Promising early data continue to roll out for Intellia Therapeutics Inc.’s hereditary angioedema (HAE) candidate, NTLA-2002, with one of the earliest treated patients in the phase I study remaining attack-free for more than a year. But it was the systemic CRISPR candidate’s potential as a one-time treatment that generated the most discussion on the company’s call as investors tried to assess its potential advantage in a crowded HAE market.
For Jeff Galvin, the CEO and founder of newly launched Addimmune Inc., HIV is not a condition that’s in the rearview mirror. It needs a functional cure to save lives, make people healthier and save money that need not have been spent. People wonder why it’s worth bothering to cure HIV, Galvin told BioWorld, when they are taking their medications every day and they are feeling pretty close to normal. But it’s not close for Galvin, who noted that there are side effects from taking the pills that can cause headaches, fatigue, nausea and diarrhea.
Following strong phase I study data in malignant melanoma released a year ago, Ultimovacs ASA’s lead candidate, UV-1, stumbled in the phase II trial, which did not meet the primary endpoint of progression-free survival in second-line treatment of malignant pleural mesothelioma.
It has been a tough spring for Fibrogen Inc., which reported another phase III miss on June 7, this time for rare disease drug pamrevlumab to treat non-ambulatory patients with Duchenne muscular dystrophy (DMD).
Standing as one of the more prominent among incurable conditions, Parkinson’s disease (PD) still hasn’t met a medicine or surgical intervention that can slow or stop progression, despite efforts of many drug developers testing new strategies.
When combined with chemotherapy, the PD-1 inhibitor antibody toripalimab reduced the risk of death by 37% over chemotherapy alone when used first line in patients with recurrent or metastatic nasopharyngeal carcinoma, Coherus Biosciences Inc. revealed at this year’s American Society of Clinical Oncology meeting (ASCO).
Gracell Biotechnologies Inc. is initiating two clinical trials in China in refractory systemic lupus erythematosus for lead candidate GC-012F, a CD19/B-cell maturation antigen dual-targeting CAR T.
Impressive data continue to roll out for Day One Biopharmaceuticals Inc.’s tovorafenib in pediatric low-grade glioma, with results from 69 evaluable patients with relapsed or progressive disease showing a 67% overall response rate, as measured by Response Assessment for Neuro-Oncology High-Grade Glioma, in the phase II Firefly-1 trial.
Positive data from two studies treating early breast cancer with CDK4/6 inhibitors presented at the American Society of Clinical Oncology (ASCO) meeting in Chicago showed progress is helping patients live longer. The two treatments and companies, Novartis AG and Eli Lilly and Co., are nearly head-to-head competitors in the niche indication.
HER2-targeting bispecific antibody zanidatamab, in development by Jazz Pharmaceuticals plc and Zymeworks Inc., produced better antitumor responses than current standard of care when used as a second-line treatment for biliary tract cancer (BTC) in a pivotal phase IIb study, bringing it closer to becoming the first therapy to target HER2-expressing BTC.
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