LONDON – In the largest-ever series A for a Spanish biotech, Splicebio S.L. has raised €50 million (US$56.9 million) to apply its protein splicing technology to the delivery of large genes that do not fit into existing vectors. The company claims its approach will overcome the capacity constraints of adeno-associated viral vectors (AAVs), by splitting genes into parcels and reconstituting the proteins they express in vivo.
Proqr Therapeutics NV stock lost three-quarters of its value as word got out that its pivotal phase II/III study of sepofarsen in treating a tough, rare and genetic retinal disease failed to hit the primary endpoint. CEO Daniel de Boer said he was “shocked by the unexpected outcome” based on data from earlier studies. He added that he is unsure if Proqr will continue developing its therapy for treating CEP290-mediated Leber congenital amaurosis 10 until it understands the new results.
After showing power in its frequency of dosing and efficacy, Vabysmo (faricimab) has been approved by the FDA for treating wet, or neovascular, age-related macular degeneration and diabetic macular edema. The bispecific monoclonal antibody was developed by Roche Holding AG units, Chugai Pharmaceutical Co. Ltd. and Genentech Inc.
Touting a series of firsts and a premium price tag to match, Immunocore Holdings plc is poised to launch the uveal melanoma drug tebentafusp in the U.S. following FDA approval for the medicine. The regulatory nod makes the drug, branded Kimmtrak, the first T-cell receptor-based therapy to reach the market, the first approval for a drug targeting gp100, and the first drug approved in 40 years for the cancer, which is the most common eye cancer in adults, though still rare.
Cellusion Inc. recently closed a ¥1.1 billion ($9.5 million) financing, bringing its total 2021 fundraising to ¥1.7 billion. The company is preparing to enter the clinic with CL-S001, its corneal endothelial substitute cell candidate for treating corneal edema due to endothelial dysfunction, or bullous keratopathy.
With the memory of Dec. 20’s stock-denting, top-line phase III fizzle by Aldeyra Therapeutics Inc. in dry eye disease (DED) still fresh, Palatin Technologies Inc. is launching a late-stage effort in the same indication. Aldeyra offered data from the Tranquility trial with reproxalap – a small-molecule, immune-modulating covalent inhibitor of reactive aldehyde species, known as RASP, formulated as an ophthalmic solution – that showed a miss on the primary endpoint of ocular redness.
DUBLIN – Novartis AG is deepening its commitment to ocular gene therapy by picking up Gyroscope Therapeutics Ltd. for $800 million up front and up to $700 million more in potential milestone payments.
What one analyst called “fantastic external validation but, even more importantly, great for the cell therapy and regenerative medicine space” arrived in the form of Lineage Cell Therapeutics Inc.’s potential $670 million deal with Roche Holding AG. With its subsidiary, Cell Cure Neurosciences Ltd., Lineage signed an exclusive worldwide collaboration and license pact with Roche and its Genentech arm.
Acelyrin Inc. closed a $250 million series B round and, at the same time, unveiled a licensing deal with Affibody AB, involving an interleukin-17A (IL-17A) inhibitor, izokibep, which it is now testing in a pivotal trial in uveitis. Solna, Sweden-based Affibody is getting $25 million up front and could earn up to $280 million more in regulatory and sales-based milestones, as well as tiered royalties, ranging from high single digits to low double digits in percentage terms.
Abbvie Inc.’s FDA clearance of Vuity (pilocarpine HCl ophthalmic solution) 1.25% for the treatment of presbyopia in adults, could bring relief to about 128 million Americans: almost half of the U.S. population, the company said. It’s the first and only eye drop given U.S. regulators’ go-ahead for what’s known as age-related blurry near vision – but plenty more have generated clinical data.
RSS
