This week on Pipeline Moves, we kick off by looking at a Phase III trial in alpha thalassaemia and beta thalassemia which met its primary endpoints. Meanwhile, a Phase II trial in propionic acidaemia and methylmalonic acidaemia was terminated.

On a positive note, a Phase II trial in non-proliferative diabetic retinopathy and proliferative diabetic retinopathy, a Phase I study of a candidate in alpha and beta-thalassaemia, a Phase I trial in myelodysplastic syndrome and chronic myeloid leukaemia all completed.

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Agios completes Phase III thalassaemia trial

Agios Pharmaceuticals’s mitapivat sulfate saw its Likelihood of Approval (LoA) rating rise in alpha thalassaemia and beta thalassaemia after a Phase III trial in the same indications reached its endpoints.

The LoA for mitapivat sulfate rose by eight percentage points from 17% to 25% in alpha thalassaemia and by nine percentage points from 42% to 51% in beta thalassaemia.

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.

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By GlobalData

Agios Pharmaceuticals announced in a press release on 3 January that the study met its primary endpoint of haemoglobin response. GlobalData evaluated the asset on 8 January.

Agios reported that statistical significance was also achieved for both key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and haemoglobin concentration.

The Phase III trial (NCT04770753) investigated mitapivat in adults with non-transfusion-dependant (NTD) alpha-thalassaemia or beta-thalassaemia.

The study enrolled 194 patients, with 130 randomised to mitapivat 100 mg twice-daily (BID) and 64 randomised to matched placebo. Additionally, 122 patients (93.8%) in the mitapivat arm and 62 (96.9%) in the placebo arm completed the 24-week double-blind period of the study.

Agios is also advancing the fully enrolled Phase III ENERGIZE-T study (NCT04770779) of the candidate in adults with transfusion-dependant alpha or beta thalassaemia. Topline data from the 48-week study is expected in mid-2024.

Following the read-out of ENERGIZE-T, Agios Pharmaceuticals intends to file for regulatory approval of mitapivat sulfate as a treatment for thalassaemia by the end of 2024.

Progression prospect for nesvategrast rises after completion

OcuTerra Therapeutics’s nesvategrast saw an increase in its Phase Transition Success Rate (PTSR) after a Phase II trial was completed. The PTSR jumped by nine points to 39% in non-proliferative diabetic retinopathy (NPDR) and to 45% in proliferative diabetic retinopathy (PDR).

PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next. Both LoA and PTSR can be calculated for a drug by considering characteristics like therapy area, indication and molecule type.

The company announced the completion of the last patient’s final visit on 3 January, with GlobalData evaluating the asset on 5 January.

The Phase II trial (NCT05409235) investigated the safety and efficacy of nesvategrast. The study enrolled 225 patients with moderately severe to severe NPDR or mild PDR with minimal vision loss.

The primary endpoints measured the proportion of participants with treatment-emergent adverse events and the proportion of participants who improved by ≥ 2 steps from baseline in Diabetic Retinopathy Severity Scale (DRSS) scores. Topline data is expected to be released in Q1 2024, as per the announcement on January 3.

Nesvategrast, also known as OTT166, is a selective small molecule RGD integrin inhibitor, which is administered as an eye drop.

Phase II trial in propionic acidaemia and methylmalonic acidaemia terminated

HemoShear Therapeutics’s HST5040 saw its PTSR decline after a Phase II trial was terminated. The PTSR dropped by 16 points to 9% in propionic acidaemia and methylmalonic acidaemia.

The trial was terminated due to business considerations by the sponsor, according to the ClinicalTrials.gov registry. The trial’s status was updated on 5 January, with GlobalData evaluating the asset on 8 January.

The Phase II trial (NCT04732429) aimed to assess the safety, efficacy, and pharmacokinetics of HST5040 in subjects with propionic or methylmalonic acidemias. The study consisted of two open-label and one randomised, double-blind portion, with the aim of assessing if the candidate can improve levels of accumulated disease-associated toxins.

Propionic and methylmalonic acidemias are inherited conditions in which the body is unable to break down certain proteins and fats.

Silence Therapeutics completes Phase I thalassemia trial

Silence Therapeutics’s SLN-124 saw its PTSR increase following the completion of a Phase I study. The drug’s PTSR rose by seven points in alpha thalassaemia, reaching 71%.

The trial’s status was updated on its ClinicalTrials.gov from active, not recruiting to completed on 3 January, and GlobalData evaluated the asset on the next day.

The randomised, placebo-controlled Phase I trial (NCT04718844) evaluated the efficacy and safety of SLN-124 in 44 adult participants with alpha-thalassaemia and beta-thalassaemia and very low- and low-risk myelodysplastic syndrome. The study’s primary endpoint aimed to assess the incidence of treatment-emergent adverse events within a timeframe of up to 84 days.

The asset is a transmembrane protease serine 6 (TMPRSS6) inhibitor, which acts by silencing TMPRSS6 to increase hepcidin levels and in turn reduce iron levels. The drug candidate elicits therapeutic action by improving erythropoiesis and reducing organ iron overload.

Amgen complete Phase I myelodysplastic syndrome and chronic myeloid leukaemia trial

Amgen’s tapotoclax saw its PTSR increase in myelodysplastic syndrome and chronic myeloid leukaemia (CML) after a Phase I trial was completed. The drug’s PTSR increased by seven points in both indications, reaching 62% and 66%.

The Phase I trial’s (NCT05209152) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 5 January and GlobalData evaluated the asset on the same day.

The purpose of the study was to assess the tolerability, safety, and efficacy of tapatoclax as a monotherapy and in combination with Bristol Myers Squibb’s Vidaza (azacitidine) for the treatment of higher risk myelodysplastic syndrome and CML. The study enrolled nine patients.

Tapatoclax acts as highly selective and reversible MCL-1 inhibitor.

Phase I glioblastoma multiforme trial completes

Diakonos Research’s dendritic cell vaccine saw its PTSR increase in glioblastoma multiforme (GBM) after a Phase I trial was completed. The drug’s PTSR increased by 12 points, reaching 77%.

The Phase I trial’s (NCT04552886) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 11 January, and GlobalData evaluated the asset on the following day.

The trial was sponsored by the New Jersey-based Cooper Health System.

The purpose of the study was to assess the safety of the dendritic cell vaccine administered to GBM patients shortly after completing standard-of-care treatments. The study enrolled 17 patients.

The cellular immunotherapy elicits antineoplastic activity by activating the immune system.

The Houston, Texas-based company is also developing the cell therapy candidate for the treatment of pancreatic ductal adenocarcinoma, cutaneous angiosarcoma, prostate cancer and triple-negative breast cancer.

Read the last edition:

Pipeline Moves: Phase III bronchiectasis trial terminated due to recruitment issues

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.