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Press Releases

Take a look at a selection of our recent media coverage:

Influenza vaccine approved for treatment and prevention in children aged one year and above

25th January 2023

Xofluza (baloxavir) has been approved in the EU for uncomplicated influenza and post-exposure prophylaxis for children aged one and older

A press release by the manufacturer Roche, states that Xofluza (baloxavir) is now approved for use for the treatment of both uncomplicated influenza and post-exposure prophylaxis of influenza in children from one year of age.

The human influenza viruses are known to cause regular epidemics creating a huge public health burden. Although influenza vaccines are available, there are also three classes of anti-viral agents that have also been used. The M2 proton channel blockers such as amantadine, neuraminidase inhibitors (e.g., oseltamivir) and finally, polymerase inhibitors like favipiravir. The influenza virus polymerase complex has become seen as a possible target for anti-viral agents and comprises three subunits: polymerase basic protein 1, polymerase basic protein 2 (PB2) and finally polymerase acidic protein (PA). These three subunits are highly conserved and PB2 is known to bind with the cap of the host cellular pre-messenger RNA and is subsequently cleaved by a cap-dependent endonuclease in the PA subunit. Xofluza contains the pro-drug baloxavir marboxil and inhibits the endonuclease activity of the polymerase acid protein.

Studies to date have shown that in adults, xofluza reduces the median time to the resolution of influenza symptoms by as much as 28 hours compared to placebo and the drug was originally indicated for use in patients from 12 years of age. The updated indication was based on the findings from two studies.

Xofluza studies in patients under 12 years of age

The first study, MiniSTONE-2 enrolled children between the ages of 1 and 12 years with a clinical diagnosis of influenza. Participants were randomised 2:1 to either a single dose of oral baloxavir or oseltamivir twice a day for 5 days. The results showed that Xofluza reduced the median time to symptom resolution to 138.1 hours compared to 150 hours with oseltamivir. The second trial examined the post-exposure prophylactic efficacy of xofluza and found that the risk of influzena was lower with baloxavir compared to placebo (adjusted risk ratio = 0.43, 95% CI 0.32 – 0.58).

The press release also cites another clinical trial designed to assess the safety and efficacy of baloxavir in healthy patients from birth to less than one year of age with influenza-like symptoms.

The updated information from the EMA can be found here.

Fidanacogene elaparvovec gene therapy effective for haemophilia B

11th January 2023

Fidanacogene elaparvovec is a novel gene therapy which has been found to effective and well tolerated in adult males with haemophilia B

In a press release from the manufacturer Pfizer, it has reported that its candidate gene therapy, fidanacogene elaparvovec, is effective in reducing the annualised bleeding rate (ABR) of total bleeds compared to a prophylaxis regimen with Factor IX (FIX) administered as part of usual care.

Haemophilia B is a rare, X-linked inherited bleeding disorders caused by mutations in the F9 gene, which results in missing or reduced production/function of clotting factor IX (FIX). The prevalence of haemophilia B is 1 in 40,000 live males although female carriers may also show some signs of bleeding. An absence or reduced level of of FIX can result in spontaneous bleeding into the joints, muscles or brain causing serious complications. Currently, the mainstay of treatment for haemophilia B involves replacement of factor IX although adeno-associated viral (AAV)-based gene therapy is one of the most emerging treatment approaches. Fidanacogene elaparvovec is a novel, investigational vector that contains a bio-engineered AAV capsid (i.e., protein shell) and a high-activity human coagulation FIX gene. The aim of such gene therapy is that once treated, individuals are able to produce FIX rather than having to regularly receive exogenous FIX. In a Phase 1/2a study, 15 adult haemophilia B patients were infused with 5 x 1011 vg/kg of fidanacogene elaparvovec and followed for at least 1 year. The study examined the ABR prior to and 52 weeks after the infusion. The results showed that the mean ABR during the first 52 weeks following fidanacogene elaparvovec infusion was 0.4 ± 1.1 compared to 8.9 ± 14.0 in the 52 weeks preceding infusion (p<0.001) and in fact, 12 patients reported zero bleeds in the 52 weeks post-infusion.

