CRISPR-based cell therapies continued to gain steam Sept. 27 with the announcements of a potentially valuable big pharma collaboration and an ambitious global regulatory push.
A prespecified interim analysis revealed that Astrazeneca plc’s oral factor D inhibitor danicopan met the primary endpoint of a phase III trial as an add-on therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH) who were also taking a C5 inhibitor, but who still experienced extravascular hemolysis.
Spectrum Pharmaceuticals Inc. celebrated a long-awaited win with the U.S. FDA’s approval late Sept. 9 of novel G-CSF drug eflapegrastim, cleared for use in chemotherapy-induced neutropenia nearly four years after the company first filed for regulatory approval. Despite moves this year to reduce its cash burn, Spectrum has ready to go a commercial team expected to sell eflapegrastim as well as cancer drug poziotinib, which is under FDA review with a PDUFA date of Nov. 24, 2022.
Shares in Forma Therapeutics Holdings Inc. gained 51% as Novo Nordisk A/S made a $20-per-share offer that values the firm’s equity at $1.1 billion. The stock (NASDA:FMTX) had closed at $13.40 prior to the disclosure of the bid on Sept. 1. It closed the day at $20.24, up $6.84, suggesting at least some optimistic investors believe the final price could go higher.
Access to advanced therapies proved to be a major talking point at a conference in London, following the U.S. approval of Bluebird Bio Inc.’s Zynteglo (betibeglogene autotemcel) cell-based gene therapy for beta thalassemia and its $2.8 million price tag. Regulators in Europe backed Zynteglo in 2019 but Bluebird opted to withdraw the therapy in 2021 after deciding that the complex thicket of pricing bodies in Europe was too difficult to negotiate.
Bluebird Bio Inc. isn’t giving out much of the information on the margins for the cost of its newly approved cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. The numbers will, the company said, come into better focus when another Bluebird drug is approved and launched.
As it moves into phase II testing with the lead candidate from its hematologic pipeline, privately held Disc Medicine Inc. is also moving to the public markets via a reverse merger agreement with struggling biotech Gemini Therapeutics Inc. Concurrent with a $53.5 million financing from investors, the deal is expected to provide Disc with a cash runway into 2025.
Pfizer Inc. plans to pay about $5.8 billion – total equity value – for Global Blood Therapeutics Inc. (GBT) and its oral sickle cell disease (SCD) treatment Oxbryta (voxelotor). The company reported the enterprise value as $5.4 billion, which includes debt and net cash. If completed, the GBT buy would be the second largest M&A in 2022 after Pfizer’s $6.7 billion buyout of Arena Pharmaceuticals Inc. Oxbryta, which netted about $195 million in sales in 2021, gained U.S. FDA approval in November 2019 for the treatment of SCD in adults and pediatric patients ages 12 and up. The FDA later expanded Oxbryta’s approved uses to SCD patients 4 years of age and older in December 2021.
As fellow gene editing firm Crispr Therapeutics AG hosted an innovation day in which it confirmed plans for regulatory filings by year-end for an ex vivo gene editing therapy in sickle cell disease and beta-thalassemia, Precision Biosciences Inc. announced plans to develop an in vivo gene editing approach through a collaboration with Novartis AG that brings Precision an initial $75 million with up to $1.4 billion in potential milestones.
New and updated preclinical and clinical data presented by biopharma firms at the European Hematology Association Congress, including: Abbvie, Affimed, Agios, Ascentage, Canbridge, Crispr, Daiichi, Disc, Genmab, Iaso, Innovent, Mustang, Novartis, Vertex, XNK.
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