PASG Passage Bio Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
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PBGM01
GM1 gangliosidosis
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Phase 1/2
Phase 1/2
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PBKR03 (GALax-C)
Krabbe Disease
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Phase 1/2
Phase 1/2
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PBFT02
Frontotemporal dementia
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Phase 1/2
Phase 1/2
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Latest News
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- Passage Bio collaborator University of Pennsylvania's Gene Therapy Program to present poster regarding development of a novel mouse model for evaluating efficacy of adeno-associated virus gene therapy for metachromatic leukodystrophy (MLD)
- Passage Bio gene therapy candidate, PBML04, showed preliminary signs of efficacy in novel mouse model of MLD
PHILADELPHIA, May 11, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ:PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, announced today the presentation by its collaborator University of Pennsylvania's Gene Therapy Program (GTP) of a digital poster at the 24th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Gourav Roy Choudhury, Ph.D., a senior research investigator at GTP, will report on the development and utility of a novel Arylsulfatase A-deficient mouse model in reproducing key aspects of human metachromatic leukodystrophy (MLD) neuropathology to enable evaluation of efficacy of adeno-associated virus (AAV) gene therapy. Preliminary findings showed the administration of Passage Bio's gene therapy candidate PBML04 (AAVhu68.GTP-207) significantly reduced the neurological deficits of MLD in this model.
"This novel animal model makes it possible to evaluate the pre-clinical efficacy of our AAV-mediated gene therapy for MLD," said Bruce Goldsmith, Ph.D., President and Chief Executive Officer of Passage Bio. "One of the major challenges in developing treatments for MLD has been the limited availability of mouse models and scarcity of phenotype in existing models. This model reproduces important aspects of human leukodystrophy neuropathology, including key biomarkers. We are encouraged by the preliminary signs of efficacy we saw with PBML04 in this study."
The biomarkers used in the Arylsulfatase A-deficient mouse model were LAMP1, GFAP, and several sulfatide species (C16:0 and C18:0). Passage Bio's gene therapy candidate PBML04, which is at the IND-enabling stage of development, utilizes an AAV viral vector to deliver a codon-optimized gene sequence encoding functional ARSA enzyme.
ASGCT Presentation Details
Title: Development and Characterization of a Novel Arylsulfatase A-deficient Mouse Model of Metachromatic Leukodystrophy to Evaluate the Efficacy of Gene Therapy
Date and time: Tuesday, May 11, 2021, 8:00am – 10:00am ET
Presenter: Gourav Roy Choudhury, Ph.D., University of Pennsylvania
Abstract number: 494About MLD
MLD is a monogenic autosomal recessive sphingolipid storage disease caused by mutations in the gene encoding the lysosomal enzyme ARSA. Patients with MLD display progressive leukodystrophy (demyelination) in the central and peripheral nervous systems, neuronal cell death, and subsequent loss of all motor and cognitive function, resulting in premature death, especially in patients with early disease onset.About Passage Bio
At Passage Bio (NASDAQ:PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania's Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at www.passagebio.com.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about 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; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "potential," "possible," "will," "would," and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
For further information, please contact:
Passage Bio Investors:
Stuart Henderson
Passage Bio
[email protected]Passage Bio Media:
Gwen Fisher
Passage Bio
215-407-1548
[email protected]
- Passage Bio collaborator University of Pennsylvania's Gene Therapy Program to present poster regarding development of a novel mouse model for evaluating efficacy of adeno-associated virus gene therapy for metachromatic leukodystrophy (MLD)
-
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PHILADELPHIA, May 10, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ:PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced it will host its second virtual Research & Development event on Monday, May 17, 2021, from 9:30 to 11:30 a.m., Eastern Time.
The event will focus on frontotemporal dementia (FTD) with granulin (GRN) mutations, providing a detailed presentation of the company's robust pre-clinical data and clinical program. Presenters for the event are:
- Eliseo O. Salinas, M.D. MSc., chief R&D officer, Passage Bio
- Christian Hinderer, M.D., Ph.D., senior research director at the Gene Therapy Program (GTP), University of Pennsylvania
- Gary Romano, M.D., Ph.D., chief medical officer, Passage Bio
- The presenters will also be joined during a Q&A session by Bruce Goldsmith, Ph.D., president and chief executive officer, Passage Bio; and James M. Wilson, M.D., Ph.D., director, GTP, University of Pennsylvania, and chief scientific advisor at Passage Bio
To register for the live event, please use the following link: https://www.webcaster4.com/Webcast/Page/359/41012
A live webcast of the presentation will be available on the Investors & Media section of Passage Bio's website at investors.passagebio.com and will remain active for 30 days.
