Q3 2021 Passage Bio Inc Earnings Call

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BF-WATCH TV: BF-WATCH TV 2021

Operator: Thank you for holding. Good morning, and welcome to the Passage Bio third quarter 2021 Financial and Operating Results conference call.

[music].

Operator: At this time, all participants are in listen-only mode. Following the formal remarks, we will open the call up for your questions. Please be advised this call is being recorded at the company's request. At this time, I'd like to turn it over to Stuart Henderson, Vice President of Investor Relations and Strategic Finance. Stuart, please proceed.

Thank you for holding good morning, and went to the passage bio third quarter of 2021 financial and operating results Conference call. I. This time, all participants are and listen only mode. Following the formal remarks, who will open the call up for your question. Please be and I says call is being recorded at the company's request.

Stuart M. Henderson: Thank you, Operator. 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. On today's call, Bruce Goldsmith, President and Chief Executive Officer, will discuss recent business highlights. Eliseo Salinas, our Chief Research and Development Officer, will review our key clinical updates, and Simona King, our Chief Financial Officer, will review our third quarter 2021 financial results.

At this time I'd like to turn it over to Stuart Henderson, Vice President of Investor Relations and strategic Finance Stuart. Please proceed.

Thank you operator.

This morning, we issued a press release that outlines the topics we plan to discuss today.

This release is available on the passage by a website under the press releases and statements section of investors in years.

On today's call Bruce Goldsmith, President and Chief Executive Officer will discuss recent business highlights.

Sales silliness, our Chief Research and development Officer will review, our key clinical updates and Simona King Our Chief Financial Officer will review, our third quarter of 2021 financial results.

Stuart M. 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, impacts of the COVID-19 pandemic on the company's operations, and its ability to manage costs along with uses of cash and other macros. 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 the, Given these risks and uncertainties, you should not place undue reliance on these forward-looking 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 the, 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 them. It is now my pleasure to pass the call over to CEO Bruce Goldsman. Bruce.

Before we begin please note that today's call me 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.

The ability of our lead product candidates treat their respective targets DNS disorder.

Manufacturing plans and strategies trends with respect to financial performance and cash flows the company's ability to fund research and development programs impacts of the COVID-19 pandemic on the company's operations and its ability to manage costs along with uses of cash and other matters.

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.

Please refer to the company's filings with the SEC for information concerning risk factors that could cause it's actual results to differ materially from expectations, including any forward looking statements made on 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.

It is nine my pleasure to pass the call over to C E O Bruce Goldsmith Bruce.

Thanks, Stuart and thank you all for joining us this morning.

Bruce Goldsmith: Thanks, Stuart, and thank you all for joining us this morning. As we near the end of 2021, we are well positioned to deliver on multiple value drivers with the advancement of our clinical stage pipeline in both pediatric and adult CNS indications, for which there is a high degree of unmet clinical need. Our efforts are underpinned by our four strategic pillars. A strong, productive partnership with Dr. Jim Wilson and his team at the University of Pennsylvania's Gene Therapy Program, or GTP. A robust and differentiated pipeline, an integrated manufacturing supply chain, and a well-positioned corporate foundation that includes a strong cash balance.

As we near the end of 2021, we are well positioned to deliver on multiple value drivers with the advancement of our clinical stage pipeline and both Paediatric and adult CNS indications for which there is a high degree of unmet clinical need.

Our efforts are underpinned by our four strategic pillars are strong productive partnership with Dr. Jim Wilson and his team at the University of Pennsylvania's gene therapy program or GTT.

A robust and differentiated pipeline and integrated manufacturing supply chain and Ah well positioned corporate foundation that includes a strong cash balance.

Our primary focus continues to be on the execution of our three global fees one two programs.

Bruce Goldsmith: Our primary focus continues to be on the execution of our three global Phase I-II programs. Since our last quarterly update, we are pleased to have activated multiple clinical trial sites across these programs. Despite challenges related to COVID-19, site activations, coupled with a variety of initiatives to support early patient identification and clinical trial enrollment, have paved the way for us to deliver on important clinical milestones by year end and into 2022. These include reporting first-in-human data from the initial cohort of patients in our global IMAGINE1 trial for the treatment of GM1 ganglia cytosis later this quarter.

Since our last quarterly update we are pleased to have activated multiple clinical trial sites across these programs.

Despite challenges related to COVID-19.

Site Activations, coupled with a variety of initiatives to support early patient identification and clinical trial enrollment have paved the way for us to deliver unimportant clinical milestones by year end and into 2022.

These include reporting first and human data from the initial cohort of patients in our global imagine one trial for the treatment of GM. One Gangliosidosis later this quarter.

Dosing, the first patient and our global uplift D study for the treatment of frontal temporal dementia with granular mutations and our global Galaxy study for the treatment of crap a disease by the year at.

Bruce Goldsmith: Dosing the First Patients in our Global Uplift-D Study for the Treatment of Frontal Temporal Dementia with Granulin Mutation and our Global Galaxy Study for the Treatment of Crab A Disease by the End of the Year and reporting initial safety and 30-day biomarker data from the first cohort of patients in our Uplift-D and Galaxy trials in the first half of 2022. These trials are part of our portfolio of seven programs focused on rare monogenic CNS disorders, and we have the option to license an additional 10 programs from GTP.

And reporting initial safety at 30 date biomarker data from the first cohort of patients in our uplift D and the galaxy trials in the first half of 2022.

These trials are part of our portfolio of seven programs focused on rare monogenic CNS disorders.

We have the option to license an additional 10 programs from GTP.

Following the transformative expansion of our strategic collaboration with GTP announced last quarter. These 10 remaining options May also include large CNS diseases.

Bruce Goldsmith: Following the transformative expansion of our strategic collaboration with GTP, announced last quarter, these 10 remaining options may also include large CNS diseases. We are initiating our expanded efforts in large CNS diseases with research programs focused on Alzheimer's disease and temporal lobe epilepsy, and we have the opportunity to explore additional large indications in the future. Passage Bio was founded with the vision of developing transformative therapies for patients with devastating CNS disorders that have limited or no treatment options, and we have assembled one of the leading genetic medicine pipelines to help these patients.

We are initiating our extended efforts and large CNS diseases with research programs focused on all timer's disease, and temporal lobe epilepsy, and we have the opportunity to explore additional large indications in the future.

Passage buyout was founded with division of developing transformative therapies for patients with devastating CNS disorders that have limited or no treatment options. We have assembled one of the leading genetic medicines pipelines to help these patients.

As many of you know our co founder and former chairman of the board Dr. Touchy Armada passed away suddenly. This summer we are proud to continue his vision and legacy with the recent appointment of our new Chairman woman of the board vaccine gallon.

