Last update 01 Nov 2024
PGN-EDO51
Last update 01 Nov 2024
Overview
Basic Info
Drug Type ASO |
Synonyms Enhanced delivery peptide-oligonucleotide conjugate for Duchenne Muscular Dystrophy Exon 51 + [1] |
Target |
Mechanism DMD exon 51 modulators(Dystrophin exon 51 modulators), Dystrophin expression stimulants, RNA interference + [1] |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePhase 2 |
First Approval Date- |
RegulationRare Pediatric Disease (US), Orphan Drug (US) |
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Related
1
Clinical Trials associated with PGN-EDO51A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)
The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period.
The study consists of 3 periods: A Screening Period (up to 45 days), a Treatment and Observation Period (16 weeks), and an Extension Period (108 weeks).
The study consists of 3 periods: A Screening Period (up to 45 days), a Treatment and Observation Period (16 weeks), and an Extension Period (108 weeks).
Start Date08 Jan 2024 |
Sponsor / Collaborator |
100 Clinical Results associated with PGN-EDO51
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100 Translational Medicine associated with PGN-EDO51
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100 Patents (Medical) associated with PGN-EDO51
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42
News (Medical) associated with PGN-EDO5108 Aug 2024
BOSTON--(
BUSINESS WIRE
)--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 and recent corporate highlights for the quarter ended June 30, 2024.
“Our July readout of the CONNECT1 5 mg/kg clinical data in Duchenne muscular dystrophy (DMD) was the culmination of years of dedicated effort from our team,” said James McArthur, Ph.D., President and CEO of PepGen. “In three months, PGN-EDO51 produced higher mean levels of exon skipped transcript at lower doses and in a shorter period than other exon 51 therapies, approved or in development. We believe this indicates our Enhanced Delivery Oligonucleotide (EDO) technology is delivering greater levels of oligonucleotide to the nuclei. We are also very pleased PGN-EDO51 was well tolerated and that all patients experienced increases in dystrophin production. We look forward to our upcoming FREEDOM-DM1 clinical results update as we believe our EDO platform has the potential to dramatically improve the lives of people living with severe neuromuscular and neurological diseases.”
Recent Program Highlights
PGN-EDO51: Duchenne Muscular Dystrophy (DMD)
Phase 2 CONNECT1-EDO51 Clinical Trial of PGN-EDO51
: In July 2024, PepGen reported clinical data from the 5 mg/kg PGN-EDO51 dose cohort, including initial safety, exon 51 skipping, and dystrophin production data. CONNECT1 is a Phase 2, open-label, multiple ascending dose (MAD) clinical trial, being conducted in Canada, evaluating PGN-EDO51 in approximately 10 male patients at least 8 years of age with DMD amenable to an exon 51-skipping approach.
At the 5 mg/kg Dose
PGN-EDO51 was well tolerated by all study cohort participants through week 13. There were no discontinuations, dose interruptions or dose reductions.
PGN-EDO51 produced mean exon skipping in biceps tissue of 2.15% at week 13 compared to baseline.
PGN-EDO51 achieved a mean muscle-adjusted dystrophin level of 1.49% of normal and a 0.70% increase from baseline after 4 doses, measured at week 13.
PGN-EDO51 achieved a mean absolute dystrophin level of
0.61% of normal and a 0.26% increase from baseline after 4 doses, measured at week 13 by Western blot analysis.
The Company plans to present additional results from the 5 mg/kg cohort at a medical meeting later in the year.
The Company expects to report initial results from the 10 mg/kg cohort of CONNECT1-EDO51 in early 2025.
Phase 2 CONNECT2-EDO51 Clinical Trial of PGN-EDO51
: Based on the data from CONNECT1, including PGN-EDO51’s emerging safety profile to date, the Company is working to optimize the design of the multinational CONNECT2 Phase 2 double-blind, placebo-controlled, MAD, 25-week trial. The CONNECT2 clinical trial is open in the United Kingdom. The Company continues to engage with regulators in the European Union and expects to open the clinical trial in the United States by year-end, subject to regulatory clearance.
