Q1 2020 Earnings Call

Good morning, and welcome to the passage bio first quarter 2020 financial and operating <unk> operating results conference call. At this time, all participants are in listen only mode.

Operator: Welcome to the Passage Bio First Quarter 2020 Financial and Operating Results Conference Call. 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. Please be advised that this call is being recorded at the company's request. At this time, I'd like to turn it over to Zoe Nita. Zoe, please proceed. Thank you, Operator.

Following the formal remarks, well open the call up for your questions.

Please be advised that this call is being recorded at the company's request.

At this time I'd like to turn it over to though we Nita Zoe. Please proceed.

[music].

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 I'd investors dot passage bio dotcom under the news and events section.

Zoe Nita: 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 at investors.passagebio.com under the News and Events section. On today's call, Bruce Goldsmith, President and Chief Executive Officer, and Rich Morris, Chief Financial Officer, will review our first quarter 2020 financial results and discuss recent business highlights. Gary Romano, our Chief Medical Officer, and Jill Quigley, our Chief Operating Officer, will also be available for the Q&A portion of the call.

On today's call Bruce Goldsmith, President and Chief Executive Officer, Enrich Morris Chief Financial Officer will review, our first quarter 2020 financial results and discuss recent business highlights.

Gary Romano, our Chief Medical Officer, and Joe quickly, our Chief operating Officer will also be available for acuity portion of the GAAP.

Before he began please note that today's call may include a number of forward looking statements, including but not limited to comment on.

Zoe Nita: Before you 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 the timing and execution of anticipated milestones, including 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 the underlying causes of their respective target monogenetic CNS disorders, manufacturing plans, and Trends with Respect to Financial Performance and Cash Flows, and the Company's Ability to Fund Research and Development Programs Impact of the COVID-19 pandemic on the company's operation and its ability to manage costs along with use 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.

Our expectations about timing execution of anticipated milestones, including initiation of clinical trial and the availability of critical data from such trials, our expectations about our collaborators and partners ability to execute key initiative the ability of our lead product candidate to treat the underlying causes a their respective target Mana genetics Deanna disorder Manny.

[music] factoring plans and strategies trends with respect to financial performance in cash flows the company's ability to fund research and development programs.

Impacts of the coping 19 Penta next on the company's operation 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 companys actual results to differ significantly from those suggested by these things.

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 FCC for information concerning factors that could cause actual results to differ materially from expectation, including any forward looking statements made on this call.

Zoe Nita: 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 its 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 statement to account for or reflect events or circumstances that occur after this call. It is now my pleasure to pass the call over to Bruce Goldsmith. Bruce.

Except as required by law the company disclaims any obligation to publicly update or revise any forward looking statement to account for reflect events or circumstances that occur. After this call. It has now my pleasure to pass the call over to preschool.

Bruce.

Bruce Goldsmith: Bruce Thank you, Zoe, and thank you all for joining us this morning. We are really excited to be here today to talk about the progress we have made since the start of the year. I will go into more detail in a moment, but since January, we successfully completed an upsized IPO to significantly improve our cash position and runway. We strengthened our board of directors and internal team across all functions to increase our capabilities, and also recently announced that we expanded our agreement with our partners at the University of Pennsylvania's gene therapy program headed by Dr. James Wilson, adding potential depth and breadth to our pipeline. Passage Bio was founded in late 2018 with the goal of truly helping patients suffering from serious, life-threatening, rare, monogenic central nervous system diseases.

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

We're really excited to be here today to talk about the progress we have made since the start of the year.

We'll go into more detail in a moment, but since January we successfully completed an upsized IPO to significantly improve our cash position and runway, we strengthened our board of directors and internal team across all functions to increase our capabilities.

Also recently announced that we expanded our agreement with our partners at University of Pennsylvania, as Gene therapy program headed by Dr., James Wilson, adding potential depth and breadth to our pipeline.

Passage bio was founded in late 2018, but the goal is truly helping patients suffering from serious life threatening rare monogenic central nervous system diseases.

Bruce Goldsmith: Our vision is to become the premier genetic medicines company by developing and ultimately commercializing transformative therapies that dramatically and positively impact the lives of patients suffering from these diseases, with patients at the center of our mission. We are focused on advancing our lead candidate into the clinic as soon as possible and plan to do so later this year. A large part of what makes our company unique is our strong partnership with TEN and the Gene Therapy Program, or GTP. This collaboration gives us access to what we believe is the best discovery, technology, and research in the field of gene therapy. This includes preclinical development and manufacturing experience that will help guide our programs as we move into the clinic. The focus of our collaboration remains on identifying and advancing transformative therapeutics that have the much-needed differentiated profiles to address patient needs. Last week, we announced that we expanded this collaboration, allowing us the opportunity to deepen our product pipeline and enhance our access to GTP's pioneering gene therapy expertise. The amendment increased the number of additional options available for us to license from 6 to 11 and extended the exercise window, including all remaining 11 options, through 2025.

Our vision is to become the premier genetic medicines company by developing and ultimately commercializing transformative therapies dramatically and positively impact the lives of patients suffering from these diseases.

With patients at the center of our mission, we are focused on advancing our lead candidate into the clinic as soon as possible and plan to do so later this year.

A large part of what makes our company unique user strong partnership with 10, and the gene therapy program or GGP. This collaboration gives us access to what we believe is the best discovery technology and research in the field of gene therapy.

This includes preclinical development and manufacturing experience that will help guide our programs as we move into the clinic.

Focus of our collaboration remains on identifying and dancing transformative therapeutics that have the much needed differentiated profiles to address patient needs.

Last week, we announced that we expanded this collaboration allowing us the opportunity to deepen our product pipeline and enhance our access to GGP is pioneering gene therapy expertise.

The amendment increase the number of additional options available roster license from six to 11 and extended the exercise window.

All remaining 11 options through 2025.

Bruce Goldsmith: We've already licensed a total of six to date, bringing our total potential product pipeline to 17 programs. Additionally, under this agreement, we have committed to fund $5 million annually for discovery research conducted by Penn. As a result, we will receive exclusive rights and licenses to certain technologies resulting from discovery research for Passage Bioproducts developed with the GTP, such as next-generation capsids, toxicity reductions, and delivery and formulation improvements. We are tremendously proud of the progress we have made to date through this collaboration. And beyond the enhanced access to technology and programs, this continues to demonstrate the extremely strong partnership between Passage Bio and Dr. Wilson's team at the GTP. Given today's environment, we are working closely with Penn, our manufacturing partners at Catalan, and our external partners across the healthcare fields as we navigate the impacts of the COVID-19 pandemic.

We've already license a total of six to date, bringing our total potential product pipeline to 17 programs.

A disagreement we've committed to fund $5 million annually for discovery research conducted by pen.

As a result, we will receive exclusive rights and licenses to certain technologies, resulting from discovery research for passage by a products developed with the GGP. So just next generation capsids talks to see reductions and delivery a formulation improvements.

We are tremendously proud of the progress we have made to date through this park collaboration and beyond beyond the enhanced access to technology and programs. It's continues to demonstrate the extremely strong partnership between passage bio and Dr. Wilsons team the GGP.

