with LEMS. Now I would like to provide an update on our ongoing global expansion initiative. As we previously announced in June, we partnered with Diode Pharma for the development and commercialization of Firdapse for the treatment of lamboride and myasthenic syndrome in Japan. Daito has made great strides initiating development activities, including consulting with the Japanese Pharmaceuticals and Medical Devices Agency to confirm clinical study parameters and requirements to obtain regulatory approval.

Speaker 3

As a result of their efforts, Dido is preparing their clinical trial notification and anticipate the small scale Phase III clinical trial will be initiated in 2022. Catalyst continues to support Dido in preparing for the clinical activities by supplying clinical trial materials as well as collaborating with Diodes and its efforts to obtain required documentation for the Japanese regulatory authorities. We have made significant progress on developing an intellectual property estate to extend the market exclusivity for Firdapse. As of the third quarter twenty twenty one, we have two patents listed for Firdapse in the FDA's Orange Book, which extends the IP portfolio to 2,034. We have several other pending patents related to the use of Firdapse filed with the U.

Speaker 3

S. Patent and Trademark Office and anticipate that they may issue later this year or in early twenty twenty two. In addition to The U. S, Firdapse currently has data exclusivity in Canada out to 2028. And in Japan, we expect Firdapse will be granted ten years of market exclusivity upon approval in that market.

Speaker 3

We will continue to seek opportunities to enhance the portfolio of state to further protect the market exclusivity geographically. During this quarter, we advanced our objectives to expand our portfolio of rare disease treatments beyond Firdapse. As Pat mentioned, we continue to actively evaluate new products or other kinds of transactions to expand both Catalyst product offerings and or our research and development pipeline. Our teams are actively and vigorously engaging in the process as we look for worthwhile prospects where we can utilize our expertise, resources and know how to expand our programs and drive growth. With a very efficient approach in place, we are enthusiastic about the path ahead and confident in our ability to identify the right opportunities to maximize our capabilities and resources.

Speaker 3

I will now turn the call over to Ali Grande, our Chief Financial Officer, to review our financial results.

Speaker 1

Thanks, Steve. We are very pleased with our financial results for the third quarter. As reported, we ended the quarter with cash and investments of $174,800,000 and no funded debt. We believe this allows us the financial flexibility to advance our R and D programs and support our strategic initiatives of acquiring earlier stage opportunities and innovative technologies to enable growth and value creation. Total net revenues for the third quarter of twenty twenty one were $36,000,000 a $22,600,000 increase when compared to total revenues of $29,300,000 for Q3 twenty twenty.

Speaker 1

Despite the competing challenges of COVID-nineteen, total Firdapse product revenue net was $35,900,000 for the third quarter of twenty twenty one, a 23% increase over net product revenue of $29,200,000 for the third quarter in twenty twenty. Net income before income taxes for Q3 twenty twenty one was $14,000,000 an approximately 20% increase when compared to $11,700,000 for Q3 twenty twenty. We reported GAAP net income of $10,300,000 for Q3 twenty twenty one or $0.10 per basic and diluted share. For Q3 twenty twenty, we reported GAAP net income of $43,300,000 or $0.42 per basic and $0.41 per diluted share. However, these figures are not comparative as the third quarter of twenty twenty benefited from the reporting of a onetime noncash deferred tax asset of approximately $31,600,000 upon reversal of the related valuation allowance.

Speaker 1

Our effective tax rate in the third quarter of twenty twenty one on an annualized basis was 24.3 as compared to 2.8% in the third quarter of twenty twenty. While in 2020, we benefited from the use of our federal net operating losses, in 2021 and future periods, we expect that we will benefit from the use of our deferred tax assets, primarily relating to state NOLs and the orphan drug tax credit, although those are subject to certain limitations, resulting in a more normalized tax rate. Because of the significant effect of the onetime reporting of our deferred tax asset in the third quarter of twenty twenty compared to the third quarter of twenty twenty one, we believe that the non GAAP measures we presented in yesterday's press release provide a more useful comparison of our results of operations for the third quarter of twenty twenty one versus the third quarter of twenty twenty. Non GAAP net income for Q3 twenty twenty one was $15,600,000 or $0.15 per basic and $0.14 per diluted share, which excludes from GAAP net income, sub based compensation expense of $1,500,000 depreciation of $31,000 and the income tax provision of $3,700,000 This compares to non GAAP net income for Q3 twenty twenty of $13,200,000 or $0.13 per basic and $0.12 per diluted share, which excludes from GAAP net income, stock based compensation of 1,500,000.0 depreciation of CAD30000 and an income tax benefit of CAD31.6 million.

