Update for the Week Ending May 9, 2020

Regulatory Update
 
The FDA approved selpercatinib (Retevmo, Lilly) on 5/8/2020, for the treatment of non-small cell lung cancer, advanced medullary thyroid cancer and advanced RET fusion-positive thyroid cancer. The FDA requires identification of a RET gene alteration must be determined before initiating treatment. Selpercatinib is taken twice a day until the disease progresses or there is unacceptable toxicity. Lilly set WAC for a 30-day supply of selpercatinib at $20,600. Selpercatinib is the first drug approved to treat cancers with mutation or fusion in the RET gene, but the drug will eventually have competition from pralsetinib, a second RET fusion mutation therapy from Blueprint Medicines. Blueprint filed an NDA for pralsetinib in 1Q2020, so a competitor for selpercatinib may be available in late 2020 or early 2021.
 
The FDA approved capmatinib (Tabrecta, Novartis) on 5/6/2020, for the treatment of metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA-approved test. Novartis has set WAC for a 28-day supply of capmatinib at $17,950. Capmatinib is the first drug approved to treat cancers with MET exon 14 skipping alterations.  The drug will eventually have competition from tepotinib, a MET inhibitor being developed by EMD Serono.
 
The FDA accepted the NDA for Bristol-Myers Squibb/Juno/Therapeutics Celgene’s lisocabtagene maraleucel for the treatment of large B-cell lymphoma and set a PDUFA date for August 2020. The PDUFA date was then delayed by three months in May 2020 to allow the FDA time to review new requested data for the drug. The new PDUFA date is 11/16/2020.
 
The FDA granted Amgen/Cytokinetics’ omecamtiv mecarbil, Fast Track designation as a treatment of chronic heart failure with reduced ejection fraction (HFrEF).
 
The EMA granted Acceleron Pharma’s sotatercept a Priority Medicines (PRIME) designation for the treatment of patients with pulmonary arterial hypertension (PAH).
 
Announced Research Updates
 
TG Therapeutics announced interim data from the 420 patient, Phase III UNITY-CLL trial, where patients treated with ublituximab plus umbralisib had an improved progression free survival compared to obinutuzumab plus chlorambucil in patients with previously untreated and relapsed/refractory chronic lymphocytic leukemia (CLL). Due to the results, the trial was ended early.
 
Akebia announced that in the 36-week, 369 patient, Phase III, INNO2VATE Correction/Conversion trial, vadadustat was non-inferior to darbepoetin in the change in hemoglobin (0.17 g/dL difference) and hemoglobin level (10.36 vs 10.53 g/dL) in patients with chronic dialysis for end-stage renal disease. Akebia announced that in the 36-week, 3,554 patient, Phase III, INNO2VATE Conversion trial, vadadustat was non-inferior to darbepoetin in the change in hemoglobin (0.31 g/dL difference) and hemoglobin level (10.36 vs 10.61 g/dL) in patients with chronic dialysis for end-stage renal disease. In the two INNO2VATE Correction/Conversion and Conversion trials, vadadustat was non-inferior to darbepoetin in the incidence of MACE (composite endpoint of all-cause mortality, non-fatal myocardial infarction, or non-fatal stroke).
 
Ovid announced that in the 12-week, 23 patient, Phase II ROCKET trial, treatment with gaboxadol resulted in a 26.2% improvement in the Aberrant Behavior Checklist-Community for Fragile X syndrome (ABC-CFXS) score and a 21.6% mean improvement in the Anxiety, Depression and Mood Scale (ADAMS) total score in patients with Fragile X syndrome.
 
Published Research Updates
 
In a 65 patient, Phase II trial, patients treated with voxtalisib plus pimasertib, the objective response rate (ORR) was 9.4% compared to 12.1% with pimasertib monotherapy in patients with recurrent unresectable borderline/low malignant potential or low-grade serous ovarian carcinoma. The trial was terminated early because over half of the patients in both arms discontinued treatment and the ORR was considered too low. 
 
In the 150 patient, Japanese PELTA trial, prophylactic landiolol for four days after surgery did not reduce the incidence of postoperative atrial fibrillation in post-cardiac surgery patients who were 70 years or older.
 
In a 12-week, 257 patient, Phase II trial, treatment with estetrol 15 mg decreased the severity of hot flushes (HFs) compared to placebo (-1.04 vs -0.66) in postmenopausal women with seven or more moderate to severe HFs per day or 50 or more moderate to severe HFs weekly.