Q4 2019 Earnings Call
[laughter].
Good day.
Fourth quarter on yearend 2019 results Conference call Today's conference is being recorded at this time.
George Kathy Galante. Please go ahead.
Okay.
But.
Good morning, welcome to I've worked last conference call, representing I've heard five today are Mr., Glenn Sblendorio, Chief Executive Officer, and President Dr., David Guy or executive Chairman.
Dr course resolved Chief Medical Officer, Mr., Dave Carroll, Chief Financial Officer, and Mr., Keith, let's be Chief operating officer.
I would like to remind you that today, we'll be making statements relating to library files future expectations regarding operational financial research and development matters, including statements regarding our expectations to use our previously announced clinical trial of the more up to the treatment of geographic atrophy as a pivotal trial I would development.
Did you Furthermore, including L. plans and expectations for second pivotal clinical trial evaluating the more for the treatment of geographic atrophy.
Hypothesis recording complement inhibition.
As a mechanism of action for the treatment of geographic atrophy, Oh projected use of cash and cash balances the timing the progress and results of clinical trials and other research and development activities.
Central utility and development potential for our product candidates.
The size of the potential markets indications l. product candidates are intended to treat.
And the potential fell business development strategy. These statements constitute forward looking statements for the purposes of the Safe Harbor provision under the private Securities Litigation Reform Act of 1995.
These statements cover many events matters that are subject to various risks that could cause actual results to differ materially from those expressed in any forward looking statements, including risks related to initiation and the progress of research and development programs and the clinical trials availability of data from these programs reliance on University club.
Operators and other third parties establishment of manufacturing capabilities expectations for regulatory matters need for additional financing in negotiation and consummation of business development transactions and other risks I refer you to our SEC filings and in particular to the risk factor section in our quarterly report on form.
10-Q filed on November 12, 2019 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 in the future. We disclaim any obligation to do so as required by law I would now like to turn the call over to blend.
Thanks, Cathy again, good morning, everyone. That's always we appreciate you joining our call. This morning, we made tremendous progress last year as we continue to build a diversified portfolio retinal diseases.
But therapeutics in June therapy.
Setting the stage for our third borrow to be a leader in developing transformative therapies to treat retinal diseases.
At the end of October last year, we achieved a major milestones as positive as a more clinical trial date geographic atrophy or G.I. secondary to age related macular degeneration War baby Andy.
These positive results.
ER physician Lezard thought it was a lead as a late stage clinical company with some more as a potential treatment option for geographic atrophy secondary to driving the potential multibillion dollar market, which there are currently no with Diego yet again proved treatments available.
I'd like to review some of the characteristics of results on this trial with you.
As reported in October 29 to individual administration of both the more two milligram and four milligram dose that the pre specified primary efficacy endpoint would statistical significance International multi center randomized double masked sham controlled clinical trial for GE.
Secondary to draw in.
Chile, 27% reduction G., a great growth over 12 month period, both the two milligrams and four milligram treatment groups.
They sort of leasing that's the guidance. We believed that this reduction in growth is not only statistically significant.
But also clinically meaningful.
Some more was well tolerated in this trial based on the potentially superior safety profile of some more observed to date, we believe that some more maybe potentially differentiate itself.
The field plus complement inhibitors.
For the treatment of GE, a secondary to draw in the elderly patients patients that we are seeking to treat.
Based on the pre specified screening trial design robust masking detailed statistical analysis with a pre specified statistical analysis plan.
Independent and mass imaging analysis before and by one of elite awaiting centers in the world for retinal imaging.
We believe that this clinical trial.
They serve as one of two pivotal trials typically requires the marketing approval.
We are leveraging our deep expertise and officials execution in retinal drug development.
The goal of brings more the patients with GE a secondary to draw it as soon as possible.
During the process of initiating the second some more pivotal trial called I see 2008, nor plenty to enrol first patient next month.
As we initiate I see 2008, we will seek to leverage our global network of top clinical trial sites for retinal diseases to assist with our planning to enroll approximately 400 patients.
