Q1 2021 Eledon Pharmaceuticals Inc Earnings Call
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Greetings and welcome to the <unk> Pharmaceuticals reports first quarter 2021 financial results. During the presentation, all participants will be in a listen only mode.
Afterwards, we will conduct a question and answer session at that time. If you have a question. Please press the one followed by the Port on your telephone should you require operator assistance at any time. Please press star Zero as a reminder, this call is being recorded today Thursday may 13th 2021, I'd now like to turn the call over to John Kuo horror senior VP of.
Finance. Please go ahead.
Good afternoon, and thank you all for joining Celadon Pharmaceuticals first quarter 2021 financial results Conference call. Joining me today, that's been members of our leadership team, David Alexander growth, Chief Executive Executive Officer, Steve Perrin, President and Chief Scientific Officer, and Paul Little Chief Financial Officer.
Earlier today <unk> issued a press release announcing our financial results for the first quarter ended March 31 2021, you.
You may access the release under the investors tab on our company website Celadon Dot com.
Before we begin I would like to remind everyone that statements made during this conference call relating to <unk> expected future performance future.
Your business prospects or future events or plans may include forward looking statements as defined under the private Securities Litigation Reform Act of 1995, all such forward looking statements are intended to be subject to the safe Harbor protection provided by the Reform Act.
Actual outcomes and results could differ materially from these forecasts due to the impact of many factors beyond the control of Celadon L. A day.
On expressly disclaims any duty to provide updates to its forward looking statements, whether as a result of new information future events or otherwise budgets.
Participants are directed to the risk factors set forth in <unk> reports filed with the Securities and Exchange Commission.
It is now my pleasure to pass the call to our CEO, Dr. David Alexander growth Yeah.
Thank you John and good afternoon, everyone.
We made good progress during the first quarter advancing both our company and our lead molecule <unk> 15 on one.
I am excited by the promise offered to us by <unk> 15, O one as a potential therapeutic for Oregon or seller transplantation, a L S and serious immunological diseases, where patients face limited treatment options.
Steve will go into the details around $18 15 on one program updates, but at a high level in the first quarter of this year, we continue to enroll patients into and remain on track for our ongoing <unk> phase II study.
We reopened our Canadian site for phase two islet cell transplantation study for the treatment of type one diabetes, and we received FDA feedback and updated our development strategy and renal transplantation.
Operationally, we completed our evolution into Eligon pharmaceuticals from Novus therapeutics.
We also bolstered our senior leadership team to help us execute on our plans and achieve our short and long term goals.
Regarding the team I am extremely pleased to introduce four new executives, Paul Little Chief Financial Officer, Jeff Bornstein S. Chief Medical Officer, David <unk>, as Chief Regulatory Officer, and Brian Smith, as General Counsel, corporate Secretary and Chief compliance Officer.
With him on board I am thrilled that we've built out a top tier leadership team that I believe positions us to achieve success for the benefit of patients and shareholders alike.
I will now turn the call over to Steve Perrin, our President and Chief Scientific officer to discuss our clinical programs after which Paul Little will provide a financial update.
Steve. Please go ahead thank.
Thank you D day.
For those of you that are new to our non story I'll start by giving a brief overview of our lead asset <unk> and IGT won anti CD 40 ligand antibody lacking FC effector function.
Physiologically the interaction of CD 40, ligand and city 40 results in clonal expansion antibody production and secretion of pro inflammatory cytokines that amplify on immune response, the CD 40, CD 40 ligand pathway attractive drug target because of the engagement of these receptors plays a pivotal.
Our role in immune system activation by media I think both antibody and cellular from the responses.
We are focusing our efforts on the development of an antagonist antibody targeting on the ligand rather than a receptor since targeting the ligand has been shown to be more efficacious in preclinical models of autoimmunity as well as on the prevention of acute and long term allograft transplant rejection.
There may be three different biological mechanisms that benefit targeting anti CD 40 ligand over targeting anti CD 40 receptor.
