New Drug Update for The Week Ending May 18

Regulatory Update

The FDA informed ImmunoGen that it would not accept sub-group data from patients with high levels of folate receptor alpha enrolled in the failed FORWARD I trial evaluating mirvetuximab soravtansine for NDA approval. ImmunoGen will need to run a new Phase III trial in patients with high folate receptor alpha expression, platinum-resistant ovarian cancer before seeking approval.

An FDA review of quizartinib questioned the credibility of some clinical trial data due to inconsistencies, lack of efficacy for secondary endpoints and imbalances between the study arms. The FDA’s Oncology Drug Advisory Committee voted 3 to 8 against recommending approval of quizartinib for the treatment of relapsed or refractory acute myeloid leukemia in May 2019.

An FDA review of pexidartinib expressed concern about data lacking for physical function, stiffness and range of motion and changes in the statistical analysis plan. The FDA’s Oncology Drug Advisory Committee voted 12 to 3 to recommending approval of pexidartinib for the treatment of giant cell tumor of the tendon sheath May 2019

The FDA’s Allergenic Products Advisory Committee will review Aimmune Therapeutics’ AR101 on 9/13/2019.  AR101 is an oral immunotherapy used to desensitize the patient to Peanut allergy.

The FDA has put a partial hold on an axalimogene filolisbac Phase III cervical cancer trial in January 2019, until Advaxis provided additional chemistry, manufacturing and controls information. Under the hold, no new patients could be enrolled in the trial, but current patients can continue to be treated. The FDA lifted the partial band on the cervical cancer trial on 5/15/2019.

Announced Research Updates

Myovant announced that in the 24-week, 388 patient, Phase III, LIBERTY trial, 73.4% of patients treated with relugolix, estradiol and norethindrone acetate achieved a menstrual blood loss volume of less than 80 mL and a 50 percent or greater reduction from baseline in menstrual blood loss volume during the last 35 days of the 24-week treatment period compared to 18.9% with placebo in patients with uterine fibroids and heavy menstrual bleeding.

UniQure announced interim data from a 52-week, 3 patient, Phase IIb trial, where after 6-months treatment with a single infusion of AMT-061 had a mean increase in Factor IX (FIX) activity of 47% (individual results: 33%, 51%, 57%) and no patient required a Factor IX infusion, experienced a bleeding event or required immunosuppression.

The Medicines Company announced that in Orion-3, a 3-year extension study of ORION-1, inclisiran lowered LDL at least 50% at all doses and frequency.

Published Research Updates

The 20-month, 572 patient, Phase III SOLAR-1 trial enrolled a population of men and postmenopausal women with HR+/HER2- advanced breast cancer whose cancer progressed after at least 12 months of an aromatase inhibitor therapy. In a subset of 341 who harbored PIK3CA mutations, PFS was 11 months with alpelisib/fulvestrant and 5.7 months with fulvestrant monotherapy. In the subset whowithout PIK3CA-mutated cancer, the overall response rate was 26.6% with alpelisib/fulvestrant compared to 12.8% with fulvestrant monotherapy. 

In a 28-day, 256 patient, Phase III trial, brimapitide did not improve hearing loss compared to placebo in patients with unilateral idiopathic sudden sensorineural hearing loss (ISSNHL). A post-hoc analysis found a benefit in patients with profound ISSNHL.

In a 187 patient, Phase I, open-label trial, treatment with erdafitinib resulted in a 46.2% overall response rate (ORR) in patients with urothelial carcinoma and a 27.3% ORR in patients with cholangiocarcinoma in patients with treatment resistant, pan-fibroblast growth factor receptor (FGFR) positive tumors. The ORR for all other tumor types of < 10%.

In a 560-patient trial, 31.4% of patients treated with amisulpride had no emesis or need of rescue medications compared to 21.5% with placebo in patients at low-to-moderate risk for PONV who had not received any prior prophylaxis.