April 2023 • PharmaTimes Magazine • 9

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Discovery programme launched by Cystic Fibrosis Syndicate

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The Cystic Fibrosis (CF) Syndicate in Antimicrobial Resistance (AMR) has announced pivotal £3m funding for a Collaborative Discovery Programme. The initiative is designed to encourage drug discovery innovators in the development of new treatments for individuals with CF.

Funded by medical research charity LifeArc, the programme will support approximately five collaborative projects – each receiving £500,000 of funding. The aim for participants is to quickly establish new antimicrobial therapies to treat chronic respiratory infections.

Researchers within small and medium enterprises (SMEs) and academia across the world are eligible to apply, with projects expected to last up to two years. Applicants are requested to address patients' priorities for improving treatment set out in the CF AMR Syndicate's recently-published Target Product Profiles for Antimicrobials.

The programme also delivers a collaborative approach to drug development, with successful applicants being able to access expert advice from managing partners, including Medicines Discovery Catapult, Cystic Fibrosis Trust and LifeArc.

Dr Paula Sommer, head of research at Cystic Fibrosis Trust, explained how important the imitative is: "People living with CF are particularly vulnerable to antimicrobial resistance. New antimicrobials that target infections caused by microbes commonly associated with lung infections in CF are urgently needed."


AstraZeneca’s IMFINZI improves lung cancer survival

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AstraZeneca has announced positive results from its analysis of the ongoing AEGEAN phase 3, placebo-controlled trial among patients with resectable early-stage non-small cell lung cancer (NSCLC).

The study showed that being treated with AstraZeneca’s IMFINZI, in combination with neoadjuvant chemotherapy, before surgery and as an adjuvant monotherapy after surgery showed a statistically significant and clinically meaningful improvement in event-free survival. The research contrasted the therapy’s performance with neoadjuvant chemotherapy alone followed by surgery.

Furthermore, IMFINZI was well tolerated and no new safety concerns were recorded in the adjuvant and neoadjuvant settings. In addition, adding IMFINZI to neoadjuvant chemotherapy was consistent with the known profile for this combination and did not increase complications or adverse events. There was also no compromise in the patients' ability to undergo surgery versus chemotherapy alone.

Susan Galbraith, Executive Vice President, Oncology R&D at AstraZeneca, reflected: “Patients with resectable non-small cell lung cancer face unacceptably high rates of recurrence, despite treatment with chemotherapy and surgery. We have shown that adding IMFINZI both before and after surgery significantly increased the time patients live without recurrence or progression events.”

John V. Heymach, Professor and Chair Thoracic/Head and Neck Medical Oncology, added: “Treating patients early with durvalumab both before and after surgery delivers a significant and clinically meaningful benefit in resectable non-small cell lung cancer.”


HOT & NOT

NICE has published final draft guidance regarding the recommendation of routine NHS funding for onasemnogene abeparvovec.

The treatment is a potentially curative one-off gene therapy for treating a rare genetic condition, spinal muscular atrophy (SMA). It will be used specifically among babies aged up to 12 months, before they develop symptoms. The drug – also known as Zolgensma – is produced by Novartis Gene Therapies.


Valneva has announced further data emerging from a study of its inactivated COVID-19 vaccine, VLA2001.

Meanwhile, the Committee for Medicinal Products for Human Use of the European Medicines Agency issued a positive opinion for the use of VLA2001 in adults of 18 to 50 years of age. This opinion is based on the vaccine being given as a booster dose, when provided at least seven months after the primary vaccination.


A clinical trial led by the University of Birmingham has discovered that patients with relentlessly painful headaches – often known as idiopathic intercranial hypertension (IIH) – could potentially be treated with an injectable peptide. The therapy is typically used for individuals with type 2 diabetes.

The research revealed the findings from a phase 2 trial of the drug exenatide – a GLP-1 receptor agonist – as a candidate treatment for IIH.


Toxic proteins within the brain that cause Parkinson’s disease and Lewy body dementia have the potential to help develop new targeted therapies.

University of Nottingham experts – led by Dr Helen Miranda Knight – have discovered that the modification of ribonucleic acid (RNA) in human brain tissue is disrupted among neurodegenerative diseases. These conditions include Lewy bodies dementia and Parkinson’s disease.


Pfizer’s rimegepant has been rejected by NICE for treating migraine headaches. It had been proposed that the drug is used for acute migraine treatment among patients who have failed to respond to two or more triptans.

In appraisal documentation from NICE, rimegepant is not being recommended within its marketing authorisation for the prevention of episodic migraine in adults, who experience at least four attacks per month.


Researchers at the Cambridge Institute of Therapeutic Immunology and Infectious Disease and Wellcome Sanger Institute showed that following SARS-CoV-2 infection, cells in the lining of the lungs, nasal cells and immune cells in the blood show a blunted inflammatory response in obese patients. This produced suboptimal levels of molecules needed to fight the infection.

Since the start of the pandemic, there have been almost 760 million confirmed cases of SARS-CoV-2 infection. There were also 6.9 million deaths.