The ligand is expressed on fewer cell types with expression generally restricted to platelets and youll see yourselves and activated lymphocytes.
Whereas the receptor is more broadly expressed on antigen presenting cells, including monocytes macrophages dendritic cells and specialized antigen presenting cells.
Targeting both the receptor and the ligand inhibits b cell activation in class switching as well as inhibiting the pro inflammatory repolarization of CD four positive helper T cells.
However, blocking CD 40 ligand also inhibits other integrin receptors, including CD 11 receptors on antigen presenting cells. Thus blocking on the pro inflammatory polarized polarization of CDA positive cytotoxic T cells as well.
And third blocking the ligand also polarizes CD four positive lymphocytes to Fox P. Three positive T regs, thus potentially creating a more taller generic environment.
Our current development strategy is to target for indications, which I'll discuss on order of clinical development.
First amyotrophic lateral sclerosis, or AOS. We're currently have a phase II trial enrolling.
Pilot cell transplantation as a potential functional cure for people living with type one diabetes, where we currently have an active clinical sites in Canada.
Third prevention of kidney allograft transplant rejection, where we have received guidance from the FDA on the path forward in the United States on a run parallel exploring a renal transplant study ex U S.
And fourth autoimmune nephritis were.
Finalizing indication selection and plan to communicate our strategy next quarter.
I will now go into more detail regarding each of these indications.
And I was on and initiated a phase two safety Tolerability and biomarker study in October of 2020. The trial is a 12 week open label dose escalating study with four doses of <unk> one.
Currently the study's enrolling at 12 sites in the United States and Canada.
Our enrollment continues to be on track and we expect to read out top line data on the first half of 2022, we will assess two primary categories of biomarker endpoints with each subject serving as their own control by comparing changes from baseline.
And the first category will assess biomarkers of CD 40 ligand target engagement Mecca.
Mechanistically blocking CD 40 ligand has profound effects on b cell maturation antibody production and the antibody class switching.
We anticipate that we will be able to assess CD 40 larger target engagement by $80 $50, one with markers of B cell function such as <unk> <unk>.
The second category of Biomarkers or changes in pro inflammatory <unk> and cytokines up regulated in people living with ALS.
There is a long history of AOS data describing increases a pro inflammatory signals on circulation, including TNF TNF Alpha MCP, one IL six and enraged we anticipate blocking of CD 40 ligand will result in an overall decrease of AOS related pro inflammatory markers in circulation.
Finally, we will also assess the other exploratory endpoints, including changes in AOS functional rating scale or <unk> for us respiratory function as well as the levels of neuro filament light chain in circulation. Since recent publications have shown a correlation between levels of <unk> light chain and the rate of Dizzy.
These progression in ALS.
In diabetes, we are focusing on people living with the brutal phase of type one diabetes, who are on chronic treatment with absorptions insulin and experienced severe swings in blood glucose levels hypoglycemic on awareness and associated Comorbidities.
Clinical trials conducted by the clinical islet transplantation consortium as well as islet cell transplants and other countries have demonstrated that islet cell transplant in patients with difficult to renewal type one diabetes may help maintain glycemic balance reinstate metabolic control and in some cases, even and eliminate the need.
For example, since insulin.
However, the current use of <unk> inhibitors, where C&I is necessary for the prevention of violence all transplant rejection.
<unk> two issues.
C&I are toxic towards transplanted islands, potentially resulting in significant islet cell loss post transplant and may lead to the requirement for multiple islet cell transplants.
Indeed in published trials I would say.
Transplant recipient recipients typically require multiple transplants with the need for a second transplant often apparent within approximately 90 days after the first transplant.
Beyond islet cell toxicity C&I is have a long list of associated toxicities, including nephrotoxicity, cardio toxicity tremor and hair loss.
As a result, we believe the replacement C&I with $80 $50 on one may improve I'll, let cell survival and clinical outcomes associated with islet cell transplant, thus potentially allowing for islet cell transplant to become a viable treatment option and even a potential functional cure for persons living with brittle type one diabetes and <unk>.
