MBNL1

- Enrollment completed for PGN-EDO51 5 mg/kg cohort in CONNECT1-EDO51 Phase 2 clinical trial in DMD patients. The Company expects to announce preliminary data from the 5 mg/kg dose cohort in mid-2024 – - CONNECT2-EDO51 Phase 2 clinical trial in DMD patients open in the U.K. - Company believes this study could potentially support accelerated approval - - FREEDOM-DM1 Phase 1 clinical trial enrolling DM1 patients with preliminary data for at least the 5 mg/kg dose cohort expected in the second half of 2024 - - Gross proceeds of approximately $80 million from February 2024 follow-on offering extends projected operating cash runway into 2026 - BOSTON, March 06, 2024 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), 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, today reported financial results for the fourth quarter and full year ended December 31, 2023, and highlighted recent corporate developments. “With important data readouts expected from our two lead programs, 2024 has the potential to be a transformational year for PepGen. In the last year, PepGen made meaningful advancements with our Enhanced Delivery Oligonucleotide (EDO) therapeutic candidates, including the initiation and dosing of patients in both our CONNECT1-EDO51 Phase 2 and FREEDOM-DM1 Phase 1 clinical trials,” said James McArthur, Ph.D., President and CEO of PepGen. “Following the recent completion of enrollment of cohort 1 of our CONNECT1-EDO51 trial, we look forward to sharing preliminary safety, exon 51 skipping, and dystrophin production data from the 5 mg/kg dose in mid-2024.” Dr. McArthur continued, “We continue to see encouraging enrollment in our ongoing FREEDOM-DM1 Phase 1 clinical trial. People with myotonic dystrophy type 1 (DM1) currently have no approved treatment options that target the root cause of the disease. We believe that PGN-EDODM1 has the potential to be disease-modifying and could generate meaningful, double-digit levels of splicing correction and corresponding correction of functional measures. We look forward to reporting preliminary data from at least the 5 mg/kg dose cohort later this year.” Recent Program Highlights PGN-EDO51: Duchenne Muscular Dystrophy (DMD) PGN-EDO51, PepGen’s lead investigational candidate in development for the treatment of DMD, utilizes the Company’s proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to target the root cause of this devastating disease. PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of people with DMD. Phase 2 CONNECT1-EDO51 Clinical Trial of PGN-EDO51 underway and 5 mg/kg cohort fully enrolled: In January 2024, PepGen announced that the first patient had been dosed in its Phase 2, open-label multiple ascending dose (MAD) CONNECT1-EDO51 clinical trial, being conducted in Canada, evaluating PGN-EDO51 in 10 male patients at least 8 years of age with DMD amenable to an exon 51-skipping approach. The starting dose will escalate from 5 mg/kg to 10 mg/kg with a potential for further dose escalations. All dose escalations will be determined based on evaluation of safety data from the prior dose cohort(s). PepGen is announcing that the 5 mg/kg PGN-EDO51 dose cohort is fully enrolled. PepGen expects to report preliminary data from this cohort in mid-2024, including initial safety, exon 51 skipping, and dystrophin production data. Initiation of CONNECT2-EDO51 Clinical Trial of PGN-EDO51: In March 2024, PepGen announced that it had received authorization from the Medicines and Healthcare products Regulatory Agency (MHRA) to initiate its Phase 2, randomized, double-blind, placebo-controlled MAD CONNECT2-EDO51 clinical trial in the United Kingdom evaluating PGN-EDO51 in approximately 20 male patients at least 6 years of age with DMD amenable to an exon 51-skipping approach. PepGen plans to extend this study to the United States and other countries, subject to regulatory authorizations. The CONNECT 2-EDO51 clinical trial, together with the data from CONNECT1-EDO51, is designed to potentially support a future accelerated approval pathway, subject to alignment with regulatory authorities. PGN-EDODM1: Myotonic Dystrophy 1 (DM1) PGN-EDODM1, PepGen’s second investigational candidate in development for the treatment of DM1, utilizes the Company’s proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1. DM1 is a progressively disabling, life-shortening genetic disorder. DM1 is estimated to affect 40,000 people in the U.S., and over 74,000 people in Europe. Fast Track Designation granted to PGN-EDODM1: In February 2024, PepGen announced that the U.S. Food and Drug Administration granted Fast Track Designation to PGN-EDODM1 for the treatment of DM1. First Patient Dosed in Phase 1 FREEDOM-DM1 Clinical Trial of PGN-EDODM1: In December 2023, PepGen announced that the first patient had been dosed in its Phase 1 single ascending dose (SAD) FREEDOM-DM1 clinical trial evaluating PGN-EDODM1 in 24 