Q3 2020 Clearside Biomedical Inc Earnings Call
Welcome to the Clearside Biomedical third quarter 2020 financial results Conference call.
Operator: Welcome to the Clearside Biomedical 3rd Quarter 2020 Financial Results Conference Call. At this time, all participants are in a listen-only mode.
At this time, all participants are in listen only mode.
Operator: After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone keypad. If you require any further assistance, during the conference, please press star then zero, and your operator will be happy to assist you. I would now like to hand the conference over to Jenny Kobin, Clearside Investor Relations. Thank you, and please go ahead, madam.
After the speaker's presentation, there will be a question answer session.
Last question during the session you will need to press star one on your telephone keypad.
If you require any further assistance.
During the conference. Please press Star then zero.
I'd be happy to assist you.
I would now one thing in the conference over to Jenny Kobin Keysight Investor Relations. Thank you. Please go ahead Madam.
Good afternoon, everyone and thank you for joining us on the call today.
Jenny R. Kobin: Good afternoon, everyone, and thank you for joining us on the call today. Before we begin, I would like to remind you that during today's call, we will be making certain forward-looking statements. Various remarks that we make during this call about the company's future expectations, plans, and prospects constitute forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. However, actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the risk factors section of our annual report on Form 10-K for the year ended December 31, 2019, our quarterly report on Form 10-Q for the quarter ended In addition, any forward-looking statements represent our views 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 in the future, we specifically disclaim any obligation to do so, even if our views change.
Before we begin I would like to remind you that during today's call, we will be making certain forward looking statements. Various remarks that we make during this call about the company's future expectations plans and prospects constitute forward looking statements.
Because of the private Securities Litigation Reform Act of 1995 actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed in the risk factor section of our annual report on form 10-K for the year ended December 31st 2019, our quarterly report on form 10-Q.
For the quarter ended September Thirtyth, 2020, and our other SEC filings available on our website.
In addition, any forward looking statements represent our views 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 in the future. We specifically disclaim any obligation to do so even if our views change.
George M. Lasezkay: On today's call, we have George Lasezkay, our Chief Executive Officer, Dr. Thomas Chula, our Chief Medical Officer and Chief Development Officer, and Charlie Deignan, our Chief Financial Officer. After our formal remarks, we will open the call for your questions. I would now like to turn the call over to George. Thank you, Jenny.
On today's call, we have George with EFI, Our Chief Executive Officer, Dr., Thomas Chong, Our Chief Medical Officer, and Chief Development Officer, and Charlie Stagnant, our Chief Financial Officer.
After our formal remarks, we will open the call for your questions I would now like to turn the call over to George.
Thank you Jenny good afternoon, and thank you for joining us on the call today.
George M. Lasezkay: Good afternoon, and thank you for joining us on the call today. The third quarter of 2020 was very productive for Clearside both operationally and clinically as we had numerous positive advances in our pipeline. As we announced on our last quarterly call, we secured acceptance of our investigational new drug application for our lead development asset, CLS-AX, our proprietary suspension of Exudinib for supracaroidal injection. With this IND, we plan to initiate our first clinical trial with CLS-AX in wet AMD patients. I'm extremely proud of the cross-functional team that's worked so diligently to take Exsidentive from just a concept a little over one year ago to our expected initiation of a Phase 1, 2A clinical trial by the end of this year. As Tom will discuss in his remarks, we are eager to advance Exsitinib into the clinic as we believe its intrinsic tyrosine kinase inhibitor characteristics can be further leverage In preclinical studies, CLSA-X, delivered suprachoroidally, was observed to be well-tolerated and showed significant tissue concentrations over time. These characteristics, if demonstrated clinically, may support suprachoroidal exsitinib's potential to reduce the profound treatment burden for patients suffering from wet AMD.
The third quarter of 2020 was very productive for Clearsign, both operationally and clinically yes, we had numerous positive advancements to our pipeline.
As we announced on our last quarterly call, we secured acceptance of our investigational new drug application for our lead development asset CLS <unk>, our proprietary suspension of accident supercritical injection.
With this R&D, we plan to initiate our first clinical trial with CLS reacts in wet AMD patients.
Extremely proud of the cross functional team. That's worked so diligently to take six isn't just a concept a little over one year ago to our expected initiation of let's say one two way clinical trial by the end of this year.
As Tom will discuss in his remarks, we are eager to advance it into the clinic as we believe its intrinsic tyrosine kinase inhibitor characteristics can be further leverage through our SCS micro injector.
In preclinical studies CLS A.X. deliberate Super currently was observed to be well tolerated and showed significant tissue concentrations over time.
These characteristics you have demonstrated clinically may support Super choroidal accident, its potential to reduce the profound treatment burden for patients suffering from wedding Andy.
Our phase one two way clinical trial for C.L.S.A.X. is expected to begin by the end of this year and.
George M. Lasezkay: Our Phase 1-2a clinical trial for CLS-AX is expected to begin by the end of this year, and we anticipate reporting initial data from the first cohort in mid-2021. In addition, we continue to progress our internal preclinical work to expand our pipeline. One of our focus areas is a non-viral vector gene therapy program that we refer to as our therapeutic biofactor. We are also working on a development program utilizing suprachoroidal administration of an integrin inhibitor small molecule suspension, initially focused on diabetic macular edema. Tom will elaborate on these early stage programs shortly. For Xypyr, our new contract manufacturer is completing the technology transfer and working diligently to prepare to manufacture the first Xypyr drug product GMP batch.
And we anticipate reporting initial data from the first cohort in mid 2021.
In addition, we continue to progress our internal preclinical work to expand our pipeline.
One of our focus areas is on not a non viral vector gene therapy program that we referred to as our therapeutic fire factory.
We're also working on a development program utilizing supercritical administration of an immigrant inhibitor small molecule suspension.
Initially focused on diabetic macular edema.
Tom will elaborate on these early stage program shortly.
[noise] reside here, our new contract manufacturer is completing the technology transfer and working diligently to prepare to manufacture decipher drug clotted GMP batches.
Once these batches are made we expect that we will be able to generate the required stability data for our new drug application resubmission to the FDA.
George M. Lasezkay: Once these batches are made, we expect that we will be able to generate the required stability data for our new drug application resubmission to the FDA. The cooperation and productivity of our new CMO, working together with the Clearside team, have been exceptional, and I'm very pleased to report that we remain on track with our expectation to resubmit our NDA in the first half of 2020. 2021, excuse me, and we expect the FDA will review the NDA within six months of the resubmission date.
The cooperation and productivity of our new CMO working together with the Clearsign team has been exceptional and I'm very pleased to report that we remain on track with our expectation to resubmit, our India in the first half of 2022.
2021 excuse me.
We expect the FDA will review the N.D.A. within six months of the Resubmission date.
We are excited by the progress made this past quarter by or development partners using our S. Micro injector deliver their drug candidates into the Supercross and space.
