Paratek Pharmaceuticals has announced positive top-line data from a Phase IIb study of oral omadacycline in adult patients with nontuberculous mycobacteria pulmonary disease (NTM-PD) caused by mycobacterium abscessus complex (MABc). The study demonstrated that omadacycline was generally safe and well-tolerated, with higher response rates compared to placebo, marking it as the first randomized, placebo-controlled trial among MABc patients.
Omadacycline is currently marketed in the US and China under the brand name Nuzyra for treating community-acquired bacterial pneumonia and acute bacterial skin infections. It has received fast-track designation from the FDA and orphan drug designation from both the FDA and EMA for NTM-PD.
Efficacy and Safety Results
The Phase IIb study evaluated the response on the NTM Symptom Assessment Scale at day 84 as the primary efficacy endpoint. Results showed that 34.1% of patients receiving omadacycline experienced an improvement of at least 50% in their NTM symptoms from baseline, without any worsening of baseline symptoms. This was compared to 20% and 12% of patients in the placebo group, respectively. Furthermore, 56.4% of patients treated with omadacycline had negative sputum cultures for MABc at day 84, compared to 29.2% of those receiving placebo.
The safety profile of oral omadacycline was consistent with its established profile. The most common adverse events reported were gastrointestinal symptoms. Treatment discontinuation due to adverse events occurred in 9.8% of patients administered omadacycline. No deaths were reported during the study.
Addressing Unmet Needs in NTM Treatment
The study results highlight omadacycline’s potential in the NTM market, where many currently used drugs are prescribed off-label. Key opinion leaders (KOLs) have expressed optimism about omadacycline's development for NTM-PD, citing the lack of novel and effective therapies. Some US KOLs have reported using omadacycline off-label to treat NTM-PD caused by MABc and M. avium complex.
Market Outlook
GlobalData estimates that oral omadacycline for NTM-PD treatment is projected to launch in the US in 2029 and in the EU in 2030. High-prescribing physicians surveyed in the US anticipate that approximately one-quarter of NTM patients will be treated with omadacycline by 2033.
The clinical success of omadacycline underscores its potential to address a critical unmet need in the NTM market and improve treatment outcomes for NTM-PD patients.
