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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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CRISPR-based asset gains momentum

Editas Medicine’s CRISPR-based asset EDIT-101 saw its PTSR in the rare ophthalmologic disorder Leber congenital amaurosis (LCA10) rise by five points to 55%, as of 4 October. This changed followed the announcement of positive interim data from a Phase I/II trial.

Initial results from the 18-subject Phase I/II study (NCT03872479) were published in a company press release on 29 September. The study sought to examine the safety and efficacy of single escalating doses of EDIT-101.

The trial measured the frequency of severe adverse events induced by EDIT-101 over a one-year timeframe as the primary outcome of which none have been noted so far, while mild events related to the drug’s subretinal injection were observed. Additionally, no dose limiting toxicities (DLTs) which would impair vision were reported.

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By GlobalData

Examining the study’s efficacy through metrics such as best corrected visual acuity (BCVA), two subjects in the mid-dose cohort were reported to have experienced in improvement in BCVA over six months.

Leber’s congenital amaurosis is a rare genetic eye disorder, which affects the retina and causes significant vision loss since infancy. The study also saw the candidate’s LoA grow by three points to 33%.

MacroGenics rises in multiple cancers

MacroGenics’ MGA271 (enoblituzumab) had its PTSR rise in multiple cancer indications on the back of a Phase I basket trial completion. Enoblituzumab’s PTSR jumped by 11 points to 76% in transitional cell cancer (TCC), a common type of bladder cancer. The asset’s PTSR also increased by 12 points in urethral and ureter cancers to 78%, as well as 12 points in squamous non-small cell lung cancer (NSCLC) to 77%.

The 145-patient Phase I’s (NCT02475213) status update went live on on 28 September. The asset’s PTSR was apprised the day after. The update also noted that the study was expanded from having two to four treatment arms and completed on 18 August. The two original arms investigated enoblituzumab with either Merck’s Keytruda (pembrolizumab) or MacroGenics’ own anti-PD1 MGA012 (retifanlimab). The two new arms investigated higher doses of enoblituzumab with Keytruda.

Enoblituzumab is a monoclonal antibody that targets B7-H3, a protein that may play a role in regulating immune response in various cancer types. Despite the PTSR changes, enoblituzumab’s LoA remained static at 1% in urethral and ureter cancers, though it increased by two points to 8% in squamous NSCLC and 1 point to 7% in TCC. The Phase I trial also recruited melanoma and head and neck cancer patients.

Merck’s Bavencio makes headway in lymphoma

A Samsung Medical Centre-sponsored study of Merck’s Bavencio (avelumab) saw its PTSR in natural killer T-cell lymphoma spike following an update extending the trial’s anticipated primary completion date to January 2022.

The Phase II study (NCT03439501) had its trial dates extended as per a 22 September update on The PTSR was updated on 5 October. In addition to extending the primary completion date to 30 January 2022, the study completion date had also been pushed back to 30 March 2022. The overall status had also changed from “enrolling by invitation” to “active, not recruiting”. Previously anticipating an enrollment of 33 anticipated subjects, the study now has an actual number of 21 participants.

Examining the rate of response of avelumab, as well as the efficacy of disease control, as the primary outcomes, the treatment cycle consists of two avelumab injections applied within 28 days. The study also saw the candidate’s LoA grow by six points to 33%.

Vedanta makes progress in infectious disease

Vedanta Biosciences’ microbiome-derived VE303 improved its chances of advancing to the next development stage in recurrent Clostridium difficile infections (CDI) by six points after its Phase II trial was completed. The drug’s PTSR reached 53% as of 30 September.

The Phase II trial (NCT03788434), dubbed CONSORTIUM, was designed to study VE303’s safety and efficacy in preventing recurrent CDI and identify a Phase III dose. CONSORTIUM recruited 79 participants and completed earlier in the month, as per a update posted on 29 September. VE303’s approvability, measured by the LoA metric, also increased by 2 points to 10%.

In July, Cambridge, Massachusetts-based Vedanta raised USD 68m with the intention of supporting a Phase III VE303 study in recurrent CDI. VE303 is the most advanced asset for the microbiome-focused company, which also has other several ongoing studies in other infectious diseases, cancer and inflammatory bowel disorders. In a previous Phase Ia/Ib trial (NCT04236778), VE303 demonstrated durable and rapid, dose-dependent colonization, as well as restoring gut microbiota after antibiotics.

Prescient bolstered by cancer trial design news

Prescient Therapeutics’ Phase I advanced cancer candidate PTX-100 saw its PTSR grow across six different oncological indications after new updates in the trial’s design. The PTSR rose by 13 points each to 64% in colorectal, pancreatic, and gastric cancers. It increased by eight points to 64% in cutaneous T-cell lymphomas, six points to 65% in angioimmunoblastic T-cell lymphomas and seven points to 64% in peripheral T-cell lymphomas (PTCL).

