On Wednesday, H.C. Wainwright maintained a Buy rating on Regulus (NASDAQ:RGLS) Therapeutics (NASDAQ:RGLS) with a price target of $10.00. This affirmation follows Regulus Therapeutics' announcement on October 8, 2024, regarding the completion of patient enrollment for Cohort 4 in their Phase 1b multiple-ascending dose study of RGLS8429. The drug is being investigated for its potential to treat Autosomal Dominant Polycystic Kidney Disease (ADPKD), a genetic disorder.
The latest cohort consists of 26 patients who are set to receive a 300 mg dose of RGLS8429 every other week over a three-month period. The study is designed to measure the changes in urinary polycystins PC1 and PC2, height-adjusted total kidney volume (htTKV), and overall kidney function as part of its endpoints.
Regulus Therapeutics has indicated that it plans to release topline data from a significant number of patients in Cohort 4 in early 2025. The data from this study is expected to contribute to the development and potential accelerated approval of RGLS8429.
In addition to the study progress, Regulus Therapeutics is also preparing for an end-of-Phase 1 meeting with the FDA, which is anticipated to occur near the end of 2024. The outcome of this meeting is expected to be instrumental in finalizing the design for the upcoming Phase 2/3 pivotal trial slated for 2025.
H.C. Wainwright's reiterated Buy rating and price target reflect the firm's continued confidence in the potential of RGLS8429 as a treatment for ADPKD and the progress of Regulus Therapeutics' clinical program.
In other recent news, Regulus Therapeutics has been making significant strides in the development of its anti-miRNA-17 therapy, currently in Phase 1b development for Autosomal Dominant Polycystic Kidney Disease (ADPKD). The therapy has shown early signs of safety and efficacy, with Canaccord Genuity maintaining a Buy rating and a $28.00 price target on the company. Notably, the Nobel Prize in Physiology or Medicine was recently awarded to Victor Ambros and Gary Ruvkun for their work on microRNA, a field closely related to Regulus's therapy.
Further, Regulus Therapeutics recently disclosed additional exploratory results from its Phase 1b study of RGLS8429, another treatment for ADPKD. The study showed that RGLS8429 consistently impacted the height-adjusted total kidney volume across high-risk classes. This development led to H.C. Wainwright and Jones Trading maintaining their Buy ratings on Regulus, with price targets of $10.00 and $8.00 respectively.
Looking ahead, Regulus Therapeutics is slated to initiate a pivotal Phase 2 trial in mid-2025, aiming for accelerated approval. The company also anticipates an end-of-Phase 1 meeting with the FDA in the fourth quarter of 2024.
InvestingPro Insights
As Regulus Therapeutics (NASDAQ:RGLS) advances its clinical program for RGLS8429, InvestingPro data provides additional context for investors. The company's market capitalization stands at $102.13 million, reflecting its current position as a clinical-stage biopharmaceutical company.
InvestingPro Tips highlight that Regulus holds more cash than debt on its balance sheet, which is crucial for funding ongoing clinical trials. This financial position aligns with the company's ability to continue its Phase 1b study and prepare for the upcoming Phase 2/3 pivotal trial in 2025.
However, investors should note that Regulus is quickly burning through cash, a common characteristic of biotech companies in the development stage. This burn rate underscores the importance of the upcoming clinical milestones and potential FDA interactions in determining the company's future funding needs.
The stock's price movements are quite volatile, as indicated by another InvestingPro Tip. This volatility is not unusual for clinical-stage biotech companies, whose valuations can fluctuate significantly based on clinical trial results and regulatory developments.
For those seeking a more comprehensive analysis, InvestingPro offers 5 additional tips that could provide deeper insights into Regulus Therapeutics' financial health and market position.
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