Q3 2020 Passage Bio Inc Earnings Call

Thank you for holding good morning, and welcome to the passage by third quarter 2020 financial and operating results conference call. At this time, all participants are in listen only mode.

Operator: Thank you for holding. Good morning, and welcome to the Passage Bio 3rd Quarter 2020 Financial and Operating Results Conference Call. At this time, all participants are in a listen-only mode.

Following the following his remarks, we'll open the call up for your questions.

Operator: Following the following 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 the call over to Zoe Meda. Zoe, please proceed.

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

At this time I'd like to turn the call over to lead me to Zoe. Please proceed.

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

Zoe Meda: 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 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 third 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. 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 resolution of FDA feedback on INDs, 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 the underlying causes of their respective target 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.

This release is available on the passage by a website at investors Dot passage buyout dotcom under the news section.

On today's call Bruce Goldsmith, <unk>, President and Chief Executive Officer, and Rich Morris Chief Financial Officer will review, our third quarter 2020, <unk> 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 the Q and a portion of the call.

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 resolution of FTD feedback on I. Andy is the initiation of clinical trials and the availability of clinical data from such trials our expectations about o'clock.

Hers and partners ability to execute key initiatives the ability of our lead product candidates to treat the underlying causes their respective target on a genetic CNS disorder manufacturing 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 COVID-19 pandemic.

On the Companys 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.

Could cause the company's actual results to differ significantly from those suggested by these statements given.

Given these risks and uncertainties you should not place undue reliance on these forward looking statements.

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

Please refer to the Companys filings with the FCC for information concerning factors that could cause 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 now my pleasure to pass the call over to CEO Frisco that's right.

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

Bruce Goldsmith: Thanks, Zoe, and thank you all for joining us this morning. This year, we have continued to make significant strides in strengthening our operations in anticipation of having our three lead programs in clinical development next year. Powering our progress has been our growing team of talented leaders dedicated to achieving our mission of providing life-transforming gene therapies for patients with rare monogenic CNS diseases, all while building lasting relationships with the communities we serve. The foundation of the company, as you know, is based on our strategic partner, Dr. James Wilson, who leads the preeminent University of Pennsylvania gene therapy program, GTP.

This year, we have continued to make significant strides in strengthening our operations and anticipation of having our three lead programs in clinical development next year.

Powering our progress has been our growing team of talented leaders dedicated switch or achieving our mission of providing life transforming gene therapies for patients with rare monogenic CNS diseases all.

All while building lasting relationships with the communities we serve.

The foundation of the company as you know is based on our strategic partner Dr., James Wilson will be leased the pre eminent University of Pennsylvania is gene therapy program GGP.

This partnership provides us with unrivalled access to cutting edge research and clinical candidates that are the result of world class expertise and resources capable of translating science ensure optimal gene therapies with a higher probability of technical and regulatory success.

Bruce Goldsmith: This partnership provides us with unrivaled access to cutting-edge research and clinical candidates that are the result of world-class expertise and resources capable of translating science into optimal gene therapies with a higher probability of technical and regulatory success. On today's call, we'll mostly focus on where we stand with our three lead programs in GM1 ganglia sedosis. Frontal Temporal Dementia, and Crab A Disease. We also have three preclinical programs in earlier stages of development for amyotrophic lateral sclerosis or ALS. Metachromatic Leukodystrophy or MLD, and Charcot-Marie-Tooth Disease Type 2a or CM2a.

On today's call will mostly focus on where we stand with our fleet three lead programs in GM one gig sentosa.

Central dementia and crabby disease.

We also have three preclinical programs in earlier stages of development and have your tropic lateral sclerosis or L.S.

Adequate medical lipodystrophy, or MLB and sure Colin Murray to disease type two way RCM two way.

In addition through our GTV partnership we have the option to license a total of 17 programs focused on rare monogenic disorders, CNS, making or potential pipeline quite robust.

Bruce Goldsmith: In addition, through our GCP partnership, we have the option to license a total of 17 programs focused on rare monogenic disorders of the CNS, making our potential pipeline quite robust. We are also well-funded to support our operations. And this financial flexibility is another key asset as we advance our initial three programs into clinical development. Today, I am excited to review some of our recent accomplishments, particularly in patient identification, clinical site preparedness, and manufacturing readiness. I will start with an update on our lead program, PDGM01. As a reminder, GM1 is a rare monogenic recessive lysosomal storage disease caused by mutations in the GLB1 gene encoding the enzyme beta-galactoside.

We are also well funded to support our operations.

This financial flexibility is another key assets be advanced initial three programs into clinical development.

Hey, I am excited to review some of our recent accomplishments, particularly across patient identification clinical site preparedness and manufacturing readiness.

I'll start with an update on our lead program P.G.M. a one.

As a reminder, G. M is a rare monogenic recessive lysosomal storage disease caused by mutations in the GLP one gene encoding that enzyme theoretical exercises we are targeting the invitro form of the disease, which is the most severe weather.