About Frontotemporal Dementia
FTD is one of the more common causes of early-onset (midlife) dementia, causing impairment in behavior, language and executive function, and occurs at similar frequency to Alzheimer's disease in patients younger than 65 years. In approximately 5% to 10% of individuals with FTD, the disease occurs because of mutations in the GRN gene, causing a deficiency of progranulin (PGRN). PGRN is a complex and highly conserved protein. The mechanism by which PGRN deficiency results in FTD is uncertain, but increasing evidence points to PGRN's role in lysosomal function. The rapid progression of FTD results in an average survival of eight years after onset of symptoms.
About PBFT02
Passage Bio is developing PBFT02, an adeno-associated virus-delivery gene therapy, for the treatment of patients with FTD with GRN mutations in a global clinical Phase 1 /2 study titled upliFT-D.
More information about upliFT-D can be found at ClinicalTrials.gov: NCT04747431.
About Passage Bio
At Passage Bio (NASDAQ:PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania's Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at www.passagebio.com.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including our planned IND submissions, 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; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "potential," "possible," "will," "would," and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
For further information, please contact:
Passage Bio Investors:
Stuart Henderson
Passage Bio
267-866-0114
[email protected]Passage Bio Media:
Gwen Fisher
Passage Bio
215-407-1548
[email protected] -
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- Dosed first patient with infantile GM1 gangliosidosis in Imagine-1 Phase 1/2 trial of PBGM01, initial safety and 30-day biomarker data expected 4Q21
- Received multiple regulatory clearances for clinical trial initiations for three most advanced pipeline programs
- Expected enrollment of first patient in Phase 1/2 FTD-GRN trial in 2Q/3Q21 and first patient in Phase 1/2 Krabbe trial in 3Q21
- Continued to advance pipeline under leadership of newly appointed Chief R&D Officer Eliseo Salinas, M.D., MSc.
- Appointed Maxine Gowen, Ph.D., an experienced biotech leader, to Passage Bio Board of Directors
- Raised $166 million, further strengthening company's cash position
- Management to host conference call today at 8:30 a.m. ET
PHILADELPHIA, May 05, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ:PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today reported financial results for the first quarter ended March 31, 2021 and provided recent business highlights.
"We are particularly proud to have recently dosed our first patient in our global Imagine-1 trial of PBGM01 for infantile GM1 gangliosidosis," said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. "This accomplishment speaks to the strength of our collaboration with the University of Pennsylvania's Gene Therapy Program. By raising an additional $166 million in the first quarter, we are well funded to advance both our clinical- and research-stage programs.
"Our primary focus in 2021 remains advancing and expanding our differentiated pipeline for patients with rare CNS disorders," Dr. Goldsmith added. "We are pleased to have successfully received a number of clinical trial regulatory approvals in a period of several months for our three most advanced programs. While some impact on clinical site initiations from the Covid-19 pandemic has occurred, which is reflected in adjusted study timelines, we look forward to reporting on several meaningful milestones throughout 2021 as we diligently work toward delivering transformative CNS therapies for patients."
Recent Highlights:
- First patient dosed in Imagine-1 study of PBGM01 for infantile GM1 gangliosidosis: The company announced in April 2021 that the first patient had been dosed in its Imagine-1 global Phase 1/2 trial of PBGM01 for the treatment of infantile GM1 gangliosidosis (GM1). Imagine-1 is a global open-label study of PBGM01 administered by a single injection into the cisterna magna in pediatric subjects with early and late infantile GM1.