Collaboration with normal Kenyon at the University of Miami, We've generated preclinical data in a non human primate model of islet cell transplant, demonstrating that <unk> hundred one in combination with other immuno.
Modulators or as monotherapy may have the ability to prevent acute and long term rejection of transplanted islands.
We currently have an active clinical site in Alberta, Canada seeking to enroll a single open arm label study primary endpoints include safety and Tolerability glucose control insulin independence reduction in HBA, one see in graft survival and function.
We will also be assessing hypoglycemic on awareness events as well as renal function.
Although Alberta has been significantly impacted by COVID-19 and the viruses complicated travel in Canada, we are still targeting to enroll the first patient this quarter and generate interim data and I look cell transplant in the first half of 2022.
With regards to data, we also look forward to having presentations and posters at the American transplant conference in June where we plan to present data from our phase one clinical study of <unk>, one as well as preclinical data, including characterization of $80 51, and in vitro assays and non human Primate studies.
<unk> in the prevention of islet cell transplantation rejection.
Moving on to renal transplantation I'd like to address the announcement, we made on April 26, updating our development strategy for <unk> and renal transplantation following our ongoing discussions with the FDA.
Recall as background that we've already studied <unk> and demonstrated a good safety profile in over 60, non human primates, including a model of islet cell transplantation, where the molecule also showed preclinical efficacy.
Moreover, multiple historical anti CD 40, login molecules similar to what <unk> have demonstrated preclinical safety and efficacy and hundreds of non human primates, including specifically in non human primate models of renal transplant patients.
Nevertheless, as part of our normal course of discussions to start the phase II trial for kidney transplantation. The FDA requested that we first provided $80 51 drug specific renal transplant data in non human primates prior to initiating a phase II trial in the U S.
To meet the agency's request, we plan to initiate a preclinical study of $850, one and our non human primate model of renal transplantation, which we expect to complete in late 2022.
Although we cannot fully predict what regulators in other jurisdictions will now require we are also exploring the ability to conduct a renal transplantation clinical trial outside the U S. In parallel to this new non human Primate study.
We will provide updates on this potential clinical trial and its design as we have discussions with and receive feedback from international regulatory agencies.
Of note the FTE on the.
Feedback on renal transplantation does not impact our other ongoing development programs in either the U S or Canada there've been no significant new drug related safety signals with $80 50, no. One in this new FTA request was not due to any specific event for data from our ongoing trials of <unk> 15 on one.
Yes.
In auto immunity, we are focusing on autoimmune nephritis, which are autoimmune diseases of the kidney.
There was a long history of preclinical and clinical data demonstrating the blocking CD 40, lagging signaling ameliorate the disease progression modifies biomarkers of disease, and improved renal function and diseases, such as focal segmental glomerular sclerosis, lupus nephritis and Iga nephropathy Mauro.
Moreover, soluble CD 40 wagon, often correlates with disease disease flares in autoimmune diseases such as these.
We look forward to providing more details on our development plans on autoimmune Freitas next quarter.
With that I'll turn the call over to Paul from a financial update.
Thank you Steve in addition to the financial results summarized in our press release, you can find additional information in our form 10-Q, which we will file later today.
The company reported a net loss of $8 5 million or <unk> 57 per share for the three months ended March 31, 2021, compared to a net loss of $8 2 million.
Or $8 32 per share for the same period in 2020 <unk>.
Research and development expenses were $5 6 million for the three months ended March 31, 2021, compared to $1 6 million for the same period in 2020.
The increase in R&D costs was primarily due to spending related to the production of clinical trial materials and other CMC activities as we advance our <unk> 15 on one program.
General and administrative expenses were $3 3 million for the three months ended March 31, 2021, compared to $1 7 million for the same period in 2020.
