Q2 2022 Passage Bio Inc Earnings Call
Operator: Good morning and welcome to the Passage Bio 2nd Quarter 2022 Financial and Operating Results Conference Call.
Good morning, and walk them through the passage bio second quarter, 2022 financial and operating results conference call.
Operator: At this time, all participants are on a listen-only mode.
At this time all participants are in a listen only mode.
Operator: Following the formal remarks, we will open the call up for your questions.
Following the formal remarks, we will open the call up for your questions. Please.
Operator: Please be advised that this call is being recorded at the company's request.
Please be advised that this call is being recorded at the company's request.
Operator: Now I'd like to turn the call over to Stuart Henderson, Vice President of Corporate Development and Investor Relations.
Now I'd like to turn the call over to Stuart Henderson, Vice President of corporate development and Investor Relations.
Operator: Stuart, please proceed.
Stuart Please proceed.
Stuart Henderson: Thank you, Operator.
Thank you operator.
Stuart Henderson: This morning, we issued a press release that outlines the topics we plan to discuss today. This release is available on the Passage Bio website under the Press Releases and Statements section of Investors & News.
We issued a press release that outlines the topics we plan to discuss today. This release is available on the passage bio website under the press releases and statements section of investors the news on.
Stuart Henderson: On today's call, Chip Hale, Interim Chief Executive Officer, will review our 2nd Quarter 2022 and recent business highlights.
On today's call chip tail interim Chief Executive Officer will review, our second quarter 2022, and recent business highlights Mark <unk>, Our Chief Medical Officer will review, our clinical programs and Simona King Our Chief Financial Officer will review, our second quarter 2022 financial results.
Stuart Henderson: Mark Forman, our Chief Medical Officer, will review our clinical programs.
Stuart Henderson: And Simona King, our Chief Financial Officer, will review our 2nd Quarter 2022 financial results.
Stuart Henderson: Before we begin, please note that today's call may include a number of forward-looking statements, including but not limited to comments on, our expectations about timing and execution of anticipated milestones, including the initiation of clinical trials and the availability of clinical data from such trials, our expectations about our collaborators' and partners' ability to execute key initiatives, the ability of our lead product candidates to treat their respective target CNS disorder, manufacturing plans and strategies, trends with respect to financial performance and cash flows, the company's ability to fund research and development programs, impact of the COVID-19 pandemic on the company's operations, and its ability to manage costs along with uses of cash and other matters.
Before we begin please note that today's call may include a number of forward looking statements, including but not limited to comment on our expectations about timing and execution of anticipated milestones, including the initiation of clinical trials and the availability of clinical data from such trials, our expectations about our collaborators and partners ability to.
Execute key initiatives.
The ability of our lead product candidate to treat their respective target CNS disorder.
Manufacturing plans and strategies.
Trends with respect to financial performance and cash flows the company's ability to fund research and development programs impact of the COVID-19 pandemic on the company's operations and its ability to manage costs along with the uses of cash and other matters.
Stuart Henderson: These forward-looking statements are based on assumptions that are subject to risks and uncertainties that could cause the company's actual results to differ significantly from those suggested by these statements.
These forward looking statements are based on assumptions that are subject to risks and uncertainties that could cause the company's actual results to differ significantly from those suggested by these statements. Given these risks and uncertainties you should not place undue reliance on these forward looking statements.
Stuart Henderson: Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements.
Stuart Henderson: Please refer to the company's filings with the SEC for information concerning risk factors that could cause its actual results to differ materially from expectations, including any forward-looking statements made on this call.
Please refer to the company's filings with the SEC for information concerning risk factors that could cause its actual results to differ materially from expectations, including any forward looking statements made on this call.
Stuart Henderson: Except as required by law, the company disclaims any obligation to publicly update or revise any forward-looking statements to account for or reflect events or circumstances that occur after this call.
Except as required by law the company disclaims any obligation to publicly update or revise any forward looking statements to account for or reflect events or circumstances that occur. After this call.
Stuart Henderson: It is now my pleasure to pass the call over to Chip Kale.
It is now my pleasure to pass the call over to chip <unk> chip.
Stuart Henderson: Chip?
Thanks, Stuart and thank you all for joining US. This morning, we continue to make important progress advancing our programs and our mission to develop transformative therapies for devastating CNS disorders with limited or no approved treatment options.
Chip Hale: Thanks, Stuart, and thank you all for joining us this morning.
Chip Hale: We continue to make important progress advancing our programs and our mission to develop transformative therapies for devastating CNS disorders with limited or no approved treatment options.
As we advance our three ongoing clinical programs, we look forward to delivering multiple value, creating clinical milestones in the second half of this year.
Chip Hale: As we advance our three ongoing clinical programs, we look forward to delivering multiple value-creating clinical milestones in the second half of this year. These milestones build upon several recent accomplishments, including, first, strong momentum in advancing our Imagine 1 trial for GM1 gangliosidosis. We have now progressed the study to recruitment of the final cohort, Cohort 4, in the dose-ascending phase of the trial, and we are excited to have begun recruiting patients for this cohort.
Milestones build upon several recent accomplishments, including first strong momentum and advancing our imagine one trial for <unk> Gangliosidosis. We have now progressed the study to recruitment of the final cohort cohort four and the dose ascending phase of the trial and we are excited to have begun recruiting patients for this.