The press release rates to the BENEGENE-2 study, which was a single arm trial, designed to evaluate the efficacy and safety of fidanacogene elaparvovec in adult male participants with moderately severe to severe haemophilia B (defined by a Factor IX circulating activity of 2% or less). The primary outcome was the ABR for total bleeds from week 12 to month 15 post-infusion. In the trial, 45 eligible participants completed at least six months of routine exogenous FIX prophylaxis therapy during the study lead-in before receiving a single intravenous dose of fidanacogene elaparvovec (5e11 vg/kg).

Fidanacogene elaparvovec and annualised bleeding rate

The press release reports that the mean ABR for all bleeds was 1.3 for the 12 months from week 12 to month 15 compared to an ABR of 4.43 during the lead-in 6 months pre-treatment period, giving in a 71% reduction in ABR (p<0.0001) after a single dose of fidanacogene elaparvovec. 

For secondary endpoints, there was a 78% reduction in treated ABR (p=0.0001) and a 92% reduction in the annualised infusion rate (p<0.0001). The mean FIX activity was 27% after 15 months and 25% at 24 months and the mean steady-state FIX concentration was significantly higher than the pre-specified threshold of 5% (p<0.0001).

The press release also notes that Fidanacogene elaparvovec has been granted breakthrough, regenerative medicines advance therapy (RMAT) and orphan drug designations from the US Food and Drug Administration, as well as PRIority MEdicines (PRIME) and orphan drug designation from the European Medicines Agency.

CHMP recommends Enhertu for HER2-low breast cancer treatment

5th January 2023

The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on Enhertu for treating HER2-low breast cancer

According to a press release by the manufacturer AstraZeneca, the  Committee for Medicinal Products for Human Use (CHMP), has adopted a positive opinion recommending a change to the marketing authorisation for Enhertu. The drug can now be used as monotherapy for the treatment of adult patients with unresectable or metastatic HER2‑low breast cancer who have received prior chemotherapy in the metastatic setting or developed disease recurrence during or within 6 months of completing adjuvant chemotherapy.

The World Health Organisation estimates that in 2020, there were 2.3 million women diagnosed with breast cancer and which led to 685 000 deaths. In Europe alone in 2020, there were 531,086 cases of breast cancer that resulted in 141,765 deaths. The human epidermal growth factor receptor 2 (HER-2) is a receptor tyrosine-protein kinase normally involved in the proliferation and division of breast cells and HER2-positive breast cancers are an aggressive type of breast cancer that tend to grow faster and are more likely to spread. It is known that around 50% of all primary breast cancers show a low-level expression of HER2 (HER2-low), defined as immunohistochemically 1+ or 2+ and lack of HER2 gene amplification measured by in situ hybridisation. Moreover, this low HER2 expression is a promising new target for antibody-drug conjugates and Enhertu (which contains trastuzumab deruxtecan) is one such specifically engineered HER2-directed antibody drug conjugate.

Enhertu clinical efficacy

CHMP based its decision of data from the DESTINY-BREAST04 trial which compared previously treated patients with HER2-low (immunohistochemistry (IHC) 1+ or IHC 2+/in-situ hybridisation (ISH)-negative) unresectable and/or metastatic breast cancer with hormone receptor (HR) positive or HR-negative disease against standard of care physician’s choice of chemotherapy. The study found that Enhertu demonstrated a 49% reduction in the risk of disease progression or death versus physician’s choice of chemotherapy in patients with HER2-low metastatic breast cancer with HR-positive disease (PFS hazard ratio, HR = 0.51, 95% CI 0.40 – 0.64, p < 0.001). In addition, the median overall survival in the hormone receptor–positive cohort was 23.9 months in the Enhertu group and 17.5 months in the physician’s choice group (HR for death = 0.64, 95% CI, 0.48 to 0.86, p = 0.003).

