This week on Pipeline Moves, we start with the completion of Novartis’s Phase II trial in ulcerative colitis. We also review the suspension of an institution-sponsored oncology Phase II trial and positive interim results from a Phase II study in growth hormone deficiency. We finish off the week with the completions of a Phase II trial in diabetic pain and a Phase I/II trial in ophthalmology indications.
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Novartis’s Phase II ulcerative colitis trial completion
Novartis’s LYS-006 saw its Phase Transition Success Rate (PTSR) in ulcerative colitis (UC) rise nine points to 40% after a Phase II trial was completed. ClinicalTrials.gov updated the trial’s status on 23 November, and the PTSR changed the following day. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
The Phase II trial (NCT04074590) was a randomised, subject-and investigator-blinded, placebo-controlled, proof-of-concept study in patients with UC. The primary outcome was measured by the clinical remission rate at the end of eight weeks. The trial enrolled 23 subjects. This was a significant reduction in participants compared to the 60 that were initially anticipated to participate.
LYS-006 is an orally administered anti-inflammatory agent. Novartis is developing the candidate for the treatment of moderate to severe inflammatory acne, non-alcoholic fatty liver disease (NAFLD), UC and moderate to severe hidradenitis suppurativa.
Suspension of institution-sponsored melanoma trial
Biohaven’s troriluzole saw its PTSR in metastatic melanoma plunge by 14 points to 12% after the suspension of a Phase II trial. The study was sponsored by the Dana-Farber Cancer Institute. Biohaven is a subsidiary of Pfizer.
The trial’s (NCT04899921) status on its ClinicalTrials.gov listing changed from recruiting to suspended due to poor enrolment, on 23 November. GlobalData appraised the asset the next day.
The study investigated the safety and effectiveness of a combination of troriluzole and Bristol-Myers Squibb’s Yervoy (ipilimumab) and Opdivo (nivolumab) in patients with melanoma that has metastasised to their brains. The study was expected to recruit 108 subjects and measured the progression-free survival until progression or death as the primary endpoint.
Troriluzole targets the glutamate neurotransmitter, where it reduces the synaptic levels of glutamate. Glutamatergic dysfunction is said to be linked to a variety of disorders.
Hormone deficiency trial sees positive interim results
Lumos Pharma LUM-201 (ibutamoren mesylate) saw a jump in its PTSR by five points settling at 36% in growth hormone deficiency. The PTSR change took place on 16 November, following the release of interim results from two Phase II trials (OraGrowtH210 and OraGrowtH212 trials) on 14 November.
The OraGrowtH210 trial (NCT04614337) is a multi-site, global trial evaluating the effectiveness of LUM-201 for the treatment of idiopathic paediatric growth hormone deficiency (PGHD). OraGrowtH212 (NCT04806854) is a single-site, open-label clinical study evaluating the pharmacokinetic (PK) and pharmacodynamic (PD) effects of LUM-201 in up to 24 treatment-naïve PGHD subjects.
The primary endpoint for the OraGrowtH210 trial is the number of participants who reached target growth as measured by their annualized height velocity (AHV) measured as standing height. According to Clinicaltrials.gov, the study plans to enrol 80 participants. The press release stated that the study is at approximately 80% enrolment.
Interim results for approximately 50% enrolment (n=41) of the OraGrowtH210 trial showed a mean AHV of 8.6 cm at six months for 1.6 mg/kg/day LUM-201, in line with the 8.3 cm AHV expected from historical database comparisons.
The interim analysis of the OraGrowtH212 trial was performed after 41 subjects, randomised into four treatment arms of approximately ten subjects, completed six months of treatment. The AHV for each arm was comparable to that observed in the OraGrowtH210 Trial. There were no treatment-related Serious Adverse Events (SAEs) in either clinical trial. The company plans to have a complete primary outcome readout for Phase II trials in H2 2023.
LUM-201 acts by increasing serum GH and cortisol levels to stimulate growth. PGHD is a disorder in which the pituitary gland releases a deficient amount of growth hormone, limiting a child’s growth.
Completion of Phase II trial in diabetic pain indication
Eisai’s LY-3526318 saw its PTSR in diabetic peripheral neuropathic pain (DPNP) rise nine points to 32% after a Phase II trial was completed. ClinicalTrials.gov updated the trial’s status from recruiting to completed on 22 November. The PTSR changed the following day.
The Phase II trial (NCT05177094) was conducted to test the efficacy and safety of LY-3526318. The trial enrolled 155 patients. The primary outcome measured the change from baseline in average pain intensity using a numeric rating scale.
LY-3526318 acts as a TRPA1 blocker. Eisai is developing the oral drug for the treatment of pain, osteoarthritis (OA) knee pain, chronic low back pain, and DPNP.
Phase I/II ophthalmology trial completed
AsclepiX Therapeutics’s synthetic peptide AXT-107 saw its PTSR improve in four ophthalmology indications after a Phase I/II trial was completed.
The PTSR increased by six points in diabetic macular oedema (DME) and wet macular degeneration, reaching 37% in both indications. The PTSR also rose by ten points in macular oedema and choroidal neovascularization reaching 41% and 31% respectively in each indication.
The trial’s status was updated on ClinicalTrials.gov from active, not recruiting to completed on 17 November, with GlobalData evaluating the asset the following day.
The open-label Phase I/II trial (NCT04697758) evaluated the safety, tolerability, bioactivity and duration of a single intravitreal injection of AXT-107 in adult patients with DME. The trial was anticipated to enrol 18 participants, but eventually recruited only six subjects.
The trial’s primary endpoint measured the drug’s safety, as assessed by the incidence of adverse events over a period of 48 weeks post-treatment. The secondary endpoints evaluated the asset’s efficacy by measuring mean changes in central retinal thickness, the mean changes and the percentage of subjects improving according to the early treatment diabetic retinopathy study chart in a time frame of up to 48 weeks.