After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding. Presented at the American Society of Hematology (ASH) annual meeting on Dec. 10, data showed 24-month results reinforced the safety of treatment, with no serious treatment-related adverse effects.
Affimed NV said the latest data with its lead innate cell engager (ICE) known as AFM-13, disclosed at the American Society of Hematology (ASH) meeting, have caused the focus to shift from monotherapy to combination regimens.
Mainstay Medical Holdings plc released data from a one-year, real-world, study of patients implanted with a neurostimulation device to treat chronic lower back pain.
MEI Pharma Inc. has discontinued the global development of its phosphoinositide 3-kinase (PI3K) delta inhibitor, zandelisib, outside Japan. “In light of FDA’s guidance, we no longer believe clinical development can be completed within a time period that would support further investment, or with sufficient certainty of the regulatory requirements to justify continued global development efforts,” said Daniel Gold, MEI’s president and CEO.
When a drug prevents bacteria from synthesizing their own folate, an essential compound for their survival, they take it directly from the host. This antibiotic resistance mechanism had not been detected until now because bacteria behave differently in the laboratory than they do in vivo during an infection.
At the ESMO Immuno-Oncology Congress 2022, Compugen Inc. shared phase I data for COM-701 in dual and triple combination with Bristol Meyers Squibb Co.’s Opdivo (nivolumab) and with or without BMS-986207. Compugen’s candidate demonstrated preliminary durable antitumor activity and immune activation in patients with platinum resistant ovarian cancer with a favorable safety and toxicity profile.
Top-line primary data from week 48 of Eiger Biopharmaceuticals Inc.'s phase III D-LIVR study of lonafarnib, a prenylation inhibitor for treating chronic hepatitis delta virus, also known as hepatitis D, hit statistical significance vs. a placebo in the composite primary endpoint.
Investigators at Relmada Therapeutics Inc. believe the same problem that plagued an earlier phase III effort called Reliance-3 – an “implausibly” high placebo response at certain sites – also foiled the latest phase III study (conducted at overlapping sites) known as Reliance-1, testing REL-1017 (esmethadone), meant as an adjunctive treatment for major depressive disorder.
Pancreatic cancer is the deadliest of cancers with just one in nine patients surviving five years after diagnosis. The low rate of survival largely results from the late stage at which the cancer is first detected, as 65% patients are not diagnosed until the disease has metastasized. Bluestar Genomics Inc. hopes it has developed an assay that can detect the cancer much sooner, allowing patients and their physicians to get ahead of the malignancy well before it spreads.
Replimune Group Inc. has found consistent treatment efficacy in its pivotal phase II Ignyte melanoma study. The interim data strongly bolstered the stock (NASDAQ:REPL), which ended Dec. 7 with a 22% increase to $23.09. The new results for the combination treatment show what Replimune called clinically meaningful durable activity across the range of anti-PD1 failed cutaneous melanoma presentations, including in patients with moderate to high tumor burden, with 85% of responses ongoing.
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