Q4 2020 Iveric Bio Inc Earnings Call
Please standby were about to begin.
Good day and welcome to the perfect bio fourth quarter and year end 2020 results Conference call. Today's conference is being recorded at this time I'd like to turn the conference over to Kathy Galante Other Investor Relations. Please go ahead.
Yeah.
Good morning, and welcome to I'm very close conference call, representing <unk> bio today are Mr. Glenn <unk>, Chief Executive Officer, and President Dr. David Guyer Executive Chairman, Mr. David Carroll, Chief Financial Officer, Dr. Praveen, do little Chief strategy and business Officer, Dr. Abraham.
Scoria, Chief Scientific Officer, and Mr. Keith Westby, Chief operating officer.
I would like to remind you that today, we will be making statements relating to <unk> bio's future expectations regarding operational financial and research and development matters, including statements regarding our expectations for patient enrollment and patient retention and gather two our second phase III clinical trial evaluating tomorrow.
Treatment of geographic atrophy secondary to age related macular degeneration, our expectations to use gather one our previously announced clinical trial and more so the treatment on T. A secondary to AMD and the phase III clinical trial.
Our development and regulatory strategy for tomorrow, and our other product candidates, including our expectations for additional indications for which we may pursue the development of the Lora and IC 500, our hypothesis regarding complement inhibition and HD one inhibition as a mechanism of action for the treatment of gea and potentially.
Other stages of AMD, our projected use of cash and cash balances the timing progress and results of clinical trials and other research and development activities and regulatory submission and the potential utility and development potential of our product candidates and the potential for our business development strategy and our personnel and human.
Capital resources.
These statements constitute forward looking statements for the purposes on the Safe Harbor provision under the private Securities Litigation Reform Act of 1995. These statements cover many events and matters that are subject to various risks that could cause actual results to differ materially from those expressed and any forward looking statements including.
Risks related to the future progression of the COVID-19, pandemic responsive measures to the pandemic and their impact on our research and development programs operations and financial position initiation and the progress of research and development programs and clinical trials, including enrollment and retention and clinical trials avail.
The ability of data from these programs.
Lions on contract development and manufacturing organizations.
University collaborators and other third parties establishment of manufacturing capabilities expectations for regulatory matters development from our competitors and the marketplace for our products need for additional financing and negotiation and consummation of business development transactions and other risk factors I refer you to our.
SEC filings and in particular to the risk factors included in our quarterly report on form 10-Q filed on November three 2020 for a detailed description of the risk factors affecting our business.
In addition, any forward looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date, while we may elect to update these forward looking statements at some point and the future. We disclaim any obligation to do so as required by law I would now like to turn the call.
Over to Glen.
Thanks, Kathy and good morning, everyone and thank you for joining us for our fourth quarter and year end conference call and hope you and your families or will.
As we get closer to turning the corner on this COVID-19 pandemic.
We start 2021 with significant momentum as we are excited to share that patients with <unk>.
And retention for Gaba too on search.
<unk> phase III clinical trial, and so as the war for the treatment of geographic atrophy as a G I.
Secondary to age related macular degeneration, or AMD is progressing well with enrollment trends and I had the schedule.
We are accelerating more tolerable to compete and completing enrollment and.
The other two to the third quarter of 2020 one.
With the 12 month results from gathered to low positive.
And final applications with the results from Gaba won our first phase III clinical trials because of more on <unk> and <unk>.
Other two with the U S food and drug administration and the.
European Medicines agency, the marketing approval of the malls could you share.
We're extremely encouraged by the progress of enrollment and gather two.
We believe patient retention is an important.
And since it is as important as patient recruitment and on <unk>.
Courage button on.
Perfect.
Programs, we have put in place to retain and patients and Kathy.
And two.
Since the initiation of gathered two and June 2020, our team has been proactive and creative and put in place multiple initiatives to tackle the many challenges that the COVID-19 pandemic has brought to conducting clinical trials.
These initiatives are designed to reduce the risk and exposure of COVID-19 to patients and the staff treating them.
Keith will review the details of our programs and more detail and a few moments.
2020 was a transformative year for a very quiet.
Please.
To recap some of the highlights from 2020, but we're most excited about.
We reported positive phase II.
Results from Jabra warm.
These data support our previously announced 12 month data.
From this trial.
But which telling point is and more met the pre specified primary efficacy endpoint with statistical significance and.
