Our development projects
Innovators at Grünenthal are pursuing a range of programmes across different modalities, targets and mechanisms of action to unlock better treatments for patients in need. Here are some highlights from our development pipeline.
Qutenza™: Reaching more patients in the US
Qutenza™ is a topical system that contains prescription-strength capsaicin. It is a non-opioid treatment that can provide prolonged pain relief for several months. Its most frequently reported adverse effects are usually transient, self-limiting, mild-to-moderate reactions on the application site.
In Europe, it is approved for treating peripheral neuropathic pain (PNP) in adults either alone or in combination with other medicinal products for the treatment of pain. In the US, Qutenza™ is approved for treating PNP associated with post-herpetic neuralgia and for treating pain associated with diabetic peripheral neuropathy (DPN) of the feet.
In 2020, the US Food and Drug Administration (FDA) approved Qutenza™ for the treatment of pain associated with DPN of the feet in adults. This marked a major milestone in our efforts to bring this treatment to more patients because painful DPN is a progressive and debilitating complication of diabetes that affected more than five million Americans in 2020. It is difficult to diagnose, treat and manage effectively.
Through the Phase III trial AV001, Grünenthal’s US affiliate Averitas Pharma investigated the efficacy, safety and tolerability of Qutenza™ in post-surgical neuropathic pain (PSNP). The trial included 409 patients from across Europe and the US who suffered from moderate to severe chronic PSNP and evaluated the reduction of average pain intensity over a total of 42 weeks. The primary endpoint was a reduction in average pain intensity at 12 weeks. Patients were randomised to receive Qutenza™ or a low-dose capsaicin control patch (0.04 percent).
The trial was designed to address differing regulatory expectations in major markets. In line with European Medicines Agency (EMA) guidance, one primary analysis assessed change in pain intensity over the 12-week period relative to baseline. In line with US Food and Drug Administration (FDA) guidance, another primary analysis assessed pain intensity at Week 12 relative to baseline.
Overall, AV001 further supported the well-established efficacy and safety profile of Qutenza™. The trial met the primary endpoint aligned with EMA guidance, demonstrating statistically significant pain relief over 12 weeks, with effects also observed over the full 42-week treatment period. The study did not meet the primary endpoint aligned with FDA guidance, as the difference versus control at Week 12 did not reach statistical significance.
Moving forward, the outcome of AV001 will support several regulatory submissions aimed at increasing Qutenza™'s availability and potential to reach more patients worldwide, including an update to the EU SmPC (Summary of Product Characteristics). A supplemental New Drug Application (sNDA) submission to the FDA will not be pursued. Publication of the AV001 data is planned in accordance with Averitas' and Grünenthal's standard publication processes, with timing currently anticipated for Q4 2026.
MPC-06-ID - Collaboration with Mesoblast on a cell therapy for chronic low back pain
We entered into a strategic partnership with Mesoblast in 2019, a world leader in cellular medicines, for the development of its investigational medicine rexlemestrocel-L (MPC-06-ID). The innovative cell therapy option is currently in clinical Phase III development for chronic low back pain (CLBP), associated with degenerative disc disease.
CLBP is a major contributory factor to the US opioid crisis, and rexlemestrocel-L has received Regenerative Medicine Advanced Therapy designation from the FDA for treatment of CLBP.
In 2021, the Phase III trial MSB-DR003 provided several important findings, including a significant and long-lasting treatment effect on pain relief. As a primary outcome measure has not yet been achieved, Mesoblast commenced MSB-DR004, a second randomised controlled Phase III trial to investigate MPC-06-ID in CLBP associated with degenerative disc disease. MSB-DR004 is expected to complete recruitment in Q2 2026 and focuses on achieving durable pain reduction with opioid-sparing activity in patients who receive a single intradiscal injection of rexlemestrocel-L + hyaluronic acid.
Tegacorat (GRM-01): Investigational treatment for Duchenne muscular dystrophy
Tegacorat is a non-steroidal selective glucocorticoid receptor agonist and modulator (SEGRAM). This Grünenthal proprietary asset is an oral investigational medicine for the treatment of Duchenne muscular dystrophy (DMD).
For families facing a DMD diagnosis, treatment options today are not curative and cannot prevent disease progression. Corticosteroids are the current standard of care and can slow down the progression of muscle deterioration, giving children more years of mobility and independence. However, they come with significant side effects including weight gain, behavioural changes, delayed puberty, increased risk of fractures, Cushingoid appearance and stunted growth.
As a result, there is an urgent need for safer and more effective treatment options. tegacorat has the potential to provide potent anti-inflammatory efficacy with reduced side effects compared to corticosteroids. Its safety profile could enable long-term treatment to address a major unmet medical need for patients with DMD that would make a meaningful positive difference to patients’ lives.
The clinical Phase I trials for tegacorat involved a total of 88 healthy participants and primarily aimed to characterise the safety and tolerability profile of tegacorat, while also confirming its pharmacokinetic characteristics.
Grünenthal aims to investigate tegacorat within a Phase II trial of boys between the ages of four and under seven who have DMD but have not taken corticosteroids. The trial will commence in 2026 at centres in the US and Europe.
NOP: Promising treatment for acute and chronic pain
Our proprietary nociceptin (NOP) receptor agonist is the culmination of many years of pioneering research in the field of NOP receptor agonists. Our molecule has a unique mechanism of action for treating acute and chronic pain and is hypothesized to deliver robust pain relief in a broad range of conditions without the side effects commonly associated with opioids. For this reason, it may provide a unique and transformative first-in-class therapy option for patients living with pain.
The NOP agonist is currently being studied in a phase I clinical trial in healthy participants. The trial aims to establish the safety and pharmacokinetic profile of the compound. Later in 2026, the molecule will enter a Phase II trial in post-surgical bunionectomy, a well-established setting to first test the efficacy and safety of our compound in acute pain.
NaV inhibitors: Creating the next generation of non-opioid pain medicines
One of Grünenthal’s most promising early research areas is our voltage-gated sodium channels (NaV) programme, where we are striving to create the next generation of non-opioid pain medicines. NaV channels can carry sodium ions into cells, resulting in an excitatory signal. If a channel's activity is modified so it can no longer carry sodium ions, it will also no longer be able to evoke excitatory signals. Of the family of nine NaV channels, we are particularly interested in those expressed in dorsal root ganglion neurones.
These specific channels play roles in triggering excitatory signals in nociceptive neurones which are felt as pain by the human brain. As well as recognising their key role in pain signalling, genetic and some clinical validation make them promising human pain targets. Manipulating these NaV channels in a way that suppresses or prevents their excitatory signalling could provide a significant analgesic effect across a range of chronic and acute pain conditions. Our lead candidate, a highly selective NaV 1.8 inhibitor, has entered clinical development in Q1 2026.