This week in clinical trials: 12th to 16th February 2024

In this instalment of our week-by-week clinical trials round-up, we look at life sciences research and development announced during the week of 12th to 16th February, from early-stage onwards. During that week, pharmaphorum covered news that Pfizer/BioNTech’s COVID jab is to be sold privately in the UK, that the first drug therapy for frostbite had been cleared by the FDA, and that Onivyde had gotten a ‘practice-changing’ FDA OK in pancreatic cancer.

Here, there’s positivity to be shared in solid tumours, dermatology, and rare disease.

Read on further for ongoing weekly news from studies across the globe.

During the week of 12th to 16th February:

Immuno-oncology platform company Medigene AG announced selection of gastric cancer, ovarian cancer, and two types of soft tissue sarcomas – myxoid/round cell liposarcoma and synovial sarcoma – as the initial clinical indications for its lead candidate MDG1015.

MD1015 is a first-in-class, third generation T-cell receptor engineered T-cell (TCR-T) therapy targeting NY-ESO-1/LAGE-1a (New York oesophageal squamous cell carcinoma 1 / L Antigen Family Member-1-a), armoured and enhanced by costimulatory switch protein PD1-41BB in the context of HLA-A*02 (HLA, human leukocycte antigen).

Pre-clinical data presented last year at the AACR and ESMO conferences demonstrated the clear potential of MDG1015 to improve clinical outcomes in solid tumours and the indications were selected predominantly based on target and/or PD-L1 expression, with 34-60%, 35-55%, and 75-80% of gastric cancer, ovarian cancer, and the two subtypes of soft tissue sarcoma expressing NY-ESO-1/LAGE-1a, respectively.

Within the current treatment landscape, high NY-ESO-1 expression is associated with a worse prognosis and expression increases with advanced disease stage. When looking only at the initially selected solid cancer indications, it is estimated that over 100,000 patients in the world’s top eight economies could be eligible for treatment with MDG1015 based on yearly incidence, target expression, and HLA-A*02 positivity.

Boston, Massachusetts-based Artax Biopharma, a clinical-stage company focused on transforming the treatment of autoimmune diseases, announced that the first patient had been dosed in its Phase 2a trial evaluating the safety and biomarker responses AX-158 in a first proof of mechanism trial of the immunomodulator in the Nck blocker class in psoriasis.

Psoriasis is a common, immune-mediated disease that affects 125 million people globally. In the US 3% of the adult population, or more than 7.5 million adults, are affected.

Immunomodulation assists the immune system in maintaining healthy control, addressing the underlying driver of autoimmune diseases. Safety and efficacy results are expected in the second half of this year, but pre-clinical data supports the potential for AX-158 to “realise effective outcomes without the immunosuppression and the side effects associated with existing autoimmune disease therapies,” stated Rob Armstrong, PhD, Artax’s chief executive officer.

KalVista Pharmaceuticals announced that the Phase 3 KONFIDENT clinical trial met all endpoints for sebetralstat, a first oral on-demand therapy for rare genetic disease hereditary angioedema (HAE).

HAE results in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body, which can be life-threatening depending on the location affected.

There is substantial treatment burden for HAE patients, currently, with the majority of approved treatments administered by injection or infusion. Sebetralstat could offer a discreet method of medication, which could be taken also at the earliest signs of an attack developing.

An investigational novel, oral plasma kallikrein inhibitor, sebetralstat received Fast Track and Orphan Drug designations from the FDA, as well as Orphan Drug Designation and an approved Paediatric Investigational Plan from the EMA.

The Phase 3 KONFIDENT study was a randomised, double blind, event-driven, crossover clinical trial that met all primary and key secondary endpoints and demonstrated a favourable safety profile. HAE attacks treated with both 300 mg and 600 mg of sebetralstat achieved the primary endpoint of beginning of symptom relief significantly faster than placebo and, consistent with previous studies, sebetralstat was well-tolerated, with a safety profile similar to placebo.