Speaker 1

The above represents an almost 18% increase of non GAAP income year over year for the third quarter. Research and development expenses were $4,500,000 for the third quarter of twenty twenty one compared to $3,700,000 for the third quarter of twenty twenty. R and D expenses remained consistent at 2021% of total operating costs for the third quarter of twenty twenty one and the third quarter of twenty twenty two twenty twenty, I'm sorry, respectively. We expect that research and development expenses will continue to be substantial in 2021 and beyond as we advance the development of our long acting formulation program for Firdapse and continue our expanded access programs. In addition, we expect R and D will also increase in future periods to be successfully executing on our strategic initiatives to acquire or in license innovative technology platforms and or earlier stage programs in other therapeutic areas outside of neuromuscular disease.

Speaker 1

SG and A expenses for the third quarter of twenty twenty one totaled NT12.2 million dollars compared to NT10 million dollars in the third quarter of twenty twenty. SG and A expenses decreased slightly as a percentage of total operating expenses to 55% for the third quarter in twenty twenty one compared to 57% for the third quarter of twenty twenty. We expect that SG and A expenses will continue to be substantial in 2021 as we continue our efforts to increase revenues from Firdapse and take steps to further expand our business. 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, November 9, and can be found on the Investor Relations page of our website at www.catalystpharma.com. And with that, I'll turn the call over to Pat.

Speaker 2

Thanks, Ali. Catalyst continued to demonstrate resilience and strong execution in the third quarter of the year, providing a very strong foundation for the company as we make progress in our business development activities and look to build a portfolio of rare disease marketed products and or other clinical programs. Finally, I'd like to thank all of our employees who have each demonstrated a dedication to making a positive impact on patients' lives. This would not happen without their passion and commitment to serving the LEMS community. This ends our prepared remarks.

Speaker 2

We will now open the call for questions.

Operator

Thank you. We'll now be conducting a question and answer session. Our first question today is coming from Charles Duncan from Cantor Fitzgerald. Your line is now live.

Speaker 5

Yes. Hey, good morning, Pat and team. Congratulations on a nice quarter of performance. Thanks for taking our questions. I had one on commercial, one on pipeline and one on strategy.

Speaker 5

Perhaps I'll start with the commercial regarding Firdapse growth drivers in 2022, not really asking for guidance here, but it might be nice to know whether or not you'd be comfortable providing guidance in 2022 later in a few weeks, maybe with start of 2022. But more importantly, when you think about the growth drivers for 2022, what is the key factor that you're focused on? Is it increasing awareness? Or is it increasing breadth or depth of prescribers?

Speaker 2

Yes. So your first question, Charles, is as far as giving guidance, as we go through the process of developing our budget for next year and our forecast, and hopefully, looking at this pandemic from the rearview mirror, I think perhaps early in Q1, we might feel comfortable in giving guidance. Think so that's our hope and desire is that we can give some revenue guidance at the beginning of the year, the New Year. In regard to the key growth factors, I'll

Speaker 4

let Jeff address that. Thanks, Charles, for the question. We see a significant opportunity in 2022 to grow. Specifically, when we look at the diagnosed patients that are out there already for LEMS that are not yet on Firdapse, we have a robust pipeline of these leads. And many of these physicians are telling us at this point that they're just waiting for these patients to come in and visit before they prescribe.

Speaker 4

So we have that. That's one. Other things, we take a look at the oncology and the tumor LEMS patients. We're applying significant resources there to educate HCPs

Speaker 2

as

Speaker 4

well as patients on tumor lens, the disease and potential treatments. So we already are seeing growth right now. I'll give you a quick specific here. In 2021, new enrollments, twenty two percent of the new enrollments are tumor LEMS patients. That's a growth of ten percent from 2019.

Speaker 4

And 2019 is only twelve percent. So we're seeing growth with the limited resources that we've applied there thus far, and we know all the work that we've done to date this year will generate new patient opportunities, oncology or tumor lEMS patients next year. The other part is, you take a look at the of the 3,000 patients that are out there, a significant portion of these patients are either undiagnosed or misdiagnosed. So we've applied resources there to help educate patients, caregivers and physicians about LEMS and treatments that are available. And we will shorten that patient journey and that will open up the opportunities for these patients to be served sooner.

Speaker 4

So that's where we see the significant growth coming to 2022.

Speaker 5

Very good. Appreciate that added color, Jeff. Regarding the pipeline question that I had, I'm actually intrigued with several of the pipeline initiatives. But I wanted to focus on the pediatric filing that you mentioned. It seems to make a lot of sense, although you said it's a very, very, very low incidence of or incidence and prevalence for pediatric patients with LEMS.