Bruce Goldsmith: As many of you know, our co-founder and former chairman of the board, Dr. Tachi Yamada, passed away suddenly this summer. We are proud to continue his vision and legacy with the recent appointment of our new chairperson of the board, Maxine Gowin. Chachi's vision is also carried by our seasoned leadership team, complemented by the recent hiring of Simona King as CFO, Mark Forman as CMO, and Maria Tornson as CCO. We believe we have the resources and expertise needed to execute and propel us forward on our path to becoming a leading CNS Genetic Medicines company. With that, I will now turn it over to Chief R&D Officer Eliseo Salinas, who will discuss our clinical programs in more detail.

<unk> vision is also carried by our season leadership team complemented by the recent hiring of Simona King CFO, Mark Forman as CMO and Maria towards as CCL. We believe we have the resources and expertise needed to execute at propel us forward on our path to becoming a leading.

C N S genetic medicines company.

With that I will now turn it over to Chief R&D Officer, Eliseo, Salinas, who will discuss our clinical programs in more detail.

Thank you Bruce.

Eliseo Salinas: First, I will start by discussing our progress with site activations and patient identification efforts for our three lead programs. Passage Bio is in a unique position with each of our three clinical trial programs operating globally, which allows us to be where the patients are. In total, we have an international network of clinical sites at various stages of activation across nine countries in three continents. Despite COVID-19 continuing to impact hospitals and clinical sites across the world, we have activated six sites in the last three months, including two international sites, and we plan to open additional sites in the U.S. and in eight other countries in the next few months.

First I will start by discussing our programs with slight deviations in patient identification airports.

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Each of them was Greek medical file programs hopefully the go button.

Which allow us to be one of the patients are.

The total we have an international miserable.

Both sides of various stages of activation across nine countries.

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Despite COVID-19, continuing to impact Husky does I'm gonna need both sides are caused the worlds we have activated six sites in the last three months, including two international sides and we plan to open additional sides in the U S and eight although coming please.

The next few months.

It just because of all international do need to file a brute I was focused on activating multiple do both sides in parallel and while I'm going patient identification initiatives that we anticipate having patients goes do you know who you're talking about bringing him before the end of the year.

Eliseo Salinas: It is because of our international clinical trial approach, our focus on activating multiple clinical sites in parallel, and our ongoing patient identification initiatives that we anticipate having patients dosed in all three of our clinical programs before the end of the year. The initiatives I'll point to as examples of our extensive patient identification efforts include, first, our collaboration with a broad network of disease experts and community leaders, and second, our partnerships with service companies offering free genetic testing and counseling for the three disease areas targeted by our LEAD program.

Then you should just I'll point to as examples of all extensive station identification and forced to include first what collaborations with the grilled networks of disease experts and community leaders and second what partnerships with service companies offering is sweetened that'd be best in and count.

So do we need these these areas targeted by our meat programs.

Do you have the desk, you just particularly important as we ask Hudson positions that this is the one of the critical Darius too early diagnosis.

Eliseo Salinas: Genetic testing is particularly important, as we have heard from physicians that this is one of the critical barriers to early diagnosis. As part of our commitment to patient and physician communities, we will continue to explore ways of reducing this barrier globally, for example.

I spotted by work commitment patient and physician communities, we will continue to explore ways of reducing this barrier GUL button.

For example, we.

Eliseo Salinas: We are working to support early and accurate identification of newborns with Crabier disease. Early diagnosis is critical because of the rapid decline of patients with this devastating disease. Much of our work in this area has been in partnership with advocacy organizations working state by state to encourage statewide adoption of newborn screening requirements.

We are working to support early an accurate identification of newborns with <unk> disease.

And I'll do diagnoses, it's great to call because of the rapid decline of patients with Disney was thinking disease.

Multiple will work in this area has been in partnership with advocacy organizations working state by state to encourage statewide adoption of newborn screening requirements.

We are pleased with our problem is I need to find patients no. That's what we are doing now will be instrumental they'll do the ongoing success, although a global multi G. Okay programs.

Eliseo Salinas: We are pleased with our progress in identifying patients and know that what we are doing now will be instrumental to the ongoing success of our global multi-year clinical program. Now, I will provide a brief update on each of our clinical programs. Our PBGN01 program utilizes a next-generation proprietary AAVH-U68 capsid to deliver a carbon-optimized GLD1 transgene to increase beta-galactosidase enzyme activity in the brain and peripheral tissues.

Now I will provide a brief update on each of our technical programs.

I want B B G N O. One program utilizes a next generation proprietary a V. A huge <unk> capsid to deliver a column optimize G. L. D. One time Jean.

Please be that got like to say these things I'm activity in the brain and three grilled dishes.

We're focused on the income that was foremost G. One blank I'm just like those is would you. The most of the appointment disease with a very rapid Dizzy schools Ah no current treatment options beyond supported can.

Eliseo Salinas: We're focused on the infantile form of GM1 glandiocytosis, which is the most severe form of the disease with a very rapid disease course and no current treatment options beyond supportive care. The IMAGINE-1 Global Phase 1-2 trial is an open-label, dose-escalation study that will enroll four distinct cohorts divided by age and dose level. The first cohort is composed of late-onset infantile patients receiving the initial dose of PBGN01. I am pleased to report that we now have four active clinical sites open for enrollment and plan to open additional sites in the U.S. and in four other countries in the next few months. We also remain on track to report first human data from cohort one later this quarter.

Imagine one global phase one to try I'll use an open label dose escalation Stabby will four distinct college divided by age and dose level.

The first Gulf War is composed of link onset and some validations receiving the initial dose B B G. M O one.

I am pleased to report that we now have four active two sides open for enrollment and clung to open additional sides <unk> and <unk> <unk> <unk>.

In the next few months.

We also remain on track to report first need to Monday depth <unk>, one laser disc water.

The primary goal of course, no one is doing.

Eliseo Salinas: The primary goal of Cohort 1 is to assess safety and tolerability, allowing for this escalation and progress into the early infantile course. The data we plan to report will include initial safety information, including results from NERSC conduct studies, as well as beta-gallons and activity levels in CSF and serum at date 30. We also plan to share the IBMC's assessment of progressing the study to the early infantile and higher dose late infantile cohorts, which may be enrolled in parallel.

Safety in Colorado alone for those is Kelly from them progress <unk> and some <unk> cohorts.

The date that you'd want to report will include initial safety information we.

We sold for most conjunction status as well as beat that Gollum Donna D V D levels D C at Stefan serum update 40.

We also plan to should I be and soon as assessment on progressing to study the earliest on file and hard those late and sometimes cohorts, which may be enrolled in town.

