Q1 2021 Iveric Bio Inc Earnings Call
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Okay.
Good morning, and welcome to the eye Varick bio first quarter 2021 earnings conference call. All participants will be in listen only mode should you need assistance. Please signal a conference specialist by pressing Star then zero on and telephone keypad.
After todays presentation, there will be and opportunity to ask questions.
Please note this call is being recorded.
I would now like to turn the conference over to Kathy Galante Senior Vice President Investor Relations. Please go ahead.
Good morning, and welcome to <unk> Conference call, representing high Barrick bio today, and Glenn and Oreo Chief Executive Officer for being Davao, President, David Carroll, Chief Financial Officer Double decide Chief Development Officer, Abraham <unk>, Chief Scientific Officer, and Keith Westby Chief <unk>.
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 some more for the treatment of geographic.
Atrophy secondary to age related macular degeneration, our expectations to use gather one our previously announced clinical trial of Nomura for the treatment of G. A secondary to AMD at the phase III clinical trial, our development and regulatory strategy for us and Lora and our other product candidates, including our.
And for additional indications for which we may pursue the development of tomorrow and I see 500, our hypothesis regarding complement inhibition and a true one inhibition and mechanism of actions for the treatment of G E and potentially other stages of a M D. Our projected use of cash and cash balances the timing progress and <unk>.
<unk> of clinical trials, and other research and development activity and regulatory submissions for potential utility and development potential of our product candidates and the potential for our business development strategy and our personnel Advisory Committee members and human capital resources. These statements constitute forward looking statements for the purpose.
And of the Safe Harbor provision under the private Securities Litigation Reform Act of 1095. 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 of the progress of research and development programs and clinical trials, including enrollment and retention and clinical trials availability of data from these programs reliance on contract.
Development and manufacturing organization University collaborators and other third parties establishment.
Establishment of manufacturing capabilities expectations for regulatory matters developments from our competitors and the marketplace for our products need for additional financing and negotiation and consummation of business development transactions and other risks I refer you to our SEC filings and in particular to the risk factors include.
And our annual report on form 10-K filed on March <unk> 2021 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.
And 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 Glenn.
Thanks, Kathy and good morning, everybody.
Thank you for joining us for our first quarter conference call.
The first quarter of 2021 has been very productive for us and our generic bio I.
I am pleased to share that we are excited to be executing and moving towards completing enrollment and to gather to clinical trial. Our second phase III clinical trial for Zamora and novel complement C. Five inhibitor for the treatment of geographic atrophy, Virginia.
Secondary to age related macular degeneration.
We expect to complete this trial and the third quarter of this year.
To date, both recruitment and retention of patients and gather two are exceeding our expectations were.
We are on track for initial topline data from Gaba too to be available approximately one year. After the enrollment of the last patient and the gathered two clinical trial.
Of course, we have to add some time for database closure and analysis of the initial top line data.
If the 12 month results from Diablo III or positive.
<unk> planned to file applications with the U S F D E.
And the European Medicines agency for marketing approval of Zamora for JA.
And also wanted to mention some organizational changes that further strengthen our management team I am thrilled to announce that Dr. Praveen Dougal, who has been promoted to president effective may one for.
<unk> has played a critical role and our execution.
Our strategy.
But the key part of the executive management team, leading the company's strategy and I want to also congratulate Kathy galante on her well deserved promotion to senior Vice President Investor Relations and <unk>.
Look forward to continuing to work with both of them at this exciting time and our company.
Finally, I wanted to acknowledge David Guyer my friend.
And who will be stepping down from our board after 14 years for his leadership.
Co founder and executive Chairman.
Sincerely thank David for his hard work and significant contributions to the company.
We wish David well has rejoined SP health investors as a venture partner.
I would now like to turn the call over to Keith who will review enrollment and retention for gather two and update you on our gene therapy pipeline and orphan inherited retinal diseases.
Following Keith per beam will discuss our strategy and AMD and the formation of our gene therapy inherited retinal disease and scientific Advisory Committee.