Given today's environment, we are working closely with 10, our manufacturing partners and cabling and our external partners across the health care fields as we navigate the impacts of the cobot 19 pandemic.

Bruce Goldsmith: Like many of our peers, our primary goal is serving patients, particularly those with no alternative, effective, and safe treatment options. As such, we remain steadfast in advancing our programs into the clinic, and we are committed to meeting our development goals. At this time, we do not anticipate any delays in trial initiations for our three lead programs. With a strong cash position from our IPO, strong collaborations, and a growing team, we feel confident in our ability to execute on these goals. While the full impact of the COVID-19 pandemic remains uncertain, so far, our employees have demonstrated their ability to be flexible and have exemplified our company's commitment to best-in-class productivity. We are focused on employee safety, physical and mental well-being, and engagement in our mission, as we believe our team is an extremely valuable asset.

Like many of our peers.

Our primary goal serving patients, particularly those with no alternative effective and safe treatment options.

As such we remain steadfast in advancing our programs into the clinic.

We're committed to meeting our development goals.

This time, we do not anticipate any delays in trial initiations for three lead programs.

With a strong cash position from our IPO strong collaborations and a growing team we feel confident in our ability to execute on these goals.

Well the full impact of the cobot 19 pandemic remains uncertain. So far our employees have demonstrated their ability to be flexible and if exemplified or companies commitment to best in class productivity.

We are focused on employee safety physical and mental wellbeing and engagement in our mission as we believe our team is an extremely valuable asset.

Bruce Goldsmith: As we do everything we can to keep our employees safe, we are also focused on the safety of frontline healthcare workers, patients, and their families as we think about our first clinical trial initiation later this year. In the spirit of patient focus, we are now going to highlight our three most advanced development programs in GM1 ganglia pseudosis, frontotemporal dementia, and CRAV-A disease. Our lead candidate for the treatment of GM1 gangliosidosis is PbGMO1, which we are developing for the treatment of the infantile form of the disease. GM1 is a rare monogenic recessive lysosomal discharge disease caused by mutations in the GLB1 gene encoding beta-galactoside A. These mutations result in an accumulation of toxic levels of GM1 gangliosides and lead to rapidly progressing neurodegeneration.

As we do everything we can to keep our employees safe.

Also focused on the safety of the frontline healthcare workers patients and their families. As we think about our first clinical trial initiation later this year.

In the spirit of patient focus, we're now going to highlight or three most advanced development programs and G. M ganglion doses, frontotemporal dementia and crabby disease.

Our lead candidate for the treatment of G. M ganglia acidosis PBGA My one which we are developing for the treatment of the infant trial form of the disease.

Jim one is a rare monogenic recessive lysosomal storage disease caused by mutations in the GLP, one gene encoding beauty collector citis.

These mutations resulted in an accumulation of toxic levels of G M gangland sides and lead to rapid.

Thirdly progressing neuro degeneration.

Bruce Goldsmith: Infantile GM1, which is characterized by onset in the first year of life, is the most severe form of the disease, reducing life expectancy to only two to four years. Infantile GM1 is the most common subtype of this disease and represents almost two-thirds of total GM1 cases. Unfortunately, patients with infantile GM1 have no effective disease-modifying treatment options available to them. Our development candidate, PBGMO1, is a next-generation HU68 AAV capsid designed to deliver a functional GLB1 gene that expresses beta-galactosidase. Based on Capsid comparison studies, this next generation Capsid was selected due to the superior transduction observed in cells of the CNS and peripheral organs, which we believe gives us the potential to treat both the CNS pathologies and the peripheral manifestations of GM1 in order to restore the developmental potential of patients.

Infantile, Jim one which is characterized by onset and the first your flight is the most severe form of the disease, reducing life expectancy to only two to four years.

Infantile G. M is the most common subtype of whose disease and regular represents almost two thirds of total Jim on cases.

Unfortunately patients with infantile Jim one has no effect in disease modifying treatment options available to them.

Our development candidate PBGA. My one is a next generation. Each you 68, A.D. capsid designed to deliver a functional GLP one gene at expresses beta blocker societies.

Based on Capsid comparison studies. This next generation capsid was selected.

The superior transduction observed in cells of the CNS and peripheral organs, which we believe it gives us the potential to treat both the CNS. That's all the geez and the peripheral manifestations of G. M in order to restored development developmental potential of patients.

Our planned phase one two trial will be an open label dose escalation study of P.G.M. or one administered by a single injection into the intracisternal Magna or I see in pediatric patients with Ensign child G M.

Bruce Goldsmith: Our planned Phase 1-2 trial will be an open-label, dose-escalation study of PVGM-01 administered by a single injection into the intracisterna magna, or ICM, in pediatric patients with infantile GM1. The primary endpoints for this study will be safety and tolerability, as well as treatment effects on the prevention of further developmental regression, restoration of developmental milestones, Prevention of Disease Progression, and Extension of Ventil

The primary endpoints for this study will be safety and Tolerability as well as treatment effects on the prevention a further developments all regression restoration of developmental milestones prevention of disease progression and extension of ventilator free survival.

Bruce Goldsmith: We will also be evaluating the effect of PBGMO1 on a number of biomarkers, including CSF and serum beta-gal enzyme activity, as well as EEG and MRI measures. We anticipate the first patient to be treated in 4Q 2020, and we expect initial 30 day safety and biomarker data in late the first half of 2021. Last month, we were happy to report that the FDA granted orphan drug designation to PBGMO1. This designation represents an important recognition of the dire need for an effective treatment option for these children and their families, while granting us financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval. In addition to the planned clinical study for PB-GMO1, we are sponsoring a natural history study in collaboration with the University of Pennsylvania's Orphan Disease Center to collect prospective data on clinical disease progression in infantile and juvenile GM1. This is the first study of its kind evaluating patients with infantile GM1. We now have three sites open, though due to the impact of the COVID-19 pandemic and concern for patient safety, we have not enrolled any patients. To address these enrollment delays, we are working with sites to explore enrolling and following patients remotely.

We will also be evaluating effective P.G.M.I., one on a number of biomarkers, including CSF and Sera beta Gal enzyme activity as well as E G and MRI measurements.

We anticipate the first patient to be treated for Q2 thousand 20, and we expect initial 30 day safety biomarker data in late first half 2021.

Last month, we were happy to report that the FDA granted orphan drug designation to PB G. M. A one this designation represents an important recognition of the dire need for an effective treatment option for these children and their families well granting us financial incentives to support clinical development and the potential for up to seven years.

Market exclusivity in the U.S. upon regulatory approval.

In addition to the planned clinical study for P.G.M., a one we are sponsoring a natural history study in collaboration with the University of Pennsylvania Orphan disease center to collect prospective data on clinical disease progression infantile and juvenile G M.

The study is the first study of its time evaluating patients with instant trial Jamel Jim one.

We now have three sites open, though due to the impact from cope at 19 pandemic and concern for patient safety, we have not enrolled any patients.

To address these enrollment delays, we are working with sites to explore enrolling in following patients remotely. We also simultaneously accessing additional sources of historical control data from this important population of children.

Now I will turn to our next most advance program PBF T or to the treatment or frontotemporal dementia or FTD.