The company had approximately $108 $6 million in cash and cash equivalents as of March 31, 2021, compared to $114 2 million in cash and cash equivalents in December as of December 31, 2020, we expect our financial resources to be sufficient to fund operations as currently planned well into 2012.
Three.
With that financial update let me turn the call back over to day to highlight some of our key upcoming milestones for 2021.
Thank you Paul.
And the early part of this year, we became Allentown pharmaceuticals, and both continued to make progress advancing <unk> hundred one and building out our team.
During the second quarter, we expect to initiate a phase II trial, and I'll, let cell transplantation and.
Type one diabetes, and we will be presenting <unk> data, including non human primate data in type one diabetes model at the American Society of transplantation annual meeting in June.
Moving forward, we will continue our efforts to advance our renal transplantation program by initiating a study in a non human primate model, while exploring a parallel track that would involve a clinical trial outside the U S.
Lastly, we will also continue to work on our preparations in the autoimmune nephritis, where we expect to launch a draw later this year.
With a world class team and strong cash position, we believe that we are well positioned to deliver on these important milestones as we develop valid on into a leading targeted immunology company focused on the CD 40, CD 40 ligand pathway.
With that I will now ask the operator to begin our Q&A session.
Operator, Sir.
Certainly and thank you very much.
I would like to register a question. Please press the one followed by the four on your telephone you will hear three total probe to acknowledge your request.
If your question has been answered and you would like to withdraw your registration. Please press. The one followed by the three once again to register a question.
Just one for new telephone keypad. Our first question comes from Alicia Young with Cantor. Your line is open.
Hi, Thanks for taking the college on.
On lithium.
Just kind of curious why you think the FDA one net.
That information.
Information related to <unk> one plan.
Given that there is already.
On the data out there from.
The competitors.
So.
I think the.
The FDA.
Was focused on.
Being able to see drug specific.
And Oregon specific data. So as you know we have done as we discussed on this call non human Primate studies looking at transportation, but with islet cells.
And here in transplantation, we're in a unique situation where.
It becomes hard to separate safety and efficacy data since one needs efficacy in order to be able to protect the Oregon.
So.
The FDA was looking for.
Was to have it.
Specific.
Information on <unk> hundred one in renal transplantation.
In order to make sure that the gift.
Give comfort that there would be not only safety, but efficacy.
In this.
Particular type of transplantation.
Okay.
And that really has to do there as you know each Oregon is it's very valuable here.
And so there is.
There is quite a high bar with transplantation.
Okay.
Just a follow up can you just maybe talk a little bit more about your potential.
Potential plans to run on ex U S study.
<unk> layered on transplant, just two instead of a channel right.
Some data here.
Exactly so these are still.
Early days in these discussions as you know we just received.
<unk> back from the agency recently.
But the goal would be to do a smaller trial than what we had initially anticipated.
But one that could be done internationally and to your point would allow us to begin to be able to show.
Data in terms of both safety and efficacy in renal transplantation in humans.
Okay got it thank you.
Great. Thank you.
And our next question comes from Remy has Cooper with life Science capital. Your line is open.
Hey, guys. Thanks for taking my question.
A quick one from me, but based on preclinical data that ITT data that you've presented do you guys envision utilizing a monotherapy arm in the upcoming phase II trial or will you test 15 on one in combo with standard of care.
Just to clarify are you talking about the renal trial.
Human islet cell transplant trial or the day.
Non human primate renal trial.
Sorry, this would be for the human islet cell transplantation trial.
Sure, let me turn that over to Steve to talk about that trial.
Yes, no currently the human <unk> cell transplant trial was not on monotherapy trial.
On a trial, where we're trying to really achieve getting rid of <unk> inhibitors due to the associated toxicities that we described we really feel that in the absence of C&I is based on our preclinical data and historical preclinical data that we'll have much better islet cell survival much better islet cell function and hopefully reduce the number of transplants required to get.
People are large enough pilots on mass.
To really impact insulin production. So it's not on monotherapy trial to trial with induction therapy plus maintenance therapy.
