PepGen to Focus on Development of Promising DM1 Program Following 10 mg/kg PGN-EDO51 Update

BOSTON--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies, today announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping, the Company will focus on advancing its promising myotonic dystrophy type 1 (DM1) program currently in Phase 2 clinical development. The Company is voluntarily discontinuing development of PGN-EDO51 and intends to wind down all DMD-related research and development activities.
In the 10 mg/kg cohort (n=4) of the CONNECT1 study, PGN-EDO51 increased exon 51 skipped transcripts to 4.26% (a mean increase of 3.5%); however, total dystrophin only increased to 0.59% of normal levels (a mean increase of 0.36%). The safety profile of PGN-EDO51 continued to be generally favorable and all treatment-related adverse events were mild in nature. No serious adverse events were reported in the study.
'We are disappointed by the dystrophin results observed in the 10 mg/kg dose cohort in CONNECT1, as it was our hope that we could improve upon existing therapies for patients in a more profound way,' said James McArthur, PhD, President and CEO of PepGen. 'As we wind down our DMD program, we would like to thank the patients, families, caregivers, investigators and study staff for their support and participation in this research. I also want to acknowledge our team's hard work and commitment to advancing new potential treatments for DMD patients.'
Paul Streck, MD, MBA, Executive Vice President, Head of R&D of PepGen continued, 'PGN-EDODM1, PepGen's investigational drug in development for DM1, has already demonstrated robust target engagement after a single 10 mg/kg dose in patients that resulted in mean mis-splicing correction of 29% with a favorable emerging safety profile as of February 24, 2025, the most recent safety update. Mis-splicing is the primary driver of DM1 pathology and currently patients have no approved treatment options for this disabling, life-shortening disorder. Going forward, we will focus our resources on advancing the Company's ongoing DM1 clinical program along with our research pipeline.'
PepGen continues to expect to report data from its FREEDOM-DM1 15 mg/kg cohort in patients with DM1 during the second half of 2025. FREEDOM is a Phase 1 single ascending dose, randomized, placebo-controlled clinical trial, with endpoints including safety, 28-day splicing correction and functional benefit measures. The Company also continues to expect to report data from the 5 mg/kg cohort of its FREEDOM2-DM1 study in DM1 patients in the first quarter of 2026. FREEDOM2 is a Phase 2 multiple ascending dose, randomized, placebo-controlled clinical trial, with endpoints following four doses that include safety, splicing correction and functional benefit measures.
About PGN-EDODM1
PGN-EDODM1, PepGen's investigational candidate in development for the treatment of myotonic dystrophy Type 1 (DM1), utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 is designed to directly address the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the dystrophia myotonic protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, disrupting the binding between the CUG repeat expansion and MBNL1. DM1 is a progressively disabling, life-shortening genetic disorder. DM1 is estimated to affect 40,000 people in the United States, and over 74,000 people in Europe. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1.
About PepGen
PepGen is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen's EDO platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.
For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as 'aims,' 'anticipates,' 'believes,' 'could,' 'estimates,' 'expects,' 'forecasts,' 'goal,' 'intends,' 'may,' 'plans,' 'possible,' 'potential,' 'seeks,' 'will,' and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential and safety profile of our product candidates, including PGN-EDO51 and PGN-EDODM1, our plans to discontinue our DMD-related research and development activities, including our CONNECT1-EDO51 and CONNECT2-EDO51 Phase 2 clinical trials, based on results from the CONNECT1 trial, the design and conduct of clinical trials, including our FREEDOM-DM1 Phase 1 trial and FREEDOM2-DM1 Phase 2 trial, and the expected timing for additional data from our FREEDOM Phase 1 trial and initial data from our FREEDOM2 Phase 2 trial.
Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to risks related to: delays or failure to successfully initiate or complete our ongoing and planned development activities for our product candidates, including PGN-EDODM1; our ability to enroll patients in our clinical trials, including FREEDOM and FREEDOM2; that our interpretation of clinical and preclinical study results may be incorrect, or that we may not observe the levels of therapeutic activity in clinical testing that we anticipate based on prior clinical or preclinical results, including for PGN-EDODM1; our product candidates, including PGN-EDODM1, may not be safe and effective or otherwise demonstrate safety and efficacy in our clinical trials; adverse outcomes from our regulatory interactions, including delays in regulatory review, clearance to proceed or approval by regulatory authorities with respect to our programs, including clearance to commence planned clinical studies of our product candidates, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our FREEDOM and FREEDOM2 clinical trials; changes in regulatory framework that are out of our control; unexpected increases in the expenses associated with our development activities or other events that adversely impact our financial resources and cash runway; and our dependence on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen's programs and operations are described in our most recent annual report on Form 10-K and quarterly report on Form 10-Q that are filed with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.
This release discusses PGN-EDO51 and PGN-EDODM1, investigational therapies that have not been approved for use in any country and is not intended to convey conclusions about their efficacy or safety. There is no guarantee that PGN-EDODM1 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval.

