Two experimental drugs that were being tested in people with dominantly inherited Alzheimer’s disease failed to meet their primary endpoint in a study sponsored by Washington University School of Medicine in St. Louis, US.
Called DIAN-TU, the study was designed to find out whether either treatment could slow the rate of cognitive decline and improve disease-related biomarkers in people known to have a genetic mutation for inherited Alzheimer’s disease.
Sanofi SA reported sales of €36 billion for 2019, up by 4.8% from a year earlier but net income plunged by 34.8% to €2.8 billion following a write-down of €3.6 billion largely due to weak sales of Eloctate, a product for haemophilia A. Eloctate and Alprolix, a product for haemophilia B, entered Sanofi’s portfolio in 2018 when it bought the Biogen Inc spin-out Bioverativ Inc. Both products are factor replacement therapies which are being challenged by new therapeutic modalities.
Denmark’s Zealand Pharma A/S has made a bid to acquire the assets of a US company in bankruptcy proceedings which, if approved, would give it a commercial footprint in the US diabetes market. The target company is Valeritas Holdings Inc which has an approved insulin delivery device in the US. The product, V-Go, is covered by Medicare and many commercial insurance plans.
A new gene therapy company, Ikarovec Ltd, has been launched in the UK to develop a treatment for diabetic macular oedema, the most common cause of sight loss in people with diabetes. The company’s founder Peter Widdowson was previously chief executive of Quethera, another UK ophthalmic gene therapy company which was sold to Astellas Pharma Inc in 2018.
Adaptimmune Therapeutics Plc has returned to Nasdaq to raise new capital with an underwritten public offering of 24 million American depositary shares (ADSs) at a price of $4 per ADS. The issue raised a net $89.8 million after underwriters exercised their option to purchase additional shares. The company will use the proceeds to advance the development of its portfolio of immunotherapies for cancer which it is doing independently and through partnerships.
Netherlands-based Azafaros BV has raised €25 million in a Series A financing round to advance compounds for rare metabolic disorders. The round was led by Forbion Capital Partners, with Participation from BioMedPartners and founding investor BioGeneration Ventures. Azafaros was created in 2018 on the basis of discoveries by scientists at Leiden University and Amsterdam University Medical Center.
The US Food and Drug Administration has issued new guidance for developers of gene therapies highlighting both the complexity of manufacture as well as the difficulty in establishing duration of response in clinical trials.
In six final guidances, the agency addresses these issues as well as the application of orphan drug regulations to gene therapy. Orphan drug eligibility is an issue because many gene therapies are being developed for rare diseases where the patient populations are small.
The European Medicines Agency has won a case in the Court of Justice of the European Union upholding its right to publicly disclose information from a pharmaceutical company’s application for marketing, once that product has been approved. The case concerns a decision by the EMA to grant a third party access to a regulatory dossier of PTC Therapeutics International Ltd in 2015 for a product for Duchenne muscular dystrophy.
GlaxoSmithKline Plc is to contribute adjuvant to an international project to create a vaccine against infections from the coronavirus which originated in China and is rapidly spreading elsewhere. The World Health Organization has declared the spread of the virus to be a public health emergency. GSK will be working with the Coalition for Epidemic Preparedness Innovations (CEPI), a group set up in 2017 to develop vaccines to stop future epidemics.
Sales of non-insulin treatments for diabetes helped sustain growth at Novo Nordisk A/S where turnover for 2019 amounted to DKK 122 billion (€16.3 billion), up by 9% on a reported basis from a year earlier and by 6% in constant exchange rates. Operating profit advanced by 11% to DKK 52.3 billion giving an operating margin of 43%.
Sales, distribution and administration costs increased during the year however spending on research and development declined partly due to a reversal of write-downs of inventory prior to the launch of the oral semaglutide drug Rybelsus.