Bruce Goldsmith: We are targeting the infantile form of the disease, which is the most severe, with a rapid disease course and no current treatment option. Our lead program, PVGM01, is a best-in-class gene therapy utilizing a next-generation proprietary AAVHU68 capsid and optimized gene expression cassette to deliver a GLB1 transgene encoding beta-gal to increase beta-gal enzyme activity in the brain and peripheral tissues. During our second quarter earnings call, we announced that FDA had placed our IMD on TBGM01 on clinical hold due to questions concerning the biocompatibility of our proposed intercisternomagma or ICM delivery device. As previously disclosed, we have now received our official letter from FDA confirming that the hold was due solely to these biocompatibility concerns. Based on guidance from FDA, we are conducting biocompatibility risk assessments and testing to resolve their questions as quickly as possible. We continue to engage with the agency and remain confident that we will be able to adequately address any concerns, and we continue to expect to receive IND clearance.

With a rapid disease course, no current treatment options.

Our lead program PV GM Award is a best in class gene therapy, utilizing next generation proprietary Avi each used 68 caps. It can optimize gene expression cassettes should deliver a GLP one transgene coding vdsl increase made ago enzyme activity in the brain peripheral tissues.

During our second quarter earnings call, we announced the F.D.A. had placed our idea P. G. M. A one clinical hold due to questions concerning the biocompatibility of our proposed interest cisterna Magna magna or ice yet delivery device.

As previously disclosed we have now received from official letter from the FDA confirming that the whole was due solely to these bio compatibility concerns.

Based on guidance from F.D., we are conducting biocompatibility risk assessment and testing resolved.

Resolved their questions as quickly as possible.

We continue to engage with the agency and remain confident that we will be able to adequately address any concerns and we continue to expect to receive I declare.

Because of our work to satisfy F. gays biocompatibility requirements. We now expect to initiate the phase one two clinical trial in the first quarter of 2021 and to report initial safety and biomarker Judah mid 2021.

Bruce Goldsmith: Because of our work to satisfy FDA's biocompatibility requirements, we now expect to initiate the Phase 1-2 clinical trial in the first quarter of 2021 and to report initial safety and biomarker data in mid-2021. Meanwhile, while we work toward R&D clearance, our clinical trial preparations remain on track. As discussed on our previous call, we have aligned with the agency on an updated trial design. As a reminder, this trial will be an open-label dose escalation study of PBGMO1, administered by a single injection into the intercisterna magna in pediatric subjects with early and late infantile GMY.

Well, we work toward R&D clearance our CFO.

Clinical trial preparations remain on track.

As we discussed on our previous call we have aligned with the agency on an updated trial design has.

As a reminder, this trial will be an open label dose escalation study of P.B.G.M. a one.

Administered by a single injection.

Instead, he is interest cisterna magnum in pediatric subjects with early and late infantile Jim one.

The dose escalation portion of the trial, we have changed the design specifically study early and late infant trial patients in separate smaller cohorts.

Bruce Goldsmith: For the dose escalation portion of the trial, we have changed the design to specifically study early and late infantile patients in separate smaller cohorts. We will now be enrolling a total of four cohorts of two patients each with separate dose escalation, for late-onset infantile GM1 and early-onset infantile. Because the safety of our patients is our top priority, in addition to the trial amendments, we have also implemented approaches for the GM1 study that address the challenges of the current COVID-19 environment. Such approaches include remote clinical assessments. Structured Video Capture of Study Subjects in the Home, and concierge services through third-party vendors to facilitate travel due to increased burdens and restrictions.

We will now be enrolling a total work horse to patients each separate dose escalation cohorts for late onset in for Jim One an early onset into child she on one.

'cause safety of our patience is our top priority. In addition to the trial amendments. We have also implemented approaches for the GM on study he did that address the challenges of the current COVID-19 environment such.

Such approaches include remote clinical assessments structured video capture study subjects in the home.

Concierge services through a third party vendors to facilitate travel due to increased burdens and restrictions.

We believe these measures will provide patient support also maintaining the rigor of our clinical trials and our ability to provide this potentially life altering therapy to patients.

Bruce Goldsmith: We believe these measures will provide patient support while also maintaining the rigor of our clinical trials and our ability to provide this potentially life-altering therapy to patients. As we have discussed, GM1 is a devastating disease that impacts patients worldwide, and we are dedicated to serving at least eight patients in the U.S. and across additional demographics and geography. Clinical supply for the Phase 1-2 trial has been manufactured and is ready for dosing. And, in addition to submitting our IND to FDA, we have also filed for clinical trial approvals in countries outside of the U.S. We are pleased with our overall progress in activating clinical sites around the world. The need for an effective therapy is underscored by our recent Orphan Disease Designation in Europe granted to PBG M01 by the European Commission in October. PBGMO-1 has previously received orphan drug and rare pediatric disease designations in the U.S. We look forward to working with these and other government agencies to fill the treatment gap for GM1 around the globe.

As we have discussed Jim one is a devastating disease that impacts patients worldwide and we are dedicated to serving at the patients in the U.S. and across additional demographics and geographies geographies.

Clinical supply from the phase one two trial has been manufacturer and is ready for dosing.

And in addition to submitting our <unk> FDA. We have also filed for clinical trial approval in countries outside of the U.S.

We are pleased with our overall progress in activating clinical sites around the world.

The need for an effective therapy is underscored by our recent orphan disease designation in Europe graduate to PV, GM or one or the European Commission in October PV.

PBG at my one previously received orphan drug and rare pediatric disease designation in the U.S.