The press release adds how the safety profile of Enhertu was consistent with previous clinical trials with the most common Grade 3 or higher treatment-emergent adverse events were neutropenia (13.7%), anaemia (8.1%), fatigue (7.5%), leukopenia (6.5%), thrombocytopenia (5.1%) and nausea (4.6%).

A summary of the CHMP provides details on the full indications for Enhertu.

mRNA-4157/V940 vaccine effective in advanced melanoma

21st December 2022

mRNA-4157/V940 which represents a personalised cancer vaccine has been found effective when combined with pembrolizumab in advanced melanoma

According to a press release by manufacturer, Moderna (in conjunction with Merck) the investigational, personalised cancer vaccine, mRNA-4157/V940, combined with pembrolizumab, was more effective than pembrolizumab alone, at reducing the risk of death or recurrence in patients with stage III/IV melanoma following complete resection. 

Melanoma of the skin is the 17th most common cancer worldwide and in 2020, there were 324,635 new cases and 57,043 deaths. Although patients diagnosed at stage 1 have an excellent prognosis, this drops significantly as the disease spreads. For example, regional melanoma (stage 3) has a 63.6% 5-year survival and this drops to 22.5% for those with stage 4 (metastatic) disease.

Pembrolizumab (brand name Keytruda) is a human, programmed death receptor-1 (PD-1) therapy and works to enable T cells to invade melanoma anywhere in the body. The drug is already licensed as monotherapy for the treatment of adults and adolescents aged 12 years and older with advanced (unresectable or metastatic) melanoma.

The press release relates to the KEYNOTE-942 study, which is an on-going phase 2b randomised study designed to assess whether postoperative adjuvant therapy with mRNA-4157/V940 and pembrolizumab improves recurrence free survival (RFS) compared to pembrolizumab alone in participants with complete resection of cutaneous melanoma and a high risk of recurrence. mRNA-4157/V940 is designed to stimulate an immune response by generating a specific T cell action based on the unique mutational signature of a patient’s tumour. In the trial, and following complete surgical resection, patients were randomised to receive mRNA-4157/V940 (nine total doses of mRNA-4157) and pembrolizumab 200 mg every three weeks up to 18 cycles (for approximately one year) or pembrolizumab alone. The primary endpoint of the trial was recurrence-free survival whereas secondary endpoints include distant metastasis-free survival and safety.

mRNA-4157/V940 preliminary efficacy data

The press release describes the results for157 patients with stage III/IV melanoma. The findings to data show that adjuvant mRNA-4157/V940 and pembrolizumab reduced the risk of recurrence or death by 44% (hazard ratio, HR = 0.56, 95% CI 0.31 – 1.08, one-sided p = 0.0266) compared with pembrolizumab alone.

In terms of safety, serious treatment-related adverse events occurred in 14.4% of patients who received the combination treatment compared to 10% with pembrolizumab alone.

In the press release, Stéphane Bancel, Moderna’s Chief Executive Officer, said ‘today’s results are highly encouraging for the field of cancer treatment. mRNA has been transformative for COVID-19, and now, for the first time ever, we have demonstrated the potential for mRNA to have an impact on outcomes in a randomised clinical trial in melanoma.’

The press release adds that the companies plan to discuss the results with regulatory authorities and initiate a Phase 3 study in melanoma patients in 2023.

Dupixent recommended for EU approval to treat moderate to severe prurigo nodularis

29th November 2022

According to a press release by the manufacturer, Sanofi, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the approval of Dupixent (dupilumab) in the EU to treat adults with moderate-to-severe prurigo nodularis who are candidates for systemic therapy. 

Prurigo nodularis (PN) is a skin disease that causes hard, pruritic nodules to form on the skin. The pruritus can be intense, causing people to scratch themselves to the point of bleeding or pain. It is categorised as a rare disease and one UK-based study estimated a prevalence of 3·27 patients per 10 000 population and the condition has a negative effect on patients’ quality of life.

