This week on Pipeline Moves, we kickoff by looking at two Phase III trials in oncology and kidney disease that failed to meet their endpoints. On a good note, we investigate completions in vitiligo, solid tumours, hepatitis B and familial amyloid cardiomyopathy.

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Phase III trial termination

Aravive’s batiraxcept saw its Likelihood of Approval (LoA) for ovarian cancer drop dramatically in ovarian cancer after a Phase III trial was terminated.

The drug’s LoA fell by 10 points to 1% in ovarian cancer. LoA is identified via GlobalData’s analysis using a combination of machine learning and a proprietary algorithm. LoA can be calculated for a drug by considering characteristics like therapy area, indication and molecule type.

The trial’s status was updated from active, not recruiting to terminated on on 26 October. According to the site, the termination is due to the trial showing no significant differences in median progression free survival (PFS) between batiraxcept and paclitaxel or paclitaxel alone arms.

The company added that there was no detriment to overall survival and no new safety signals were identified. GlobalData evaluated the asset on 31 October.

The randomised and double-blind Phase III trial (NCT04729608) compared the efficacy and safety of batiraxcept in combination with paclitaxel versus placebo in combination with paclitaxel in patients with platinum-resistant recurrent ovarian cancer.

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Batiraxcept acts by inhibiting growth arrest-specific protein 6 (GAS6). Activation of the AXL receptor occurs when it binds to a GAS6 ligand on tumor cells, which causes tumor cell proliferation. The drug candidate blocks this binding and prevents tumour progression.

Phase III trial failure

Travere Therapeutics’ Filspari (sparsentan) saw its LoA drop after a Phase III trial in focal segmental glomerulosclerosis (FSGS) failed to meet its endpoints. The LoA for Filspari fell by nine points from 23% to 14%.

A paper published in the New England Journal of Medicine states that among patients with FSGS, there were no significant between-group differences in eGFR slope at 108 weeks, despite a greater reduction in proteinuria with Filspari than with irbesartan. GlobalData evaluated the asset on 6 November.

The Phase III trial (NCT03493685) was investigating the long-term nephroprotective potential of Filspari compared to an angiotensin receptor blocker, irbesartan, in patients with primary and genetic FSGS.

Filspari is a selective dual-acting receptor antagonist with affinity for endothelin (A type) and angiotensin II receptors (type 1). Endothelin and angiotensin II are the vasoconstrictor substances that also can exert proliferative and proinflammatory effects.

Filspari was originally developed by Bristol Myers Squibb before being licensed to Pharmacopeia, which was acquired by Ligand Pharmaceuticals Travere acquired the drug from Ligand in 2012.

Phase Ib trial completion

VYNE Therapeutics‘ VYN201 saw its Phase Transition Success Rate (PTSR) grow in vitiligo after the announcement of positive data from a Phase Ib trial. The drug’s PTSR grew by 14 points to 79%. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

On 30 October, the company announced positive clinical data from the Phase Ib trial in a press release. The trial studied the safety, tolerability and pharmacokinetics of three different doses of once-daily topical VYN201 in 29 subjects. Specifically, the study tested 0.5%, 1% and 2% doses of VYN201.

The trial measured the treatment’s potential efficacy and effects on the progression of skin depigmentation and repigmentation through the facial vitiligo area scoring index (F-VASI).

Significant clinical improvements were observed in both the 1% and 2% cohorts with a dose dependent response, based on the 30 October press release. Specifically, 50% of subjects in the 1% cohort achieved an improvement in their F-VASI score that was equal or greater than 25%. The treatment was broadly well-tolerated, and no serious adverse events were reported.

VYN-01 is a locally-administered pan-bromodomain BET inhibitor that has so far shown reductions in pro-inflammatory and disease-related biomarkers, based on the 30 October press release.

According to the press release, the company now plans to advance the asset into a Phase IIb study in H1 2024.

Moderna Phase I trial completion

Moderna’s MEDI1191 saw its PTSR increase in solid tumours after a Phase I trial was completed. The drug’s PTSR increased by 12 points, reaching 49%.

The Phase I trial’s (NCT03946800) status was updated from active, not recruiting to completed on on 31 October, and GlobalData evaluated the asset on the following day.

The open-label, dose-escalation, and expansion study evaluated MEDI1191 in combination with AstraZeneca’s Imfinzi (durvalumab) in adult patients with advanced solid tumours. The study enrolled 61 patients.

The gene therapy acts as an IL-12 activator and is under development for the treatment for solid tumours.

Hepatitis B Phase I trial completion

Drug Farm’s DF-006 saw its PTSR increase in hepatitis B after a Phase I trial was completed. The drug’s PTSR grew by six points to 58%.

The Phase I trial’s (ACTRN12621000592842) status was updated to completed on The Australian and New Zealand clinical trial registry on 27 October, and GlobalData evaluated the asset on 1 November. The purpose of this placebo-controlled study was to evaluate the safety, tolerability, and pharmacokinetics of DF-006.

DF-006, an alpha-protein kinase 1 inhibitor (ALPK1) activator, is being developed by Drug Farm, which has offices in the US and China. The small molecule drug candidate is under development for the treatment of hepatitis B virus (HBV) infection and hepatocellular cancer.

Cardiology trial completion

Alexion Pharmaceuticals’s ALXN-2220 saw its PTSR increase in familial amyloid cardiomyopathy (ATTR-CM) after a Phase I trial was completed. The drug’s PTSR increased by seven points, reaching 80%.

The Phase I trials (NCT04360434) status was updated from active, not recruiting to completed on on 3 November, and GlobalData evaluated the asset on 6 November.

The randomised, placebo-controlled, double-blind, dose-escalation study evaluated ALXN-2220 in patients with amyloid transthyretin cardiomyopathy. The study enrolled 46 patients.

ALXN-2220 acts by inhibiting transthyretin (TTR) protein and is under development for the treatment of ATTR-CM.

Read the last edition:

Pipeline Moves: Advancement prospects drop for hearing loss drug after trial termination

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 datapoints from individual drugs, clinical trials, regulatory milestones, company, and financial databases.