Q2 2023 Fulcrum Therapeutics Inc Earnings Call
Yeah.
[music].
Okay.
Good morning, and welcome to Fulcrum Therapeutics second quarter, 2023 financial results.
Update conference call.
Currently all participants are in a listen only mode.
It is being webcast live on the investors section of full crumbs website at W. W. W Dot com.
Dot com and is being recorded please be reminded that remarks made during this call may contain forward looking statements within the meaning of the private Securities Litigation Reform Act of 1995.
May include statements about the company's future expectations and plans, including the clinical hold on F. T X Dash 6058 clinical development timelines and financial projections. While these forward looking statements represent fulcrum view as of today.
It should not be relied upon as representing the company's views in the future talk from me update these statements in the future, but it's not taking on an obligation to do so.
Please refer to fulcrum its nice recent filings with the Securities and Exchange Commission for a discussion of certain risks and uncertainties associated with the company's business.
Leading the call today will be Alex Sapir, CEO and President Paul Krump, joining Alex on the call today are a doctor Ian phrase shirts interim Chief Medical Officer, and Gregg terrain, Joe Paul Krump Principal accounting officer after providing updates on our key programs there will be a brief <unk>.
You and I am, which Alex Ian and Greg will be available to answer your questions with that it's my pleasure to turn the call over to Alex. Please go ahead.
Thank you operator, and thanks to all of you for taking time to join US today, It's truly an honor for me to have the opportunity to lead fulcrum at this important time and to build on the company is strong.
Foundation, as we work towards advancing our pipeline and delivering on our commitment to improve the lives of patients with rare genetic diseases.
So what I'd like to do this morning is to provide a brief update on our two key programs most map, Vermont for patios, GAAP Yolo humoral muscular dystrophy, our fsh deeper short.
And F T X $6 58 for sickle cell disease.
After that I'll provide a couple of corporate update and then turn it over to Greg for financial highlights and after Greg We'll open it up for questions. So.
So let's start with less map of Mod our most advanced program as a quick reminder, loves map of mine a selective P 38 Alpha beta map kinase inhibitor is currently in phase III development for the treatment of Fsh D. A form of muscular dystrophy with an estimated patient population of 30.
And in the U S now.
However, safety get characterized by relentless and accumulated loss of muscle function over many years, resulting in the inability to perform daily like life activities like putting away the dishes or lifting a cup of coffee activities that you and I take for granted.
Now even more to show Green is the fact that these patients have no approved treatment options for their disease.
These are the factors that drove us to embark on this journey to find options for these patients that had none.
So in June of 2022, we initiated our phase III trial for <unk>, which we call. The reach study, let me give a bit of background on reach its a 48 week trial intended to be registration, enabling both in the U S and in ex U S geographies. The primary endpoint for this study is the change.
From baseline in reachable workspace or our Ws, a quantitative measure of upper extremity range of motion and function that specifically evaluates shoulder and arm mobility using <unk> motion sensor technology.
Now preserving this upper extremity function is critical for these patients to maintain their independence and their ability to perform some of these activities of daily living that I talked about earlier.
As part of this study will also be looking at some other important secondary endpoints like muscle fat infiltration or MSI, which is an important marker of disease pathology as well as self reported quality of life measures.
And health care utilization questionnaires that will really help inform our thinking on our payer strategy as we prepare for a commercial launch.
I'm really excited to share that.
Screening in the reach study has now closed and we expect enrollment to complete later this quarter and with this being a 48 week study we plan to report top line data in the fourth quarter of 2024.
For us and more importantly for the patients. This brings us one step closer to delivering the first ever FDA approved therapy for patients with Fsh date.
So let me now move on to $6 58.
$6 58 is our oral hbf inducer for the potential treatment of patients with sickle cell disease. As previously announced we received a clinical hold letter from FDA on February 24, and at that point immediately suspended dosing and paused enrollment in the phase <unk> trial for $6 50.
Yes.
And the initial feedback provided by FDA. They stated that the hold was related to preclinical data that we submitted in April October and December of 2022, as well as other non clinical and clinical evidence of Hematological malignancies observed with other <unk> inhibitors.
Yes.