We look forward to working with these and other government agencies to fill the treatment gap for GM one around the globe.

Our aim is to keep patients at the center of PPG emo ones development and to work with families and physicians to drive the best possible outcome for patients.

Bruce Goldsmith: Our aim is to keep patients at the center of PBGMO1's development and to work with families and physicians to drive the best possible outcome for patients. As we prepare for clinical site activations, we are ramping up patient identification and education efforts, starting with our recently announced partnership with InVita, a leading medical genetic testing company, to increase genetic testing and support early identification of GM1 through a program called Detect Lysosomal Storage Disorder. The Detect LSDs program offers free genetic testing and counseling to encourage earlier diagnosis of lysosomal storage diseases like GM1.

As we prepare for clinical site Activations, we're ramping up patient identification and education efforts, starting with our recently announced partnership with in vitro a leading medical genetic testing company.

Increased genetic testing and support early identification of Jim One group.

Program called detect lysosomal storage disorders.

The detect Lsts program offers free genetic testing and counseling to encourage earlier diagnosis of lysosomal storage diseases like GM one.

Additionally, in DJ will be reaching out.

Bruce Goldsmith: Additionally, Invitae will be reaching out to health care providers who have patients that have tested positive for GM1 to provide educational and clinical trial information. We believe this partnership will be a crucial resource, not only for our clinical program, but also for the patient community more broadly, as we aim to shorten the timeline for reliable diagnosis, treatment, and ultimately halting the progression of this disorder. We continue to engage with patient advocacy groups on important initiatives, such as newborn screening. Currently, we are sponsoring the New York Screen Plus pilot program, which will offer newborn screening for rare diseases, including GM1, to 150 to 175,000 ethnically diverse babies in eight hospitals across the state of New York. Additionally, we are involved in other meetings and initiatives sponsored by our advocacy partners to determine ways to support further adoption of newborn screening. Preclinical data, including recently published data in the peer-reviewed journal Human Gene Therapy, continue to support the potential of PBGMO1 as a treatment for the underlying cause of GM1. In a murine model, the data showed that by increasing beta-gal activity in the brain and peripheral tissues, dbGMO1 was able to reduce neuronal lysosomal storage lesions, improve neurological function, and increase survival.

Our health care providers, who have patients that have tested positive for Jim wants to provide educational and clinical trial information.

We believe this partnership will be a priest crucial resource not only for our clinical program, but also for the patient community more broadly as we aim to shorten the time line for reliable diagnosis treatment and ultimately halting the progression of this disorder.

We continue to engage with patient advocacy groups, an important initiatives such as newborn screening.

Currently we are sponsoring the New York screen, plus pilot program, which will offer newborn screening for rare diseases, including GM, one to 150 to 275000.

Technically diverse babies in heat hospitals across the state of New York.

Additionally, we are involved in other meetings and initiatives sponsored by our advocacy partners to determine ways to serve support further adoption of newborn screening.

Preclinical data, including recently published data in the peer reviewed journal human gene therapy.

Continue to support the potential of PV GMR, one as a treatment for the underlying cause of Gen. One.

And I'm hearing model the data showed that by increasing baby girl activity in the brain and peripheral tissues PBG handle one was able to reduce neuronal lysosomal storage lesions improves neurological function and increased survival.

This data is encouraging and suggest immense potential for Jim one patients.

Bruce Goldsmith: This data is encouraging and suggests immense potential for GM1 patients. We are excited to advance PBGMOI toward clinical development and begin dosing patients. Based on the support of preclinical data, physician and advocacy engagement, and productive interactions with FDA, we look forward to getting this trial started as soon as possible. As for our other lead programs, PBFTO2 for FTD and PBKRO3 for Crab A Disease, we plan to file INDs for both once we receive FDA clearance for our GM1 program. Based on our current timelines, we are preparing for Phase 1-2 clinical trial initiations for PBFTO2 and PBKRO3 in the first half of next year. As we finalize the protocols of our FTD and CREB-A clinical trial programs, we plan to incorporate feedback from FDA regarding our GM1 program as applicable in order to design the safest, most efficient trials possible for these two indications.

We are excited to advance PV GM Hawaiian toward clinical development and begin dosing patients.

Based on the support of preclinical data physician and advocacy engagement and productive interactions with FDA. We look forward to getting this trial started as soon as possible.

As for our other lead programs PBF T O two for FTD and TDK, our three for Krave disease, we plan to file ideas for both once we receive FDA clearance for our GM one program.

Based on our current timelines, we are preparing for phase one two clinical trial initiations for PBF tier two TB Taro three the first half of next year.

As we finalize the protocol of our FTD and kind of a clinical trial programs. We plan to incorporate feedback from the FDA regarding our GM one program as applicable in order to design the safest most efficient trials possible for these two indications.

As a reminder, PDF tier two was a gene therapy that utilizes a b one factor to deliver to the brain functional granular Angie and cutting the programming on protein complex and highly conserved protein thought to have multiple roles and cell biology and installation.