Chip Hale: Second, presentation of encouraging longer term safety biomarker and clinical efficacy, data from Cohort 1 of our Imagine 1 trial at the American Society of Gene and Cell Therapy, or ASGCT, annual meeting in May.
Cohort.
Presentation of encouraging longer term safety biomarker and clinical efficacy data from cohort one of our imagine one trial at the American Society of gene and cell therapy or <unk> annual meeting in May.
Chip Hale: Mark will review these data in more detail shortly, and we look forward to reporting, initial data from Cohorts 2 and 3 later this year.
Mark will review these data in more detail shortly and we look forward to reporting initial data from cohorts two and three later this year.
Chip Hale: Finally, FDA clearance of our IND application for a Phase 1 study of PBML04 in metachromatic, leukodystrophy. This marks our fourth consecutive IND clearance as a company, and our third rare pediatric, lysosomal storage disorder program to reach clinical development. This clearance is a testament to the strong CMC and analytics capabilities we have developed, internally at our state-of-the-art CMC laboratory in New Jersey, and the high-quality preclinical data supporting the program through our strong partnership with Penn's gene therapy program.
Finally, FDA clearance of our IND application for a phase one study of <unk>, four and Metachromatic Luca dystrophy. This marks our fourth consecutive IND clearance as a company and our third rare pediatric lysosomal storage disorder program to <unk>.
Each clinical development. This clearance is a testament to the strong CMC and analytics capabilities. We have developed internally at our state of the art CMC Laboratory in New Jersey, and the high quality preclinical data supporting the program through our strong partnership with <unk> gene therapy program.
Chip Hale: In addition, we continue to recruit additional patients in Cohort 1 of our GALAXY clinical, trial for early infantile CRAV-A disease, and look forward to reporting initial data from a subset of Cohort 1 by year-end.
In addition, we continue to recruit additional patients in cohort one of our Galaxy clinical trial for early infantile krabbe disease and look forward to reporting initial data from a subset of cohort one by year end.
Chip Hale: Consistent with our current guidance, we also expect to dose the first patient in our UPLIFT-D, clinical trial for frontotemporal dementia shortly.
Consistent with our current guidance, we also expect to dose the first patient in our uplift the clinical trial for frontier.
Dementia shortly.
Chip Hale: Clinical trial execution remains a core priority for us. We have established a global network of trial sites across each of our ongoing clinical, programs, with active sites in the United States, Brazil, Canada, the UK, Israel, and the Netherlands.
Clinical trial execution remains a core priority for US we have established a global network of trial sites across each of our ongoing clinical programs with active sites in the United States, Brazil, Canada, The U K, Israel and the Netherlands. We also continue to employ various strategies to <unk>.
Chip Hale: We also continue to employ various strategies to drive patient identification across our, programs, including several initiatives focused on identifying frontotemporal dementia patients with a granulin mutation. These efforts have increased genetic testing among FTD patients over the last few months.
<unk> patient identification, our cross our programs, including several initiatives focused on identifying frontal temporal dementia patients with a granular mutation.
These efforts have increased genetic testing among FTB patients over the last few months.
Chip Hale: Underpinning our execution is a strong cash position, which we estimate will fund our, continued operations into the second quarter of 2024.
Underpinning our execution as a strong cash cash position, which we estimate will fund our continued operations into the second quarter of 2024.
Chip Hale: Lastly, we were pleased to have recently added Michael Kamarck, PhD, to our board of directors. Dr. Kamarck is a seasoned biopharmaceutical executive with over 40 years of experience, in discovery research, process development, and technical operations. We look forward to drawing on his expertise.
Lastly, we were pleased to have recently added Michael Kamarck ph D to our board of directors. Dr. <unk> is a seasoned biopharmaceutical executive with over 40 years of experience in discovery research process development and technical operations, we look forward to drawing on his expertise.
Chip Hale: With that, I will now turn it over to our chief medical officer, Mark Forman, who will, review the progress made across each of our ongoing clinical programs.
With that I will now turn it over to our Chief Medical Officer, Mark Forman, who will review the progress made across each of our ongoing clinical programs.
Mark Forman: Thank you, Chip.
Thank you chip first let me discuss our lead program <unk> one for <unk> Gangliosidosis.
Mark Forman: First, let me discuss our lead program, PBGM01, for GM1 ganglia sedosis. GM1 is a fatal neurological lysosomal storage disease caused by a GLB1 gene mutation that, results in low activity of the beta galactosidase enzyme. This leads to a rapid neurological decline and, in the most severe forms, unfortunately, to a mortality within several years, years. Patients with GM1 are a rare and underserved population, and there are no disease-modifying therapies for the disorder currently.
<unk> is a fatal neurological lysosomal storage disease caused by <unk>, one gene mutation that results in low activity of the beta galactosidase enzyme.
This leads to a rapid neurological decline and in the most severe form unfortunately to mortality within several years.
Patients with <unk> are rare and underserved population and there are no disease modifying therapies for that disorder currently.
Mark Forman: Our IMAGINE-1 clinical trial focuses on the early and late infantile forms of GM1, which are the most severe forms of the disease. This global Phase I-II trial is an open-label dose escalation study with pbGMO1, enrolling four distinct cohorts divided by age and dose level.