George M. Lasezkay: We are excited by the progress made this past quarter by our development partners using our SES microinjector to deliver their drug candidates into the supracarotid state. Regenexx Myo has enrolled and dosed multiple patients in its Phase II clinical trial to evaluate the supercoronal delivery of RGX-314, an adeno-associated virus gene therapy, using our SES microinjector for the treatment of wet AMD. Based on our licensing agreement with Regenexx Bio, this progress triggered a milestone payment to us in the third quarter. Regenexx also announced that it received IND clearance by the FDA to evaluate the supercoroidal delivery of RGX-314 in patients with diabetic retinopathy. This trial is now active, and Regenexx Bio expects to begin enrolling patients with diabetic retinopathy by the end of this year. Meanwhile, our oncology licensing partner, Aura Bioscience, announced that they have dosed the first patient in their Phase 2 clinical trial evaluating the safety and efficacy of supracaroidal administration of AU011 as a potential first-line treatment for patients with primary caroidal melanoma.
Rejects bio has enrolled and dosed multiple patients in its phase two clinical trial to evaluate the supercritical delivered.
Our GXT Threeq 14, and had no associated virus gene therapy using.
Using our S U S micro injector for the treatment of wedding Andy.
Based on our licensing agreement with rejects filed this progress triggered a milestone payment to us in the third quarter.
Rejects bio also announced that they received R&D clearance by the FDA to evaluate supercritical delivery of RG X 314 in patients with diabetic retinopathy. This trial is now active energenic bio expects to begin enrolling patients with diabetic retinopathy by the end of this year.
Our own college in licensing partner or bio Sciences announced they have dosed the first patient in their phase two clinical trial evaluating the safety and efficacy of Suprachoroidal administration of a use 011, that's a potential first line treatment for patients with primary choroidal melanoma.
The initiation of these clinical trials represent major milestones for us as they are the first to partner programs to utilize our S. U S micro injector as the motive administration for the partners therapeutic products.
We believe we are the partner of choice for accessing the supercritical space as our SCS micro injector has been used in multiple clinical trials with consistent safety and reliability.
George M. Lasezkay: The initiations of these clinical trials represent major milestones for us as they are the first partnered programs to utilize our SES microinjector as the mode of administration for the partner's therapeutic product. We believe we are the partner of choice for accessing the supracaroidal space as our SCS microinjector has been used in multiple clinical trials with consistent safety and reliability. As such, we continue to expand our intellectual property portfolio in the U.S. and in Europe.
As such we continue to expand our intellectual property portfolio in the U.S. and in Europe.
We now have 21 granted U.S. patents and 20 European patents, providing extensive coverage of our SCS micro injector and its use administration of any drug into the suprachoroidal space by injection as well as coverage for specific supercar it'll product candidates.
We are committed to extending our global patent estate as we continue to expand our internal product pipeline and increased patient access to innovative therapies through our supercross injection platform.
George M. Lasezkay: We now have 21 granted U.S. patents and 20 European patents providing extensive coverage of our SES microinjector and its use, administration of any drug into the supracaroidal space by injection, as well as coverage for specific supracaroidal product candidates. We are committed to extending our global patent estate as we continue to expand our internal product pipeline and increase patient access to innovative therapies through our supercorroidal injection platform. With that, I will now ask Dr. Tom Chula, our Chief Medical Officer and Chief Development Officer, to elaborate on our research and clinical development programs.
With that I will now ask Dr., Charles <unk>, Dr., John Ciulla, our Chief Medical Officer, and Chief Development Officer to elaborate on our research and clinical development programs Huh.
Thank you George we've made significant advances in our pipeline over the last several months and I'm pleased to share. These updates with you today.
As George mentioned, our clinical development team is on track to initiate our CLS X days, one to a clinical trial by the end of this year and patients with let Andy.
Our outside clinical research organization has been chosen we're working with them to prepare the trial sites for next season.
As a reminder, the phase one two trial will be an open label dose escalation trial to assess the safety and Tolerability of a single dose, let's see let's say I could administered through the Suprachoroidal injection procedure.
Dr. Thomas Chula: Thank you, George. We've made significant advances in our pipeline over the last several months, and I'm pleased to share these updates with you today. As George mentioned, our clinical development team is on track to initiate our CLS-AX Phase 1-2A clinical trial by the end of this year in patients with wet AMD. Our outside clinical research organization has been chosen, and we are working with them to prepare the trial sites for initiation. As a reminder, the Phase 1-2A trial will be an open-labeled dose-escalation trial to assess the safety and tolerability of a single dose of CLSA-X administered through the supracortial injection procedure. Eligible patients are those who demonstrate stable visual acuity following two or more previous injections with Fliverset, an anti-VEGF agent.
Eligible patients had though to demonstrate stable visual acuity falling two or more previous injections with an.
An anti VEGF agent.
As a tyrosine kinase inhibitor that inhibits that different sectors, one two and three extending its hands that Jeff inhibition could be more effective than current focus anti Jeff Hey, inhibition, and there's already independent literature, demonstrating its potential efficacy in preclinical models of corneal retinal and colorectal cancer Genesis.
Also in preclinical models and that's been shown to be more potent and have better ocular biocompatibility than other T.I.s, suggesting the potential for efficacy and safety advantages.
And we believe that delivery of the agency Suprachoroidal injection not only has the potential to leverage the attributes of exited but may also allow for less frequent dosing and therefore, a lower treatment burden for patients, which addresses a large unmet need.
Because this is a first in human trial. Our primary objective is to assess the safety and Tolerability us you'll see us and these let him be patients.
We will also evaluate secondary metrics, including pharmacokinetics visual function ocular anatomy and the need for additional treatment with Intravitreal aflibercept.
Dr. Thomas Chula: As a tyrosine kinase inhibitor that inhibits VEGF receptors 1, 2, and 3, excitinib's pan-VEGF inhibition could be more effective than current-focused anti-VEGF-A inhibition, and there is already independent literature demonstrating its potential efficacy in preclinical models of corneal, retinal, and choroidal angiogenesis. Also, in preclinical models, it has been shown to And we believe that delivery of the agent via supracortial injection not only has the potential to leverage the attributes of excitinib but may also allow for less frequent dosing and therefore a lower treatment burden for patients, which addresses a large unmet need. Because this is a first-in-human trial, our primary objective is to assess the safety and tolerability of CLSA-X in these wet AMD patients. We will also evaluate secondary metrics, including pharmacokinetics, visual function, oculoanatomy, and the need for additional treatment with intravitreal effluvircepts. The trial of approximately 15 patients in three cohorts will include a total of five patient visits. With the timeline for each cohort at around 6 months, we expect initial safety data from the first cohort in mid-2021.
The trial of approximately 15 patients in three cohorts will include a total of five patient visits with the timelines for each cohort at around six months, we expect initial safety data from the first cohort and mid 2021.
In addition to see a lot of PX, we remain dedicated to expanding our internal pipeline. As noted previously we have been interested in Super quarterly administration of gene therapy recently published preclinical paper for me. We're now the ocular gene therapy group at the University of Iowa demonstrate that Super corridor, where we it's a viable route.
He'd be mediated retinal transduction and they concluded that further investigation for potential human gene therapy is warranted.
We are pursuing a therapeutic bio factory approach to the delivery of gene therapy, Thier, DNA nanoparticles and our preclinical research has demonstrated several promising characteristics.
First supercritical administration of DNA nanoparticles has yielded similar marketing activity compared to sub retinal administration.
Second we have observed that supercritical demonstration of DNA nanoparticles containing therapeutic transgene is better tolerated then went into Dan went deliberate individually.
Third we have seen preliminary signs of protein expression for the duration of the preclinical studies.
And finally, O.C.T. imaging demonstrates opening up a suprachoroidal space posted really all the way to the optic nerve.