adult patients with DM1 in the U.S., Canada, and the United Kingdom. The starting dose of 5 mg/kg can escalate to 10 mg/kg, and then 20 mg/kg based on evaluation of safety data from the prior dose cohort(s). PepGen anticipates reporting preliminary data from at least the 5 mg/kg PGN-EDODM1 dose cohort, including safety, splicing correction, and functional outcome measures, in the second half of 2024. In addition to the ongoing FREEDOM-DM1 trial, PepGen expects to open its FREEDOM2-DM1 placebo controlled, MAD clinical trial in DM1 patients in the second half of 2024. Preclinical PGN-EDO53: DMD PGN-EDO53 is PepGen’s third investigational candidate for the treatment of DMD that utilizes the Company’s proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to target the root cause of this devastating disease. PGN-EDO53 is designed to skip exon 53 of the dystrophin transcript, an established therapeutic target for approximately 8% of people with DMD. PepGen plans to advance PGN-EDO53 into investigational new drug (IND) and clinical trial application (CTA) enabling nonclinical studies. AdditionalCorporate Highlights $80 Million Underwritten Offering of Common Stock: In February 2024, PepGen priced an underwritten offering of 7,530,000 shares of common stock at a price to the public of $10.635 per share, for aggregate gross proceeds to PepGen of approximately $80 million, before deducting commissions and expenses payable by PepGen. Financial Results for the Three Months and Twelve Months ended December 31, 2023 Cash and cash equivalents were $110.4 million as of December 31, 2023. Based on the Company's current operating plan and projections, it believes that current cash, cash equivalents and marketable securities, together with the net proceeds from its recent common stock offering, will be sufficient to fund projected operating requirements into 2026. Research and development expenses were $16.3 million for the three months ended December 31, 2023, compared to $13.2 million for the same period in 2022. Research and development expenses were $68.1 million for the year ended December 31, 2023, compared to $54.1 million for the same period in 2022. The increase in research and development expenses was primarily due to costs associated with the advancement of the Company’s PGN-EDO51 and PGN-EDODM1 programs, including preclinical, clinical, and manufacturing costs for our ongoing and future clinical trials. General and administrative expenses were $4.5 million for the three months ended December 31, 2023, compared to $4.0 million for the same period in 2022. General and administrative expenses were $16.6 million for the year ended December 31, 2023, compared to $14.2 million for the same period in 2022. The increase in general and administrative expenses was primarily due to increased personnel-related costs. Net loss was $19.5 million for the three months ended December 31, 2023, compared to $14.9 million for the same period in 2022. Net loss was $78.6 million for the year ended December 31, 2023, compared to $69.1 million for the same period in 2022. PepGen had approximately 23.8 million shares outstanding on December 31, 2023. About PepGen PepGen Inc. 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 Enhanced Delivery Oligonucleotide, or 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 that are designed to target the root cause of serious diseases. 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 pro our product candidates, including PGN-EDO51, PGN-EDODM1 and PGN-EDO53, our technology, including our EDO platform, the design, initiation and conduct of clinical trials, including expected timelines, dose levels and dose escalation, including for our CONNECT2-EDO51 Phase 2 trial and our FREEDOM2-DMI trial, as well as preliminary data reports from our CONNECT1-EDO51 Phase 2 trial and FREEDOM-DM1 Phase 1 trial, the advancement of PGN-EDO53 into IND/CTA enabling studies, regulatory interactions, including development pathway for our product candidates, and our financial resources and expected cash runway. 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-EDO51, PGN-EDODM1 and PGN-EDO53; our ability to enroll patients in our clinical trials, including CONNECT1-EDO51, CONNECT2-EDO51, FREEDOM-DM1and FREEDOM2-DM1; 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; our product candidates, including PGN-EDO51 and 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, including CONNECT2-EDO51, or other regulatory feedback requiring modifications to our development programs; changes in regulatory framework that are out of our control; our ability to obtain, maintain and protect our intellectual property; our ability to enforce our patents against infringers and defend our patent portfolio against challenges from third parties; competition from others developing therapies for the indications we are pursuing; 