Dupixent is a fully human monoclonal antibody that inhibits the signalling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways. The drug currently has several indications including the treatment of moderate-to-severe atopic dermatitis in adults and adolescents 12 years, as add-on maintenance treatment for severe asthma and as an add-on therapy with intranasal corticosteroids for the treatment of adults with severe Chronic rhinosinusitis with nasal polyposis

Dupixent clinical data

The efficacy data for Dupixent which led to the CHMP approval came from two randomised trials, PRIME and PRIME 2. In both PRIME and PRIME 2 the primary objective was to demonstrate the efficacy of dupilumab on itch response in patients with prurigo nodularis and which was inadequately controlled on topical prescription therapy or when those therapies are not advisable. The PRIME trial included adults with PN and at least 20 nodules and severe itch who were randomly assigned to 300 mg dupilumab subcutaneously after a 600-mg loading dose or to matching placebo, every two weeks for 24 weeks. The primary endpoint was the number of patients who experienced at least a 4-point reduction in the Worst-Itch Numerical Rating Scale (WI-NRS) score from baseline to week 24. Overall, 60% of dupixent patients achieved the primary endpoint, compared to placebo patients (18%, p < 0.0001). In the second trial, PRIME 2, the primary endpoint was the proportion who experienced a clinically meaningful reduction in itch from baseline at week 12. This occurred for 37% of Dupixent patients compared to 22% of placebo patients (p = 0.0216).

The press release also reports that Sanofi and Regeneron (a partnering biotechnology company) are studying the use of dupilumab in a broad range of diseases including paediatric eosinophilic oesophagitis, hand and foot atopic dermatitis, chronic inducible urticaria-cold, chronic spontaneous urticaria and chronic obstructive pulmonary disease with evidence of type 2 inflammation.

RSV vaccine shows high efficacy in older adults

19th October 2022

An RSV vaccine provided a high overall efficacy in older adults especially among patients with a severe lower respiratory tract infection

RSV vaccine candidate, RSVPreF3, manufactured by GSK provided a high overall efficacy in a phase 3 trial among older adults according to a press release by the manufacturer.

Respiratory syncytial virus (RSV) is one of the common viruses that cause coughs and colds and causes severe respiratory illnesses in infants and older adults who frequently require hospitalisation. The condition creates a severe disease burden upon sufferers and the global number of hospital admissions for RSV-acute respiratory infections (ARI) in older adults has been estimated at 336000 and which lead to an estimated 14 000 in-hospital-related deaths. Currently, not a single RSV vaccine has been approved although in September 2022, it was reported that Pfizer was ready to file for FDA approval of its candidate RVS vaccine.

The GSK press release relates to their randomised, placebo-controlled trial which was designed to evaluate the efficacy of the RSVPreF3 at preventing lower respiratory tract disease (LRTD) caused by RSV in adults ≥ 60 years of age following a single dose of the vaccine. GSK announced preliminary findings from the study in June 2022 although no data was presented in the release apart from the fact that the vaccine showed statistically significant and clinically meaningful efficacy in adults aged 60 years and above. While the current findings have yet to be published in a peer-reviewed article, the press release does provide plenty of data.

RSV vaccine results

The findings show that among those assigned to RSVPreF3 there were 7 lower respiratory tract RVS cases compared to 40 in the placebo group, giving an overall vaccine efficacy of 82.6% (96.95% CI, 57.9 – 94.1, p < 0.0001). The vaccine also demonstrated a high efficacy against both type A (84.6%) and type B (80.9%) RVS.

In addition, there was a consistently high vaccine efficacy across a range of pre-specified secondary endpoints. For example, among those with a severe lower respiratory tract infection, there was only 1 case in the RSV vaccine group compared to 17 in the placebo arm, giving an efficacy of 94.1% (95% CI, 62.4 – 99.9, p < 0.0001). Among patients with pre-existing comorbidities, such as underlying cardiorespiratory and endocrino-metabolic conditions, vaccine efficacy was 94.6% (95% CI, 65.9 – 99.9) and 93.8% (95% CI, 60.2-99.9) in adults aged 70-79 years.

Among patients with RSV confirmed acute respiratory infections, vaccine efficacy whilst lower, was still high with an overall efficacy of 71.7% and again, this was similar for type A (71.9%) and type B (70.6%).