Supports the potential to address macular disorders. In addition to peripheral retinal disorders.
We are excited about the potential of this platform using our S. Micro injector for both a bio factory in native gene replacement approach.
I'm also encouraged by our integrated inhibitor program as interruptions represent a novel target with limited competition.
Integrates a multifunctional cell adhesion molecule that regulate critical cell processing, such as it heesen migration with duration invasion survival in Aptoses.
Dr. Thomas Chula: In addition to CLSAx, we remain dedicated to expanding our internal pipeline. As noted previously, we have been interested in supracortical administration of gene therapy. Recently, a published preclinical paper from the renowned Ocular Gene Therapy Group at the University of Iowa demonstrated that supercortical delivery is a viable route for AAV-mediated retinal transduction, and they concluded that further investigation for potential human gene therapy is warranted. We are pursuing a therapeutic biofactory approach to the delivery of gene therapy via DNA nanoparticles.
Additionally, they play a critical role in Pathlogic process, he started inflammation yesterday Genesis and fibrosis.
We believe that Super called Liberty been integral inhibitor suspension me provide targeting compartmentalization and durability advantages over topical intravitreal delivery.
What weve observed in other preclinical studies of small molecule for sanctions like trying some alone and accident.
Therefore, we have been running a series of preclinical studies with her and her inhibitors ascension to assess the articular tolerability distribution and pharmacokinetics.
We expect our first set of data from these preclinical studies in the first half of next year.
Ultimately, we hope to address the pathlogic processes in diabetic macular edema and macular degeneration.
As George mentioned, we're very pleased with the progress made by our development partners over the last several months last week, our partner Genex Bio analysis. It is enrolled and dosed multiple patients and its phase two gene therapy clinical trial for the treatment of what AOMT, you utilizing Archie X 314.
Dr. Thomas Chula: And our preclinical research has demonstrated several promising characteristics. First, supracortial administration of DNA nanoparticles has yielded similar marker gene activity compared to subretinal administration. Second, we have observed that supercortical administration of DNA nanoparticles containing therapeutic transgenes is better tolerated than when delivered individually. Third, we have seen preliminary signs of protein expression for the duration of the preclinical study. And finally, OCT imaging demonstrates opening of the supracortial space posteriorly all the way to the optic nerve, which supports the potential to address macular disorders in addition to peripheral retinal disorders. We are excited about the potential of this platform using our SCS microinjector for both a biofactory and a native gene replacement approach. I'm also encouraged by our endocrine inhibitor program, as endocrines represent a novel target with limited competition. Integrins are multifunctional cell adhesion molecules that regulate critical cell processes such as adhesion, migration, proliferation, invasion, survival, and apoptosis. Additionally, they play a critical role in pathologic processes such as inflammation, angiogenesis, and fibrosis.
This trial is evaluating the efficacy safety and Tolerability Super Cornel delivery of RPX, We 14, using our S. U S micro injector.
The study entitled Aviate is a multicenter open label randomized active controlled dose escalation trial, that's expected to enroll approximately 40 patients with severe wet AMTI across two cohorts.
The primary endpoint of the trial and mean change in vision and patients dose of RG ex reporting compared to patients receiving monthly injections of ran it doesn't matter.
Other endpoints include mean change in central retinal thickness and number of anti VEGF Intravitreal injections received following administration Super core it'll RPX reporting.
This study is first in human gene therapy trial, utilizing our SCS micro injector I want to remind everyone that gene therapy has typically been delivered via sub retinal injection, which involves parts one of the truck fleet in the operating room and creation of regret not any followed by limited retinal detachment therapies injected.
Into the sub retinal space.
The sub retinal injection procedure has been undergoing optimization for over a decade. We believe it's simple quarterly injection should expose more surface area to the gene therapy, providing the potential to create large amounts of retinal lessen basically.
Since we're in the early stages of this approach you may still be more optimizing could do as the most appropriate dosing schedule is determined each therapeutic.
I'd like to note that Energenic Spiles earnings conference call. This last week.
And commenting on the 88 trial using Clearsight SCS micro injector. They stated that our GXT reporting that's been well tolerated to date, including no evidence of inflammation.
Dr. Thomas Chula: We believe that supracortial delivery of an integrin inhibitor suspension may provide targeting, compartmentalization, and durability advantages over topical or individual delivery, similar to what we've observed in other preclinical studies of small molecule suspensions like triamsomalone and excitin. Therefore, we've been running a series of preclinical studies with our integrin inhibitor suspension to assess its ocular tolerability, distribution, and pharmaco We expect our first set of data from these preclinical studies in the first half of next year. Ultimately, we hope to address the pathologic processes in diabetic macular edema and macular degeneration.
The tolerance, a super quarter, we administered gene therapy in humans, especially with a prophylactic steroid therapy would.
Would represent an important initial sign of progress.
Dennis bio expects to complete enrollment of the first cohort by the end of 2020 and report initial safety data from the first cohort in early 2021.
We are excited about this groundbreaking approach and look forward to the important safety signals from this first in man dose cohort of a super quarterly administered gene therapy.
In addition, we connect file has received acceptance of its R&D application by the U.S. happy to evaluate the Suprachoroidal deliveries Archie X 314 in patients with diabetic retinopathy.
This phase two trial entitled altitude is now.
What's your next bio expects to begin dosing patients across 15, leading U.S. retina centers by the end of 2020 and plan to report interim data from this trial in 2021.
Dr. Thomas Chula: As George mentioned, we are very pleased with the progress made by our development partners over the last several months. Last week, our partner Regenexx Bio announced that it had enrolled and dosed multiple patients in its Phase II gene therapy clinical trial for the treatment of wet AMD utilizing RGX314. This trial was evaluating the efficacy, safety, and tolerability of supracortal delivery of RGX314 using our SCS microinjector. The study, entitled Aviate, is a multi-center, open-label, randomized, active-control, dose-escalation trial that is expected to enroll approximately 40 patients with severe wet AMD across two cohorts. The primary endpoint of the trial is mean change in vision at the dose of RGX314 compared to patients receiving monthly injections of ranibizumab. Other endpoints include mean change in central retinal thickness and number of anti-VEGF individual injections received following administration of supracoronal RGX314. This study is the first in-human gene therapy trial utilizing our SCS microinjector.
We're very excited about the mechanics bio clinical trials for two important reasons first office based Super Crowed administration potentially avoid the risks associated with corresponding attracted me retinal sodomy and sub retinal injection, especially in diabetic patients and second the ability for physicians to treat patients in their offices could substantially increased.
<unk> access to care compared to current models of referring patients to regional Okcular gene therapy surgical treatment centers.
We're also encouraged by the progress fire oncology partner oral bio sciences.
It's a temporary they announced the dosing of the first patient in their phase two clinical trial evaluating the Super Cuomo administration of Hey, you 011 patients with Kuwait a melanoma.
This trial, whereas utilizing clearsight Fcs Michael exactly to the liver easier a one one to the post your segment of the <unk> record of melanoma are located.
The objective of this study include assessment of safety and preliminary efficacy in the treatment of choroidal melanoma utilizing Super Court administration at AIU 011. Other objectives include determined determining the highest tolerable dosing regimen as long as assessing immunogenicity, notably the first cohort of the phase two study demonstrated favorable.
Safety data with no adverse events noted.
His preliminary safety results or is by old man viral nanoparticle conjugates.