Importantly, we believe the other one is currently the oldest and.
Completed phase III clinical trial showed suppression of Gi growth with can treat with continuous treatment effect over time.
We dosed, our first patient and gathered to clinical trial can serve as an important step and potentially delivering a clinically meaningful therapy safely to patients would you add.
The 12 month results from our gather one clinical trial were published and a highly respected journal ophthalmology.
Initial journal of the American Academy of Ophthalmology.
Over the course of the year and number of leading Kols presented to gather one data with numerous medical conferences and most recently Purdue presented together one results at the Bascom Palmer angiogenesis and explanation meeting last month.
The FDA.
The us FDA granted fast track designation for some more for the treatment of G. A secondary to AMD.
We also increased the enrollment target target and our ongoing sales to be screening trial of the more up to the treatment of autosomal recessive store growth disease.
Moving to enroll an additional 25 patients consistent with our original goal of enrolling a total of 120 patients.
And our ICEE 500 program, we are pursuing each tiara one inhibition is an important target and the treatment of G E.
Essentially earlier stages of the M D.
So we will provide your applause for ice and 500 and a few moments.
And our gene therapy programs, we are progressing to finalize the dosing levels for IC 100, and our product candidate for the treatment of adoption mediated autosomal dominant retinitis pigmentosa.
Clients to file on R&D and moving into the clinic.
Phase, one and two trial, beginning and the second half of 2021.
We're also progressing the development of <unk> 201 product candidate for the treatment of best one related IR dues with clients to follow and IMT and move with it.
And to the clinic with a phase one and $2 12 also beginning and the second half of 2021.
Keith will provide more details for both programs and a few moments on.
On the financial foot and 2000, Twenty's and strength of our balance sheet with an underwritten public offering and a concurrent private placement with diebold capital and some sales.
And the bio capital raising approximately $160 million and gross proceeds.
On the corporate front and 2020, we expanded our board of directors and executive management team by adding three leading industry experts, Dr. Mark Bloom and price to our board.
And doctors, moving Google and double decided to our management team and.
And we continue to selectively expand our workforce and the expertise and retina CMC and regulatory.
And I'll turn the call over to Q2 will review the other one and gather two clinical trials and ups.
Great you on our gene therapy pipeline and orphan inherited retinal diseases cheap.
Thank you Glenn and good morning, everyone. We are extremely pleased to accelerate our timeline for completing enrollment and gather two to the third quarter of 2021.
We are in and unusually fortunate position of having already published a positive phase III study gather one and a major peer reviewed journal we.
We believe this is our most impactful recruitment tool with investigators and gather too.
Further the early and continuous treatment effect demonstrated in the gather one trial along with Zamora is well tolerated safety profile that was maintained throughout the 18 months trial are key motivators per patient retention and together two trial.
We continue to work with our investigators to provide a safe environment for patients, which we believe increases the patient comfort and confidence to participate and the gathered two clinical trial during the Covid pandemic, we implemented a number of initiatives to reduce the risk and exposure to COVID-19 for our patients and the staff treating.
We're committed to continuing patient enrollment and retention aggressively and the gather two clinical trial, while prioritizing patient safety.
I would like to outline some other steps our clinical operations team has taken to drive recruitment and retain patients throughout the trial.
We increased the number of clinical trial sites participating and gather two as compared together one.
We are providing private transportation per patients to and from their scheduled office visits.
Pfizer setup for social distancing, and we are providing PPE to patients and site personnel.
As Glenn mentioned earlier retention is as important as patient recruitment.
We are proactively monitoring visit scheduling and patient follow through to ensure scheduled treatments are maintained.
We also continue to monitor the COVID-19 situation closely and explore additional ways, we can support our patients and clinical trial sites.
In total we are planning to enroll approximately 400 patients and the gathered two clinical trial.
Patients were randomized one to one into two cohorts. The first cohort receiving monthly administration of Zamora, two milligram for 12 months and.
And the second cohort receiving monthly administration of Sham.
Similar to the gather one clinical trial the Prespecified primary efficacy endpoint will be the mean rate of change and GE growth over 12 months.
And by fund this auto fluorescence at three time points baseline month, six and month 12 by square root transformation.
If the pre specified primary efficacy endpoint is met at month 12, we plan to file for marketing approval and do more for the treatment of GA secondary to AMD with the FDA and EMA.
