Shares of PepGen, Inc. (PEPG) have gained 22.6% over the past four weeks to close the last trading session at $5.7, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $10 indicates a potential upside of 75.4%.The mean estimate comprises six short-term price targets with a standard deviation of $5.83. While the lowest estimate of $3.00 indicates a 47.4% decline from the current pric ...

U.S. stock futures were mixed this morning, with the Dow futures falling around 0.1% on Monday.Shares of Meihua International Medical Technologies Co., Ltd. (NASDAQ:MHUA) fell sharply in pre-market trading after the company announced that it will be delisted from the Nasdaq.Meihua International Medical Technologies shares dipped 32.9% to $9.41 in pre-market trading.Here are some other stocks moving lower in pre-market trading.SMX (Security Matters) Public Limited Company (NASDAQ:SMX) shares dipped 17.2% to ...

U.S. stock futures were mixed this morning, with the Dow futures falling around 0.1% on Monday.Shares of Meihua International Medical Technologies Co., Ltd. (NASDAQ:MHUA) fell sharply in pre-market trading after the company announced that it will be delisted from the Nasdaq.Meihua International Medical Technologies shares dipped 32.9% to $9.41 in pre-market trading.Here are some other stocks moving lower in pre-market trading.SMX (Security Matters) Public Limited Company (NASDAQ:SMX) shares dipped 17.2% to ...

Core Insights - Passage Bio and PepGen are small-cap medical companies being compared based on various financial and operational metrics to determine which stock is more favorable [1][3][4]. Institutional Ownership - Passage Bio has 53.5% of its shares owned by institutional investors, while PepGen has 58.0% [1]. - Insider ownership stands at 5.0% for Passage Bio and 5.2% for PepGen, indicating a similar level of insider investment [1]. Analyst Ratings - Passage Bio has a consensus target price of $42.67, suggesting a potential upside of 363.26% [3][4]. - PepGen's consensus target price is $10.00, indicating a potential upside of 83.82% [3][4]. - Passage Bio has a stronger consensus rating with a score of 2.50 compared to PepGen's score of 2.33 [3]. Earnings and Valuation - Passage Bio reported a net income of -$64.77 million with an earnings per share (EPS) of -$14.40 [5]. - PepGen's net income is -$89.98 million with an EPS of -$2.82 [5]. - Passage Bio has a higher price-to-earnings ratio compared to PepGen, which is currently trading at a lower valuation [5]. Profitability - Passage Bio has a net margin of -102.09% and a return on equity of -52.97% [6]. - PepGen's net margin is -84.15% with a return on equity of -65.59% [6]. Risk & Volatility - Passage Bio has a beta of 1.86, indicating it is 86% more volatile than the S&P 500 [7]. - PepGen has a beta of 1.91, meaning it is 91% more volatile than the S&P 500 [7]. Summary - PepGen outperforms Passage Bio in 6 out of 11 factors compared [8].

Summary of PepGen Conference Call Company Overview - **Company**: PepGen (NasdaqGS:PEPG) - **Focus**: Development of EDO platform technology for treating myotonic dystrophy type 1 (DM1) Key Points Clinical Study Updates - **FREEDOM-DM1 Clinical Study**: - Early data showed significant splicing improvements: - 5 mg/kg: 12% improvement - 10 mg/kg: 29% improvement - 15 mg/kg: over 50% improvement, a first in DM1 [2][3] - Upcoming data from multiple ascending dose (MAD) study expected in Q1 next year, with a focus on 5 mg/kg and 10 mg/kg doses [3][22] Efficacy and Mechanism - **EDO Technology**: - Engineered to enhance nuclear delivery of therapeutic agents, achieving nearly 100-fold higher delivery compared to traditional methods [5][6] - Targets pathogenic RNA specifically, reducing off-target effects [6] - **Expectations for Splicing Improvement**: - Anticipated that multiple doses will yield greater splicing improvements than single doses, with functional benefits expected to translate from splicing improvements [7][10] Patient Selection and Functional Measures - **Patient Selection for MAD**: - Adjustments made to baseline myotonia requirements to minimize variability in results [9] - **Functional Measures**: - Focus on hand strength and ankle dorsiflexion as key indicators of disease progression [10] - VHOT (Voluntary Hand Opening Test) is a primary endpoint, but the company is exploring other functional measures for potential registration [11][12] Safety Profile - **Safety Data**: - No treatment-emergent adverse events related to kidney observed at 5 and 10 mg/kg doses in DM1 patients [17] - Previous DMD program showed mild to moderate renal biomarker changes, but these were transient and reversible [15][21] - **Dosing Strategy**: - 10 mg/kg is expected to be safe and well-tolerated, with potential for further exploration of higher doses [18] Regulatory Considerations - **FDA Perspective**: - Emphasis on benefit-risk assessment, particularly regarding transient and reversible side effects [20] - The company aims to establish reliable and predictable efficacy endpoints for potential registration [11] Future Plans - **Geographic Expansion**: - Plans to expand clinical studies into multiple geographies to facilitate timely data collection [27] - **Upcoming Data Releases**: - Full data from the 5 mg/kg cohort expected in Q1 next year [25][26] Additional Insights - **Natural History of Disease**: - Limited understanding of how splicing levels correlate with functional outcomes, but improvements in splicing are anticipated to enhance functional benefits [10][12] - **Patient-Centric Focus**: - The company aims to address broader patient needs beyond VHOT, including improvements in mobility, digestion, and overall quality of life [13] This summary encapsulates the key discussions and insights from the PepGen conference call, highlighting the company's advancements in clinical studies, safety profiles, and future directions in the treatment of DM1.