Bruce Goldsmith: We are also simultaneously accessing additional sources of historical control data from this important population of children. Now I will turn to our next most advanced program, PBFTO2, for the treatment of Frontotemporal Dementia or FTD. FTD is a neurodegenerative disease and one of the most common causes of early-onset dementia, causing impairment in behavior and language and executive function with further progression to immobility and death. Currently, there are no approved disease-modifying therapies for these patients. In approximately 5 to 10% of patients with FTD, or a prevalence of approximately 3,000 to 6,000 patients in the United States, the disease is caused by mutations in the granulin gene, which leads to a deficiency of progranulin. Emerging evidence suggests that progranulin's pathogenic contribution to FTD and other neurodegenerative disorders relates to a critical role in lysosomal function.

FTD is in neuro degenerative disease, and one of the most common causes of early onset dementia, causing impairment in behavior and language and executive function with further progression in mobility.

Currently there are no approved disease modifying therapies for these patients.

In approximately 5% to 10% of patients with FTD for prevalence of approximately 3000 to 6000 patients in the United States diseases caused by mutations in the granule energy, which leads to the efficiency of pro granular.

Emerging evidence suggests that programming olin's pathogenic contribution to FTD and other neurodegenerative disorders relates to a critical role laces almost function.

Our product candidate PBF T. Two there's an 81 viral vector that is designed to deliver a modified DNA in coatings yearend Asian cells.

Preclinical studies have demonstrated its ability to significantly increase program going above normal levels in the CSF.

As part of a preclinical studies, we tested I see M. administration of the 81 capsid against other capsid ties in non human primates and found that 81 provided the strongest transduction.

Bruce Goldsmith: Our product candidate, PBFT02, is an AAV1 viral vector that is designed to deliver a modified DNA encoding GRN to the patient's cells. Preclinical studies have demonstrated its ability to significantly increase progranulin above normal levels in the CSF. As part of our preclinical studies, we tested ICM administration of the AAV1 capsid against other capsid types in non-human primates and found that AAV1 provided the strongest transduction and also achieved up to 50 times the normal human CSF level of programming.

And also achieved up to 50 times, the normal human CSF level of programming.

Based on the encouraging safety and efficacy data for a preclinical studies, we plan to initiate a phase one two trial in the first half of 2021.

Our second most advanced pediatric program is an inventory crowd they disease, a rare and often life threatening lysosomal storage disease that results in progressive damage to both the brain and peripheral nervous system.

Bruce Goldsmith: Based on the encouraging safety and efficacy data from our preclinical studies, we plan to initiate a phase 1, 2 trial in the first half of 2021. Our second most advanced pediatric program is infantile Crab A disease, a rare and often life-threatening lysosomal storage disease that results in progressive damage to both the brain and peripheral nervous system. Krabbe disease is caused by mutations in the galactosil-ceramidase gene, or GALC gene, that leads to the accumulation of cytosine, which in turn kills myelin-producing cells in the CNS and PNS. Without myelin, nerves in the brain and body cannot properly transmit signals, leading to rapidly progressing neurodegeneration in infants. This results in a short life expectancy, which is only two years for those diagnosed with the early form, which manifests before 6 months of age, and approximately 5 years with the late form, which manifests between 7 and 12 months of age.

Crabby diseases caused by mutations in nickel actor, so ceramic days or Gal see gene that leads to accumulation of psychosis.

Which in turn kills Mylan producing cells in the CNS npls.

Without mylan nerves in the brain body can up properly transmit signals leading to rapidly progressing neuro degeneration since.

This results in a short life expectancy, which is only two years for those diagnosed with the early form.

Which manifest before six months of age and approximately five years with a late form which manifest manifest between seven and 12 months of age.

There are no current disease modifying therapies available to these patients and we believe incidents maybe higher than reported due to lack adequate squint screening at birth.

Tomorrow, there will be a presentation of data.

From our Crabby program at a GCT by Juliet hurdle from GGP.

While the full dataset will be presented it tomorrow data from the tweets your mouse and the naturally occurring crabby talk model. So that administration of a Phd 60, carrying a functional galaxy gene into the CSS resulted in normalization of plc enzyme activity and approve improved all parameters of Krave disease, We bill.

Bruce Goldsmith: There are no current disease-modifying therapies available to these patients, and we believe incidents may be higher than reported due to the lack of adequate screening at birth. Tomorrow there will be a presentation of data from our CRAB-A program at ASGCT by Juliette Hordeau from GTP. While the full data set will be presented tomorrow, data from the twitcher mouse and the naturally occurring Crab A dog model show that administration of AAVHE68, carrying a functional GALC gene into the CSF, resulted in normalization of GALC enzyme activity and improved all parameters of Crab A disease. We believe these data are very supportive of our clinical approach. Our development candidate, PBKRO3, utilizes the same next-generation AABHU68 capsid as used on the GM1 program. For PV-KRO3, AAVHU68 will be used to deliver DNA encoding the GALC enzyme to patient cells to reduce cyclosine accumulation and restore myelin. We believe that PV-KRO3 has the potential to address the underlying cause of disease and plan to initiate a Phase 1-2 trial in the first half of 2021. In addition to these LEAD programs, we also have three additional CNS programs in the Discovery and Candidate Selection stage. PBM-004 for My Little Dog.

Leave these data are very supportive of our clinical approach.

Our development can be PB care, a three utilizes the same next generation 88, you 68 capsid has used on the GM one program.

For B for PV care of three Aviate, you succeed will be used to deliver DNA encoding the galaxy enzyme to patient cells to reduce cycle seen accumulation and restore mylan. We believe that PB care of three has the potential to address the underlying cause a disease and plan to initiate a phase one trial.

The first half of 2021.

In addition to these lead programs. We also have three additional CNS programs in discovery and candidate selection stage TB and allow for for MLB PD L. five for Alice with a Cnine wharf 70 to gain a function mutation NPV CMO six for CN T. too.

Okay.

We are currently coordinating with GGP to conduct discovery stage preclinical studies for these programs and look forward to progressing needs into India, enabling studies.

Lastly, I want to take a moment to discuss manufacturing was which is a key aspect of our business.

Bruce Goldsmith: AL05 for ALS with a CA9-ORF72 gain of function mutation, and PD-CM06 for CMT2A. We are currently coordinating with GTP to conduct discovery stage preclinical studies for these programs and look forward to progressing these into IND-enabling studies. Lastly, I want to take a moment to discuss manufacturing, which is a key aspect of our business. Earlier this quarter, we finalized a development services and clinical supply agreement with Catalan to secure clinical-scale manufacturing capacity for our gene therapy program. This agreement formalizes the supply terms for the previously announced collaboration with Catalin for Passage Bio's dedicated manufacturing suite, which we expect to be functional by year end. Once it's up and running, the CGMP suite will be capable of meeting production requirements for our currently product candidates for any clinical needs through early commercialization.

Earlier this quarter, we finalized a development services and clinical supply agreement with catalyst to secure clinical scale manufacturing capacity for our gene therapy programs.

Disagreement formalizes the supply trends for the previously announced collaboration with handling for passage by as dedicated manufacturing speed, which we expect to be functional by yearend.

