PepGen Reports Promising Clinical Data from DM1 Trial
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 17 hours ago
0mins
Should l Buy PEPG?
PepGen announced promising clinical data from the 5 mg/kg multiple ascending dose cohort of the ongoing Phase 2 FREEDOM2-DM1 trial in patients with myotonic dystrophy type 1. The Company believes the totality of safety and efficacy results support the potential of the ongoing 10 mg/kg dose cohort, with clinical results expected in the second half of 2026. "We are excited to share data from the lowest dose cohort in our multiple ascending dose study of PGN-EDODM1 in patients with DM1," said Paul Streck, MD, EVP Research and Development. "Overall, we are encouraged by the favorable safety and tolerability profile, and the positive trends exhibited with splicing improvement and vHOT. We look forward to reporting data from the ongoing 10 mg/kg multiple ascending dose cohort of FREEDOM2, which is more than halfway enrolled and remains on track to read out in the second half of this year." FREEDOM2 is a Phase 2 MAD, randomized, placebo-controlled clinical trial evaluating PGN-EDODM1 in patients with DM1, with planned dose escalation up to 12.5 mg/kg. The 5 mg/kg cohort enrolled eight patients, who received PGN-EDODM1 or placebo every four weeks over a 12-week period. Key endpoints include safety, splicing correction and functional outcome measures. The data cutoff was March 4, 2026. Patients (n=6) treated with PGN-EDODM1 at 5 mg/kg demonstrated a mean splicing correction of 7.3%, compared to 6.8% in placebo-treated patients (n=2). Excluding an outlier, patients showed a mean splicing correction of 22.9%. The outlier patient exhibited a worsening in splicing correction (70.8%), reducing the overall group mean to 7.3%. Middle finger vHOT in the treatment group showed a positive trend of improvement versus a worsening observed in the placebo group. Both returned to baseline at Week 16. Mean muscle tissue concentrations of PGN-EDODM1 available in 5 of 6 treated patients were 158 ng/g, as measured approximately one week after the fourth dose in the MAD study; one concentration readout remains pending. No meaningful improvements were observed in 10-meter walk/run test (10MWRT) or handgrip strength at the starting PGN-EDODM1 dose of 5 mg/kg.
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Analyst Views on PEPG
Wall Street analysts forecast PEPG stock price to rise
2 Analyst Rating
1 Buy
0 Hold
1 Sell
Hold
Current: 4.230
Low
3.00
Averages
11.50
High
20.00
Current: 4.230
Low
3.00
Averages
11.50
High
20.00
About PEPG
PepGen Inc. is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies. The Company’s Enhanced Delivery Oligonucleotide (EDO) platform leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Its EDO peptides are engineered to optimize tissue penetration, cellular uptake and nuclear delivery. Using these EDO peptides, the Company is generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases. It is focused on addressing the underlying cause of Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1), which have high unmet need. Its pipeline consists of two clinical stage programs, PGN-EDO51 for DMD patients who are amenable to an exon 51-skipping approach and PGN-EDODM1 for DM1 patients, and several additional preclinical stage programs. Its product candidates also include PGN-EDO53, PGN-EDO45 and PGN-EDO44.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
PepGen Shares Plunge 44% After Mixed Mid-Stage Trial Results
- Disappointing Trial Results: PepGen (PEPG) shares plummeted approximately 44% in after-hours trading following the mid-stage trial results for its lead treatment PGN-EDODM1, which showed no significant improvements in handgrip strength or 10-meter walk/run tests at the 5 mg/kg dose cohort.
- Ongoing Research Plans: Despite the disappointing results, the company plans to continue studying the 10 mg/kg dose cohort, with data expected in H2 2026, indicating the company's ongoing confidence in the drug's potential.
- Divergent Analyst Opinions: While Leerink Partners analyst Joseph Schwartz highlighted the mixed nature of the data, Oppenheimer's Kostas Biliouris argued that the selloff was overdone, maintaining that PGN-EDODM1 still shows positive drug effects and may yield better results at higher doses.
- Market Reaction Analysis: Despite some analysts' optimism regarding the drug, PepGen's stock remains under pressure due to a partial FDA hold on the mid-stage trial, reflecting market sensitivity to clinical data and concerns about the company's future prospects.
See More
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- PEPG Trial Disappointment: PepGen's Phase 2 Freedom 2 study results showed no significant improvements in walking speed or hand strength at the starting dose of 5 mg/kg, leading to a 45% plunge in premarket trading, reflecting investor disappointment in the therapy's efficacy.
- SGMO Earnings Miss: Sangamo Therapeutics reported a Q4 loss per share of $0.11, wider than the expected $0.05 loss, despite revenue rising to $14.23 million, indicating pressure on the company's funding outlook that may impact future operations.
- PHR Revenue Outlook Cut: Phreesia lowered its fiscal 2027 revenue outlook to $510 million to $520 million, below analyst expectations of $552.1 million, resulting in a 27% drop in share price and prompting downgrades from multiple brokerages.
