Pfizer, Sanofi, J&J among big pharmas boosting drug approval chances
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Regulatory roundup: Sanofi/Regeneron, J&J and Eyenovia boost approval chances with Phase III updates

By Manasi Vaidya 16 Aug 2021 (Last Updated August 17th, 2021 12:13)

Trial updates and data releases across the pharma space moved the needle when it came to several drugs’ approval and trial progress prospects.

Regulatory roundup: Sanofi/Regeneron, J&J and Eyenovia boost approval chances with Phase III updates
Credit: Halawi / Shutterstock.com

Need to Know:

GlobalData’s proprietary model uses a combination of machine learning and an algorithm to calculate an individual drug’s Phase Transition Success Rate (PTSR) and Likelihood of Approval (LoA). While LoA provides the probability of a drug ultimately receiving market authorisation, PTSR indicates the probability of a drug’s advancement to the next stage of clinical development. The model uses data points from the individual drugs, clinical trials, regulatory milestones, company, and financial databases.

Sanofi/Regeneron’s Libtayo sees 7% LoA bump

Earlier this month, Sanofi and Regeneron Pharmaceuticals stopped their Phase III trial with Libtayo (cemiplimab) in combination with chemotherapy, thereby boosting its LoA in non-small cell lung cancer (NSCLC) to 73%, as of 7 August.

The trial’s Independent Data Monitoring Committee (IDMC) recommended the Phase III study be stopped at an interim protocol-defined analysis after the combination reduced the risk of death by 29%, compared to chemotherapy alone, as per a 5 August press release. The result and subsequent trial stoppage increased the LoA by seven points, up from 66%. While Libtayo is currently approved as a first-line monotherapy to treat NSCLC with PD-L1 expression levels of greater than or equal to 50%, this Phase III study had enrolled patients with locally advanced or metastatic disease across PD-L1 expression levels.

Margenza PTSR soars to 60% in gastric cancer

The completion of a Phase Ib/II trial meant that MacroGenics’ Margenza (margetuximab) saw its PTSR jump by 16 points in HER2+ gastric and gastroesophageal junction (GEJ) cancers. The Phase Ib/II was investigating Margenza in combination with Merck’s Keytruda (pembrolizumab) and its listing on ClinicalTrials.gov was updated as ‘completed’ on 2 August.

The drug now has a PTSR of 60% in gastric cancer and 54% in GEJ, as of 5 August. Margenza is a HER2/neu receptor antagonist, which garnered an FDA approval in December 2020 for metastatic HER2-positive breast cancer patients who have received two or more prior lines of anti-HER2 regimens.

J&J’s congenital heart disease candidate looks poised for approval

Johnson & Johnson’s Opsumit (macitentan) also had its LoA rise by five points in congenital heart disease after its Phase III trial’s status was changed to ‘completed’ on ClinicalTrials.gov. The 142-patient Phase III RUBATO trial (NCT03153137) was designed to test Opsumit’s ability to boost exercise capacity through cardiopulmonary exercise testing versus placebo and was completed on 20 July as per the study’s listing.

This trial was conducted in adult and adolescent patients born with congenital heart disease who had undergone the palliative Fontan operation. The endothelin receptor antagonist Opsumit was FDA-approved in 2013 for pulmonary arterial hypertension.

Strong results boost approval chances for Pfizer’s hair loss drug

Pfizer’s PF-06651600 (ritlecitinib) saw its LoA jump by 10 points to 23% in alopecia areata after positive topline Phase IIb/III trial data was revealed. The LoA was updated on 6 August, two days after the ALLEGRO trial results were announced on 4 August.

As per the update, ritlecitinib’s 30mg and 50mg once-daily oral doses achieved the primary endpoint of proportion of patients with ≤20% scalp hair loss after six months in ALLEGRO. All 718 patients had at least 50% scalp hair loss at baseline. However, the 10mg ritlecitinib arm did not demonstrate statistically significant improvement over placebo. ALLEGRO and the long-term Phase III ALLEGRO-LT trial (NCT04006457) data will be submitted for ritlecitinib’s regulatory filings.

Outlook improves for Eyenovia vision therapy

Eyenovia’s pilocarpine for presbyopia saw its PTSR catapult 16 points up to 35% after its Phase III trial was completed. The PTSR score change occurred on 10 August after the study’s ClincalTrials.gov entry was updated from ‘enrolling’ to ‘completed’ on 5 August. Presbyopia is a type of age-related farsightedness.

The Phase III VISION-1 trial tested a 1% pilocarpine and a 2% pilocarpine solution administered via Optejet, an Eyenovia device intended to apply the medication efficiently and horizontally to the eyes. According to the topline results released on 15 June, a greater percentage of patients met the primary endpoint with the 2% pilocarpine solution compared to placebo (p<0.05). The completed trial also resulted in a 10-point boost in the drug’s Likelihood of Approval (LoA), which rose to 22%. Eyenovia also released positive topline study results on 15 June.

Study failure sees PTSR plummet for Servier/Novartis cancer therapy

On the other hand, the PTSR for Servier’s S64315 dropped 45 points to 21% once its Phase I trial in collaboration with Novartis was suspended due to lack of efficacy, as of 5 August 2021. As per a disclosure document released by Novartis on 21 June, the study was terminated following a temporary halt in 2019. Novartis then decided to end the study given the limited clinical activity observed with S64315 and the current competitive landscape for anticancer therapies, as per the same document. The terminated trial also resulted in a 5-point drop in the drug’s LoA in multiple myeloma, which fell to 2%.