Goal
Clinical trials offer opportunity to patients, healthcare providers, and those in biopharma. This is a large and diverse group of people, which have different amounts of time and scientific knowledge. Thus, brief summaries of clinical trial data in terms that are understandable to this broad audience would be highly impactful. Here, I will write brief summaries on recently published clinical trial data.
Posted: 9/9/24
Arrowhead Pharmaceuticals publishes Phase III results from the PALISADE study
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) published results from a Phase III clinical trial studying the efficacy and safety of plozasiran (ARO-APOC3), an RNAi interference agent, in patients with Familial Chylomicronemia Syndrome (FCS).
The primary endpoint of the study was percent change in fasting triglyceride levels, which are high in FCS patients and can cause pancreatitis. Patients treated with 25 mg plozasiran and those treated with 50 mg exhibited an 80% and 78% decrease in triglyceride levels, respectively. Incidence of acute pancreatitis was also lower in the plozasiran groups compared to the placebo group (OR: 0.17; 95% CI: 0.03-0.94; P = 0.03). Thus far, plozasiran appears to be an effective treatment in FCS patients.
Plozasiran was quite safe, as adverse effects (AEs) occurred at a similar rate between groups. Notably, severe and serious AEs actually occurred more in the placebo group. Hyperglycemia was observed in prediabetic and diabetic patients treated with plozarisan.
According to a press release, Arrowhead plans to file a New Drug Application with by the end of 2024, which would provide the first approved treatment for FCS in the US.
Posted: 9/3/24
Daiichi Sankyo and AstraZeneca publish Phase II results of the DESTINY-CRCO2 Trial
Daiichi Sankyo (Tokyo: 4568) and AstraZeneca (NASDAQ: AZN) published data from Phase II of the DESTINY-CRCO2 trial in The Lancet Oncology, testing the safety and efficacy of Trastuzumab deruxtecan (Enhertu) in HER2-positive colorectal cancer patients.
82 patients were treated with 5.4 mg/kg, while 40 patients were treated with 6.4 mg/kg. The lower dose yielded an objective response rate (ORR) of 37.8%, while the higher dose yielded an ORR of 27.8%. Grade 3 adverse effects were also lower in patients given the 5.4 mg/kg dose, and these adverse effects were relatively manageable.
In April, Daiichi Sankyo and AstraZeneca announced that Enhertu is the first drug to receive tumor-agnostic approval of a HER2-targeted therapy by the FDA. This comes after three successful trials where Enhertu showed promising efficacy and safety profiles across a wide-range of patients with various types of HER2-positive solid tumors (see DESTINY-PanTumor02 and DESTINY-Lung01).
Posted: 8/26/24
Ono Pharmaceutical Co. releases an update on the MOTION trial
Deciphera Pharmaceuticals (NASDAQ: DCPH), a subsidiary of Ono Pharmaceutical Co (OTCMKTS: OPHLY), recently published Phase I/II (NCT03069469) and Phase III (NCT05059262) data evaluating the dosage, safety, and efficacy of vimseltinib for treating tenosynovial giant cell tumor (TGCT) that is not amenable to surgery.
In the Phase I/II study, dosage was determined to be 30 mg twice weekly without a loading dose for a median duration of 25.1 months. Objective response rate (ORR) was 72% and treatment was well tolerated.
In the Phase III study, the ORR was 40% (33/83) in the vimseltinib group and 0% (0/40) in the placebo group and treatment was well tolerated, similar to the Phase I results.
The NDA for Vimseltinib was accepted by the FDA for priority review and PDUFA target date was set for February 17, 2025. Vimseltinib was already approved for marketing in Europe and has initiated the EMA’s centralized review process, per an Ono Pharmaceutical news release.
Posted: 8/19/24
Syndax releases phase II AUGMENT-101 data
Syndax (NASDAQ: SNDX) published phase II clinical trial data (AUGMENT-101, NCT04065399) assessing the safety and efficacy of SNDX-5613 (revumenib) in relapsed and refractory KMT2a-rearranged (R/R KMT2Ar) acute leukemia patients.
The study reported a high objective response (63.2) rate, and a remission rate that is significantly higher than historical remission rates in patients with this type of leukemia (22.8% compared to 10%).
Treatment was well tolerated, as most adverse effects were managed by dose adjustment or treatment with corticosteroids and hydroxyurea.
Syndax reported that an NDA filing is currently under review by the RTOR program with a PDUFA date set for December 26, 2024.
Posted: 8/12/24
Updated efficacy and safety of LOXO-292 in thyroid cancer patients
Eli Lilly and Co. (NYSE: LLY) published updated efficacy and safety data in a phase I/II clinical trial on LOXO-292 (selpercatinib) treatment of 324 patients with RET-mutant medullary thyroid cancer (MTC) and 66 patients with RET fusion-positive thyroid cancer (TC).
The study includes data from 228 additional patients, sustainable objective response rates, and continued tolerability relative to the initial report on the LIBRETTO-001 trial in the New England Journal of Medicine. The group also reported three-year progression-free survival rates of 75.2% and 87.3% in naïve MTC and naïve TC, respectively.
Butsch Science Communication 