News

Novartis has decided to price its new gene therapy for spinal muscular atrophy using a formula that measures the performance of the drug over five years. The therapy, Zolgensma (onasemnogene abeparvovec-xioi), is a one-time treatment for a genetic disease that affects a child’s ability to swallow and breath, frequently leading to death.

The US Food and Drug Administration has approved a gene therapy to treat children under the age of two years who have spinal muscular atrophy, a rare genetic disease that affects a child’s ability to swallow and breath, frequently leading to death.

Evotec SE is to expand its pharmaceutical services business into biologics with the acquisition of Just Biotherapeutics Inc of Seattle, Washington, US. The deal will give the Germany-based company a capacity to discover and develop protein therapeutics in addition to its existing small molecule business. Evotec will pay an initial $60 million for the company, a figure that could rise to $90 million depending on the achievement of certain milestones within the next three years.

Syncona Ltd, an active investor in cell and gene therapy, has launched a new company, Quell Therapeutics Ltd, which will develop cell therapies to treat autoimmune conditions based on engineered T regulatory (Treg) cells. Tregs are a subset of T cells with the potential to down-regulate the immune system.

Rinri Therapeutics Ltd, a spin-out of Sheffield University in the UK, has received £1.4 million in seed funding from a European syndicate in order to advance a prospective stem cell therapy to restore hearing. The company aims to reverse neuropathic sensorineural hearing loss by repairing the damaged cytoarchitecture of the inner ear.

Storm Therapeutics Ltd has raised an additional £14 million in Series A money bringing the total for the early investment round up to £30 million. The new money includes funding from the new investor, Seroba Life Sciences, as well as from existing investors, Cambridge Innovation Capital, M Ventures, Pfizer Ventures, Taiho Ventures and IP Group

The extension will enable to the company to advance its preclinical pipeline of products that modulate RNA modifying enzymes for oncology.

A new French company working in the field of stem cell culture has raised €7.1 million in a Series A financing round to advance its technology for mass producing stem cells. TreeFrog Therapeutics SAS was founded in November 2018 on the basis of years of academic research.

Following a first quarter in which revenue increased by 27%, Evotec SE expects its business will grow by 10% this year – confirming earlier forecasts. The march forward reflects continued demand for its drug discovery services as well as ongoing milestone payments from drug development partners such as Bayer AG, Boehringer Ingelheim GmbH and Second Genome Inc.

AstraZeneca Plc’s Bruton tyrosine kinase inhibitor Calquence, already approved in the US for mantle cell lymphoma, has delivered positive results in a Phase 3 trial of previously-treated patients with chronic lymphocytic leukaemia. The trial showed a statistically-significant and clinically-meaningful improvement in progression-free survival and will stop early, the company announced on 6 May. Calquence was administered as a monotherapy and compared with a combination of rituximab and a physician’s choice of either idelalisib or bendamustine.

MorphoSys AG has reported positive data from a single-arm Phase 2 study of its investigational monoclonal antibody tafasitamab with the cancer drug lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma. The drug, previously known as MOR208, is being prepared for a regulatory submission to the US Food and Drug Administration by the end of the year.