Got it thank you very much.
Thank you.
Thanks, Jeremy.
Yes.
Our next question comes from Thomas Smith with SVP Leerink. Your line is open.
Hey, guys. Good afternoon. Thanks for taking the questions I guess first just on the updated plan on renal transplant can you provide a little more color on the plans for running the additional.
Non human Primate study I know you've laid out some initial timelines, but just a sense of I guess size and scope and specifically what what youre looking for.
Okay.
Yes, so we're still on discussions with the agency about the design.
Obviously, we're trying to be sensitive for the utilization of non human primates in these types of experiments, but understand.
The ability to generate that type of data.
The guidance from the agency they want to show our ability to prevent transplant rejection. So that was probably the most important requirements as we think about our design with them, but we're still on a dialogue on what that design needs to look like it will most likely include monotherapy.
In order to be able to show the effect.
Specifically our antibody.
Okay, Great and then.
Follow up question just in terms of.
The ALS study enrollment as we're starting to see some improvement in COVID-19 infection rates, maybe if you can just talk a little bit about enrollment trends are you guys seeing any meaningful changes in terms of.
Patient enrollment or cadence that gives you a little bit more confidence in the first half 'twenty two.
Timeline for data.
So in terms of the AOS trial as we mentioned were.
We're on track.
Overall, we've been on.
On track for.
Pretty much the whole.
The whole time since we've launched the trial.
We did see some slowdowns that were associated with the various COVID-19 peaks.
Here in the United States, but maybe because we're an enough sites and the sites are distributed across the United States and Canada.
It did not have a meaningful impact.
In terms of.
Recruitment and enrollment.
So with regards to that one trial, we continue to see.
Good interest as we've seen.
But at least that specific trial.
As Ben so far quite independent.
What's been happening with COVID-19.
Okay, Great I appreciate the color thanks, guys.
Okay.
And as a brief reminder to all to register for a question. It is one four on your telephone keypad next.
Next question comes from Matt Kaplan with Ladenburg Thalmann. Your line is open.
Hi, Good evening guys. Thanks for taking the question.
Just wanted to follow up a little bit on the islet cell transplant trial, it sounds like you're about to.
Have your first patient pay.
Patient enrolled in that near term.
How should we think about kind of the rollout of data on that from that study is it going to be.
As you reach.
Certain co.
Cohort number or when you announced that <unk> has as you get.
Kind of completion towards the completion on the study.
Great.
Thank you very much Matt, let me turn that over to Steve.
Yes, it's a great question, Matt I mean, obviously, we want a cohort of patients to be able to determine readouts on the study. So we're enthusiastic about.
The site is active in.
We're looking forward to our first patient coming in but we need three or four subjects worth of data to really understand the.
The outcomes that we're looking at in the study, which is islet cell function met.
Metabolic control quite semic awareness. So I think we're still on track to be able to acquire that data for late next year, but we're not going to be announcing data on a subject by subject basis. So we will announce the data on the first upset in the first half of next year and then we would expect to have the full number of patients enrolled.
And to get that data that full topline data in the second half.
Okay. That's very helpful. Thank you and then second question.
You mentioned some upcoming.
Presentations at the American Society of transplantation.
Can you give us a little bit more color in terms of what to look for there and as you announce.
That does.
Those datasets in June.
Yes, sure there's four presentations, that's going to happen up a transplant Congress, there's two page two posters and two oral presentations.
One of the posters is focusing on the historical design of <unk> 15 on one and its characterization primarily in in vitro studies with.
With a receptor binding FC effector function.
And lack of platelet activation.
The second poster is focused on the non human primate work that we did initially with a $2 15, no. One so talking about the pharmacokinetics in non human primates as well as reviewing data from our two <unk>.
<unk> toxicity studies that we did in non human primates.
<unk> week study as well as the subsequent 26 week study.
