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Pfizer and BioNTech have announced what the world has been waiting for – positive interim Phase III results for their mRNA-based COVID-19 vaccine, BNT162b2, suggesting that it is 90% effective in preventing infection.
While the full data have yet to be published in a peer-reviewed journal, the topline result is a huge step forward in the fight against COVID-19, and looks to be compelling enough to secure regulatory approval, which the companies said they would file for after the third week of November.
While China and Russia have already granted emergency approval for their own COVID-19 vaccines, this is the first time that any candidate has produced data from a Phase III efficacy trial.
Stock markets have surged on the news, as effective vaccines are seen as key to overcoming the pandemic, which has crippled the global economy. The SARS-CoV-2 virus has infected 50.5 million worldwide since the start of the year, and claimed the lives of over 1.26 million people.
The companies said BNT162b2 had demonstrated evidence of efficacy against COVID-19 in trial participants without prior evidence of infection in an interim analysis conducted on 8 November by an independent data monitoring committee (DMC).
The analysis was based on 94 patients showing symptoms of infection in the trial of 43,538 participants. While the companies have not revealed full details, they said that the case split between vaccinated individuals and those who received the placebo indicated a vaccine efficacy rate above 90%, at 7 days after the second dose. This means that protection is achieved 28 days after the initiation of the vaccination, which consists of a two-dose schedule.
The 90% rate is much higher than the 50% mark that the US Food and Drug Administration had stipulated as a minimum, although the companies noted that as the study continues, the final efficacy percentage may change.
Pfizer and BioNTech are continuing to accumulate safety data and currently estimate that a median of two months of safety data following the second (and final) dose of the vaccine candidate – the amount of safety data specified by the FDA in its guidance for potential Emergency Use Authorization – will be available by the third week of November. So far, the DMC has not reported any serious safety concerns.
“Today is a great day for science and humanity. The first set of results from our Phase III COVID-19 vaccine trial provides the initial evidence of our vaccine’s ability to prevent COVID-19,” said Pfizer’s CEO Albert Bourla.
“We are reaching this critical milestone in our vaccine development program at a time when the world needs it most with infection rates setting new records, hospitals nearing over-capacity and economies struggling to reopen.”
PFIZER'S ALBERT BOURLA
The Phase III readout is a remarkable achievement for the companies, having only started work on the vaccine together in March, accelerating the normal development times hugely with the collaboration of governments, regulators and trial sites globally.
The companies are also working to prepare the necessary safety and manufacturing data to gain US FDA approval for the manufacturing of the vaccine. Based on current projections the companies expect to produce globally up to 50 million vaccine doses in 2020 and up to 1.3 billion doses in 2021.
The allocation of these first doses 50 million doses will also be contentious, as the partners have signed a number of multi-billion dollar deals with the richest nations, but have not yet agreed to supply the COVAX facility, aimed at guaranteeing supplies for poorer nations.
Another major hurdle will be in shipping and storage of the vaccine, which requires storage at -70°C and is otherwise only viable for 24 hours at room temperature.
BIONTECH'S UGUR SAHIN
“The first interim analysis of our global Phase III study provides evidence that a vaccine may effectively prevent COVID-19. This is a victory for innovation, science and a global collaborative effort,” said Ugur Sahin, BioNTech co-founder and CEO.
“When we embarked on this journey 10 months ago this is what we aspired to achieve. Especially today, while we are all in the midst of a second wave and many of us in lockdown, we appreciate even more how important this milestone is on our path towards ending this pandemic and for all of us to regain a sense of normality."
Unlike many other frontrunners, the partners did not work under the US government led Operation Warp Speed initiative, but did align on clinical trial protocols including for targets for ethnic diversity in participants.
While the partners have already secured advance contracts around the world for the vaccine, their potential first-to-market status could help them accumulate many more orders. Analysts at Mizuho said they expected an EUA by December and revenues of $8.5bn in sales for the vaccine in 2020-2021 alone.
Pfizer and BioNTech revealed that after discussion with the FDA, they had decided to drop the first planned interim analysis, which would have occurred at 32 cases. News had been eagerly awaited at the end of October, but the partners chose to wait longer in order to increase their chances of generating compelling data at the first interim ‘peek’ at the results. (Also see "Pfizer's Bourla Urges Patience As COVID-19 Vaccine Data Timeline Lags" - Scrip, 27 Oct, 2020.)
That meant they elected to wait until a minimum of 62 cases had accrued. And by the time these discussions with the FDA had ended, the evaluable case count had reached 94 and the DMC performed its first analysis.
The trial is continuing and a final analysis is planned when a total of 164 confirmed COVID-19 cases have accrued.
In addition to the primary efficacy endpoint evaluating confirmed COVID-19 cases accruing from 7 days after the second dose, the final analysis now will include, with the approval of the FDA, new secondary endpoints evaluating efficacy based on cases accruing 14 days after the second dose as well.
The companies said these secondary endpoints will help align data across all COVID-19 vaccine studies and allow for cross-trial learnings and comparisons between novel vaccine platforms being used by COVID-19 vaccine developers. This will be significant as the first interim results from other vaccine developers read out. First among these is likely to be Moderna, also using an mRNA platform, which is also expected to unveil results this month.
Additionally, participants will continue to be monitored for long-term protection and safety for an additional two years after their second dose.
The companies also confirmed plans to submit data from the full Phase III trial for scientific peer-review publication.
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