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Welcome to the Catalyst Biosciences Corporate Update Conference Call. This call is being recorded. [Operator Instructions] I would now like to turn the call over to Catalyst Biosciences. Please go ahead.
Thank you, operator, and good morning, everyone. Earlier today, we issued a press release announcing our corporate strategy update. You may access that release and the company's website under the Investors tab. With us today are Dr. Nassim Usman, President and Chief Executive Officer; and Dr. Grant Blouse, Chief Scientific Officer.
Following our prepared remarks, we will open the call for a question-and-answer session. Before we begin, I would like to remind you that various remarks that we make on this call contain forward-looking statements, including statements regarding expected regulatory and clinical actions and development and projected operating expenses. These statements are subject to risks, uncertainties and other factors that could cause actual results to differ materially from those expressed or implied in the statements themselves.
All forward-looking statements made on this call are made based on the beliefs of Catalyst as of this date only. Future events or simply the passage of time may cause these beliefs to change. For a more detailed description of the risks that impact these forward-looking statements, please refer to our most recent quarterly report on Form 10-Q and our annual report on Form 10-K. Please be aware that you should not place undue reliance on the forward-looking statements made today.
Finally, this conference call is the property of Catalyst Biosciences, and any recording, other duplication or rebroadcast without the expressed written consent of Catalyst is prohibited. With that, I will turn the call over to Nassim Usman, President and Chief Executive Officer.
Thank you, Ana, and thank you, everyone, for joining us this morning. Our call today is to discuss our strategic decision to stop the clinical development of MarzAA and focus solely on our complement programs and protease medicines platform going forward. It is important to note this decision is not based on any concerns about the efficacy of MarzAA or the ultimate success of the trial.
Rather, it is a strategic decision based on several factors, including a recently updated feasibility assessment in which we determined that it was not in the best interest of our stockholders to continue to finance MarzAA through the completion of the ongoing trials based on anticipated time lines and expense. Enrollment in our MarzAA clinical trial has been very slow. The pandemic created several logistical challenges that were difficult to overcome. Also, we faced increasing competition for study subjects due to the increasing availability of prophylaxis therapy, both approved and in late-stage development globally. Due to these factors, we estimate that the MAA-304 or Crimson-1 study could be completed by late 2023, a time frame, however, and financing hurdle we could not overcome without a partner.
As a protease platform company with a diverse pipeline, we face a strategic decision to either continue as a late-stage hemophilia company developing MarzAA or pivot to an earlier-stage protease medicines company developing our complement portfolio. Our protease platform and complement programs are very exciting and address multiple therapeutic areas ranging from ultra-orphan to large markets in areas such as nephrology, inflammation and ophthalmology.
We believe that this shift offers the best forward for the long-term success of the company and value creation for our stockholders. We plan to report data obtained in the MAA-304 or Crimson-1 trial to date.
The data will show MarzAA has successfully treated bleeds subcutaneously and that we have not observed any treatment-related adverse or thrombotic events. We are exploring opportunities to license or sell our MarzAA and DalcA portfolio.
Turning to our balance sheet for a moment. We reported cash of $64.5 million as of September 30, 2021. Halting development of MarzAA while we search for a partner will allow us to reduce our burn by approximately 40% through headcount and CRO cost reductions and invest our resources in our complement therapeutics and protease medicines platform. Complement is a very large and growing commercial opportunity that is only starting to be tapped. We believe that our key advantage is that our protease platform allows us to develop candidates with a differentiated approach to complement regulation, one that can rapidly engage and degrade high-abundancy targets in a way antibodies and small molecule inhibitors cannot.
We believe complement will open up opportunities in multiple disease settings ranging from ultra-orphan to large markets. We will move forward with our clinical development candidate, CB 4332, a subcu enhanced complement factor I as swiftly as possible and continue to generate candidates from our platform that we will either license or develop on our own. We believe the complement market holds tremendous potential that investing our resources in these programs is the optimal strategy going forward.
Now I will turn the call over to Grant Blouse, our Chief Scientific Officer, who will give more color on our complement pipeline, where it stands as of today and what you can expect in the near term. Grant?
Thank you, Nassim. Our complement portfolio is led by the development candidates, CB 4332 and CB 2782-PEG, both of which originated from our internal discovery platform. CB 4332, a subcu-dosed enhanced complement [ back drive ] protease is designed to address the rare immunodeficiency where there is no current standard of care and potentially other complement-related disorders.
CB 2782-PEG is our C3 degrader product candidate, which we've licensed to Biogen and is in preclinical development for the treatment of dry AMD. We plan to complete the transfer of Catalyst-supported activities to Biogen for this product candidate.
We recently presented preclinical data on CB 4332, indicating its potential as an effective, longer-acting subcu therapy in CFI-deficient patients, that's complement factor I, by replacing the underlying deficient protease. We also enrolled the first 2 complement factor I-deficient subjects in our ConFIdence natural history study designed to assess and follow the clinical outcomes of patients with complement factor I deficiency and, therefore, support the CB 4332 development program. We plan on submitting an IND for 4332 in 2022 and initiating the first-in-human trial with this product candidate.
