PASG Passage Bio

Passage Bio Reports First Quarter 2021 Financial Results and Recent Business Highlights

Passage Bio Reports First Quarter 2021 Financial Results and Recent Business Highlights

  • 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 . 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 .

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

 

Media:

Gwen Fisher

Passage Bio

215.407.1548

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)

 



EN
05/05/2021

Underlying

To request access to management, click here to engage with our
partner Phoenix-IR's CorporateAccessNetwork.com

Reports on Passage Bio

 PRESS RELEASE

Passage Bio Seeks Pennsylvania College Student Candidates for Third An...

Passage Bio Seeks Pennsylvania College Student Candidates for Third Annual Tachi Yamada Scholarship Program $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 ...

Laura Chico
  • Laura Chico
 PRESS RELEASE

Passage Bio Reports Fourth Quarter and Full-Year 2023 Financial Result...

Passage Bio Reports Fourth Quarter and Full-Year 2023 Financial Results and Provides Recent Business Highlights 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 Q...

Wedbush Research
  • Wedbush Research
2454 MEDIATEK INC.
MTB M&T BANK CORPORATION
UBI UBISOFT ENTERTAINMENT SA
VMW VMWARE INC. CLASS A
XNCR XENCOR INC.
XENE XENON PHARMACEUTICALS INC
WDC WESTERN DIGITAL CORPORATION
WBS WEBSTER FINANCIAL CORPORATION
VYGR VOYAGER THERAPEUTICS INC.
VLY VALLEY NATIONAL BANCORP
TTWO TAKE-TWO INTERACTIVE SOFTWARE INC.
TRIP TRIPADVISOR INC.
SIMON SILICON MOTION TECHNOLOGY CORPORATION SPONSORED ADR
SGMO SANGAMO THERAPEUTICS INC.
RHP RYMAN HOSPITALITY PROPERTIES INC.
RARE ULTRAGENYX PHARMACEUTICAL INC.
PZZA PAPA JOHN'S INTERNATIONAL INC.
PSTG PURE STORAGE INC. CLASS A
PEB PEBBLEBROOK HOTEL TRUST
PCRX PACIRA BIOSCIENCES INC.
NYCB NEW YORK COMMUNITY BANCORP INC.
NVDA NVIDIA CORPORATION
NVCR NOVOCURE LTD.
NTAP NETAPP INC.
NBIX NEUROCRINE BIOSCIENCES INC.
MU MICRON TECHNOLOGY INC.
KURA KURA ONCOLOGY INC.
KPTI KARYOPHARM THERAPEUTICS
INTC INTEL CORPORATION
GOOGL ALPHABET INC. CLASS A
GLPI GAMING AND LEISURE PROPERTIES INC.
GERN GERON CORP.
FATE FATE THERAPEUTICS INC
EXPE EXPEDIA GROUP INC.
CTMX CYTOMX THERAPEUTICS INC.
CMRX CHIMERIX INC
CFG CITIZENS FINANCIAL GROUP INC.
BPMC BLUEPRINT MEDICINES CORP.
BMRN BIOMARIN PHARMACEUTICAL INC.
BKU BANKUNITED INC.
BIIB BIOGEN INC.
BBY BEST BUY CO. INC.
AXTI AXT INC.
ARDX ARDELYX INC
HST HOST HOTELS & RESORTS INC.
GRBK GREEN BRICK PARTNERS
FRX_CN FENNEC PHARMACEUTICALS
7974 NINTENDO CO. LTD.
2330 TAIWAN SEMICONDUCTOR MANUFACTURING CO. LTD.
2303 UNITED MICROELECTRONICS CORP.
EA ELECTRONIC ARTS INC.
HPE HEWLETT PACKARD ENTERPRISE CO.
UTHR UNITED THERAPEUTICS CORPORATION
CLSD CLEARSIDE BIOMEDICAL INC
ALBO ALBIREO PHARMA
PK PARK HOTELS & RESORTS INC.
REAL REAL MATTERS
ANAB ANAPTYSBIO INC.
KALA KALA PHARMACEUTICALS INC.
ARGX ARGENX ADS
MRSN MERSANA THERAPEUTICS
GTHX G1 THERAPEUTICS
ALPN ALPINE IMMUNE SCIENCES
VRNA VERONA PHARMA ADS
VICI VICI PROPERTIES INC
AMZN AMAZON.COM INC.
AMD ADVANCED MICRO DEVICES INC.
AAPL APPLE INC.
MSFT MICROSOFT CORPORATION
APLS APELLIS PHARMACEUTICALS
BKNG BOOKING HOLDINGS INC.
DNLI DENALI THERAPEUTICS INC
RCUS ARCUS BIOSCIENCES
AVGO BROADCOM INC.
ZS ZSCALER INC.
SURF SURFACE ONCOLOGY
SRRK SCHOLAR ROCK HOLDING CORPORATION
KNSA KINIKSA PHARMACEUTICALS
LQDA LIQUIDIA CORPORATION
