Blog: BLUEBIRD BIO, INC. : Regulation FD Disclosure, Other Events, Financial Statements and Exhibits (form 8-K) – Marketscreener.com

Item 7.01 Regulation FD Disclosure.

On September 16, 2022, bluebird bio, Inc. (the “Company”) issued a press release
regarding the Accelerated Approval of SKYSONA® (elivaldogene autotemcel), also
known as eli-cel. A copy of the press release is attached as Exhibit 99.1.

The information contained in this item, including Exhibit 99.1 attached hereto,
is being furnished and shall not be deemed “filed” for purposes of Section 18 of
the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or
otherwise subject to the liabilities of that section, nor shall it be deemed
incorporated by reference in any filing under the Securities Act of 1933, as
amended, or the Exchange Act, except as expressly set forth by specific
reference in such filing.


Item 8.01   Other Events.


On September 16, 2022, the Company announced that the U.S. Food and Drug
Administration (the “FDA”) has granted Accelerated Approval of SKYSONA to slow
the progression of neurologic dysfunction in boys 4-17 years of age with early,
active cerebral adrenoleukodystrophy (“CALD”). The Company has agreed to provide
confirmatory long-term clinical data to the FDA as a condition of the SKYSONA
Accelerated Approval, and continued approval for this indication may be
contingent upon verification and description of clinical benefit in a
confirmatory trial(s).

The Accelerated Approval of SKYSONA is based on data from the Company’s Phase
2/3 study ALD-102 (Starbeam) (N=32) and Phase 3 study ALD-104 (N=35), and is
based on 24-month Major Functional Disability (“MFD”) free survival. A post-hoc
enrichment analysis in symptomatic patients assessed MFD-free survival from
onset of symptoms (neurologic function score (“NFS”) ? 1) in SKYSONA treated
(N=11) and untreated patients (N=7). SKYSONA treated patients had an estimated
72 percent likelihood of MFD-free survival at 24 months from time of first NFS ?
1, compared to untreated patients who had only an estimated 43 percent
likelihood of MFD-free survival.

The SKYSONA Biologics License Application (“BLA”) was reviewed by the FDA under
priority review, and the Company received a rare pediatric priority review
voucher upon approval. On September 15, 2022, prior to the completion of its
review of the SKYSONA BLA, the FDA lifted the clinical hold on SKYSONA that was
put in place in August 2021.

The Company anticipates that commercial product will be available by the end of
2022 through a limited number of Qualified Treatment Centers (“QTCs”) in the
United States, including Boston Children’s Hospital and Children’s Hospital of
Philadelphia. The Company has set the wholesale acquisition cost of SKYSONA in
the U.S. at $3.0 million.

Cautionary Statement Regarding Forward-Looking Statements

This Current Report on Form 8-K contains “forward-looking statements” within the
meaning of the Private Securities Litigation Reform Act of 1995. All statements
that are not statements of historical facts are, or may be deemed to be,
forward-looking statements, including statements regarding the availability of
SKYSONA as a commercial product, including the availability at certain QTCs and
the timing thereof. Such forward-looking statements are based on historical
performance and current expectations and projections about the Company’s future
goals, plans and objectives and involve inherent risks, assumptions and
uncertainties, including internal or external factors that could delay, divert
or change any of them in the next several years, that are difficult to predict,
may be beyond the Company’s control and could cause its future goals, plans and
objectives to differ materially from those expressed in, or implied by, the
statements. No forward-looking statement can be guaranteed. Forward-looking
statements in this Current Report on Form 8-K should be evaluated together with
the many risks and uncertainties that affect the Company’s business,
particularly those identified in the risk factors discussion in the Company’s
Annual Report on Form 10-K for the year ended December 31, 2021, as updated by
its subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and
other filings with the Securities and Exchange Commission. These risks and
uncertainties include, but are not limited to: the risk that the efficacy and
safety results from the Company’s prior and ongoing clinical trials will not
continue or be seen in the commercial treatment context; the risk that
additional insertional oncogenic or other safety events associated with
lentiviral vector, drug product, or myeloablation will be discovered or reported
over time; the risk that the results of confirmatory studies may fail to support
full approval of SKYSONA and, if not, additional studies may be required; the
risk that the Company may not be able to obtain adequate price and reimbursement
for any approved products, including the potential for delays or additional
difficulties for SKYSONA in light of the FDA granting Accelerated Approval; the
risk that the Company may encounter delays in the initiation of its commercial
operations in the United States; the risk that the Company is not able to
activate QTCs on the timeframe that we expect; the risk that the QTCs experience
delays in their ability to enroll or treat patients; the risk that the Company
experiences delays in establishing operational readiness across its supply chain
following approval to support treatment in the commercial context; and the risk
that any one or more of the Company’s product candidates will not be
successfully developed, approved by the FDA or commercialized. The
forward-looking statements included in this Current Report on Form 8-K are made
only as of the date

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hereof and except as otherwise required by applicable law, the Company
undertakes no obligation to publicly update or revise any forward-looking
statement, whether as a result of new information, future events, changed
circumstances or otherwise.



Item 9.01   Financial Statements and Exhibits.



(d)  Exhibits

    Exhibit
      No.                                             Description
     99.1               Press release     of     bluebird bio, Inc.     dated     September
                      16    , 2022  .
      104             Cover Page Interactive Data File (embedded within the Inline XBRL document)





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