- Several recent regulatory milestones achieved in a period of three months for three most advanced pipeline programs:
- PBGM01 for GM1 and PBKR03 for Krabbe disease received regulatory clearances for clinical trial initiations from U.S. Food and Drug Administration (FDA), UK MHRA, and Health Canada
- PBGM01 also received regulatory clearance for clinical trial initiation from the Brazilian Regulatory Health Agency
- PBFT02 for frontotemporal dementia with granulin mutations (FTD-GRN) received regulatory clearances for clinical trial initiation from FDA and Health Canada
- The European Commission granted Orphan designation for PBKR03 for the treatment of early infantile Krabbe disease.
- Announced collaboration with InformedDNA, the nation's leading genetics services organization: The collaboration will provide no-cost genetic counseling and testing for adults who have been diagnosed with FTD. The testing program will facilitate identification of FTD patients with certain inherited genetic mutations as well as support for clinical trial recruitment and enrollment. It is estimated that approximately 5 to 10 percent of FTD is caused by a GRN gene mutation.
- Timelines adjusted for global Phase 1/2 clinical trials for PBGM01, PBFT02 and PBKR03 due primarily to impacts associated with Covid-19: The initial safety and 30-day biomarker data for Imagine-1 for PBGM01 for GM1 is expected to read out in 4Q21. The global clinical trial upliFT-D for PBFT02 for FTD-GRN is expected to initiate in 2Q/3Q 2021 with initial safety and 30-day biomarker data to be reported in 1H22. The global clinical trial GALax-C for PBKR03 for Krabbe disease is expected to initiate in 3Q21 with initial safety and 30-day biomarker data to be reported in 1H22.
- Eliseo O. Salinas, M.D., MSc, appointed as chief research & development officer: In March 2021, the company appointed Dr. Salinas as the company's chief research and development (R&D) officer. Dr. Salinas has extensive experience in the development of small molecules, biologics and cell therapy and has been directly involved with numerous investigational new drug (IND) application submissions and regulatory approvals in the United States and globally.
- Maxine Gowen, Ph.D., appointed to Passage Bio board of directors: In February 2021, the company announced the appointment of Dr. Gowen to its board of directors. She brings significant public company leadership and clinical development expertise, having co-founded and led a start-up biotech company and served in a variety of leadership roles at GlaxoSmithKline over a period of 15 years.
- Raised $166 million in public offering of common stock: In January 2021, the company announced a public offering of 7,000,000 shares of its common stock at a price of $22 per share. The underwriters also exercised their option to purchase an additional 1,050,000 shares of common stock for total offering net proceeds of $166 million.
Anticipated Upcoming Milestones
- Host R&D day focused on FTD-GRN, May 17.
- Open CMC research and development site in Hopewell, NJ, by the end of the second quarter of 2021.
- Report initial safety and 30-day biomarker data from Phase 1/2 PBGM01 trial in 4Q21.
- Dose first patient in Phase 1/2 FTD-GRN trial in 2Q/3Q21; report initial safety and 30-day biomarker data in 1H22.
- Dose first patient in Phase 1/2 Krabbe trial in 3Q21; report initial safety and 30-day biomarker data in 1H22.
- Continue to advance preclinical programs for PBML04 (Metachromatic leukodystrophy), PBLA05 (Amyotrophic lateral sclerosis) and PBCM06 (Charcot-Marie-Tooth Disease Type 2A), and an undisclosed adult CNS program.
First Quarter 2021 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were $437.6 million as of March 31, 2021 as compared to $304.8 million as of December 31, 2020. This includes $166 million in net proceeds from the company's public offering in January 2021.
- Research and Development (R&D) Expenses: R&D expenses were $25.0 million for the first quarter ended March 31, 2021, compared to $13.1 million for the same quarter in 2020. Acquired in-process R&D expenses were $1.5 million for the first quarter ended March 31, 2021, compared to none in the same quarter of 2020.
- General and Administrative (G&A) Expenses: G&A expenses were $12.5 million for the first quarter ended March 31, 2021, compared to $4.8 million for the same quarter in 2020.
- Net Loss: Net loss was $38.9 million, or a net loss of $0.76 per basic and diluted share, for the quarter ended March 31, 2021 compared to $17.6 million, or a net loss of $1.00 per basic and diluted share, for the same quarter in 2020.
