In this week’s edition of Pipeline Moves, we take a look at Aslan Pharmaceutical’s completion of its Phase II/III gastric cancer trial and RegenxBio’s Phase I/II interim data in mucopolysaccharidosis type I.
We also have a variety of Phase I updates. We cover Ocular Therapeutix in open-angle glaucoma and ocular hypertension, Bayer for its inflammation asset, AnaMar in idiopathic pulmonary fibrosis and systemic sclerosis, and Menarini’s solid tumour asset felezonexor.
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Aslan completes gastric cancer trial
Aslan Pharmaceuticals’ ASLAN001 (varlitinib) had its Likelihood of Approval (LoA) rise in gastric cancer after the completion of a Phase II/III trial. The candidate’s LoA increased by seven points to 56%.
GlobalData’s review happened on 16 February following the ClinicalTrials.gov update on 15 February. LoA is identified via GlobalData’s analysis using a combination of machine learning and its proprietary algorithm.
The Phase II/III study (NCT03130790) enrolled 52 patients with first-line advanced or metastatic gastric cancer. Patients received the chemotherapy cocktail modified FOLFOX6 with either varlitinib or placebo. The trial has two coprimary endpoints investigating tumour size at week 12 at the trial’s phase II portion, and overall survival at the phase III portion.
Regenxbio reports positive interim data
Regenxbio’s RGX-111 for mucopolysaccharidosis type I (MPS I) saw its Phase Transition Success Rate (PTSR) jump five points to 33% after interim Phase I/II data was reported. Regenxbio released interim results in a 9 February press release, and the PTSR change took effect on 11 February. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
The company reported data from the first five patients and has plans to expand enrollment by up to six additional patients. In the Phase I/II trial (NCT03580083), the first five patients had no reported serious drug-related AEs, in the study’s primary endpoint. As for the secondary endpoints, the study reported positive biomarker activity.
MPS I is an autosomal genetic disease characterized by cell, tissue, and organ dysfunction and is caused by a deficiency in the lysosomal enzyme alpha-L-iduronidase (IDUA). RGX-111 is a single-dose gene therapy that delivers the IDUA gene to the central nervous system, which could prevent the progression of cognitive deficits in MPS I.
Ocular shares positive Phase I
Ocular Therapeutix’s OTX-TIC (travoprost) saw an increase in its PTSR after the announcement of positive Phase I results. The PTSR grew by 10 and 11 points to 84% in open-angle glaucoma and ocular hypertension, respectively. GlobalData’s update happened on 16 February after a company presentation on 11 February.
The Phase I (NCT04360174) enrolled 19 patients, with participants receiving a travoprost implant in one eye and topical travoprost in the other as an active comparator. Results show that the travoprost implant produced intraocular pressure (IOP)-lowering effects as early as two days following administration.
The hydrogel-based travoprost implant is injected into the anterior chamber of the eye and gradually delivers the treatment over several months. Topical travoprost is administered once a day to the eye.
Bayer completes Phase I
Bayer’s BAY1830839 saw its PTSR rise in inflammation by nine points to 63% after completion of a Phase I trial. The update on 14 February followed the new information on ClinicalTrials.gov on 11 February.
The Phase I study (NCT05003089) enrolled 72 healthy male participants. In addition to receiving BAY1830839, participants received imiquimod and lipopolysaccharide, which are intended to cause irritation and inflammation to the skin and in the blood, respectively. This would allow the study to see if the asset can reduce such symptoms.
BAY1830839 is an interleukin-1 receptor associated kinase-4 inhibitor, which prevents the production of proinflammatory cytokines by targeting the enzyme.
AnaMar concludes Phase I study
AnaMar’s AM-1476 saw its PTSR rise in idiopathic pulmonary fibrosis (IPF) and systemic sclerosis after a Phase I trial was completed. The PTSR rose six points to 54% in IPF and seven points to 61% in systemic sclerosis. ClinicalTrials.gov updated the trial to “completed” on 16 February, and the PTSR change took effect the next day.
Gothenburg, Sweden-based AnaMar designed the Phase I trial (NCT04691115) to assess the safety and pharmacokinetics of AM-1476 in 72 healthy subjects. AM-1576 targets the 5-hydroxytryptamine receptor 2B (5-HT-2B), which is implicated in fibrosis development.
Menarini terminates solid tumour trial
Menarini’s felezonexor saw its PTSR decline across multiple indications after its Phase I solid tumour trial was terminated. GlobalData’s appraisal happened on the 14 February following the ClinicalTrials.gov update on 11 February.
The PTSR decreased by 35 points in colorectal cancer and gastrointestinal tumours, landing at 31% and 33%, respectively. There was also a 34-point decline to 32% in urinary tract cancer, 29 points to 62% in non-small cell lung cancer, and 25 points to 29% in neuroendocrine tumours. A 21-point drop to 18% in solid tumours and 13 points to 52% in breast cancer were also recorded.
The Phase I trial (NCT02667873) enrolled 72 patients with advanced solid tumours. Patients received oral felezonexor during a 28-day cycle, with a primary endpoint looking into the asset’s safety profile.
Felezonexor was added to Menarini’s pipeline after it acquired Stemline Therapeutics in May 2020. Felezonexor is an oral, small-molecule reversible inhibitor of exportin-1 (XPO1), a key nuclear transport protein.
Need to Know:
GlobalData’s proprietary model uses a combination of machine learning and an algorithm to calculate an individual drug’s PTSR and LoA. While LoA provides the probability of a drug ultimately receiving market authorization, PTSR indicates the probability of a drug’s advancement to the next stage of clinical development. The model uses data points from the individual drugs, clinical trials, regulatory milestones, company, and financial databases.