Q1 2021 Caladrius Biosciences Inc Earnings Call
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Welcome to the Collagenase Biosciences first quarter 2021 financial results and business update conference call for it.
All participants are in a listen only mode. Following management's prepared remarks, we will hold a Q&A session to ask a question at that time. Please press the star key followed by one on your Touchtone phone.
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As a reminder, this call is being recorded today Thursday may 6th 2021, I will now turn the call over to John.
Vice President of Investor Relations.
For Communications Accolade ran please go ahead sir.
Thank you operator, and good afternoon, everyone. Welcome to <unk> first quarter 2021 conference call to discuss our financial results and provide a business update.
Joining me today from our management team are Dr. David Mazzo, President and Chief Executive Officer, and James <unk>, Vice President Finance and Treasury.
Earlier today, we issued a press release announcing our first quarter 2021 financial results, which is available under the investors and news sections of our company website. If you have not received this news release or if you'd like to be added to the company's email distribution list. Please email me at Jason do you do at collateral Dot com.
Before we begin I will remind you that comments made by management. During this conference call will contain forward looking statements that involve risks and uncertainties regarding the operations and future results for the full address I encourage you to review the Companys filings with the Securities and Exchange Commission, including without limitation. Its forms 10-K, 10-Q, and 8-K, which identify specific.
Civic.
Factors that may cause actual results or events to differ materially from those described in the forward looking statement for.
Furthermore, the content of this conference call contains time sensitive information that is accurate only as of the date of the live broadcast Thursday may six 2021, because I just biosciences undertakes no obligation to update no.
Obligation to revise or update any statements to reflect events or circumstances. After the date of this top line. Please.
Please keep in mind that the company continues to conduct calls from different locations. During the COVID-19 1919 pandemic. So we appreciate your patience should we have any tech.
With that I will now turn the call over to Dr.
Dave.
Thank you John Good afternoon, everyone and thank you for joining us on today's call to discuss our first quarter 2021 financial results and recent business highlights.
During the first quarter of 2021, we continued to advance our clinical development programs and strengthen our financial position, giving us the confidence and means to fund operations for the next several years based on our current development plans, while also allowing us to explore additional pipeline expansion opportunities.
Over the last several months, we delivered on a number of strategic priorities in support of our robust autologous CD 34 positive cell technology based on clinical pipeline on which I will further expand in a few moments following the prepared remarks, covering the financial results with that I will now turn the call over to James NIPSCO, Our vice.
President of Finance and Treasury, who will review and provide commentary on our quarterly financial results.
Thanks, Dave and good afternoon, everyone I'm pleased to join you today and provide a review of our first quarter financial results.
Starting with our operating expenses.
Research and development expenses for the three months ended March 31, 2021 were $5 1 million compared to one 5 million for the three months ended March 31 2020.
Research and development in the current year period focused on the advancement of our schema repair platform and related to.
Expenses associated with efforts to advance C. L. B F 16, and the phase two B freedom trial, which now has multiple sites actively screening and enrolling patients and on.
Ongoing expenses for hone edra, and critical limb ischemia and burgers disease in Japan for which we continue to focus spending on patient enrollment and Japanese rolling NDA preparation and.
Expenses associated with the planning and preparation of an eye on B and proof of concept protocol for C. L. B S 201, as a treatment for diabetic kidney disease.
General and administrative expenses, which focus on general corporate related activities were $3 million for the three months ended March 31, 2021 compared to $2 6 million for the three months ended March 31 2020.
Representing an increase of 18% as a result of our performance Stock Award vesting, one off consulting expenses and a large increase in directors and officers liability insurance premiums as experienced throughout our industry.
Overall net losses were $8 1 million and $4 million for the three months ended March 31, 2021 and 'twenty 'twenty respectively.
Turning now to our balance sheet and cash flow.
In January 2021, we announced that we had closed on the 25 million capital raise through the sale of the company's common stock for several institutional and accredited investors and a private placement price at the market under NASDAQ rules.
Shortly thereafter in February 2021 the company announced that it closed a $65 million capital raised through the sale of its common stock for several institutional and accredited investors and two registered direct offerings price that the market under NASDAQ rules.
In recent months, many small biopharma companies are experiencing an increasingly difficult time as they compete for capital.
Ever. Despite these market hurdles, we have successfully and opportunistically secured $90 million in new capital growth proceeds year to date in 2021.
