→ Having followed Milan-based EryDel and its CEO for years, Sofinnova Partners is jumping in for the ride, leading its latest €26.5 million ($32.4 million) round. The funds will cover the registrational Phase III clinical trial of EryDel’s lead product, EryDex, which uses the biotech’s red-blood-cell-based drug delivery technology to treat ataxia telangiectasia. The trial, dubbed ATTeST, is currently enrolling in Europe, Asia, Australia and the US. Sofinnova is joined by existing shareholder Genextra SpA and Innogest SGR, all chipping in to push the company along in its international development. “We are thrilled to welcome Sofinnova Partners as our lead investor,” CEO Luca Benatti said in a statement. “Their unique international reach and recognized experience for building global leaders represent a huge asset at a time when EryDel is about to accelerate its growth plan.”
→ More than three years into its partnership with Zymeworks $ZYME, Celgene $CELG is exercising an option to expand its use of the Azymetric platform. The big biotech now has rights to develop and commercialize 10 bispecific antibody candidates coming out of that platform, up from eight. For Vancouver-based Zymeworks, that means an unspecified expansion as well as an extra potential $328 million in milestones ($164 million for each product, as established in original agreement). The research program term has also been extended by two years.
→ Alnylam’s lead drug patisiran just got one step closer to approval with positive data reported out this morning from a Phase III trial. In a new post-hoc analysis, patisiran reduced composite rate of all-cause hospitalization and mortality by about 50% relative to placebo. The drug also demonstrated an improvement in multiple quality of life measurements when compared to placebo. The Cambridge/Boston RNAi specialist has expressed much confidence in patisiran for hereditary ATTR amyloidosis, having restructured its deal with Sanofi to re-gain global rights just ahead of the JP Morgan health care conference in January. An approval — by the PDUFA date of August 11 — is widely expected. “We believe these results, along with previously presented APOLLO data that show halting or reversal of neuropathy progression in a majority of patients treated with patisiran, strengthen the body of evidence demonstrating that patisiran, if approved, has the potential to be a transformative treatment for patients with all forms of hereditary ATTR amyloidosis,” said Eric Green, VP and general manager of the TTR program at Alnylam in a statement. “We continue to work collaboratively with the FDA and EMA through patisiran’s review process, with the goal of making this medicine available to patients as quickly as possible, upon approval.”
→ UK-based Orchard Therapeutics is opening up a Boston office in the Seaport District, fast on the heels of its acquisition of GSK’s rare disease unit. The new US office for the gene therapy company will be for “commercial operations and several key financial and administrative functions.”
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