Course will review the design device in 2008 in more detail in a moment.
The results from Oh, P.H. 2003 is a more pivotal trial have already been presented were planned to be presented at several prestigious retinal meetings around the world, including the Mekeel Society in San Diego.
And your Genesis oxidation and degeneration, leaving in Miami.
Be association for research and vision in ophthalmology or ARVO meeting in Baltimore.
The American society of retinal specialist.
Yes, all Russ.
And to be held in Seattle.
The National Symposium on molecular pharmacology therapeutics in Spain.
The retina 2020, new trends conference in Italy, and.
Asia Pacific Central Retinal Society meeting in China.
The safety and efficacy of more as being assessed in an ongoing phase to be randomized double masked sham controlled screening trial.
Patients with autosomal recessive.
Organic disease. This is an important inherited retinal degeneration disease.
Although autosomal recessive storgard disease is a monogenic retinal disease caused by genetic mutations.
The baby see a fourg.
Complement activation may play a role in this where disease.
20, Nigeria, we completed the enrollment of notifications in this trial.
The trial is on track.
Initial topline data expected during the second half of 2020.
Although brings more to patients is our top priority, we continue to advance our gene therapy portfolio orphan inherited retinal diseases.
Natural history studies in R&D, enabling activities for IC 100.
Which is intended to treat rhodopsin mediated 80 yorkie.
I see 200, which is intended to treat best ones related retinal diseases or both on track and we expect to identify the lead Lilly June construct for LCH Center.
By mid year.
Over the past several months, we hosted three research and development Symposium with catalyst that include Cup retinal specialists from the U.S. and around the World gene therapy scientist and shale wells, providing insight and sharing their expertise and feedback in regard towards a more into therapy programs.
We hope that these R&D supposing provided a deep understanding about programs and want to thank all the investors and analysts who tend to these leases meetings and listened to the corresponding webcast.
The symposiums are currently posted on our website than we'd like to invite you.
To participate for those that did not participate lives can listen to them online.
In November we had the privilege of announcing Dr. globe can go as our chief strategist gene therapy at I've ever bio.
Well paid brings deep to therapy expertise with over 30 years of scientific research experience in a b vectors and GE based treatments.
That's one of the world's leading gene therapy experts won't things highly distinguished career includes is major contributions to the development of the dental associated [noise].
Virus June delivering technology.
Well two is currently president of the American Society of gene and cell therapy.
He is also director of the Jorge Gene Therapy Center co director of the researchers just stupid so two for rare diseases.
Professor of microbiology because of the geological systems at the University of Massachusetts Medical School.
Our goal is to combine well things deep expertise and gene therapy in advisory capacity together with our expertise in drug development.
Retinal diseases to shape, there, but as gene therapy strategy in the coming years.
We also happy to welcome Dr. Abraham story or to the position of Chief Scientific Officer, He joined US in October.
They then leaves the company's research and preclinical gene therapy activities.
His extensive experience includes positions at Genzyme, Sanofi and most recently to Serbia therapeutics, leading multi for multiple ocular gene therapy programs.
Extremely happy I'm pleased to be working with both clumping and neighborhood.
On the financial fun.
We continued to build our financial position than simply due to successful.
Follow on public offering of our common stock in pre funded warrants, resulting in net proceeds from the company of approximately 42.6 million.
December 2019.
This transaction result in the company, finishing 2019 with approximately 126 million in cash cash equivalents.
With the addition of this capital we expect them you have sufficient cash cash equivalents to Thunder operations like capital expenditures as currently planned into the beginning of 2022.
We are currently we are continuing to explore all options for future development of potential commercialization of Zamora.
Including plans for potential partner.
We are pleased about the excitement generated by some more data and the progress energy with their programs. We are excited about towards potential to positively impact and transform the lives of the many patients with GE, a who currently do not have any treatment options available to them.