Try Our AI Features

Explore what Daily8 AI can do for you:

Learn with AIFact CheckingText to SpeechAI Summary

Related Articles

Associated Press

7 hours ago

• Associated Press

ROSEN, NATIONAL INVESTOR COUNSEL, Encourages PepGen Inc. Investors to Secure Counsel Before Important Deadline in Securities Class Action

NEW YORK, June 21, 2025 (GLOBE NEWSWIRE) -- WHY: Rosen Law Firm, a global investor rights law firm, reminds purchasers of securities of PepGen Inc. (NASDAQ: PEPG) between March 7, 2024 and March 3, 2025, both dates inclusive (the 'Class Period'), of the important August 8, 2025 lead plaintiff deadline. SO WHAT: If you purchased PepGen securities during the Class Period you may be entitled to compensation without payment of any out of pocket fees or costs through a contingency fee arrangement. WHAT TO DO NEXT: To join the PepGen class action, go to or call Phillip Kim, Esq. at 866-767-3653 or email [email protected] for more information. A class action lawsuit has already been filed. If you wish to serve as lead plaintiff, you must move the Court no later than August 8, 2025. A lead plaintiff is a representative party acting on behalf of other class members in directing the litigation. WHY ROSEN LAW: We encourage investors to select qualified counsel with a track record of success in leadership roles. Often, firms issuing notices do not have comparable experience, resources, or any meaningful peer recognition. Many of these firms do not actually litigate securities class actions, but are merely middlemen that refer clients or partner with law firms that actually litigate the cases. Be wise in selecting counsel. The Rosen Law Firm represents investors throughout the globe, concentrating its practice in securities class actions and shareholder derivative litigation. Rosen Law Firm achieved the largest ever securities class action settlement against a Chinese Company at the time. Rosen Law Firm was Ranked No. 1 by ISS Securities Class Action Services for number of securities class action settlements in 2017. The firm has been ranked in the top 4 each year since 2013 and has recovered hundreds of millions of dollars for investors. In 2019 alone the firm secured over $438 million for investors. In 2020, founding partner Laurence Rosen was named by law360 as a Titan of Plaintiffs' Bar. Many of the firm's attorneys have been recognized by Lawdragon and Super Lawyers. DETAILS OF THE CASE: According to the lawsuit, throughout the Class Period, defendants made false and misleading statements and/or failed to disclose that: (1) PGN-EDO51 was less effective and safe than defendants had led investors to believe; (2) the CONNECT2 study was dangerous or otherwise deficient for purposes of U.S. Food and Drug Administration ('FDA') approval; (3) as a result of all the foregoing, PepGen was likely to halt the CONNECT2 study, and PGN-ED051's clinical, regulatory, and commercial prospects were overstated; and (4) as a result, defendants' public statements were materially false and misleading at all relevant times. When the true details entered the market, the lawsuit claims that investors suffered damages. To join the PepGen class action, go to or call Phillip Kim, Esq. at 866-767-3653 or email [email protected] for more information. No Class Has Been Certified. Until a class is certified, you are not represented by counsel unless you retain one. You may select counsel of your choice. You may also remain an absent class member and do nothing at this point. An investor's ability to share in any potential future recovery is not dependent upon serving as lead plaintiff. Follow us for updates on LinkedIn: on Twitter: or on Facebook: Attorney Advertising. Prior results do not guarantee a similar outcome. ------------------------------- Contact Information: Laurence Rosen, Esq. Phillip Kim, Esq. The Rosen Law Firm, P.A. 275 Madison Avenue, 40th Floor New York, NY 10016 Tel: (212) 686-1060 Toll Free: (866) 767-3653 Fax: (212) 202-3827 [email protected]