Providing us with the financial security to focus on execution of our business plan.
As of March 31, 2021, Collaborates had cash cash equivalents and marketable securities of approximately $111.5 million.
On existing programs and projections, we remain confident that the current cash balances will fund operations for the next several years.
Notably through study completion for the phase two being freedom trial of C. L. B F 16.
Through the registration eligible study completion for whom they had dropped.
And through the phase two proof of concept study for C. L. B S 201.
While still providing capital to explore additional pipeline expansion opportunities.
That completes the financial overview with that let me turn the call back to Dave.
Sorry, I was on mute everyone apologize for the delay thank you James.
As I do on all of our results calls I will begin by providing a high level summary of what we are doing a collagenase and why we believe our development programs remain a relevant and attractive investment opportunity today.
Classroom is focused on the development of autologous cellular therapies designed to reverse disease we.
We have late stage clinical programs underway based on our large database of human clinical data to date, our therapies have shown strong signs of effectiveness and durability with a pristine safety profile. Unlike many allogeneic therapies and present the possibility of substantial pharmacodynamic benefit.
Most importantly, we remain focused on the development of personalized curative cell therapy products that will restore human health and improved quality of life with a single administration of the therapy, rather than one that requires frequent re administration.
Our CD 34 positive cell therapy technology has led to the development of therapeutic product candidates designed to address diseases and conditions caused by ischemia, a condition in which the supply of oxygenated blood to healthy tissue is restricted.
Previously published preclinical and human clinical studies have demonstrated that the administration of CD 34 positive cells induces angiogenesis of the micro vasculature that is that these cells prompt the development of new blood capillaries, thereby contributing to the prevention of tissue death by facilitating blood flow to the area of ischemic.
Consult.
We believe that several chronic conditions caused by underlying underlying ischemic injury can be improved through the application of our CD 34 positive cell technology, including but not limited to coronary microvascular dysfunction or C. M D.
Critical limb ischemia or CLI.
Burgers disease, diabetic kidney disease decay D and no option refractory disabling angina or noida.
I will now speak to the specifics on each of our development programs kicking off with C. L. P. S. 16 are promising CD 34 positive cell therapy product for the treatment of coronary microvascular dysfunction.
All of our CD 34 positive cell therapy product candidates <unk> uses a proprietary and patented formulation of CD 34 positive sales specifically designed for an injection at or near the site of ischemic insult, which in the case of CMT is an infusion into the coronary artery.
There'll be a 16 was the subject of the completed escape <unk> trial, a 20 patient proof of concept clinical trial evaluating <unk> 16, as a treatment for coronary microvascular dysfunction, a disease involving damage to the micro circulation in the heart with no accompanying discernible large vessel blockages.
CMT patients have an equally poor prognosis related to major adverse cardiac events and debt as to patients who have identifiable large vessel blockages.
As a result of the lack of discernible large vessel disease, CMT sufferers are often underdiagnosed misdiagnosed or untreated.
It's especially important to note that CMT is even more profit and females than males, making this and more important emerging women's health issue.
We remain committed to raising awareness of this growing womens health crisis, and finding an effective treatment for it.
<unk> 16 is designed to address and reverse the underlying pathology of C. M. D by employing the CD 34 positive cells innate ability to increase micro circulation and thereby hopefully improve the long term outcomes and quality of life and those living with CMT.
In May 2020, the company announced the full data results from the escape CMT trial at the society for cardiovascular angiography and interventions, where Skye 2020, scientific session's virtual conference showing a highly statistically significant improvement in coronary flow reserve correlating with symptom relief.
For patients with CMT after a single intra coronary injection of single out <unk> 16.
Consequently, the company recently initiated a rigorous phase two b clinical trial known as the freedom trial, which is currently recruiting and treating patients at multiple sites in the United States.
Studies designed to complete enrollment by the end.
Sorry, the studies targeted to complete enrollment by the end of 2021 with top line data anticipated for the third quarter of 2022, barring any impact on enrollment due to COVID-19, or other unforeseen challenges.
<unk> hundred five patient double blind randomized placebo controlled phase two b clinical trial will evaluate the efficacy and safety of delivering autologous CD 34 positive sales in subjects with CMT and without instructive coronary arterial disease.
And supported the freedom trial. The company is engaging with the American Heart Association for a variety of initiatives designed to raise awareness of C. M D.
