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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

Eli Lilly’s Alzheimer’s drug Kisunla has secured regulatory approval in the UK, but has been rejected for widespread use in NHS. Credit: Michael Vi via Shutterstock. Eli Lilly’s Kisunla (donanemab) will not yet be made available to Alzheimer’s patients in the UK after the National Institute for Health and Care Excellence (NICE) said it doesn’t demonstrate good value for UK’s NHS. On 23 October, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granted approval to the therapy for the treatment of mild cognitive impairment or mild dementia due to Alzheimer’s disease in some adults. This follows on from the US Food and Drug Administration (FDA)’s green light in July 2024 . However, NICE, which acts as the UK’s price watchdog, said that although the monthly injection slows disease progression by four to seven months, as demonstrated in the Phase III TRAILBLAZER-ALZ 2 (NCT04437511) study, there are “significant uncertainties about how much benefit donanemab provides, and how long this lasts for after stopping treatment”. It also said that more evidence is needed on the clinical and cost-effectiveness of Kisunla. A NICE committee also explained that one-third of patients in the trial had signs of brain swelling or brain bleeding, as determined by amyloid-related imagining abnormalities (ARIAs). Similar concerns were raised in the FDA’s June 2024 advisory committee convened to discuss the drug’s approval. Patients in Lilly’s trial were closely monitored due to this, with MRIs done at four, 12, 24, 52, and 76 weeks in addition to allowing for unscheduled MRIs at investigator discretion. This frequent monitoring can rack up increased costs for payers , as well as bringing up accessibility issues. In the announcement accompanying the decision, Helen Knight, director of medicines evaluation at NICE said: “The cost-effectiveness estimate for donanemab is 5 to 6 times above what NICE normally considers an acceptable use of NHS resources.” See Also: TreeFrog and University of Pittsburgh probe TLS role in immuno-oncology Be Bio raises $82m to advance haemophilia B gene therapy to Phase I/II trial In the US, Kisunla has a price tag $695.65 per vial. This means that the cost of treatment for a six and 12 month-plan would amount to $12,522 and $32,000, respectively. Kisunla isn’t the only Alzheimer’s therapy on the market. Eisai and Biogen ’s Leqembi (lecanemab) secured UK approval in August 2024, but NICE published a separate draft guidance that said it leans against reimbursing the drug. Leqembi has an annual cost of $26,500 per year in the US, and its infusions are administered every two weeks for a minimum of 18 months. In July, Leqembi faced rejection from the European Medicines Agency (EMA) , while the agency is continuing to review Kisunla. Kisunla and Leqembi are monoclonal antibodies that work by targeting and clearing amyloid beta plaques from the brain, aiming to slow early cognitive decline by reducing toxic buildup linked to the disease. It is estimated that around 70,000 adults in England would have been eligible for treatment with Kisunla, according to NICE.

J&J’s Darzalex-VRd can now be given to newly diagnosed MM patients eligible for ASCT. Credit: Michael Vi/Shutterstock The European label for Johnson and Johnson Innovation Medicine’s (J&J) Darzalex (daratumumab) has been expanded to include patients with newly diagnosed multiple myeloma (MM) eligible for autologous stem cell transplant (ASCT). The label expansion for the anti-CD38 antibody was supported by data from the ongoing Phase III PERSEUS trial that was submitted to to the European Medicines Agency in March 2024. Darzalex initially received approval as a monotherapy for MM from the EC in May 2016. Along with MM, the antibody is marketed for treating primary system amyloidosis, and is in clinical studies for a range of oncological, immunological, and metabolic disorders. Projections from GlobalData estimate the drug will generate $11.5bn in revenue this year, set to increase to an annual $20bn in revenue by 2030. GlobalData is the parent company of Pharmaceutical Technology . See Also: TreeFrog and University of Pittsburgh probe TLS role in immuno-oncology Be Bio raises $82m to advance haemophilia B gene therapy to Phase I/II trial Co-formulated with Halozyme’s Enhanze drug delivery technology, Darzalex is the only subcutaneous, CD38-targeting MM treatment approved. The PERSEUS trial (NCT03710603) found that a Darzalex-VRd quadruple regimen resulted in a 58% reduced risk of disease progression or death compared to bortezomib, lenalidomide, and dexamethasone alone. Among 709 randomised MM patients, Darzalex therapy led to improved minimal residual disease (MRD) negativity (75.2% vs 47.5%), complete response (87.9% vs 70.1%), and sustained MRD negativity for ≥12 months (64.8% vs 29.7%). The quadruple regimen of Darzalex with bortezomib, lenalidomide, and dexamethasone received US Food and Drug Administration (FDA) approval for the same indication in July 2024, following FDA approvals to treat MM for Darzalex with bortezomib, thalidomide and dexamethasone (VDd) in 2019 and as a monotherapy in 2015. In the UK, Darzalex monotherapy was approved to treat MM by the National Institute for Health and Care Excellence (NICE) in January 2018. NICE later published guidance recommending Darzalex VDd for MM in April 2023.