Clinical Trials - The 15 mg/kg cohort of the FREEDOM study demonstrated a mean splicing correction of 53.7%, the highest reported to date in DM1 patients [3]. - All patients in the 5 mg/kg cohort of the FREEDOM2 trial have been enrolled and received at least one dose, with results expected in Q1 2026 [4]. - The company expects to initiate dosing in the 10 mg/kg cohort of the FREEDOM2 trial in Q1 2026, with results anticipated in the second half of 2026 [4]. - PGN-EDODM1 has received Orphan Drug and Fast Track Designations from the U.S. FDA for the treatment of DM1 [9]. Financial Performance - The company raised $115 million in a public offering, extending its cash runway into the second half of 2027 [8]. - Cash, cash equivalents, and marketable securities totaled $163.7 million as of September 30, 2025, compared to $120.2 million at the end of 2024 [20]. - Research and Development Expenses were $13.4 million for the three months ended September 30, 2025, down from $17.7 million in the same period in 2024, a decrease of approximately 24% [8]. - General and Administrative Expenses were $5.2 million for the three months ended September 30, 2025, slightly down from $5.4 million in the same period in 2024 [8]. - The net loss for the three months ended September 30, 2025, was $18.0 million, or $(0.52) per share, compared to a net loss of $21.4 million, or $(0.66) per share, for the same period in 2024 [8]. - Total operating expenses for the three months ended September 30, 2025, were $18.7 million, down from $23.2 million in the same period in 2024, a reduction of approximately 19% [18].

Financial Performance - PepGen reported net losses of $71.3 million and $67.7 million for the nine months ended September 30, 2025, and September 30, 2024, respectively [100]. - For the nine months ended September 30, 2025, the net cash used in operating activities was $65.8 million, compared to $62.9 million for the same period in 2024, reflecting a net loss of $71.3 million in 2025 versus $67.7 million in 2024 [138][139]. - Total operating expenses increased slightly by $0.5 million to $73.9 million compared to $73.4 million in 2024 [120]. - For the three months ended September 30, 2025, total operating expenses decreased by $4.5 million to $18.7 million from $23.2 million in the same period of 2024, primarily due to a reduction in research and development expenses [115]. Cash and Liquidity - As of September 30, 2025, PepGen had cash, cash equivalents, and marketable securities totaling $163.7 million, with an accumulated deficit of $342.8 million [100]. - The company expects its existing cash and marketable securities to fund operations into the second half of 2027, with no expected revenue from product sales until regulatory approval is obtained [101]. - As of September 30, 2025, the company had $163.7 million in cash, cash equivalents, and marketable securities, indicating a strong liquidity position [145]. - The company raised gross proceeds of $107.6 million from the sale of 31.25 million shares at $3.20 per share in the 2025 Offering [131]. Research and Development - Research and development expenses for the three months ended September 30, 2025, totaled $13.4 million, compared to $17.7 million for the same period in 2024 [106]. - The ongoing FREEDOM study for PGN-EDODM1 reported a mean splicing correction of 53.7% following a single 15 mg/kg dose, with all drug-related adverse events being mild or moderate [91]. - PepGen has decided to discontinue development of PGN-EDO51 and wind down all DMD-related research and development activities [94]. - Research and development expenses for the nine months ended September 30, 2025, were $57.2 million, a decrease of $0.3 million from $57.5 million in 2024, mainly due to a $3.9 million decrease in preclinical costs [122]. Operating Expenses - General and administrative expenses decreased by $0.2 million to $5.2 million for the three months ended September 30, 2025, primarily due to a reduction in personnel-related costs [117]. - General and administrative expenses increased by $0.8 million to $16.7 million for the nine months ended September 30, 2025, driven by increased consulting and legal expenses [123]. - Research and development expenses for the three months ended September 30, 2025, were $13.4 million, down from $17.7 million in 2024, reflecting a $1.4 million decrease in personnel-related costs and a $1.2 million decrease in manufacturing costs [116]. Financing Activities - Net cash provided by financing activities increased to $108.2 million for the nine months ended September 30, 2025, compared to $88.6 million in 2024, driven by proceeds from the 2025 Offering [142][143]. - The net cash provided by financing activities in 2025 included $108.1 million from the 2025 Offering, highlighting successful capital raising efforts [142]. Regulatory and Designation Updates - The FDA has granted orphan drug designation and Fast Track designation for PGN-EDODM1 for the treatment of DM1 [93]. - The company has filed a shelf registration statement covering the offering of up to $300 million in various securities, effective June 16, 2023 [127]. Other Financial Metrics - Interest income for the three months ended September 30, 2025, was $0.6 million, down from $1.8 million in the same period of 2024 [118]. - Non-cash adjustments in operating activities included $8.2 million of stock-based compensation and $1.1 million of depreciation expense for the nine months ended September 30, 2025 [138]. - The company experienced a decrease of $3.5 million in accrued expenses and a decrease of $2.3 million in operating lease liabilities during the nine months ended September 30, 2025 [138]. - The company reported a decrease in accrued expenses primarily due to lower research and development accruals, amounting to $5.8 million for the nine months ended September 30, 2024 [139]. - The company’s investments in marketable securities resulted in $58.0 million in maturities during the nine months ended September 30, 2025 [140]. - There were no material changes to the company's critical accounting policies and estimates from those described in the previous financial statements [144].

