Finance, Grants, Deals

European investors, including INKEF Capital and the Dementia Discovery Fund, have joined a large syndicate of other institutions to raise $42 million in Series A money for QurAlis Corp of Cambridge, US enabling it to progress new therapies in neurodegeneration. Besides INKEF and Dementia Discovery, the round was led by LS Polaris Innovation Fund and Mission BioCapital. QurAlis intends to use the funds to support development of therapies for amyotrophic lateral sclerosis (ALS) as well as the genetically related frontotemporal dementia.

Netherlands-based Kiadis Pharma NV has raised €17 million in two private share placements enabling the cell therapy company to advance products for patients with both liquid and solid tumours. On 6 May, not long after the financing completed, the company received notice from US Food and Drug Administration that it could proceed with a Phase 2 study of its lead product – an adjunctive cell therapy for blood cancer patients undergoing a haploidentical haematopoietic stem cell transplant.

The UK Medical Research Council, a public body, and LifeArc, an independent charity, are making £16 million available to establish a network of centres that will offer clinical grade viral vectors and translational and regulatory guidance to support academic-led patient trials of new gene therapies.

Grants for up to five years will support these gene therapy innovation hubs which will form a centrally coordinated network designed to enable the sharing of knowledge across all of the units, the two organisations said on 4 May.

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.

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.

A candidate vaccine against Lyme disease has been given a boost following a collaboration announced on 30 April between the vaccine’s developer Valneva SE and Pfizer Inc. The vaccine, VLA15, is currently in Phase 2 as a prophylactic treatment against Lyme disease. Under the agreement, Valneva will fund 30% of development costs up to regulatory submission. Pfizer will lead late-stage development and be responsible for commercialisation, paying Valneva tiered royalties on product sales starting at 19%.

Bavarian Nordic A/S has received a new order under a 2017 contract with the US government to supply its non-replicating smallpox vaccine to the US strategic national stockpile. The vaccine, Jynneos, is indicated for people who have been determined to be at high risk for infection from smallpox or monkeypox, which both belong to the Orthopoxvirus genus of viruses. It was approved by the Food and Drug Administration in September 2019. According to the Centers for Disease Control and Prevention, the last natural outbreak of smallpox in the US occurred in 1949.