Over the last seven months, our industry has been working around the clock to combat the COVID-19 virus, including developing effective therapeutics to treat COVID-19 and vaccines to prevent future infections.
I had the opportunity to connect with Ramona Sequeira, President of Takeda Pharmaceuticals U.S.A, about the company’s efforts to fight COVID-19.
Stephen Ubl (SU): Ramona, while there has been a lot of discussion around the development of COVID-19 vaccine candidates, can you tell me more about the plasma-derived therapy Takeda and other global plasma leaders are exploring to treat those fighting the virus?
Ramona Sequeira (RS): Yes, I would be delighted to share more about the potential plasma-derived therapy we are working on as part of the CoVIg-19 Plasma Alliance. Essentially, we are pooling and processing plasma from recovered COVID-19 patients – also known as ‘convalescent plasma’ – to develop a non-branded plasma-derived therapy known as a hyperimmune globulin (H-Ig) that contains consistently high levels of antibodies to the new coronavirus. What’s important to understand is that our process purifies the donated plasma to remove or inactivate any viruses and standardizes it, so it has a consistent level of virus-specific antibodies in each unit. Because hyperimmune therapy has already been shown to be effective in treating severe viral respiratory infections, we believe it may have the potential to treat people at risk for serious complications from COVID-19.
We are collaborating with the National Institute of Allergy and Infectious Diseases (NIAID) at the National Institutes of Health (NIH) on a global multi-center, double-blind, placebo-controlled, randomized clinical trial to test the safety, tolerability and efficacy of the hyperimmune globulin (H-Ig) medicine for COVID-19. As the sponsor of the study, NIAID is managing the execution of the trial.
A sufficient amount of CoVIg-19 product has been manufactured by the CoVIg-19 Plasma Alliance and is ready for clinical evaluation. A summary of trial details such as enrollment targets, study design and sites will be available on www.clinicaltrials.gov. Once completed, the study will form the basis for starting submissions for regulatory authorization.
We have worked closely with the FDA and EMA to understand the trial design requirements for regulatory authorization. We are pleased with the remarkable progress the Alliance has made in just a matter of months - we have established the processes to collect convalescent plasma, manufactured our H-Ig and coordinated with the NIH on a rigorous multi-national pivotal study protocol.
While our program is unrelated to the recent decision regarding an emergency use authorization for the direct transfusion of COVID-19 convalescent plasma (CCP), we welcome all efforts that can contribute potential therapeutic solutions for COVID-19.
SU: Very exciting and promising to know that therapies are being explored that can aid in this fight. Can you tell us a bit more about the CoVIg-19 Plasma Alliance, namely how it has - and will - contribute to the development of treatments?
RS: We believe the pandemic demands bold moves. Which is why, when faced with the unprecedented challenges presented by COVID-19, Takeda formed an Alliance of world-leading plasma companies. Takeda and members of the Alliance believe the plasma community has a special responsibility – and opportunity – to make a difference in the interest of global public health. Instead of pursuing individual projects, we have all set aside our company interests to work together.
The Alliance includes 12 leading plasma companies, including Takeda, all of whom have deep experience in discovering, developing, and producing plasma-derived therapies. The Alliance also includes global organizations from outside the plasma industry that are providing support to encourage more people to donate plasma. Together, Alliance members are working to accelerate the development of this potential medicine, improve our chances of success and increase the supply and availability of the potential medicine, if approved.
Finally, to ensure we maintain our momentum, our first and most important priority in the development of this potential treatment is our ability to collect enough convalescent plasma. Plasma can’t be made in a lab or by artificial methods, so we urgently need plasma donations from people who have recovered from COVID-19.
That’s why the Alliance joined The Fight Is in Us coalition that brings together all parties interested in advancing investigation into multiple plasma-based approaches. Together, the coalition points interested donors to more than 2,200 locations across the U.S., making it more likely for people to find a facility close to where they live.
Survivors of COVID-19, or those who know someone who has successfully recovered should visit the coalition website at www.thefightisinus.org where a clever “plasmabot will help them locate the closest plasma collection site in their area.
