Corticosteroids are a standard first-line treatment for Duchenne muscular dystrophy, but these drugs have many side effects. FDA approval of Italfarmaco’s Duvyzat is the first nonsteroidal drug to pass the agency’s regulatory bar for treating this rare disease.
Contineum Therapeutics, which emerged from a Verant Ventures startup creation engine, has filed paperwork for an IPO. The biotech’s clinical-stage pipeline spans multiple sclerosis, idiopathic pulmonary fibrosis, and depression.
Despite achieving its Phase 3 study's main goal, the results for Point Biopharma's therapy are short of what Novartis radiopharmaceutical Pluvicto achieved in a similarly designed clinical trial. Nevertheless, the data readout should clear the way for Eli Lilly to complete its $1.4 billion acquisition of Point.
Inato recently raised $20 million in Series A2 funding for its tech platform, which enables access to inclusive clinical trials. The two-way platform brings exposure to community-based providers who often are overlooked by Big Pharma for clinical trial sites by allowing providers to apply for clinical trials in which they're interested.
Marburg virus has no FDA-approved vaccines or therapies, but a vaccine candidate from the National Institutes of Health now has encouraging results from a U.S. clinical trial. Next steps include tests in more countries including Ghana, where a Marburg outbreak was declared last summer.
Crescendo Health recently launched with a $3.4 million initial investment. The startup provides clinical researchers with software tools that make it easier to gather study participants' longitudinal health data.
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Jazz Pharmaceuticals is paying Zymeworks $50 million up front for rights to develop zanidatamab, a bispecific antibody cancer currently in two pivotal clinical trials. It’s the second bispecific antibody pact of the week, following Gilead Sciences’ deal to secure an option on a MacroGenics drug in early clinical development.
Bluebird Bio gene therapy Skysona has won FDA approval, making it the first FDA-approved therapy for cerebral adrenoleukodystrophy, an ultra-rare and fatal neurological disorder. Skysona’s regulatory green light follows the August approval of Zynteglo, a Bluebird gene therapy for a rare blood disorder.
Akero Therapeutics’ experimental NASH drug, efruxifermin, met the main and secondary goals of Phase 2b clinical trial. The biotech will still need to show that the drug’s efficacy can hold up in a pivotal study, but the results so far are a positive sign for the NASH field, which has been marred by clinical trial failures.
Decentralized and hybrid clinical trials have a faster enrollment rate and a higher retention rate than centralized models, research shows.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
A small Memorial Sloan Kettering Cancer Center study found that all rectal cancer patients treated with GSK drug Jemperli showed a complete response at six months—there were no detectable signs of the cancer. These early data presented at ASCO suggest that rectal cancer with a particular genetic signature might be treatable with this immunotherapy in lieu of the standard regimen of chemotherapy, radiation, and surgery.
A Rocket Pharmaceuticals gene therapy for the rare immune disorder leukocyte adhesion deficiency-I has met the survival goals of a pivotal study, paving the way for regulatory submissions that the company expects to file in early 2023.
An antiviral that Enanta Pharmaceuticals is developing for respiratory syncytial virus infection failed to beat a placebo in its first Phase 2 test. But the biotech notes that these study participants weren’t very sick and may have recovered on their own, so it’s now pinning its hopes on ongoing studies in higher-risk groups, such as transplant patients.
The FDA placed a partial hold on a Curis drug in development for treating leukemia after a patient treated in a clinical trial died. The biotech company said the patient experienced a complication that is a known risk of the small molecule drug.
The FDA approved Pluvicto, a Novartis radiopharmaceutical, as a new treatment option for advanced prostate cancer. Novartis added the therapy via its $2.1 billion acquisition of Endocyte four years ago.