PGN-EDODM1: Myotonic Dystrophy 1 (DM1)
Phase 1 FREEDOM-DM1 Clinical Trial of PGN-EDODM1
: PepGen anticipates reporting clinical results from the FREEDOM clinical trial, including safety, splicing correction, and functional outcome measures, in the fourth quarter of 2024. FREEDOM is a Phase 1 single ascending dose clinical trial evaluating PGN-EDODM1 in approximately 24 adult patients with DM1 in the United States, Canada, and the United Kingdom.
Phase 2 FREEDOM2-DM1 Clinical Trial of PGN-EDODM1
: PepGen is announcing today that both Health Canada and the United Kingdom Medicines and Healthcare products Regulatory Agency have cleared the Company’s clinical trial application (CTA) submissions for the FREEDOM2 trial, and PepGen expects to initiate patient dosing in the second half of 2024. FREEDOM2 is a Phase 2 randomized, double-blind, placebo-controlled, MAD clinical trial evaluating PGN-EDODM1 in approximately 24 adult patients with DM1 in Canada, the United Kingdom, and in the United States, subject to regulatory clearance.
PGN-EDODM1 Poster:
In April 2024, PepGen presented a poster on the PGN-EDODM1 program at the 14
th
International Myotonic Dystrophy Consortium (2024 IDMC-14) Meeting.
Poster title: FREEDOM-DM1: Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1.
The PGN-EDODM1 poster presented at the 2024 IDMC-14 Meeting is available on the Investors page of PepGen’s website under Scientific Publications.
PGN-EDO53 (DMD)
PepGen continues to advance PGN-EDO53
in investigational new drug (IND) and CTA enabling nonclinical studies.
Financial Results for the Three Months Ended June 30, 2024
Cash, cash equivalents and marketable securities
were $161.3 million as of June 30, 2024, which is anticipated to fund currently planned operations into 2026.
Research and Development expenses
were $25.1 million for the three months ended June 30, 2024, compared to $16.9 million for the same period in 2023.
General and Administrative expenses
were $5.4 million for the three months ended June 30, 2024, compared to $4.2 million for the same period in 2023.
Net loss
was $28.3 million, or $(0.87) basic and diluted net loss per share, for the three months ended June 30, 2024, compared to $19.5 million, or $(0.82) basic and diluted net loss per share, for the same period in 2023. PepGen had approximately 32.6 million shares outstanding on June 30, 2024.
About PGN-EDO51
PGN-EDO51, PepGen's lead clinical candidate for the treatment of Duchenne muscular dystrophy (DMD), utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic phosphorodiamidate morpholino oligomer (PMO) 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 DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein. The U.S. Food and Drug Administration has granted PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations for the treatment of patients with DMD amenable to an exon-51 skipping approach.
About
PGN-EDODM1
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, a key RNA splicing protein. 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 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 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-EDODM1 and, based on early data, PGN-EDO51, the potential of our EDO platform to deliver higher levels of oligonucleotide to the nuclei and to dramatically improve the lives of people living with severe neuromuscular and neurological diseases, the design, initiation and conduct of clinical trials, including expected timelines for our CONNECT2 Phase 2 trial and FREEDOM2 Phase 2 trial, the expected timing for additional results from our CONNECT1 Phase 2 trial and results from our FREEDOM Phase 1 trial, ongoing and planned regulatory interactions, the advancement of PGN-EDO53 in IND/CTA enabling studies, and our financial resources and 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, CONNECT2, 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-EDO51 and PGN-EDODM1; 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, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our CONNECT1, CONNECT2, 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, an investigational therapy that has not been approved for use in any country, and is not intended to convey conclusions about its efficacy or safety. There is no guarantee that PGN-EDO51 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval.