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. PepGen Inc. Condensed Statement of Operations (in thousands, unaudited) Twelve Months Ended December 31, Three Months Ended December 31, 2023 2022 2023 2022 Operating expenses: Research and development $ 68,126 $ 54,077 $ 16,300 $ 13,166 General and administrative 16,640 14,224 4,511 4,047 Total operating expenses $ 84,766 $ 68,301 $ 20,811 $ 17,213 Operating loss $ (84,766 ) $ (68,301 ) $ (20,811 ) $ (17,213 ) Other income (expense) Interest income 6,400 2,793 1,346 1,591 Other income (expense), net (187 ) 110 43 (28 ) Total other income, net 6,213 2,903 1,389 1,563 Net loss before income tax $ (78,553 ) $ (65,398 ) $ (19,422 ) $ (15,650 ) Income tax (expense) benefit (73 ) (3,706 ) (73 ) 714 Net loss $ (78,626 ) $ (69,104 ) $ (19,495 ) $ (14,936 ) PepGen Inc. Condensed Balance Sheet Data (in thousands, unaudited) December 31, 2023 2022 Assets Cash, cash equivalents and marketable securities $ 110,407 $ 181,752 Other assets 32,645 35,688 Total assets $ 143,052 $ 217,440 Liabilities and stockholders’ equity Liabilities $ 34,631 $ 37,809 Stockholders’ equity: 108,421 179,631 Total liabilities and stockholders’ equity $ 143,052 $ 217,440 Investor Contact Laurence Watts Gilmartin Group Laurence@gilmartinir.com Media Contact Sarah Sutton Argot Partners pepgen@argotpartners.com

-ATX-01 is the industry's first microRNA therapeutic to be evaluated for DM1 and the first program from ARTHEX's pipeline to enter the clinic –-First patient expected to be enrolled in Q2 2024- VALENCIA, Spain, Feb. 28, 2024 /PRNewswire/ -- ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, announced that the U.S. Food and Drug Administration (FDA) cleared the Company to initiate the Phase I-IIa ArthemiR™ study of ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1). Continue Reading ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic to be investigated for DM1 and is the first therapeutic from ARTHEx's pipeline to enter the clinic. miR-23b is known to be associated with regulating the expression of MBNL proteins involved in the pathogenesis of DM1, a devastating, rare neuromuscular disorder that causes muscle weakness and other life-limiting complications. There are currently no disease-modifying treatments for DM1. "Receiving FDA clearance to initiate our first-in-human study, ArthemiR™, for ATX-01 in DM1 is a major milestone for ARTHEX and for DM1 patients and their families who are in need of an approved therapeutic option," said Dr. Judith Walker, Chief Medical Officer of Arthex. "ATX-01 holds significant potential to deliver therapeutic benefit to DM1 patients, based on its dual mechanism of action that targets both the toxic DMPK mRNA and the reduced active MBNL levels. We plan to launch the ArthemiR™ study first in the US, followed by Canada and Europe." The ArthemiR™ trial is a Phase I-IIa double-blind, placebo-controlled, dose escalation study expected to enroll participants with classic Myotonic Dystrophy Type 1 (DM1). The primary objective is to determine the safety and tolerability of single and multiple ascending doses of ATX-01 in DM1 participants. ARTHEx will also investigate target engagement at the muscle level through biomarkers, including MBNL levels and splicing index. In addition, the clinical endpoints from the trial will include measures related to muscle function, patient-reported outcomes, and quality of life measures. Dr. Nicholas Johnson, Professor, Vice Chair of Research in the Department of Neurology at Virginia Commonwealth University, and the lead investigator for the ArthemiR™ trial, commented, "It is exciting to see new agents coming to clinical trials, especially compounds with different mechanisms of action. This increases our hope of one day having effective and safe, disease modifying treatments for this multisystemic condition. Patients are eager for new trials, and we are delighted to start enrolment in the coming months." About ATX-01 ATX-01 is an antimiR oligonucleotide designed to target microRNA 23b (miR-23b), which is associated with regulating the expression of MBNL proteins involved in the pathogenesis of DM1. It has been demonstrated in human DM1 myoblast cell lines that ATX-01 has a unique, dual mechanism of action which reduces toxic DMPK mRNA and increases MBNL protein levels. Toxic DMPK and reduced levels of MBNL have been identified as the molecular underpinnings of DM1. ATX-01 will shortly be evaluated in the Phase I-IIa ArthemiR™ trial for the treatment of DM1. ATX-01 has received Orphan Drug Designation for ATX-01 in DM1 from the US and European authorities. ATX-01 was discovered through ARTHEx's in-house discovery engine, which is designed to identify and optimize novel microRNA modulators and ensure their preferential delivery to target tissues, for the treatment of diseases in which microRNAs are involved in the disease pathogenesis. About Myotonic Dystrophy