Keith.
Thank you Glenn and good morning, everyone. It's.
As Glenn mentioned.
Our main priority is to aggressively drive patient recruitment and retention and the gather two clinical trial.
And the enthusiasm resiliency and dedication of patients physicians and their staff are exceeding our expectations.
With the reduction and the mean rate of <unk> growth over 12 months at 27.38% a P value of 0.0072 for there is it more of two milligram group as compared to the corresponding Sham control group and the gather one clinical trial.
We believe many principal investigators are enthusiastic about enrolling patients for the gather two clinical trial levered.
Leveraging the quality of the positive gather one results to maximize both patient recruitment and retention for gather too.
These positive 12 month data are further supported by positive 18 months efficacy results and a favorable safety profile that was maintained throughout the 18 months trial.
Further we believe that the early and continuous treatment effect demonstrated and gather one is a key motivator for patient retention and gather too.
In total we are planning to enroll approximately 400 patients and the gather two clinical trial.
We continue to be on track and look forward to completing enrollment and gather two and the third quarter of this year.
Our phase <unk> screening clinical trial of Zamora and for the treatment of autosomal recessive <unk> disease.
Referred to as the Star trial is ongoing with the goal of enrolling approximately 120 patients and results from this clinical trial are expected. After the 12 months initial topline data from gatherer too.
There are currently no therapies approved for <unk> disease, and either the U S or the European Union.
Turning to our gene therapy programs, we are excited about the progress and the development of our product candidate for the treatment of best one related inherited retinal diseases or IRR DS IC 200.
We are completing a toxicology study and the naturally occurring canine model of best disease. As a reminder, we have data demonstrating long term rescue and this model following a single sub retinal injection.
We are on track to release, the recently manufactured GMP batch of IC 200, and preparation for the planned IND filing and plan to move IC 200 into a phase one two clinical trial and the second half of 2021.
We are not aware of any ongoing competitive programs for best run related IR DS and believe IC 200 has the potential to be both first and best in class.
Regarding IC 100, our product candidate for the treatment of rhodopsin mediated autosomal dominant retinitis Pigmentosa. The company continues to evaluate the results from its preclinical toxicology studies.
And our preclinical efficacy and toxicology study and a naturally occurring canine model and ROE a DRP.
Efficacy was demonstrated at all three doses tested.
We also tested the same three doses and a GOP toxicology study in non human primates.
Ocular inflammation on clinical exam was observed and the high dose group and canine and to varying degrees at different dosing levels tested in non human primates.
Due to the different findings and two different species and our high commitment to the safety of our patients. We are planning to discuss the design of our planned first in human clinical trial with regulators prior to submitting an IND.
We now believe that IC 100, and will likely be delayed from entering into a phase one two clinical trial this year.
We are thrilled about the progress of our many gene programs and collaboration with the University of Massachusetts Medical School and we're pleased to report that we have recently identified a lead construct for leber congenital amaurosis type 10 for LCA 10, mini Sep 290 program.
And currently considering development plans for this program.
Also during the second quarter, we expect to obtain additional results from our Star Guard disease. Many ABC for program and we expect to obtain preliminary results from our <unk> related IR day program during the second half of this year we.
We will continue to keep you updated on our many gene programs.
For your time I will now turn the call over to provision.
Thank you Kim.
Thank you all for joining the call this morning.
I hope that you're all well.
A key goal of ours is to expand and advance our footprint and multiple stages and types of AMD.
And we intend to execute a development strategy that will involve both us and Lora and IC and 500, and a complementary fashion to impact multiple for arms and stages of AMD.
I would like to let everyone know that we are planning to host a dry AMD virtual symposium for investors and analysts on Friday June 18th from 10 am to noon eastern time.
The event will include presentations and discussions with retinal specialists and key opinion leaders and Zamora and IC 500, and the dry AMD landscape.
Guest speakers will include Dr. Frank holds University a bond.
Dr. Peter Kaiser Cleveland Clinic, Learner College of Medicine, KOL Eye Institute.