Bruce Goldsmith: As a reminder, PDFTO2 is a gene therapy that utilizes an AAV1 vector to deliver to the brain a functional granulone gene encoding the progranulone protein, a complex and highly conserved protein thought to have multiple roles in cell biology and inflammation. In September, we announced the publication in a peer-reviewed journal of preclinical data from our partners at HensGTP. In mass data, a single administration of an optimized AAV1GRN vector of PVF-T02 showed the greatest increase in CSF progranular levels when compared to two other AAV vectors, reaching more than 50-fold the normal human CSF progranular level. The data also show that AAV1-GRN also appeared to demonstrate extensive transduction of the ependymal cells, which line the ventricles of the brain and are Following these increased progranulid levels, subjects showed reduced lysosomal storage lesions, normalized lysosomal enzyme expression, and corrected microgliosis.

September we announced the publication in a peer reviewed journal preclinical data for our partners attends GTP.

In mouse data a single administration of an optimized avi ones year and vector of PBF T. T O. Two should the greatest increase in CS have progressed no levels when compared to two other avi vectors, reaching more than 50 fold the normal human CSF program levels.

The data showed that 81 GRM also appeared to demonstrate extensive transduction of the dependable cells, which line the ventricles of the brain that are involved in the production of CSS.

Following these increased progression of levels subject showed reduced lysosomal storage lesions normalized lysosomal enzyme expression and corrected micro clearances.

In addition to demonstrating the ability of Tcfs tier two to drive key biomarker results data from this study also supported our IC ATM, we're delivery, which was shown to be well tolerated and minimally invasive.

Bruce Goldsmith: In addition to demonstrating the ability of PBFCO2 to drive key biomarker results, data from the study also supported our ICM route of delivery, which was shown to be well-tolerated and minimally invasive. Our use of the AAV1 capsid in ICM's delivery is a result of rigorous preclinical development work conducted by our partners at GTP, illustrating again the benefits of our core business model of unparalleled access to cutting- We are also preparing our third program, PBKRO3, for a Phase 1-2 trial for the treatment of Crab A disease, a rare and often life-threatening glycosomal storage disease that results in progressive damage to both the brain and peripheral nervous system. As we discussed in detail on our last call, data from preclinical models show that PVKRO3, which utilizes the AAVEG68 capsid to deliver a functional GALC gene, increased We recently announced that FDA granted Orphan Drug and Rare Pediatric Disease designations to PBKR03 for Crabbe disease, further demonstrating its potential and emphasizing the need for effective treatment options.

Our use of the one capsid Tonight, she answered leverage route of delivery as a result of rigorous preclinical development work conducted by our partners at GGP illustrating again, the benefits of our core business model of unparalleled access to cutting edge science and research.

We are also preparing our third program PB care, a three for a phase one trial for the treatment of Krave disease, a rare and orphan life threatening lysosomal storage disease that results in progressive damage to both the brain peripheral nervous system.

As we discussed in detail on our last call data from preclinical models should that TV, Kara three which utilizes the e. cheese 60, a capsid to deliver a functional gal CG increased kelcy enzyme activity reduced segment seen accumulation and restored Mylan <unk>.

Leading to a phenotypic correction and increased survival in a naturally occurring canine mouse model.

We recently announced that FDA granted orphan drugs and rare pediatric disease designation to PV care three for crabby disease.

There is adjusting its potential and emphasizing the need for effective treatment options. We are encouraged that FDA recognizes the critical unmet need in this patient population and we believe the PB care three has the potential to be a life altering therapy that can address the underlying cause of disease.

Bruce Goldsmith: We are encouraged that FDA recognizes the critical unmet need in this patient population, and we believe that TBKR03 has the potential to be a life-altering therapy that can address the underlying cause of the disease. We look forward to initiating our Phase 1-2 trial in the first half of 2021. Finally, turning to our earlier stage pipeline, we continue to progress our earlier programs for the treatment of MLZ, ALS, and CMT2A. We look forward to providing further updates as these programs move closer to clinical development. Beyond our clinical programs, we are also expanding our operational capabilities and are excited to announce the completion of our dedicated GMP manufacturing suite at Catalyst. Construction of the suite is now complete and has undergone qualifications for GMP manufacturing readiness.

Look forward to initiating our phase one two trial in the first half of 2021.

Finally, turning to our earlier stage pipeline, we continue to progress our earlier programs for the treatment of MSC Payless and CMT today, we look forward to providing further updates as these programs move closer to clinical development.

Beyond our clinical programs. We are also expanding our operational capabilities and are excited to announce the completion of our data dedicated GMP manufacturing suite catalent.

Instruction of the suite is now complete and has undergone qualifications for GMP manufacturing readiness we.

We plan to initiate manufacturing activities that are dedicated suite. This quarter, we'll be able to meet production requirements for our lead pipeline products through early commercialization.

Bruce Goldsmith: We plan to initiate manufacturing activities at our dedicated suite this quarter. We'll be able to meet production requirements for our lead pipeline products through early commercialization. Manufacturing capacity has been a key tenet of our strategy since launching the company.

Manufacturing capacity has been a key tenet of our strategy since launching the company. This milestone marks important progress towards securing control of our supply chain, which we believe will set us up for both clinical and commercial success.

Bruce Goldsmith: This milestone marks important progress for securing control of our supply chain, which we believe will set us up for both clinical and commercial success. To support our continued growth and upcoming trial initiations, we continue to make key hires across our clinical, regulatory, manufacturing, and corporate teams. I am delighted Passage Bio's mission has resonated with so many talented individuals, and we feel we are assembling the right group of dedicated, experienced leaders to accomplish our goals. And with that, I will turn the call over to Rich to give a financial update. Thank you, Bruce. As we reported in our press release this morning, we ended the quarter with cash, cash equivalents, and marketable securities of approximately $336 million, as compared to $159 million as of December 31, 2019.