I imagine one clinical trial focuses on the early and late infantile form of GM, one which are the most severe forms of the disease.
This global Phase <unk> trial is an open label dose escalation study with <unk>, one enrolling four distinct cohorts divided by age and dose level.
Mark Forman: As a reminder, our approach uses a next-gen proprietary AAV-HUSA6 capsid administered via the cisterna magna to deliver a codon-optimized GLB1 transgene to increase beta-galactosidase enzyme activity in the brain and peripheral, tissues.
As a reminder, our approach using our next generation proprietary AAV <unk> a six.
<unk> administered via the Cisterna Magna to deliver a codon optimized youll be one transgene to increase beta galactosidase enzyme activity in the brain in peripheral tissues.
Mark Forman: We were pleased to report updated positive interim safety, biomarker, and clinical efficacy data from cohort 1 at ASGCT in May. These data show that the low dose of pbGMO1 was well-tolerated and had a favorable safety profile, with patient 1 data through 13 months and patient 2 data through seven months. No serious adverse events, no complications related to ICM administration, and no evidence of dorsal root ganglion toxicity were observed in either patient.
We were pleased to report updated positive interim safety biomarker and clinical efficacy data from cohort one at <unk> in May.
These data showed that the low dose of <unk>, one was well tolerated and had a favorable safety profile with patient one data through 13 months and patient two data through seven months.
No serious adverse events no complications related to ICM administration and no evidence of dorsal root ganglion toxicity were observed in either patients.
Mark Forman: Both patients showed continued improvement across developmental areas on the Vineland-2, a caregiver-assessed scale, and Bayley-3, an investigator-assessed scale following pbGMO1 administration. Patient 1 showed improvement across all developmental areas through the 12-month assessment, with notable progression in motor and language domains. Patient 1 progressed from taking a few steps to jumping and climbing stairs.
Both patients showed continued improvement across developmental areas on the Vineland II, a caregiver assessed scale and daily three and investigator assessed scale following <unk> administration.
Patient one showed improvement across all developmental areas through the 12 month assessments with notable progression in motor and language domains.
Patient one progress from taking a few steps to jumping and climbing stairs.
Mark Forman: Patient 2 showed improvement across multiple developmental domains, despite having severe, developmental delay at baseline and a prior history of regression. This patient progressed from standing with support for five seconds to standing with support for one to three minutes and walking without support.
Patient two showed improvement across multiple developmental domains, despite having severe developmental delay at baseline and a prior history of regression.
This patient progressed from standing with support for five seconds to standing with support for one to three minutes and walking without support.
We were extremely pleased to see continued and meaningful improvement in developmental milestones cities children, including regaining of lost milestones.
Mark Forman: We were extremely pleased to see continued and meaningful improvement in developmental milestones for these children, including regaining of lost milestones.
Mark Forman: Volumetric MRI data showed meaningful growth in brain volume for patient 1, who was 15 months of age at gene transfer, patient 2, who had severe developmental delay at baseline and was 31 months of age at gene transfer, showed a slight decline in brain volume from baseline at six months. Longer-term biomarker data for beta-galactosidase enzyme activity in CSF and serum showed functional transgene expression.
Volumetric MRI data showed meaningful growth in brain volume for patient one who was 15 months of age at gene transfer.
Patient two who had severe developmental delay at baseline and was 31 months of age of gene transfer showed a slight decline in brain volume following days from baseline at six months.
Longer term biomarker data for beta galactosidase enzyme activity in CSF and serum showed functional transgene expression.
Mark Forman: As Chip mentioned, our IMAGINE-1 trial has experienced strong momentum. To date, we have dosed a total of five patients in the study. We have completed dosing of cohorts 1 and 3, low-dose in late and early infantile patients respectively, and have dosed the first patient in the high-dose late infantile cohort 2.
As chip mentioned, our imagine one trial has experienced strong momentum to date, we have dosed a total of five patients. In this study we have completed dosing of cohort one and three low dose in late and early infantile patients respectively and have dosed. The first patient in the high dose late infantile cohort two.
Following positive review of interim safety data from cohort three by the independent data monitoring Committee. We are excited to have now advance the study to recruitment of patients for cohort four high dose early infantile GM one.
Mark Forman: Following positive review of interim safety data from cohort 3 by the Independent Data Monitoring Committee, we are excited to have now advanced the study, to recruitment of patients for cohort 4, high-dose early infantile GM1. This is the final patient cohort in the dose ascending phase of the study.
This is the final patient cohort in the dose ascending phase of the study.
Mark Forman: We remain on track to report initial, 2.
We remain on track to report initial safety and biomarker data from cohorts two and three in the second half of 2022.
Mark Forman: Moving on to our global PBKRO3 program, GALX-C, which is an open-label dose escalation study, in patients with early infantile Krabbe disease. Krabbe disease is a condition that progresses rapidly, damaging both the brain and the peripheral, nervous system, resulting in a life expectancy of only two years in the severe cases. Krabbe disease is a fatal neurological lysosomal storage disease caused by a GALX-C gene mutation, that results in decreased enzyme activity of galactosylseramidase.
Moving on to our global <unk> III program Galaxy, which is an open label dose escalation study in patients with early infantile krabbe disease.
<unk> disease is a condition that progresses rapidly damaging both the brain and the peripheral nervous system tilting in a life expectancy of only two years in the severe cases.