Potentially confronted with irreversible bilateral vision loss as well as the potential to bring our gene therapy to patients with warfare inherited retinal disease, who also do not have any treatment options available to them.
I'll now turn the call over the course.
Thank you Glenn and good morning, everyone.
As you had said Michael or degeneration, it's characteristically disease of the elderly.
As the leading cause of usual dolls individuals a 50 years of age or older in developed countries.
The United States, if it's estimated that approximately 11 million individuals or affected the a and b, we're going to be booked its global prevalence of approximately 270 million individuals.
Because I've been keeping life expectancies developed and developing countries. The elderly sector of the general population is expected to continue to increase and becoming decades.
While the one eight Americans was considered to be elderly 1994.
It is expected by the one in five they fall into this category like 2030.
As a and B progressed as we age generally pokerstars to either didn't know vascular or doctoral sick dry form of Andy.
For the meal basketball form of the disease or wet AMD.
Hey, good draw that topic for the mall photo receptors. These are the south that person like.
Our T cells use of the cells that support the Fulton receptors.
Awesome shipped this quarter kept motors, which would be lot supply needs to the formation of the geographic atrophy.
Currently approximately 1.5 million individuals are deposits will have geographic atrophy in the United States.
Incidence of approximately how does on 69000 patients every year, which is even slightly higher than what is your pork. That's what I know what each event for wet AMD.
Furthermore.
The last month or progression of geographic atrophy overtime is a common cause of vision ball in patients diagnosed with the web form up and.
Or generally to the anti VEGF therapy.
Indicating that the many patients regardless of whether they have to dry or what form of a and b. The final automic outcome, leading to lots of vision is geographic atrophy.
Hardly at all no it's the a or B M. A approved treatments for GA.
Which leads to bilateral irreversible loss of vision and its large group of patients.
As many pointed out we are excited about the results of our first pivotal trial for Zimura in geographic atrophy second due to a and b, indicating that the mortgage was well tolerated over 12 months.
And was able to slow down big brought up GA over the 12 month period.
In a statistically significant degree.
Following this results we immediately started the process to initiate our second pivotal trial in G.A. I see 2000 <unk>.
We did go up enrolling our first patient next month.
Recently that we are on track.
I see 2008, he's an international randomized double masked sham controlled multicenter pivotal clinical trial evaluating the safety and efficacy obviously more up to me the ground in patients with geographic atrophy country to drive a and B.
As you may recall in the first pivotal clinical trial board Nimodipine milligram and be model for me gone cohorts demonstrated a statistically significant reduction in they mean, great of GE a grow over 12 months when compared to the corresponding sham controlled cohorts with a seminar 27%.
Reduction.
And since the more up to me to ground is administered as a single intravitreal injection, whereas the move a four milligram requires to interpret told injections, we selected that being one of two milligram dose for evaluation into I see 2000, I'm a clinical trial.
Our understanding from the FDA is that regarding because they were up two milligram goes for marketing approvals purposes from a safety perspective at least 300 patients need to be treated with monthly the more up to me the ground ordered the higher dose what are the ratio of at least 12 months with a portion of these patients.
Good for 24 months.
[laughter]. Therefore, we are planning to enroll approximately 400 patients and I see 2000 and a trial.
These patients would be a randomized one to one to one into two parts.
The first cohort receiving mums administration of the moved up to me to grab for 12 months.
And a second cohort receiving Monday administration of shopping.
The pre specified primary efficacy endpoint would be the same as our first people trial and he's the me rates up change and geographic atrophy growth over 12 months measure by Fundus Autofluorescence, that's three timeframes baseline Onesix I'm htwo.
If you do see positive 12 month data from I see 2008, we plan to file for marketing approval for is the more rock hope that you had been of D.A., we'd ask D.A.M.A.
I'd Montreal, you plan to randomize patients into the more about tumor the Graham our to receive either monthly or every other month administration, obviously move up too many Graham.
All patients initially there was a lumpy administrations of Sham, we've continued to receive monkey administrations of shot.