Once it's up and running the cgmp suite will be capable of meeting production requirements for our currently product candidates for any clinical needs through early commercialization.

Having our own dedicated suite is an important step toward our goal of having expanded control of the supply chain, which we believe will set us up for both clinical and commercial success by allowing for greater flexibility and control in terms of scalability and prioritization as we move products through development.

And with that I will turn the call over to rich to give a financial and operations update.

Thank you Bruce.

As we reported in our press release. This morning, we ended the quarter with cash and cash equivalent of approximately $367 million as compared to approximately $159 million as of December 31st 2019 and.

Bruce Goldsmith: Having our own dedicated suite is an important step toward our goal of having expanded control of the supply chain, which we believe will set us up for both clinical and commercial success by allowing for greater flexibility and control in terms of scalability and prioritization as we move products through development. And with that, I will turn the call over to Rich to give a financial and operations update.

First quarter of 2020, we raised approximately $228 million in net proceeds from our IPO.

We expect our current cash balance to fund our operation and clinical expenses into 2023.

R&D expenses were approximately $13 million for the quarter ended March 31st.

Rich Morris: Thank you, Bruce. As we reported in our press release this morning, we ended the quarter with cash and cash equivalents of approximately $367 million, as compared to approximately $159 million as of December 31st, 2019. In the first quarter of 2020, we raised approximately $228 million in net proceeds from our IPO. We expect our current cash balance to fund our operations and clinical expenses into 2023.

Impaired to approximately $3 million the same quarter in 2019.

The increase was primarily due to an increase of approximately $5 million in R&D costs incurred with Penn in connection with the preparation.

Overall, R&D filings as well as an increase in other research costs.

Approximately $3 million as we prepare for our clinical trials to begin in the second half with 20 Twond and early 2021.

We also had an approximately 2 million dollar increase in personnel related costs and approximately $200000.

Rich Morris: R&D expenses were approximately $13 million for the quarter ended March 31st, compared to approximately $3 million for the same quarter in 2019. The increase was primarily due to an increase of approximately $5 million in R&D costs incurred with Penn in connection with the preparation of several I&D filings, as well as an increase in other research costs of approximately $3 million, as we prepare for our clinical trials to begin in the second half of 2020 and early 2021. We also had an approximately $2 million increase in personnel-related costs and approximately $200,000 increase in facility and other costs due to increases in employee headcount in the R&D function. G&A expenses were approximately $5 million for the quarter ended March 31, compared to approximately $1 million for the same quarter in 2019.

Increase in facility and other costs due to increases in employee head count in the R&D function.

GNS expenses were approximately $5 million for the quarter ended March 31st compared to approximately $1 million for the same quarter in 2019.

The increase was primarily due to an approximately 2 million dollar increase in personnel related and share based compensation expense due to increases in employee head count.

Our professional fees and facility costs also increased by approximately $600000 and approximately $800000 respectively.

As we expanded our operations to support our research and development efforts.

Net loss was approximately $18 million for the first quarter of 20 point compared to approximately $8 million in the same quarter of 2019.

Net loss per basic and diluted share was one dollar in the first quarter of 2020 compared to a dollar and 83 cents per net loss per basic and diluted share in the first quarter of 2019.

Rich Morris: The increase was primarily due to an approximately $2 million increase in personnel-related and share-based compensation expense due to increases in employee headcount. Our professional fees and facility costs also increased by approximately $600,000 and approximately $800,000, respectively, as we expanded our operations to support our research and development efforts. The net loss was approximately $18 million for the first quarter of 2020 compared to approximately $8 million in the same quarter of 2019. The net loss per basic and diluted share was $1 in the first quarter of 2020 compared to $1.83 net loss per basic and diluted share in the first quarter of 2019.

As part of ongoing efforts to continue to grow our organization, we have signed an agreement for new office space.

Which we will move into in early 2021.

We also continue our active recruitment effort focused on leveraging the Virgin cell and gene therapy at the center and our unique access to a strong local candidates in Philadelphia.

We have made a number of key hires over the past quarter and are excited to continue to grow our team of talented dedicated scientists business personnel.

With that let me turn the call back to Bruce for closing remarks.

Thanks Rich.

In closing I'd like to reiterate our commitment to continue to make 2020, a transformative year for passage bio and the patients we serve.

Bruce Goldsmith: As part of ongoing efforts to continue to grow our organization, we have signed an agreement for new offices, which we will move into in early 2021. We also continue our active recruitment efforts focused on leveraging the burgeoning cell and gene therapy epicenter and our unique access to a strong pool of local candidates in Philadelphia. We have made a number of key hires over the past quarter and are excited to continue to grow our team of talented, dedicated scientists and business people. With that, I will turn the call back to Bruce for his closing remarks. Thanks, Ray.

As a newly public company. We believe we are well positioned with internal financial operational strength external collaborations and a continued focus on health care providers patients and their families.

This isn't important time for passage by up to the key remaining milestones in 2020, which are initiation of Pdgm a one clinical study for the treatment of patients with instant trial, Jim one the first patient enrollment anticipated and for Q2 thousand 20.

Advancement of two programs in FTD and crabby towards clinical study initiation in first half 2021, and continued expansion of the clinical manufacturing and operations team to support these programs.

Operator: In closing, I would like to reiterate our commitment to continuing to make 2020 a transformative year for Passage Bio and the patients we serve. As a newly public company, we believe we are well positioned with internal financial and operational strength, external collaborations, and a continued focus on healthcare providers, patients, and their families. This is an important time for Passage Bio for the key remaining milestones in 2020, which are initiation of the PBGM01 clinical study for the treatment of patients with infantile GM1, with first patient enrollment anticipated in 4Q2020; advancement of two programs in FTD and CRAB-A towards clinical study initiations in the first half 2021; and continued expansion of the clinical manufacturing and operations team to support these programs. I'm absolutely confident in our team's ability to continue to execute, and we look forward to updating you on our progress. We'd now like to open up the call to your questions. Operator?

I'm, absolutely confident in our team's ability to continue to execute and we look forward to updating you on our progress.

We'd now like to open up the call for your questions operator.

Thank you.

Ladies and gentlemen, if you have a question at this time. Please press the star followed by the number one key on your Touchtone telephone. If your question has been answered or you wish or move yourself from the Q. Please press the pound team once again to ask a question. Please press Star then one now.

And our first question comes from Salveen Richter from Goldman Sachs. Your line is open.

Thanks for taking the questions. Its Andrei on her Salveen I was just hoping you could speak a little bit more on how you're thinking about conducting your child's an echo bit 19 environment.

You mentioned I guess on this call here that you're thinking about potentially remote enrolling patients and just given how ratcheted fee that is are there additional hurdles associated with merval versus traditional methods and then how are you thinking about patient monitoring and data collection.

Hi, Andrew This is Bruce thanks, very much for your question.

Operator: Thank you. Ladies and gentlemen, if you have a question at this time, please press the star followed by the number one key on your touchtone telephone. If your question has been answered or you wish to remove yourself from the queue, please press the pound key. Once again, to ask a question, please press the star and then one now. And our first question comes from Salveen Richter of Goldman Sachs. Your line is open. Thanks for taking the question. This is Andrea on behalf of Salveen.