On the two oral presentations one of them is focusing on a presentation of our phase one data of <unk> hundred 51, primarily in healthy volunteers, but we did have a cohort of AOS patients on that study and again focusing on safety Readouts.
Pharmacokinetics.
Responses as well as we did do an immune challenge showing functional activity of the antibody in that study.
And then the last presentation will be presented actually by normal Kenyon our collaborator on Miami, who has worked with $850 one in her nonhuman primate.
Sell model and she'll be presenting data on 851 in combinations with other traditional immune module Tory drugs that are used in the context of transplant rejection as well as presenting data on with several doses of <unk>, one either as a monotherapy and presenting data on the ability of <unk>.
Didn't want to prevent acute and long term islet cell transplant rejection.
Great. Thanks, Thanks, a lot for the added color Steve.
Youre welcome.
And all this brief reminder.
To register a question its one four on your phone next question comes from Vernon Bernardino.
Bernardino My apologies from Hs.
<unk> Your line is open.
Thanks for taking my question noise, you might get from where you are able to plan if you say that though.
Just a follow up on Matts question, Steve can you describe a little bit.
What.
On the immune challenges and how it's done.
Yes, Theres a couple of different ways to do immune challenges what we typically it's done with any type of a foreign antigen. So tetanus toxin is one that's not uncommon.
<unk> is another one that's very common and that's the one that we used to say a protein that's derived from shellfish and so you can imagine and we try to avoid people with this type of an experiment production of shellfish allergies, but if you take a foreign antigen like from Shellfishing injected subcutaneously under the skin it would generate a very.
Acute hypersensitivity reaction because your body has never seen that protein before so the way. These experiments are typically done and we did it a little bit different typically what one would do is enroll your subjects into the study.
And probably three to seven days before you do that foreign protein challenge you would start treating them with your immune mall modular Tory immunosuppressive drug to start tampering down their immune system and then you come in and do your sub Q challenge to see if you can block the body's immune system from recognizing that foreign antigen.
What we did in our phase one study was actually quite more aggressive the match, we actually injected the KLA each sub Q at the exact same time that we did our IV infusion of <unk> 51, and yet we demonstrated the ability to completely block the immune system's recognition of that for <unk>.
And in two or three subjects and we did get a pretty good attenuation even in that third subjects. So under a very difficult challenge was the first data to show we had functional activity of $80 51 in humans.
And so you'll do the same.
With the current Alice studied that is on the same day.
So on the AOS study, we're not planning on doing any KH challenges in that in that phase II study in ALS.
Okay.
And then.
Would that data for the immune challenged served as.
The result, you would.
Keep in mind as far as the <unk>.
Results concern across the board as far as the different studies that's free.
Oh in Ireland.
I mean, the goal of the KLA study on the Phase. One study was really to just show the first signs of functional activity for $80 50 don't want on humans and our subsequent studies were more focused on disease specific biomarkers to show functional activity.
As I described in our <unk> study there was a couple of different biomarker components to that study.
One of them being the ability to look at $850 <unk> changing b cell function.
As I described when you block the CD 40 ligand pathway. It has a dramatic impact on B cell maturation antibody production class switching and one of the ways that you can measure of that is markers of b cell function on one of them is <unk> 13.
Another way to measure whether <unk> 51.
As having effects on AOS is to look at those pro inflammatory markers that have been described in multiple studies that are elevated and people with AOS compared to healthy controls.
<unk> modulates the immune system should have an impact on those pro inflammatory markers.
Perfect.
Looking forward to the.
ALS data at.
Moving to phase one data of transplantation and continued progress with the ALS and all that in vivo studies. Thank you for taking my question.
Thank you. Thank you thanks Vernon.
And there are no further questions at present time, please continue with your presentation or closing remarks.
Thank you again to everyone for joining us on the call.
We are pleased you could join us today to hear our progress during the first quarter and we look forward to keeping you updated on our programs.
And that does conclude the call for today, we thank you very much for your participation NFC. Please disconnect your lines.
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