In order for us to restart the phase <unk> study. The agency has requested that program further define the patient population, where the potential benefit of country continued treatment was $6 58 outweighs potential risk.
I do think it's important to mention that at this stage. The FDA has not requested any additional preclinical or clinical data as a prerequisite to restarting the phase <unk> study in patients.
Just on preliminary clinical data that we obtained prior to the clinical hold $6 58 has demonstrated dose dependent increases in total fetal hemoglobin or hbf of a magnitude that we believe has the potential to lead to a meaningful improvement in disease severity.
We believe that $60 58 is an oral hbf inducer has the potential to provide a differentiated therapeutic option for patients living with sickle cell disease and that the clinical and preclinical data generated to date demonstrate a favorable benefit risk profile.
Overall, our interactions with FDA have been productive and collaborative and we look forward to continuing our interactions as we work toward resolving the clinical hold as quickly as possible.
I will provide an update once we have more clarity on the regulatory path forward and I intend to provide specifics regarding this more narrowly defined patient population once we have agreement with FDA.
So that covers the updates on our two key clinical programs before turning it over to Greg Let me give a quick update on two other important topics.
As we remain committed to delivering groundbreaking therapies for underserved communities.
In July of this year, we obtained an exclusive global license from camp for therapeutics to acquire intellectual property arising from camp for his preclinical research program in Diamond Black fan anemia, or DBA for short.
Under the terms of disagreement fulcrum Wil research investigational oral compounds for the potential treatment of DBA, a congenital rare blood disorder that affects an estimated 5000 individuals worldwide.
Our agreement with <unk> for further strengthens our discovery pipeline and we are excited to expand on <unk> foundational preclinical work, which we believe has potential broad applications and a unique opportunity for growth.
Additionally, solidifying our leadership team is one of my key priorities and I'm pleased to announce the appointment of Alan Musso to the position of Chief Financial Officer effective August seven.
I have known Alan for some time now and thus have firsthand knowledge of his financial acumen. His keen strategic insights on a range of complex financial decisions that faced the company of our size and most importantly his character.
His leadership experience within the Biopharma industry will be invaluable as the company enters its next stage of development and welcome aboard Allen.
And so with that let me turn it over to Greg to give you an update on our financials Greg.
Thanks Alan.
We ended June 32023, with cash cash equivalents and marketable securities of $278 2 million as compared to $202 $9 million as of the <unk>.
Number 31 2022.
We continue to operate from a strong financial position and we expect our cash cash equivalents and marketable securities to fund our operating expenses into mid 2025.
This projection assumes a timely resolution of the Fts 50, 58 clinical whole.
Collaboration revenue was <unk> 9 million for the second quarter of 2023 as compared to $1 9 million for the second quarter of 2022.
Research and development expenses were $17 8 million for the second quarter of 2023 as compared to $25 million for the second quarter of 2020.
The decrease of $7 $2 million was primarily attributable to a $5 million milestone due to GSK that we achieved during the second quarter of 2022 upon the initiation of reach as well as decreased costs as a result of the clinical hold correct yes.
Sure.
General and administrative expenses were $10 3 million for the second quarter of 2023 as compared to $11 1 million for the second quarter of 2022.
Decrease of Europe was $8 million was primarily due to decreased professional services costs.
Net loss was $23 8 million for the second quarter of 2023 as compared to $34 $1 million for the second quarter of 2022 and with that let me turn it back over to Alan that's great. Thanks, Greg and with that overview operator, Let's go ahead and open it up for questions.
As a reminder to ask a question. Please press star one one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one one again, please standby, while we compile the Q&A roster.
A comment that I heard.
Regarding the F D as the F. D. A has not asked for any more preclinical data in order to get off of hold I'm. Just giving you you are doing some more non talks preclinical studies does does that does that suggest that really the key to getting off of hold.
Lies solely on the ability to define a patient population where there's.
A an attractive risk benefit relationship.
Can you can you help us can you just explain this this concept a little bit more yeah, absolutely Joe and thanks for thanks for asking the question I'll I'll start off and then I'll turn it over to Ian to provide a bit more insight. So you're you're absolutely right. The F. D. A has not requested any additional preclinical.