We intend to treat on follow all patients for 24 months.
The inclusion and exclusion criteria for I see 2008 are similar to offers the most people trial.
However in the first trial when patients reported to have developed probably doesn't elastomers vision or CMV. In this study I during the trial they would exclude itself for the participation. In this study because we were concerned that fundus autofluorescence images could not be reliably evaluate that in the presence of C. N b in this study out.
After discussions with our independent Reading Center, who has your view the images from our ongoing as the motor GA trial.
That many of these images quick potentially be reliably assessed by fundus autofluorescence.
For the upcoming I see 2008 trial, you're planning to keep patients who develop CMV in this study I in the trial and there wasn't much of these efficiency a size will be included in the primary efficacy analysis as long as their fundus autofluorescence images can be reliably assessed by the mass leading cycle.
As Glenn mentioned earlier.
Met its pre specified primary endpoint in the first pivotal trial like reducing the rate of growth in patients with dry a and B you know international multicenter randomized double map sham controlled clinical trial.
You're right the reduction in the main group at rates of GE a growth over 12 months was 27.38 person with a P value of 0.0072 or does the mood or two milligram group as compared to the corresponding Sham control group.
I'm, 27.81% with a P value of 0.0051, what is the more affirming they've gone through as compared to the corresponding Sham control group.
These data for both those groups were statistically significant and based on a recent that's good guidance, we've come to them to be a clinically meaningful.
Most importantly, based on our preliminary review of the safety data to date, the more was generally well tolerated over 12 month of administration.
But just 12 month period, there were no investigated related that's a good report that the motor related adverse events no cases of Zimura related he dropped or examination no oculus suitors said, there's event no cases of doing whatever they didn't increase even talk about pressure milk isn't going to fund Midas I know discontinuations attributed to it.
Yes, you get it just anymore.
Further the investigator report that rate of quarter busbar membrane incidence appears to be lower than what has been report that infrastructure for complement inhibition in GA.
There's some recent big picture this may potentially be do demora blocking becoming cascade downstream identiv enough C five and melt blocking the cleavage of C.
We believe that potentially Supergirl safety profile, obviously more up to date could potentially differentiate anymore in the field of complement inhibitors for the treatment of GE, a and b I barely patients.
The design of our first pivotal clinical trial for is the Mora in patients with GE, a second only to draw a and B I was just a few important characteristics that differentiated from other place to be trials done into field and we believe supported to qualify as a pivotal trial.
We'd like to go over some of these important differentiating factors.
This trial was designed as it seems to be screening trial, which means that it designed and conducted by the phase three trial with all necessary phase three departments, but with a smaller number of patients.
The transmitted by design of a screening trial will likely take this study drug is education and off duty to pick a significant with a smaller sample size than trial could qualify as a pivotal clinical trial.
I was just put that earlier at the reduction of D.A. broke reach statistical significance for both of the move to many Gram dose I'm told me ground goes it we'd compared to the corresponding sampling told arms among 12.
It looks like it's important criteria.
To minimize bias in this trial.
Patients evaluating physicians I, but its body was the sponsor and independent reading center, but all boss to the treatment that the individual patients receiving throughout the trial.
It's pretty specified statistical analysis plan or Sep was used for said he's been analysis based on the pre specified criteria Inglis Sep. It goes up zimura wouldn't be statistically significantly more effective and a sham controlled.
The strength up evidence that this down to requirement of a euro point, you don't want to five one sided false positive at a rate incorporating an adjustment for multiplicity arising from comparing each dose with a sham control.
Further robust pre specified that'd be analysis was plan I'm for from which indicated the analysis results for the boss don't missing data.
[noise], what the assessment of the primary endpoint the images been evaluated by a leading independent en masse reading center.
They did views were completely masks and its visit and each visit was evaluate that independently.
As indicated earlier I was personally what a pivotal trial is ongoing and patients continue to be treated on followed until they reach the month 18 timeframes.