Turning now to <unk> 12, or <unk> in Japan on a product candidate for the treatment of critical limb ischemia and burgers disease in Japan.
As we have described previously CLI is characterized by a severe obstruction of the arteries that significantly reduces blood flow to the lower extremities, principally the feet and legs and represents the end stage of peripheral arterial disease.
CLI patients often experience severe rest pain limited mobility, non healing skin ulcers, and if not successfully treated eventual interpretation.
<unk> was awarded a soccer cocky designation from the Japanese regulatory authorities for the treatment of CLI and brokers disease.
Succour cocky designation is akin to an <unk> designation in the United States and affords the recipient prioritized regulatory consultation a dedicated review system to support the development and review process, including the option of a rolling registration submission as well as reduced review time of six months.
For the registration application once filed.
On Azure is also eligible for early conditional approval and possibly full approval in Japan based on the compelling nature of the complete data from our ongoing prospective randomized controlled open label Multicenter study in CLI and burgers disease patients, which was designed in direct collaboration with the Japanese TDMA.
Note that conditional approval of a soccer gawky product only requires for demonstration of a trend toward therapeutic effect along with acceptable safety.
The ongoing <unk> study in Japan comprises subjects divided into two cohorts totaling 37 patients for number agreed with the Japanese regulatory authorities. Specifically there is a 30 subject group with traditional arterial sclerotic no option CLI and the seven subject group with burgers disease a subcategory.
On a CLI that its orphan inside and it's often associated with heavy tobacco for nicotine product use.
These subjects, who are allocated to treatment are dosed with <unk> in a single treatment involving a series of intramuscular injections. In addition to receiving standard of care pharmacotherapy subjects randomized to the control arm only receive standard of care with drugs approved in Japan, including anti platelet agents anticoagulants invasive dialogue.
<unk> the choice of which is made by the investigators according to the protocol for.
Study allows for the rescue of subjects in the control arm by indicating the crossover to treatment if their disease is deemed to be progressing.
The primary objective of this study is to show that for <unk> can prevent the serious consequences of CLI and burgers disease by reverting the patients to a CLI free condition through improved blood flow in the afflicted limb.
Your life free status is defined as two consecutive monthly visits in which rest pain is absent and previous non healing skin ulcers are completely healed as determined by an independent adjudication Committee.
As previously reported and as you can review in our corporate presentation on our corporate website. The burgers disease cohort is completely enrolled and the results from that group are very positive and consistent with the beneficial therapeutic effect and safety profile as reported by previously published clinical trials in Japan and.
In the United States for <unk>.
Patients with burgers disease, amputation, and EBIT death are likely outcomes and no available pharmacotherapy prevent amputation, however subjects in the burgers disease cohort in our study have shown a very strong signal with for seven subjects meeting the primary CLI free endpoint, which is enough.
Standing on the positive results for these patients who normally see continued progression leading to amputation.
Of course, we are very encouraged by the study results to date and believe that they suggest a positive outcome for the overall trial recognizing however.
Net the final conclusions of the trial will be dependent on all full data results from all subjects. In addition, the company was pleased to report that the FDA recently granted orphan designation to <unk> as a treatment for brokers disease in the United States.
Well the other studies enrollment continues to be significantly slowed by the COVID-19 pandemic and the repeated state of emergency Declaration in Japan, We remain encouraged by the current patient Prescreening pipeline as we work doggedly toward enrollment completion, noting however that the exact date of completing enrollment it's impossible to predict.
Due to the continuing impact of COVID-19 on clinical studies like ours in Japan.
Regarding commercialization our strategy remains to license or partner <unk> in Japan and to that end our conversations continue with prospective partners and we continue to seek to consummate a deal in concert with the completion of the study if not before.
Moving on to <unk> 201 for the treatment of diabetic kidney disease.
Most recently proposed development program <unk> 201 is designed to assess the safety and efficacy of CD 34 positive cell therapy as a treatment for patients with diabetes, who are also suffering from chronic kidney disease, but not yet requiring dialysis.
We refer to these patients as having diabetic kidney disease with Dk day.
Based on a wealth of published preclinical and early clinical data. It appears that the innate ability of CD 34 positive sales to promote the growth of new micro vasculature could be a means to attenuate the progression of decay D or even reverse its course, we are currently engaging FDA discussions designed to finalize an initial.