Or clinical data prior to restarting our phase one b study in impatience, what they've have requested is some additional what I would define as pharmacology data before moving into before moving into healthy volunteers. So would that sort of broad overview, let me just turn it over to eat.
And he can go into a little bit more detail.
Yeah. Thanks, Alex I think it's just important to make that differentiation between the two aspects of the hold and the two aspects of the clinical program. The one around the patients with sickle cell disease and the one be studying and as Alex has said the request. There is is around defining the appropriate patient population.
That there's a preclinical pharmacology study to evaluate <unk>.
Target engagement off to short number of doses.
In order to provide reassurance that.
Giving shortly giving a small number of doses to help the volunteers is not associated with any long term durations. So so that's the work this ongoing but it's specifically for the healthy volunteer population and as we've said before the healthy volunteer population is a nice to have as part of the drug development program.
But it's not an absolute requirements and our priority is very much around getting back into the patient populations for the one b.
Next question operator.
Standby for the next question.
The next question comes from Edward 10 task with Piper Sandler your line of sight fence.
Great. Thank you and just two questions or primary firstly walk home Port Allen [laughter].
<unk> with respect to the question. So you'll go back into healthy volunteer first most likely is that true.
Define what would be a long term follow up there and how quickly do you think you could get back into sickle cell patients [laughter] and then just wanted to follow up is there any specific that needs to be done with <unk>.
Other than just executing on the trial to report the data <unk> next here. Thanks, Yeah.
Yeah, It's great. Let me, let me turn it over to Ian for the first question that I'm happy to take the second the second question, there and thanks for asking though.
Yes, Thanks, Alex.
To be clear the healthy volunteers and since.
Since are not linked in any way and they're getting back into the clinic.
The healthy volunteer studies that are are typically done at this stage are things like Ah Radiolabeled add me study, which is a single dose study or a formulation switch study again typically a single dose crossover type study.
Are supportive for the for the overall program, they're not required and so again priority is to get back into the patients that is not a requirement that there be any healthy volunteers studies initiated first it's getting back into the patients and then then the healthy volunteers studies will proceed in parallel.
As necessary and if there are any issues and getting back into healthy volunteers. Those types of studies can be done in the patient population if needed. So so it's not a requirement helpful clarification. Okay. That's great and and then this is Alex in terms of your your second question yesterday, we had we had announced in our.
Earlier comments were delighted that screen is close this is a study that has enrolled very quickly we would expect enrollment to close sometime later this quarter. So if you add 48 weeks to that which is the.
The duration of the study that would put us out until sometime in the fourth quarter of next year or two released top line and what you would see during that top line releases basic would essentially be what you would expect to see will provide patient disposition the balance between the two arms will be providing the primary and.
Point as well as the secondary endpoints well, we'll report out on safety and we we we are we would expect to see similar improvements that we saw in the redox for study across all of those key primary and secondary endpoint. So we're we're certainly looking forward to add to that that day.
Great. Thank you so very much and said.
Please stay on line for the next question.
The next question comes from day, John How're Stiefel your line of <unk>.
Hello. This is <unk> can you guys hear me now yeah, that's much better. Thank you so much.
Alright, so one of our questions have there been any additional discussions with the F. D. A regarding tax for as a biomarker Netflix H D and <unk> for like a doctor has been reached with minimal effect on tax for a composite score by like a note noteworthy benefit in a reasonable workplace, how would they interpret that and what about the ear.
I see that as well.
Great question I think the answer that let me turn that over to to Ian.
That has not been a topic of discussion with the regulators. The focus is on the endpoints as defined for the reached study which for the primary endpoint is is obviously the reasonable work space and and.
And not the biomarker the biomarker.
Is not being evaluated in in the reach study enrolled in that as I say has not been a topic of discussion.
Okay excellent. Thank you so much thank you.
Standby for the next question.
The next question comes from Matthew vehicular with O P. C. O. Your line is open.
Hey, guys. This is Matt <unk>. Thanks for taking my question I wanted to ask just given the variability inherent in F. S. H D. Could you talk about what gives you confidence that that one year timeline and then she studied will be long enough.
To make a meaningful impact on how it comes thanks, so much yeah N.
