Nxera Pharma Reports Positive Phase 2 Data for Schizophrenia Candidate NBI-1117568

Nxera Pharma Co., Ltd. (Nxera), previously known as Sosei Group or Sosei Heptares, has announced promising Phase 2 results for NBI-1117568 (NBI-'568), an oral, muscarinic M4 selective receptor agonist developed under a collaboration with Neurocrine Biosciences Inc. (Neurocrine). This investigational drug aims to treat schizophrenia and is the first of its kind in clinical development for this purpose.

The Phase 2 clinical trial evaluated the efficacy, safety, and tolerability of a once-daily 20 mg dose of NBI-'568 in adults with schizophrenia. The study successfully met its primary endpoint, showing a statistically significant improvement in the Positive and Negative Syndrome Scale (PANSS) total score, with a 7.5-point reduction compared to placebo at week 6. The drug also showed an 18.2-point improvement from baseline PANSS total score, demonstrating its potential as an effective treatment option.

In addition to the primary outcome, NBI-'568 showed significant improvements in secondary endpoints, such as the Clinical Global Impression of Severity (CGI-S) scale and Marder Factor Score for both Positive and Negative Symptom Change. These results underscore the drug’s potential to address various facets of schizophrenia symptoms.

The study design was robust, involving multiple arms to assess different dosages. NBI-'568 was generally safe and well-tolerated across all doses tested. Treatment discontinuation due to adverse events was comparable between the drug and placebo groups. Common adverse events included somnolence, dizziness, and headache. Gastrointestinal issues like nausea and constipation were infrequent and similar to placebo. Cardiovascular events were minimal and not clinically significant, and there was no notable weight gain compared to placebo. Few extrapyramidal symptoms were reported.

Neurocrine plans to advance NBI-'568 into Phase 3 trials in early 2025, reflecting their commitment to addressing the unmet needs in schizophrenia treatment. The collaboration between Nxera and Neurocrine began in 2021, focusing on developing muscarinic receptor agonists for various neurological and neuropsychiatric disorders. This partnership has been fruitful, with Nxera receiving significant financial milestones and being eligible for up to $2.6 billion in development, regulatory, and commercial milestones, plus product royalties.

Nxera's EVP, Matt Barnes, highlighted the importance of these Phase 2 results, emphasizing that they validate Nxera’s NxWave™ discovery platform. The data suggests that NBI-1117568 could provide a once-daily oral therapeutic option for schizophrenia patients, offering a competitive and beneficial risk profile. The successful advancement of four development candidates from Nxera’s portfolio further demonstrates the effectiveness of their collaboration with Neurocrine.

Eiry Roberts, M.D., Chief Medical Officer at Neurocrine, reiterated the significant need for new schizophrenia treatments and expressed optimism about NBI-'568's potential as it progresses to Phase 3 trials.

In summary, NBI-1117568 has shown promising Phase 2 results, indicating its potential as a novel treatment for schizophrenia. With favorable efficacy, safety, and tolerability profiles, its development continues under the collaborative efforts of Nxera and Neurocrine, aiming to offer new hope for patients with schizophrenia.