Opelconazole

The decision was based on an interim analysis of the phase 3 Opera-T study.\n The show is over for Pulmocide’s phase 3 trial after patients who received its investigational inhaled antifungal had lower response rates and a higher number of deaths than those in the control arm.The decision was based on an interim analysis of the phase 3 Opera-T study, conducted after around half of the patients had been randomized and had reached day 84 of treatment, Pulmocide said in a Jan. 7 release. This analysis was done to recalculate the trial’s sample size.The London-based biotech was trialing opelconazole, a nebulized azole compound, in treatment-resistant invasive pulmonary aspergillosis. Aspergillosis is an infection of the lungs caused by mold in the genus Aspergillus, and is most common in patients with compromised immune systems, like those who have received lung transplants.In the most severe invasive form of the infection, the fungus has spread from the lungs to other parts of the body, such as the skin, brain, heart or kidneys.Opera-T was set to enroll around 123 patients and compare twice-daily opelconazole and placebo in patients also being treated with other antifungals. Enrolled patients were “severely immunocompromised,” Pulmocide said in the release, “most of whom had a life-threatening underlying hematological malignancy or were recipients of a stem cell or solid organ transplant.”Based on the analysis, most of the patients’ disease had not responded to at least two earlier antifungal treatments before joining the study, the British biotech added. “The analysis showed a numerically lower favorable response rate and a numerically higher mortality rate in the opelconazole arm compared to the control arm,” the company explained.None of the undisclosed number of patient deaths were attributed to opelconazole itself. The early termination has put the entire opelconazole program on the rocks. Pulmocide is now performing “a thorough review of the unblinded data” from Opera-T to determine the program’s future, according to the release.Pulmocide previously reported in April 2024 that opelconazole, its sole drug candidate, had appeared to eradicate Aspergillus from six patients infected with the fungus in the phase 2 Opera-S trial, with substantially lower levels of treatment discontinuation compared to control. However, the primary goal of Opera-S was the prevention of aspergillosis in lung transplant patients, not invasive fungal infections like in Opera-T.The fungal-minded biotech pulled in a $147.5 million series C spread across 2021 and 2022 to fund development of opelconazole. At the time, the company was riding the high of “very remarkable responses” seen in case studies of patients given the antifungal candidate through the U.K.’s compassionate use program, CEO Daniel Burgess told Fierce back in May 2021.

We met with management teams from several private biotech companies during last week's JPM Healthcare Conference. We highlight some of the more interesting names in this note (in alphabetical order): AltruBio, ARTHEx Biotech, Congruence Therapeutics, Ensho Therapeutics, Evox Therapeutics, Octant Bio, Odyssey Therapeutics, Paratus Sciences, Pulmocide, Sorriso Pharmaceuticals, and Stealth Biotherapeutics. •AltruBio: AltruBio is a company in the I&I space developing a subQ antibody targeting PSGL-1, a unique immune checkpoint target that regulates T-cell homeostasis. Lead asset ALTB-268 is designed to downregulate chronically activated T-cells and restore immune system homeostasis, and the anti-PSGL-1 mechanism could have broader potential across T-cell-mediated diseases. ALTB-268 is currently in development for UC (Ph2a) and Psoriatic Arthritis (Ph2a planned). •ARTHEx Biotech: ARTHEx is a company in the rare neuromuscular space developing a potentially differentiated DM1 therapy. Lead asset ATX-01 is an ASO targeting miR-23b, designed to modulate the important MBNL and DMPK protein targets. ATX-01 is currently in a Ph1/2 trial in DM1, and initial results could be available in 2H25. Additional ATX-01 indications include DM2 (preclinical) and congenital DM1 (preclinical). •Congruence Therapeutics: Congruence is utilizing its proprietary Revenir™ computational discovery platform to identify novel binding sites for small molecule correctors of difficult-to-target genetically-validated, pathogenic proteins. The preclinical pipeline includes programs in Genetic Obesity (MC4R target), GBA Parkinson's (GCase target) and Alpha1-Antitrypsin Deficiency (AAT target). •Ensho Therapeutics: Ensho is a company in the I&I space developing a potential best-in-class oral α4β7 inhibitor for UC/Crohn's that could be a competitor to TAK's Entyvio (anti-α4β7 antibody) and LLY's ORF-057 (oral α4β7 inhibitor, MORF acquisition for $3.2B in 2024). Lead asset NSHO-101 is set to start a Ph2 trial in UC in 1H25 and a Ph2 trial in Crohn's could follow in 2027. •Evox Therapeutics: Evox is developing proprietary exosome therapeutics to treat rare genetic disorders. The preclinical pipeline includes Spinocerebellar Ataxia Type 2 (SCA2) and Amyotrophic Lateral Sclerosis (ALS) programs. •Octant Bio: Octant is leveraging its proprietary Navigator platform to design oral small molecule correctors of difficult-to-target proteins. Preclinical programs include Retinitis Pigmentosa (RHO-adRP), Fabry Disease, and p53 cancers. •Odyssey Therapeutics: Odyssey is a company in the I&I space and is developing a suite of novel small-molecule and protein therapeutics. Lead program OD-07656 is an oral RIPK2 inhibitor in development for UC/Crohn's. The company filed an S-1 on 1/17/25. •Paratus Sciences: Paratus is a preclinical-stage company in the I&I and cardiometabolic space. The company is leveraging its proprietary bat genomics/transcriptomics library to identify novel, human-relevant therapeutic targets. •Pulmocide: Pulmocide is developing nebulized opelconazole for the treatment of refractory invasive fungal infections in the lung. Opelconozole, a novel azole antifungal, has shown positive efficacy and safety results in Ph2 and compassionate use trials across multiple indications. A Ph3 superiority trial vs SoC in invasive pulmonary aspergillosis is ongoing, with data expected in 2026. •Sorriso Pharmaceuticals: Sorrisso is a clinical-stage company in the I&I space, developing novel, oral "vorabodies" designed towards multi-targeting for higher efficacy and lower systemic safety risk. Lead asset SOR102 is a gut-directed dual-targeting TNFα/IL-23 oral antibody in Ph2a development in UC/Crohn's. •Stealth Biotherapeutics: Stealth is a clinical-stage company developing mitochondria-targeted therapies. Lead asset elamipretide is under FDA review for treating the ultra-rare cardioskeletal disease Barth Syndrome (4/29/25 PDUFA). A Ph1 trial for pipeline asset bevemipretide (dry AMD) could start in 1H25. View Full Report with Disclaimers Find More top More News