We have also my cigarettes, you can put earliest enrolling patients into perspective G. M. One not too Oh, she's still study with more than 10 patients now enrolled.

Eliseo Salinas: We have also made significant progress enrolling patients in the prospective GM1 natural history study, with more than 10 patients now enrolled. This study will provide important data to improve understanding of overall disease progression and meaningful outcome measures. We look forward to discussing the imaging 1 data with you before the end of this year.

This study will provide important data to improve understanding overall disease progression a meaningful outcome measures.

We look forward to discussing the image you one data with you before the end of this year.

Moving on to the global P. B T R three programs and crabby disease Cold Galaxy.

Eliseo Salinas: Moving on to our global PBKRO3 program in cardiac disease called GALAX-C. Cravet disease is a condition that progresses rapidly, damaging both the brain and the peripheral nervous system and resulting in a life expectancy of only two years in the severe case. So far, we have activated two sites and are making significant progress in opening additional sites in the U.S. and in four other countries, paving the way for us to dose the first patient in the fourth quarter of 2021. GALAXY is an open-label dose escalation study of PBKR03 in patients with early infantile cardiac disease.

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Paving the way for us to do the first station pulled this study in the fourth quarter of 2021.

Galaxy is an open table those escalations Daddy of P. B K out of a tweak in patients with early on file cabinet disease.

Eliseo Salinas: The study will run similarly to our Imagine 1 trial, first evaluating an initial dose of PBKR03 in late-onset patients and then progressing into early-onset and high-dose cohorts after an assessment of Cohort 1. Additionally, there will be a confirmatory expansion cohort for both age groups once the dose escalation phase of the study is completed. The main goal of the study is to assess the safety and tolerability of ascending doses of PD-KRO3 in patients with early infantile Krabi disease, as well as to assess the impact of GAL-C on CSF and serum.

You gotta be with one similarly, do our email one trial.

First evaluate the initial dosage P. T O tweet late onset patients and then progressing into early all set on high dose <unk> ask you are in the system and of course one.

Additionally, there will be a confirmatory expansion gulfport for both age groups. Once the bills is condition phase of the study is completed.

The main goal of the salaries to assist in safety until the ability of offending doses P. D. K O three in patients with early and <unk> <unk> <unk> <unk> <unk> <unk> <unk> <unk> <unk> <unk>.

Eliseo Salinas: Turning to our third program, for Frontal Temporal Dementia with Granulomycase. STD is one of the more common causes of early-onset dementia where there is impermanent behavior, language, and executive function and occurs at a similar frequency to Alzheimer's disease in patients younger than 65 years old.

Starting to have a search program.

D S. P O two who frontotemporal dementia with grunting mutations.

Excuse me is one of the more common causes of early onset dementia wood. There is an apartment in behavior language Unexcited dysfunction and of course, a similar sequence to do all that and much disease in patients younger than 65 years old.

Rapid progression of Ft view results you can never at survival of a G. S. After one set of symptoms.

Eliseo Salinas: The rapid progression of FTD results in an average survival of eight years after the onset of symptoms. We are specifically focused on STD granule, where the disease occurs because of mutations in the granule gene causing a deficiency of progranules. It is estimated that about 5-10% of SQD is caused by a granule mutation.

We have specific comes with <unk> with the disease of course because of mutations Gwendolyn Jim calls me at the <unk> <unk>.

It is estimated that about 5% to 10% of 50 D is caused by a grandma's mutation.

Eliseo Salinas: Our Global Phase I-II Clinical Trial, APLISP-V, is an open-label dose escalation study of PBFTO2 in FPD granulin patients. So far, we have received regulatory authorizations to initiate clinical trials in both the United States and Canada, and we expect three additional country authorizations in the next three months. We plan to enroll two co-hosts, three patients each receiving two different ascending doses of PVFDO2, with an option for a third cohort treated with a higher dose, depending on safety and enzyme results observed in the first two cohorts.

Our global saves one two clinical trial I believe it was an open label those Escalations tabby C. B S. C O two E S. P D grandly patients.

So far we have received regular until you have legislation resupplies I'll try I'll see those neither states and Canada, you expect to be additional counter yep, Oh sensations in the next three months.

We plan to go to calls three patients at risk.

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Eliseo Salinas: We have opened our first clinical trial slide and are, as we said, on track to dose the first patient in the fourth quarter of this year. We also have made significant progress advancing site initiation activities at other sites and expect to open those sites soon. Like our other lead programs, our key goals are to assess the safety and durability of ascending doses of EDFTO2, as well as its impact on programming. In addition to our progress with our three lead clinical programs, we're making significant strides with our earlier stage pipeline programs and our new discovery research efforts in temporal lobe epilepsy and Alzheimer's disease.

We have opened our first can you tell tall side and as you said on track two bills before space in the fourth quarter of this year.

We also have made significant progress advanced insight initiation like D V D. At other sides unexpected to open those sides soon.

Like our hold on the programs what key goals are to US says the safety and the ability of ascending doses.

C B S P O T as well as its impact on programming levels.

In addition to our progress would that went through three you can think of programs, where making significant strides with our earlier stage five blend program and I want a new discovery research and 14 temporal lobe epilepsy and old timers disease.

Eliseo Salinas: We look forward to sharing more about these efforts in 2020. Advancing programs for CNA diseases requires tremendous support from a variety of stakeholders, and I would like to conclude by recognizing and thanking the caregivers, health care providers, advocacy organizations, and patients in the GM1, carbid disease, and STD communities. With that, I will now turn the call over to Simona to discuss our finances.

We look forward to sharing more about this it was in 2022.

I've always been program for C. N N diseases requires tremendous support from a variety of stakeholders.

And I would like to conclude the break up nice and I'm thinking that could give us.

Health care providers advocacy organizations and patients in the gym, one COVID-19 disease, an S U V communities.

Simona King: Thank you, LSAO. I would like to start by saying how pleased I am to be part of the Passage Bio team. I am honored to work alongside a group of dedicated and passionate leaders who are committed to bringing transformative medicine to patients and families whose lives are impacted by CNS disorder. As we reported in our press release this morning, we ended the quarter with approximately $354 million in cash, cash equivalents, and marketable securities, as compared to $305 million as of December 31, 2020.

With that I will not turn the call over to <unk> to review our financials.

Thank you Alex a L. I would like to start by saying help police Diane to be part of the passage of I O T. I am honored to work alongside a group of dedicated and passionate leaders who are committed to bringing transformative medicine to patients and families whose lives are impacted by C. N S. <unk>.

Orders.

And can we reported in our press release. This morning, we ended the quarter with approximately $354 million in cash cash equivalent and marketable securities as compared to $305 million at that December 31st 2020, Alright.

