The Pharma Data

SEPTEMBER 2, 2020

This decision was based on analyses from the pivotal RMS and PPMS studies presented at the American Academy of Neurology Annual Meeting 2019, which showed higher OCREVUS exposure was associated with lower B-cell levels and with greater control of disability progression, without impacting safety. In 2019, Roche invested CHF 11.7

Clinical Trials 52

Clinical Trials Treatment Disease Therapies 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

AUGUST 9, 2021

HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumours, including breast, gastric, lung and colorectal cancers. 3 HER2 protein overexpression may occur as a result of HER2 gene amplification and is often associated with aggressive disease and a poor prognosis in breast cancer.

Trials 52

Trials Treatment Clinical Trials Protein Expression 52

The Pharma Data

SEPTEMBER 22, 2020

About Zolgensma ® ( onasemnogene abeparvovec ) Zolgensma ® is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion.

Clinical Development 52

Clinical Development Therapies Clinical Trials Treatment 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

The Pharma Data

APRIL 8, 2021

EVRYSDI was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively. Food and Drug Administration (FDA) approved EVRYSDI for the treatment of SMA in adults and children 2 months of age and older. About OCREVUS ® (ocrelizumab).

Clinical Trials 52

Clinical Trials Trials Disease Therapies 52

The Pharma Data

JANUARY 15, 2021

3 HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumors including breast, gastric, lung and colorectal cancer. 2,5 In the U.S., it is estimated that 27,600 new cases of gastric cancer were diagnosed in 2020 and more than 11,000 people died from the disease.

Treatment 52

Treatment Trials Disease Therapies 52

The Pharma Data

OCTOBER 26, 2020

Increased infiltrates, expression of immune related genes and higher PD-L1 protein expression were observed across all patients suggesting a remodeling of the tumor microenvironment consistent with a switch toward a “hot tumor” phenotype. Clin Lung Cancer 2019; e601 – e608. 2019; 153(3):521-529.

Vaccine 40

Vaccine Trials Treatment Virus 40