On Friday, H.C. Wainwright maintained a Buy rating and a $37.00 stock price target for Pharming Group (NASDAQ:PHAR), following the company’s announcement of a new clinical trial. Pharming Group has initiated a Phase 2 proof-of-concept study to assess leniolisib (Joenja) in patients with primary immunodeficiencies (PIDs) related to immune dysregulation from altered PI3Kδ signaling.
The trial is a single-arm, open-label, dose-ranging study projected to enroll around 12 patients. It will focus on evaluating safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical efficacy. The outcomes of this Phase 2 trial are expected to inform the design of a potential Phase 3 study.
According to the company, the pivotal study might be a placebo-controlled, randomized trial with a larger patient enrollment than the previous APDS program, which included about 30 patients. Moreover, the company anticipates that at least one year of exposure data will likely be necessary to support the supplemental new drug application (sNDA).
The conditions targeted in the study include ALPS-FAS, CTLA4 haploinsufficiency, NFKB1 haploinsufficiency, and PTEN deficiency. These PIDs are often severe and life-threatening, and currently, there are no approved therapies addressing the underlying immune dysfunction.
Pharming’s move to progress leniolisib into Phase 2 trials and demonstrate clinical efficacy in PIDs could potentially be a significant advancement for the company. It would not only expand its clinical portfolio but also create additional value. The firm’s analyst has reiterated the Buy rating and price target based on these developments.
In other recent news, Pharming Group has seen robust growth in its financial results and patient enrollment for its key products, RUCONEST and Joenja. The company reported a 23% increase in Q2 sales for RUCONEST and a significant sales increase for Joenja.
In addition, Pharming Group completed enrollment for the first leniolisib study and is initiating a Phase 2 study. The company’s full-year revenue guidance remains between $280 million and $295 million, with a Q2 revenue growth of 35% and a stable gross profit of 89%.
Furthermore, Pharming Group’s drug, Joenja, received regulatory approval from the Medicines and Healthcare products Regulatory Agency (MHRA) in the U.K. for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), marking the first specific approval for APDS treatment in the country. This development follows the drug’s successful approval in the United States and Israel, expanding its global presence.
H.C. Wainwright maintained its Buy rating for Pharming Group, indicating confidence in the company’s prospects. The firm highlighted the potential for further expansion of Joenja’s market reach, given ongoing regulatory reviews in other countries. These are recent developments in the company’s journey, showcasing its progress in the biopharmaceutical industry.
InvestingPro Insights
Pharming Group’s recent initiation of a Phase 2 proof-of-concept study for leniolisib aligns with its strong financial performance and market position. According to InvestingPro data, the company has demonstrated impressive revenue growth of 34.54% over the last twelve months as of Q2 2023, reaching $277.56 million. This growth trajectory supports Pharming’s ability to invest in new clinical trials and expand its product pipeline.
InvestingPro Tips highlight Pharming’s impressive gross profit margins, which stand at 88.55% for the same period. This financial strength provides the company with resources to fund research and development initiatives, such as the newly announced clinical trial.
While the company is currently not profitable, with a P/E ratio of -42.82, the initiation of new clinical trials like the leniolisib study for PIDs could potentially lead to future revenue streams and profitability. Investors should note that Pharming operates with a moderate level of debt, which may provide flexibility for its research and development efforts.
For readers interested in a more comprehensive analysis, InvestingPro offers 8 additional tips for Pharming Group, providing a deeper understanding of the company’s financial health and market position.
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