Ya 2023.
I sold some $2.50 April calls for .13 cents.
Good article on BLUE:
Investors Take Flight After Bluebird Bio’s Sickle Cell Delay | Scrip | March 30, 2023
Executive Summary
Bluebird’s first two gene therapies are on the market, but a new delay to its biggest hope, the sickle cell disease candidate lovo-cell, has spooked investors.
Bluebird Bio has seen its share price drop by 25% after the company announced a further delay to the filing of its sickle cell disease gene therapy candidate, lovotibeglogene autotemcel, or lovo-cel.
The US biotech had expected to file with the US Food and Drug Administration by the end of this week, but announced on 29 March that this would now been delayed by at least a few weeks.
Timing is significant as Bluebird is in a race to market with a rival sickle cell disease (SCD) candidate, Vertex and CRISPR Therapeutics’s exagamglogene autotemcel (exa-cel), which began a rolling submission in November.
The news added to long-running problems at the gene therapy company and its potentially most lucrative candidate. Lovo-cel’s filing was previously set back 12 months in late 2020 when the FDA requested more data on planned manufacturing processes for the gene therapy.
The latest sell-off left its share price at $3.12, representing a 97% decline over the past five years, a period which included the spin-off of its oncology portfolio as 2seventy Bio, Inc.. (Also see "Bluebird Spinoff 2seventy Bio Gets Its Own Wings" - Scrip, 19 Oct, 2021.)
Speaking on its Q4 call with analysts, Bluebird CEO Andrew Obenshain said the company was likely to miss a target to file in Q1, but believed lovo-cel was still on track for a likely early 2024 launch.
Bluebird also recently launched its first two gene therapies onto the US market – transfusion-dependent beta thalassemia treatment Zynteglo (betibeglogene autotemcel) and Skysona (elivaldogene autotemcel) for rare disease cerebral adrenoleukodystrophy – priced at $2.8m and $3m respectively.
The company provided an update of the first handfuls of commercial patients undergoing cell collection in preparation to receive the treatments, but Bluebird will not report any revenues until Q1 results in May.
Updated Gene Therapy Manufacturing Queried
The delay to lovo-cel’s filing centers on a drug product comparability analysis which Bluebird submitted to the FDA in December. The company recently introduced a new and improved approach to manufacturing the gene therapy, but needs to demonstrate to the US regulator that a new method produces comparable product to that used in its pivotal trials.
The FDA gave feedback to Bluebird last month, and posed further questions that it wants to see answered before the company begins its filings. The company has now provided the regulator with a full chemistry, manufacturing, and controls module, along with responses to the extra queries. Obenshain said there was no timeline for the FDA to respond, but that it had committed itself to a “timely response”.
He added that the full filing was otherwise complete and ready for submission, and expressed confidence in its pivotal trial data in SCD.
Bluebird’s filing will be based on the efficacy results from 36 patients in the HGB-206 Group C cohort and will include a median of 32 months of follow-up and more than 4.5 years of follow-up for some patients. It also expects to include efficacy results from two patients with 18 months of follow-up in the HGB-210 study.
Bluebird believes it is in good shape to be a long-term leader in the field. Obenshain highlighted the fact that it now has 12 activated qualified treatment centers (QTCs) in the US up and running for Skysona and Zynteglo, with a further 30 preparing to come on stream.
Obenshain said having these centers set up to deliver Zynteglo for beta thalassemia would “directly translate” into allowing it to bring lovo-cel to patients with the closely related disease SCD. This, Bluebird believes, will give it an advantage over Vertex.
“We will have the same treating physicians, the same QTC network and the same payer relationships,” said Obenshain. “Bluebird has over a year head start, launching a gene therapy on inherited hemoglobin disorders versus any other gene therapy program.”
Analysts at SVB Securities were however pessimistic on the latest developments, and reduced their ‘possibility of success’ rating for lovo-cel in SCD to 50% from 70%, reflecting the uncertainty around the FDA’s information request and submission delay.
A further cause for concern was the company’s disclosure in a 10-K filing that a patient previously involved in the lovo-cel partial clinical hold had been diagnosed with myelodysplastic syndrome, a type of rare blood cancer. However, the patient has been reported to have no need for interventional therapy or transfusions, and there was no evidence of insertional oncogenesis, a risk linked to gene therapy.
Dane Leone, analyst at Raymond James, took a more phlegmatic approach to the news, commenting that the lovo-cel CMC issues seemed “straightforward" and saw no significant concerns about the MDS, concluding that “the current negative stock reaction is over-done.”
The next significant development in the market is likely to be news from Vertex/CRISPR Therapeutics. The companies had predicted their filing of exo-cel would be completed in Q1, but without any update as yet, look likely to run over into Q2.