Biopharma Spotlight: RedHill’s Neuroblastoma Drug; Versant and Novartis Launch Borealis; Tonix and Bilthoven Partner on Mpox Vaccine
Shine a spotlight on today’s top life science and biopharma news stories
The U.S. FDA granted RedHill Biopharma (Tel Aviv, Israel and Raleigh, NC) Orphan Drug Designation to opaganib for treatment of neuroblastoma, a form of childhood cancer. Neuroblastoma is rare, but the most common malignancy in infants, with a median age of diagnosis of 17 months. In the U.S., it makes up about 10% of all childhood cancer cases and 15% of pediatric cancer-related deaths. Earlier, the FDA granted the same drug orphan drug designation for cholangiocarcinoma, a bile duct cancer (CCA). Orphan Drug Designation offers seven-year marketing exclusivity if it is approved for the indication and may also bring accelerated development and review times, possible grant funding and tax credits.
“Opaganib has broad oncology potential with promising preliminary clinical data in solid tumor cancers such as prostate cancer and CCA, and data from a range of U.S. government supported and Apogee conducted preclinical studies in various indications, including radioprotection, and also in combination with RedHill’s RHB-107,” said Dr. Mark Levitt, CSO at RedHill. “We also see such utility extending to the potential for opaganib to have a sensitizing effect in hormone receptor pathway inhibition therapy, which the Company expects to test in a planned externally funded Phase II study.”
Opaganib is a first-in-class, oral sphingosine kinase-2 (SPHK2) selective inhibitor. It has anticancer, anti-inflammatory and antiviral activity and targets multiple possible diseases, including obesity-related syndromes, prostate cancer, CCA, gastrointestinal acute radiation syndrome, Sulfur Mustard exposure, COVID-19, Ebola and other viruses.
Versant and Novartis Launch Borealis Biosciences to Focus on Kidney Disease
Vancouver-based Borealis Biosciences emerged from stealth mode with $150 million in combined Series A financing from investor Versant Ventures and Novartis AG. It also has strategic research collaboration funding from Novartis. Borealis was created on the theory that RNA therapeutics can be used to treat kidney diseases. It is built on the success of Chinook Therapeutics, a kidney disease company founded by Versant in 2019 and acquired by Novartis in 2023 for up to $3.5 billion. Borealis was founded around key members of Chinook’s Vancouver-based research team. Novartis committed up to $100 million in upfront plus near-term research funding. Novartis has the option to acquire two future development-ready programs from Borealis, with Borealis eligible for up to $750 million in various milestones.
Tonix and Bilhoven Bio Partner on Tonix’s Mpox Vaccine
Tonix Pharmaceuticals (Chatham, NJ) inked a collaboration deal with Bilthoven Biologicals (BBio), part of the world’s largest vaccine manufacturer, the Cyrus Poonawalla Group, which includes The Serum Institute of India. They will work to advance TNX-801, Tonix’s mpox vaccine candidate. TNX-801 (recombinant horsepox virus) is a live replicating, attenuated virus based on horsepox. It is in preclinical development to prevent mpox (formerly called monkeypox) and smallpox.
The technology underpinning TNX-801 is a viral vector platform from which recombinant version can be developed against other infectious diseases. BBio was chosen by the European Union for its “ever-warm” vaccine manufacturing companies for Europe’s pandemic preparedness program.
“The recent mpox outbreak exemplifies precisely why we built the pandemic preparedness facility at BBio,” said Jurgen Kwik, CEO of Bilthoven. “The establishment of the ‘ever-warm’ facility for pandemic preparedness underscores the critical importance of readiness in the face of global health emergencies, such as mpox. This collaboration encapsulates the essential role of the facility in bolstering pandemic preparedness and response capabilities.”