Amgen has announced that its phase 3 trial evaluating once-daily oral Lumakras (sotorasib) met its primary endpoint of progression-free survival, demonstrating ‘statistical significance and superiority’ over standard of care Docetaxel chemotherapy in KRAS G12C-mutated non-small cell lung cancer (NSCLC).

The phase 3 CodeBreaK 200 trial assessed the safety and efficacy of Lumakras in 345 previously treated patients with KRAS G12C-mutated NSCLC who had received at minimum, prior platinum-based doublet chemotherapy and checkpoint inhibitor therapy.

Amgen has not yet shared any information on whether Lumakras improved overall survival, a key secondary endpoint of the study. The company did however release results from the CodeBreaK 100 phase 1/2 trial in patients with KRAS G12C-mutated advanced NSCLC in April, which demonstrated a two-year overall survival of 32.5% in patients treated with Lumakras.

KRAS G12C is the most common KRAS mutation in NSCLC, with around 13% of patients with NSCLC harbouring the KRAS G12C mutation. Treatment options are limited for NSCLC patients with the KRAS G12C mutation whose first-line treatment has failed to work or has stopped working, and the outcomes with other approved therapies are suboptimal, Amgen reported.

Research published in the Journal of Clinical Oncology found that relatively low-cost PCR tests were able to successfully detect endometrial cancer (EC) using samples taken from the cervix or vagina.

Developed by researchers at the University of Innsbruck in Austria and University College London, the tests could significantly reduce the time to diagnosis for patients with EC.

The study emphasisd that a simple test to triage women with at least equivalent accuracy to current standards, without the need for specialist referral, is ‘urgently needed’.

Early results demonstrated the test was as effective for all participants, regardless of menopausal status, age, stage of cancer, ethnicity and histology.

Most importantly, the test successfully detected all eight of the womb cancers that were mixed among 63 women who presented consecutively because of postmenopausal bleeding at 89% specificity, with only a few women without cancer receiving a positive result.

Not only does the data collected indicate that the test performs equally as well as other strategies used to screen and triage patients with EC, but the results also suggest that the test may have the potential to enable targeted, more frequent monitoring of individuals at risk of being diagnosed with EC.

Sanofi and Swedish Orphan Biovitrum’s (Sobi) Biologics License Application (BLA) for efanesoctocog alfa (BIVV001) for the treatment of haemophilia A has been accepted by the US Food and Drug Administration (FDA) for priority review.

The application is supported by data from the pivotal XTEND-1 phase 3 study, which showed adults and adolescents 12 years and older treated once weekly with efanesoctocog alfa experienced clinically meaningful prevention of bleeds and superiority to prior factor prophylaxis based on an intra-patient comparison.

Efanesoctocog alfa was well tolerated, and no inhibitor development to factor VIII was detected.

People living with haemophilia A do not have sufficient functioning factor VIII protein to help their blood clot. The condition occurs in around one in 5,000 male births annually, and more rarely in females.

Efanesoctocog alfa is a novel and investigational recombinant factor VIII therapy designed to extend protection from bleeds with once-weekly dosing.

The treatment was granted Breakthrough Therapy Designation by the FDA in May 2022, having received Fast Track designation in February 2021 and Orphan Drug designation in August 2017.

The European Commission (EC) also granted efanesoctocog alfa Orphan drug designation in June 2019.

Sanofi outlined that regulatory submission in the EU will follow the availability of data from the ongoing XTEND-Kids paediatric study, with both events expected in 2023.

The European Commission (EC) has granted BioMarin Pharmaceutical’s (BioMarin) Roctavian (valoctocogene roxaparvovec) gene therapy a conditional marketing authorisation (CMA) for the treatment of severe haemophilia A.

The authorisation is specifically for the treatment of adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).

People living with haemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and potentially life-threatening bleeds from even modest injuries.

Roctavian works as a one-off infusion delivering a functional gene that is designed to enable the body to produce Factor VIII on its own without the need for continued haemophilia prophylaxis, thus relieving patients of their treatment burden, relative to the therapies that are currently available.

The application was supported by a significant body of data from the Roctavian clinical development programme, including two-year outcomes from the global GENEr8-1 phase 3 study that demonstrated stable and durable bleed control.

This included a reduction in the mean annualised bleeding rate (ABR) and the mean annualised Factor VIII infusion rate.

Of the 8,000 adults with severe haemophilia A in the 24 countries within BioMarin’s footprint covered by the approval, there are an estimated 3,200 patients who will be indicated for Roctavian, the company said.

Novartis’ Scemblix (asciminib) has been approved by the European Commission (EC) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia (CML) in chronic phase, previously treated with two or more tyrosine kinase inhibitors (TKIs), the company announced.

CML is a type of cancer in which the body produces cancerous white blood cells and almost all CML patients have an abnormality known as the Philadelphia chromosome, which causes malignant white blood cells to proliferate.

Scemblix is the first CML treatment that acts as a STAMP inhibitor, specifically targeting the ABL myristoyl pocket. This treatment approach may help address resistance in patients with CML previously treated with two or more TKIs and overcome mutations at the defective BCR::ABL1 gene, which is associated with the over-production of leukaemic cells.

The approval is based on results from the pivotal phase 3 ASCEMBL trial, which demonstrated a near doubling in maternal mortality ratio (MMR) rate for patients treated with Scemblix compared to Pfizer’s Bosulif (bosutunib).

Moreover, the discontinuation rate due to adverse reactions of those treated with Scemblix was more than three times lower.

The results were confirmed in the 96-week longer-term follow-up where the MMR rate was more than double with Scemblix compared to Bosulif, and the discontinuation rate due to adverse reactions was 7.7% for Scemblix and 26.3% for Bosulif.