The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. This guidance provides clarity on the path to registration for AVXS-101 IT.

Clinical Development 52

Clinical Development Therapies Clinical Trials Treatment 52

The Pharma Data

JANUARY 13, 2021

Structure 3:1031-1039 (1995) 3 Waters J, Biocentury ; October 26, (2018) 4 NCT02273960; ClinicalTrials.gov ; “Study to Evaluate Safety and Efficacy in Adult Subjects With ITP (ITP)”; results posted April 1, 2019, updated July 29, 2019 and accessed Jan 11, 2021 5 Ferrant JL et al., International Immunol. (11):1583

Pharmaceuticals 52

Pharmaceuticals Small Molecule Vaccine Protein Expression 52

Alta Sciences

APRIL 17, 2024

Ketamine is a dissociative anesthetic approved in 2019 by the U.S. They work by binding to specific sequences of nucleotides present within the mRNA structure and can induce mechanisms that either decrease, restore, or modify protein expression. Its use in the treatment of substance use disorders is still under study.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

The Pharma Data

APRIL 8, 2021

Roche will present data from five studies from the EVRYSDI clinical development programme, which was designed to represent a broad spectrum of people living with SMA. The full range of data from Roche’s clinical development programme in neuroscience being presented at 2021 AAN include: Medicine and/or Therapeutic Area.

Clinical Trials 52

Clinical Trials Trials Disease Therapies 52

The Pharma Data

AUGUST 9, 2021

Enhertu is being further assessed in a comprehensive clinical development programme evaluating efficacy and safety across multiple HER2-targetable cancers, including breast, gastric, lung and colorectal cancers. 1 More than two million patients with breast cancer were diagnosed in 2020, resulting in nearly 685,000 deaths globally.

Trials 52

Trials Treatment Clinical Trials Protein Expression 52

The Pharma Data

SEPTEMBER 2, 2020

We are exploring potential optimisation of OCREVUS, our first-in-class B-cell therapy, in a large Phase IIIb clinical trial programme to evaluate the impact of higher dose OCREVUS on reducing disability progression in relapsing MS (RMS) and primary progressive MS (PPMS). In 2019, Roche invested CHF 11.7

Clinical Trials 52

Clinical Trials Disease Treatment Therapies 52

The Pharma Data

OCTOBER 28, 2020

In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons.

Protein Expression 40

Protein Expression Therapies Treatment Disease 40