mkendis

June 04, 2024 09:00 AM Eastern Daylight Time

CAMBRIDGE, Mass.–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

Contacts

Investor Contact:
ir@be.bio

Media Contact:
media@be.bio

BE-101 is the first engineered B Cell Medicine to Enter Clinical Trials for Hemophilia B

Initiation of BeCoMe-9 Phase 1/2 Clinical Study Expected in the Second Half of 2024

May 28, 2024 09:00 AM Eastern Daylight Time

CAMBRIDGE, Mass.–Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), today announced the clearance of its Investigational New Drug application (IND) from the U.S. Food and Drug Administration (FDA) for BE-101, a first-in-class BCM in development for the potential treatment of hemophilia B. The Phase 1/2 clinical trial, BeCoMe-9, is a multi-center, first-in-human dose escalation study designed to assess the safety and preliminary efficacy of BE-101 in adult participants with moderately severe to severe hemophilia B. Be Bio expects to begin dosing participants in the second half of 2024.

“Be Bio was founded to develop transformative medicines, with eyes locked on the patient. Adults and children with hemophilia B have long sought a single dose Factor IX replacement therapy with extreme durability while retaining the dosing flexibility of the current standard of care. BE-101 seeks to deliver active and sustained levels of Factor IX using a patient’s own B cells in a simple infusion that requires no preconditioning,” said Joanne Smith-Farrell, Chief Executive Officer. “The IND clearance is a major milestone in our journey to offer a new, transformative standard of care for people with hemophilia B, and drives Be Bio’s transition to becoming a clinical-stage company.”

“Hemophilia B is not yet solved as bleeding events can create long-term consequences, such as significant chronic pain and irreversible joint damage,” said Dr. Steven Pipe, Medical Director of the Pediatric Hemophilia and Coagulation Disorders Program and Medical Director of the Special Coagulation Laboratory at the University of Michigan. “BE-101 has the potential to be disease modifying by providing long-lasting FIX protection using the patient’s own B cells while providing the option to be titratable and redosable as necessary. If proven safe and effective in adults, the ability to treat children and transform the phenotype as early as possible would be a game changer.”

In preclinical studies, a single dose of BE-101 has demonstrated the ability to deliver active and sustained FIX levels. The data confirmed the expected biodistribution of FIX-expressing BCMs in bone marrow tissue, where they engraft stably over time. Additionally, the redosability of BE-101 has been demonstrated, resulting in a predictable increase in plasma FIX levels.

About BE-101

BE-101 is an autologous 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.

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

May 21, 2024 09:00 AM Eastern Daylight Time

CAMBRIDGE, Mass. — Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), today announced the addition of Paula Cannon, Ph.D., to its Scientific Advisory Board. Dr. Cannon, Distinguished Professor of Molecular Microbiology and Immunology at the Keck School of Medicine of the University of Southern California, was recently appointed President of the American Society for Gene and Cell Therapy (ASGCT). Dr. Cannon will work with other members of the Scientific Advisory Board to provide scientific and clinical guidance as the Company advances its pipeline of B Cell Medicine (BCM) programs, including its lead program, BE-101, a first-in-class BCM for the treatment of hemophilia B.

“Be Bio is pioneering the development of engineered B cell medicines to create a new paradigm for therapeutic proteins, and Paula’s experience is an ideal fit for our esteemed Scientific Advisory Board,” said Joanne Smith-Farrell, Ph.D., Chief Executive Officer of Be Bio. “There are few individuals that have the combination of gene editing know-how and B cell biology as Paula, and we look forward to drawing on her extensive research and clinical experience as we advance our novel BCM platform across protein classes and therapeutic areas and progress BE-101, our lead program, in hemophilia B.”

“Engineered B cell medicines have the potential to disrupt traditional biologics by leveraging the immune system’s biosynthetic powerhouse, the B cell, to develop a new class of therapeutics,” said Dr. Cannon. “Cell and gene therapies are literally changing lives, and I am delighted to join Be Bio’s Scientific Advisory Board and look forward to working with other industry leaders to advance the potential of engineered B Cell Medicines.”

Dr. Cannon was recently named the President of ASGCT, the largest professional organization for academic and industry scientists working in this area of medicine. Her background as a virologist studying HIV and other RNA viruses led to an interest in how viruses could be exploited as gene therapy vectors. More recently her lab has made advances engineering B cells to express customizable antibodies and non-antibody molecules, in a way that could take advantage of the natural response of B cells to vaccination. She is joined on the Scientific Advisory Board by experts in the fields of B cell biology, cell and gene therapy, genetic and infectious disease, and oncology.