REPL REPLIMUNE GROUP
AX AXOS FINANCIAL INC.
ARVN ARVINAS HOLDING
YMAB Y-MABS THERAPEUTICS
STRO SUTRO BIOPHARMA
LYFT LYFT
UBER UBER TECHNOLOGIES INC.
BCEL ATRECA
DELL DELL TECHNOLOGIES INC CLASS C
STOK STOKE THERAPEUTICS
IGMS IGM BIOSCIENCES
SWTX SPRINGWORKS THERAPEUTICS
AQST AQUESTIVE THERAPEUTICS
IDYA IDEAYA BIOSCIENCES
PASG PASSAGE BIO
BDTX BLACK DIAMOND THERAPEUTICS
ACET ADICET BIO INC
ITOS ITEOS THERAPEUTICS
ORIC ORIC PHARMACEUTICALS
ZNTL ZENTALIS PHARMACEUTICALS
PRAX PRAXIS PRECISION MEDICINES
U UNITY SOFTWARE
TVTX TRAVERE THERAPEUTICS INC
DASH DOORDASH
ABNB AIRBNB INC. CLASS A
LSEA LANDSEA HOMES CORP (A)
VRDN VIRIDIAN THERAPEUTICS INC
PLTK PLAYTIKA HOLDING
NUVB NUVATION BIO INC (A)
RBLX ROBLOX
DAWN DAY ONE BIOPHARMACEUTICALS INC
ELEV ELEVATION ONCOLOGY
OMGA OMEGA THERAPEUTICS
LBPH LONGBOARD PHARMACEUTICALS
THRX THESEUS PHARMACEUTICALS
MRVL INC.
STX MARVELL TECHNOLOGY INC
APP SEAGATE TECHNOLOGY HLDGS PLC
DSGN APPLOVIN CORP
ATXS DESIGN THERAPEUTICS INC
AVTE ASTRIA THERAPEUTICS INC
GFS AEROVATE THERAPEUTICS INC
RLYB GLOBALFOUNDRIES INC
KRTX RALLYBIO CORP
FUSN KARUNA THERAPEUTICS INC
YOU FUSION PHARMACEUTICALS INC
VIGL CLEAR SECURE INC
MLTX VIGIL NEUROSCIENCE INC
SOUN MOONLAKE IMMUNOTHERAPEUTICS
EWTX SOUNDHOUND AI INC
DDI EDGEWISE THERAPEUTICS INC
SKLZ DOUBLEDOWN INTERACTIVE CO LTD
EMBRAC B SKILLZ INC
EMBRACER GROUP AB
Andreas Argyrides ... (+11)
  • Andreas Argyrides
  • Caroline Pocher
  • David Carlson
  • David Nierengarten
  • Dennis Pak
  • Laura Chico
  • Liana Moussatos
  • Martin Fan
  • Ritika Das
  • Robert Driscoll
  • Sam Ravina
XNCR XENCOR INC.
XENE XENON PHARMACEUTICALS INC
VYGR VOYAGER THERAPEUTICS INC.
SGMO SANGAMO THERAPEUTICS INC.
RARE ULTRAGENYX PHARMACEUTICAL INC.
PCRX PACIRA BIOSCIENCES INC.
NVCR NOVOCURE LTD.
NBIX NEUROCRINE BIOSCIENCES INC.
KURA KURA ONCOLOGY INC.
KPTI KARYOPHARM THERAPEUTICS
GERN GERON CORP.
FATE FATE THERAPEUTICS INC
CTMX CYTOMX THERAPEUTICS INC.
CMRX CHIMERIX INC
BPMC BLUEPRINT MEDICINES CORP.
BMRN BIOMARIN PHARMACEUTICAL INC.
BIIB BIOGEN INC.
ARDX ARDELYX INC
FRX_CN FENNEC PHARMACEUTICALS
UTHR UNITED THERAPEUTICS CORPORATION
CLSD CLEARSIDE BIOMEDICAL INC
ALBO ALBIREO PHARMA
ANAB ANAPTYSBIO INC.
KALA KALA PHARMACEUTICALS INC.
ARGX ARGENX ADS
MRSN MERSANA THERAPEUTICS
GTHX G1 THERAPEUTICS
ALPN ALPINE IMMUNE SCIENCES
APLS APELLIS PHARMACEUTICALS
DNLI DENALI THERAPEUTICS INC
RCUS ARCUS BIOSCIENCES
SURF SURFACE ONCOLOGY
SRRK SCHOLAR ROCK HOLDING CORPORATION
KNSA KINIKSA PHARMACEUTICALS
LQDA LIQUIDIA CORPORATION
REPL REPLIMUNE GROUP
ARVN ARVINAS HOLDING
YMAB Y-MABS THERAPEUTICS
STRO SUTRO BIOPHARMA
BCEL ATRECA
STOK STOKE THERAPEUTICS
IGMS IGM BIOSCIENCES
SWTX SPRINGWORKS THERAPEUTICS
AQST AQUESTIVE THERAPEUTICS
IDYA IDEAYA BIOSCIENCES
PASG PASSAGE BIO
BDTX BLACK DIAMOND THERAPEUTICS
ACET ADICET BIO INC
ITOS ITEOS THERAPEUTICS
ZNTL ZENTALIS PHARMACEUTICALS
PRAX PRAXIS PRECISION MEDICINES
TVTX TRAVERE THERAPEUTICS INC
VRDN VIRIDIAN THERAPEUTICS INC
DAWN DAY ONE BIOPHARMACEUTICALS INC
ELEV ELEVATION ONCOLOGY
OMGA OMEGA THERAPEUTICS
LBPH LONGBOARD PHARMACEUTICALS
THRX THESEUS PHARMACEUTICALS
DSGN INC.
ATXS DESIGN THERAPEUTICS INC
AVTE ASTRIA THERAPEUTICS INC
RLYB AEROVATE THERAPEUTICS INC
KRTX RALLYBIO CORP
FUSN KARUNA THERAPEUTICS INC
VIGL FUSION PHARMACEUTICALS INC
EWTX VIGIL NEUROSCIENCE INC
EDGEWISE THERAPEUTICS INC

ResearchPool Subscriptions

Get the most out of your insights

Get in touch