A Week of Sharp Turns Across the PRISM Emerging Biotech Index

Long-term gene therapy data, a discontinued cancer program, a $110 million raise and a year-end readout defined the week for five index companies.

Companies across the PRISM Emerging Biotech Index reported news this week ranging from long-term clinical data and regulatory progress to a program shutdown and a nine-figure capital raise.

uniQure Releases 48-Month AMT-130 Data After BLA Submission

uniQure (NASDAQ: QURE) released new 48-month data for AMT-130, its investigational gene therapy for Huntington’s disease, weeks after submitting a Biologics License Application to the FDA.

The longer-term analysis continued to show slower disease progression in high-dose patients, although the 44% slowing on the composite Unified Huntington’s Disease Rating Scale did not reach statistical significance at 48 months. The company’s earlier 36-month analysis remains the primary basis of its BLA. The FDA had previously told uniQure that the three-year analysis could support an accelerated-approval application.

Foghorn and Lilly Stop FHD-909

Foghorn Therapeutics (NASDAQ: FHTX) and Eli Lilly decided not to advance FHD-909 after Phase 1 data did not show enough efficacy to justify expansion. The partners are also discontinuing their selective SMARCA2 degrader program and do not expect further activity under the collaboration. Foghorn is cutting approximately 40% of its workforce and concentrating resources on its wholly owned pipeline. The company expects the restructuring to extend its cash runway into the second half of 2029.

Capricor Presents 24-Month HOPE-3 Data Ahead of November PDUFA Date

Capricor Therapeutics (NASDAQ: CAPR) is presenting new 24-month analyses from the HOPE-3 program at the World Muscle Society Congress this week, extending the dataset for deramiocel in Duchenne muscular dystrophy. The analyses build on earlier Phase 3 results showing a 54% slowing in upper-limb function decline at 12 months. Capricor included the analyses in a recent major amendment to the drug’s BLA. With the FDA actively reviewing the application ahead of a November 22 PDUFA target date, the longer-term data are part of the evidence now before regulators.

Pyxis Raises $110 Million With Warrants Tied to MICVO Survival Data

Pyxis Oncology (NASDAQ: PYXS) closed a public offering generating approximately $110 million in upfront gross proceeds. Warrants included in the financing could bring total gross proceeds to as much as $282.6 million if fully exercised for cash. The warrants are tied to future overall survival data from MICVO, Pyxis’ antibody-drug conjugate for recurrent or metastatic head and neck cancer. The company expects that data in the first half of 2027. The structure ties additional funding directly to MICVO’s next major dataset.

Oncolytics Targets Interim Colorectal Update by Year-End

Oncolytics Biotech (NASDAQ: ONCY) has activated nearly all planned sites in Part A of REO 033, its randomized study of pelareorep in RAS-mutant, microsatellite-stable metastatic colorectal cancer, with multiple patients enrolled. The company expects to have enough data for an interim clinical update by year-end and has said it is aligned with the FDA on a potential accelerated-approval path based on objective response rate. Oncolytics also announced a peer-reviewed publication this week showing that pelareorep altered the tumor environment in liver cancer in ways that could make tumors more responsive to immunotherapy.

Capricor’s November 22 PDUFA date is the next scheduled regulatory decision among the companies covered this week.