Welcome to the latest edition of our weekly EuroBiotech Report. The week pencilled in as a possible end to uniQure’s woes came and went with a whimper. Despite Spark Therapeutics' data renewing safety worries and uniQure showing long-term symptom relief, the Dutch biotech’s failure to close the gap in Factor IX activity means it remains stuck with also-ran status in the eyes of many. TiGenix is hoping its pitch to Wall Street investors is better received. The stem cell therapy specialist took another step toward an IPO intended to give it $42.9 million to advance a Crohn’s disease program. Nordic Nanovector’s own pitch for cash went well, resulting in it securing $60 million for a phase 2 cancer trial pairing its Betalutin with Roche’s Rituxan. Amryt Pharma sorted out its own clinical trial financing plans late last week by securing €20 million from the European Investment Bank. The European Medicines Agency accepted Sanofi and Regeneron’s dupilumab for review. And more. Nick Taylor
UniQure has posted fresh data from a phase 1/2 trial of its hemophilia B gene therapy. The 12-month low-dose data and six-month high-dose results link AMT-060 to the near-cessation of spontaneous bleeding and a sharp drop in Factor IX (FIX) replacement therapy, but the gene therapy continues to be dogged by comparisons to Spark Therapeutics’ rival program.
EMA has accepted Regeneron and Sanofi’s dupilumab for review. Dupilumab’s filing is chock-full of clinical trial data showing it improves multiple outcomes in patients with moderate-to-severe atopic dermatitis, leading to the IL-4/IL-13 inhibitor being widely seen as the next blockbuster to roll off the lot at Regeneron.
TiGenix has taken another step toward its delayed Nasdaq IPO. The stem cell therapy specialist is aiming to net $42.9 million (€39.8 million) through the listing, approximately half of which will go toward a U.S. late-phase trial of Crohn's disease candidate Cx601.
Nordic Nanovector has raised $60 million in an oversubscribed private placement. The cash injection will enable Nanovector to test its CD37-targeting antibody-radionuclide conjugate in combination with rituximab, better known as Roche’s cancer powerhouse Rituxan.
Amryt Pharma has secured a €20 million loan from the European Investment Bank (EIB) to fund a pivotal trial of its lead candidate in epidermolysis bullosa. The specialty pharma, which is chaired by Shire founder Harry Stratford, is set to start the phase 3 clinical trial early next year.