In parallel, we are generating additional leads from our platform technologies, providing an opportunity to either out-license or develop internally. We presented preclinical data last month and expect to nominate our next development candidate originating from our protein discovery platform in 2022.
Thank you, Grant. The progress and opportunities in our complement programs are quite impressive, which validates our decision to make the pivot now. We thank everyone who has been involved in the MarzAA program over the last several years, particularly our study subjects, clinical trial investigators and site staff, employees and investors.
It is our hope to see MarzAA on the market one day. We look forward to executing as a protease platform and complement company. With that, I will turn the call back over to the operator, and we will take questions. Operator?
[Operator Instructions] Our first question is from Chris Raymond with Piper Sandler.
This is Ally Bratzel on for Chris. So I guess, first, on CB 4332, I think you indicated you've enrolled the first 2 CFI-deficient patients in the natural history study. Can you just talk to early observations on that like how you got to find those patients? And then outline your goals for enrollment in the natural history trial prior to the IND filing. And also, clarify what clinical data we can expect to see from that program next year.
Yes. Thanks. Let me take that in a couple of parts. So -- and just to make sure I get them all, so your first question was how the ConFIdence natural history study works and what the first 2 patients are, what's going on with the ones that we've enrolled. So the way that study works is there's 2 parts. There's ConFIrm, which is screening, and ConFIdence, which then once they've identified a CFI deficiency, they enroll in ConFIdence.
This allows us to follow them kind of on their standard of care, which there really is none for complement factor I deficiency, but how they're treated by their clinicians, what their progress is, most of these patients have recurrent bacterial infections, some have autoimmune encephalitis. So we're able to follow kind of the prevalence of their disease and how they do now in the absence of 4332. This allows us to then be able to compare back to kind of pre-drug when we start trials where we're interventional and looking at the long-term benefits of having 4332 on board. So that's the idea behind the natural history studies.
They also allow us now in our proactive search and outreach with KOLs to identify those patients that we have an opportunity to enroll in the first-in-human studies next year. So all this is going on now while we work towards filing an IND in 2022. And then -- so the first-in-human study, to the second part of your question, is designed to be a standard safety and a PK/PD study.
So you'll have a single ascending dose and a multiple ascending dose regimens for the study, the first of which is single ascending dose, and we expect to be able to have data by the end of the year on the initial PK, and we'll be looking for biomarkers as well because given the introduction of CB 4332, which is the CFI replacement therapy, we'll be able to follow complement biomarkers and then look for a rebalance of those biomarkers in the presence of complement factor I. Does that answer all your parts there?
Yes, yes. That's helpful. And maybe just a second one. Could you remind -- on that big partnership, can you just remind us of the Catalyst flow for 2782-PEG? And any event -- or what are the next events that would trigger milestone payments? And related to that, just on the cash runway. I think by our math, the cash burn has been a little over $20 million a quarter in 2021. So 40% reduction would take that to around $13 million a quarter. So that gives you 5 quarters of runway. So I guess, is that in line with your thinking? And just -- and any other -- or outlining the Catalyst flow over the next 5 quarters, again, I think, would just be helpful.
Yes. With respect to 2782-PEG, which is partnered with Biogen, Biogen is controlling the news flow around that molecule. So there's not much more I can say except that we are currently supporting them in IND-enabling activities, and we'll have to wait for them to update the street on what their next step is.
Our next milestone from that partnership would be around entering the clinic. With respect to your question on the cash runway, yes, we estimate approximately 1 year is what we have at the moment. And I think that answers all of your questions, I believe?
Yes. And then maybe just one last one, just on the kind of timing of the strategic decision and the decision process just for halting MarzAA. I think as recently as maybe September, the target was still to have that for submission by year-end and pivotal data in '22. So just wondering if there's anything like specific you can point to that changed over the last 2 months, that really drove the announcement of the strategic decision today. Or was it really just entirely driven by the pace of enrollment?
Well, it's mostly the pace of enrollment in order -- we were very close to being able to do the DSM-V, but it's the long-term that was impacted by the recent assessment because if you do the math, it's the last patient in that counts, and it's about 14 months for that patient to get through their 10 bleeds. And so when we mapped that all out, the data was getting pushed into the latter part of 2023. And so with slow enrollment, that's what made our decision to switch to the new strategy.
Our next question is from Timur Ivannikov with Raymond James.
Can you guys just talk about Phase I/II MarzAA in rare blood disorders? Does that -- that basically cannot work without a Phase III study? And how far along was that Phase I/II study in terms of enrollments?
So you are correct that, that study could not -- it was not registrational. It was a rather small study looking at PK/PD. We will be sharing the big details of what's come out of that study along with the 304 study. But that study was also facing enrollment challenges. And without the 304 study leading to an approval, it was not viable as a stand-alone. And again, we just did not feel we had the capital to take that all the way to the end.
Okay. Got it. And then just in terms of the timing for the strategic decision, I think you mentioned there was a recent feasibility analysis. I'm not sure how much time you've had to think about this shutdown program halt. And I was wondering whether you had a chance to reach out to any potential partners already, including perhaps Novo Nordisk.
