Edasalonexent

Patients receiving STAR-0215 once or twice over six months saw monthly HAE attack rates decline by 90% to 96%, according to Astria. Astria Therapeutics is planning a phase 3 launch for its star monoclonal antibody after early data demonstrated a favorable safety profile and reduction in attack rates of a rare immune disorder. The initial phase 1b/2 results, shared Monday morning, assessed Astria’s STAR-0215, a monoclonal antibody inhibitor of plasma kallikrein, among 16 patients with hereditary angioedema (HAE). The trial, dubbed ALPHA-STAR, is measuring the number of adverse events reported, along with other secondary endpoints like duration and severity of HAE attack. The initial data found STAR-0215 to be generally well tolerated with no serious treatment-emergent adverse events (TEAEs) occurring. No discontinuations were recorded and the two TEAEs reported were both mild: one case of dizziness and one rash at injection site.     The study includes three dosing cohorts. Participants in the first arm received one 450-mg dose of STAR-0215, while participants in the second group received a 600-mg dose followed by a 300-mg dose three months later, and patients in the final cohort were given a 600-mg dose and then another 600-mg dose a month later. When evaluating monthly HAE attack rate, the first cohort saw a 92% reduction at a six month follow-up; group two recorded a 96% reduction at 84 days; and cohort three demonstrated a 90% decrease at Day 28. Astria said these findings support chronic dosing two or four times per year. The phase 1b/2 trial has a primary completion date of September 2025, according to ClinicalTrials.gov. “The initial results of the ALPHA-STAR trial represent a very exciting step forward in the HAE treatment landscape,” Marcus Maurer, M.D., executive director of the Institute of Allergology at the Universitatsmedizin Berlin, said in a March 25 release. “STAR-0215 has the potential to help patients manage their disease with a mechanism and modality that they trust, but with a substantially improved dosing regimen and the ability to administer without pain,” Maurer added. “Based on this profile, STAR-0215 has the potential to normalize the lives of people living with HAE.” With the early results in hand, Astria intends to launch STAR-0215 into phase 3 development by the first quarter of 2025, pending discussions with regulatory agencies.  To get STAR-0215 to market as fast as possible, Astria wants to focus the potential late-stage program on dosing every three months, immediately followed by a second trial to support label expansion with dosing every six months. Astrai has its origins in Catabasis Pharmaceuticals. When Catabasis' doomed Duchenne muscular dystrophy drug edasalonexent flamed out in phase 3, the company rebranded in 2021 and shifted focus to STAR-0215.

A few months after finally admitting defeat on their failed Duchenne MD program, Catabasis Pharmaceuticals is heading in a new direction with the purchase of Quellis Biosciences and their lead antibody for hereditary angioedema. Catabasis shared the news on Friday that they’re buying out Quellis, a rare disease-focused biotech that was unveiled over a year ago by Chris Garabedian and Perceptive Advisors’ PXV Fund. With it, Catabasis is snapping up QLS-215, a monoclonal antibody inhibitor of plasma kallikrein. The candidate is currently in preclinical development — but won’t be for long, according to Catabasis. In addition to the buyout, the Boston-based biotech closed a $110 million private placement to fund the completion of IND-enabling, Phase Ia and Phase Ib/II studies. Catabasis and Quellis are keeping the terms of their deal under wraps. But after the acquisition and private placement, Catabasis expects to have a cash runway through 2023. The company plans on filing an IND application for QLS-215 in the first half of 2022, and reading out initial Phase I results by the end of 2022. If all goes well, they hope to launch a Phase Ib/II trial in 2023, with initial results by the end of that year. Back in October, Catabasis acknowledged a final failure for its Duchenne MD drug edasalonexent. Three years earlier, CEO Jill Milne and the crew insisted they found reason for hope in data from a failed study, and that it justified a try in Phase III. After the primary, change in baseline on the North Star Ambulatory Assessment, and the secondary on timed function tests both came up short of statistical significance, Catabasis killed the effort — leaving them with one other drug in the pipeline, a preclinical program on autophagy. Drug hunters and developers working in neurological diseases will now have a new place to turn to for insights. The Gladstone-UCSF Center for Neurovascular Brain Immunology is launching with a $2.5 million donation and neuroimmunologist Katerina Akassoglou as the founding director. “Historically, neurological diseases have been classified as being only degenerative, inflammatory, or vascular,” she said in a statement . “But we now know, given recent insights from clinical research and the failure of many clinical trials, that this classification cannot explain how diseases start and progress, nor has it been able to identify the best potential drug candidates.” By integrating basic and clinical research on vascular biology, immunology and neuroscience, scientists at the new center aim to shed light on overlapping mechanisms behind diseases like multiple sclerosis, Alzheimer’s disease and traumatic brain injury — and have implications extending as far as neonatal brain injury, epilepsy, infectious diseases and psychiatric disorders. Add it all together, Akassoglou said, and their approaches in imaging and drug discovery may just “transform biomedicine.” To uncover the role of vascular abnormalities in the brain, they will bring together basic and clinical experts and develop new molecular diagnostics, while testing new therapies and biomarkers.