At month 12, we plan to re randomize patients and is it more a two milligram arm to receive either monthly or every other month administration of <unk> and more of two milligram.
And the patients in the Sham cohort will continue to receive monthly Sham administration.
The final safety evaluation will be performed at month 24 for all patients.
As previously reported and together one clinical trial.
And that its pre specified primary efficacy endpoint at 12 months with statistical significance.
The other one was and international multicenter randomized double masked sham controlled phase III clinical trial for G. A secondary to AMD.
The reduction in the mean rate of <unk> growth over 12 months was 27, 38% a P value of 0.00 <unk> for this and more accumulative Graham group as compared to the corresponding Sham control group.
And 27 and eight 1% of.
A P value of 0.0051 for the some more on four milligram group as compared to the corresponding Sham control group.
These data for both dose groups were statistically significant.
These positive 12 month data are further supported by the 18 month results, which we reported in June 2020.
So Morris favorable safety profile was maintained throughout the 18 months trial.
Regarding our gene therapy programs, we continue to advance our IC 100, and IC 200 programs for ROE AARP and best one related inherited retinal diseases, respectively.
We have engaged a gene therapy, CDM Ho, who has completed GMP manufacturing for phase <unk> clinical supply of both IC 100, and IC 200.
IND, enabling toxicology studies of IC 100, our complete and are currently ongoing for IC 200.
We are planning for our phase one two clinical trial for IC, 100, including and offering site selection and other start up activities.
We plan to meet with regulatory authorities to discuss our selected doses per our first in human clinical trial before filing the IND for IC 100.
Based on this we anticipate filing the IND and initiating the clinical study and the second half of 2021.
For IC 200, and we plan to file the IND and initiate a phase one and two clinical trial and the second half of 2021.
Our many gene programs in collaboration with the University of Massachusetts Medical School and continue to progress we are optimizing the mini gene construct score on many step to 90 program and plan to select the lead contract and the second quarter of 2021.
Thank you for your time and I'll now turn the call over to per beam.
Thank you Keith Thank you all for joining and if you all this morning.
I hope that you are all well.
Looking ahead, we plan on building upon our position and GAA by exploring the potential development and Zamora and 504 other forms and stages of AMD.
We expect that our strategy will involve boat and <unk>, five and <unk> tiara, one inhibition and a complementary fashion.
And provide some further details.
Clearly.
Our lead asset Zamora.
However.
Our intention is to potentially expand the reach of the more beyond G E.
Earlier stages of AMD as well as Neovascular macular degeneration or wet AMD, and then potentially pair that development with the development of our HD already one inhibitor <unk> and 500.
We expect that our HCR <unk> inhibitor can be used to target select patients with genotype that aren't particularly at risk to develop AMD.
<unk> is a primary ex U.
That is widely expressed in the retina and.
And the arms to each tiara, one Lucas has a compelling genetic association with AMD.
<unk> expression is quite far upstream and the pathogenesis of AMB.
We anticipate these at risk patients, maybe identified and free to earlier.
Painfully prevent vision loss.
Is it more on the other hand targets C. Five.
Which is further downstream and the pathogenesis of AMD.
The hypothesis force <unk> inhibition in the context of AMD.
As Jean agnostic.
Therefore, these two assets have the potential of being complementary.
With these two assets, we may have the ability to treat specific genotype.
Or to treat AMD more broadly independent of genetics, and potentially and both earlier and later stages of AMB.
This fits nicely with our vision five Eric bio.
And not just developed products.
Build franchises.
Treating retinal diseases.
Early in 2021.
We completed a review of our HQ or <unk>, one inhibitor ICU and 500 development program.
And we believe that Ipi 500 may have the potential for less frequent dosing and then other competitive assets currently in development.
In other words.
We believe items 500 has the potential to be.
The best in class.
As a result and.
We have revised our development plans and timelines for ice and 500.
Currently we are conducting additional formulation activities and planning for cgmp manufacturing activities for ice and 500, while planning to initiate a number of preclinical safety and pharmacology studies.
Based on our current time lines and subject to successful preclinical development and cgmp manufacturing, we expect to file an IND for <unk> 500 during the second half.
Of 2022.
We look forward to keeping you updated on our progress and we continue to move our pipeline of therapeutics and gene therapy product candidates forward.
We appreciate the support and for our shareholders.
Thank you for your time.
I will now turn the call over to Dave.
Thank you Praveen and good morning, everyone.