Business Wire

11 hours ago

• Business Wire

GE HealthCare drives innovation in theranostics with latest technological advances

CHICAGO--(BUSINESS WIRE)--At this year's Society of Nuclear Medicine and Molecular Imaging (SNMMI) Annual Meeting, GE HealthCare is spotlighting the future of precision care with its innovative portfolio of theranostics-enabling solutions designed to help drive clinical and operational excellence. Making its debut, GE HealthCare's MIM Software introduces LesionID Pro with automated zero-click pre-processing i – an AI-powered innovation to help aid physician decision making and therapy response monitoring. 'Precision care is the future of oncology—and theranostics is at the heart of that future. The integration of advanced imaging and AI-powered software is accelerating the adoption of theranostics in clinical practice,' shares Shyam Srinivas, MD, PhD, Chief Share With cancer accounting for over 10 million deaths globally each year, ii the rise of precision care – particularly theranostics – is offering new hope to patients. By combining advanced diagnostic imaging and radiopharmaceuticals with targeted therapies, theranostics enables a personalized, patient-centric approach that may help improve disease detection, treatment accuracy, and overall quality of life. 'Precision care is the future of oncology—and theranostics is at the heart of that future. The integration of advanced imaging and AI-powered software is accelerating the adoption of theranostics in clinical practice,' shares Shyam Srinivas, MD, PhD, Chief of Nuclear Medicine, Associate Clinical Professor, Department of Radiological Sciences, University of California, Irvine. 'With tools like GE HealthCare's Omni Legend, StarGuide, and MIM software at our disposal, we now have the ability to visualize disease with great clarity, quantify tumor burden efficiently, and make fast, informed decisions. These advancements are not only helping enhance diagnostic accuracy and therapy monitoring but are also opening the door to dosimetry—ultimately helping improve outcomes for our patients. This is precision care in action, and it's making a real difference in patients' lives.' Central to the practice of theranostics is molecular imaging, such as positron emission tomography (PET) and single photon emission computed tomography (SPECT), which provides detailed, patient-specific insights to guide and monitor treatment. However, accessing these insights – like whole-body tumor burden, which represents the total amount of cancer is in the body – has traditionally required time-consuming manual analysis, resulting in clinical and operational challenges. In response, GE HealthCare's MIM Software is introducing LesionID Pro with automated zero-click pre-processing, i designed with AI-powered automation to help physicians access reliable whole-body tumor burden statistics without having to spend hours manually segmenting lesions, removing normal physiologic uptake, and registering multiple patient images for comparison. In addition to turning manual pre-processing into a zero-click experience, this new version of LesionID Pro comes with significant algorithm improvements that provide physicians with a precise whole-body tumor volume to review and finalize. Intuitive, user-friendly tools were intentionally designed with input from leading theranostics practitioners with the ultimate goal of making whole-body tumor burden analysis a practical clinical reality and help shorten physicians' time-to-report. 'At GE HealthCare, we are dedicated to providing clinicians the precision care tools needed for the adoption and practice of theranostics,' shares Jean-Luc Procaccini, President & CEO, Molecular Imaging & Computed Tomography, GE HealthCare. 'We designed our portfolio of precision care solutions to evolve with healthcare system needs and help support a patient's entire care journey – from the imaging equipment needed for a noninvasive look at a patient's anatomy and treatment monitoring, to novel radiopharmaceuticals used to diagnose and monitor disease and the systems required to produce them, to the software optimized to enable data-driven decision-making. In the hands of clinicians, these tools help advance the global practice of personalized medicine and help improve patient outcomes.' Also on display at #SNMMI25, as part of GE HealthCare's comprehensive portfolio of theranostics-enabling solutions for clinical and operational excellence, are the following innovations: MINItrace Magni, iii GE HealthCare's newest cyclotron technology, designed with a small footprint (about the size of a commercial refrigerator) and the goal of providing an easy-to-site, easy-to-install solution for the reliable, in-house production of commercial PET tracers and radiometals, including Gallium-68, used in diagnostic imaging to support personalized care plans. Adoption of such easy-to-site, easy-to-install technology may help enhance the capabilities of the healthcare system but also grant clinicians the ability to offer a variety of tracers to their patients and encourage the practice of precision care locally, helping fuel inhouse Theranostics capabilities. Omni Legend is a performance-focused PET/CT designed to evolve and help meet growing healthcare system demands by enabling clinicians to reduce dose by up to 40% iv while maintaining