Week of November 15

COVID-19

• Moderna, Pfizer and BioNTech announced positive interim Phase 3 results regarding their COVID-19 vaccine candidates. From Pfizer/BioNTech’s trial, 94 COVID-19 cases were detected. With 86 cases having received the placebo, the vaccine demonstrated an efficacy rate above 90% at 7 days after the second dose. Of the 95 COVID-19 cases in Moderna’s trial, 90 participants had received the placebo, while 5 had received both doses of the vaccine leading to a 94.5% efficacy at 14 days after the second dose. Participants in both trials experienced minor adverse events, including injection site pain, fatigue, myalgia, and headache. Final analysis will occur when COVID-19 cases numbers reach 164 and 151 for Pfizer/BioNTech and Moderna, respectively. An advantage of the Pfizer/BioNTech candidate is that in addition to participants generating a strong neutralizing antibody response, participants also produce a strong CD8+ response. At this time, there is no information on Moderna’s candidate on eliciting a CD8+ response, which may be an indicator for long-lasting immunity. An advantage of Moderna’s candidate is that it is more heat tolerant compared to Pfizer/BioNTech’s candidate, which requires being kept on dry ice. One concern arises for both vaccines: compliance. Both require two doses that are separated by 21 to 29 days, which may influence willingness to return for a second dose, let alone receive a vaccine in the first place. A good review of both mRNA vaccines can be found at Evaluate: Link 1, Link 2, Link 3

• FDA granted Emergency Use Authorization for Eli Lilly’s COVID-19 neutralizing antibody bamlanivimab to treat mild or moderate COVID-19 patients who are at high risk of progressing to severe COVID-19 and/or hospitalization. This authorization is based on Phase 1 results demonstrating that recently diagnosed patients treated with bamlanivimab reduced viral load and hospitalization rates. Phase 2 and Phase 3 trials are ongoing, investigating bamlanivimab treatment in the ambulatory setting and at long-term care facilities, respectively. Link

• Medicago and GSK announced the start of their Phase 2/3 plant-derived COVID-19 vaccine. The vaccine, a more traditional approach compared to Moderna’s and Pfizer/BioNTech’s mRNA candidates, is composed of recombinant spike protein expressed as virus-like particles. The Phase 2 trial (n= 300) is composed of healthy and elderly adult cohorts, which will receive the vaccine or a placebo and will be followed for 12 months. Link

Oncology

• FDA approved Merck’s combination of Keytruda and chemotherapy to treat patients with metastatic TNBC expressing PD-L1. This follows Phase 3 results demonstrating that the combination reduced the risk of disease progression by 35% in PD-L1-expressing tumors, compared to chemotherapy alone. Link

• EMA approved Roche’s combinational subcutaneous (SC) injection of Perjeta and Herceptin (Phesgo) to treat HER2+ breast cancer. Phase 3 results for Phesgo demonstrated equivalent levels of Perjeta and Herceptin in the blood compared to IV infusion, while reducing treatment administration time to around 5 minutes compared to 210-300 minutes of treatment and subsequent maintenance. Link

• Johnson & Johnson submitted applications to the FDA and EMA seeking approval for Darzalex (daratumumab) subcutaneous (SC) formulation in combination with pomalidomide and dexamethasone to treat patients with relapsed or refractory multiple myeloma. The SC formulation shortens the administration of Darzalex to a few minutes compared to several hours when given IV. Link

• At the upcoming annual American Society of Hematology meeting, GSK will present new data on Blenrep, an anti-BCMA therapy, to treat multiple myeloma patients. Data will include clinical results of Blenrep in combination with bortezomib and dexamethasone for refractory or relapsed multiple myeloma at the 2L or 3L setting. Link

CNS Disorders and Neurodegeneration

• To help maintain the Parkinson’s disease registry in California during the COVID-19 pandemic, The Michael J. Fox Foundation for Parkinson’s Research (MJFF) gave $380,000 in extension funding to help continue the registry until June 2021. This registry maintains data on Parkinson’s disease trends related to socioeconomic status, race, ethnicity, and exposure to environmental factors. Link

Industry and Corporate Developments

• Intellia Therapeutics secured funding from the Bill & Melinda Gates Foundation for the development of their in vivo sickle cell disease (SCD) treatments, which utilize CRISPR/Cas9 genome editing technology. Funding will be used for advancing preclinical validation of Intellia’s proprietary in vivohematopoietic stem cell (HSC) genome editing method. This methods seeks to avoid the need for bone marrow transplantation surgery to treat various blood disorders. Link

• Seed Therapeutics, a subsidiary of BeyondSpring, and Eli Lilly announced a collaboration and licensing agreement to identify new targeted protein degradation (TPD) therapeutics intended to target proteins previously thought to be undruggable. Seed Therapeutics will receive a $20M upfront payment and equity investment, with the potential to receive roughly $780M in milestone payments. Link

• UCB Pharma acquired Handl Therapeutics for an undisclosed amount and announced a collaboration with Lacerta Therapeutics in an effort to advance UCB’s genomic and epigenomic research platforms to identify new therapeutic targets. Link