Yeah. Thanks man. So so you're correct that there is a heterogeneity in Fsh D. I think that's that's pretty well described we're very much encouraged by the the data from the redox for phase two study in 80 patients on the ritual work space, which did show.
Ah nominally statistically significant difference.
Between less mathematics placebo in that study. So I think we're encouraged by that as we move into a larger number of patients 230 in in the reach study.
And the the powering in that study is based on the observed effects seen in the redox for phase two study so that with respect to the primary and then one of the secondary endpoints in the reach study is an M. R. I M. R. I based endpoint, which because it is a whole body MRI assess.
<unk> accounts for the heterogeneity at least across different muscles in any given patient.
The muscles broadly across each patient. So I think that's helpful. In that it's a more integrated evaluation, it's not focused purely on the upper extremity.
Which the reasonable work space does.
Thanks, guys. Thank you <unk>.
It's a reminder to ask a question. Please press star one one on your telephone please stand by for the next question.
The next question is from Judah Frommer with credit Suisse. Your line is open.
Yeah, Hi, guys. Thanks for taking my question. Congrats on the progress made me it has to put a finer point on the updates we can expect and <unk> I got some if all goes as planned.
What would be the next update we would hear related to patient and what would be the next update we would hear related to a healthy volunteers just trying to figure out if those would come at the same time and which would come first.
Yeah, great Great questions unit. Thanks for asking let me, let me set the stage a little bit in terms of our thoughts on when we would expect it to come off clinical hold and then I'll I'll turn it over to Ian to get more specifically into the the questions that you pose so.
When the when the whole first came out of the end of February I think it was around the beginning of March that we had guided too that it would take at least a minimum of six months in order to reach resolution with FDA, so that sort of puts us out until you know the the September timeframe. So I I would expect that we would be.
Able to share with everybody. The resolution that has been reached and the password with the F. D. A sometime probably in the fourth quarter of this year or possibly even the first quarter.
Of of next year as I mentioned that the conversations with FDA continue to go well I would define them as productive and very collaborative, but but as many of you know it just there there there's a process when engaging with the F. D. A in that in that process simply takes time and then once we get off home.
Obviously, you know one of our key priorities.
Adds an organization is to Reinitiate that phase one the study begin dosing patience again with the 12 milligram.
Ideally 10 patients and then moved to the 20th and our belief that that will continue to see these dose dependent increases in H B F. S that we've been so happy with prior to the Hall I think it's probably a little bit premature at this stage to to.
Guide when we would be able to report out either data from healthy volunteers or data from the from from the other two cohort of 12 milligram and the 20 milligram cohort that I mentioned in the <unk> favour <unk> study, but I don't know, even if you have anything else you'd Wanna share Mmm, no just to reemphasize, what what I.
I mentioned earlier in response to a question that too.
Populations that healthy volunteer in the sickle cell patients are really quite distinct in terms of what is required to get back to those populations and then our primary focus is around getting back to the <unk> patients until we will announce when.
We've achieved agreement.
Agreement on that and moving forward with this study.
In the patient population for the healthy volunteers it'll likely be whatever the next healthy volunteer study happens to be.
That's likely what will be announced but they're not they're not linked and they're not sequence.
That's helpful. Thank you thanks to you too.
This concludes the question and answer portion of the call I will now turn the call back over to fall Crum C. E O Alex foreclosed seeming Mark Alec that's great. Thanks, Michelle So overall I would say I'm I'm very encouraged by the continued progress in the first half of 2020.
Three three and with our strong cash position out until mid 2025.
We have the team look forward to executing on many of the key priorities for our two clinical programs in the months add Uhm and finally, let me just take this opportunity to express my sincere appreciation and gratitude to my fellow fulcrum teammates to the physicians, we work with to advance our clinical studies.
And finally, and most importantly to the patient and their family. That's the efforts of this collective group of people that each day brings us one step closer to treating the root cause of genetically defined rare diseases, and bringing transformative therapy to patients, but thanks again, everyone for joining us on the call. This morning, I look forward to seeing many of you at at at.
Some of the upcoming fall investor conferences have a great rest of the day. Thanks.
This concludes today's conference call. Thank you for participating you may now disconnect.
Mmm.
[music].
Mmm.
[music].
Mmm.
[music].
Mmm.
[music].