With the drug already under review by the U.S. FDA for dry eye disease (DED), Aldeyra Therapeutics Inc.’s reproxalap hit statistical significance in the second phase III study for allergic conjunctivitis (AC), and the firm is mulling what CEO Todd Brady called the “high-class problem” of how to best commercialize the product. A partner may help puzzle that out.
A higher rate than expected of serious bleeding, including five deaths, in ovarian cancer patients receiving upifitamab rilsodotin (UpRi) led the U.S. FDA to place a partial clinical hold on two trials, possibly delaying Mersana Therapeutics Inc.’s BLA filing targeted for later this year.
Patients with nonalcoholic fatty liver disease (NAFLD) and type 2 diabetes had a 44% reduction in liver fat after receiving Inventiva Pharma SA’s PPAR agonist, lanifibranor, in a phase II trial, findings that caused the Daix, France-based company’s stock to soar.
The death of a patient caused Seattle Children's to pause its phase I study of relapsed/refractory pediatric acute myeloid leukemia (AML). It’s another hitch in the world of ground-breaking CAR T treatments that can have a serious downside.
Since late 2006, when hiked levels of aldosterone and increases in blood pressure foiled Pfizer Inc.’s torcetrapib, researchers have been wary of cholesteryl ester transfer protein (CETP) inhibitors. But not all researchers. Among the still-hopeful is Newamsterdam Pharma NV, which recently rolled out positive phase II data with obicetrapib, and the company is forging ahead despite other CETPs that have not fared well in late-stage testing after performing nicely in phase II, including candidates from Merck & Co. Inc. and Eli Lilly and Co.
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.
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).
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