<unk> disease is a fatal neurological lysosomal storage disease caused by a galaxy gene mutation that results in decreased enzyme activity of Galactic <unk>.
Mark Forman: Like GM1, this is also a pediatric leukodystrophy and lysosomal storage disease with an underserved, population and devastating disease progression.
Like GM. One this is also a pediatric Luca dystrophy, and lysosomal storage disease, with an underserved population and devastating disease progression.
Mark Forman: Our approach to treating Krabbe is similar to our approach to GM1, utilizing the same, proprietary capsid and ICM delivery to potentially address both CNS and peripheral disease manifestation.
Our approach to treating <unk> is similar to our approach to GM, one utilizing the same proprietary capsid and ICM delivery to potentially address both CNS and peripheral disease manifestations.
Mark Forman: We have now opened six sites for recruiting globally, including in the U.S., Canada, the Netherlands, Israel, and the U.K. We have dosed the first patient in the study and are actively recruiting additional patients, in cohort one.
We have now opened six sites for recruiting globally, including in the U S, Canada, and Netherlands, Israel and the U K.
We have dosed the first patient in the study and are actively recruiting additional patients in cohort one.
Mark Forman: We look forward to reporting initial safety and biomarker data from a subset of cohort, one by the end of 2022.
We look forward to reporting initial safety and biomarker data from a subset of cohort one by the end of 2022.
Mark Forman: Our third clinical program, PBFTO2, is for frontal temporal dementia with granular mutations. FTD is a devastating form of early-onset dementia affecting patients between the ages, of 40 to 65. The form of the disease we are seeking to treat with our therapy is caused by a granulin, or GRN gene mutation, which results in a deficiency of progranulin. It's estimated that about 5 to 10% of FTD is caused by a GRN mutation.
Our third clinical program PV FTE <unk> is for frontal temporal dementia with granular mutations.
<unk> is a devastating form of early onset dementia affecting patients between the ages of 40 to 65.
The form of the disease, we are seeking to treat with our therapy is caused by a granular or GRM gene mutation, which results in a deficiency of programming right.
It's estimated that about 5% to 10% of FTE is caused by a GRM mutation.
Mark Forman: We are utilizing the AAV1 capsid to deliver the GRN gene via ICM delivery to the CSF. The goal of this treatment and delivery approach is to potentially provide higher-than-normal, levels of the progranulin protein to the CNS to overcome the progranulin deficiency in, GRN gene mutation carriers.
We are utilizing the AAV one capsule to deliver the GRM gene by ICM delivery to the CSS.
The goal of this treatment and delivery approach is to potentially provide higher than normal levels of the program human proteins to the CNS to overcome the program in deficiency in GRN gene mutation carriers.
Mark Forman: As a reminder, the study design consists of two cohorts of three patients, each receiving, two different ascending doses of PBFTO2, with an optional third cohort treated with a higher dose, depending on safety and biomarker data results observed in the first two cohorts.
As a reminder, the study design consists of two cohorts of three patients each receiving two different as sending doses of <unk> with an optional third cohort treated with a higher dose depending on safety and biomarker data results observed in the first two cohorts.
Mark Forman: We now have three active clinical trial sites, including two in the U.S. and one in Brazil, and activation efforts at additional sites continue.
We now have three active clinical trial sites, including two in the U S and one in Brazil and activation efforts at additional sites continue.
Mark Forman: We also continue to employ a variety of patient identification initiatives, including efforts, to increase genetic testing among FTD patients.
We also continue to employ a variety of patient identification initiatives, including efforts to increase genetic testing among SCD patients.
Mark Forman: We've now identified prospective patients with a granulin mutation and expect to dose, the first patient in the study shortly.
We've now identified prospective patients with a granular.
Shouldn't expect to dose the first patient in this study shortly.
Mark Forman: Turning lastly to PBML04, our program for metachromatic leukodystrophy, or MLD.
Turning lastly to <unk> for a program frenetic chromatic Luca dystrophy or MLP.
Mark Forman: Infantile MLD is a fatal inherited disease caused by mutations in the ARSA, or the arylsulfatase, A gene, which reduces enzyme activity. MLD is characterized by muscle weakness, rigidity, gait disorder, and developmental delays, and, unfortunately, children typically die by the age of five.
Infantile MLD is a fatal inherited disease caused by mutations in the Asa where the arrow sulfatase, a gene which reduces enzyme activity.
MLG is characterized by muscle weakness rigidity gait disorder, and developmental delays and unfortunately children that typically die by the age of five.
Mark Forman: Worldwide incidence is approximately 1 in 100,000 live children.
Worldwide incidence is approximately $1 100000 live births.
Mark Forman: Our approach is similar to our approach to GM1 and CRAB-A, utilizing the same proprietary capsid, AAV, HU68, and ICM delivery to express ARSA and potentially address both central nervous system and peripheral manifestations of this devastating disease.
Our approach is similar to our approach to <unk> utilizing the same proprietary capsid AAV <unk> 68, an ICM delivery to express Orissa and potentially address both central nervous system and peripheral manifestations of this devastating disease.
Mark Forman: We announced in June that the FDA cleared our IND, marking our fourth IND clearance as a company, and our third pediatric lysosomal storage disorder program to reach clinical development. We're thrilled to have achieved this milestone and are excited by the promise of our potential therapy.