The RSV vaccine was also well tolerated with observed solicited adverse events typically mild-to-moderate in severity and transient, with the most frequent being injection site pain, fatigue, myalgia, and headache.

The company expects to undertake regulatory submissions based on the phase III data in the second half of 2022.

Sotorasib and panitumumab effective in treatment-resistant metastatic colorectal cancer

21st September 2022

Sotorasib with panitumumab provided a 3-fold higher objective response rate than sotorasib mono-therapy in metastatic colorectal cancer

Sotorasib and panitumumab are a safe and effective combination for patients with chemo-refractory metastatic colorectal cancer according to the findings of a phase 1b trial presented at the European Society for Medical Oncology (ESMO) Annual Meeting in Paris, France.

Colorectal cancer (CRC) is the third most common cancer type worldwide and in 2020 there were an estimated 1.93 million cases and 916,000 deaths. However, among patients with metastatic disease, fewer than 20% survive beyond 5 years from their diagnosis. KRAS is a commonly mutated oncogene in CRC, and present in approximately 40% of all cases and sotorasib is a small molecule that selectively and irreversibly targets KRAS. Panitumumab is the first fully human monoclonal antibody directed against the epidermal growth factor receptor, leading to increased apoptosis, reduced proliferation of tumour cells and reduced angiogenesis with the result that tumour growth so that the development of metastases are prevented. Moreover, in patients with wild-type KRAS tumours, panitumumab is a useful option in combination with chemotherapy for the first- and second-line treatment of metastatic colorectal cancer.

In a previous single-arm, phase 2 trial, adult patients with KRAS mutated advanced colorectal cancer, mono-therapy with sotorasib led to a 9·7% overall response rate. In the current phase 1b trial, researched assessed the impact of adding panitumumab and eligible patients were those with refractory metastatic colorectal cancer on or after prior fluoropyrimidine, oxaliplatin, irinotecan and an anti-angiogenic agent. The researchers set the primary endpoint as safety but also examined the secondary endpoints of anti-tumour activity, progression-free survival, overall survival and pharmacokinetics.

Sotorasib objective response rate

While the trial is on-going, to date 40 patients (median age 57.5 years, 75% female) have been enrolled and received oral sotorasib 960 mg daily and panitumumab 6 mg/kg IV every 2 weeks. Overall, patients had a median of 2 prior lines of chemotherapy. In an associated press release by the manufacturer, Amgen, the median duration of treatment was described as 5.9 months with 25% of patients remaining on treatment at the time of data cut-off. With a median follow-up time of 8.8 months, the press release reports that median overall survival (OS) was not yet reached.

The confirmed objective response rate in the current trial was 30% (95% CI 16.6 – 46.5) and the disease control rate was 90% (95% CI 76.3 – 97.2). In addition, tumour shrinkage of any magnitude was observed in 87.5% of patients.

Treatment-related adverse events of any grade occurred in 92.5% of patients with grade 3 adverse events in 22.5% of patients although no adverse events were greater than grade 3 or led to treatment discontinuation.

The authors concluded that the combination therapy provided a 3-fold higher response than previously observed with sotorasib mono-therapy.

In the press release, David M. Reese, executive vice president of Research and Development at Amgen said ‘we are thrilled to see these CodeBreaK 101 results, which show that LUMAKRAS (sotorasib) plus Vectibix (panitumumab) achieved a 30% confirmed objective response rate in patients with KRAS G12C-mutated metastatic colorectal cancer. Treatment response rates can be as low as 2% in this patient population, and the current standard of care offers a median progression-free survival benefit of two months, so developing new treatment options is critically important for patients.’

Abstract
Sotorasib in combination with panitumumab in refractory KRAS G12C-mutated colorectal cancer: Safety and efficacy for phase Ib full expansion cohort

RSVpreF vaccine candidate effective against respiratory syncytial virus

31st August 2022

RSVpreF a vaccine candidate against respiratory syncytial virus showed a high level of efficacy in older adults with more severe infection

Pfizer’s vaccine candidate RSVpreF for respiratory syncytial virus (RSV) showed a high level of efficacy in older patients with more severe lower respiratory tract illness which was defined by three or more RSV-associated symptoms according to a press release from the company.

