Hypophosphatasia

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

▎药明康德内容团队编辑 今日，致力于开发B细胞疗法的Be Biopharma宣布达到多个关键里程碑。用于治疗血友病B的主打项目BE-101进入临床开发阶段；同时该公司宣布选定第二个候选药物，用于治疗低磷酸酶症（HPP）。这两个项目均基于Be Biopharma公司强大且高效的B细胞药物（BCM）平台，该平台利用基因编辑技术改造B细胞，使其能够持续产生治疗性蛋白质，最终开发出具有持久性、可滴定且可重复给药的候选药物，无需预处理，具有成为潜在“best-in-class”基因疗法的潜力。 该公司最近达到的里程碑包括： 2024年5月，Be Biopharma宣布其潜在“first-in-class”B细胞疗法BE-101的IND申请获得FDA的许可。BE-101的设计目的是无需预处理，即可让B细胞持续产生恒定水平的凝血因子IX，具有持久性、可重复给药性及可滴定性。首个人体临床试验BeCoMe-9已开始招募患者，这是一项多中心的人体剂量递增研究，旨在评估BE-101在中度至重度血友病B成人患者中的安全性和初步疗效。FDA最近已授予BE-101快速通道资格。 该公司宣布BE-102已被选为治疗HPP的候选药物，HPP是一种严重的遗传疾病，具有极高的未满足医疗需求。它的特征是由于ALPL基因的功能缺失突变，导致碱性磷酸酶（ALP）活性不足，从而引起骨骼和牙齿的发育不良和脆弱。现有的获批疗法需要每周多次注射，且仅限于儿科发病的形式。此前展示的数据表明，BE-102有潜力在体内生成活性ALP并且持续维持ALP水平。 该公司还宣布已完成8200万美元的融资，得到了多家风险投资公司和医药公司的支持，包括ARCH Venture Partners、Atlas Venture、RA Capital Management、Alta Partners、Longwood Fund、百时美施贵宝和武田创投。获得资金将用于实现BE-101的临床概念验证，并推进BE-102的开发。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Be Bio Announces $82 Million Financing and Transition to Clinical Stage Company. Retrieved October 22, 2024, from https://www.businesswire.com/news/home/20241022283327/en 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

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.