We announced in June that the FDA cleared our IND.
Our fourth IND clearance as a company our third pediatric lysosomal storage disorder program to reach clinical development.
We're thrilled to have achieved this milestone and are excited by the promise of our potential therapy.
Mark Forman: As we continue to evaluate our resources and operating expenses, we've made the decision to hold advancement of clinical development activities for the program at this time.
As we continue to evaluate our resources and operating expenses. We've made the decision to hold the advancement of clinical development activities for the program at this time.
Mark Forman: With that, I will now turn the call over to Simona to review our financials.
With that I will now turn the call over to Simona to review our financials.
Simona King: Thank you, Mark.
Simona King: Before reviewing our expenses for the quarter, I want to reiterate our strong cash position. As we reported in our press release this morning, we ended the second quarter with approximately $239.3 million in cash, cash equivalents, and marketable securities, compared to $267.1 million as of March 31, 2022.
Thank you Mark before reviewing our expenses for the quarter I want to reiterate our strong cash position that we're in.
Reported in our press release. This morning, we ended the second quarter with approximately $239 3 million in cash cash equivalents and marketable securities compared to $267 1 million as of March 31 2022.
Simona King: We continue to expect these existing cash resources to fund operations into Q2 2024, and continue to manage our cash carefully to ensure we can deliver on meaningful clinical milestones over the coming quarters. R&D expenses were $26.8 million for the quarter ended June 30, 2022, compared to $33.1 million for the same quarter in 2021. The decrease was primarily due to a decrease of $10.9 million in clinical manufacturing expenses, which relates to the timing of our manufacturing activities. This amount was partially offset by $4.6 million of increases in clinical operations expenses, expenses associated with the PEN agreement and facility and other expenses.
We continue to expect these existing cash resources to fund operations into Q2 in 2024 and continue to manage our cash carefully to ensure we can deliver on meaningful clinical milestones over the coming quarters.
R&D expenses were $26 $8 million for the quarter ended June 32022, compared to $33 1 million for the same quarter in 2021.
The decrease was primarily due to a decrease of $10 9 million in clinical and manufacturing expenses, which relate to the timing of our manufacturing activities.
This amount was partially offset by $4 6 million of increases in clinical operations expenses expenses associated with the Penn agreement and facility and other expenses.
Simona King: DNA expenses were $13 million for the quarter ended June 30, 2022, compared to $15.4 million for the same quarter ended June 30, 2022, compared to $15.4 million for the same quarter in 2021. The decrease was primarily due to a $2.3 million reduction in personnel-related and share-based compensation expense, related to our workforce restructuring, which was partially offset by seven expenses incurred in the three months ended June 30, 2022, related to the separation of our chief executive officer.
G&A expenses were $13 million for the quarter ended June 32022, compared to $15 4 million for the same quarter.
Quarter ended June 32022, compared to $15 4 million for the same quarter in 2001.
The decrease was primarily due to a $2 $3 million reduction in personnel related and share based compensation expense related to our workforce restructuring, which was partially offset by severance expenses incurred in the three months ended June related to our workforce restructuring, which was partially offset by 700.
Simona King: Net loss was $39.5 million for the quarter ended June 30, 2022, compared to $48.4 million for the same quarter in 2021.
Expenses incurred in the three months ended June 32022 related to the separation of our Chief Executive Officer.
Simona King: Let me now turn it back to Chip for closing remarks.
Net loss was $39 5 million for the quarter ended June 32022, compared to $48 4 million for the same quarter in 2021.
Chip Hale: Thank you, Simone.
Chip Hale: With three ongoing clinical programs and state-of-the-art analytics capabilities, we believe we are well-positioned to achieve several important milestones over the coming months. We continue to experience strong momentum in our IMAGINE-1 trial, and expect to report initial data from cohorts two and three in the coming months.
Let me now turn it back to chip for closing remarks.
Thank you Simona with three ongoing clinical programs and state of the art analytics capabilities. We believe we are well positioned to achieve several important milestones over the coming months, we continue to experience strong momentum in our imagine one trial and expect to report initial data.
Chip Hale: We expect to report initial data from cohorts two and three in the coming months, and expect to report initial data from cohorts two and three in the second half of 2022.
From cohorts two and three in the second half of 2022 additional expected near term milestones include first reporting initial safety and biomarker data from a subset of cohort one of our Galaxy trial by the end of 2022 and second consistent with our guidance dosing.
Chip Hale: Additional expected near-term milestones include, first, reporting initial safety and biomarker data from a subset of cohort one of our GALAX-C trial by the end of 2022, and second, consistent with our guidance, dosing the first patient in our UPLIFT-D trial soon.
Our first patient in our uplift D trial soon prior to taking your questions I would like to thank the patients families and trial investigators as well as the employees at passage bio and our colleagues at the gene therapy program for their support and dedication to our mission with that I'd like to open the call for your quest.
Chip Hale: Program.
Chip Hale: Prior to taking your questions, I would like to thank the patients, families, and trial, investigators, as well as the employees at Passage Bio and our colleagues at the Gene, Therapy Program for their support and dedication to our mission.
Chip Hale: With that, I'd like to open the call for your questions.
<unk> operator.
Operator: Operator?
Operator: Thank you.