So there's two aspects of what we'd like to highlight for for corporate 19.

First the remote monitoring that you you highlighted was actually in reference to our natural history study and after after I make some introductory remarks, all all our ask Gary Romano, our Chief Medical officer to highlight that that's part one and then the second part is that.

We do realize that.

Andrea: I was just hoping you could speak a little bit more on how you're thinking about conducting your trials in a COVID-19 environment. You've mentioned, I guess, on this call here that you're thinking about potentially remote enrolling patients. And given how rare the disease is, are there additional hurdles associated with remote versus traditional methods? And then how are you thinking about patient monitoring and data collection?

Coke at 19, certainly we're looking at the global impact of potential delays et cetera.

The <unk>.

The trial start for GM, one for PBGA PBB GM at one for the treatment of patients within Patel GM will one as of today, we don't anticipate any impact on the I'd filing and.

Bruce Goldsmith: Hi Andrea, this is Bruce. Thanks very much for your question. There are two aspects of what we would like to highlight for COVID-19. First, the remote monitoring that you highlighted was actually in reference to our natural history study, and after I make some introductory remarks, I'll ask Gary Romano, our Chief Medical Officer, to highlight that. That's part one.

You know, we're certainly in discussions with our sites as well as.

The network of other bio pharma and clinical teams in CRM Rose and we certainly think that there there are impacts globally around.

Hope at 19 and site openings et cetera, but we also believe that hospitals will continue to prioritize trials with life threatening and catastrophic illnesses to prioritize opening those so for the code for the PBGA My won a therapeutic treatment for GE Evo, Jim one that we anticipate.

Gary Romano: And then the second part is that we do realize that COVID-19, certainly we're looking at the global impact of potential delays, et cetera, on the trial start for GM1, for PVGMO1, for the treatment of patients with infantile GMO1. As of today, we don't anticipate any impact on the IND filing, and we're certainly in discussions with our sites as well as the network of other biopharmaceuticals and clinical teams and CROs, and we certainly think that there are impacts globally around COVID-19 and site openings, et cetera. But we also believe that hospitals will continue to prioritize trials with life-threatening and catastrophic illnesses to prioritize opening those. So, for the PVGMO1 therapeutic treatment for GM1, which we anticipate trial initiation for the back half of this year, in the fourth quarter, we don't see any specific impact on COVID-19 at this time. What we're focused on is the identification of patients and then the subsequent safe treatment of those patients as they come into the site. And Gary, perhaps you can add some additional color commentary around the natural history study and the remote monitoring that we are anticipating.

Trial initiation for for the back half of this year.

In the fourth quarter, we don't see any specific impacts on.

Looking at 19 at this time.

What we're focused on is the identification of patients and then the subsequent seed treatment of those patients as they do come into the sites.

Gary perhaps you can add some additional color commentary around the natural history study and the remote monitoring that we are anticipating.

Yes, Thank you Chris.

I would just add backs.

We are.

Putting into place.

Capability to remotely monitor patients in the natural history study that includes.

Conducting the assessment assessments of developmental scales.

Over the phone and over video.

We.

Our also exploring how we might use structured.

Okay data collection.

Gary Romano: Yes, thank you, Bruce. Yeah, I would just add that we are putting into place the capability to remotely monitor patients in a natural history study that includes conducting the assessments of developmental scales over the phone and via video. We are also exploring how we might use structured video data collection to evaluate patients' progress and treatment response in the trials. This is going to start as a natural history study, and the learnings that we take from that, we will apply as well to the interventional trials for patient follow-up, as necessary.

To evaluate patients progress.

And treatment response in the trials. This is going to start in the natural history study and the learnings that we take from that we will apply as well to the interventional trials for patient follow up visits.

As necessary.

And then maybe just one additional question if I may just as it relates to the extended collaboration with Penn on could you provide some color maybe on the thinking of what spread the decision and and why it.

Andrea: Great, and then maybe just one additional question, if I may, just as it relates to the expanded collaboration with Penn, could you provide some color on the thinking of what spurred the decision and why it's, you know, why now?

Why now.

Sure no great Thanks, Andrew and.

What we what we thought about is.

In terms of the timing.

It really builds on the collaboration that we have in the successes we've had to date with that with Jim and mid GTP team and.

Bruce Goldsmith: Sure. Great. Thanks, Andrea.

Bruce Goldsmith: And what we thought about was, in terms of the timing, it really builds on the collaboration that we have and the successes we've had to date with Jim and the GTP team. And what we thought about were the specific avenues of research that the GTP is conducting in terms of thinking not only of our lead programs but also our future programs. And when we looked at the ability to not only access, thinking about the next generation capsids that GTP has been discovering and focused on, but also the other technologies that they're approaching in terms of toxicity reduction approaches and delivery and formulation improvements. We thought that this was the appropriate time to build on that collaboration because we want to have the ability, first of all, to continue to support innovative research, and the second is to apply that innovative research to our either ongoing programs, as appropriate, or future programs.

What we thought about was the.

Civic avenues of research that that the GGP is conducting in terms of thinking not only of our lead programs, but also our future programs and when we looked at the ability to not only.

Access thinking about the next generation Capsids that GTP has been discovering and focused on but also the other.

Technologies that they're approaching in terms of toxicity reduction, but approaches and delivery and formulation improvements.

We thought that this would be appropriate time to build on that collaboration because we want to have the ability first of all to continue to support innovative research and the second is to apply that innovative research to our either ongoing programs as appropriate or future programs.

Bruce Goldsmith: So, and the way we think about this is certainly the technology access, but the overarching benefit is also that we are adding five additional programs for an additional cost of an additional, for a total of 11 programs that we could access. And the other piece was that now we have five years to execute on those programs and initiate the research, as opposed to under the initial agreement, where we would have only had two years to start that. So, there are a number of reasons why we thought that this was the appropriate time to both access technology, expand the collaboration, as well as extend the time.

So.

And the way we think about this is certainly the technology out access, but the the overarching.

Benefit is also that we are adding.

Five additional programs for an additional.

For a total of 11 programs that we could access.

And the other piece was too.

Have a five years now to execute on those programs answers initiate initiate that research as opposed to under the initial agreement we would've only had two years to a two to start that so there are number of reasons why we thought that this was the appropriate time to both access technology.

We expand the collaboration as well as extend the time.

Great. Thanks, so much.

Thank you. Our next question comes from Yaron Werber from Cowen Your line is open.

Yaron Werber: Thank you. Our next question comes from Yaron Werber of Cowan. Your line is open.

Operator: I turned it on for you.

Hi, guys. This is dependent on for your own thanks very much for taking the question bank Congrats on progress so far.

Operator: [inaudible]

Operator: IJEM, Laura Chico, Stuart Henderson, William Chou, Mark Forman, Passage Bio

I actually just really wanted to ask you a little bit more broadly on kind of how you're thinking to prioritized some of the programs a little bit farther down the road.

Operator: to really maintain, you know, initiation of CREVE and FQD really simultaneously, and then actually beyond there, how you kind of think about which opportunities to pursue first, and then also, I was actually hoping to just get a little bit more of your thoughts on whether there are any updates on the newborn screening progress. I know this is actually going to come up quite a bit.