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.

Contacts

Investors:
ir@be.bio

Media:
media@be.bio

• Preclinical research demonstrates that CRISPR/Cas9-based precision B cell gene engineering coupled with artificial intelligence-guided protein design produces active tissue-nonspecific alkaline phosphatase (“ALP”)
• Data presented at American Society of Gene & Cell Therapy 27th Annual Meeting

May 10, 2024, 12:00 PM Eastern Daylight Time

CAMBRIDGE, Mass., Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), today presented results from new preclinical research demonstrating production of active ALP by a BCM, which highlights BCMs as a potential treatment for Hypophosphatasia (HPP). Researchers used CRISPR/Cas9 precision gene engineering and artificial intelligence-guided protein design to modify primary human B cells to produce ALP, an enzyme deficient in people living with HPP. The findings were presented during a poster presentation at the American Society of Gene & Cell Therapy (ASGCT) 27^th Annual Meeting on Friday, May 10, at 12:00pm ET.

HPP is a rare genetic disease caused by loss of function mutations in the ALPL gene which leads to deficient ALP activity. People living with HPP can experience wide ranging systemic complications, with impaired bone mineralization, such as rickets/osteomalacia, being a major clinical hallmark of severe disease. The only approved therapy for HPP is an enzyme replacement therapy, asfotase alfa, which requires multiple injections every week and is approved only for use in patients with pediatric-onset forms of HPP.

“This study demonstrates how BCMs coupled with artificial intelligence-guided protein design broaden the potential of our medicines to express highly effective conjugated therapeutic proteins such as ALP-Fc fusion proteins,” said Rick Morgan, Chief Scientific Officer. “BCMs are designed to provide constant and durable protein levels without preconditioning, are redosable, and can be applied to a wide range of diseases, including a potential first-in-class medicine for people living with HPP.”

In this study, primary human B cells were expanded and precision engineered by CRISPR/Cas9 genome editing with AAV-mediated homology directed repair (HDR) to insert an ALP gene expression cassette into various loci, including CCR5 (a safe harbor locus). Guided by an artificial intelligence-based ALP protein structure design engine, protein constructs were optimized for activity and stability of ALP-Fc fusion proteins. Engineered BCMs secreted active ALP proteins up to 200 ng/1e6 cells/24hr. Robust in vitro phenotypic correction using ALP secreting BCMs (ALP-BCMs) was demonstrated in the MC3T3 osteoblast precursor mineralization model.

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, and 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.

Contacts

Investors:
ir@be.bio

Media:
media@be.bio

Cambridge, MA – Be Biopharma, Inc. (“Be Bio”), a company pioneering the development of engineered B Cell Medicines (BCMs), today announced that it will present at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting being held May 7-11, 2024, in Baltimore, MD.

Details regarding the Be Biopharma presentation at the conference are as follows:

Title: CRISPR/Cas9-based precision B cell gene engineering coupled with artificial intelligence-guided protein design produces active and sustained levels of tissue nonspecific alkaline phosphatase for the treatment of Hypophosphatasia

Presenter: Monika Musial-Siwek, Ph.D., Director, Protein Sciences, Be Biopharma

Date: May 10, 2024

Time: 12:00 PM ET

Session Title: Friday Posters: Musculo-Skeletal Diseases
Session Room: Exhibit Hall
Final Abstract Number: 1649

The abstract highlights Be Bio’s novel approach using engineered B-cell Medicines (BCMs) as a potential new treatment for Hypophosphatasia (HPP), a genetic disorder characterized by loss-of-function mutations in the ALPL gene which impairs the mineralization of bones. Researchers used CRISPR/Cas9 precision gene engineering and artificial intelligence-guided protein design to modify primary human B cells to produce tissue nonspecific alkaline phosphatase (ALP), an enzyme deficient in people living with HPP.  The nonclinical data demonstrates the engineered BCM successfully produces active ALP , highlighting the therapeutic potential of the BCM platform as a novel treatment modality for HPP.  BCMs have key attributes of plasma cells, including natural longevity, high levels of protein secretion, the ability to engraft without host preconditioning, and the ability to be re-dosed, making them an attractive platform for sustained supply of biologics.

For more information, please visit the conference website https://annualmeeting.asgct.org/.

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.

Investor Contact:

ir@be.bio

Media Contact:

media@be.bio