exceptional image quality. Supportive of the diagnostic portion of theranostics, the system continues to gain in popularity, representing the company's fastest-ever-selling PET/CT. v StarGuide is a digital SPECT/CT with a 12 CZT detector design that delivers high-quality 3D images and short scan times. Optimized for certain theranostic procedures, the system is designed to help clinicians pinpoint the size, shape, and position of lesions and monitor therapy with exceptional precision. Its flexibility in patient scanning and workflow efficiencies also support high patient throughput and help reduce complexity. For oncology patients, especially those in pain, short scans can help enhance comfort and overall experience. Aurora is an advanced dual-head SPECT/CT designed with excellent diagnostic capabilities vi and streamline workflows, offering clinicians excellent image quality and operational efficiency. Its CT has a 40 mm detector – twice the detector coverage compared to CTs of other hybrid systems vii – with the ability to reduce the dose up to 82%, viii support accurate quantitation, and help clinicians make the personalized care decisions that are at the heart of theranostics. Theranostics Pathway Manager Tile is an easy-to-use application, available on GE HealthCare's Command Center software, that is designed to simplify the time-consuming task of coordinating the theranostics care pathway. It does so by tracking patient readiness for therapy, eliminating the need for manual data gathering across disparate systems (e.g., labs, scheduling, ordering, spreadsheets), and providing a unified, up-to-date view of each patient's treatment journey. Oregon Health & Science University will be an early adopter. 'Every day counts when it comes to cancer care. The latest theranostics solutions will help our care teams more quickly and easily keep tabs on patient readiness and reduce patient coordination time—freeing up more time for clinicians to focus on direct patient care,' says Erik Mittra, M.D., Ph.D., professor of diagnostic radiology in the at Oregon Health & Science University. Altogether, GE HealthCare has the unique ability to provide solutions along every step of the theranostics care pathway. Our integrated portfolio of solutions provides clinicians with the isotopes, imaging, informatics, and molecular imaging agents necessary for the practice and advancement of precision care. For more information on GE HealthCare's innovative portfolio of theranostics-enabling solutions, please visit SNMMI show attendees are also encouraged stop by the company's booth (#638 and #1023) at New Orleans Ernest N. Morial Convention Center in New Orleans, Louisiana from June 21-24. About GE HealthCare Technologies Inc. GE HealthCare is a trusted partner and leading global healthcare solutions provider, innovating medical technology, pharmaceutical diagnostics, and integrated, cloud-first AI-enabled solutions, services and data analytics. We aim to make hospitals and health systems more efficient, clinicians more effective, therapies more precise, and patients healthier and happier. Serving patients and providers for more than 125 years, GE HealthCare is advancing personalized, connected and compassionate care, while simplifying the patient's journey across care pathways. Together, our Imaging, Advanced Visualization Solutions, Patient Care Solutions and Pharmaceutical Diagnostics businesses help improve patient care from screening and diagnosis to therapy and monitoring. We are a $19.7 billion business with approximately 53,000 colleagues working to create a world where healthcare has no limits. GE HealthCare is proud to be among 2025 Fortune World's Most Admired Companies™. Follow us on LinkedIn, X, Facebook, Instagram, and Insights for the latest news, or visit our website for more information. i LesionID Pro with automated zero-click pre-processing is 510(k)-pending with the U.S. FDA. Not CE Marked and not licensed in accordance with Canadian law. Not available for sale in the United States, Europe, Canada, or any other region. ii Cancer. World Health Organization. Published February 3, 2022. Accessed March 2, 2023. iii Technology in development that represents ongoing research and development efforts. These technologies are not products and may never become products. Not CE marked. iv Omni Legend 21cm as compared to Discovery MI Gen1 20cm. As demonstrated in phantom testing. v Based on orders data of GE HealthCare PET/CT systems since 2010. vi Compared to NM/CT 870 DR. vii As compared to NM/CT 870 DR with Optima 540 CT. viii a ASiR-V reduces dose by 50% to 82% relative to FBP at the same image quality (Image quality as defined by low contrast detectability). viii b In clinical practice, the use of ASiR‐V may reduce CT patient dose depending on the clinical task, patient size, anatomical location, and clinical practice. A consultation with a radiologist and a physicist should be made to determine the appropriate dose to obtain diagnostic image quality for the particular clinical task. Low Contrast Detectability (LCD), Image Noise, Spatial Resolution and Artifact were assessed using reference factory protocols comparing ASiR‐V and FBP. The LCD was measured using 0.625 mm slices and tested for both head and body modes using the MITA CT IQ Phantom (CCT183, The Phantom Laboratory), using a model observer method.