If you're still thinking to kind of do really maintain.

Initiation of Krave and the FTD really simultaneously and then actually beyond there, how you're kind of think about which opportunities to pursue first.

And then also I was actually hoping to just get a little bit more.

Just your thoughts on if theres any update on the newborn screening progress I have this is actually going to come up quite a bit across kind of industry in times of covered so I was just wondering if there's any kind updates there thanks very much.

Bruce Goldsmith: across kind of the industry in times of COVID. So I was just wondering if there's any kind of updates there. Thanks very much. Thank you for the question. So I'll begin and I think I'll make a short comment about newborn screening and maybe turn this over to Jill to expand on just our general efforts there. So, in terms of FTD and CRAB-A, yes, we are on track for the initiation of clinical studies in the first half of 2021. And that remains consistent with our previous guidance, our previous statements that we think that we can advance both FTD and CRAB-A generally in the same quarter, or sorry, in the same half to start the studies. And, you know, it really depends on all of the progress towards IND filing, manufacturing, clinical site initiations, and, obviously, patient recruitment that will define the actual timing, the precise timing of clinical trial initiation.

Thank you for the question.

So I'll begin and.

Think I'll make.

Short comment about newborn screening and maybe turn this over two to gel to expand about just our general efforts there.

So in terms of FTD and Crabby, Yes, we are on track for de initiation of clinical studies in the first half of 2021.

And that remains consistent with our our previous guidance our previous statements that we think that we can advance of FTD and craft a.

Generally.

Generally in the same quarter or sorry in the same have to our to start the studies and you know it really depends on on all of the progress towards a I into filing manufacturing.

Clinical site initiations, and obviously patient recruitment that will define the actual timing a precise timing of clinical trial initiation, but we are building the capacity internally in terms of the clinical team and the manufacturing team in order to just support the parallel development of those of those programs I think that was your first.

Bruce Goldsmith: But we are building the capacity internally in terms of the clinical team and the manufacturing team in order to support the parallel development of those programs. I think that was your first question, and that remains parallel and on track. The second question you asked was, how do we continue to prioritize and select new programs? You know, the way we think about this is that we are primarily focused on patient needs and thinking about this both internally at Passage Bio with our advisors, both at our board level and externally, and, of course, with Jim Wilson and his extensive team at GTP. So we think about patient need and then our ability to de-risk those with Jim and his team at GTP in all of the aspects that we've talked about, whether that is delivery method, whether it's biodistribution, evaluating different capsids, etc., so that we can make sure that we have a differentiated and optimized approach.

Question and that remains in parallel and on track.

The second question you said was how do we continue to prioritize and select new programs.

The way we think about this is we are primarily focused on the patient needs and thinking about this both internally at passage bio with our AR.

Advisors, both at our board level and externally and of course with Jim Wilson and his extensive team at GTP. So we think about patient need and and then in our ability to to de risk of those with Jim and his team at GGP.

In all of the aspects, we've talked about whether that is.

Delivery method, whether it's by a distribution evaluating different capsids et cetera. So that we can make sure that we have a differentiated and optimized approach. So.

Bruce Goldsmith: So.

Bruce Goldsmith: Those are the ideas behind the next series of selected programs that we have in mind. And we think that we can continue to diversify our portfolio as we have already accomplished with our first six programs. These are all obviously rare monogenic CNS disorders, but some are very, very rare pediatric disorders. Some are like FDD and progranulin, a bit more expensive, et cetera. So we're trying to create a balanced and diversified portfolio in terms of the indications we're selecting as well. And, in general, we're just engaging in.

Those are the there was the ideas behind the next series of selected programs that we have in mind.

And we're thinking that we can continue to diversify our portfolio as we already have accomplished with us for six programs. These are all obviously rare monogenic CNS is disorders, but some are very very rare pediatric disorders.

Our like for like FTD and programming Olin a bit more expensive.

Et cetera, So we're trying to create a balanced and diversified portfolio in terms of the indications for selecting as well and in general we're just engaging in active and collaboration discussion with discussions with Dr. Wilson.

Bruce Goldsmith: General.

Bruce Goldsmith: and discussions with Dr. Wilson and his team around indications with these concepts in mind and will continue to do so. So that was, I think, the first two questions. And then the third question was on newborn screening, and I'll turn it over to Jill in a moment. I just wanted to say that we are partnering as closely as possible in this era of COVID-19 with both advocacy groups and academic and clinical leaders to think about how to implement this and expand this not only for GM1, but also to continue to collaborate with CRAB-A. But Jill, do you want to talk in general about how we think about the necessity and expansion of newborn screening?

Team.

Around indications with these concepts in mind and we'll continue to so.

So that was I think the first two questions and then the third question was on.

Newborn screening and I'll turn it over Jill in a moment I just wanted to say that we are partnering as closely as possible in this area code at 19.

With both advocacy groups and.

An academic and clinical leaders to think about how to implement this and expand this not only for GM, one but also to continue to collaborate with crabby.

Joe do you want to talk in general about how we how we think about the necessity and expansion of.

Jill Quigley: Sure, Bruce. Yes, newborn screening is going to be very important, of course, for early identification of patients eventually when there's treatment available for various different diseases. We continue to have strong relationships with patient advocacy groups and key opinion leaders (KOLs). Both patient advocacy groups and key opinion leaders are absolutely critical as we think about newborn screening and a strategy around that to support the patients that we're serving. So we've identified a few different opportunities to support the advancement of newborn screening, specifically with GM1. There are already programs, I think, as everyone knows, in place for CROB-A. And we will continue to support those opportunities as we work towards newborn screening, not only at a pilot state level but also continue to try to find a way to move towards a RUST application.

Work screening.

Sure first yes, everything newborn screening is going to be very important and of course for early identification of patients.

Actually when there's a treatment available for various different diseases. We continue to have strong relationships with patient advocacy groups entails.

Pete advocacy groups and halos, you're absolutely critical as we think about newborn screening and its strategy around that to support the patients that were serving so we've identified.

A few different opportunities to support the advancement of newborn screening specifically with the GM. One there are already programs I think everyone has in place for probably.

And we will continue to support those opportunities as we work towards newborn screening not only at pilot street level, but continue to try to find a way to move towards the rest application.

Thanks, Joe and just because they put in that context with Covance incumbent 19, which I think was also your question.

Jill Quigley: Thanks, Jill. And just putting that in context with COVID-19, which I think was also your question, yeah, I think we recognize that all of the aspects of newborn screening, patient identification, patient recruitment are challenging. So, we're partnering with groups that are in touch with patients and looking to, despite COVID-19, initiate some of those programs and partnerships. But you're absolutely right that these remain significant challenges in the era of the

Yeah, I think we recognize that.

All of the aspects of a newborn screening patient identification patient recruitment are challenging. So we're we're partnering with groups that are in touch with patience and looking to despite cobot 19 initiate some of those programs and partnerships.

But you're absolutely right that these are these remain significant challenges in the error or dependent.