[noise] patients if I don't think division the independent reading sender and we have to sponsor continues to be Basque regarding that you've been group to each each individual patients randomized and expect to them en masse until the patients you each month 18.
It is important to point out that Ms trial with not designed to assess the statistical significance between individual cohorts that month 18.
And any month 18 results would be descriptive on that.
We expect to report month 18 data by the end of second quarter.
To conclude.
<unk> is a significant caused a bilateral you'll give us a bolt ons to be lost a functional vision with a major impact on the quality by an independent about elderly patients.
Although anti VEGF therapy is available for treatment of with A.M.D. No XT eight or M.A. approved treatments are currently available for geographic atrophy.
Further developmental personal geographic atrophy overtime, the common cause of vision ball the pension diagnose we'd like a and B was being treated you done type I just thought.
Indicating that regardless of whether patients have been blogs orthovisc form of a and b. The final anatomic outcome, leading to lots of vision in many patients these geographic atrophy.
Yeah, I think of treatment options for geographic atrophy represents an area of and urgent unmet medical need and a major public health concern for the expanding elderly population.
We look forward to keeping you updated regarding the progress of obviously moved up program.
Two.
Potentially help these patients I would now like to turn the call over today they.
Thank you cost and good morning, everyone I'd like to highlight a few items from our press release of this morning. It provides some guidance on your expected yearend 2020 cash balance of cash runway.
The quarter, our net loss totaled 17.5 million the 39 cents per share compared to net income of 104.1 million were $2. A 62 cents per share for Q4 2018 as Q4 2018 reflects $125 million gain on extinguishment of a royalty purchase liability payable to Novo holdings.
I get lost between 18 totaled 58.9 million or $1.39 per share compared to net income of $63.1 million 70 per share for 2018.
2018 reflects the impact of the aforementioned gain on extinguishment of a royalty purchase liability.
Turning to our expected you're in cash balancing cash runway as we previously announced our cash balance at December 31st was approximately 126 million. We now estimate our year end 2020 cash balance will range between 60 to 70 million.
We also less to make it of our available cash will be sufficient to fund our operations and capital expenditures. That's currently planned into the beginning of 2022.
Yes. Its are based on our current business plan, which includes the initiation of our IC 2008 trial and the continuation of our R&D of our R&D programs.
Of course, he's actually it's not reflect any additional expenses, resulting from the potential in licensing or acquisition of additional product candidates were technologies.
Associated down with the company may pursue.
I now turn call back over to Glenn. Thank you for your time.
Well. Thank you everybody for listening this morning, and I'm, we're focused on execution over the next few months. The first obviously is to get patients into a second pivotal trial.
Next month, and and obviously, we're looking forward to the 18 month data.
That would come in the second quarter. So it's a focus on execution working with our collaborators to.
To to move so these programs forward. So thanks again, thanks for your continued support and operator will you. Please open the lines for some questions.
Thank you if he would like to ask a Christian p. signal by pressing star one on your telephone keypad using speakerphone. Please make sure. Your mute function is turned off unfamiliar signal to each or equipment.
Can I start wanting to ask your question.
No took a first question comes from Ken Cacciatore of Cowen and company.
Hey, good morning team just a congratulations on all the progress you've been making just a few questions here first I just wondering if youve actually did thinking about it entered into any actually west partnering discussions and trying to think of non dilutive ways. You may be looking to bolster your cash position.
And then secondly, I know it's difficult to dealing you're just kind of get going here on enrollment, but can you give us some thoughts are ways of putting perspective around the timing to complete enrollment for the phase three program.
And then lastly, palaces clearly running a program as well and we understand the doing two studies versus your one given you're already have a pivotal completed but can you talk about any subtleties in differences between the two programs that you'd like to share. Thank you so much.
Yeah. Thank you Ken for those three questions I'll take the first two I'm on the partnering we had a very good J.P. Morgan and we said that opportunity to talk with a number of potential partners or we're going to continue to speak to those partners. What I was encouraged by was sort of the strength of the data obviously a trucks.