Dr Arshad commodity.
The error I associates and University of Nevada Reno Dr.
Doctor and not loans.
From a faculty of medicine at Tel Aviv University, and Tel Aviv Medical Center.
Dr. Charles Wykoff.
Retina consultants of Texas, and blends and I and Institute Houston Methodist Hospital, and Dr. Trent Woodruff and University of Queensland.
We hope you will be able to join us at our virtual symposium on June 18th.
Please reach out to Kathy if you have any questions.
Earnings for IC 500.
We announced in March 2021.
We revised our development plans for IC and 500 to include the investigation of multiple dosing schedules for our product candidates and.
April 2021, we commenced our first preclinical.
Tolerability study and we're currently planning additional preclinical studies, including pharmacokinetic and target engagement studies.
Formulation and optimization and other manufacturing activities are also ongoing.
We continue to remain excited about the development potential of IC 500, and expect to submit an IND to the FDA for IC 500, and G. A secondary to AMD and the second half of 2022.
As a reminder.
We believe IC 500 has the potential to be best in class.
As it inhibits H tiara, one both intra and extra seven lately.
As Keith mentioned.
While IC 100 is delayed.
Our IC 200, and many gene programs are progressing well.
We're excited to announce today.
In addition to our visionary stirring and imaging advisory committees for.
<unk>.
And Ive, Eric Bio gene therapy inherited retinal disease Scientific Advisory Committee, which.
Which brings together and.
We know and group of thought leaders.
<unk> extensive clinical experience and a deep understanding of gene therapy to treat inherited retinal diseases.
Advisory Committee will work closely with senior management as we advance our gene therapy programs.
Advisory Committee members include Dr. Elias for both <unk> and co.
Cole Eye Institute, Cleveland Clinic, Learner College of Medicine, Dr. Andreas Lauer, Casey Eye Institute University of Oregon.
Dr. Bart low.
And <unk> University Hospital and.
And children's hospital of Philadelphia Dr.
Dr Mark and they've seen Casey Eye Institute University of Oregon.
And Doctor Eleanora Lab, Duke Reading Center, Duke University Medical Center.
We remain committed to our mission statement.
The developed.
Formative therapies and retinal diseases.
We look forward to keeping you updated on our progress and the months to come.
Thank you for your time.
I will now turn the call over to Dave.
Thank you Praveen and good morning, everyone I'd like to highlight a few items from our press release of this morning and update our expected year end cash balance and confirm our expected cash runway for the quarter. Our net loss totaled $26 8 million with 29 per share compared to a net loss of $15 one.
$1 million or 28 per share for Q1 and 2020.
This increase and net loss was driven primarily by increases in both R&D and G&A expenses and the Q1 and 2020 impact of a favorable settlement of a state corporate tax audit.
R&D expenses increased due to the progression of our <unk> clinical programs and the progression of our preclinical gene therapy and IC for 100 programs.
During the quarter, we continued our recruitment and retention activities for gathered two and continued as a more and manufacturing scale up activities.
G&A expenses increased primarily due to an increase and legal costs associated with our ongoing litigation.
Turning to our expected year end cash balance and cash runway.
As Keith and Glenn have described we are aggressively driving gathered to recruitment and retention. We're also investing and the manufacturers and more drug substance and drug product, including required starting materials as we aim to scale up and validate the manufacturing process.
As expected both of these activities have impacted our Q1 cash burn however, we expect our quarterly cash burn to decrease as the year progresses.
We now expect our year end cash balance will range between 125 and $135 million.
And our long term cash forecast is unchanged, we 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> for any commercialization expenses for Zamora.
These estimates are based on our current business plan, which includes the continuation of our ongoing clinical development programs with Nomura the progression of our IC 100, 200 programs into the clinic and advancement of our IC 500 development program.
These estimates also assume that we will enroll approximately 400 patients and the <unk> trial.