Bruce Goldsmith: All right. Great. Thanks very much. Thank you. Our next question comes from Anupam Rana from J.P. Morgan. Your line is open. Hey guys, thanks so much for taking the question. A quick one for me, and I'm sorry if you guys already answered this because we've been hopping on a few different calls this morning, but can you give us an update?

Alright, great. Thanks very much.

Thank you Sir our next question comes from on a Palm Renault from JP Morgan Your line is open.

Hey, guys. Thanks, so much for taking the question.

Hi equipment for me and I'm, sorry, if you guys already answered this because we've been hopping on a few different calls this morning, but he was an update on the natural history strategy.

Anupam Rana: Update on the natural history strategy

Anupam Rana: for both GM1 and CREB-A specifically. Yeah, it's a little bit of a follow-on to the prior question, but any update there would be helpful.

For both GM wanting crime base specifically.

Yes, it's a little bit will follow onto the prior question, but any update there would be helpful. Thank you.

Anupam Rana: be helpful. Thank you.

Bruce Goldsmith: Sure, I'll make a brief comment and then turn it over to Gary. I think for the natural history study, we're still, maybe Gary can comment on the number of sites we've opened and also, you know, the patient enrollment status, which has admittedly been very challenging in the COVID-19 era, as well as complementary approaches that we're trying to do both from remote screening as well as other data sources. And then Gary, maybe you can comment on where we think we are with CRABE from external sources.

Sure I'll make a brief comment and then turn it over to Gary I think for the natural history study we're still.

Maybe Gary can comment on.

The number of sites we've opened it also.

The patient enrollment status, which is admittedly been very challenging into cobot 19 era as well as.

Complimentary approaches that we're trying to do both from remote screening as well as other data sources and then and then Gary maybe you can comment on where we think we are with crabby from external sources.

Gary sure. Thanks, Jeff I'm here. Thanks, Rich, yes, so let me start with GM. One if you can hear me okay.

Gary Romano: Gary. Sure. Thanks. Yeah, I'm here.

Gary Romano: Thanks. Yeah. So, let me start with GM1. If you can hear me,

Gary Romano: Okay. Our strategy for accessing natural history data for comparisons to our interventional trial data for GM1 includes, firstly, a prospective collection of that data in a protocol that is sponsored by the Orphan Disease Center, PEN, as you know, which is a protocol that is nearly identical to the DM1 interventional protocol that we are going to be using for our study. So, that study is running at sites around the world, including the same sites that we are using for our interventional study, which we think will really add to the quality of that data, given that it takes into account site-specific factors. For that study, we have three open sites as of today, with several more sites coming online very soon. We have yet to enroll a patient in that study, but we are moving to be able to enroll patients remotely and then follow those patients. And that's possible because many of those patients are very well known, and so the baseline assessments can be done remotely. They've already been clearly diagnosed by the sites.

Yeah.

Our next mystery stride, our strategy for accessing natural history data.

For comparisons.

To our interventional trial data for GM. One includes firstly prospective collection that that data in a in a protocol that is sponsored by.

The orphan disease Center pen as you know, which you say a protocol that is.

Nearly identical to the to the T. M. Interventional protocols that we are we're going to be using in our scar study.

So.

And that that study is running at sites around the globe, including the same sites.

That we are using for our interventional study, which we think we'll we'll really add since about two the.

Quality that data given that it takes into.

Account site specific.

Factors.

That study is how do we have three open sites as well as of today.

With several more sites coming online very soon.

We have yet to enroll a patient in that study, but we are moving to be able to enroll patients.

Yes.

Remotely and then follow those patients and that's possible there because many of those patients are very well known and so the.

The baseline assessments can be done remotely they've already been currently diagnosed by the sites.

We're going to also supplement that data from the perspective natural history study with retrospective natural history data and we're working with some of the centers around the around the globe that have the most.

Gary Romano: We're going to also supplement that data from the prospective natural history study with retrospective natural history data, and we're working with some of the centers around the globe that have the most longitudinal data on these very same patients, and we already have agreements in place to access that data. So, in the end, we'll have prospectively collected data and retrospectively collected data for GM1. For CRAB-A, our strategy is really to rely on retrospective natural history data. And that's because there is rather abundant natural history data for CRAB-A. For example, we are already working closely with universities across the globe who have this data, and in that case, we're writing protocols that will extract this retrospective data from their databases. And all of this work, by the way, for GM1 and for CRAB-A, we will be doing with early and frequent communications with regulators about our strategy, our statistical analysis strategy for using the natural history data as comparative controls for our single arm.

Longitudinal data on these very sick patients.

And we're already have agreements in place to to access that.

Yes that data so in the end well have prospectively collected data.

Retrospectively collected data for GM one.

For Crabby are our strategy is really to rely on retrospective natural history data and that's because there is rather abundant natural history data for it for crab base.

For example, we are.

We are already in working closely with universities across the globe, who have to stay to.

And that case, we're writing.

Protocols that will extract from their databases this retrospective data.

And all of this work by the way for GM, one and for credit they will be doing.

With.

Early and often communications with regulators about our strategy our statistical analysis strategy.

For using the natural history study data as compared to controls to our single arm trials.

Gary Romano: Great, thanks for taking our question. Thanks very much. Thank you. And again, ladies and gentlemen, to ask a question, please press star and then one now. And our next question comes from Gola Amusa from Chardon. Your line is open. Hi, it's Bola Musa from Chardon.

Great. Thanks for taking my question.

Thanks.

Thank you and again, ladies and gentlemen to ask the question. Please press Star and then one now.

And our next question comes from Golar, Melissa from Chardan. Your line is open.

I will answer from Charles Thanks for taking my call I'm, just a big picture question on your vision I think you've mentioned, becoming a purpose premier genetic medicines company.

Gola Amusa: Thanks for taking my call. Just a big picture question about your vision. I think you've mentioned becoming a premier genetic medicines company, the premier genetic medicines company rather than just a gene therapy company. And not to get into semantics, but could you discuss whether you have access to non-AAV-based genetic medicines capabilities coming out of UPenn with the collaboration or whether you hope to obtain such capabilities from an external source? And then secondly, the first quarter in terms of expenses probably isn't representative going forward. So, could you talk qualitatively or directionally about the expense ramp from 1Q to 2Q to 3Q to 4Q and then from 2020 to 21 and 22, given that you have a pretty long runway into 2023? I'll pause there for a moment.

The Premier genetic medicines company, rather than just a gene therapy company and not to get into semantics, but could you discuss whether you have access to non HBP east genetic medicines capabilities coming out at U. Penn with the collaboration or whether you hope to obtain such capabilities from an external source.

And then secondly, first quarter in terms of.

<unk> expenses, probably isn't representative going forward. So could you talk qualitatively or directionally about to be expense ramp from wants you to twoq to Threeq to Fourq you amount from 2020 to 21 and 22, given that you have a pretty long runway into 2023 Approx there.

Great. Thanks for the question. So I'll take the first part and turn to separate sensor rich, but for some comments around the burn.

Bruce Goldsmith: Great, thanks for the question. So I'll take the first part and turn this over to Rich for some comments around the burn. So you're absolutely right; I think it's a great observation that we

So you're absolutely right I think it's a great observation that we do.

Bruce Goldsmith: we do.

