PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
- Preliminary data from the 5 mg/kg PGN-EDO51 dose level in the CONNECT1-EDO51 Phase 2 trial are expected mid-2024
- Phase 1 results following a single dose of 10 mg/kg of PGN-EDO51 in healthy volunteers demonstrated peak exon 51 skipping of 3.8% with 100% of subjects demonstrating exon skipping
- PepGen Inc. is committed to developing therapies with the potential to improve the lives of people living with DMD
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The initiation of the CONNECT1-EDO51 Phase 2 clinical trial by PepGen Inc. marks a significant progression in the development of a potential treatment for Duchenne muscular dystrophy (DMD), specifically for patients amenable to exon 51 skipping therapies. The trial's objective to evaluate the safety, tolerability and efficacy of PGN-EDO51 is a crucial step in the drug development process. The reported Phase 1 data indicating tolerability up to 15 mg/kg and the highest levels of exon skipping in comparison to other similar therapies, if validated by subsequent trials, could position PGN-EDO51 as a leading candidate in the exon-skipping treatment landscape.
From a medical research perspective, the focus on oligonucleotide muscle concentrations and dystrophin protein production is noteworthy. Dystrophin is integral for muscle function and its absence is the hallmark of DMD. Thus, the ability of PGN-EDO51 to induce exon skipping and restore dystrophin production could potentially translate into clinical benefits for patients. The upcoming data readout in mid-2024 will be pivotal in understanding the drug's therapeutic potential and guiding dosage escalation decisions.
For investors, PepGen Inc.'s announcement is a material event as it directly relates to the company's core product pipeline and future revenue potential. The transition from Phase 1 to Phase 2 trials signifies a de-risking of the investment, as the drug has passed initial safety hurdles. The biotechnology sector is highly sensitive to clinical trial outcomes and positive preliminary data in mid-2024 could significantly affect the company's stock valuation. However, investors should also be aware of the inherent risks in drug development, including the potential for adverse safety findings or insufficient efficacy in later-stage trials.
It is also essential to consider the competitive landscape and the market size for DMD treatments. If PGN-EDO51 continues to show promise, it could capture a substantial market share, especially given the reported superior exon skipping levels. However, the long-term financial impact will depend on the drug's final approval, pricing, insurance coverage and competition from other DMD therapies.
The advancement of treatments like PGN-EDO51 has broader economic implications beyond the stock market. The development of effective therapies for rare diseases like DMD can reduce long-term healthcare costs by potentially lessening the need for extensive supportive care and delaying disease progression. For the economy, this translates to potential savings in healthcare expenditures and improved productivity as individuals with DMD may have an improved quality of life and increased independence.
Additionally, the biotech industry's investment in research and development often acts as a catalyst for job creation and economic growth. The success of clinical trials can lead to increased investment in the sector, fostering innovation and potentially leading to breakthroughs in other areas of medicine. The economic impact of drug development, therefore, extends beyond immediate financial returns to encompass broader societal benefits.
- Preliminary data from the 5 mg/kg PGN-EDO51 dose level in the CONNECT1-EDO51 Phase 2 trial are expected mid-2024: including initial safety, exon 51 skipping and dystrophin protein production data -
- Phase 1 results following a single dose of 10 mg/kg of PGN-EDO51 in healthy volunteers demonstrated peak exon 51 skipping of
BOSTON, Jan. 08, 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 announced that the first patient has been dosed in its CONNECT1-EDO51 Phase 2, open-label multiple ascending dose (MAD) clinical trial evaluating PGN-EDO51 for the treatment of Duchenne muscular dystrophy (DMD) patients amendable to an exon 51 skipping therapy.
“We are pleased to have dosed the first patient in our CONNECT1-EDO51 clinical trial, which marks another milestone in our commitment to developing therapies with the potential to truly improve the lives of people living with DMD,” said James McArthur, Ph.D., President and CEO of PepGen. “Based on the levels of exon skipping achieved following a single dose of PGN-EDO51 in our Phase 1 healthy volunteer trial, we are looking forward to our initial planned data readout in DMD patients at the 5 mg/kg PGN-EDO51 dose level for CONNECT1-EDO51 in the middle of 2024.”
PepGen previously reported data from a Phase 1 trial evaluating PGN-EDO51 in 32 healthy adult volunteers. In the Phase 1 trial, PGN-EDO51 was generally well-tolerated through 15 mg/kg. PGN-EDO51 also produced the highest levels of exon 51 skipping in healthy volunteers following a single dose when compared to publicly available clinical data for other exon 51 skipping approaches. Following a single dose of 10 mg/kg, PGN-EDO51 achieved an average level of
The CONNECT1-EDO51 Phase 2 clinical trial is an open-label, MAD study, enrolling approximately 10 male patients of at least 8 years of age with DMD amenable to an exon 51 skipping approach to evaluate the safety and tolerability of PGN-EDO51. In addition to safety, oligonucleotide muscle concentrations, exon skipping and dystrophin protein production in muscle will be assessed at week 12 following 4 monthly doses of PGN-EDO51. Per the protocol, the starting dose will escalate from 5 mg/kg to 10 mg/kg. Further dose escalation will be determined based on evaluation of safety data from prior dose cohorts. (ClinicalTrials.gov identifier: NCT06079736)
About PGN-EDO51
PGN-EDO51, PepGen’s lead clinical candidate 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
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is an X-linked recessive muscle-wasting disease that predominantly affects males. This debilitating 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 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, 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, our technology, including our EDO platform, the design, initiation and conduct of clinical trials, including the CONNECT1-EDO51 clinical trial, including expected timelines, dose levels, 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; our ability to enroll patients in and complete our clinical trials, including the CONNECT1-EDO51 clinical trials; 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 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 PGN-EDO51, or other regulatory feedback requiring modifications to our development 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.
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