Simona King: RMD expenses were $26.6 million for the third quarter ended September 30th, compared to $20.8 million for the same quarter last year. The increase was primarily due to $3.1 million in clinical development and professional services expenses, $2.7 million in personnel-related expenses resulting from an increase in employee headcount, $1.1 million in clinical manufacturing expenses, and $1.2 million in facility and other expenses. These increases were partially offset by a $2.3 million decrease in research and development expenses associated with the PEN agreement, which relates to expenses incurred in the three months ended September 30, 2020, for preclinical work performed in preparation for IND filings for our LEAD program.

Alright, Andy expensive $26 $6 million for the third quarter ended September 30th compared to $28 million for the same quarter in 2020 the.

The increase was primarily due to 3.1 million dollar and clinical development and professional services expense at $2.7 million and personnel related expenses, resulting from an increase in employee head count $1.1 million and clinical manufacturing next benefit.

And $1.2 million and facility and other expenses deep.

These increases were partially offset by a 2.3 million dollar decrease in research and development expenses associated with the Penn agreement, which relates to expenses incurred in the three months ended September 30th 2024 preclinical work performed in preparation for IMT filings are are.

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Acquired in process, R&D expenses or $5.5 million for the third quarter and that's September 30th compared to zero expensive in the same quarter of 2020.

Simona King: Acquired in-process R&D expenses were $5.5 million for the third quarter ended September 30th, compared to zero expenses in the same quarter of 2020. We incurred $0.5 million in license fees and $5 million in fees related to the August 2021 amendment with Penn, which extended our partnership by one year and extended our rights to large CNS indications. G&A expenses were $15 million for the third quarter ended September 30th, compared to $7.8 million for the same quarter in 2020.

We encourage that zero point $5 million and license fees and $5 million in fees related to the August 2021 Amendment with pen, which extended our partnership and buy one year and expanded our right into large CNS indications.

GMA expenses were $15 billion for the third quarter ended September 30th compared to $7.8 million for the same order in 2020.

Simona King: The increase was primarily due to $5.3 million in personnel-related and share-based compensation expenses resulting from an increase in employee headcount. Professional fees and other expenses also increased $1.8 million as we expanded our operations to support our research and development efforts and incurred expenses associated with operating as a public company. The net loss was $46.9 million for the third quarter of 2021 compared to $28.5 million in the same quarter of 2020. The net loss per basic and diluted share was $0.87 in the third quarter of 2021 compared to $0.63 in the third quarter of 2020.

The increase was primarily due to $5.3 million and personnel related and shared based compensation expenses, resulting from an increase in employee headcount prep.

Professional fees and other expenses also increased $1.8 million as we expanded our operation to support our research and development effort and incurred expenses with operating as a public company.

Net loss plus $46 $9 million for the third quarter of 2021 compared to $28.5 million in the same quarter of 2020 net loss per basic and diluted share with 87 cents in the third quarter of 2021 compared to 63 cents and the third call.

Of 2020.

Simona King: As we continue to advance our pipeline and invest in our capabilities, we are supported by the strength of our balance sheet. We expect our current cash, cash equivalents, and marketable securities to fund our operations for at least the next 24 months. I will now turn it back to Bruce for closing remarks.

And can we continue to advance our pipeline and invest in our capabilities. We are supported by the strength of our balance sheet. We expect our current cash cash equivalent and marketable securities to fund our operations for at least the next 24 months let.

Let me now turn it back to Bruce for closing remarks.

Thank you Simona Ah.

Bruce Goldsmith: Across our three lead programs, we are extremely proud of the accomplishments of our passionate team at Passage Bio. Our clinical operations team has been tireless in their efforts to open clinical trial sites around the world. We also continue to invest in our in-house CM&C capabilities. This investment includes expanding our manufacturing team, who have made significant progress since opening our new CM&T laboratory in Hopeville, New Jersey last June. This group has been instrumental in developing and advancing differentiated analytics using the latest in technologies and methodologies. Our team's work builds upon GTP's world-class vector analytical method.

Across our three lead programs, we are extremely proud of the accomplishments of our passionate team at passage bio are clinical operations team has been tireless in their efforts to open clinical trial sites around the globe.

We also continue to invest in our in house <unk> capabilities. This investment includes expanding our manufacturing team who have made significant progress since opening our new <unk> Laboratory and hope out New Jersey last June.

This group has been instrumental in developing and advancing differentiated analytics using the latest in technologies and methodologies.

Our teams work builds upon gtp's world-class vector analytical methods.

Our patient engagement team has also built productive partnerships with the advocacy community, which has helped tremendously in the raising awareness of our clinical trial programs Wheeler.

Bruce Goldsmith: Our patient engagement team has also built productive partnerships with the advocacy community, which has helped tremendously in raising awareness of our clinical trial progress. We look forward to delivering on multiple clinical milestones over the next few months, including reporting initial safety and 30-day biomarker data for the Phase 1-2 trial for infantile GM1, as well as dosing the first patients in our trial for FTD-GRN and our trial for early infantile Crab-AIDS. In the first half of 2022, we also look forward to reporting initial safety and 30-day biomarker data from our FTD and CRAB-A clinical trials.

We look forward to delivering on multiple clinical milestones over the next few months, including reporting initial safety and 30 day biomarker data for the phase went to trial for infantile G. M. One as well as dosing the first patients and our trial for F. T. D. G. R. N N R trial for early infantile crabby disease.

In first half 2022, we also look forward to reporting initial safety and 30 day biomarker data from our F T D and krabbe a clinical trials.

Before taking questions I'd like to thank the incredible passage by our team and Jim Wilson and his team at G. T. P for their hard work, bringing are clinical trials to the point, where they are today.

Bruce Goldsmith: Before taking questions, I'd like to thank the incredible Passage Bio team and Jim Wilson and his team at GTP for their hard work bringing our clinical trials to the point where they are today. One of the benefits we have at Passage Bio is being able to draw on the deep scientific expertise of GTP, a world-renowned organization unmatched in its pioneering research contributions to AAV gene therapy. We also want to recognize our partnership with caregivers, health care professionals, scientists, and advocacy groups who share and support our vision to transform the lives of patients impacted by devastating CNS disorders. With that, I would like to open the call to questions, Operator. As a reminder, to ask a question, you will need to press...

One of the benefits we have a passage bio is being able to draw on the deep scientific expertise of G. T. P. A world renowned organization unmatched in its pioneering research contributions to a V gene therapy.

We also want to recognize our partnership with caregivers health care professionals scientist and advocacy groups, who share and support our vision to transform the lives of patients impacted by devastating CNS disorders.