People that have interest so as you know these are and we've been saying for that we're committed to exploring partner and a the keep you updated it over the coming months, if those discussions for glass, others said I want to put on the timelines or commitments on that but your thought about a ways to further strengthen the company not only in terms of collateral.
Operation, but.
Also non dilutive financing is something we're thinking about as well.
On the timing on enrollment or let us get started first oh.
Obviously, there is always very competitive questions as to enrollment.
Just focus you know in my summary, I talked about well focus on execution I think you'll see the sand intensity once we get the trial up and going as we move forward.
If possible world, we'll update the other community, but right now that's something we want to hold a little close.
You know that we can do this based on a test track record so stay tuned on that too and I'm, sorry for not being more specific.
First do you want to take or the third question about.
Differences.
In the trial the short I'm in a yell at me I can tell you a little bit our.
Phase two trial, that's funded out in great detail during our call was a a.
Hey, I'm, a precipitous like see skewing, a screening trial, which are with certain qualifications that I went over with the pre specified statistical significance at the but is beautiful and she will want to five one sided and also was a ER doubled last child and that's what is quadruple bypass because you know the pace.
Our liquidity position the a independent reading center responds around depiction of our old mass.
Pointed out of continues to be bass.
And I think the.
The primary efficacy endpoint is very similar they're worth of D.A., losing over three time points, which we believe is a accepted by the regulatory agency and are meant to be a meaningful and a you know we looked at a as pointed out we looked at on the two minute ground because for many groundless two injections.
What didn't believe you want to start every other month from that gets goal because it's already been solved.
That is a less.
Theres less efficacy when do you do every other month, but we wanted to see after 12 months, how would that looks like for the long term two minutes for patients.
Thank you.
Thanks Kim.
Yeah.
Thank you and when I moved your next question comes from David Nierengarten of Wedbush Securities.
Good morning, this is Matt on for David.
It was I just want to get a little bit of insight on shore.
Saturday.
Personally for 12 months on the 2008 trial, you're going to has.
Option for in every other month dosing and I just wanted to get counted your thoughts on line.
To achieve from.
This cohort.
Sure insight on what you think.
You might see this work as and.
Maintenance.
And also with the 2003 trial, you mentioned that you'll have to month month 18 data by the second quarter, Iceland, what others have us analyses you make feels like hills.
We'll take a few different thanks for taking each will be presenting that.
Thank you.
Well thanks.
No.
Good course.
Thank you for the question, so again I pointed out.
You know for to be.
And just stepped up after obviously the primary endpoint is about 12.
And after that month, while we are looking to see whether it's you treat these patients for the year on a monthly basis, whether you thought you can acquire down the disease that in the second year, you may not need to treat them are frequently and they've got obviously, we know from previously published literature that what you do.
There are lots on the Gulf you wouldn't have no what efficacy and that's why we didn't do that's on the gets all but whether that would still be the case. After one year that is not known and that's what are we were going to find out to whether you want and need to continue to monthly sort of longer period of time or a 12 month would be sufficient enough that you can go to every or other months.
Treatment would you. Please repeat your second question for me we of course I have it it's about the 18 month period and all lumped concerned it's somebody I want to really stressed but was talked about in the.
In the call. So it's important to point out that.
This is the first trial. This trial was not designed not designed to assess statistical summit significance between individual cohorts at month 18, because it's something that's very important any 18 months results will be descriptive. The primary endpoint is at the 12 months and a as we also said we expect to report that data by the end of the set of course.
So compete completes the trial. The you know the emphasis is on the 12 and we'll have some descriptive analysis from the 18.
Which are really focuses on safety only [noise].
[noise] that anything else.
Does it your two questions.
Yes, all right. Thank you.
Okay, a that concludes the questions for today, a upward I'd just like to thank everybody.
For joining and look forward to speaking over the coming weeks and months. Thanks.
This concludes todays call. Thank you for your participation you may now disconnect.
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