Important because they may further support the potential for viral vectors and supercritical states.
Dr. Thomas Chula: I want to remind everyone that gene therapy is typically delivered via subretinal injection, which involves parsed plantar tractomy in the operating room and creation of a retinotomy followed by a limited retinal detachment as therapy is injected into the subretinal space. This subretinal injection procedure has been undergoing optimization for over a decade. We believe that supracortial injection should expose more surface area to the gene therapy, providing the potential to treat large amounts of retina in a less invasive way. Since we are in the early stages of this approach, there may still be more optimizing to do as the most appropriate dosing schedule is determined for each therapeutic. I'd like to note that in Regenexx Bio's earnings conference call last week, in commenting on the ADA trial using Clearside's SCS microinjector, they stated that RGX314 has been well tolerated to date, including no evidence of inflammation. The tolerance of supercordially administered gene therapy in humans, especially without prophylactic steroid therapy, would represent an important initial sign of progress.
Furthermore, quidel melanoma is a rare and aggressive type of eye cancer and is the most common intractable cancer in adults with a high potential becoming that static.
This that's a critical treatment need and we are in contact with potential for 8011 to treat this devastating disease and improve the lives of patients.
Our medical Affairs team continues to proactively data scientific and medical community.
Several clinical data presentations were given that the annual scientific meeting of the society.
We also do a corporate and clinical overview at the retinal features form.
We look forward consenting data at the American Academy of Ophthalmology annual meeting later this week.
Importantly, we also know that data from a phase two clinical trial in diabetic macular edema was published an ophthalmology retinal appeared be Medline index retina Journal of the American Academy of Ophthalmology, the trial untitled I'd be evaluated supercritical side, you're going to use with entered between administered.
Patients with DMD over a six month evaluation period.
This early data suggest the potential Wolf has lived here if approved reducing treatment burden for DMD patients.
I believe we have just published a critical characterization of where to inject a procedure cost reimbursable disorders.
The article peer reviewed medical and external translational visual science and technology.
Describe newspaper Democratic Super One Jackson was well accepted by physician investigators.
The Boston Procedurally accommodates a wide range of anatomical demographic variables peak.
These studies suggest that single quarterly Jack and can be readily adopted and clinical crackers for targeted compartmentalize library of argument there.
Dr. Thomas Chula: Regenexx Bio expects to complete enrollment in the first cohort by the end of 2020 and report initial safety data from the first cohort in early 2021. We are excited about this groundbreaking approach and look forward to the important safety signals from this first in-man dose cohort of a super-coherently administered gene therapy. In addition, Regenexx Bio has received acceptance of its IND application by the U.S. FDA to evaluate the supercortical delivery of RGX314 in patients with diabetic retinopathy. This Phase 2 trial, entitled Altitude, is now at; Regenexx expects to begin dosing patients across 15 leading U.S. retina centers by the end of 2020 and plans to report interim data from this trial in 2021. We are very excited about the Regenexx bioclinical trials for two important reasons. First, office-based supercoronal administration potentially avoids the risks associated with parsed plantar patrectomy, retinotomy, and subretinal injection, especially in diabetic patients.
Altogether, we've made tremendous progress both internally and with our development partners to broaden the scope of development activities for Super Credo injection platform. It.
Most importantly, we look forward to continuing this positive trajectory in 2021.
I will now turn the call over to our CFO Charlie technique to review our financial results.
Thank you Tom.
<unk> financial results for the third quarter were published this afternoon and our press release and are available on our website. Therefore, I will summarize our current financial status.
As we reported our cash and cash equivalents at the end of September 20, Twond told approximately $15 million.
Our quarterly cash burn remains consistent with spending focused primarily on resubmission of the ending acres IPO and our anticipated clinical trial initiation of course CLS acts.
Planned investments in our preclinical work are also incorporated into our operating plans.
As George and Tom discussed our partner Bridgend expire initiated their phase two clinical trial, utilizing our SCS micro injector, which triggered a milestone payment from them of $3 million with this additional licensing revenue we are able to extend our cash runway and currently expect to have sufficient resources to fund plan.
The operations into the third quarter of next year.
We remain active participants in sell side sponsored events and look forward to presenting upsets people virtual health care Conference next week.
Dr. Thomas Chula: And second, the ability for physicians to treat patients in their offices could substantially increase patient access to care compared to current models of referring patients to regional ocular gene therapy surgical treatment centers. We are also encouraged by the progress made by our oncology partner, Oral Bioscience. In September, they announced the dosing of the first patient in their Phase 2 clinical trial evaluating the supracarotal administration of AU011 in patients with carotid melanoma. For this trial, ORA is utilizing Clearside's SES microinjector to deliver AE011 to the posterior segment of the eye, where chordal melanomas are located. The objectives of this study include assessment of safety and preliminary efficacy in the treatment of chordal melanoma utilizing superchordal administration of AU011. Other objectives include determining the highest tolerable dose regimen, as well as assessing immunogenicity.
I will now turn the call back over to George for his closing remarks.
Thank you Charlie.
In August of last year, we introduced a new strategic direction for clear side.
The two prong strategy included first building, an internal research and development pipeline in areas such as novel small molecules and non viral gene therapy.
Second establishing external collaborations with other companies to allow them to access supercar rental space and specific therapeutic areas, we could not or did not intend to pursue.
As you have heard in our remarks today the strategy does come to fruition through the hard work and dedication of our team over the last 15 months.
We have built a diversified pipeline of clinical and preclinical programs we.
We have focused our spending on the growth a key assets and we have secured non dilutive funding via upfront and milestone based payments.
Dr. Thomas Chula: Notably, the first cohort of this phase two study demonstrated favorable safety data with no adverse events noted. These preliminary safety results with ORAS viral nanoparticle conjugates are important because they may further support the potential for viral vectors in the supercorridor space. Furthermore, chordal melanoma is a rare and aggressive type of eye cancer and is the most common intraocular cancer in adults with a high potential for becoming metastatic.
Potential future royalty revenue stream.
We expect this momentum momentum to continue into 2021 with the potential approval of zipper receipt of initial clinical data for CLS X in wet AMD patients and the advancement of our preclinical programs.
I would now like to ask the operator to open the call up for questions.
Ladies and gentlemen, this you have a question at this time, please press star and the number one on your Touchtone telephone. If your question has been answered your question look yourself from the queue. Please press the pound key.
Dr. Thomas Chula: This presents a critical treatment need, and we are exploring the potential for AE011 to treat this devastating disease and improve the lives of patients. Our medical affairs team continues to proactively engage the scientific and medical community; several clinical data presentations were given at the annual scientific meeting of the Rettner Society. We will also deliver a corporate and clinical overview at the Retina Futures Forum, and we look forward to presenting data at the American Academy of Ophthalmology annual meeting later this week. Importantly, we also announced that data from a Phase II clinical trial in diabetic macular edema were published in Ophthalmology Retina, the peer-reviewed Medline Index Retina Journal of the American Academy of Ophthalmology. The trial entitled TYPE-E evaluated supracortial xytherapy when used with individually administered Fliversep to patients with DME over a six-month evaluation period.
Please stand by while we compile the kewaunee roster.
Our first question comes from the line of Liana Moussatos with Wedbush. Your line is open.
Hi, This is for myself liana. Thank you for taking our question Ben Congrats on all the progress.
