Our lead projects
To move closer to our vision of a world free of pain, we are currently pursuing a range of programmes across different modalities, targets and mechanisms of action. Here are some selected highlights from our development pipeline.
RTX (resiniferatoxin)
In April 2021, we acquired the Swiss biotech company Mestex AG and its lead compound resiniferatoxin (RTX), a highly potent TRPV1 agonist. Grünenthal initiated a global Phase III clinical development programme in 2022 to evaluate the efficacy and safety of intra-articular injections of RTX in adults with moderate to severe pain associated with osteoarthritis of the knee for whom available treatment options provide inadequate relief.
The programme consists of three trials – two pivotal trials (KF7039-01 and KF7039-02) and an open-label safety study (KF7039-03). As primary endpoints, the pivotal trials evaluated the change in pain score on the Western Ontario and McMaster Universities (WOMAC) Osteoarthritis Index. At the same time, the trials measured several secondary endpoints, including change in pain and physical function scores on the WOMAC Osteoarthritis Index.
Both pivotal trials did not meet their primary endpoints. Grünenthal will conclude this development programme with the completion of the open-label safety study KF7039-03 in the second half of 2025. To date, RTX has shown a favourable safety profile and has been well tolerated.
Qutenza™ Life Cycle Management
Qutenza™ is a patch containing prescription-strength capsaicin. In Europe, it is approved for the treatment of peripheral neuropathic pain. In the US, it is approved for the treatment of neuropathic pain associated with post-herpetic neuralgia, and in 2020 it also received approval for the treatment of neuropathic pain associated with diabetic peripheral neuropathy (DPN) of the feet in adults.
Our life-cycle management efforts focus on making Qutenza™ more widely available by expanding the label further, particularly in the United States. Specifically, we have started a pivotal Phase III study in post-surgical neuropathic pain. We are also pursuing selected further exploratory activities in other indications with external partners.
Phase III trial AV001 with QUTENZA®: Recruitment completed
MPC-06-ID
In 2019, we have embarked on a partnership with Mesoblast to develop a highly innovative mesenchymal precursor cell therapy for patients with chronic low back pain
associated with degenerative disc disease who have not found effective relief from available treatment options.
Early in 2021, Mesoblast published results from the ongoing Phase III trial MSBDR003 that was carried out in the US and Australia. The trial provided a number of important findings, including a significant and long-lasting treatment effect on pain relief. However, it did not achieve its primary outcome measure between the treatment groups.
After having analysed the data obtained through this trial, Mesoblast anticipates to conduct another confirmatory trial in the US, and to design this trial to support potential parallel product approvals in both the US and Europe.
Glucocorticoid Receptor Modulator (GRM)
Glucocorticoids, such as prednisolone, are known to be highly effective anti-inflammatory drugs. However, they come with several significant side effects, including reduced bone formation that may lead to osteoporosis, as well as increased glucose levels, which raises the risk of diabetes. These side effects are a strong limitation for the long-term use of glucocorticoids, despite their efficacy. With our GRM programme, we are pursuing the development of clinical candidates for oral treatment with broad anti- inflammatory efficacy and the potential of significantly reduced side effects when compared to available glucocorticoid-based therapies. The most advanced compound within our GRM programme is currently in Phase I clinical development and has shown positive results in its First in Human trial.
Nociceptin/Orphanin FQ receptor Peptide agonist (NOP)
Although several different treatment options are available, many patients with neuropathic pain still suffer from treatment non-response or insufficient pain relief. With our NOP programme, we are pursuing the development of a selective, peripherally-restricted oral treatment with a unique mechanism of action for chronic pain that offers a more favourable safety profile than current therapies. This programme is based on our many years of intense and ground-breaking research in the field of NOP receptors, and opens up a unique opportunity for a transformative first-in-class treatment.
First participants enrolled in first-in-human Phase I clinical trial with nociceptin (NOP) receptor agonist
Read more in our press release