A Phase 2a study that was evaluating a potential treatment for paediatric patients with non-alcoholic fatty liver disease was stopped early in August after the first three patients showed an increase in liver fat and elevations in serum alanine transaminase (ALT) levels, raising concerns about liver toxicity.
Orchard Therapeutics Ltd has raised $150 million in a Series C financing round to advance its portfolio of ex vivo gene therapies, the most advanced of which is in registration trials for the treatment of an ultra-rare disease that damages the immune system.
The oversubscribed financing round was led by Deerfield Management Company LP with significant new investments from RA Capital Management and nine other funds. Existing investors also participated in the round including Temasek, Baillie Gifford and Cowen Healthcare Investments.
A study published in the journal Clinical Cancer Research concludes that elevated levels of kidney injury molecule-1 (KIM-1), a transmembrane protein in blood plasma, can predict renal cell carcinoma (RCC) incidence up to five years before a diagnosis.
An early warning of kidney cancer could make the difference between life and death. When diagnosed early, eight out of 10 people will survive for five years or more. But if the disease is diagnosed late, only one out of 10 survive, according to the charity Cancer Research UK.
Oncology drugs that promise to meet a major public health need may in future be eligible for a clinical development pathway that is both simpler and less costly than traditional methods, the US Food and Drug Administration announced on 10 August.
A study published in the American Journal of Respiratory and Critical Care Medicine by researchers at the Helmholtz Zentrum München and the University of Denver in the US suggests that idiopathic pulmonary fibrosis (IPF) may be related to an increase in extracellular vesicles that relay WNT5A signals to the lungs for an increase in connective tissue.
Extracellular vesicles are tiny pouches released by cells that can contain a large number of messenger substances, such as proteins and nucleic acids.
The US Food and Drug Administration has approved an RNA therapeutic developed by Alnylam Pharmaceuticals Inc to treat patients with a rare genetic disease characterised by the build-up of abnormal amyloid protein in peripheral nerves and other organs.
Artios Pharma Ltd, which is developing a novel approach for destroying cancer by acting on DNA repair mechanisms, has raised £65 million from venture capital companies in a “highly oversubscribed” series B round. Among its investors were four large pharmaceutical companies.
H. Lundbeck A/S, the Danish specialist in brain diseases, reported a 9.8% rise in second quarter revenue to DKK 4.7 billion (€631 million) while its operating profit rose by 21.7% in the period to DKK 1.66 billion. The buoyant results were attributed to large increases in sales of the company’s schizophrenia, major depression and hypotension drugs. However the effect of price rises, if any, was not disclosed.
For the first half year, Lundbeck’s revenue rose by 9.3% to nearly DKK 9.3 billion while its operating profit increased by 34% to DKK 3.5 billion.
Evotec AG reported double-digit increases in revenue for its drug discovery and service businesses for the 2018 first-half year, driven by new business from Aptuit, its recently acquired contract research unit. Revenue for the year is expected to grow by more than 30%.
Group revenue was €173.8 million for the first half, up by 67%. Drug discovery revenue was €32 million, up by 52%, while revenue from services to pharmaceutical partners was €163.3 million, up by 61%. There was an overlap of €21.5 million between the two units.
Allergan Pharmaceuticals International Ltd has exercised an option to develop and commercialise a pre-clinical drug candidate that uses the CRISPR gene editing tool to potentially correct mutations in Leber Congenital amaurosis (LCA), a serious eye disorder.