We don't comment on any specific outreach, but we're always engaged in business development.
Okay. Okay. Got it. And then just one more question on the -- so in terms of the complement factor I program, can you just remind us how confident you are, it will not be causing immunogenicity since it is a modified complement factor I?
So the 4332 for CFI deficiency -- and thanks for the question, Timur. It's engineered for extended half life, and we've done -- and are in the process of doing a full immunogenicity risk assessment on that molecule. And we don't feel that there's any -- going to be any issues with that molecule and are very confident with its profile for going into the clinic. And what we do with these molecules is we do a full assessment in silico. We look at potential T-cell epitopes. We look at T-cell regitopes. We look at how the drug product does on its own when, kind of, if you will, fed to antigen-presenting cells and whether or not they induce a T-cell response.
So we really do the full gamut of all preclinical risk assessment we can on 4332 as well as all our molecules going forward to ensure that they're not developing an immune response that would be concerning in the clinic.
Okay. Got it. And then maybe one final question. And with regarding to the prevalence of CFI deficiency, it appears to be an ultra-orphan type of indication. I think your KOL, a few months back, Dr. Haerynck, she showed several patients in her practice. But I'm just trying to understand to what extent you know the prevalence in the U.S. and outside.
Yes. We have that ongoing right now. We've got boots on the ground looking and talking to KOLs and outreach to recruit for the trials. And just as a little bit of color, Dr. Haerynck herself has 12 patients with CFI deficiency. And we've built a pretty solid KOL network across the globe of researchers and clinicians that do have cohorts of CFI-deficient patients that are interested in partnering with us to go forward with the trials for these individuals. So we continue that enrollment and, of course, getting KOLs and patients in the U.S. and the North American content is quite important for us as well. So we're definitely doing that.
Our next question is from Sam Slutsky with LifeSci Capital.
I guess, first, just after the SAD/MAD data with 4332, just remind me where you go from there in terms of indication selection within CFI deficiency and then just general potential opportunity with the asset.
Right. So after a SAD/MAD, so we would still be looking at moving forward. We're thinking about a Phase 2 would be in CFI-deficient patients. These patients have recurrent bacterial infections with or without the advent of vasculitis. And that's probably about 85% of the population of CFI, full CFI deficiency. And then you have another subgroup that has a prevalence of kind of a nephropathy -- not a nephropathy. We have those as well in partial deficiencies. That's an opportunity for expansion later.
On the first study after the SAD/MAD would probably be focused on the fully deficient patients and the opportunities for them. You can think of 4332, it's given as CFI as a natural regulator to the complement cascade. We think there might be potential in other complement-mediated diseases where you add in more complement [ back drive ]. And so we're exploring those opportunities now. And when we've kind of settled on where those opportunities lie, we'll talk more to them.
Got it. Okay. And then just lastly, so regarding the MarzAA studies that were ongoing, anything to comment on immunogenicity in any of this?
No, nothing to comment on today. We're completing all the follow-up studies, and we'll share that along with the efficacy results soon.
[Operator Instructions] Our next question is from Catherine Novack with Jones Research.
Just with regard to the partial MarzAA efficacy safety data that you'd have on hand from the trial as it was conducted to date, when do you think you will be able to share that? And what aspects of that data set would be potentially attractive to a buyer or a partner? And how proactive do you expect to be in looking for a partner or buyer for the asset?
Well, it will take a couple of months to wind down the trial because there are mandatory safety things we have to do for the patients who are still in the trial. And we have to clean the data, of course, and then we'll be able to share that data publicly. So a few months is our sort of general guidance. With respect to finding a partner, we will do that as expeditiously as possible.
Got it. And then in terms of the complement degrader programs, after CB 4332 is in the clinic, how are you prioritizing the rest of the complement program in development in terms of indications?
Yes. So good, great question. So I mean, what we're looking for in indications is that we obviously want the most successful with our next molecule that we're going to nominate as a development candidate next year. And so we're looking both at the indications market prevalence in the competitive market when we go forward. But really, we're also trying to identify opportunities where we, in disease areas such as nephrology and inflammation, where we think our differentiated protease approach will really have advantages over the current modalities that are targeting those indications.
So we're really kind of looking at a whole global aspect of going forward for opportunity, matching the precision medicine of the protease platform approach. And really, the areas that we're focusing on is nephrology and inflammation.
Okay. And then one last question. In terms of the Biogen collaboration, what's left to complete the transfer of CBIO-supported activities for the C3 degrader?
I mean, so we don't really talk about specific details of the activities, but what we've spoken to publicly before with the 2782-PEG program is that we've been responsible for certain IND-enabling studies as well as CMC development. So when our part of those packages are complete, then they're handed over for Biogen to run with internally.
We have reached the end of the question-and-answer session, and I will now turn the call over to management for closing remarks.
Thank you, everyone, for joining us today. We look forward to keeping you updated on our progress.
This concludes today's conference, and you may disconnect your lines at this time. Thank you for your participation.