Consolidated Statements of Operations
(unaudited, in thousands)
Three Months Ended
June 30,
2024
2023
Operating expenses:
Research and development
$
25,063
$
16,926
General and administrative
5,362
4,218
Total operating expenses
$
30,425
$
21,144
Operating loss
$
(30,425
)
$
(21,114
)
Other income (expense)
Interest income
2,121
1,684
Other income, net
(31
)
(62
)
Total other income (expense), net
2,090
1,622
Net loss before income tax
$
(28,335
)
$
(19,522
)
Income tax expense
—
—
Net loss
$
(28,335
)
$
(19,522
)
Net loss per share, basic and diluted
$
(0.87
)
$
(0.82
)
Weighted-average common shares outstanding, basic and diluted
32,469,187
23,790,430
Condensed Consolidated Balance Sheets
(unaudited, in thousands)
June 30,
December 31,
2024
2023
Assets
Cash, cash equivalents and marketable securities
$
161,306
$
110,407
Other assets
32,204
32,645
Total assets
$
193,510
$
143,052
Liabilities and stockholders’ equity
Liabilities
$
38,470
$
34,631
Stockholders’ equity:
155,040
108,421
Total liabilities and stockholders’ equity
$
193,510
$
143,052
Clinical ResultPhase 2Phase 1Orphan DrugFast Track
31 Jul 2024
– PGN-EDO51 at 5 mg/kg was well tolerated, and all patients continued to long-term extension portion of trial. Dosing of second cohort at 10 mg/kg is ongoing –
– Four doses of PGN-EDO51 at 5 mg/kg achieved mean exon skipping levels of 2.15% after three months of dosing –
– PGN-EDO51 at 5 mg/kg showed mean muscle-adjusted dystrophin level of 1.49%, a 0.70% increase from baseline, after three months of dosing –
– PGN-EDO51 at 5 mg/kg showed mean absolute dystrophin level of 0.61%, a 0.26% increase from baseline, after three months of dosing –
BOSTON, MA, USA I July 30, 2024 I
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 positive clinical data from the first dose cohort (5 mg/kg) of PGN-EDO51, its lead investigational candidate for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach. In the ongoing CONNECT1-EDO51 Phase 2 open-label trial, PGN-EDO51 demonstrated higher levels of exon skipping than previously reported studies with other oligonucleotide therapies at similar PMO dose levels in DMD patients. The Company also reported that change from baseline in total dystrophin production and muscle-adjusted dystrophin production was comparable to, or higher than, previously reported studies with other oligonucleotide therapies at similar PMO dose levels in DMD patients. Today at 4:30 p.m. ET, the Company will host a conference call with the CONNECT1 lead investigator Dr. Hugh McMillan to discuss the data being presented.
“We are encouraged by the early data from our CONNECT1 clinical trial of PGN-EDO51 in people with DMD. In three months, the starting monthly dose of 5 mg/kg achieved high levels of exon skipping and all patients showed increases in dystrophin. PGN-EDO51 produced meaningfully higher levels of exon skipped transcript at lower doses and in a shorter time period compared to other exon 51 therapies, approved and in development, indicating that our Enhanced Delivery Oligonucleotide technology is delivering higher levels of oligonucleotide to the nuclei,” said James McArthur, Ph.D., President and CEO of PepGen. “Importantly, PGN-EDO51 has demonstrated a favorable safety profile, supporting our ongoing evaluation of the 10 mg/kg monthly dose cohort in CONNECT1. We intend to leverage the early observations from CONNECT1 to optimize our CONNECT2-EDO51 Phase 2 trial. Based on these initial results, we are optimistic about the possibility that higher levels of dystrophin production will be observed in the 10 mg/kg cohort of CONNECT1. We also look forward to reporting data from the first cohort of our placebo-controlled multinational study CONNECT2.”
“People with DMD and their families constantly hope for effective therapies with the potential to change the course of this relentlessly progressive neuromuscular disease. I was pleased to see that the 5 mg/kg dose was well tolerated and that all three participants demonstrated an increase in dystrophin production and exon skipping after only three months of treatment with PGN-EDO51. I look forward to seeing the results of exon skipping and dystrophin production at 10 mg/kg in both CONNECT1 and CONNECT2,” said Dr. Hugh McMillan, Pediatric Neurologist at the Children’s Hospital of Eastern Ontario, and Professor in the Department of Pediatrics at the University of Ottawa.
