In this week’s edition of Pipeline Moves, the Clinical Trials Arena team reports on a Phase I hepatitis B trial termination and a Phase Ib pancreatic cancer study suspension. We also review a Phase II solid tumour trial termination, leading to an asset’s likelihood of further investigation to drop in endometrial cancer.

On to more positive news, the team reports on the further study prospects of a cannabidiol transdermal gel in DiGeorge Syndrome after positive Phase II data, as well as the Likelihood of Approval of a prurigo nodularis asset on the back of positive Phase III results.

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Hepatitis B trial terminated

VenatoRx Pharmaceuticals’s hepatitis B asset VNRX-9945 saw a drop in its Phase Transition Success Rate (PTSR) after a Phase I study was terminated. The PTSR dropped by 24 points to 25%. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

According to the study’s (NCT04845321) listing, the trial had its part 2 terminated, unrelated to safety data. The study’s status changed on June 21, with the PTSR change happening the following day.

The 32-subject study aimed to test single and multiple ascending doses of VNRX-9945 in healthy adult volunteers. In part one, participants were given one dose of VRNX-9945 or the placebo. The first part also had a food effect cohort, which received treatments in a fasted and fed state, making a total of two doses.

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By GlobalData

The second part planned to dose the subjects with VNRX-9945 or placebo once a day for 14 days. In both parts, the primary endpoint was the number of subjects with adverse events up to eight days after the last dosing. 

VNRX-9945 is a capsid protein inhibitor, which blocks hepatitis B virus replication at multiple steps in the viral lifecycle. By blocking the viral capsid assembly, the treatment interferes with virus production.

Pancreatic cancer study suspended

SignalChem Lifesciences’s SLC-0111 saw it PTSR in pancreatic ductal adenocarcinoma (PDAC) drop after an investigator-led Phase Ib trial was suspended. The PTSR decreased by 18 points to 15%.

The suspension was due to restructuring at the trial’s pharma partner, requiring the need for a change in partner altogether. The Phase Ib trial (NCT03450018) lists British Columbia Cancer Agency as the sponsor, while the Canadian Cancer Society and Welichem Biotech, SignalChem’s development partner, are collaborators. GlobalData evaluated the asset on June 24 after a update the day prior.

The open-label, single-arm study intended to measure the safety and tolerability of oral SLC-0111 in combination with chemotherapy gemcitabine. It anticipated to enrol 30 patients with metastatic PDAC who are positive for carbonic anhydrase IX. SLC-0111 acts as carbonic anhydrase IX inhibitor and induces cancer call apoptosis.

Pemazyre solid tumour cancer study terminated

Incyte’s Pemazyre (pemigatinib) saw its PTSR dive in endometrial cancer following a Phase II trial termination. The PTSR declined by 11 points, settling at 33%.

It was a business decision to discontinue further enrolment in the Phase II trial (NCT03822117), with the listing further noting there were no safety concerns that contributed to the decision. The status of the trial was updated on June 20, with GlobalData evaluating the asset on June 22. The trial was previously marked as completed on March 29. 

The Phase II study aimed to evaluate Pemazyre in locally advanced, metastatic, or inoperable solid tumour malignancies with activating FGFR mutations or translocations. The trial was initially anticipated to recruit a total of 170 participants but ended up recruiting 111 patients. The study’s primary endpoint is objective response rate up to approximately six months.

Pemazyre was FDA approved for metastatic bile duct cancer in April 2020. It is under development for multiple oncology indications such as gastric, breast, colorectal, non-small cell lung cancers, among others.

Cannabidiol gel trial reveal positive results

Zynerba Pharmaceuticals’s cannabidiol transdermal gel ZYN002 (Zygel) saw its PTSR rise in DiGeorge Syndrome after a Phase II trial completion and announcement of positive topline results. The PTSR increased by 15 points to 50%.

GlobalData evaluated the asset on June 22 after the company issued a press release on the same day. The open-label, single-arm Phase II (NCT05149898) enrolled 20 patients, aged four to 15, with genetically confirmed 22q11.2 DiGeorge syndrome.

In the Phase II’s primary endpoint, topline results show Zygel was well tolerated and safe. The asset also showed statistically significant improvements at 14 weeks of treatment compared to baseline data in the secondary efficacy endpoints Anxiety, Depression and Mood Scale (ADAMS), Aberrant Behavior Checklist – Community (ABC-C), and Pediatric Anxiety Rating Scale (PARS-R).

Galderma report positive Phase III data

Galderma’s Mitchga (nemolizumab) saw its Likelihood of Approval (LoA) increase by eight points reaching 27% in prurigo nodularis, after positive Phase III trial data was announced. The company announced the positive topline data on June 22, with GlobalData evaluating the asset on June 27.

LoA is identified via GlobalData’s analysis using a combination of machine learning and its proprietary algorithm and can be calculated by considering characteristics like therapy area, indication and molecule type.

The randomised, double-blind Phase III OLYMPIA-2 study (NCT04501679) evaluated the efficacy and safety of Mitchga in adult patients with moderate-to-severe prurigo nodularis. The trial met both primary endpoints of improving skin lesions and itchiness in prurigo nodularis patients after a 16-week treatment period. The company is also investigating the drug’s efficacy in prurigo nodularis in the ongoing phase III OLYMPIA-1 trial.

Prurigo nodularis is a rare, inflammatory skin condition, characterised by skin nodules causing an intense and chronic itching sensation, and has no approved therapies. Mitchga is a monoclonal antibody that inhibits IL-31 signaling, which plays a key role in prurigo nodularis pathophysiology. The drug is marketed in Japan for pruritus associated with atopic dermatitis.

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.