Core Insights - PepGen Inc. has received a U.S. patent for its proprietary PGN-EDODM1 molecule, which is a significant addition to its patent portfolio and is expected to provide exclusivity until the second half of 2042 [1][2]. Group 1: Patent and Exclusivity - The newly issued composition of matter patent covers PGN-EDODM1, which utilizes PepGen's Enhanced Delivery Oligonucleotide (EDO) platform [1][2]. - The patent is expected to offer exclusivity in the U.S. until the second half of 2042, with potential for extension following FDA approval [1][2]. Group 2: Product Development - PGN-EDODM1 is designed to treat myotonic dystrophy type 1 (DM1), a severe neuromuscular disease with no approved therapies targeting its underlying cause [2][3]. - The investigational therapy aims to restore normal splicing function of MBNL1, addressing the negative effects of CUG repeat expansion in DMPK transcripts [3]. Group 3: Company Overview - PepGen Inc. is a clinical-stage biotechnology company focused on developing next-generation oligonucleotide therapies for severe neuromuscular and neurological diseases [4]. - The EDO platform is based on over a decade of research, utilizing cell-penetrating peptides to enhance the uptake and efficacy of oligonucleotide therapeutics [4].

Summary of PepGen Conference Call Company Overview - **Company**: PepGen - **Event**: Guggenheim's 2025 Healthcare Innovations Conference - **Key Presenters**: James McArthur (President and CEO), Paul Strak (EVP and Head of R&D) [1][2] Industry Focus - **Industry**: Neuromuscular diseases - **Technology**: EDO platform technology for delivering therapeutic oligonucleotides [3] Core Points and Arguments 1. **Clinical Study Results**: - Reported a 54% splicing improvement at the top dose of 15 mg/kg in the Freedom clinical study, significantly higher than the previous 22% improvement achieved with a different approach [3][4] - The study showed that the treatment was generally safe, with no serious adverse events reported [4] 2. **Future Expectations**: - Anticipates reporting results from a multiple ascending dose study in Q1 2024, aiming to build upon the 12% splicing improvement seen with a single dose [4][9] - Plans to report data from a second cohort at 10 mg/kg in the second half of 2024, expecting to improve upon the 29% splicing improvement previously reported [4] 3. **Market Potential**: - The DM1 patient population is significantly larger than that of SMA, with no approved therapies currently available for DM1 [5][18] 4. **Therapeutic Index**: - Observed a better-than-dose-dependent increase in muscle concentration and splicing, with mild transient changes in renal function noted [4][5] 5. **Preclinical Data**: - In a mouse model, a three-and-a-half-fold increase in oligo concentration in muscle was observed with multiple doses, leading to nearly complete splicing improvement [6] 6. **Safety Monitoring**: - Safety is monitored in real-time during trials, with a focus on ensuring no adverse effects from the treatment [7] 7. **Regulatory Pathways**: - The company is considering both accelerated approval pathways and a single large phase three study, depending on the outcomes of the ongoing studies [16] 8. **Differentiation from Competitors**: - Emphasizes the unique targeting approach of the EDO platform, which focuses on pathogenic RNA, potentially leading to better outcomes compared to existing therapies [13][18] 9. **Secondary Endpoints**: - Plans to monitor additional endpoints such as 10-meter walk/run and hand grip strength, with optimism for improvements in larger muscle groups [14] 10. **Future Indications**: - While currently focused on DM1, the company is exploring other neuromuscular indications like Charcot-Marie-Tooth for future development [20] Additional Important Points - **Delivery Method**: Currently focused on IV formulation, with no immediate plans to shift to subcutaneous delivery [19] - **Patient Enrollment**: Plans to enroll patients both in the U.S. and internationally for ongoing studies [22] - **Timeline for Data**: Expected data for the 5 mg/kg dose in Q1 2024 and for the 10 mg/kg dose in the second half of 2024 [21][22]

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