Oh, how long will dictate what the new CMO did you did registration batches and then from there how long would dictate to get good to stay bleak data to submit to resubmit the abuse.
No I have one question on CNS effects. When you reported the safety data May 21 next year do you also plan to report any secondary endpoints.
Okay well. Thank you for that question I'll, let Tom take the second question in a minute I'll take the first one and I fear.
We.
We did you know disclosed again today that our timing on the NDA Resubmission would be no later than the first half of next year 2021, where things are going very well with the new CMO too.
Dr. Thomas Chula: This early data suggests a potential wolf enzyme here is approved in reducing the treatment burden for DMV patients. Finally, we have just published a clinical characterization of the supracortial injection procedure across three ventral disorders in the Arvo Peer-Reviewed Medical Index Journal, Translational Visual Science and Technology. The studies described in this paper demonstrate that supercritical injection is well-accepted by physician investigators and that the device and procedure may accommodate a wide range of anatomic and demographic variables.
To be precise once we make the registration batches it will take us three months to produce the stability data.
Once we re submit we expect that we will get a action on our resubmission within six months of the Resubmission date.
Tom you want to answer the question about CLS CX.
Sure.
Just a quick review so it's a phase one to a study is an open label dose escalation study the three cohorts of five patients each.
Charles A. Deignan: These studies suggest that supracortal injections could be readily adopted in clinical practice for targeted, compartmentalized delivery of ocular therapy. Altogether, we've made tremendous progress, both internally and with our development partners, to broaden the scope of development activities for our supercritical injection platform. And most importantly, we look forward to continuing this positive trajectory in 2021. I will now turn the call over to our CFO, Charlie Deignan, to review our financial results. Thank you, Tom. Our financial results for the third quarter were published this afternoon in our press release and are available on our website. Therefore, I will summarize our current financial status. As we reported, our cash and cash equivalents at the end of September 2020 totaled approximately $15 million.
I will begin at the 0.03 milligram dose and we have multiple endpoints that will include a predominately safety, but we'll also be assessing the tolerability visual function ocular anatomy a need for additional therapies.
We will be.
Presenting all of those endpoints.
In mid 2021.
Great. Thank you.
Our next question comes from the line of Big Bad job, along with Roth Capital Partners. Your line is open.
Hi, Thanks for taking my question just have a couple here. The first one I think we still feel like the gap I just kind of wondering what this body of work.
To have data from all book, what I did I hear you, Tom say that well just have data from cohort one and then I'd expect that we will see efficacy data from Jeff.
You know below us. So for example, the jopling 30 milligram or I do expect efficacy higher guess it.
Well thanks for the question first of all.
So as you know there's five patients per cohort.
And again, it's really key towards safety it'll be hard to make.
Make any efficacy conclusion said, a small sample size, but nevertheless, we will have some signals a biologic effect, including visual acuity RCT angiographic parameters.
Charles A. Deignan: Our quarterly cash burn remains consistent with spending focused primarily on resubmission of the NDA for Zyper and our anticipated clinical trial initiation for CLS-AS. The planned investments in our preclinical work are also incorporated into our operating plan. As George and Tom discussed, our partner Regenexx Bio initiated their Phase II clinical trial utilizing our SES microinjector, which triggered a milestone payment from them of $3 million. With this additional licensing revenue, we are able to extend our cash runway and currently expect to have sufficient resources to fund planned operations into the third quarter. We remain active participants in CellSide-sponsored events and look forward to presenting at the Stieple Virtual Healthcare Conference next year. I will now turn the call back over to George for his closing remarks. Thank you, Charlie.
We will of course the presenting.
Data from each cohort.
As we accumulate that data and escalate the dose.
Thanks, Tom and then another follow up when yeah, I know what the data from the Denny I coming up early next year I think a lot of people aren't going to be interested in your Apis with non bouncing delivery. So can you just talk about where you are with that five isn't a little bit more detail.
Sure so.
As I mentioned in my prepared remarks.
We have.
Two programs that were working on and.
We've seen some interesting results so far.
Huh.
What we've already reported is that Super choroidal delivery of a marketing versus Intravitreal delivery results in the same activity if that market teams, which I provided.
Provided us.
Some some hope about going forward. So we're now working with therapeutic transgene, what weve observed. So far is that we can open the Super Quidel Stakes acutely in this rabbit model all the way to the poster your coal, which suggests that we can treat macular disorders as well as peripheral disorders. We've.
George M. Lasezkay: In August of last year, we introduced a new strategic direction for Clearside. The two-pronged strategy included, first, building an internal research and development pipeline in areas such as novel small molecules and non-viral gene therapy, and second, establishing external collaborations with other companies to allow them to access the suprachoroidal space in specific therapeutic areas we could not or did not intend to pursue. As you have heard in our remarks today, this strategy has come to fruition through the hard work and dedication of our team over the last 15 months. We have built a diversified pipeline of clinical and preclinical programs. We have focused our spending on the growth of key assets.
We've also seen expression of therapeutic gene has a duration of the study.
And we've also observed that when we inject super currently versus intellectually on clinical exam will be threatened the suprachoroidal delivery seems to be a better tolerated.
We're in the process of further.
Quantifying these results and we hope to present this sometime in 2021.
Thank you and then the final one your friend, who I think is the high level one Opco George maybe maybe have already asked this in the past, but you know I do think about I'd have a different pipeline program. On you also have to and stuff that you're working on are you thinking about partnership at all or do you anticipate no independent we are moving.
Operator: And we have secured non-dilutive funding via up-front and milestone-based payments with a potential future royalty revenue stream. We expect this momentum to continue into 2021 with the potential approval of Xipyr, receipt of initial clinical data for CLS-AX and WET-AMD patients, and the advancement of our preclinical program. I would now like to ask the operator to open the call for questions. Ladies and gentlemen, if you have a question at this time, please press star and the number one on your touchtone telephone. If your question has been answered or you wish to remove yourself from the queue, please press the pound key.
These programs into the clinic and beyond.
Thanks, Doug I appreciate that.
Question I think.
The difference between now in 14 15 months ago is that we have these choices to make.
When we didn't last this time last year and that's a good thing.
We are going to be actively evaluating you know.
How we go forward with our various programs I Wouldnt say that collaborations are out of the picture, but right now what we're really focused on is getting the zipper and D.A. resubmitted in the most timely fashion to kick off the CLS Adx clinical program and to generate more data on those pre.
Operator: Please stand by while we compile the Q&A roster. Our first question comes from the line of Liana Moussakos with Wedbush. Your line is open. Hi, this is Shweta for Liana.
Clinical programs. So we can make we can make the decision as to whether there.
Programs that we can afford to bring forward because they justify that or they should be programs that we might be able to do better with a partner, but our our strategy is still to develop in the internal pipeline as well as external collaborations and net that's not changing.
Shweta: Thank you for taking our questions and congratulations on all the progress. For Xipyr, how long would it take for the new CMO to produce the registration batches? And then from there, how long would it take to generate the stable data to resubmit the NDA? And I have one question on CLS-AX.
Thanks, George and congrats on the strong progress.
Thank you appreciate it.
Shweta: When you report the safety data mid-21 next year, do you also plan to report any secondary endpoints? Okay, well, thank you for that question. I'll let Tom take the second question in a minute. I'll take the first one on Zyphyr.
[music].
And our next question is from the line of Annabel Samimy with Stifel. Your line is open.
