PGRN

LILLE, France--(BUSINESS WIRE)-- ALZPROTECT is thrilled to announce that it has received favorable feedback from both the American (FDA) and European (EMA) authorities regarding the regulatory path for advancing the clinical development of Ezeprogind/AZP2006 for patients with PSP. This significant milestone follows the already very promising results of the phase 2a study in PSP and marks a decisive advancement. It will provide a clear path for the further development of Ezeprogind/AZP2006 within both scientific and regulatory expectations. This press release features multimedia. View the full release here: Regulatory authorities have provided crucial insights into various design elements, with a particular focus on selecting study endpoints pertinent to the PSP indication and ensuring that the demographic and characteristic pro the study sample closely mirrors the global PSP population. This feedback has armed Alzprotect with a valuable roadmap, enabling the refinement of the intended Phase 2/3 study protocol to ensure seamless alignment with scientific standards and regulatory requirements. With well-defined regulatory pathways for the PSP indication, Alzprotect's forthcoming Proof of concept randomized controlled PROMISE-PSP trial, scheduled to commence in late 2024, is set to deliver outcomes that align with scientific and regulatory expectations across the United States and the European Union. Dr. Artin Karapet, our Chief Medical Officer, expressed his heartfelt joy stating, "I am truly delighted to witness this alignment between agencies from different shores on critical program elements. Their guidance not only paves a feasible path forward but also brings AZP2006 one step closer to our patient community." Phil Verwaerde, Chief Executive Officer, added, "The alignment with regulatory authorities marks a pivotal moment for ALZPROTECT. It validates our vision and the potential of Ezeprogind/AZP2006 not only for PSP but also for a broader neurodegenerative disease spectrum. We are dedicated to accelerating our efforts to meet the urgent needs of patients." The molecule, AZP2006, proposes a fundamentally distinct mechanism of action compared to existing treatments: Targeting the root causes of neurodegeneration by stimulating the action of a protein essential to the functioning of the brain, Progranulin. AZP2006 has shown excellent safety and promising efficacy in human trials, including 102 healthy subjects across three Phase I trials and 36 PSP patients in a Phase 2a trial. Alzprotect plans to initiate a randomized Phase 2b/3 proof of concept for PSP and expand its drug development to include Parkinson's disease, Alzheimer's disease, and amyotrophic lateral sclerosis in the coming years. View source version on businesswire.com: Contacts p.verwaerde@alzprotect.com Source: Alzprotect Smart Multimedia Gallery Video Alzprotect's corporate video Logo View this news release online at:

$10,000 scholarship to be awarded to a rising junior with a life science major attending a Pennsylvania-based four-year college or university Scholarship application deadline is April 26 for the initial award in the fall of 2024 PHILADELPHIA, March 05, 2024 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today announced the opening of its application process for its third annual Tachi Yamada Scholarship program. The scholarship program is for junior students enrolled in life science programs at four-year colleges and universities in Pennsylvania and who plan to have a career in the life sciences industry. Passage Bio will select one sophomore student annually to award $10,000 in scholarship funding – $5,000 over two consecutive years to be used toward college-related expenses. Interested sophomore students have until April 26, 2024, to apply for the scholarship. Passage Bio first announced its plans to launch the annual Tachi Yamada Scholarship Program in October 2021 to continue the legacy of Tadataka (Tachi) Yamada, M.D., a visionary biopharma industry leader who passed away in August 2021. A co-founder of Passage Bio and chairman of the company's board of directors, Tachi demonstrated a lifelong commitment to mentoring and fostering the development of the upcoming generation of scientists. “Entering its third year, the Tachi Yamada Scholarship program stands as a proud testament to our ongoing dedication to nurturing students who aspire to drive meaningful change through the realms of science. Through this initiative, we pay homage to Tachi's enduring legacy and his invaluable contributions to our field," said Will Chou, M.D., president and chief executive officer of Passage Bio. “We look forward to welcoming and supporting the next exceptional scholar who will undoubtedly make an impact in the life sciences industry.” In addition to the financial award, the selected scholar will be matched with a Passage Bio mentor for professional growth opportunities over the two-year scholarship period. To apply, students must be sophomores, who will be juniors in the fall of 2024, attending four-year colleges or universities in Pennsylvania and majoring in a life science discipline. They must have a cumulative unweighted grade point average of 3.0 and be a United States citizen or permanent resident of the United States or hold a U.S. student visa. The student