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GlobalData’s Investigative News team reviews data generated by an in-house model that combines machine learning and its proprietary algorithm. Likelihood of Approval (LoA) provides the probability of a drug in securing market authorization; Phase Transition Success Rate (PTSR) indicates the probability of a drug advancing to the next stage of development. The model uses data points from individual drugs, clinical trials, regulatory milestones, company, and financial databases.

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Novartis’s SERD terminated in breast cancer

Novartis’s selective ER antagonist and degrader (SERD), LSZ102, had its PTSR dive by 33 points to 40% in ER-positive Her2-negative breast cancer. This is on the back of a Phase I/Ib LSZ102 trial termination. The Phase I/Ib (NCT02734615) was terminated by Novartis, the trial’s sponsor, according to an 11 October update posted on The PTSR was appraised the next day (12 October).

The trial investigated the oral LSZ102 as a monotherapy or in combination with Novartis’s own Kisqali (ribociclib) or Piqray (alpelisib) in patients with oestrogen receptor-positive (ER+) breast cancer who have progressed after endocrine therapy. Due to the Phase I/Ib termination, LSZ102’s LoA also dropped by six points to 7%.

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On 25 August, primary endpoint safety data was reported, showing LSZ102 was well tolerated as a monotherapy and with Kisqali, and had a manageable safety profile with Piqray. Preliminary LSZ102 clinical activity was also seen with the combination, particularly with Kisqali.

J&J reports positive RVS vaccine results

Johnson & Johnson’s (J&J) positive Phase IIb results for its RSV vaccine propelled the candidate ahead in its development journey with a 15-point rise in its PTSR. Its LoA also rose by 5 points edging to 15%.

On 2 October, the company announced the positive results where the trial met its primary and secondary endpoints as part of a late-breaking abstract at the virtual ID Week conference. The vaccine displayed an 80% efficacy rate in preventing RSV-associated lower respiratory tract disease in adults who are 65 years or older. The PTSR for the vaccine stands at 52%, as of 5 October.

The Phase IIb (NCT03982199) CYPRESS study evaluated a combination of the company’s investigational vaccine candidate Ad26.RSV.preF with a prefusion F (preF) protein designed to induce a more optimal response.

Based on signals seen in that trial, J&J launched a Phase III trial (NCT04908683) that will enroll approximately 23,000 adults who are 60 years and older at different sites across North America, Europe, Asia and the Southern Hemisphere. The vaccine has a Breakthrough Therapy designation for the prevention of lower respiratory tract disease caused by RSV in adults who are 60 years or older.

Enanta’s FXR agonist NASH plans revised

Enanta Pharmaceuticals’ farnesoid X receptor (FXR) agonist EDP-305 had its PTSR drop by 11 points to 42% in nonalcoholic steatohepatitis (NASH). On 4 October, the company announced it no longer plans to continue further internal development, and it will instead prioritize combination approaches via an outlicensing strategy for the asset. The same decision was made with its other FXR agonist EDP-297.

EDP-305’s PTSR was updated on 5 October. As a result of the company decision, the Phase IIb ARGON-2 trial (NCT04378010) studying EDP-305 monotherapy is discontinued, the announcement states. Based on available data from its ARGON development program, the 1mg EDP-305 dose provided the best balance between tolerability and efficacy. On the back of Enanta’s update, EDP-305’s LoA also dropped by 2 points to 6%.

Idorsia fails Phase III Fabry disease test

Idorsia Pharmaceutical’s Fabry disease candidate lucerastat saw its PTSR in neuropathic pain and Fabry disease on the heels of negative Phase III results. The PTSR fell by 31 points to 29% and by 36 points to 39%, in these indications, respectively.

The 118-subject Phase III study (NCT03425539) had its results announcement in a company press release on 11 October. GlobalData had its latest update on 12 October. The LoA also fell, dropping by 22 points to 21% in neuropathic pain and by 17 points to 19% in Fabry disease.

The trial sought to examine the use and effect of lucerastat in patients suffering from neuropathic pain caused by Fabry disease. While the press release noted a “substantial and consisted reduction of plasma Gb3”, the study saw no reduction in neuropathic pain, the primary endpoint, after six months. The study also investigated the change from baseline until the sixth month in plasma globotriaosylceramide (Gb3).

Garetosmab progress shot rise after trial completion

Regeneron Pharmaceuticals’ REGN2477 (garetosmab) saw its PTSR in fibrodysplasia ossificans progressive (FOP) spring by 40 points to 76% after the study’s completion. The LoA also grew, rising by four points to 7%. The Phase II study (NCT03188666) was completed as per a 6 October update on, with the trial status changing from “suspended” to “completed”. GlobalData had its latest result on 8 October.

The 44-subject study examined the safety and effects of REGN2477 on patients suffering from abnormal bone formations caused by FOP. Measuring the incidence and severity of adverse events from the drug’s first injection over a 28-week timeframe as the primary outcome, the study also sought to measure the change from baseline in heterotopic ossification in participants as the primary efficacy objective. In addition, the study also examined the time-weighted average of the change in daily pain caused by FOP through a daily numeric rating scale across a 28-week time frame.

Momentum builds for Eubiologics vaccines

Eubiologics’s vaccines for pneumococcal and meningococcal infections made gains in their potential to advance to the next development stage after completing their respective Phase II trials. The company’s pneumococcal conjugate vaccine EUC-003 saw its PTSR rise by 7 points to 47%, while the PTSR for meningococcal conjugate vaccine EUC-004 was also bumped by 11 points to 62%.

Both the respective Phase II trials—(NCT04830358) and (NCT04714229)— were completed as per updates made to their entries on 7 October. The new PTSR is as of 9 October. While the trial updates did not change EUC-003’s LoA, it did prompt a 5-point rise in EUC-004’s LoA to 25%.

