NEW YORK – The US Food and Drug Administration this week granted orphan drug designation to Fore Biotherapeutics' BRAF inhibitor, FORE8394, for the treatment of primary brain and central nervous system (CNS) malignancies. The drug is currently being studied in a Phase II trial in advanced unresectable solid or primary CNS tumors harboring BRAF alterations. An orphan drug designation is granted to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval. Last year, FORE8394 was also granted fast track designation by the FDA.
RayzeBio this week said it completed enrollment in the Phase Ib part of its ongoing ACTION-1 trial of radiopharmaceutical RYZ101 in patients with SSTR-positive neuroendocrine tumors of the gastrointestinal tract and pancreas who have progressed on a lutetium-177-labeled somatostatin analog. All patients passed the eight-week period for evaluating dose-limiting toxicities and none were observed. The company plans to begin a Phase III study after establishing a recommended dose in the Phase Ib portion of the trial.
Separately, the firm also announced that it has homed in on a targeted radiopharmaceutical that it will advance to clinical trials in liver cancer patients. The firm discovered the agent, which targets Glypican-3 (GPC3) on the cell surface, through a research collaboration with PeptiDream. RayzeBio is now advancing the candidate into investigational new drug application-enabling studies.
Gilead Sciences said this week that its CD19-directed CAR T-cell therapy Yescarta (axicabtagene ciloleucel) resulted in an overall survival benefit versus historical standard-of-care therapy for relapsed or refractory large B-cell lymphoma patients in the Phase III ZUMA-7 study. Yescarta was already approved for second-line treatment of LBCL in April of last year based on the study's primary endpoint, event-free survival. Now, the overall survival endpoint has reached maturity and, according to Gilead, demonstrated a significant benefit for these patients. The drugmaker plans to present full data at an upcoming medical meeting.
Atossa Therapeutics said this week that its selective estrogen receptor modulator, (Z)-endoxifen, will be evaluated as a neoadjuvant treatment in a study arm of the ongoing I-SPY 2 clinical trial. The therapy will be studied under I-SPY's Endocrine Optimization Pilot Protocol, which is enrolling patients with newly diagnosed estrogen receptor-positive invasive breast cancer whose tumors are predicted to be sensitive to endocrine therapy and who may not see benefit from chemotherapy. About 20 patients will be enrolled in the (Z)-endoxifen study arm to be treated for up to 24 weeks prior to surgery.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Oncology News.