With that I would like to <unk> open to call up for questions operator.

As a reminder to ask a question you want need to press star one on your telephone too.

Operator: As a reminder, to ask a question, you will need to press star one on your telephone. It's Romero. Your line is now open.

Yeah.

Romero.

Your line is now open.

Hi, good morning eyes. Thanks her color here.

Tessa Romero: Hi, good morning guys. Thanks for all the color here.

So my question is around the F. D D T G R N and <unk>.

Tessa Romero: So my question is around the FDD-GRN and CRABAE studies. You made some comments on site activation progress for CRABAE and regulatory authorizations for FDD-GRN. Can you lay out for us where you are in the process in terms of sites opening, screening, enrollment, or dosing for each of these studies?

You made some comments on site activation progress for krabbe by and the regulatory authorization for F. D. T. T. R. M. Can you lay out trash, where you are in the process in terms of sites opening screening enrollment or dosing for each of these studies.

Bruce Goldsmith: Yes, Tessa, thanks very much for the question. Yeah, we're making great progress with COVID-19. We've been very, I think, open in the past and transparent about, you know, the challenges that COVID first had and then the, I think, the lingering kind of operational challenges just working with sites as the number of studies post-COVID is kind of ramping up. We've been very successful at moving some of the studies forward. I think we have four sites open with the GM1 program, two with CRAB-A, and we do have one site open, which is the University of Pennsylvania for the frontal temporal dementia program, and we anticipate additional sites opening in the near term as well.

Yeah.

Yes, Tessa thanks very much for the.

The question.

We're making great progress amid COVID-19, we've been very I think open in the past and transparent about the challenges that COVID-19.

First had and then I think the the lingering kind of operational challenges just working with cites as the as the number of studies of post Covid is kind of wrapping up we've been very successful at moving some of the studies forward I think we have four site site to open with the G. M. One program to with crab.

And we do have one site open which is the university of Pennsylvania for the frontal temporal dementia program and we anticipate.

Additional sites opening in the near term as well. So we've had we've had a great deal of progress on the site activation. You also asked about that kind of patient identification and maybe I will say you could come at a little bit about the various programs in terms of how we are identifying patients in collaboration with the sites and other other groups.

Bruce Goldsmith: So we've had a great deal of progress on the site activations. You also asked about patient identification, and maybe I'll say you could comment a little bit about the various programs in terms of how we're identifying patients in collaboration with the sites and other groups.

Yes so.

Eliseo Salinas: Yes, so in all the programs, we have a vast network of physicians, experts, advocacy organizations, and patient organizations to identify new patients. It's different in the two pediatric leukodystrophies than in FTD, but essentially, there are strong similarities.

You know the programs, we have a vast network of.

Physicians experts advocacy organizations and patient organizations to identify new patients it's different in the two pediatric Luca these trophies.

Then an F T V, but essentially.

There are strong similarities so we're very well connected with oldest those <unk> and now the the Red leave me do step for us is not.

Eliseo Salinas: So we are very well connected with all those networks, and now the rate-limiting step for us is not the identification of patients; the rate-limiting step now for us is opening the sites that we are opening, as Bruce said. One quick reminder that, you know, when you open sites for a study like this one, it's not an arithmetic function with a constant rate; it's a sigmoid function. The first part is

Division of Asian, originally be to step now for that just opened and besides that.

Openness Bruce said, one quick reminded when you open sides for a study like this one is not in order to submit the function with that comes down to rate is is a sigmoid function. The first part is exponential. So we open during the first part of the year one side for <unk>.

Eliseo Salinas: So we open, during the first part of the year, one site for GM1. We opened six in the last four months, and we plan to open more than twice that number in the next four months. So we're very happy with the progress, and we think that we are in good shape to enroll in those trials. Great. Thanks so much for taking our questions, guys. Thanks, Tessa.

One we open six and the last four months and we plan to open more than twice that number in the next four months. So so we're very happy with the progress and and we think that we are in good shape doing really those trials.

Great. Thanks, so much for taking our questions.

Nina Petrito-Gard: Thank you. Our next question comes from the line of Nina Petrito-Gard from Citi. Your line is now open. Bye.

Operator.

Thank you. Our next question comes from the line Nina <unk>.

<unk> Guard from city. Your line is now open.

Nina Petrito-Gard: Thanks for taking the question. So I just wanted to ask a question about timing for the FTD-TRN data, just so I make sure that I understand. I know there are 60, kind of 60 days between the dosing of the patients, and then we should get the first 30 days of biomarker data 60 days after the third patient is dosed, I believe. So if I do the math on that, I think I'm getting close to like a June-July kind of timeframe for the first data, just given that there are three patients in that first cohort. I guess just help me understand if that's kind of the right way to think about it.

Hey, guys. Thanks for taking the question. So I just wanted to ask a question about timing for the F. T. D. TR N data, so I make sure that I understood and I know, we there's 60 60 days between dosing of the patient and then we should get kind of the first.

30 day biomarker data 60 days after the third <unk> I believe so if I do the math out on that I think I'm getting close to like June July kind of time frame for that first Donna just given that there's three patients in that.

Bruce Goldsmith: You're Matt, and I'm Nina, and thanks for the question. And your math is absolutely correct. So there is an interval between each patient, and that's kind of FDA mandated so that we do have to do, you know, so-called sentinel patients where you dose one at a time with gene therapy. You know, as we, just as an aside, as we create a safety database, there's always an opportunity to interact with FDA and see if the accumulated safety could accelerate that, but you're absolutely right, as we've disclosed in all our slides, there is an interval.

First cohort Uhm I guess, just help me understand if that's kind of the right way to think about it.

You are.

I need to and thanks for the question and your math is absolutely correct. So there is an interval between each patient and that's kind of FDA mandated so that we do have to do.

So-called sensor locations, where your dose one at a time with gene therapy.

As we just as an aside as we do create a safety database, there's always an opportunity to interact with FDA and see if.

Cumulative safety could accelerate that but you are absolutely right as we can disclose and all our slides. There is an interval and you are also absolutely right on the math that we do expect it to be late in the first half of 2022 and as we have in the past as we enter 2022, I will certainly update and as we enroll the first patient.

Bruce Goldsmith: And you're also absolutely right on the math that we do expect it to be late in the first half of 2022, and as we have in the past, as we enter 2022, we'll certainly update, and as we enroll the first patient that we anticipate by the end of this year, we will update timelines as appropriate, and, you know, if that moves back because of the kind of mandated timelines, we'll certainly be transparent in that, but we certainly also believe we can meet the first half 2022, but you're absolutely right that it'll be late in the half.