Thank you.
Operator: We will now take any questions you may have.
We will now take any questions you may have and we have a question. Please press star one one and you will put be put into the queue.
Operator: If you have a question, please press R11, and you will be put into the queue.
Operator: Please stand by while we compile the Q&A roster.
Please standby, while we compile the Q&A roster.
Okay.
Operator: Our first question comes from the line of Brendan Smith with Cowen.
Our first question comes from the line of Brendan Smyth with Cowen Your line is open.
Operator: Your line is open.
Brendan Smith: Hi, guys.
Brendan Smith: Thanks very much for taking the questions.
Hi, guys. Thanks, very much for taking the questions.
Brendan Smith: Just a couple quick ones from us.
A couple of quick ones from us.
Brendan Smith: Sorry if I missed this, but for the initial CREB-A data, and actually maybe for the GM-1, update as well in the second half, is this something we should probably expect in like a press release, or are you targeting something more formal as like a medical meeting?
If I missed this but for the initial <unk> data and actually maybe for the Jan one update as well in second half or is this something we should probably expecting like a press release that you were targeting something more formal at a medical meeting.
Brendan Smith: And then just really quickly for MLD, can you maybe just expand a bit on your decision, for clinical development for the time being?
And then just really quickly for MLD can you, maybe just expand a bit on your decision.
Medical development, all time being is it.
Brendan Smith: Is this probably something that we would see for additional pipeline programs?
Probably something that we would get for additional pipeline programs is it more of a way to strategize and prioritize the current programs you have in the clinic.
Brendan Smith: Is it more of a way to strategize and prioritize the current programs you have in the clinic?
Brendan Smith: Or does it have something to do more with the drug itself?
Or does it have something to do more with the drug itself.
Brendan Smith: Thanks very much.
Thanks very much.
Chip Hale: Great.
Chip Hale: Hey, Brendan.
Chip Hale: Thank you.
Great Hey, Brendan Thank you thanks for those questions.
Chip Hale: Thanks for those questions.
Chip Hale: So first, I think on the CREB-A data and the GM-1 data, we are currently evaluating the, best way to release that data.
So first I think on the <unk> data and the GM one data.
We are currently evaluating the best way to release that data.
Chip Hale: We will be looking to see if it makes sense to do it at a scientific conference.
We will be looking to see if it makes sense to do it at a scientific conference, but if that doesn't it depends on when the data comes in and the schedule of those meetings or we would do a press release. So that is still to be determined but we're looking at all different alternatives.
Chip Hale: But if that doesn't, you know, it depends on when the data comes in and the schedule, of those meetings, or we would do a press release.
Chip Hale: So that is still to be determined, but we're looking at all different alternatives.
Chip Hale: On your question about MLD and the decision to pause the further development, as we said, earlier, it's really, it's not about the asset itself. It is about our evaluation of our current financial situation and priorities.
On your question about MLD and the decision to pause the further development as.
As we said earlier, it's really it's not about the asset itself. It is about our evaluation of our current.
Our financial situation and priorities.
Chip Hale: And so we're just evaluating the best way to go forward and to prioritize the assets, that we need to prioritize.
And so.
We're just evaluating the best way to go forward and to prioritize.
The assets that we need to prioritize.
Chip Hale: Got it.
Chip Hale: Yep.
Chip Hale: Makes a lot of sense.
Got it it makes a lot sense. Thanks guys.
Chip Hale: Thanks, guys.
Chip Hale: Thank you.
Thank you.
Operator: Please stand by for our next question.
Please standby for our next question.
Operator: Our next question comes from the line of Nina Baitrido-Garge with Citi.
Our next question comes from the line of Neena <unk> Garg with Citi. Your line is open.
Operator: Your line is open.
Nina Baitrido-Garge: Hey, guys.
Nina Baitrido-Garge: Thanks for taking the question.
Nina Baitrido-Garge: I was just wondering if you could give us an update on some of the safety events that, you saw in the initial patient that was dosed with PBKR03, and I'm just curious how many additional patients you've dosed.
Hey, guys. Thanks for taking the question I was just wondering if you could give us an update on some of the safety events that you saw in the initial patient that was dosed.
Tdkr three and I'm just curious how many additional patients you built and if you've seen any similar safety signals and then on <unk>.
Nina Baitrido-Garge: And if you've seen any similar safety signals.
Nina Baitrido-Garge: And then on PBFTO2, it sounds like you have identified some patients, so just kind of, curious what the gating factor is to getting at least the first patient dosed there.
It sounds like you have identified some patients. So just kind of curious what the gating factor is to.
Getting at least the first patient dose.
Nina Baitrido-Garge: Thanks.
Chip Hale: Great.
Chip Hale: Thank you, Nina.
Great. Thank you Nina I'll hand, it over to tomorrow to talk about the safety events that we've seen with PV KR or three.
Mark Forman: I'll hand it over to Mark to talk about the safety events that we've seen with PBKR03.
Mark Forman: But on FTD, yeah, consistent with our guidance, we're happy to announce that we expect to, be dosing shortly, and that is a result of, we think, ongoing patient identification activities that we've been doing.
But on the.
On FTB, yes.
Consistent with our guidance, we are happy to announce that we will be we expect to be dosing shortly.
And and that is a result of we think ongoing patient identification activities that we've been we've been doing.
