Prime Medicine Publishes Phase 1/2 Data for PM359, Demonstrating Significant Efficacy
Written by Emily J. Thompson, Senior Investment Analyst
Source: Newsfilter
Updated: 10 hour ago0mins
- Clinical Data Release: Prime Medicine published Phase 1/2 clinical data for PM359 in the New England Journal of Medicine, demonstrating that two patients achieved 69% and 83% neutrophil engraftment by Day 30, significantly exceeding the 20% minimum threshold for clinical benefit, marking the first human validation of Prime Editing's safety and efficacy.
- Sustained Efficacy: Both patients remained free of new CGD-related complications post-treatment, with Patient 1 stopping mesalamine without CAC flare-ups and Patient 2 showing a substantial decrease in fecal calprotectin levels, indicating PM359's potential to enhance patient quality of life.
- Safety Assessment: No clinically significant adverse events attributable to PM359 were observed during treatment, with all noted toxicities consistent with busulfan-based conditioning, further supporting the safety profile of Prime Editing technology.
- Future Development Potential: This data release not only showcases PM359's early clinical efficacy but also provides critical insights into the broad applicability of Prime Editing technology in gene editing, potentially opening new avenues for treating various genetic and immunological diseases.
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Analyst Views on PRME
Wall Street analysts forecast PRME stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for PRME is 6.08 USD with a low forecast of 4.25 USD and a high forecast of 9.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
6 Analyst Rating
3 Buy
3 Hold
0 Sell
Moderate Buy
Current: 3.850
Low
4.25
Averages
6.08
High
9.00
Current: 3.850
Low
4.25
Averages
6.08
High
9.00
JPMorgan
Tessa Romero
Neutral
maintain
2025-11-18
Reason
JPMorgan
Tessa Romero
Price Target
2025-11-18
maintain
Neutral
Reason
JPMorgan analyst Tessa Romero kept a Neutral rating with no price target on Prime Medicine following a transfer of coverage. The firm says it would consider recommending the shares as the company moves closer to "value-creating milestones."
Citi
Samantha Semenkow
Neutral
downgrade
$5
2025-11-11
Reason
Citi
Samantha Semenkow
Price Target
$5
2025-11-11
downgrade
Neutral
Reason
Citi analyst Samantha Semenkow lowered the firm's price target on Prime Medicine to $4.25 from $5 and keeps a Neutral rating on the shares.
Citi
Neutral
maintain
$4 -> $5
2025-10-29
Reason
Citi
Price Target
$4 -> $5
2025-10-29
maintain
Neutral
Reason
Citi raised the firm's price target on Prime Medicine to $5 from $4 and keeps a Neutral rating on the shares. The firm adjusted biotech price targets as part of a Q3 earnings preview.
Citi
Buy
to
Neutral
downgrade
$10
2025-05-26
Reason
Citi
Price Target
$10
2025-05-26
downgrade
Buy
to
Neutral
Reason
Citi downgraded Prime Medicine to Neutral from Buy with a price target of $1.50, down from $10. The shares are trading near cash, reflecting the market's uncertainty on the company's path to potential value inflection, the analyst tells investors in a research note. The firm says Prime's cash runway supports operations only into the first half of 2026 after implementing cost-cutting measures, which is well short of management's guidance of initial in-vivo clinical data for Wilson's and Alpha-1 antitrypsin deficiency in 2027.
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About PRME
Prime Medicine, Inc. is a biotechnology company engaged in creating and delivering gene editing therapies to patients. It deploys its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated one-time curative genetic therapies. It is advancing a set of high-value programs across its core areas of focus (hematology, immunology and oncology, liver, and lung). These include ex vivo hematopoietic stem cell (HSC) programs for the treatment of p47phox chronic granulomatous disease CGD and X-linked CGD; a lipid nanoparticle (LNP) Prime Editor for the treatment of Wilson’s Disease; a LNP or adeno-associated virus Prime Editors for the treatment of cystic fibrosis; and ex vivo T-cell therapies. PM359, its product candidate within its hematology, immunology and oncology area of focus, targets the p47phox variant of CGD. Its PM577 comprises a Prime Editor encapsulated as RNA cargo in a liver-targeted LNP.
About the author
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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.
Sigh Up to Get Intellectia Insights
What is the significance of the 69% and 83% neutrophil engraftment results?
How does PM359 demonstrate safety and efficacy in the Phase 1/2 trial?
Will PM359's success lead to accelerated approval for other genetic therapies?
How likely is Prime Editing to become a standard treatment for CGD?