Conference Call Details
Passage Bio will host a conference call and webcast today at 8:30 a.m. ET. To access the live conference call, please dial 833-528-0605 (domestic) or 830-221-9711 (international) and reference conference ID number 6366724. A live audio webcast of the event will be available on the Investors & Media section of Passage Bio's website at investors.passagebio.com. The archived webcast will be available on Passage Bio's website approximately two hours after the completion of the event and for 30 days following the call.About Passage Bio
At Passage Bio (NASDAQ:PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania's Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at www.passagebio.com.Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about 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; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "potential," "possible," "will," "would," and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.For further information, please contact:
Investors:
Stuart Henderson
Passage Bio
267.866.0114
[email protected]Media:
Gwen Fisher
Passage Bio
215.407.1548
[email protected]Passage Bio, Inc.
Balance Sheets
(Unaudited)(in thousands, except share data) March 31, 2021 December 31, 2020 Assets Current assets: Cash and cash equivalents $ 249,521 $ 135,002 Marketable securities 188,068 169,815 Prepaid expenses 3,374 1,405 Prepaid research and development 9,986 10,961 Total current assets 450,949 317,183 Property and equipment, net 6,574 2,795 Other assets 7,649 8,029 Total assets $ 465,172 $ 328,007 Liabilities and stockholders' equity Current liabilities: Accounts payable $ 6,248 $ 5,265 Accrued expenses and other current liabilities 13,221 15,910 Total current liabilities 19,469 21,175 Deferred rent 4,199 2,077 Other liabilities — 41 Total liabilities 23,668 23,293 Commitments and Contingencies Stockholders' equity: Common stock, $0.0001 par value: 300,000,000 shares authorized; 53,977,484 shares issued and 53,848,324 shares outstanding at March 31, 2021 and 45,917,084 shares issued and 45,614,807 shares outstanding at December 31, 2020 5 4 Additional paid‑in capital 651,283 475,617 Accumulated other comprehensive loss (7 ) (12 ) Accumulated deficit (209,777 ) (170,895 ) Total stockholders' equity 441,504 304,714 Total liabilities and stockholders' equity $ 465,172 $ 328,007 .
Passage Bio, Inc.
Statements of Operations and Comprehensive Loss
(Unaudited)Three Months Ended March 31, (in thousands, except share and per share data) 2021 2020 Operating expenses: Research and development $ 24,970 $ 13,117 Acquired in‑process research and development 1,500 — General and administrative 12,464 4,795 Loss from operations (38,934 ) (17,912 ) Interest income, net 52 327 Net loss $ (38,882 ) $ (17,585 ) Per share information: Net loss per share of common stock, basic and diluted $ (0.76 ) $ (1.00 ) Weighted average common shares outstanding, basic and diluted 51,331,449 17,624,011 Comprehensive loss: Net loss $ (38,882 ) $ (17,585 ) Unrealized gain on marketable securities 5 — Comprehensive loss $ (38,877 ) $ (17,585 ) -
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- No-cost genetic screening and counseling program aimed at identifying FTD patients who have certain inherited genetic mutations to guide early treatment intervention and awareness of clinical trials
- Passage Bio is evaluating gene therapy candidate, PBFT02, for treatment of FTD with granulin mutations
PHILADELPHIA, May 03, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ:PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders; and InformedDNA, the nation's leading genetics services organization, today announced a collaboration to provide no-cost genetic counseling and testing for adults who have been diagnosed by their physicians with Frontotemporal Dementia (FTD).
The testing program will facilitate identification of patients with FTD with certain inherited genetic mutations, providing an important step for early and precise treatment intervention, as well as supporting clinical trial recruitment and enrollment. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. Approximately 30 percent of all FTD is hereditary and most commonly involves a mutation of the granulin (GRN), C9orf72 or MAPT genes – all of which are tested for in this program.
"FTD is a life-threatening condition that progresses rapidly and has an average survival of eight years after onset of symptoms, so it is critical that patients are identified as early as possible to achieve the best outcomes for them," said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. "By partnering with InformedDNA, we are able to offer patients with FTD an option to potentially identify whether there is an inherited genetic mutation causing their disease. This will enable clinicians to intervene sooner with an appropriate treatment approach for their specific form of the disorder. We believe this collaboration can serve as a valuable resource for the FTD community, allowing for earlier treatment or participation in clinical trials aimed at finding innovative treatment options."