Development program for <unk> to zero, one and continued to plan to initiate a phase II proof of concept study in the third quarter of this year.
And lastly, I'll ago for the treatment of no option refractory disabling angina.
As disclosed on previously on previous quarterly calls <unk> acquired the rights to data and regulatory filings for a CD 34 positive cell therapy program for ignore that patients that had been advanced to phase III by a previous sponsor.
Based on the clinical evidence from the completed studies that a single administration of all ago reduces mortality improves angina and increases exercise capacity in patients with otherwise untreatable answered on this product received regenerative medicine advanced therapy, or our math designation from the FDA.
Since then we have been in discussions with the FDA regarding the size and scope of our phase III trial of appropriate and practical size, which in combination with the previously filed phase one two and three data could be considered for the registration of all ago.
Currently the FDA maintained that a 400 patient study, including a 50 patient standard of care arm and a 150 patient placebo arm is their requirement.
Epidemiological data indicates that the available nor to population in the United States is orphan inside an addition.
Put from Kols has convinced us that enrollment in this study for nor the patients where there is a large chance of being randomized to standard of care or placebo will seriously deter subject enthusiasm for participation and we'll also have a negative impact on enrollment consequently, our position vis vis FCA remains debt.
Phase III study, requiring a large number of patients and with a combined placebo and standard of care arm.
We will take an unacceptably long time to enroll if enrollment completion would be possible at all.
We'll only initiate a phase III study for all ago. If we can reach agreement with FDA on a study design that is practical and affordable.
So in closing we are very pleased with the corporate and development achievements made during the first quarter of 2021 further attesting to our ability to successfully manage the current global volatile business environment.
To maintain this momentum in the coming quarters as we focus on execution to achieve our project development milestones and with that overview. Operator, we are now ready to take questions.
As a reminder to ask a question. Please press the star key followed by one on your touch.
Please offer only one question per listener at a time and then be prepared to return to the queue for any additional questions.
Our first question is from Joe.
With H C Wainwright.
Okay.
Yeah.
Hi, Alex from firing at calling in for Joe. Thank you. So much for taking my question and my question is really focused on how are your speaking for the COVID-19 impact on the freedom trial I know you touched on them for a little bit, but how are you viewing slightly openings on the impact of enrolling patients.
To free them currently and maybe looking out to the rest of the year.
Well thanks, Sarah for the question and let me be clear about a couple of things here so far we've.
We've had minimal impact.
<unk>.
On the freedom trial, which is the phase II B for <unk> 16, due to COVID-19. So we are we have opened the number we want to say we are on track. According to projections with site openings and have a number of other sites in the process and so.
Identifying and opening sites does not seem to have been impacted by the pandemic and early indications are that patient recruitment is not being impacted by the pandemic. So we will see as more sites get open enrollment really begins to pick up.
But for now our COVID-19 does not seem to be a major.
Challenge for recruitment in the freedom trial.
Contrary to that okay.
Okay, I'll, just clarify for hone net debt in Japan.
For the 19 has a major impact on enrollment and we have been stalled enrolling patients for the better part of 2020 and early 2021 due to the state of emergencies that had been declared in Japan due to the pandemic.
We're still hopeful though that with only a few patients left to enroll that once things are back to normal there will be able to complete the trial in due course.
Alright. Thank you. Thank you for the clarification.
Sure.
Your next question is from Sam Hindu.
Right with Brookline.
I am sure handle on them.
On behalf of Kumar from Brooklyn.
Moving to update.
Hi.
Its hard to question in terms of patient recruitment for the phase <unk> freedom from for <unk>.
And since we know the agenda.
Ex <unk> and other cardiac outcomes I was wondering if for Europe and for their duty patient enrollment.
Yes, we are just to be clear the typical <unk> population is characterized by.
Patients who tend to be.
Younger than 65.
Juruti of which our female generally somewhere between a half for two thirds are female most of those females of premium and and they seem to.
Have a not any particular history of heart disease.
They are scattered around the country and based upon the available data we've calculated there somewhere between 5 million and $1 5 million patients who would be eligible to be treated by <unk> 16, because underlying coronary microvascular dysfunction, and we've taken that into accounts.
In terms of.
The recruitment and site our projections.
Thank you it sounds great. Thanks for taking my questions certainly.
Certainly.
Your next question is from Pete Enderlin MAZ partners.
Hi, Thank you Hi, Dave James.
