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Monthly News Roundup - February 2021

FDA Authorizes Emergency Use of Janssens’s One-Shot COVID-19 Vaccine 

The FDA has issued an emergency use authorization (EUA) for the Janssen COVID-19 Vaccine, the third vaccine in the U.S. for the prevention of COVID-19 disease. The Janssen COVID-19 Vaccine is used in individuals 18 years of age and older, is given as a one-time shot, and can be shipped and stored under refrigeration. 

The new vaccine is manufactured differently than the mRNA vaccines from Moderna and Pfizer/BioNTech: it uses the adenovirus type 26 (Ad26), a weakened cold virus to deliver DNA to temporarily make the  “spike” protein of the SARS-CoV-2 virus. It does not cause COVID disease or a cold but helps to trigger the immune response against the SARS-CoV-2 virus.

  • In the ongoing Phase 3 ENSEMBLE studies from South Africa, Latin America and the U.S, over 44,000 participants aged 18 years and older received the vaccine or saline placebo and were followed for a median of 8 weeks.
  • Across geographies, the vaccine was approximately 66.9% effective in preventing moderate to severe/critical COVID-19 two weeks after vaccination and 66.1% effective in preventing moderate to severe/critical disease four weeks after vaccination. Vaccine efficacy against severe/critical COVID-19 at least 14 days after vaccination was 76.7% and 85.4% at least 28 days after vaccination. None of the nearly 22,000 vaccinated people in the trial died of COVID-19 compared to five deaths in the placebo group.
  • Side effects were mild to moderate and usually cleared up in 1 to 2 days. The most commonly reported side effects were injection site pain, headache, fatigue, muscle aches, and nausea. 
  • The vaccine should be stored at  2°C to 8°C (36°F to 46°F) and protected from light. Do not store frozen. Unpunctured vials of Janssen COVID-19 vaccine may be stored between 9°C to 25°C (47°F to 77°F) for up to 12 hours. Each vial contains five doses and does not contain a preservative. Shipping is expected to start in the U.S. during the first week of March. 

FDA Approves Breyanzi CAR-T Cell Therapy for Large B-cell Lymphoma

Bristol-Myers Squibb’s Breyanzi (lisocabtagene maraleucel), a chimeric antigen receptor (CAR) T-cell therapy, was approved for the treatment of adults with relapsed or refractory large B-cell lymphoma (LBCL). It is used when at least 2 previous treatments have not worked or have stopped working. 

  • Breyanzi is a CAR-T cell therapy made from a patient’s white blood cells that are genetically modified. It is given as an intravenous (IV) infusion in a certified healthcare facility.
  • In studies, 268 patients with relapsed or refractory LBCL received Breyanzi in both the inpatient and outpatient settings. Of these patients, 73% achieved a complete response (CR), including 54% who had minimal or no detectable lymphoma remaining following treatment, and 19% who achieved a partial response. Median duration of response was 16.7 months in all responders.
  • Common, serious and fatal side effects can occur with Breyanzi. Cytokine release syndrome (CRS), which may be fatal, and serious neurologic toxicities are outlined in a Boxed Warning. In studies, CRS resolved in 119 of 122 patients (98%) with a median duration of 5 days (range: 1 to 17 days). Other common side effects include fatigue, difficulty breathing, fever, chills, and confusion, among others.

FDA Approves Ukoniq for Two Lymphoma Indications

In February, the FDA cleared TG Therapeutics once daily oral Ukoniq (umbralisib) under accelerated approval for two lymphoma uses: to treat adults with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one prior anti-CD20 based regimen, and for adults with relapsed or refractory follicular lymphoma (FL) who have received at least three prior lines of systemic therapy. 

  • Ukoniq is a unique dual inhibitor of phosphoinositide 3 kinase (PI3K) delta and casein kinase 1 (CK1) epsilon.
  • In the Phase 2 UNITY-NHL open-label trial, patients received Ukoniq 800 mg orally once daily. In the group being treated for MZL, the ORR was 49% (34/69 patients) and in the FL group the ORR was 43% (50/117 patients).
  • Serious and common side effects included diarrhea-colitis, pneumonia, elevated creatinine, fatigue, nausea and neutropenia, among other adverse events.

Cosela First Therapy OK’d to Help Protect Bone Marrow Before Chemotherapy

In February, the FDA approved G1 Therapeutics’ Cosela (trilaciclib) 300 mg injection to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer.

  • Cosela is the first therapy to help protect bone marrow when given prior to chemotherapy. Myelosuppression can occur when there is damage to the bone marrow from chemotherapy and can increase the risk of infections, severe anemia, and bleeding.
  • Cosela is classified as a cyclin-dependent kinase 4/6 (CDK4/6) inhibitor. It is given as a 30-minute intravenous (IV) infusion completed within 4 hours prior to the start of chemotherapy on each day chemotherapy is administered.
  • The most common side effects (≥10%) included fatigue, low calcium levels (hypocalcemia), low potassium levels (hypokalemia), low phosphate levels (hypophosphatemia), increased liver enzymes (aspartate aminotransferase), headache, and pneumonia.

Regeneron’s Evkeeza is a First-in-Class Treatment for High Cholesterol

Homozygous familial hypercholesterolemia (HoFH) is a rare, inherited condition in which patients cannot remove unwanted low-density lipoprotein-cholesterol (LDL-C or “bad” cholesterol) from their body. Patients with HoFH often die from a heart attack before the age of thirty.

  • This past month, the FDA approved Evkeeza (evinacumab-dgnb) to be used in addition to other LDL-C lowering therapies to treat adults and children, aged 12 years and older, with HoFH.
  • Evkeeza is classified as an angiopoietin-like 3 (ANGPTL3) inhibitor, a new classification of therapy that binds and blocks ANGPTL3, a protein that plays a key role in lipid metabolism. It is given as a 60 minute intravenous (IV) infusion once every 4 weeks.
  • In studies, common side effects (≥5%) were nasopharyngitis (common cold), influenza-like illness, dizziness, rhinorrhea (runny nose), and nausea.

Tepmetko: First Oral Once-Daily MET Inhibitor for Aggressive Form of Lung Cancer

This past month, the FDA approved EMD Serono’s Tepmetko (tepotinib) under accelerated approval. Tepmetko is an oral MET inhibitor for the treatment of adults with metastatic non-small cell lung cancer (NSCLC) with mesenchymal-epithelial transition (MET) exon 14 skipping alterations. Tepmetko inhibits the oncogenic MET receptor signaling caused by MET (gene) alterations. METex14 skipping occurs in approximately 3% to 4% of NSCLC cases.

  • Tepmetko monotherapy is being evaluated in the ongoing Phase II VISION study of 152 patients with a median age of 73 years. Tepmetko has demonstrated an overall response rate of 43% in treatment-naïve patients and 43% in previously treated patients. Median duration of response (DOR) was 10.8 months and 11.1 months among treatment-naïve and previously treated patients, respectively.
  • Tepmetko is given as two 225 mg tablets (450 mg) orally once daily with food until the disease worsens or there is unacceptable toxicity.
  • Common adverse reactions include edema (fluid build-up), fatigue, nausea, diarrhea, muscle and bone pain, and shortness of breath (dyspnea). There is an increased risk of interstitial lung disease/pneumonitis (severe or life-threatening swelling of the lungs), liver toxicity, and the potential risk of fetal harm in pregnancy that requires the need for contraception.

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