First Patient Enrolled in Japan for Groundbreaking Clinical Trial of Leniolisib for APDS Treatment!

Pharming Group N.V. is pleased to announce that the first patient has successfully been enrolled in its groundbreaking Phase III clinical trial to evaluate leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in Japan. This trial will assess the efficacy of leniolisib in both adults and pediatric patients aged 12 years and older, offering hope to the many individuals suffering from this rare primary immunodeficiency.

Pharming is set to evaluate the safety, tolerability, and efficacy of leniolisib in a single-arm, open-label clinical trial with three patients who are 12 years of age or older and have a confirmed APDS diagnosis. For an assessment period spanning 12 weeks, each patient will receive a weight-based dose of up to 70mg of leniolisib twice daily. The aim of the trial is to evaluate the same primary and secondary efficacy endpoints that were used for assessing the leniolisib APDS trials.

Pharming is pushing to get leniolisib approved for use in Japan, and to make this happen, they’ve launched a trial to help demonstrate its efficacy. All eligible participants enrolled in the trial will receive the investigational drug for a full year, with the plan being to use the data collected in a future registration application to the Pharmaceuticals and Medical Devices Agency (PMDA). In addition, and to ensure that their results are as comprehensive as possible, an open-label extension trial will be made available to ongoing study participants.

Hirokazu Kanegane, Professor of the Department of Child Health and Development, Tokyo Medical and Dental University, commented:

The launch of this clinical trial offers a glimmer of hope to the APDS community in Japan. Although support treatments are currently available, a disease-modifying treatment would represent a major breakthrough for patients, their families, caregivers and doctors. If successful, this new therapy could shape the future of rare primary immunodeficiency treatment in Japan.

Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:

I am very proud to announce the start of our Phase III trial in Japan, furthering our ambition to provide an innovative and effective oral therapy to patients with APDS. Our Phase II/III trial results have proven Joenja®’s potential in this rare and progressive disease, and now, we are actively working with regulatory authorities to supply this revolutionary treatment to those in need across the globe. It is our hope that Joenja® will alter the course of APDS and give patients in Japan the quality of life that they deserve.

The Ministry of Health, Labour and Welfare of Japan has granted orphan drug designation (ODD) to leniolisib for the treatment of APDS in May 2023. This is the first approved therapy in Japan for this complex and progressive disease, providing a much-needed ray of hope for patients affected by APDS.

The Japanese Ministry of Health, Labour and Welfare has introduced an innovative system to drive the development of treatments for diseases requiring urgent attention. Through its Observational Drug Development (ODD) program, investigators can receive guidance, subsidies, and preferential consideration for drug approval applications for conditions impacting less than 50,000 people across the country. In this way, ODD provides vital support for the development of desperately-needed treatments.

In April 2023, the U.S. Food and Drug Administration gave the green light to Leniolisib, allowing the drug to be commercially launched as Joenja® for the treatment of APDS in patients over the age of 12. This is a major breakthrough that could provide immense relief to those patients who have been struggling for years with this debilitating condition.

About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)

APDS (Primary Immunodeficiency) is a rare and life-threatening condition, affecting only around one to two people in every million worldwide. First identified in 2013, APDS is caused by variants in two genes known as PIK3CD and PIK3R1, which when overly active, disrupt the normal development and function of essential immune cells in the body. This leads to a host of symptoms including frequent and recurrent chest and sinus infections, lymphoproliferation, autoimmunity, and enteropathy.

Unfortunately, due to these symptoms being associated with so many conditions, it has been reported that APDS sufferers can often endure a long and arduous diagnostic journey, with the median delay standing at as much as seven years. Regrettably, this delay can cause an accumulation of damage which could lead to dire and lasting consequences including permanent lung damage and even lymphoma. The only way to accurately diagnose APDS is through genetic testing.

About Joenja® (leniolisib)

Joenja® (leniolisib), the first and only targeted treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome available in the US, has demonstrated clinical efficacy in a randomized, placebo-controlled Phase II/III clinical trial. Not only did the treatment show statistically significant improvement in immune dysregulation, but it also resulted in the normalization of immunophenotype within these patients.

With plans for further regulatory approvals in the UK, Canada, Australia and Japan, Joenja® is a safe and effective treatment for APDS in adult and pediatric patients 12 years of age and older, with a Phase III clinical trial in children aged 4 to 11 underway and another planned in children aged 1 to 6 years.

About Pharming Group N.V.

Pharming Group N.V. is a pioneering biopharmaceutical company that is devoted to improving the lives of those living with rare, life-threatening conditions. With its transformative portfolio of precision medicines and protein replacement therapies at various stages of development, Pharming is delivering hope across North America, Europe, the Middle East, Africa, and Asia-Pacific. Headquartered in Leiden, Netherlands, Pharming’s dedicated employees around the globe are dedicated to bringing relief to patients in over 30 markets.

Forward-Looking Statements

Pharming is striving for success in its preclinical studies and clinical trials of its product candidates. However, there are still various risks and uncertainties that could alter the magnitude of the expected outcomes. Among these are the scope, progress, and expansion of clinical trials, along with potential cost implications.

Furthermore, numerous scientific, regulatory, and technical developments could significantly impact the clinical and commercial prospects of Pharming. As such, readers should be aware that actual results could differ significantly and adversely from those previously expected or projected. To ensure transparency, this press release has incorporated a cautionary section to inform readers of all the risks associated with Pharming’s operations and performance.

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