In this week’s edition of Pipeline Moves, Clinical Trials Arena reviews four studies that were either suspended or terminated: a blood cancer platform trial, one study in primary systemic amyloidosis, another in tuberculosis, and the fourth being a trial in uterine leiomyoma.

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Novartis suspends platform trial

Novartis’s four oncology assets see their Phase Transition Success Rates (PTSR) plummet following a Phase I/II platform trial suspension. The study’s suspension was due to challenges in identifying patient populations as specified per protocol. The PTSR-affected oncology assets are Adaveko (crizanlizumab), siremadlin, sabatolimab, and NIS-793.

The trial’s listing also states Novartis’s portfolio is under re-evaluation. There were no new safety concerns among study subjects. GlobalData evaluated the assets on July 5 after a update on the same day. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

In myelofibrosis, Adaveko’s and sabatolimab’s PTSR dropped 21 points to 14% and 13%, respectively. NIS-793’s PTSR also decreased by 21 points to 16%, while siremadlin’s dove by 12 points to 12%.

In post-polycythemia vera myelofibrosis (PPV-MF), NIS-793’s and sabatolimab’s respective PTSRs dipped 18 points to 20% and 17%. Adaveko’s PTSR decreased 17 points to 19%, and siremadlin’s diving 15 points to 16%.

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As for post-essential thrombocythemia myelofibrosis (Post-ET MF), NIS-793’s and Adaveko’s PTSR fell 23 points to 25% and 24%, respectively. Sabatolimab’s rate also decreased 23 points to 22%, while siremadlin reduced 19 points to 20%.

The Phase I/II open-label platform trial (NCT04097821) anticipated to enrol 243 myelofibrosis patients. The study was designed to investigate Incyte’s Jakafi (ruxolitinib), as a combination partner of each of the four candidates. LTT462 is the fifth asset under investigation as a combo in the platform trial.

Adaveko secured FDA approval for sickle cell disease in November 2019. Siremadlin inhibits the binding of the human double minute 2 homolog (HDM2) protein to tumor suppressor protein p53, to induce tumor cell apoptosis. Sabatolimab elicits antitumor response by targeting T-cell immunoglobulin mucin-3 (TIM3). Meanwhile, NIS-793 acts by inhibiting transforming growth factor beta 1 (TGFb1).

Primary systemic amyloidosis study terminated

Acrotech Biopharma’s melphalan saw its PTSR plunge in primary systemic amyloidosis after the termination of a Phase II study sponsored by Boston Medical Center. The PTSR dove by 25 points to 30%.

The Phase II study (NCT02994784) was halted early having encountered slow accrual due to the Covid-19 pandemic, according to its listing. The study aimed to enrol 43 subjects but ended with 28. The termination was also due to alternative primary systemic amyloidosis treatments being available, it states. The trial’s status changed on July 6, with GlobalData appraising the candidate the next day.

The Phase II investigated propylene glycol-free melphalan hydrochloride in subjects with amyloid light chain (AL) amyloidosis before receiving autologous stem cell transplantation. AL amyloidosis is also known as primary amyloidosis. Amyloidosis refers to several conditions, which feature the build-up of abnormal amyloid proteins in the body. The trial had a primary endpoint looking into the candidate’s safety profile.

Melphalan is a phenylpropanoic acid derivative, which acts as an antineoplastic agent. It is marketed in the US under the brand name Evomela for the treatment of multiple myeloma. In March 2019, Spectrum Pharmaceuticals announced the completion of its sale of Evomela, alongside other treatments, to Acrotech. Acrotech is a subsidiary of Aurobindo Pharma.

Tuberculosis trial terminated

Archivel Farma’s therapeutic vaccine, under the name RUTI, saw its PTSR decrease in tuberculosis following a Phase IIa trial termination. The PTSR dropped by 11 points, settling at 45%.

The Phase IIa study (NCT02711735) was terminated due to lack of enrolment according to its listing. The trial initially anticipated to recruit 27 participants but ended up only recruiting nine subjects. The study recruited from Ukraine with three trial sites. Its status was updated on July 4, with GlobalData evaluating the asset on July 06. Archivel is based in Barcelona, Spain. 

The Phase IIa aimed to recruit patients with multidrug-resistant tuberculosis (MDR-TB). RUTI is a therapeutic vaccine designed to be administered weeks upon the start of standard MDR-TB treatment. The trial’s primary endpoint investigated the candidate’s safety profile over eight weeks after vaccination. 

RUTI is a therapeutic vaccine also in clinical trials in latent tuberculosis, non-muscle-invasive bladder cancer (NMIBC), and Covid-19. The vaccine’s mechanism is linked to the induction of a Th1 response that stimulates specific immunity against antigens.

Bayer terminates uterine leiomyoma trial

Bayer’s vilaprisan (BAY-1002670) saw its Likelihood of Approval (LoA) in uterine leiomyoma (uterine fibroids) drop by eight points to 23% after a Phase III trial was terminated. The study closed prematurely due to changes in its development program.

The drug was evaluated by GlobalData on July 2 after its entry was updated from completed to terminated on June 30. Vilaprisan is a selective progesterone receptor agonist. LoA is identified via GlobalData’s analysis using a combination of machine learning and its proprietary algorithm.

The double-blind, placebo-controlled Phase III study (NCT03400956) enroled 103 subjects with uterine fibroids. The primary objective of the trial was to show the superiority of vilaprisan compared to placebo in the treatment of heavy menstrual bleeding.

Participants received vilaprisan, placebo and vilaprisan, or vilaprisan and placebo in two period treatments for 12 weeks separated by one bleeding week. Amenorrhea or absence of menstruation, as a primary endpoint was detected in 90.6% of participants in the first arm, 3% in second arm, and 80.6% in the third arm.

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.