factor IX

Be Bio’s BE-101 works by inserting the human Factor IX (FIX) gene into primary human B cells. Image credit: Shutterstock/ ardhikauw. Be Biopharma has raised $82m in financing to advance a gene therapy targeting hemophilia B to a Phase I/II trial, whilst also adding an ex-Roche exec who was involved with the launch of a haemophilia A treatment to its roster. The financing had big pharma involvement in the form of Bristol Myers Squibb and Takeda Ventures, with venture capital firms ARCH Venture Partners, Atlas Venture, and RA Capital Management participating. Be Bio said the funds, which were also from Alta Partners and the Longwood Fund, will be allocated to the hemophilia B candidate BE-101 and  a newly unveiled programme for hypophosphatasia called BE-102. Alongside the funds, the US biopharma added Suha Patel and Dr. Kiran Patki to its ranks, who will serve as senior vice president of commercial & franchise strategy and senior vice president of clinical development, respectively. Patel was involved in launching the haemophilia A treatment Hemlibra (emicizumab-kxwh) at Roche’s Genentech, whilst Patki previously worked at rare disease biotech Rally Bio. Be Bio’s CEO Dr. Joanne Smith-Farrell said that “some valued colleagues will be moving on” as the company shifts resources toward product development. Be Bio’s BE-101 is a genetically engineered B cell therapeutic that works by inserting the human Factor IX (FIX) gene into primary human B cells, which allows for continuous expression of active FIX levels with a single infusion. Haemophilia B, which affects around 40,000 globally, is an X-linked recessive bleeding disorder caused by a deficiency in clotting factor IX and has traditionally been treated with regular infusions of the protein. The advancement of gene therapies is changing the treatment paradigm for this disorder, with single infusions reducing the burden on patients taking repeated treatments. There are currently two US Food and Drug Administration (FDA)-approved gene therapies for haemophilia B – CSL Behring’s Hemgenix (etranacogene dezaparvovec) and Pfizer’s Beqvez (fidanacogene elaparvovec ). Be Bio’s candidate has an FDA orphan drug designation and fast track status, which confers benefits such as the possibility of seven years of market exclusivity. See Also: TreeFrog and University of Pittsburgh probe TLS role in immuno-oncology Lilly’s Kisunla does not demonstrate “good value” for UK’s NHS The FDA approved an investigational new drug application (IND) in March this year, greenlighting the company to initiate a trial evaluating the gene therapy. The multi-centre, first-in-human dose escalation study (NCT06611436), called BeCoMe-9, will evaluate the safety and preliminary efficacy of intravenously administered BE-101 in adults with moderately severe to severe haemophilia B. The trial is estimated to enrol 24 patients and has a primary endpoint of adverse events and serious adverse events one year post dose. Smith-Farrell said: “We are eager to clinically demonstrate BE-101’s potential to provide a highly durable FIX replacement therapy for Hemophilia B patients. Additionally, we are excited to advance BE-102 to address the needs of a large patient population with few to no therapeutic options.” Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Be Bio’s $82 million round announced Tuesday will help the Cambridge, MA-based biotech fund work for two of its drugs in development, including the first-ever B cell therapy to enter human studies for hemophilia B. While the new round is smaller than its $130 million Series B from 2022, the company plans to use the new funding to reach clinical proof-of-concept for its hemophilia B cell therapy and advance a second candidate for hypophosphatasia, a rare genetic disease that disrupts the development of bones and teeth. CEO Joanne Smith-Farrell told Endpoints News the amount was “what we needed to actually get this program all the way to where we’ve got definitive proof-of-concept.” The startup plans to present clinical data next year for hemophilia B. For hypophosphatasia, it expects to seek an IND in 2026, Smith-Farrell said. Companies developing B cell therapies hope they can essentially be long-term protein factories in the body, but without some of the dangers and side effects that come with other cell and gene therapies. The treatments, if they work, could address a range of diseases where people are missing an essential protein. The $82 million round included investors such as ARCH, Atlas Venture, RA Capital and others. Seattle’s Immusoft in October gave a glimpse at results from the first patient to receive a B cell therapy. Immusoft is developing its treatment for mucopolysaccharidosis type I, a genetic disease in which people are missing an enzyme needed to break down sugars. Be Bio has opened enrollment for a Phase 1/2 study of its hemophilia B therapy, BE-101, to adults with moderate-to-severe disease. The experimental treatment is meant to produce Factor IX, a blood clotting factor, without needing preconditioning. The hope is that the therapy, while durable, will also be redosable if needed.

Pfizer’s anti-tissue factor pathway inhibitor Hympavzi (marstacimab-hncq) has been approved by the US Food and Drug Administration (FDA) to treat haemophilia A or B in adult and paediatric patients aged 12 years and older. Hympavzi, which is now the first in its drug class to be approved in the US for haemophilia A or B, has been specifically authorised for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with haemophilia A without factor VIII inhibitors or haemophilia B without factor IX inhibitors. More than 800,000 people globally are affected by haemophilia, a family of rare genetic blood disorders caused by a clotting factor deficiency. Despite recent progress in haemophilia treatment, many patients continue to experience repeated bleeding episodes and manage their condition with frequent intravenous infusions that may need to be given multiple times a week. Administered as a once-weekly subcutaneous injection, Hympavzi works by reducing the amount of the naturally occurring anticoagulation protein, tissue factor pathway inhibitor. This increases the amount of thrombin, an enzyme critical in blood clotting, that is generated. The FDA’s decision was supported by positive results from the late-stage BASIS trial, in which Hympavzi reduced the annualised bleeding rate for treated bleeds by 35% and 92% after a 12-month active treatment period compared to routine prophylaxis and on-demand treatment, respectively, in patients with haemophilia A or B without inhibitors. Ann Farrell, director of the division of non-malignant haematology in the FDA’s Center for Drug Evaluation and Research, said the approval “provides patients with haemophilia a new treatment option that is the first of its kind to work by targeting a protein in the blood clotting process”. The authorisation comes less than six months after the FDA approved Pfizer’s one-time haemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt) for use in certain adults with moderate-to-severe cases of the disorder. The company also has a range of recombinant factor treatments. Aamir Malik, Pfizer’s chief US commercial officer and executive vice president, said: “We look forward to launching this latest medical breakthrough and to now offer three distinct classes of haemophilia medicines… that can meet the unique treatment needs of a wide range of patients.”