This week on Pipeline Moves, we start by looking at Alnylam’s Phase III win with Amvuttra in ATTR amyloidosis with cardiomyopathy.
Meanwhile, Junshi Biosciences’s toripalimab saw a Phase II investigator-led ovarian cancer trial suspended.
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On a positive note, Shiongi’s S-309309 reported a Phase II trial completion in obesity.
Interested in more news in your inbox? Sign-up for our daily newsletter.Alnylam win in Phase III ATTR amyloidosis trial
Alnylam Pharmaceuticals’s Amvuttra (vutrisiran) saw its Likelihood of Approval (LoA) rating increase in transthyretin (ATTR) amyloidosis with cardiomyopathy after a Phase III trial in the same indication met all endpoints.
The LoA for Amvuttra shot up by 11 percentage points from 55% to 66% in ATTR amyloidosis with cardiomyopathy. LoA is calculated by 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.
A press release from Alnylam Pharmaceuticals, released on 24 June 2024, states that the HELIOS-B trial achieved statistical significance on the primary and all secondary endpoints. GlobalData evaluated the asset on 26 June 2024.
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By GlobalDataThe press release says that the trial achieved 28% and 33% reduction of all-cause mortality and recurrent cardiovascular events in the overall and monotherapy populations, respectively.
The Phase III trial (NCT04153149) evaluated the efficacy and safety of Amvuttra in patients with ATTR amyloidosis with cardiomyopathy.
The trial randomised 655 adult patients with ATTR amyloidosis with cardiomyopathy at a 1:1 ratio for Amvuttra or placebo. The drug was administered subcutaneously once every three months during a treatment period, which went up to 36 months.
Alnylam will now move ahead with global regulatory submissions later this year, including filing a supplemental New Drug Application with the FDA using a Priority Review Voucher.
Phase II trial of Shionogi’s obesity candidate completion
Shionogi’s S-309309 saw its Phase Transition Success Rate (PTSR) increase following a Phase II trial completion in obesity. The drug’s PTSR rose by nine points to 34% in obesity. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
The trial’s status was updated on its ClinicalTrials.gov from active, not recruiting to completed on 28 June and GlobalData evaluated the asset on 1 July.
The Phase II trial (NCT05925114) evaluated the efficacy of S-309309 in 365 obese adult subjects, compared to placebo. The trial’s primary endpoint evaluated the change from baseline in body weight for a time period of up to 24 weeks post-treatment.
The asset is a monoacylglycerol acyltransferase 2 (MGAT2) inhibitor. The drug candidate is an oral asset that plays a major role in lipid metabolism by inhibiting MGAT2, which is highly expressed in the small intestine.
Junshi Biosciences’s ovarian cancer loss in Phase II trial
Shanghai Junshi Biosciences’s toripalimab saw a 10-point fall in its PTSR in ovarian cancer following the extended suspension of a Phase II institution-led study.
PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next. It can be calculated for a drug by considering characteristics like therapy area, indication and molecule type.
The PTSR change took place on 28 June, following a change in the trial’s start date (NCT05479045) on ClinicalTrials.gov from June 2024 to August 2024 on 25 June. As per the ClinicalTrials.gov entry, the protocol amendment is pending and its status remained suspended.
The Georgetown University-led Phase II study, which also lists the US Department of Defence as a partner, is designed to investigate a combination therapy strategy to prevent anti-PD-1 therapy resistance in metastatic ovarian cancer patients. The updated study protocol swapped Bristol Myers Squibb’s Opdivo (nivolumab) with toripalimab in a combination regimen with a New York-esophageal cancer 1 (NY-ESO-1) peptide vaccine. The trial measured median progression-free survival as its primary endpoint.
Toripalimab is a PD-1 monoclonal antibody that prevents binding of PD-1 with PD-L1 and PD-L2, blocking downstream signalling. This acts to restore immune function through the activation of T cells and T-cell-mediated immune responses against tumour cells. Toripalimab is currently approved for the treatment of nasopharyngeal carcinoma under the US brand name Loqtorzi, and marketed as Tuoyi in China. The Shanghai, China-headquartered company is also investigating the therapy as a treatment for breast cancer, small cell lung cancer, and liver cancer amongst other conditions.
Cartesian’s cell therapy falls in multiple myeloma
Cartesian Bio’s Descartes-08 saw its PTSR decrease in multiple myeloma after a Phase II trial was terminated. The PTSR dropped by ten points to 24%.
The Phase II trial’s (NCT04816526) status was updated from completed to terminated on ClinicalTrials.gov on 27 June, and GlobalData evaluated the asset on the following day. According to the study’s ClinicalTrials.gov listing, the sponsor did not disclose the reason for terminating the study.
The purpose of the study was to assess the minimal residual disease (MRD)-negative complete response rate after consolidation treatment with Descartes-08 in patients with high-risk myeloma who have residual disease following induction therapy. The study enrolled 13 patients.
Descartes-08 exhibits antineoplastic activity by targeting cells expressing B cell maturation antigen (BCMA) and is under development for the treatment of multiple myeloma (third-line therapy), myasthenia gravis, systemic lupus erythematosus and unspecified autoimmune diseases.
Phase II multiple sclerosis trial completes
Hope Biosciences’s HB-adMSCs saw a nine-point jump in its PTSR, landing at 36% in relapsing/remitting multiple sclerosis (RRMS), after a Phase II trial was completed.
The study’s ClinicalTrials.gov listing (NCT05116540) was updated from active, not recruiting to completed on 20 June. GlobalData evaluated the asset on 24 June. The randomised, double-blind Phase II study compared the efficacy and safety of autologous HB-adMSCs compared to placebo in multiples sclerosis patients. Led by the Hope Biosciences Stem Cell Research Foundation, the trial’s primary endpoint evaluated the efficacy of intravenous infusions of the therapy against placebo in patients, measured as improvements in the Multiple Sclerosis Quality of Life (MSQOL) 54 Instrument.
Sugar Land, Texas-based Hope Biosciences’s cell therapy uses adipose-derived mesenchymal stem cells to treat RRMS. The treatment acts by eliciting regenerative effects as the cell therapy differentiates into numerous cell types. The company is also investigating the cell therapy for its use in several other conditions including cerebral palsy, Parkinson’s disease, and traumatic brain injury.
Note: The indication in paragraphs one, four and five has been updated.
Read the last edition:
Pipeline Moves: Approval prospects for BioTissue’s diabetic foot ulcer drug up after Phase III completionNeed 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 advancemen