And I'd like to highlight a few items from our press release of this morning, and provide some guidance on our expected year end cash balance and our expected cash runway.
For the quarter, our net loss totaled $25 4 million were 27 per share compared to a net loss of $17 5 million or <unk> 39 per share for Q4 2019.
This increase and net loss was driven primarily by and increase in R&D expenses associated with or is it more a clinical programs and manufacturing activities.
And third the programs and the progression of our IC 500 program during.
During the quarter, we continued our startup and recruitment activities for gathered to and continue to on manufacturing scale up.
Our net loss for 2020 totaled $84 5 million or $1 14 per share compared to a net loss of $58 9 million and $1 39 per share for 2019 again, primarily due to an increase in R&D expenses.
Turning to our expected year end cash balance and cash runway.
And we estimate our year end cash balance will range between $130 and $140 million. We also estimate that our cash will be sufficient to fund our capital expenditures and operating expenses as currently planned into 2024.
Excluding any potential approval, our sales milestones payable to <unk> ready commercialization expenses for Zamora.
These estimates are based on our current business plan, which includes the continuation of our ongoing clinical development programs for Zamora and.
The progression of our IC 100, 200 programs into the clinic and the advancement of our IC 500 development program.
These estimates also assume that we will enroll approximately 400 patients and the gather two trial.
These estimates reflect and additional expenditures related to potentially studying zamora and any other indications, resulting from the potential in licensing or acquisition of additional product candidates or technologies. The commencement of any new sponsored research programs revenue associated development that we may pursue on.
I'll now turn the call back over to Glenn. Thank you for your time.
Thank you Dave.
2021 will be another important year for volume.
All about continued ex.
<unk> to finalize enrollment.
And our gathered two trial and Q3 and also importantly, as we've talked about a lots of day to retain these patients and the trial. So thanks for your time this morning.
And the continued support we will now turn the call over to the operator, so that we can open up the low and for any questions operator.
Thank you and if you would like to ask a question. Please signal by pressing star one on your telephone keypad.
If you are using a speaker phone. Please make sure you on mute function is turned off by your signal to reach our equipment again press star one to ask a question and.
We'll pause for just a moment to allow everyone an opportunity to signal for questions.
We'll go first to Tiago <unk> with credit Suisse.
Thank you and congrats on all the progress.
I just have one on <unk> Mora, so as we're thinking about the potential sector differentiation relative to what other couple of other approaches.
And I can help us contextualize this heavy rates seen and gather one and potential baseline characteristics of the population and the role that might have influenced that somehow and.
How that could look like and gather too.
So wondering if there are any relevant protocol on that validate and ethanol changes.
And our investigators report events are healthy and then just to confirm.
And could also result, and differences between debt are one and get into on the safety.
As we're talking about C and D rates. Thanks.
And.
Thiago Thanks for the question for me and I think Thats one for you.
Sure. Thank you Glenn on Thiago. Thank you for the question, but on a lot of questions within that but a very important question, but let's just talk about the <unk>.
The first thing I'd like to share with you is why we excluded patients with CMV and other fellow alive.
It was entirely due to concern about compliance and protection of the integrity of the data.
Such a patient on whatever likelihood to return to our clinic multiple times for more than a month.
More than a year.
And multiple times per month for more than a year and <unk>.
Thought that was unreasonable, especially inevitable patients we wanted to minimize the patient dropout and protect the integrity of the data and that's the reason.
And we excluded patients with <unk> and the follow on it.
And regards to conversion to wet macular degeneration, we are starting to learn.
The risk factors there are many such risk factors, we know that and having a and b M and the fellow <unk>, maybe one of many risk factors. However, having a geographic atrophy that have a higher rate of metabolism, because it grows faster and recall that we recruited patients with extra four wheel geographic atrophy the fabs.
Just go out and geographic atrophy may also.
Theoretically a greater risk factors. So it's important to recognize as you well know the limitation of comparing results across trials when the patient populations are desktop, but it's also important to remember the magnitude of difference for instance, or the ratio of conversion between drug and.
Sam and gather one and was about four times, however, with <unk> three inhibition and Theyre reported rates of 17 times. So comparing the magnitude is also important so as we've mentioned many times before we believe that theres solid signs of support the beneficial efficacy and safety.
Profiles that we have demonstrated with C. Five inhibition to date now your question regarding gather one and gather too.
The inclusion criteria are exactly the same we are also studying patients with extra full wheel geographic atrophy, the only difference and.
