FDA Grants Orphan Drug Designation for BE-101, a Novel Engineered B Cell Medicine, for the Treatment of Hemophilia B
June 04 2024 - 9:00AM
Business Wire
Be Biopharma, Inc. (“Be Bio”), a company pioneering the
discovery and development of Engineered B Cell Medicines (BCMs),
today announced that the U.S. Food and Drug Administration (FDA)
has granted Orphan Drug Designation to BE-101, a novel engineered B
Cell medicine (BCM) being developed for the treatment of Hemophilia
B. Be Bio is on track to initiate its Phase 1/2 study, BeCoMe-9,
evaluating BE-101 in adults with severe or moderately severe
Hemophilia B in the second half of 2024.
"Despite recent advances, patients with hemophilia B still
suffer from bleeding events, joint damage and chronic pain," said
Joanne Smith-Farrell, Ph.D., Chief Executive Officer of Be Bio.
"BE-101 has the potential to be the first and only Factor IX
replacement therapy that is extremely durable, re-dosable and
titratable, representing an opportunity to dramatically improve the
treatment paradigm in hemophilia B. The Orphan Drug Designation
reinforces the potential of this therapy and underscores the need
for improved therapeutic options for these patients."
The FDA grants Orphan Drug Designation to drugs or biologics
designed to treat rare diseases or conditions affecting fewer than
200,000 people in the United States. This designation offers
several significant advantages, including a seven-year period of
exclusive marketing rights following approval, exemption from user
fees, and eligibility for tax credits on qualified clinical
trials.
About BE-101
BE-101 is a first-in-class B Cell Medicine (BCM) that is
engineered to insert the human FIX gene into primary human B cells,
allowing for expression of active FIX for the treatment of
Hemophilia B. BE-101 has the potential to express sustained
therapeutic FIX activity levels with a single infusion with the
flexibility to be re-dosed, if needed. The potential to maintain
therapeutic FIX activity levels while reducing dosing frequency
associated with current FIX replacement regimens would address the
considerable infusion burden associated with current therapies and
potentially drive significant reductions in the annualized bleeding
rates and FIX usage. Be Bio expects to initiate a phase 1/2 study,
BeCoMe-9, evaluating BE-101 in patients with severe or moderately
severe Hemophilia B in the second half of 2024.
About Hemophilia B
Hemophilia B is an X-linked recessive bleeding disorder that
affects approximately 1:20,000 males. It is caused by mutations in
the gene that encodes for the FIX protein, an essential enzyme in
the coagulation cascade. This can lead to spontaneous bleeding as
well as bleeding following injuries or surgery.1 People with
hemophilia B bleed longer than other people. Bleeds can occur
internally, into joints and muscles, or externally, from minor
cuts, dental procedures or trauma.2 While an adeno-associated virus
(AAV) vector-based gene therapy has been approved for some adults
as a potential new option, the current standard of care and only
treatment for children remains prophylactic administration of
exogenous FIX derived from recombinant protein. The short
biological half-life of FIX requires frequent infusions to maintain
therapeutic levels.
About Engineered B Cell Medicines – A New Class of Cellular
Medicines
The B cell is a powerful cell that produces thousands of
proteins per cell per second at constant levels, over decades.
Precision genome editing can now be used to engineer B Cells that
produce therapeutic proteins of interest, driving a new class of
cellular medicines – Engineered B Cell Medicines (BCMs) – with the
potential to be durable, allogeneic, redosable and administered
without pre-conditioning. The promise of BCMs could transform
therapeutic biologics with broad application — across protein
classes, patient populations and therapeutic areas.
About Be Biopharma
Be Biopharma (“Be Bio”) is pioneering Engineered B Cell
Medicines (BCMs) to dramatically improve the lives of patients who
are living with Hemophilia B and other genetic diseases, cancer,
and other serious conditions. With eyes locked on the patient, our
team of purpose-driven scientists, technologists, manufacturing
experts and business builders collaborate to create a bold new
class of cell therapies. Be Bio was founded in October 2020 by B
cell engineering pioneers David Rawlings, M.D., and Richard James,
Ph.D., from Seattle Children’s Research Institute. Be Bio is backed
by ARCH Venture Partners, Atlas Venture, RA Capital Management,
Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda
Ventures, Seattle Children’s Research Institute and others. Since
our founding, Be Bio’s investors have committed over $180 million
to enable the Company to re-imagine medicine based on the power of
B cell therapy. For more information, please visit us at Be.Bio and
our LinkedIn page.
References
1What is Hemophilia? U.S. Centers for Disease Control and
Prevention. Accessed November 9, 2023
https://www.cdc.gov/ncbddd/hemophilia/facts.html 2Hemophilia B.
National Bleeding Disorders Foundation. Accessed November 9, 2023
https://www.hemophilia.org/bleeding-disorders-a-z/types/hemophilia-b
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