SU: That is a great approach to an interesting situation. The past serval months have presented challenges to drug development that we have never seen before. Can you talk about any of these challenges and how Takeda has been able to navigate them?
RS: There’s no question that investigators and site staff around the world have faced extraordinary circumstances operating clinical studies while managing the additional burdens caused by COVID-19. Takeda is no exception and we’ve worked really hard to adjust in creative and meaningful ways to keep our clinical trials moving forward as safely and quickly as possible.
Given the fluidity of the pandemic, Takeda is evaluating each of our clinical trials on a fit-for-purpose basis taking into consideration the varying impact of COVID-19 in different regions of the world. For example, in some places, patients have been unable to physically be at study sites, so where possible, we worked quickly and creatively to implement solutions such as direct-to-patient delivery of study medicines and re-evaluating study design to account for potential disruptions. We also continue to assess and build out digital technology to enable more remote monitoring of patients enrolled in clinical trials.
Looking forward, we are optimistic that as the industry continues to work with regulators, academics and study sites, many of the new processes and technologies that are being adopted in response to COVID-19 will help Takeda move our medicines through the clinical trial process more quickly, allowing potential treatments to get to patients faster.
SU: Speaking of the future, the complexities introduced by COVID-19 have the potential to accelerate changes to the status quo for how companies approach Research & Development. Can you talk about how some of the innovative approaches Takeda has taken in response to COVID-19 that could be applied to R&D beyond the pandemic?
RS: In the early days of the pandemic, Takeda helped establish the COVID R&D Alliance comprising more than 20 biopharma companies, academic institutions, not- for-profit organizations and venture capital firms who are collaborating to rapidly advance COVID-19 therapies and vaccines. The Alliance is organized around seven working groups, each led by a different R&D leader: clinical phase repurposing, preventive vaccines, novel antibodies, novel small molecule antivirals, preclinical repurposing, clinical trial acceleration, and finally data sharing – which is being led by Takeda.
The new ways of thinking about data sharing, real-world data and adaptive studies that we are applying to develop COVID-19 therapies and vaccines can absolutely be applied more broadly to get innovative treatments to patients with unmet needs more quickly and efficiently.
For example, within weeks of being created, the Alliance’s data-sharing working group established a cross-functional team to develop data-sharing platforms to collect, share and analyze COVID-19 related data among participating organizations to accelerate the development of therapeutics and vaccines. The unprecedented level of collaboration among the Alliance sets a new standard that could potentially change the data-sharing ecosystem beyond the pandemic. More broadly, real-world data has the potential to improve trial design and can be used to define patient segments, accelerate enrollment and inform safety profiles. Real-world data is also useful in the expansion of approved products into new indications where regulators can evaluate a drug’s efficacy in a new population while considering the existing safety profile.
Another concept that could be applied beyond the pandemic is the use of adaptive trial platforms. Through our work with the COVID R&D Alliance, Takeda is also participating in the I-SPY COVID Trial, a pre-competitive adaptive platform for COVID-19 acute respiratory distress syndrome (ARDS) ICU patients. The I-SPY COVID Trial utilizes Quantum Leap Healthcare Collaborative's adaptive platform trial design which allows multiple agents to be evaluated -- adding or removing them from the trial without stopping. This increases trial efficiency by minimizing the number of participants as well as the time required to evaluate potential treatments.
Personally, I am very optimistic that our industry will leverage the learnings from the pandemic to improve our approach to R&D to accelerate the delivery of safe and effective new treatments and medicines to the patients who need them.
As always, you can stay up-to-date with the work the biopharmaceutical industry is doing to combat COVID-19 here.
Stephen J. Ubl Stephen J. Ubl is president and chief executive officer of PhRMA. Mr. Ubl leads PhRMA’s work preserving and strengthening a health care and economic environment that encourages medical innovation, new drug discovery and access to life-saving medicines. Ubl is recognized around the world as a leading health care advocate and policy expert who collaborates successfully with diverse stakeholder groups – including patient and physician groups, regulators, public and private payers, and global trade organizations – to help ensure timely patient access to innovative treatments and cures.