Hi, Thanks for taking my question and I agreed to a lot of great progress and a big difference from last year.
So.
I want to get a sense you made a comment earlier that.
The ophthalmology meeting is coming much more comfortable if that's your platform and an excuse I guess my question is how are they getting that comfort and that experience.
George M. Lasezkay: We've, you know, disclosed again today that our timing on the NDA resubmission would be no later than the first half of next year, 2021. Things are going very well with the new CMO. To be precise, once we make the registration batches, it will take us three months to produce the stability data. Once we resubmit, we expect that we will get action on our resubmission within six months of the resubmission date. Tom, you want to answer the question about CLSAx? Sure, just a quick review.
There aren't any products on the market yet is that primarily.
Through some of the clinical work that you're doing or is it just primarily through the published work or they are these physicians working with US yes platform at all and getting much more comfortable with the delivery into into that area of the eye.
I ask the second question I had is with regard to.
Some expectations around milestones and when we might see some obviously you've got a lot of clinical and regulatory events coming up that may trigger. Some so for example with resubmission.
Dr. Thomas Chula: So this is phase one of a study. It's an open-label dose escalation study. There are three cohorts of five patients each, and we'll begin at the 0.03 milligram dose.
Dr. Thomas Chula: And we have multiple endpoints that will include predominantly safety, but we'll also be assessing tolerability, visual function, ocular anatomy, and need for additional therapies. And we will be presenting all of those endpoints in the mid-20s. Great, thank you. And our next question comes from the line of Zegba Jalla with Roth Capital Partners. Your line is open. Hello, thanks for taking my question. I just have a couple here.
[music].
Besides here or is that on Resubmission is it only on approval are you going to get any milestones with.
Upcoming data and then.
Then I guess finally in this.
This kobin environment, it's it's pretty clear that ophthalmology has been the worst hit.
One of the worst hit areas in terms of clinical development. So are you seeing any slowdown.
Not necessarily of your.
Partner its activity, but in terms of additional partnership talks that Hello.
Or any yet additional request that you are get inbound requests from from partner. She is your technology. Thanks.
Zegba Jalla: The first one, I think, relates to CLS effects. Just kind of wondering, for this study, if we're going to have data from all of the cohorts, or did I hear you, Tom, say that we'll just have data from cohort one? And then, do you expect that we will see efficacy data from just the lowest dose, for example, the 0.03 milligram? Or do you expect efficacy at higher doses? Well, thanks for the question, first of all. As you know, there are five patients per cohort. And again, it's really keyed towards safety.
Hmm.
Oh, that's a lot to digest.
I can break it down if you forget [laughter] I'm just trying to take notes, while you're talking on the on the Supercross hurdle.
There's maybe a couple of ways, we can answer that I'll, let Tom speak.
A moment, but.
The question was about people, becoming more comfortable with Suprachoroidal I think there's a couple of reasons for that and then I'll, let Tom elaborate on that and then you might talk about the cobot question as well.
First of all our team has done a tremendous job in getting the word out about how safe and how reliable and how easy to use the Super Choroidal administration has been okay. We've done a number of presentations a bold in person before cobot hit and virtually since Cogut has made most of these conference all these conferences.
Dr. Thomas Chula: It'll be hard to make any efficacy conclusions with a small sample size, but nevertheless, we'll have some signals of biological effect, including visual acuity, OCT, and angiographic parameters. We will, of course, be presenting data from each cohort as we accumulate that data and escalate. And then another follow-up one year later, you know, with the data from Regenify coming up early next year, I think a lot of people are also going to be interested in your efforts with non-viral gene delivery. So can you just talk about where you are with that progress in a little bit more detail? Uh, sure.
Go virtual so we've really gotten the news out we've established a scientific advisory board with some key opinion leaders that really buy into the Super Corrado approach. There's also been a fair number of preclinical work specifically work by Peter can Fitzgerald, that's really just started to create some justice.
Dr. Thomas Chula: So, uh... As I mentioned in my prepared remarks, we have two programs that we're working on, and we've seen some interesting results so far. What we've already reported is that supercordial delivery of a marker gene versus individual delivery results in the same activity of that marker gene, which gives us some hope about going forward. So we're now working with therapeutic transgenes. What we've observed so far is that we can open the supracrital space acutely in this rabbit model all the way to the posterior pole, which suggests that we can treat macular disorders as well as peripheral disorders. We've also seen expression of this therapeutic gene for the duration of the study, and we've also observed that when we inject supercordially versus intubitually on clinical examination of these retinas, the supercordial delivery seems to be better tolerated. We're in the process of further quantifying these results, and we hope to present this sometime in 2021. And again, in the final year for me, I think it's just a high level one for George, maybe.
Vacation for people to be interested in the space both for viral and non viral.
Gene therapy, he has done it both and that's probably what led to our Oh.
Leading reason why reject expire wanted departure us partner with us last year. So there's the preclinical work that's going on there is the information that's being spread by our medical affairs team and just all the work we've done with or with Bausch with rejects in terms of training their people and then.
Turning around and training their investigators the word is spreading that this is a fairly straight forward process to use and the data is starting to be accumulated that looks like the super choroidal space can make a difference in in many conditions that are being treated or with certain therapeutic agents.
Delivery to the behind the visual field might be preferable to.
Administration in front of the visual field, Tom do you want to comment any further on that.
What seems to be.
A growing acceptance and interest in the Suprachoroidal space.
Sure I think you summarized it really well I just wanted to sort of start from scratch in mentioned that we've done over a thousand injections, We fund global.
George M. Lasezkay: And maybe I've already asked this in the past, but you know, as you think about having different pipeline programs, you also have the intervention stuff that you're working on. Are you thinking about partnerships at all? Or do you anticipate, you know, independently moving these programs into the clinic and being able to do that? Thanks. I appreciate that question. I think the difference between now and 14-15 months ago is that we have these choices to make when we didn't this time last year, and that's a good thing. We're going to be actively evaluating, you know, how we go forward with our various programs. I wouldn't say that collaborations are out of the picture, but right now, what we're really focused on is getting the Zyphyr NDA resubmitted in the most timely fashion to kick off the CLS-AX clinical program and to generate more data on those preclinical programs so we can make the decision as to whether they're programs that we can afford to bring forward because they justify that, or they should be programs that we might be And that's not changing.
Global trials phase one into phase 123, global trials and we trained hundreds of clinician investigators we found that it was a very well adopted and then as George mentioned, there's been some preclinical gene therapy studies using supercritical approach. He mentioned Johns Hopkins, but also one of the highlights universe.
Steve Iowa has also published a paper recently purchased.
Pursuing suprachoroidal injection Preclinically of gene therapy, and then as George mentioned, there's a whole host of trials now actually using our very micro injector. So they'll be for clinical trials. This year assessing three assets and a question you mentioned magenis bio they'll have.
Two trials for a empty and Danny.
Our investigators have all undergone training.
Or a bio sciences. Similarly has their phase two trial up and running with her SCS micro injector. Their investigators are also oh.
We have also received and also undergoing a training we'll have our own phase 128 trial with except NIM will be training investigators for that.
And I also should mentioned that even globally Arctic.
Plans to pursue a clinical trial.
In China.
Our training programs and as a bonus.
Medical Affairs will also be very actively engaging.
Retina community as it plans for lunch and then finally, just wanted to highlight or amplify what George mentioned, our medical affairs team has been incredibly active this year, we had over 35 presentations at multiple retina Congresses.
