A malaria vaccine, developed by researchers at Oxford University, has demonstrate high-level efficacy of 77% in a phase 2b trial – an ‘extremely positive result’.

Results from a phase 2b trial of the vaccine candidate – R21/Matrix-M – have been published on SSRN/Preprints with The Lancet.

In their findings, the Oxford researchers noted that their vaccine is the first to meet the World Health Organization’s (WHO) goal for a malaria vaccine with at least 75% efficacy.
The phase 2b trial conducted at the Clinical Research Unit of Nanoro (CRUN) in Burkina Faso recruited 450 participants aged five to 17 months from the catchment area of Nanoro, covering 24 villages with an approximate population of 65,000 people.

Two groups were allocated to received the R21/Matrix-M vaccine – either with a low dose or high dose of Novavax’s Matrix-M adjuvant – while the third control group received a rabies vaccine.

In the higher-dose adjuvant group, the vaccine demonstrated 77% efficacy and 71% efficacy in the lower-dose adjuvant group after 12 months of follow-up. No serious adverse events related to the vaccine were observed, the researchers noted.

After these positive results the trial was extended, with a booster vaccination to be administered before the next malaria season one year later.

bluebird bio is anticipating a potential lift of all clinical holds on its LentiGlobin gene therapy for sickle cell disease (SCD) following the re-classification of one of the previously reported suspected unexpected serious adverse reactions (SUSAR).

In February, bluebird bio said that it had received reports of a case of myelodysplastic syndrome (MDS) in a patient from group C of the phase 1/2 HGB-206 study of LentiGlobin (betibeglogene autotemcel; beti-cel) for SCD.

At the same time, bluebird bio said that another SUSAR of acute myeloid leukaemia (AML) had been reported in the HGB-206 study.

bluebird bio has now announced that after further assessment following the review of results from additional tests of the MDS case, the treating investigator has concluded the patient did not have MDS and revised the diagnosis instead to transfusion-dependent anaemia.

The biotech company said it is continuing to work with the treating investigator to identify the cause of the anaemia.

bluebird bio also reported last month that it is ‘very unlikely’ that the SUSAR of AML was related to the BB305 lentiviral vector (LVV) used for LentiGlobin gene therapy.

With this assessment, as well as the revision of the MDS case, bluebird bio is hoping to potentially see a lift of all clinical holds on the HGB-206 and HGB-210 clinical studies of LentiGlobin for SCD, as well as the holds on the HGB-207 and HGB-212 studies of beti-cel for beta-thalassaemia.

Biogen has announced that it will offer its investigational drug tofersen to patients with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) who have no other options from mid-July.

The move follows pressure from ALS campaigners and patients to open access to the medicine, which is still being evaluated in a phase 3 clinical trial.

In a statement, Biogen said that although it does not “believe it is fair to ask participants in this study to continue to receive placebo while other SOD1-ALS patients are offered access to tofersen, access could be provided as soon as the placebo-controlled study has ended”.

“Until the safety and efficacy have been established, we will prioritise early access for a subset of the most rapidly progressing patients with this rare, severe disease,” the company added.

Biogen is aiming to start offering compassionate use access to a subset of SOD1-ALS patients with the ‘most rapidly progressive disease’ after patients in the late-stage study on placebo transition to tofersen treatment.

This stage will come before the safety and efficacy of the drug are established, Biogen noted.

Following this, if the results from the phase 3 study show that tofersen is safe and effective and no additional studies are needed, Biogen will then launch an early access programme (EAP) for the broad SOD1-ALS population from autumn 2021.

Eli Lilly has revealed new early-stage data for its RET inhibitor Retevmo in new cancer types, as the company looks beyond the drug’s approved lung and thyroid cancer indications.

The phase 1/2 LIBRETTO-001 trial – presented at the virtual American Association for Cancer Research (AACR) annual meeting – enrolled 32 patients with 12 unique RET fusion-positive advanced cancer types, including pancreatic, colon, breast and salivary cancer, with 62.5% having gastrointestinal tumours.

Across all 32 patients who received Retevmo (selpercatinib), the researchers observed a confirmed objective response rate (ORR) of 47%, with the median duration of response not yet reached with a follow-up of 13 months. According to Lilly, responses were ongoing in 73% of responding patients in the study.

“We are excited to broaden the body of evidence for Retevmo in RET fusion-positive cancers beyond lung and thyroid tumours,” said David Hyman, chief medical officer, oncology at Lilly.

“These encouraging outcomes, including in difficult-to-treat GI malignancies, support a growing body of evidence that RET fusions are potentially actionable in a wide range of tumour types,” he added.

Hyman said that Lilly will discuss the new data in the broad range of cancer types with regulatory authorities this year.