To support our continued growth and upcoming trial initiations, we continue to make key hires across our clinical regulatory manufacturing and corporate teams I'm delighted passage bias mission has resonated with so many talented individuals and we feel we are assembling the right group of dedicated experienced leaders to accomplish our goals.

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

Thank you Bruce.

As we reported in our press release. This morning, we ended the quarter with cash cash equivalents and marketable securities.

Proximately $336 million as compared to $159 million as of December 31st 2019.

We expect our current cash balances to fund our operations into 2023.

R&D expenses were $20.8 million for the quarter ended September thirtyth compared.

Compared to $10.4 million for the same quarter in 2019.

The increase was primarily due to an increase of $8.3 million in.

Rich Morris: We expect our current cash balance to fund our operations into 2023. R&D expenses were $20.8 million for the quarter ended September 30, compared to $10.4 million for the same quarter in 2019. The increase was primarily due to an increase of $8.3 million in clinical manufacturing costs and a $1.9 million increase in clinical development costs as we prepare for a clinical trial to begin in early 2021. These increases were offset by a $4.3 million decrease in preclinical research and development costs incurred as we finalized work associated with our lead indications and preparations for IND filing. We also had a $4.5 million increase in personnel-related costs, including share-based compensation, due to increases in employee headcount in the R&D function. G&A expenses were $7.8 million for the quarter ended September 30th, compared to $1.2 million for the same quarter in 2019.

Clinical manufacturing costs.

And $1.9 million increase in clinical development costs as we prepare for our clinical trials to begin in early 2021.

These increases were offset by a $4.3 million decrease in preclinical research and development costs incurred as we finalize work associated with our lead indications and preparations for IB filings.

We also had a 4.5 million dollar increase in personnel related costs, including share based compensation.

Increases in employee head count and the R&D function.

Gene and expenses were $7.8 million for the quarter ended September thirtyth.

Compared to $1.2 million for the same quarter in 2019.

The increase was primarily due to a $4.4 million 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 $8.6 million.

And $1.6 million, respectively, as we expanded our operations to support our research and development efforts.

Rich Morris: The increase was primarily due to a $4.4 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 $0.6 million and $1.6 million, respectively, as we expanded our operations to support our research and development efforts. The net loss was $28.5 million for the third quarter of 2020 compared to $11.4 million in the same quarter of 2019. The net loss per basic and diluted share was $0.63 in the third quarter of 2020 compared to a $2.02 net loss per basic and diluted share in the third quarter of 2019.

Net loss was $28.5 million for the third quarter 2020.

Compared to $11.4 million in the same quarter of 2019.

Net loss per basic and diluted share was 63 cents in the third quarter of 2020 compared to year end $2 in two cents net loss per basic and diluted share in the third quarter of 2019.

Now I will turn the call back to Bruce for closing remarks.

Thanks, Rich as we are close to the end of this year I want to take a moment to reflect on our progress throughout 2020. This year, we expanded the foundation of the company as we continue to prepare the clinical stage development of three promising pipeline assets next year, taking us closer to the two.

Bruce Goldsmith: Now I will turn the call back to Bruce for his closing remarks. Thanks, Rich. As we are close to the end of this year, I want to take a moment to reflect on our progress throughout 2020. This year, we expanded the foundation of the company as we continued to prepare for the clinical phase development of three promising pipeline assets next year, taking us closer to our goal of offering innovative, safe, and effective therapies to patients who currently have limited or no treatment options. As I shared earlier, we are engaging with FDA to resolve their questions regarding device biocompatibility, and we anticipate dosing our first patient in the Phase 1-2 trial of PBGM01 in the first quarter of 2021. We continue to prepare our clinical sites for dosing in both the U.S. and abroad and are undergoing measures to support patient safety amidst the evolving COVID-19 pandemic. We expect to report initial 30-day biomarker and safety data for the first cohort of late-onset infantile GM1 patients in mid-2021.

Our goal of offering innovative safe and effective therapies to patients who currently have limited or no treatment options.

As I shared earlier, we are engaging with FDA to resolve their questions regarding device biocompatibility.

We anticipate dosing our first patient in the phase one two trial PBG ever won.

In the first quarter of 2021.

We continue to prepare our clinical sites for dosing in both the U.S. and abroad, and our ongoing undergoing measures to support patient safety mitts evolving COVID-19 pandemic.

We expect to report initial 30 day biomarker safety data from the first cohort of late onset is Intel GM on patients.

B 2021.

We also remain on track to initiate phase one two trials for our FTD and crabby programs in the first half of 2021.

As I mentioned earlier, we continue to extend our clinical manufacturing and operations teams to support these programs along with the future development of additional pipeline candidates all with the goal of transforming the lives of patients suffering from serious life threatening rare monogenic central nervous system Caesars.