EUC-003, also known as EuPCV15, consists of 15 serotypes of pneumococcal polysaccharides conjugated to the CRM197 protein. EUC-004 is the first four-valent meningococcal vaccine produced in Korea and consists of the four serotypes of meningococcal polysaccharide conjugated to the CRM197 protein.

Cerecin in migraine bolstered by trial update

Cerecin’s tricaprilin saw its PTSR in migraine grow by 12 points to 42% once its enrolment status was updated. The ongoing Phase II study’s (NCT04437199) status was updated from “recruiting” to “active, not recruiting” on a 10 October on GlobalData had its latest update on 11 October.

The Phase II trial is designed to examine tricaprilin’s efficacy and safety in treating participants suffering from frequent migraines, by measuring the change in the number of migraine headache in the third month of treatment. The study is also tracking the proportion of subjects with a 50% reduction from baseline in number of migraine headache days in treatment months one, two and three, as the secondary outcome.

Tricaprilin’s LoA in migraine remained firm at 28%. Tricaprilin is an oral version of a medium chain triglyceride designed to induce ketosis and thereby improve mitochondrial metabolism. This in turn is intended to address deficient glucose metabolism which is seen in neurological conditions. Apart from migraines, the Singapore-headquartered company is developing tricaprilin in Alzheimer’s disease, Parkinson’s disease and epilepsy.

Retinitis candidate see modest bump

4D Molecular Therapeutics’ 4D-125 for retinitis pigmentosa saw its PTSR bump five points to 47% after reporting interim data from an ongoing Phase I/II trial. 4D-125 is a gene replacement therapy for patients with genetically confirmed X-linked retinitis pigmentosa (XLRP) caused by mutations.

In the Phase I/II trial (NCT04517149), 4D-125 was well-tolerated in the eight patients treated to date, with no reported dose-limiting toxicities or serious adverse events, according to interim results presented at the American Society of Retina Specialists meeting on 10 October. Initial clinical activity via measurements of reduced photoreceptor loss was also observed, and the company announced plans to continue enrolling the trial.

Overall, the Phase I/II trial is expected to enroll 43 patients, with the incidence and severity of adverse events as the primary endpoint. Interim results also bumped the drug’s LoA by two points up to 23%.

T cell therapy advancement shot increases

Repertoire Immune Medicines’ trial initiation spurred its autologous T cell therapy’s PTSR to rise in cervical cancer, head and neck cancer squamous cell carcinoma (HNSCC), and melanoma. RPTR-168’s PTSR grew by ten points to 32% in cervical cancer and 31% in melanoma, and increased by seven points to 32% in HNSCC.

The Cambridge, Massachusetts-based company announced the dosing of the first patient in the Phase I portion of the 24-subject, open-label Phase I/II study (NCT04762225) in a 11 October release. GlobalData’s PTSR algorithm had its latest update on 12 October.

The trial seeks to investigate its safety and tolerability in patients suffering from relapsed metastatic HPV-16 positive tumors like cervical cancer and HNSCC, and melanoma. Examining the number of subjects with dose-limiting toxicities in a 28-day timeframe as the primary outcome, the study also measures the best overall response on a six-month timeframe as one of its secondary outcomes.

RPTR-168 is an autologous multi-targeted T cell (MTC) therapy tethered with IL-12 and can target five different antigens linked to HPV-16 positive tumor development. The T cell therapeutic’s LoA also rose, increasing by four points to 13% in cervical cancer, as well as by four and three points to 8% in melanoma and HNSCC, respectively.

Dacogen basket trial terminated

The termination of a University of Heidelberg-sponsored Phase I study of J&J’s Dacogen (decitabine) led to the drug’s PTSR plunge across nine oncological indications. The most significant drops in PTSR were of 30 points to 43% in penile and vulvar cancers and 29 points to 41% in vaginal cancer. Dacogen is currently used to treat myelodysplastic syndromes, and has approved generic alternatives.

The open-label Phase I study (NCT04252248) was terminated due to “organizational reasons” as per an update posted on 6 October on As of 8 October, Dacogen’s PTSR fell by 26 points to 38% and 40% in cervical and oral cavity (mouth) cancer, as well as by 25 points to 36%, 38% and 37% in head and neck cancer, uterine cancer and oropharyngeal cancer respectively. Similarly, it fell by 27 points to 38% in anal cancer.

The trial sought to investigate the efficacy and safety of Dacogen for patients suffering from HPV-caused anogenital and head and neck cancers. While initially aimed at enrolling 18 subjects, the study accrued only three.

The study also saw a reduction in Dacogen’s LoA in those indications. The biggest changes in the drug’s LoA included an eight-point drop to 11% in vaginal cancer, and a six-point drop to 8% in both vulvar and oropharyngeal cancers.

Kineret trial in intracerebral haemorrhage completed

A University of Manchester-sponsored Phase II study of Swedish Orphan Biovitrum’s Kineret (anakinra) saw its PTSR in intracerebral haemorrhage rise by six points to 52% after the trial’s completion. The 25-subject Phase II study (NCT03737344) was completed as per a 4 October update on GlobalData had its latest update on 6 October. The study saw the candidate’s LoA stand at 2%.

The trial sought to examine the use of Kineret, an immunosuppressive drug, in reducing blood inflammation levels in subjects who have previously suffered from a haemorrhagic stroke. The patients included in the study had to be admitted within eight hours of symptoms onset, suffering from no underlying macrovascular causes.

Measuring the oedema extension distance (OED) over a 72-hour timeframe as the primary outcome, subjects were injected with 100mg doses twice-daily within eight hours of symptoms onset. Amongst other secondary outcomes, the were tested for inflammatory markers from the baseline until day four.