This week, in honor of Breast Cancer Awareness month, we outline breast cancer treatments in the pipeline of major players and discuss the FDA’s consideration of expanded access or emergency use authorization for COVID-19 vaccines. We’ve compiled a breakdown of noteworthy developments in biotech, that focus on the economics of innovation.
Major Players Breast Cancer Treatment Pipeline
Roche’s Venclexta for HR-Positive Breast Cancer
When breast cancer has a significant number of receptors for estrogen or progesterone it’s considered hormone-receptor (HR) positive. Venclexta is currently being assessed in a phase II clinical trial evaluating it for the treatment of second line or later HR-positive breast cancer. Venclexta is a novel small molecule Bcl-2 selective inhibitor designed to restore apoptosis, also known as programmed cell death, by blocking the function of a pro-survival Bcl-2 family protein. The Bcl-2 family proteins, which are expressed at high levels in many tumors, play a central role in regulating apoptosis and, consequently, are thought to impact tumor formation, tumor growth, and resistance. Venclexta is currently used to treat adults with chronic lymphocytic leukemia, small lymphocytic lymphoma , or acute myeloid leukemia.
Novartis’ Spartalizumab for Triple-Negative Breast Cancer
Triple-negative breast cancer (TNBC) is cancer that tests negative for estrogen receptors, progesterone receptors, and excess HER2 protein. About 10-20% of breast cancers are triple-negative. TNBC does not respond to hormonal therapy medicines or medicines that target protein receptors. Novartis is currently testing Spartalizumab, a monoclonal antibody directed against the human programmed death-1 (PD-1) receptor, to treat TNBC. The Phase I clinical trial is an open-label, dose escalation study of Spartalizumab & LAG525 in combination with other antitumor and antibody medications.
Bristol-Myers Squibb’s Opdivo for Triple-Negative, HR-Positive, and HR-Negative Breast Cancer
Bristol Myers Squibb Company (BMS) will perform a clinical trial collaboration with Ubivac, a clinical stage immunotherapy company. The trial will evaluate the safety, tolerability, and preliminary efficacy of UbiVac’s investigational product, DPV-001, a first-in-class cancer vaccine that exploits autophagy, in combination with BMS’s immune checkpoint inhibitor, Opdivo. The Phase 1 trial will test the hypothesis that combination immunotherapy with the DPV-001 cancer vaccine and Opdivo will augment anticancer immunity in patients with advanced triple negative breast cancer. This is the first clinical trial to combine a cancer vaccine (DPV-001) with a T cell agonist (Opdivo), that amplifies immune system activity. BMS is also currently completing a randomized multi-arm study to evaluate the efficacy and safety of Opdivo versus placebo in combination with chemotherapy and endocrine therapy. The therapy is being tested in participants with high-risk, estrogen receptor-positive, human epidermal growth factor receptor 2-negative (ER+, HER2-) early stage breast cancer.
FDA Vaccine Committee Meeting, Expanded Access or Emergency Use
Last week, the FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) met to discuss the development, authorization and licensure of vaccines to prevent COVID-19. At the meeting Marion Gruber, the director of the FDA’s Office of Vaccines Research and Review, presented a pertinent issue of whether issuing an emergency use authorization (EUA) would interfere with long-term assessment of safety and efficacy and jeopardize future approval for every COVID-19 vaccine. The developer of a COVID-19 vaccine that is given an EUA might not be able to generate enough additional data to ever successfully apply for a full license. This issue hinges on whether once a vaccine has been cleared for use by the FDA, the randomly assigned participants that receive a placebo in its clinical trial must be informed and offered a vaccine. Vaccinating the participants in a trial’s control arm would end the ability to continue to compare the two groups.
Many of the vaccine developers will only have a few months worth of efficacy data and if control arm participants are vaccinated the likelihood of a full approval is slim. The FDA is urging vaccine manufacturers to keep their trials blinded as long as possible, to collect as much data as they can. Randomized controlled trials are the best method for discerning efficacy, duration of effect, and population variance. Some authorities in FDA approvals, recommended expanded access in contrast to EUA, to ensure the continuation of the clinical trials. Expanded access is the use of an investigational new drug outside of a clinical trial in patients for the diagnosis, monitoring, or treatment of a serious disease or condition. In contrast, participants in clinical trials/studies are considered human subjects, whether they are patients or healthy volunteers. Pfizer and BioNTech are expected to apply for an emergency use authorization mid-November. The FDA will have to seriously consider whether to issue an EUA or emergency use, as they have indicated they plan to unblind their trial and offer people in the placebo arm a vaccination.