- Market Sentiment Fluctuates: While retail sentiment for PEPG, SGMO, and PHR on Stocktwits was 'extremely bullish', the disappointing performance and unclear outlook have severely undermined investor confidence, reflecting a cautious attitude towards biotech stocks.
See More
PepGen Under Investigation for Securities Fraud Claims
- Securities Fraud Investigation: Pomerantz LLP is investigating on behalf of PepGen Inc. investors whether the company and its officers have engaged in securities fraud or other unlawful business practices, highlighting serious concerns regarding corporate governance.
- Partial Clinical Hold: On March 4, 2026, PepGen announced that the FDA placed a partial clinical hold on its PGN-EDODM1 trial, raising questions about previously submitted pharmacology and toxicology studies, which could impact the company's future R&D efforts.
- Significant Stock Drop: Following the FDA announcement, PepGen's stock price fell by $1.25, or 18.57%, closing at $5.50 per share on March 5, 2026, reflecting market concerns about the company's outlook.
- Legal Firm Involvement: Pomerantz LLP, a prominent securities class action firm with over 85 years of experience, focuses on fighting for the rights of victims of securities fraud, indicating heightened investor scrutiny on corporate governance and transparency.
See More
PepGen Under Investigation for Securities Fraud
- Investigation Launched: Pomerantz LLP is investigating on behalf of PepGen Inc. investors regarding potential securities fraud or other unlawful business practices, indicating serious concerns about corporate governance.
- Clinical Trial Halted: On March 4, 2026, PepGen announced that the FDA placed a partial clinical hold on its PGN-EDODM1 trial, raising questions about previously submitted pharmacology and toxicology studies, which could impact the company's future R&D efforts.
- Stock Price Plummets: Following the FDA announcement, PepGen's stock price fell by $1.25, or 18.57%, closing at $5.50 per share on March 5, 2026, reflecting market pessimism regarding the company's outlook.
- Potential Legal Consequences: Pomerantz LLP, a prominent securities litigation firm, may initiate a class action against PepGen and its executives, further exacerbating the legal risks and financial pressures faced by the company.
See More
PepGen Under Investigation for Securities Fraud Claims
- Securities Fraud Investigation: Pomerantz LLP is investigating claims on behalf of PepGen Inc. investors regarding potential securities fraud or unlawful business practices, indicating significant scrutiny over the company's governance.
- Clinical Trial Hold: On March 4, 2026, PepGen announced that the FDA placed a partial clinical hold on its PGN-EDODM1 trial, raising concerns related to previously submitted pharmacology and toxicology studies, which could impact the company's future R&D efforts.
- Stock Price Decline: Following the FDA announcement, PepGen's stock price fell by $1.25, or 18.57%, closing at $5.50 per share on March 5, 2026, reflecting market concerns about the company's outlook.
- Law Firm's Reputation: Pomerantz LLP, a leading firm in securities class action litigation with over 85 years of experience, focuses on protecting the rights of victims of securities fraud and corporate misconduct, underscoring its authority and influence in the industry.
See More
PepGen Faces FDA Clinical Hold Investigation
- FDA Clinical Hold: On March 5, 2026, PepGen disclosed that the FDA imposed a partial clinical hold on its PGN-EDODM1 FREEDOM2-DM1 trial due to concerns regarding previously submitted pharmacology and toxicology studies, which could significantly impact the company's research and development trajectory and market confidence.
- Sharp Stock Decline: Following the FDA announcement, PepGen's stock price plummeted during intraday trading on March 5, 2026, indicating investor anxiety over regulatory developments and uncertainty surrounding the FREEDOM2 clinical program, potentially leading to decreased investor confidence and capital outflows.
- Legal Investigation Initiated: Johnson Fistel, PLLP is investigating whether PepGen and its executives violated federal securities laws, and investors who suffered losses from PepGen stock are encouraged to join the investigation, which may affect the company's legal liabilities and future financial performance.
- Investor Rights Protection: Johnson Fistel, PLLP is a nationally recognized shareholder rights law firm, ranked among the Top 10 Plaintiff Law Firms in 2024, reflecting its effectiveness in advocating for investors, which may attract more affected investors seeking legal recourse.
See More
PepGen Shares Plunge 44% After Mixed Mid-Stage Trial Results
- Disappointing Trial Results: PepGen (PEPG) shares plummeted approximately 44% in after-hours trading following the mid-stage trial results for its lead treatment PGN-EDODM1, which showed no significant improvements in handgrip strength or 10-meter walk/run tests at the 5 mg/kg dose cohort.
- Ongoing Research Plans: Despite the disappointing results, the company plans to continue studying the 10 mg/kg dose cohort, with data expected in H2 2026, indicating the company's ongoing confidence in the drug's potential.
- Divergent Analyst Opinions: While Leerink Partners analyst Joseph Schwartz highlighted the mixed nature of the data, Oppenheimer's Kostas Biliouris argued that the selloff was overdone, maintaining that PGN-EDODM1 still shows positive drug effects and may yield better results at higher doses.
- Market Reaction Analysis: Despite some analysts' optimism regarding the drug, PepGen's stock remains under pressure due to a partial FDA hold on the mid-stage trial, reflecting market sensitivity to clinical data and concerns about the company's future prospects.