Hum.
First question I'll try to sort of combine two questions into one.
Abide by your protocol.
I have $112 million.
On the statement is its enough for several years.
No.
And my I remember from English class with several typically means three or more.
And so you.
Burnt $8 million in the last quarter quarter just reported.
And.
If you annualize that.
That would be basically about three years' worth now so excluding all ago. So is that the way you see the deferred rate going on how would you see it progressing over the next.
Two years lets say I know what it'll be.
Most volatile depending on when things started to.
The pace of enrollment on all that but are we looking at you know roughly $8 million a quarter on average going forward.
I think well first of all thanks for your question Pete.
And the reason that we used a qualitative term like several rather than being specific is because.
$8 million a quarter for the next few quarters as we are ramping up and completing enrollment in freedom and then in the sequential fashion wrapping up in completing enrollment in the free to into two O. One proof of concept trial.
Okay that that helps a lot and related to that in the press release.
And before you've said Ah you know what you would have.
Capital for additional pipeline expansion opportunities.
So can you just.
Any way you, possibly can elaborate on that I mean are we talking about things basically close to your ischaemic.
Technology C D 30 for.
Or would it be a little further afield on that and how for the field do you think you can go with your technology base.
Well.
There's a bunch of questions [laughter] hold on one bed [laughter], but but I'll do my best to Anthropy that the fact is that we're fortunate that.
My personal background as well as the background of most of the people on my team is such that we have experience and expertise working across multiple therapeutic areas of course, we've been focused on cardiovascular disease, but we are venturing now intermingled disease as well with two O. One.
Because that's where the angiogenic properties of C. D 30 for seem to be most obviously initially place, but we could at.
At least from a company perspective easily manage programs in almost any other therapeutic category and also with almost any other modality of therapy that is small molecules therapeutic proteins antibodies.
Et cetera, because we all have that kind of experience. So right now our pipeline expansion explorations are I would say opportunistic we are looking at everything that would meet the criteria of being of course affordable have a high probability of clinical <unk>.
Access and would treat a highly unmet medical need and a competitive way and that could be you know of course, we give preference to those things that are closer to the therapeutic area in which for currently working but that doesn't mean that will discount other technologies, because we can manage them from the perspective of capacity.
And expertise.
Thanks, I'll I'll get back in the queue.
Thank you for <unk>.
No question on Friday.
H C anyway.
Hi, Sarah on forget I, just had one line follow up question.
I know you had off that touched upon your interest actual room packing regarding no idea with are there any other at camp ending discussion point aside from the patient population size for enrollment that you guys have yet to agree on on in regard to it and sent to a pivotal study.
No I mean, sorry that that's the frustrating the frustrating situation. So we have I would say tacit agreement with F. D. A and certainly you know a strong database that indicates that noida is.
<unk> orphan incise, alright, it doesn't have an orphan designation, but there's somewhere between 30000 and 100000 available patients in the United States and so for something of that size doing a 400 patient click.
Clinical trial is enormous and then when you make that.
Study design essentially.
Give the patient or the subject of 50 per cent chance of being randomize to a non treatment arm.
These are patients who are having seven plus angina episodes of day and with you know as the name implies no option nothing's working everything's refractory, they're not going to tolerate being.
Randomize the standard of care, which is nothing or to placebo they'll drop out. So there's no point in us initiating a trial, which as suggested by FDA by our best guests would cost over $70 million, but have a very very low probability I've ever being able to be completely enrolled but we're we're.
Being with them at Seeber to see if we can come to an agreement on Ah Ah Ah Ah study size, that's much smaller and with a design that is more attractive to get people to join the trial and he can't get there for whatever reason then we simply wouldn't be bad business to run that trial. So <unk>.
We're we're still in discussion we haven't given up.
But you know it's been slow going mostly because F. D. A has been preoccupied with lots of other things as last year, but uhm, we'll keep at it and hopefully one day, we'll be able to reach agreement on the pivotal trial design that we can actually execute.
Alright. Thank you good luck.
Thanks, a lot.
Okay.
Question of the free.
And now I can't.
Call back on.
I.
Again, thank you all for participating on today's call. We look forward to speaking with you again during our next quarterly conference call and to continuing to provide updates on our achievements in progress we remain grateful for your continued interest in in support of Collagic Biosciences, and we ask that you stay well and have a good evening goodbye.
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