RSV is a major viral pathogen causing severe lung disease in the adult population, particularly among the elderly and which constitutes a substantial disease burden. The global number of hospital admissions for RSV-ARI in older adults has been estimated to be 336,000 leading to about 14,000 in-hospital deaths. Currently, there is no specific treatment for the virus apart from supportive care. RSV has two molecular subtypes A and B and RSVpreF is bivalent vaccine based on the crystal structure of pre-fusion F and which is a vital form of the viral fusion protein (F) that RSV uses to attack human cells. The vaccine itself contains two preF proteins which protect against the two main form of RSV, A and B which actually have multiple genotypes within each of them.

The press release provides top-line data from a phase 3 trial, RENOIR, designed to assess the efficacy, immunogenicity and safety of RSVpreF in adults. While RENOIR is not complete, the release describes how to date, the trial has enrolled approximately 37,000 participants and who were randomised to receive 120μg RSVpreF or placebo in a 1:1 ratio. Enrolment up to approximately 40,000 participants continues in the Southern Hemisphere to accumulate cases during their first season.

The information in the press release describes the findings of a pre-planned, interim analysis to assess protection against RSV-associated lower respiratory tract illness defined by two or more symptoms. The analysis determined a vaccine efficacy of 66.7% (96.66% CI: 28.8% – 85.8%). Based on this positive result, the manufacturer turned to the more severe disease primary endpoint defined by three or more symptoms and where the vaccine efficacy was 85.7% (96.66% CI: 32.0% – 98.7%). Moreover, an independent, external Data Monitoring Committee indicated the investigational vaccine was well-tolerated, with no safety concerns.

Commenting on these interim findings, Annaliesa Anderson, senior vice president and chief scientific officer, Vaccine Research and Development at Pfizer said ‘Scientists and researchers have worked to develop RSV vaccines with little success for over half a century. These findings are an important step in our effort to help protect against RSV disease.’

Pfizer is also investigating the efficacy and safety of RSVpreF in infants born to women vaccinated during pregnancy.

Bivalent Moderna vaccine likely to protect against Omicron sub-variants

26th June 2022

A bivalent Moderna vaccine was found to produce a potent neutralising antibody response against two Omicron sub-variants BA.4 and BA.5

According to a press release a bivalent Moderna vaccine is able to generate a large neutralising antibody response against two of the Omicron sub-variants, BA.4 and BA.5 and therefore likely to protect against these variants.

Both the BA.4 and BA.5 sub-variants of Omicron were detected in South Africa in January and February 2022, respectively. Moreover, there have recently been concerns raised over these two sub-variants especially after a study published in the New England Journal of Medicine, found that the sub-variants, substantially escaped neutralising antibodies induced by both vaccination and infection. The results of the study showed that among individuals who had received a third (i.e., booster) dose of BNT162b, compared with the response against the original COVID-19 isolate, the neutralising antibody titre was lower by a factor of 21 against BA.4 or BA.5. In other words, it appears highly likely that even among those who have been fully vaccinated, the BA.4 and BA.5 can lead to re-infection.

The bivalent Moderna vaccine has been studied in a phase 2 and phase 3 trial which has yet to be published. In the study, all participants who had previously received 2 or 3 doses of an approved COVID-19 vaccine were then given mRNA-1273.529, mRNA-1273.214, or mRNA-1273 as the 4th dose.

Bivalent Moderna vaccine and Omicron sub-variants

According to the press release, for the COVID booster candidate, mRNA-1273.214, only one month after administration in previously boosted participants, a 50 µg booster dose elicited potent neutralising antibody responses against the Omicron sub-variants BA.4 and BA.5 in all participants. In fact, mRNA-1273.214 was able to increase the level of neutralising antibody titres against BA.4/BA.5 by 5.4-fold (95% CI 5.0 – 5.9) above baseline in all participants regardless of prior infection and by 6.3-fold (95% CI 5.7 – 6.9) in the subset of seronegative participants.