And the exclusion criteria is really.
Paul is that we are continuing to follow those patients who developed and neovascular membrane for the entirety of the study we call them and gather one the Duke reading and central was not certain whether those patients could have inadequate measurement of the geographic atrophy and growth and then.
Looking back at the gather one study that confident that those patients can be accurately methods. So we will continue to follow those patients for the entirety of the study and hopefully.
If you have if that answers your question, but thank you for the question.
Thank you very much.
Yes.
We will go next to Stacy <unk> with Cowen and company.
Good morning, Thanks for taking my question, great to hear about the enrollment and retention progress I have just one for wondering if theres any reason that's more I can't be formulated and extended release formulation.
And seeing a number of different and Accenture with these technologies for wet AMD, Jamie and just curious about any progress here. Thanks.
Thank you for that question Keith do you want to.
And take that question.
Sure happy to do that thank you and great question.
No to answer your question no. There's no reason why we actually think that the properties of Zamora, which is an aptamer chemically synthesized aptamer lend itself quite well to those type of technologies and a.
Of course, we are always looking at those types of technologies in the background.
And I'll just add debt.
It's early days or focus is on getting the trial completed and hopefully.
And being able to flow.
Hello.
And with the regulatory agencies and score.
And the approval of this product that's greatly and use implementations clearly.
And extended release is a key part of lifecycle.
And think we have mentioned the lifecycle and the plan for Zamora.
We're looking at a number of things.
But more to come on that.
And the future. Thank.
Thank you for the questions.
Thanks.
We will go next to David and Theyre on Garden with Wedbush Securities.
<unk>.
And we're just a quick question from me.
I have missed it earlier, but do you attribute the enrollments and the positive turns on enrollment and a purely on the enrollment and patient recruitment or.
How is the dropout rate.
Looking at compared to your expectations in other words and it was a dropout rate better and what's your had anticipated and.
And then on price and 100.
And again, maybe I missed this but.
A little bit of a push out on the R&D filing is manufacturing and related or.
Wrapping up preclinical.
Data assets or kind of what's the cause there.
Yeah, David and its Glenn and thank you to the two questions I'll take the first one and I'll ask.
Keith will take the second one.
And as we said on the call.
Happy with the enrollment and the trial.
Trending ahead of schedule.
Early days, so as it relates to.
Drop out so it's not something we've talked about growth will be important point is.
But you also spoke about retention and.
That retention with just important and Keith total number of the initiatives there.
So we have thought about retention rates on the beginning if you remember we had to.
Deferred the start of this trial, which was a difficult decision.
Beginning with Covid and try to pick the right point, so obviously, our efforts around retention, which will minimize.
Miss visits et cetera.
And those type of metrics Jos.
Too early to report those I think you should take away from this debt.
At this point that we're happy with the progress of the trial.
Keith do you want to take the.
Second question and obviously one of it.
Sure. Thanks Glenn.
And so we had provided general guidelines initially as we complete our preclinical development for our first gene therapy asset to hopefully entered the clinic and we just wanted to be sure that we're bringing forward a plan that has a high probability of success and an area with significant unmet medical need. So we remain very excited about the collaboration with the University of Penn and and University of floor.
Arda.
And we will continue to develop the unique retinal asset.
And it's capable of simultaneously knocking down and replacing the toxic mutant gene. So as we discussed we would be wrapping.
Wrapping up the and we completed the non clinical studies and we're in the process of site.
<unk> and starting up to.
Starting up activities, we will plan to meet with the regulatory authorities to discuss the selected doses for this first in human trial before we file the IND.
And then debt that's why the <unk> now and the second half that's approached and posted the first half.
David I think this will be our first clinical trial in gene therapy. So we want to be sure that we're.
Taking all the right steps.
And doing all the right things importantly, as Keith said.
The preclinical talks work done.
And I do want to mention and congratulate our operations team on the work that on with our outside CMO on on manufacturing so the materials on payout.
But on.
I think since this will be our first on the gene therapy program I wanted to share that we're taking all the right steps.
Thank you.
At this time there are no further question and I will turn the call back to Glenn for closing remarks.
Well operator, thank you for hosting the call and again, thank you to everybody for listening today, and we look forward to continue dialog on that.
And then.
On the second quarter.
Thank you goodbye.
This does conclude today's conference we thank you for your participation.
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Okay.
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