CONNECT1 Results for the 5 mg/kg Starting Dose Cohort (n=3)
Exon 51 Skipping and Dystrophin Production Data
Safety and Tolerability Data
The 5 mg/kg dose of PGN-EDO51 was well tolerated by all study cohort participants through week 13. There were no discontinuations, dose interruptions or dose reductions.
The Company plans to present additional results from the 5 mg/kg cohort at a medical meeting later in the year.
Update on PGN-EDO51 10 mg/kg Cohort
As of July 29, 2024, two participants have received a total of four doses at 10 mg/kg in the ongoing CONNECT1 study. To date, PGN-EDO51 has been generally well tolerated at this dose level and the Company expects to report initial results from the 10 mg/kg cohort in early 2025.
Update on CONNECT2-EDO51 Clinical Trial
Based on the data from CONNECT1, including PGN-EDO51’s emerging safety profile to date, the Company is working to optimize the design of the CONNECT2 Phase 2 double-blind, placebo-controlled 25-week multinational trial. The CONNECT2 clinical trial is open in the United Kingdom. The Company continues to engage with regulators in the European Union and expects to open the clinical trial in the United States by year-end.
Conference Call Details
PepGen will host a conference call and webcast today at 4:30 p.m. ET to review the data being presented. To access the call, please dial (866) 400-0049 and provide the Conference ID 9666330. A live webcast of the presentation will be available on the Events & Presentations section of the PepGen investor website,
investors.pepgen.com
.
About PGN-EDO51
PGN-EDO51, PepGen’s lead clinical candidate for the treatment of Duchenne muscular dystrophy (DMD), utilizes the Company’s proprietary Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic phosphorodiamidate morpholino oligomer (PMO) 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 DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein. The U.S. Food and Drug Administration has granted PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations for the treatment of patients with DMD amenable to an exon-51 skipping approach.
About the CONNECT Clinical Program
CONNECT1-EDO51 is an open-label, multiple ascending dose Phase 2 trial designed to evaluate PGN-EDO51 at up to three different dose levels starting with 5 mg/kg administered intravenously once every four weeks for 12 weeks in patients with DMD amenable to an exon 51-skipping approach. The key endpoints for this trial are safety, dystrophin production and exon skipping. Multiple pharmacokinetic parameters are also being assessed as part of the trial protocol.
CONNECT2-EDO51 is a double-blind, placebo-controlled, multiple ascending dose, multinational Phase 2 trial designed to evaluate PGN-EDO51 at up to three different dose levels intravenously once every four weeks for 24 weeks in patients with DMD amenable to an exon 51-skipping approach. Endpoints included in this trial are safety, dystrophin production, exon skipping and clinical assessments of mobility, pulmonary function and quality of life.
About Duchenne Muscular Dystrophy (DMD)
DMD is an X-linked recessive muscle-wasting disease that predominantly affects males. This progressively debilitating and fatal disease is caused by genetic mutations in the gene encoding dystrophin, a protein critical for healthy muscle function, and is one of the most prevalent rare genetic diseases, with an incidence rate of approximately one in every 3,500 to 5,000 male births. DMD is characterized by progressive muscle weakness, which leads to patients losing the ability to walk, a loss of upper body function, cardiac issues and difficulties breathing. DMD is invariably fatal by young adulthood. Despite significant advances in treatments for this devastating disease, current exon skipping therapies are limited by poor delivery to muscle tissue nuclei and have yet to establish meaningful clinical benefit for DMD patients.
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 (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
www.pepgen.com
. Follow PepGen on
LinkedIn
and
X
.
