Pfizer confirms post-election status for COVID-19 vaccine
Pfizer said late last week that it will not ask the FDA for emergency-use authorisation (EUA) of its investigational COVID-19 vaccine (which is being co-developed with BioNTech) before next month’s US Presidential election. Pfizer may have efficacy data in hand by that point, but will not have the required safety data until late November, confirmed CEO Albert Bourla.
In a poll of 61 US infectious disease specialists, 43% told FirstWord they would be less confident in the effectiveness of any vaccine approved ahead of election-day. More here.
The US government said this week it is nevertheless hopeful of having enough supply of one or more effective vaccines for seniors and healthcare workers by the end of the year.
Other COVID-19 news
It is reported that the US trial of AstraZeneca's COVID-19 vaccine AZD1222 is expected to resume imminently after the FDA completed its review of a serious adverse event that had prompted a hold on global testing last month. The Phase III trial has been suspended in the US since September 6, after a participant in the company's UK trial fell ill with what was suspected to be transverse myelitis. Whilst testing has since resumed in the UK and other countries, it remains stalled in the US, where the FDA has been conducting its own review of the incident.
In a note to investors, analysts at Jefferies speculated this week that data from the UK trial of AZD1222 may read out by mid-November or early December, earlier than expected due to an escalating COVID-19 infection rate in the UK since mid-September.
The UK government has also signed a contract with Open Orphan's hVIVO subsidiary to develop a model for human challenge studies of COVID-19 vaccines, and is supporting the effort with an investment of £33.6 million pounds ($43.5 million). The government said that vaccine candidates "proven to be safe in initial trials" will be given to "carefully selected" volunteers. Using controlled doses of virus, researchers will first try to determine the smallest amount it takes to cause COVID-19 infection in small groups of healthy young people, aged between 18 and 30 years, with up to 90 volunteers involved at this stage. The participants will be continuously monitored to see exactly how the vaccine works and to identify any side effects. More here.
Elsewhere, interim findings from the World Health Organization's (WHO) global Solidarity trial suggest that Gilead Sciences' Veklury (remdesivir) and other drug regimens tested had "little or no effect" on 28-day mortality or the in-hospital course of COVID-19 among hospitalised patients. More here.
Biogen all in on aducanumab
With an FDA advisory committee a matter of weeks away, the shadow of aducanumab – Biogen's experimental Alzheimer's disease treatment – looms over the company and its outlook.
The outcome of this meeting – to be held on November 6 and a potential precursor to US approval of aducanumab – looks like an increasingly 'make or break' moment for the biotech.
The company presented its third-quarter results on Wednesday, showing that key franchises such as Tecfidera (multiple sclerosis) and Spinraza (spinal muscular dystrophy) are under increasing pressure from competitors. To make matters worse, Biogen confirmed that it is scrapping once promising R&D projects in both of these areas.
Mixed data for CRISPR's allogeneic CAR-T therapy
This week, CRISPR Therapeutics released the first results from its closely watched allogeneic CAR-T cell platform, with early data from the CARBON study of CTX110 in patients with CD19-expressing B-cell malignancies.
However, its search for unequivocal validation came up short, as the company's otherwise acceptable demonstration of efficacy was complicated by a sub-optimal safety profile.
Results were reported from 11 patients who received one of four ascending doses of the cell therapy, finding that dose group 1 produced "suboptimal" efficacy, with three of three patients experiencing progressive disease within one month. Responses improved in dose group 2, where responses were observed in two of three patients, but ultimately resulted in progressive disease. In dose group 3, four of four patients responded, with two ongoing complete responses (CRs) after about three months of follow-up.
But results from dose group 4 were more concerning; while a 74-year old patient with transformed follicular lymphoma had achieved a CR within one month, he subsequently developed febrile neutropaenia, confusion and memory loss, and reactivation of HHV-6 infection. The patient was treated for immune effector cell‐associated neurotoxicity syndrome (ICANS), and died about two months following CTX110 administration.
More analysis here.
Tracking a potential Keytruda 'killer'
Having passed an interim analysis looking at response rate, a head-to-head study evaluating Merck KGaA and GlaxoSmithKline's bintrafusp alfa against Merck & Co.'s PD-1 inhibitor Keytruda in high PD-L1 metastatic NSCLC patients will continue. Despite some confusion, FirstWord understands the cohort enrolled in this study has not been expanded, suggesting confidence that its current size is sufficient to show a benefit in favour of bintrafusp alfa.
LUNG037 is evaluating bintrafusp alfa, a potential first-in-class TGF-beta/PD-L1 bi-functional fusion protein, head-to-head against Keytruda. Keytruda monotherapy is the standard-of-care treatment for advanced NSCLC patients whose tumours express PD-L1 at levels of 50% or higher. The decision to run a head-to-head study against such a strong incumbent is described by analysts at Wolfe Research as a "brave, go for broke" trial design.
Although there is a high level of uncertainty around the outcome of this study, key opinion leaders describe it as being well designed and likely to change treatment for patients with high PD-L1 expression if positive.
As one expert noted, "when you've got a trial like that, it's make or break. If it turns out better than Keytruda, you then potentially have a huge market, if you've got a survival or PFS difference."
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