These estimates do not reflect and additional expenditures related to potentially studying zamora and other indications, resulting from the potential in licensing or acquisition of additional product candidates or technologies, where commencement of any new sponsored research programs, where any associated development that we may pursue.
I'll now turn the call back over to Glenn. Thank you for your time.
Well, thanks, Dave and thank you everyone for your time this morning, and your continued support I'll now ask the operator.
If you can open up for the line for questions. Thank you.
We will now begin the question and answer session.
And ask a question you May press Star then one on your telephone keypad.
If you are using a speakerphone please pick up your handset before pressing the keys.
And at any time for your question has been addressed and you would like to withdraw your question. Please press Star then two.
At this time, we will pause momentarily to assemble our roster.
The first question.
Comes from.
Thiago vote with credit Suisse. Please go ahead.
Hey, Thanks for taking the question. So just a quick confirmation here, so and ambition to get to one data is the <unk>.
12 months together two data sufficient to support the filing from a safety or exposure perspective, or do you need any additional follow up there.
And another follow up on the broader competitive landscape and Jay So again theres a lot of focus on Q3 and true five and ambition because there is clinical data for for those approaches.
But curious about your thoughts on longer term potential approaches like gene therapies targeting <unk>, 15, 59, or with covenant covenant and sector age proteins and other emerging therapies out there what are some frozen cause thank you.
Thiago Thank you Glenn and thank you for the question. So I'll take the first question.
<unk>.
The other one trial and all.
Loss per game to take the second question, Yes, we believe the combination of gathered volume gathered two will be sufficient for a regulatory filing.
As we have said in the past and similar disclosures and pad.
Numerous conversations and formal conversations with the FDA and it's given us comfort and the.
Yeah.
And our strategy.
With the two trials, so yes, we're comfortable and as I said today.
Completion of Gaba true our intent is to.
<unk> analyzed top level data and if it's sufficient and we would file for regulatory approval.
For me.
Thank you Glenn and good morning, Thiago and thank you for the question.
What I would say is where we're delighted that there are so many companies that are looking at the complement pathway because it validates the target.
We remain quite convinced that our target has a great deal of scientific backing and scientific evidence and in fact, we feel that our results.
Reflect what would be consistent with previously known science and previously published science and <unk>.
Terms of gene therapy, I think it's important to understand that.
And that there is a different challenge to gene therapy for chronic disease as opposed to an orphan disease.
Gene therapy is very exciting, but again and again the challenges remain for a chronic disease, which may be more different than orphan diseases. We've seen so we support all of these companies were.
Delighted that there are more people that are in the complement pathway as I say because it validates the target. However, we remain convinced.
Net science stance with our target selection and the inhibition of <unk> five.
Got it understood all right. Thank you very much.
The next question comes from Stacy <unk> with Cowen. Please go ahead.
Yes.
Good morning, Thanks for taking my questions and congratulations on the continued progress and this year and.
And so so first I'm wondering if you guys would be willing to provide any additional commentary on retention on enrollment and for instance have you seen and even greater acceleration and the past few months for should we just expect kind of a steady addition of patients and.
And then my second question is another competitive one as they are.
Approach Apollo with data read out and can you remind us the main differences and study design and specifically the reasoning behind the key and Christian criteria for extra for the LDS and thank you.
And with phase III, thanks for the compliment on execution and thanks for the questions.
So on the first the retention.
And the updates on the color I'll ask Keith to do that and on the second question.
Perfect and will take that.
Thanks Glenn.
Thanks Stacy.
Yes.
And the enrollment was was good from the onset. So it's been continued to be quite we're quite happy with that and.
And I think Theres, a number of things that really drive that money is the results from the gather one which were published the 12 month results as well as well as the 18 month follow on results from gather one.
Which really helped drive the enrollment and as well as the retention and gather too. So we're quite pleased with that and very much on track for the third quarter to complete that.
Yes, thanks for the only thing I'd add in terms of color I think it goes back to what we said in the past on the preparation and this COVID-19 environment.