George M. Lasezkay: Thanks, George, and congratulations on the steady progress. Thank you. I appreciate it. And our next question is from the line of Annabel Samimy with Stiefel. Your line is open.
Annabel Eva Samimy: Hi, thanks for taking my question, and I agree there's been a lot of great progress and a big difference from last year. So, you know, I want to get a sense of how the ophthalmology community is becoming much more comfortable with the SDS platform and its use. I guess my question is, how are they getting that comfort and that experience?
I did mentioned earlier that we just published its available fully online download we just published a clinical characterization of the Super Cordele injection procedure using a micro injector.
That showed that the procedure is very well accepted by.
Clinician investigators and that showed that the or suggest that the procedure could be very rapidly adopted in clinical practice.
Annabel Eva Samimy: You know, there aren't any products on the market yet. Is it primarily through some of the clinical work that you're doing, or is it just primarily through published work? Are these physicians working with the SDS platform at all and getting much more comfortable with the delivery into that area of the eye? I guess the second question I have is with regard to some expectations around milestones and when we might see some. Obviously, you've got a lot of clinical and regulatory events coming up that may trigger some. So, for example, with the resubmission of Xipir, is that on resubmission? Is it only on approval?
Tom do you want to just comment very briefly and then we'll get the her final question on coated impacting our recruitment.
Sure. So the American Society retina specialists has put out guidelines for coated.
And basically the guidelines.
Essentially state that that.
Essential.
Trapeze like anti VEGF therapies for Hey, Andy.
That should not be halted because obviously patients required and Tom by and large the retinal community has adopted as adopted quite well to continue to treat patients with anti VEGF therapies for Mds DMD and cardio we.
Annabel Eva Samimy: upcoming data. And then, I guess, finally, in this COVID environment, it's pretty clear that ophthalmology has been the worst hit, one of the worst hit areas in terms of clinical development. So are you seeing any slowdown, not necessarily of your partners' activity, but in terms of additional partnership talk that, you know, or any additional requests that you're getting, inbound requests from partners to use your technology? Thanks. That's a lot to digest. I can break it down if you forget. I was trying to take notes while you were talking.
We think for our.
Phase one two way clinical trial as I mentioned the initial cohort will just be five stations, we have multiple sites that are.
Initiating.
Each of these sites.
He has been up and running a treating wet AMD patients without interruption.
They are all very experienced investigators would actually have a capital injection procedure as well as experienced with our.
Well, we face wet AMD trial, so we're fully confident that we'll be able to recruit.
Hi patients for our initial cohort without any significant interruption.
George M. Lasezkay: On the supercoroidal, there's maybe a couple of ways we can answer that. I'll let Tom speak in a moment, but the question was about people becoming more comfortable with supercoids. I think there are a couple of reasons for that, and then I'll let Tom elaborate on that, and then he might talk about the COVID question as well. First of all, our team has done a tremendous job of getting the word out about how safe and how reliable and how easy to use the supercoroidal administration has been. We've done a number of presentations, both in person before COVID hit and virtually since COVID has made most of these conferences virtual.
And I'll just make one final comment I haven't seen cobot really affect anything in terms of potential.
Central partnership discussions or anything interesting collaborations I haven't seen that impact.
Any noticeable degree.
And.
So that's that's all I can say, we've actually not really been impacted in any significant way Michael in terms of our business.
Fortunately for us.
Okay, Great and then the last question regarding a new way that we can start thinking about how to frame some milestones that might be coming in from.
From various.
Catalysts or developments you have with partners.
Or that has not been disclosed and they won't be disclose at any point going forward until they come along.
George M. Lasezkay: We've really gotten the news out. We've established a scientific advisory board with some key opinion leaders that really buy into the supercoroidal approach. There's also been a fair amount of preclinical work, specifically work by Peter Campechero that's really started to create some justification for people to be interested in the space, both for viral and non-viral gene therapy. He's done both, and that's probably what led to a major reason why Regenexx Bio wanted to partner with us last year.
I think we'll let Charlie handle that one.
Hi, Annabel yet unfortunately.
To the company and should be confidential agreements and the license deals we can't disclose those but the most important the majority of the big milestone comes on approval MSI periods, we've disclosed bausch, there's a $15 million payment there and Arctic also our Chinese licensee also.
George M. Lasezkay: There's the preclinical work that's going on. There's the information that's being spread by our medical affairs team, and just all the work we've done with Aura, with Foush, with Regenexx in terms of training their people and them turning around and training their investigators. The word is spreading that this is a fairly straightforward process to use, and data is starting to accumulate that looks like the supercoroidal space can make a difference in many conditions that are being treated or with certain therapeutic agents for which delivery behind the visual field might be preferable to administration in front of the visual field. Tom, do you want to comment any further on that, what seems to be a growing acceptance and interest in the supercoroidal space? Sure, I think you summarized it really well.
There's some money associated with that but.
We can't give the details until they come in and many others.
Okay. If I may just ask one more question with regard to.
I guess I have here with but Andy I know that you've been presenting I'm not named Im sorry, Danny Me, then presenting your data or publishing rotated a tidy data on it.
You know obviously, there's other programs and development and Yeah me, whether it's gene therapy for your partners are.
And you know moving on with wet AMD do with your own exceptional.
And possibly moving into another area is.
Pushing forward time sooner alone NTM me I guess thought of as an older technology that maybe you're more interested in moving onto some newer technologies.
Non steroid based.
Dr. Thomas Chula: I just wanted to sort of start from scratch and mention that, you know, we've done over a thousand injections. We've run global trials, phase 1 and 2, phase 1, 2, 3 global trials, and trained hundreds of clinician investigators. We found that it was very well accepted.
Technology that may have better impact.
Impacting the marketplace.
The the answer to that I think is fairly straightforward that develop further development of zipper here.
Is entirely up to Bausch and lomb.
It's their decision whether they want to push that forward for DMD. We've shared all our data that weve generated both in the RV show in Dnbi with them we've.
Dr. Thomas Chula: And then, as George mentioned, there's been some preclinical gene therapy studies using super coroidal approaches. He mentioned Johns Hopkins, but I also wanted to highlight the University of Iowa has recently published a paper recently pursuing super corridal injection preclinical gene therapy. And then, as George mentioned, there's a whole host of trials now actually using our very own micro-inductor. So there'll be four clinical trials this year assessing three assets. And, of course, we mentioned Regenexx Bio. They'll have two trials for AMD and DME.
We've had conversations with them.
To bring them up to speed so they know, but the decision about zipper, you're going into additional indications is entirely in their control.
We're we're working on other things on our internal pipeline and that's their product they've license that product and the development is up to them.
Okay, great. Thank you.
Sure.
Thank you and our next question comes from the line of John Waldron with JMP Securities. Your line is open.
Hey, Thanks for taking the questions just a couple from me.
Dr. Thomas Chula: Their investigators have all undergone training. Oura Biosciences similarly has their phase 2 trial up and running with our SCS micro-injector. Their investigators have also received and are also undergoing training. We'll have our own phase 1, 2, and A trial with Exitinib, and we'll be training investigators for that.
I'm wondering can you comment on the design of the sale of ACS trial on why the requirement for the anti VEGF treatment and should you expect to see the same response and bulk VEGF naive and VEGF experienced patients and then based on your preclinical data is three months, what you're expecting for the duration of effect.