Mark Forman: So I'll hand it over now to Mark to talk about the safety issues on the CREB-A asset.
So I'll hand, it over now to Mark to talk about the safety issues on.
On the <unk> assets.
Mark Forman: All right.
Mark Forman: So thanks, Chip.
Thanks Chip so as we disclosed previously yet our first patient.
Mark Forman: So as we disclosed previously, our first patient approximately 30 days after administration, developed the adverse event of acute hydrocephalus, that patient had a ventricular peritoneal shunt placed, and that was to reduce the CSF buildup in the brain, which this is a standard treatment for hydrocephalus. This patient has done well following this intervention and is currently stable, and, the SAE has been updated to resolve it.
Approximately.
On May 30 days after administration developed.
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The adverse event of acute hydrocephalus.
That patient.
Ventricular peritoneal shut in place and that was.
Without that was to reduce the CSF buildup in the brain, which is this is a standard treatment for hydrocephalus on.
This patient has done well following this intervention and is currently stable and the SAE has been updated to resolve it.
Mark Forman: As a result of that SAE, and as we disclosed previously, we've updated the protocol to, include additional safety monitoring, and that has now, and now we're actively recruiting additional patients for that study, but we haven't dosed anyone to date.
As a result of that SAE and as we disclosed previously we have updated the protocol.
Nina Baitrido-Garge: Got it.
To include additional safety monitoring and we're and that has now and now we're actively recruiting additional patients for that study, but we haven't dosed anyone to date.
Yes.
Nina Baitrido-Garge: Thank you.
Got it thank you.
Nina Baitrido-Garge: Thank you.
Thank you.
Please standby for our next question.
Operator: Please stand by for our next question.
Our next question comes from the line of Madison Al Saadi with Raymond James Your line is open.
Operator: Our next question comes from the line of Madison El Saadi with Raymond James.
Please check to see if you're on mute Madison.
Operator: The line is open.
I'm sorry about that this is Alex on for Danielle Thrill.
Nino you saw my questions, but just wanted to see what.
Expansion for additional sites for FCB recruitment, how that effort is going.
And how kind of the patient influx.
I think COVID-19 was a major concern about screening efforts and wanted to hear how if those are impacted at some.
Whatever various move.
Moving forward for patient recruitment in that study.
Operator: Please check to see if you're on mute, Madison.
Great. Thank you, Alex I think I'll hand, it over to Mark to talk about the latest on our site expansion and our patient I'd activities and how that maybe helping screening, but just as a general comment as we've commented over the past few quarters. We have we have been focusing on.
A lot on our patient I'd efforts.
And.
And we believe that we are beginning to see some benefit from that so but with that I'll hand, it over to mark.
Operator: I'm sorry about that.
Alex: This is Alex on for Danielle Thrill.
Thanks Chip.
So consistent with all of our programs for the Sdd program, we are taking a global approach to.
Alex: Nina, you stole my questions, but I just wanted to see what expansion for additional sites for SED recruitment, how that effort is going and how kind of the patient influx.
Alex: You know, I think COVID was a major concern about screening efforts and wanted to hear how influencers are impacted by some of these VA point whatever variants moving forward for patient recruitment in that study.
To date, we have three active clinical sites.
Following the recent activation of trial sites in the us in Houston in Brazil. So we now have two sites open in the U S and one in Brazil, we are actively working towards opening of sites in additional an additional countries and we anticipate those.
Alex: Great.
<unk> bear fruit in the coming in the coming months.
Chip Hale: Thank you, Alex.
With regard to patient identification again as chip said, we're really excited that we.
We expect to be dosing, our first patient soon and we believe this is a direct result of our patient identification efforts that we were we've employed a number of different strategies.
Chip Hale: I think I'll hand it over to Mark to talk about the latest on our site expansion and our patient ID activities and how that may be helping screening.
To help improve rates of genetic testing among <unk> patients, we're supporting genetic testing in partnership with informed DNA and VK and are working with health care providers and that study investigators to provide these kits free of charge and to offer these genetic testing program.
Chip Hale: But just as a general comment, as we've commented over the past few quarters, we have been focusing a lot on our patient ID efforts, and we believe that we're beginning to see some benefit from that.
We're also working closely with each of our study investigators to develop individualized plans for patient identification and recruitment.
Chip Hale: So, but with that, I'll hand it over to Mark.
Lastly, we're conducting extensive outreach to health care providers to educate them on the availability and benefits all of our genetic testing program.
Mark Forman: Thanks, Jim.
Mark Forman: So, consistent with all of our programs, for the FDD program, we are taking a global approach. To date, we have three active clinical sites following the recent activation of trial sites in Houston and Brazil. So, we now have two sites open in the U.S. and one in Brazil. We are actively working towards opening sites in additional countries, and we anticipate those to bear fruit in the coming months.
These efforts have hedged translates excuse me have translated into a significantly observed increase in genetic testing among our FTE among SCD patients with our sponsor genetic testing programs over the last few months and are quite encouraging and we have identified a number of individuals of number of patients with GRN mutations in there.
Are the patients that are currently being evaluated excuse me evaluated for.
For study availability, but again, we expect.
Mark Forman: With regard to patient identification, again, as Chip said, we're really excited that we expect to be dosing our first patient soon, and we believe this is a direct result of our patient identification efforts where we've employed a number of different strategies.