Speaker 5

I guess I'm wondering if you anticipate being able to provide some PK data or other supportive evidence for that filing? And then is it possible that pediatric LEMS is actually underdiagnosed? And where would the source of pediatric LEMS be from? Could it be for small cell lung cancer patients that are pediatric, which I'm sure is very few? Or what is the source of pediatric LEMS, genetic?

Speaker 2

Charles, I'll let Steve Miller answer that question.

Speaker 3

Well, thanks for the questions, Charles. First, let me speak to the origin of pediatric LEMS. It is autoimmune, like the LEMS for adults. It is essentially the same disease. As you probably know, all autoimmune conditions typically occur later in life, although they can occur in pediatric patients, but it is relatively low frequency.

Speaker 3

And that is the origin of why the numbers are relatively small. It is possible that there is some undiagnosed cases that are out there, but the reality is that the number of patients is relatively small. At this point in time, we believe there may be perhaps ten to fifteen pediatric LEMS patients that are known currently. There may be others out there as well. And as we move our commercial efforts into the pediatric LEMS space upon approval, we may be able to find those patients.

Speaker 3

The most important thing is that in spite of the fact that it's just a few patients, it's very important for us to ensure that we can make sure that every single LEMS patient, regardless of who they are, what age are or where they are, is able to get an approved therapy for their disease.

Speaker 5

Okay. That's very helpful. Appreciate the focus on delivering for patients. Last question I'm not sure if you're going to be able to answer this, but I'll give it a shot anyway.

Speaker 5

So you said that you've got you're happy with the eleventh Court of Appeals designation, terms of orphan, possible en banc hearings. What's your sense of being able to say with the definitive statement on the outcome of that? And regarding legal interactions with wannabe competitors, if you will, how would clarity on the outcome, especially a win with that, putting it to bed, change your perspectives on investment in Firdapse marketing, the pipeline and potential in licensing?

Speaker 2

So Charles, as we've previously stated, the FDA and Jacobus had forty five days from the opinion to seek an inbox hearing or rehearing of the opinion. And that date is approximately November 15. And the statistics bear out that less than 5% of the requests actually ever get reheard. And so we don't know what other strategies may be incorporated by the other side, if you will. But that would be the first step.

Speaker 2

And of course, the FDA, as we understand it, would require actually the approval of the Solicitor General's office to even make that request. So we don't know what the next steps will be. We believe even if a request is submitted that it's likely based on the results of the three judge panel and the opinion, which was strongly worded, we think that it's very unlikely that a rehearing would be granted. And so of course, the other option is cert in applying request to the Supreme Court for a hearing. And less than 2% of cases that are sent to the Supreme Court are actually heard by the Supreme Court.

Speaker 2

So we are from a commercial perspective, and a patient advocacy perspective, we're ready at any point for what the decision of the court may be ultimately and the overturning, if you will, of the FDA approval for Resurgi. And what the results of that would be in terms of transitioning a warm transition of patients to amifampridine.

Speaker 5

Okay. That answers my question. We'll look forward to some visibility on that and appreciate the focus on, like I said, serving the patients. For taking my questions.

Speaker 2

Thank you, Charles.

Operator

Thank you. Your next question today is coming from Scott Henry from Roth Capital. Your line is now live.

Speaker 6

Thank you and good morning. Pat, sort of a follow-up to the appellate court question. Obviously, we can look at the organic growth of Firdapse and chart that out over the next next couple of years. But if Resurgi is pulled off the market, can you talk about how that would change the outlook? Sort of what could be the incremental upside to the revenue line if that event were to occur?

Speaker 6

Yes.

Speaker 2

Scott, that's a great question. And we're reluctant really to talk much about that. Really the concern would be the first concern would be a smooth transition where no patient ever misses a dose of their drug. That's our biggest concern. And I think that we're prepared for that.

Speaker 2

I know we're prepared for that. We previously have stated, when asked how many patients are on REZERGI. We, of course, don't have access to that information. But based on the number of patients that we lost when REZERGI was approved and likely some growth, we've said that we believe that there's somewhere between one hundred to one hundred and twenty five patients that are currently receiving Rosurgi. We know a number of those patients are at higher doses than what our average dose patient is, which is on average about sixty milligrams for an adult patient.

Speaker 2

I think those are the only metrics that we can talk about at this point. And perhaps in giving guidance, if we do so early next year, we can be a bit more specific than we can right now.

Speaker 1

Okay. And thank you, Pat. And if you

Speaker 6

were to pick up those patients, would that be sort of a step increase? Or would you expect them to come in gradually or kind of overnight? Just trying to think about that.

Speaker 2

I think we're seeing some patient migration actually right now to a lesser degree. But I would expect that it would be a fairly large bolus over a thirty to sixty day period of time.

