Damoctocog Alfa Pegol

Pictured: Baisong Mei/Nicole Bean for BioSpace Baisong Mei is no stranger to leadership roles. At just 15 years old, he was already in medical school leading a group of students 10 years his senior. (In China, medical students skip undergraduate degrees and instead go straight to a doctoral program.) That was just one of many instances of leadership peppered throughout the career of Editas’s current chief medical officer. Mei was born in a small town in the southern part of Anhui province in China. He skipped grades a couple of times, which allowed him to start college at Bengbu Medical College (now Bengbu Medical University) at just 15 years old, the first time in his life he went to a bigger city, Bengbu. Because he had done well in high school, scored reasonably well on the college entrance examination, held leadership positions and participated in competitions, he was made the head of a group in the class. This entailed leading older students in group activities, an experience he called “challenging.” But that didn’t discourage him. After graduating from Bengbu Medical College at the age of 20, he went on to earn a master’s degree at Wuhan University School of Medicine, where he then worked as a faculty member for five years. At 28, he left China to move to Ohio with only two suitcases. There, Mei earned a Ph.D. in molecular biology and biochemistry from the University of Toledo. He then completed fellowships at the University of California, San Francisco and UC Berkeley; held leadership positions at Bayer, Biogen and Sanofi; and co-invented an approved drug, Jivi, an injectable medicine from Bayer for people with hemophilia A. Mei’s laundry list of accomplishments goes on. But he said that one of the most rewarding successes of his career is seeing patients in clinical trials that he runs sign on for an extension. He explained that this usually means patients feel like the drug is working for them without too many complications, which is a huge success in the R&D world. It’s happened many times throughout his career, he said, the first time with a hemophilia B drug at Biogen that was later approved by the FDA and marketed as Alprolix. Learning to Adapt Mei’s position at early-stage biotech Editas is more entrepreneurial than his earlier career, allowing him to remain close to the science and to clinical trials. But after coming from academia and Big Pharma, learning how to operate in a genome editing biotech with a few hundred employees involves a learning curve, he said. Fortunately for him, his formula for success is all about continuously learning and adapting. The most valuable thing he’s learned since starting at Editas in 2022 is how to communicate in a way that inspires confidence in the company, he said, so that stakeholders know that it has a solid strategy and can deliver helpful products. He’s been part of the executive leadership team working to transform Editas from a technology platform company to a commercial therapeutics company and leader in in vivo programmable gene editing. The team made changes to sharpen the company’s strategic focus and emphasize execution of clinical programs. “When you become a CMO in a private company, in addition to driving all these clinical trials, you also [have to think about] how the company can grow and what is the strategy of the company so that [it] can help more patients and help the stakeholders of the company,” he said. With these additional responsibilities, “I feel that I’m able to make more impact, not only to the company, [but] to the patients.” Mei categorizes the challenges he’s encountered in his career into two types: cultural and career-related. Most of his cultural challenges happened when he first came to the U.S. and had to adapt to food, language, TV, interpersonal interactions and many other aspects of daily life that were completely different from China. And his cultural environment has varied greatly even throughout his time in the U.S., he said, since he’s now lived in the Midwest and on both coasts. “The education system in China really . . . set a foundation for me to be able to adapt here in United States,” he said, by emphasizing math, physics and chemistry, and putting a lot of value on hard work and discipline. For example, in high school, he often studied past midnight and in medical school he had over 30 hours of classes each week. “This allowed me to really focus on learning and be humble, and that really helped me a lot.” When he first came to the U.S. for his Ph.D., it was uncertain whether he would be able to remain in the country to work after his student visa period. So he viewed it as an experiment, completing his Ph.D. without knowing how the rest of his career would turn out. If he couldn’t stay in the U.S., his backup plan was to bring his learning back to China. “And now, of course, after more than 30 years, I feel I’m American,” he said. “I feel the United States has given immigrants like myself so many opportunities. I feel so grateful for that . . . this is actually an amazing place [that] can provide people opportunity. And I understand why many people want to come.” Mollie Barnes is a freelance science writer based in Los Angeles. Reach her at mollie@100yearsco.com. Follow her on Threads and Instagram @shejustlikedtogo and see more of her work at molliebarnes.contently.com.