That we anticipate by the end of this year, we will update timelines is appropriate and if that moves back because of the kind of mandated timelines will will certainly be transparent and that but we certainly also believe we can meet the first half of 2022, but you are absolutely right that it'll be late in the in the App.

Okay perfect. Thank you so much.

Nina Petrito-Gard: Okay, perfect, thank you so much. Thank you, thank you. Our next question comes from the line of Laura Chico from Wedbush. Your line is now open.

Thank you.

Thank you. Our next question comes from the line of Laura Chico. So glad Bush. Your line is now open.

Laura Kathryn Chico: Hey, good morning guys. Thanks for taking the questions. I guess I'd like to drill into the site activation comments a little bit more. You mentioned, if I heard correctly, that six sites were activated in the last three months. And on clintrials.gov, it looks like across the three studies, there are six recruiting sites. So I guess I wanted to understand, are the constraints on site activation improving? And really trying to understand what the key barriers are actually moving towards getting a recruitment started. And then I have one quick follow-up. Thanks.

Hey, good morning, guys. Thanks for taking my questions I guess I'd like to drill into the site activation comments, a little bit more you mentioned, if I heard correctly six sites, where activate in the last three months and by Colin trials that kind of it it looks like a cross as three studies. There are six recruiting sites. So I guess I I wanted to understand <unk>.

Are the constraints on site activation, improving and really trying to understand what the key barriers are actually moving towards getting a recruitment started and then I have one quick follow up thanks.

Bruce Goldsmith: Sure. Thanks, Laura, and I appreciate the question. So it is an operational consideration. It has to do with, you know, getting budgets back from the sites, and you'd think that there would be an opportunity to accelerate that because it's a budget, but in fact, any budgets actually come very late in the process because if there are any considerations by the individual reviews, then they have to be incorporated, et cetera, and there are multiple departments in each hospital that we're

Sure, Thanks, Laura and and I appreciate the question.

So it is an operational consideration it has to do with.

Getting budgets back from the sites get and you would think that there would be an opportunity to accelerate that because it's a budget, but in fact any.

Budget's actually come usually very late in the process because if there are any considerations by the individual reviews, then they have to be incorporated et cetera, and there's multiple multiple departments in each hospital that we're dealing with for example, the pharmacy is is one of them and it really does come back down to operational considerations.

Bruce Goldsmith: For example, the pharmacy is one of them, and it really does come back down to operational considerations. The reason is that when we've done benchmarking and looked at the time to kind of open sites and enroll first patients, especially in gene therapy, it does, you know, we're not so far out of that realm, especially because these are academic centers. The first center that we opened for the GM1 study was a community center with a centralized IRB.

The reason, we've done benchmarking and looked at the.

Time to kind of open sites and enroll first patients, especially in gene therapy. It does we're not so far out of that realm, especially because these are academic centers. The first senator that we open for.

For the Jam one study was it was a community center with a centralized IRB. Each of these new centers are just taking a bit of time because of the the <unk> the budget negotiations et cetera.

Bruce Goldsmith: Each of these new centers is just taking a bit of time because of the IRBs, the budget negotiations, et cetera. So it really is down to that. You know, at the same time, we're not sitting kind of idly by as these sites are getting up and running. There's a lot of interaction between the patient engagement groups, the clinical operations team, and the clinical teams, and our leadership with each individual site to work with them to identify potential patients along the way. But, of course, it's a balance because until the site is open, patients can't be officially screened. Alassay, do you have any other comments, or is that fairly comprehensive? It is very comprehensive.

So it really is down to that at the same time, we're not sitting kind of idly by as these sites are kind of getting up and running there's a lot of interaction between the patient engagement groups. The clinic operations team in the clinical teams and our leadership with each individual site too.

Work with them to identify potential patients along the way but of course, it's a balance because until the site is open patients can't be officially screamed Jose do you have any other comments or is that fairly comprehensive.

It is very comprehensive, but I would add only want one thing.

Eliseo Salinas: It's very comprehensive, but I would add only one thing. If you want to plot a curve, you need three points. For the first six months of the year, we open one site, St. Peter's Community Hospital. For the last four months. Three months.

If you want to applaud occur three three points now the first six months of the year, we opened one side Saint Peters has become.

The community hospital the last four months three months, we open six sides.

Eliseo Salinas: We opened six sites, and in the next four months, we plan to open two or three times that number. So if you plot the three, you have a sigmoid function, which is exponential growth. So what happened in the beginning part of the curve does not predict the second part of the curve. What happened in the recent past, the six sites we opened in four months, that's predicted to continue to grow. And then we will reach the plateau, the number of sites we want. At that time, the rate-limiting step would be, you know, the availability and incidence of the patients. But now we are done.

And the next four months, we plan to open two or three times without number. So if you plug. The three you have a sigmoid function, which is an explanation growth. So what happened in the beginning part of the curve does not predict the second part of the good what happened in the recent past six sides. We open at four months.

That's predictive continued to growth and then we will we will reach the plateau a number of sites. We won at that time, the readily be to step would be nobody.

<unk> of the patients, but now we were done yet.

Bruce Goldsmith: Yeah, and I guess, as Alastair was speaking, the other thing we are doing is working across the globe. So these are not just U.S. sites. We have sites now in the U.S. and Canada. We've been very open and transparent on clinicaltrials.gov that we're also opening sites in Brazil, the U.K., Europe, and the Middle East. And you know, there are individual complexities for each review of gene therapy, especially gene therapy that creates some challenges, et cetera.

Guess.

I'll say it was speaking and the other thing we are doing is working across the globe. So these are not just use sites. We have sites now in the U S and Canada, we we've been very open and transparent unqualified dot Gov that we're also opening sites in Brazil, the UK Europe, and the middle East and.

There are individual complexities for each.

Review of the.

The gene therapy specially.

That creates some challenges et cetera. So have we were very pleased with the with the first sites that are that are outside of the United States being opened as well. So all of that is coming together to say that.

Bruce Goldsmith: So I mean, we're very pleased with the, you know, with the first sites that are outside the United States being opened as well. So all of that's coming together to say that we very strongly believe that we're in a great position and we have probably the biggest network in these studies, we believe, and we're very proud of that.

Very strongly believe that we're in great position and we have the probably the biggest network in these studies.

We believe and we're very proud of that.

Operator: That's super helpful.

Alright, you're very helpful.

Laura Kathryn Chico: Yeah, you had a follow-up, please.

Please yeah, if you don't mind, it just kind of related more to imagine one and obviously, it's early in the process for recruitment, but I'm just curious about screening failures and are there any aspects of the inclusion exclusion criteria that might be impacting recruitment thus far thanks guys.

