First it was the manufacturing process. Now, the novel coronavirus has forced Bluebird Bio to further delay treating patients with its gene therapy Zynteglo in Europe.
On Thursday, Bluebird said it’s now expecting to dose the first commercial patient with Zynteglo (LentiGlobin) in Germany in the second half of 2020, rather than in the first half of the year as the company communicated earlier, “given the evolving COVID-19 situation.”
As for the U.S., on top of the COVID-19 impact, Bluebird still hasn’t reached an agreement with the FDA over new data the agency has required to complete its review. Therefore, the Cambridge, Massachusetts-based biotech said it doesn’t expect to wrap up is rolling application for the drug until mid-2021.
Bluebird has already faced multiple delays on Zynteglo in both territories. After winning its first-ever nod for beta-thalassemia in Europe in March 2019, the actual launch was then pushed back to early 2020 after the EMA demanded changes to commercial drug specification and related manufacturing processes.
EU regulators approved the refined standards in October. And in January, before the global coronavirus outbreak, Bluebird unveiled its first launch in Germany. At that time, chief commercial officer Alison Finger said it was expecting to dose the first patient “soon.”
Now that that plan’s been disrupted, the company’s using the lag to continue its payer discussions and other commercial preparation activities. However, it warned the pandemic would likely “impact its ability to achieve market access and reimbursement in Europe.”
In the U.S., Bluebird was originally expecting to finish filing an application to the FDA by early 2020. But as part of the submission, the agency recently asked for additional data on release assays measuring viral potency, which the two parties have yet to shake hands on. Development and validation of these assays for use in its commercial product are likely to be affected by the COVID-19 outbreak, Bluebird added.
There is but one piece of good news coming out of the delay. Bluebird said it may allow the company to seek approval for a broader beta-thalassemia patient population, including those with β0/β0 genotypes.
Meanwhile, existing clinical studies of Zynteglo for beta-thalassemia and for sickle cell disease, as well as Lenti-D for cerebral adrenoleukodystrophy and Bristol Myers Squibb-partnered CAR-T therapy bb21217 for multiple myeloma, may experience disruptions from the COVID-19 pandemic, the company said.
Though the FDA application for another CAR-T, ide-cel (bb2121), remains on track for a filing in the first half of the year, partner Bristol Myers has suspended screening, enrollment and dosing of existing subjects in the KarMMa-2, KarMMa-3 and KarMMa-4 studies in different lines of multiple myeloma. The timely FDA approval of ide-cel by March 2021 is one of three elements tied to a contingent value right Bristol will be paying Celgene shareholders in their $74 billion merger.
“Generally, the company expects the COVID-19 pandemic to shift the timing of enrollment and completion of clinical studies by at least three months,” Bluebird said in its update.
If the COVID-19 pandemic drags on, Bluebird might have to worry about its balance sheet, SVB Leerink analyst Mani Foroohar wrote in a Friday note to clients. With about $1.2 billion in cash as of the end of 2019, management believes it can last until the second half of 2021 on the current operating plan, Foroohar said. The company has highlighted nondilutive options to bolster the balance sheet if that happens.
Bluebird isn't the only biopharma company that's facing regulatory or clinical disruptions because of COVID-19. The FDA has postponed an advisory committee discussion of Intercept Pharmaceuticals' nonalcoholic steatohepatitis contender, currently sold in another indication as Ocaliva. Pfizer has paused enrollment in many clinical trials in response to the pandemic. Eli Lilly is also holding off on starting most new studies or recruitment of new patients into its existing trials.