And Keith and his team work very closely not only with the sites, but took it into account also the impact potentially on patients and how do we keep them safe.
So I think that early planning and.
If you recall, we did delay the trial in early 2020 until we can better understand what was happening with the pandemic and.
And that was about a three or four month delay and that allowed us really to take a step back analyze what was happening with the docs.
And well and I think we're seeing the fruits of that planning and the execution now and as you know we said at the end of the year that we did move up the completion date from.
The end of the year or closer to fourth quarter, two and for third quarter. So.
Maybe a little bit more color for you.
At this point in time.
So for me and ill give you the second question.
Yes, Thank you Glen and Stacy. Thank you for your question. So what would have been known for quite a long time more than half essentially is that theres, a very stereotypical way that geographic atrophy advances and what it does is that it advances fairly rapidly and and circumferential fashion and by rapidly.
And a matter of several months and and you can see this from the <unk> studies that Genentech has published in regards to their lamp alleging that program.
And then once that advances relatively rapidly circumferential once the full gets involved the geographic atrophy tends to slow down.
Once the soldiers involved so.
The bottom line is that there are lots and lots of patients out there with excellent visual acuity, the visual acuity and maybe refractive or for 2020, but their visually dysfunctional because of the geographic atrophy that they have this extra coal. If you will that may not allow them to function. They may not may not be able to see a straight line.
If you and architect or certainly from an engineer.
They may not be able to finish reading, a spreadsheet or sentiment sicker and accounting for a lawyer. So there are lots of those patients out there and the logic behind the selection of extra for wheel geographic atrophy.
And is not only to address this enormous unmet need but also to be able to note that if we're able to slow down but fastest growing geographic atrophy, we've met a higher bar of scrutiny, and then slowing down and a slower growing geographic atrophy.
And finally, what I'd also say is that the ability to protect the fovea from being affected by geographic atrophy is terribly valuable and we believe that we'll be able to demonstrate that as well. So for all of those reasons important to understand why we selected this patient population and also as you've noticed it's important to note that our pace.
And population.
It is quite different than the appellate patient population and should be regarded that way as well when you analyze the data and thank you for your question.
Thank you.
The next question comes from David near and Garden with Wedbush Securities. Please go ahead.
Okay.
Mr. Nir and Garden. Your line is open. Please go ahead with your question or perhaps two.
Thanks.
The old mute button.
And I'll ask gene therapy questions today on those two programs.
Or are there any differences and.
Manufacturing or.
And anything else that would lead to differences and the animal outcomes or you know.
Obviously, the the gene being inserted is different.
Do you think it's isolated to maybe inflammation and caused by the Jeep product or again are there differences and manufacturing or other things that might.
It might be leading to those cases of inflammation and the animals. Thanks.
David Thank you for your question Keith.
Sure. Thanks, David.
Very good question, so and in terms of the manufacturing we feel confident that we did produce.
Good quality materials for these.
And it's a similar manufacturing process for for both.
No.
Looking at the data, we see the discrepancy and the two different species. So we just thought it would be prudent to take a step back and and really analyze that data and have some discussions with regulators prior to initiating the IND filing for the program.
That's for IC 100 for IC 200, we are very much on track.
Completed the manufacturing of the GMP batch for the clinical trial, and that's and the process of being released now.
And could.
Could you remind us on the rhodopsin protein being.
Delivered by the factor or is it what.
Species is that coming from and in those studies and becoming the human rhodopsin protein or a different source.
It is it's a great question.
And is the human rhodopsin, that's being used for for both.
And non human Primate studies as well as the canine.
As the same material, we plan to move forward into a clinical trial.
Got it thank you.
Yes.
There are no other questions I would like to turn the conference back over to Glen and <unk> <unk> for any closing remarks.
Thank you operator, and so we're very happy to have the opportunity to update you. This morning about our continued execution and progress and.
And I'd like to wish everybody and good morning, and thanks for listening.
Hi.
The conference has now concluded. Thank you for attending today's presentation you may now disconnect.
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Okay.
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