The 24-subject, open-label Phase I trial (NCT03900442) had its details changed in a 22 September update on, with the PTSR updated on 29 September. Seeking to examine the pharmacodynamics, pharmacokinetics, and safety of PTX-100 IV infusions, the update included the drug’s doses, ranging from 500 to 2000 mg/m2. Measuring the time and dose dependent PK of several PTX-100 doses in subjects over a 14-day timeframe as one of its primary outcomes, the update also included the revelation of biomarkers such as Tmax. The trial’s anticipated completion date had also been pushed to 30 April 2023.

The study also saw changes in the asset’s LoA in some of the six indications. For colorectal and gastric cancers, the LoA remained at 3%, as well as stagnating at 1% in angioimmunoblastic T-cell lymphoma. The LoA grew by one point to 1% in pancreatic cancer, by one point to 6% in cutaneous T-cell lymphoma and by one point to 8% in peripheral T-cell lymphoma.

Alzheimer’s disease approach likely to advance

Neuronascent’s NNI-362 for Alzheimer’s disease saw its PTSR leap 17 points to 61% following positive Phase Ia results.

The Clarksville, Maryland-based Neuronascent enrolled 56 healthy volunteers aged 50–72 years in the Phase Ia dose-escalation study (NCT04074837), which randomized subjects to placebo or varying doses of NNI-362. Safety was the study’s primary endpoint, and there were no serious or dose-dependent adverse events reported in a 29 September company press release.

NNI-362 can combat age-related neurodegenerative disorders through the restoration of lost neurons. These initial Phase Ia results did not change the drug’s LoA, which remains at 1%.

Allena gets bump in gout and kidney disease

Allena Pharmaceuticals’ ALLN-346 got a bump in its potential to advance to the next development stage after a Phase IIa study was initiated. The trial is enrolling patients with hyperuricemia and gout, and with mild-to-moderate chronic kidney disease (CKD). With the trial start, ALLN-376’s PTSR rose by 6 points to 32% as of 1 October.

On 29 September, Allena said it had dosed its first patient in the 24-patient Phase IIa study (NCT04987294). The trial is looking at the incidence of treatment-related adverse events as a primary endpoint and is also measuring serum urate changes as a secondary outcome. It is scheduled to complete in July next year.

Allena has another ongoing 24-patient Phase II study (NCT04987242) exploring the same drug in patients with hyperuricemia and gout. Oral ALLN-346 features an enzyme that can break down urate in the gastrointestinal tract, and thus aims to address hyperuricemia seen in several indications like gout and CKD. ALLN-346’s approvability, measured by the LoA metric, also rose by 1 point to 6%.

Adlai Nortye asset jumps in rectal cancer

Adlai Nortye’s palupiprant saw its PTSR spring nine points to 17% for rectal cancer after its Phase Ib trial completed.

The China-based biotech enrolled 29 participants with locally advanced rectum cancer in the open-label Phase Ib study (NCT03152370) of palupiprant with preoperative chemotherapy, according to a 4 October update to In addition to assessing the safety and tolerability of palupiprant, the dose-finding trial also looked for early efficacy signals in its expansion arm after determining the recommended Phase II dose.

Palupiprant is a small molecule prostaglandin E2 receptor EP4 subtype antagonist. The drug, also known as E7046, was the focus of a Phase I trial (NCT02540291) run by Eisai. However, the trial was terminated after Eisai licensed the drug to Adlai Noryte, granting the company exclusive rights to research, develop, manufacture and market palupiprant, according to a July 2018 update to The Phase Ib completion did not change the drug’s LoA, which remains at 0%.

Cystic fibrosis asset completes Phase II

Laurent Pharmaceuticals’ fenretinide for cystic fibrosis saw its PTSR bounce five points to 38% after its Phase II trial completed.

The Montreal, Canada-based biotech enrolled 166 patients with cystic fibrosis in the global, placebo-controlled Phase II APPLAUD trial (NCT03265288). The study, which investigated the absolute change in percent predicted forced expiratory volume in one second (FEV1) as a primary endpoint, completed enrollment on 15 September, according to a 5 October update to Patients underwent six 21-day dosing cycles of either placebo or treatment followed by seven days of drug-free periods, for a total of six months.

Fenretinide, also known as LAU-7b, has anti-inflammatory effects that can preserve lung function in CF and defend against resistant bacteria. The Phase II completion also resulted in a modest bump to the drug’s LoA, which jumped two points to 10%.