Dr. Thomas Chula: And I also should mention that even globally, Arctic plans to pursue clinical trials in China, and they'll have training programs. As for those medical affairs, we'll also be very actively engaging the retina community as it prepares for launch. And then finally, I just wanted to highlight or amplify what George mentioned. Our medical affairs team has been incredibly active this year. We've had over 35 presentations at multiple retina congresses, and I did mention earlier that we just published it. It's available fully online download.
Or do you think it could have a longer impact. Thank you.
I think thats for Tom Tom do you want to address that.
Sure. So let me start with the first question. The design. So as you know as you alluded to were requiring patients to offend treatment experience.
And the reason.
For patient safety HM.
This is first in man is the first time actually first time any tyrosine kinase inhibitors and injected the suprachoroidal space. So so we want patients whove already been treated and we want to we want to maintain efficacy.
Obtained with.
Initially anti VEGF therapy.
Dr. Thomas Chula: We just published a clinical characterization of the supracortial injection procedure using a micro-injector, and that showed that the procedure is very well accepted by clinicians, and it showed that, or suggests that the procedure could be very rapidly adopted in clinical practice. Tom, do you want to just comment very briefly, and then we'll get to her final question on COVID impacting recruitment? Sure, so the American Society of Retinal Specialists has put out guidelines for COVID, and basically, the guidelines essentially state that essential therapies like anti-VEGF therapies for AMD should not be halted because, obviously, patients require them, and by and large, the retina community has adopted has adopted quite well; they continue to treat patients with anti-VEGF therapies for AMD and DME and RDO. We think for our Phase 1, 2A clinical trial. As I mentioned, the initial cohort will just be five patients. We have multiple sites that are initiating. Each of these sites has been up and running treating wet AMD patients without interruption.
However that doesn't preclude later studying exiting it as as primary therapy, but but we want it to go with a more conservative route because this is the first time in any any tyrosine kinase inhibitors been injected in the Suprachoroidal space.
I think.
Your next question.
Well actually before I finish that question.
Also want to add that other companies looking at novel therapies have also adopted a similar approach for their initial.
Phase one studies and patients will have been treated as well so it's a fairly common.
Quite typical trial design.
Your next question about expected duration.
We expect to see multiple month duration.
We know that the.
Levels, and the Suprachoroidal space and retina, what some of the doses that were anticipating using will be above the IC 50 for many many months on this could very well be a six month therapy, obviously its too soon to tell without any clinical data, but we're expecting multiple month.
Really.
And I think was your third question.
No you touched on it thank you very much and congrats on the progress.
Thank you.
Thank you and I'm not showing any further questions I'll now turn the call back over to George has that gone for closing remarks. Please go ahead.
Dr. Thomas Chula: They are all very experienced investigators with our supracortial injection procedure as well as experienced with early phase wet AMD trials. So we're fully confident that we'll be able to recruit five patients for our initial cohort without any significant interruption. And I'll just make one final comment. I haven't seen COVID really affect anything in terms of potential partnership discussions or anything, interesting collaborations. I haven't seen that impact to any noticeable degree.
Thank you and thank you for joining us on the call. This afternoon. We appreciate your continued interest in clear side and we look forward to updating you on our progress in future. Operator, you may now disconnect.
Ladies and gentlemen, this does conclude the program. Thank you for participating everyone have a great day.
[music].
George M. Lasezkay: That's so that's all I can say. We've actually not really been impacted in any significant way by COVID, fortunately. And then the last question regarding any way that we can start thinking about how to frame some milestones that might be coming in from various catalysts or developments you have with partners, or have those not been disclosed, and they won't be disclosed at any point going forward until they come. I think we'll let Charlie handle that. Hey, Annabel.
Charles A. Deignan: Yeah, unfortunately, due to confidential agreements and the license deals, we can't disclose those. But the most important, the majority, the big milestone comes with approval of Xypyr, as we've disclosed, Bausch. There's a $15 million payment there, and Arctic also, our Chinese licensee, there's some money associated with that.
Charles A. Deignan: But we can't give the details until they come in on any other. If I may just ask one more question with regard to... I guess I will appear with WebAMD.
Annabel Eva Samimy: I know that you've been presenting, I'm not WebAMD, I'm sorry, it was DME. You've been presenting your data or publishing your data, the Tybee data. You know, obviously, there's other programs in development in DME, whether it's gene therapy for your partners or in, you know, moving on with what AMD has with your own exigena and possibly moving into another area is, Pushing forward with Trimed Simulon in DME, I guess, thought of as an older technology that maybe you're more interested in moving on to some newer technologies, you know, technology that may have a better impact The further development of Xipir is entirely up to Baosheng Long.
George M. Lasezkay: It's their decision whether they want to push that forward for DME. We've shared all our data that we've generated both in RVO and DME with them. We've had conversations with them to bring them up to speed, so they know, but the decision about Xipir going into additional indications is entirely in their control. We're working on other things in our internal pipeline, and that's their product. They've licensed that product, and the development is up to them.
George M. Lasezkay: Okay, great. Thank you. Thank you. And our next question comes from the line of John Wolleben with J&P Security. Your line is open. Hey, thanks for taking the questions. Just a couple for me.
[music].
Jonathan Patrick Wolleben: I'm wondering, can you comment on the design of the CLS-AX trial and why the requirement for anti-VEGF treatment and should you expect to see the same response in both VEGF-naive and VEGF-experienced patients? And then, based on your preclinical data, is three months what you're expecting for the duration of effect, or do you think it could have a longer impact? Thank you. I think that's for Tom. Tom, do you want to address that? Sure, so let me start with the first question, the design.
Dr. Thomas Chula: So as you know, as you alluded to, we're requiring patients to have treatment experience, and the reason really is for patient safety. This is the first in man, the first time, actually the first time, any tyrosine kinase inhibitor has been injected into the supracortal space, so we want patients who've already been treated, Initial Anti-Digest Therapy. However, that doesn't preclude later studying exitinib as primary therapy, but we wanted to go with a more conservative route because this is the first time that any tyrosine kinase inhibitor has been injected into the supracortal. And I think your next question... Well, actually, before I finish that question, I also want to add that other companies looking at novel therapies have also adopted a similar approach for their initial phase one studies, so patients will have been treated as well.
Dr. Thomas Chula: So it's a fairly common and quite typical trial. And I think your next question about expected duration is right; we expect to see multiple month duration. We know that the levels in the supracortal space and retina with some of the doses that we're anticipating using will be above the IC50 for many, many months. This could very well be a six-month therapy. Obviously, it's too soon to tell without any clinical data, but we're expecting multiple months of durability.
Dr. Thomas Chula: And I think, was there a third question? No, you touched on that. Thank you very much and congratulations on the progress. Thank you. Thank you, and I'm not showing any further questions, so I'll now turn the call back over to George Lasezkay for closing remarks. Please go ahead. Thank you, and thank you for joining us on the call this afternoon. We appreciate your continued interest in Clearside, and we look forward to updating you on our progress in the future.
George M. Lasezkay: Operator, you may now disconnect. Ladies and gentlemen, this does conclude the program. Thank you for participating, and everyone have a great day.
Operator: The film is a work of fiction. Any resemblance to actual persons, living or dead, is coincidental. The film is a work of fiction, and any resemblance to actual persons, living or dead, is coincidental. The [inaudible]