In this edition of Pipeline Moves, the Clinical Trials Arena team kicks off with an analysis of the termination of an investigator-led Phase I/II cystic fibrosis trial. The study was on an asset owned by Horizon Therapeutics. We continue with Ascendis Pharma’s sharing of positive topline Phase III data in hypoparathyroidism. We close off with the completion of two Phase I trials, one by Novartis in uveal melanoma and the second by Sunhawk Vision Biotech in myopia.

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Investigator-led cystic fibrosis clinical trial terminated

Horizon’s Ravicti (glycerol phenylbutyrate) saw its Phase Transition Success Rate (PTSR) in cystic fibrosis plunge 19 points to 18% after an investigator-led Phase I/II trial was terminated. The study (NCT02323100) was sponsored by National Jewish Health, with Horizon listed as one of the collaborators.

The trial’s ClinicalTrials.gov listing was updated to “terminated (funding ended)” on 21 March, and the PTSR change took effect the next day. The study had a target enrollment of 36 patients, but ultimately enrolled 13 prior to the study’s termination. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

Ravicti, which has FDA approval for urea cycle disorders, provides an alternative pathway to urea that could lower ammonia in patients with cystic fibrosis.

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Ascendis reports Phase III data

Ascendis’ ACP-014 saw its Likelihood of Approval (LoA) leap 11 points to 76% in hypoparathyroidism following positive topline Phase III data. LoA is identified via GlobalData’s analysis using a combination of machine learning and its proprietary algorithm.

Some 78.7% of ACP-014 patients achieved the primary endpoint of serum calcium levels reaching the normal range and independence from therapeutic levels of conventional therapy, compared to 4.8% of subjects in the placebo arm (p<0.0001). The company reported topline data from the 82-patient Phase III PaTHway trial (NCT04701203) on March 13, and the LoA change took effect March 21.

Hypoparathyroidism is characterised by abnormally low levels of parathyroid hormones, which regulate the balance between calcium and phosphorus in the body. Subcutaneously administered ACP-014, also known as TransCon PTH, is a long-acting prodrug of parathyroid hormones.

Novartis completes Phase I

Novartis’ HDM201 saw its PTSR in metastatic uveal melanoma rise after the completion of a Phase I study that studied HDM201 as a combination partner. HDM201’s PTSR grew by seven points to 65% upon the completion of the Phase I study (NCT02601378) investigating darovasertib (LXS196) as a monotherapy and in combination with HDM201.

The 107-subject Phase I was updated to “completed” in a 21 March update on ClinicalTrials.gov, with GlobalData appraising the asset the next day. The study primarily investigated for safety but had efficacy, pharmacokinetic, and pharmacodynamic secondary endpoints.

HDMI201 is a human double minute 2 homolog (HDM2) inhibitor, which is intended to restore p53 signaling and p53-mediated induction of tumor cell apoptosis. Darovasertib is a protein kinase C inhibitor, designed to induce the apoptosis of cancer cells and inhibits tumor cell proliferation.

Sunhawk concludes myopia trial

Sunhawk Vision Biotech’s SHJ002 saw its PTSR in myopia rise after the completion of a Phase I study. The PTSR grew by seven points to 62%.

The open-label study (NCT04928144) had its status changed to “completed” in a 23 March update on ClinicalTrials.gov. GlobalData had its latest update on 24 March.

The 12-subject, dose-escalation study investigated the safety and tolerability of SHJ002 in paediatric subjects. The trial had coprimary endpoints measuring adverse events and the change from baseline in best corrected visual acuity. The trial examined the asset in four different concentrations: 0.025%, 0.080%, 0.25% and the fourth arm measuring an unnamed maximum tolerated concentration of the ophthalmic solution.

SHJ002 is an antisense RNAi oligonucleotide, which targets microRNA-328. By inhibiting the expression of microRNA-328, the asset is intended to increase retinal pigment epithelium cell proliferation and elicit a therapeutic intervention.

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.