The well-established matching-adjusted indirect comparison (MAIC) methodology was used to compare the efficacy of personalised prophylaxis with Nuwiq® against other recombinant FVIII products Personalised prophylaxis with Nuwiq® can lead to higher rates of patients with zero bleeds compared with other recombinant FVIII products, albeit with higher weekly doses The data were published recently in the European Journal of Haematology LACHEN, Switzerland, Oct. 31, 2023 /PRNewswire/ -- Data from a matching-adjusted indirect comparison (MAIC) of Nuwiq® (simoctocog alfa) versus extended half-life recombinant factor VIII (rFVIII) concentrates in haemophilia A patients undergoing personalised prophylaxis were published in the European Journal of Haematology1. Preliminary data from the MAIC were presented by Dr Craig Kessler, Lombardi Comprehensive Cancer Center and Georgetown University Medical Center, at the 65th ASH Annual Meeting & Exposition in December 20222. Continue Reading Octapharma AG: Indirect comparison data indicate that personalised prophylaxis with Nuwiq® may lead to fewer patients with haemophilia A having bleeds than with other recombinant factor VIII products The analysis used MAIC – a well-established statistical approach – to compare the efficacy of pharmacokinetic (PK)-guided, personalised prophylaxis with Nuwiq® (simoctocog alfa) versus Eloctate® (efmoroctocog alfa), Jivi® (damoctocog alfa pegol), and Adynovate® (rurioctocog alfa pegol). The analysis used data from prospective clinical trials of personalised prophylaxis in adults with severe haemophilia A. Dr Kessler commented, "This MAIC exercise provides important comparative data on the efficacy and consumption of various recombinant FVIII products in adults with haemophilia A on personalised prophylaxis. The results can be considered by clinicians and patients when treatment decisions are being made." MAICs are a way of comparing the efficacy of different medical interventions or products in the absence of head-to-head randomised trials3. By matching patient populations based on clinically relevant baseline characteristics, it reduces inherent differences between the studies and can provide comparative evidence of relative treatment effects. Individual patient data from patients treated with Nuwiq® in the NuPreviq study were compared with aggregated data from 117 patients treated with Eloctate® in the personalised prophylaxis arm of the A-LONG study, 110 patients treated with Jivi® in the PROTECT VIII study, and patients treated with Adynovate® in the PROPEL study (which had two arms targeting different FVIII trough levels: 1–3% (57 patients) and 8–12% (58 patients)). A significantly higher percentage of patients with zero bleeds was reported with Nuwiq® compared with Eloctate® (75% vs 45%), Jivi® (77% vs 38%) or Adynovate® (target trough level 1–3%; 78% vs 42%). The efficacy of Nuwiq® was similar to that of Adynovate® in the 8–12% arm (77% vs 62%). The mean annualised bleeding rate was significantly lower with Nuwiq® than with Jivi® (1.5 vs 4.9). Consistent with the approved dosing, the mean FVIII weekly dose was significantly higher for Nuwiq® than Eloctate®, Jivi® or Adynovate® 1–3%, but lower than for Adynovate® 8–12%. Further details of the study can be found in the European Journal of Haematology publication. In addition, a short video summarising the results is available in the online version of the article. About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Haematology and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centres across Europe and the US. Octapharma has 40 years of experience in patient care. About Nuwiq® Nuwiq® (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein4. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor4. Nuwiq® treatment has been assessed in nine4-6 completed clinical trials which included 201 previously treated patients (190 individuals)4 and 108 previously untreated patients5 with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations7. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups7. References Kessler CM et al. Eur J Haematol 2023; 111:757–67. Kessler CM and Tiede A. Blood 2022; 140(Suppl. 1): 2755–6. Signorovitch JE et al. Value Health 2012; 15:940–7. Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. Liesner RJ et al. Thromb Haemost 2021; 121:1400–8. Octapharma AG, data on file. Nuwiq® Summary of Product Characteristics. Logo - Video - SOURCE Octapharma AG