Bruce Goldsmith: We also remain on track to initiate Phase 1-2 trials for our FTD and CRAB-A programs in the first half of 2021. As I mentioned earlier, we continue to expand our clinical manufacturing and operations teams to support these programs, along with the future development of additional pipeline candidates, all with the goal of transforming the lives of patients suffering from serious, life-threatening, rare, monogenic central nervous system diseases. The reason we have been able to be so productive despite a challenging year is because of our talented, committed team of employees at Passage Bio. I'd like to take this opportunity to recognize and thank them for their flexibility, hard work, and diligence as we head into the final months of this year. It's because of them that we are well prepared to take on what promises to be a milestone-filled year in 2021. At this time, we'd like to open the call up to your questions. Operator.

The reason, we have been able to be so productive despite a challenging year it because of our talented committed team of employees that passage bio.

I'd like to take this opportunity to recognize and thank them for their flexibility hard work and diligence as we head into the final months of this year.

It's because of them that we are well prepared to take on what promises to be a milestone sales year 2021.

At this time, we'd like to open the call up for your questions operator.

Thank you Sir we will now take any questions you may have.

Do you have a question press star, one and you'll be put into the Q.

If you'd like to remove yourself from the queue. Please press the pound key.

The first question comes from I know Palm Rama from JP Morgan. Please go ahead.

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

Just a quick one for me.

Operator: Thank you, sir. We will now take any questions you may have. If you have a question, press star 1 and you'll be put into the queue. If you would like to remove yourself from the queue, please press the pound key. The first question comes from Anupam Rama from J.P. Morgans.

For the NB take collaboration with Kobe also being like some storage disorder are they.

Airplanes ultimately to include this in indication as part of the collaboration for two facilitating genetic testing and patient identification that if I could squeeze one more in on the biocompatibility risk assessments and testing of the IC device, maybe any more color on the final gating factor, it's kind of coming.

Anupam Rama: Please go ahead. Hey guys, thanks so much for taking the question. Just a quick one for me, for the In-Vitae collaboration with PREV-A also being a lysosomal storage disorder, are there plans ultimately to include this indication as part of the collaboration, you know, facilitating genetic testing and patient identification? If I could squeeze one more in on the biocompatibility risk assessments and testing of the ICM device, maybe any more color on the final gaining factors, kind of completing this and This is Gary.

Leading this and and.

Discussions with the FDA. Thanks, so much.

[noise].

Yes. This is Gary.

May have lost reassess connection so let me okay Gary.

Apologies.

Tom Thanks, very much I was on a double mute as opposed to single me at that time.

Gary Romano: We may have lost Bruce's connection, so let me try. Okay. Go ahead, Gary. I apologize. Autopalm, thanks very much. I was on double mute as opposed to single mute at that time.

So thanks for your question. Thanks for joining the call apologies for the delay so I'll take the second question and then I'll turn it over to Joe for a discussion on the on the and the VJ calibration and other approaches were taking on patient identification. So yeah. We're we're essentially the.

Gary Romano: So thanks for your question. Thanks for joining the call. Apologies for the delay.

Gary Romano: So I'll take the second question, and then I'll turn it over to Jill for a discussion on the EVT calibration and other approaches we're taking on patient identification. So, yeah, we're essentially, the gating items and the discussions with the FDA are really completing some of the testing and finalizing the reports, and essentially moving forward with the final risk assessment. And that's based on an interactive discussion with the FDA since receiving a letter earlier this year and simply completing those tests. And we had indicated back in September and August that those were probably going to take about seven to eight weeks, and we're generally on that timeline and still on track for our initiation of the clinical study in the first quarter of next year. And that's for GM1, and then we'll file, as we said, for FTD and CRAB-A sequentially after that.

The gating items and the discussions with the FDA are really completing some of the testing and and finalizing the reports and essentially moving forward with the the final risk assessment and that's been based on.

An interactive discussion with the FDA since receiving a letter earlier this year and just simply completing those tests and we had indicated.

Back in I think September and August that those were probably going to take about seven to eight weeks and we're generally on that timeline and still on track for our.

The initiation of the clinical study in the first quarter of next year.

So.

And that's for GM, one and then then we'll file as we said for FTD and crap a sequel.

Sequentially after that so initiation of those studies should be high in the first half of next year. So overall still moving forward with the initial plans and the gating items are really just finalizing all of those tests and risk assessment.

Gary Romano: So initiation of those studies should be in the first half of next year. So overall, still moving forward with the initial plans, and the gating IJMs are really just finalizing all of those tests and risk assessments. Jill?

Joe.

Sure.

Jill Quigley: Sure, so Ana Pond, that's a great question about the newborn screening initiative for CRABE. Currently, CRABE is not included in New York Screen Plus, but CRABE, as you know, has newborn screening pilots in states across multiple states. I think there are up to about nine states that are now screening for CRABE.

So in a kind of it's a great question about that.

That the newborn screening initiative for crowd Bank I'm currently crowd. They it's not included on your screen Pos, but probably as you know has newborn screening pilot in states across multiple I'll see if I can get to about nine states that are now screening for Krave and one of the reasons. We initially focused on the core.

Jill Quigley: One of the reasons we initially focused on supporting the inclusion of GM1 for New York Screen Plus is because there are no states doing any newborn screening for GM1 currently. And certainly, as the program progresses, we can consider adding CRABE, although there are multiple states that have pilot programs already for that. Yeah, and the same and the same approach for in vitae as well, which is that, you know, the lysosomal storage disease. The Detect Program is certainly able to be expanded as we move forward.

