Switzerland-based Santhera Pharmaceuticals Holding AG is to work with Peter Yurchenco of Rutgers University, US on a novel gene therapy approach for treating a congenital muscular dystrophy caused by mutations in the LAMA2 gene. Santhera has entered into two agreements, the first with the university and the second with Prof Yurchenco, to research gene replacement as a therapy for LAMA2-related muscular dystrophy.
Belgium-based Bone Therapeutics SA has extended its financial runway into the second quarter of 2021 after negotiating equity, bond and loan deals that yielded €15 million in new capital. The proceeds will be used to advance two lead assets for bone health into late-stage development.
BioMarin Pharmaceutical Inc has taken steps to extend its footprint in gene therapy with a preclinical collaboration with Dinaqor AG of Switzerland to develop therapies for rare genetic cardiomyopathies. Cardiomyopathies are diseases of the heart muscle, a significant proportion of which are inherited. Dinaqor will received an undisclosed upfront payment from BioMarin and is eligible for development, regulatory and commercial milestones. BioMarin is also investing in the Swiss company.
Sales of the glucagon-like peptide 1 (GLP-1) drugs for diabetes lifted Novo Nordisk A/S in the first quarter, more than offsetting the slow growth of the company’s insulins. Group sales amounted to DKK 33.9 billion (€4.54 billion) up by 16% in actual exchange rates and by 14% at constant rates. The operating profit was DKK 16.3 billion, up by 14%, giving an operating margin of 48.1%. The wide margin reflected low administrative costs and relatively low R&D spending. While spending on R&D increased by 41% from a year earlier, as a percentage of sales it was a smaller 11.1%.
Galapagos NV increased its staff by 45% in the year to 31 March in anticipation of the launch of its first pharmaceutical, filgotinib, on global markets. The Belgian company reported an operating loss of €44.6 million in the first quarter, but this was dwarfed by cash and cash equivalents of €5.7 billion, the result of successful equity offerings and a lucrative partnership with Gilead Sciences Inc for filgotinib, which is in registration for rheumatoid arthritis.
MorphoSys AG achieved a dramatic increase in liquidity in the first quarter following a new licensing deal with Incyte Corp and ongoing milestone payments from its partner Janssen Biotech Inc. Total liquidity amounted to €1.1 billion at the end of the quarter, up from €357.4 million on 31 December 2019. At an analyst briefing on 7 May, Jean-Paul Kress, the chief executive, said the cash will finance the company’s commercial operations in the US ahead of the expected launch of the cancer drug tafasitamab. It will also pay for the in-licensing of new assets.
A new serology test designed to detect protective antibodies in people who have been exposed to the SARS-CoV-2 virus has been given emergency use authorisation by the US Food and Drug Administration, the developer Roche announced on 3 May. The test has also received a CE mark and is eligible for use in Europe. Roche said the test has a specificity of greater than 99.8% and a 100% sensitivity. The sensitivity of a diagnostic test is the proportion of true positives that are correctly identified whereas the specificity is the proportion of true negatives.
A drug originally approved to treat diabetes has now received clearance to also be used for patients with heart failure. The drug, Farxiga (dapagliflozin), is a sodium-glucose co-transporter 2 (SGLT2) inhibitor which works by helping the kidneys lower blood glucose levels. On 6 May, the US Food and Drug Administration authorised Farxiga to treat adults with heart failure with reduced ejection fraction with and without Type 2 diabetes.
The cancer charity Cancer Research UK has chosen a bispecific protein therapeutic as a new development project, electing to take the molecule, CB213, into a Phase I clinical trial in patients with solid tumours. The therapeutic is being developed by Crescendo Biologics Ltd, which announced a partnership with the charity on 5 May. Crescendo has a preclinical portfolio of molecules targeting a range of cancers. The molecules are small, and based on the variable domain of the heavy chain of an antibody.
Norway-based BerGenBio ASA has raised NOK 500 million (€45.4 million) in an oversubscribed private share placement to support the development of its oncology portfolio which focuses on AXL inhibition. The lead candidate, bemcentinib, is in five Phase 2 studies as a single agent, or in combination, in acute myeloid leukaemia and non-small cell lung cancer.