must also write a 500-word essay as part of the application process. The official rules and application details are available on Passage Bio’s website in the Careers section at Passage Bio, Inc. – Careers. Applications must be submitted by April 26, 2024 and will be evaluated by a panel of Passage Bio employees; the winner will be selected by Passage Bio at its discretion. About Passage Bio Passage Bio (Nasdaq: PASG) is a clinical stage genetic medicines company on a mission to improve the lives of patients with neurodegenerative diseases. Our primary focus is the development and advancement of cutting-edge, one-time therapies designed to target the underlying pathology of these conditions. Passage Bio’s lead product candidate, PBFT02, seeks to treat neurodegenerative conditions, including frontotemporal dementia, by elevating progranulin levels to restore lysosomal function and slow disease progression. To learn more about Passage Bio and our steadfast commitment to protecting patients and families against loss in neurodegenerative conditions, please visit: www.passagebio.com. For further information, please contact: Investors: Stuart Henderson Passage Bio 267.866.0114 shenderson@passagebio.com Media:Mike BeyerSam Brown Inc. Healthcare Communications312.961.2502MikeBeyer@sambrown.com

Continued momentum in upliFT-D trial of PBFT02 for the treatment of patients with frontotemporal dementia (FTD) with granulin mutations (GRN) with clinical trial site expansion into Europe and seven global sites now activatedPursuing new indications for PBFT02, including adult neurodegenerative diseases FTD-C9orf72, amyotrophic lateral sclerosis (ALS) and Alzheimer’s diseaseEnded 2023 with robust balance sheet sufficient to deliver meaningful clinical data; with cash runway into Q4 2025 PHILADELPHIA, March 04, 2024 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today reported financial results for the fourth quarter and year ended December 31, 2023 and provided recent business highlights. "In 2023, we achieved a significant milestone by announcing encouraging data from three patients in Cohort 1 of our FTD-GRN program. This data showcased the ability of PBFT02 to raise CSF progranulin to supraphysiologic levels, even at the lowest dose, Dose 1, and positions PBFT02 as a potential best-in-class treatment for FTD-GRN. Our global upliFT-D study continues to see strong momentum, and we're excited to announce the activation of our first clinical trial site in Europe to further bolster recruitment efforts. We are on track to complete dosing for Cohort 1 patients in the first half of this year and share 6-month additional safety and biomarker data from Cohort 1 in the second half of 2024," said Will Chou, M.D., president and chief executive officer of Passage Bio. "Buoyed by this promising data and additional evidence supporting progranulin's role in neurodegeneration, we are optimistic about expanding the application of PBFT02 beyond FTD-GRN and into new indications with significant unmet needs, including FTD-C9orf72, ALS, and Alzheimer's disease. We eagerly anticipate a catalyst-rich 2024, supported by our robust balance sheet, bringing us closer to improving patient outcomes across neurodegenerative diseases." Recent Highlights Sustained momentum in global Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for frontotemporal dementia (FTD) with granulin mutations (GRN), marked by the activation of two new trial sites and expansion into Europe: Initial data from the first three patients in Cohort 1 demonstrated elevated CSF progranulin levels to supraphysiologic levels at the lowest tested dose, Dose 1, up to six months post-treatment (n=1). Additionally, Dose 1 of PBFT02 continued to be generally well-tolerated in patients who received an enhanced steroid regimen for immunosuppression. In the first half of 2024, the company expects to complete dosing for Cohort 1 patients and report 6-month safety and biomarker data in the second half of 2024. Following the positive data, multiple GRN+ patients have been identified and are being evaluated for eligibility across seven clinical trial sites in Brazil, Canada, the United States and Europe.Expansion of PBFT02 into additional adult neurodegenerative indications: In response to the promising initial data for PBFT02 in FTD-GRN and evidence supporting progranulin’s role in neurodegeneration, the company is currently evaluating the therapeutic potential of PBFT02 in additional indications, including FTD-C9orf72, ALS, and Alzheimer’s disease. The company is preparing for regulatory discussions and expects to obtain regulatory feedback on the clinical pathway to treating FTD-C9orf72 and ALS patients with PBFT02 in 2H 2024.Robust balance sheet to support operations: The company’s decision to focus resources on the exploration of PBFT02 in additional indications, while simultaneously pursuing potential out-licensing opportunities for clinical-stage pediatric programs in GM1 gangliosidosis, Krabbe disease and metachromatic leukodystrophy, provides sufficient cash runway into Q4 2025 to execute through critical value-inflecting milestones. Appointed Kathleen Borthwick as Chief Financial Officer, effective March 1, 2024: Ms. Borthwick previously served as the company’s Senior Vice President, Finance and Interim CFO and