Disc Medicine Inc.’s positive phase II data from an ongoing, open-label trial called Beacon with oral bitopertin in erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) whetted investor appetite for the results of the other mid-stage Disc experiment known as Aurora with the compound, due early next year.
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.
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.
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.
In the flurry of presentations on early detection of cancer at the 2023 American Society of Clinical Oncology Annual Meeting, Grail LLC stood out for the number of sessions and the strength of its results. In a real-world study presented, Grail’s Galleri multi-cancer early detection (MCED) test cancer signal origin (CSO) demonstrated accuracy of 91%.
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).
Additional data from a phase II trial of S1P modulator icanbelimod (CBP-307) reinforced earlier positive results in ulcerative colitis (UC), potentially putting Connect Biopharma Holdings Ltd. in a better negotiating position as it seeks a partner to take the program forward, while in-house activities remain focused on an early 2024 regulatory filing for atopic dermatitis candidate CBP-201.
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