Bruce Goldsmith: believe that we have the potential and certainly our vision is to become the premier genetic medicines company and and right now our strategy in terms of the foundation that we've laid with Penn is certainly on gene therapy for patients with rare monogenic CNS disorders and and we think that with that foundation that will allow us to build the clinical manufacturing and commercialization efforts, that are going to be supportive of that goal, you know, we do recognize that there is more to genetic medicines than specifically the gene therapy that's supported by the GTP current relationship and we think that there's a number of ways of accessing additional technologies that could support that goal and one is obviously through an expanded relationship that may be either selective or broad with our existing partners such as the GTP group and then the other is other opportunities external to that which could come either from either academic partnerships or biotech partnerships. I think that's the long-term goal and the long-term vision because we don't want to be distracted from the near-term goals of building the clinical and manufacturing and commercial infrastructure around our extensive portfolio of up to 17 programs and so the way we think about this is what we're laying out and executing on right now is the foundation and the genetic medicines vision or mission if you will from a long-term perspective is just that, a goal that we want to achieve over time as those technologies

Believed that we have the potential and certainly our vision is to become the premier genetic medicines company and and right now our strategy in terms of the foundation that we played with Penn is certainly on gene therapy for patients with rare monogenic CNS disorders, and we think that with that foundation.

That will allow us to build the clinical manufacturing and commercialization efforts.

That are going to be supportive of that goal.

We do we do recognize that there is more to genetic medicines than it and then specifically that the gene therapy that supported by.

The GTB current relationship and we think that there's there's number of ways of of accessing.

Additional technologies that could support that goal and one is obviously through an expanded relationship.

That may be either selective abroad, with our existing partners such as such as the GGP group.

And then the other is other other opportunities external to that.

Which could come either from either academic partnerships or order.

Biotech partnerships I think thats the long term goal in the long term vision.

Because we don't want to be distracted from from the near term.

Goals of building, the clinical and manufacturing and commercial infrastructure around.

Our extensive portfolio of up to 17 programs and so the way we think about this is.

What we're laying out and executing on right now is the foundation and the genetics medicines vision or core mission. If you will from a long term perspective is just that a goal that we want to achieve overtime as those technologies and quite frankly, the the ability to address a patient the same goal pace.

Bruce Goldsmith: Quite frankly, the ability to address the same goal of patients with significant unmet medical need with transformative therapies is coming to be realized. So I would balance the initial focus with a long-term focus in my answer to your question. Rich, do you want to talk a little bit about the burn, or do you have a follow-up, sorry?

Once with significant unmet medical need with transformative therapies.

Comes to be realized so I would balance the initial focus with a long term focus in my answer to your question.

Rich do you want to talk a little bit about the burn or are doing a follow up sorry.

I will after the burn question.

Bruce Goldsmith: I will after the burn question.

Great. Good morning, good morning.

Rich Morris: Great, great. Morning, morning.

Rich Morris: Thanks for joining the call. Obviously, the first quarter will not be reflective of the balance of the year on a quarterly basis. What you can anticipate is that obviously the quarterly expense and expense from year to year will grow. We're going to see a significant growth in headcount over the course of the year, which will affect not only the first half, the second half of our first half done this year, but then we'll carry it forward in the next year. In addition, you'll see our clinical trials, and I'm going to do this, there are three that we'll be ramping up, mostly in the back half of this year. So there'll be a significant increase in R&D expense as we turn those trials on, and then we'll obviously have a full year's worth of expense for those trials in 2021 compared to 2020. All that's been considered in our guidance, and we're pretty excited that we have a strong cash balance, and then we'll get into early 2023. Great.

Thanks for joining the call. So obviously, the first quarter I will not be reflective of the the balance of the year on a quarterly basis.

What you can anticipate is that obviously the quarterly expense fund from year to year will grow over grow.

We're going to see a significant growth in headcount over the course of year.

Which will affect not only the first the first half over the second half or first half and this year, but then will carry forward into next year.

In addition, you'll see our clinical trials and I'm wondering if theres three that will be ramping up mostly in the back half of this year. So there will be a significant increase in R&D expense as we.

Turn those trials on and then we'll obviously have a full year worker expense for those trials.

2021, compared to 20 point.

All that been considered in our in our guidance.

And we're pretty excited the real strong cash balance.

And then we'll get into early 2023.

Great just a quick follow up but I think there was a paper dot by Dr. Wilson in April is one that went public in molecular therapy, Matheson clinical development compared I see I'm injections to other types of injections.

Gola Amusa: Right, and just a quick follow-up, I think there was a paper by Dr. Wilson in April, it was one of the public intellectual therapy methods in clinical development that compared ICM injections to other types of injections for CNS indications. Do you find that there's anything in that paper that the market under-appreciates about your ICM approach?

For CNS indications it do you find that theres anything in that paper that the market under appreciates about your I'd see I'm approach.

So I think.

Bruce Goldsmith: So I think the ICM approach, as we look at the volume of publications and also our internal data, we believe it's a very effective, very safe approach to date, and obviously, we have to show that and demonstrate it in clinical studies as well, but we are also aware that other groups are also using ICM. The advantage is a very good distribution and transduction that we've seen from non-human primate studies, and we've also seen in the partnership with Penn that that study and approach in terms of safety and delivery through the blood-brain barrier, that's guided obviously by imaging. Gary, I don't know if you have any additional comments, but I think that's the general approach, but I don't know if you have any additional comments on the kind of proof of safety and efficacy of the delivery method with ICM.

Yes, I see him approach.

As we look at the the volume of publications and also our internal data.

I think we believe it's a very effective very safe approach to date and obviously, we have to show that demonstrate in clinical studies as well, but we also are aware that other groups or are also using I see.

The advantage is a very good distribution and transduction that we've seen from them.

Non human Primate studies, and we've also seen.

In the partnership with Penn.

That that is.

The study and other studies have supported a very good approach in terms of safety and delivery through the blood brain barrier, that's guided obviously by by imaging.

Gary I don't know if you have any additional comments, but I think thats. The general approach, but I don't know if you have an additional comments on on that that.

Could improve safety and efficacy.

Efficacy of the delivery method good idea.

No I want me to reiterate that.

Gary Romano: No, only to reiterate that the greatest biodistribution or most complete biodistribution is seen with ICM approaches versus other intrathecal, such as lumbar puncture. And we also believe that there are important safety and biodistribution advantages over intracerebral ventricular approaches.

The.

Best by Mr. Do greatest by a distribution are most complete by distribution seem with I see approaches versus other interests equal.

Such as lumbar puncture and we also believe that there are important safety.

And by a distribution advantages over two Super particular approaches.

Great. Thank you. Thank you.

Gary Romano: Great, thank you. Thank you, and I am showing no further questions in the queue at this time. Ladies and gentlemen, thank you for participating in today's conference. This does conclude your program. You may now disconnect. Everyone, have a great day.

Thank you and I am showing no further questions in the queue at this time.

Ladies and gentlemen, thank you participating in today's conference. This does conclude your program you may now disconnect everyone have a great day.

Operator: BF-WATCH TV 2021

Operator: [inaudible]

Operator: BF-WATCH TV 2021

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