SOURCE:
PepGen
Orphan DrugClinical ResultPhase 2Oligonucleotide
14 May 2024
– CONNECT1-EDO51 trial preliminary data from 5mg/kg dose cohort expected mid-2024 – – FREEDOM1-DM1 trial preliminary data from at least 5mg/kg dose cohort expected second half 2024 – – Net proceeds of $86.3 million from common stock offerings extending cash runway into 2026 – BOSTON, May 14, 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 and recent corporate highlights for the quarter ended March 31, 2024. “Our team has made exceptional progress in the first quarter advancing multiple clinical trials for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1),” said James McArthur, Ph.D., President and CEO of PepGen. “We are on track to achieve several significant milestones during the remainder of 2024, including sharing preliminary data from both the CONNECT1-EDO51 and FREEDOM-DM1 clinical trials and initiating the FREEDOM2-DM1 Phase 2 clinical trial in people living with DM1.” 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 Enhanced Delivery Oligonucleotide (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 DMD patients. Phase 2 CONNECT1-EDO51 Clinical Trial of PGN-EDO51: In March 2024, PepGen announced that the 5 mg/kg PGN-EDO51 dose cohort was fully enrolled. PepGen expects to report preliminary data from this cohort in mid-2024, including initial safety, exon 51 skipping, and dystrophin production data. CONNECT1-EDO51 is a Phase 2, open-label multiple ascending dose (MAD) clinical trial, being conducted in Canada, evaluating PGN-EDO51 in approximately 10 male patients at least 8 years of age with DMD amenable to an exon 51-skipping approach.Phase 2 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 CONNECT2-EDO51 in the United Kingdom. CONNECT2 is a Phase 2, randomized, double-blind, placebo-controlled MAD clinical trial, 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 CONNECT2 clinical trial, together with the data from CONNECT1, is designed to potentially support a future accelerated approval pathway, subject to regulatory authority feedback.Orphan Drug and Rare Pediatric Disease Designations granted to PGN-EDO51: In March 2024, PepGen announced that the U.S. Food and Drug Administration has granted both Orphan Drug and Rare Pediatric Disease Designations for PGN-EDO51 for the treatment of patients with DMD amenable to an exon-51 skipping approach.In March 2024, PepGen presented two posters on the PGN-EDO51 program at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. Poster title: Single- and Repeat-Dose Nonclinical Data for PGN-EDO51 Demonstrated Potential for the Treatment of DMD.Poster title: CONNECT1-EDO51 and CONNECT2-EDO51: Phase 2 Study Designs to Evaluate Safety and Efficacy for DMD Amenable to Exon 51 Skipping.
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, a key RNA splicing protein. 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. Phase 1 FREEDOM-DM1 Clinical Trial of PGN-EDODM1: 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. FREEDOM-DM1 is a Phase 1 single ascending dose (SAD) clinical trial evaluating PGN-EDODM1 in approximately 24 adult patients with DM1 in the U.S., Canada, and the United Kingdom.In April 2024, PepGen presented a poster on the PGN-EDODM1 program at The 14th International Myotonic Dystrophy Consortium (2024 IDMC-14) Meeting. Poster title: FREEDOM-DM1: Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1. In March 2024, PepGen presented two posters on the PGN-EDODM1 program at the 2024 MDA Clinical & Scientific Conference. Poster title: PGN-EDODM1 Single- and Repeat-Dose Nonclinical Data Indicated Mechanistic and Meaningful Activity for Potential Treatment of DM1.Poster title: FREEDOM-DM1: Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for DM1.
