On Thursday, H.C. Wainwright reaffirmed its Buy rating and $37.00 price target for Pharming Group (NASDAQ:PHAR), representing significant upside potential from the current price of $9.27. According to InvestingPro data, analyst targets range from $14 to $37, with the company showing impressive gross profit margins of 89.39% in the last twelve months.
The announcement of positive topline results from a Phase 3 clinical trial comes as the stock has gained over 22% in the past week.
The trial evaluated the safety, tolerability, and efficacy of leniolisib (Joenja) in treating children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS) across various regions including the U.S., Europe, and Japan. The company's strong financial position, with a current ratio of 3.53 indicating ample liquidity to fund its research programs, supports its ongoing clinical development efforts.
The study met its primary efficacy endpoints, which included a reduction in index lymph node size and an increase in the proportion of naïve B cells out of total B cells from baseline at 12 weeks. The secondary endpoint was to assess improvement in health-related quality of life through a patient questionnaire. The findings indicated improvement in lymphoproliferation, immunophenotype correction, and consistent improvements across all dose levels, which are in line with previous findings in adolescent and adult patients.
The trial also reported that all treatment-emergent adverse events were mild to moderate, with no drug-related serious adverse events, and all patients completed the 12-week treatment period. These patients are now continuing treatment with leniolisib for an additional year through an open-label extension trial to further evaluate its long-term safety, tolerability, and efficacy.
Moreover, a separate Phase 3 trial is currently in progress to test a new pediatric formulation of leniolisib for children aged 1 to 6 years with APDS. These 12-week results underscore leniolisib's potential to meet the treatment needs of pediatric patients with this rare immune disorder.
The continued Buy rating and price target reflect confidence in the drug's market potential following its FDA approval in March 2023 for treating APDS in adults and pediatric patients aged 12 years and older.
With revenue growth of 30.64% in the last twelve months and an overall "GOOD" financial health rating from InvestingPro, which offers 6 additional key insights about Pharming Group's investment potential in its comprehensive Pro Research Report.
In other recent news, Pharming Group NV reported a 12% quarter-over-quarter increase in Q3 revenues, totaling $74.8 million, slightly below the $78 million expected by Oppenheimer and consensus estimates. Despite this, the company confirmed its full-year 2024 revenue guidance to be between $280 million and $295 million.
Oppenheimer has since revised its revenue estimate for Pharming Group down from $297 million to $287 million for the year. In light of these recent developments, Oppenheimer has maintained an Outperform rating for Pharming Group, albeit with a revised price target.
Jefferies also initiated coverage on Pharming Group shares with a Buy rating, highlighting the company's strong revenue growth. The company's key product, Joenja, grew by a substantial 73% and is expected to tap into new commercial opportunities in other significant markets, including Europe and Japan, starting from 2026.
Pharming Group is actively seeking to expand its portfolio by acquiring additional late-stage assets in the rare disease category. This strategy could not only boost Pharming's revenue but also increase profit margins, especially if the new assets can be integrated within the existing commercial framework. Despite the upcoming retirement of long-time CEO Sijmen de Vries and potential competition for its product Ruconest, Pharming Group's resilience and growth potential remain evident.
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