Mark Forman: To help improve rates of genetic testing among FTD patients, we're supporting genetic testing in partnership with Informed DNA and V-TEG, and are working with healthcare providers and study investigators to provide these kits free of charge and to offer these genetic testing programs.
Mark Forman: We're also working closely with each of our study investigators to develop individualized plans for patient identification and recruitment.
Mark Forman: And lastly, we're conducting extensive outreach to healthcare providers to educate them on the availability and benefits of our genetic testing program.
To dose our first patient.
In the near future.
Okay.
Great. Thanks, so much.
Thank you please standby for our next question.
Mark Forman: These efforts have translated into a significantly observed increase in genetic testing among FTD patients with our sponsored genetic testing programs over the last three months, and are quite encouraging. We have identified a number of patients with GRN mutations, and those are the patients that are currently being evaluated for study availability.
Our next question comes from the line of Laura Chico with Wedbush. Your line is open.
Mark Forman: But again, we expect to dose our first patient in the near future.
Mark Forman: Great, thank you so much.
Hey, good morning, guys I apologize I joined a little bit late.
Alex: Thank you.
Two questions first.
The FTB Richard I guess I just wanted to clarify is this necessarily is the bottleneck here.
Operator: Please stand by for our next question.
A diagnosis issue or is there something else how does that play in terms of just the competitive environment and then I apologize I missed this.
Operator: Our next question comes from the line of Laura Chico with WETBUSH.
Any updates related to the CEO search thanks very much.
Operator: Your line is open.
Great. Thank you Laura I think I'll I'll take your second question first and then hand, it over to Mark to talk about FTE recruitment, but on the CEO search.
Laura Chico: Hey, good morning, guys.
Laura Chico: I apologize.
Laura Chico: I joined a little bit late.
The board has engaged Russell Reynolds to conduct a public search for the new CEO . The searches active and the board is evaluating potential candidates and is making good progress while the searches ongoing are strong capable executive leadership team remains focused on driving execution of our near term milestones and managing our cash.
Laura Chico: I just had two questions.
Laura Chico: First, on the FTD recruitment, I guess I just wanted to clarify, is this necessarily, – is the bottleneck here a diagnosis issue, or is there something else kind of at play in terms of just the competitive environment?
Laura Chico: And then, I apologize, I missed this.
With that I'll hand, it over to Mark to talk about.
What what the issue is and what we're doing to resolve the issues with the FTB recruitment.
Sure, Yes, thanks chip.
I think.
The challenges.
With the recruitment that we've experienced I think are.
Brazil are multifactorial on first there is.
A critical issue is the identification of patients and so that is where our extensive outreach efforts are working to sort of increase that sort of throughput that are coming through.
Yes, certainly.
Competition with other companies that are <unk> are having asked that do have active trials.
<unk>.
But lastly, as the patients that are identified obviously have to meet eligibility criteria. So it's a combination of all of those factors, but again I think to reiterate we've.
The efforts that we have been making to do our outreach and to increase our throughput of genetic testing are clearly bearing fruit and we now have.
A cohort of individuals that are being evaluated for eligibility.
Yes.
Thank you.
And I'm showing no further questions in the queue at this time.
Laura Chico: Any updates related to the CEO search?
Ladies and gentlemen, thank you for participating in today's conference call. This does conclude your program and you may disconnect everyone have a great day.
Laura Chico: Thanks very much.
To raise Johan during Q&A, you can dial one one.
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Chip Hale: Great.
Chip Hale: Thank you, Laura.
Chip Hale: I think I'll take your second question first and then hand it over to Mark to talk about, FTD recruitment.
Chip Hale: But on the CEO search, the board has engaged Russell Reynolds to conduct the public search, for the new CEO.
Chip Hale: The search is active, and the board is evaluating potential candidates and is making good progress. While the search is ongoing, our strong, capable executive leadership team remains focused, on driving execution of our near-term milestones and managing our cash.
Chip Hale: With that, I'll hand it over to Mark to talk about what the issues and what we're doing, to resolve the issues with the FTD recruitment.
Mark Forman: Sure.
Mark Forman: Yes.
Mark Forman: Thanks, Chip.
Mark Forman: So, I mean, I think the challenges with the recruitment that we've experienced, I think, are multifactorial.
Mark Forman: First, there is, you know, I mean, a critical issue is the identification of patients.
Mark Forman: And so, that is where our extensive outreach efforts are working to sort of increase that, sort of throughput that are coming through.
Mark Forman: There's certainly competition with other companies that are having, that do have active trials.
Mark Forman: But lastly, is the patients that are identified, obviously, have to meet eligibility criteria.
Mark Forman: So, it's a combination of all of those factors.
Mark Forman: But again, I think, to reiterate, we've, the efforts that we have been making to do our, outreach and to increase our throughput of genetic testing are clearly bearing fruit.
Mark Forman: And we now have a cohort of individuals that are being evaluated for eligibility.
Mark Forman: Thank you.
Operator: And I'm showing no further questions in the queue at this time.
Operator: Ladies and gentlemen, thank you for participating in today's conference call.
Operator: This does conclude your program, and you may disconnect.
Operator: Everyone, have a great day.
Operator: To raise your hand during Q&A, you can dial star 11.
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