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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)

Osteoarthritis (OA) is a progressive condition that currently cannot be cured. The inflamed, swollen and painful joints lead to limitation on mobility of the affected patients and may impact their quality of life significantly. Millions of OA patients currently receive intra-articular corticosteroids or need to undergo knee replacement surgery as last remaining treatment option.

RTX (resiniferatoxin) is an investigational medicine for the intra-articular treatment of pain associated with OA of the knee. The highly potent TRPV1 agonist currently concludes Phase II of clinical development. Its mechanism of action is well validated and initial data shows a long-lasting and significant analgesic effect and functional improvements compared to placebo (saline injection), as well as a favourable safety profile.

We are currently preparing two pivotal Phase III studies to investigate the efficacy, safety and tolerability of RTX in patients with pain associated with OA of the knee. The studies will start in 2021 and they are part of a global development programme aimed at meeting the requirements for approval in the EU, the US, Japan and China.

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.

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.

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 peripheral neuropathic 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. The most advanced compound within our NOP programme entered clinical development in late 2020.

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 entered clinical development in early 2021.