This week on Pipeline Moves, we kick off by looking at Pfizer’s topline data from a Phase II trial of ponsegromab on cancer cachexia.
Meanwhile, IMPACT Therapeutics senaparib has seen its phase transition prospects drop in solid tumours after a Phase II termination.
Go deeper with GlobalData
On a positive note, Merck’s Keytruda has seen its transition chances increase in thymic carcinoma and of Teva Pharmaceuticals has completed a Phase II trial of Austedo in dystonia.
Interested in more news in your inbox? Sign-up for our daily newsletter.Pfizer scores in Phase II cancer cachexia trial
Pfizer’s ponsegromab saw a 16-point increase in its Phase Transition Success Rate (PTSR), settling at 42% in cancer cachexia or cancer-related anorexia following the release of Phase II results. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
Pfizer presented data from its Phase II ponsegromab study (NCT05546476) at the European Society for Medical Oncology (ESMO) 2024 Congress on 14 September. GlobalData evaluated the asset on 17 September.
The Phase II double-blind trial evaluated the efficacy, safety and tolerability of ponsegromab compared to placebo in patients with cancer cachexia. The trial investigated the change from baseline in body weight with ponsegromab compared to placebo as its primary endpoint.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataCachexia is a disorder that causes weight loss and muscle wasting that cannot be reversed by consuming more calories. In the Phase II study, ponsegromab demonstrated significant increases in body weight after 12 weeks across all doses, reaching a 5.6% mean increase at the highest dose at 12 weeks. No clinically significant adverse trends were reported in the treatment group. Furthermore, Pfizer shared that treatment-related adverse events occurred in 8.9% of patients taking placebo compared to 7.7% of patients taking ponsegromab. Based on the positive readout, Pfizer has begun discussing plans to start registration-enabling studies for the therapy in 2025, as per a 17 September press release.
Ponsegromab is a monoclonal antibody therapy that treats cachexia by inhibiting growth differentiation factor 15 (GDF 15) to prevent skeletal muscle wasting induced by the condition. Pfizer is also investigating ponsegromab in a Phase II trial (NCT05492500) for heart failure.
Merck’s Keytruda chances rise in thymic carcinoma
Merck’s Keytruda (pembrolizumab) saw its PTSR in thymic carcinoma increase after Phase II trial results were released at a recent conference. The drug’s PTSR rose by 17 points to 43%.
On 13 September, results from the open-label, single-arm PECATI trial (NCT04710628) were presented at the ESMO congress. GlobalData evaluated the asset on 17 September.
The trial enrolled 43 pre-treated thymic carcinoma and B3-thymoma patients. Participants received a course of 20mg oral lenvatinib daily and 200mg intravenous Keytruda once every three weeks for a maximum of 35 three-week cycles. Lenvatinib is a tyrosine kinase inhibitor (TKI) marketed by Eisai Co as Lenvima to treat thyroid cancer, renal cell carcinoma, and other cancer types.
The five month-progression free survival (PFS) rate with Keytruda was 91%, meeting the study’s primary endpoint. After one year, the overall survival rate was 85%. The rate of treatment-related adverse events that were grade 3 or above was 34.9% with no treatment-related deaths.
Keytruda is a major blockbuster drug approved for several cancer indications. Its sales are expected to peak at over $34bn in 2027 before the company loses its key patents the following year, as per GlobalData. Keytruda is a monoclonal antibody that blocks Programmed Cell Death Protein 1 (PD-1), a T cell surface receptor upregulated in some tumours. This action lifts the inhibition of the anti-tumour immune response.
Phase II dystonia Phase II trial completes
Teva Pharmaceuticals Austedo (deutetrabenazine) saw a nine-point rise in its PTSR in dystonia, settling at 56% following the completion of a Phase II investigator-led trial.
The study’s ClinicalTrials.gov listing (NCT04173260) was updated from active, not recruiting to completed on 6 September. GlobalData evaluated the asset on 23 September. The open-label Phase II AUDYT study investigated the safety and tolerability of Austedo in adult subjects with dystonia. Led by the University of Pennsylvania, the Phase II trial evaluated the proportion of study subjects able to titrate up to the maximum tolerated dose of Austedo as its primary endpoint.
Austedo treats dystonia by limiting the release of dopamine in the body, which prevents the stimulation of certain nerves and involuntary movements. Dystonia is a movement disorder associated with involuntary muscle contraction.
The FDA first approved Austedo in April 2017 for the treatment of chorea associated with Huntington’s disease, with a subsequent approval for tardive dyskinesia in August 2017. The agency has since approved a once-daily, extended-release tablet formulation of the therapy for both indications in May 2024.
Phase II solid tumour trial terminated
IMPACT Therapeutics’s senaparib saw an 11-point decrease in its PTSR, falling to 9% in solid tumours, following a Phase II trial termination.
The study’s ClinicalTrials.gov listing (NCT05824455) was updated from recruiting to terminated on 12 September. GlobalData evaluated the asset on the same day.
The Phase II open-label, multicentre study investigated senaparib in combination with irinotecan in patients with solid tumours. The trial evaluated the incidence of dose-limiting toxicities, the incidence and severity of adverse events, and abnormal changes in laboratory tests as its primary endpoints.
Senaparib is a targeted therapy that inhibits poly (ADP-ribose) polymerase2 (PARP2) and poly (ADP-ribose) polymerase 1 (PARP1). This causes cancer cell death by preventing the cells from repairing damaged cell DNA. IMPACT is also developing the therapy for prostate cancer, liver cancer, and small cell lung cancer.
Phase I large B cell lymphoma trial suspended
Acrotech Biopharma’s Beleodaq (belinostat) saw its PTSR in diffuse large B cell lymphoma decrease by 34 points to 36% after a Phase I study was suspended.
The Phase I trial’s (NCT05627245) status was updated from recruiting to suspended on ClinicalTrials.gov on 19 September, with GlobalData evaluating the product on the following day.
The purpose of the study was to test the safety, side effects, and best dose of the combination of Ipsen’s Tazverikin (tazemetostat) and Beleodaq in treating subjects with lymphomas. According to ClinicalTrials.gov, the trial was suspended by the National Cancer Institute because it was pending amendment.
The East Windsor, New Jersey-based Acrotech is developing Beleodaq, an inhibitor of histone eacetylase, for the treatment of various cancer indications. The drug is approved for treating relapsed or refractory peripheral T cell lymphoma (PTCL).
Premature ovarian cancer candidate chances drop after Phase I suspension
Vitti Labs’s EV-PURE saw its PTSR in premature ovarian failure decrease by 31 points to 27% after a Phase I study was suspended.
The Phase I trial’s (NCT06072794) status was updated from recruiting to suspended on the ClinicalTrials.gov on 19 September, with GlobalData evaluating the product on the following day.
The purpose of the study was to evaluate the efficacy and safety of EV-PURE in premature ovarian failure. According to ClinicalTrials.gov, the trial was suspended following a request from the sponsor.
EV-PURE elicits therapeutic intervention by an undisclosed mechanism of action and is under development for the treatment of chronic obstructive pulmonary disease (COPD), osteoarthritis, traumatic brain injury, Crohn’s disease, COVID-19-associated acute respiratory distress syndrome, and Alzheimer’s disease.
Read the last edition:
Read OnNeed 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.