These results follow on from the data in a press release in early June 2022, in which Moderna announced that a booster dose with mRNA-1273.214 increased neutralising geometric mean titres (GMT) against the Omicron variant by approximately 8-fold above baseline levels. Moreover, a 50 μg booster dose of mRNA-1273.214 was well-tolerated in the 437 study participants and both the safety and reactogenicity profile of mRNA-1273.214 was similar to that of mRNA-1273 when these vaccines were administered as a second booster dose.

Based on these preliminary findings, Moderna is now working hard to complete all the necessary regulatory submissions in the coming weeks requesting to update the composition of the booster vaccine to mRNA-1273.214.

Paxlovid of no benefit to low risk patients with COVID-19

21st June 2022

Paxlovid is of no benefit to patients at a low risk of either being hospitalised or death after infection with COVID-19

According to the results of a press release from the manufacturer, Pfizer, paxlovid does not benefit patients who are either unvaccinated or vaccinated and who are deemed not at a high risk of severe complications such as hospitalisation or death if infected with COVID-19.

Paxlovid is a protease inhibitor antiviral therapy against COVID-19 and was developed to be taken orally, at the first sign of infection or at first awareness of an exposure. The early use of the drug could therefore help patients avoid severe illness and which might lead to hospitalisation or death, or avoid disease development following contact with an infected individual.

The June 2022 press release, follows on from the success of the results from an earlier trial, EPIC-HR, in which symptomatic, unvaccinated, non-hospitalised adults, at high risk for progression to severe COVID-19, were assigned in a 1:1 ratio to receive either 300 mg of nirmatrelivir plus 100 mg of ritonavir (i.e., Paxlovid) or placebo every 12 hours for 5 days.  The results of EPIC-HR were published in February 2022 and showed that treatment of symptomatic COVID-19 with paxlovid reduced the risk of progression to severe COVID-19 by 89% compared to placebo and without any major safety concerns. Based on these encouraging results, both the FDA and EMA approved the use of paxlovid for the treatment of mild-to-moderate COVID-19 in adults and children (12 years and older) who were deemed to be at high risk for progression to severe COVID-19, including hospitalisation or death.

With the success of their drug in high-risk patients, Pfizer proceeded with EPIC-SR, a phase 2/3 trial entitled, Evaluation of Protease Inhibition for COVID-19 in Standard-Risk Patients (EPIC-SR). This time, the trial was designed to evaluate efficacy and safety in patients with a confirmed diagnosis of COVID-19 infection and who were deemed to be at standard risk (i.e., having a low risk for either hospitalisation or death). Enrolled participants were randomised 1:1, to receive paxlovid or matching placebo orally twice daily for five days.

Paxlovid in low-risk patients

The latest press release provides an updated analysis of the results from the EPIC-SR trial. The trial’s primary endpoint was self-reported, sustained alleviation of all (COVID-19) symptoms for four consecutive days. In an earlier press release from December 2021, Pfizer reported that primary endpoint in EPIC-SR was not met although this was based on an analysis of only 45% of the trial’s planned enrolment. Based on an updated analysis of 1,153 patients enrolled through December 2021, the current press release re-affirms, the results of the earlier, interim analysis. The data show that there was a non-significant 51% relative risk reduction in the primary outcome and in a sub-group analysis of 721 vaccinated adults who had at least one risk factor for progression to severe COVID-19, there was also a 57% non-significant relative risk reduction in hospitalisation or death.

However, not all of the current data is negative. When performing an additional analysis of secondary endpoint data, treatment with Paxlovid resulted in a nominally significant 62% decrease in COVID-19-related medical visits per day across all patients, relative to placebo (p = 0.0228).

Following these results, the press release reports that Pfizer has decided to cease enrolment into EPIC-SR due to low rate of hospitalisation or death in the standard-risk population but will continue to evaluate treatment in populations with high unmet need.