And the inclusion of GM, one for New York screen process, because there are no states doing any newborn screening for GM. One currently and certainly as you know as the program progresses, we can consider adding crabby. Although there are multiple states that pilot programs are ready for that.

Yes, and the sales and the same approach for in vitro as well, which is that the lysosomal storage disease.

Detect program is certainly.

To able to to be expanded as we move forward.

Thanks, so much for taking our questions.

Jill Quigley: Thanks so much for taking our questions. Thank you. I show our next question comes from the line of Salveen Richter.

Thank you.

Our next question comes from the line of Salveen Richter.

Go ahead.

Operator: Go ahead. Hi, this is Sonia on behalf of Salveen. You recently announced a partnership with NVTA towards the goal of increasing patient identification efforts in GM1. Could you comment on the number of patients you would expect to identify from the partnership? And then, have you had discussions with the FDA? Have your discussions with the FDA impacted your filing plans for either of the diseases on GM1? Yes, so I taught the first part, and that was on Envitae.

Hi.

Mrs Sonia on for Salveen.

You recently announced a partnership with NBT towards the goal of increasing patient identification efforts and GM. One could you comment on the number of patients you would expect to identify from the partnership.

And then have you had discussions with the FDA have your discussions with the FDA impacted your filing plans for either of the diseases on GM <unk> June one.

Yes so.

I taught the first part and that was done on an VK. So.

Bruce Goldsmith: So, you know, the way that we think about this Envitae and also the combination of that with pilot programs like New York Screen Plus is to do two things. One is to identify patients and essentially get the patients that are coded and have a genetic screen and are identified with GM1. Right now, the incidence is predicted to be about 0.5 in 100,000 live births, which is really just around, you know, 50 patients potentially per year, 50 children. But we also recognize that the underdiagnosis of a disease that does not have a standardized genetic screen that is being used in practice may increase the identification of the disease.

The way you do we think about this is in VJ and also the combination of that with.

Let programs like the New York screen plus is to do two things one is to identify.

Patients and so.

Essentially get to get the patients that are coded and have a genetic screen and our identified with GM one right.

Right now the incidence is predicted to be about 2.5, and 100000 life first which is really just around.

50, 50 patients potentially per year 50 children.

But we also recognize that.

The under diagnosis of a disease that does not have a standardized genetic screen that is in that is being used in practice.

May increase the identification of the disease. So we're partnering within retail on two aspects. One is to as testing is needed to allow for free testing, which should hopefully lower an economic barrier for potential screening of GM, one which may increase both the incidence and the.

Bruce Goldsmith: So we're partnering with NVTA on two aspects. One is to, as testing is needed, allow for free testing, which should hopefully lower an economic barrier to the potential screening of GM1, which may increase both the incidence and the detection of that incidence. And then the second part is for those patients who have already been identified; there's an educational and awareness campaign to talk about clinical studies that are available. So in all of those, the idea, as well as New York Screen Plus, is to raise awareness and potential identification of the patients. So hopefully, all of the actual incident patient population is eventually diagnosed and brought into potential therapy. So we don't have a specific number because the gap right now that exists is for those patients who may have the disease but are not aware. And NVTA's program, as well as partnerships like New York Screen Plus and partnerships with advocacy groups, is hoping to increase awareness and the identification of patients. Yeah, yeah.

Detection of that incidence and then the second part is for those patients who have already been identified.

There's a educational and awareness campaign to talk about clinical studies that are available. So in all of those the idea as well as the New York screen plus is to raise awareness and potential identification of the of the patients. So hopefully that all of the actual in the patient population is eventually.

Question brought into potential therapy. So we don't have specific number because the gap right now that exists is.

For those patients who may have the disease, but are not aware and didn't vtcs program as well as partnerships like near screen, plus and partnerships with advocacy groups are hoping to increase the awareness and the identification of patients.

And could you repeat your second question.

Yeah. Yeah. So you noted the plan to incorporate feedback from FDA on Jan one as applicable to prescribe an FTD indication.

Bruce Goldsmith: So you noted the plan to incorporate feedback from FDA on GM1 as applicable for CRAB and STD indications. So we were just wondering how those discussions with the FDA have impacted your filing plans for other diseases. Sure. If you don't mind, maybe I'll turn it over to Gary to talk about the filing plans for FTD and CRAB-A. Sure. Thanks, Bruce.

So we were just wondering how those discussions with the FDA have impacted your filing plans for other diseases.

Sure.

If you don't mind, maybe I'll turn it over to Gary to talk about the filing plans for FTD and craft a word.

Sure. Thanks, Chris Yes. Thanks.

Gary Romano: Thanks. So, thanks for the question. We are still planning to submit our INDs for FTD and CRAB-A, probably in the early first quarter of next year, first half of next year. So again, as Bruce mentioned, this is going to follow the FDA, getting off the clinical hold from the GM1 program. So immediately following getting off the hold, we intend to move forward with the other ID submissions. As Bruce mentioned, that should happen towards the end of this year, beginning of May.

So thanks for the question, we are still planning to submit our ideas for FTD and crabby.

[music].

In the Oh.

Probably to the early early first quarter.

Next year first half of next year.

So.

Again as Bruce mentioned this is going to follow.