brings 25 years of pharmaceutical and biotech strategic financial leadership experience. Anticipated Upcoming Milestones: FTD-GRN Initiate dosing for Cohort 2 FTD-GRN patients in 1H 2024Report 6-month safety and biomarker data from Cohort 1 patients in 2H 2024Announce 12-month follow-up data from Cohort 1 patients in 1H 2025Report initial safety and biomarker data from Cohort 2 patients in 1H 2025 FTD-C9orf72 and ALS Obtain regulatory feedback on the pathway to treating FTD-C9orf72 and ALS patients with PBFT02 in 2H 2024 Fourth Quarter and Full-Year 2023 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $114.3 million as of December 31, 2023, as compared to $189.6 million as of December 31, 2022. The company expects current cash, cash equivalents and marketable securities to fund operations into Q4 2025.Research and Development (R&D) Expenses: R&D expenses were $12.1 million for the quarter ended December 31, 2023, and $61.4 million for the year ended December 31, 2023, compared to $17.7 million and $86.1 million for the same quarter and year ended in 2022, respectively.General and Administrative (G&A) Expenses: G&A expenses were $6.3 million for the quarter ended December 31, 2023, and $41.6 million for the year ended December 31, 2023, compared to $10.6 million and $49.3 million for the same quarter and year ended in 2022, respectively.Net Loss: Net loss was $16.8 million, or $0.30 per basic and diluted share, for the quarter ended December 31, 2023 and $102.1 million, or $1.86 per basic and diluted share, for the year ended December 31, 2023, compared to a net loss of $27.1 million, or $0.50 per basic and diluted share, for the quarter ended December 31, 2022 and $136.1 million, or $2.50 per basic and diluted share, for the year ended December 31, 2022. About Passage Bio Passage Bio (Nasdaq: PASG) is a clinical stage genetic medicines company on a mission to improve the lives of patients with neurodegenerative diseases. Our primary focus is the development and advancement of cutting-edge, one-time therapies designed to target the underlying pathology of these conditions. Passage Bio’s lead product candidate, PBFT02, seeks to treat neurodegenerative conditions, including frontotemporal dementia, by elevating progranulin levels to restore lysosomal function and slow disease progression. To learn more about Passage Bio and our steadfast commitment to protecting patients and families against loss in neurodegenerative conditions, please visit: www.passagebio.com. Forward-Looking StatementsThis 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 the progress 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; our expectations about potential out-licensing opportunities related to PBGM01, PBKR03 and PBML04; and the ability of our product candidates to treat their respective target 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; 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. Passage Bio, Inc.Balance Sheets December 31,(in thousands, except share and per share data) 2023 2022Assets Current assets: Cash and cash equivalents $21,709 $34,601 Marketable securities 92,585 155,009 Prepaid expenses and other current assets 923 926 Prepaid research and development 2,742 6,508 Total current assets 117,959 197,044 Property and equipment, net 15,295 22,515 Right of use assets - operating leases 16,858 19,723 Other assets 433 4,267 Total assets $150,545 $243,549 Liabilities and stockholders’ equity Current liabilities: Accounts payable $1,298 $4,065 Accrued expenses and other current liabilities 11,670 11,011 Operating lease liabilities 3,373 3,275 Total current liabilities 16,341 18,351 Operating lease liabilities – noncurrent 22,921 23,832 Total liabilities 39,262 42,183 Stockholders’ equity: Preferred stock, $0.0001 par value: 10,000,000 shares authorized; no shares issued and outstanding at both December 31, 2023 and December 31, 2022 — — Common stock, $0.0001 par value: 300,000,000 shares authorized; 54,944,130 shares issued and outstanding at December 31, 2023 and 54,614,690 shares issued and outstanding at December 31, 2022 5 5 Additional paid‑in capital 705,789 694,733 Accumulated other comprehensive income (loss) (43) (966)Accumulated deficit (594,468) (492,406)Total stockholders’ equity 111,283 201,366 Total liabilities and stockholders’ equity $150,545 $243,549 Passage Bio, Inc.Statements of Operations and Comprehensive Loss Year Ended December 31,(in thousands, except share and per share data) 2023 2022Operating expenses: Research and development $61,419 $86,053 Acquired in‑process research and development — 3,000 General and administrative 41,580 49,341 Impairment of long-lived assets 5,390 — Loss from operations (108,389) (138,394)Other income (expense), net 6,327 2,269 Net loss $(102,062) $(136,125)Per share information: Net loss per share of common stock, basic and diluted $(1.86) $(2.50)Weighted average common shares outstanding, basic and diluted 54,743,490 54,429,023 Comprehensive loss: Net loss $(102,062) $(136,125)Unrealized gain (loss) on marketable securities 923 (553)Comprehensive loss $(101,139) $(136,678) For further information, please contact: Investors:Stuart HendersonPassage Bio267.866.0114shenderson@passagebio.com Media:Mike BeyerSam Brown Inc. Healthcare Communications312.961.2502MikeBeyer@sambrown.com