Business Insider

17 hours ago

• Business Insider

TruGolf Announces Reverse Stock Split

Salt Lake City, Utah, June 18, 2025 (GLOBE NEWSWIRE) -- TruGolf Holdings, Inc. (NASDAQ: TRUG), a leading provider of golf simulator software and hardware, today announced that it filed an amendment to its amended and restated certificate of incorporation with the Secretary of State of the State of Delaware to effect a 1-for-50 reverse stock split of its Class A common stock. The reverse stock split will take effect at 12:01 am (Eastern Time) on June 23, 2025, and the Company's Class A common stock will open for trading on The Nasdaq Capital Market on June 23, 2025 on a post-split basis, under the existing ticker symbol 'TRUG' but with a new CUSIP number 243733409. As a result of the reverse stock split, every fifty shares of the Company's Class A common stock issued and outstanding prior to the opening of trading on June 23, 2025 will be consolidated into one issued and outstanding share. Proportionate adjustments will be made to the exercise prices and the number of shares underlying the Company's outstanding equity awards, as applicable, as well as to the number of shares issuable under the Company's equity incentive plans. The Class A common stock issued pursuant to the reverse stock split will remain fully paid and non-assessable. The reverse stock split will not affect the number of authorized shares of Class A common stock or the par value of the Class A common stock. No fractional shares will be issued if, as a result of the reverse stock split, a stockholder would become entitled to a fractional share because the number of shares of Class A common stock they hold before the reverse stock split is not evenly divisible by the split ratio. Instead, the stockholder will be entitled to receive a cash payment in lieu of a fractional share. As a result of the reverse stock split, the number of shares of Class A common stock outstanding will be reduced from approximately 40.5 million shares to approximately 0.8 million shares, and the number of authorized shares of Class A common stock will remain at 650 million shares. About TruGolf, Inc. Since 1983, TruGolf has been passionate about driving the golf industry with innovative indoor golf solutions. TruGolf builds products that capture the spirit of golf. TruGolf's mission is to help grow the game by attempting to make it more Available, Approachable, and Affordable through technology - because TruGolf believes Golf is for Everyone. TruGolf's team has built award-winning video games ("Links"), innovative hardware solutions, and an all-new e-sports platform to connect golfers around the world with E6 CONNECT. Since TruGolf's beginning, TruGolf has continued to attempt to define and redefine what is possible with golf technology. Forward-Looking Statements This news release contains certain statements that constitute 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements that are not of historical fact constitute 'forward-looking statements' and accordingly, involve estimates, assumptions, forecasts, judgements and uncertainties. Forward-looking statements include, without limitation, the timing of the reverse stock split. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. The Company has attempted to identify forward-looking statements by terminology including ''believes,'' ''estimates,'' ''anticipates,'' ''expects,'' ''plans,'' ''projects,'' ''intends,'' ''potential,'' ''may,'' ''could,'' ''might,'' ''will,'' ''should,'' ''approximately'' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors. Any forward-looking statements contained in this release speak only as of its date. The Company undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in the Company's Annual Report on Form 10-K and subsequently filed Quarterly Reports on Form 10-Q and Current Reports on Form 8-K filed with the SEC, which are available on the SEC's website,