Adults who have been diagnosed with FTD by a doctor are eligible for genetic counseling and testing for certain genetic mutations at no cost through the Passage Bio-sponsored program. Individuals will receive initial and post-test genetic counseling over the phone from InformedDNA genetic counselors who are highly trained in hereditary neurological conditions. If the individual elects to proceed with genetic testing following initial counseling, then the genetic counselor will coordinate the test order and sample collection. Test results will be available in approximately three weeks from laboratory receipt of the individual's sample. If patients test positive for a genetic mutation, in addition to counseling, they will be given information about potential treatment and clinical trial options specific to their mutation, if available.
"It's estimated that FTD affects 50,000-60,000 Americans and it is the leading cause of dementia for those under the age of 65. We're proud to leverage our deep expertise in the genetics of neurodegenerative diseases and our extensive community-based referring physician network to increase access to genetics services for patients with frontotemporal dementia," said Karmen Trzupek, director of clinical trial services at InformedDNA.
Additional information on the FTD genetic counseling and testing process offered through the program, as well as tools and information for healthcare providers, patients and caregivers, is available at https://informeddna.com/passagebio-ftd/.
Passage Bio Plans to Initiate a Phase 1/2 Clinical Study for an FTD Gene Therapy
Passage Bio is developing PBFT02, an adeno-associated virus (AAV)-delivery gene therapy, for the treatment of patients with FTD with granulin (GRN) mutations. FTD is one of the more common causes of early-onset (midlife) dementia, causing impairment in behavior, language and executive function, and occurs at similar frequency to Alzheimer's disease in patients younger than 65 years. In approximately 5 to 10 percent of individuals with FTD – 3,000 to 6,000 people in the United States – the disease occurs because of mutations in the GRN gene, causing a deficiency of PGRN, a complex and highly conserved protein. The mechanism by which PGRN deficiency results in FTD is uncertain, but increasing evidence points to PGRN's role in lysosomal function.
More information about the global Phase 1/2 PBFT02 study, upliFT-D, can be found at ClinicalTrials.gov: NCT04747431.
About Passage Bio
At Passage Bio (NASDAQ:PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania's Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at www.passagebio.com.
About InformedDNA
InformedDNA is the authority on the appropriate use of genetic testing. It leverages the expertise of the largest, independent staff of board-certified genetics specialists in the U.S. to help ensure that patients, clinicians, health plans, health systems, employers, pharmaceutical companies and all stakeholders have access to the highest quality genetic services. Key offerings include clinical genetic counseling, genetic testing utilization management, genetic testing payment integrity, and expert genetics support for clinical trials. For more information: https://www.informeddna.com/.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about 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; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "potential," "possible," "will," "would," and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
For further information, please contact:
Passage Bio Investors:
Stuart Henderson
Passage Bio
267-866-0114
[email protected]Passage Bio Media:
Gwen Fisher
Passage Bio
215-407-1548
[email protected]InformedDNA Media:
Savannah Matthews Lentz, MERGE
803- 883-6628
[email protected] - No-cost genetic screening and counseling program aimed at identifying FTD patients who have certain inherited genetic mutations to guide early treatment intervention and awareness of clinical trials
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PHILADELPHIA, April 29, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ:PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that it will host a conference call and live webcast at 8:30 a.m. ET on Wednesday, May 5, 2021 to report its first quarter 2021 financial results and discuss recent business highlights.
To access the live conference call, please dial 833-528-0605 (domestic) or 830-221-9111 (international) and refer to conference ID 6366724. A live audio webcast of the event will be available on the Investors & Media section of Passage Bio's website at investors.passagebio.com. The archived webcast will be available on Passage Bio's website approximately two hours after the completion of the event and for 30 days following the call.
About Passage Bio
At Passage Bio (NASDAQ:PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania's Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at www.passagebio.com.
For further information, please contact:
Passage Bio Investors:
Stuart Henderson
Passage Bio
267-866-0114
[email protected]Passage Bio Media:
Gwen Fisher
Passage Bio
215-407-1548
[email protected]