Bluebird Ventures into Uncharted Waters as FDA Decision, Zynteglo Uptake Looms Large Despite Cash Crunch

Despite the hampered state of its finances, bluebird bio remains steadfast in its operations through the end of 2021. Massachussetts-based gene therapy company announced the news on Tuesday during its second-quarter earnings conference. The company declared that the majority of its restricted cash is currently inaccessible, but that it is confident in its approach and resourceful in its endeavors.

Over the coming months, Bluebird Bio has an ambitious plan in place to propel its business forward, centred around the scaling up of its FDA-approved gene therapy Zynteglo to treat beta-thalassemia, as well as the potential FDA approval of its sickness cell disease treatment lovotibeglogene autotemcel (Lovo-cel). This could be a game-changer for those looking for a breakthrough in the gene-therapy space.

Despite having approximately $45 million in restricted cash, bluebird CEO Andrew Obenshain warned during an investor call Tuesday that the company’s runway would keep it afloat only through the second quarter of 2024.

At the end of the second quarter in 2023, bluebird held a healthy cash position of $291 million, with plans to keep its yearly cash burn within the range of $270 million to $300 million.

In just five short years, bluebird bio’s Zynteglo and lovo-cel treatments are projected to result in a combined multibillion-dollar revenue opportunity in the U.S. – but only if they are approved by the FDA. According to bluebird Chief Executive Officer Nick Obenshain, the company is on a path toward profitability in the near term and will see significant growth and scale in the years to come.

Bluebird soared to a stunning $6.9 million in revenue for the second quarter – quadruple the $1.5 million it brought in during the same period the year prior. This impressive growth was driven by Zynteglo and its cerebral adrenoleukodystrophy gene therapy Skysona (elivaldogene autotemcel), both of which have already been used to treat 16 patients.

Zynteglo, the revolutionary ex-vivo lentiviral gene therapy, made history when it was granted FDA approval in August 2022. Despite its monumental impact, uptake has been somewhat sluggish, with only 11 individuals beginning treatment. In stark contrast, Skysona, which was granted approval just a few weeks later, has been initiated in five patients.

Bluebird is expanding its reach of treatment centers with qualified assistance for Zynteglo and Skysona to 50 by next year, extending its comprehensive network from current 15 active centers! With this, the company is continuously working to make life-changing therapies more accessible.

Tuesday’s call shone a light on bluebird’s possible financial future – if all 16 initiated patients go through with infusions, revenues estimated to pass $45 million could give the business an unprecedented boost. On top of that, FDA approval of lovo-cel could be the key to sustaining bluebird’s operations for a long time to come.

For sufferers of the life-threatening condition Sickle Cell Disease (SCD), breakthrough one-time gene therapy lovo-cel could provide a much-needed reprieve. With a potential market of 100,000 people aged 12 or over with a history of Vaso-occlusive Events, the Food and Drug Administration (FDA) accepted Bluebird’s Biologics License Application for lovo-cel and granted it Priority Review status in June 2023. The FDA has until December 20 to make a decision – with luck, lovo-cel could provide long-term relief for SCD sufferers across the globe.

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