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Several late-stage biotech drug candidates fail in pivotal trials when conducted in all-comers. If the true purpose is to provide efficacy and value on a patient-by-patient basis in treatment settings, these questions must be addressed head on in pivotal trials.
To solve this challenge, we have built proprietary precision medicine platforms to better conduct pivotal trials in mechanistically and molecularly defined patient populations.
This translates to innovative trials designs, accelerated development and approvals, refined drug labels–all leading to efficient commercialization and treatment.
We are building a pipeline of pivotal stage assets by capitalizing on our strengths in patient-centric precision medicine, and by adopting a heavily de-risked, asset-centric development strategy.
Several diseases are a lot more complex than we once thought. For example, breast cancer and non-Hodgkin’s lymphoma (B-NHL) are umbrella diseases with more than 10–30 and 60 molecular types, respectively.
Subtypes are also recognized in Alzheimer’s disease, several cancers, and autoimmune diseases such as lupus and rheumatoid arthritis. What was long considered one, homogeneous disease is indeed a collection of rare diseases.
Several pivotal trials fail and the odds of failure rates are much higher when we impose the one-size-fits-all pivotal trial strategy on such heterogeneous diseases. When the trials succeed, a majority of biotech drugs including breakthrough therapies on the market provide excellent responses in only 5–20% of patients, if not fewer.
Thus, it is becoming increasingly relevant that pivotal trials enroll molecularly and(or) immunologically defined patient populations. Such trials can be smaller, and smarter, and potentially lead to shorter development timelines.
Three proprietary disease-specific precision medicine platforms are developed that are ready to be deployed in pivotal trials. The platforms, used individually or in combination, are applicable in several diseases including Alzheimer’s disease, autoimmune and inflammatory disorders, rare diseases, and a select set of cancers.
The platforms enable patient stratifications by disease subtypes, disease severity, DNA and immune biomarkers, as well as to correlate mechanism of action of drugs. Pivotal trials thus performed could be significantly smaller than for all-comers trials, for instance, 400 patients instead of 2,000 patients.
Such pivotal trials further provide the capacity to conduct patient-centric (N=1) trials by evaluating individual variability in genetics and immunology.
Such a clinical development strategy could eliminate middlemen–the entities that do not provide any significant value in the commercialization phase. Thus, biotech drugs developed through this model could be priced sustainably, allowing faster adoption by payers and accelerated access for patients.
Alzheimer’s disease (AD) is a complex, progressively debilitating, and fatal neurodegenerative disease. There are currently over 6 million individuals with AD in the US. Effective therapies do not exist and there is a significant need for developing therapeutics that can halt or reverse the underlying pathology of AD.
Across all types of AD therapies, the failure rate in clinical trials is more than 95%. This demands a re-examination of the drug development process.
Currently approved or about-to-be approved immunotherapies targeting beta-amyloid plaques are not curative but may provide at best modest clinical improvement in some subset(s) of patients.
We plan to launch Phase-2b trials in mechanistically stratified AD subsets to evaluate the therapeutic effectiveness of three immunotherapy assets that target novel immune mechanisms. The objective is to develop these assets as precision immunotherapies that can significantly prevent or slow the progression of AD on a subject-by-subject basis.