PGN-EDO51 and PGN-EDODM1 posters presented at the 2024 MDA Conference, and PGN-EDODM1 poster presented at the 2024 IDMC-14 Meeting, are available on the Investors page of our website under past events within the Events & Presentations page of the News & Events section. PGN-EDO53: DMD PGN-EDO53, PepGen's third investigational candidate 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-EDO53 is designed to skip exon 53 of the dystrophin transcript, an established therapeutic target for approximately 8% of DMD patients. PepGen continues to advance PGN-EDO53 into investigational new drug (IND) and clinical trial application (CTA) enabling nonclinical studies. Other Corporate Updates In February 2024, PepGen received $9.9 million from its at-the-market offering program and $76.4 million from an underwritten public offering, resulting in net proceeds of $86.3 million. Financial Results for the Three Months Ended March 31, 2024 Cash, cash equivalents and marketable securities were $175.2 million as of March 31, 2024, inclusive of net proceeds from the recent common stock offerings, which is anticipated to fund currently planned operations into 2026.Research and Development expenses were $14.7 million for the three months ended March 31, 2024, compared to $14.4 million for the same period in 2023.General and Administrative expenses were $5.1 million for the three months ended March 31, 2024, compared to $3.7 million for the same period in 2023.Net loss was $18.0 million for the three months ended March 31, 2024, compared to $16.3 million for the same period in 2023. PepGen had approximately 32.4 million shares outstanding on March 31, 2024. 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 profile of our product candidates, including PGN-EDO51 and PGN-EDODM1, our technology, including our EDO platform, the design, initiation and conduct of clinical trials, including expected timelines for our CONNECT2-EDO51 Phase 2 trial and FREEDOM2-DM1 Phase 2 trial and 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 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 and FREEDOM-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, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our CONNECT1-EDO51, CONNECT2-EDO51, FREEDOM-DM1 and FREEDOM2-DM1 programs; 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. Investor ContactNoel DonnellyChief Financial Officerndonnelly@pepgen.com Media ContactSarah SuttonArgot Partnerspepgen@argotpartners.com Condensed Consolidated Statements of Operations(unaudited, in thousands) Three Months EndedMarch 31, 2024 2023 Operating expenses: Research and development $14,732 $14,360 General and administrative 5,066 3,671 Total operating expenses $19,798 $18,031 Operating loss $(19,798) $(18,031)Other income (expense) Interest income 1,735 1,792 Other income, net 43 (80)Total other income (expense), net 1,778 1,712 Net loss before income tax $(18,020) $(16,319)Income tax expense — — Net loss $(18,020) $(16,319)
Condensed Consolidated Balance Sheets(unaudited, in thousands) March 31, December 31, 2024 2023 Assets Cash, cash equivalents and marketable securities $175,223 $110,407 Other assets 31,777 32,645 Total assets $207,000 $143,052 Liabilities and stockholders’ equity Liabilities $28,001 $34,631 Stockholders’ equity: 178,999 108,421 Total liabilities and stockholders’ equity $207,000 $143,052
Phase 2Phase 1Orphan DrugOligonucleotideFinancial Statement
100 Deals associated with PGN-EDO51
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R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Muscular Dystrophy, Duchenne | Phase 2 | CA | 18 May 2023 |
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Clinical Result
Clinical Result
Indication
Phase
Evaluation
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| Study | Phase | Population | Analyzed Enrollment | Group | Results | Evaluation | Publication Date |
|---|
Phase 2 | Muscular Dystrophy, Duchenne exon 51-skipping | 3 | PGN-EDO51 5 mg/kg | (qklvhiurua) = ufbqzhuztn thlhufocfg (wbzpwywhlm ) View more | Positive | 31 Jul 2024 | |
Phase 1 | - | (wfvznsunyj) = The majority of treatment-emergent adverse events were mild and resolved without intervention, including transient, reversible changes in kidney biomarkers (n=9) and hypomagnesemia (n=2) at the highest doses, with no significant clinical sequelae cyqrtbkzpu (szafrhdsgu ) | Positive | 01 May 2023 | |||
Phase 1 | - | swrhgzdicl(qirygmqftl) = transient, reversible changes in kidney biomarkers (n=9) and hypomagnesemia (n=2) at the highest doses; with no significant clinical sequelae. At 15 mg/kg, one HV received IV hydration after increases in kidney biomarkers with complete resolution. ubaaxcdrpq (nvjkmomubg ) View more | - | 25 Apr 2023 | |||
Phase 1 | Muscular Dystrophy, Duchenne kidney biomarkers | hypomagnesemia | - | (tyfqocbaxp) = The majority of treatment-emergent adverse events were mild and resolved without intervention, including transient, reversible changes in kidney biomarkers (n=9) and hypomagnesemia (n=2) at the highest doses, with no significant clinical sequelae icyyeluprf (rsgbqihqwv ) | Positive | 19 Mar 2023 | ||
GlobeNewswire Manual  | Phase 1 | Muscular Dystrophy, Duchenne Exon 51 | 32 | (qztxgywyxe) = mild and resolved without any intervention awvwohpxwp (khkeyhktaz ) View more | Positive | 28 Sep 2022 | |
Placebo |
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