Speaker 6

Okay, great. Thank you for taking that question. Just a small follow-up for Ali, a couple of model questions. First, the tax rate was 27% in the third quarter. What should we think about as the ongoing tax rate?

Speaker 6

I would expect to be perhaps close to 22%, 23%. And then even though R and D is expected to be substantial, should we expect that to trend down given some of the programs that are not getting resources currently?

Speaker 1

So first, to address your tax question, we expect our future rate to be more of a normalized rate. So we expect it to be a fair statutory rate of 21%, but approximately from 2% to 4%. On your R and D questions, while you are correct that some of our programs are winding down, we do expect to have some R and D expenses from our future programs that we might pick up in the future based on our growth strategy.

Speaker 2

Scott, I'll add to that. Thank you, Ali. That for modeling purposes, I don't think you're going to see R and D expenses go down. They'll probably accelerate some as we bring in new projects and opportunities, which we expect in 2022. So I don't think that, that number is going to decline.

Speaker 1

Yes. We also continue to have expenses from our expanded access programs, including and we'll have some expenses from our continuing we expect to have expenses from our continued MuSK MG patients that we'll continue to supply.

Speaker 5

Okay, great. Thank you for taking the questions.

Speaker 2

Thank you, Scott.

Operator

Thank you. Our next question is coming from Joe Catanzaro from Piper Sandler. Your line is now live.

Speaker 7

Hey, guys. Thanks so much for taking my question. Maybe just one quick one for me. I was just wondering maybe over the last year or so of your team's BD diligence, what have you found the most challenging? Has it been asset quality, deal terms or something else?

Speaker 7

And what specifically led you not to pursue that one asset that you alluded to?

Speaker 2

Yes. Good question, Joe. Thank you. The issue really came down to when we brought in subject matter experts and our team was able to do a deeper dive. The issue really was about valuation more than anything.

Speaker 2

The number that in looking at public disclosures when we dug in a little bit, we just couldn't get comfortable with valuation of the transaction. And we did spend a lot of time and effort. And so and we were certainly quite invested in the project. And so I think it took a lot of discipline on our part to walk away. Was unfortunate.

Speaker 2

But I think the biggest challenge has been until we opened up to other therapeutic areas, the biggest challenge was trying to find something in neuro. And as you know, we recently decided that we're going to look outside of neuro and everything, any therapeutic area outside of oncology. And so that's opened up quite a vast opportunities for us that we're looking at. So and I think that valuations as you look at products and or companies that might have some innovative technology, those valuations have come down a bit as you guys know since the beginning of this year. And so I think it's at the time, it was pretty much a seller's market.

Speaker 2

I think it's a little more balanced at this point. And I think that companies are who are in need for additional capital are looking around what their strategic options may be to just doing another financing.

Speaker 7

Okay, got it. That's very helpful. Thanks for taking my question.

Speaker 2

Thanks, Joe.

Operator

Thank you. Our next question is coming from Joon Lee from Truist. Your line is now live.

Speaker 4

Good morning. This is Les on for Jun. And thank you for taking my questions. I have two. First, on the small trial in Japan with your partner, Dido.

Speaker 4

Can we just kind of get an idea of what the study design looks like and time lines? And then second, what have been your challenges of getting the diagnosed patient population on to Firdapse? Well,

Speaker 3

thanks for the question, June. We're not going to go into any detail about the study design because confidentiality reasons with DYNO. I can tell you that it's smaller than the studies that we ran in The United States, as we had previously discussed publicly. And it should be a relatively short period of time to recruit and complete the study, shorter than we spent on studies in The United States.

Speaker 2

Could you repeat the second part of your question, please?

Speaker 1

Sure. I just wanted

Speaker 4

to get an idea of what have been your challenges in reaching the diagnosed LEMS population and putting them on Firdapse? It's I'll take that answer. And it's really not the challenge of reaching these patients because we know the patients and who is treating these patients, these physicians. And many times, it's just the logistics behind it. There was a backlog in patient visits because of the Delta variant and other reasons.

Speaker 4

So the physicians, what they're telling our field force is that as soon as that patient comes back in, they will then put that patient on Firdapse, if appropriate. So that's we feel like there is a backlog pent up demand that will happen once these patients can get back in there. But we are extremely proud of our growth that we've had, and we see this as being incremental growth moving forward. Does that address your question? It does.

Speaker 4

Thank you.

Operator

Thank you. We reached the end of our question and answer session. I'd like to turn the floor back over to management for any further or closing comments.

Speaker 2

Thanks, everyone, for joining our call today. We look forward to our next corporate update. Have a great day.

Operator

Thank you. That does conclude today's teleconference and webcast. You may disconnect your line at this time and have a wonderful day. We thank you for your participation today.

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