The FDA.

Getting off the clinical hold from the GM one program so immediately.

Immediately following getting also hold we intend to move forward with the other ideas submissions.

As Bruce mentioned that should happen towards the end of this year beginning of next.

Thank you.

Gary Romano: As a reminder, to ask a question, you would need to press star 1 on your telephone. To withdraw your question, please press the pound key. Our next question comes from the line of Yarin Weber from Cowan. Please go ahead. Hi guys, this is Brendan on for yourself. Congratulations on the quarter. Thanks again for taking the question. Just a couple quick ones from us.

Thank you as a reminder to ask a question you would need to press star one on your telephone to withdraw your question. Please press the pound key.

I show. Our next question comes from the line of Yaron Weber from Cowen. Please go ahead.

Hi, guys. This is brendan on for your own correct. The quarter. Thanks again for taking the question. So couple of quick ones from us.

I guess first just really kind of wanted to get a sense of where recruitment stands for these programs and obviously you can't treat with the hold in place, but have been able to kind of recruit some patients for any of these but I suppose particularly for GM. One there may be a bit of a bolus, they're ready for when the hold is lifted.

Operator: I guess first, just really kind of wanted to get a sense of where recruitment stands for these programs. Obviously, you can't treat with the hold in place, but have you been able to recruit some patients for any of these? I suppose, particularly for GM1s, there may be a bit of a bolus there ready for when the hold is lifted. Yeah, I'll start and then I'll turn it over to Gary.

Yeah, I'll start and then I'll turn it over to Gary I mean it.

Bruce Goldsmith: I mean, you know, Gary can talk about maybe some of the patient recruitment efforts we're undertaking and the connections with sites, but I do want to mention that one of our approaches has always been that if a patient is in need of therapy, they should get therapy. So we're, you know, while sites are certainly engaged, if there are other alternatives, we're really trying to be patient-focused here. And since there has been uncertainty, obviously, with our timelines, even though we have a projected timeline, we've been very careful not to ask sites to hold patients, for example, because, as you know, these patients, GM1, CRAB-A, and patients with FTD, are in a very severe state. So we've been very patient-focused in kind in our approach and know that once our therapies are approved, the clinical hold is lifted, and that further INDs are filed, we'll obviously be focused on patient recruitment.

Gary can talk about maybe some of the.

Agent recruitment efforts were.

We're undertaking in the connections with sites, but I do want to mention that whatever our approach has always been that if a patient is in need of therapy. They should get therapy. So we are we are you know well well sites are certainly engaged if there are other alternatives. We're we're really trying to be patient focus here and is there.

As there has been uncertainty obviously with our timelines, even though we have a projected timeline, we've been very careful not to.

ASP ESCO sites to hold patients for example, because as you know these patients.

Jim one krave and patients with FTD are in a very severe state. So we've been very patient focus in kind of our approach and know that once our therapy is the clinical holds are the clinical hold is lifted and the further ideas are filed.

We'll obviously be then focused on patient recruitment, having said that we've certainly been in touch with sites and physician.

Bruce Goldsmith: Having said that, we've certainly been in touch with sites and physicians and patient advocacy groups. And maybe, Gary, you can offer some additional commentary about how that reaction is in patients that are interested in participating in the study or family. Yeah, thanks, Bruce. Yeah, so we've been working with centers of excellence around the world for GM1. So this includes North America, Europe, South America, and the Middle East.

Physicians and patient advocacy groups and maybe Carey you can offer some additional commentary about.

How how the reaction is in patients that are interested in participating in the study or families I should say.

Yes, Thanks Bruce.

Yeah. So we've been working with centers of excellence around the world for GM one.

So this includes.

North America, Europe, South America Middle East. These are the sort of centers to get referrals for these patients.

Gary Romano: These are the centers that get referrals for these patients throughout the world. So our really, our intention is to be able to bring patients from different demographics and geographies as quickly as we can into the study. We are, as Bruce mentioned, moving forward with these filings to open sites across the world, and we expect that we will have sites ready to enroll as soon as we come off the clinical hold, which we anticipate to be right around the early, early 2021. So we are anticipating that there's been a lot of enthusiasm from all these sites. And as we just mentioned, we're not holding patients in a queue at this point, but we know the patients are out there, and the way that our trial will be well positioned to enroll them. Thanks very much, guys, and I'm showing no further questions in the queue at this time. Ladies and gentlemen, this concludes your program, and you may now disconnect. Everyone have a great day.

Throughout the world.

So we're really.

Our intention is to be able to bring patients from different demographics and geographies as quickly as we can into the study.

We are as.

As Bruce mentioned, we are moving forward with these.

Filings to open sites across the world and.

We expect that we will have sites ready.

To enroll so.

And as we come off the clinical hold.

Which we anticipate to be right around the.

Early in early 2021.

[music].

Yeah. So.

We are anticipating that theres been a lot of enthusiasm from all these sites and.

As Bruce mentioned, we're not we're not holding patients in a queue at this point, but we know the patients are out there and.

And where they are.

Well the.

Well positioned to enroll.

Okay. Thanks, very much guys.

Thank you.

And I'm showing no further questions in the queue at this time, ladies and gentlemen, thank you for participating in today's conference. This concludes your program. You may now disconnect everyone have a great day.

[music].