Laura Kathryn Chico: Yeah, if you don't mind, it's kind of related more to Imagine One, and obviously, it's early in the process for recruitment, but I'm just curious about screening failures, and are there any aspects of the inclusion-exclusion criteria that might be impacting recruitment thus far? Thanks, guys.

Thanks, So I.

Bruce Goldsmith: Thanks Laura. So I don't believe it's the, well, let's be careful about the definition. So for each of these studies, there are age limits that, and also developmental stage and developmental milestones that are required because of both the interaction with the regulatory agencies, interaction with the community of advocacy folks that have had input in these studies, and also, of course, the treating clinicians.

I don't I don't believe it's the well so let let's let's be careful about the definition. So for each of these studies there are in the pediatric population there are age limits that and also developmental stage.

And developmental milestones that are required of because of both the interaction with the regulatory agencies interaction with the community of advocacy.

Folks that have had input in their studies and also of course, the treating clinicians. So I don't want to say that there are no criteria that are affecting enrollment because.

Bruce Goldsmith: So I don't want to say that there are no criteria that are affecting enrollment because, you know, two things can happen to these children because they have really devastating diseases and an incredibly fast progression sometimes. So they may, you know, kind of have deficits that preclude initial entry into the study. They may be patients that we can hopefully address later on, but there are those limitations. And the second thing is, and this has been a focus and something that I alluded to earlier, as we're working with sites and advocacy groups and the testing programs in Vitae and Informed DNA to identify patients, there may be patients that are identified, but without sites open at the time, such as really early this year, patients could then age out and be too old for the studies.

Two things can happen to these children, because they have really devastating diseases and incredibly fast progression, sometimes so they may.

Good.

Kind of have.

Deficits that preclude initial entry into this study that may be patients that we can hopefully address later on but there are those limitations and the second thing is.

And this is this has been a focus.

Focus on something that I alluded to earlier as we're working with sites too and advocacy groups and the testing programs in VJ added form DNA to identify patients. There may be patients that are identified but without sites open at the time such as really early this year patients could then age out and be too.

Old for the studies, so I wanted to be very careful to say, it's not specifically entry criteria that are inappropriate that we believe are constraining enrollments and screening, but it's rather I think the consistent application of those across the patient population and the difficulty that the.

Bruce Goldsmith: So I want to be very careful to say it's not specifically entry criteria that are inappropriate that we believe are constraining enrollment and screening, but it's rather, I think, the consistent application of those across the patient population and the difficulty that these patients face.

Patients face.

That's helpful. Bruce Thank you.

Laura Kathryn Chico: That's helpful, Bruce. Thank you. Thank you.

Thank you.

Thank you. Our next question comes from the lineup you song from BT Igene. Your line is now open.

Operator: Thank you. Our next question comes from the line of Yun Zong from BTIG. Your line is now open.

<unk> from the Gaijin. Your line is now open please start hearing about.

Yun Zong: Your line is now open. Please check your mute button. Yeah, I forgot to mute myself. Apologies.

I forgot.

Myself I apologize and so on the three day by market data that you'd expect to reported in fourth quarter R.

Yun Zong: And so on the 30-day biomarker data that you expect to report in the fourth quarter, are you expecting to report the substrate reduction as well? And also on the beta-gal in the CSF, I believe other companies have talked about the challenge of quantification in the CSF. So can you talk about the comfort level that you believe that the data will accurately reflect how much enzyme you're producing in the CSF, please?

Are you expecting to report the substitute production as well and also on the right account in the CSF I believe.

Other companies have talked about the challenge and.

Quantification in the CSS so.

Can you talk about the comfort level that you believe the data will.

Currently reflect how much and some of your produce an assistant please.

Bruce Goldsmith: Thanks for the call and for the question and joining the call. So, what we've committed to, and we've been, I think, very transparent in doing this, disclosing the beta-galactosidase enzyme activity in CSF and serum and, of course, the safety, which is the principal endpoint, so that we can move on to higher doses and also include earlier stage, younger age patients. The substrates we've been a little bit careful because although other companies have disclosed this, we want to make sure that we look at the pharmacodynamics and understand the longitudinal view of that, and the same comments apply to, for example, clinical endpoints.

Thanks for the for the question and joining the call.

So what we've.

Committed to is and we've been I think very transparent and doing this is disclosing the betagalactosidase enzyme activity and CSF in Sierra and of course, the safety, which is the principal.

And so that we can move on to higher dosing and also two including earlier stage there earlier AIDS patients.

The substrates, we've been we've been a little bit careful because.

Although other companies have disclosed this we want to make sure that we look at the pharmacodynamics and understand the longitude no view of that and the same comments apply to for example, clinical endpoints. So we have not committed to disclosing that at this point, but we really focused on CSS.

Bruce Goldsmith: So we're not committed to disclosing that at this point, but we've really focused on CSF. Well, obviously, as we move forward, you know, continue to think about this, and then your second question was kind of about the measurement of serum and CSF. And Alessio, do you want to comment on that and Yoon's comment or question? Yes. Admittedly, it's a challenging asset to develop.

Well, obviously as we move forward continue to to think about this.

And then your second question was kind of on the measurement of serum and and CSF.

And I will say or do you want to comment on that and comment or question.

Admittedly it is a challenging asset to develop.

Eliseo Salinas: Yes, admittedly, it's a challenging asset to develop. You know, some sponsors have struggled with that. As you know, the concentrations of the CSF are much smaller, so it requires a very sensitive but also very specific test. We're confident with the test we have developed, and because of the relevant administration and the type of disease we're dealing with, we think that that's very, very important.

Some some sponsors have struggled with that.

As you know the the concentrations of the CSA much smaller so.

Wire in a very a very sensitive but also very specific days.

We're confident with the test we have develop and because of the route of administration and the type of disease, we're dealing with.

Think that that's very very important.

Operator: Thank you. Our next question comes from the line of Daniel Brill from Raymond James. Your line is now open.

Thank you. Our next question comes from the lineup den Umbriel for Raymond James Your line is now open.

Daniel Brill: Hi guys, good morning and thanks for the question. So, sorry if I'm beating a dead horse here, but I just wanted to clarify for the FTD program specifically, are you actually screening and identifying patients now that you've got some sites open? And then, if I understand correctly, it sounds like you'll update timelines when you enroll the first patients in these studies. I wonder if you'll provide an update when subsequent patients are also enrolled?

Hi, good morning, and thanks for the question, so sorry, if I'm, beating a dead horse here, but I just wanted to clarify the television programs. Specifically are you actively screening in identifying patients now that you've got some sites open and then if I understood correctly. It sounded like you'll update timelines when you enroll the first patients in the study is wondering it.