See More
PEPG, SGMO, and PHR Shares Decline Amid Disappointing Results
- PEPG Trial Disappointment: PepGen's Phase 2 Freedom 2 study results showed no significant improvements in walking speed or hand strength at the starting dose of 5 mg/kg, leading to a 45% plunge in premarket trading, reflecting investor disappointment in the therapy's efficacy.
- SGMO Earnings Miss: Sangamo Therapeutics reported a Q4 loss per share of $0.11, wider than the expected $0.05 loss, despite revenue rising to $14.23 million, indicating pressure on the company's funding outlook that may impact future operations.
- PHR Revenue Outlook Cut: Phreesia lowered its fiscal 2027 revenue outlook to $510 million to $520 million, below analyst expectations of $552.1 million, resulting in a 27% drop in share price and prompting downgrades from multiple brokerages.
- Market Sentiment Fluctuates: While retail sentiment for PEPG, SGMO, and PHR on Stocktwits was 'extremely bullish', the disappointing performance and unclear outlook have severely undermined investor confidence, reflecting a cautious attitude towards biotech stocks.
See More
PepGen Under Investigation for Securities Fraud Claims
- Securities Fraud Investigation: Pomerantz LLP is investigating on behalf of PepGen Inc. investors whether the company and its officers have engaged in securities fraud or other unlawful business practices, highlighting serious concerns regarding corporate governance.
- Partial Clinical Hold: On March 4, 2026, PepGen announced that the FDA placed a partial clinical hold on its PGN-EDODM1 trial, raising questions about previously submitted pharmacology and toxicology studies, which could impact the company's future R&D efforts.
- Significant Stock Drop: Following the FDA announcement, PepGen's stock price fell by $1.25, or 18.57%, closing at $5.50 per share on March 5, 2026, reflecting market concerns about the company's outlook.
- Legal Firm Involvement: Pomerantz LLP, a prominent securities class action firm with over 85 years of experience, focuses on fighting for the rights of victims of securities fraud, indicating heightened investor scrutiny on corporate governance and transparency.
See More
PepGen Under Investigation for Securities Fraud
- Investigation Launched: Pomerantz LLP is investigating on behalf of PepGen Inc. investors regarding potential securities fraud or other unlawful business practices, indicating serious concerns about corporate governance.
- Clinical Trial Halted: On March 4, 2026, PepGen announced that the FDA placed a partial clinical hold on its PGN-EDODM1 trial, raising questions about previously submitted pharmacology and toxicology studies, which could impact the company's future R&D efforts.
- Stock Price Plummets: Following the FDA announcement, PepGen's stock price fell by $1.25, or 18.57%, closing at $5.50 per share on March 5, 2026, reflecting market pessimism regarding the company's outlook.
- Potential Legal Consequences: Pomerantz LLP, a prominent securities litigation firm, may initiate a class action against PepGen and its executives, further exacerbating the legal risks and financial pressures faced by the company.
See More
PepGen Under Investigation for Securities Fraud Claims
- Securities Fraud Investigation: Pomerantz LLP is investigating claims on behalf of PepGen Inc. investors regarding potential securities fraud or unlawful business practices, indicating significant scrutiny over the company's governance.
- Clinical Trial Hold: On March 4, 2026, PepGen announced that the FDA placed a partial clinical hold on its PGN-EDODM1 trial, raising concerns related to previously submitted pharmacology and toxicology studies, which could impact the company's future R&D efforts.
- Stock Price Decline: Following the FDA announcement, PepGen's stock price fell by $1.25, or 18.57%, closing at $5.50 per share on March 5, 2026, reflecting market concerns about the company's outlook.
- Law Firm's Reputation: Pomerantz LLP, a leading firm in securities class action litigation with over 85 years of experience, focuses on protecting the rights of victims of securities fraud and corporate misconduct, underscoring its authority and influence in the industry.
See More
PepGen Faces FDA Clinical Hold Investigation
- FDA Clinical Hold: On March 5, 2026, PepGen disclosed that the FDA imposed a partial clinical hold on its PGN-EDODM1 FREEDOM2-DM1 trial due to concerns regarding previously submitted pharmacology and toxicology studies, which could significantly impact the company's research and development trajectory and market confidence.
- Sharp Stock Decline: Following the FDA announcement, PepGen's stock price plummeted during intraday trading on March 5, 2026, indicating investor anxiety over regulatory developments and uncertainty surrounding the FREEDOM2 clinical program, potentially leading to decreased investor confidence and capital outflows.
- Legal Investigation Initiated: Johnson Fistel, PLLP is investigating whether PepGen and its executives violated federal securities laws, and investors who suffered losses from PepGen stock are encouraged to join the investigation, which may affect the company's legal liabilities and future financial performance.
- Investor Rights Protection: Johnson Fistel, PLLP is a nationally recognized shareholder rights law firm, ranked among the Top 10 Plaintiff Law Firms in 2024, reflecting its effectiveness in advocating for investors, which may attract more affected investors seeking legal recourse.
See More