Type 1 (DM1) Myotonic dystrophy type 1 (DM1) is a highly disabling disease affecting more than one million people worldwide. The condition affects muscles and other tissues (causing respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment). Most commonly, it manifests during adulthood (classic DM1), although DM1 can develop at birth in a congenital form, or during childhood. Although signs and symptoms vary among affected individuals, sadly, with progression of the disease, DM1 patients experience a reduction in the ability to perform activities of daily living. Moreover, patients have a significantly shortened lifespan and there is currently no approved treatment to slow the progression of the disease. About ARTHEx Biotech ARTHEx Biotech is a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs. The Company's lead investigational compound, ATX-01, is being evaluated for the treatment of myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder, in the Phase I-IIa ArthemiR™ trial. ARTHEx is also advancing its in-house discovery engine to identify and develop microRNA modulators for other disorders with high unmet medical needs, including genetically-driven diseases like DM1. The Company headquarters are in Valencia, Spain. For more information, please visit and engage with us on LinkedIn. SOURCE ARTHEx Biotech

Feb. 20, 2024 -- PepGen Inc. (Nasdaq: PEPG), 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, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PGN-EDODM1, an investigational candidate for the treatment of myotonic dystrophy type 1 (DM1). “Receiving Fast Track designation from the FDA for PGN-EDODM1 is a significant milestone in our efforts to deliver a potentially transformative therapy to the DM1 community,” said James McArthur, Ph.D., President and CEO of PepGen. “Patients with DM1 currently have no available treatment options that target the root cause of the disease, which leads to progressive neuromuscular symptoms and reduction in life expectancy. Following robust preclinical data, we are now evaluating PGN-EDODM1 in the ongoing FREEDOM-DM1 Phase 1 trial and expect to report preliminary data later this year. We believe that PGN-EDODM1 has the potential to be disease-modifying and improve outcomes for patients living with DM1.” The FDA’s Fast Track designation is a process designed to facilitate the development and expedite the review of drug candidates that treat serious conditions and fill an unmet medical need, with the goal of getting important new drugs to the patient earlier. Once a drug candidate receives Fast Track designation, early and frequent communication between the FDA and the drug company is encouraged throughout the entire drug development and regulatory review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients. For more information about Fast Track designation, please visit the FDA website (www.fda.gov). PGN-EDODM1 is currently being evaluated in the ongoing FREEDOM-DM1 Phase 1 clinical trial for the treatment of people living with DM1. The Company expects to report initial data from the trial in 2024. (ClinicalTrials.gov identifier: NCT06204809) PGN-EDODM1 is an investigational candidate designed to deliver a peptide-conjugated antisense oligonucleotide (ASO) to restore cellular function. DM1 is caused by an expansion of CUG repeats that form hairpin loops in the DMPK RNA, resulting in sequestration of the MBNL1 protein, a key RNA processing factor. The sequestration of MBNL1 results in downstream mis-splicing events and aberrant expression of many proteins that play a critical role in muscle and other systemic functions (e.g. endocrine, gastrointestinal, central nervous system). By specifically blocking the toxic DMPK transcript CUG repeats, the goal of PGN-EDODM1 is to liberate MBNL1 protein and to restore functional downstream splicing and muscle and other systemic functions. Myotonic dystrophy type 1, or DM1 (also known as Steinert’s disease), is a progressively disabling, life-shortening genetic disorder. DM1 is the most prevalent form of the disease and generally the most severe. DM1 is estimated to affect 40,000 people in the U.S., and over 74,000 people in Europe. The average life expectancy for people living with DM1 is 45-60 years old. People living with DM1 typically present with myotonia (stiff or contracted muscles), muscle weakness, and cardiac and respiratory abnormalities. Many people living with DM1 also experience excessive daytime sleepiness, fatigue, and issues with gastrointestinal or cognitive dysfunction that significantly affect their quality of life. PepGen Inc. 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 Enhanced Delivery Oligonucleotide, or 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 that are designed to target the root cause of serious diseases. Thecontent above comes from the network. if any infringement, please contact us tomodify.