Yeah, I'll I'll provide an update when like subsequent patients are also enrolled.

Daniel Brill: Thanks, Danielle. So, yeah, I appreciate the question. So, we have focused on sites such as the University of Pennsylvania that have relatively intact and large numbers of patients that are being actively either monitored or tracked and followed from a family perspective, from an FTD perspective. And so other sites are similar to that, with various degrees of numbers of patients that are already in the treatable system, for example. But we're supplementing that by also supporting informed DNA from a genetic screening and counseling perspective.

Thanks, Thanks that now so.

Yeah I appreciate the question so.

We have.

Focused on sites such as the University of Pennsylvania.

That have a relatively.

Intact and large comparatively.

Patients that are being actively either monitored or.

Attract and followed from our family perspective from from an FTB perspective.

And so other sites are similar to that with various degrees of numbers of patients that are already in the the treatable system for example, but.

But we're supplementing that by also.

Supporting and foreign DNA from a genetic screening and counseling perspective. So we are actively working as we have across all of our studies to identify patients.

Daniel Brill: So we are actively working, as we have across all of our studies to identify patients. We can't obviously bring patients into screening formally until the studies are open, but we can certainly and have, across the various sites and interactions with physicians, pre-identified patients that may be eligible. But obviously, the final determination is when they're actually at the site. So that's a little bit of a combination answer to your question.

We couldn't obviously bring patients into screening formally until the studies are open, but we can certainly in half across the across the various sites and interactions with physicians pre identified patients that may be eligible, but obviously the final determination as when they are actually have the site.

So that's a little bit of a combination.

Of an answer to your question.

Bruce Goldsmith: The other point I'll make is that there are large natural history studies, such as the GENPHY study and all the FTD studies, that we're also looking at those sites that have those relationships as well. So that is part of the patient population that is already kind of involved with the medical community and the sites that we're interested in, to an extent. To your second question, I think I said we may update this when we have the first patient dosed.

The other point I'll make is that there are large natural history studies such as the <unk> study in the all Std's studies that we're also looking at those sites that have those relationships as well. So that is part of the patient population that is already kind of involved with the with the medical community and the sites that were interested in <unk>.

An extent.

To your second question I think I said, we may update this when we have the first patient dosed, we typically update our timelines and think about this.

Bruce Goldsmith: We typically update our timelines and think about this when we, you know, enter into early 2022 and we set out our goals. So we'll have to look at the enrollment of the first patient and our estimation of other sites coming on. As Alessio said, we're making great progress on that. And then there is the projection of patient enrollment. So it's a combination of all of those things, and it may not be at the time of the first patient dose.

When we enter into early 2022, and we set out our goals.

So we'll have to look at at the enrollment of the first station in our estimation of of other sites coming on as a Seo said, we're making great progress on that and then the projection of patient enrollment. So it's a combination of all of those things and it may not be at the time of first patient dosed.

Bruce Goldsmith: And then I think the third part of your question is, are we going to update as we enroll individual patients? And what we've done in the past, as we did with the GM1 study, is announcing the first patient dose and then the target date of data disclosure. And we don't really want to get into the granular detail of each individual patient's timing because it just isn't an individual corporate goal. We're really interested in the enrollment of the first patient and data disclosure. And so that's what our approach has been.

And then I think the third part of your question is are we going to update as we enroll individual patients and what we've been doing in the past as we have with the GM. One study is announcing the first patient dosed and then the target date of data disclosure and we don't really want to get into the granular detail of each individual.

<unk> timing.

Because it just isn't it as an individual corporate goal.

Interested in the enrollment of the first patient and a data disclosure and and so that's what our approach has been.

Daniel Brill: That's really helpful. Thanks so much for clarifying. Thank you. Our next question comes from the line of Yaron Werber from Cowan. Your line is now open.

Necessarily have all thanks, so much for clarifying.

Thank you. Our next question comes from the line of year round Weber from Cower. Your line is now open.

Operator: Hi, thanks very much for taking the questions. This is Brandon on For Your Own.

Hi, Thanks, very much for taking the questions as it burned down for your own actually just a quick one on the earlier pipeline sorry, sorry, if I missed has been bouncing around a little bit, but I was actually wondering if we might be able to see some especially M. L. D. R.

Brandon: Actually, just a quick one on the earlier pipeline, sorry.

Brandon: on the earlier pipeline. Sorry if I missed it, it's been bouncing around a little bit, but I was actually wondering if we might be able to see some, especially MLD or ALS data, any of the preclinical data at all next year and maybe when we might be able to expect that and kind of how to think about it. Thanks.

S data any of the preclinical data at all next year and maybe when we might be able to expect that in and kind of kind of think about those things.

No. It takes for the answer to the question and I appreciate you being on the call. So.

Bruce Goldsmith: Thanks for the question, and I appreciate you being on the call. So again, we certainly are focused on that, and we understand the depth of our pipeline is, I think, probably one of the strongest genetic medicines pipelines in CNS that we are really proud of and continues to grow with not only those programs, which are the first seven, but also obviously additional options that we may exercise and then the addition of Alzheimer's and the TLA and potentially LARGE.

Again, we certainly are focused on that and we understand the the depth of our of our pipeline is is I think probably one of the strongest genetic medicines.

Pipeline and CNS.

That is that we are really proud of and continues to grow with the not only those programs, which are the first seven.

But also obviously additional options that we may exercise and then the addition of the Alzheimer's and the.

<unk> and potentially large.

Bruce Goldsmith: To go specifically to your question, we haven't specifically updated the timelines, but we may do so as we enter early 2022 and think about the data that is available and may become public. What we've said in the past is that our ability to share this information is really in partnership with the gene therapy program and Jim Wilson. As they advance and feel comfortable with the data that they're generating, then we feel comfortable sharing it, part of this is publications, and part of this is public disclosures. So I think it's a great question for us to consider as we enter 2022, and we'll maybe look forward to having that conversation as we enter the year.

Go specifically to your question.

We haven't specifically updated the timelines we may do so as we enter early 2022, and we think about.

The data that is available and may become public what we've said in the past is that our ability to share. This information is really in partnership with the gene therapy program and Jim Wilson as they advanced and feel comfortable with the data that they're generating that we feel comfortable than sharing it and part of this is.

Publications and part of this is public disclosures.

So I think it's a great question for us to consider as we entered 2022 and will will maybe look forward to having that conversation as we entered the year.

Alright, Yep makes sense okay.

Brandon: Alright, yep, that makes sense. Thanks guys.

Operator: Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect.

Thank you. This concludes today's conference call. Thank you for participating and May now disconnect.

BF-WATCH TV: BF-WATCH TV 2021

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